Sun 8 Nov
7:00 - 18:00
Registration Hours
Session Type: General Meeting
8:00 - 12:00
Developing Decision-Grade Real-World Evidence
Session Type: Short Course
Topics: Real World Data & Information Systems
Track: Real-World Evidence (RWE)
Level: Intermediate
Separate registration required.
In this course, participants will be introduced to the principles of what makes real-world evidence (RWE) decision-grade, including an extended example. In the first half of the course, we will review the most recent RWE frameworks and guidelines and examine case studies in which RWE was used in regulatory and HTA approval. The second half of the course is an extended example in which participants will examine a study that could support an indication expansion and interactively discuss how choices made in the design and implementation may affect the meaning and interpretability of results.
PREREQUISITE: Students are expected to be familiar with relevant concepts and methodologies for analyzing real-world data.
Speakers
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Jeremy Rassen, ScD
Aetion, Inc., New York, NY, United States
Jeremy A. Rassen, MS, ScD is a pharmacoepidemiologist with 25 years of academic and industry experience. He is cofounder, president, and chief technology officer at Aetion, a healthcare technology company that delivers real-world evidence for life sciences companies, payers, and regulatory agencies. Prior to founding Aetion, Dr. Rassen was assistant professor of medicine at Harvard Medical School, where he focused on methods to improve the quality and validity of real-world data studies. He also worked in Silicon Valley in a variety of tech companies. Dr. Rassen received his bachelor’s degree in computer science from Harvard College and his master’s and doctorate degrees in Epidemiology from the Harvard TH Chan School of Public Health.
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Jennifer Polinski, ScD, MPH, MSc
Datavant, Boston, MA, United States
Dr. Polinski serves as senior vice president and head of science delivery at Datavant. Dr. Polinski is an epidemiologist and pharmacoepidemiologist with more than 25 years of experience in the healthcare industry and academia. Her specialties include real-world evidence generation from real-world data in the comparative safety and effectiveness space as well as health economics and outcomes research. Before joining Datavant and its predecessor Aetion, Dr. Polinski held analytics leadership roles at CVS Health and at Haven, the Amazon, Berkshire Hathaway, JPMorgan Chase healthcare venture. Prior to her industry experience, she spent 11 years at the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital. She was an Assistant Professor at Harvard Medical School and the Harvard T.H. Chan School of Public Health. She has published more than 75 articles in peer-reviewed medical journals. Dr. Polinski received her bachelor's degree from the University of Virginia, master's degrees from both Emory University (Public Health) and Harvard T.H. Chan School of Public Health (Epidemiology), and a doctorate degree in Epidemiology from the Harvard T.H. Chan School of Public Health.
Prompt Engineering for HEOR: Practical Skills and Use Cases for HEOR Professionals
Session Type: Short Course
Topics: Methodological & Statistical Research
Track: AI
Level: Introductory
Separate registration required.
Prompt engineering—the art and science of designing effective inputs for generative AI—has become a critical skill for health economists and outcomes researchers. Mastery of prompt engineering can significantly enhance productivity, accuracy, and innovation in HEOR, unlocking the full potential of large language models (LLMs) and other AI tools. This course delivers a comprehensive introduction to prompt engineering, tailored specifically for the HEOR context. Participants will gain hands-on experience with practical prompt strategies for systematic literature reviews (SLRs), economic modeling, real-world evidence generation, and more. The curriculum also addresses current best practices and common pitfalls, equipping attendees to confidently apply prompt engineering in regulated and high-stakes settings.
PREREQUISITE: Basic knowledge of systematic literature reviews and economic modeling will be helpful. No prior knowledge or use of AI is required.
Speakers
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Jag Chhatwal, PhD
Harvard Medical School / Massachusetts General Hospital, Boston, MA, United States
Jag Chhatwal, PhD, is the director of the Institute for Technology Assessment at Massachusetts General Hospital and an associate professor at Harvard Medical School. He also serves as core faculty at the Center for Health Decision Science, Harvard T.H. Chan School of Public Health. Dr. Chhatwal has co-authored more than 125 original research articles and editorials in leading peer-reviewed journals. His research has informed health policy decisions at prominent organizations including the White House, the World Health Organization, and the CDC, and has been featured in major media outlets such as CNN, Forbes, National Public Radio, The New York Times, and The Wall Street Journal. Dr. Chhatwal serves as an associate editor of Value in Health and as guest editor for its special issue on artificial intelligence. He is also a member of the ISPOR Generative AI Working Group.
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Dalia Dawoud, BSc, MSc, PhD
Cytel, London, United Kingdom
Dalia Dawoud, PhD, is Research Principal, HTA Policy and Strategy. She is also the Director and CEO of PEHTA Consulting Ltd. and holds a professor position at the Faculty of Pharmacy, Cairo University. She has over 15 years experience as a health economist and researcher. Her work is largely focused on the application of HEOR in HTA and clinical guideline development. She worked at leading organizations including NICE, where she led a portfolio of HORIZON Europe projects such as HTx, EDiHTA and SUSTAIN HTA, and the Royal College of Physicians, London. She is widely published in the areas of health economics and outcomes research and serves as associate editor for Value in Health and as director on ISPOR Board of Directors (2023-2026). She is also a member of the ISPOR AI Working Group and ISPOR Living HTA Working Group.
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Turgay Ayer, PhD
Value Analytics Labs, Boston, MA, United States
Turgay Ayer, PhD, holds the Virginia C. and Joseph C. Mello Chair and serves as the research director for Healthcare Analytics and Business Intelligence at the Center for Health & Humanitarian Systems at Georgia Tech. He is also the chief technology officer at Value Analytics Labs. Dr. Ayer holds a courtesy appointment at Emory Medical School where he teaches Big Data Analytics courses and serves as a Senior Scientist at the Centers for Disease Control and Prevention (CDC). Dr. Ayer’s research focuses on health economics modeling (HEOR), real-world evidence, data science, machine learning, econometric modeling, and healthcare analytics. He has published over 80 peer-reviewed journal papers and more than 300 conference abstracts, with his work featured in top-tier business, engineering, medical, and health policy journals. His research has attracted substantial attention from major media outlets, including The Wall Street Journal, The Washington Post, US News, and NPR. A recognized expert in HEOR, Dr. Ayer has been at the forefront of applying generative AI to navigate healthcare systems and support better decision-making. He has contributed significantly to the development of advanced models for predicting healthcare outcomes and designing innovative cost-effectiveness analysis frameworks. Under his leadership, Value Analytics Labs has focused on the development of cutting-edge technologies, including ValueGen.AI, to enhance healthcare analytics and improve the efficiency of healthcare decision-making processes.
Applied Generative AI for HEOR: Introduction
Session Type: Short Course
Topics: Methodological & Statistical Research
Track: AI
Level: Introductory
Separate registration required.
The rapid advancement in generative artificial intelligence (GenAI) presents an opportunity for transformative potential in the field of health economics and outcomes research (HEOR). This course provides an introductory understanding of generative AI models with a particular focus on large language models (LLMs), which are transforming the field of HEOR. Participants will be provided with an overview of the most appropriate ways to access LLMs, going beyond the use of chatbots. Further, they will be given insights into how to use prompt engineering, retrieval-augmented generation (RAG) and agents to conduct scientific research and gain an understanding on issues pertaining to privacy and security when using GenAI for HEOR. Participants will further explore specific applications of these models for conducting robust scientific HEOR research in, for example, systematic literature reviews (SLR) and economic evaluation. The course aims to equip participants with the knowledge to begin to use generative AI techniques for specific HEOR contexts and to appreciate how these innovative approaches can enhance HEOR activities. Practical exercises using Python and relevant AI frameworks will be incorporated for participants to follow along.
PREREQUISITES: Students should have a general understanding of common HEOR concepts such as SLRs and cost-effectiveness models. Knowledge of Python or similar programming languages such as R is considered a benefit but not required.
Speakers
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Sven L Klijn, MSc
Bristol Myers Squibb, Princeton, NJ, United States
Sven Klijn is Director at Bristol Myers Squibb in the Global HEOR Evidence Acceleration & Innovation group, where he leads the innovative modeling agenda in hematology and cell therapy. In addition, Sven has an active role in providing modeling and Generative AI education at international congresses. He has widely published on innovative methods, especially in the fields of survival extrapolation and Generative AI. Sven has a training in public health and health economics and previously had various roles in CROs related to health-economic modeling.
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William Rawlinson
Estima Scientific, London, United Kingdom
Will is a senior health economist at Estima Scientific holding a degree in Physics and Philosophy from the University of Oxford. Will has 4 years’ experience developing cost-utility models and has specialized in applications of generative AI to health economic modelling. Will has published on the automation of R modelling using large language models (LLMs), and more recently has focused on applications of LLMs to Excel modelling and model reporting.
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Timothy Reason, BSc, MSc
Estima Scientific, London, United Kingdom
Tim Reason is co-founder of Estima Scientific and specializes in AI and evidence synthesis, having spent 15 years in the field of HEOR and technology. Tim is managing director of Estima, driving business activities, innovation and strategy for the company. Tim’s specializes in the intersection of HEOR, software development and AI to drive better outcomes for patients. Tim is the lead author on 2 seminal papers in AI for HEOR, showing that AI can be used to automate health economic modelling and NMA.
Risk-Sharing/Performance-Based Arrangements in Developing Countries
Session Type: Short Course
Topics: Health Policy & Regulatory
Track: Access and Pricing
Level: Intermediate
Separate registration required.
During recent years, Managed Entry Agreements (MEAs) have become instrumental in ensuring the access of innovative medicines. This course is designed for healthcare professionals (including public decision-makers, academia, and industry) involved in pricing and reimbursement decisions who are wishing to understand the applicability and technical aspects of managed entry agreements (MEAs) in countries with severe economic constraints and explicit cost-effectiveness criterion. The topic will be introduced with key features of pricing and reimbursement systems in representative countries to understand why special methods are needed to facilitate evidence-based reimbursement policies of new health technologies. Faculty will present an economic model to explain the methodology and implications of managed entry agreements in cost-effectiveness and budget impact analysis. Participants will then have the opportunity to apply what they have learned through a hands-on exercise on making pricing and reimbursement decisions. A decision algorithm will be presented to support evidence and value-based policy decisions of high-cost new technologies. A series of password protected economic models will add more and more complexity to a pragmatic case study on a new pharmaceutical product in oncology. To close the course faculty will lead a discussion on the applicability of a pragmatic decision tool illustrating the pros and cons of different managed entry agreements and their usefulness in CEE settings. Participants who wish to gain hands-on experience must bring their laptops with Microsoft Excel for Windows installed.
Speakers
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Bertalan Németh, PhD
Syreon Research Institute, Budapest, Hungary
Bertalan Németh PhD graduated from Corvinus University of Budapest (MSc in Quantitative economics and Operation research), Eötvös Loránd University (Pharmaceutical economics and drug policies), and Semmelweis University School of PhD Studies (PhD in Pharmacoeconomics). Between 2010 and 2015 he was a Health Economist at the Hungarian HTA office. Since August 2015 Bertalan has been a Senior Health Economist, and since 2019 a Principal Researcher at Syreon research Institute. Bertalan was lead author or co-author of more than 50 peer reviewed publications. Bertalan was the President of the ISPOR Hungary Chapter and was the Chair of the ISPOR CEE Consortium. He was a participant in the international EUnetHTA project, the ISPOR HTA Roundtable Europe, and the Scientific Committee of the Annual Conference of the ISPOR Hungary Chapter. Bertalan is also a faculty member of ISPOR HTA Trainings and was the module leader of Health Technology Assessment for the MSc program at Eötvös Loránd University.
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Rok Hren, MSc, PhD
Syreon Research Institute, Budapest, Hungary
Rok Hren has more than 15 years of commercial experience in pharmaceutical industry and more than 12 years on a board level in both (1) line management, which has included full P&L responsibility for operations in Slovenia and Romania, and (2) leadership regional functions in Central and Eastern Europe. He regularly presents on the topic of pharmaceutical economics and policies at conferences in Europe and is well experienced in healthcare media business.
He received his PhD in Physiology and Biophysics from Dalhousie University, Canada and MSc in International Health Policy (Health Economics) with Distinction from London School of Economics and Political Science, UK while he was a post-Doctoral Fellow at Nora Eccles Harrison Cardiovascular Research and Training Institute, University of Utah Medical School, USA. He is also an assistant professor at the University of Ljubljana and past president of the ISPOR Slovenia Regional Chapter. In total, his publications gathered 300/394 citations (excluding self-citations) in WoS/Scopus (as of September 10, 2016).
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Katarzyna Kolasa, PhD
Kozminski University, Warsaw, Poland
Driven with passion to improve healthcare, Katarzyna has focused her academic and business career on health economics.
She has been working with multiple pricing and reimbursement challenges worldwide for the last 25 years, while holding various regional and global leadership positions at Astra Zeneca, BMS, Biogen Idec, Lundbeck, GE Healthcare, Straub Medical, BD, and the Swedish County Council of Kalmar. Katarzyna is mentor and consultant to start ups involved in the development of innovative medical devices and digital health solutions from both Holland and Poland.
Since 2000, she has been an academic teacher and supervisor for over 30 MBA and PhD students. In partnership with the Polish Medical Research Agency, Deloitte Digital and the Polish Central Hospital of Ministry of Interior Affairs, she founded the first Digital Health 6 months educational program designed for digital transformation leaders working in the healthcare sector. Katarzyna developed an innovative Master Program Health Economics & Big Data (HEBDA) with the first edition being financed by EU Power Grant 2018 as well.
She is the founder of the Global Special Interest Group Digital Health and short courses “The Role of Digital Endpoints in the Value Generation for Health Technologies”, “Risk-Sharing/Performance-Based Arrangements in Developing Countries” for ISPOR, The Professional Society for Health Economics and Outcomes Research. She is currently a member of the ISPOR Education Council and a previous member of the ISPOR Health Science Policy Council as well.
Katarzyna has dedicated her academic research towards methodological advancements into the value assessment of pharmaceuticals, medical devices, and digital health solutions. Passionate about Big Data, she led the first project of machine learning adaptation for the optimal utilization of CT scanners granted by the Polish Ministry of Health. Since January 2022, she is the leader of AI special interest group at the Polish Chamber of Physicians. With the patronage of the Polish Parliamentary Commission for Innovation & Digitalization, she organized the first dialog about the societal preferences towards the adoption of AI in the healthcare in Poland.
Being a coauthor of more than 50 IF publications, she has presented her research at more than 60 international scientific conferences. As of 2022, Google Scholar reports over 730 citations to her work.
Health Economic Modeling in R for Decision Making: Assessment, Adaptation, and AI-Assisted Validation
Session Type: Short Course
Topics: Economic Evaluation
Level: Intermediate
Separate registration required.
Health economic models developed in R are playing an increasingly prominent role in reimbursement and health technology assessment decisions. While numerous resources exist for building models in R, comparatively little attention has been devoted to reviewing, validating, and adapting existing models. In practice, decision-makers and analysts frequently encounter complex R-based models that they must evaluate, verify, and modify, often with limited familiarity with the original codebase. This course aims to address that gap, drawing on the instructors' direct experience working with and advising HTA bodies including the UK National Institute for Health and Care Excellence (NICE), the Irish National Centre for Pharmacoeconomics (NCPE), and familiarity with the processes of the Dutch Zorginstituut Nederland (Zin) and the Canadian Drug Agency (CDA).
Using a Markov cost-effectiveness model in Atrial Fibrillation as the case study, based on a model developed for UK National Institute for Health and Care Excellence (NICE) guidelines, participants will work through a structured sequence covering model execution, code quality assessment, manual and AI-assisted validation, and practical modification for sensitivity and scenario analyses.
The course begins with an introduction to decision modeling in R, establishing foundational concepts and workflow conventions for reproducible health economic analyses. Participants will then examine the Atrial Fibrillation model's structure, inputs, and outputs before executing it in R to generate base case results.
With the model running, the course turns to coding practice. Participants will learn to recognize well-structured R code, including clear naming conventions, modular organization, documentation standards, and reproducibility safeguards. They will assess whether the case study model adheres to these standards.
Validation progresses through two complementary approaches. Participants will first perform manual extreme-value and unit tests, designing targeted checks that probe model behavior at boundary conditions and verify that individual components produce expected outputs.
The course then introduces an agentic artificial intelligence approach that automates the same structured validation process, demonstrating how AI can replicate and extend what participants learned to do manually. This pairing illustrates the progression from understanding validation principles to scaling them efficiently, while the analyst retains focus on substantive judgment. R's transparent and readable code provides a natural advantage for AI-assisted review compared to spreadsheet-based models where logic is dispersed across cells and tabs.
The course also covers NICE's position statement on the use of AI in evidence generation and reporting and shows it in action: as participants use AI to quality-control the case study model, they will see how to declare and describe AI use, keep the analyst accountable, and maintain transparency and reproducibility.
Finally, participants will modify the R model to implement sensitivity and scenario analyses, adjusting parameters, restructuring assumptions, and generating alternative results. This exercise consolidates skills from the full course, requiring participants to understand the model well enough to make targeted, purposeful modifications.
By the end of the course, participants will be equipped to independently assess, validate, and adapt R-based health economic models encountered in HTA submissions, academic review, and research collaboration. Participants who wish to gain hands-on experience must bring their laptops with R/R Studio installed. An online version of RStudio will be provided prior to the course as a backup.
PREREQUISITES: Basic R usage, Health economic decision modeling, modeling in health technology assessment, and Markov models.
Speakers
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Howard Thom, MSc, PhD
Bristol, United Kingdom
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Felicity Lamrock, BSc, PhD
Queen's University Belfast, Belfast, United Kingdom
Dr. Felicity Lamrock is a senior lecturer in Data Analytics at Queen’s University Belfast. She is currently the Director of the Northern Ireland Centre for Health Analytics and Decision Science (NI-CHADS) with a focus on the analysis of health data for decision modelling. Current projects include a range of disease areas including cancer, rare diseases, diabetes, COVID-19, and cardiovascular disease. Felicity was previously a statistician at the National Centre for Pharmacoeconomics (NCPE) working with a team of pharmacists and clinicians on Health Technology Assessments to advise the Health Service Executive on the recommendation of new drug therapies in Ireland. She remains involved with NCPE as a statistical advisor and is exploring how Northern Ireland could benefit from more decision modelling/pharmacoeconomic assessment.
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Eline Krijkamp, PhD, MSc
Erasmus School of Health Policy and Management, Rotterdam, Netherlands
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Baris Deniz, MSc
chapel hill, NC, United States
Getting Closer to the Target: Practical Strategies to Hit the Mark in Trial Emulation
Session Type: Short Course
Topics: Methodological & Statistical Research, Epidemiology & Public Health
Track: Real-World Evidence (RWE)
Level: Intermediate
Separate registration required.
Target trial emulation (TTE) has become a cornerstone of real-world evidence (RWE) generation, particularly for external control arms and hybrid trial designs. However, translating the conceptual framework into credible, decision-ready evidence requires careful alignment of key design elements—including eligibility criteria, index date selection, follow-up, and confounding control. Small deviations in these choices can introduce substantial bias, including immortal time bias, prevalent user bias/ left truncation, informative censoring, and residual confounding driven by disease trajectory.
This course provides a comprehensive, applied framework for designing fit-for-purpose TTE studies, with a focus on time-related design challenges. Drawing on recent methodological advances and real-world applications, we integrate three core components: (1) foundational TTE principles and sources of bias, (2) a structured decision framework for index date selection, and (3) practical strategies for addressing time-related biases, including time-varying confounding.
Participants will engage with a series of case-based exercises that simulate real-world study design decisions. Through live polling and interactive dashboards, attendees will evaluate tradeoffs across alternative design strategies—such as line-of-therapy selection, comparator definition, and confounding adjustment—and observe how these decisions influence study outputs in real time (e.g., survival curves, hazard ratios, covariate balance, and weighting diagnostics).
By integrating conceptual guidance with hands-on application, this course equips researchers, regulators, and decision-makers with practical tools to operationalize TTE principles and generate credible, transparent, and defensible RWE across therapeutic areas.
PREREQUISITES: Basic familiarity with observational research and real-world data. Prior exposure to causal inference concepts is helpful but not required.
Speakers
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Neisha Opper, MPH, PhD
Landmark Science, La Crescenta, CA, United States
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Shivani Aggarwal, MS, PhD
Landmark Science, Inc, Los Angeles, CA, United States
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Hoa Le, PhD, MD
The University of North Carolina at Chapel Hill, Chapel Hill, NC, United States
Experienced Leader with expertise in RWE, Biostatistics, Pharmacoepidemiology, Drug Safety, AI, GenAI, External Control Arms and Digital Twins
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Jennifer B. Christian, PharmD, MPH, PhD, FISPE
Durham, NC, United States
8:00 - 17:00
Reimbursement Systems for Pharmaceuticals in Europe
Session Type: Short Course
Topics: Health Policy & Regulatory
Track: Access and Pricing
Level: Intermediate
Separate registration required.
Pharmaceutical reimbursement systems in Europe are complex, diverse, and heterogeneous, shaped by national policies, healthcare priorities, regulatory frameworks and underlying epistemological choices. This short course offers an in-depth exploration of these systems, focusing on the decision-making processes that determine whether and how new medicines are reimbursed or accessible across key European markets.
Unlike marketing authorization for pharmaceuticals (mainly regulated at the European level by EMA), pricing and reimbursement decisions in Europe are managed by individual member states. Health care services are generally covered by a single public health insurer operating under the Ministry of Health supervision. As a monopoly buyer (monopsony), this situation provides a leading position for the public health insurer to set reimbursement conditions. On the other side, pharmaceutical companies may be in a monopoly situation with a single provider or very few for the same medicinal class. Therefore, based on each country’s set of regulations, processes, and values, wide variations exist in pricing and reimbursement decisions of pharmaceuticals driven by power positions and desirability of new products. This course is essential for professionals involved in market access, health economics, regulatory affairs, and policymaking, providing the tools and knowledge needed to navigate the evolving landscape of pharmaceutical reimbursement in Europe.
Speakers
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Mondher Toumi, MSc, PhD, MD
Aix-Marseille University, Marseille, France
Professor Mondher Toumi is an MD by training and holds 2 MSc in Biostatistics, and in Biological Sciences (option pharmacology) and a PhD in Economic Sciences. He is a professor of Public Health at Aix-Marseille University. After working for 12 years as a research manager in the Department of Pharmacology at the University of Marseille, he joined the Public Health Department in 1993. In 1995, he entered the pharmaceutical industry and worked there for 13 years.
Mondher Toumi was appointed global vice president at Lundbeck A/S in charge of health economics, outcome research, pricing, market access, epidemiology, risk management, governmental affairs, and competitive intelligence. In 2008, he founded Creativ-Ceutical, an international consulting firm dedicated to support health industries and authorities in strategic decision-making.
In February 2009, he was appointed professor at Lyon I University in the Department of Decision Sciences and Health Policies. He launched the first European University Diploma of Market Access (EMAUD), an international course already followed by more than 500 students. Additionally, he recently created the Market Access Society to promote research and scientific activities around market access, public health and health economic assessment. He is chief editor of the Journal of Market Access and Health Policy (JMAHP).
Since September 2014, he joined the research unit EA3279 of the Public Health Department, at Aix-Marseille University (France) as Full Professor. Mondher Toumi is also a visiting professor at Beijing University (Third Hospital).
In June 2022 Mondher Toumi founded InovIntell, an international venture dedicated to AI in life sciences.
He is a recognized expert in health economics and an authority on market access and risk management. He published more than 200 scientific publications and authored or co-authored several books predominantly in the fields of market access and health economics.
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Frank-Ulrich Fricke, PhD, MSc
Technische Hochschule Nürnberg, Nuremberg, Germany
Frank-Ulrich Fricke is a professor of health economics at the Technische Hochschule Nürnberg Georg Simon Ohm and an impartial member of the arbitration board on drug prices in the German healthcare system (Schiedsstelle nach § 130b SGB V) since 2011. He has served as a faculty dean since 2017. After studying business administration and a PhD in economics, Frank-Ulrich worked in industry and in consulting for several years. Main areas of interest have been market access, pricing and reimbursement, health policy and health economic evaluations. Frank-Ulrich is a member of several national as well as international professional associations.
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Keith H Tolley, BA, MPhil, MPP
Tolley Limited, Buxton, United Kingdom
Keith has over 35 years’ experience in health economics across academia (University of York - Centre for Health Economics and University of Nottingham, UK 1987-1997), for several pharmaceutical companies, including GSK, Pfizer, and Ortho Biotech (1997-2005), and in consultancy as a Director at Mapi (Adelphi) Values and now Tolley. He has direct experience of HTA as performed by NICE and SMC, and reimbursement and pricing issues around Europe. Keith has managed and strategically contributed to company submissions to NICE and SMC across a range of disease areas. He has also reviewed and been involved in the development of health economic models for NICE and SMC and other HTA bodies and has reviewed economic models for their suitability (eg, structure, data inputs) for drug reimbursement purposes.
Keith is also a health economics assessor with the SMC, a position he has held since 2005, having previously been an industry representative on the NDC. In 2013, Keith also became an assessor for the All Wales Medicine Strategy Group (AWMSG) and has provided expert advice as part of the NICE Early Scientific Advice Program.
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Krzysztof Kloc
Clever-Access, Kraków, Poland
Krzysztof Kloc is senior principal consultant in pricing, reimbursement, and market access (PRMA), vice head of the PRMA department, and co-founder of Clever-Access. Based in Krakow, Poland, he holds a master’s degree in applied biotechnology and has over 15 years of experience in market access and health technology assessment. Krzysztof has been engaged in consultancy projects in Poland related to the introduction and revisions of the Reimbursement Act, as well as in international projects, including stakeholder and pathway mapping, positioning and pricing strategy, evidence generation plans, and value communication. He was a speaker at the HTA Symposium in Krakow and is an active trainer for the International Market Access Upper Degree (IMAUD).
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Oriol INNOVATION TECHNOLOGY TRANSFER Solà-Morales Serra, PhD
Barcelona, Spain
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Claudio Jommi, MSc
Universita del Piemonte Orientale, Milan, Italy
11:00 - 12:00
First-Time Attendee Orientation
Session Type: General Meeting
New to ISPOR? Join us for this engaging and informative session designed to help first-time attendees make the most of their experience at ISPOR Europe 2026. You’ll gain insights into the conference structure, key sessions, and networking opportunities while connecting with fellow newcomers and ISPOR leaders. Whether you’re looking to navigate the agenda, maximize learning, or build professional relationships, this session will set you up for success. Don’t miss this chance to start your ISPOR journey with confidence!
12:00 - 13:00
Break (Lunch on Own)
Session Type: General Meeting
13:00 - 17:00
Causal Inference and Causal Estimands from Target Trial Emulations Using Evidence from Real-World Observational Studies and Clinical Trials
Session Type: Short Course
Topics: Real World Data & Information Systems
Track: Real-World Evidence (RWE)
Level: Advanced
Separate registration required.
In recent years, real-world evidence (RWE) has been increasingly used to inform regulatory, payer, and health technology assessment (HTA) decisions, as well as clinical guideline development. In addition, it has been recognized that the analysis of hypothetical estimands in clinical trials is necessary when the standard intention-to-treat (ITT) analysis does not answer the decision problem, usually because of treatment switching. An innovative framework for causal inference methods, target trial emulation, causal estimands and causal modeling guides the design and analysis of observational studies and clinical trials. This course will (1) introduce causal principles, causal diagrams (directed acyclic graphs; DAGs), and target trial emulation to avoid self-inflicted biases (eg, time-zero bias, immortal time bias), (2) provide an overview of causal methods for baseline confounding (multivariate regression, propensity scores) and time-varying confounding (eg, g-formula, marginal structural models with inverse probability of treatment weighting, and rank-preserving structural failure-time models with g-estimation), (3) propose appropriate estimands to ensure decision problems are directly addressed when analyzing observational data or data from clinical trials affected by treatment switching, (4) present lessons learned from applied case examples in HTA, such as single arm-trials with external control arms or trials affected by treatment switching, (5) provide recommendations regarding the use of causal inference methods and estimands and their application in causal modeling, and (6) discuss acceptance and barriers from an HTA agency perspective. The target audience includes all stakeholders and researchers from all fields in health and healthcare.
PREREQUISITE: Students are expected to have a basic knowledge in epidemiologic studies and methods (including the concept of confounding).
Speakers
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Uwe Siebert, MPH, MSc, ScD, MD
UMIT TIROL - University for Health Sciences and Technology; Harvard Chan School of Public Health, Hall in Tirol, Austria
Uwe Siebert, MD, MPH, MSc, ScD, is a professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL-University for Health Sciences and Technology in Austria and director of the Division for HTA in the ONCOTYROL–Center for Personalized Cancer Medicine in Austria. He is also adjunct professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health and Affiliated Researcher in the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston.
After medical school, he worked for several years as a physician in international public health projects in West Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in decision sciences at the Harvard School of Public Health.
His research interests include applying real-world evidence-based quantitative, causal and translational methods from public health, epidemiology, artificial intelligence, comparative effectiveness research, health services and outcomes research, economic evaluation, modeling, and health data a d decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, clinical guideline development, public health policies and patient guidance. His research focuses on cancer, infectious disease, cardiovascular disease, neurological disorders, and others.
He has been leading projects/work packages in several EU FP7, H2020 and Horizon Europe projects (eg, ELSA-GEN, BiomarCaRE, MedTecHTA, DEXHELPP, EUthyroid, FORECEE, MDS-RIGHT, RECETAS, CORE-MD, EUREGIO-EFH, CIDS, OnCoVID, 4D PICTURE, CATALYSE). He teaches HTA, health economics, modeling, epidemiology, causal inference and target trial emulation, and data and decision science for academia, industry, and health authorities in Europe, North and South America, and Asia. He directs the Continuing Education Program on Health Technology Assessment & Decision Sciences (htads.org).
He has served as member of the ISPOR Directors Board and as president of the Society for Medical Decision Making (SMDM). He is a leadership member of the ISPOR Personalized/Precision Medicine SIG, a member of the Latin America Consortium Advisory Committee of ISPOR, and co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force. He is a member of the Oncology Advisory Council and the National Committee for Cancer Screening of the Austrian Federal Ministry of Health.
He has authored more than 400 publications (> 30,000 citations, H index > 80), and is editor of the European Journal of Epidemiology. Further information Internet: http://htads.org, umit-tirol.at/dph, hsph.harvard.edu/uwe-siebert, Twitter: @UweSiebert9, LinkedIn: uwe-siebert9.
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Felicitas Kuhne, MSc, PhD
Pfizer Pharma GmbH, Berlin, Germany
Felicitas Kühne is a senior scientist and deputy coordinator at the Institute of Public Health, Medical Decision Making and Health Technology Assessment at the Department of Public Health, Medical Decision Making and Health Technology Assessment, UMIT TIROL - University for Health Sciences and Health Technology in Hall in Tirol, Austria. She is co-leading the Program on Causal Inference in Science and is the director of the HTADS course “Causal Inference for Assessing Effectiveness in Real-World Data and Clinical Trials: A Practical Hands-on Workshop”. Further, Felicitas Kühne is an outcomes research manager at Pfizer Pharma GmbH, Germany.
Felicitas Kühne holds a doctoral degree in Health Technology Assessment from UMIT TIROL as well as a master’s degree in health policy and management from the Harvard TH Chan School of Public Health, Boston, USA. She received her state approval as physiotherapist from the Georg-August-University of Göttingen, Germany and participated in Health-Economic Program of the University of Cologne, Germany. Before she started her position at UMIT TIROL in 2011, she worked as a consultant for pharmaceutical companies and healthcare providers, conducting several decision-analytic, real-world evidence, epidemiologic, and costing studies in a variety of disease areas.
Her research interests include evaluating public health interventions by applying advanced evidence-based quantitative methods from epidemiology, comparative effectiveness research, health services and outcomes research, economic evaluation, machine learning, and decision sciences. Her current substantive research focuses on identifying synergies of causal inference and decision science. The main disease areas are cardiovascular diseases, cancer, and infectious diseases including HIV/AIDS, hepatitis C, and pneumococcal disease. She teaches courses in decision-analytic modeling, economic evaluation, analysis of big data, and advanced causal epidemiologic methods at several universities and for industry in Europe and the USA.
She has authored several publications including textbook chapters and scientific articles and disseminated her finding at several conferences. She received financial support for her studies and research from several national and international organizations.
Felicitas Kühne is an active advisory board member for a NIMHD K01 award as well as a member of the editorial board of the journal of Medical Decision Making (MDM) and the journal of MDM Policy & Practice (MDM P&P). She is a member of the Working Group "Medical Decision Making" of the German Society for Medical Informatics, Biometry and Epidemiology (GMDS). She is also a member of the Professional Society for Health Economics and Outcomes Research (ISPOR), and the Society for Medical Decision Making (SMDM).
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Nicholas Latimer, MSc, PhD
SCHARR, University of Sheffield, Nottingham, United Kingdom
Nick joined the University of Sheffield in June 2008. Previously he worked as a research officer and analyst at NERA Economic Consulting, a Health Economics Fellow at Queen Mary, University of London (QMUL), and a Health Economist at Roche Products Ltd.
Nick has worked on several clinical trials, NICE Technology Appraisals and Public Health guidelines, and consultancy projects. Much of his work focuses on survival analysis and adjusting for treatment switching in clinical trials and he has completed NIHR Doctoral and Post-Doctoral Research Fellowships on these topics. In 2024, Nick completed a Senior Research Fellowship funded by Yorkshire Cancer Research in which he investigated the application of causal inference techniques to estimate comparative effectiveness from cancer registry datasets. He has authored NICE Decision Support Unit technical support documents on survival analysis (TSD14, 2011; TSD21, 2020), treatment switching (TSD16, 2014; TSD24, 2024), and partitioned survival analysis (TSD19, 2017), and was a member of Technology Appraisal Committee B for 5 years. He works part-time for Petauri Evidence.
https://www.sheffield.ac.uk/scharr/sections/heds/staff/latimer_n
Integrating Patient, Payer, and Investor Perspectives on Valuing Innovative Medicines for Orphan Diseases
Session Type: Short Course
Topics: Health Policy & Regulatory, Epidemiology & Public Health
Track: Expanded Value Measures
Level: Intermediate
Separate registration required.
Explore the value assessment of innovative drugs from the perspectives of relevant stakeholders, their respective data requirements, and their methods and processes. Gain a better understanding of the value assessment from the investor perspective, with a focus on orphan drugs and advanced therapy medical products (ATMPs).
The value of medical innovation depends on a stakeholder's perspective in different decision contexts. Regulatory authorities (EMA, FDA) mainly consider the clinical value of medical innovation. In the context of coverage decisions, national health authorities may adopt a broader perspective by including clinical, economic criteria, and sometimes even other criteria such as equity and social values. For pricing and reimbursement, "value-based pricing" is the most widely accepted approach across countries, but it can vary from a narrow concept based on the incremental cost-effectiveness ratio (ICER) threshold to broader societal or holistic approaches.
Value-based pricing determines the maximum price from the national payer perspective. In the context of the investment decision, this price should exceed the minimum price for the investor acting in the international financial market to make a financial valuation. Furthermore, there are numerous other stakeholders, eg, patients, physicians, healthcare insurers, and employers--with their specific assessment of the value of medical innovation including, for example, patient and family quality of life, real-world effectiveness, budget impact, and the costs of lost productivity. Familiarity with health economic evaluation is desirable, but the course assumes little or no familiarity with economic valuation theory.
Speakers
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Lou Garrison, PhD
The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, Seattle, WA, United States
Lou Garrison, PhD, is professor emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
For the first 13 years of his career, Dr. Garrison worked in non-profit health policy at Battelle and then the Project HOPE Center for Health Affairs, where he was the Director from 1989-1992. Following this, he worked as an economist in the pharmaceutical industry for 12 years. From 2002-2004, he was vice president and head of Health Economics & Strategic Pricing in Roche Pharmaceuticals, based in Basel, Switzerland.
Dr. Garrison received a BA in Economics from Indiana University, and a PhD in Economics from Stanford University. He has more than 150 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies.
Dr. Garrison was elected as ISPOR President for July 2016-June 2017, following other leadership roles since 2005. He recently co-chaired the ISPOR Special Task Force on US Value Frameworks. He was selected in 2017 by PharmaVOICE as being among “100 of the Most Inspiring People” in the industry. He recently received the PhRMA Foundation and Personalized Medicine Coalition 2018 Value Assessment Challenge First-Prize Award as lead author on a paper on “A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine.”
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Marlene Gyldmark, MPhil
BeiGene, Basel, Switzerland
In her current role, Marlene leads the EU HTA organizational readiness at BeiGene in the Global Value, Access, and Pricing group.
Marlene’s prior life science industry experience includes vice president global head Access Evidence at Idorsia, Switzerland; global head Health Policy and Outcomes Research at Roche Diabetes Care, Switzerland; global head Modelling, Outcomes Research, Statistics and Epidemiology, Roche Pharma, Switzerland; health economist at Pfizer Denmark, and Pricing and Economic Analyst at Novo Nordisk, Denmark. Before joining the life science industry, she worked as a researcher in the Danish Hospital Institute, Denmark and at University of Copenhagen, Denmark. Since 1996 Marlene has been an external lecturer at University of Copenhagen, Denmark.
Other work experiences include serving as a member of the board of directors (2000-2012) at EASE Consulting, Denmark and member of the board of the Institute of Neurodiversity (2021- 2025). She has been a long-term member of ISPOR and served as member of the Board of Directors between 2021-2024. Currently, Marlene also acts as a Copenhagen Goodwill ambassador.
She holds a master’s in economics and policy sciences from University of Copenhagen, Denmark, and a MPhil in health economics from York University, UK.
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Mark J Nuijten, MBA, PhD, MD
A2M, Amsterdam, Netherlands
Mark Nuijten is a medical doctor, health economist, valuation economist, and healthcare publicist. He is a visiting professor at Ben-Gurion University in Israel, setting up the department on Clinical and Economic Valuation of Medical Innovation. He has become a leading health policy and economics expert over the last 2 decades, reflected in more than 200 publications and leading positions in scientific societies and editorial boards. Dr. Nuijten was board director of ISPOR (2002-2004) and chair of the Management Board of Value in Health (2002-2004). He was a member of the Editorial Advisory Board of Value in Health. He obtained his PhD in health economics (2003) on the thesis “In search for more confidence in health economic modelling” at the Erasmus University, Rotterdam.
Mark Nuijten is founder of A2M (Ars Accessus Medica) and founding partner of the Minerva International Health Economic Network. He was trained as a physician and worked in clinical research before obtaining his international MBA from Erasmus University, Rotterdam, where he later was a senior staff member. Prior to setting up Ars Accessus Medica, Dr. Nuijten was the founding managing director of the IQVIA Quintiles office in the Netherlands, which included European responsibility for the policy and health economic division.
He is a pioneer in the field of healthcare innovation in biotechnology and has been the first classical health economist successfully applying and developing Discounted Cash Flow methodologies for valuation of biotechnology innovation (eg, a pricing model to assess prices of expensive orphan drugs from an investor’s perspective—published in a Nature journal). He also developed an integrated valuation model, an interactive dynamic tool for the economic valuation of R&D projects, which can be used to optimize the initial clinical program (eg, indication, comparator, outcomes, and study design), and the associated pricing and market access pricing strategy.
Applied Generative AI for HEOR: Robust Architectures
Session Type: Short Course
Topics: Methodological & Statistical Research
Track: AI
Level: Intermediate
Separate registration required.
Generative AI (GenAI) is rapidly transforming how HEOR and market access work is conducted, from literature reviews and evidence synthesis to dossier development and HTA submissions. As the field moves beyond experimentation, a new challenge emerges for subject matter experts: how to design and build AI tools that are rigorous enough to withstand scrutiny in regulatory, HTA, and payer engagement environments.
This intermediate-level course is designed for health economists, outcomes research professionals, market access specialists, and other HEOR subject matter experts who want to move beyond general-purpose chatbots and one-off pilots, and instead learn how to architect robust, purpose-built AI solutions. The emphasis throughout is on design: participants will learn how architectural choices determine whether an AI tool produces outputs that are reliable, traceable, and defensible.
The course is organized around three core themes, each illustrated with two applied examples that run throughout the course. These are complemented by ad hoc examples that showcase the diversity of possible architectures and applications across HEOR and market access.
Context Engineering
Large language models are only as good as the information they are given. Participants will learn how to design context: how external knowledge (eg, clinical data, published evidence, HTA guidance) is retrieved and incorporated into GenAI workflows. Retrieval-Augmented Generation (RAG) is treated as a cornerstone architecture, alongside complementary techniques such as tool use, that together determine factual accuracy, traceability, and domain fit.
Agentic AI
Participants will take an in-depth look at how autonomous and semi-autonomous AI agents can coordinate multi-step HEOR processes, such as structured data extraction and drafting workflows, while maintaining control, monitoring, and accountability. Faculty will discuss how to set boundaries for agents, orchestrate tasks, and design for human oversight.
Rigor for HTA
Building AI tools whose outputs will be scrutinized by HTA bodies, regulators, and payers demands a different standard than rapid prototyping. Faculty will address what separates "vibe-coded" solutions from HTA-ready tools and demonstrate how to evaluate and validate GenAI systems in terms of reliability, reproducibility, and regulatory alignment, drawing on frameworks such as ELEVATE-GenAI and guidance from NICE and the FDA. Ethical considerations around the application of AI are discussed in the same context. Participants will also learn how to set up a professional working environment, including effective use of an IDE, AI-assisted development, and reusable components such as skills.
The course provides full, runnable code for a worked example, discussed from a design and architecture perspective and highlighting a small number of key functions. Participants can run and adapt this example themselves after the course.
By the end of this course, participants will understand how to design AI architectures that live up to the evidentiary standards of HEOR and market access. They will leave with concrete design patterns, runnable reference implementations, and validation frameworks to build GenAI tools whose outputs can withstand scrutiny in HTA, regulatory, and payer settings. A basic understanding of Python or other similar scripting languages is recommended to get the most benefit from the provided worked examples.
PREREQUISITES: Attendance at “Applied Generative AI for HEOR: Introduction” or familiarity with concepts such as prompt engineering, APIs, and LLM workflows are necessary.
Speakers
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Sven L Klijn, MSc
Bristol Myers Squibb, Princeton, NJ, United States
Sven Klijn is Director at Bristol Myers Squibb in the Global HEOR Evidence Acceleration & Innovation group, where he leads the innovative modeling agenda in hematology and cell therapy. In addition, Sven has an active role in providing modeling and Generative AI education at international congresses. He has widely published on innovative methods, especially in the fields of survival extrapolation and Generative AI. Sven has a training in public health and health economics and previously had various roles in CROs related to health-economic modeling.
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Rajdeep Kaur, PhD
Pharmacoevidence Pvt. Ltd., Mohali, India
Dr. Rajdeep Kaur is the Lead of AI Sciences at Pharmacoevidence, with a Ph.D. in Computer Science and Engineering and 17+ years of expertise in advanced technologies. Her work focuses on Generative AI, machine learning, and cloud-enabled data systems, with a strong emphasis on real-world healthcare applications. She has successfully led multiple GenAI projects, combining deep technical expertise to deliver impactful AI-driven solutions.
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Ghayath Janoudi, PhD, MD
Loon, Ottawa, ON, Canada
Dr. Ghayath Janoudi, MBBS, MSc, PhD, is the Founder and CEO of Loon, an AI-driven clinical research and market access company developing scientifically validated AI agents for Health Economics and Outcomes Research (HEOR), Health Technology Assessment (HTA), and reimbursement strategy.
A medical doctor and health outcomes researcher by training, Dr. Janoudi holds a PhD in Clinical Epidemiology with a specialization in artificial intelligence for clinical research. He previously held senior leadership roles at Canada’s Drug Agency (formerly CADTH) and at clinical research organizations, where he led work on HTA, drug reimbursement policy, and value evidence evaluation.
A recognized thought leader in AI for clinical discovery, Dr. Janoudi is a well-published author in AI-enabled evidence synthesis, and was named Canada’s 2024 Emerging Healthcare Leader for his contributions to accelerating timely and equitable access to innovative therapies.
Practical Applications of Large Language Models for Real-World Evidence Generation and HEOR
Session Type: Short Course
Topics: Methodological & Statistical Research
Track: AI
Level: Intermediate
Separate registration required.
Examine large language models (LLMs) from industry leaders such as OpenAI, Anthropic AI, Google, and Meta, focusing on their application in real-world evidence generation and HEOR. The course covers technical LLMs, including their architecture, processing layers, attention mechanisms, embeddings, context window, hallucinations, risk-based frameworks, and current task-specific live benchmarks used for model assessment.
Participants will learn prompt engineering through hands-on, practical examples, empowering them to utilize commercially available LLMs. These examples include scientific literature retrieval, PICO extraction and processing, extracting and handling numerical data, summarizing tables and figures, automating captions, and generating code.
Upon completing this in-depth course, participants will gain the competencies needed to use LLMs responsibly for practical applications in RWE and HEOR, while remaining mindful of regulatory obligations. To participate in practical exercises, attendees are required to bring a personal laptop and have access to a personal or corporate LLM account with file upload functionality.
PREREQUISITE: General knowledge of chat-based LLMs (GPT, Claude, etc) is important. This is an intermediate course, and students should have prior knowledge of AI and have used chat based LLMs in a professional/work setting.
Speakers
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Manuel Cossio
Cytel Inc. Dubendord, Zürich, Switzerland; Universitat de Barcelona, Dubendorf, Switzerland
AI Engineer and Head of AI Solutions at Cytel with 13+ years of experience in HEOR. I lead the development of AI-driven solutions for evidence generation, economic modeling, and HTA landscaping—including EU JCA and market access. With expertise across both pharma and CRO consulting, I’m committed to advancing patient care through smarter, AI-enabled decision-making.
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Benjamin Bray, MD, MBChB, MSc, FFBCS
Lane Clark and Peacock, London, United Kingdom
Dr. Ben Bray is a medical doctor and epidemiologist and is Evidence Generation lead at LCP Health Analytics. He has been working in health data science and epidemiology for over 12 years and has extensive experience in the development and validation of machine learning models and in applications of AI using health data. He has authored over 60 publications including in The Lancet, BMJ and PLOS Medicine and has co-authored multiple reviews on the use of AI and machine learning in various therapy areas. He holds an Honorary Senior Clinical Lecturer post at King’s College London, focusing on research into machine learning analytics using large health databases.
Designing a Patient-Centered Strategy for Drug Development and Value
Session Type: Short Course
Topics: Patient-Centered Research
Level: Advanced
Separate registration required.
This course provides an in-depth discussion of the steps needed to successfully implement patient-reported outcomes (PRO) measurement within the drug development program to generate data to support patient-centered value messages. Formulation of a successful PRO strategy requires an understanding of PRO instrument selection, psychometric evaluation, data capture, and interpretation to negotiate regulatory, reimbursement, and market access drug development hurdles. Judging PRO instrument quality and appropriateness can be challenging.
The course will present the key elements to consider at each step in reviewing and selecting PRO measures and determining the need for new instruments. In addition, participants will gain a better understanding of regulatory expectations for qualitative and quantitative evidence to support the quality of PRO measures and aspects to consider when interpreting meaningful change. The course will include interactive discussions of PRO success stories and common pitfalls to watch out for during PRO implementation in clinical trial programs.
Participants will gain the knowledge and skills required to take on a more active and confident role in the PRO strategy and implementation process.
PREREQUISITE: This course assumes that participants will have a basic knowledge of key PRO-related concepts (eg, health-related quality of life, symptoms, impacts, a general knowledge of the PRO development steps, and a working knowledge of PRO measurement within clinical programs.)
Speakers
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Ari Gnanasakthy, MBA, MSc
RTI Health Solutions, Research Triangle Park, NC, United States
Ari Gnanasakthy is head of Patient-Reported Outcomes at RTI-HS. Prior to RTI-HS.
Mr. Gnanasakthy was the executive director and head of the Patient-Reported Outcomes Center of Excellence at Novartis Pharmaceuticals. He has almost 25 years of experience in the pharmaceutical industry. At Novartis, he worked in several departments, including Biostatistics, Health Economics, Pricing, and Outcomes Research. After receiving his bachelor's degree in mathematics, statistics, and computing, Mr. Gnanasakthy joined Rothamsted Experimental Station (UK), where he was responsible for the statistical analysis of survey data of agricultural soil in England and Wales. He then joined the Milk Marketing Board (UK), where he was a part of the team responsible for modeling lactation curves of dairy cows. Mr. Gnanasakthy's extensive experience in the field of statistics and outcome research has resulted in numerous abstracts and almost 40 publications. Throughout his career, Mr. Gnanasakthy has developed and validated over a dozen patient-reported outcomes instruments and currently serves in the editorial board of Cancer Clinical Trials and a reviewer for many professional journals, including Value in Health.
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Lynda Doward, MSc
RTI Health Solutions, Manchester, United Kingdom
Ms. Doward has over 30 years of experience conducting patient-centered outcomes research including the provision of strategic advice to pharmaceutical companies in the incorporation of the patient voice into drug development programs. Ms. Doward is an expert in the development of clinical outcome assessment (COA) strategies including the development of patient-centered clinical trial endpoints, the implementation of patient-reported and other COA outcome measures in clinical trial programs, and the inclusion of PRO and other COA value messages at key drug development hurdles. Ms. Doward has extensive experience in supporting pharmaceutical clients in their COA-related submissions to regulatory agencies in Europe and the US and advises on health-utility measurement strategies for reimbursement agencies in Europe. Ms. Doward has led the development of over 40 COA questionnaires that have been adapted and validated for use in over 60 languages worldwide.
Ms. Doward currently serves on the ISPOR COA Special Interest Group (leadership committee) and the ISPOR Patient Council (member) and was a member of the leadership committee of the completed ISPOR Good Research Practices Task Force for the measurement of health state utilities in clinical trials. Ms. Doward has acted as a consultant to the World Health Organization and has served as a Research Advisor to the UK Department of Health, and medical charities in the United Kingdom.
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Shanshan Qin, PhD
RTI Health Solutions, Research Triangle Park, NC, United States
Shanshan Qin, PhD, received her training on Qualitative Methodology (including statistic inference and estimation, traditional and modern testing theories, structural equation modeling, and mixed and mixture modeling) at University of Georgia. She has over 10 years of experience in leading, planning, and conducting psychometric analyses to evaluate measurement properties and interpretability of clinical outcome assessment (COA) scores; and supporting regulatory submission and publication of COA evidence. She has extensive experience with COAs in a variety of therapeutic areas, including mental and behavioral disorders, dermatology, oncology, gastroenterology, obesity, and ophthalmology.
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Rebecca Crawford, MA
RTI Health Solutions, Manchester, United Kingdom
Ms. Crawford has 13 years of experience providing consultative support to pharmaceutical companies with a focus on the development of patient-reported outcome (PRO) measurement strategies to best meet the needs of their clinical trial programs.
Ms. Crawford has developed, culturally adapted, and validated clinical outcome assessment measures, including PROs for several different therapeutic areas. Ms. Crawford has expertise in research design and in the application of both traditional and innovative qualitative research methods, including the collection and analysis of social media data to provide insights into the patient disease and treatment experience.
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Nicholas J. Rockwood, PhD
RTI Health Solutions, Bend, OR, United States
Nicholas Rockwood, PhD, is a senior psychometrician in the Patient-Centered Outcomes Assessment group with RTI Heath Solutions and has been working on psychometric evaluations of clinical outcome assessments. Prior to joining RTI-HS, Dr. Rockwood was an assistant professor within the School of Behavioral Health at Loma Linda University, where he conducted quantitative research, taught doctoral-level statistics courses, and provided statistical consulting services to medical and behavioral health faculty and researchers. His statistics and psychometrics research, which has been published in top psychometrics journals such as Psychometrika and Multivariate Behavioral Research, broadly focuses on the development and evaluation of generalized latent variable modeling methods (eg, item response theory, multilevel modeling, structural equation modeling).
Mon 9 Nov
7:00 - 8:30
Morning Coffee Service
Session Type: General Meeting
Don't miss the start of the day with the Plenary Session. Enjoy your morning coffee as you listen to dynamic presentations intended to inspire and empower.
7:00 - 17:00
Registration Hours
Session Type: General Meeting
8:30 - 10:00
Plenary Session 1: Welcome Remarks
Session Type: Plenary
Join us as we kick off ISPOR Europe 2026. Under this year’s conference theme, Shaping the Future of Access through Science and Policy, ISPOR’s Chief Executive Officer will set the stage with opening remarks highlighting the transformative role of health economics and outcomes research in advancing patient-centered care, strengthening global health systems, and addressing pressing challenges such as affordability, adoption, and health disparities.
Immediately following the opening presentations, the scientific plenary panel will take the stage.
Speaker
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Rob Abbott
ISPOR, Lawrenceville, NJ, United States
Plenary Session 1: The Science of What Matters: Rethinking Value in Healthcare
Session Type: Plenary
Topics: Health Technology Assessment, Patient-Centered Research, Economic Evaluation
Track: Expanded Value Measures
Level: Intermediate
This opening plenary challenges a fundamental assumption in HEOR: that current approaches to measurement fully capture what matters in healthcare decision-making. Drawing on behavioral science, wellbeing research, and health economics, the session will examine how widely used measures—while valuable—may overlook broader dimensions of value, including wellbeing, happiness, daily functioning, societal contributions, and lived experience.
The discussion will explore how what we choose to measure shapes priorities, incentives, and decisions across healthcare systems—and the risks of optimizing for what is easily quantified rather than what truly matters. A moderated interview and audience discussion will consider how broader measures of value could be incorporated into policy and HTA frameworks, the trade-offs involved, and whether today’s systems are equipped to support a more holistic, outcomes-driven approach to healthcare decision-making.
Moderator
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Sandra Nestler-Parr, MPhil, MSc, PhD
Biocryst Pharmaceuticals, Weybridge, United Kingdom
9:30 - 19:00
Exhibit Hall Hours
Session Type: General Meeting
9:45 - 10:15
Coffee and Connect
Session Type: General Meeting
Head to the exhibit hall to connect with fellow attendees and exhibitors over a steaming cup of coffee.
10:30 - 11:30
Beyond the Index Event: Aligning Hospital Decision-Making with Expanded Value Measures for Medical Devices and Diagnostics
Session Type: Issue Panel
Topics: Medical Technologies, Health Policy & Regulatory, Health Service Delivery & Process of Care
Track: Expanded Value Measures
Level: Introductory
ISSUE: As health technology assessment and value assessment frameworks increasingly incorporate expanded value measures, gaps remain in how these elements influence real-world decision making. Hospital technology adoption decisions for medical devices and diagnostics (MDD) may favor short-term value associated with the index event, which may limit the extent to which expanded value frameworks can shape patient access to innovation. This issue panel will examine opportunities to better align expanded value measures with hospital-level adoption and procurement decision making for MDD and how hospital decision-making processes can better reflect long-term patient and health system value.
OVERVIEW: Health technology assessment and value assessment frameworks are increasingly incorporating expanded value measures that consider patient-centered outcomes and long-term health impacts. MDD value is often generated downstream through improved long-term outcomes, reduced complications, and improved quality of life rather than the index event itself. Hospitals may prioritize short-term value over longer-term patient and system value since they typically bear the upfront cost of technologies while some downstream benefits may emerge later or outside the hospital setting. In this context, long-term patient benefits may not always be fully captured at the point of hospital decision making.
The moderator will open the session providing an overview of the issue to be discussed for 5 minutes, each speaker will provide their perspective for 15 minutes, and 10 minutes will be left for discussions and questions.
Moderator
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Belinda A Mohr, PhD
Medtronic, Phoenix, AZ, United States
Belinda A. Mohr has over 13 years of experience in the health economics and outcomes research (HEOR) medical devices space. She’s currently HEOR consultant on the corporate HEOR team at Medtronic and serves as the chair of the ISPOR Medical Devices and Diagnostics Special Interest Group. Prior to joining Medtronic, she was associate director of HEOR at Philips, where she spent 4 years leading HEOR real-world data and evidence initiatives and studies. Prior to Philips, Belinda held positions at W.L. Gore & Associates, Inc. for 9 years, including as a clinical strategist and as a health economist. She holds a PhD in economics from the University of California, Santa Barbara.
Speakers
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Marjan Hummel, PhD
Amsterdam, Netherlands
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Sally Lewis
United Kingdom
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Huw Thomas, MSc, CA
Camarthen, United Kingdom
Humans at the Helm: Navigating Increasing AI Autonomy in HEOR and HTA
Session Type: Spotlight
Topics: Health Technology Assessment, Organizational Practices, Health Policy & Regulatory
Track: AI
Level: Intermediate
ISSUEArtificial intelligence (AI) is rapidly transforming HEOR and HTA workflows, with increasing interest in moving from simple prompting and workflow automation toward more autonomous agentic AI systems. While AI offers significant opportunities to enhance productivity and augment expertise, its increasing autonomy also raises important questions around accountability, transparency, trust, and scientific rigour. As AI capabilities continue to advance, how should the HEOR and HTA community navigate increasing levels of AI autonomy while ensuring that human judgement and accountability remain central to decision-making? What guardrails that should be in place?This panel will explore how organisations can responsibly navigate increasing AI autonomy, examining when greater autonomy adds value and the governance and validation needed to maintain trust, confidence, and human judgement in decision-making.
OVERVIEWThe moderator, Bill Malcolm, will introduce the topic and frame the discussion on responsibly navigating increasing AI autonomy in HEOR and HTA. (6 min)Dalia Dawoud will provide the HTA and policy perspective (13 min), discussing the role of human oversight, transparency, and governance in AI-enabled workflows and the implications for evidence generation and decision-making.Jaime Caro will provide a methodological and research perspective (13 min), exploring how increasing AI autonomy may reshape professional practice, where additional complexity adds value versus unnecessary risk, and what should remain uniquely human in decision-making.Siguroli Teitsson will draw on practical industry experience (13 min) discussing how organisations assess and deploy different levels of AI autonomy, and how workflow design, governance and human judgement remain critical to successful adoption.The session will conclude with a moderated panel discussion and audience Q&A (15 min) exploring practical approaches to navigating increasing AI autonomy while maintaining human judgement, accountability, and trust across the HEOR and HTA ecosystem. To encourage active participation, the session will incorporate live audience polling and voting on key questions.
Moderator
Speakers
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Dalia Dawoud, BSc, MSc, PhD
Cytel, London, United Kingdom
Dalia Dawoud, PhD, is Research Principal, HTA Policy and Strategy. She is also the Director and CEO of PEHTA Consulting Ltd. and holds a professor position at the Faculty of Pharmacy, Cairo University. She has over 15 years experience as a health economist and researcher. Her work is largely focused on the application of HEOR in HTA and clinical guideline development. She worked at leading organizations including NICE, where she led a portfolio of HORIZON Europe projects such as HTx, EDiHTA and SUSTAIN HTA, and the Royal College of Physicians, London. She is widely published in the areas of health economics and outcomes research and serves as associate editor for Value in Health and as director on ISPOR Board of Directors (2023-2026). She is also a member of the ISPOR AI Working Group and ISPOR Living HTA Working Group.
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Jaime Caro, PhD
Boston, United States
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Siguroli Teitsson, BSc, MSc
Bristol Myers Squibb, Denham, United Kingdom
Siguroli Teitsson is a Director in Global HEOR Economic & Predictive Modeling at Bristol Myers Squibb. In his role, Siguroli leads the advancement of innovative modeling and analytics in oncology, and drives the integration of cutting-edge AI automations in HEOR and market access, streamlining workflows to accelerate patient access to medicines. With a background in engineering and health economics, he has previously held senior roles in CROs and has extensive publication record in innovative analytics within the field of HEOR, contributing to advancements in methodology and practice.
Capturing Patient and Caregiver Experiences in Psychological and Cognitive Conditions
Session Type: Research Podiums
Patient-centered assessment requires measures that reflect the full impact of disease on patients and caregivers. This session focuses on cognitive symptoms, caregiver burden, quality of life, and meaningful change in outcomes across psychological and neurological conditions, providing evidence to support more comprehensive and patient-centered evaluation of treatment benefit.
POPULATION PREFERENCES FOR MODE OF ADMINISTRATION IN EARLY ALZHEIMER'S DISEASE THERAPY: UTILITY DECREMENTS ESTIMATED BY A DISCRETE CHOICE EXPERIMENT IN JAPAN
OBJECTIVES: To quantify treatment preferences of the Japanese general population for early Alzheimer's disease (AD) as utility decrements (disutilities), covering both clinical attributes and treatment-related burden, with particular focus on different modes of administration, namely subcutaneous injection (SC) and intravenous infusion (IV), using a discrete choice experiment (DCE).
METHODS: A web-based DCE was administered to 2,194 Japanese adults (aged 40-79 years), yielding 65,820 observations. Health states were defined by five attributes: survival years, memory, mood, ability to perform housework, and treatment-related burden. The treatment-related burden attribute captured route of administration, visit frequency, administration time, and setting (self-administration versus administration at a healthcare facility). A conditional logit model was estimated with survival years as a continuous variable and other attributes as interactions with survival years. Each level's disutility was obtained by dividing its coefficient by the survival-year coefficient. Subgroup (age, sex, caregiving experience, contact with patients) and sensitivity analyses by response quality were performed.
RESULTS: The relative importance of attributes was mood (29.9%), memory (27.2%), treatment-related burden (25.5%), and housework ability (17.5%). Both modes of administration produced disutilities relative to a hypothetical reference of monthly outpatient visits without injection: weekly SC -0.339 and biweekly IV -0.388, indicating a lower burden for SC than for IV (disutility difference 0.049). The disutility associated with treatment-related burden remained directionally stable across subgroups and sensitivity analyses.
CONCLUSIONS: Among the Japanese general population, treatment burden for early AD differed by mode of administration, with SC showing a lower burden than IV. As these utility estimates reflect general-population preferences, they can inform value assessment of early AD therapies that differ in administration burden.
CHARACTERIZING THE PATIENT EXPERIENCE OF COGNITIVE IMPAIRMENT ASSOCIATED WITH BIPOLAR DISORDER: A QUALITATIVE INTERVIEW STUDY
OBJECTIVES: People living with bipolar disorder (BD) can experience cognitive symptoms even in the stable (euthymic) phase of the disease. The objective of this study was to identify the most relevant and impactful concepts of cognitive impairment (CI), to support the selection / development of patient-centered clinical outcome assessments (COAs) for use in clinical trials.
METHODS: A non-interventional qualitative interview study was conducted in the United States. Adults living with BD with confirmed CI (based on clinician impression) associated with their BD (CIABD) and caregivers participated in semi-structured, one-to-one concept elicitation interviews exploring patient-reported and caregiver-observed symptoms, impacts, and patient experience with CIABD. Transcripts were analyzed using a combined deductive and inductive coding approach. Concept saturation was assessed across interview waves.
RESULTS: A total of 35 participants were interviewed (26 patients and 9 caregivers). Seven CI concepts were identified across six DSM‑5 neurocognitive domains: attention, executive functioning, memory, learning, language, social cognition, and perceptual motor function. Problems with attention and executive functioning were reported by all participants (100%), followed by language (89%) and memory (86%). Difficulties with attention - particularly concentration and sustained attention - were frequently described and rated as bothersome (mean rating 7/10). Participants reported that CI impacts multiple aspects of daily life, including day-to-day functioning, emotional wellbeing, work or school performance, and social relationships, highlighting the broad and multidimensional burden of CIABD. Caregivers reported similar observations and described additional impacts related to caregiving and their own emotional burden. Concept saturation was achieved for symptom and impact concepts .
CONCLUSIONS: This study provides novel patient-centered evidence for CIABD that described salient symptoms and impact domains relevant to those living with the condition. The findings support the evaluation of the most suitable COAs that reflect concepts that are relevant and meaningful to patients in line with patient-focused drug development efforts in CIABD.
IS EQ-5D APPROPRIATE FOR MEASURING THE HEALTH-RELATED QUALITY OF LIFE OF CAREGIVERS OF PEOPLE WITH DEMENTIA DUE TO ALZHEIMER'S DISEASE? A SYSTEMATIC LITERATURE REVIEW
OBJECTIVES: There is disagreement about whether EQ-5D is appropriate for measuring caregiver’s health-related quality of life (HRQoL) in Alzheimer’s Disease, because studies report small changes in carers’ EQ-5D as patient severity increases. We aimed to understand if EQ-5D is appropriate in this context by systematically examining if it is statistically significantly associated with factors related to patient health and caregiving burden.
METHODS: We consolidated evidence from two National Institute for Health and Care Excellence appraisals and conducted a systematic literature review using 4 databases to identify studies that analysed the relationship between caregivers’ EQ-5D scores and disease-related or caregiving variables. We extracted data from studies that used multivariate regression or longitudinal analyses to control for potential confounding.
RESULTS: We included 46 studies: 5 were longitudinal, 15 reported multivariate analyses, 1 used both, and 26 were only univariate analyses. We grouped studies which analysed the effect of similar variables on carers’ HRQoL. 6 studies analysed the effect of caregiving burden (4 using Zarit Burden Interview (ZBI)): all found significant results. 3 studies analysed proxy-reported patient HRQoL: 2 found significant results. 5 studies analysed the effect of patient disease severity: 3 found significant results. 5 studies analysed the effect of patient symptoms/needs: 3 found significant results. Carers’ EQ-5D was not statistically significantly associated with care setting (5 studies), caring duration (2 studies), or patient-reported HRQoL (2 studies). A one unit increase on the ZBI 12-item 0-48 scale corresponded to an EQ-5D disutility of 0.002-0.006.
CONCLUSIONS: EQ-5D demonstrates validity in caregivers via its responsiveness to changes in caregiving burden. Predicting caregivers’ HRQoL from patient severity alone oversimplifies the caregiving experience and is inaccurate and insufficient for economic evaluation. Standard psychometric tests using univariate analysis are poorly suited to analysing caregivers’ HRQoL data, as they do not control for the multiple inter-related factors that affect caregivers.
DOES THE EQ-HWB-9 CAPTURE CAREGIVER BURDEN? EVIDENCE FROM FAMILY CAREGIVERS OF PEOPLE WITH SCHIZOPHRENIA IN CHINA
OBJECTIVES: This study examined the construct validity of the 9-item EQ Health and Wellbeing instrument (EQ-HWB-9) in caregivers of people with schizophrenia. We also explored how the psychometric performance of EQ-HWB-9 and EQ-5D-5L differed in this caregiver population.
METHODS: Primary family caregivers of people with schizophrenia were recruited from a mental health hospital in Nanjing, China, between January and November 2025. Caregivers completed paper-based questionnaires including sociodemographic questions, ZBI-22, EQ-5D-5L, and EQ-HWB-9. EQ-5D-5L and EQ-HWB-9 index scores were derived using the Chinese and experimental UK value sets, respectively. Convergent construct validity was assessed using correlations with ZBI-22. Known-groups validity was assessed by comparing groups known to differ in daily caregiving hours, patient stable status duration, and caregivers’ health conditions using Cohen’s effect size (ES) and relative efficiency (RE).
RESULTS: Among 300 caregivers, mean age was 57.0 years and 33.0% were female. Mean EQ-5D-5L, EQ-HWB-9, and ZBI-22 scores were 0.879, 0.809, and 32.6, respectively. Exhaustion was the most frequently affected EQ-HWB-9 dimension (65.4%). ZBI-22 was strongly correlated with the EQ-HWB-9 index (r=-0.68), and moderately correlated with the EQ-5D-5L index (r=-0.45). At the item level, ZBI-22 was strongly correlated with six EQ-HWB-9 items (r=0.50-0.60), but only with the EQ-5D-5L anxiety/depression dimension (r=0.53). Both ZBI-22 and EQ-HWB-9 scores significantly differentiated all caregiver subgroups. However, EQ-5D-5L index scores of subgroups differing in patient stable status duration were not significantly different. EQ-HWB-9 showed greater discrimination in caregiving hours (ES: 0.60 vs 0.37; RE=2.68) and patient stable status duration (ES: 0.35 vs 0.02; RE=709.76), whereas EQ-5D-5L discriminated better for caregivers’ own health conditions (ES: 0.58 vs 0.38; RE=2.33).
CONCLUSIONS: EQ-HWB-9 demonstrated good construct validity among Chinese family caregivers of people with schizophrenia. Although EQ-5D-5L captured caregiver burden to some extent, EQ-HWB-9 appeared more sensitive to differences in caregivers’ wellbeing.
Early Value Assessment When There Are Multiple External Comparators: Introducing Value-Based Decision Boundary Analysis (VB-DBA)
Session Type: Workshop
Topics: Methodological & Statistical Research, Study Approaches, Health Technology Assessment
Track: Expanded Value Measures
Level: Intermediate
PURPOSE: Health technology developers (HTDs) currently use early cost-effectiveness models to determine economically justifiable prices (EJPs) based on target product profiles (TPPs). These are then used to evaluate future commercial decisions, such as whether to move forward with development or how to optimize launch. Whilst this analysis provides important insights, a major limitation is only being able to evaluate a single TPP at a time. This workshop demonstrates a novel methodology, termed Value-Based Decision Boundary Analysis (VB-DBA), which enables practitioners to use network meta-analysis to determine minimum TPP requirements that satisfy different decision-making criteria and give rise to a range of EJPs. Attendees will learn how to utilize and deploy this methodology to support early commercial feasibility assessments for their assets.
DESCRIPTION: Attendees will learn how to implement VB-DBA and the value of this type of analysis. Workshop contents include a review of existing approaches and challenges, detailed VB-DBA methodology and execution, and the relevance to HTDs. Ronan Mahon will moderate the session and introduce the topic in the context of drug development (8 minutes). This will include real-time audience polling regarding HEOR approaches currently in use during early asset assessments. Subhajit Gupta will provide a detailed walkthrough of statistical methods, data requirements and assumptions required to deploy these methods (20 mins). George Joseph and Kokuvi Atsou will illustrate how these methods are being used in drug development and how they may affect launch strategy in practice (10 mins each). Additional audience participation will include identifying challenges and solutions for a hypothetical case study in renal cell carcinoma (7 mins). This will form a worked example presented by Ronan Mahon. The final 5 minutes will be audience questions and feedback. This interactive informative workshop will be valuable to researchers, pharmaceutical executives and health economic/evidence synthesis analysts interested in combined health economic/evidence synthesis methods required for decision-making within early phase drug development.
Moderator
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Ronan Mahon
University of Galway, Dun Laoghaire, Ireland
Speakers
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Subhajit Gupta
India
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George Joseph, MS, PhD
Cambridge, MA, United States
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Kokuvi ATSOU, PhD
Chilly-Mazarin, France
Navigating Europe’s New Era of Health Policy Reform: How Will the EU HTA Regulation, Pharmaceutical Legislation Revision, Critical Medicines Act, and Biotech Act Shape Medicines Development and Patient Access?
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment, Organizational Practices
Track: Access and Pricing
Level: Intermediate
ISSUE: The European health policy environment is experiencing its most significant transformation in decades. In January 2025, the EU HTA Regulation began to apply. Simultaneously, revisions to the Pharmaceutical Legislation and prospective Critical Medicines Act (CMA) and Biotech Act are reshaping market access strategy.
OVERVIEW: Four interconnected legislative shifts are reshaping how medicines are developed, assessed, and accessed. JCAs under the EU HTA Regulation are now underway for oncology and ATMPs, expanding to orphan medicines in 2028 and all medicines in 2030. Manufacturers face new requirements for harmonised evidence dossiers, accelerated timelines, and coordinated multi-stakeholder engagement. Reforms to the Pharmaceutical Legislation introduce changes to regulatory exclusivity periods, new launch obligations, and procedural timelines with direct consequences for development strategy, pricing negotiations, and portfolio planning. CMA will seek to improve access to not just critical medicines but also medicinal products of common interest: therapies that are not sufficiently available in several countries, including orphan drugs. The Biotech Act aims to strengthen Europe's biotechnology competitiveness, streamline regulatory pathways for innovative biologics, and address supply chain resilience while raising questions about alignment with existing HTA and regulatory processes. Not only are these four political landscape changes significant individually, they also influence one another. These reforms demand unprecedented coordination across regulatory, HTA, and market access functions among manufacturers, government stakeholders, and academia. This panel will provide authoritative perspectives on implementation status, practical challenges, and strategic intent behind each initiative; examine interdependencies between regulatory approval, JCA, and national HTA/pricing processes; and identify actionable insights for all stakeholders navigating this landscape. Panellists include the Chairs from the EMA Management Board and JCA Subgroup, and a MA specialist offering regulatory, HTA, and commercial perspectives on Europe's evolving policy environment.
Moderator
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Suzette Matthijsse
Lumanity, Sheffield, United Kingdom
I am passionate about accelerating patient access to innovative therapies by shaping evidence, strategy, and cross-functional decision-making across the product lifecycle. With 15 years of experience spanning academia and HEOR consulting, I specialize in guiding pharmaceutical and biotech partners through the evolving EU HTAR landscape from early development to Joint Clinical Assessments since the EU HTAR entered into force in 2022.
Speakers
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Rui SANTOS IVO, Assoc. Prof.
European Medicines Agency, Lisbon, Portugal
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Anne Willemsen, MSc
Zorginstituut Nederland, Diemen, Netherlands
Anne Willemsen joined the Dutch National Healthcare Institute in 2016, and currently is Chair of the Joint Clinical Assessment (JCA) subgroup under the EU HTA Regulation’s Coordination Group, after having served the JCA Subgroup as co-chair for the past three years (2023-2026). She leads the subgroup through the preparatory phase of the JCA process and will guide it through the production of Joint Clinical Assessments started as of January 2025. Previously she held several roles within the European Network for HTA (EUnetHTA), including spearheading the Secretariat responsible for coordinating JCAs and joint work with the European Medicines Agency. Her work also focused on patient involvement and conflict of interest procedures. With a background in Health Sciences and a recently started PhD, she brings extensive experience in European HTA collaboration, focusing on predictability of the procedures, transparency and stakeholder engagement in JCA.
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Neil Grubert
Neil Grubert Consulting, Basildon, United Kingdom
Neil Grubert spearheaded the development of Decision Resources Group’s international market access business, most recently as Vice-President of Global Market Access Insights. Since October 2014, he has been working as an independent global market access consultant, trainer, and writer. He has completed projects for 14 of the world’s top 20 pharmaceutical companies, smaller manufacturers and industry associations. He is a leading voice on pharmaceutical pricing and market access, with more than 26,000 followers of his daily posts on LinkedIn for insights into global trends and policy developments.
Patient-Reported Outcomes in Federated Real-World Data: Infrastructure Ready, Evidence Fit for HTA?
Session Type: Issue Panel
Topics: Patient-Centered Research, Real World Data & Information Systems, Health Technology Assessment
Track: Patient-Centered Evidence
Level: Intermediate
ISSUE
Health Technology Assessment (HTA) bodies, regulators, and healthcare decision-makers increasingly require high-quality longitudinal patient-reported outcome (PRO) evidence to evaluate real-world treatment effectiveness and safety, evidence that trial environments alone cannot provide. The infrastructure to support prospective PRO collection now exists: validated instruments such as the EQ-5D-5L, digital collection platforms, and expanding evidence networks such as DARWIN EU® have meaningfully expanded opportunities to generate, characterize, and reuse PRO data in real-world settings. Yet despite this maturity, PRO evidence rarely reaches decision-makers in credible, reusable form. Collection remains fragmented and episodic, fit for individual studies but not designed for repeated use across research questions and decision contexts. The gap is no longer primarily technical. It is structural.
OVERVIEW
The panel brings together complementary perspectives across the PRO evidence lifecycle: European RWE infrastructure with Prof. Verhamme, patient-centered outcomes implementation with Dr. Rylands, operational evidence generation with Dr. Lawes, and HTA decision science with Prof. Siebert. The session moves from infrastructure realities through to HTA utility, building toward a shared examination of why meaningful generation and reuse of longitudinal PRO evidence continues to lag behind both infrastructure capacity and decision-maker demand. Panelists will explore unresolved tensions around continuity, sustainability, and what "reusable" longitudinal real-world PRO evidence – evidence designed from the outset for repeated use across studies settings, and stakeholders – actually requires in practice. Audience polling will be used at key moments to surface where participants see the sharpest barriers, grounding the moderated discussion in real-world priorities rather than theoretical ones.
Moderator
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Uwe Siebert, MPH, MSc, ScD, MD
UMIT TIROL - University for Health Sciences and Technology; Harvard Chan School of Public Health, Hall in Tirol, Austria
Uwe Siebert, MD, MPH, MSc, ScD, is a professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL-University for Health Sciences and Technology in Austria and director of the Division for HTA in the ONCOTYROL–Center for Personalized Cancer Medicine in Austria. He is also adjunct professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health and Affiliated Researcher in the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston.
After medical school, he worked for several years as a physician in international public health projects in West Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in decision sciences at the Harvard School of Public Health.
His research interests include applying real-world evidence-based quantitative, causal and translational methods from public health, epidemiology, artificial intelligence, comparative effectiveness research, health services and outcomes research, economic evaluation, modeling, and health data a d decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, clinical guideline development, public health policies and patient guidance. His research focuses on cancer, infectious disease, cardiovascular disease, neurological disorders, and others.
He has been leading projects/work packages in several EU FP7, H2020 and Horizon Europe projects (eg, ELSA-GEN, BiomarCaRE, MedTecHTA, DEXHELPP, EUthyroid, FORECEE, MDS-RIGHT, RECETAS, CORE-MD, EUREGIO-EFH, CIDS, OnCoVID, 4D PICTURE, CATALYSE). He teaches HTA, health economics, modeling, epidemiology, causal inference and target trial emulation, and data and decision science for academia, industry, and health authorities in Europe, North and South America, and Asia. He directs the Continuing Education Program on Health Technology Assessment & Decision Sciences (htads.org).
He has served as member of the ISPOR Directors Board and as president of the Society for Medical Decision Making (SMDM). He is a leadership member of the ISPOR Personalized/Precision Medicine SIG, a member of the Latin America Consortium Advisory Committee of ISPOR, and co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force. He is a member of the Oncology Advisory Council and the National Committee for Cancer Screening of the Austrian Federal Ministry of Health.
He has authored more than 400 publications (> 30,000 citations, H index > 80), and is editor of the European Journal of Epidemiology. Further information Internet: http://htads.org, umit-tirol.at/dph, hsph.harvard.edu/uwe-siebert, Twitter: @UweSiebert9, LinkedIn: uwe-siebert9.
Speakers
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Angela Jane Rylands
United States
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Katia Verhamme
Rotterdam, Netherlands
Katia Verhamme is Associate Professor of Use and Analysis of observational data. Katia graduated as medical doctor from the University of Ghent and trained as General Practitioner. Within the department of medical informatics, she is leading the pharmacoepidemiology group and is an expert on multi-database studies. Since her PhD graduation as pharmaco-epidemiologist from the Erasmus University Medical Centre, Katia has successfully developed a research group conducting pharmaco-epidemiological research, using electronic patient records as compiled in various observational health care databases. She has a specific interest in the field of respiratory pharmaco-epidemiology and has successfully coordinated multiple Post-Authorisation Safety Studies requested by the EMA. She currently is Deputy Director of Darwin EU® and leads the Study Operations Pillar within Darwin EU®. She is also guest professor Pharmaco-epidemiology at the Faculty of Pharmaceutical Sciences at the University of Ghent.
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Mario Lawes
Data4Life, Potsdam, Germany
Converting Vaccine Evidence to Policy
Session Type: Research Podiums
This session explores the broader societal, economic, and equity benefits of vaccines, highlighting how vaccination can improve population wellbeing and healthcare system sustainability.
IMPROVING INFLUENZA VACCINATION UPTAKE AMONG HEALTHCARE PROFESSIONALS: A GLOBAL UMBRELLA REVIEW IDENTIFYING BEHAVIORAL AND SYSTEM-LEVEL LEVERS FOR POLICY ACTION
OBJECTIVES: Seasonal influenza imposes a recurrent and preventable burden on healthcare systems, with substantial clinical and economic impact. Healthcare professionals (HCPs) play a critical role in both transmission control and vaccination advocacy; however, vaccination uptake remains persistently suboptimal. This study aims to identify the key determinants of influenza vaccination uptake among HCPs and inform policy strategies to improve coverage at scale.
METHODS: An umbrella review was conducted in accordance with PRISMA 2020 guidelines. PubMed, Cochrane Library, and Google Scholar were systematically searched for review articles published between 2000 and 2025 examining HCPs’ beliefs, attitudes, and behaviors toward influenza vaccination. Eligible studies included systematic and narrative reviews focusing on HCPs. Methodological quality was assessed using AMSTAR-2. Determinants were synthesized across three domains: behavioral, professional, and system-level drivers.
RESULTS: Twenty-five reviews were included, encompassing diverse healthcare settings and regions. Vaccination uptake among HCPs remains below recommended targets, with variation across contexts. Behavioral drivers included perceived protection of self and family and confidence in vaccine effectiveness as facilitators, while safety concerns and low perceived disease risk were principal barriers. Professional factors showed higher uptake among physicians and frontline clinical staff compared with non-clinical personnel, with younger professionals more likely to vaccinate and recommend vaccination. System-level interventions, including free and on-site vaccination, organizational programs, and mandatory vaccination policies, were strongly associated with higher uptake, often achieving near-target coverage. HCP vaccination behavior was consistently associated with higher likelihood of recommending vaccination, amplifying population-level impact.
CONCLUSIONS: Influenza vaccination uptake among HCPs is driven by the interaction between behavioral factors and health system design rather than knowledge alone. Educational interventions alone are unlikely to achieve meaningful improvements. Strategies should integrate behavioral interventions with structural measures, including facilitated access and institutional policies. Strengthening HCP vaccination represents a scalable, system-level intervention with potential to improve population-level vaccination coverage and reduce influenza burden.
GREENHOUSE GAS EMISSIONS AVOIDED THROUGH HPV VACCINATION IN CERVICAL CANCER AND CIN3 CARE PATHWAYS
OBJECTIVES: Vaccination programmes deliver clinical and economic benefits, yet their environmental value remains unquantified. Healthcare systems contribute approximately 4-5% of global greenhouse gas (GHG) emissions, with care pathways, including surgery, radiotherapy, chemotherapy, and patient travel, representing a meaningful share of this burden. Preventive interventions that reduce disease incidence offer potential environmental co-benefits alongside health. The human papilloma virus (HPV) vaccination substantially reduces cervical cancer and cervical intraepithelial neoplasia (CIN3) incidence, yet GHG emissions avoided have not been quantified. This research aims to estimate GHG emissions associated with cervical cancer and CIN3 care pathways in England, and quantify emissions avoided under observed HPV vaccination versus a counterfactual unvaccinated scenario.
METHODS: Published estimates of cervical cancer (n=687) and CIN3 (n=23,192) cases prevented through HPV vaccination in England by June 2020 were used to parameterise an environmental model comparing GHG emissions under vaccinated and counterfactual unvaccinated scenarios. Care pathways were mapped by disease stage using published clinical guidance. Stage IV was limited to first-line treatment due to subsequent heterogenous management. GHG emissions were assigned to surgery, inpatient stay, outpatient treatment, radiotherapy/chemotherapy sessions, and patient travel using Sustainable Healthcare Coalition emission factors. Emissions were calculated per patient and across the modelled population.
RESULTS: Total cervical cancer treatment emissions decreased from 380,254kg carbon dioxide equivalents (CO₂e) for those unvaccinated to 202,423kg CO₂e for vaccinated patients, avoiding 177,832kg CO₂e (-46.8%).The largest absolute reduction occurred in stage IB/IIA disease, avoiding 135,588kg CO₂e, followed by stage IIB/III/IVA with 32,612kg CO₂e. Total CIN3-related emissions decreased from 1,465,187kg CO₂e to 736,958kg CO₂e, avoiding 728,229kg CO₂e (-49.7%). Total average per-patient emissions were 31.4kg CO₂e for CIN3 and 259.1kg CO₂e for cervical cancer.
CONCLUSIONS: HPV vaccination substantially reduces healthcare-related GHG emissions by mitigating the need for cervical cancer and CIN3 treatment. These findings highlight an additional environmental value of vaccination programs beyond clinical and economic benefits.
THE POLICY CASE FOR PAEDIATRIC INFLUENZA VACCINATION STRATEGIES: LAIV MODELING ANALYSIS OF PUBLIC HEALTH IMPACT AND COST SAVINGS IN EUROPE
OBJECTIVES: Childhood vaccination with live attenuated influenza vaccine (LAIV) is a cost-effective strategy for protecting children and reducing influenza-like illness across Europe. Vaccinating children can interrupt transmission across the broader population, thereby reducing infections across age groups. This study evaluated the epidemiological and economic impact of increasing paediatric LAIV vaccination (children aged 2-17 years) across five European markets.
METHODS: A compartmental dynamic transmission model simulated influenza circulation within a population over one year under current vaccination practices (inactivated influenza vaccine only; reference case). A linked decision tree translated symptomatic infections into clinical outcomes, healthcare resource use, and mortality. Scenarios that increase paediatric vaccination coverage (VCR) to 25% and 50% were compared with the reference case for each market (Austria, Catalonia, Czechia, Romania, and Sweden). Outcomes included infections, hospitalisations, deaths, costs and quality-adjusted life years, capturing clinical and economic burden of seasonal influenza under current and alternative vaccination strategies.
RESULTS: Increasing paediatric LAIV coverage generated consistent benefits across all five countries/regions. Expanding VCR to 25-50% reduced overall infections by 34-48% and 57-71%, respectively, accompanied by marked declines in hospitalisations and mortality. While 40-78% of infections were prevented in the target group, 32-69% of influenza cases were prevented in adults due to LAIV expansion. Despite heterogeneity in demographics and baseline influenza burden, results were directionally consistent. LAIV expansion was cost‑effective across all scenarios and was dominant in two markets from a payer perspective and in most scenarios from a societal perspective. Owing to the substantial indirect effects of LAIV expansion, up to $8.6 in total savings (direct and indirect costs) per $1 invested could be achieved.
CONCLUSIONS: Increasing paediatric LAIV coverage substantially reduces influenza burden, with pronounced indirect protection in adults. Expanding childhood vaccination represents a high-impact, cost-effective strategy to improve population health and relieve pressure on healthcare systems.
PHARMACIST-LED VACCINATION IN FRANCE: DRIVERS OF ACCEPTABILITY ACROSS HEALTHCARE USERS, GENERAL PRACTITIONERS, AND PHARMACISTS
OBJECTIVES: In 2023, pharmacists in France were allowed to prescribe and administer recommended vaccines to individuals aged ≥11 years. This study evaluated factors influencing acceptability among healthcare users, general practitioners (GPs), and pharmacists.
METHODS: In 2025, 300 pharmacists, 300 GPs, and 3,000 adult healthcare users, representative of the French population, completed a web-based cross-sectional survey. Acceptability of pharmacists’ prescribing and administering vaccines was measured using Likert scales. Linear Discriminant Analysis (LDA) identified variables discriminating between higher and lower acceptability.
RESULTS: Over the past two years, 53.1% of healthcare users were vaccinated in pharmacies, rising to 77.9% among those ≥65 years and 76.8% among infectious risk seniors. LDA showed age as the strongest discriminator of acceptability, with heterogeneous effects. Acceptability increased with age among users, from a mean (SD, age group) of 7.4/10 (2.2, 18-29 years) to 8.6/10 (1.9, ≥75 years), while it declined among pharmacists from 9.8/10 (0.7, <45 years) to 9.0/10 (1.5, >55 years). GPs showed the lowest scores, decreasing from 5.6/10 (3.1) to 4.8/10 (3.0) across age groups comparable to pharmacists. Pharmacists were most comfortable prescribing (96.0%) and administering (97.7%) vaccines to users ≥65 years without chronic conditions, and least comfortable with immunocompromised patients (50.0% prescription, 76.7% administration) and those with allergies (53.7%, 65.7%). GPs were favourable to pharmacists’ prescription (52.0%) and administration (59.0%) to older users without chronic conditions, but less favourable for immunocompromised patients (26.7%, 37.3%), pregnant women (30.0%, 37.0%), patients on anticoagulants (30.7%, 36.3%) and those with allergies (24.0%, 32.0%).
CONCLUSIONS: Stakeholders’ age and healthcare users’ characteristics influence acceptability of pharmacy-based vaccination. Older users and even those at infectious risk show the greatest engagement and acceptability. Conversely, pharmacists and GPs reported lower confidence in the management by pharmacists of certain profiles, such as immunosuppression, highlighting the need for targeted training and stronger interprofessional collaboration to maximise public health impact.
When Global Policy Agendas Collide – Evidence, Value, and Pricing Tensions at the Intersection of EU and US Reform
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment
Track: Access and Pricing
Level: Introductory
Issue: Market access strategy is being reshaped by evolution of EU and US policy reforms that place competing emphasis on evidence generation and pricing. EU Joint Clinical Assessment (JCA) increases the need for robust, standardized comparative evidence that withstands scrutiny across Member States. Emerging US pricing policies threaten to link some US prices to lower international benchmarks. Together with revisions to EU General Pharmaceutical Legislation, these shifts create unprecedented pressure on manufacturers to generate evidence that delivers both EU wide clinical value and price corridors aligned to the evolving US landscape.
Overview: This panel explores how EU and US policy intersection is reshaping evidence strategy, pricing, and development decision making, through the distinct perspectives of industry, EU HTA, and US policy.
Aodan Tynan provides an industry view on how JCA, compounded by US MFN pricing, is already altering clinical development priorities: While development programs should be redesigned early to accommodate EU wide comparators, endpoints and populations, focus is shifting to supporting pricing strategies compatible with the evolving US landscape.
Niklas Hedberg argues, as an EU HTA stakeholder, that JCA’s emphasis on robust, patient relevant evidence applicable across Member States is both appropriate and overdue. JCA represents a step forward in improving consistency and equity in European decision making, and its scientific ambition should not be compromised by short term pricing dynamics outside the EU.
Duane Schulthess offers a complementary US perspective, highlighting that MFN style reforms are grounded in principles of greater US EU equity in funding pharmaceutical innovation. While disruptive, this objective is viewed as legitimate and necessary but may have implications for EU access.
Each participant presents their perspective (30 mins), followed by moderated discussion and audience Q&A (~20 mins) to highlight consensus and disagreements on how goals can be reconciled without undermining evidence, innovation incentives, or patient access – a topic relevant to industry, HTA and policy stakeholders.
Moderator
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Oliver Staples, PhD
Basel, Switzerland
Speakers
The Historical Linear Life Science Model is Dead and Requires Rethinking to Adapt to a Rapidly Changing Ecosystem
Session Type: Issue Panel
Topics: Organizational Practices, Health Technology Assessment
Level: Intermediate
Issue:
The life sciences ecosystem is often described as a linear pathway from discovery through clinical development, regulatory approval, health technology assessment (HTA), adoption, and patient care. This model has shaped the system, but critics argue it no longer reflects modern healthcare innovation.
Advances in personalised medicine, RWE, AI, and learning health systems have exposed the limitations of a linear framework. Feedback loops between patients, health systems, policymakers, regulators, industry and HTA bodies remain fragmented, leading to evidence gaps, misaligned incentives, inefficiencies, and delays in translating innovation into improved outcomes.
This panel will debate whether the traditional ecosystem should be replaced by a dynamic “infinite-loop” model characterised by continuous feedback, bidirectional knowledge flows, and proactive demand signalling.
A central question is whether HTA should evolve from a downstream assessment process into a strategic integrative mechanism linking policy, innovation, adoption, and population health outcomes.
Overview:
This 60-minute issue panel will bring together representatives from HTA, policy, industry, academia, healthcare delivery, and patient communities to debate the future architecture of the life sciences ecosystem.
The moderator will provide a five-minute introduction, followed by brief opening perspectives from each panellist.
A structured discussion with the panel and audience will explore the issue from a 360-degree system perspective, identify areas of consensus and divergence, and consider key design elements of a future model, including roles, feedback mechanisms, and system components.
Audience participation will be integrated throughout the discussion, and the session will conclude with a summary and next steps.
The panel will be of interest to HTA professionals, policymakers, payers, regulators, industry, clinicians, and researchers. By presenting multi-stakeholder perspectives, the panel will challenge assumptions and stimulate discussion on how HTA can contribute to a more responsive, equitable, and sustainable ecosystem.
Moderator
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Carole Longson, PhD
University of Manchester, Manchester, United Kingdom
Carole Longson has over 30 years of executive and board experience in life sciences and is an internationally respected leader in Health Technology Assessment and Market Access. After a career in drug discovery at GSK, she spent 18 years at NICE pioneering its Technology Appraisal and Health Tech Evaluation programs, later serving as Chief Scientific Officer at the Association of the British Pharmaceutical Industry. A former HTAi President and EUnetHTA Executive Committee member, she has advised the European Commission and WHO. She is currently Vice Chair of Medicines Discovery Catapult UK and senior counsel in HTA and market access.
Speakers
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Eric Low
Eric Low Consulting, Haddington, United Kingdom
Eric has worked in medical research, health technology appraisal, market access, health policy and patient organisations for over 30 years.
He established Myeloma UK in 1996, developing it from an idea to a successful and respected organisation he led as Chief Executive until 2017. During this time, he designed and implemented a disease blueprint and bench-to-bedside strategic model to accelerate and prioritise the discovery, development of and access to effective new treatments, best practices, as well as information and support for patients and their families
In 2017, Eric established a small specialist consultancy specialising primarily in strategic HTA and market access, life sciences and healthcare policy, and patient and patient group engagement.
Eric is committed and focused on improving patient outcomes. He has a strong track record of delivery and success in building multi-stakeholder research collaborations, patient coalitions, market access strategies, changing policy, and delivering innovative solutions to complex issues, challenges and barriers in progress.
Eric also holds several Board, honorary, advisory and voluntary positions. He also advises numerous medical and health-focused charities on a pro bono basis. He was awarded an OBE for services to charity in the Queen’s Birthday Honours 2012.
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Jane Adam
United Kingdom
Who Decides Which Covariates Matter? Aligning Regulatory and HTA Perspectives in Indirect Treatment Comparisons
Session Type: Issue Panel
Topics: Health Technology Assessment, Methodological & Statistical Research, Health Policy & Regulatory
Track: Access and Pricing
Level: Intermediate
ISSUE: Indirect treatment comparisons (ITCs) are essential for health technology assessment (HTA) when head-to-head evidence is unavailable. Advances in methods, including population-adjusted indirect comparison, multilevel network meta-regression, and real-world evidence, have increased the sophistication of comparative effectiveness analyses. However, a persistent challenge remains: which covariates should be included in ITC adjustment models? Covariate misspecification can affect the feasibility, precision and validity of ITCs.
HTA guidance, including the Joint Clinical Assessment (JCA) Guidelines and NICE Technical Support Documents, emphasise adjustment for treatment effect modifiers and prognostic factors, but give limited direction on identifying, justifying and validating them. In practice, covariate selection varies across submissions, raising concerns about transparency, reproducibility and bias.
Regulatory agencies such as the European Medicines Agency (EMA) routinely assess clinically relevant covariates in trials and for marketing authorisation. Whether and how regulatory evidence should inform HTA covariate identification remains an important question, especially as stakeholders seek greater alignment between regulatory and HTA evidence requirements.
OVERVIEW: representatives from HTA, regulatory, academic and industry will debate key questions on covariate identification in ITCs. The moderator will introduce current challenges in covariate selection across ITC submissions. Panellist 1 will discuss HTA expectations for robust covariate adjustment. Panellist 2 will examine how regulators consider clinically meaningful treatment effect modifiers and prognostic factors during evidence evaluation. Panellist 3 will present a structured framework using publicly available regulatory documentation to support transparent covariate selection in ITCs and evaluate it through a previous NICE case study. 10-15 minutes for each panellist followed by 20 minutes for panel discussion and Q&A to determine next steps towards more transparent and decision-relevant covariate identification practices in ITC.
Moderator
Speakers
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Kate Ren
University of Sheffield; ConnectHEOR Limited, Sheffield, United Kingdom
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Anja E Schiel, PhD
Norwegian Medicines Agency, Oslo, Norway
Anja Schiel has studied Biology at the Johannes Gutenberg-University, Mainz, Germany. She received her PhD from the Free University in Amsterdam in 2006 and worked several years as Post-Doc on a range of subjects focusing on oncology, immunology and molecular biology, first at the University of Leiden and later at the University of Oslo, before starting at the Nor-wegian Medical Products Agency (NOMA) in 2012. At NOMA she is working as Special Adviser/Statistician/Methodologist both on regulatory and HTA projects. She has been Chair of EMA’s Biostatistics Working Party (2017 – 2019) and dur-ing 2019 – 2022 she was Chair of EMA’s Scientific Advice Working Party (SAWP). She contin-ues currently as alternate member at the SAWP and is member of the recently established Methodology Working Party (MWP) at EMA. She has been involved in EUnetHTA JA3 and its successor, EUnetHTA 21, with particular focus on parallel EMA-HTA scientific advice (joined scientific consultations). As one of the vice Chairs of the JSC Committee for Scientific Consistency and Quality (JSC CSCQ) she was in-volved in the preparation of the implementation of developer support under the Regulation on Health Technology Assessment (HTAR, to apply from January 12th 2025). Until January 2025 she was a member of the HTA coordination groups (HTACG) Joined Scientific consultation sub-group (JSC SG) and the Methodological and Procedural sub-group (MPG SG ).
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Dominik Karres
European Medicines Agency, Netherlands
10:30 - 13:30
Poster Session 1
Session Type: Research Posters
Poster Tours 11:45–12:30 | Presenters will be with their posters from 12:30–13:30
11:15 - 13:00
Lunch Service (Exhibit Hall)
Session Type: General Meeting
As you enjoy your lunch in the Poster and Exhibit Hall, seize the opportunity to engage in meaningful conversations with fellow attendees. Take this time to exchange ideas, forge new partnerships, or simply enjoy casual conversations.
11:30 - 12:15
HEOR Impact Cases Poster Tour
Session Type: HEOR Impact Cases
This tour will take place during Poster Session 1, Posters will be hung from 10:30-13:30.
Determinants of Unregistered Medicines Importation in Saudi Arabia: A National Regulatory Analysis
Problem Statement: Unregistered medicines are routinely imported into Saudi Arabia through regulated pathways to address drug shortages and unmet clinical needs. However, policymakers lacked empirical evidence on what drives the volume of these imports whether shortages alone explain demand, or whether broader therapeutic and market factors play an equally important role. Without this understanding, regulatory responses remained reactive, limiting the system's ability to anticipate supply stress and prioritize registration efforts strategically.
Description: This study used national regulatory data from Saudi Arabia's import clearance system (Faseh) to analyze 19,004 unregistered medicine import entries (2024 - 2025), representing 350.1 million units. Multivariable negative binomial regression was used to identify determinants of import volume per entry. Key predictors included Essential Drug List (EDL) status, shortage history, availability of registered therapeutic alternatives, route of administration, company type, and therapeutic class. Two pre-specified interaction terms assessed whether shortage effects were modified by alternative availability and EDL status.
Lessons Learned: Unregistered medicine importation is not shortage-driven alone it reflects a structured pattern shaped by therapeutic priority, market structure, and supply disruption history. EDL-listed medicines were associated with more than twice the import volume of non-EDL products, demonstrating that clinical priority independently drives importation demand regardless of shortage status. Medicines with registered therapeutic alternatives paradoxically showed higher import volumes, revealing a critical gap between nominal registry presence and functional market availability. The significant interaction between shortage status and alternative availability indicated that even nominally available alternatives provide partial buffering during shortages attenuating but not eliminating the surge in unregistered importation. This distinction has direct policy relevance: expanding the medicine registry is insufficient without ensuring that registered alternatives are actively stocked, distributed, and clinically accessible. Importation analytics can serve as a measurable indicator of pharmaceutical system stress, supporting proactive regulatory planning rather than reactive case-by-case responses.
Stakeholder Perspective: This evidence was generated from a government regulatory perspective, specifically within the Saudi Food and Drug Authority (SFDA). Findings are directly relevant to national regulators and health system planners seeking to strengthen supply resilience, prioritize market authorization, and reduce dependency on unregistered importation pathways. The results support transitioning from reactive importation management toward anticipatory governance using structured importation data to identify registration gaps, guide market entry facilitation programs, and improve pharmaceutical supply resilience across both routine and crisis conditions.
Implementing Value-Based Care in Maxillofacial Procedures: A Pilot Study on Protocol-Driven Decision Making, Economic Outcomes, and Patient Satisfaction in a Brazilian Payer System
Problem Statement: The process of selecting high-cost surgical materials (specifically for maxillofacial, BMF, procedures) within the healthcare system is characterized by a significant asymmetry of information and objectives between primary stakeholders. Healthcare providers prioritize technical autonomy and brand preference, while health insurance operators (payers) focus on economic sustainability and cost containment. This divergence often leads to inefficient resource allocation, increased costs, and conflicts, highlighting the need for a value-based approach.
Description: This paper describes the implementation of a pilot project involving organizational and care-delivery changes aimed at restructuring the flow of requests for BMF procedures within a mid-sized health insurance operator. The project, conducted in Bahia, Brazil (August-December 2024), utilized patient journey mapping as a central framework to redesign the care pathway, adopting a model based on validated clinical protocols and enhanced clinical governance. Key interventions focused on efficiency, safety, and humanization of care: 1. Joint elaboration and implementation of specific clinical protocols for BMF procedures; 2. Restructuring the accreditation process, focusing on credentialing a network of referenced and qualified providers; 3. Implementation of an automated system to block requests from non-qualified providers, followed by the assisted redirection of patients to the qualified network; 4. Creation of a new, humanized welcoming service (patient navigation) to guide beneficiaries.
Lessons Learned: The results of the pilot study were expressive, demonstrating both economic gains and improvements in care quality. Of the 66 requests analyzed under the new governance model, 71% of the procedures initially demanded were deemed unnecessary based on protocol adherence, generating an estimated saving of R$ 14.8 million for the payer. Furthermore, litigation was significantly reduced, with the number of related lawsuits/injunctions dropping from 28 to just 1. Beneficiary satisfaction (measured by Net Promoter Score - NPS) reached an average of 4.2 out of 5, indicating high approval of the new standardized flow.The findings suggest that transitioning from an autonomy-based model to a protocol-driven, value-based healthcare model is highly effective in optimizing resource allocation in supplementary health. Implementing robust clinical governance mechanisms can drastically reduce unnecessary procedures and litigation while maintaining high levels of patient satisfaction. This model provides a blueprint for other payers seeking to balance economic efficiency with evidence-based quality care.
Stakeholder Perspective: Payer (Health Insurance Operator)
Scan-2030: building a decision-ready health technology assessment ecosystem in hong kong for sustainable access to innovative medicines
Problem Statement: Hong Kong’s publicly funded Hospital Authority (HA) must decide which high-cost innovative medicines to list, subsidise or negotiate prices for while preserving equity and sustainability. Its Cost Assessment Panel negotiates with manufacturers, assesses cost-effectiveness and budget impact, and commissions economic evaluations. These decisions require reusable local Health Economics and Outcomes Research (HEOR) evidence linking medicine pipelines, disease burden, unmet need, comparative effectiveness, affordability and value.
Description: Horizon Scanning of Chronic Disease Burden and Care Needs to 2030 (SCAN-2030), funded by the University Grants Committee Research Impact Fund, built a decision-ready health technology assessment (HTA) and HEOR platform for Hong Kong’s learning healthcare system. Its Innovative Medicine Database tracks more than 50 pipeline medicines in depression, inflammatory bowel disease (IBD) and prostate cancer. Territory-wide HA electronic health records generated real-world data (RWD)-based burden forecasts, comparative effectiveness analyses and decision-analytic models for formulary, safety-net, cost-assessment and price-negotiation questions. Depression modelling projected 55,849-57,896 prevalent cases to 2032, with all-cause care costs exceeding US$322.3 million; treatment-resistant or comorbid cases represented fewer than 20% of patients but 31%-54% of costs. Economic modelling of a novel antidepressant identified price negotiation, delivery redesign and dosing optimisation as value levers. IBD forecasts projected age-standardised prevalence reaching 105.88 per 100,000 by 2032. Prostate cancer analyses showed comparable survival between novel hormonal agents and chemotherapy, with lower annual health resource use among novel hormonal agent users. A biosimilar model in 25,099 rheumatoid arthritis patients found biosimilar tumour necrosis factor inhibitors generated higher quality-adjusted life-years and lower lifetime costs than conventional therapy. SCAN-2030 translated HEOR outputs into policy briefs, dashboards, HA-facing briefings, international HTA exchanges and HEOR101 lectures. These outputs informed HA Cost Assessment Panel deliberations and formulary discussions by structuring evidence requirements, quantifying budget consequences, identifying medicines requiring formal economic evaluation and strengthening negotiation preparedness.
Lessons Learned: SCAN-2030 demonstrates HEOR-in-action by moving local evidence generation from one-off studies to reusable decision support for HA. Its workflow identifies technologies, quantifies burden and unmet need, compares effectiveness, models value and affordability, communicates uncertainty and trains users. This approach converted RWD and modelling into practical inputs for prioritisation, safety-net planning, commissioned evaluations and sustainable access decisions, while strengthening local HTA and HEOR capacity.
Stakeholder Perspective: From a government-affiliated payer and HTA system-building perspective, SCAN-2030 supported HA cost assessment, formulary readiness, safety-net planning and sustainable access. Clinicians, researchers and patient-facing stakeholders used the HEOR outputs to interpret unmet need, affordability and value.
From Evidence to Elimination: A Decade of HEOR informing Taiwan's National Hepatitis C DAA Reimbursement Policy
Problem Statement: Taiwan faced deep uncertainty in 2016 when committing to national DAA reimbursement for hepatitis C, with budget projections reaching NTD 600-800 billion at prevailing prices. The MOHW established a dedicated National Hepatitis C Program Office to operationalize the Minister’s 2025 elimination mandate, translating political commitment into evidence-based plans, implementation strategies, and outcome monitoring through sustained HEOR capacity across the full policy cycle.
Description: HEOR informed every phase of Taiwan's elimination program. At the planning stage, epidemiological modeling estimated 400,000 chronic HCV patients and projected treatment need; a natural history Markov model compared DAA, interferon, and no-treatment scenarios, projecting that reaching 250,000 treated patients by 2025 would reduce HCC cases by 24,000 and liver-related deaths by 54,000 by 2040. Budget impact analysis estimated NTD 45.7 billion in treatment and testing costs, offset by NTD 37.6 billion in avoided complication costs through 2040. At implementation, a national patient registry for all 173,747 reimbursed patients captured fibrosis stage, age, and SVR12 outcomes, enabling pharmaceutical risk-sharing agreements in 2017-2018. A real-time elimination monitoring network (TWNHCP-MIN) integrated NHI claims, screening, and seven subpopulation databases to generate monthly care cascade reports, identifying gaps and guiding adaptive policy adjustments. Retrospectively, a Markov-based ROI analysis valued 614,980 discounted QALYs at NTD 672 billion against NTD 28.7 billion in NHI spending, yielding an ROI of 22.4. By June 2025, Taiwan had achieved WHO Path to Elimination Gold Tier programmatic targets, with 90.2% of chronic HCV patients diagnosed and 92.6% of those diagnosed treated.
Lessons Learned: Four lessons emerged. First, front-loading HEOR investment at the policy design stage, through disease modeling and budget impact analysis, enabled credible target-setting and multi-year budget commitment under uncertainty. Second, registry-based risk-sharing reduced financial exposure while generating real-world effectiveness data. Third, integrated real-time monitoring with subpopulation disaggregation enabled adaptive management rather than static implementation. Fourth, retrospective ROI analysis reframes treatment expenditure as public investment, strengthening the evidence base for sustained universal access.
Stakeholder Perspective: Government (MOHW and NHIA, Taiwan)
The Impact of HTA on Vitamin D Testing in Switzerland
Problem Statement: Testing for vitamin D deficiency had been observed to increase in Switzerland, despite concerns regarding the benefits of vitamin D testing prior to vitamin D supplementation, thereby contributing to increased costs for the Swiss mandatory health insurance system.
Description: The Swiss Federal Office of Public Heath (FOPH) initiated an HTA to evaluate the available clinical and economic evidence on vitamin D testing. The HTA report published in 2020 found a lack of available evidence for vitamin D testing for at-risk groups. In 2021, Choosing Wisely Switzerland independently published their recommendation against routine vitamin D testing for patients without risk factors. In 2022, the FOPH restricted coverage of vitamin D tests under compulsory health insurance, limiting reimbursement to specific conditions. An academic research group subsequently conducted an independent retrospective analysis of health insurance claims data to assess the effects of the measures taken since publication of the HTA report. This analysis could demonstrate that the Choosing Wisely recommendation reduced the number of tests prescribed per physician by around 6% within 12 months following the intervention. Coverage restriction reduced the monthly number of tests by around 58% within 6 months.
Lessons Learned: HTA is a powerful tool to induce change in behaviours regarding potentially low-value care.
Stakeholder Perspective: The HTA (payer's perspective) was commissioned by the Swiss Federal Office of Public Heath (FOPH).
Regulatory Assessment of Emergency and Critical Care Services in Dubai: Using Evidence to Drive System-Level Reform
Problem Statement: Problem statement Emergency and critical care services are considered among the main health services in any health system and continue to have high demand in the region. system gaps have been identified by stakholders within patient flow and resource alignment leading to delay in care. Dubai Health Authority identified that despite strong individual hospital performance and patient accessibility, the broader emergency system required integrated governance, standardized triage protocols, tiered facility services, and coordinated hospital pathways — creating preventable treatment delays and uneven patient outcomes across trauma and emergency cases.
Description: Description Dubai Health Authority conducted a multi-stakeholder, mixed-methods assessment over seven months. The project encompassed: (1) on-site assessments of 30 acute care hospitals using standardized assessment criteria based on emergency, stroke and trauma standards aligned with international standards; (2) an EMS system assessment covering dispatch, prehospital protocols, and interfacility transfer operations; (3) international benchmarking against other local and international health systems; (4) patient pathway, referral, and transfer mapping for trauma and emergency cases; (5) development of evidence-based key performance indicators (KPIs). The process included stakeholder engagement and validation through meetings, and workshops. Assessment and data collection were done through site visits, document review, and interviews. Benchmarking revealed that although the local system has strong collaboration and alignment between ambulance services and healthcare providers, there was a need to strengthen the implementation of tiered care, bypass protocols, and centralized registries.
Lessons Learned: Lessons learned Lessons include: (1) strengthening facility-level and regional system-level quality indicators, a tiered designation framework for emergency service providers (adapted from American College of Surgeons guidelines) was recommended (2) adopting clear bypass protocols to reduce secondary transfers — evidence-based bypass protocols were recommended with stakeholder consensus; (3) establishment of unified trauma registries, a real-time bed dashboard, and KPI dashboards; (4) clear emergency policy (5) stakeholder validation workshops were critical for prioritization and buy-in, ensuring recommendations were actionable. The process demonstrated that structured, multi-method evidence generation can bridge the gap between individual hospital practices and system-level performance transformation.
Stakeholder Perspective: Stakeholder perspective This case is presented from the perspective of a government health regulatory authority, Dubai Health Authority is the regulatory licensing entity that also establishes health policies and standards based on local regulations and international best practices. Recommended decisions include: facility tiering system, emergency policy including EMS bypass protocol, regional registry, and KPI and monitoring tools adoption.
Oncology Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 1, Posters will be hung from 10:30-13:30.
Posters featured in this tour:
PT1: HOW SHOULD PICOS EVOLVE WHEN TREATMENT SEQUENCING CHANGES? A LIVING EVIDENCE CASE STUDY IN TRIPLE-CLASS EXPOSED MULTIPLE MYELOMA (TCE-RRMM)
PT2: REAL-WORLD EPIDEMIOLOGY, TREATMENT PATTERNS, AND CLINICAL OUTCOMES OF METASTATIC COLORECTAL CANCER IN TAIWAN: A NATIONWIDE POPULATION-BASED STUDY, 2015-2021
PT3: FROM BARRIERS TO ENABLERS: IDERHA POLICY RECOMMENDATIONS TO ENABLE HEALTH DATA SHARING FOR SECONDARY USE IN THE EUROPEAN HEALTH DATA SPACE
PT4: ESMO-MCBS: DO CURRENT CRITERIA UNDERVALUE CLINICAL BENEFIT IN POOR-PROGNOSIS POPULATIONS
PT5: A GENERAL FRAMEWORK FOR IPD META-ANALYSIS OF COMPETING RISKS DATA WITH FLEXIBLE PARAMETRIC SURVIVAL MODELS: ESTIMATION, PREDICTION AND SOFTWARE
PT6: SAME EVIDENCE, DIFFERENT DECISION? A COMPARATIVE REVIEW OF ONCOLOGY HTA RECOMMENDATIONS ACROSS NICE AND CDA-AMC
Moderator
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Abeer A Al Rabayah, MBA, MSc, DrPH
King Hussein Cancer Center, Amman, Jordan
Dr. Abeer Ahmad Al Rabayah leads the Center for Drug Policy and Technology Assessment (CDPTA) at the King Hussein Cancer Center (KHCC) in Amman, Jordan. In addition to her role at KHCC, Dr. Abeer holds a research associate position at the Institute of Public Health, Medical Decision Making, and Health Technology Assessment at UMIT TIROL – University for Health Sciences and Technology, Hall i.T., Austria.
As the director of the Hospital-Based Health Technology Assessment (HB-HTA) program and the founder of the Health Technology Assessment (HTA) fellowship program at KHCC, In 2024, the KHCC HB-HTA program received the King Hussein Cancer Research Award for Academic Program Excellence. Dr. Abeer has significantly contributed to the field. She has authored over 20 publications in HTA and Health Economics research, covering areas such as decision analytical modeling, HTA processes and methods, valuation of health outcomes, and Health-Related Quality of Life research.
Dr. Abeer is influential at the national, regional, and international levels in advancing the implementation and development of HTA. She is an active member of numerous HTA steering committees. She is the chair of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) HTA round table for the Middle East and Africa (2024-2026). Currently, Dr. Abeer is appointed by the World Health Organization to serve as a member of the Technical Advisory Group on Pricing Policies for Medicines (TAG-PPM), providing evidence-based technical advice to improve affordable access to essential and priority medicines.
Dr. Abeer holds a BSc in Pharmacy, a Master of Business Administration (MBA) from Jordan University, and a Master of Science (MSc) in International Health Technology Assessment from the University of Sheffield, United Kingdom. She is a PMP-certified project manager and a final-year Ph.D. candidate at UMIT TIROL – University for Health Sciences and Technology, Hall i.T., Austria.
12:15 - 13:15
ISPOR Strategic Dialogues: Integrated Evidence Generation Across the Product Lifecycle
Session Type: Forums
Topics: Organizational Practices, Health Policy & Regulatory
Level: Intermediate
Even when regulators, HTA bodies, and payers seek answers to many of the same scientific questions, evidence generation remains largely sequential, siloed, and awfully inefficient. This fragmentation creates avoidable translation loss between decision systems, triggers late evidence requests, and often requires sponsors to generate multiple versions of "decision-grade" evidence for different stakeholders. Drawing on insights from the ISPOR Strategic Dialogues pilot in the EU/EEA, this panel will examine whether Europe has reached an inflection point in evidence generation. With implementation of the EU HTA Regulation, the introduction of Joint Scientific Consultation (JSC), and increasing collaboration among regulators, HTA bodies, and innovators, is Europe creating the conditions for a more integrated approach to evidence planning across the product lifecycle? Panelists representing regulatory agencies, HTA, industry, and clinical research will discuss whether JSC can serve as a building block for integrated evidence generation and how early scientific dialogue can evolve into coordinated evidence strategies that reduce duplication while respecting national decision-making. The discussion will explore what integrated evidence generation means for innovator companies as they rethink clinical development programs, real-world evidence strategies, and evidence packages to support multiple decision makers. Finally, the panel will consider whether Europe is uniquely positioned to become the global leader in integrated evidence generation, what barriers remain, and what practical steps are needed to translate alignment into more efficient evidence development, greater evidence reuse, and ultimately faster patient access to innovation.
Moderator
Speakers
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Michael Berntgen
Amsterdam, Netherlands
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Carlos Martin Saborido
Roche, Switzerland
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Beate Wieseler, PhD
IQWiG, Köln, Germany
Dr. Beate Wieseler is Head of the Department of Drug Assessment at the German Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, IQWiG), Germany’s Health Technology Assessment agency. At IQWiG she is responsible for the scientific assessment of pharmaceuticals, the development of assessment methods as well as in the Institute’s collaboration with German and international external parties. Currently, Beate Wieseler also is the Chair of the Methodological and Procedural Guidance Subgroup of the European HTA Coordination Group.
Prior to joining IQWiG in 2005, Beate Wieseler worked in clinical research and regulatory affairs for about 10 years. Beate Wieseler holds a Diploma in Biology from the University of Bonn and a Dr. rer. nat. from the University of Freiburg, Germany.
The Non-3G (GENEROUS, GLOBE, GUARD) Pricing Paradox: Navigating the "MFN Trap" Opportunities and Risk in Latin America, the Middle East, and Asia
Session Type: Forums
Topics: Health Policy & Regulatory, Health Technology Assessment, Economic Evaluation
Level: Intermediate
MFN is reshaping how companies prioritize launch markets, with 19 markets currently included across the GENEROUS, GLOBE, and GUARD programs. While these “non-3G” markets may represent opportunities for earlier launches following the US, their health systems continue to face significant budget constraints driven by macroeconomic pressures, including inflation, currency devaluation, and geopolitical instability. This cross-regional forum will examine the opportunities and risks created by this evolving pricing environment across Latin America, the Middle East, and Asia, including the potential implications for access in neighboring lower-income markets.
OVERVIEW
Presented by the ISPOR Latin America Consortium, ISPOR Asia Pacific Consortium, and ISPOR Arab Network, this forum evaluates three regional realities covering four markets:
• LATAM (G20 UMICs, near-to-cross to HIC): Argentina and Mexico.
• GCC (HIC, part of the GCC bloc's joint negotiations and reference pricing): UAE.
• AP (LMIC yet the world's 3rd largest pharma market): India.
Panelists representing each network's leadership will debate three cross-regional pressures:
1. Macroeconomic Headwinds: The conflict between rigid GNI-per-capita graduation metrics and the fiscal reality of health systems enduring hyperinflation, war, or currency devaluation.
2. Pooled Procurement: How high-income pricing demands clash with consolidated buying blocs, such as joint GCC negotiations and reference pricing or PAHO Regional Revolving Funds in Latin America.
3. Neighboring Country Impact: The operational risk to lower-priced neighboring countries that border a non-3G market, where high commercial prices set in the bordering UMIC or HIC destabilize affordability and access in the adjacent, lower-price country.
Moderator
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Manuel Antonio Espinoza, MSc, PhD, MD
Hong Kong, Hong Kong
Speakers
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Diego Guarin, MPH, MSc, MD
ISPOR Latin America Consortium Chair, Rahway, NJ, United States
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Manuel Antonio Espinoza, MSc, PhD, MD
Hong Kong, Hong Kong
How Can HTA and Regulatory Guidelines and Reviews Strengthen the Case for More Patient Centered Evidence Creation?
Session Type: Workshop
Topics: Patient-Centered Research, Health Technology Assessment, Clinical Outcomes
Track: Patient-Centered Evidence
Level: Intermediate
Purpose:
The purpose of the workshop is to have an interactive session with input from participants on expanding the use of patient evidence to support reimbursement, and explore whether the use of regulatory and HTA guidelines is working well or should be further strengthened.
The topic will focus on whether globally patient evidence has a higher valuation due to more inclusion in regulatory and health technology guidelines, and how sponsors can include it to strengthen their case for a better value and creation of health.
Participants are expected to leave the workshop empowered and better informed.
Description:
Katja Rudell has analysed the 2025 EMA JCA guideline and workshop slides to evaluate the impact of patient centred evidence which has always been considered nice to have in EMA 2005 regulatory guidelines on QoL. She will outline the intent of the two concepts -conceptual model and measurement model that are introduced to reduce burden and increase chances for positive outcome on trials and patient evidence. Dan Ollendorf will describe ICER’s patient engagement process in detail and highlight recent work to pilot patient-informed economic model development as well as the efforts of ICER’s overarching Patient Council. Elisabeth Oehrlein will outline frameworks to plan the inclusion of patient centered evidence that sponsors could use to plan ahead. She will draw on her experience as a patient advocate.
The session is then planned to move to open-ended discussion and improvement suggestions. The interactive part will include - 2 surveys one at the start and one at the end of the session as well as knowledge checks in between the presenters. We hope to get questions about improvements to guidelines live and prepare for active participation.
Moderator
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Katja Rudell, BSc, MSc, PhD, Other
Kielo Research, York, United Kingdom
Speakers
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Dan Ollendorf, MPH, PhD
Institute for Clinical and Economic Review (ICER), Boston, MA, United States
Dan Ollendorf, PhD is Chief Scientific Officer and Director of Health Technology Assessment (HTA) Methods and Engagement at the Institute for Clinical and Economic Review (ICER), with responsibility for managing the organization’s internal health economics capacity and program of external collaboration with academic researchers and institutions, as well as collaboration with international HTA bodies and other stakeholders on key process and methods topics. From 2007-2018, Dr Ollendorf was chief scientific officer for ICER, where he oversaw the broader HTA process and managed relationships with multiple stakeholders and research collaborators.
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Elisabeth Oehrlein, MS, PhD
Applied Patient Experience, LLC, Washington, DC, United States
My Friend Claude: An ISPOR–SMDM Frontiers in Modeling Session on AI Skills for Model Conceptualization and Transportability
Session Type: Forums
Topics: Methodological & Statistical Research
Level: Advanced
ISPOR and the Society for Medical Decision Making (SMDM) jointly present a scientific methods session examining how early adopters are applying custom Claude Skills to health economic and decision modeling. Bringing together leaders in decision modeling, the session features Beth Devine, Beate Jahn, Jagpreet Chhatwal, Ian Cromwell, and Laura Pizzi. The session will focus on two custom Claude Skills developed to support decision modeling workflows. The Conceptualization Skill synthesizes published evidence, clinical practice guidelines, disease pathways, and stakeholder perspectives to identify decision-relevant health states, causal relationships, structural assumptions, alternative model structures, and evidence gaps. The skill produces a documented conceptual model with evidence traceability, providing a transparent foundation for expert review and model development. The Transportability Skill evaluates whether an existing model can be adapted to a new healthcare setting or patient population. The skill systematically compares epidemiology, treatment pathways, clinical practice, healthcare resource utilization, costs, utilities, and other contextual factors between source and target settings. It identifies model components requiring modification, distinguishes transferable from setting-specific parameters, documents supporting evidence, and generates a structured transportability assessment. Drawing on their experiences as early adopters, panelists will discuss practical lessons learned from integrating Claude Skills into modeling workflows, including the impact on efficiency, transparency, documentation, reproducibility, and validation. The discussion will also examine where Claude Skills add the greatest value, where expert judgment remains essential, and priorities for future methods research. Attendees will gain practical insight into how custom Claude Skills can support model conceptualization and transportability, along with an evidence-informed perspective on their appropriate role in advancing health economic and decision modeling.
Moderator
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Beth Devine, MBA, MSc, PharmD, PhD
University of Washington, Seattle, WA, United States
Speakers
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Beth Devine, MBA, MSc, PharmD, PhD
University of Washington, Seattle, WA, United States
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Jag Chhatwal, PhD
Harvard Medical School / Massachusetts General Hospital, Boston, MA, United States
Jag Chhatwal, PhD, is the director of the Institute for Technology Assessment at Massachusetts General Hospital and an associate professor at Harvard Medical School. He also serves as core faculty at the Center for Health Decision Science, Harvard T.H. Chan School of Public Health. Dr. Chhatwal has co-authored more than 125 original research articles and editorials in leading peer-reviewed journals. His research has informed health policy decisions at prominent organizations including the White House, the World Health Organization, and the CDC, and has been featured in major media outlets such as CNN, Forbes, National Public Radio, The New York Times, and The Wall Street Journal. Dr. Chhatwal serves as an associate editor of Value in Health and as guest editor for its special issue on artificial intelligence. He is also a member of the ISPOR Generative AI Working Group.
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Beate Jahn, PhD
UMIT – University for Health Sciences, Medical Informatics and Technology, Hall i.T., Austria
AI in HEOR: Advancing Evidence Generation for Joint Clinical Assessment
Session Type: Forums
Topics: Health Policy & Regulatory
Level: Introductory
Artificial intelligence (AI) is rapidly transforming how health economics and outcomes research (HEOR) is conducted across the pharmaceutical industry. As the Joint Clinical Assessment (JCA) takes place across Europe under the EU Health Technology Assessment (HTA) Regulation, the potential for AI use has attracted interest among many HEOR researchers. From accelerating literature reviews and predicting PICO scenarios to streamlining data extraction and evidence synthesis, AI has the potential to improve efficiency while reducing manual workload. As AI adoption continues to expand, organizations must also address key considerations regarding transparency, validation, reproducibility, data privacy, and regulatory acceptance. Understanding where AI can add value and where human expertise is needed is essential for HEOR professionals, especially for students and early-career professionals entering the field. This session invites students and young professionals to explore current and emerging applications of generative AI throughout the HEOR workflow, with a focus on evidence generation for Joint Clinical Assessments. Participants will hear perspectives from experienced professionals across academia, industry, and consulting on practical use cases, opportunities to improve efficiency, current limitations, governance considerations, and the evolving skills needed for the future HEOR workforce. The session will conclude with an interactive discussion, giving attendees the opportunity to engage directly with panelists, ask questions, and gain practical insights into how AI is reshaping evidence generation, market access, and career development in HEOR. This session is brought to you by the ISPOR Student Network.
Moderator
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Nga Weng Leong, BSc, MSc
University of Mississippi, Oxford, MS, United States
13:45 - 14:45
Are Patient Values Genuinely Shaping Value Assessment, or Do They Remain Acknowledged but Underutilized?
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment, Patient-Centered Research
Track: Patient-Centered Evidence
Level: Intermediate
ISSUE: Across global HTA systems, broader value elements—caregiver burden, disease severity, financial toxicity, and whole-health impacts on families—are increasingly recognized conceptually. Collaborative initiatives like BRAVER and APACE have strengthened this evidence base, and HTA bodies formally acknowledge the need for patient-centered value. Yet, the practical reality remains: these elements rarely influence final assessments or pricing recommendations in a consistent, meaningful way. This gap is both methodological and structural. As HTAs evolve toward lifecycle approaches and precision medicine, evidence complexity has grown, but the capacity of patients and organizations to engage throughout the evidence lifecycle has not kept pace, leaving patient values acknowledged but structurally underutilized.
OVERVIEW: The session is structured for maximum debate: Ramiro Gilardino will frame the operational friction points, followed by four sharp, 5-minute stakeholder perspectives:
Dan Ollendorf (ICER) will frame and reflect on ICER’s experience integrating patient perspectives, including the challenges of operationalising broader value elements transparently. Lotte Steuten (OHE) will draw on BRAVER and APACE to examine how broader value elements remain difficult to quantify despite growing recognition. A patient expert will bring the patient community perspective on patient experience data frameworks and the practical realities patients face when attempting to influence HTA and payer decision-making. In a moderated discussion, the panel will examine what methodological, structural, and technological shifts are required to close the enduring gap between recognising patient values and embedding them as practical infrastructure to drive decisions.
Moderator
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Ramiro E Gilardino, MSc, MD
Insights & Impact, Zurich, Switzerland
I lead the access and policy strategies that enable innovative medicines to reach patients across Europe, Latin America, and global markets. With 16+ years at the intersection of HTA, market access, and health policy, I have led access strategy in some of the most complex regulatory environments in the industry, including EU Joint Clinical Assessment, IRA, and evolving global pricing scrutiny, helping organizations anticipate and navigate change across launch and lifecycle.
I focus on oncology, rare disease, and specialty portfolios. I have supported 20+ global product launches and built market access capabilities from the ground up, translating complex policy environments into actionable pricing, reimbursement, and evidence strategies.
I bring a combination of clinical training, health economics expertise, and policy fluency, operating at the executive level across industry, consulting, and global institutions. My work includes shaping HTA frameworks and access environments through board roles at ISPOR, advisory engagement with the World Health Organization and World Bank, and 40+ publications in leading HEOR, HTA, and policy journals.
Speakers
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Dan Ollendorf, MPH, PhD
Institute for Clinical and Economic Review (ICER), Boston, MA, United States
Dan Ollendorf, PhD is Chief Scientific Officer and Director of Health Technology Assessment (HTA) Methods and Engagement at the Institute for Clinical and Economic Review (ICER), with responsibility for managing the organization’s internal health economics capacity and program of external collaboration with academic researchers and institutions, as well as collaboration with international HTA bodies and other stakeholders on key process and methods topics. From 2007-2018, Dr Ollendorf was chief scientific officer for ICER, where he oversaw the broader HTA process and managed relationships with multiple stakeholders and research collaborators.
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Chris Skedgel
United States
How Will the Most Favoured Nation (MFN) policy Influence Medicines Pricing and Access in Europe and How Should Policy Makers Respond?
Session Type: Workshop
Topics: Health Policy & Regulatory, Economic Evaluation, Health Technology Assessment
Track: Access and Pricing
Level: Intermediate
Purpose: The Most Favoured Nation (MFN) policy being pursued by the US administration has the potential to influence prices and access for new pharmaceuticals across high income countries. This workshop will identify how MFN is likely to affect access and pricing globally and what European policymakers should do in response.
Description: MFN drug pricing aims to anchor US drug prices to those paid in other high income (reference) countries, addressing longstanding concerns that US prices exceed those elsewhere. Commentators have raised concerns that the policy will lead manufacturers to increase prices, or delay or forgo launches in European markets to protect US revenues. In this workshop, we will show how economic models can predict the outcomes of MFN in Europe and inform healthcare policy development.
Professor Sullivan will moderate and introduce MFN policy and its motivation from a US perspective [5 minutes]. Iria Alvarez Novoa will provide a pharmaceutical industry perspective on how MFN is expected to change launch sequence decisions in European markets and how manufacturers are preparing [12 minutes]. Professor Sa will show that MFN may lead manufacturers to narrow covered patient populations in Europe—restricting access via coverage even under full insurance—while reducing global revenues unless reference countries adjust pricing rules [12 minutes]. Dr Woods will then show how different policy responses—including changes to cost effectiveness thresholds or other pricing approaches—are likely to influence pricing, innovation, and long term population health outcomes [12 minutes].
The workshop will use real-time polling to ask participants to consider how they would react to MFN as a manufacturer or health system actor and to consider the trade-offs between price, access and long-term innovation. This interactive workshop will be valuable for those involved in pricing and access decisions within industry, and those working on pharmaceutical policy across governments, academia, and health systems.
Moderator
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Sean D Sullivan, PhD
University of Washington, Seattle, WA, United States
Sean D. Sullivan, BScPharm, MSc, PhD, is Professor and Dean, School of Pharmacy. He holds a joint appointment as Professor of Health Services in the School of Public Health. He holds adjunct appointments in the School of Medicine, the Public Health Sciences Division at the Fred Hutchinson Cancer Research Center, and at the Kaiser Permanente Washington Health Research Institute. He completed training in pharmacy at Oregon State University in 1983, obtained a master’s degree at the University of Texas in 1986 and a PhD in health economics and policy at the University of California, Berkeley in 1992. Dr. Sullivan has authored more than 400 journal articles, book chapters, task force reports and organizational and governmental publications. In many of these writings, he has assessed the evidence and applications of medical technology in relation to coverage and reimbursement decisions. His research interests include technology assessment, medical decision-making, and economic evaluation of medical technology. He is past president of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and past chair of the Academy of Managed Care Pharmacy (AMCP) Executive Committee of the Format for Formulary Submissions – the United States evidence-based guidelines for formulary decision making. Dr. Sullivan served as a past member of the Medicare Evidence Development and Coverage Advisory Committee, a past member of the Regence Blue Shield and Premera Blue Cross P/T Committee, and the Chair of the Premera Blue Cross Value Assessment Committee. He is also on the editorial boards of Value in Health, PharmacoEconomics, Journal of Medical Economics, and the European Journal of Health Economics. He was awarded the 2014 Stephen G. Avey Lifetime Achievement Award from the Academy of Managed Care Pharmacy (AMCP) and the 2015 APhA Academy of Pharmaceutical Research Sciences (APRS) Research Achievement Award.
Speakers
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IRIA ALVAREZ NOVOA IGLESIAS
Spain
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Luis Sa
University of Porto, Portugal
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Beth Woods, BA, MSc
University of York, York, United Kingdom
The Future of Causal Inference: How Large (or Small) Role Will AI Play?
Session Type: Issue Panel
Topics: Methodological & Statistical Research, Real World Data & Information Systems, Health Policy & Regulatory
Track: AI
Level: Intermediate
Issue:
As real-world evidence takes an increasingly important role in regulatory and health technology assessment decision making, causal inference implemented via a target trial framework is becoming more widely adopted to improve the credibility and interpretability of non-randomized evidence. At the same time, rapid advances in artificial intelligence are reshaping how healthcare data are generated, curated, and analysed. These parallel developments raise a question regarding the future of evidence generation: will causal inference continue to rely primarily on explicitly designed epidemiologic frameworks, or will it also use AI-driven systems which increasingly infer causal relationships directly from large-scale healthcare data?
Overview:
This issue panel will debate whether current approaches to causal inference are scalable and sustainable in an era of rapidly expanding data complexity, and whether AI can meaningfully augment (and at one end of the spectrum, potentially replace) traditional causal inference paradigms. Panelists will also discuss the implications for transparency, reproducibility, and regulatory trust. The discussion will also explore whether future "decision-grade" RWE systems will require fundamentally new architectures integrating AI-enabled longitudinal data capture, automated endpoint derivation, and dynamic protocol emulation.
Moderator
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Radek Wasiak, PhD
Adigens Health Limited, London, United Kingdom
Speakers
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Miguel Hernan, PhD
Boston, MA, United States
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Gorana Capkun, PhD
Allschwil, Switzerland
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Michael Haft
Xplain Data, Zorneding, Germany
Dr. Michael Haft is Founder and CEO of Xplain Data. He holds a Ph.D. in Theoretical Physics and Neuroinformatics and has more than 20 years of experience developing advanced analytics technologies at Siemens, Accenture, and SAP, where he served as Chief Architect for Big Data Analytics. At Xplain Data, he pioneers Causal AI solutions that uncover cause-and-effect relationships from observational real-world data, enabling hypothesis-free causal discovery and supporting researchers and clinicians in understanding disease drivers, treatment effects, and evidence-based decision-making.
Health Utility Measurement: EQ-5D Innovations and Broader Perspectives
Session Type: Research Podiums
Health utility measures play a central role in health economics, outcomes research, and HTA. This session explores advances in EQ-5D measurement alongside broader challenges in preference-weighted outcomes research, including content validity, symptom-specific measurement, utility estimation, and analytical tools that support robust and reproducible evidence generation. Together, these studies highlight opportunities to improve the relevance, sensitivity, and application of patient-centered outcome measures in healthcare decision-making.
EQ5DSUITE: AN R PACKAGE AND WEB INTERFACE FOR DESCRIBING AND ANALYSING EQ-5D DATA
OBJECTIVES: Although guidance exists for the analysis of EQ-5D data, implementation in practice often relies on bespoke spreadsheets and scripts, limiting reproducibility and comparability across studies. We developed eq5dsuite, an open-source R package with a companion web-based interface that provides a unified framework for EQ-5D valuation and descriptive analyses. Its application is demonstrated using real-world patient outcomes data.
METHODS: eq5dsuite supports valuation of EQ-5D-3L, EQ-5D-5L, and EQ-5D-Y-3L responses using published country-specific value sets and established crosswalk methods. The package provides a structured set of functions for analysing EQ-5D profiles, EQ-5D values, and EQ-VAS data, implementing analyses recommended in published guidance in a consistent and reproducible manner. To illustrate its functionality, the package was applied to publicly available English NHS PROMs data to examine pre- and post-operative outcomes among hip replacement patients and to compare baseline health status between patients undergoing knee replacement and groin hernia repair. A companion Shiny application provides equivalent functionality through a graphical user interface.
RESULTS: Among hip replacement patients, problems before surgery were most frequently reported in the pain/discomfort, usual activities, and mobility dimensions. Mean EQ-5D values increased from 0.365 before surgery to 0.793 after surgery, while mean EQ-VAS scores increased from 65.5 to 77.0. In a separate baseline comparison, pre-operative knee replacement patients reported lower mean EQ-5D values than groin hernia patients (0.404 vs 0.788), consistent with greater impairment across most dimensions among knee replacement patients.
CONCLUSIONS: eq5dsuite provides a standardised framework for valuation and descriptive analysis of EQ-5D data within reproducible analytical workflows. Equivalent implementations in Stata and Excel allow the framework to be applied in settings where R is not routinely used, facilitating consistent and reproducible reporting of EQ-5D outcomes in health economics and outcomes research.
EXPLORING CONTENT VALIDITY OF GENERIC PREFERENCE-WEIGHTED MEASURES: A SCOPING REVIEW
OBJECTIVES: Content validity is a fundamental property of patient-reported outcome measures (PROMs). Although general guidance exists, no framework is tailored specifically to generic preference-weighted measures (PWMs), potentially leading to suboptimal study design and misleading conclusions about validity across populations. This scoping review aimed to identify and summarise qualitative methods used to assess the content validity of generic PWMs.
METHODS: The review followed Joanna Briggs Institute guidance. Searches of eight databases identified peer-reviewed studies reporting primary qualitative research on relevance, comprehensibility, and/or comprehensiveness of generic PWMs. Two reviewers independently screened studies and extracted data on study characteristics, participants, instruments, and methods. Study trustworthiness was evaluated using Lincoln and Guba’s framework (credibility, transferability, dependability, and confirmability).
RESULTS: Searches conducted in December 2025 and updated in April 2026 identified 46 eligible studies. Most were undertaken in a single country (42/46), commonly the UK (15/46). Nineteen PWMs were evaluated, most frequently the EQ-5D-5L (31 studies). Thirty-two studies were qualitative and 14 used mixed-methods. Evidence relating to relevance was reported in 37 studies, comprehensibility in 29, and comprehensiveness in 32. However, key instrument features were assessed less often, including completion instructions (6 studies), recall period (26), and response levels (30). Reporting was generally limited: 42 studies provided only illustrative quotations, three reported no qualitative data, and only one reported a complete dataset. Study trustworthiness varied across domains, with credibility scoring lowest (22%). Overall transparency was poor, with frequent omission of methodological details; only 14 studies reported using a reporting checklist.
CONCLUSIONS: Considerable variation exists in qualitative approaches to assessing the content validity of generic PWMs, with inconsistent alignment to good practice guidance. Important gaps remain in evaluating instrument design features, while limited reporting restricts assessment of study quality and trustworthiness. PWM-specific guidance is needed to improve the design, conduct, reporting, and interpretation of qualitative content validity research.
A REVIEW OF UTILITY VALUES IN NATIONAL INSTITUTE FOR HEALTH AND CARE EXCELLENCE (NICE) HEALTHTECH GUIDANCE
OBJECTIVES: The NICE HealthTech programme produces guidance on non-drug technologies, including medical devices, digital and diagnostic technologies. This study aimed to review the instruments used to derive utility values in economic models developed for HealthTech assessments. Utility values reflect the health-related quality of life (HRQoL) of a given health state and are used to calculate quality-adjusted life years, NICE’s preferred approach to expressing health effects.
METHODS: All HealthTech guidance published in 2025 that included an economic evaluation was reviewed. Data was extracted on the characteristics of the economic model, utility value sources, instruments used to obtain utility values, how those values were used, and whether quality of life was identified as a key issue or future research priority.
RESULTS: Seventeen HealthTech assessments were included. Thirteen assessments included modelling of health effects. In almost all of those (11/13 models), utility values were sourced from multiple published studies. For the remaining 2, all utility values were sourced from a single source and only one of these was a pivotal trial of the technology. The EQ-5D was the most used instrument to derive health state utilities (10/13 models), with one model using utilities derived with the CHU9D tool and the remaining 2 models only using disutilities with no reporting of the source. Where EQ-5D was used, the EQ-5D-3L and EQ-5D-5L were used in 7 and 3 cases, respectively. Where EQ-5D-5L was used, appropriate mapping using the Hernandez algorithm was reported in 2 cases, with no mapping reported in the remaining case. Committee members considered HRQoL a key issue in 6/17 assessments. However, in 10/17 assessments they recognised HRQoL as an area for future research.
CONCLUSIONS: Utility values used in NICE HealthTech assessments are sourced heterogeneously. The EQ-5D was the most commonly used instrument, consistent with the NICE reference case, with predominant use of the older EQ-5D-3L.
DO WE NEED A SKIN IRRITATION BOLT-ON FOR THE EQ-5D-5L? A SYSTEMATIC REVIEW OF THE MEASUREMENT PROPERTIES OF THE EQ-5D-5L IN CONDITIONS WITH ITCH
OBJECTIVES: Itch is a burdensome symptom across dermatological and non-dermatological conditions, impacting health-related quality of life (HRQoL). This systematic literature review aimed to identify and summarise published evidence relating to the EQ-5D-5L’s measurement properties in conditions where itch is a key symptom for adults.
METHODS: A systematic literature search was conducted in Embase and MEDLINE in February 2025 (PROSPERO No.: CRD420250650978). Included articles underwent quality assessment using an adapted Grading of Recommendations Assessment, Development and Evaluation (GRADE) for quantitative studies and the Critical Appraisal Skills Programme (CASP) checklist for qualitative studies.
RESULTS: Overall 73 articles were eligible, assessing 17 dermatological (e.g. psoriasis) and 9 non-dermatological conditions (e.g. chronic kidney disease, CKD). Qualitative studies (n=5) reported the EQ-5D-5L comprehensible and relevant. Itch was reported missing in one study and was the most frequent spontaneously reported symptom in another. Ceiling effect ranged between 15% (hidradentis supprativa) and 55% (urticaria). Some evidence of known-groups validity was identified, however only one article (CKD) defined groups by itch severity, showing worse EQ-5D-5L values with more severe itch. There was mixed evidence for convergent validity between the EQ-5D-5L and disease-specific HRQoL measures, correlations ranged from weak (-0.10, actinic keratosis) to strong (-0.79, pemphigus). Of the conditions included, psoriasis and CKD had the most evidence supporting the EQ-5D-5L psychometric properties, however for both conditions there was only limited evidence of content validity, evidence of a ceiling effect and no evidence of test-retest reliability.
CONCLUSIONS: This systematic review found mixed evidence of the EQ-5D-5L’s psychometric properties in conditions where itch is a key symptom, predominantly in dermatological conditions. Overall, the findings suggest potential limitations in the EQ-5D-5L’s ability to fully capture the burden of itch. A bolt-on measuring skin irritation (itch) is available; research should explore the impact this has on the EQ-5D-5L’s psychometrics in these conditions.
COAlescing the oncology COA strategy: A provocative look at why oncology COA strategies often fail - and how to fix them
Session Type: Issue Panel
Topics: Clinical Outcomes, Patient-Centered Research, Health Policy & Regulatory
Track: Patient-Centered Evidence
Level: Intermediate
Issue
Clinical Outcome Assessments (COAs) are central to patient-centered oncology trials and regulatory decision-making, yet many COA strategies fail to produce interpretable or label-enabling evidence. A key reason is that COAs are too often treated as downstream measurement tools rather than integral parts of trial design. This leads to fragmentation across endpoint selection, operational implementation, and statistical analysis, with decisions made in isolation from the research question, feasibility constraints, and analytic needs. The result is often missing, biased, or non-actionable data. These failures are not inevitable - they reflect avoidable design and implementation choices. Without a more integrated, end-to-end approach, oncology COA strategies will continue to underdeliver.
Overview
This panel will examine why oncology COA strategies fail, where they succeed, and how to improve them. It will begin by addressing a core weakness: poorly specified objectives and endpoints that undermine downstream decisions.
An industry perspective will explore the tension between scientific ambition and operational feasibility, with lessons from global oncology programs on cross-functional alignment and development planning.
An implementation perspective will show how burden, inconsistent training, and system-level issues can compromise data quality, while highlighting practical solutions.
An FDA representative will offer a regulatory perspective reviewing existing FDA guidance and recent labeling examples to show how rigorously defined and collected COA-based endpoints can support oncology product labeling.
The panel will open up for a 20-minute Q&A with the audience, welcoming attendees’ successes and failures to stress-test the strategies proposed. Together, the panel will move beyond diagnosis to challenge entrenched practices and offer a concrete path toward more rigorous, aligned, and decision-relevant COA strategies.
Moderator
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Konstantina Skaltsa, BSc, PhD
IQVIA, Barcelona, Spain
Konstantina provides scientific oversight for the Patient-Centered Solutions team, ensuring alignment with best practices while driving thought leadership in cutting-edge areas. A statistician by training with over 15 years of academic and industry experience, she is a subject-matter expert in psychometrics and statistical analysis of clinical outcome assessment (COA) data.
Her advisory work spans the full COA endpoint strategy lifecycle, including defining meaningful COA objectives, aligning study design and endpoints, leading estimand discussions, and proposing statistical methodologies aligned with selected estimands. She has supported FDA and EMA interactions for COA-based label claims and contributed to payer dossiers across Europe, Canada and Australia.
Konstantina has extensive experience across oncology, dermatology, rheumatology, and respiratory diseases. She has a strong interest in the implementation of the estimand framework and co-chairs the PSI Patient-Focused Drug Development (PFDD) Special Interest Group, promoting best practices in estimands and estimators for COA endpoints. Additional interests include the analysis and interpretation of tolerability data in oncology trials.
Her market access expertise includes utility estimation, mapping methods, survival analysis, treatment-switching adjustment techniques, and network meta-analysis. She has co-authored multiple peer-reviewed publications and is an active member of PSI, ISPOR, and ISOQoL.
Konstantina holds a PhD in Biostatistics from the University of Barcelona and a BSc in Applied Mathematics from the University of Crete.
Speakers
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Bryan Bennett, BSc, PhD
Jazz Pharma, Berwick-upon-Tweed, United Kingdom
Bryan Bennett, PhD, is the Head of Patient-Centered Outcomes and Senior Director at Jazz Pharmaceuticals, where he leads a dedicated team of Outcomes Scientists developing novel Clinical Outcome Assessment (COA) strategies across neuroscience and oncology. With over three decades of experience spanning clinical practice, academia, consultancy, and the pharmaceutical industry, Dr. Bennett is a passionate advocate for patient-focused drug development. He has consistently driven efforts to ensure the patient's voice is integrated into every stage of the therapeutic lifecycle. Dr. Bennett is an active member of the ISPOR community, recently serving on the ISPOR Europe Program Committee and the COA Special Interest Group Leadership team.
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Lindsay Hughes, PhD
IQVIA, Parsippany, NJ, United States
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Flora Mulkey
Food and Drug Administration, Silver Spring, MD, United States
Beware (non-proportional) Hazard! Alternative Routes for Dealing with Time-Varying Treatment Effects in Evidence Synthesis for Health Technology Assessment (HTA) Decision Making
Session Type: Issue Panel
Topics: Methodological & Statistical Research, Health Technology Assessment, Clinical Outcomes
Track: Access and Pricing
Level: Intermediate
ISSUE: Often when conducting meta-analyses (MA) of time-to-event (TTE) results, especially in Health Technology Assessment (HTA), a hazard ratio (HR) scale is used due to the ease with which a treatment effect can be incorporated into a cost-effectiveness decision model. However, issues arise when there is evidence of non-proportional hazards in some of the trials/studies included. A number of methods have been advocated, including; fractional polynomial models, flexible M-splines, parametric models, flexible parametric models, piecewise exponential models, Restricted Mean Survival Time (RMST) models, and more recently a time-varying HR approach. However, the use of some methods has been limited by either their complexity and/or the ease of incorporating the results into cost-effectiveness decision models. This issue panel introduces, debates and reviews both more flexible and simpler methods, and discusses the relative merits of the most commonly used and alternative approaches.
OVERVIEW: Keith Abrams will introduce and provide an overview on the use of methods for dealing with non-proportional hazards in HTA (10 minutes – including audience participation via the ISPOR app), and the three panellists will then consider the different methods in HTA using illustrative examples (10 minutes each) – David Phillippo will discuss the use of spline-based approaches to dealing with non-proportional hazards and how these capture time-varying effects, Rhiannon Owen will discuss the use of simpler time-varying hazard ratio approaches and how these can be more useful in a HTA setting, and Shannon Cope will discuss the relative merits of all the methods available, especially regarding implications for integrating the results into modelling and Joint Clinical Assessment (JCA). Keith Abrams will then moderate a discussion involving the audience and panellists on the relative merits of using the different methods available in a HTA setting, before eliciting the audience’s final views via the ISPOR app.
Moderator
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Keith R Abrams, BSc, MSc, PhD
University of Warwick, Coventry, United Kingdom
Keith R Abrams is Professor of Statistics & Data Science in the Department of Statistics and Adjunct Professor of Biostatistics in Warwick Medical School (WMS) at the University of Warwick and a National Institute for Health and Care Research (NIHR) Senior Investigator Emeritus. He is also Honorary Professor in the Centre for Health Economics at the University of York and Associate Director of Health Data Research (HDR) UK. His research centres around the development, evaluation, application and translation of (Bayesian) statistical methods in Health Technology Assessment (HTA) and Health Data Science. Prof Abrams is a member of NICE’s Decision & Technical Support Unit (DTSU) and has previously served on NICE’s Diagnostics Advisory and Technology Appraisal Committees. He is a Fellow of the Royal Statistical Society, and a Chartered Statistician. He is also Chief Statistical & HTA Adviser at Visible Analytics Limited.
Speakers
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David Phillippo, BSc, MSc, PhD
University of Bristol, Bristol, United Kingdom
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Rhiannon K Owen, BSc, MSc, PhD
Swansea University, Swansea, United Kingdom
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Shannon Cope, MSc
Precision AQ, VANCOUVER, Canada
Shannon Cope is a leader in evidence generation and synthesis supporting the comparative effectiveness of new treatments required for regulatory and health technology assessments. Based in Vancouver, Canada, she serves as a Senior Advisor to Precision AQ, where she leads health economics and outcomes (HEOR) studies and supports Joint Clinical Assessments in Europe.
She has expertise in network meta-analysis, external control analyses, population-adjusted analyses, trial and individual-level surrogacy analyses, multi-level network meta-regressions that integrate data from randomized controlled trials with patient and study-level evidence, expert elicitation for time-to-event outcomes, and AI for evidence synthesis. Her work has contributed to numerous peer-reviewed publications and conference presentations, particularly in oncology and chronic disease management.
She holds a Master of Science degree in Health Administration from the University of Toronto and has been recognized for her contributions to methodological innovation in HEOR.
Tackling Societal Challenges Facing Public Health
Session Type: Research Podiums
This session explores how inequalities in healthcare access, social determinants of health, and economic factors shape population health outcomes across different settings.
DISAGGREGATING SOCIAL DETERMINANTS OF HEALTH TO PREDICT COLORECTAL CANCER SCREENING: A TRACT-LEVEL DOMINANCE ANALYSIS
OBJECTIVES: Colorectal cancer is among the most commonly diagnosed cancers in the United States and the leading cause of cancer death for men and women combined, yet it is also one of the most preventable and early-stage detection carries a 91% five-year survival rate compared to 14% after distant spread. The communities where screening is lowest are often the same communities where late-stage diagnosis is most common, a convergence that demands precise identification of which neighborhood barriers most strongly suppress screening uptake. Composite social deprivation indices such as the CDC Social Vulnerability Index (SVI) are widely used to study these disparities but obscure which specific determinants drive screening variation, and standard regression with correlated predictors is difficult to interpret. This study applies dominance analysis, a variable-importance method designed for correlated predictors, to rank neighborhood-level determinants of tract-level screening prevalence and test whether those rankings vary across states.
METHODS: We constructed a cross-sectional dataset of U.S. census tracts linking CDC PLACES screening prevalence, SVI measures, individual American Community Survey predictors (e.g., poverty, unemployment, insurance coverage, education, limited English proficiency), USDA Rural-Urban Commuting Area codes, and Opportunity Atlas intergenerational mobility estimates harmonized to 2020 boundaries. Sequential models assessed explanatory gains from disaggregation. LASSO cross-validation will pre-select predictors before dominance analysis, conducted nationally and within six focal states: Georgia, Texas, Iowa, New York, California, and Massachusetts.
RESULTS: Adjusted R² increased from 0.398 (SVI composite) to 0.522 (SVI themes) to 0.650 (individual predictors), demonstrating substantial explanatory heterogeneity masked by composite indices. Adding Opportunity Atlas mobility raised adjusted R² to 0.653 on 81,498 tracts, statistically significant but modest in practical magnitude.
CONCLUSIONS: Individual tract-level SDOH measures explain substantially more screening variation than composite indices. Dominance analysis will provide definitive variable-importance rankings (eg. demographic & socioeconomic factors), clarify whether intervention priorities differ across states, and designing policies to address SDoH.
QUANTIFYING UNMET CARE GAPS IN CHRONIC DISEASE AND CARDIOMETABOLIC MULTIMORBIDITY: THE CLINICAL, ECONOMIC AND MACROECONOMIC CASE FOR CLOSING THEM
OBJECTIVES: Underdiagnosis and suboptimal treatment of long-term conditions drive inefficiency and poor outcomes, with documented care gaps across hypertension, diabetes and respiratory disease. This analysis quantifies the clinical and economic burden of cardiovascular-kidney-metabolic (CKM) multimorbidity and models a prevention-first approach across population, system, employer and macroeconomic dimensions in England.
METHODS: CF used NHS England secondary-care cost data (2023/24), HES/ONS data across five condition clusters (cardiovascular disease, type 2 diabetes, obesity, chronic kidney disease, dementia). Diagnosis and treatment gaps were estimated from population prevalence and registry data against NICE-aligned pathways; healthcare resource utilisation savings were modelled under optimised risk-factor management. Obesity-associated relative risks were derived from HES comparisons, and per-person cost variation assessed across regions by deprivation.
RESULTS: CKM conditions account for ~45% of chronic-disease burden and ~56% of acute costs in England (~£26bn annually), yet few achieve optimal treatment (37% of CVD patients at lipid targets; 28% of eligible type 2 diabetes at HbA1c target; 54% of people with obesity diagnosed). Obesity (BMI >30) raised relative risk for hypertensive heart disease (2.6), type 2 diabetes (2.5), CKD (2.1), osteoarthritis (2.7) and gout (2.5). Better detection and management could avert 3.4 million CVD events and save ~£12bn/year in secondary care and ~£2.2bn in primary-care/polypharmacy avoidance; CF sizes £6.1-9.2bn from closing care gaps alone. Per-person acute cost for most-deprived 20% patients with multimorbidity (£3.54k) closely tracked the rest of the population (£3.50k) but with materially greater between-area variation indicating systematic underservice in deprived areas.
CONCLUSIONS: The consequences of unmet care extend beyond NHS budgets to employers, insurers and the Treasury. Earlier intervention on upstream drivers, particularly obesity, and equitable delivery to most deprived populations are the highest-yield opportunities; realising this value requires targeted eligibility, diagnostic-led risk stratification and funding models that align incentives across payers, systems and government.
TERRITORIAL INEQUALITIES OF PRACTICING PHYSICIANS IN HUNGARY AND THE DACH COUNTRIES IN 2024: A GINI AND THEIL INDEX-BASED ANALYSIS
OBJECTIVES: To assess and compare spatial inequalities in physician supply across Hungary and the DACH countries in 2024 using population- and area-based indicators, with emphasis on Gini and Theil inequality measures.
METHODS: We conducted a cross-sectional study of spatial inequalities in physician supply in Hungary, Austria, Germany, and Switzerland in 2024. Practicing physicians were examined across functionally comparable administrative units (federal states, counties, and cantons). Physician availability was measured using two indicators: number of physicians per population and per area. Lorenz curves demonstrating the physician distribution were constructed from cumulative shares. Inequalities were quantified using the Gini coefficient as a weighted measure. Theil T (T) and Theil L (L) indices were calculated using population- and area-based indicators. Data were obtained from OECD Health Statistics database, Statistics Austria, the German Medical Association, the Federal Statistical Office of Germany, the Hungarian Central Statistical Office, the FMH Swiss Medical Association, and the Federal Statistical Office of Switzerland.
RESULTS: Substantial cross-country and within-country differences were observed in 2024. Among the included countries, Austria had the highest physician density per 100,000 population (568), while Hungary had the lowest (448). Internal disparities were most pronounced in Switzerland (6.59-fold) and Hungary (3.45-fold), whereas Austria and Germany showed smaller differences (1.59-fold and 1.92-fold, respectively). Population-based inequalities were moderate: Gini coefficients ranged from 0.058 (Germany) and 0.096 (Austria) to 0.153 (Switzerland) and 0.226 (Hungary). Theil indices confirmed low-to-moderate inequality (Germany: T=0.008, L=0.007; Austria: T=0.015, L=0.014; Switzerland: T=0.041, L=0.040; Hungary: T=0.087, L=0.084). Area-based inequalities were substantially higher. Gini values reached 0.501 in Hungary and 0.557 in Switzerland, while Austria and Germany remained more moderate (G=0.353; G=0.373). Theil indices (Germany: T=0.357, L=0.251; Austria: T=0.909, L=0.321; Switzerland: T=0.711, L=0.618; Hungary: T=1.056, L=0.466) highlighted strong spatial concentration.
CONCLUSIONS: Population-based indicators substantially underestimate spatial inequalities in physician supply compared with area-based measures.
JOB LOSS AND CARDIOVASCULAR HEALTH: A LARGE-SCALE ANALYSIS ON THE IMPACT OF UNEMPLOYMENT ON MAJOR ADVERSE CARDIOVASCULAR EVENTS AND ALL-CAUSE MORTALITY
OBJECTIVES: To investigate the short- and mid-term effects of job loss on the incidence of major adverse cardiovascular events (MACE) and all-cause mortality in a large, unselected German cohort.
METHODS: This retrospective study utilized data from the German Analysis Database for Evaluation and Health Services Research (2013-2023), encompassing approximately 4.4 million individuals. Inclusion required one baseline year, a reporting year, and three follow-up years or death after baseline. Job loss was defined as employment during the baseline year followed by unemployment in the reporting year. MACE included acute coronary syndrome, stroke, and incident heart failure. A 1:1 exact matching for age, gender, and reporting year, and propensity score matching for traditional coronary artery disease risk factors and Charlson Comorbidity Index (CCI) were applied to generate a control cohort. Odds ratios (OR) were calculated using the Baptista-Pike method, with significance tested by Fisher’s exact test.
RESULTS: Among 57,203 individuals with job loss, 2% were unmatched and excluded. Both cohorts had a mean age of 50 ± 11 years, with 47.2% women. During follow-up, 3,899 MACE occurred in the job loss cohort versus 3,310 in controls. Job loss was associated with a increased risk of MACE (OR 1.21, 95% CI 1.16-1.26). The risk was modest in the reporting year (OR 1.07, 95% CI 1.02-1.13), peaked in the first follow-up year (OR 1.32, 95% CI 1.20-1.45), and declined gradually thereafter. Men showed a slightly higher risk (OR 1.21) compared to women (OR 1.16). All-cause mortality was also elevated in the job loss group (OR 1.17, 95% CI 1.08-1.26).
CONCLUSIONS: Job loss significantly increases the risk of major cardiovascular events and all-cause mortality, particularly within the first year following unemployment. These findings support recognizing job loss as a cardiovascular risk factor and underscore the need for targeted preventive strategies.
Should real-world evidence play a role in reimbursement decisions on decommissioning health technologies?
Session Type: Issue Panel
Topics: Real World Data & Information Systems, Health Technology Assessment, Health Policy & Regulatory
Track: Real-World Evidence (RWE)
Level: Intermediate
Issue: As healthcare systems face growing pressure to allocate scarce resources to innovative health technologies, real-world evidence (RWE) is increasingly considered to support access decisions. However, the use of RWE to inform reassessment and potential decommissioning of existing health technologies is less established. Is RWE robust enough to justify withdrawal, reimbursement restrictions on specific patient populations, and what evidence is required within a transparent and ethical governance framework? The session will discuss key uncertainties around using RWE to reassess the value of health technologies and inform potential decommissioning, including pre-specified reassessment criteria, analytic standards, funding, governance, independent review of data generation, and safeguards to manage bias and conflicts of interest when manufacturer-sponsored analyses may influence reimbursement restrictions.
Overview: This panel will debate whether and how real-world evidence should influence reimbursement reassessment and decommissioning decisions. Julie De Almeida will open by framing the debate using examples including NICE's use of the SACT dataset in oncology reassessment and the CanREValue framework.
Prof. Durand-Zaleski will provide an HTA perspective, discussing how RWE could trigger reassessment and decommissioning decisions by identifying low-value prescribing, limited effectiveness in routine practice, safety concerns, inappropriate use, or subgroups where reimbursement is no longer justified.
From an industry perspective, Dr. Balp will highlight challenges of using observational data to support decommissioning decisions, including the potential impact on patients already treated, incentives for evidence generation, and implications for innovative therapies. A key question is who should fund RWE that may result in decommissioning or reimbursement restrictions?
Prof. Jönsson will provide an independent academic perspective, challenging both viewpoints and asking whether there should be pre-specified reassessment criteria at initial coverage making RWE critical across the lifecycle of a health technology.
Moderator
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Julie De Almeida, BSc
Acumetis, Doncaster, United Kingdom
Speakers
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Isabelle Durand-Zaleski, MPP, PhD, MD
Paris, France
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Maria-Magdalena Balp, MASc, MD
Novartis Pharma AG, Basel, Switzerland
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Linus Jönsson, MSc, PhD, MD
Karolinska Insitutet, Stockholm, Sweden
First Wave, First Lessons: Multi-Stakeholder Insights from the initial Joint Clinical Assessments Under the European Regulation on Health Technology Assessment
Session Type: Issue Panel
Topics: Health Technology Assessment
Track: Access and Pricing
Level: Introductory
ISSUE: January 2025 marked the start of the European Regulation on Health Technology Assessment (EU HTAR), initiating Joint Clinical Assessments (JCAs) for the first eligible technologies. Health technology developers (HTDs), HTA bodies, and payers are now seeking early insights on how procedural and methodological requirements are being implemented in practice, and what this means for submission and capacity planning, assessment quality, evidence expectations, national pull-through and use, facilitating patient access to new technologies. This panel will examine early experiences from the first JCAs, focusing on observed process dynamics, facilitators and barriers to effective engagement, and will translate these into practical lessons learned and opportunities for continuous improvement.
OVERVIEW: At the time of ISPOR EU 2026, an estimated 6-8 JCA dossiers and reports will have been published, having gone through this new process. These cases provide a unique opportunity to reflect on how requirements, timelines, interactions, and evidence expectations are being implemented, providing valuable insights into optimal preparations for the JCAs to come. The panel will begin with a concise overview of the EU HTAR and the JCA process, including key milestones, roles, and deliverables. Panellists will then discuss hands-on experience from multiple perspectives on key dimensions, such as PICO scoping, evidence requirements, stakeholder engagement, management of uncertainty, timeline adherence, and national procedure integration. A thematic synthesis of cross-case findings will highlight areas of emerging alignment and practical benefits, recurring challenges, and variation in interpretation. The discussion will integrate perspectives from a JCA assessor, an HTA body representative, and a HTD representative. The session will conclude with actionable recommendations for evidence planning, cross-functional alignment, procedural learnings, and effective stakeholder engagement to support high-quality assessments and facilitate patient access.
Moderator
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Suzette Matthijsse
Lumanity, Sheffield, United Kingdom
I am passionate about accelerating patient access to innovative therapies by shaping evidence, strategy, and cross-functional decision-making across the product lifecycle. With 15 years of experience spanning academia and HEOR consulting, I specialize in guiding pharmaceutical and biotech partners through the evolving EU HTAR landscape from early development to Joint Clinical Assessments since the EU HTAR entered into force in 2022.
Speakers
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Anne Willemsen, MSc
Zorginstituut Nederland, Diemen, Netherlands
Anne Willemsen joined the Dutch National Healthcare Institute in 2016, and currently is Chair of the Joint Clinical Assessment (JCA) subgroup under the EU HTA Regulation’s Coordination Group, after having served the JCA Subgroup as co-chair for the past three years (2023-2026). She leads the subgroup through the preparatory phase of the JCA process and will guide it through the production of Joint Clinical Assessments started as of January 2025. Previously she held several roles within the European Network for HTA (EUnetHTA), including spearheading the Secretariat responsible for coordinating JCAs and joint work with the European Medicines Agency. Her work also focused on patient involvement and conflict of interest procedures. With a background in Health Sciences and a recently started PhD, she brings extensive experience in European HTA collaboration, focusing on predictability of the procedures, transparency and stakeholder engagement in JCA.
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James Ryan, BA, MSc
AstraZeneca, Cambridge, United Kingdom
James is a health economist with 25 years' experience and a passionate HTA practitioner. As Director, HTA Policy at AstraZeneca, he is leading AstraZeneca's preparation for the EU HTA Regulation, as well as their external advocacy, including publications, forums, and panel discussions. James is vice-chair of EFPIA’s HTA Working Group and co-lead of their JCA methods workstream. He is also a representative for AstraZeneca on both the HTAi Global Policy Forum and HTA meetings for the Centre for Innovation in Regulatory Science.
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Niklas Hedberg, MSc
TLV, Stockholm, Sweden
Niklas Hedberg is the Chief Pharmacist at the Dental and Pharmaceutical Benefits Agency (TLV) in Sweden.
Niklas is the HTAR Coordination Group co-chair for medicinal products. He was the Chair of the Executive Board for EUnetHTA21 (2021-2023) and for EUnetHTA JA 3 (2018- 2021).
Niklas has been working with HTA and Pricing and Reimbursement since 2001.
Reading Between the Endpoints: Translating Trials from Regulatory Estimands to HTA/JCA Decision Making
Session Type: Workshop
Topics: Health Technology Assessment, Methodological & Statistical Research, Clinical Outcomes
Track: Access and Pricing
Level: Intermediate
Purpose
Estimands are now firmly embedded in regulatory guidance and are gaining prominence in HTA, with formal relevance under the EU JCA. Despite this shared framework, challenges remain in translating trial results across decision contexts. Regulatory estimands are typically defined to support benefit–risk assessment under controlled trial conditions, whereas HTA/JCA decision making often require comparative effectiveness aligned with clinical practice. These differing objectives can have implications from trial design to evidence interpretation.
A key methodological tension arises between the JCA scope, formulated using a PICO framework, and the estimand attributes required to explicitly address intercurrent events. While PICO specifies population, intervention, comparator, and outcome, it does not fully articulate how events such as treatment discontinuation, switching or subsequent therapies should be handled, whereas estimands require these assumptions to be made explicit. Ultimately, analyses described in the estimand format are required for JCA submission. As such, misalignment between PICOs and estimands can complicate interpretation, limit reuse of regulatory analyses, and reduce the relevance of trial evidence for JCA assessment.
Description
This workshop aims to compare regulatory and HTA/JCA requirements and their implications for estimand definition; examine how intercurrent events and trial design choices influence the applicability of estimands across decision contexts; and identify practical strategies to improve early alignment and transparency to support JCA relevant comparative effectiveness assessment.
The session will feature perspectives from industry, regulatory, academic/HTA and EFPSI with experience in estimands and real-world evidence. With practical examples, speakers will discuss intercurrent event handling, trial design constraints, and considerations when extending the estimand framework beyond clinical trials to JCA. Interactive polling and moderated audience discussion will identify common challenges and actionable approaches to improve alignment, interpretability, and decision relevance across the evidence continuum.
Moderator
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Miranda Cooper, BSc, MSc
AstraZeneca, Barcelona, Spain
Speakers
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Uwe Siebert, MPH, MSc, ScD, MD
UMIT TIROL - University for Health Sciences and Technology; Harvard Chan School of Public Health, Hall in Tirol, Austria
Uwe Siebert, MD, MPH, MSc, ScD, is a professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL-University for Health Sciences and Technology in Austria and director of the Division for HTA in the ONCOTYROL–Center for Personalized Cancer Medicine in Austria. He is also adjunct professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health and Affiliated Researcher in the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston.
After medical school, he worked for several years as a physician in international public health projects in West Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in decision sciences at the Harvard School of Public Health.
His research interests include applying real-world evidence-based quantitative, causal and translational methods from public health, epidemiology, artificial intelligence, comparative effectiveness research, health services and outcomes research, economic evaluation, modeling, and health data a d decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, clinical guideline development, public health policies and patient guidance. His research focuses on cancer, infectious disease, cardiovascular disease, neurological disorders, and others.
He has been leading projects/work packages in several EU FP7, H2020 and Horizon Europe projects (eg, ELSA-GEN, BiomarCaRE, MedTecHTA, DEXHELPP, EUthyroid, FORECEE, MDS-RIGHT, RECETAS, CORE-MD, EUREGIO-EFH, CIDS, OnCoVID, 4D PICTURE, CATALYSE). He teaches HTA, health economics, modeling, epidemiology, causal inference and target trial emulation, and data and decision science for academia, industry, and health authorities in Europe, North and South America, and Asia. He directs the Continuing Education Program on Health Technology Assessment & Decision Sciences (htads.org).
He has served as member of the ISPOR Directors Board and as president of the Society for Medical Decision Making (SMDM). He is a leadership member of the ISPOR Personalized/Precision Medicine SIG, a member of the Latin America Consortium Advisory Committee of ISPOR, and co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force. He is a member of the Oncology Advisory Council and the National Committee for Cancer Screening of the Austrian Federal Ministry of Health.
He has authored more than 400 publications (> 30,000 citations, H index > 80), and is editor of the European Journal of Epidemiology. Further information Internet: http://htads.org, umit-tirol.at/dph, hsph.harvard.edu/uwe-siebert, Twitter: @UweSiebert9, LinkedIn: uwe-siebert9.
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Antonia Morga, PhD
Astellas Pharma Ltd, Addlestone, United Kingdom
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Dominik Karres
European Medicines Agency, Netherlands
14:45 - 15:15
Coffee and Connect
Session Type: General Meeting
Head to the exhibit hall to connect with fellow attendees and exhibitors over a steaming cup of coffee.
15:15 - 16:15
From Principles to Practice: Preliminary Good Practice Recommendations from the Joint ISPOR-SMDM Model Validation II Task Force
Session Type: Forums
Topics: Economic Evaluation, Methodological & Statistical Research, Health Technology Assessment
Level: Intermediate
Health economic models are widely used to inform healthcare reimbursement decisions and pricing negotiations, but in practice there is no consistent approach to validating them or reporting how validation has been conducted. While existing ISPOR-SMDM guidance highlights the importance of validation, so far this has not been consistently applied in research or economic evaluation of health technology. This session will be delivered as an interactive panel discussion. After a short introduction by Dr. Salah Ghabri, task force members will present the joint task force’s draft recommendations. These recommendations build on recent advances in modelling, software development, real-world evidence, and the growing use of artificial intelligence (AI). The task force’s aim is to provide practical and flexible guidance that can be applied across different model types, decision settings, and stakeholder groups. Dr. Beate Jahn will discuss draft recommendations and practical challenges covering key aspects of model validation, including conceptual model validation, input data validation, model verification and validation of model outcomes. Dr. Isaac Corro Ramos will present draft recommendations for terminology, available tools, and validation planning, documentation and reporting. Finally, Dr. Mafalda Ramos will provide a stakeholder analysis, discussing the extent to which these draft recommendations can be applied in practice, and what could be the role/responsibility of various stakeholders, including HTA agencies, industry, CROs, journal editors and academic researchers. Lastly, the audience will then be invited to provide feedback to help shape the final recommendations before publication, ask questions, and share their experiences.
Moderator
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Salah Ghabri, MSc, PhD, HDR
French National Authority for Health (HAS), Paris, France
Speakers
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Beate Jahn, PhD
UMIT – University for Health Sciences, Medical Informatics and Technology, Hall i.T., Austria
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Isaac Corro Ramos, MSc, PhD
Erasmus University, Rotterdam, Netherlands
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Mafalda Ramos, MSc
From Value Assessment to Delivery Reality: Integrating HEOR and Health Systems Perspectives in Coverage Decisions for GLP-1 Therapies
Session Type: Forums
Topics: Health Policy & Regulatory, Economic Evaluation, Health Service Delivery & Process of Care
Level: Intermediate
Hosted by the ISPOR Institute for Healthcare Transformation, this forum explores how evidence of value can be translated into sustainable implementation within real-world health systems. A moderated panel will bring together complementary perspectives shaping coverage decisions for GLP-1 therapies. The HEOR perspective will frame the evidence base for coverage, including cost-effectiveness, budget impact, and outcomes modeling adapted to regional settings. The health systems perspective will shift the discussion from whether therapies should be covered to whether systems can support appropriate initiation, follow-up, supply forecasting, prescribing governance, and equity monitoring. The policy and payer perspective will examine how these considerations translate into phased adoption, conditional coverage, and governance mechanisms.
Moderator
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Kelly Saldana
ISPOR, Lawrenceville, NJ, United States
Speakers
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Sara Al Dallal
United States
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Sabry Hamza, PhD
Amman, Jordan
Prof. Sabry Hamza, MD
Sabry Hamza, MD, is Professor of Obstetrics and Gynecology at Ain Shams University, Cairo, with more than 30 years in academic medicine and over 25 years leading large donor-funded health programs across Egypt, Jordan, and the wider MENA region. As a USAID Chief of Party and principal evaluation lead, he directed health systems portfolios exceeding US$200 million, spanning health financing, service delivery, quality assurance, and clinical governance, and worked closely throughout with ministries of health, USAID, UNFPA, and UN agencies, including in humanitarian and refugee settings.
His research on low-cost interventions for obstetric hemorrhage, conducted with the University of California, San Francisco and partners in Egypt and Nigeria, became part of the evidence base underpinning World Health Organization (2012) and FIGO guidance. He led the establishment of Jordan’s Maternal Mortality Surveillance and Response System and co-authored national reproductive health strategies, curricula, and maternal mortality reports in both Egypt and Jordan.
His work sits where value assessment meets delivery reality: what health systems can actually absorb, finance, and sustain once a coverage decision is made.
From Policy to Practice: The Evolution of HEOR and Healthcare Decision-Making in China — Experiences, Insights, and Lessons Learned
Session Type: Forums
Topics: Health Policy & Regulatory, Real World Data & Information Systems, Economic Evaluation
Level: Intermediate
China’s healthcare system has undergone profound transformation in recent years, with Health Economics and Outcomes Research (HEOR) emerging as a critical discipline supporting evidence-based policy development and healthcare decision-making. Driven by national healthcare reforms and initiatives from the National Healthcare Security Administration (NHSA), the integration of health technology assessment (HTA) and real-world evidence (RWE) has accelerated the transition toward value-based healthcare, enabling more effective resource allocation, reimbursement decisions, and patient access to innovative therapies.
This forum will bring together leading experts to discuss the latest advances in HEOR, healthcare reform, and evidence-informed decision-making in China. Through diverse perspectives and practical experiences, speakers will examine the development of healthcare policies, advances in innovative drug development, evolving reimbursement and pricing frameworks, and the expanding role of real-world evidence in shaping healthcare decisions.
Moderated by Dr. Jing Wu, the session will provide an overview of China’s healthcare system, key achievements, and ongoing challenges. Dr. Yue Yang will discuss recent developments in drug approval pathways and emerging trends in innovative medicine development. Dr. Wen Chen will examine national reimbursement policies, drug price negotiations, and evolving evidence requirements for healthcare decision-making. Dr. Tao Guo will share real-world case studies highlighting how RWE is being applied to inform policy, optimize healthcare delivery, and improve patient outcomes.
Together, the forum will provide valuable insights into China’s journey from policy evolution to practical implementation, highlighting lessons learned and opportunities for the future of evidence-based healthcare.
Moderators
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Jing Wu, PhD
Tianjin, China
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Jing Wu, RPh, PhD
Tianjin University, Beijing, China
Prof. Wu has dedicated nearly 20 years to research and teaching in health economics and pharmacoeconomics. Her expertise spans health policy evaluation, pharmacoeconomic evaluation, real-world data analysis, and health-related quality of life and health utility study. She earned her PhD in Pharmacoeconomics through a joint program between Shenyang Pharmaceutical University and Peking University in 2007 and was a visiting scholar at the Schaeffer Center for Health Policy and Economics at the University of Southern California in 2013. Prof. Wu has authored over 100 high-impact scientific articles, led three National Natural Science Foundation of China projects, contributed to multiple pharmacoeconomics textbooks, and spearheaded the development of the China Guidelines for Pharmacoeconomic Evaluations (2020) and the Companion Guide to the China Guidelines for Pharmacoeconomic Evaluations (2022), promoting the development and standardization of pharmacoeconomics in China. She currently serves as Chair of the Pharmacoeconomics Committee of the Chinese Pharmaceutical Society, Vice Chair of the Pharmacoeconomics Committee of the China Health Insurance Research Association, Vice Chair of the Pharmacoeconomics Committee of the Chinese Society of Research Hospitals. She co-chaired the ISPOR Asia-Pacific Conferences in 2018 and 2022, served as Executive Chair of the 10th Huaxia Pharmacoeconomics and HTA Forum in 2019, and founded the China Young Scholars Forum on Pharmacoeconomics in 2017—organizing around 40 high-level academic meetings to date. As a national-level advisor, she provides strategic guidance to China’s National Healthcare Security Administration, playing a key role in reimbursement policy and healthcare-system reform.
Speakers
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Yue Yang
Beijing, China
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Wen Chen, PhD
School of Public Health, Fudan University, Shanghai, China
Professor CHEN received his PhD degree in social medicine and health management from Shanghai Medical University in 1998 and completed research fellowship at School of Public Health, University of California at Berkeley from August of 2000 to May of 2001.
Currently he serves as the director of the Center for Yangtze River Delta Healthcare Security Research and Center for Pharmacoeconomic Evaluation and Research, Fudan University. He was the Dean of the School of Public Health at Fudan University from April 2013 to June 2017. Prof. Chen is often invited as investigator and advisor by national and municipal governments for various research programs in the field of Chinese healthcare system, national and provincial health insurance, pharmacoeconomics and pharmaceutical policy, health financing, and etc. He had more than 160 publications on international and Chinese health economics and management journals.
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Tao Guo, MA
The Guiding Center of Pharmaceuticals and Medical Service Pricing, Tendering and Purchasing,National, Beijing, China
Updating HTA Methods in Central and Eastern Europe: Can National Guidelines Converge While Remaining Locally Relevant?
Session Type: Issue Panel
Topics: Health Technology Assessment, Methodological & Statistical Research, Organizational Practices
Track: Access and Pricing
Level: Intermediate
ISSUE:
HTA guidelines in Central and Eastern Europe are increasingly expected to address rapid methodological and policy change, including real-world evidence, disease severity, digital health technologies, artificial intelligence, cost-effectiveness thresholds and the implementation of EU Joint Clinical Assessment. The key debate is whether CEE countries should move toward greater methodological convergence or preserve nationally tailored approaches reflecting local payer needs, data availability and institutional capacity.
OVERVIEW:
This issue panel will compare recent HTA guideline developments in Hungary, Czechia, Ukraine and Poland. The Hungarian perspective will present a regular, predefined process of methodological guideline revision, including recent changes related to willingness-to-pay thresholds and the potential use of additional health-economic tools such as EQ-5D-5L and bolt-on dimensions. The Czech perspective will focus on the ongoing update of national HTA guidance, including real-world evidence, QALY shortfall for severity assessment, digital health technologies and vaccines. The Ukrainian perspective will discuss the third update of national HTA guideline for medicines, including recent developments in evaluating added clinical benefit, the first guideline for medical devices, and institutional implementation in a rapidly evolving healthcare system. The Polish perspective will cover the fourth update of national HTA guidelines, including changes resulting from Joint Clinical Assessment, patient involvement, artificial intelligence and other methodological clarifications. Each panelist will provide a 10-minute country perspective, followed by 20 minutes of moderated debate and audience discussion. The session will benefit HTA agencies, payers, policymakers, academics, consultants and industry representatives interested in translating emerging methodological standards into practical national guidance. The panel aims to identify where regional convergence is useful, where national flexibility remains essential, and how CEE countries can learn from one another when updating HTA methods.
Moderator
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Maciej Niewada, PhD
ISPOR Poland Chapter, Warszawa, Poland
Speakers
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Magdalena Rdzanek
AOTMiT, Warszawa, Poland
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Oresta Piniazhko, PhD
State Expert Center of the Ministry of Health of Ukraine, Kyiv, Ukraine
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Tomáš Doležal, PhD, MD
Value Outcomes s.r.o., Prague 2, Czech Republic
The HEMA Debate: The Value Judgements Involved in Deciding How “Value” Is Measured: A Value in Health Forum
Session Type: Forums
Topics: Methodological & Statistical Research, Economic Evaluation, Health Technology Assessment
Level: Intermediate
This Value in Health Forum features a live debate between Mark Sculpher and Darius Lakdawalla on a key issue arising from the 2026 Health Economics Methods Advisory (HEMA) report: how should the benefits of healthcare be measured and valued in the economic evidence used to inform health technology assessment decisions? While there has been much discussion at previous ISPOR meetings about practical ways of extending the elements of value included in value assessment, this debate directly tackles the deeper issue that sit at the heart of these methods choices. What is the appropriate normative or conceptual framework for economic evaluation used in health technology assessment? And given the fundamental differences between competing approaches, who should decide what “value” means in a healthcare system?
Devlin will introduce the session on behalf of Value in Health, including some reflections on the role of value judgements in HEOR science.
McGuire will introduce the speakers and moderate the debate.
Lakdawalla will argue that methods should be driven by the principles of welfare economics, based on measured individual preferences. Sculpher will argue that, over many years, welfare economics has adapted to key features of collectively funded health systems and normative choices need to be taken by accountable policy makers—not economists. McGuire will discuss these contrasting positions, indicating areas for compromise and new research.
This session will feature a Q&A and audience participation is welcomed.
Moderator
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alistair mcguire, BA, MPhil, PhD
Speakers
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Nancy J Devlin, PhD
University of Melbourne, Melbourne, Australia
Nancy is Professor of Health Economics at the University of Melbourne and Editor-in-Chief at Value in Health. Her principal areas of research expertise are the measurement and valuation of patient reported health outcomes; the cost effectiveness thresholds used in making judgments about value for money in health care; and priority setting in health care.
Nancy has published >200 original peer reviewed journal articles on a wide range of empirical and theoretical topics in health economics, and is co-author of Economic Analysis in Health Care, a textbook on health economics widely used in the UK and elsewhere. In 2025 Nancy was named in the Stanford/Elsevier ‘top 2% scientists’. In 2024, she was ranked 3rd in the world’s ‘Highly Ranked Scholars’™ in Health Economics based on productivity, impact and research quality. Her work was highlighted in the UK’s NIHR 10-year anniversary report, which noted ‘The impact of her research is worldwide and highly significant in improving health and health care decision making’ . Her research, submitted as a case study to the UK’s 2014 REF exercise, was judged by the sub-panel as ‘demonstrating very considerable impact in terms of reach and significance' .
She was the lead investigator on QUOKKA, a multi-million dollar programme of research on health outcomes in children, funded by the Medical Research Future Fund (Australia) (2020 – 2025), results from which have been described as “…a huge leap forward for the field of health-related quality of life measurement in child health”
She is the elected Chair of the Board of the EuroQol Research Foundation (2020-2025), the European-based not for profit body that developed the EQ-5D, the world’s leading generic measure of patient reported outcomes. Nancy has also served as the 2019/20 elected international President of ISPOR, the peak international professional society for health economics and outcomes research.
Prior to joining the University of Melbourne in 2019, Nancy was Director of Research at the Office of Health Economics, London for ten years, leading the development of its research programme, culminating in OHE achieving Independent Research Organisation (IRO) status in 2019. Prior to OHE she was Professor of Economics at City University of London, where she held the positions of Head of the Economics Department and Dean of Social Sciences. She has 40 years of experience as a researcher and as an advisor to health care organisations, both in the public and private sectors, in the UK and internationally.
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Darius Lakdawalla, PhD
University of Southern California, Los Angeles, CA, United States
Darius Lakdawalla is a widely published, award-winning researcher and a leading authority on health economics and health policy. He holds the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation at the University of Southern California, where he sits on the faculties of the Alfred E. Mann School of Pharmacy and Pharmaceutical Sciences and the Sol Price School of Public Policy. He is also the Chief Scientific Officer at USC’s Leonard D. Schaeffer Center for Health Policy and Economics, one of the nation’s premier health policy research centers. His research has focused primarily on the economics of risks to health, the value and determinants of medical innovation, the economics of health insurance markets, and the industrial organization of healthcare markets. His academic work has appeared in leading peer-reviewed journals of economics, health policy, and medicine. His work has also been featured by prominent popular press outlets, such as the Wall Street Journal, National Public Radio, Forbes, and the New York Times. Dr. Lakdawalla received the PhRMA Foundation Value Assessment Challenge Award, designed to encourage innovative approaches to defining and measuring value in health care, in 2019 (third place) and 2020 (first place). He is the co-author of the recent book, Valuing Health: The Generalized and Risk-Adjusted Cost-Effectiveness (GRACE) Model, published by Oxford University Press. Dr. Lakdawalla is also a newly elected member (2025) of the National Academy of Medicine.
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Mark Sculpher, PhD
University of York, York, United Kingdom
Shaping the Future of HEOR: A Special Preview of ISPOR's Updated Science Strategy
Session Type: Forums
Topics: Organizational Practices
Level: Introductory
Healthcare decision makers have never faced greater expectations for rigorous, timely, and actionable evidence. As artificial intelligence accelerates evidence generation, real-world evidence becomes increasingly central to regulatory and HTA decisions, and healthcare systems face growing pressure to improve outcomes while managing finite resources, the science of health economics and outcomes research (HEOR) must continue to evolve to meet these demands. In this special forum, ISPOR leaders—including members of the Health Science Policy Council (HSPC)—will offer an exclusive preview of the Society's updated Science Strategy, a forward-looking framework designed to ensure that ISPOR remains at the forefront of scientific innovation and continues to meet the evolving evidence needs of healthcare decision makers around the world. This session will share the approach to its development, the emerging scientific priorities under consideration, and the opportunities where ISPOR can make its greatest contribution. Discussion will explore the future of artificial intelligence, real-world evidence, patient-centered methods, health technology assessment, evidence synthesis, and the next generation of ISPOR Good Practices and scientific initiatives. Importantly, this will be a conversation. ISPOR and the Health Science Policy Council leaders want to hear from the community about the proposed scientific priorities. If you care about where the science of HEOR is headed—and how ISPOR will lead it—this is a discussion you won't want to miss.
Moderator
Speakers
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Deborah A Marshall, PhD
University of Calgary, Calgary, AB, Canada
Professor and Svare Chair in Health Economics, Value and Impact, Cumming School of Medicine, University of Calgary. Her applied research programme assesses the value and impact of health services using health economics, socioeconomic benefits, patient preferences research for national and international research programmes in precision health and patient-oriented research, particularly in child health and rare disease and precision health. She supports embedded research fellows as the Nominated Principal Applicant of the CIHR Health Systems Impact Fellowship National Cohort Training Program to advance capacity for sustainable and patient-centered learning health systems across Canada”. She is a founding co-investigator of the innovative Patient and Community Engagement Research (PaCER) programme at the University of Calgary which trains patients to design and conduct health research, using specific adapted methods of qualitative inquiry. A former President and member of the Board of Directors, incoming Chair of Health Science Policy Council (HSPC) she remains an active member of ISPOR, as chair and member of Task Forces and SIGs.
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Zsombor J Zrubka, MBA, PhD, MD
Initium Zrt., Budapest, III., Hungary
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Peter Neumann, ScD
Tufts Medical Center, Boston, MA, United States
16:00 - 16:45
Artificial Intelligence Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 2, Posters will be hung from 16:00-19:00.
Posters featured in this tour:
PT7: NICE'S APPROACH TO TRIAGING AI USE CASES TO EXPLORE POTENTIAL USE FOR GUIDANCE PRODUCTION TASKS
PT8: ESTIMATING THE COSTS AND BENEFITS OF AI-SUPPORTED DETECTION OF PULMONARY MALIGNANCIES ON ROUTINE CHEST X-RAYS USING REAL-WORLD DATA
PT9: EVALUATING AI VERSUS HUMAN-LED INDUCTIVE THEMATIC ANALYSIS OF PATIENT INTERVIEWS: A CASE STUDY IN TREATMENT PREFERENCES FOR NON-MUSCLE-INVASIVE BLADDER CANCER
PT10: AN INTEGRATED THREE-PLATFORM AI WORKFLOW FOR EU JOINT CLINICAL ASSESSMENT: AI-ASSISTED PICO DEFINITION, REAL-WORLD EVIDENCE VALIDATION, AND AI DOSSIER AUTHORING
PT11: WHERE MUST THE HUMAN REMAIN IN THE LOOP? EVALUATING EXPERT-VALIDATION CHECKPOINTS IN AI-ASSISTED HTA EVIDENCE WORKFLOWS
PT12: AI-ASSISTED DENTAL CARIES DETECTION WITH RADIOGRAPHY: AN EARLY ECONOMIC EVALUATION IN ENGLAND
Patient-Centered Evidence Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 2, Posters will be hung from 16:00-19:00.
Posters featured in this tour:
PT13: CONTENT VALIDITY EVALUATION OF SELECTED PATIENT REPORTED OUTCOME MEASURES FOR PAIN, FATIGUE, AND PHYSICAL FUNCTIONING IN ADULTS, ADOLESCENTS AND CHILDREN LIVING WITH OSTEOGENESIS IMPERFECTA
PT14: CONTENT VALIDATION OF THE NTDT-PRO IN ALPHA-THALASSEMIA: A STANDALONE HYBRID CONCEPT ELICITATION AND COGNITIVE INTERVIEW STUDY
PT15: EXPLORING THE BURDEN OF CHRONIC INDUCIBLE URTICARIA AND THE MEANINGFULNESS OF TREATMENT USING IN-TRIAL INTERVIEWS
PT16: FUNCTIONAL, DISABILITY-RELATED AND CAREGIVER PRODUCTIVITY BURDEN IN PULMONARY FIBROSIS IN GREECE
PT17: STRUCTURAL EQUATION MODELING (SEM) OF PATIENT SATISFACTION WITH ISATUXIMAB ADMINISTRATION: PERCEIVED TIME SAVINGS AND A LATENT INJECTION EXPERIENCE CONSTRUCT INDEPENDENTLY PREDICTED SATISFACTION
PT18: WAITING FOR EVIDENCE OR EARLIER ACCESS? PREFERENCES FOR ONE-TIME THERAPIES AMONG CANCER PATIENTS AND THE GENERAL PUBLIC
Moderator
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Bryan Bennett, BSc, PhD
Jazz Pharma, Berwick-upon-Tweed, United Kingdom
Bryan Bennett, PhD, is the Head of Patient-Centered Outcomes and Senior Director at Jazz Pharmaceuticals, where he leads a dedicated team of Outcomes Scientists developing novel Clinical Outcome Assessment (COA) strategies across neuroscience and oncology. With over three decades of experience spanning clinical practice, academia, consultancy, and the pharmaceutical industry, Dr. Bennett is a passionate advocate for patient-focused drug development. He has consistently driven efforts to ensure the patient's voice is integrated into every stage of the therapeutic lifecycle. Dr. Bennett is an active member of the ISPOR community, recently serving on the ISPOR Europe Program Committee and the COA Special Interest Group Leadership team.
16:00 - 19:00
Poster Session 2
Session Type: Research Posters
Poster Tours 16:00–16:45 | Presenters will be with their posters from 18:00–19:00
16:45 - 17:45
Case Studies in Med Tech Evaluation
Session Type: Research Podiums
This session explores economic evaluation case studies from countries with diverse income levels, healthcare system structures, and stages of health technology assessment (HTA) development. It brings together examples of both sophisticated, high-technology medical interventions and public health programs, illustrating the breadth of economic evaluation across traditional and non-traditional health technologies. Recognizing that non-pharmaceutical technologies often present unique methodological and implementation challenges, the session will examine how economic evaluations can be designed and conducted for these technologies. Drawing on real-world experiences from different healthcare systems, the session will also highlight the policy implications of economic evaluation findings and how they can support evidence-informed decision-making across a range of healthcare contexts.
Moderator
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Abeer A Al Rabayah, MBA, MSc, DrPH
King Hussein Cancer Center, Amman, Jordan
Dr. Abeer Ahmad Al Rabayah leads the Center for Drug Policy and Technology Assessment (CDPTA) at the King Hussein Cancer Center (KHCC) in Amman, Jordan. In addition to her role at KHCC, Dr. Abeer holds a research associate position at the Institute of Public Health, Medical Decision Making, and Health Technology Assessment at UMIT TIROL – University for Health Sciences and Technology, Hall i.T., Austria.
As the director of the Hospital-Based Health Technology Assessment (HB-HTA) program and the founder of the Health Technology Assessment (HTA) fellowship program at KHCC, In 2024, the KHCC HB-HTA program received the King Hussein Cancer Research Award for Academic Program Excellence. Dr. Abeer has significantly contributed to the field. She has authored over 20 publications in HTA and Health Economics research, covering areas such as decision analytical modeling, HTA processes and methods, valuation of health outcomes, and Health-Related Quality of Life research.
Dr. Abeer is influential at the national, regional, and international levels in advancing the implementation and development of HTA. She is an active member of numerous HTA steering committees. She is the chair of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) HTA round table for the Middle East and Africa (2024-2026). Currently, Dr. Abeer is appointed by the World Health Organization to serve as a member of the Technical Advisory Group on Pricing Policies for Medicines (TAG-PPM), providing evidence-based technical advice to improve affordable access to essential and priority medicines.
Dr. Abeer holds a BSc in Pharmacy, a Master of Business Administration (MBA) from Jordan University, and a Master of Science (MSc) in International Health Technology Assessment from the University of Sheffield, United Kingdom. She is a PMP-certified project manager and a final-year Ph.D. candidate at UMIT TIROL – University for Health Sciences and Technology, Hall i.T., Austria.
WHAT PREDICTIVE ACCURACY IS REQUIRED FOR AN ARTIFICIAL INTELLIGENCE TOOL TO BE COST-EFFECTIVE IN PREDICTING DIALYSIS NEED AMONG PATIENTS WITH CHRONIC KIDNEY DISEASE?
OBJECTIVES: Chronic Kidney Disease (CKD) impacts 11 to 13% of adults, and 50% of patients progressing to kidney failure initiate dialysis unexpectedly in hospital. Unplanned dialysis leads to worse clinical outcomes, lower quality of life, and substantially higher healthcare costs. An artificial intelligence (AI) tool has been developed to predict dialysis need at 6 and 12 months using routinely collected CKD clinic data. This study determined the minimum predictive accuracy required for the tool to be cost-effective.
METHODS: A state-transition Markov model was developed to estimate lifetime costs (2025 Canadian dollars) and quality-adjusted life-years (QALYs) for the AI tool compared with usual care from the Canadian public healthcare payer perspective. The model simulated adults with CKD transitioning among CKD without dialysis, pre-emptive kidney transplant, hemodialysis, peritoneal dialysis, unplanned dialysis, and kidney transplant health states over 8 years. AI tool accuracy, costs, and utilities were obtained from the published literature. Time-varying transition probabilities between health states were informed by real-world administrative data. Costs and QALYs were discounted by 1.5% annually. Threshold and probabilistic analyses were conducted.
RESULTS: At a sensitivity of 0.651 and specificity of 0.97, the AI tool resulted in 0.00055 additional QALYs and $26.95 per patient compared to usual care, resulting in an incremental cost-effectiveness ratio (ICER) of $51,964/QALY gained. Threshold analyses demonstrated substantial sensitivity of economic outcomes to predictive performance. Reducing sensitivity from 0.651 to 0.645 increased the ICER to CAD $187,305/QALY gained. Results were primarily driven by reductions in unplanned dialysis initiation and downstream costs and outcomes.
CONCLUSIONS: The economic value of AI-enabled dialysis prediction depends on maintaining high predictive performance, highlighting the importance of rigorous validation and ongoing monitoring of AI tools before large-scale implementation. These findings provide decision-makers with a quantitative performance threshold to inform adoption, reimbursement, and evaluation of AI technologies in CKD care.
COST-EFFECTIVENESS OF A PUBLICLY FUNDED ELECTIVE EGG FREEZING PROGRAM FOR WOMEN DELAYING CHILDBEARING IN GERMANY
OBJECTIVES: Women increasingly delay childbearing for financial, professional, or personal reasons, potentially limiting family-building opportunities. Elective egg freezing (EEF) allows oocyte cryopreservation at a reproductively optimal age, potentially improving future live birth (LB) outcomes. This analysis evaluated the cost-effectiveness of a public EEF program versus no EEF for women delaying childbearing, from a German healthcare perspective.
METHODS: A decision-tree model was developed comparing EEF versus no EEF. In the EEF arm, women (25-34 years) underwent one ovarian stimulation (OS) and oocyte retrieval cycle with oocytes cryopreserved for future use. In both arms, women attempted natural conception at 40 years; those without a LB proceeded to assisted reproductive technology (ART) at age 41. ART in the EEF arm included up to five embryo transfer cycles using thawed oocytes; ART in the no EEF arm included up to four OS cycles with up to five embryo transfers per cycle. Model inputs were derived from the Deutsches IVF-Register (D·I·R), the German statutory health insurance outpatient fee schedule (EBM/KBV), and other published and publicly available sources. Costs were discounted at 4% annually. Parameter uncertainty and modelling assumptions were assessed using probabilistic and deterministic sensitivity analyses and scenario testing.
RESULTS: EEF was associated with 64% more LBs versus no EEF (45 vs 27 per 100 women) driven by higher ART-related LBs (27 vs 9). Total discounted costs were higher with EEF (€6,203 vs €4,455 per woman), but costs per LB were lower (€13,875 vs €16,339). At a willingness-to-pay threshold of €30,000/LB, EEF was cost-effective with an incremental cost of €10,021 per additional LB. Results were robust in sensitivity and scenario analyses.
CONCLUSIONS: From the German healthcare perspective, EEF is a cost-effective strategy compared with no EEF for improving LBs outcomes among women delaying childbearing, supporting the establishment of a publicly funded EEF program in Germany.
COST-EFFECTIVENESS OF AI-POWERED PRE-SCREENING FOR GERMLINE BRCA TESTING IN HR+/HER2-NEGATIVE EARLY BREAST CANCER IN FRANCE
OBJECTIVES: Germline BRCA mutation (gBRCAm) testing identifies HR+/HER2-negative early breast cancer (eBC) patients eligible for adjuvant olaparib, but testing remains suboptimal due to low mutation prevalence, next-generation sequencing (NGS) costs, and limited genetic counselling capacity. We evaluated the cost-effectiveness of AI-powered histopathology pre-screening to identify patients at elevated gBRCAm risk before confirmatory NGS testing in France.
METHODS: A decision-tree model compared current standard of care with AI pre-screening over a lifetime horizon. Under standard care, NGS testing rates were 70% for patients at high personal mutation risk and 40% for those at low risk. With pre-screening, 65% of low-risk patients underwent AI analysis of H&E whole-slide images before NGS referral; high-risk patients and remaining low-risk patients followed standard care. AI sensitivity and specificity were 93% and 41%, respectively. Costs, quality-adjusted life-years (QALYs), and life-years were derived from a French health technology assessment partitioned survival model for olaparib. The population included 38,600 HR+/HER2-negative eBC patients, 80% classified as low risk. AI pre-screening and NGS costs were €150 and €882.90, respectively.
RESULTS: AI pre-screening identified 303 additional gBRCAm patients versus standard care (1,214 vs. 911). Incremental costs were €13.7 million, with 344 additional QALYs and 421 additional life-years gained. The incremental cost-effectiveness ratio was €39,900/QALY, and incremental net monetary benefit was €3.5 million at a €50,000/QALY willingness-to-pay threshold. Tests needed to identify one gBRCAm-positive patient decreased from 19.49 to 17.67.
CONCLUSIONS: AI-powered histopathology pre-screening before confirmatory gBRCA NGS testing was cost-effective in France and improved identification of patients eligible for adjuvant olaparib. By increasing diagnostic yield and reducing tests per positive case, this strategy may support more efficient use of NGS and genetic counselling resources while expanding access to precision oncology.
COST-EFFECTIVENESS OF VOLUME-BASED LOW-DOSE CT LUNG CANCER SCREENING IN A HIGH-RISK POPULATION IN MOROCCO: A COHORT SIMULATION INFORMED BY THE NELSON STUDY
OBJECTIVES: Lung cancer is a leading cause of cancer mortality in Morocco, where most cases are diagnosed at advanced stages due to the absence of a national screening programme. This study evaluated the cost-effectiveness of volume-based low-dose computed tomography (CT) lung cancer screening (LCS) versus no screening for in a high-risk population in Morocco.
METHODS: A cost-effectiveness analysis compared 17 annual rounds LCS with volume CT versus no screening in adults aged 50-74 with significant smoking history, from a Moroccan payer perspective over a lifetime horizon. A decision tree modelled NELSON’s study outcomes, while a state-transition Markov model simulated treatment costs and long-term survival per stage at diagnosis. Model inputs were informed by the Grand Casablanca Cancer Registry, National Reference Pricing (TNR), Moroccan epidemiological data, and 10 key opinion leaders interviews. A screening uptake rate of 30% yielded 292,568 participants. Costs and health outcomes were discounted at 3.5%. Deterministic, probabilistic, and scenario sensitivity analyses were performed.
RESULTS: LCS shifted 75% of diagnoses to stage I (versus 3% without screening), reducing stage IV diagnoses by 91%. Over a lifetime horizon, screening averted 7,083 premature lung cancer deaths, generated 342,505 additional quality-adjusted life years (QALYs) and 476,824 life-years gained. The incremental cost-effectiveness ratio (ICER) was MAD 18,189/QALY (USD 2,001/QALY), well below the estimated willingness-to-pay threshold (WTP) of MAD 108,898/QALY (3×GDP per capita). Probabilistic sensitivity analysis confirmed robustness, with 97% of simulations falling below the threshold. Findings remained cost-effective under conservative uptake assumptions (10% and 20%).
CONCLUSIONS: Annual volume-based low-dose CT LCS for high-risk adults maybe highly cost-effective in Morocco, with an estimated ICER far below the estimated national willingness-to-pay threshold. Policymakers can consider these findings as foundational economic evidence supporting implementation of a national LCS program to reduce lung cancer mortality through earlier detection.
Mixture Cure Models: Do They Create More Issues Than They are Trying to Solve?
Session Type: Workshop
Topics: Methodological & Statistical Research, Health Technology Assessment, Economic Evaluation
Level: Intermediate
PURPOSE: The objectives are to present theoretical and practical issues of applying mixture-cure models (MCMs) in health technology assessment (HTA) settings for interventions with curative potential (IwCP), present a case-study to help demonstrate these challenges and propose practical solutions.
DESCRIPTION: Extrapolation of time-to-event outcomes for IwCP requires careful consideration to ensure clinical plausibility of estimated long-term survival. MCMs are commonly used in these settings to explicitly model survival for the “cured” and “uncured” cohorts. Despite being commonly used across recent HTA submissions of IwCP, both theoretical and practical issues remain when leveraging MCMs for nested outcomes, such as PFS, PFS2 and OS, in oncology. Relative survival frameworks (RSFs), cure fraction linking, non-MCMs (nMCMs) and Bayesian hierarchical MCMs (BH-MCMs) have been proposed to address MCMs limitations.
Mr Conlon will summarise existing HTA guidelines on cure models and their importance to health economics and HTA (5 min.). Professor Baio will briefly describe MCM background and limitations that RSFs, cure fraction linking, nMCMs and BH-MCMS aim to solve (15 min.). Mrs Proskorovsky will present practical statistical and modelling challenges arisen from a case-study in first-line advanced ovarian cancer where MCMs, RSFs and cure fraction linking were explored (15 min.). Professor Owen will discuss a proposed preliminary framework for navigating these challenges, including key considerations and recommendations on best practices for HTA submissions from a NICE committee member and outline priorities for future methodological research (15 min.). Audience participation will involve real-time polling at the start and end of the session, to assess MCMs familiarity, key challenges experienced when using MCMs and what they would have done in the case-study presented, with time reserved at the end for a Q&A session (10 min.). This workshop will be valuable for researchers and professionals interested in statistical methods research and its application to economic modelling and HTA.
Moderator
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Seán Conlon, BSc, MSc, Other
United Kingdom
Speakers
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Gianluca Baio, PhD
University College London, London, United Kingdom
Gianluca is professor of Statistics and Health Economics in the Department of Statistical Science at University College London. He graduated in Statistics and Economics from the University of Florence, Italy and then completed a PhD program in Applied Statistics again at the University of Florence, after a period at the Program on the Pharmaceutical Industry at the MIT Sloan School of Management, Cambridge (USA). He worked as a research fellow and then Lecturer in the Department of Statistical Sciences at University College London, UK. His main interests are in Bayesian statistical modelling for cost effectiveness analysis and decision-making problems in the health systems, hierarchical/multilevel models and causal inference using the decision-theoretic approach. Gianluca leads the Statistics for Health Economic Evaluation research group within the department of Statistical Science, whose activity revolves around the development and application of Bayesian statistical methodology for health economic evaluation, eg, cost-effectiveness or cost-utility analysis. He also collaborates with the UK National Institute for Health and Care Excellence (NICE) as a scientific advisor on Health Technology Appraisal projects. He has developed and maintains several R packages, many specifically devoted to health economic evaluation, including BCEA, survHE and missingHE.
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Irina Proskorovsky
Thermo Fisher Scientific, Valencia, Spain
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Rhiannon K Owen, BSc, MSc, PhD
Swansea University, Swansea, United Kingdom
Joint Clinical Assessment (JCA) and Pan-European Solidarity-Based Reimbursement Model (PANSOL) - Is collaborative assessment meeting the needs of EU member states?
Session Type: Workshop
Topics: Health Policy & Regulatory, Health Service Delivery & Process of Care, Health Technology Assessment
Track: Access and Pricing
Level: Intermediate
PURPOSE: This workshop will examine how a pan-European solidarity-based reimbursement model (PANSOL) could complement EU HTA processes by translating evidence into joint access decisions for high-cost and orphan medicines. Using PANSOL as the working case, participants will test which elements of a shared reimbursement pathway are scientifically defensible, politically feasible and operationally acceptable across European health systems.
DESCRIPTION: Professor Lieven Annemans will chair the session and frame the access problem: persistent launch/reimbursement disparities, fragmented national pricing and reimbursement processes, and the boundary between Joint Clinical Assessment and national appraisal. Tanja Novakovic (Speaker 1; 10 min.) will define the equity and affordability challenge from a payer/HTA perspective, focusing on where current pathways create delays, duplication and unequal negotiating capacity. Krzysztof Landa (Speaker 2; 10 min.) will introduce PANSOL as a conceptual model: voluntary or enhanced-cooperation participation, light-touch EU-level HTA/appraisal, pooled GDP-proportional financing, centralised negotiation, horizon scanning and risk-sharing for high-cost and orphan medicines. Mark Parker (Speaker 3; 15 min.) will assess implementation requirements, including governance, selection criteria, value and affordability thresholds, evidence development, confidentiality, national competence, and safeguards against unintended incentives.
Moderator
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Lieven Annemans
Ghent University, GENT, Belgium
Speakers
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Mark Parker
Belgrade, Serbia
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Krzysztof M Landa, MD
Watch Health Care Foundation, Warszawa, Poland
MD, CEO of MedInvest Scanner, 27 years of experience in R&P, HTA and MA. He used to be the Deputy MoH responsible for reimbursement in Poland 2015-2017 and the Director of Drug Policy Dept. at the Central Office of NHF 2006-2007. The Chairman of the Health Committee of the Polish-Ukrainian Chamber of Commerce. Plus various other systemic roles.
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Tanja Novakovic
Beograd Vracar, Serbia
Artificial Intelligence for Evidence Generation and Synthesis
Session Type: Research Podiums
Artificial intelligence is rapidly transforming the way evidence is identified, synthesized, and interpreted in health economics and outcomes research. This session showcases innovative applications of AI to systematic literature reviews, evidence identification, and evidence synthesis workflows. Presentations will highlight methodological advances that improve efficiency, reproducibility, and transparency while maintaining scientific rigor. Together, these studies demonstrate how AI can support high-quality evidence generation for health technology assessment and healthcare decision-making.
MULTI-COUNTRY HEOR LANDSCAPE ASSESSMENT FOR MULTIPLE SCLEROSIS USING A MULTI-AGENT GENAI SYSTEM
OBJECTIVES: Generative AI can accelerate HEOR and market access tasks, but commonly used models often lack the accuracy, depth, and traceability required for rigorous evidence synthesis. We used a HEOR-specific multi-agent AI system, ValueGen.AI, to address these limitations. This study evaluated the system’s ability to autonomously generate a comprehensive, multi-country HEOR landscape assessment for multiple sclerosis (MS).
METHODS: We used a hierarchical “Deep Agent” architecture using the LangGraph framework to orchestrate over 1,000 specialized sub-agent invocations across three layers: 1) A Main Orchestrator decomposing queries and evaluating information sufficiency; 2) A Deep Agent Layer executing parallel retrieval; and 3) A Tool Layer utilizing the Model Context Protocol (via FastMCP) to interface with heterogeneous data sources (NICE, G-BA/IQWiG, ICER, INAHTA, PubMed, clinical trial registries, and web repositories). The platform was tasked to generate a landscape report for MS covering epidemiology; clinical management across all MS phenotypes (RRMS, SPMS, PPMS); outcomes evidence; economic evidence; payer and reimbursement environment; competitor and pipeline analysis; stakeholder mapping; and evidence gaps, spanning US, UK, Germany, France, Italy, Spain, and China. Content and references were verified via human review.
RESULTS: ValueGen.AI generated a 300-page, seven-country HEOR landscape assessment for MS in under 48 hours. The report synthesized over 380 verifiable references across 12 sections with 126 country-specific subsections. Key findings included cross-country prevalence estimates ranging from 2.2 per 100,000 (China) to 335 per 100,000 (Germany); over 15 approved DMTs with anti-CD20 antibodies dominating the approximately $16.1 billion global market; a late-stage pipeline centered on BTK inhibitors with fenebrutinib showing positive Phase 3 results; and progressive MS as the principal unmet need.
CONCLUSIONS: Multi-agent AI workflows can dramatically improve HEOR productivity by compressing months of work into days. This study demonstrates that structured AI architectures can perform complex, multi-country HEOR landscape assessments without compromising the scientific rigor and traceability essential for decision-making.
READY FOR PRIME TIME? EVALUATING LARGE LANGUAGE MODELS FOR AUTOMATED EXTRACTION OF COST-EFFECTIVENESS MODELS FROM HTA SUBMISSIONS
OBJECTIVES: Large language models (LLMs) can automate extraction of health economic data (HED) from published technology assessments (TAs). Previous evaluations (ChatGPT-4) demonstrated suboptimal performance despite evaluating multiple prompting strategies. This study assessed whether a reasoning-enabled LLM, combined with domain-specific extraction guidance, can support accurate and automated extraction of HED from TAs.
METHODS: A custom GPT agent based on ChatGPT-5.5 (Thinking-Light) was developed to extract structured HED exclusively from uploaded TA documents, without access to external sources. The agent was guided by sixteen detailed domain-specific knowledge files, covering both simple domains (e.g. population, model structure) and advanced domains (e.g. committee critiques, modelling assumptions). For each domain, the agent reported the extracted information, supporting text excerpt, and source location to facilitate subject matter expert (SME) verification. Nine TAs across NICE (UK), CADTH/CDA (Canada), and ICER (US) were reviewed and extracted using the custom GPT agent. Outputs were compared against an SME reference standard and scored using domain-specific Likert scales (four-points for simple domains, five-point for advanced domains). Automated extraction and scoring of nine additional TAs are ongoing.
RESULTS: Each TA was fully extracted within minutes, compared with 60-90 minutes of SME extraction. The GPT-agent extractions demonstrated excellent agreement to SME extractions across simple domains (all extractions rated as correct or partially correct). Across advanced domains, more than 85% of extractions were rated as Good or Excellent, with only the committee critiques (55.6% Good/Excellent; 44.4% Fair) and modelling assumptions (66.7% Good/Excellent; 33.3% Fair/Poor) domains showing lower performance. No hallucinated extractions were identified.
CONCLUSIONS: A reasoning-enabled GPT, guided by domain-specific knowledge, achieved high agreement with SME reference extractions across most domains, while substantially reducing extraction time. These findings demonstrate the feasibility of AI-assisted, traceable extraction workflows for HTA evidence synthesis, with human validation remaining essential, especially for advanced domains.
A HYBRID AI-ASSISTED SCREENING FRAMEWORK FOR SYSTEMATIC LITERATURE REVIEWS IN HER2-POSITIVE METASTATIC BREAST CANCER
OBJECTIVES: Recent position statements from health technology assessment (HTA) bodies, including NICE (UK) and CDA (Canada), emphasize responsible use of artificial intelligence (AI) for evidence generation, aided by appropriate human oversight. Building on the first NICE‑accepted AI‑assisted systematic literature review (SLR), this study utilizes a hybrid AI-human framework to identify outcomes beyond those conventionally predefined in treatment‑ or outcome‑focused SLRs.
METHODS: A literature search was conducted across EMBASE®, MEDLINE®, CENTRAL, and the Cochrane Database of Systematic Reviews (CDSR) from January 2019 to December 2025. Using the AI‑assisted SLR methodology described by Makhija et al., the Pharmacoevidence AI‑SLR tool was applied to an oncology use case in human epidermal growth factor receptor 2 (HER2)-positive metastatic breast cancer (mBC). Outcomes included: trastuzumab deruxtecan (T-DXd) discontinuation, efficacy, safety, tolerability, and subsequent therapy patterns post T-DXd. The AI system served as an independent second reviewer for screening and data extraction.
RESULTS: A total of 2,082 citations were screened using the hybrid framework. A pilot sample of 100 citations was screened to optimize prompts. Title and abstract screening performance was high (accuracy: 96.5%, precision: 87.8%). An accuracy of ~98% was attained for both full-text screening and data extraction of study outcomes. Overall, the hybrid approach markedly improved efficiency, delivering twice the speed of traditional human review while maintaining accuracy above the ideal 95% benchmark, compared with less than 90% for conventional review. These gains translated into an estimated 50% reduction in operational costs and time.
CONCLUSIONS: This study demonstrates that AI‑assisted SLR with a human-in-the-loop, can be efficiently integrated into SLR workflows. Unlike traditional SLRs, which typically focus on a single treatment, this study examined a broader range of therapies used post T-DXd. Agreement rates across first- and second-stage screening outperformed the traditional two-human review (reaching the near-ideal 95% benchmark), with significant efficiency gains and scalability for future HTA submissions.
AI DELPHI: A FRAMEWORK FOR BENCHMARKING THE WRITING QUALITY OF LARGE LANGUAGE MODELS (LLMS) IN HEALTH ECONOMICS AND OUTCOMES RESEARCH (HEOR) SETTINGS, A CANADIAN PROOF-OF-CONCEPT
OBJECTIVES: To develop and validate AI Delphi, a reproducible multi-criteria framework for evaluating AI performance in HEOR, and compare ten LLMs across five providers using Canadian HTA documentation.
METHODS: AI Delphi uses four blinded AI judges (Claude Opus 4.8+RAG, GPT 5.5+RAG, Gemini 3.1 Pro+RAG, Claude Sonnet 4.6+RAG) scoring outputs across nine criteria: page/token budget (5%), writing quality and HEOR/payer style (25%), market access/payer insights, economic evaluation, clinical evaluation, comparative effectiveness, disease burden/unmet need, missing key information, and hallucination (10% each). To reduce bias, self-preferential scores were excluded. Convergence was assessed iteratively: scores <5% were averaged; divergent criteria re-evaluated with cross-judge information; unresolved discordance underwent senior HEOR adjudication. Ten LLMs generated a formatted abstract and four-page payer-centric summary of a Canadian HTA report for sacituzumab govitecan in HR+/HER2− metastatic breast cancer post-endocrine therapy and at least two metastatic systemic therapies. Models included four Anthropic, two OpenAI, two Google, Kimi K2.6, and DeepSeek V4 Pro. Each was tested with and without HEOR-specific hybrid RAG anchored by a human-authored, Canadian Grounding Codex.
RESULTS: Anthropic and OpenAI achieved the highest provider-level scores (both 75.5/100), followed by DeepSeek/Kimi combined (69.2), then Google (66.5). Top-performing configurations were Claude Opus 4.8+RAG (79.1) and GPT 5.5+RAG (78.5). RAG improved high-reasoning models but yielded inconsistent or negligible gains for others. Writing quality varied, with Google models producing bullet-point outputs incompatible with deliverable standards. Missing key information was most heterogeneous, while hallucination rates were low. Delphi convergence occurred in Round 1 for 30.8%, Round 2 for 61.1%, and required human adjudication in 8.1%.
CONCLUSIONS: AI Delphi provides a reproducible framework to evaluate LLM performance in HEOR. High-reasoning models with domain-specific RAG generated the strongest payer-centric outputs, however RAG benefits were model-dependent. Formatting gaps demonstrate that HEOR AI evaluation must assess both structure and content. Future work should expand testing across HTA bodies and indications.
Whole Health at risk: Does EU Joint Clinical Assessment conflict with Europe’s Antimicrobial Resistance ambitions?
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment
Track: Expanded Value Measures
Level: Introductory
The Joint Clinical Assessment (JCA), introduced under the EU HTA Regulation, focuses on patient level efficacy and safety of new health technologies. This debate will explore whether this narrow focus is at odds with the concept of Whole Health, particularly with the backdrop of EU Antimicrobial Resistance (AMR) priorities. The current JCA assessment necessarily has a strictly defined focus, but does this mean patient and broader population level benefits will be inadequately captured, particularly for antimicrobials? Does the tight framework risk a focus on inappropriate comparators and outcomes for some types of technologies? Does the existing approach to JCA run counter to other EU priorities, such as AMR and the General Pharmaceutical Legislation. As the first JCAs are finalised, the panel will explore the uncertainties in both the willingness and capacity for methodological evolution. The debate becomes more consequential as the scope of JCA expands to all health technologies by 2030.
This panel will examine whether JCA could hinder rather than support the achievement of Whole Health in Europe, particularly in the context of AMR. The session brings together perspectives from EU HTA bodies, industry and the HEOR community to explore limitations and opportunities for JCA evolution. The moderator will introduce the topic and policy context (6 minutes). Panellist 1 will outline how JCA is expected to evolve and if this can address concerns expressed regarding the existing framework (8 minutes). Panellist 2 will highlight emerging challenges for industry and contextualises it with wider AMR policies at EU level (8 minutes). Panellist 3 will assess JCA limitations from an HEOR perspective and propose recommendations for methodological reform (8 minutes). Interactive audience engagement (incl. polling) will be done throughout the session (5 min). The session will conclude with audience Q&A (25 min). This session will be highly relevant for policymakers, HTA bodies, regulators, industry leaders, HEOR researchers and patient representatives seeking to understand whether JCA can fully support whole health ambitions and how it may need to evolve to do so.
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Moderator
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Grace Hampson
Office of Health Economics, London, United Kingdom
Speakers
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Anja E Schiel, PhD
Norwegian Medicines Agency, Oslo, Norway
Anja Schiel has studied Biology at the Johannes Gutenberg-University, Mainz, Germany. She received her PhD from the Free University in Amsterdam in 2006 and worked several years as Post-Doc on a range of subjects focusing on oncology, immunology and molecular biology, first at the University of Leiden and later at the University of Oslo, before starting at the Nor-wegian Medical Products Agency (NOMA) in 2012. At NOMA she is working as Special Adviser/Statistician/Methodologist both on regulatory and HTA projects. She has been Chair of EMA’s Biostatistics Working Party (2017 – 2019) and dur-ing 2019 – 2022 she was Chair of EMA’s Scientific Advice Working Party (SAWP). She contin-ues currently as alternate member at the SAWP and is member of the recently established Methodology Working Party (MWP) at EMA. She has been involved in EUnetHTA JA3 and its successor, EUnetHTA 21, with particular focus on parallel EMA-HTA scientific advice (joined scientific consultations). As one of the vice Chairs of the JSC Committee for Scientific Consistency and Quality (JSC CSCQ) she was in-volved in the preparation of the implementation of developer support under the Regulation on Health Technology Assessment (HTAR, to apply from January 12th 2025). Until January 2025 she was a member of the HTA coordination groups (HTACG) Joined Scientific consultation sub-group (JSC SG) and the Methodological and Procedural sub-group (MPG SG ).
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Libby Hamson
GSK, London, United Kingdom
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Patrick K Hopkinson, MBA
Independent Consultant, United Kingdom
Beyond “Using RWE”: What It Takes to Make Real-World Evidence Decision-Grade for HTA?
Session Type: Workshop
Topics: Health Technology Assessment, Real World Data & Information Systems, Economic Evaluation
Track: Real-World Evidence (RWE)
Level: Intermediate
Purpose:
Real-world evidence (RWE) is increasingly discussed in health technology assessment (HTA), economic evaluation, managed access, and reassessment. However, the presence of real-world data (RWD) in an evidence package does not necessarily make the evidence decision ready. This workshop will examine how HTA systems can judge whether RWE is fit for purpose for specific decision problems, including initial reimbursement, managed access, and reassessment.
Description:
HTA systems are moving beyond whether RWE can be used, towards when, how, and for what purpose it should inform decisions. Guidance from NICE and Canada’s CDA-AMC emphasises data relevance, quality, transparent reporting, appropriate methods, and alignment between evidence and the decision problem. In China, RWE is also developing rapidly through regulatory science initiatives and growing policy interest in pharmacoeconomic and reimbursement decisions.
This workshop will focus on decision-ready RWE across jurisdictions. Tina Wang will open with an international HTA systems perspective on how RWE is used to manage uncertainty across initial assessment, managed access, post-launch evidence generation. Huajie Jin will provide a methodological perspective on RWE in economic evaluation, focusing on when RWD can support parameter estimation, model structure, care pathways, resource use, costs, and uncertainty analysis. Recent academic work on schizophrenia economic evaluations will illustrate the gap between available RWD and decision-ready evidence. Jing Wu will discuss China’s evolving policy environment for RWE, pharmacoeconomic evaluation, reimbursement decision-making, and the challenges of using RWE in a rapidly developing HTA system.
Participants will review a short hypothetical RWE-informed economic evaluation case and vote on whether different RWE components should be used as core evidence, supportive evidence, scenario analysis, or rejected as insufficiently decision-ready. The workshop will conclude with practical appraisal questions for judging whether RWE is fit for purpose in HTA.
Moderator
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Mark Sculpher, PhD
University of York, York, United Kingdom
Speakers
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Tina Wang, PhD
CIRS, London, United Kingdom
Tina Wang is the Director of the HTA Programme and Strategic Partnerships at the Centre for Innovation in Regulatory Science (CIRS), an international think tank dedicated to advancing regulatory and HTA policies and practices worldwide.
Tina leads CIRS’ global work on HTA strategy and research, driving initiatives that inform evidence-based policy development and foster alignment across regulatory and HTA systems. She oversees the conceptualisation, design, and delivery of HTA-related projects, ensuring that CIRS’ research agenda reflects the evolving needs of the global HTA landscape.
With extensive experience working in complex, multi-stakeholder environments, Tina builds and manages strategic partnerships with regulatory agencies, HTA bodies, industry, academia, and patient groups. Her work focuses on facilitating policy dialogue, advancing best practices, and strengthening global collaboration to support efficient, transparent, and patient-centred HTA decision making.
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Huajie Jin, BSc, MSc, PhD
King's College London, London, United Kingdom
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Jing Wu, RPh, PhD
Tianjin University, Beijing, China
Prof. Wu has dedicated nearly 20 years to research and teaching in health economics and pharmacoeconomics. Her expertise spans health policy evaluation, pharmacoeconomic evaluation, real-world data analysis, and health-related quality of life and health utility study. She earned her PhD in Pharmacoeconomics through a joint program between Shenyang Pharmaceutical University and Peking University in 2007 and was a visiting scholar at the Schaeffer Center for Health Policy and Economics at the University of Southern California in 2013. Prof. Wu has authored over 100 high-impact scientific articles, led three National Natural Science Foundation of China projects, contributed to multiple pharmacoeconomics textbooks, and spearheaded the development of the China Guidelines for Pharmacoeconomic Evaluations (2020) and the Companion Guide to the China Guidelines for Pharmacoeconomic Evaluations (2022), promoting the development and standardization of pharmacoeconomics in China. She currently serves as Chair of the Pharmacoeconomics Committee of the Chinese Pharmaceutical Society, Vice Chair of the Pharmacoeconomics Committee of the China Health Insurance Research Association, Vice Chair of the Pharmacoeconomics Committee of the Chinese Society of Research Hospitals. She co-chaired the ISPOR Asia-Pacific Conferences in 2018 and 2022, served as Executive Chair of the 10th Huaxia Pharmacoeconomics and HTA Forum in 2019, and founded the China Young Scholars Forum on Pharmacoeconomics in 2017—organizing around 40 high-level academic meetings to date. As a national-level advisor, she provides strategic guidance to China’s National Healthcare Security Administration, playing a key role in reimbursement policy and healthcare-system reform.
The Cost of Waiting: Governing Agentic AI When Guidance Lags the Field
Session Type: Issue Panel
Topics: Organizational Practices, Health Policy & Regulatory, Real World Data & Information Systems
Track: AI
Level: Intermediate
Issue:
AI governance in evidence synthesis usually focuses on what could go wrong if teams adopt AI too quickly. That caution is necessary, but incomplete. The field also faces a guidance gap: evidence teams are already experimenting with AI, while formal recommendations often lag behind current workflows using retrieval, agents, audit trails, local validation sets, and structured human adjudication. Without practical internal and community-developed frameworks, the risk is not simply slower adoption. It is inconsistent, poorly documented, or inappropriate AI use spreading before the field has agreed how to govern it. HEOR, medical information, and market-access teams still face pressure for faster, more current evidence, especially under JCA and other timelines. The debate is therefore not acceleration versus caution. It is how organizations can use controlled, risk-tiered AI governance to prepare for formal guidance, reduce poor AI use, and keep evidence workflows transparent, auditable, and reviewable.
Overview:
This panel will examine the cost of waiting for formal AI guidance and the governance models organizations can use while the field catches up. The discussion will focus on practical decision-making: which evidence tasks can move now, which require stronger controls, and what documentation makes AI-supported work defensible to medical, HTA, regulatory, and market-access reviewers. The panel combines perspectives from a technology builder, pharma medical information, evidence-production operations, and an HTA-methods moderator. The aim is to help attendees distinguish unmanaged adoption from controlled, risk-tiered use of AI in evidence workflows.
Moderator
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Raphael Sonabend-Friend
NICE, London, United Kingdom
Dr Raphael Sonabend-Friend is a Scientific Adviser in the Science, Evidence and Analytics team at the National Institute for Health and Care Excellence (NICE). Raphael supports the testing of artificial intelligence, particularly focused on generative artificial intelligence methods. Raphael holds a PhD in the accessible and transparent use of machine learning in healthcare settings. Raphael has over a decade of experience in the healthcare sector ranging from large philanthropies, small local charities, governmental bodies, and private sector consulting for UK and international organisations.
Speakers
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Amir Saeidmehr
Zenbase AI, San Francisco, CA, United States
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Thomas Paulsson, BSc, MSc, PhD
Recordati Rare Diseases, LONDON, United Kingdom
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Amanda Hansson Hedblom
Valid Insight (Bioscript Group), London, EC1R 3AW, United Kingdom
Integrating Environmental Sustainability into HTA: Global Policy Archetypes and Future Directions for Access Decision-Making
Session Type: Workshop
Topics: Health Technology Assessment, Health Policy & Regulatory
Track: Expanded Value Measures
Level: Introductory
Purpose: This workshop will examine emerging international approaches for incorporating environmental sustainability (ES) into health technology assessment (HTA) and explore how these approaches may influence future healthcare access, pricing, and value assessment decisions. Attendees will gain practical insight into the policy rationale, methodological challenges, and trade-offs associated with integrating ES considerations into HTA frameworks.
Description:
As climate change increasingly affects population health and healthcare system resilience, HTA agencies are beginning to explore whether ES should be incorporated into healthcare decision-making. This debate aligns with the growing focus on expanded value assessment and shaping future access through science and policy. However, there is currently limited consensus regarding the role, methodology, and evidentiary standards for including ES within HTA processes.
The workshop moderator will introduce the session (3 mins) by outlining the growing relevance of ES within HTA and framing the discussion in the context of evolving global access and value assessment priorities.
Speaker one will present the industry perspective on why sustainability integration is becoming an important global policy issue and discuss potential implications for innovation and market access.
Speaker two will present findings from international landscaping research and introduce a four-part HTA archetype framework: “green pioneers,” “clinical arbiters,” “industrial strategists,” and “price-dominant/fragmented” systems.
Speaker three will present case studies demonstrating the methods and policy approaches currently being explored across representative countries within each archetype.
Following on from the presentations, the workshop moderator will facilitate real-time audience polling on preferred approaches for integrating ES into HTA. Followed by an interactive classification exercise where attendees assign countries/agencies to HTA sustainability archetypes.
Finally, a live Q&A session with panellists will spotlight feasibility and implementation considerations across jurisdictions.
Moderator
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Anke-Peggy Holtorf, MBA, PhD
Health Outcomes Strategies, GmbH, Basel, Switzerland
Speakers
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Sandro Gsteiger, PhD
Roche, Basel, Switzerland
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Fiona Adshead, MSc
Sustainable Healthcare Coalition, London, United Kingdom
Fiona chairs the Sustainable Healthcare Coalition that inspires partnerships and action on sustainable healthcare and is an independent expert advisor and board member for national and global organisations on wellbeing and sustainability.
A high-profile public health leader with a track record of reframing thinking and developing innovative strategy and programmes at the heart of business and government, her previous roles include Deputy Chief Medical Officer and Director General in the UK Government, Director of Chronic Disease and Health Promotion at the World Health Organisation and Bupa’s Chief Wellbeing Officer.
Fiona is a visiting Professor at UCL and is a Senior Associate at Cambridge Institute for Sustainability Leadership.
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Stuart Mealing, MSc
York Health Economics Consortium, York, United Kingdom
EU HTA system readiness for Orphan Medicinal Products ahead of 13 January 2028
Session Type: Issue Panel
Topics: Health Technology Assessment, Methodological & Statistical Research, Clinical Outcomes
Track: Access and Pricing
Level: Intermediate
Issue:
Orphan Medicinal Products will become subject to mandatory Joint Clinical Assessments, from 13 January 2028 and we only have a few months to prepare the system to accommodate OMPs, that often have a different trial design.
The Regulation (EU) 2021/2282 on health technology assessment recognises the ethical and practical challenges inherent to study design and evidence generation in the area of rare diseases – however, the current methodological guidance documents do not explicitly account for the specificities of OMPs.
Overview:
The panel will explore the rare disease companies’ requirement for clarity ahead of entering the JCA in 2028, and the readiness of the EU HTA as it relates to OMPs, covering inter alia the methodological guidance, interactions and advice opportunities and expert involvement in rare and ultra-rare diseases.
The panel will also reflect on the challenge for smaller companies that often specialise in rare diseases in navigating the JCA.
During the panel speakers representing rare disease companies, the EU HTA Coordination Group and patients will discuss the readiness of the EU HTA system for OMPs, and the implications for national access decisions, that will be based in part on the JCA report.
Moderator
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Alexander Natz
EUCOPE, Brussels, Belgium
Alexander Natz is the Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) in Brussels and advises innovative pharmaceutical and biotech companies, including start-ups, in regulatory and pricing & reimbursement matters from the EU law and German law perspective.
From 2008 to 2013, he was Head of the Brussels Office of Bundesverband der Pharmazeutischen Industrie e.V. (BPI). Before, he worked as a lawyer at Sträter Law Firm in Germany with a special focus on managed entry agreements and licensing of pharmaceuticals. Dr. Natz has also worked in the field of competition law with the European Commission and in the pharmaceutical industry.
Speakers
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Pierrick Rollet
Rare Diseases, Alexion, Rixensart, Ireland
Currently Vice President Global Head Value-Access & Pricing at Alexion Astra Zeneca Rare Diseases, Pierrick is a life science C level accomplished executive with over 25 years of international experience across senior general management roles at global, region and local level leading global commercial/market access strategy, business local-regional operations, global franchise development programs and global Business Development.
With a large experience across 4 continents and speaking 5 languages, Pierrick is an agile international enterprise leader with integrated strategic thinking built upon an end-to-end understanding of leading and developing strategy & operations at global, region and local levels within both established and growth stage commercial biopharmaceutical companies.
As Rare Diseases global executive committee team member, international Board director (JV board member, JV CEO in Japan) or Vice President global business development, Pierrick has a proven track record in structuring strategic access partnerships while establishing-nurturing collaborative relationships with governments, scientific community and the external community at large.
Pierrick holds an MBA from IMD Lausanne in Switzerland, a MA in international economy from Paris Dauphine University & a BA in Japanese language from Paris Sorbonne Oriental Languages University
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Francois Houyez
European Organisation for Rare Diseases (Eurordis), Paris, France
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Niklas Hedberg, MSc
TLV, Stockholm, Sweden
Niklas Hedberg is the Chief Pharmacist at the Dental and Pharmaceutical Benefits Agency (TLV) in Sweden.
Niklas is the HTAR Coordination Group co-chair for medicinal products. He was the Chair of the Executive Board for EUnetHTA21 (2021-2023) and for EUnetHTA JA 3 (2018- 2021).
Niklas has been working with HTA and Pricing and Reimbursement since 2001.
ICH E22 – General Considerations for Patient Preference Studies: Are We Ready to Move from Guidance to Practice?
Session Type: Issue Panel
Topics: Patient-Centered Research, Health Policy & Regulatory
Track: Patient-Centered Evidence
Level: Introductory
ICH issued a draft guideline on General Considerations for Patient Preference Studies (E22) for public consultation in November 2025 with the final guideline expected in 2027. The purpose of the guideline is to “outline[s] general considerations about the use, design, conduct, analysis, and submission of PPS aimed at informing drug development, regulatory submission and evaluation, drug approvals and maintenance of such approvals”. This statement implies that regulatory bodies will consider PPE in regulatory decisions related to drugs. However, despite years of discussion related to the use of PPE to inform regulatory decisions, there are few concrete examples of drug-product decisions in which regulators acknowledged the role PPE played in the decision.
The session will first introduce potential uses of PPE to inform regulatory and development decisions. According to the draft guideline, these include everything from understanding patients’ willingness to participate in clinical trials to informing selection of clinical trial endpoints, through quantitative benefit-risk assessment (Hauber – 10 minutes). The session will then review the key examples of regulatory decisions in which PPE was publicly acknowledged by regulators as being considered in decision making (Berlin – 10 minutes). The session will then present a regulatory view of the potential for PPE to inform regulatory decisions (Pignatti – 10 minutes). The final panelist (Gonzalez – 10 minutes) will describe potential methodological and procedural challenges to implementing the ICH E22 guideline which may result in developing standards for high quality research with uncertain impacts on regulatory decision-making. Panelists will then discuss the potential future of PPE in regulatory decision making.
Moderator
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Brett Hauber, PhD
Pfizer, New York, NY, United States
Speakers
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Conny Berlin, MSc
Basel, Switzerland
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Francesco Pignatti, MD
European Medicines Agency, Amsterdam, Netherlands
Francesco Pignatti graduated as Medical Doctor at the University of Rome. In 1995 he became Research Fellow at the EORTC Data Center in Brussels, Belgium. In 1997 he obtained a Master of Science degree in Biostatistics from the University of Limburg, Belgium. In 1999 he joined the European Medicines Agency (EMA), which is located in Amsterdam since 2019. From 2009 to 2023 he was appointed Head of the Office of Oncology and Haematology, he is currently Scientific Adviser for Oncology. His main regulatory science interests include cancer drug regulation, benefit-risk analysis, and stated preference studies.
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Juan M Gonzalez, PhD
Duke Clinical Research Institute, Cary, NC, United States
18:00 - 19:00
Welcome Reception
Session Type: General Meeting
Women in HEOR Reception
Session Type: General Meeting
Tue 10 Nov
7:00 - 8:30
Morning Coffee Service
Session Type: General Meeting
Don't miss the start of the day with the Plenary Session. Enjoy your morning coffee as you listen to dynamic presentations intended to inspire and empower.
7:00 - 17:00
Registration Hours
Session Type: General Meeting
8:30 - 10:00
Plenary Session 2: We Optimize Treatments—But How Do We Optimize the System?
Session Type: Plenary
Topics: Health Policy & Regulatory, Health Service Delivery & Process of Care, Health Technology Assessment
Track: Expanded Value Measures
Level: Intermediate
Building on the opening plenary’s exploration of what value means, this session shifts the focus to where value is created across healthcare systems. HEOR has developed sophisticated methods for evaluating individual health technologies, yet existing practices, care pathways, and delivery models that may provide limited value are often subject to far less scrutiny.
Through brief expert perspectives and an interactive moderated discussion, the session will examine how HEOR can evolve from product-centric assessment toward system-level value creation. Speakers will explore the barriers to more holistic resource allocation—including system inertia, political will, professional resistance, and methodological limitations—and highlight opportunities to identify low-value activities, optimize care delivery, and redirect resources toward innovations that improve population health and system performance.
Moderator
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Rasmus Gjesing, Masters
Copenhagen, Denmark
Plenary Session 2: Welcome, Presidential Address, and Keynote Speaker
Session Type: Plenary
Begin day two of ISPOR Europe 2026 with inspiring insights from ISPOR’s President Beth Devine and our Keynote speaker Ricardo Baptista Leite, CEO of NOVA Center for Global Health.
Immediately following the opening presentations, the scientific plenary panel will take the stage.
Speakers
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Beth Devine, MBA, MSc, PharmD, PhD
University of Washington, Seattle, WA, United States
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Ricardo Baptista Leite, M.D.
Geneva, Switzerland
9:30 - 19:00
Exhibit Hall Hours
Session Type: General Meeting
10:30 - 11:30
Bridging China and Europe: When Is China-Generated Evidence Fit for European HTA?
Session Type: Spotlight
Topics: Health Technology Assessment, Real World Data & Information Systems, Health Policy & Regulatory
Track: Real-World Evidence (RWE)
Level: Intermediate
ISSUE:As China’s biopharmaceutical ecosystem matures, China-generated clinical trial and real-world evidence are increasingly used to inform global development and access strategies. The key debate is no longer whether such evidence is credible, but whether it is sufficiently relevant, transferable, and decision-ready for European HTA and payer contexts. Clinical trial evidence may be more transferable when design, endpoints, comparators, populations, and execution quality align with international expectations. Real-world evidence is more context-dependent, with challenges around data provenance, coding systems, clinical pathways, standards of care, patient representativeness, and local resource use. The central question for Europe is whether China-generated evidence can serve as core evidence in HTA, or should remain supportive unless transferability is explicitly demonstrated.
OVERVIEW:This issue panel will bring together China HTA, European academic HTA, and European industry perspectives. Jing Wu will discuss how China’s HTA, pharmacoeconomic, and RWE landscape is evolving, and what this means for global evidence generation. Huajie Jin will examine transferability, uncertainty, and how China-generated evidence may be incorporated into European reimbursement and economic evaluation. Ross Selby will discuss evidence planning across markets, access strategy, and post-launch evidence generation from an industry perspective. The session will focus on practical methodological and policy principles for integrating China and European evidence streams in ways that are scientifically credible, HTA-relevant, and supportive of timely patient access. The moderator will use short audience polling questions to stimulate debate, including whether China-generated clinical trial evidence can ever serve as core evidence in European HTA, and whether China-generated RWE should be used as core evidence, supportive evidence, or scenario analysis only.
Moderator
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Lung-I Cheng, PhD
AESARA, Somerville, MA, United States
Global market access and value executive with 15+ years of experience across oncology, cell and gene therapy, and rare diseases. Led pricing, HTA, reimbursement, and evidence strategies across the US, Europe, China, and emerging markets, spanning pipeline through launch and lifecycle. Experience includes leadership roles at Takeda, Amgen, and Cencora, with a strong track record in cross-functional alignment, global access strategy, and translating evidence into decisions that expand patient access and portfolio value.
Speakers
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Jing Wu, RPh, PhD
Tianjin University, Beijing, China
Prof. Wu has dedicated nearly 20 years to research and teaching in health economics and pharmacoeconomics. Her expertise spans health policy evaluation, pharmacoeconomic evaluation, real-world data analysis, and health-related quality of life and health utility study. She earned her PhD in Pharmacoeconomics through a joint program between Shenyang Pharmaceutical University and Peking University in 2007 and was a visiting scholar at the Schaeffer Center for Health Policy and Economics at the University of Southern California in 2013. Prof. Wu has authored over 100 high-impact scientific articles, led three National Natural Science Foundation of China projects, contributed to multiple pharmacoeconomics textbooks, and spearheaded the development of the China Guidelines for Pharmacoeconomic Evaluations (2020) and the Companion Guide to the China Guidelines for Pharmacoeconomic Evaluations (2022), promoting the development and standardization of pharmacoeconomics in China. She currently serves as Chair of the Pharmacoeconomics Committee of the Chinese Pharmaceutical Society, Vice Chair of the Pharmacoeconomics Committee of the China Health Insurance Research Association, Vice Chair of the Pharmacoeconomics Committee of the Chinese Society of Research Hospitals. She co-chaired the ISPOR Asia-Pacific Conferences in 2018 and 2022, served as Executive Chair of the 10th Huaxia Pharmacoeconomics and HTA Forum in 2019, and founded the China Young Scholars Forum on Pharmacoeconomics in 2017—organizing around 40 high-level academic meetings to date. As a national-level advisor, she provides strategic guidance to China’s National Healthcare Security Administration, playing a key role in reimbursement policy and healthcare-system reform.
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Huajie Jin
United Kingdom
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Ross A Selby, BSc, MSc, PharmD
Italfarmaco SpA, United Kingdom
Generating Decision-Ready Evidence for HTA and JCA
Session Type: Research Podiums
As HTA and Joint Clinical Assessment (JCA) requirements continue to evolve, decision-makers increasingly rely on evidence beyond direct head-to-head clinical trials. This session showcases the application of matching-adjusted indirect comparisons (MAICs), network meta-analysis (NMA), real-world evidence (RWE), and surrogate endpoint validation to address evidence gaps and support robust comparative effectiveness assessments. Presentations highlight methodological innovations and practical approaches for generating decision-ready evidence in areas where traditional clinical trial data alone may be insufficient.
MATCHING-ADJUSTED INDIRECT COMPARISON OF ETRANACOGENE DEZAPARVOVEC VERSUS MARSTACIMAB FOR THE TREATMENT OF SEVERE HEMOPHILIA B
OBJECTIVES: To compare the efficacy of a single dose of etranacogene dezaparvovec (EDZ) versus routine prophylaxis with marstacimab in the treatment of adults with severe hemophilia B (HB), using matching-adjusted indirect comparisons (MAIC).
METHODS: Unanchored MAICs compared EDZ individual patient data (IPD) from HOPE-B trial and publicly available summary-level data (SLD) from BASIS trial for subjects with severe HB without inhibitors who received prior factor IX (FIX) prophylaxis and received routine prophylaxis with marstacimab. Due to lack of HB subgroup data in the primary publication of BASIS, publicly available SLD was obtained from the German Gemeinsamer Bundesausschuss (G-BA) dossier, French Haute Autorité de Santé (HAS) decision document, and conference materials. Patients from HOPE-B were matched to BASIS by excluding those who would not be eligible for or enrolled in BASIS. Then, HOPE-B patients were weighted to align with BASIS on age, BMI, and geographic region. The following outcomes were assessed: mean annualized bleeding rate (ABR), mean annualized spontaneous bleeding rate (AsBR), mean annualized joint bleeding rate (AjBR), proportion of patients without bleeds, and annualized FIX consumption. Bleeding outcomes were based on treated bleeds.
RESULTS: After adjustment for above factors, EDZ was associated with statistically significantly lower mean ABR using G-BA (rate ratio [RR]: 0.06 [95% confidence interval (CI): 0.01, 0.43]) and HAS data (RR: 0.06 [95% CI: 0.06, 0.42]), AsBR using G-BA data (RR: 0.06 [95% CI: 0.01, 0.56]), AjBR using G-BA data (RR: 0.06 [95% CI: 0.01, 0.54]), along with statistically significantly higher proportion of patients without bleeds using G-BA (odds ratio [OR]: 8.67 [95% CI: 1.36, 55.41]) and HAS data (OR: 10.84 [95% CI: 1.67, 70.26]), and statistically significantly lower mean FIX consumption using conference materials (difference: -285.75 IU/kg/year [95% CI: -442.99, -128.52]).
CONCLUSIONS: MAICs suggest favorable efficacy for EDZ versus prophylaxis with marstacimab based on available data.
OPERATIONALISING JCA REQUIREMENTS FOR INDIRECT TREATMENT COMPARISONS: AN UPDATED SLR-BASED FRAMEWORK FOR IDENTIFYING TREATMENT EFFECT MODIFIERS AND PROGNOSTIC VARIABLES
OBJECTIVES: The EU Joint Clinical Assessment (JCA) requires a systematic approach to identify treatment effect modifiers (TEMs) and prognostic variables (PVs) for indirect treatment comparisons (ITCs) but provides limited operational guidance on how this should be implemented. Following presentation of our TEMs/PV identification framework at ISPOR Europe 2025, updated JCA methodological guidance and JCA assessments published to date have provided further insight into assessor expectations. This study updates the framework with a particular focus on systematic literature review (SLR) identification and extraction.
METHODS: The original five-step TEMs/PVs identification framework was reviewed against emerging evidence sources including updated methodological JCA guidance (May 2026), JCA reports and appendices published to date (e.g., tovorafenib, April 2026), and assessor - developer clarification exchanges. Evidence sources were reviewed to identify new expectations regarding TEM/PV identification, evidence requirements and reporting. Findings were mapped to the existing framework and used to refine framework components and develop a standardised variable-level extraction template.
RESULTS: Updated JCA guidance and reporting confirm that an SLR is now expected for TEMs/PV identification prior to an ITC, with an existing review accepted only if recent and considered to be of adequate quality covering the full decision-problem. The completeness of the candidate set of TEMs/PVs in the submission and the clinical rationale for inclusion/exclusion were scrutinized. We converted the extraction step of the framework into a standardised template capturing, per variable: operational definition, outcome and PICO segment; source and analysis type with sample sizes; effect-modification evidence (interaction p-value, within- vs across-subgroup direction, confidence intervals, magnitude, pre-specification); prognostic evidence (uni-/multivariable analyses, adjustment); credibility (plausibility, consistency, risk of bias).
CONCLUSIONS: Early JCA experience signals a clear expectation for rigorous, SLR-based identification of TEMs and PVs, with a transparently documented extraction process. The updated framework presented offers practical, actionable guidance including a standardized extraction template for developers preparing ITCs for JCA.
EXPLORING THE RELATIONSHIP BETWEEN SURROGATE ENDPOINTS AND CLINICAL OUTCOMES IN SYSTEMIC SCLEROSIS-ASSOCIATED INTERSTITIAL LUNG DISEASE (SSC-ILD): A SYSTEMATIC LITERATURE REVIEW
OBJECTIVES: SSc-ILD is a leading cause of mortality in systemic sclerosis, but mortality is rarely used as a primary trial endpoint due to the long follow-up required. Consequently, forced vital capacity (FVC) and diffusing capacity of the lungs for carbon monoxide (DLCO) are widely used as surrogate markers of disease progression. This systematic literature review (SLR) evaluates their association with mortality in patients with SSc-ILD.
METHODS: Embase and MEDLINE® were searched in May 2026, from database inception, to identify studies reporting data on association of FVC/DLCO with mortality.
RESULTS: A total of 44 studies were included in this SLR, assessing the association between FVC and/or DLCO and all-cause or SSc-ILD-related mortality. Of these, six were interventional studies (including randomized controlled trials and real-world studies), and 38 were observational. FVC was reported heterogeneously, including baseline thresholds of <70% predicted (n=5), <80% predicted (n=2), and as a continuous % predicted measure (n=16). Baseline % predicted FVC was significantly associated with mortality, with multivariable HRs of 0.95-0.99 per 1% increase across six studies. Additionally, 24 studies assessed FVC decline, most commonly using thresholds of >5%, >10%, and >15%, with 15 reporting a significant association between worsening FVC and increased mortality risk. Similarly, baseline DLCO % predicted was significantly associated with mortality, with multivariable HRs ranging from 0.95-0.98 per 1% increase in three studies. Among interventional trials, SLS-1 and SLS-2 showed that declines in FVC and DLCO over 2 years were better predictors of mortality than baseline values.
CONCLUSIONS: Baseline percent-predicted FVC and DLCO, and their changes over time, predict mortality in SSc-ILD. However, heterogeneity in thresholds, endpoint definitions, and follow-up durations limits comparability. Standardized thresholds and prospective surrogacy validation are needed to strengthen the evidence.
A LIVING NETWORK META-ANALYSIS (NMA) FRAMEWORK INCORPORATING REAL-WORLD EVIDENCE (RWE) IN CHRONIC LYMPHOCYTIC LEUKEMIA (CLL) USING THE ONCOSCOPE REAL-TIME AI-ASSISTED SYSTEMATIC LITERATURE REVIEW (REAL-SLR)
OBJECTIVES: The optimal choice of a specific therapy in first-line (1L) and relapsed/refractory (R/R) CLL remains uncertain as randomized-controlled trials (RCTs) and anchored indirect comparisons do not cover all relevant treatment options. Evolving evidence requirements under EU-JCA increasingly require timely comparative effectiveness evidence. We propose a living RWE-informed NMA framework using Oncoscope REAL-SLR database to integrate RCT and comparative RWE to address evidence gaps in CLL.
METHODS: A living NMA framework was developed using the PRISMA-compliant Oncoscope REAL-SLR Database. RCTs and comparative RWE studies reporting adjusted relative effect on overall and progression-free survival (OS/PFS) are identified within 1L and R/R CLL populations. Relevant effect modifiers identified through REAL-SLR are documented in a living catalogue. After assembling of RCT-only network, comparative RWE data is evaluated for bridging any of the identified gaps. RWE integration uses bias-adjusted informative prior or three-level hierarchical/bias-adjustment.
RESULTS: The living NMA supports inclusion of new RCTs and/or comparative RWE studies as they are identified in the daily-updated REAL-SLR. As of June 15th 2026, REAL-SLR database included 81 RCTs and 72 comparative RWE studies. In 1L and R/R CLL, respectively, 32 studies (31 RCTs) and 15 studies (13 RCTs) informed PFS analyses, while 21 studies (10 RCTs) and 12 studies (8 RCTs) informed OS analyses. The treatment-naïve PFS network was the most complete; comparative RWE could bridge evidence gaps between acalabrutinib and ibrutinib. For OS, RWE data could potentially connect venetoclax+obinutuzumab, ibrutinib, acalabrutinib, and zanubrutinib. In previously treated population, RWE data could provide additional OS comparison between venetoclax-monotherapy and venetoclax+rituximab/obinutuzumab-combinations.
CONCLUSIONS: The Oncoscope REAL-SLR Database enables a living NMA framework that incorporates emerging trial and real-world evidence to address evidence gaps in CLL This approach provides a decision-relevant assessment of comparative effectiveness and a complete mapping of the multi-PICO evidence for JCA in rapidly evolving oncology landscapes.
VBHC x HEOR: From Shared Principles to Better Decisions - Are We There Yet?
Session Type: Issue Panel
Topics: Health Service Delivery & Process of Care, Patient-Centered Research, Economic Evaluation
Track: Expanded Value Measures
Level: Intermediate
Issue:
VBHC was designed as a framework for patient-centred care, condition-specific outcomes, and the use of PROs to support benchmarking and inform value-based payment (VBP). Despite this conceptual clarity, VBHC has struggled to achieve consistent operationalisation. Its growing relevance is reflected in the ISPOR Top 10 HEOR Trends, where VBHC now ranks third. In parallel, HEOR provides mature methods for measuring outcomes and costs and informing decisions, yet these tools are not systematically incorporated into VBHC initiatives. The core issue to be debated is whether VBHC and HEOR are sufficiently aligned to support robust, comparable, and decision-relevant assessments of healthcare performance, and what methodological and practical gaps must be addressed for this alignment to be implemented at scale.
Overview:
This 60 minute panel will examine the conceptual, empirical, and implementation dimensions of aligning VBHC and HEOR. The session will begin with a 15 minute presentation by Jens Grueger, who will outline the conceptual foundations of VBHC–HEOR alignment and the role of HEOR frameworks in defining and measuring value. This will be followed by a 15 minute presentation from Borja García Lorenzo, who will discuss empirical applications demonstrating how established HEOR methods can be transferred into VBHC contexts to operationalise the value equation and support more consistent assessment of healthcare performance. Lotte Steuten will then provide a 15 minute of critical perspective on the practical conditions that shape implementation in health systems, reflecting on the factors that influence whether conceptual and empirical advances can translate into routine practice. The final 15 minutes will be dedicated to audience discussion and debate, focusing on the methodological, operational, and system level implications of VBHC–HEOR alignment. The session will be of particular interest to stakeholders involved in decision making and resource allocation, including payers, HTA bodies, clinicians engaged in outcomes measurement, health system managers, and researchers working on VBHC and HEOR.
Moderator
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William V Padula, PhD
University of Southern California, Rancho Palos Verdes, CA, United States
Speakers
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Jens Grueger, PhD
CHOICE Institute, University of Washington, Seattle, WA, United States
Jens Grueger is a Senior Advisor at the Boston Consulting Group advising life sciences companies on pricing and market access, and a Senior Advisor at Curta, focusing on evidence, value, access and pricing. He is an Affiliate Professor of Health Economics at the CHOICE Institute, University of Washington School of Pharmacy and ISPOR’s 2020-2021 President.
Previously, Jens had a distinguished career in the pharmaceutical industry. He was Senior Vice President and Head of Global Access for F. Hoffmann-La Roche (2011-2019). Jens was Vice President and Head of Global Market Access Primary Care at Pfizer (2009-2011), Head of Global Pricing & Health Economics at Novartis (1999-2009), and Director of Health Economics at the German affiliate of SmithKline Beecham Pharma (1994-1997). He founded Diversified Health Systems, an internet-based disease management services start-up (1997-1999).
Jens holds a MSc in Medical Statistics and Theoretical Medicine and a PhD in Mathematical Statistics from the Technical University of Dortmund, Germany and has authored more than 40 publications.
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Borja Garcia-Lorenzo
Biosistemak Institute for Health Systems Research, Bilbao, Spain
Borja García-Lorenzo holds a PhD in Economics from Universidad de Las Palmas de Gran Canaria and an MSc in Industrial Economics from Universidad Carlos III de Madrid. He is currently a health economist at Biosistemak – Institute for Health Systems Research, where he leads work on Value-Based Health Care (VBHC) and contributes to the development of value-oriented approaches for health system performance and decision-making.
His current work focuses on applying VBHC principles to outcome measurement, performance assessment and the use of evidence to inform resource allocation. He collaborates with hospitals, regional health services and research partners in projects aimed at strengthening value-driven governance and improving the alignment between outcomes, costs and decisions.
He previously worked as a health economist at Hospital Clínic Barcelona and at the Spanish Network of Agencies for Health Technology Assessment. He has held research positions at Université de Bordeaux and the Institut de Recerca en Avaluació i Polítiques Públiques (IRAPP), and has been a visiting researcher at the Toulouse School of Economics and the Groupe d’Analyse de Théorie Économique (GATE) in Lyon. He has taught applied quantitative methods in economics at several universities, and currently teaches health economic evaluation in postgraduate programmes.
His research interests include VBHC, health technology assessment, public health policy evaluation and efficiency analysis. He has contributed to publicly funded research projects and to reports for the Spanish Ministry of Health, and his work has been published in Q1 journals in health economics.
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Lotte Steuten, MSc, PhD
Office of Health Economics, London, United Kingdom
Lotte Steuten, PhD Deputy Chief Executive of the Office of Health Economics; former Member, Board of Directors, ISPOR
Prof Lotte Steuten is deputy chief executive of the Office of Health Economics (OHE), the world’s oldest independent health economics research organization, based in London, UK, and a globally recognized expert in health economics and outcomes research (HEOR).
Her research addresses challenges in valuing and paying for innovative therapies, with the aim of achieving effective, accessible, affordable, and efficient healthcare for all. She has published over 150 peer-reviewed papers on topics including the value of novel treatments, diagnostics and prevention for a wide range of non-communicable and infectious diseases.
With 2 decades of experience across Europe, the United States, and Asia Pacific, she advises governments, industry, and other organizations worldwide. She is frequently sought by media and international stakeholders for expert commentary on HEOR, value assessment, health policy innovation, and evolution of health technology assessment globally.
Alongside her position at OHE, Prof Steuten is a visiting honorary professor at City St George’s, University of London. Prior to joining OHE, she held academic faculty positions at the Fred Hutch Cancer Research Center and the University of Washington in the United States. She earned her PhD (with honors) from Maastricht University in the Netherlands.
Convergence and the Transatlantic Bargain: US Pricing Reform, the EU Pharma Package, and What Either Means for Innovation and Access
Session Type: Issue Panel
Topics: Health Policy & Regulatory
Track: Access and Pricing
Level: Intermediate
ISSUE: Pharmaceutical pricing on both sides of the Atlantic is being reformed simultaneously — but in ways that may pull against each other. In the US, the IRA's Medicare negotiation programme is forcing prices toward international levels, while MFN and tariff-linked arrangements press other wealthy countries to pay more. In Europe, the EU Pharma Package resets data-protection baselines, broadens the Bolar exemption, and allows Member States to strip protection where products are not launched locally. Industry warns that reforms weaken innovation incentives. Payers and patient advocates argue the old settlement failed on access and affordability. The panel asks whether the two reforms are on a collision course, a convergence path, or are unexpectedly complementary.
OVERVIEW: This panel brings together European Commission, US policy, and health economics perspectives to examine two landmark reforms and what their interaction means for pricing, innovation, and access globally. Indranil Bagchi (8 mins) will open by framing the dual reform landscape from a global industry perspective, setting out the key tensions and questions for the panel. Ferenc Marojka (10 mins) will present the EU Pharma Package and Biotech Act — their incentive redesign, protection changes, and intended effects on launch behaviour and affordability across Member States. Sean Sullivan (10 mins) will describe the US reform agenda, covering the IRA negotiation programme, MFN, and tariff arrangements, and what each is designed to achieve. Amanda Cole (10 mins) will examine the combined economics of both agendas, exploring whether the simultaneous pressure to lower US prices and raise international ones creates a sustainable equilibrium — or a damaging squeeze on the revenues that fund future innovation. At least 20 minutes will be reserved for audience discussion, with a focus on whether transatlantic coordination is feasible and what a more coherent global pricing settlement might look like.
Moderator
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Indranil Bagchi, MS, PhD
GSK US, Collegeville, PA, United States
Dr. Indranil Bagchi is the Global Head of Pricing & Market Access at GSK. In this role, Indranil drives the overall strategy on value demonstration and market access across all product areas in the GSK portfolio, to secure access to medicines for our patients and to maximize the value of our portfolio.
Indranil has more than two decades of experience in market access across several major companies in the pharmaceutical industry. In 2014, Indranil received the Outstanding 50 Asian Americans in Business award and in 2010, Indranil was recognized in Pharmaceutical Executive magazine's annual roster of Emerging Leaders, The New Breed of Leadership. Indranil is a frequent speaker and contributor to forums, articles and conferences addressing issues related to access to medicines.
Prior to GSK, Indranil was Senior Vice President and Worldwide Head of Value and Access at Novartis Oncology. Previously at Pfizer, he was Vice President and Global Head of Payer Insights and Access and prior to that, he was with GlaxoSmithKline in Health Economics and Outcomes Research. Dr. Bagchi has an undergraduate degree in Pharmacy, a masters degree in Pharmacy and Healthcare Administration and a doctoral degree in Pharmaceutical Socioeconomics.
Speakers
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Ferenc Marofka, MSc
Brussels, Belgium
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Sean D Sullivan, PhD
University of Washington, Seattle, WA, United States
Sean D. Sullivan, BScPharm, MSc, PhD, is Professor and Dean, School of Pharmacy. He holds a joint appointment as Professor of Health Services in the School of Public Health. He holds adjunct appointments in the School of Medicine, the Public Health Sciences Division at the Fred Hutchinson Cancer Research Center, and at the Kaiser Permanente Washington Health Research Institute. He completed training in pharmacy at Oregon State University in 1983, obtained a master’s degree at the University of Texas in 1986 and a PhD in health economics and policy at the University of California, Berkeley in 1992. Dr. Sullivan has authored more than 400 journal articles, book chapters, task force reports and organizational and governmental publications. In many of these writings, he has assessed the evidence and applications of medical technology in relation to coverage and reimbursement decisions. His research interests include technology assessment, medical decision-making, and economic evaluation of medical technology. He is past president of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and past chair of the Academy of Managed Care Pharmacy (AMCP) Executive Committee of the Format for Formulary Submissions – the United States evidence-based guidelines for formulary decision making. Dr. Sullivan served as a past member of the Medicare Evidence Development and Coverage Advisory Committee, a past member of the Regence Blue Shield and Premera Blue Cross P/T Committee, and the Chair of the Premera Blue Cross Value Assessment Committee. He is also on the editorial boards of Value in Health, PharmacoEconomics, Journal of Medical Economics, and the European Journal of Health Economics. He was awarded the 2014 Stephen G. Avey Lifetime Achievement Award from the Academy of Managed Care Pharmacy (AMCP) and the 2015 APhA Academy of Pharmaceutical Research Sciences (APRS) Research Achievement Award.
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Amanda Cole, BSc, PhD
Office of Health Economics, London, United Kingdom
Amanda Cole is a Director at the Office of Health Economics (OHE). She leads OHE’s Economics of Innovation theme and is an Honorary Professor of Practice at University College London, as well as a member of ISPOR’s Health Science Policy Council.
Her research focuses on value based payment models, incentives for innovation, pharmaceutical market design, and how HTA policy shapes R&D decisions. She is particularly interested in aligning reimbursement practices with emerging drug development paradigms, and in advancing the use of real world evidence to support technology development and adoption.
Amanda has a strong interest in rare diseases and serves on several advisory groups in the rare disease space. Before joining OHE, she was a research fellow at the University of Birmingham, where she earned her PhD in Health Economics in 2013.
Beyond the trial: real-world evidence and lifecycle evidence generation in NICE existing-use HealthTech assessments
Session Type: Issue Panel
Topics: Real World Data & Information Systems, Health Technology Assessment, Medical Technologies
Track: Real-World Evidence (RWE)
Level: Introductory
ISSUE: As health systems seek to optimise decisions on technologies already embedded in routine care, real-world evidence (RWE) is becoming crucial. NICE's existing-use HealthTech assessments (formerly late-stage assessments, LSA) apply lifecycle thinking to established products, drawing on registry data, claims data, observational studies, and patient-reported outcomes to address what pre-market evidence was not designed to answer. LSAs are inherently challenging, with key issues including limited direct comparative evidence between similar products, evidence-supported price variation, and uncertainty over whether incremental product innovation translates into meaningful benefit, particularly where evidence base remains limited. These challenges reflect feasibility of generating evidence that is sufficiently timely and decision-relevant. Stakeholders and patients also raised concerns about how LSAs may affect continued access to commonly used technologies in NHS.
This panel will explore how a proactive lifecycle evidence strategy, with anticipated future evidence needs for reassessment, can help demonstrate and sustain product value. Using contrasting case studies: drug-eluting stents for coronary artery disease and slide sheets, panellists will discuss which types of RWE are most influential in NICE LSA decisions and how evidence gaps can be planned earlier in a product's commercial cycle. OVERVIEW Prof Lee will introduce the session (5 minutes). Mr Kolovetsios will share views from the industry perspective and challenges in RWE generation in fast-moving med tech industry (12 minutes). Dr Chang-Douglass will present the committee perspective, focusing on decision-making under uncertainty, limitation, and values of RWE (12 minutes). Dr Chalkidou will outline NICE lifecycle approach and the need for closer industry/ stakeholder collaboration for future RWE in HTA. (12 minutes). The session will conclude with moderated audience discussion. This session is relevant to policymakers, clinicians, industry representatives, and patients. Attendees will gain insights into assessing established technologies, the use of RWE, and implications for future HTA processes.
Moderator
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Dawn Lee, MSc
University of Exeter PenTAG, Exeter, United Kingdom
Speakers
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Michael Kolovetsios, MSc
UK, United Kingdom
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Stacey Chang-Douglass, PhD
Clarivate, London, United Kingdom
Stacey is Head of Health Economics at Clarivate, where she oversees the delivery of economic modelling to support HTA submissions, global model development and country adaptations. With over 15 years of health economics experience spanning positions in consulting and at NICE, she provides strategic insights and technical guidance on projects across an extensive range of therapy areas. She also serves as a standing committee member at the NICE Medical Technologies Advisory Committee since 2022, a health economist member of the committee updating NICE guideline NG208, and an External Oversight Group member of the NICE Listens project on AI in health and care guidance. She holds an MSc from the London School of Economics and a PhD from the University of York.
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Anastasia Chalkidou
London, United Kingdom
Can Agentic AI Deliver HTA-Ready Health Economic Models? Governance, Validation, and Trust
Session Type: Issue Panel
Topics: Methodological & Statistical Research, Economic Evaluation, Health Technology Assessment
Track: AI
Level: Advanced
ISSUE:
Large language model (LLM) agents can now construct Excel-based health economic models, execute multi-phase quality control protocols, generate HTA-compliant technical reports, and produce structured bibliographies—all with validated accuracy. Yet no consensus exists on whether AI-generated outputs that pass identical verification standards to human-generated work should be treated equivalently in HTA submissions. The field faces a governance gap: proof-of-concept demonstrations have outpaced frameworks for responsible production deployment. This panel debates whether current validation standards suffice for LLM-generated health economic outputs, or whether new governance is required.
OVERVIEW:
Three speakers from different sectors present empirical perspectives within a 60-minute session. The industry speaker (10 min) presents results from a multi-model validation programme: automated QC achieving concordance with human reviewers across 10+ models, technical reports reaching zero expert revision, functional cost-effectiveness models built in under 3 hours, and reference management with full structural accuracy. The consultancy speaker (10 min) addresses scalability and trust, reporting significant timeline reductions but arguing that the critical success factor is decomposing modelling tasks into auditable sub-steps, with governance calibrated to use case—from early asset valuation to submission-grade models. The academic speaker (10 min) examines where current reporting frameworks—including ELEVATE-GenAI—fall short for autonomous agent workflows, proposing adapted criteria including specification completeness scoring and assumption provenance tracking. A 15-minute moderated discussion addresses: (1) Is an LLM-generated model that passes ISPOR-SMDM equivalent to a human-built model that passes ISPOR-SMDM? (2) What frameworks must HTA agencies implement to audit AI involvement, and where is the threshold between AI-assisted and AI-generated modeling? (3) Where is human oversight essential versus performative? This benefits health economic modellers, HTA assessors, pharmaceutical submission teams, and HEOR consultants navigating AI adoption.
Moderator
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Andrew Briggs, DPhil
London School of Hygiene & Tropical Medicine, London, United Kingdom
Speakers
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Andre Verhoek, MSc
AstraZeneca, Barcelona, Spain
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Jag Chhatwal, PhD
Harvard Medical School / Massachusetts General Hospital, Boston, MA, United States
Jag Chhatwal, PhD, is the director of the Institute for Technology Assessment at Massachusetts General Hospital and an associate professor at Harvard Medical School. He also serves as core faculty at the Center for Health Decision Science, Harvard T.H. Chan School of Public Health. Dr. Chhatwal has co-authored more than 125 original research articles and editorials in leading peer-reviewed journals. His research has informed health policy decisions at prominent organizations including the White House, the World Health Organization, and the CDC, and has been featured in major media outlets such as CNN, Forbes, National Public Radio, The New York Times, and The Wall Street Journal. Dr. Chhatwal serves as an associate editor of Value in Health and as guest editor for its special issue on artificial intelligence. He is also a member of the ISPOR Generative AI Working Group.
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Xiaoyan Wang, PhD
New Orleans, United States
Embracing the Opportunity of AI in Economic Modelling Responsibly Through Agentic Workflows and Guardrails
Session Type: Workshop
Topics: Economic Evaluation, Health Technology Assessment
Track: AI
Level: Intermediate
PURPOSE: Health economic models must be reproducible, auditable, and clinically credible. As agentic AI tools proliferate, modelers face a practical question: which parts of the modelling workflow benefit from AI assistance, and which are too consequential to delegate? Speakers will share perspectives from industry, academia, HTA, and vendor experience, addressing where human oversight remains non-negotiable and exploring the emerging potential of agentic AI in model development, validation, and “living HTA.”
DESCRIPTION: This workshop gives attendees a cross-stakeholder view of agentic AI in health economic modelling. [Beth] will open the session by framing the core challenge: agentic AI is a powerful, general-purpose technology that can be directed at nearly any task, but not every task it can do should be delegated to it. (10 min.) [Tim] will demonstrate where AI provides clear value in the health economic modelling workflow: data extraction, incorporating “live” NMA results into decision models, model building and rapid scenario testing, and report generation. (15 min.) [Liza] will share practical lessons from developing agentic health economic modelling systems, making the case for deliberately excluding AI from model calculations, with computation instead delegated to open-source packages such as heemod and PyDICE. (10 min.) [Dan] will bring an HTA perspective, describing work done to date on model testing, validation, and replication as well as conversion (e.g., Excel to R), and will discuss the prospects of agentic AI for model development and support of "living HTA" activities. (10 min.) [Beth] will then facilitate audience interaction with probing questions (15 min.). This workshop is intended for health economists and HTA practitioners who want a practical perspective on where the technology earns trust—and where it does not.
Moderator
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Beth Devine, MBA, MSc, PharmD, PhD
University of Washington, Seattle, WA, United States
Speakers
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Timothy Reason, BSc, MSc
Estima Scientific, London, United Kingdom
Tim Reason is co-founder of Estima Scientific and specializes in AI and evidence synthesis, having spent 15 years in the field of HEOR and technology. Tim is managing director of Estima, driving business activities, innovation and strategy for the company. Tim’s specializes in the intersection of HEOR, software development and AI to drive better outcomes for patients. Tim is the lead author on 2 seminal papers in AI for HEOR, showing that AI can be used to automate health economic modelling and NMA.
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Liza Zasukhina
MSD, Prague, Czech Republic
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Dan Ollendorf, MPH, PhD
Institute for Clinical and Economic Review (ICER), Boston, MA, United States
Dan Ollendorf, PhD is Chief Scientific Officer and Director of Health Technology Assessment (HTA) Methods and Engagement at the Institute for Clinical and Economic Review (ICER), with responsibility for managing the organization’s internal health economics capacity and program of external collaboration with academic researchers and institutions, as well as collaboration with international HTA bodies and other stakeholders on key process and methods topics. From 2007-2018, Dr Ollendorf was chief scientific officer for ICER, where he oversaw the broader HTA process and managed relationships with multiple stakeholders and research collaborators.
Responsible AI in HTA - Introducing the NICE AI Methods Framework
Session Type: Other Breakout Session
Topics: Health Technology Assessment
Track: AI
Level: Introductory
PURPOSE: The objectives of this session are to introduce the NICE AI Methods Framework, to discuss what would make AI acceptable and trustworthy in HTA decision-making, and to gather stakeholder perspectives to inform public feedback.
DESCRIPTION: AI has the potential to be transformative across HTA. However, there is limited consensus regarding acceptable use, evidence standards, transparency, reproducibility, and oversight for AI applications in HTA.
This session will introduce the NICE AI Methods Framework, a best practice framework developed to support the use of AI throughout the HTA pipeline. The session will begin with an introduction outlining the importance of responsible AI in HTA, the immediate need for best practice guidance to enable innovation, and the global context for AI governance and implementation in evidence generation and decision-making (7 minutes, Higashi). A walkthrough of the AI Framework will then be presented, including the draft AI principles and best practice guidance for specific use cases; areas for stakeholder feedback will also be highlighted (10 minutes, Sonabend-Friend). A 7-minute reflection on the Framework will be provided by Klijn.
The session will continue with a moderated discussion exploring what would make AI acceptable and trustworthy in HTA decision-making (24 minutes, Higashi moderating; Sonabend-Friend, Fotheringham, Klijn, and Ho participating). Discussion topics will include opportunities enabled through AI, priority use cases for the framework, and operational considerations for implementing best practice AI methods guidance.
The session will conclude with audience interaction, including live polling, targeted discussion prompts, and selected audience questions (12 minutes, moderated by Higashi). Illustrative topics will include current AI usage, organizational readiness for AI in HTA, barriers to adoption, and challenges in operationalizing AI frameworks. This session may benefit HTA agencies, payers, industry, HEOR professionals, policymakers, and researchers involved in evidence generation and decision-making.
Moderator
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Mitch Higashi, PhD
ISPOR, Villanova, PA, United States
Speakers
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Raphael Sonabend-Friend
NICE, London, United Kingdom
Dr Raphael Sonabend-Friend is a Scientific Adviser in the Science, Evidence and Analytics team at the National Institute for Health and Care Excellence (NICE). Raphael supports the testing of artificial intelligence, particularly focused on generative artificial intelligence methods. Raphael holds a PhD in the accessible and transparent use of machine learning in healthcare settings. Raphael has over a decade of experience in the healthcare sector ranging from large philanthropies, small local charities, governmental bodies, and private sector consulting for UK and international organisations.
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Sven L Klijn, MSc
Bristol Myers Squibb, Princeton, NJ, United States
Sven Klijn is Director at Bristol Myers Squibb in the Global HEOR Evidence Acceleration & Innovation group, where he leads the innovative modeling agenda in hematology and cell therapy. In addition, Sven has an active role in providing modeling and Generative AI education at international congresses. He has widely published on innovative methods, especially in the fields of survival extrapolation and Generative AI. Sven has a training in public health and health economics and previously had various roles in CROs related to health-economic modeling.
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Yan Teck Ho, PhD
Singapore, Singapore
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James Fotheringham, PhD
University of Sheffield, Sheffield, United Kingdom
Having trained in kidney medicine, healthcare data analysis and causal inference, James joined NICE as a committee member in 2021. He became Chair of Technology Appraisal Committee C in 2025, and has interests in the health economics of kidney disease and HTA methods including reflecting the environment in HTA decision-making.
Patient engagement in economic modelling: bridging the gap between what’s measured and what matters
Session Type: Other Breakout Session
Topics: Patient-Centered Research, Economic Evaluation, Real World Data & Information Systems
Track: Patient-Centered Evidence
Level: Introductory
PURPOSE:
Patient engagement has been widely recognised as an important component of HEOR, yet its integration into economic model development remains variable. This session will demonstrate how early patient engagement can guide real-world evidence generation, challenge and/or redefine existing model concepts, and improve the relevance of economic evaluations for decision making, using choroidal melanoma as an illustrative example.
DESCRIPTION:
Economic models are typically conceptualised around clinical trial endpoints and precedent, but these may not always fully capture the lived experience of patients, potentially limiting the relevance of model outputs in real-world decision making. Using uveal melanoma as an example, this session will examine how patient perspectives can be more systematically incorporated into model conceptualisation and data analysis.
The introduction will contrast traditional model conceptualisation with a patient-informed framework, presenting a practical roadmap for integrating patient insights into model development (Ross Goldstein, 9 minutes).
Jo Gumbs will discuss current opportunities for patient involvement in evidence generation, highlighting the types of insights patients can contribute and how these can complement traditional data sources. Practical recommendations for improving engagement with patients in health economics activities will be presented (12 minutes).
Ariel Ong will address methodological considerations for capturing patient experience, including the integration of multiple real-world data sources, use of unstructured patient-level information, and advanced analytical methods (12 minutes).
Noémi Muszbek will present model structures to illustrate how data, patient experience and precedent may all be considered. The importance of non-conventional outcomes, and the difficulty in translating them into costs and utilities to be accounted for in decision-making, will be discussed (12 minutes).
The session will conclude with audience discussion, using interactive polling to share experiences and perspectives on translating patient experience into data and cost-effectiveness modelling (15 minutes).
Moderator
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Ross Goldstein, MD, MBA
Brighton, MA, United States
Speakers
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Ariel Yuhan Ong, MD
Moorfields Eye Hospital NHS Foundation Trust, United Kingdom
Ariel is a senior ophthalmology registrar in the Oxford deanery and NIHR Doctoral Fellow at University College London and Moorfields Eye Hospital in London. She is also the Data Lead for the INSIGHT Health Data Research Hub. Ariel’s research focuses on validating and deploying safe and effective AI systems to enhance clinical decision-making and scientific discovery, using retinal disease as an exemplar. She is also working on building the data infrastructure and evidence frameworks needed to scale AI innovation safely. Other research interests include improving the management of eye disease in pregnancy as well as sustainability in eye care.
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Jo Gumbs, MSc
Slough, United Kingdom
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Noemi Muszbek, MSc
Visible Analytics Ltd, Reading, United Kingdom
Patient Preferences in Healthcare Decision-Making: Global Insights from Discrete Choice and Preference Studies
Session Type: Research Podiums
Understanding what matters most to patients is essential for patient-centered care and evidence-based decision-making. This session highlights preference studies across diverse healthcare settings, including digital health, oncology, diagnostics, and chronic disease management. The presentations demonstrate how patients weigh benefits, risks, costs, convenience, and trust, providing insights that can inform clinical practice, product development, and policy decisions.
QUALITY OF LIFE VERSUS LENGTH OF LIFE: PREFERENCES OF CANCER PATIENTS FROM EIGHT EUROPEAN COUNTRIES
OBJECTIVES: Shared decision-making combines patients’ values, goals, and preferences with physicians’ medical expertise. Evidence on cancer patients’ attitude towards treatment, particularly whether they prioritize quality of life (QoL) over length of life (LoL), remains limited. This study aimed to assess patients’ preference toward QoL versus LoL.
METHODS: Within the 4D PICTURE project, patients diagnosed with cancer from eight European countries (Austria, Denmark, Germany, the Netherlands, Slovenia, Spain, Sweden, and UK) completed a digital questionnaire. Using a 5-point Likert scale, patients indicated whether they considered QoL more important than LoL. Additionally, demographic and clinical data were collected, including age, sex, marital status, cancer type, treatment type, and time since diagnosis. Responses were analyzed descriptively and with mixed-effects ordinal regression including country as a random factor. Sensitivity analyses using subgroup interaction models were conducted to assess the robustness and generalizability of the findings. Outcomes are reported as odds ratios (OR) with 95% confidence intervals (95% CI).
RESULTS: Among patients with cancer (n = 2,284; 71.4% women; mean age 59.7 ± 12.1 years), 61.4% reported that QoL was more important than LoL. Preferences for QoL over LoL varied across countries. After adjustment for demographic, clinical, and cross-country heterogeneity, older age (per 10 years increase) (OR = 1.15, 95% CI 1.10 - 1.21, p<0.01), having treatment completed (OR = 1.17, 95% CI 1.00-1.37, p=0.04782), and certain cancer types, particularly brain/CNS (OR = 3.88, 95% CI 1.51 - 10.01, p<0.01) and lung/pleural cancers (OR = 1.48, 95% CI 1.03 - 2.14, p<0.01) were associated with higher odds of prioritizing QoL over LoL. Sensitivity analyses supported the robustness of the findings.
CONCLUSIONS: Across eight European countries, patients with cancer tend to place greater value on QoL than on LoL, emphasizing the importance of considering patients’ preferences for QoL in the shared decision-making process in oncology.
PUBLIC PREFERENCES FOR LUNG NODULE BIOPSY AHEAD OF NATIONAL LUNG CANCER SCREENING: A UK DISCRETE CHOICE EXPERIMENT
OBJECTIVES: Lung cancer is the leading cause of cancer death in the UK, with poor survival driven by late diagnosis. Expanding low-dose CT screening increases early detection and biopsy demand. Selecting between robotic-assisted bronchoscopy (RAB) and CT-guided transthoracic needle aspiration (TTNA) is clinically important. These approaches differ in technique, complication risk, anaesthesia, pneumothorax requiring hospitalisation and repeat procedures, but evidence on how these attributes are valued is limited. We quantified UK public preferences for lung nodule biopsy attributes.
METHODS: A discrete choice experiment (DCE) was conducted in a representative UK general-population sample (N=223). Participants chose between biopsy options defined by method, anaesthesia, complication risk and repeat-biopsy probability, reflecting RAB and TTNA characteristics. Attributes were informed by literature and clinical input. Preferences were analysed with conditional and random parameter logit models, plus subgroup and latent class analyses. Time trade-off (TTO) and anxiety-based choice were included.
RESULTS: Respondents placed greatest weight on minimising complication risk (relative attribute importance RAI 55.2%) and avoiding repeat biopsy (RAI 37.6%); anaesthesia (6.3%) and biopsy method (0.8%) were less important. Latent class analysis identified a risk-averse segment prioritising safety and diagnostic certainty, consistent with the anxiety-based task. In the time trade-off exercise, willingness to undergo biopsy rather than wait increased from 58.3% to 67.7% as complication risk fell from 20% to 5%, with mean utility rising from 0.64 to 0.67.
CONCLUSIONS: This is the first UK DCE to quantify preferences distinguishing RAB and TTNA. The most valued factors, low complication risk and avoiding repeat biopsy, are where these approaches differ, with RAB offering TTNA-like yield at lower complication rates. Because patients prioritise outcomes over modality, biopsy selection should reduce avoidable harm by minimising complications and repeat procedures. As screening expands biopsy demand, findings support the patient-centred component of NICE evaluations. Confirmatory work in patients referred for lung nodule biopsy is warranted.
USER PREFERENCES FOR HEALTH APPLICATIONS: POPULATION-REPRESENTATIVE CONJOINT EXPERIMENTS IN AUSTRIA AND ITALY
OBJECTIVES: Although digital health applications have considerable potential to support disease management and promote well-being, adoption rates remain relatively low. In order to support design, implementation, and dissemination of health applications, this study aimed to identify factors influencing individuals' preferences for using them.
METHODS: We conducted a survey with an embedded conjoint experiment among population-representative samples of 3444 Austrians and 2994 Italians in second half of 2025. We tested attributes such as who recommended the App, content, functionality, developer, data usage and cost. Average marginal component effects (AMCE) with 95% confidence intervals (95%CIs) were calculated.
RESULTS: In both Austria and Italy, participants strongly preferred health applications recommended by a healthcare professional rather than a friend (Austria: AMCE=0.466, 95% CI 0.334-0.598; Italy: AMCE=0.423, 95% CI 0.291-0.555), whereas AI-generated recommendations reduced preferences (Austria: AMCE=-0.310, 95% CI -0.441 to -0.179; Italy: AMCE=-0.181, 95% CI -0.313 to -0.049). Compared with peer-to-peer chat functions, Austrian participants preferred applications that enabled booking appointments with healthcare professionals (AMCE=0.321, 95% CI 0.188-0.455) and asking health-related questions (AMCE=0.268, 95% CI 0.137-0.399), while in Italy only appointment booking increased preferences (AMCE=0.169, 95% CI 0.042-0.297). Applications developed by university hospitals were preferred over national and international companies in Austria (AMCE=0.213, 95% CI 0.096-0.330), but not in Italy. Compared with personal data use only, Austrian participants were less likely to prefer applications that used their data to train AI models (AMCE=-0.153, 95% CI -0.282 to -0.023), whereas no such effect was observed in Italy. In both countries, applications covered by health insurance were preferred over self-funded applications.
CONCLUSIONS: Preferences for health applications were primarily driven by trust-related factors, including recommendations from healthcare professionals, health insurance coverage, and, in Austria, development by a university hospital.
PREFERENCES FOR TARGETED THERAPY ATTRIBUTES AMONG CHINESE PATIENTS WITH ALK-POSITIVE ADVANCED NON-SMALL CELL LUNG CANCER: A DISCRETE CHOICE EXPERIMENT
OBJECTIVES: To quantify treatment preferences among Chinese patients with ALK-positive advanced non-small cell lung cancer (NSCLC) with prior ALK tyrosine kinase inhibitors (ALK-TKI) therapy.
METHODS: An online discrete choice experiment (DCE) was conducted from May to June 2025 through a national patient organization. Seven attributes with three levels each: median progression-free survival (PFS), objective response rate (ORR), constipation, central nervous system (CNS) adverse events, rash, liver injury, and monthly treatment cost. An orthogonal fractional factorial design generated nine choice sets with two treatment alternatives and an opt-out option. A quality-control task and consistency checks were applied. Data were analyzed using mixed logit models to evaluate relative importance, choice probability, risk-benefit trade-offs, and subgroup variations.
RESULTS: Of 269 questionnaires, 251 met prespecified quality criteria. Safety profiles dominated patient preferences, with liver injury (28.02%) and CNS adverse events (22.46%) carrying the highest relative importance, followed by ORR (17.93%). Patients favored higher ORR and longer PFS, with ORR yielding greater preference weights. Elevating ORR from 70% to 80% and 90% increased choice probability by 14.17% and 17.36%, respectively, whereas extending median PFS from 30 months to 60 months increased it by 11.87%. Notably, ORR improvements could offset moderate CNS adverse events or liver injury, whereas PFS extensions could not. Neither severe CNS adverse events nor severe liver injury could be compensated for by efficacy gains. Subgroup analyses showed that females, patients with comorbidities, patients with stage IV disease, and those who had received only one prior ALK-TKI exhibited stronger avoidance of moderate CNS adverse events.
CONCLUSIONS: Preferences for ALK-TKI treatments among Chinese patients are primarily dictated by hepatic and neurological safety, followed by ORR benefits. These findings offer pivotal insights for patient-centered clinical decision-making and preference-informed evaluations of ALK-TKIs.
10:30 - 13:30
Poster Session 3
Session Type: Research Posters
Poster Tours 11:45–12:30 | Presenters will be with their posters from 12:30–13:30
11:15 - 13:00
Lunch Service (Exhibit Hall)
Session Type: General Meeting
As you enjoy your lunch in the Poster and Exhibit Hall, seize the opportunity to engage in meaningful conversations with fellow attendees. Take this time to exchange ideas, forge new partnerships, or simply enjoy casual conversations.
11:30 - 12:15
Real-World Evidence Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 3, Posters will be hung from 10:30-13:30.
Posters featured in this tour:
PT25: INTEGRATING PATIENT-CENTRICITY INTO REAL-WORLD EVIDENCE GENERATION: INSIGHTS FROM SOCIAL MEDIA LISTENING
PT26: GLUCAGON-LIKE PEPTIDE-1-BASED ANTI-OBESITY MEDICATIONS REDUCE INCIDENT OBSTRUCTIVE SLEEP APNEA IN OLDER ADULTS: REAL-WORLD EVIDENCE
PT27: REAL-WORLD EVIDENCE TO ASSESS THE FEASIBILITY OF LOW-DOSE CT LUNG CANCER SCREENING IN THE BRAZILIAN UNIFIED HEALTH SYSTEM (SUS)
PT28: CHARACTERIZING PEOPLE WITH UNTREATED MULTIPLE SCLEROSIS IN REAL-WORLD EVIDENCE: INSIGHTS FROM LITERATURE AND THE GERMAN NEUROTRANSDATA REGISTRY (2018-2024)
PT29: REAL-WORLD EVIDENCE ON THROMBOTIC MICROANGIOPATHIES IN GREECE: PATIENT CHARACTERISTICS AND CLINICAL OUTCOMES FROM REAL-WORLD HOSPITAL DATA OVER A 20-YEAR PERIOD
PT30: A SYSTEMATIC REVIEW OF REAL-WORLD EVIDENCE IN HEALTH TECHNOLOGY ASSESSMENTS FOR MULTIPLE MYELOMA IN EUROPE
Expanded Value Measures Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 3, Posters will be hung from 10:30-13:30.
Posters featured in this tour:
PT19: VACTS-PSO: A PAYER-ORIENTED VALUE FRAMEWORK INTEGRATING CLINICAL OUTCOMES, QUALITY OF LIFE, AND REAL-WORLD INTENSIFICATION COSTS IN PSORIASIS
PT20: EMPIRICAL ECONOMIC EVALUATION AND ENVIRONMENTAL IMPACT ASSESSMENT OF REUSABLE VERSUS SINGLE-USE CATHETERS FOR CLEAN INTERMITTENT CATHETERISATION
PT21: DIFFERENCES IN SOCIAL RISK, MENTAL HEALTH, AND CLINICAL PROFILES AMONG ADULTS WITH OBESITY TREATED WITH GLP-1 VERSUS NON-GLP-1 ANTI-OBESITY MEDICATIONS USING REAL-WORLD DATA
PT22: UNDERSTANDING HOW TO MEASURE WHOLE HEALTH: AN EXPLORATIVE STUDY TO IDENTIFY WHOLE HEALTH TOOLS FOR OUTCOMES RESEARCH
PT23: INCORPORATING ENVIRONMENTAL IMPACT INTO COST-UTILITY ANALYSIS: A PROOF-OF-CONCEPT APPLICATION IN SEVERE ASTHMA BIOLOGICS
PT24: THE IMPACT OF STROKE SEVERITY ON INFORMAL CAREGIVER BURDEN: A TARGETED LITERATURE REVIEW
12:15 - 13:15
Are New EU Policies Really Going to Improve RWD Availability to Inform Access Decisions?
Session Type: Forums
Topics: Health Policy & Regulatory, Real World Data & Information Systems
Level: Intermediate
Several EU policy initiatives aim to strengthen research, innovation and decision-making on access to medicines, including the European Health Data Space (EHDS), the EU HTA Regulation, and broader pharmaceutical, biotech and AI legislation. Together, these frameworks could improve availability and use of real-world data (RWD) and real-world evidence (RWE) in regulatory and HTA processes. However, it remains unclear whether these policy initiatives are sufficiently aligned, operationally mature, and responsive to payer and HTA needs to be able to inform access decisions in practice. Bhattacharjee (moderator) will open with reflections on the geopolitical environment, national and international RWD initiatives, Europe’s competitiveness in life sciences innovation, and the potential role of RWD and RWE in demonstrating value in European healthcare systems.
Schutte will discuss implementation of the EHDS, including emerging guidelines and technical specifications formulated by Joint Action TEHDAS2, which facilitates access to health data and strengthen cross-border collaboration, outlining implications for HTA and payers.
Hedberg will examine how RWE has featured in Joint Scientific Consultations and Joint Clinical Assessments under the HTA Regulation since January 2025, and how the role of RWE may evolve as implementation expands to additional therapeutic areas and high-risk medical devices.
Natz will address opportunities arising from the EHDS, General Pharmaceutical Legislation, the Biotech and AI Acts for RWE development to inform Outcomes-Based Managed Entry Agreements, reflecting on increased healthcare digitalization but continued slow uptake of RWE in HTA and payer decision-making.
After 10 minutes for each presentation, there will be 20 minutes for panel discussion. This session will be relevant to life sciences industry stakeholders, HTA bodies, payers, regulators, data custodians, patient organizations, and evidence-generation experts. Attendees will gain practical insight into how these policy frameworks may shape RWD availability, evidence expectations, stakeholder collaboration and use of RWE in access decisions across Europe.
Moderator
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Sandipan Bhattacharjee, MS, PhD
Bayer U.S. LLC, Belle Mead, NJ, United States
Speakers
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Niklas Hedberg, MSc
TLV, Stockholm, Sweden
Niklas Hedberg is the Chief Pharmacist at the Dental and Pharmaceutical Benefits Agency (TLV) in Sweden.
Niklas is the HTAR Coordination Group co-chair for medicinal products. He was the Chair of the Executive Board for EUnetHTA21 (2021-2023) and for EUnetHTA JA 3 (2018- 2021).
Niklas has been working with HTA and Pricing and Reimbursement since 2001.
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Nienke Schutte, PhD
Brussels, Belgium
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Alexander Natz
EUCOPE, Brussels, Belgium
Alexander Natz is the Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) in Brussels and advises innovative pharmaceutical and biotech companies, including start-ups, in regulatory and pricing & reimbursement matters from the EU law and German law perspective.
From 2008 to 2013, he was Head of the Brussels Office of Bundesverband der Pharmazeutischen Industrie e.V. (BPI). Before, he worked as a lawyer at Sträter Law Firm in Germany with a special focus on managed entry agreements and licensing of pharmaceuticals. Dr. Natz has also worked in the field of competition law with the European Commission and in the pharmaceutical industry.
Beyond Pricing: How HTA Informs Health System Decisions Worldwide
Session Type: Forums
Topics: Health Technology Assessment, Health Policy & Regulatory
Level: Intermediate
Health technology assessment (HTA) is a multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle, with the goal of informing decisions that promote an equitable, efficient, and high-quality health system. By systematically assessing clinical benefit, comparative effectiveness, and real-world impact, HTA ensures that coverage, reimbursement, and resource allocation decisions are informed by evidence, context, and patient need, rather than price alone. Despite its central role in healthcare decision making, the value and application of HTA remain the subject of ongoing debate among different stakeholders including policymakers and health technology developers. Globally, HTA is applied across a wide range of decision contexts beyond pricing, including priority setting, reimbursement, and procurement.
On 8 November 2026, ISPOR convened its first in person Global HTA Roundtable, bringing together 90 participants from 59 countries and jurisdictions to explore areas of consensus on the value of HTA and how it can be used most effectively across health systems. In this session, organized by the ISPOR HTA Council, attendees will hear from three HTA bodies or payers who participated in the Roundtable and will share their perspectives on the importance of global collaboration and key takeaways from the discussion. The session will conclude with an open audience discussion, providing participants with the opportunity to ask questions and share their own experiences and insights.
Moderator
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Jessica Daw, MBA, PharmD
Sentara Health Plans, Virginia Beach, VA, United States
Speakers
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Abeer A Al Rabayah, MBA, MSc, DrPH
King Hussein Cancer Center, Amman, Jordan
Dr. Abeer Ahmad Al Rabayah leads the Center for Drug Policy and Technology Assessment (CDPTA) at the King Hussein Cancer Center (KHCC) in Amman, Jordan. In addition to her role at KHCC, Dr. Abeer holds a research associate position at the Institute of Public Health, Medical Decision Making, and Health Technology Assessment at UMIT TIROL – University for Health Sciences and Technology, Hall i.T., Austria.
As the director of the Hospital-Based Health Technology Assessment (HB-HTA) program and the founder of the Health Technology Assessment (HTA) fellowship program at KHCC, In 2024, the KHCC HB-HTA program received the King Hussein Cancer Research Award for Academic Program Excellence. Dr. Abeer has significantly contributed to the field. She has authored over 20 publications in HTA and Health Economics research, covering areas such as decision analytical modeling, HTA processes and methods, valuation of health outcomes, and Health-Related Quality of Life research.
Dr. Abeer is influential at the national, regional, and international levels in advancing the implementation and development of HTA. She is an active member of numerous HTA steering committees. She is the chair of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) HTA round table for the Middle East and Africa (2024-2026). Currently, Dr. Abeer is appointed by the World Health Organization to serve as a member of the Technical Advisory Group on Pricing Policies for Medicines (TAG-PPM), providing evidence-based technical advice to improve affordable access to essential and priority medicines.
Dr. Abeer holds a BSc in Pharmacy, a Master of Business Administration (MBA) from Jordan University, and a Master of Science (MSc) in International Health Technology Assessment from the University of Sheffield, United Kingdom. She is a PMP-certified project manager and a final-year Ph.D. candidate at UMIT TIROL – University for Health Sciences and Technology, Hall i.T., Austria.
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Saudamini Dabak, MA
Nonthaburi, Thailand
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Claire Telford, MSc, PhD
Pfizer, Gaithersburg, MD, United States
Opportunity Cost, or Opportunity Lost? Sacred Principle, Functional Fiction, or Evolving Concept in HTA?
Session Type: Forums
Topics: Economic Evaluation, Health Technology Assessment
Level: Intermediate
The concept of opportunity cost — the health foregone elsewhere when a new intervention is funded — has been central to HTA methodology for decades, yet agreement on how to measure and apply it remains elusive. Debates over supply-side cost-effectiveness thresholds have generated important methodological advances, but have also exposed the limits of empirical consensus: the assumptions required to translate marginal productivity estimates into operational thresholds are consequential, and the stakeholders most affected by those thresholds have little structural incentive to converge on any single estimate. In this respect, the problem may be as much political-economic as it is methodological. At the same time, there is a growing need and momentum to move beyond health-sector-only value frameworks. Broader value elements - productivity, informal care, social participation, and cross-sectoral spillovers - now feature prominently in research agendas, HTA discussions, and frameworks such as ISPOR's Whole Health approach. This raises a fundamental tension: a narrow opportunity cost frame, anchored in health budget displacement, cannot fully accommodate these broader dimensions; yet widening the frame to cross-sectoral resource allocation makes the empirical challenge substantially harder, not easier. The risk is that in the pursuit of theoretical completeness, practical tractability is lost. The question this panel will interrogate is therefore not whether opportunity cost matters - it does - but whether the current framing is adequate for the decisions HTA is now being asked to support. Methods for estimating broader value have advanced considerably, and imperfect evidence about broader value is not obviously worse than imperfect evidence about narrow displacement costs. What would it mean, in practice, to implement a broader opportunity cost perspective in HTA - one that takes displacement seriously across sectors without abandoning the principled foundations that make HTA defensible? And who should bear the burden of proof?
Moderator
Speakers
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Dan Ollendorf, MPH, PhD
Institute for Clinical and Economic Review (ICER), Boston, MA, United States
Dan Ollendorf, PhD is Chief Scientific Officer and Director of Health Technology Assessment (HTA) Methods and Engagement at the Institute for Clinical and Economic Review (ICER), with responsibility for managing the organization’s internal health economics capacity and program of external collaboration with academic researchers and institutions, as well as collaboration with international HTA bodies and other stakeholders on key process and methods topics. From 2007-2018, Dr Ollendorf was chief scientific officer for ICER, where he oversaw the broader HTA process and managed relationships with multiple stakeholders and research collaborators.
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Nancy J Devlin, PhD
University of Melbourne, Melbourne, Australia
Nancy is Professor of Health Economics at the University of Melbourne and Editor-in-Chief at Value in Health. Her principal areas of research expertise are the measurement and valuation of patient reported health outcomes; the cost effectiveness thresholds used in making judgments about value for money in health care; and priority setting in health care.
Nancy has published >200 original peer reviewed journal articles on a wide range of empirical and theoretical topics in health economics, and is co-author of Economic Analysis in Health Care, a textbook on health economics widely used in the UK and elsewhere. In 2025 Nancy was named in the Stanford/Elsevier ‘top 2% scientists’. In 2024, she was ranked 3rd in the world’s ‘Highly Ranked Scholars’™ in Health Economics based on productivity, impact and research quality. Her work was highlighted in the UK’s NIHR 10-year anniversary report, which noted ‘The impact of her research is worldwide and highly significant in improving health and health care decision making’ . Her research, submitted as a case study to the UK’s 2014 REF exercise, was judged by the sub-panel as ‘demonstrating very considerable impact in terms of reach and significance' .
She was the lead investigator on QUOKKA, a multi-million dollar programme of research on health outcomes in children, funded by the Medical Research Future Fund (Australia) (2020 – 2025), results from which have been described as “…a huge leap forward for the field of health-related quality of life measurement in child health”
She is the elected Chair of the Board of the EuroQol Research Foundation (2020-2025), the European-based not for profit body that developed the EQ-5D, the world’s leading generic measure of patient reported outcomes. Nancy has also served as the 2019/20 elected international President of ISPOR, the peak international professional society for health economics and outcomes research.
Prior to joining the University of Melbourne in 2019, Nancy was Director of Research at the Office of Health Economics, London for ten years, leading the development of its research programme, culminating in OHE achieving Independent Research Organisation (IRO) status in 2019. Prior to OHE she was Professor of Economics at City University of London, where she held the positions of Head of the Economics Department and Dean of Social Sciences. She has 40 years of experience as a researcher and as an advisor to health care organisations, both in the public and private sectors, in the UK and internationally.
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Lotte Steuten, MSc, PhD
Office of Health Economics, London, United Kingdom
Lotte Steuten, PhD Deputy Chief Executive of the Office of Health Economics; former Member, Board of Directors, ISPOR
Prof Lotte Steuten is deputy chief executive of the Office of Health Economics (OHE), the world’s oldest independent health economics research organization, based in London, UK, and a globally recognized expert in health economics and outcomes research (HEOR).
Her research addresses challenges in valuing and paying for innovative therapies, with the aim of achieving effective, accessible, affordable, and efficient healthcare for all. She has published over 150 peer-reviewed papers on topics including the value of novel treatments, diagnostics and prevention for a wide range of non-communicable and infectious diseases.
With 2 decades of experience across Europe, the United States, and Asia Pacific, she advises governments, industry, and other organizations worldwide. She is frequently sought by media and international stakeholders for expert commentary on HEOR, value assessment, health policy innovation, and evolution of health technology assessment globally.
Alongside her position at OHE, Prof Steuten is a visiting honorary professor at City St George’s, University of London. Prior to joining OHE, she held academic faculty positions at the Fred Hutch Cancer Research Center and the University of Washington in the United States. She earned her PhD (with honors) from Maastricht University in the Netherlands.
13:45 - 14:45
AI Applications in Health Technology Assessment
Session Type: Research Podiums
This session aims to evaluate the extent to which emerging use of AI applications (e.g. generative AI) can be useful to support health Technology assessment (HTA). This section identifies and discusses specific issues related to the use of generative AI in health-economic modeling.
SCALING RARE DISEASE INSIGHTS THROUGH AI-DRIVEN PHENOTYPE SEARCH ON FEDERATED NETWORKS
OBJECTIVES: Rare hereditary diseases often face a prolonged "diagnostic odyssey" and significant data scarcity.
This study aims to validate a Longitudinal Patient Similarity (LPS) framework deployed across a federated network of hospitals. The primary objective is to demonstrate that advanced AI can identify high-fidelity patient cohorts suitable for accelerating diagnosis, constructing synthetic clinical trial arms, and enabling federated HEOR analyses.
METHODS: Leveraging the EHDEN network infrastructure, we implemented a federated, privacy-preserving AI framework across multiple hospital sites. Using the OMOP CDM to harmonize electronic health records (EHR), pharmacy, and laboratory data, we applied a vector-similarity AI model. This model was trained on small "seed cohorts" (n=10-22 confirmed patients) to identify undiagnosed individuals by matching their unique clinical trajectories, the chronological sequence and timing of health events, against known disease phenotypes. Validation was conducted retrospectively across 1.2 million patient records and prospectively in routine clinical workflows for five distinct pathologies: Alport Syndrome, Fabry disease, Primary Hyperoxaluria, and Tuberous Sclerosis Complex.
RESULTS: The LPS framework demonstrated robust generalizability across multiple independent hospital sites. In a preselected cohort of 72,611 patients with Chronic Kidney Disease, the model achieved a sensitivity >84% for all disease cohorts. The effectiveness of the screening was confirmed by a clinically manageable Number Needed to Screen (NNS), with a median of 30 patient reviews required to identify one confirmed case. High-similarity cohorts were validated as representative of the target rare disease phenotypes, confirming their suitability for secondary research use cases, such as the generation of large-scale, real-world evidence pools and the construction of synthetic control arms for clinical trial augmentation.
CONCLUSIONS: This study demonstrates that federated RWD networks augmented by AI-driven longitudinal similarity, provide a scalable "safety net" for rare disease diagnosis. Beyond diagnostic utility, these high-fidelity cohorts bridge the gap between rare disease populations and rigorous RWE generation.
VALIDATION OF A GENERAL-PURPOSE AI AGENT (CLAUDE) FOR KAPLAN-MEIER CURVE DIGITIZATION AND PARAMETRIC SURVIVAL EXTRAPOLATION
OBJECTIVES: Survival extrapolation from published Kaplan-Meier (KM) curves is widely used in health-technology assessment (HTA) when individual patient data are unavailable. However, digitisation of KM curves, reconstruction of pseudo-individual patient data (pIPD), and parametric extrapolation are time-consuming and may be affected by analyst-dependent variability. This study evaluated whether a general-purpose artificial intelligence (AI) agent (Claude, Anthropic) could perform this end-to-end workflow and reproduce results comparable to the conventional manual workflow.
METHODS: We analysed 30 trials (60 figures, 118 KM curves). Using Claude, KM curves were digitised, pIPD were reconstructed using the Guyot algorithm, and six standard parametric survival models were fitted and extrapolated over an 80-year horizon. AI-generated results were compared with a reference workflow consisting of manual KM curve digitization, pIPD reconstruction, and parametric survival modelling in R. Agreement was assessed using RMSE, MAE, and maximum vertical deviation (MVD, worst-point survival difference). We additionally compared fitted curves and concordance in best-fitting model selection (AIC, BIC).
RESULTS: AI-derived KM reconstructions closely matched the reference workflow, although visual review remained necessary. Median RMSE and MAE were 0.018 and 0.014 survival probability units, respectively. The median MVD was 0.040, indicating that AI-reconstructed curves typically remained within four percentage points of the reference throughout observed follow-up. Agreement remained high for parametric extrapolations (median RMSE 0.004; median MAE 0.001; median MVD 0.028), although divergence increased over longer extrapolation horizons. The largest discrepancies occurred for monochrome figures in which treatment arms were distinguished only by line style.
CONCLUSIONS: An AI agent was able to reproduce a standard KM digitisation and survival extrapolation workflow with mostly good agreement to a validated R-based approach. While results support the potential of AI to accelerate routine HTA modelling tasks, sensitivity in long-term extrapolation and model selection highlights the need for expert oversight, particularly when source figures have low visual discriminability.
CAN GENERATIVE AI RELIABLY IDENTIFY PAYER DECISIONS AND REPLACE MANUAL HTA REVIEW? A COMPARISON OF AI AND HUMAN EXTRACTION OF A FRENCH HTA REPORT
OBJECTIVES: Despite growing interest in applying GenAI to analyse health technology assessment (HTA) reports which inform market access decision-making, its ability to accurately identify payer decision drivers and the rationale underpinning these decisions remains uncertain.
METHODS: We assessed the performance of three GenAI models (ChatGPT 5.5 Pro, Claude Opus 4.8 Max, and Gemini 3.1 Pro extended) in extracting key payer insights from the French HTA of valoctocogene roxaparvovec in severe haemophilia A using a standardized extraction prompt run twice per model to assess reproducibility. Outputs were assessed against a human reference extraction. Evaluation focused on the accuracy of HTA recommendations, submitted evidence, and identification of decision drivers and their associated rationale. Both qualitative and quantitative differences between GenAI and human extraction approaches were documented.
RESULTS: Compared with human-only extraction, GenAI models offered substantial time savings; however, performance differed in the interpretation of payer reasoning. GenAI models were generally effective at identifying overall HTA conclusions but were less consistent in distinguishing supporting evidence from payer critique and in accurately attributing the rationale underlying positive and negative payer commentary. Variability was observed across models in the completeness and precision of extracted findings. Human review identified omissions, occasional misclassification of payer perspectives, loss of contextual detail, and some instances of hallucinations, all of which could influence the downstream interpretation of HTA outcomes. While GenAI substantially reduced extraction time, human-led extraction remained necessary to ensure accuracy and distinguish high-level conclusions from the specific rationale informing HTA decisions.
CONCLUSIONS: While future use of GenAI-assisted HTA extraction can accelerate evidence review, current models did not consistently capture the full depth of payer reasoning and decision-making context. Human validation remains essential to ensure accurate identification and interpretation of HTA commentary, decision drivers, and supporting rationale.
GOVERNANCE AND SAFEGUARDS TO EVALUATE GENERATIVE AI TOOLS AT NICE
OBJECTIVES: AI has the potential to accelerate health technology assessment (HTA) and guideline development. Adoption requires organisations to move from fully human processes towards workflows in which AI augments decision making. Robust validation is essential to ensure tools are safe, reliable, and suitable for high-stakes environments. Our objective is to describe the internal governance and validation guide developed at NICE to evaluate AI tools, particularly large language models.
METHODS: NICE have developed an internal validation guide supported by a Python-based testing infrastructure hosted within an environment independent from NICE production servers. The guide operates across three levels. First, testing progresses through defined phases from proof-of-concept to shadow testing against real NICE workflows. Second, experiments follow a validation protocol in which development and evaluation datasets are separated, success metrics are predefined, and experiments are executed and documented in reproducible environments using Jupyter notebooks. Third, model optimisation follows a structured iterative process, including prompt design, k-shot prompting, reasoning prompt engineering where appropriate, and parameter setting. Configurations are recorded in a structured format and evaluated using in-house software. Technical safeguards include system prompts to prevent misuse and limiting analyst interaction to only adjusting predefined settings.
RESULTS: Several use cases have been identified that are using this guide, with initial testing evaluating Committee Discussion and Interpretation of the Evidence. The supporting infrastructure includes automated testing, version control and tracking software issues, enabling transparency and technical robustness. The guide was independently reviewed by an external partner to focus on sociotechnical perspectives; in light of the review the guide was updated to include more detail on accountability, responsibility, and consideration for the use of a holdout dataset as part of a prompt test suite.
CONCLUSIONS: NICE's evaluation guide provides a structured approach for safely evaluating AI tools and may offer a model for organisations considering responsible adoption of AI-assisted methods.
Developing qualitative vignettes to explore patient perspectives of meaningful change in clinical outcomes assessment: AI-based and manual methodological applications
Session Type: Workshop
Topics: Patient-Centered Research, Clinical Outcomes, Methodological & Statistical Research
Track: Patient-Centered Evidence
Level: Intermediate
PURPOSE: It is critical to establish meaningful change thresholds for clinical outcomes assessments (COA) used to evaluate key efficacy endpoints in clinical trials for new therapies. Qualitative methods are a complementary approach to quantitative methods, but often require complex scoring algorithms to be presented to patients to seek their perspectives on multi-item measure changes. This workshop will focus on how to generate vignettes to discuss varying levels of change in COA scores with patients, and considerations for implementation and analyses. Participants will generate vignettes and practice exploring changes in outcomes using AI-based and manual methods.
DESCRIPTION: Workshop attendees will obtain a working knowledge of applying vignette-style methods in qualitative interviews to explore meaningful change in COA scores with patients. The workshop moderator, Helen Kitchen, will introduce the topic by providing background on the importance of estimating meaningful change thresholds to interpret clinical trial COA scores, describing methodologies referenced in the FDA Patient-Focused Drug Development (PFDD) Guidance (10 min). One such method, vignettes, has been used by the presenters in multiple qualitative studies to explore patient perspectives in meaningful change. Dr Piper Fromy will describe the triangulation of these qualitative methods and quantitative methods to establish COA interpretation thresholds (10 min). Dr Catherine Bottomley will guide workshop participants in developing their own vignettes to explore meaningful change in complex multi-item COA scores (20 min). Natalie Aldhouse will demonstrate AI-based and manual methods for implementing vignettes in qualitative interviews and conducting real-time analyses (20 min).
This interactive workshop will be valuable to COA researchers working in industry, academic, and clinical settings who are interested in using qualitative methods to explore meaningful change thresholds in complex COA scores.
Moderator
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Helen Kitchen, MSc
Clarivate, London, United Kingdom
Speakers
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Piper Fromy, PhD
Saumur, France
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Catherine Bottomley, PhD
Clarivate Analytics, London, United Kingdom
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Natalie Aldhouse, MSc
Nottingham, United Kingdom
Train the Economist or Hire the Engineer? The Future of Expertise in AI-Native HEOR
Session Type: Issue Panel
Topics: Organizational Practices, Economic Evaluation, Real World Data & Information Systems
Track: AI
Level: Intermediate
ISSUE: Artificial intelligence (AI) is moving from experimental use to routine application across HEOR, including evidence synthesis, economic modeling, real-world evidence, comparative effectiveness research, and value communication. While attention focus is heavily placed on software capabilities, a fundamental challenge has emerged: who is qualified to lead AI-enabled HEOR workflows? Should HEOR professionals acquire technical expertise to direct and govern AI analyses, or should AI data scientists be cross-trained in health economics, epidemiology, and clinical evidence evaluation? The answer impacts scientific quality, organizational structure, and the baseline identity of the HEOR profession.
OVERVIEW: This panel explores competing approaches to building the next generation of AI-native HEOR capabilities. Ramiro Gilardino will frame the structural debate, focusing on the friction between advanced engineering agility and the deep methodological rigor required for healthcare decision-making. Jaime Caro will argue that robust HEOR necessitates expert mastery of causal inference, economic evaluation, and evidence synthesis, meaning AI tools must be adopted safely under the strict stewardship of grounded domain specialists. Manuel Cossio will counter that sophisticated multi-agent AI architectures and automated analytical workflows demand native data science expertise, making the training of technical specialists in core HEOR principles the only scalable path forward. Mitch Higashi will provide an institutional leadership perspective, detailing future-ready competency frameworks, dual-mentorship governance models, and collaborative workforce strategies that move past binary either-or approaches. Through moderated debate and real-time audience polling, this interactive session outlines non-delegable competencies and details how global organizations must prepare for an AI-integrated future.
Moderator
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Ramiro E Gilardino, MSc, MD
Insights & Impact, Zurich, Switzerland
I lead the access and policy strategies that enable innovative medicines to reach patients across Europe, Latin America, and global markets. With 16+ years at the intersection of HTA, market access, and health policy, I have led access strategy in some of the most complex regulatory environments in the industry, including EU Joint Clinical Assessment, IRA, and evolving global pricing scrutiny, helping organizations anticipate and navigate change across launch and lifecycle.
I focus on oncology, rare disease, and specialty portfolios. I have supported 20+ global product launches and built market access capabilities from the ground up, translating complex policy environments into actionable pricing, reimbursement, and evidence strategies.
I bring a combination of clinical training, health economics expertise, and policy fluency, operating at the executive level across industry, consulting, and global institutions. My work includes shaping HTA frameworks and access environments through board roles at ISPOR, advisory engagement with the World Health Organization and World Bank, and 40+ publications in leading HEOR, HTA, and policy journals.
Speakers
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Jaime Caro, PhD
Boston, United States
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Manuel Cossio
Cytel Inc. Dubendord, Zürich, Switzerland; Universitat de Barcelona, Dubendorf, Switzerland
AI Engineer and Head of AI Solutions at Cytel with 13+ years of experience in HEOR. I lead the development of AI-driven solutions for evidence generation, economic modeling, and HTA landscaping—including EU JCA and market access. With expertise across both pharma and CRO consulting, I’m committed to advancing patient care through smarter, AI-enabled decision-making.
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Mitch Higashi, PhD
ISPOR, Villanova, PA, United States
New Forces Shaping Global Pricing
Session Type: Research Podiums
This session explores how US Most Favored Nation pricing, the EU Joint Clinical Assessment, and China’s 2026 drug pricing reforms are reshaping global launch strategies, evidence requirements, and access pathways. It will also examine innovative value-based pricing approaches to balance affordability, equitable access, and incentives for pharmaceutical innovation.
BEYOND INCOME TIERS: AN ADAPTIVE VALUE-BASED PRICING FRAMEWORK TO RECONCILE ACCESS, EQUITY, AND INNOVATION FOR ON-PATENT MEDICINES IN LMICS
OBJECTIVES: This study characterizes the limitations of conventional tiered pricing and develops a capacity-calibrated, dynamically re-priced value-based pricing framework that aligns prices with local health value while remaining feasible across varying LMIC institutional capacities.
METHODS: We conducted a structured review of peer-reviewed and grey literature published between 2003 and 2025 using PubMed, Scopus, ISPOR/Value in Health, WHO, BMJ, and Lancet sources. Evidence related to pharmaceutical pricing, value-based pricing, HTA, and market access in LMICs was synthesized alongside comparative analysis of tiered pricing, external reference pricing, and managed-entry agreements. Drawing on opportunity-cost threshold theory and adaptive HTA principles, we developed an AVBP framework and assessed its components against affordability, equity, budget impact, and implementation feasibility criteria.
RESULTS: Core failures of conventional tiered pricing: arbitrary income-band stratification, weak alignment of price with local value, and limited purchaser bargaining power-excluding middle-income populations through binary high/low tiers. Components of the capacity-calibrated, dynamically re-priced AVBP framework:
-Anchors launch price to country-specific, opportunity-cost willingness-to-pay thresholds.
-Adds disease-burden and budget-impact ceilings to safeguard population-level affordability.
-Embeds iterative re-pricing via outcomes-based managed-entry agreements and confidential rebates.
-Calibrates implementation to national HTA/data maturity, using adaptive-HTA shortcuts where infrastructure is nascent.
-Stress-testing suggested that AVBP could improve affordability and equity while maintaining manufacturer incentives through value- and volume-contingent pricing across varying levels of HTA capacity.
CONCLUSIONS: AVBP offers a feasible, value-anchored evolution beyond static tiered pricing, reconciling affordability, equity, and innovation incentives-a transparent, capacity-sensitive negotiation tool for LMIC policymakers and HTA agencies, and predictable, value-justified market entry for manufacturers. The essential next step is prospective pilot implementation across diverse LMIC settings to validate the framework under real-world conditions.
FROM PRICE COMPRESSION TO LIFECYCLE VALUE MANAGEMENT: IMPLICATIONS OF CHINA'S 2026 DRUG PRICING REFORM
OBJECTIVES: China’s drug pricing system is undergoing a major transformation following April 2026 State Council guidance on drug price formation. This study assesses how the reform shifts pricing logic across the product lifecycle and implications for pricing predictability, evidence requirements, and innovative drug access.
METHODS: A structured policy and literature review was conducted using April-June 2026 national guidance, policy commentary, and secondary analyses. The reform was assessed against the historical framework across three dimensions: (1) launch pricing formation, (2) post-launch price evolution and NRDL integration, and (3) evidence requirements and governance mechanisms.
RESULTS: Historically, China’s P&R system has been characterized by free pricing at launch followed by significant price compression through NRDL negotiations, with reported price reductions of ~60% post-NRDL reimbursement. The 2026 reform introduces a more structured, value-based launch pricing approach. A unified quantitative scoring framework (170-point scale) classifies products into three tiers, aligning innovation level, clinical value, and pricing flexibility. This shifts the system from reactive price correction toward front-end value-based price setting. At a system level, the reform reduces pricing fragmentation by moving from province-level processes toward coordinated national mechanisms, with expected reductions in time to listing and improved launch predictability. Pricing is increasingly managed over the lifecycle rather than at a single timepoint. High-value products may benefit from multi-year price stability and potential upward reassessment based on real-world evidence, while lower-value or competitive products remain subject to ongoing price erosion. This indicates a more explicit balancing of innovation reward versus long-term cost containment.
CONCLUSIONS: China’s 2026 reform represents a strategic shift from front-loaded price compression toward lifecycle-based value governance. While it may improve early pricing visibility and differentiate true innovation, it also requires sustained value demonstration across the lifecycle. Success will increasingly depend on integrating launch pricing, evidence generation, and reimbursement strategy into a coordinated, long-term approach.
MOST-FAVORED-NATION PRICING POLICIES AND THE GLOBAL PRICE CASCADE: IMPLICATIONS FOR DIFFERENTIAL PRICING AND EQUITABLE ACCESS IN LOW- AND MIDDLE-INCOME COUNTRIES
OBJECTIVES: Most-favored-nation (MFN) pricing policies, increasingly adopted in high-income markets, are typically evaluated for their domestic cost-saving effects alone. This analysis examines how MFN provisions interact with external reference pricing (ERP) networks to affect differential pricing sustainability and vaccine access in low- and middle-income countries (LMICs).
METHODS: A conceptual framework was developed incorporating three dimensions: health system financing structure, institutional procurement architecture (Gavi, PAHO, and Global Fund versus commercial channels), and the strategic pricing flexibility, a calibration window, that ERP systems have historically preserved. The framework was applied to three vaccine markets, pneumococcal conjugate (PCV), human papillomavirus (HPV), and malaria, using documented list- and procurement-price data.
RESULTS: MFN provisions eliminate the calibration window manufacturers use to sustain differential pricing, producing upward price cascades through ERP chains. Middle-income countries face compounded exposure: ineligible for donor-subsidized prices, yet losing the flexibility ERP previously preserved. PCV pricing shows a roughly six-fold gap between Gavi and PAHO middle-income prices (US$2 vs. US$12.83 to 14.50 per dose); HPV shows the steepest gradient (US$4.50 to 96.32 per dose) with a post-graduation cost cliff exceeding US$10 million annually; malaria vaccine pricing reveals an analogous donor-financed cascade (US$0.20 to 9.30 per dose across tiers), with inter-donor competitive benchmarking documented in a 2025 manufacturer price reduction.
CONCLUSIONS: PPP-based analyses of MFN effects are insufficient: differential pricing sustainability instead depends on health system financing structure, institutional procurement architecture, and the strategic pricing flexibility that MFN provisions eliminate. Middle-income countries, caught between donor and commercial pricing tiers, bear the greatest equity burden. MFN policy design should incorporate explicit income-level carve-outs protecting Gavi, PAHO, and Global Fund procurement from comparator baskets. LMIC health ministries should engage proactively in international pricing governance to safeguard the differential pricing architectures on which equitable access depends.
US MOST FAVOURED NATION PRICING AND EU JOINT CLINICAL ASSESSMENT: A CEE-WEIGHTED PULSE CHECK ON LAUNCH STRATEGY, EVIDENCE GENERATION AND ACCESS RISK
OBJECTIVES: To assess how market access professionals perceive global launch, pricing and evidence-generation implications of two concurrent policy shifts: the US Most-Favoured-Nation (MFN) pricing framework and EU Joint Clinical Assessment (JCA).
METHODS: An online pulse-check survey was conducted from 14 May to 23 June 2026 among market access, HEOR, commercial and leadership professionals. Twenty-six responses were analysed descriptively as a global industry readout with a Central and Eastern Europe (CEE)-weighted case study, as 69% were CEE-based. Likert items were reported as top-2-box agreement; free text was reviewed thematically. AI-assisted tools supported survey design and analysis; outputs and interpretations were reviewed and approved by the authors.
RESULTS: Respondents reported moderate MFN awareness (58% somewhat, 15% very familiar). 69% reported MFN impact varying by drug portfolio; 58% reported potential reference-basket expansion was already influencing pricing outside baskets; 69% were more cautious about list prices and 54% reported accelerating confidential pricing. The leading perceived MFN risk was upward price pressure reducing patient access (42%), followed by launch delays (35%). JCA was perceived less as simplification than evidence-system shift: 62% agreed it would increase launch complexity and cost, 58% that organisations were investing earlier in integrated evidence generation, and 58% that it would strengthen smaller-market HTA capacity; only 31% agreed JCA would enable simultaneous EU launches (versus 38% disagreeing). The future role of smaller/lower-GDP markets remained contested (35% more important; 31% no change; 23% less important). CEE respondents were directionally more optimistic than non-CEE respondents on non-basket launch attractiveness and future market importance.
CONCLUSIONS: This global, CEE-weighted pulse check suggests MFN and JCA are prompting reassessment but not decisive resequencing. The central tension is whether MFN’s affordability-driven pricing pressure and JCA’s harmonisation-driven evidence requirements may reshape launch incentives in ways that increase pricing opacity, evidence demands and access uncertainty in smaller markets.
Are HTA and Health Funding Systems Failing to Incentivise Investment in Disease Prevention?
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Epidemiology & Public Health, Health Technology Assessment
Track: Expanded Value Measures
Level: Introductory
ISSUE: European health systems consistently state that prevention is a priority, yet funding and evaluation arrangements historically prioritise treating disease. Prevention interventions generate benefits over long time horizons across sectors. These benefits may not be fully recognised within existing HTA and funding structures, which typically assess value based on direct patient outcomes and within annual spending cycles. As a result, prevention may be supported in principle but disadvantaged in practice. This panel will examine the drivers of underinvestment in prevention and debate how HTA methods, policy and funding arrangements need to evolve to realise the long-term, cross-sector and societal value of preventive interventions.
OVERVIEW: Alex Porteous will highlight dissonance between European commitments to disease prevention, limited funding dedicated to preventive care, and the continuing clinical and economic burden of preventable illness. Alex will argue that progress will continue to be limited without conceptual and structural shifts in HTA and health funding systems that incentivise investment (10 minutes).
Grace Hampson will discuss the economics of prevention, including challenges associated with economic evaluation, funding, and adoption of preventive health interventions, followed by opportunities to promote investment and uptake through innovative financing. An International Policy Speaker will argue that prevention should be viewed through the lens of capital investment and economic development, examining governance barriers, budget silos and short political time horizons that drive underinvestment, and highlighting the need for integrated governance models and international coordination. James Jarrett will highlight how these challenge manifest in practice, outlining challenges in the evaluation of HIV prevention.
Moderator
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Alex Porteous, BA, MSc
Costello Medical, London, United Kingdom
Speakers
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Grace Hampson
Office of Health Economics, London, United Kingdom
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James Jarrett, BA, MA, PhD
Gilead Sciences Inc, London, United Kingdom
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Rosanna Tarricone, PhD
Bocconi University, Rome, Italy
Beyond the Chinese Market: How China-Generated Evidence is Reshaping Global HTA, Access, and Reimbursement Decisions
Session Type: Issue Panel
Topics: Real World Data & Information Systems, Health Technology Assessment, Health Policy & Regulatory
Track: Real-World Evidence (RWE)
Level: Intermediate
Issue
China’s biopharmaceutical ecosystem has undergone a profound transformation over the past decade, emerging as a significant contributor to global drug discovery, clinical development, and real-world evidence generation. Increasing licensing agreements between multinational companies and Chinese innovators, alongside the growing participation of Chinese sites in multinational studies, have expanded the visibility of data originating from China in regulatory, reimbursement, and access discussions beyond its borders.
This evolution raises important questions for HTA bodies, payers, and industry decision-makers. Under what circumstances can evidence generated in one healthcare setting inform decisions in another? How should stakeholders assess the relevance of clinical and real-world data derived from Chinese populations when evaluating effectiveness, value, and uncertainty? As healthcare systems face mounting pressure to accelerate patient access while maintaining rigorous evidence standards, these questions are becoming increasingly consequential.
Overview
This issue panel will examine the growing influence of evidence generated in China on global health technology assessment, reimbursement, and access decisions. Drawing on perspectives from industry, academia, and HTA stakeholders, the discussion will explore how evolving research capabilities, regulatory reforms, and expanding data infrastructure are shaping evidence packages used to support market access across regions.
Panelists will discuss opportunities to leverage data generated in China to address evidence gaps, improve development efficiency, and support decision-making throughout the product lifecycle. Equally, the session will consider ongoing challenges related to transferability, methodological expectations, population heterogeneity, and jurisdiction-specific evidence requirements. Through a balanced and forward-looking discussion, the panel will explore whether the emergence of China as a major evidence-generation hub is reshaping global approaches to assessing value, managing uncertainty, and enabling timely patient access to innovation.
Moderator
Speakers
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Lizheng Shi, PhD
Tulane University School of Public Health and Tropical Medicine, New Orleans, LA, United States
Lizheng Shi, PhD, MsPharm, MA, is the Neal A. and Mary Vanselow Endowed Chair in the Department of Health Policy and Management at the School of Public Health and Tropical Medicine of Tulane University. He is the founding director of Tulane’s Health Systems Analytics Research Center (HSARC). Dr. Shi’s current health services research interest focuses on innovative health technologies to improve healthcare quality, access, and cost of patient-centered care from the equity perspective, using pharmaco-economics, health technology assessment, health analytics, and policy evaluation. Dr. Shi is dedicated to disseminating and translating population health knowledge at the local, national, and international levels. He is the associate editor of Value In Health and co-editor in chief for Pharmacoeconomics and Policy.
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Minghuan Jiang, PhD
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Le Gao
Xi'an JiaoTong University, China
Parallel Joint Scientific Consultations under the EU HTA Regulation: What Have We Learned from the First Procedures?
Session Type: Issue Panel
Topics: Health Technology Assessment
Track: Access and Pricing
Level: Intermediate
Issue
Joint Scientific Consultations (JSCs) are designed to facilitate early dialogue between health technology developers (HTDs), HTA bodies, regulators, patients and healthcare professionals. By enabling discussion on evidence generation plans early on, JSCs aim to support more informed decision-making and contribute to the generation of evidence relevant to multiple stakeholders.
The first parallel JSC procedures involving both HTA bodies and regulators have now been completed. The experience gained raises a number of important questions. How useful was the advice provided through parallel consultations? To what extent did the process influence evidence generation strategies and development plans? How were patient and clinical perspectives integrated into the discussions? What operational, methodological or procedural challenges were encountered by participants? Most importantly, what lessons can be drawn to strengthen future JSC procedures?
For the first time, stakeholders directly involved in parallel JSCs can move beyond expectations and discuss real-world experience. This Issue Panel will explore what has been learned from the first procedures and how the JSC framework should evolve to maximise its value for all participants.
Overview
The moderator will introduce the role of JSCs within the EU HTA framework and the objectives of parallel consultations involving HTA bodies and medicines regulators. Industry representative will share their practical experience, highlighting both the value generated by the process and the challenges encountered when translating advice into evidence generation strategies. Representatives from HTA bodies will reflect on the strengths of the process, key lessons learned and opportunities for future improvement. Patient organisations involved will discuss the contribution of patient and clinical expertise to early evidence planning. The panel will conclude with an interactive discussion among speakers and audience members on how the JSC framework can continue to evolve as experience accumulates, ensuring that it delivers meaningful value for developers, decision-makers, healthcare professionals and patients.
Moderator
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Michael Berntgen
Amsterdam, Netherlands
Speakers
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Karina Hansen, PhD
NOVARTIS, Basel, Switzerland
Head of HEOR &HTA, International at Novartis since 2024. 25 years of global and international experience within the Pharmaceutical Industry in senior management roles within HEOR, HTA, OR/RWE and Access at AbbVie, Allergan, and Lundbeck, building and developing high performing teams and individuals. PhD in Public Health & Health Economics from Paris XI, France. MSc in Economics, Copenhagen University, Denmark.
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Stephanie Said, PhD
Berlin, Germany
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DIMITRIOS ATHANASIOU, MBA
World Duchenne Organization, ARGIROUPOLIS, Greece
Delay or Prepare? Navigating EU HTA in the Era of MFN-Driven Launch Uncertainty
Session Type: Issue Panel
Topics: Health Technology Assessment, Health Policy & Regulatory, Organizational Practices
Track: Access and Pricing
Level: Intermediate
ISSUE
Global pricing pressures, including Most Favored Nation (MFN) and intensified reference pricing (ERP), are reshaping pharmaceutical launch strategies. Companies are delaying or deprioritizing European launches to protect global pricing corridors. At the same time, the EU Joint Clinical Assessment (JCA), introduces a mandatory centralized clinical evaluation requiring early preparation on populations, comparators, endpoints, and evidence generation.
This creates a strategic dilemma for manufacturers: How much should companies invest in JCA readiness when European launch is uncertain or delayed?
The issue extends beyond operational readiness and raises broader questions around launch sequencing, portfolio prioritization, patient access, and interaction between global pricing policies and EU HTA requirements.
OVERVIEW
This panel will provide a practical framework to guide JCA investment decisions under MFN uncertainty and evolving global pricing dynamics. The discussion will focus on how different levels of JCA and payer evidence preparation may influence pricing lifecycle management, launch sequencing, evidence investment and HTA strategy, and time to patient access.
The session will introduce a three-level readiness framework: Compliance Only – minimal JCA preparation with risk of delayed access or weaker positioning, Targeted Readiness – focused preparation balancing pricing protection and access readiness, and Full Readiness approach – comprehensive preparation to optimize future JCA and pricing outcomes
Panelists will consider these approaches across three strategic scenarios: short-term MFN dominance, transitional/selective European launch strategies, and long-term rebalancing if MFN pressures decline.
The panel will specifically debate: business risks of under-preparation, evidence investment across portfolios and geographies, whether “minimum viable JCA readiness” is sufficient, and how delayed launches affect patient access and equity. The session will be particularly relevant for global market access, HEOR, pricing, and HTA professionals navigating evolving EU and US policy dynamics.
Moderator
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Eric C Faulkner, MPH
Passage Health Associates, Durham, NC, United States
Speakers
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Joerg Ruof, MBA
Basel, Switzerland
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Jon D Campbell, MS, PhD
National Pharmaceutical Council, Washington, DC, United States
Jon D. Campbell, MS, PhD, is the Chief Science Officer (CSO) of the National Pharmaceutical
Council (NPC), which conducts policy-relevant research and communicates it with impact.
As CSO, Dr. Campbell leads NPC’s research agenda in concert with NPC’s board and research team.
After training at the University of Washington, he joined the faculty at the University of Colorado Anschutz Medical Campus, earning multiple appointments in Pharmacy and Public Health. Dr. Campbell is an author of over 350 manuscripts and abstracts in the field of health economics and health policy research. He holds an adjunct faculty appointment within the Center for Evaluation of Value and Risk in Health at Tufts University School of Medicine.
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Gregory Daniel, MPH, PhD
Eli Lilly & Co. Ltd., Washington, DC, United States
Gregory Daniel, PhD, MPH is Vice President, Head of Policy Innovation, Evidence & Engagement at Eli Lilly and Company. Greg has led the Global Public Policy group, responsible for evaluating and developing public policy concepts in areas such as prescription drug access, affordability and innovation, global value-based healthcare, health equity, and other high priority policy areas. Greg also leads policy evidence development and external engagement, developing partnerships with academic groups, think-tanks, and other policy research organizations to advance policies that support innovation, access and affordability. He is also currently the Editor-in-Chief of Therapeutic Innovation and Regulatory Science (TIRS), the scientific journal of DIA. Greg also serves on the Board of Directors and is Past President for the Innovation in Value Initiative (IVI) Foundation, is Adjunct Associate Professor at the Eshelman School of Pharmacy at the University of North Carolina-Chapel Hill and is Visiting Scholar at the Center of Innovation in Regulatory Science at the Duke-National University of Singapore.
Prior to joining Eli Lilly, Greg held the positions of Head of US Healthcare Policy at Edwards Lifesciences; Deputy Center Director and Clinical Professor at the Duke-Margolis Center for Health Policy; Fellow and Managing Director in the Center for Health Policy at the Brookings Institution; and Vice President, Government and Academic Research at Carelon (formerly HealthCore, subsidiary of Elevance).
Greg received his PhD in pharmaceutical economics, policy and outcomes from the University of Arizona, and an MPH focused in biostatistics, MS, and BS in Pharmacy all from The Ohio State University.
Modern Synthesis Methods for Estimating Subgroup-Specific Treatment Effects
Session Type: Workshop
Topics: Methodological & Statistical Research, Health Technology Assessment, Study Approaches
Level: Intermediate
PURPOSE: Individual clinical trials are rarely powered to detect treatment effect modifiers, leaving subgroup analyses underpowered and prone to spurious findings. Combining information across trials makes identifying such subgroups more feasible. The canonical example is tamoxifen in breast cancer: individual trials were too small to determine whether the drug worked differently by oestrogen receptor status, but the EBCTCG patient-level meta-analysis demonstrated this convincingly, reshaping clinical practice. Identifying which patients benefit most is central to coverage and reimbursement decisions, yet available data vary widely — from full individual participant data (IPD) to aggregate subgroup summaries only. Suitable methods require careful adjustment to the setting at hand. DESCRIPTION: Dr. Remiro-Azócar will frame the problem: why subgroup-specific estimates matter for HTA, why subgroup analyses from individual trials are surprisingly unreliable, and why evidence synthesis offers a path forward (7 min). Dr. Jansen will present the network meta-analysis framework for subgroup analysis, including shrinkage estimation to stabilize uncertain subgroup-specific estimates, network meta-regression with aggregate and individual participant data, and how models can be set up to minimize ecological bias when patient-level effect modifiers are only available as study-level summaries (13 min). Renato Panaro will address subgroup-specific meta-analysis using aggregate subgroup data, showing how standard subgroup meta-analysis can disagree with interaction meta-analysis when subgroup contributions vary across studies, and presenting contribution-adjusted methods that resolve this inconsistency (15 min). Dr. Campbell will introduce new methods for leveraging subgroup-level summary data — frequently reported but typically ignored — within population-adjusted network meta-analysis (15 min). The session will conclude with a scenario involving a treatment network with varying data availability. The audience will be asked, via live polling, to identify appropriate methods, required assumptions, and to interpret results considering key sources of bias (10 min).
Moderator
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Antonio Remiro-Azócar, BSc, MSc
Speakers
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Jeroen Jansen
United States
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Renato Panaro
Germany
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Harlan Campbell
Precision AQ, Rossland, BC, Canada
Methods for Health State Utility Estimation: Task Framing, Current Practice, and Future Directions in HTA
Session Type: Other Breakout Session
Topics: Methodological & Statistical Research, Health Technology Assessment, Patient-Centered Research
Track: Patient-Centered Evidence
Level: Intermediate
Health state utility (HSU) estimation underpins cost utility analysis, yet methodological choices are often poorly aligned with the underlying analytic objective. This workshop brings together academic and industry perspectives to examine how HSUs are conceptualised and estimated, and the implications for health technology assessment (HTA).
We begin by outlining the importance of clearly framing HSU estimation as a descriptive, predictive, or causal task (Dr?Franklin). This framing has major implications for study design, data needs, interpretation, and use within decision models. Using philosophical arguments and directed acyclic graphs (DAGs), we show how lack of clarity leads to inconsistent practice and potentially biased model inputs. We will also present findings from an umbrella review of applied HSU estimation approaches, synthesising methods used across disease areas and data sources. From 185 primary studies identified across 19 systematic reviews, we highlight substantial variability in methodological choices, justification, and reporting standards.
We then examine current HTA practice and guidance gaps, drawing on a review of HTA submissions and findings from a Delphi study conducted by a NICE External Assessment Group (Prof?Lee). We find that inconsistent, poorly reported, and methodologically variable analyses of patient level utility data in NICE appraisals create avoidable uncertainty, and we summarise consensus based recommendations to support more robust and standardised modelling.
Finally, we discuss practical challenges from an industry perspective (Dr?Lloyd), including concerns that EQ 5D may not fully capture lived experience of many conditions, leading companies to feel that important aspects of treatment value are overlooked. We also highlight the persistent tension between methodological rigour, feasibility, timelines, and cost; particularly when early real world data collection is encouraged, despite the high likelihood that many products will not reach the market.
The session will give attendees a clearer conceptual framework for HSU estimation and practical insights to improve methodological consistency and decision relevance.
Moderator
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Matthijs M Versteegh, BSc, MA, PhD
Huygens & Versteegh, Zwijndrecht, Netherlands
Speakers
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Dawn Lee, MSc
University of Exeter PenTAG, Exeter, United Kingdom
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Matthew Franklin, BA, MSc, PhD
SCHARR, University of Sheffield, Sheffield, United Kingdom
My research has broadly focussed on estimating the comparative cost-effectiveness of different health and social care interventions using economic evaluation methods both within-study and for modelling-based analyses. I am particularly interested in the conduct of economic evaluations that use real-world data to support trials and observational studies. I have a keen interest in the philosophies of data science tasks, such as working out what we want to estimate, why, and to inform whom, and then working through if this is a description, prediction, or causal inference task. By understanding these philosophies, we can better work through the appropriate methods for the task at hand. I am particularly interested in tasks when causal inference is of interest, which is often the key interest in economic evaluation. My University of Sheffield staff page and publication list can be found here: https://sheffield.ac.uk/smph/matthew-franklin
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Andrew Lloyd, DPhil
Acaster Lloyd, London, United Kingdom
14:45 - 15:15
Coffee and Connect
Session Type: General Meeting
Head to the exhibit hall to connect with fellow attendees and exhibitors over a steaming cup of coffee.
15:15 - 16:15
Using Patient Centered Digital Endpoints to Measure Whole Health
Session Type: Forums
Topics: Patient-Centered Research, Clinical Outcomes, Medical Technologies
Level: Intermediate
Instead of treating a specific disease/symptom, whole health focuses on restoring health, promoting resilience, and preventing disease across the lifespan. ISPOR notes that Whole Health expands the definition of value to ask if evidence captures the full range of outcomes and costs recognizing the physical, behavioral, socioeconomic, environmental and spiritual dimensions of health.
A key component of whole health is that it prioritizes outcomes of importance to individuals while promoting well-being for all. Digital devices can measure biomarkers such as physical function, speech, eye movement, and metabolic and physiological markers which can inform endpoints that matter to patients, e.g. ability to walk without stopping to catch a breath.
This forum will explore three perspectives on how digital technologies may offer opportunities to deliver on the concept of whole health by measuring not only whether an intervention treats disease, but whether it improves people’s functioning, well-being, equity, self-management, and life context in ways that are clinically meaningful and economically relevant.
The first speaker will address the concept of whole health focusing on the payer perspective. What aspects have payers accepted as digital endpoints and how can these be integrated into HTA. The second speaker will suggest co-creation with patients of digital endpoints may support the user’s ability to function, participate, manage daily life, and experience well-being within their real social and environmental context. The third speaker will ground the discussion in practice, presenting a real-world example of a digital health technology whose digital mobility outcomes capture meaningful, real-time measures that support the whole health concept.
This session convenes experts who will share experiences and offer solutions to align expectations of the promise of digital technologies to incorporate Whole Health across patient research and payer perspectives.
Moderator
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Annette Champion, BSc, MBA
Healthcare Research Insights, Inc, Lake Forest, IL, United States
President/Founder Healthcare Research Insights, Inc., Lake Forest, IL, providing strategic research and consulting services to pharmaceutical and biotech companies, focusing on development and communication of clinical and HEOR evidence to support product licensing, development, launch, and new indications. Co-lead of ISPOR Digital Health SIG PICOTS-ComTeC project. Previously, Abbvie/Abbott Director of Clinical Epidemiology, Director of Scientific Communications, Metabolics and Hypertension Therapeutic Executive Committee member, Director Knoll Cardiovascular Products, International HEOR Group founder; Aventis/RPR Director of Socioeconomics; Arthur D. Little Health Economics & Healthcare Policy lead.
Speakers
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Anita D Burrell, BA, MA, MBA
Anita Burrell Consulting LLC, Flemington, NJ, United States
Anita Burrell is a dynamic, dedicated professional with extensive knowledge of health economics and global market access for pharmaceutical products as well as a regular speaker in the industry.
Anita has led teams to success across a wide variety of roles and geographies. At Sanofi, she headed Global Health Economics, Worldwide Pricing and Reimbursement and was an R&D champion bringing Aubagio, the oral MS therapy, to market ahead of schedule. She has served on the HBA Board of Directors and led the ISPOR Special Interest Group for Digital Health
As a consultant Anita has helped companies to:
Understand market dynamics and payer behavior across many diseases including immunology, cardiovascular, oncology, hematology and endocrinology in the top 11 markets including Europe, Japan and the US
Design integrated evidence plans to demonstrate robust demonstration of product benefits for faster market uptake
Establish credibility for a Global Market Access Insights function through thought leadership, strategic vision and producing three product lines within 9 months
Understand the Global Evolution of the Biosimilar Market and prepare for the future implications in Europe and the United States
Anita holds a BA (Hons) Economics from the University of Stirling, an M.A. Economics from Dalhousie University, Nova Scotia, Canada, and an MBA from Kingston University, Surrey UK. She speaks French at an advanced level.
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Anke-Peggy Holtorf, MBA, PhD
Health Outcomes Strategies, GmbH, Basel, Switzerland
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Nadine Jamous
ZimmerBiomet, Burnham, United Kingdom
Generative AI for Systematic Literature Reviews: ISPOR Good Practices Task Force Findings
Session Type: Forums
Topics: Health Technology Assessment, Methodological & Statistical Research, Study Approaches
Level: Introductory
PURPOSE: Systematic literature reviews (SLRs) are foundational to evidence-based medicine, health technology assessment (HTA), yet they remain labor-intensive and resource-demanding. Generative AI (GenAI) tools are increasingly being applied across SLR workflows, with early studies demonstrating promise for screening, data extraction, and report drafting. However, key concerns persist regarding hallucinated outputs, reproducibility, prompt sensitivity, and variable performance across tasks. This workshop will share the findings and recommendations of the ISPOR Task Force on GenAI for SLRs, equipping attendees to responsibly integrate GenAI into evidence synthesis while preserving research integrity. DESCRIPTION: This forum presents the final findings and recommendations of the ISPOR Good Practices Task Force on GenAI for SLRs. The Task Force conducted a rapid evidence assessment identifying 115 studies (November 2022--July 2025) and applied a structured assessment framework across eight evaluation domains to seven core SLR tasks: search strategy development, title/abstract screening, full-text screening, data extraction, risk of bias assessment, qualitative summarization, and report writing. Task Force members will present task-level performance summaries, recommended accuracy metrics, and consensus-based good practice recommendations. Key findings indicate that GenAI can augment--but not replace--human expertise. The strongest evidence supports title/abstract screening and structured data extraction within recall-oriented, human-in-the-loop workflows. Interpretive tasks such as risk of bias assessment demonstrated the lowest readiness for GenAI integration. Autonomous deployment was not supported for any SLR task. The forum will present the task force recommendations addressing: (1) appropriate and unsupported uses of GenAI by SLR task; (2) required human oversight and accountability safeguards; (3) recommended performance metrics for each task; and (4) reporting and transparency standards aligned with the ELEVATE- GenAI framework. The session will close with a facilitated audience discussion.
Moderator
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Dalia Dawoud, BSc, MSc, PhD
Cytel, London, United Kingdom
Dalia Dawoud, PhD, is Research Principal, HTA Policy and Strategy. She is also the Director and CEO of PEHTA Consulting Ltd. and holds a professor position at the Faculty of Pharmacy, Cairo University. She has over 15 years experience as a health economist and researcher. Her work is largely focused on the application of HEOR in HTA and clinical guideline development. She worked at leading organizations including NICE, where she led a portfolio of HORIZON Europe projects such as HTx, EDiHTA and SUSTAIN HTA, and the Royal College of Physicians, London. She is widely published in the areas of health economics and outcomes research and serves as associate editor for Value in Health and as director on ISPOR Board of Directors (2023-2026). She is also a member of the ISPOR AI Working Group and ISPOR Living HTA Working Group.
Speakers
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Jag Chhatwal, PhD
Harvard Medical School / Massachusetts General Hospital, Boston, MA, United States
Jag Chhatwal, PhD, is the director of the Institute for Technology Assessment at Massachusetts General Hospital and an associate professor at Harvard Medical School. He also serves as core faculty at the Center for Health Decision Science, Harvard T.H. Chan School of Public Health. Dr. Chhatwal has co-authored more than 125 original research articles and editorials in leading peer-reviewed journals. His research has informed health policy decisions at prominent organizations including the White House, the World Health Organization, and the CDC, and has been featured in major media outlets such as CNN, Forbes, National Public Radio, The New York Times, and The Wall Street Journal. Dr. Chhatwal serves as an associate editor of Value in Health and as guest editor for its special issue on artificial intelligence. He is also a member of the ISPOR Generative AI Working Group.
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Raphael Sonabend-Friend
NICE, London, United Kingdom
Dr Raphael Sonabend-Friend is a Scientific Adviser in the Science, Evidence and Analytics team at the National Institute for Health and Care Excellence (NICE). Raphael supports the testing of artificial intelligence, particularly focused on generative artificial intelligence methods. Raphael holds a PhD in the accessible and transparent use of machine learning in healthcare settings. Raphael has over a decade of experience in the healthcare sector ranging from large philanthropies, small local charities, governmental bodies, and private sector consulting for UK and international organisations.
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Sven L Klijn, MSc
Bristol Myers Squibb, Princeton, NJ, United States
Sven Klijn is Director at Bristol Myers Squibb in the Global HEOR Evidence Acceleration & Innovation group, where he leads the innovative modeling agenda in hematology and cell therapy. In addition, Sven has an active role in providing modeling and Generative AI education at international congresses. He has widely published on innovative methods, especially in the fields of survival extrapolation and Generative AI. Sven has a training in public health and health economics and previously had various roles in CROs related to health-economic modeling.
From Evidence to Enterprise Value: Positioning HEOR in the Boardroom
Session Type: Forums
Topics: Organizational Practices
Level: Introductory
HEOR leaders are increasingly asked to translate value concepts to C-suite leaders and external audiences who may be less familiar with technical details. Additionally, executives often prioritize financial impact, strategic positioning, and shareholder value, which requires HEOR leaders to adapt their communication strategies. The session will focus on the language and framing necessary to connect HEOR to business initiatives, while emphasizing cross-functional collaboration across any organization.
The session will begin with an overview of the different perspectives and the importance of communication, framing, and cross-departmental engagement to strengthen the business case for HEOR evidence (10 minutes, Craig). This will be followed by a discussion across of variety of focus areas including how to effectively communicate and contextualize HEOR outputs to the c-suite, aligning HEOR outputs with broader strategic imperatives to ensure resonance in the boardroom, important communication tools, and tangible examples of HEOR creating business value. Panelists include Craig Roberts (Merck), Goncalo Jesus (Viiv), Robyn Carson (Abbvie), Paulo Carita (Sanofi), Clark Paramore (Biogen). An audience Q&A will follow.
Audience members will leave with actionable strategies for translating HEOR into terms that internal executives and non-HEOR external audiences understand, thereby strengthening organizational understanding of HEOR and demonstrating the function as a critical driver of business value.
Moderator
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Clark Paramore, MSPH
Biogen, Cambridge, MA, United States
Speakers
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Craig Roberts, MBA, PharmD
Merck, North Wales, PA, United States
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Gonçalo Jesus
ViiV Healthcare, London, United Kingdom
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Robyn Carson, MPH
AbbVie, Morris Plains, NJ, United States
Robyn T. Carson, MPH, is Vice President & Head of Patient-Centered Outcomes Research and HEOR-Strategy Aesthetics where she leads teams focused on integrating the patient voice through generation of patient experience data (PED) and ensuring deployment of best practices for clinical outcome assessment (COA) development, validation, and implementation across therapeutic areas at AbbVie. Robyn has driven patient-centered research in the pharmaceutical industry for 19 years where she has held roles in of increasing responsibility at Pfizer, Forest Labs, Actavis, Allergan and AbbVie. During her career, Robyn has made significant contributions to major product approvals and launches, as well as the development of multiple novel patient-reported outcome (PRO) instruments and innovative real-world research platforms. In addition, Robyn has led key departmental and enterprise-wide initiatives related to patient-focused drug development (PFDD) and patient-centricity. Robyn has also been a leader within the cross-industry Critical Path Institute PRO Consortium since 2008, currently serving as the Industry Co-Director. Prior to joining the pharmaceutical industry, Robyn conducted research at Columbia University, NYC Department of Health & Mental Hygiene, and served as a Research Fellow at the National Cancer Institute. Robyn holds a MPH in Epidemiology from Columbia University.
Vaccination Coverage an opportunity lost: Are European Health Systems Leaving Health Benefits on the Table?
Session Type: Issue Panel
Topics: Epidemiology & Public Health, Health Policy & Regulatory, Health Service Delivery & Process of Care
Track: Expanded Value Measures
Level: Intermediate
ISSUE: Vaccination remains one of the most cost-effective public health interventions available, yet coverage rates across Europe fall consistently below WHO targets for influenza, COVID-19, and childhood immunisation. Hesitancy, access barriers, and entrenched delivery models leave substantial at-risk populations, including minority communities, elderly adults, and vulnerable groups, systematically underreached. Innovative delivery strategies exist. Pharmacist-led programmes have demonstrated improved uptake and favourable cost-effectiveness across European settings. Community-based outreach has shown measurable impact where traditional channels fail. Yet these approaches remain inconsistently embedded in national immunisation programmes, and HTA frameworks have been slow to evaluate non-traditional delivery models with the same rigour applied to vaccines. The core issue is whether European health systems have the HTA tools and political will to adopt and scale innovative vaccination strategies, or whether structural inertia leaves proven solutions outside the funded care pathway.
OVERVIEW: This panel examines how innovative vaccination delivery strategies can be embedded within European public health systems. Moderator Cornelis Boersma will introduce the issue (5 min), followed by three perspectives: cost-effectiveness of pharmacist-led vaccination (Adina Turcu-Stiolica), national immunisation policy in a non-EU context (Aleksandra Grozdanova), and the patient perspective from current research (Tarik Catic), each presenting for 10 min.
HTA professionals, payers, public health academics, and patient advocates will benefit from a moderated debate (15 min) on three unresolved questions:
-Should HTA frameworks evaluate vaccination delivery models, not just vaccines, as reimbursable interventions, and what evidence standards apply to pharmacist-led programmes?
-What governance mechanisms are needed for non-EU systems to adopt and scale evidence-based vaccination delivery innovations?
-How should patient perspectives on vaccination barriers be incorporated into HTA evaluations of innovative delivery strategies?
The session concludes with audience Q&A (10 min).
Moderator
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Cornelis Boersma, PhD
Health-Ecore, Zeist, Netherlands
Speakers
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Adina Turcu-Stiolica, PhD
University Of Medicine And Pharmacy Of Craiova, Craiova, Romania
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Aleksandra Grozdanova
Skopje
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Tarik Catic, PhD
Sarajevo, Bosnia And Herzegovina
Inside the EMA: A Bird's-Eye View of Europe's Medicines Regulator
Session Type: Forums
Topics: Health Policy & Regulatory
Level: Introductory
Europe's medicines regulator is operating in an increasingly complex environment. Implementation of the EU Health Technology Assessment Regulation, rapid advances in artificial intelligence, geopolitical uncertainty, evolving expectations for scientific transparency, and growing pressure on public institutions are reshaping the landscape in which regulatory decisions are made. In this exclusive conversation, EMA Chief Medical Officer Steffen Thirstrup will provide a rare inside view of how the European Medicines Agency is responding to these challenges while remaining committed to its core mission of protecting public health through scientific excellence. Interviewed by Laura Pizzi, Chief Science Officer of ISPOR, Dr. Thirstrup will discuss the Agency's strategic priorities, how leadership is balancing finite resources against expanding responsibilities, and the opportunities that lie ahead for regulatory science in Europe. The conversation will explore how the EMA is implementing the EU HTA Regulation and strengthening collaboration with HTA bodies, while also addressing broader issues that will shape the future of medicines regulation—including the responsible integration of artificial intelligence, maintaining public trust in science, responding to an increasingly complex geopolitical environment, and fostering international collaboration with partners such as the US Food and Drug Administration (FDA) and the International Council for Harmonisation (ICH). Designed as an interview rather than a technical update, this session offers attendees an opportunity to hear directly from one of Europe's foremost regulatory leaders about the strategic decisions shaping the Agency today and the future of medicines regulation tomorrow. The conversation will conclude with audience questions, allowing participants to engage directly with Dr. Thirstrup on the issues defining the next era of regulatory science.
Moderator
Speakers
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Steffen Thirstrup, PhD, MD
European Medicines Agency (EMA), Amsterdam, Netherlands
Steffen Thirstrup is a medical doctor and board-certified specialist in clinical pharmacology and therapeutics. He holds a PhD in pharmacology and has a long background in clinical internal medicine with special emphasis on adult respiratory medicine. Additionally, Dr. Thirstrup was appointed adjunct professor in pharmacotherapy at the Faculty of Health Sciences, University of Copenhagen, in 2012.From 2004-09 Steffen Thirstrup worked at Danish Medicines Agency first as the Danish member of CHMP at the European Medicines Agency (EMA) for five years including 10 months as joint CHMP- and CAT-member, followed by a short period as head of Danish Institute for Rational Pharmacotherapy dealing with HTA and best practice guidelines for primary care. In 2011 Prof. Thirstrup rejoined the licensing division at the Danish Medicines Agency acting as Head of Division for Medicines Assessment and Clinical Trials. During this period Prof Thirstrup co-chaired the European Commission’s working group on market access for biosimilars medicinal products and acted as key scientific contact for the managing entity of the IMI beneficiaries for the PROTECT collaboration (Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium).
In March 2013, Prof Thirstrup joined the pharmaceutical consultancy company NDA Group AB as a full-time medical advisor on NDA’s regulatory advisory board. In April 2014 Prof Thirstrup was appointed as director for the Regulatory Advisory Board at NDA Regulatory Services Ltd.
Since June 2022 Prof Thirstrup has been the Chief Medical Officer at the European Medicines Agency, Amsterdam, The Netherlands
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Julia Chamova
ISPOR, LIMHAMN, Sweden
Shaping the Global Research and Policy Agenda in HEOR in the Next Decade: A Value in Health Regional Issues Forum
Session Type: Forums
Topics: Study Approaches, Economic Evaluation, Health Technology Assessment
Level: Introductory
Health economics and outcomes research (HEOR) has matured substantially over the past decades, generating robust methodological standards and well-defined research priorities in jurisdictions with long-established health technology assessment (HTA) systems. However, this maturity is not evenly distributed across the globe. In many regions—including Asia Pacific, Latin America, Africa, the Middle East, and Eastern Europe—the research and policy agenda for HEOR remains far less defined. Identifying which topics most urgently require attention and upskilling in these settings is therefore a pressing and largely unresolved question.
This question is of particular relevance to Value in Health Regional Issues (VIHRI), a journal that since its inception has served as a global platform for scientific dissemination, giving voice to regional perspectives often underrepresented in mainstream HEOR literature. This forum aims to bring together leading voices in the field to discuss and debate the priority research and policy topics that should define the HEOR agenda over the next decade, with particular attention to the needs of emerging and diverse healthcare systems worldwide.
The insights generated during this session will directly inform the journal's editorial strategy, guiding the development of new theme sections and content priorities. For the audience, this forum represents a valuable opportunity to identify potential avenues for contribution, including the submission of original research in the thematic areas emerging from the discussion.
Espinoza will open the session with a brief overview of the journal's recent development and growing regional footprint and will moderate the debate. Steuten, Villanueva, and Wu will contribute their perspective on priority topics and methodological gaps, including global policy leadership, regional editorial experience, and cross-regional scientific perspective, fostering a dynamic and multi-angle discussion. The session will conclude an open Q&A that encourages audience members to share their own views on the emerging global HEOR agenda.
Moderator
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Manuel Antonio Espinoza, MSc, PhD, MD
Hong Kong, Hong Kong
Speakers
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Lotte Steuten, MSc, PhD
Office of Health Economics, London, United Kingdom
Lotte Steuten, PhD Deputy Chief Executive of the Office of Health Economics; former Member, Board of Directors, ISPOR
Prof Lotte Steuten is deputy chief executive of the Office of Health Economics (OHE), the world’s oldest independent health economics research organization, based in London, UK, and a globally recognized expert in health economics and outcomes research (HEOR).
Her research addresses challenges in valuing and paying for innovative therapies, with the aim of achieving effective, accessible, affordable, and efficient healthcare for all. She has published over 150 peer-reviewed papers on topics including the value of novel treatments, diagnostics and prevention for a wide range of non-communicable and infectious diseases.
With 2 decades of experience across Europe, the United States, and Asia Pacific, she advises governments, industry, and other organizations worldwide. She is frequently sought by media and international stakeholders for expert commentary on HEOR, value assessment, health policy innovation, and evolution of health technology assessment globally.
Alongside her position at OHE, Prof Steuten is a visiting honorary professor at City St George’s, University of London. Prior to joining OHE, she held academic faculty positions at the Fred Hutch Cancer Research Center and the University of Washington in the United States. She earned her PhD (with honors) from Maastricht University in the Netherlands.
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Rafael Pinedo-Villanueva, BA, MSc, PhD
University of Oxford, Oxford, United Kingdom
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Jing Wu, RPh, PhD
Tianjin University, Beijing, China
Prof. Wu has dedicated nearly 20 years to research and teaching in health economics and pharmacoeconomics. Her expertise spans health policy evaluation, pharmacoeconomic evaluation, real-world data analysis, and health-related quality of life and health utility study. She earned her PhD in Pharmacoeconomics through a joint program between Shenyang Pharmaceutical University and Peking University in 2007 and was a visiting scholar at the Schaeffer Center for Health Policy and Economics at the University of Southern California in 2013. Prof. Wu has authored over 100 high-impact scientific articles, led three National Natural Science Foundation of China projects, contributed to multiple pharmacoeconomics textbooks, and spearheaded the development of the China Guidelines for Pharmacoeconomic Evaluations (2020) and the Companion Guide to the China Guidelines for Pharmacoeconomic Evaluations (2022), promoting the development and standardization of pharmacoeconomics in China. She currently serves as Chair of the Pharmacoeconomics Committee of the Chinese Pharmaceutical Society, Vice Chair of the Pharmacoeconomics Committee of the China Health Insurance Research Association, Vice Chair of the Pharmacoeconomics Committee of the Chinese Society of Research Hospitals. She co-chaired the ISPOR Asia-Pacific Conferences in 2018 and 2022, served as Executive Chair of the 10th Huaxia Pharmacoeconomics and HTA Forum in 2019, and founded the China Young Scholars Forum on Pharmacoeconomics in 2017—organizing around 40 high-level academic meetings to date. As a national-level advisor, she provides strategic guidance to China’s National Healthcare Security Administration, playing a key role in reimbursement policy and healthcare-system reform.
16:00 - 16:45
Access and Pricing Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 4, Posters will be hung from 16:00-19:00.
Posters featured in this tour:
PT31: FORECASTING UK BRANDED MEDICINES SPEND UNDER VPAG: A MACHINE-LEARNING MODEL INTEGRATING NHS PRESCRIBING AND COMMERCIAL SALES DATA
PT32: CRITICAL ASSESSMENT OF PRICING PATHWAYS AND REGULATORY CLASSIFICATION OF ATMPS APPROVED IN BRAZIL
PT33: HOW NEW GLOBAL PRICING & ACCESS POLICIES COULD RESHAPE PRICE EROSION IN EUROPEAN MARKETS
PT34: PAYMENT DESIGN AS A BARRIER TO PERITONEAL DIALYSIS IN MOROCCO: A PROVIDER-MARGIN FRONTIER AND PD-FIRST BUDGET-IMPACT ANALYSIS
PT35: MITIGATION STRATEGIES FOR US INTERNATIONAL REFERENCE PRICING: A QUALITATIVE ASSESSMENT OF POLICY AND MANUFACTURER RESPONSES
PT36: POTENTIAL MEDICARE PART B SAVINGS FROM ACCELERATED BIOSIMILAR ADOPTION
Student Research Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 4, Posters will be hung from 16:00-19:00.
Posters featured in this tour:
PT37: ESTABLISHING THE VALUE OF HOSPITAL ELECTRONIC MEDICAL RECORD DATA IN THE HEALTH TECHNOLOGY ASSESSMENT AND REASSESSMENT PROCESS FOR ONCOLOGY: HEPATOCELLULAR CARCINOMA IN THE CHANG GUNG RESEARCH DATABASE AS A CASE STUDY
PT38: SOCIETAL COSTS IN DUTCH PHARMACEUTICAL REIMBURSEMENT DOSSIERS: EVIDENCE QUALITY AND IMPACT ON COST-EFFECTIVENESS OUTCOMES OF PRODUCTIVITY LOSSES AND INFORMAL CARE USE
PT39: COST-UTILITY ANALYSIS OF REMOTE ISCHEMIC CONDITIONING FOR ACUTE MODERATE ISCHEMIC STROKE BASED ON THE RICAMIS TRIAL
PT40: ASSESSING CROSS-COUNTRY AGREEMENT IN HTA RATINGS: THE CASE OF PORTUGAL AND FRANCE
PT41: EVALUATING THE IMPACT OF FRANCE'S DEROGATORY DIRECT ACCESS SCHEME:INSIGHTS FROM THE PUBLIC HOSPITALS OF PARIS (AP-HP) FRAMEWORK
PT42: PATTERNS AND OUTCOMES OF ADVERSE EVENTS ASSOCIATED WITH HYDROCEPHALUS SHUNTS: EVIDENCE FROM THE MAUDE DATABASE
Moderator
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Nga Weng Leong, BSc, MSc
University of Mississippi, Oxford, MS, United States
16:00 - 19:00
Poster Session 4
Session Type: Research Posters
Poster Tours 16:00–16:45 | Presenters will be with their posters from 18:00–19:00
16:45 - 17:45
How to Unlock the Usage of Real-World Data for Medical Devices in Health Technology Assessments?
Session Type: Issue Panel
Topics: Medical Technologies, Real World Data & Information Systems, Health Technology Assessment
Track: Real-World Evidence (RWE)
Level: Intermediate
ISSUE: Medical devices present specific assessment challenges because of their heterogeneity, frequent product updates, dependence on context of use, and potential to alter care pathways. Demonstrating their clinical and economic value is therefore often more complex and less standardized than for other health technologies.
Real-world data (RWD) and the resulting real-world evidence (RWE) can help address these challenges by showing how devices perform in routine clinical practice, including effectiveness, safety, and patterns of use across broader patient populations than those typically included in pre-market studies.
Despite the growing use and submission of real world data and evidence, why do HTA bodies still often consider it insufficient for decision making?
This session will explore three questions: What criteria do decision makers use to assess the relevance of RWD for medical devices? Which types of RWD are most useful across the technology lifecycle? What solutions could strengthen the RWD/RWE ecosystem and improve its acceptability for HTA?
OVERVIEW: Medical devices require a tailored and continuous approach to assessment because of their clinical, economic, and organizational impacts. While RWD/RWE can contribute meaningfully, HTA for medical devices is not systematic across all European countries and is not always directly linked to decision-making. The EU HTA Regulation, which includes high-risk medical devices from June 2026, aims to better align clinical assessments across Member States. In parallel, the EHDS Regulation may facilitate secondary use of health data and improve its fitness for purpose.
The moderator will open with a 10-minute overview, followed by a 40-minute panel discussion and 10 minutes for Q&A audience.
This session will be of particular interest to medical device industry representatives, HTA stakeholders, and HEOR researchers.
Moderator
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Frederique Debroucker
Medtronic, Paris, France
Speakers
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Raphael Sonabend-Friend
NICE, London, United Kingdom
Dr Raphael Sonabend-Friend is a Scientific Adviser in the Science, Evidence and Analytics team at the National Institute for Health and Care Excellence (NICE). Raphael supports the testing of artificial intelligence, particularly focused on generative artificial intelligence methods. Raphael holds a PhD in the accessible and transparent use of machine learning in healthcare settings. Raphael has over a decade of experience in the healthcare sector ranging from large philanthropies, small local charities, governmental bodies, and private sector consulting for UK and international organisations.
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Jana Boleckova, PhD
Edwards Lifesciences Sàrl, Prague, Czech Republic
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Daniel Prieto-Alhambra
Oxford, United Kingdom
Addressing the Challenges in Rare Disease Methods
Session Type: Research Podiums
This session highlights multiple areas of challenge in generating robust evidence for value assessment in rare diseases and provides examples of how these challenges play out in varying access contexts (conditional access agreements, joint clinical assessment and national-level HTA).
STATISTICAL LESSONS FROM EARLY EU JOINT CLINICAL ASSESSMENTS AND IMPLICATIONS FOR RARE-DISEASE HTA ACROSS EU-25: THE ROLE OF EARLY AUTOMATION IN IDENTIFYING EVIDENCE GAPS
OBJECTIVES: The EU Joint Clinical Assessment (JCA) sets harmonised expectations for comparative evidence, indirect treatment comparisons (ITCs), and uncertainty analysis. As orphan medicines enter scope from 2028, concerns persist about alignment between JCA methodological requirements and the statistical characteristics of rare disease evidence, which often relies on small samples, heterogeneous populations, and real world evidence (RWE). This study assessed alignment using lessons from early JCA experience and evaluated how early stage tools can identify evidence gaps, ITC feasibility issues, and dataset requirements.
METHODS: A targeted review was conducted of HTA Coordination Group (HTACG) methodological guidance, the 2026 Implementation Report, JCA reports, and withdrawal cases. EU‑25 HTA methodological guidance updates (2024-2026) were examined to identify expectations relevant to rare disease evidence. Statistical issues were mapped across comparator selection, ITC feasibility, effect modifier handling, and uncertainty quantification. An R Shiny application was developed to highlight where additional evidence may be needed and summarise requirements.
RESULTS: Early JCA signals showed recurring issues with comparability and evidence structure, including disconnected networks and effect modifier imbalances limiting feasibility of traditional ITCs. Misaligned or infeasible comparators, weakly justified comparisons, and immature survival data contributed to substantial uncertainty. Two withdrawals in 2026 followed requests for improved comparator justification, deeper exploration of effect modifiers, or RWE triangulation. Limited updates to national methodological guidance highlight uncertainty around alignment between EU JCA and HTA decisions. Applied to a rare disease pilot (SMA), the R Shiny application enabled identification of effect‑modifier constraints, assessment of ECA or NMA feasibility, and highlighted where additional data or RWE would be required.
CONCLUSIONS: Early JCA experience highlights statistical misalignment between JCA expectations and feasible evidence generation in rare diseases. Early stage automation can strengthen planning by identifying evidence gaps and data needs, supporting more efficient translation of JCA outputs into national HTA across EU‑25.
RACING AGAINST RARITY: CAN EVIDENCE GATHERED BETWEEN ENTRY AND EXIT OF CONDITIONAL ACCESS AGREEMENTS RESOLVE UNCERTAINTY IN RARE DISEASES?
OBJECTIVES: For interventions with plausible potential to be cost-effective despite clinical and/or economic uncertainty, NICE managed access agreements (MAAs) and SMC ultra-orphan evidence generation periods provide interim access conditional on additional data collection to resolve uncertainties. Evidence generation in rare diseases is constrained by small populations, less conventional study designs and poorly established outcomes, among other challenges. Therefore, we explored whether conditional access periods allow key evidence gaps to be addressed to support routine reimbursement in rare diseases.
METHODS: NICE highly specialised technology (HST) MAA exit appraisals and SMC ultra-orphan reassessments published between January 2022 and May 2026 were identified; appraisals with accessible entry and exit documentation were selected. Key conclusions on changes to the evidence base between entry and exit were extracted.
RESULTS: Eight NICE HST and SMC ultra-orphan exit appraisals were identified. Two SMC and three NICE exit appraisals with corresponding conditional access entry documentation were selected for analysis, with a mean duration of 50 months (range: 10−80) between entry and exit. At entry, principal uncertainties were the lack of evidence for long-term treatment effects and implications for modelling long-term disease progression. Real-world evidence, collected as part of the conditional access or conducted separately, largely reduced the uncertainties. Committees explicitly noted uncertainty remained around long-term treatment effects in two appraisals, although this did not prevent routine reimbursement. Only one appraisal was not approved for routine reimbursement at exit, resulting from commercial considerations rather than persisting uncertainty.
CONCLUSIONS: Only one of five re-appraisals did not achieve routine reimbursement at conditional access exit, suggesting that uncertainty can sufficiently be resolved within current conditional access timelines for rare diseases. Positive re-appraisals were generally based on new or longer-term data becoming available. However, the high rate of positive re-appraisal suggests conditional access agreements may be preferentially applied to technologies already expected to achieve routine reimbursement.
EU JOINT CLINICAL ASSESSMENT OF TOVORAFENIB: METHODOLOGICAL LEARNINGS FOR FUTURE ORPHAN DOSSIERS
OBJECTIVES: To review and evaluate the tovorafenib JCA report to identify key learnings for evidence generation and dossier development for future JCA orphan submissions.
METHODS: We conducted a structured analysis of the published JCA report in June 2026 to identify methodological challenges related to PICO definition, evidence availability and identification, and indirect treatment comparisons (ITCs).
RESULTS: Of eight PICOs defined across three patient populations, six contained no comparative data and one was excluded by the assessors for abstract-only comparator evidence; only one MAIC was assessed. In terms of ITCs the following issues were identified: the PV/EM identification was insufficiently documented with three unadjustable confounders; both sensitivity scenarios increased risk of bias by using subsets of the base-case adjustment PV/EMs; the ITC SAP designated no primary analysis, rendering all p-values nominal; both unmeasured-confounding sensitivity tools were excluded because of a shifted null hypothesis without pre-specification, inappropriate odds-ratio approximations, and E-values using point estimates rather than confidence interval bounds contrary to cited good practice thereby downplaying unmeasured confounding. The HTD's post-hoc attempt to qualify its own submitted PFS ITC based on limitations in study comparability was not accepted. Although carer input was documented it was not clearly linked to assessment scope or conclusions, while several outcomes were unreported or omitted due to missing PICO 5 data or methodological issues.
CONCLUSIONS: Key learnings for future orphan submissions include the following: prospective ITC SAP must include hypothesis testing; PV/EM identification must be fully documented; sensitivity analyses should address uncertainty; and pivotal trial design should align with anticipated comparator studies. JCA may be more valuable when assessing medicines supported by large randomised controlled trials with head-to-head comparisons, established endpoints and accepted comparators. Applying the same comparative framework and methodological rigour to orphan drugs raises the likelihood of exposure to broad critique.
WHEN UTILITY DATA ARE SCARCE: METHODS AND ACCEPTABILITY IN NICE RARE DISEASE APPRAISALS
OBJECTIVES: In rare diseases, the collection of robust health-related quality of life (HRQoL) data is often challenging due to small and geographically dispersed patient populations. Furthermore, many rare diseases involve cognitive impairment or complex symptom profiles, limiting the feasibility and validity of preference-based instruments and necessitating the use of proxy or vignette-based approaches. The objective of this study was to examine how health state utilities have been sourced or estimated for rare diseases when conventional utility data are limited or unavailable in National Institute for Health and Care Excellence (NICE) technology appraisals (TAs) and highly specialised technologies (HSTs). This included assessing the extent to which different approaches were accepted or critiqued, and identifying factors influencing preferred methods.
METHODS: TAs and HSTs published in the last 10 years (June 2016 to mid-June 2026) were reviewed. Only appraisals of rare diseases, defined according to the European Medicines Agency (EMA) orphan designation criteria were included. Rare oncology, haematological, and immunological conditions were excluded. Withdrawn or terminated appraisals were not considered. Committee papers were reviewed, and approaches used to estimate patient HRQoL were further analysed.
RESULTS: A total of 48 appraisals were selected, of which 22 were TAs and 26 were HSTs. In general, the number of appraisals between 2016 and 2026 increased over the years, with the lowest number assessed in 2018 and 2020 (n=1) and the highest in 2023 (n=10). Trial-based utility data were available in most TAs, whereas HSTs more frequently relied on alternative approaches due to limited data availability.
CONCLUSIONS: In rare disease NICE appraisals, limitations in HRQoL data necessitate the use of diverse utility estimation approaches. The acceptability of these approaches depends on the availability of supporting evidence and the extent to which they are validated and triangulated. These findings highlight the need for greater methodological guidance on utility estimation in rare diseases.
Advances in Decision Analytics and Economic Evaluation
Session Type: Research Podiums
Advances in decision analytics continue to shape the methodological foundations of health economic evaluation. This session presents innovative approaches to decision modelling, uncertainty analysis, expert elicitation, value of information analysis, and economic evaluation methods. The presentations address methodological challenges commonly encountered in health technology assessment while proposing practical analytical solutions. Together, they demonstrate how methodological innovation can support more transparent, efficient, and evidence-informed healthcare decisions.
JOINT ESTIMATION OF MULTI-STATE TRANSITION PROBABILITIES USING KAPLAN-MEIER SURVIVAL CURVES
OBJECTIVES: Clinical trials often report results using Kaplan-Meier (KM) survival curves measuring progression-free survival (PFS) and overall survival (OS). For settings with multiple health states, such as cancer, survival curve data typically includes events which happen in subsequent health states. However, since trials do not typically report whether deaths occur pre- or post-progression, available methods are inadequate to estimate state transition probabilities and hazard rates using only OS and PFS KM survival curves. Our goal is to introduce a publicly accessible method which fills this gap.
METHODS: We analyze the structure of KM survival curve data in the context of multi-state settings to probe why traditional methods are lacking. We then develop a general methodological approach to jointly estimate multi-state transition probabilities using KM survival curves. Using this general approach, we develop a flexible and simple hands-on form that can be used by the public in an open-source statistical software program.
RESULTS: We find that underlying issue is that the structure of typically available KM survival curve data, even with infinitely large samples, is consistent with a range of parameter combinations. We describe this so-called partial identification problem. We then develop and describe a general method to solve it to jointly estimate state transition probabilities using PFS and OS Kaplan-Meier survival curves. We also develop and describe a specific and flexible ready-to-use functional form, implementing it in an R package which allows users to estimate multi-state transition probabilities from KM survival curves and visualize results.
CONCLUSIONS: Estimating state-specific parameters is necessary settings such as modelling sequential treatment paths, but current methods are often restrictive or otherwise lacking. We describe the underlying problem and using this knowledge, we develop a methodological approach and software implementation allowing researchers to jointly estimate multi-state transition probabilities from Kaplan-Meier survival curve data, enabling more flexible and accurate multi-state modelling.
EXPLORING STATISTICAL UNCERTAINTY OF HEALTH INTERVENTION EFFECTS IN DISCRETE CHOICE EXPERIMENTS: A FLU VACCINE CASE STUDY
OBJECTIVES: Inputs for attributes and attribute levels in preference studies, such as estimates of risks and effectiveness, are subject to various kinds of statistical uncertainty. To date, such uncertainty is rarely incorporated, and it remains unclear whether and how it can be operationalized. This study investigates the effect of incorporating statistical uncertainty into a discrete choice experiment (DCE) on comprehension and study outcomes.
METHODS: A DCE measuring preferences for flu vaccination among elderly (aged 60 or older) individuals in the Netherlands (n=526) served as a case study. The DCE included five attributes: vaccine effectiveness, risk of mild side effects, risk of severe side effects, protection duration, and incubation time. Statistical uncertainty was incorporated for the attribute ‘vaccine effectiveness’ only. Mean vaccine effectiveness levels of 20%, 30%, 60% and 70% were described numerically and graphically, with statistical uncertainty expressed as ranges of +/-5%, +/-10% and +/-15%. Multinomial logit and latent class models were used for the preference analyses, and comprehension, perceived understanding, and use of heuristic decision-making were investigated.
RESULTS: Respondents generally understood the mean vaccine effectiveness, but many had difficulty interpreting or applying the exact uncertainty ranges. Overall, higher mean effectiveness was associated with higher utility, whereas disutility was associated with higher statistical uncertainty. Incorporating statistical uncertainty also increased respondents’ attention to the vaccine effectiveness attribute, with fewer respondents ignoring this attribute, but also led to increased dominant decision-making.
CONCLUSIONS: Incorporating statistical uncertainty into DCEs is feasible and could be valuable in situations in which attributes are considered as uncertain. Future DCEs, especially those used to inform market forecasts or policy decisions as well as clinical context, should consider including statistical uncertainty in the design of their attribute levels to closely mimics real-world decision-making.
DECISION RULES FOR MULTI-TREATMENT RECOMMENDATIONS BASED ON NET MONETARY BENEFIT, FOR RISK-NEUTRAL AND RISK-AVERSE DECISION-MAKERS
OBJECTIVES: Healthcare evaluations often use the expected value (EV) of net benefit (NB) to recommend a single best treatment, under the risk neutrality assumption. However, recommending multiple-treatment options may be desirable, and risk-averse decision-makers may wish to consider uncertainty. The loss-adjusted expected value (LaEV) and Grading of Recommendations Assessment, Development and Evaluation (GRADE) approaches have been proposed to account for uncertainty and make multi-treatment recommendations based on a single efficacy outcome. We aimed to extend the EV approach to multi-treatment recommendations, and adapt LaEV and GRADE approaches to NB-based decisions.
METHODS: We took a 2-stage approach to EV multi-treatment decisions. The first stage identifies the optimal treatment. At the second stage, all treatments superior to the reference are compared with the optimal treatment within a minimally important difference (MID) in NB (MID-NB), which is calculated from MID in quality-adjusted life years (MID-Q). LaEV follows a similar approach but penalises EV by the expected loss due to decision-making under uncertainty. GRADE involves multiple stages where treatments are excluded until no treatment exceeds a threshold probability of being superior by MID-NB to at least one other treatment in the recommendation set. We applied and compared these methods using one hypothetical example and three cost-effectiveness models with multiple options, where disease-specific MID-Q is derived from a rapid review.
RESULTS: The LaEV approach recommends the same treatments or fewer than EV, as high uncertainty options are excluded. Depending on circumstances, GRADE may recommend more treatments than EV, potentially excluding the highest-NB treatment. GRADE results can depend on the selection of the reference treatment. All approaches are sensitive to MID-NB, but this remains to be further explored in future research.
CONCLUSIONS: Within the NB framework, the LaEV approach is conservative and reliable for risk-averse decision-makers, who have the flexibility to set a MID-NB threshold that reflects their risk tolerance.
MAKING VALUE-OF-INFORMATION TRACTABLE FOR PATIENT-LEVEL SIMULATION: A MACHINE-LEARNING EMULATOR FOR PROBABILISTIC ANALYSIS AND EVPPI OF DISCRETE-EVENT MODELS
OBJECTIVES: Patient-level and discrete-event simulation (DES) models capture heterogeneity and event-history dependence that cohort Markov models cannot, but their runtime makes full probabilistic sensitivity analysis (PSA) and value-of-information (VOI) computation - particularly EVPPI/EVSI - frequently infeasible. We developed and validated a machine-learning emulator (surrogate model) that makes these analyses practical.
METHODS: Using a space-filling (Latin hypercube) design over the model's input distributions, we ran the DES at 1,000 parameter sets and trained emulators (Gaussian process regression and gradient-boosted trees) to predict incremental costs, QALYs and net benefit. Predictive accuracy was assessed by out-of-sample cross-validation and probabilistic calibration/coverage. The validated emulator then generated the full PSA, cost-effectiveness acceptability curves, EVPI and EVPPI, with EVPPI cross-checked against established non-parametric regression methods (GAM; GP/INLA). Case study: a patient-level DES of treatment sequencing in metastatic colorectal cancer.
RESULTS: Emulators predicted incremental net benefit with out-of-sample R²=0.98 and 95% prediction-interval coverage of 94%. Full PSA requiring ~38 hours of direct simulation (10,000 runs) was reproduced by the emulator in under 10 minutes; emulator-based EVPPI agreed with GAM/GP reference estimates within 3%, while nested Monte Carlo EVPPI on the native model was computationally infeasible (estimated >2 months). Predictive accuracy degraded only in the extreme tails of time-to-event parameters.
CONCLUSIONS: A validated ML emulator reproduces patient-level simulation outputs with high fidelity and cuts the cost of rigorous uncertainty and value-of-information analysis by orders of magnitude, bringing EVPPI/EVSI within reach for complex individual-level models. This supports better-targeted research prioritisation and generalises across disease areas and model types.
Most Favoured Nation Pricing: Will International Price Alignment Improve Affordability or Restrict Access to Innovation? Insights from Central and Eastern Europe
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment, Health Service Delivery & Process of Care
Track: Access and Pricing
Level: Introductory
**Issue**
Most Favoured Nation (MFN) pricing policies and international price alignment mechanisms are increasingly shaping pharmaceutical pricing and reimbursement discussions worldwide. Governments view these approaches as tools to improve affordability and strengthen negotiating power. However, concerns are growing that reliance on international price benchmarking may create unintended consequences for patient access, innovation incentives, and global launch strategies.
Central and Eastern Europe (CEE) provides a particularly relevant case study. Due to widespread use of external reference pricing, many CEE countries are highly sensitive to pricing decisions made elsewhere. As manufacturers face pressure to coordinate prices across markets with different economic capacities, lower-priced countries may experience delayed launches and reduced access to innovative medicines.
**Overview**
This issue panel will examine whether MFN-style pricing policies can achieve affordability objectives without undermining access to innovation. Using CEE as an example of a region particularly exposed to international pricing dynamics, panelists representing global market access, HTA, healthcare policy, and payer perspectives will debate the implications of international price alignment for affordability, equity, innovation, and healthcare sustainability.
The discussion will explore whether international price benchmarking improves market efficiency and affordability or whether it discourages manufacturers from launching products in lower-income countries and contributes to access inequalities. Each panelist will deliver a 10–15-minute presentation, followed by a moderated discussion and audience debate. The session will identify practical approaches to balancing affordability with sustainable access to innovative therapies.
Moderator
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Roman Casciano, MS
Certara USA Inc., Mamaroneck, NY, United States
Speakers
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Marcin Czech, MBA, PhD, MD
Institute of Mother and Child, Warsaw, Poland
Marcin Czech is a full professor and head of the Department of Pharmacoeconomics at the Institute of Mother and Child in Warsaw, postgraduate courses director at Warsaw University of Technology, Business School, Past President of ISPOR, Poland Chapter, former Undersecretary of State/ Vice Minister at the Ministry of Health.
He is the author of over 300 articles, books and reports in the field of management, health economics, pharmacoeconomics and quality of life.
A medical doctor by education, specialist in epidemiology and public health, specialist in psychiatry, holding PhD degrees in medicine and management, MBA; completed postgraduate studies in Health Economics, Leadership Academy, university trainings at the University of York, University of St. Andrews, Mc. Master University.
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Michal Worotynski, MSc
Ewopharma, Schaffhausen, Switzerland
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Sean Donovan
EMD Serono, United States
Accounting for Preference Heterogeneity in Stated Choice Data: From Good-Practice Methods to Decision-Relevant Interpretation
Session Type: Workshop
Topics: Patient-Centered Research, Methodological & Statistical Research, Health Policy & Regulatory
Track: Patient-Centered Evidence
Level: Intermediate
PURPOSE: This workshop will provide guidance from the ISPOR Good Practice Task Force for accounting for preference heterogeneity in stated choice data to support researchers and decision makers in moving beyond averages in data analysis, reporting, and results interpretation.
DESCRIPTION: Stated choice data is increasingly considered in regulatory, HTA, policy, and clinical decisions. While best-practice guidance for generating, analyzing and applying stated choice data exists, clear recommendations concerned with preference heterogeneity remain limited. This is in stark contrast to the importance of the topic. Accounting for differences in healthcare preferences between individuals is decision-relevant and avoids bias. Practical guidance can also reduce the perceived complexity of various analytical methods. Using an interactive benefit-risk case study, the workshop will highlight the relevance of the topic, outline considerations during data analysis, and make recommendations for results interpretation.
The workshop will be moderated by Deborah Marshall (10 minutes). Francesco Pignatti will introduce the case study, that considers the regulatory approval of a health technology with benefits, uncertainty, treatment burden, and adverse event risks. He will specifically reflect on how preference heterogeneity can inform regulatory decision making (10 minutes). The remaining speakers will draw on the case study when presenting key elements of the ISPOR Good Practice Task Force. Sebastian Heidenreich will discuss key methodological considerations for data analysis (10 minutes). Axel Mühlbacher will deliver guidance on results presentation and interpretation (10 minutes). The workshop will conclude with practical recommendations for communicating heterogeneity to regulators, HTA bodies, payers, clinicians, and patient organizations (5 minutes).
Participants will actively take part by judging whether technology in the case study is acceptable for the full population, subgroups, or not acceptable. Live polling will be conducted before and after the discussion from a regulatory perspective. The workshop will end with questions and answers.
Moderator
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Deborah A Marshall, PhD
University of Calgary, Calgary, AB, Canada
Professor and Svare Chair in Health Economics, Value and Impact, Cumming School of Medicine, University of Calgary. Her applied research programme assesses the value and impact of health services using health economics, socioeconomic benefits, patient preferences research for national and international research programmes in precision health and patient-oriented research, particularly in child health and rare disease and precision health. She supports embedded research fellows as the Nominated Principal Applicant of the CIHR Health Systems Impact Fellowship National Cohort Training Program to advance capacity for sustainable and patient-centered learning health systems across Canada”. She is a founding co-investigator of the innovative Patient and Community Engagement Research (PaCER) programme at the University of Calgary which trains patients to design and conduct health research, using specific adapted methods of qualitative inquiry. A former President and member of the Board of Directors, incoming Chair of Health Science Policy Council (HSPC) she remains an active member of ISPOR, as chair and member of Task Forces and SIGs.
Speakers
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Francesco Pignatti, MD
European Medicines Agency, Amsterdam, Netherlands
Francesco Pignatti graduated as Medical Doctor at the University of Rome. In 1995 he became Research Fellow at the EORTC Data Center in Brussels, Belgium. In 1997 he obtained a Master of Science degree in Biostatistics from the University of Limburg, Belgium. In 1999 he joined the European Medicines Agency (EMA), which is located in Amsterdam since 2019. From 2009 to 2023 he was appointed Head of the Office of Oncology and Haematology, he is currently Scientific Adviser for Oncology. His main regulatory science interests include cancer drug regulation, benefit-risk analysis, and stated preference studies.
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Sebastian Heidenreich, BSc, MSc, PhD
Thermo Fisher Scientific, London, United Kingdom
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Axel C Mühlbacher, PhD
HS Neubrandenburg, Neubrandenburg, Germany
From Evidence to Access in Asia: Lessons on RWE and Lifecycle HTA for Sustainable Reimbursement
Session Type: Workshop
Topics: Real World Data & Information Systems, Health Policy & Regulatory, Health Technology Assessment
Track: Real-World Evidence (RWE)
Level: Introductory
PURPOSE: Asian health systems are rapidly adapting reimbursement, health technology assessment (HTA), and evidence-generation policies to balance timely patient access, affordability, and uncertainty. Recent reforms in China, South Korea, and Taiwan illustrate different approaches to using real-world evidence (RWE) in reimbursement policy, strengthening RWE guidance and evidence standards for healthcare decision-making, and linking earlier access with parallel review, conditional listing, HTA, and health technology reassessment (HTR). This workshop will provide a practical, comparative overview of how RWE and lifecycle HTA approaches are being used across these three systems to support access to innovation while maintaining health system sustainability.
DESCRIPTION: Dr. Wu will discuss the initiation, development, and application of RWE in China’s reimbursement policies, highlighting how evidence generation is being integrated into coverage and access decisions. Professor Suh will present South Korea’s evolving use of RWE in reimbursement and healthcare decision-making, focusing on evidence requirements, decision uncertainty, and HIRA’s 2026 RWE guidance. Professor Ou will share Taiwan’s lifecycle HTA approach to accelerating patient access while managing uncertainty, including parallel review, conditional listing, and the growing role of RWE in HTA/HTR across the drug life cycle.
The session will be structured as three presentations (12 minutes each), followed by facilitated comparison and audience interaction. Live polling will be used to engage participants regarding which RWE and lifecycle HTA tools are most transferable across settings, what evidence gaps most limit accelerated access, and how RWE should be governed when reimbursement decisions are made under uncertainty. Moderated Q&A will draw out cross-country lessons for Europe and global stakeholders. The workshop will benefit HTA bodies, payers, policymakers, researchers, industry, and clinicians interested in sustainable access, lifecycle value assessment, and the policy use of RWE.
Moderator
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Feng Xie, PhD
McMaster University, Hamilton, ON, ON, Canada
Speakers
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Jing Wu, RPh, PhD
Tianjin University, Beijing, China
Prof. Wu has dedicated nearly 20 years to research and teaching in health economics and pharmacoeconomics. Her expertise spans health policy evaluation, pharmacoeconomic evaluation, real-world data analysis, and health-related quality of life and health utility study. She earned her PhD in Pharmacoeconomics through a joint program between Shenyang Pharmaceutical University and Peking University in 2007 and was a visiting scholar at the Schaeffer Center for Health Policy and Economics at the University of Southern California in 2013. Prof. Wu has authored over 100 high-impact scientific articles, led three National Natural Science Foundation of China projects, contributed to multiple pharmacoeconomics textbooks, and spearheaded the development of the China Guidelines for Pharmacoeconomic Evaluations (2020) and the Companion Guide to the China Guidelines for Pharmacoeconomic Evaluations (2022), promoting the development and standardization of pharmacoeconomics in China. She currently serves as Chair of the Pharmacoeconomics Committee of the Chinese Pharmaceutical Society, Vice Chair of the Pharmacoeconomics Committee of the China Health Insurance Research Association, Vice Chair of the Pharmacoeconomics Committee of the Chinese Society of Research Hospitals. She co-chaired the ISPOR Asia-Pacific Conferences in 2018 and 2022, served as Executive Chair of the 10th Huaxia Pharmacoeconomics and HTA Forum in 2019, and founded the China Young Scholars Forum on Pharmacoeconomics in 2017—organizing around 40 high-level academic meetings to date. As a national-level advisor, she provides strategic guidance to China’s National Healthcare Security Administration, playing a key role in reimbursement policy and healthcare-system reform.
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Hae Sun Suh, MA, MS, PhD
Kyung Hee University, Seoul, Korea
Dr. Hae Sun Suh, BPharm, MPharm, MA, Ph.D., serves as a professor in the College of Pharmacy and holds the position of Chair of the Department of Regulatory Science and Institute of Regulatory Innovation through Science (IRIS) at Kyung Hee University, Seoul, South Korea. She previously served as President of the ISPOR Korea Chapter and currently acts as the ISPOR Asia Consortium Chair. She also participates in various committees within the Ministry of Health and Welfare in Korea, where evidence for coverage and reimbursement decisions increasingly reflects RWE and patient perspectives.
Before assuming her current roles, Dr. Suh held positions as an associate professor at the College of Pharmacy at Pusan National University, an associate research fellow at the Korean Health Insurance Review and Assessment Service (HIRA), and as director of the Department of Health Technology Assessment Research at the National Evidence-based Healthcare Collaborating Agency (NECA).
Dr. Suh earned her Bachelor of Pharmacy and Master of Pharmacy degrees from Ewha Womans University in Korea, and subsequently her Master of Arts in Economics and her Doctor of Philosophy in Pharmaceutical Economics and Policy at the University of Southern California in the U.S.
Dr. Suh's research spans healthcare economics and health outcomes research across clinical, economic, and humanistic dimensions, with a strong emphasis on capturing the patient's voice through patient-reported outcomes and patient-centered evidence. Her current work applies causal artificial intelligence (AI) and Real-World Data to evaluate the effectiveness and safety of interventions in ways that reflect real patient experience. Drawing on her regional leadership and HTA background, she is committed to translating local patient-engagement experiences across Asia Pacific into coordinated regional action—the central aim of this workshop.
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Huang-tz Ou, PhD
National Cheng Kung University, Tainan, Taiwan
Dr. Huang-tz (Anita) Ou is a health economist and Professor at National Cheng Kung University, specializing in real-world evidence (RWE), cost-effectiveness modeling, and policy-relevant health economics and outcomes research (HEOR). She is a core advisor to Taiwan’s National Health Insurance (NHI) reimbursement system and leads multi-country research collaborations and value framework initiatives adopted by national authorities and payers. Dr. Ou currently serves as Executive Director of the Taiwan Society for Pharmacoeconomics and Outcome Research (TaSPOR). She is a core expert for Taiwan’s Drug Benefit Evaluation, Health Technology Re-assessment (HTR), and First-in-Class Drug Pricing Committees, and serves as an official delegate to the Pharmaceutical Benefit and Reimbursement Scheme Joint Committee under the NHI Administration. Her research leverages national claims, surveys, clinical registries, and electronic health records to generate robust RWE, supporting health policy and reimbursement decisions. She has published extensively on clinical effectiveness, economic burden, and patient preferences, and advocates for HEOR’s strategic use to guide evidence-based policymaking, resource optimization, and high-value care. Dr. Ou is Associate Editor for Cardiovascular Diabetology and Pharmacoeconomics. Her research and service have been recognized with major honors, including the ISPE Rising Star Award (2015), Taiwan’s Ta-You Wu Memorial Award (2020), Young Scholars’ Creativity Award (2021–2024), and the Excellent Young Scholars Award (2023–2026). In 2025, she received the prestigious LEAP Fellowship (2025–2027) from the Foundation for the Advancement of Outstanding Scholarship in Taiwan. Dr. Ou earned her PhD in Pharmacy Administration from the University of Michigan in 2010.
Could a Higher Willingness-to-Pay for 'Innovative' Therapies in Cost Effectiveness HTAs Help Enable Europe to Retain R&D and Manufacturing?
Session Type: Issue Panel
Topics: Health Technology Assessment, Economic Evaluation
Track: Access and Pricing
Level: Intermediate
Issue
• It takes increasingly innovative approaches to deliver further health gains for many therapy areas, but such approaches are inherently higher risk for manufacturers.
• New pharmacological modalities benefits may go beyond the QALY so how can this be captured? How can this be fairly and transparently reflected in decision making?
• With the aim of supporting the development and commercialisation of innovative technologies, should they be assigned additional value in HTA?
• If it is progressive and appropriate to give additional willingness-to-pay to innovative therapies, should willingness-to-pay be lowered for non-innovative therapies?
• How would we define 'innovative'? How do we stop all products having an ‘innovation’ claim, how do we define true innovation?
• Would a higher willingness-to-pay for innovative therapies reflect public preferences?
Overview
This issue panel examines whether and how we should consider innovation in HTA, should true innovation be rewarded with a higher willingness-to-pay to recognise benefits outside the QALY. Prof Lieven Annemans (Belgium) will introduce the concept of paying more for innovation in HTA and frame the debate. Prof Lotte Steuten (Office of Health Economics) will present the rationale for increased flexibilities and higher willingness-to-pay for innovative treatments. Prof Mark Sculpher (UK) will articulate the case against, including considering the opportunity cost for other patients and the potential for double counting.
Natalie Bennett (Industry representative) will highlight the rationale for change and inclusion, setting out the need for transparency and fairness of consideration.
Moderator
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Lieven Annemans
Ghent University, GENT, Belgium
Speakers
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Natalie Bennett
Novartis Pharmaceuticals UK Limited, Surrey, United Kingdom
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Mark Sculpher, PhD
University of York, York, United Kingdom
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Graham Cookson, MA, MSc, PhD
Office of Health Economics, London, United Kingdom
Graham Cookson, an econometrician by training, focuses on health system efficiency, policy evaluation, and pricing and reimbursement in pharmaceutical markets. Alongside his position at OHE, Graham is an Honorary Professor at City, University of London and is a Fellow of Royal Statistical Society, Higher Education Academy and member of Royal Economic Society.
His current research interests include the measurement and determinants of productivity in healthcare especially labour productivity; the industrial organisation of healthcare especially tariffs and competition; real-world evidence in health economic evaluation; and big data in the health and life sciences. He is best known for this work on the economics of staffing and skill mix in the English NHS, which was critical to the development of the NICE Guidelines on Safe Staffing.
Impact of Agentic AI on Redefining Productivity in HEOR: From Deliverables to Decision Impact
Session Type: Other Breakout Session
Topics: Organizational Practices, Study Approaches, Health Technology Assessment
Track: AI
Level: Intermediate
Purpose:
Generative AI (GenAI), particularly agentic AI systems, is rapidly transforming HEOR. While early applications suggest substantial productivity gains, the extent of these benefits and their implications for scientific rigor remain unclear. At the same time, evidence requirements are becoming increasingly complex, requiring HEOR teams to generate high-quality insights faster than ever. This panel will explore how productivity in HEOR should be redefined beyond time and cost savings to include evidence quality, decision impact, and patient outcomes. Panelists will discuss how AI can enhance speed, scale, and scientific excellence while maintaining methodological standards expected by HTA agencies.
Description:
Moderated by Uwe Siebert (past ISPOR President), the session will begin with an overview of recent advances in GenAI and their implications for HEOR workflows, evidence generation, and quality assurance.
Jag Chhatwal will present academic applications of GenAI in health economic modeling, including AI-driven model verification conducted with NICE and full replication of a published ICER model. These examples demonstrate how tasks traditionally requiring weeks can be completed in hours while preserving transparency and methodological rigor.
Turgay Ayer will provide an AI developer perspective, showcasing a large-scale agentic AI system comprising more than 1,000 specialized agents. He will demonstrate its use in producing comprehensive landscape assessment reports within 48 hours and discuss how multi-agent architectures can coordinate evidence identification, synthesis, and reporting while minimizing hallucinations.
Ipek Stillman will offer an industry perspective on GenAI-driven efficiency gains, examining whether productivity improvements can translate into meaningful organizational value. She will discuss implementation challenges and propose how agentic AI can help HEOR evolve from an evidence-generation function to one focused on enabling better decisions.
The session will conclude with a discussion on how regulators, HTA agencies, and other stakeholders may redefine expectations as AI adoption accelerates.
Moderator
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Uwe Siebert, MPH, MSc, ScD, MD
UMIT TIROL - University for Health Sciences and Technology; Harvard Chan School of Public Health, Hall in Tirol, Austria
Uwe Siebert, MD, MPH, MSc, ScD, is a professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL-University for Health Sciences and Technology in Austria and director of the Division for HTA in the ONCOTYROL–Center for Personalized Cancer Medicine in Austria. He is also adjunct professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health and Affiliated Researcher in the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston.
After medical school, he worked for several years as a physician in international public health projects in West Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in decision sciences at the Harvard School of Public Health.
His research interests include applying real-world evidence-based quantitative, causal and translational methods from public health, epidemiology, artificial intelligence, comparative effectiveness research, health services and outcomes research, economic evaluation, modeling, and health data a d decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, clinical guideline development, public health policies and patient guidance. His research focuses on cancer, infectious disease, cardiovascular disease, neurological disorders, and others.
He has been leading projects/work packages in several EU FP7, H2020 and Horizon Europe projects (eg, ELSA-GEN, BiomarCaRE, MedTecHTA, DEXHELPP, EUthyroid, FORECEE, MDS-RIGHT, RECETAS, CORE-MD, EUREGIO-EFH, CIDS, OnCoVID, 4D PICTURE, CATALYSE). He teaches HTA, health economics, modeling, epidemiology, causal inference and target trial emulation, and data and decision science for academia, industry, and health authorities in Europe, North and South America, and Asia. He directs the Continuing Education Program on Health Technology Assessment & Decision Sciences (htads.org).
He has served as member of the ISPOR Directors Board and as president of the Society for Medical Decision Making (SMDM). He is a leadership member of the ISPOR Personalized/Precision Medicine SIG, a member of the Latin America Consortium Advisory Committee of ISPOR, and co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force. He is a member of the Oncology Advisory Council and the National Committee for Cancer Screening of the Austrian Federal Ministry of Health.
He has authored more than 400 publications (> 30,000 citations, H index > 80), and is editor of the European Journal of Epidemiology. Further information Internet: http://htads.org, umit-tirol.at/dph, hsph.harvard.edu/uwe-siebert, Twitter: @UweSiebert9, LinkedIn: uwe-siebert9.
Speakers
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Jag Chhatwal, PhD
Harvard Medical School / Massachusetts General Hospital, Boston, MA, United States
Jag Chhatwal, PhD, is the director of the Institute for Technology Assessment at Massachusetts General Hospital and an associate professor at Harvard Medical School. He also serves as core faculty at the Center for Health Decision Science, Harvard T.H. Chan School of Public Health. Dr. Chhatwal has co-authored more than 125 original research articles and editorials in leading peer-reviewed journals. His research has informed health policy decisions at prominent organizations including the White House, the World Health Organization, and the CDC, and has been featured in major media outlets such as CNN, Forbes, National Public Radio, The New York Times, and The Wall Street Journal. Dr. Chhatwal serves as an associate editor of Value in Health and as guest editor for its special issue on artificial intelligence. He is also a member of the ISPOR Generative AI Working Group.
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Turgay Ayer, PhD
Value Analytics Labs, Boston, MA, United States
Turgay Ayer, PhD, holds the Virginia C. and Joseph C. Mello Chair and serves as the research director for Healthcare Analytics and Business Intelligence at the Center for Health & Humanitarian Systems at Georgia Tech. He is also the chief technology officer at Value Analytics Labs. Dr. Ayer holds a courtesy appointment at Emory Medical School where he teaches Big Data Analytics courses and serves as a Senior Scientist at the Centers for Disease Control and Prevention (CDC). Dr. Ayer’s research focuses on health economics modeling (HEOR), real-world evidence, data science, machine learning, econometric modeling, and healthcare analytics. He has published over 80 peer-reviewed journal papers and more than 300 conference abstracts, with his work featured in top-tier business, engineering, medical, and health policy journals. His research has attracted substantial attention from major media outlets, including The Wall Street Journal, The Washington Post, US News, and NPR. A recognized expert in HEOR, Dr. Ayer has been at the forefront of applying generative AI to navigate healthcare systems and support better decision-making. He has contributed significantly to the development of advanced models for predicting healthcare outcomes and designing innovative cost-effectiveness analysis frameworks. Under his leadership, Value Analytics Labs has focused on the development of cutting-edge technologies, including ValueGen.AI, to enhance healthcare analytics and improve the efficiency of healthcare decision-making processes.
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Ipek Ozer Stillman, MBA, MSc
Takeda, Cambridge, MA, United States
Bridging the Gap Between Broader Value Frameworks and Research-Ready Decision-Making
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Real World Data & Information Systems, Methodological & Statistical Research
Track: Expanded Value Measures
Level: Intermediate
Issue
Broader value dimensions are increasingly recognized across healthcare systems, yet HTA frameworks continue to focus primarily on clinical outcomes and healthcare costs. At the same time, policymakers face growing challenges related to aging populations, labor shortages, caregiver burden, and long-term system sustainability.
While expanded value assessment may improve HTA and decision-making in reflecting the wider consequences of health interventions, important questions remain regarding HTA remit, normative and methodological standards, evidentiary robustness, consistency across disease areas, and the risk of selective application. Additional challenges include fragmented data infrastructures, limited measurement of non-health outcomes, insufficient interoperability, and a lack of consensus on how broader value dimensions should be operationalized within HTA processes.
Overview
This panel will explore not only whether broader value dimensions should be incorporated into HTA and healthcare decision-making, but also how implementation can be carried out in a transparent, credible, and methodologically rigorous manner. Systematically including broader dimensions of value requires clear guidance on evidence standards, modeling approaches, data infrastructure, governance structures, and cross-sector collaboration.
The discussion will also address key tensions associated with expanded value assessment: how to balance broader societal outcomes against traditional health outcomes; avoid double-counting, inconsistent application across technologies or patient groups; manage differences between stakeholders generating evidence and those making funding decisions; and whether current healthcare systems are sufficiently prepared to operationalize broader value frameworks in routine practice.
After an introduction (10min), speakers will present their perspectives (30min), followed by a moderated discussion on implementation challenges and future directions for expanded value assessment (10min) and an interactive audience debate (10min). The panel provides insights relevant for researchers, HTA bodies, policymakers, healthcare leaders, and industry stakeholders.
Moderator
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Malina Müller, BA, MA, PhD
WifOR Institute, Darmstadt, Germany
Speakers
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Petra Došenovic Bonca, PhD
Ljubljana, Slovenia
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Valentina Prevolnik Rupel, PhD
Ljubljana, Slovenia
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Wim Goettsch, MSc, PhD
Utrecht University; Zorginstituut Nederland, Utrecht; Diemen, Netherlands
AI-Assisted Health Economic Modeling: Balancing Human-AI Workflows to Ensure Efficient, Robust, Transparent & Compliant Decision Making for HTAs
Session Type: Issue Panel
Topics: Economic Evaluation, Health Technology Assessment
Track: AI
Level: Introductory
ISSUE: Health economic models can now be rapidly developed using artificial intelligence (AI) agents to create complex cost-effectiveness models (CEMs). This approach can substantially reduce development times and increase model flexibility and scope (as they can be rapidly restructured or rescoped at short notice). However, this approach may lack clinical validity and an inherent understanding of the decision problem, unmet need, and value drivers. LLM-based agents used for model development are also probabilistic and therefore not necessarily reproducible, and the decision-making process for model construction is opaque (with justification of model assumptions being made by humans post-hoc). Large language models (LLMs) on which the AI agents rely may also be biased, based on their training data, and lacking original targeted solutions. These issues need to be resolved before AI-developed CEMs are able to significantly contribute to decision-making within HTAs. This session will detail the current risks and concerns around AI-assisted modeling, and how current methods may be misaligned with the use of AI agents and LLMs. It will also describe what actions need to be taken by relevant stakeholders and what guidelines and guardrails are required to increase confidence in models built using AI and enable them to be used within HTAs. OVERVIEW: Ray Gani will moderate and provide an assessment of current AI capabilities for developing CEMs, and current guidelines and advice on their use within HTA, covering perceived opportunities, ongoing initiatives, and major reservations. Páll Jónsson will describe the steps being taken at NICE to address the current issues described above and potential guidance updates. Ronan Mahon will discuss the implications of different types of risks, in particular fundamental (inherent) vs. manageable (via human oversight). Peter Quon will describe work ongoing within industry to resolve these issues and integrate AI workflows into CEM development. Each speaker will have 10 mins, with 20 mins for audience questions, feedback, and discussion. The intended audience is health economists, health-economic modelers, and HTA organizations.
Moderator
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Ray Gani, BSc, MSc, PhD
PharmaQuant, Hook, United Kingdom
Dr Ray Gani is PharmaQuant’s strategic and operational lead for HEOR, providing traditional and AI HEOR strategies and solutions for the biopharmaceutical, biotechnology, and medical device companies. Ray has 20+ years of experience in HEOR from consultancy, industry, and public health, serving in various leadership positions in HEOR, market access and real-world evidence.
Ray has over 50 peer-reviewed publications (https://scholar.google.com/citations?user=yYhcZ78AAAAJ&hl=en), is leading the commercialization of AI modelling and NMA tools (ModeLynx and HELiQ), as well as modelling, evidence synthesis, and JCA projects.
Speakers
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Pall Jonsson, BSc, PhD
NICE, Manchester, United Kingdom
Páll Jónsson is Programme Director for Data and Evidence at UK’s National Institute for Health and Care Excellence (NICE). He has a strategic role in ensuring NICE is at the forefront of harnessing new and emerging opportunities to inform NICE’s guidance for the health and care sectors. His remit includes data access and analysis, real-world evidence methods, implementation of AI in HTA and guidelines, and evidence services.
Páll holds a doctorate in biochemistry and bioinformatics from the University College London. Prior to his current role, he served as Associate Director for Science Policy and Research, overseeing a portfolio of international research projects in areas such as big data and real-world evidence.
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Ronan Mahon
University of Galway, Dun Laoghaire, Ireland
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Peter Quon, MPH
Regeneron, Lake Success, NY, United States
18:00 - 19:00
Networking Reception
Session Type: General Meeting
Wed 11 Nov
7:00 - 8:00
Coffee and Connect
Session Type: General Meeting
Head to the breakout rooms to connect with fellow attendees over a steaming cup of coffee.
7:00 - 13:00
Registration Hours
Session Type: General Meeting
8:00 - 9:00
Preparing CEE Countries for JCA: Aligning European HTA Standards with National Access Decisions
Session Type: Issue Panel
Topics: Health Technology Assessment, Organizational Practices
Track: Access and Pricing
Level: Introductory
ISSUE
The EU HTA Regulation and Joint Clinical Assessments introduce a common European approach to clinical evidence assessment, but countries differ in HTA maturity, resources, procedures, and readiness to use JCA outputs in national decision-making. This panel will debate how Central and Eastern European countries and neighbouring systems can prepare for JCA while maintaining national responsibility for pricing, reimbursement, and access decisions. The key issue is whether JCA will reduce duplication, improve consistency, and support faster access to innovation, or whether evolving HTA systems may face methodological, procedural, and capacity-related challenges. The discussion will focus on practical readiness and the ability to translate European assessments into locally relevant decisions.
OVERVIEW
With the first JCA reports for medicinal products expected to become available in June, this panel comes at a timely moment to discuss how these early assessments may resonate across CEE countries and influence national HTA, pricing, reimbursement, and access processes. The issue panel will bring together WHO/Europe and national HTA authority perspectives on JCA preparation in Central and Eastern Europe. Tarang Sharma will present the “Strengthening the National Health Technology Assessment Framework” initiative, supported by WHO/Europe and the European Commission through DG REFORM and technical support mechanisms, using a CEE country as an example of HTA capacity-building aligned with European norms. Jacek Walczak will present Poland’s preparation process, focusing on institutional and methodological adaptation. Boryana Ivanova will present Bulgaria’s preparation process, highlighting alignment with European HTA developments while preserving national pricing and reimbursement responsibilities. Dr. Sharma will deliver a 20-minute overview of the WHO programme for HTA in CEE countries, followed by 10-minute country presentations and 20 minutes of moderated discussion and debate. The panel will benefit HTA bodies, payers, policymakers, industry, patients, and researchers interested in European HTA implementation and equitable access to innovation.
Moderator
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Magda Chatzikou, PhD
Pharmecons Easy Access, Rafina, United Kingdom
Speakers
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Tarang Sharma, BSc, MPH, MS, PhD
WHO, Copenhagen, Denmark
Tarang Sharma has over two decades of experience in health policy development and implementation. She is the founder and Director for Evidence to Policy and currently works as a Senior Consultant Advisor for the World Health Organization for Immunization and Vaccine Policy and as a Senior Researcher for MAGIC Evidence Ecosystem Foundation, supporting evidence informed policy decisions globally. She is also part of the Steering Group for the Interest Group for HTA for Medical Devices for HTAi. Prior to this she was with the WHO Regional Office for Europe as a Technical Officer for the Novel Medicines Platform and Health Technology Assessment, working to support strategic cooperation between all stakeholders for improved access of novel technologies to patients and supporting HTA capacity building for the Region.
Previously she worked for the Agenda, Policy & Strategy unit in the Immunization, Vaccines and Biologicals department at the WHO HQ in Geneva, supporting evidence to policy recommendations for the new COVID-19 vaccines and has also worked for evidence to policy impact and health research mechanisms both at WHO HQ and WHO Europe as a senior consultant and a technical officer.
She has worked for three different national governments, in USA (AMNH in New York City as a Research Associate), in UK (for NICE as a Senior Analyst) and in Denmark (for the Danish Medicines Council: Medicinrådet, as a Specialist Consultant Advisor). She has also worked for non-governmental agencies/non-state actors with Cochrane Denmark in Copenhagen as well as being part of the Cochrane Central Executive Team at their headquarters in London, UK and previously for Akanksha and Kutumb Foundations in India. She has worked as a health economist for the University of Sheffield in UK and for Novo Nordisk A/S in Denmark and on pre-clinical research as a Research Scientist for Ranbaxy laboratories Ltd (now bought by Sun Pharma) in India.
She has published several peer-reviewed articles, national and international reports and presented at various national and international meetings and conferences. She has a MS in Biology (specialised in Applied Recombinant DNA Technology, Jan 2003) from New York University, a MPH (specialised in Health Economics, November 2008) from University of Sheffield and MPH (July 2008) and a PhD in Public Health and Epidemiology from University of Copenhagen (May 2018).
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Jacek Walczak, MD
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Boryana Ivanova, PhD
Sofia, Bulgaria
Can We STEDI the Course? Antimicrobial Value Assessment at a Crossroads
Session Type: Issue Panel
Topics: Health Technology Assessment
Track: Expanded Value Measures
Level: Intermediate
Issue:
Antimicrobial resistance (AMR) represents a critical global health challenge: we urgently need new antibiotics, yet current HTA approaches have become an active barrier to this innovation. The STEDI framework (Spectrum, Transmission, Enablement, Diversity, and Insurance value) emerged as an experiment in methods evolution—designed to capture the full value of antimicrobial products by adopting a "whole health" perspective that extends beyond traditional cost-effectiveness analysis. However, progress toward demonstrating STEDI in practice has stalled, raising essential questions about whether this represents the future of value assessment.
Critically, AMR exposes a structural limitation of traditional HTA and reimbursement models: society needs antibiotics even when appropriate stewardship aims to keep their use low. This panel brings together stakeholders from divergent perspectives to debate what STEDI's stalled momentum reveals about the real appetite for broader, holistic measures of value in healthcare decision-making—and whether this experiment can ultimately reshape how we assess antimicrobial innovation.
Overview:
Research demonstrates that rigorous STEDI quantification remains elusive, with decision-makers resorting to manual adjustments and substantial upward revisions of initial valuations. Meanwhile, the May 2024 UK antimicrobial subscription framework appears to have evolved away from strict STEDI adherence toward more pragmatic criteria-based assessment. Yet international jurisdictions (Spain, Greece, Canada) are cautiously exploring STEDI-inspired approaches despite evidence of substantial uncertainty. An alternative pathway is emerging in Italy, where access to WHO 'Reserve' antibiotics is enabled through a dedicated national fund combined with AIFA monitoring registries—prioritizing access, stewardship and real-world tracking over full STEDI quantification.
This session directly addresses the current inflection point in antimicrobial economics policy and will generate essential debate as global health systems finalize their approaches.
Moderator
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Laith Yakob
Adelphi Values PROVE, Bollington, United Kingdom
Speakers
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Edward I Broughton, BSc, MPH, PhD
Pfizer, Muenchen, Germany
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Grace Hampson
Office of Health Economics, London, United Kingdom
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Entela Xoxi, MSc, PharmD, PhD
Independent Consultant, Roma, Italy
Three Pathways from HTA to Access in China, the UAE, and South Africa: Price Negotiation, Value-Based Decision-Making, and Equity-Driven Priority-Setting
Session Type: Other Breakout Session
Topics: Health Technology Assessment, Health Policy & Regulatory
Track: Access and Pricing
Level: Intermediate
PURPOSE: The purpose of this session is to compare how emerging and evolving HTA systems in China, the United Arab Emirates, and South Africa are being shaped by different policy contexts and how they are used to support access decisions for innovative health technologies.
DESCRIPTION: Many countries are building or strengthening HTA systems at a time when health systems face growing pressure to improve access to innovative technologies while maintaining affordability, equity, and sustainability. China, the United Arab Emirates, and South Africa represent three different emerging and evolving HTA contexts. Although these systems differ in scale, financing, institutional design, and policy priorities, they all face the challenge of translating evidence into practical decisions on pricing, reimbursement, procurement, coverage, and prioritization.
This session will compare three pathways from HTA to access. China illustrates a large-scale system where HTA evidence is increasingly connected with price negotiation, reimbursement-list decisions, and efforts to expand affordable access to innovation. The United Arab Emirates represents a rapidly developing, policy-driven environment where HTA is being positioned within value-based healthcare, payer decision-making, and access management. South Africa highlights the role of HTA and priority-setting in the context of equity, universal health coverage, and allocation of limited healthcare resources.
The session will describe how these countries are developing HTA functions, generating and interpreting evidence, and linking assessment outputs to access decisions. The discussion will examine how each system balances innovation, affordability, equity, budget impact, and sustainability, and what lessons may be transferable across different settings.
By comparing price negotiation, value-based decision making, and priority-setting, the session will provide practical insights for policymakers, HTA agencies, payers, researchers, clinicians, patient representatives, and industry stakeholders interested in making HTA a more actionable instrument for improving access to high-value health technologies.
Moderator
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Nuriya Musina, PhD
Sechenov University, Dubai, United Arab Emirates
Speakers
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Kefah Ali Alqawasmeh
AUH, United Arab Emirates
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Jani Mueller, PhD
Jani Mueller, Johannesburg, South Africa
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Yingyao Chen, PhD
Fudan University, Shanghai, China
From Static to Dynamic Value: Can Living HTA and AI Reshape the Innovation Cycle?
Session Type: Issue Panel
Topics: Health Technology Assessment, Real World Data & Information Systems
Track: Real-World Evidence (RWE)
Level: Intermediate
ISSUE: Health Technology Assessment (HTA) has traditionally been conducted as static, one-off value assessments at launch, which can become outdated as evidence evolves. This panel will explore the emergence of living HTA, where conditional reimbursement is linked to RWE-driven reassessment to enable earlier patient access while managing uncertainty. Topics include (a) whether AI can catalyze living HTA through evidence surveillance and reassessment support, while managing bias, transparency and governance risks, (b) how national health priorities influence HTA flexibility, funding signals and downstream R&D investment, and (c) operational challenges in defining reassessment triggers and decision relevant endpoints. The panel will also examine implications for manufacturers as value assessment shifts from a single upfront decision to a dynamic lifecycle process, as well as challenges for public authorities actively managing conditional decisions, including potential withdrawal of approvals.
OVERVIEW: HTA decision making is becoming more complex as technologies reach the market with greater uncertainty and expectations for post-launch evidence generation. In response, decision makers are exploring conditional reimbursement, structured reassessment and expanded RWE use to balance timely patient access with robust evaluation. Alongside advances in AI, these developments have increased interest in living HTA.
An introductory overview, 10 minutes, will outline the rationale for living HTA and key drivers of change. Subsequent panellists will offer contrasting perspectives on AI-enabled reassessment, the influence of national priorities on policy choices, and practical challenges in operationalizing lifecycle evidence generation. A manufacturer perspective will address how dynamic value assessment reshapes evidence strategies and lifecycle processes.
This panel will debate whether living HTA represents an incremental evolution of current practices or a more fundamental shift in how value is assessed and rewarded, and whether it can improve alignment between public health benefit, patient access, financial sustainability and long-term innovation incentives.
Moderator
Speakers
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Olivia Wu, MSc, PhD
University of Glasgow, Glasgow, United Kingdom
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Herwig Ostermann, MSc, PhD
Vienna, Austria
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Nicholas Adlard, MA, MBA, MSc
Novartis Pharma, Basel, Switzerland
How Should Evidence be Assessed in Rare Diseases?
Session Type: Issue Panel
Topics: Health Technology Assessment, Methodological & Statistical Research, Health Policy & Regulatory
Track: Access and Pricing
Level: Intermediate
Issue: Assessing treatments for rare diseases presents fundamental challenges for HTA. Small populations with systemic, evolving, life-limiting diseases with a range of symptoms make it difficult to plan and execute sufficiently large randomized-controlled trials (RCTs). As a result, evidence is typically generated through small, short-term RCTs or single-arm trials, with outcomes that may not best reflect patient benefit. These may be augmented by evidence from patient registries, case series, case reports, patient experience data, etc. but such evidence is considered unreliable in existing HTA frameworks. This often results in high uncertainty about added benefit for high-cost therapies and negative recommendations for use.
Overview: HTA critical assessment methods are rooted in the discipline of evidence-based medicine, guided by standards such as the Cochrane Handbook and GRADE. GRADE uses a hierarchy of evidence to judge the certainty of the treatment effect based on the entire body of evidence. This is up- or down-graded, depending on a range of factors. This hierarchy and adjustment factors are challenging for rare diseases.
This panel will present ongoing work proposing novel approaches to evidence assessment that are better aligned with the nature of evidence available for rare disease therapies.
After the moderator’s brief introduction, in 10-minute presentations, Drs Caro and Tunis will present research that has led to development of novel approaches to use the totality of evidence available from a range of sources to evaluate and communicate the added benefit of rare disease treatments. Dr Goetz will critique the practical application of the frameworks in an HTA system, reflecting on his experience in the Austrian HTA system and plans for EU Joint Clinical Assessments. In the final 25-minutes, the moderator will orchestrate audience discussion about feasibility of the new approaches and what is needed to operationalize a new paradigm for evidence assessment in rare diseases.
Moderator
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Karen Facey, BSc, PhD
Universities of Oxford, Utrecht, Edinburgh and RWE4Decisions, Utrecht, Netherlands
Karen Facey worked as a senior statistician in pharma and medicines regulation before moving to Scotland in 2000 to setup the first national HTA agency. Since 2003, she has been an independent consultant working with all stakeholders on matters related to HTA, real-world data and patient involvement. She has also served for 10 years on the governing board of regional and national health boards in Scotland and is a member of the national non-medicines appraisal committee. Karen is now an senior HTA advisor to the RWE4Decisions learning network and the Guidelines for RWE Generation and SUSTAIN-HTA projects via the Universities of Oxford and Utrecht. She is also a member of the Myeloma Patients Europe patient evidence research committee.
Speakers
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Jaime Caro, PhD
Boston, United States
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Sean Tunis
United States
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Gregor Goetz, DrPH
Vienna, Austria
Children or Parents - Whose preferences matter? That depends on the decision making context
Session Type: Workshop
Topics: Patient-Centered Research, Economic Evaluation, Methodological & Statistical Research
Track: Patient-Centered Evidence
Level: Intermediate
PURPOSE: Patient preferences are fundamental for health economics and outcomes research. Preferences inform utility estimates for economic evaluation and are important for reflecting patient perspectives for patient-centered care. This workshop will focus on how approaches to elicit and interpret preferences from children/youths and parents can be used in decision making from a methodological perspective in health economics and outcomes research and practical perspective in clinical practice. Participants will learn good research practices for collecting, analyzing and using these preferences.
DESCRIPTION: In health economics and outcomes research in childhood disease, preferences of parents and children often differ. Questions arise regarding whose preferences matter, and does the decision-making context matter?
Workshop attendees will gain knowledge of good practices for stated preferences research with children/youths. Kerrie-Anne will moderate the session and introduce why it is relevant to directly capture the children’s perspectives and not just parents. Nancy will review the challenges and recommendations for valuing child and adolescent health states to derive utilities for use in economic evaluation from the recent ISPOR Task Force report. She will highlight new research underway to address how to handle differences in utilities when transitioning between child and adult HRQoL states (10 minutes). Deborah will provide an overview of stated preferences research methods in children from a systematic review and discuss the challenges of collecting patient preferences in children including ethical aspects and issues of interpretation of adult proxy responses. Drawing on case examples, she will then explore interpretation of factors important to children that differ from parents (15 minutes).
Audience participation will include identifying problems and solutions for a hypothetical case study in inflammatory chronic diseases in children (20 minutes). This interactive and informative workshop will be valuable to researchers, clinicians, and industry analysts who are interested conducting patient-preference research in children/youths.
Moderator
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Kerrie-Anne Ho, PhD
UCB, Slough, United Kingdom
Speakers
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Nancy J Devlin, PhD
University of Melbourne, Melbourne, Australia
Nancy is Professor of Health Economics at the University of Melbourne and Editor-in-Chief at Value in Health. Her principal areas of research expertise are the measurement and valuation of patient reported health outcomes; the cost effectiveness thresholds used in making judgments about value for money in health care; and priority setting in health care.
Nancy has published >200 original peer reviewed journal articles on a wide range of empirical and theoretical topics in health economics, and is co-author of Economic Analysis in Health Care, a textbook on health economics widely used in the UK and elsewhere. In 2025 Nancy was named in the Stanford/Elsevier ‘top 2% scientists’. In 2024, she was ranked 3rd in the world’s ‘Highly Ranked Scholars’™ in Health Economics based on productivity, impact and research quality. Her work was highlighted in the UK’s NIHR 10-year anniversary report, which noted ‘The impact of her research is worldwide and highly significant in improving health and health care decision making’ . Her research, submitted as a case study to the UK’s 2014 REF exercise, was judged by the sub-panel as ‘demonstrating very considerable impact in terms of reach and significance' .
She was the lead investigator on QUOKKA, a multi-million dollar programme of research on health outcomes in children, funded by the Medical Research Future Fund (Australia) (2020 – 2025), results from which have been described as “…a huge leap forward for the field of health-related quality of life measurement in child health”
She is the elected Chair of the Board of the EuroQol Research Foundation (2020-2025), the European-based not for profit body that developed the EQ-5D, the world’s leading generic measure of patient reported outcomes. Nancy has also served as the 2019/20 elected international President of ISPOR, the peak international professional society for health economics and outcomes research.
Prior to joining the University of Melbourne in 2019, Nancy was Director of Research at the Office of Health Economics, London for ten years, leading the development of its research programme, culminating in OHE achieving Independent Research Organisation (IRO) status in 2019. Prior to OHE she was Professor of Economics at City University of London, where she held the positions of Head of the Economics Department and Dean of Social Sciences. She has 40 years of experience as a researcher and as an advisor to health care organisations, both in the public and private sectors, in the UK and internationally.
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Deborah A Marshall, PhD
University of Calgary, Calgary, AB, Canada
Professor and Svare Chair in Health Economics, Value and Impact, Cumming School of Medicine, University of Calgary. Her applied research programme assesses the value and impact of health services using health economics, socioeconomic benefits, patient preferences research for national and international research programmes in precision health and patient-oriented research, particularly in child health and rare disease and precision health. She supports embedded research fellows as the Nominated Principal Applicant of the CIHR Health Systems Impact Fellowship National Cohort Training Program to advance capacity for sustainable and patient-centered learning health systems across Canada”. She is a founding co-investigator of the innovative Patient and Community Engagement Research (PaCER) programme at the University of Calgary which trains patients to design and conduct health research, using specific adapted methods of qualitative inquiry. A former President and member of the Board of Directors, incoming Chair of Health Science Policy Council (HSPC) she remains an active member of ISPOR, as chair and member of Task Forces and SIGs.
Biases that Generate Immortal Time: A Structural Diagnosis and Practical Solutions for HTA Submissions
Session Type: Workshop
Topics: Methodological & Statistical Research, Study Approaches, Real World Data & Information Systems
Track: Real-World Evidence (RWE)
Level: Intermediate
Purpose
Artificial introduction of immortal-time due to study design choices remains one of the most consequential and under-recognized threats to the validity of real-world evidence (RWE) used in market access and health technology assessment (HTA). When eligibility criteria are applied after treatment assignment, or when patients are misclassified into treatment strategies, estimated treatment effects can be biased — with direct implications for reimbursement decisions, comparative effectiveness claims, and external control arm acceptability. This workshop will equip attendees with a structural framework to diagnose design decisions that introduce immortal-time and provide concrete, implementable solutions grounded in the target trial framework. Participants will learn to identify the two principal mechanisms generating immortal time, apply design fixes, and critically appraise RWE submitted in HTA dossiers.
Description
Attendees will gain a working knowledge of how to classify, according to their structure, biases that generate immortal time and some design choices that prevent it. The workshop will cover a) the formal classification of biases that generate immortal time, b) resources to eliminate them at the study design stage, and c) case studies illustrating such applications. Xabier Garcia de Albeniz will introduce, using causal diagrams, the structure of biases that introduce immortal time (15 min.). Thibaut Sanglier will present cloning-censoring-weighting as a tool to address these biases in a case study for breast cancer (15 min.). Julia Vila, will illustrate analytical resources consisting of sequential emulation in a real-world colorectal cancer study and show the consequences of wrong design choices on effect estimates (15 min.). The workshop will close with an exercise to prompt audience participation and reaction to alternative study designs for hypothetical studies susceptible to incurring in immortal time generation. A structured discussion of remediation strategies will be offered (15 min.). This workshop will be especially valuable to RWE generators, HTA assessors, and market access professionals.
Moderator
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Miguel Hernán
Harvard T.H. Chan School of Public Health, Boston, MA, United States
Miguel Hernán is the Director of CAUSALab, the Kolokotrones Professor of Biostatistics and Epidemiology at the Harvard T.H. Chan School of Public Health, and faculty at the Harvard-MIT Division of Health Sciences and Technology. He and his collaborators repurpose real world data into evidence for the prevention and treatment of infectious diseases, cancer, cardiovascular disease, and mental illness. This work has contributed to shape health research methodology worldwide.
Speakers
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Xabier Garcia de Albeniz
Adigens Health Limited, Sant Cugat del Valles, Spain
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Thibaut Sanglier, PharmD, MPH, PhD
Basel, Switzerland
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Julia Vila, PhD
RTI Health Solutions, Spain
Julia Vila Guilera has over 8 years of experience in epidemiology and health research across academic and consulting settings. In her current role at RTI Health Solutions, she designs pharmacoepidemiologic studies and contributes to the development of study protocols, analysis plans, and study reports supporting regulatory decisions across a range of therapeutic areas. Her work focuses on the design and conduct of real-world evidence studies using causal inference methods, the target trial emulation framework, and external control arm studies.
No Longer Inevitable: Avoidable Hospitalisations As a Catalyst for Health System Transformation
Session Type: Issue Panel
Topics: Health Service Delivery & Process of Care, Health Policy & Regulatory, Economic Evaluation
Track: Expanded Value Measures
Level: Introductory
Issue: Over 3 billion people today are living with chronic disease, rare disease and cancer – this already significant burden is set to increase further as populations age. Robust evidence shows that avoidable hospitalisations represent both a major cost driver and a signal of upstream system failure. They reflect gaps in prevention, early diagnosis, continuity of care, and optimal use of guideline-directed medical therapy (GDMT), making them a powerful indicator of primary care performance and overall system effectiveness.
This panel will highlight the significant impact of NCD-related avoidable hospitalisations and explore the opportunities and policy levers for health systems to address these through proactive, integrated, and outcomes-focused care.
Overview:
Andreas Heddini will introduce how addressing avoidable hospitalisations can drive care pathway transformation and deliver benefits for patients, health systems and the wider economy. This will be supported by findings from a new study demonstrating the impact of GDMT optimisation on outcomes and costs (10 minutes).
Prof York Zöllner will examine the economic burden of rising NCD-related avoidable hospitalisations, and how health systems can use this metric to identify inefficiencies, strengthen accountability, and improve outcomes (10 minutes).
Penilla Gunther will provide pragmatic and realistic contextual considerations of how effective policy options can be implemented to improve health system outcomes. (10 minutes)
Dr Richard Russell will provide frontline operational perspective on how to prevent avoidable hospitalisations (e.g. through increasing GDMT uptake), outlining challenges and identifying practical solutions to improve patient care and improving strain on healthcare workforce. (10 minutes)
A moderated discussion will engage panellists and the audience on implementation, risks, and policy implications (20 minutes).
Stakeholders who would benefit from attending: Health and finance policymakers, primary care leaders, clinical leaders, patient advocates, payers, population health leaders
The study presented was funded by AstraZeneca.
Moderator
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Andreas Heddini
AstraZeneca, Switzerland
Speakers
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York Francis Zoellner
Hamburg, Germany
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Penilla Gunther, .
Stockholm, Sweden
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Richard Russell, MBBS, PhD
London, United Kingdom
8:30 - 10:00
Networking Breakfast Bites (Exhibit Hall)
Session Type: General Meeting
Join us for Networking Breakfast Bites, designed to kickstart the final day of ISPOR Europe 2026, with light refreshments and valuable networking opportunities.
8:30 - 11:30
Exhibit Hall Hours
Session Type: General Meeting
8:45 - 9:45
Navigating PICOs in the Era of JCA
Session Type: Research Podiums
This session examines key methodological and evidence-generation challenges emerging from the EU Joint Clinical Assessment, drawing lessons from early assessments, comparator selection, and evolving PICO requirements.
Moderator
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Steffen Wahler, MA, MD
St. Bernward, Hamburg, Germany
ARE APPROVED THERAPIES ALWAYS THE MOST RELEVANT COMPARATORS? A LIVING EVIDENCE ANALYSIS OF REGULATORY-GUIDELINE-REIMBURSEMENT DIVERGENCE ACROSS SIX ONCOLOGY INDICATIONS
OBJECTIVES: Health technology assessment (HTA) and Joint Clinical Assessment (JCA) require identifying clinically relevant comparators. As standards of care evolve, guideline recommendations may diverge from regulatory approvals through biomarker-driven therapies, sequencing changes, and off-label strategies. We evaluated the concordance between European Medicines Agency (EMA) approvals, European guideline recommendations and National Institute for Health and Care Excellence (NICE) decisions across six oncology indications and assessed implications for comparator selection through a Real-Time AI-Assisted Living Systematic Literature Review (REAL-SLR).
METHODS: A PRISMA-compliant, continuously updated REAL-SLR was used to identify interventional studies evaluating therapies with EMA approval and/or major European guideline recommendations in non-small cell lung cancer (NSCLC), bladder cancer (BC), prostate cancer (PC), pancreatic ductal adenocarcinoma (PDAC), multiple myeloma (MM), and chronic lymphocytic leukemia (CLL). Alignment between approval status, guideline recommendations and NICE decisions was monitored and assessed across indications.
RESULTS: As of June 17, 2026, the REAL-SLR contained 1,726 studies in NSCLC, 862 in PC, 751 in MM, 582 in PDAC, 554 in BC, and 341 in CLL. Of these, 412, 128, 182, 72, 28, and 97, respectively, evaluated EMA-approved/guideline-recommended therapies. Concordance between EMA-approved therapies and guideline recommendations varied across indications, from 92% in NSCLC and 91% in PC, 78% in CLL (78%), 75% in BC, 74% in MM, and only 49% in PDAC. Furthermore, only 36%-69% of studies with EMA-approved therapies have both guideline recommendations and NICE positive decisions. Divergence was primarily driven by older therapies no longer guideline-recommended, emerging therapies not yet approved, sequencing changes, and tumor-agnostic biomarker-driven strategies not yet adopted by guidelines.
CONCLUSIONS: Substantial differences exist between regulatory approvals, guideline recommendations and reimbursement decisions across oncology. Regulatory approvals alone may overestimate clinically relevant comparators, while guidelines reflect emerging standards of care. Living evidence monitoring enables earlier identification of comparator shifts, supporting HTA, JCA, reimbursement, and evidence-generation planning.
EVIDENCE GAPS AND STRATEGIC LESSONS FROM THE TOVORAFENIB CASE - A CRITICAL APPRAISAL OF FIRST EU JCA REPORT
OBJECTIVES: The European Commission has released its first Joint Clinical Assessment (JCA) report, a major milestone for the European Union (EU) Health Technology Assessment (HTA) framework. The evaluation of tovorafenib for paediatric low-grade glioma (LGG) is the first real-world example of how comparative clinical evidence will be assessed at the EU level. This study aims to analyze the key lessons emerging from the first EU JCA and their implications for future evidence generation and EU HTA submission strategies.
METHODS: The JCA report for tovorafenib, published by the European HTA bodies through the EU HTA framework, was reviewed to assess evidence requirements, comparator selection, indirect treatment comparisons, patient-reported outcomes, and implications for future submissions.
RESULTS: The critical review of the report revealed the assessment highlighted substantial limitations in the evidence base analysed. No direct comparative data was available. Only one of eight PICOs could be evaluated using an unanchored matching-adjusted indirect comparison (MAIC), which highlighted the importance of comparative evidence under EU HTA. The pivotal single arm study, FIREFLY-1 limited conclusions on relative effectiveness, while concerns about residual confounding, limited covariate adjustment, poor population overlap, reduced sample size, and inconsistent outcome definitions created substantial uncertainty. In addition, patient-centred outcomes, such as health-related quality of life and symptom measures, were largely unavailable, limiting the assessment of patient-centered benefit. The assessment underscores the need to align evidence strategies with both regulatory and HTA requirements, with EU HTA success increasingly dependent on comparative, PICO-aligned, and patient-centred evidence rather than regulatory efficacy data alone.
CONCLUSIONS: The first EU JCA signals a shift toward comparative, PICO aligned, and patient-centred evidence for HTA success. Early integration of HTA requirements into clinical development will be essential for future EU market access.
METHODOLOGICAL FIT BETWEEN THE EU JOINT CLINICAL ASSESSMENT AND EVIDENCE GENERATION FOR ADVANCED THERAPY AND ORPHAN MEDICINES
OBJECTIVES: The EU Joint Clinical Assessment (JCA) requires evidence on relative effectiveness and safety across Member State PICO questions. Advanced therapy medicinal products (ATMPs) and orphan medicines often have small populations, limited comparator availability, single arm pivotal studies, surrogate outcomes and immature durability data at launch. We assessed how JCA evidence expectations align with the evidence base typically available for ATMPs and orphan medicines before the 2028 scope expansion
METHODS: We synthesized published JCA methodological and procedural guidance and evidence reviews of EU authorized ATMPs. Evidentiary features were extracted for trial design, comparator availability, endpoint maturity, use of external controls, indirect treatment comparisons and long term follow up. Features were mapped against JCA requirements for comparative evidence across PICO defined populations and comparators.
RESULTS: The evidence base for ATMPs and orphan medicines frequently relies on single arm trials, surrogate or intermediate endpoints, natural history data, external controls and post launch follow up. These features create challenges under a JCA framework centered on comparative estimates for each relevant population and comparator. A stakeholder analysis of 18 EU authorized ATMPs estimated that 16 would not have demonstrated added benefit under baseline methods proposed for JCA, mainly because randomized evidence or long term durability data were unavailable at launch. PICO multiplication may further widen evidence gaps when Member States identify multiple subpopulations or comparators not represented in pivotal trials. These risks are expected to increase when orphan medicines enter JCA scope in 2028 and all new medicines enter scope in 2030.
CONCLUSIONS: A methodological fit issue exists between JCA comparative evidence requirements and the evidence generation constraints of ATMPs and orphan medicines. Developers should seek early scientific advice, plan indirect or external control strategies prospectively, justify surrogate outcomes and define post launch durability evidence plans before JCA scope expansion.
HOW MANY PICOS CAN ONE ASSESSMENT CARRY? SCOPE AND FEASIBILITY IN EU JOINT CLINICAL ASSESSMENT
OBJECTIVES: A single Joint Clinical Assessment (JCA) was meant to replace duplicated national reviews — but how many PICOs can one assessment realistically carry? The EU HTA Regulation (HTAR) requires assessment scopes to reflect Member States' needs through the Population-Intervention-Comparator-Outcome (PICO) framework. This study examined whether operational PICO scoping remains compatible with the workload assumptions underpinning the HTAR and whether extensive subgroup requests generate a burden exceeding the system's original design parameters.
METHODS: A legal-policy analysis was conducted using Regulation (EU) 2021/2282, implementing legislation, published methodological guidance, European Commission impact-assessment documents, and early operational JCA experience. Particular attention was given to the interaction between Member State PICO requests and subgroup specifications. The observed operational model was compared with the workload assumptions described during the legislative development of the HTAR and assessed against principles of feasibility and proportionality.
RESULTS: The analysis identified a structural divergence between the anticipated and operational assessment scope. While early stakeholder communications suggested a manageable number of PICOs per assessment, the aggregation of Member State subgroup requests creates a combinatorial expansion of analytical requirements. Multiple stratification requests based on biomarkers, prior therapies, disease stage, age, and treatment line transform a limited assessment scope into a substantially larger evidence-generation exercise. This multiplication increases demands on manufacturers and assessors without a corresponding adjustment of procedural timelines. The principal driver of scope inflation is not the number of comparators but the accumulation of subgroup analyses, many of which operate below conventional statistical thresholds for robust inference.
CONCLUSIONS: Inclusiveness without prioritisation risks defeating the very goal of reducing duplication. Explicit prioritisation criteria, proportionality filters, and statistical-feasibility thresholds would keep PICO scoping both manageable and scientifically valid.
Beyond the Kaplan–Meier Curve: Advances in Survival Analysis
Session Type: Research Podiums
Survival analysis remains central to HTA, health economic modelling and regulatory decision making. This session presents methodological advances in analyzing, extrapolating and synthesizing time-to-event data, with particular emphasis on addressing complex censoring mechanisms, external evidence and competing risks.
Moderator
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Martin Scott, BSc, MSc
Numerus, Reutlingen, Germany
INTERVAL-CENSORED PROGRESSION-FREE SURVIVAL IN ONCOLOGY TRIALS - ESTIMATOR BIAS, PARAMETRIC EXTRAPOLATION, AND IMPLICATIONS FOR RESTRICTED MEAN SURVIVAL TIME
OBJECTIVES: In oncology trials, progression-free survival (PFS) is inherently interval-censored: progression is detected only at scheduled clinic visits, so the true event time lies within a known interval rather than being observed exactly. Despite this, progression is commonly assumed to occur at the scheduled visit. This simulation study quantified the impact of different approaches to interval-censoring on bias, long-term extrapolation and restricted mean survival time (RMST).
METHODS: A single-arm trial (n=200) with maximum follow-up of 36 months was simulated with true progression times following a Weibull distribution and deaths following an exponential distribution. Four non-parametric estimators were compared, assuming 3-monthly scheduled visits: a reference truth estimator (no interval censoring), a right-censored Kaplan-Meier, a midpoint-censored Kaplan-Meier, and a Turnbull non-parametric maximum likelihood estimator. Bias relative to the truth was estimated for each estimator. Parametric extrapolation was undertaken using the seven standard HTA distributions for each estimator, comparing estimated survival at different landmarks and RMST.
RESULTS: All three approaches introduced absolute bias in the survival curve through the observed period, with right-endpoint and midpoint censoring producing larger bias than the Turnbull estimator. This absolute bias also distorted the parametric model selected based on AIC, causing fits to diverge substantially from those based on the truth reference beyond the data cut-off. At a 10-year horizon, survival estimates from the censoring methods ranged from 0.77 (right-censoring) to 7.50 (midpoint-censoring) times the truth reference, corresponding to survival of 1.00% to 9.79%. At 50-year horizons, RMST differed from the truth reference by up to 18 months.
CONCLUSIONS: These findings highlight that the choice of censoring mechanism for progression events is not only a technical detail but can be a major source of bias with direct implications for survival extrapolation and potentially cost-effectiveness conclusions.
BAYESIAN INTEGRATION OF EXTERNAL TRIAL DATA IMPROVES LONG-TERM SURVIVAL EXTRAPOLATION IN MULTIPLE MYELOMA
OBJECTIVES: Survival extrapolation in oncology is essential to inform cost-effectiveness in health technology assessment (HTA). HTA guidelines recommend incorporating external evidence to improve long-term plausibility, but how this should be operationalised, and how robust the estimates are, remains poorly characterised. This study assessed whether integrating external trial data into a Bayesian framework improves long-term overall survival (OS) accuracy versus standard parametric survival models (PSMs).
METHODS: Clinical trials assessing melphalan-prednisone (MP) in transplant-ineligible newly-diagnosed multiple myeloma were identified through a literature review. OS from VISTA (75-month follow-up; restricted mean OS 42.4 months) was truncated at 30 months to emulate immature data. Seven standard PSMs were fitted to the truncated data. Bayesian PSMs (BPSMs) were then fitted using informative priors on shape parameters from eight external MP trials, under three prior strengths (weak: SD=20% of mean; moderate: 5%; strong: 0.1%). Accuracy was quantified as the area between each extrapolation and the full Kaplan-Meier curve in months (ΔAUC).
RESULTS: Standard PSMs overestimated long-term OS, with ΔAUC of 1.9 to 4.5 months (4.5% to 10.6% of restricted mean survival). In contrast, BPSM extrapolations were distributed around the Kaplan-Meier curve rather than systematically above it. Under moderate informative priors, adding an external prior improved accuracy versus the same distribution without a prior in 83% of cases. Moderate priors outperformed weak priors on average, while strong priors produced more variable results. For every external trial, at least one BPSM outperformed all PSMs, and seven of eight matched the long-term Kaplan-Meier curve (ΔAUC<1 month). AIC, BIC and DIC on the Bayesian fit poorly predicted accuracy; goodness-of-fit on the external trial provided a useful but imperfect guide to distribution selection.
CONCLUSIONS: BPSMs prevented the systematic overestimation observed with standard PSMs and should be explored in HTA submissions. Distribution selection should always be validated against clinical expert opinion to ensure long-term plausibility.
WHEN THE TAIL WAGS THE DOG III: A PERSIST-VERSUS-FADE EXTRAPOLATION CHOICE AND THE SURVIVAL RATIO SHIFT (RASOLUTE302)
OBJECTIVES: Partitioned survival models rely on extrapolated curves yet seldom report where the modelled benefit falls: inside the observed data, or in the assumed tail. Using RASolute302 in pancreatic cancer, where a large overall-survival (OS) effect remains immature, we applied the Extrapolation Benefit Share (EBS: the share of incremental mean survival arising beyond the observed data) and the Survival Ratio Shift (SRS: whether the advantage is tail-concentrated), with a relative-survival floor for OS.
METHODS: Pseudo-individual patient data were reconstructed (SurvdigitizeR) from the published progression-free survival (PFS) and OS Kaplan-Meier curves (daraxonrasib vs investigator's choice of chemotherapy; metastatic pancreatic cancer). Eight parametric distributions were fitted jointly (shared distribution, treatment covariate). Mean survival was decomposed into observed and extrapolated components over a 40-year horizon, with boundary determined by reverse-Kaplan-Meier diagnostic. OS was additionally modelled as relative survival (excess hazard plus ONS UK general-population mortality).
RESULTS: With nine months' median follow-up, both endpoints were extrapolation-dominated, and the modelled OS gain exceeded the PFS gain. For PFS, EBS was 37-69% with incremental survival 3-5 months; for OS, EBS was 65-86% with incremental survival 5-13 months. The population floor barely altered OS (relative survival ≈ all-cause; excess mortality dominates). In the extrapolation period distributions split sharply: increasing-hazard fits keep the excess hazard rising (persist), whilst long-tailed fits turn it over (fade) - a data-unresolved choice swinging the OS gain ~2.5-fold. Crucially, SRS exceeded 1 under every distribution (≈1.9-3.7): the OS advantage is loaded into extrapolated tail whichever extrapolation is chosen.
CONCLUSIONS: A large, significant OS effect can rest almost entirely on extrapolation. It’s lifetime magnitude hinging on an open persist-versus-fade choice that a population-mortality floor cannot resolve. Reporting EBS with SRS makes explicit not only how much of the benefit is assumed, but that it is structurally a tail phenomenon.
SURVIVAL MODELING USING COXIAN PHASE-TYPE DISTRIBUTION IN DISCRETE STATE-TRANSITION MODELS
OBJECTIVES: State-transition models with time-dependence in non-initial states typically account for that time-dependence using tunnel states. However, this considerably increases the model size in case of long-term time-dependence. We used the Coxian phase-type distribution as an alternative approach, where patients progress stochastically through sequential phases, enabling long-term time-dependent transitions and rewards to be captured even with just a few phases. This study evaluates the feasibility and validity of using the Coxian phase-type distribution on cost and utility estimates compared with conventional approaches.
METHODS: We applied the Coxian and tunnel state approaches in a heart transplantation model, with monthly cycles. Hazard rates for the Coxian phase-type distribution were estimated through optimization and subsequently used to derive transition probabilities. We performed survival extrapolation using a three-phase Coxian distribution and compared fit with the tunnel-state approach. State rewards for each phase were calibrated to the time-dependent costs and utilities for each cycle. We compared approaches according to visual fit, and estimated lifetime costs and utilities associated with post-transplantation states.
RESULTS: The tunnel-state approach required a tunnel length of 13, while the Coxian approach only required 3 phases and provided better visual fit. The Coxian approach captured a non-monotonic hazard pattern, with the highest hazard occurring in the initial cycles, followed by a substantially lower intermediate hazard, and a modest increase in long-term hazard. Estimated lifetime costs are €13,876 using tunnel states and €14,108 using the Coxian phase-type distribution, while utilities are 22.61 in the 13-month tunnel-state approach and 22.60 with the Coxian approach.
CONCLUSIONS: The Coxian phase-type approach offers a flexible alternative to tunnel state for long-term extrapolation by avoiding limitations imposed by finite tunnel lengths. This approach can improve long-term fit in non-initial states while accounting for time-dependent hazards and rewards.
9:00 - 11:30
Poster Session 5
Session Type: Research Posters
Presenters will be with their posters from 9:00–10:00
10:00 - 11:00
From Framework to Practice: Piloting a Harmonized EU Approach to Early Feasibility Studies to Shape the Future Medical Device Regulatory Framework
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Medical Technologies, Patient-Centered Research
Level: Intermediate
ISSUE The MDR is currently being reviewed, creating a timely opportunity to address gaps in the EU regulatory framework that are preventing the uptake of EFS in Europe, which are a critical step in the clinical evidence pathway for certain devices. This session will explore the harmonized framework for EFS developed by the HEU-EFS project, highlighting both areas of alignment with current regulatory landscape and recommendations for future improvements. Attention will be given to how the framework could bring a more structured, predictable, and harmonized approach to EFS across EU. The panel will also discuss the pilot phase currently underway to test the framework in real-world settings, with the aim of generating practical recommendations and support future policy and regulatory developments.
OVERVIEW An overview of the harmonised HEU-EFS framework will be provided by Prof. Giuditta Callea (SDA Bocconi, HEU-EFS Coordinator) (10 minutes). Dr. Alexandra Poulsson (NIPH) will describe the opportunities offered by the HEU-EFS framework to address key challenges within the current EU regulatory landscape for MDs (10 minutes). Mr. Andrea Rappagliosi (Edwards Lifesciences) will then present the objectives and expected outcomes of the HEU-EFS pilot phase, focusing on the results of the open call for pilots launched by the project and the work conducted by the HEU-EFS Screening Committee in selecting candidate pilots (10 minutes). Ms. Marta Bragagnolo (GHH) will present key elements of the HEU-EFS Roadmap for structured patient involvement in EFS. The final 20 minutes will be dedicated to an interactive discussion with the audience, moderated by Prof. Callea. Questions and comments from participants will be discussed in a roundtable format with panellists.
This session will be of particular interest to regulators, HTA bodies, notified bodies, patient organizations, decision-makers, ethics experts, clinical investigators, and medical device manufacturers seeking to contribute to the future development of EFS in EU.
Moderator
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Giuditta Callea, PhD
SDA Bocconi School of Management, Milano, Italy
Giuditta Callea holds a degree in Management Engineering from the University of Brescia and a Ph.D. in Economics and Technology Management from the University of Bergamo.
Associate Professor of Practice at SDA Bocconi School of Management, she coordinates the MASAN Observatory on management of healthcare procurement and contracting.
Her research focuses on public procurement of healthcare technologies, HTA, and funding and reimbursement.
She is Principal Investigator of the "Harmonised Approach to Early Feasibility Studies for Medical Devices in the European Union" (HEU-EFS) project funded by the Innovative Health Initiative and serves as Topic Editor for Clinical Therapeutics in Pharmacoeconomics, Outcomes, and Health Policy.
Speakers
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Alexandra H.C. Poulsson, PhD
Oslo, Norway
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Andrea Rappagliosi
Edwards Lifesciences Sàrl, Nyon, Switzerland
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Marta Bragagnolo
Galway, Ireland
Marta is the EU Advocacy Manager for Global Heart Hub, the international alliance of cardiovascular patient organizations, leading stakeholder engagement strategies to support the organisation’s policy and advocacy objectives and coordinating the organisation’s work in EU-funded projects that advance cardiovascular health across Europe and beyond.
Marta also represents Global Heart Hub as vice-chair of the equity and access working group of the WHO/Europe Public Health Innovation Platform for the years 2026-2028.
The Triple Challenge: How do global pricing dynamics, with US MFN policy, China’s rise, and European policies converge and impact European competitiveness?
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment
Track: Access and Pricing
Level: Intermediate
ISSUE:
Three converging forces are reshaping pharmaceutical innovation — where it happens, who accesses it, and on what terms. Does Europe adequately recognise and reward innovation, and what are the consequences if not?
Global pricing dynamics are sending complex signals to innovators. The US, long the anchor of pharmaceutical revenue, is introducing Most-Favored-Nation (MFN) pricing, adding further uncertainty.
China's rapid emergence as a biopharmaceutical force illustrates what happens when an ecosystem deliberately rewards innovation. Now rivalling established leaders, China serves as a mirror — forcing Europe to question its positioning.
Value assessment is deeply interconnected across markets, with decisions in one region rippling into others. Europe's environment — characterised by protracted timelines, fragmentation, and price erosion — risks sending negative signals to investors.
Europe's policy reforms — including the HTA Regulation, Pharmaceutical Legislation, Biotech Act, and Critical Medicines Act — carry potential. But the test is whether patients, innovators, and investors experience them as a coherent signal valuing medical progress.
This panel will explore what it will take for Europe to remain a place where innovation is genuinely sustained: where value is recognised, access is achieved, and investment follows.
OVERVIEW: Introduction provided by Casper Paardekooper (moderator), introducing the panel and the Triple Challenge for Europe (10 min).
Neil Grubert will share his views on global policy shifts and global pricing dynamics (12 min).
Eelko den Breejen will share his views on Europe’s competitiveness and its positioning in the global innovation landscape from an industry perspective (12 min).
Ferenc Marofka will shed light on the latest developments in EU policy and share his views on the expected impacts of ongoing and upcoming reforms (12 min).
Time is allowed for audience questions and a panel discussion (15 min).
This session will benefit stakeholders in European market and patient access, either from a patient, industry or payer perspective.
Moderator
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Casper Paardekooper, MSc
Cencora, Voorburg, Netherlands
Casper is passionate about health and access policy: working together with stakeholders in the health and life sciences ecosystem to improve the system and to ensure patients have access to innovations that can make the difference for them.
Casper is VP, Health Policy & Strategy at Cencora Global Consulting Services. He oversees all activities related to health policies affecting Europe and drives strategy across practice areas. He maintains strong connections with all key stakeholders in the European health and life sciences ecosystem. He is seen as a thought leader when it comes to health and access policy, authoring multiple influential publications. In that capacity, he regularly serves as moderator and speaker at key conferences and webinars. He is a trusted advisor to many biopharma companies, bringing 20 years of consulting and life sciences experience.
Speakers
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Neil Grubert
Neil Grubert Consulting, Basildon, United Kingdom
Neil Grubert spearheaded the development of Decision Resources Group’s international market access business, most recently as Vice-President of Global Market Access Insights. Since October 2014, he has been working as an independent global market access consultant, trainer, and writer. He has completed projects for 14 of the world’s top 20 pharmaceutical companies, smaller manufacturers and industry associations. He is a leading voice on pharmaceutical pricing and market access, with more than 26,000 followers of his daily posts on LinkedIn for insights into global trends and policy developments.
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Eelko den Breejen
Pfizer (Global), Capelle a.d. IJssel, Netherlands
Eelko is Global Access, Strategy & Pricing Oncology Team Lead and acts as the Global Head of Oncology Portfolio & Market Engagement at Pfizer. He joined Pfizer via the integration of Seagen where he served as the Head of International Market Access & Policy. Eelko has over 15 years industry experience in market access and policy functions with increasing responsibility at Global, Regional and Local level, e.g. previously he served as Head of Market Access and Corporate Affairs for Biogen in the Netherlands and has worked as the Global Policy Lead for Precision Oncology at Roche in Switzerland. Eelko holds a BSc in Physiotherapy, a MSc in Health Economics, Policy & Law and followed the Political Science program at the Netherlands Institute of Government.
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Ferenc Marofka, MSc
Brussels, Belgium
Can CEE Healthcare Systems Afford Innovation Without Reforming Procurement and Financing Models?
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Epidemiology & Public Health, Health Service Delivery & Process of Care
Track: Access and Pricing
Level: Introductory
**Issue**
Healthcare systems across Central and Eastern Europe (CEE) face increasing pressure to provide access to innovative therapies while operating within constrained budgets and ageing populations. Current procurement and financing models are often focused on short-term cost control and annual budget cycles, which may not adequately capture the long-term value of innovation, prevention, improved adherence, and better patient outcomes. As the costs of novel therapies continue to rise, policymakers, payers, and industry stakeholders are increasingly questioning whether existing financing mechanisms can sustain equitable access to innovation. Can CEE healthcare systems continue to afford innovation under current procurement and financing approaches, or are broader reforms necessary to ensure long-term sustainability?
**Overview**
This issue panel will bring together perspectives from payers, industry, and health system experts to debate whether existing healthcare financing and procurement models are fit for the future. The discussion will examine how European healthcare systems can balance financial sustainability with timely patient access to innovation, and whether current reimbursement and procurement approaches adequately reward long-term health outcomes. Particular attention will be given to the challenges posed by demographic change, increasing demand for healthcare services, rising costs of innovative therapies, and limited public resources.
Each panelist will deliver a 10–15-minute presentation outlining their perspective, followed by an interactive discussion with the audience. Moderated by Izabela Pieniazek (Certara), the debate will encourage participants to challenge assumptions, compare experiences across healthcare systems, and identify practical solutions that could help CEE countries maintain access to innovation while ensuring long-term sustainability of healthcare financing.
Moderator
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Izabela Pieniazek, MSc
Certara, KRAKOW, Poland
Izabela is a Senior Director at Certara's Global Evidence Synthesis Department, where she leads the Centre of Excellence. She has worked in Health Technology Assessment and Market Access since 2006.
Drawing on her extensive experience in health economics, systematic reviews and value and access, as well as her long-standing collaboration and management of multidisciplinary teams, she excels at overseeing all aspects of multidimensional market access projects.
She has co-authored numerous peer-reviewed publications and conference papers and has shared her knowledge of market access by moderating or speaking at scientific conferences.
She holds an MSc in Mathematics from the Cracow University of Technology. She has also completed the following postgraduate studies: Practical Forecasting and Time Series Analysis at Cracow University of Economics and StatSoft Poland, and Human Resources Management at the School of Banking and Management in Cracow.
Speakers
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Joanna Lis, PhD
Warsaw, Poland
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Krzysztof M Landa, MD
Watch Health Care Foundation, Warszawa, Poland
MD, CEO of MedInvest Scanner, 27 years of experience in R&P, HTA and MA. He used to be the Deputy MoH responsible for reimbursement in Poland 2015-2017 and the Director of Drug Policy Dept. at the Central Office of NHF 2006-2007. The Chairman of the Health Committee of the Polish-Ukrainian Chamber of Commerce. Plus various other systemic roles.
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Elena Chitan, MPH, PharmD
Nicolae Testemitanu State University of Medicine and Pharmacy, Chisinau, Moldova
HTA as Enabler, Not Blocker: Which Redesign Should Come First to Support Procurement Decisions in Smaller European Health Systems?
Session Type: Issue Panel
Topics: Health Technology Assessment, Health Policy & Regulatory
Track: Access and Pricing
Level: Intermediate
Issue
Many smaller European countries, especially but not exclusively in Central Europe, have adopted HTA frameworks modelled on the UK cost-effectiveness paradigm, yet they operate in fundamentally different institutional contexts, with resource constraints, limited data availability, greater sensitivity to short-term budget impact than to cost-effectiveness and institutional traditions where negotiation is the primary mechanism to decide about access. The result is a mismatch: heavy cost-per-QALY assessments consume significant resources and delay access while not necessarily providing payers with the information they actually need to negotiate procurement deals across price, volume, indication, and lifecycle dynamics. Although this problem statement is widely shared, reform proposals considerably differ in which redesign should come first.
Overview
This panel debates competing variations for procurement-focused HTA. The four participants agree that HTA must move from blocker to enabler of procurement, but they prioritize the first step differently. The moderator, Lou Garrison frames procurement-focused HTA and the three competing proposals for redesign (5 min). Each panellist (10 min) advocates for a different first move: proportionate review leveraging evidence generated elsewhere, with governance to adapt it locally (Jorge Mestre-Ferrandiz); rapid prioritization by health gain and disease burden (Dávid Dankó); and integrated added-benefit assessment driving price-volume agreements as system-level contracts, drawing on French and Italian practice (Olivier Wong). The moderator then opens the debate, pressing each panellist on the role of formal cost-effectiveness analysis, the transferability of foreign assessments, and whether the cost-effectiveness threshold should serve as a negotiation anchor. Audience polling will identify which redesign carries most potential for smaller European markets. The session is aimed at HTA agency leaders, payers, manufacturers, and policy researchers working with smaller or transitioning European health systems.
Moderator
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Lou Garrison, PhD
The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, Seattle, WA, United States
Lou Garrison, PhD, is professor emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
For the first 13 years of his career, Dr. Garrison worked in non-profit health policy at Battelle and then the Project HOPE Center for Health Affairs, where he was the Director from 1989-1992. Following this, he worked as an economist in the pharmaceutical industry for 12 years. From 2002-2004, he was vice president and head of Health Economics & Strategic Pricing in Roche Pharmaceuticals, based in Basel, Switzerland.
Dr. Garrison received a BA in Economics from Indiana University, and a PhD in Economics from Stanford University. He has more than 150 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies.
Dr. Garrison was elected as ISPOR President for July 2016-June 2017, following other leadership roles since 2005. He recently co-chaired the ISPOR Special Task Force on US Value Frameworks. He was selected in 2017 by PharmaVOICE as being among “100 of the Most Inspiring People” in the industry. He recently received the PhRMA Foundation and Personalized Medicine Coalition 2018 Value Assessment Challenge First-Prize Award as lead author on a paper on “A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine.”
Speakers
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Jorge Mestre-Ferrandiz, BA, MSc, PhD
Profesor Asociado, Madrid, Spain
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Dávid Dankó, MSc, PhD
Ideas Solutions Kft, Budapest, Hungary
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Olivier Wong
MEDI-QUALITE OMEGA, PARIS, France
- Former Voting Member of the Transparency Committee and the Clinical Practice Guidelines Committee at HAS (Haute Autorité de Santé) and ANSM (French National Agency for Medicines and Health Products Safety)
- Chief Medical Officer of Medi-Qualité Omega, a highly specialized boutique consultancy focused on early pipeline assessment, evidence generation strategy, and early asset development with a strong market access and payer-perspective focus
- Health Technology Assessment (HTA) expert & Speaker in Public Health and Health Economics & Outcomes Research (HEOR) for academic institutions, government agencies, and healthcare organizations
- Former Auditor and Advisor to the French National Health Insurance Funds, Ministry of Health, and national Electronic Medical Record (eMR) initiatives
- Former Drug Development Lead for a rare disease therapy
- Former Academic Family Physician at Paris Cité University and Necker-Enfants Malades Teaching Hospital
- Extensive involvement in the design and implementation of disease management programs at both national and regional levels, including cancer care and screening, rare diseases, avoidable hospitalizations, early discharge pathways, and healthcare provider networks
Issues in Evaluating AI-Enabled Healthcare
Session Type: Issue Panel
Topics: Study Approaches, Health Technology Assessment, Clinical Outcomes
Track: AI
Level: Introductory
ISSUE: As AI-enabled healthcare technologies pursue reimbursement, a fundamental methodological question confronts evaluators: how should the study population be defined? Should economic evaluation encompass all individuals with access to AI-enabled care, analogous to an intent-to-treat framework, or be restricted to those who actively engage with the technology? This distinction is consequential. An access-based population captures the marginal benefit of reaching individuals who would not have sought care otherwise—people newly engaged through AI-enabled pathways who generate incremental health outcomes. An engagement-based cohort yields more precise effectiveness estimates among users but may understate the technology’s broader value by excluding these marginal gains. The choice directly affects cost-effectiveness conclusions and reimbursement decisions for an emerging class of health technologies.
OVERVIEW: Seamus Kent will moderate. Anastasia Chalkidou (10 minutes) will open with a framing of how the UK and Europe consider the value of clinical AI technologies seeking reimbursement and why population definition matters for evidence generation, drawing on their experience with UK and European HTA processes. Blythe Adamson (15 minutes) will argue for evaluating the broader access-based population, presenting her work on the outcomes and economics of an autonomous AI doctor and demonstrating how economic modeling extends observed clinical outcomes to estimate population health impact, including the value of AI models that expand the reach of healthcare. Ian Hooley (15 minutes) will present his evaluation of the outcomes and economics of a virtual maternity care program at Pomelo Care, advocating for engagement-based cohorts with propensity score methods to achieve balanced treatment populations and produce more credible effectiveness estimates for payers. Twenty minutes will be reserved for audience discussion and debate. This session will benefit health technology assessors, health economists, regulators, AI developers, and payers seeking rigorous evaluation frameworks for AI-enabled care.
Moderator
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Seamus Kent, MSc, PhD
Erasmus University Rotterdam, Amsterdam, Netherlands
Speakers
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Anastasia Chalkidou
London, United Kingdom
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Blythe Adamson, MPH, PhD
Doctronic, New York, NY, United States
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Ian J Hooley, BS
Pomelo Care, New York, NY, United States
From National HTA to Joint Clinical Assessment: Exploring Similarities and Differences in Comparator and Outcome Selection Across Six European Health Technology Assessment Agencies
Session Type: Issue Panel
Topics: Health Technology Assessment, Health Policy & Regulatory
Track: Access and Pricing
Level: Intermediate
ISSUE: The HTA Regulation (HTAR) came into effect on January 2025 and the first Joint Clinical Assessments (JCA) on medicinal products started. To understand the predictability of the PICOs (Patient Population, Intervention, Comparators and Outcomes) in JCAs, comparison are made between the Outcomes and Comparators requested by six European HTA bodies on 21 oncology/ATMP drugs in a retrospective study. Further, this comparisons looks at the data studied in the trial and compares this to the HTA bodies needs. The panel explores whether there are differences or similarities between the Outcomes and Comparators the HTA bodies assess in their reports. Another question is whether this variation stems from the different approaches they have on a scoping phase. Further, the panel explores whether data studied in the trials reflects relevant questions HTA bodies require information on. After the panel discussion, the audience will understand how each HTA body decides on their Outcomes and Comparators and also how this may impact the predictability of the assessment scope of the JCAs.
OVERVIEW: The panel starts with a brief scene-setting presentation with results of the retrospective study in which comparators and outcomes six European HTA bodies on 21 drugs are compared. In a moderated discussion, each panelist reflects on the questions how their HTA body decides on the PICO and how the joint work under the HTAR may impact the availability of data to answer their questions. While each panellist is from an HTA organisation, each organisation has a different approach on conducting a scoping phase prior the assessment or assessing based on what the industry submits for national reimbursement applications. Therefore, this session is very relevant to understand the HTA bodies perspective and needs and will help to understand the expectations for the JCA work. The panel ends with a Q&A with the audience.
Moderator
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Anne Willemsen, MSc
Zorginstituut Nederland, Diemen, Netherlands
Anne Willemsen joined the Dutch National Healthcare Institute in 2016, and currently is Chair of the Joint Clinical Assessment (JCA) subgroup under the EU HTA Regulation’s Coordination Group, after having served the JCA Subgroup as co-chair for the past three years (2023-2026). She leads the subgroup through the preparatory phase of the JCA process and will guide it through the production of Joint Clinical Assessments started as of January 2025. Previously she held several roles within the European Network for HTA (EUnetHTA), including spearheading the Secretariat responsible for coordinating JCAs and joint work with the European Medicines Agency. Her work also focused on patient involvement and conflict of interest procedures. With a background in Health Sciences and a recently started PhD, she brings extensive experience in European HTA collaboration, focusing on predictability of the procedures, transparency and stakeholder engagement in JCA.
Speakers
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Roisin Adams
Dublin, Ireland
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Johanna Seeger
Germany
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Camillle Thomassin, PhD
Paris, France
Novel Value Elements and Approaches in HTA
Session Type: Research Podiums
Navigating the New Frontier of Health Technology Assessment and the complex shifts in market access requires balancing strict clinical demands with economic viability and societal equity. This intensive, 1-hour workshop breaks down five seminal pieces of Health Technology Assessment policy research, promoting novel values and explore debatable insights. Participants will gain an actionable blueprint for aligning technical innovation with evolving market access frameworks, addressing critical cost-effectiveness thresholds, real-world data (RWD) integration, and equity mandates.
EXAMINING HOW HEALTH EQUITY IS CONSIDERED IN HEALTH TECHNOLOGY ASSESSMENT: AN ANALYSIS OF DOCUMENTS FROM FOUR BODIES
OBJECTIVES: Recent initiatives by ISPOR, HTAi and international HTA bodies have emphasized the importance of health equity within value assessments. Despite this attention, there is limited understanding of how equity is operationalized within HTA processes. A review of HTA appraisals was conducted to examine how equity is explicitly considered across four major bodies (CDA-AMC, ICER, NICE, and PBAC).
METHODS: We conducted a review of 362 HTA appraisals published between January 2024 and April 2025. We extracted information on the presence of explicit equity considerations, their respective equity subgroup(s) and whether equity considerations were noted in final recommendations. We screened appraisals and supporting documents using PROGRESS-Plus equity keywords. Individuals from CDA-AMC, ICER, and NICE formed an informant panel who validated the approach and provided contextual insights into processes for their respective HTA bodies.
RESULTS: Overall, 241/362 (66.6%) appraisals explicitly referenced health equity. Most equity considerations were descriptive/qualitative in nature, with four using quantitative approaches to assess equity impact directly. Across all appraisals, equity considerations most commonly related to place of residence (37.7%), age (31.7%), and socioeconomic status (26.7%) (subgroups not mutually exclusive). Of the 66.6% appraisals explicitly referencing equity considerations, only 16% of reports explicitly mentioned equity in the phrasing of recommendations. Equity considerations appeared more likely to be explicitly incorporated into recommendation wording where technologies involved complex access barriers for underserved populations, or where equity was a key consideration in the deliberation. These findings were validated with the HTA group to better understand context and current work in this area.
CONCLUSIONS: Health equity is increasingly recognised in HTA. However, our study shows that there are opportunities to strengthen its systematic and transparent integration into decision-making. Clarifying the circumstances in which equity is considered, broadening available methodological approaches, and offering more explicit guidelines could support greater consistency of equity considerations in assessments and decision-making.
REAL-WORLD DATA FOR MEDICAL DEVICE ARTIFICIAL INTELLIGENCE: A EUROPEAN HTA BODIES SURVEY
OBJECTIVES: The EU Health Technology Assessment (HTA) Regulation (HTAR) and European Health Data Space (EHDS) are expected to increase use of real-world data (RWD) in HTA. For Medical Devices Artificial Intelligence (MDAI), RWD may help address evidentiary uncertainty across lifecycle. This study explored European HTA bodies’ perspectives on MDAI assessment, RWD and Health Data Access Bodies (HDABs).
METHODS: A semi-structured online survey was developed within the EU-funded SHAIPED project and distributed to 63 HTA bodies (HTAb) across 30 EU/EEA countries. Closed-ended and open-text questions addressed MDAI evaluation challenges, RWD use, fit-for-purpose criteria and HDAB enabling conditions. Quantitative responses were descriptively analyzed, and qualitative responses thematically analyzed.
RESULTS: Twenty-one responses were received from 16 EU/EEA countries. Preliminary findings indicated limited MDAI-specific formal HTA pathways, with several reporting assessments through general medical-device/digital-health routes. The main HTA challenge identified was the lack of adequate methods to assess technologies that may change over time. A second challenge concerned data-related issues, relevant to adaptive and static MDAI, including data protection and security. Respondents highlighted the need to adapt traditional HTA methods by moving beyond one-time assessment. Suggested adaptations included extending assessment timeframes, defining reassessment triggers, periodically monitoring algorithm drift and setting up conditional reimbursement agreements. Informers emphasized the importance of assessing data-source quality. The prioritized data-quality domains were representativeness, validation against controlled data, and completeness and standardization. HDABs were seen as potentially helping resolve RWD access challenges, by promoting interoperability and data harmonization, enabling secure and timely access to data and supporting data quality, completeness and accuracy.
CONCLUSIONS: This expert consultation gathered EU HTAb perspectives on existing methods for HTA of MDAI and the role of RWD. HTAb highlighted the need for methodologies that accommodate the evolving nature of MDAI and strong reliance on RWD. Experts reported that HDABs under EHDS could help effectively support HTA processes for MDAI.
CRITICAL ANALYSIS OF THE HISTORY AND IMPACT OF COST-EFFECTIVENESS THRESHOLDS: LESSONS AND INSIGHTS FOR DECISION-MAKERS
OBJECTIVES: Conduct a critical analysis of the conceptual basis of, and pragmatic experience with, cost-effectiveness thresholds (CETs) in healthcare policy and decision-making, their roles, limitations and open issues.
METHODS: An integrative review and ‘snowballing’ approach across textbooks, databases, grey literature, and seminal papers is being conducted. Of 3,586 records initially identified, 154 theoretical, 198 empirical, and 47 policy-related papers were considered eligible and included.
RESULTS: Analysis from this ongoing research uncovered at least six dominant themes: 1. Historical CET experiments: Some systems have chosen to leverage CETs; others have chosen not to, either based on principled reasoning and/or their perception of limited utility of CETs as a prioritization tool. 2. Establishing a CET: There is no uncontroversial scientific basis for the determination of any level of CET. Thresholds have been assigned differently across markets, contingent on the theoretical approach, local preferences, and political & technocratic pressures. 3. Ethical considerations: CETs drive equality of QALYs, but do not treat patients as persons equally. Social preferences and the social (distributive) goals of collectively financed health schemes beyond allocative efficiency defined in terms of health maximization are not addressed. 4. Usefulness in decision-making: CETs do not account for rights/based reasoning, and derived priorities inherent in constitutional provisions across nations. 5. Empirical issues: Apart from measurement challenges, CETs are by definition blind to budget impact. 6. Real-world implications: CETs can lead to delayed or reduced access to effective medicines meeting medical needs, resulting in ‘modifications’ to funding and access pathways and threshold levels to address social needs and preferences.
CONCLUSIONS: Cost-effectiveness thresholds raise fundamental ethical and methodological issues, as well as pragmatic questions on impact and the social dimensions of healthcare. Given the potentially far-reaching consequences for both patients and payers of selecting and utilizing an ill-defined CET, these issues should be openly discussed in each health system.
READINESS OF HEALTH TECHNOLOGY DEVELOPERS FOR EU JOINT CLINICAL ASSESSMENT: EVIDENCE REQUIREMENTS, OPERATIONAL CHALLENGES, AND NATIONAL HTA IMPLICATIONS
OBJECTIVES: Regulation (EU) 2021/2282 has shifted EU Health technology assessment (HTA) from voluntary cooperation model to mandatory Joint Clinical Assessment (JCA), beginning in 2025 for oncology medicines and advanced therapy medicinal products. This transition creates new requirements for evidence planning, multi-country PICO alignment, dossier preparation, and national HTA adaptation. This study assessed what JCA “readiness” now means for health technology developers, focusing on evidence requirements, operational bottlenecks, and the residual adaptation.
METHODS: A targeted document analysis was conducted using primary EU sources published since implementation of Regulation (EU) 2021/2282 implementation including the JCA procedural guidance, scoping guidance, dossier-template guidance, methodological Q&A, the 2026 HTACG work program, and first published medicinal-product JCA report (tovorafenib). National HTA guidance from NICE, G-BA, IQWiG, HAS, and AIFA was reviewed to map alignment and divergence in comparators, outcomes, indirect comparisons, real-world evidence, and transparency requirements.
RESULTS: EU JCA requires a PICO-specific dossier for an inclusive, multi-member-state assessment scope, with systematic searches across bibliographic databases, trial registries, HTA reports, EMA files, and patient registries; standard dossier submission is due within 100 days of the first request and no later than 45 days before envisaged CHMP opinion. In tovorafenib JCA, five PICOs were defined but comparative results were submitted for one, assessors issued two formal information requests and the only comparative analysis relied on an unanchored Matching-Adjusted Indirect Comparison (MAIC) with an effective sample size of 5.81. National expectations remain partly divergent: Germany prioritizes nationally specified comparators and adjusted indirect evidence, France emphasizes clinically relevant comparators and overall survival in advanced cancer, and Italy requires broader clinical and economic reimbursement documentation.
CONCLUSIONS: JCA readiness should be treated as a launch capability rather than a submission event, requiring early comparator planning, robust evidence-synthesis, proactive confidentiality management, and pre-defined national overlays for post-JCA access discussions.
Small Sample, Big Decisions: Patient Preference Data for Regulatory and Payer Uses in Rare Diseases
Session Type: Issue Panel
Topics: Patient-Centered Research, Health Policy & Regulatory, Health Technology Assessment
Track: Patient-Centered Evidence
Level: Intermediate
ISSUE: Development and commercialisation of therapies for rare diseases is difficult for a variety of reasons, including lack of established endpoint hierarchy or inadequate understanding of the distribution of symptoms and their impacts in the population. Recent guidelines from the European Medicines Agency/International Committee for Harmonisation, and from the US Food and Drug Administration, position patient preferences as key evidence for regulatory decision-making. Several HTA agencies and payers welcome patient preference data, especially in rare disease contexts where generic quality-of-life instruments are often inadequate. Patient preference elicitation in rare diseases, however, is challenging due to limited sample sizes and heterogeneous disease manifestations. How can we effectively elicit decision-grade patient preference data for regulatory and payer uses?
OVERVIEW: The panel will discuss how patient preferences are incorporated into the regulation and reimbursement of rare disease therapies, and the challenges sponsors face when omitting patient preference data, such as justifying endpoint selection, inadequately powered benefit-risk assessments, and patient value of the therapy. Dr Tervonen will open the panel by discussing key challenges and opportunities with patient preference studies in rare disease research. Dr Ho will then present the sponsor's perspective and discuss what good and inadequate patient-preference studies in rare diseases look like. Mr Low will discuss how patient preferences have shaped reimbursement decisions in the UK, and how patient preference data can be complemented with other real-world data for access discussions. Dr Pignatti will discuss current guidance and regulatory expectations for patient preference data, including possibilities to inform orphan drug designation. The panel will close with a discussion between the panellists and the audience about their experience in the rare disease context. HEOR scientists, regulatory and clinical outcome assessment scientists benefit from attending this intermediate panel.
Moderator
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Tommi Tervonen, PhD
Kielo Research, Zug, Switzerland
Speakers
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Kerrie-Anne Ho, PhD
UCB, Slough, United Kingdom
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Eric Low
Eric Low Consulting, Haddington, United Kingdom
Eric has worked in medical research, health technology appraisal, market access, health policy and patient organisations for over 30 years.
He established Myeloma UK in 1996, developing it from an idea to a successful and respected organisation he led as Chief Executive until 2017. During this time, he designed and implemented a disease blueprint and bench-to-bedside strategic model to accelerate and prioritise the discovery, development of and access to effective new treatments, best practices, as well as information and support for patients and their families
In 2017, Eric established a small specialist consultancy specialising primarily in strategic HTA and market access, life sciences and healthcare policy, and patient and patient group engagement.
Eric is committed and focused on improving patient outcomes. He has a strong track record of delivery and success in building multi-stakeholder research collaborations, patient coalitions, market access strategies, changing policy, and delivering innovative solutions to complex issues, challenges and barriers in progress.
Eric also holds several Board, honorary, advisory and voluntary positions. He also advises numerous medical and health-focused charities on a pro bono basis. He was awarded an OBE for services to charity in the Queen’s Birthday Honours 2012.
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Francesco Pignatti, MD
European Medicines Agency, Amsterdam, Netherlands
Francesco Pignatti graduated as Medical Doctor at the University of Rome. In 1995 he became Research Fellow at the EORTC Data Center in Brussels, Belgium. In 1997 he obtained a Master of Science degree in Biostatistics from the University of Limburg, Belgium. In 1999 he joined the European Medicines Agency (EMA), which is located in Amsterdam since 2019. From 2009 to 2023 he was appointed Head of the Office of Oncology and Haematology, he is currently Scientific Adviser for Oncology. His main regulatory science interests include cancer drug regulation, benefit-risk analysis, and stated preference studies.
From PICO to Practice: Advancing Evidence Synthesis
Session Type: Research Podiums
As evidence requirements become increasingly complex, innovative approaches are needed to synthesize data across multiple studies and populations. This session focuses on PICO prioritization, indirect treatment comparisons, network meta-analysis and reporting standards that strengthen evidence submitted to HTA agencies extrapolating and synthesizing time-to-event data, with particular emphasis on addressing complex censoring mechanisms, external evidence and competing risks.
FORWARD-LOOKING PICO: A PRIORITIZATION AND OPTIMIZATION FRAMEWORK TO SUPPORT GLOBAL HTA STRATEGY IN THE ERA OF JOINT CLINICAL ASSESSMENTS
OBJECTIVES: This study aimed to develop a structured framework for systematic prioritization and optimization of PICO scenarios to support early HTA evidence planning.
METHODS: The framework was developed through synthesis of evidence from published JCA scoping documents, six official HTACG PICO scoping exercises, over 30 published studies, and ISPOR MCDA guidelines. An initial framework was constructed and iteratively refined through three consensus-based internal review cycles involving four HEOR and market access experts, to ensure operational relevance, usability, and alignment with real-world HTA expectations.
RESULTS: The proposed framework establishes a four-phase integrated workflow. Phase 1 (Evidence Mapping) involves targeted landscape searches and clinical guideline reviews to extract and standardize PICO-relevant parameters. Phase 2 (Structured Scoring and Prioritization) generates PICO scenarios through systematic combination of elements, which are evaluated using a weighted multi-criteria scoring system (1-5 scale) across five domains: evidence frequency, clinical relevance, HTA alignment, country-specific factors, and future market dynamics. Element-level scores are aggregated to derive composite scenario scores, enabling ranking into high, moderate, and low priority tiers. Phase 3 (Expert validation) refines prioritized scenarios through stakeholder input and alignment. Phase 4 (Decision Optimization) applies a 2×2 matrix assessing likelihood of HTA request against feasibility of evidence generation to identify decision-critical scenarios.
CONCLUSIONS: This framework provides a transparent and reproducible approach to early PICO planning in the evolving JCA landscape. By integrating likelihood of HTA request with evidence feasibility, it supports more structured evidence planning, enables early identification of data gaps, and helps mitigate potential risks during HTA submissions.
EXTENDING MULTILEVEL NETWORK META-REGRESSION TO UNANCHORED NETWORKS AND SINGLE-ARM STUDIES
OBJECTIVES: Health Technology Assessment decisions require reliable estimates of relative treatment effects between multiple interventions. Population adjustment methods are used to obtain estimates adjusting for differences between study populations. Increasingly, these methods are used to make “unanchored” comparisons with disconnected networks and single-arm studies, relying on the stringent, as-yet untestable assumption of conditional constancy of absolute effects. Multilevel Network Meta-Regression (ML-NMR) is a population adjustment method that coherently synthesises individual participant data and aggregate data from multiple studies. Unlike other methods, ML-NMR provides estimates for any target population, scales to networks of any size, and allows key assumptions to be tested. However, ML-NMR has not yet been proposed for disconnected networks.
METHODS: We extend the ML-NMR framework to unanchored scenarios. Networks are reconnected using either a fixed baseline model that combines similar studies, or a two-stage random baseline model that captures uncertainty from unobserved prognostic factors. To guide anchor selection, we assess population similarity using propensity score overlap and clinical expertise. We propose methods to evaluate the conditional constancy assumption, using cross-validation to assess predictive accuracy and the random baseline variance to quantify unobserved differences between studies. We illustrate different bridging strategies using a manually disconnected network of plaque psoriasis treatments, benchmarking against the fully connected network.
RESULTS: Bridging comparisons between disconnected studies produced unbiased estimates when populations were highly similar but introduced 6-18% bias when populations differed substantially. The random baseline model incorporated additional uncertainty due to unobserved differences which here was small (𝜏; = 0.10 [0.03, 0.32]). Careful anchor selection, based on population similarity and clinical judgement, was essential for valid treatment comparisons.
CONCLUSIONS: Extending ML-NMR to disconnected networks and single-arm studies brings the advantages of this approach to unanchored settings. Practical methods to assess the strong assumptions are provided, and methods are implemented in the multinma R package.
INDIRECT TREATMENT COMPARISON FEASIBILITY ASSESSMENTS IN HEALTH TECHNOLOGY SUBMISSIONS: A REVIEW OF NICE TECHNOLOGY APPRAISALS
OBJECTIVES: Feasibility assessments are a critical preliminary step in determining whether indirect treatment comparisons (ITCs) can be robustly conducted and which methods are most suitable. However, approaches to assessing feasibility are not standardised and are often inconsistently reported. This study reviewed National Institute for Health and Care Excellence (NICE) technology appraisals (TAs) to characterise how ITC feasibility assessments are conducted, identify common practices, and explore gaps in reporting.
METHODS: A targeted review of NICE TAs published between April 2023 and April 2026 was conducted. Terminated appraisals were excluded. Data extraction was based on initial company evidence submissions and focused on: 1) whether an ITC was conducted and its type, 2) whether an ITC feasibility assessment was conducted and terminology used, and 3) the aspects of ITC feasibility assessed.
RESULTS: Of the 213 TAs identified, 144 included an ITC. Feasibility assessments were reported in 138 initial submissions (126 with an ITC, 12 without). Terminology and reporting varied considerably across appraisals. While 73% explicitly used the term “feasibility assessment”, others described the process using non-standardised terminology. Across key components of feasibility assessments, population characteristics and study design were most consistently assessed (80 and 76%, respectively), followed by outcome definitions and reporting (71%). Explicit reporting of treatment effect modifiers and prognostic variables was less frequent (64%). Notably, 55% of TAs indicated that more detailed feasibility assessment findings were provided in appendices that were often not publicly available.
CONCLUSIONS: ITC feasibility assessments in NICE TAs show substantial variability and limited transparency, with detailed reporting often confined to non-public appendices. With the emergence of the EU Joint Clinical Assessment (JCA) and its accompanying methodological guidance, the need for consistent and transparent feasibility assessments is becoming increasingly important. Development of a general feasibility framework could improve standardisation and enable clearer, publicly accessible reporting, thereby strengthening confidence in ITC-related evidence.
THE PRISMA-ECONEVAL 2026 STATEMENT: EXTENSION OF PRISMA AS A REPORTING GUIDELINE FOR SYSTEMATIC REVIEWS OF HEALTH ECONOMIC EVALUATIONS
OBJECTIVES: Systematic reviews of health economic evaluations (SR-HEEs) are increasingly used to inform policies and decisions about the allocation of healthcare resources, including reimbursement and coverage decisions by health technology assessment (HTA) agencies, and to identify methodological gaps in health economic evaluation. Transparent, complete, and accurate reporting of SR-HEEs is therefore essential to support critical appraisal and appropriate use of review findings. We here present the extension to the Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) 2020 statement for SR-HEEs: the PRISMA-EconEval 2026 statement.
METHODS: The development of PRISMA-EconEval 2026 followed four stages according to recommendations on reporting guideline development and modelled on approaches used in previous extensions of PRISMA: (1) scoping review of methodological literature on conduct and reporting of SR-HEE; (2) multi-round Delphi survey of interest holders; (3) consensus meeting; and (4) post-consensus piloting. A Public Involvement Reference Group comprising patient and public representatives participated in all stages.
RESULTS: PRISMA-EconEval 2026 statement includes 34 reporting items and is designed for all SR-HEEs, irrespective of their objective(s). Twelve items remained unchanged from PRISMA 2020, 15 were modified, and seven were new additions including conceptual framework, patient and public involvement and engagement, and artificial intelligence and language model use. The statement is primarily designed for systematic reviews of full health economic evaluations, rather than non-systematic reviews and systematic reviews of partial economic evaluations. The statement is intended to guide reporting of SR-HEEs. Readers are encouraged to consult the accompanying Explanation and Elaboration paper, which describes the essential and additional elements of each reporting item and provides exemplars.
CONCLUSIONS: PRISMA-EconEval 2026 statement provides guidance for researchers reporting SR-HEEs, as well as reviewers and editors involved in evaluating SR-HEEs. It may also support systematic reviewers during review planning and conduct and facilitate healthcare decision-making by HTA agencies and other decision-makers.
11:00 - 11:30
Break (Exhibit Hall)
Session Type: General Meeting
11:30 - 13:00
Plenary Session 3: Closing Reflections and ISPOR’s Scientific Future
Session Type: Plenary
As ISPOR Europe 2026 draws to a close, join us for final reflections and forward-looking perspectives from the Society’s leadership. ISPOR’s Chief Executive Officer will highlight key takeaways from the conference, while the Chief Science Officer and Executive Director of the new ISPOR Institute for Healthcare Transformation will share updates on ISPOR’s evolving scientific priorities and opportunities for member engagement.
Immediately following the opening presentations, the scientific plenary panel will take the stage.
Speakers
Plenary Session 3: We Optimize Treatments—But How Do We Optimize the System?
Session Type: Plenary
Topics: Health Policy & Regulatory, Real World Data & Information Systems, Methodological & Statistical Research
Track: AI
Level: Intermediate
Bringing the conference to a forward-looking conclusion, this plenary asks: If healthcare systems are asking new questions about value, what evidence will they need—and how must HEOR evolve to deliver it?
As priorities expand to include population health, system resilience, sustainability, and long-term societal outcomes, decision-makers need evidence that extends beyond individual interventions and spans pharmaceuticals, medical technologies, care delivery, prevention, and public health. Through perspectives from payers, clinicians, evidence-generation experts, and data science leaders, the session will explore how more integrated evidence ecosystems can support complex resource-allocation decisions and align stakeholders around shared outcomes.
The discussion will also examine the potential of artificial intelligence and advanced analytics to enable faster, more adaptive evidence generation, continuous monitoring, and predictive decision support. It will consider the accompanying challenges of credibility, trust, methodological rigor, and implementation—and what HEOR must do to remain relevant and actionable in an increasingly interconnected healthcare landscape.
Moderator
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Malina Müller, BA, MA, PhD
WifOR Institute, Darmstadt, Germany
