Program

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

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

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.
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

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.
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.
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

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.
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.
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

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

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.
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).
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.

8:00 - 17:00

Reimbursement Systems for Pharmaceuticals in Europe

Session Type: Short Course

Topics: Health Policy & Regulatory

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

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.
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.
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.
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).

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

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

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.
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).
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

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

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.”
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.
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 health economics and outcomes research (HEOR) is conducted from literature reviews and evidence synthesis to economic modeling and HTA submissions. As the field moves beyond experimentation, professionals face a new challenge: how to responsibly validate, implement, and scale GenAI solutions in real-world HEOR settings.

This intermediate-level course builds upon basic concepts and is designed for HEOR professionals, data scientists, and decision makers seeking to understand not only how GenAI works, but how to implement and evaluate it effectively within regulated and evidence-driven environments.

The course provides a practical framework for moving “from prototype to practice,” describing the lifecycle of GenAI implementation—from early sprints and pilot projects to production deployment. Participants will explore both technical and organizational perspectives, including workflow orchestration, modularization, scaling, and change management.

Retrieval-Augmented Generation (RAG) is a cornerstone architecture that integrates external knowledge bases into LLM workflows. Faculty will discuss why RAG is particularly relevant for HEOR, demonstrating how external information (eg, clinical data, published evidence, HTA guidance) can be incorporated in GenAI workflows according to best practice standards and used to improve factual accuracy and traceability. A guided practical session is included so participants become familiar with how to implement a simple RAG pipeline, learning how to chunk data, generate embeddings, and augment prompts for domain-specific use.

The course will also provide an extensive overview of Agentic AI, a fast-evolving frontier in AI automation. Participants will examine how autonomous AI “agents” can coordinate multi-step HEOR processes—such as literature updates, model maintenance, or simulated committee reviews—while maintaining control and accountability. A second practical session will demonstrate an agentic workflow in action, showcasing task orchestration, monitoring, and boundary setting.

Beyond technical topics, there will be a focus on evaluation and validation of GenAI solutions for HEOR, where participants will learn how to critically assess GenAI tools in terms of reliability, reproducibility, and regulatory alignment. This will also be discussed in the context of potential ethical concerns around the application of AI. Using frameworks such as ELEVATE-GenAI, and referencing NICE and FDA guidance, participants will learn how to ensure that AI-driven outputs meet HEOR’s high standards for quality and transparency.

By the end of this course, participants will understand how to bridge the gap between exploratory AI use and operational excellence. They will leave with actionable frameworks and hands-on knowledge to evaluate, implement, and govern GenAI tools that enhance productivity, transparency, and scientific integrity across HEOR activities.

PREREQUISITES: Completion of the “Applied Generative AI for HEOR: Introduction” ISPOR course or familiarity with concepts such as prompt engineering, APIs, and LLM workflows. A basic understanding of Python or other similar scripting languages is recommended to get the most benefit from the guided practical sessions.

Speakers

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.
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.
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.
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.

Practical Applications of Large Language Models for Real-World Evidence Generation and HEOR

Session Type: Short Course

Topics: Methodological & Statistical Research

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

Manuel Cossio, MMed, MEng
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.
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

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.
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.
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.
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.
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

Rob Abbott
ISPOR, Lawrenceville, NJ, United States

Plenary Session 1: When Metrics Miss the Point: 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

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

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.

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

Bill Malcolm, MSc
Bristol Myers Squibb, Middlesex, United Kingdom
Bill Malcolm Leads the Economic & Predictive Modeling Team for Global HEOR at BMS. He has over 20 years of experience across a broad range of therapeutic areas and has published extensively in the field of HEOR. His current focus is transforming HEOR workflows using Generative AI methods and he has published several key papers on this innovative topic area. Bill is a member of the ISPOR AI Working Group.

Speakers

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.
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.

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

Ronan Mahon
University of Galway, Galway, Ireland

Speakers

Subhajit Gupta
India
George Joseph, MS, PhD
Cambridge, MA, United States
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

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

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.
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

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.

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

Oliver Staples, PhD
Basel, Switzerland

Speakers

Aodan Tynan, MA
London, United Kingdom
Niklas Hedberg, MSc
TLV, Stockholm, Sweden
Duane Schulthess
VitalTransformation BVBA, Wezembeek Oppem, Belgium

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

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.

Speaker

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.

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

Mikkel Oestergaard, PhD
Merck Sharp & Dohme International Service B.V., Zurich, Switzerland
Executive Director, HTA Statistics, MSD

Speakers

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
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 ).

10:30 - 13:30

Poster Session 1

Session Type: General Meeting

Poster Tours will be from 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.

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

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

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.
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.

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

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.

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.

Speaker

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.

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

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.

Speaker

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.

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

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.

Speaker

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.

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

Julie De Almeida, BSc
Doncaster, United Kingdom

Speakers

Isabelle Durand-Zaleski, MPP, PhD, MD
Paris, France
Maria-Magdalena Balp, MASc, MD
Basel, Switzerland
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

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

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.
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.

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.

Speaker

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.

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.

16:00 - 19:00

Poster Session 2

Session Type: General Meeting

Poster Tours 16:00–16:45 | Presenters will be with their posters from 18:00 – 19:00

16:45 - 17:45

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.

Speaker

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.

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.

Speaker

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.

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. .

Speaker

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 ).

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.

Speakers

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.
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

Raphael Sonabend-Friend
NICE, 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.

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.

Speaker

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.

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

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.

Speaker

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

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.

Speaker

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.

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

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

Beth Devine, MBA, MSc, PharmD, PhD
University of Washington, Seattle, WA, United States
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

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.

Speaker

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.

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.

Speakers

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.
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.
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

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

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.
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.

Speaker

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.

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.

Speaker

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.

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.

Speakers

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.
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.

Speakers

Raphael Sonabend-Friend
NICE, 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.
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.

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).

Speaker

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.

10:30 - 13:30

Poster Session 3

Session Type: General Meeting

Poster Tours will be from 11:30–12:15 | Presenters will be with their posters from 12:30–13:30

11:15 - 13:00

Lunch Service (Exhibit Hall)

Session Type: General Meeting

13:45 - 14:45

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

Helen Kitchen, MSc
Clarivate, London, United Kingdom

Speakers

Piper Fromy, PhD
Saumur, France
Catherine Bottomley, PhD
Clarivate Analytics, London, United Kingdom
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

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.

Speaker

Manuel Cossio, MMed, MEng
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.

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

Alex Porteous, BA, MSc
Costello Medical, London, United Kingdom

Speakers

Grace Hampson
Office of Health Economics, London, United Kingdom
James Jarrett, BA, MA, PhD
Gilead Sciences Inc, London, United Kingdom

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.

Speaker

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.

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.

Speaker

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.

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.

Speakers

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.
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

Antonio Remiro-Azócar, BSc, MSc

Speakers

Jeroen Jansen
United States
Renato Panaro, B.S., M.Sc.
Göttingen, Germany
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

Matthijs M Versteegh, BSc, MA, PhD
Huygens & Versteegh, Zwijndrecht, Netherlands

Speakers

Dawn Lee, MSc
University of Exeter PenTAG, Exeter, United Kingdom
Matthew Franklin, BA, MSc, PhD
SCHARR, University of Sheffield, Sheffield, United Kingdom
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

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

Cornelis Boersma, PhD
Health-Ecore, Zeist, Netherlands

Speakers

Adina Turcu-Stiolica, PhD
University Of Medicine And Pharmacy Of Craiova, Craiova, Romania
Aleksandra Grozdanova
Skopje
Tarik Catic, PhD
Sarajevo, Bosnia And Herzegovina

16:00 - 19:00

Poster Session 4

Session Type: General Meeting

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.

Speaker

Raphael Sonabend-Friend
NICE, 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.

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.

Speaker

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.

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

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.

Speaker

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 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.

Speakers

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.
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.
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.

Speaker

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.

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

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

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.
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.

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.

Speaker

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.

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.

Speaker

Pall Jonsson, BSc, PhD
National Institute for Health and Care Excellence (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.

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.

Speaker

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).

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

Laith Yakob
Adelphi Values PROVE, Bollington, United Kingdom

Speakers

Edward I Broughton, BSc, MPH, PhD
Pfizer, Muenchen, Germany
Grace Hampson
Office of Health Economics, London, United Kingdom
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

Nuriya Musina, PhD
Sechenov University, Dubai, United Arab Emirates

Speakers

Kefah Ali Alqawasmeh
AUH, United Arab Emirates
Jani Mueller, PhD
Johannesburg, South Africa
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

Esther Nzenza, BSc

Speakers

Olivia Wu, MSc, PhD
University of Glasgow, Glasgow, United Kingdom
Herwig Ostermann, MSc, PhD
Vienna, Austria
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

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.

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.

Speakers

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.
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

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.

Speaker

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

Andreas Heddini
AstraZeneca, Switzerland

Speakers

York Francis Zoellner
Hamburg, Germany
Penilla Gunther, .
Stockholm, Sweden
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

9:00 - 11:30

Poster Session 5

Session Type: General Meeting

Presenters will be with their posters from 09:00 – 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

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.

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

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 Head of Pricing, Policy & Stakeholder Engagement at Cencora Global Consulting Services. He oversees all activities related to global pricing strategies, as well as all activities related to European health policies. 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

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.
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.

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

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.

Speaker

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.

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

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.”

Speaker

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

Seamus Kent, MSc, PhD
Erasmus University Rotterdam, Amsterdam, Netherlands

Speakers

Anastasia Chalkidou
London, United Kingdom
Blythe Adamson, MPH, PhD
Doctronic, New York, NY, United States
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

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.

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.

Speakers

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.
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.

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.

Speaker

Laura Pizzi, MPH, PharmD
ISPOR, Lawrenceville, NJ, United States
Laura Pizzi is Chief Science Officer at ISPOR.

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

Malina Müller, BA, MA, PhD
WifOR Institute, Darmstadt, Germany

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