Sun 9 Nov
7:00 - 18:00
Registration Hours
Session Type: General Meeting
8:00 - 12:00
Advanced Patient-Reported Outcomes
Session Type: Short Course
Topics: Patient-Centered Research
Level: Advanced
Separate registration required.
This course provides an in-depth discussion of the steps needed to successfully implement patient-reported outcomes (PRO) measurement within the drug development program to generate data to support patient-centered value messages. Formulation of a successful PRO strategy requires an understanding of PRO instrument selection, psychometric evaluation, data capture, and interpretation to negotiate regulatory, reimbursement, and market access drug development hurdles. Judging PRO instrument quality and appropriateness can be challenging.
The course will present the key elements to consider at each step in reviewing and selecting PRO measures and determining the need for new instruments. In addition, participants will gain a better understanding of regulatory expectations for qualitative and quantitative evidence to support the quality of PRO measures and aspects to consider when interpreting meaningful change. The course will include interactive discussions of PRO success stories and common pitfalls to watch out for during PRO implementation in clinical trial programs.
Participants will gain the knowledge and skills required to take on a more active and confident role in the PRO strategy and implementation process.
PREREQUISITE: This course assumes that participants will have a basic knowledge of key PRO-related concepts (eg, health-related quality of life, symptoms, impacts, a general knowledge of the PRO development steps, and a working knowledge of PRO measurement within clinical programs).
Speakers
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Rebecca Crawford, MA
RTI Health Solutions, Manchester, United Kingdom
Ms. Crawford has 13 years of experience providing consultative support to pharmaceutical companies with a focus on the development of patient-reported outcome (PRO) measurement strategies to best meet the needs of their clinical trial programs.
Ms. Crawford has developed, culturally adapted, and validated clinical outcome assessment measures, including PROs for several different therapeutic areas. Ms. Crawford has expertise in research design and in the application of both traditional and innovative qualitative research methods, including the collection and analysis of social media data to provide insights into the patient disease and treatment experience.
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Shanshan Qin, PhD
RTI Health Solutions, Durham, 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 10 years of experience as a psychometric analyst and statistic consultant and has been working on psychometric evaluation of clinical outcome assessments (COAs) and support of regulatory review of COA labeling claims in various therapeutic areas, including dermatology, gastroenterology, diabetes, oncology, ophthalmology, and mental disorder. She is an experienced programmer of SAS, R, and IRT PRO.
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Lynda Doward, MSc
RTI Health Solutions, Manchester, United Kingdom
Ms. Doward has over 30 years of experience conducting patient-centered outcomes research including the provision of strategic advice to pharmaceutical companies in the incorporation of the patient voice into drug development programs. Ms. Doward is an expert in the development of clinical outcome assessment (COA) strategies including the development of patient-centered clinical trial endpoints, the implementation of patient-reported and other COA outcome measures in clinical trial programs, and the inclusion of PRO and other COA value messages at key drug development hurdles. Ms. Doward has extensive experience in supporting pharmaceutical clients in their COA-related submissions to regulatory agencies in Europe and the US and advises on health-utility measurement strategies for reimbursement agencies in Europe. Ms. Doward has led the development of over 40 COA questionnaires that have been adapted and validated for use in over 60 languages worldwide.
Ms. Doward currently serves on the ISPOR COA Special Interest Group (leadership committee) and the ISPOR Patient Council (member) and was a member of the leadership committee of the completed ISPOR Good Research Practices Task Force for the measurement of health state utilities in clinical trials. Ms. Doward has acted as a consultant to the World Health Organization and has served as a Research Advisor to the UK Department of Health, and medical charities in the United Kingdom.
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Nicholas J. Rockwood, PhD
RHI 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).
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, will review the most recent RWE frameworks and guidelines, and examine case studies in which RWE was used in regulatory and HTA approval. The second half of the course is an extended example in which participants will examine a study that could support an indication expansion, and interactively discuss how choices made in the design and implementation may affect the meaning and interpretability of results.
PREREQUISITE: Students are expected to be familiar with relevant concepts and methodologies for analyzing real-world data.
Speakers
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Jeremy Rassen, ScD
Aetion, Inc., New York, NY, United States
Jeremy A. Rassen, MS, ScD is a pharmacoepidemiologist with 25 years of academic and industry experience. He is cofounder, president, and chief technology officer at Aetion, a healthcare technology company that delivers real-world evidence for life sciences companies, payers, and regulatory agencies. Prior to founding Aetion, Dr. Rassen was assistant professor of medicine at Harvard Medical School, where he focused on methods to improve the quality and validity of real-world data studies. He also worked in Silicon Valley in a variety of tech companies. Dr. Rassen received his bachelor’s degree in computer science from Harvard College and his master’s and doctorate degrees in Epidemiology from the Harvard TH Chan School of Public Health.
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Shirley Wang, PhD
Brigham & Women's Hospital, Harvard Medical School, Boston, MA, United States
Dr. Wang is an associate professor at Brigham and Women’s Hospital, Harvard Medical School and lead epidemiologist for the Food and Drug Administration’s (FDA) Sentinel Innovation Center. She leads the Meta-Research in Pharmacoepidemiology program, with recent projects aimed at improving the transparency, reproducibility, and robustness of evidence from healthcare databases (www.repeatinitiative.org) and informing when and how real-world evidence studies can draw causal conclusions to inform regulatory or other healthcare decision-making (www.rctduplicate.org). She is currently PI on multiple NIH R01s and is also funded by FDA. Her methods work has received 3 awards from international societies.
Health Economic Modeling in R: A Hands-on Introduction
Session Type: Short Course
Topics: Economic Evaluation
Level: Introductory
Separate registration required.
This highly practical course will outline the computational and transparency advantages of using R, for those used in health economic modelling using Microsoft Excel. This course explores the use of R for health economic modelling in the context of health economics and outcomes research (HEOR) and faculty will guide the participants through practical examples of HEOR. The faculty are expert speakers who have diverse experience in academia, national Health Technology Assessment agencies (NICE, NCPE), and industry. The faculty will lead participants through practical examples of health economic modelling including using R for Markov models from deterministic analysis through to probabilistic sensitivity analysis and EVPI. Additional useful packages for modelling using R will also be discussed. All sessions will interchange between descriptive lectures and hands-on exercises. Participants will be provided with materials, including model examples in R and information on where to go for further learning. This course is designed for those with some familiarity with modelling techniques, such as the concepts of discrete time cohort Markov models and probabilistic sensitivity analysis, but familiarity with R coding is not required. Attendees will require a laptop with RStudio (v1.1.0 or higher) and R (v4.2.1 or higher) downloaded and installed.
Speakers
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Felicity Lamrock, PhD
Queen's University Belfast, Belfast, Ireland
Dr. Felicity Lamrock is a senior lecturer in Data Analytics at Queen’s University Belfast. She is currently the Director of the Northern Ireland Centre for Health Analytics and Decision Science (NI-CHADS) with a focus on the analysis of health data for decision modelling. Current projects include a range of disease areas including cancer, rare diseases, diabetes, COVID-19, and cardiovascular disease. Felicity was previously a statistician at the National Centre for Pharmacoeconomics (NCPE) working with a team of pharmacists and clinicians on Health Technology Assessments to advise the Health Service Executive on the recommendation of new drug therapies in Ireland. She remains involved with NCPE as a statistical advisor and is exploring how Northern Ireland could benefit from more decision modelling/pharmacoeconomic assessment.
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Gianluca Baio, PhD
University College London, London, United Kingdom
Gianluca is professor of Statistics and Health Economics in the Department of Statistical Science at University College London. He graduated in Statistics and Economics from the University of Florence, Italy and then completed a PhD program in Applied Statistics again at the University of Florence, after a period at the Program on the Pharmaceutical Industry at the MIT Sloan School of Management, Cambridge (USA). He worked as a research fellow and then Lecturer in the Department of Statistical Sciences at University College London, UK. His main interests are in Bayesian statistical modelling for cost effectiveness analysis and decision-making problems in the health systems, hierarchical/multilevel models and causal inference using the decision-theoretic approach. Gianluca leads the Statistics for Health Economic Evaluation research group within the department of Statistical Science, whose activity revolves around the development and application of Bayesian statistical methodology for health economic evaluation, eg, cost-effectiveness or cost-utility analysis. He also collaborates with the UK National Institute for Health and Care Excellence (NICE) as a scientific advisor on Health Technology Appraisal projects. He has developed and maintains several R packages, many specifically devoted to health economic evaluation, including BCEA, survHE and missingHE.
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Howard Thom, BA, MSc, PhD
Bristol Medical School : Population Health Sciences, Bristol, United Kingdom
Howard Thom has worked for over 10 years developing health economic models in R. These have been in many disease areas, including heart disease, stroke, physiotherapy, mental health, rheumatology, dermatology, and oncology. His methodological interests are structural uncertainty, value of information analysis, and the use of R for efficient modelling. He founded and co-chairs the R for Health Technology Assessment (HTA) scientific committee, organizing annual workshops on the use of R in HTA. He currently works at both the University of Bristol and at Clifton Insight, giving him the perspective of both commercial consulting and academia.
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Rose Hart, BSc, PhD
Dark Peak Analytics, Sheffield, United Kingdom
Rose Hart is a health economist with over 8 years of consultancy experience, specialising in health economic modelling and the development of advanced, reproducible tools in R and R Shiny. She has particular expertise in health technology assessment, early modelling, and value communication, and is known for building scalable solutions that integrate economic and statistical methods. Rose is passionate about embedding emerging technologies and efficient coding practices into modelling workflows.
Introduction to Applied Generative AI for HEOR
Session Type: Short Course
Topics: Methodological & Statistical Research
Level: Introductory
Separate registration required.
The rapid advancement in generative artificial intelligence (Gen AI) 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 revolutionizing 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 to conduct scientific research and gain an understanding on issues pertaining to privacy and security when using Gen AI for HEOR. Participants will further explore specific applications of these models for conducting robust scientific HEOR research in systematic literature review (SLR), real-world evidence analysis, 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. Participants who wish to gain hands-on experience are required to bring their laptops with Python installed.
PREREQUISITES: Students should have a general understanding of common HEOR concepts such as SLRs and cost-effectiveness models. Knowledge of Python or similar programming languages such as R is considered a benefit, but not required.
Speakers
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Sven L Klijn, MSc
Bristol Myers Squibb, Princeton, NJ, United States
Sven Klijn is director at Bristol Myers Squibb in the Global HEOR Economic & Predictive Modeling group, where he leads the innovative modeling agenda in hematology and cell therapy. In addition, Sven has an active role in providing modeling education and masterclasses at international congresses. He has widely published on innovative methods, especially in the field of survival extrapolation and Generative AI. Sven has training in public health and health economics and previously had various roles in CROs related to health economic modeling.
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William Rawlinson
Estima Scientific, London, United Kingdom
Will is a senior health economist at Estima Scientific holding a degree in Physics and Philosophy from the University of Oxford. Will has 4 years’ experience developing cost-utility models and has specialized in applications of generative AI to health economic modelling. Will has published on the automation of R modelling using large language models (LLMs), and more recently has focused on applications of LLMs to Excel modelling and model reporting.
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Tim Reason, MSc
Estima Scientific, South Ruislip, 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
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Bertalan Németh, PhD
Syreon Research Institute, Budapest, Hungary
Bertalan Németh, PhD, graduated from the Corvinus University of Budapest (MSc in Quantitative economics and Operation research), the Eötvös Loránd University (Pharmaceutical economics and drug policies), and the Semmelweis University School of PhD Studies. 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 is responsible for strategic consulting, and he is involved in various projects that include modelling, economic evaluation in health, health technology assessment and health statistics as well. 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 META Conference. Bertalan was also a faculty member of the global ISPOR HTA Training and was the module leader of Health Technology Assessment for the MSc program at Eötvös Loránd University.
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Rok Hren, MSc, PhD
Hren & Partner d.o.o., Ljubljana, Slovenia
Rok Hren has more than 15 years of commercial experience in pharmaceutical industry and more than 12 years on a board level in both (1) line management, which has included full P&L responsibility for operations in Slovenia and Romania, and (2) leadership regional functions in Central and Eastern Europe. He regularly presents on the topic of pharmaceutical economics and policies at conferences in Europe and is well experienced in healthcare media business.
He received his PhD in Physiology and Biophysics from Dalhousie University, Canada and MSc in International Health Policy (Health Economics) with Distinction from London School of Economics and Political Science, UK while he was a post-Doctoral Fellow at Nora Eccles Harrison Cardiovascular Research and Training Institute, University of Utah Medical School, USA. He is also an assistant professor at the University of Ljubljana and past president of the ISPOR Slovenia Regional Chapter. In total, his publications gathered 300/394 citations (excluding self-citations) in WoS/Scopus (as of September 10, 2016).
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Katarzyna Kolasa, PhD
Kozminski University, Warsaw, Poland
Driven with passion to improve healthcare, Katarzyna has focused her academic and business career on health economics.
She has been working with multiple pricing and reimbursement challenges worldwide for the last 25 years, while holding various regional and global leadership positions at Astra Zeneca, BMS, Biogen Idec, Lundbeck, GE Healthcare, Straub Medical, BD, and the Swedish County Council of Kalmar. Katarzyna is mentor and consultant to start ups involved in the development of innovative medical devices and digital health solutions from both Holland and Poland.
Since 2000, she has been an academic teacher and supervisor for over 30 MBA and PhD students. In partnership with the Polish Medical Research Agency, Deloitte Digital and the Polish Central Hospital of Ministry of Interior Affairs, she founded the first Digital Health 6 months educational program designed for digital transformation leaders working in the healthcare sector. Katarzyna developed an innovative Master Program Health Economics & Big Data (HEBDA) with the first edition being financed by EU Power Grant 2018 as well.
She is the founder of the Global Special Interest Group Digital Health and short courses “The Role of Digital Endpoints in the Value Generation for Health Technologies”, “Risk-Sharing/Performance-Based Arrangements in Developing Countries” for ISPOR, The Professional Society for Health Economics and Outcomes Research. She is currently a member of the ISPOR Education Council and a previous member of the ISPOR Health Science Policy Council as well.
Katarzyna has dedicated her academic research towards methodological advancements into the value assessment of pharmaceuticals, medical devices, and digital health solutions. Passionate about Big Data, she led the first project of machine learning adaptation for the optimal utilization of CT scanners granted by the Polish Ministry of Health. Since January 2022, she is the leader of AI special interest group at the Polish Chamber of Physicians. With the patronage of the Polish Parliamentary Commission for Innovation & Digitalization, she organized the first dialog about the societal preferences towards the adoption of AI in the healthcare in Poland.
Being a coauthor of more than 50 IF publications, she has presented her research at more than 60 international scientific conferences. As of 2022, Google Scholar reports over 730 citations to her work.
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
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Mondher Toumi, PhD
Inovintell, Luxembourg, Luxembourg
Professor Mondher Toumi is an MD by training and holds 2 MSc in Biostatistics, and in Biological Sciences (option pharmacology) and a PhD in Economic Sciences. He is a professor of Public Health at Aix-Marseille University. After working for 12 years as a research manager in the Department of Pharmacology at the University of Marseille, he joined the Public Health Department in 1993. In 1995, he entered the pharmaceutical industry and worked there for 13 years.
Mondher Toumi was appointed global vice president at Lundbeck A/S in charge of health economics, outcome research, pricing, market access, epidemiology, risk management, governmental affairs, and competitive intelligence. In 2008, he founded Creativ-Ceutical, an international consulting firm dedicated to support health industries and authorities in strategic decision-making.
In February 2009, he was appointed professor at Lyon I University in the Department of Decision Sciences and Health Policies. He launched the first European University Diploma of Market Access (EMAUD), an international course already followed by more than 500 students. Additionally, he recently created the Market Access Society to promote research and scientific activities around market access, public health and health economic assessment. He is chief editor of the Journal of Market Access and Health Policy (JMAHP).
Since September 2014, he joined the research unit EA3279 of the Public Health Department, at Aix-Marseille University (France) as Full Professor. Mondher Toumi is also a visiting professor at Beijing University (Third Hospital).
In June 2022 Mondher Toumi founded InovIntell, an international venture dedicated to AI in life sciences.
He is a recognized expert in health economics and an authority on market access and risk management. He published more than 200 scientific publications and authored or co-authored several books predominantly in the fields of market access and health economics.
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Frank-Ulrich Fricke, PhD
Nuremberg, Germany
Frank-Ulrich Fricke is a professor of health economics at the Technische Hochschule Nürnberg Georg Simon Ohm and an impartial member of the arbitration board on drug prices in the German healthcare system (Schiedsstelle nach § 130b SGB V) since 2011. He has served as a faculty dean since 2017. After studying business administration and a PhD in economics, Frank-Ulrich worked in industry and in consulting for several years. Main areas of interest have been market access, pricing and reimbursement, health policy and health economic evaluations. Frank-Ulrich is a member of several national as well as international professional associations.
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Maarten Jacobus Postma, PhD
Professor Maarten J. Postma holds the chair Global Health Economics at the University Medical Center Groningen (UMCG) and the faculty of Economics & Business, both at the University of Groningen. Also, he is Prof in Pharmacoeconomics at the Departments of Pharmacy at the University of Groningen, Pharmacology at the Airlangga University (Surabaya, Indonesia) and Pharmaceutical Care Innovation at Universitas Padjadjaran (Bandung, Indonesia).
He did his MSc in econometrics and his PhD in health economics. He specifically leads a team of 100 staff, PhD and post-doc researchers in health and pharmacoeconomics, contributing to many international research networks and scientific communications. He specializes in the role of pharmacoeconomics/health economics in the reimbursement process. He serves (served) on various committees advising the Dutch government on reimbursement of drugs and vaccines. Also, he is an advisor to various health economics consultancy companies and pharmaceutical companies worldwide, Ministries of Health in neighboring countries, member of editorial boards of scientific journals (eg, Value in Health), on advisory boards for pharmaceutical companies and expert consultant for WHO. He is a member of the UK’s Joint Committee of Vaccination & Immunization since 2014.
He has almost 1000 peer-reviewed international publications, an H-factor of 100 and extensive teaching/lecturing experience (Groningen, Amsterdam, Utrecht, Heidelberg, Bielefeld, Ankara, Sofia, various universities in Indonesia, JoBurg, Moscow and Hanoi). He ranks 2nd (1st) most innovative (health) economist of the Netherlands. He attracted 25 EU-projects, 10 WHO-projects, various national projects from the National Institute of Public Health and the MoH, projects from NGOs and multiple projects from pharma, aggregating over 25 million EUR of funds for university in the last decade. His research often features in newspapers and Dutch television. He is involved in 5 spin-off consultancy companies from the University in various roles, inclusive director, shareholder and advisor. He was listed as the second most innovative economist of the Netherlands for 2024.
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Keith H Tolley, BA, MPhil, MPP
Tolley Limited, Buxton, United Kingdom
Keith has over 35 years’ experience in health economics across academia (University of York - Centre for Health Economics and University of Nottingham, UK 1987-1997), for several pharmaceutical companies, including GSK, Pfizer, and Ortho Biotech (1997-2005), and in consultancy as a Director at Mapi (Adelphi) Values and now Tolley. He has direct experience of HTA as performed by NICE and SMC, and reimbursement and pricing issues around Europe. Keith has managed and strategically contributed to company submissions to NICE and SMC across a range of disease areas. He has also reviewed and been involved in the development of health economic models for NICE and SMC and other HTA bodies and has reviewed economic models for their suitability (eg, structure, data inputs) for drug reimbursement purposes.
Keith is also a health economics assessor with the SMC, a position he has held since 2005, having previously been an industry representative on the NDC. In 2013, Keith also became an assessor for the All Wales Medicine Strategy Group (AWMSG) and has provided expert advice as part of the NICE Early Scientific Advice Program.
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Krzysztof Kloc
United States
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).
12:00 - 13:00
Break (Coffee Service, Lunch on Own)
Session Type: General Meeting
13:00 - 17:00
Budget Impact Analysis II: Practical Steps to Building a BIA and Enhanced Applications
Session Type: Short Course
Topics: Economic Evaluation
Level: Intermediate
Separate registration required.
This course presents an overview of budget impact analysis, the 6-step approach to developing budget impact analyses, and various practical applications. The course will review the basics of budget impact analysis, interpretation of results, and simplicity versus accuracy and face validity. Technical topics will include static versus dynamic budget impact models, considerations for device and diagnostic technologies, and realistic features such as patient copayments and use of generics. The instructors will walk through two different budget impact analyses programmed in Excel (one static and one dynamic) and work with participants during hands-on exercises to enhance these models. The Excel-based budget impact models used for the course will be provided to participants in advance of the presentation. This course is designed for those who are interested in learning the basics of budget impact analysis and desire exposure to these analyses in Excel. Participants who wish to gain hands-on experience must have Microsoft Excel for Windows installed on their computers.
PREREQUISITE: Working knowledge of Microsoft Excel is required.
Speakers
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Stephanie Earnshaw, PhD
RTI Health Solutions, Pittsboro, NC, United States
Stephanie Earnshaw has performed health outcomes and health services research for 30+ years. Her research focuses on applying decision-analysis techniques (e.g., decision trees, Markov processes, simulation) to industry-related issues and health care problems. In addition to developing budget-impact and cost-effectiveness models to support health technologies for the pharmaceutical, biotechnology, and diagnostic and medical device industry, she has developed innovative mathematical models using these methods to determine pricing strategy, predict clinical outcomes, allocate resources, and cost care pathways particularly in support of medical diagnostics.
Dr Earnshaw received her PhD in Industrial Engineering at North Carolina State University and is a member of ISPOR and the Institute for Operations Research and Management Sciences. She has presented her work at professional conferences and has published in several peer-reviewed journals. She has presented workshops and various courses on decision-analytic modeling techniques for pharmaceutical companies and organizations such as ISPOR, the Academy of Managed Care Pharmacy (AMCP), and the Centers for Disease Control and Prevention (CDC). Dr. Earnshaw has served on the ISPOR Board of Directors and as Chair of the Audit Committee and Educational Council. She has held an Adjunct Faculty appointment at the University of North Carolina’s Eshelman School of Pharmacy, Division of Pharmaceutical Outcomes and Policy, is honored as a Distinguished Alumni in Industrial and Systems Engineering at North Carolina State University and is one of the lead authors of “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
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Anita Brogan, MSc, PhD
RTI Health Solutions, San Diego, CA, United States
Anita Brogan has 20+ years of experience in using analytical techniques to assess and present the health and economic impact of emerging pharmaceutical and biotechnology interventions. She has led the development of user-friendly and transparent cost-effectiveness, cost-utility, and cost-consequence models; budget-impact models; resource allocation models; and infectious disease transmission models programmed in Microsoft Excel and other platforms. She has experience with numerous types of modeling procedures, including Markov and other stochastic models, simulation, regression, linear and nonlinear programming, and various types of sensitivity analysis. Dr. Brogan has developed models and analyses in the areas of HIV, hepatitis C, RSV, Ebola, influenza, cystic fibrosis, bone health, mental health, women’s health, oncology, osteoporosis, chronic pain, age-related macular degeneration, hospital-acquired infection, financial portfolio optimization, and vehicle routing.
Dr. Brogan holds a PhD in Operations Research from the University of North Carolina. She has presented workshops and short courses on decision-analytic modeling techniques in a variety of venues, including meetings of ISPOR and the Academy of Managed Care Pharmacy (AMCP). Dr. Brogan has held an adjunct professorship at the Fermanian School of Business at Point Loma Nazarene University. She is also a co-author of the book “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
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Thor-Henrik Brodtkorb
United States
Thor-Henrik Brodtkorb, PhD, is executive director in Health Economics at RTI Health Solutions (RTI-HS). He holds a PhD in Health Technology Assessment from the University of Linköping and has been with RTI-HS for 12 years. He has been teaching courses in decision-analytic modeling at Linköping University as well as presented workshops and short courses on decision- analytic modeling techniques for organizations such as Pharma Industry Sweden, Swedish Agency for Health Technology Assessment and Assessment of Social Services (SBU), and ISPOR.
At RTI-HS, Dr. Brodtkorb leads the development of cost-effectiveness, cost- utility, cost-consequence, and budget-impact models for pharmaceutical, device, and diagnostic technologies. These models have been used to support reimbursement decisions in more than 15 European countries including NICE in UK, SMC in Scotland, TLV in Sweden, and NOMA in Norway. He has developed models and analyses in the areas of oncology, alcohol dependence, major depressive disorder, Alzheimer’s disease, dermatology, multiple sclerosis, cardiology, orthopedics, and asthma. His research has been presented at professional conferences and published in peer-reviewed journals. He is also a co-author of the book “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
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Ashley Davis, PhD
RTI Health Solutions, Research Triangle Park, NC, United States
Ashley Davis is a senior director within the Health Economics division at RTI Health Solutions (RTI-HS). She holds a PhD in Industrial Engineering and Management Sciences from Northwestern University and has been with RTI- HS for 10 years. She has presented short courses on budget impact modeling techniques in a variety of venues, including meetings of The Professional Society for Health Economics and Outcomes Research (ISPOR).
In her role at RTI-HS, Dr. Davis uses analytical methodologies to evaluate the clinical and economic value of upcoming pharmaceutical products and changes to healthcare policies. She has developed user-friendly and transparent cost- effectiveness, cost-utility, and cost-consequence models; budget-impact models; resource allocation models; and infectious disease transmission models programmed in Microsoft Excel and other platforms. She has experience with numerous types of modeling techniques, including Markov and other stochastic models, simulation, statistical analysis, linear and nonlinear programming, robust optimization, and various types of sensitivity analysis. Dr. Davis has developed models and analyses in the areas of HIV, hepatitis C, cystic fibrosis, herpes zoster, influenza, pneumococcal disease, respiratory syncytial virus, severe asthma, chronic obstructive pulmonary disease, eosinophilic esophagitis, spinal surgery, cytomegalovirus, and organ transplantation. Her research has been presented at various professional conferences and published in peer- reviewed journals.
Prompt Engineering for HEOR: Practical Skills and Use Cases for HEOR Professionals
Session Type: Short Course
Topics: Methodological & Statistical Research
Level: Introductory
Separate registration required.
Prompt engineering—the art and science of designing effective inputs for generative AI—has become a critical skill for health economists and outcomes researchers. Mastery of prompt engineering can significantly enhance productivity, accuracy, and innovation in HEOR, unlocking the full potential of large language models (LLMs) and other AI tools. This course delivers a comprehensive introduction to prompt engineering, tailored specifically for the HEOR context. Participants will gain hands-on experience with practical prompt strategies for systematic literature reviews (SLRs), economic modeling, real-world evidence generation, and more. The curriculum also addresses current best practices and common pitfalls, equipping attendees to confidently apply prompt engineering in regulated and high-stakes settings.
PREREQUISITE: Basic knowledge of systematic literature reviews and economic modeling will be helpful. No prior knowledge or use of AI is required.
Speakers
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Rachael Fleurence, MSc, PhD
Value Analytics Labs, Boston, MA, United States
Rachael L. Fleurence, PhD, MSc, is the head of Evidence and AI Solutions, at Value Analytics Lab, a life sciences consultancy. A health economist by training, she previously served as senior advisor to Dr. Francis Collins at the National Institutes of Health, where she led a national initiative to eliminate Hepatitis C in the US. She also served as an advisor to the National Institute of Biomedical Imaging and Bioengineering (NIBIB), focusing on artificial intelligence and machine learning. Previously, Dr. Fleurence was a senior health policy advisor in the Biden-Harris White House and Senior Advisor to the NIH Director. She played a key role in the federal COVID-19 response, leading the “Say Yes! COVID Test” program and serving on White House pandemic policy groups. Prior to her federal service, she led the National Evaluation System for health Technology Coordinating Center (NESTcc) and PCORnet at PCORI and spent several years in the private sector in health economics and outcomes research (HEOR) consulting. Dr. Fleurence has received multiple NIH Director’s Awards, the HHS Secretary’s Award for Distinguished Service, and the National Champion for Global Hepatitis Elimination award. She co-led the ISPOR Task Force on EHR Data for Health Technology Assessment, serves on the ISPOR Working Group on Generative AI, and is an associate editor for Value in Health. She also sits on the boards of CTTI (the Clinical Trials Transformation Initiative) and ICN (the ImproveCareNow network). She holds degrees from Cambridge University, ESSEC Business School (Paris), and the University of York (UK).
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Jag Chhatwal, PhD
Harvard Medical School / Massachusetts General Hospital, Boston, MA, United States
Jag Chhatwal, PhD, is the director of the Institute for Technology Assessment at Massachusetts General Hospital and an associate professor at Harvard Medical School. He also serves as core faculty at the Center for Health Decision Science, Harvard T.H. Chan School of Public Health. Dr. Chhatwal has co-authored more than 125 original research articles and editorials in leading peer-reviewed journals. His research has informed health policy decisions at prominent organizations including the White House, the World Health Organization, and the CDC, and has been featured in major media outlets such as CNN, Forbes, National Public Radio, The New York Times, and The Wall Street Journal. Dr. Chhatwal serves as an associate editor of Value in Health and as guest editor for its special issue on artificial intelligence. He is also a member of the ISPOR Generative AI Working Group.
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Dalia Dawoud, BSc, MSc, PhD
Cairo University, Cairo, Egypt
Dalia Dawoud, PhD, is the director of PEHTA Consulting Ltd. She is also a professor 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, IMI EHDEN 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.
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Turgay Ayer, PhD
Value Analytics Labs, Boston, MA, United States
Turgay Ayer, PhD, holds the Virginia C. and Joseph C. Mello Chair and serves as the research director for Healthcare Analytics and Business Intelligence at the Center for Health & Humanitarian Systems at Georgia Tech. He is also the chief technology officer at Value Analytics Labs. Dr. Ayer holds a courtesy appointment at Emory Medical School where he teaches Big Data Analytics courses and serves as a Senior Scientist at the Centers for Disease Control and Prevention (CDC). Dr. Ayer’s research focuses on health economics modeling (HEOR), real-world evidence, data science, machine learning, econometric modeling, and healthcare analytics. He has published over 80 peer-reviewed journal papers and more than 300 conference abstracts, with his work featured in top-tier business, engineering, medical, and health policy journals. His research has attracted substantial attention from major media outlets, including The Wall Street Journal, The Washington Post, US News, and NPR. A recognized expert in HEOR, Dr. Ayer has been at the forefront of applying generative AI to navigate healthcare systems and support better decision-making. He has contributed significantly to the development of advanced models for predicting healthcare outcomes and designing innovative cost-effectiveness analysis frameworks. Under his leadership, Value Analytics Labs has focused on the development of cutting-edge technologies, including ValueGen.AI, to enhance healthcare analytics and improve the efficiency of healthcare decision-making processes.
Using LLMs to Simplify Real-World Evidence Research
Session Type: Short Course
Topics: Real World Data & Information Systems
Level: Intermediate
Separate registration required.
This course introduces how large language models (LLMs) can enhance real-world evidence (RWE) research, highlighting practical applications and best practices. Participants will explore how LLMs streamline tasks such as literature reviews, clinical documentation analysis, patient phenotyping, and data interpretation through real-world examples.
Significant focus is given to interactive exercises that provide hands-on experience with LLM-assisted workflows, including synthesizing literature, interpreting complex data, and efficiently reviewing reports. Ethical and regulatory considerations critical to the responsible use of AI in healthcare research are also addressed, emphasizing transparency, bias mitigation, and compliance.
The course concludes by considering future AI trends, such as multimodal integration and evolving regulatory environments, preparing participants to effectively integrate these advancements into their research strategies. Participants who wish to gain hands-on experience must bring their laptops with Microsoft Excel for Windows installed.
PREREQUISITE: This course assumes that participants are familiar with the standing challenges and opportunities for RWE in research.
Speakers
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Dan Drozd, MSc, MD
PicnicHealth, San Francisco, CA, United States
Daniel R. Drozd, MD, MSc, is the chief medical officer at PicnicHealth. Prior to joining PicnicHealth he was on faculty at the University of Washington in the Department of Allergy & Infectious Diseases where he led research into the use of electronic health record data to power observational research and enhance the understanding of the chronic burden of HIV infection. At PicnicHealth he oversees scientific collaborations with PicnicHealth’s industry and academic partners and works extensively with both the product and commercial teams. Prior to attending medical school, he worked for numerous technology start-ups as an engineer and at the University of Washington in the Clinical Informatics Research Group where he led the development of a large EHR data integration platform used to power HIV real-world research.
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Troy Astorino
PicnicHealth, San Francisco, CA, United States
Troy Astorino is the co-founder and chief technology officer at PicnicHealth. With deep AI expertise from MIT and SpaceX, Troy co-founded PicnicHealth to make it easier to capture patient-centered data and improve healthcare. As the CTO, he leads product development, overseeing engineering, product, and design. Troy's expertise lies in AI-driven observational research and data quality. He has developed a system that continuously audits and improves data accuracy, ensuring that research insights generated by PicnicHealth address research questions with the highest level of reliability.
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Kieran Mace, PhD
Plinth Analytics, Chicago, IL, United States
Kieran Mace is a passionate data scientist and entrepreneur who combines deep technical expertise with exceptional leadership abilities. As the co-founder and CEO of Plinth Analytics, he leads a boutique real-world data science consultancy dedicated to improving lives through analytics.
His career spans critical roles at the intersection of healthcare, data science, and technology, where he has consistently delivered innovative solutions to complex problems. From developing novel pan-tumor prognostic models at Genentech to building clinical data transformation pipelines at PicnicHealth, Kieran has demonstrated an exceptional ability to translate complex data into actionable insights.
With a PhD in Computational Biology/Biomedical Informatics from UCSF and extensive experience in clinical RWD, genomics, systems biology, and software engineering, Kieran brings both technical depth and strategic vision to every project. His research has been published in prestigious journals including Cell, PLoS One, and ACS Synthetic Biology, with his work cited over 1,400 times in scientific literature.
Kieran excels at building and leading high-performing teams, believing deeply that "there is nothing better than achieving something together that we could not achieve alone." His collaborative approach and ability to communicate complex concepts to diverse stakeholders make him particularly effective at bridging the gap between technical implementation and business impact.
Valuation of Innovative Drugs
Session Type: Short Course
Topics: Health Policy & Regulatory
Level: Intermediate
Separate registration required.
The value of medical innovation depends on the perspective. Registration authorities (EMA, FDA) mainly consider the clinical value of the medical innovation, whereas national health authorities take a broader perspective by including clinical, economic criteria, and potential other criteria like equity and social values. Value-based pricing is the most widely accepted approach in the pricing and reimbursement process in Europe, which varies from the narrow concept based on the incremental cost-effectiveness ratio (ICER) threshold to broader approaches. Value-based pricing determines the maximum price from the national payer perspective. This price should exceed the minimum price for the investor acting in the international financial market, which is based on economic valuation theory. Finally, there are other stakeholders, eg, patients, physicians’ healthcare insurers, employers, with their specific assessment of the value of medical innovation varying from, respectively, quality of life, effectiveness, budget impact, and costs of lost productivity. This course offers an overview of the perspectives of the relevant stakeholders and their respective data requirements for value assessment of innovative drugs. The course will then describe in-depth description of the various value-based pricing methods, eg, ICER, multicriteria decision analysis (MCDA), comparative effectiveness research (CER), and relative effectiveness (RE). We include examples of orphan drugs and ATMPs which are most striking to illustrate the concepts, but we also include value assessment for more traditional innovative drugs in broad indications. Familiarity with health economic evaluation is desirable, but the course assumes little or no familiarity with economic valuation theory.
Speakers
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Lou Garrison, PhD
The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, Seattle, WA, United States
Lou Garrison, PhD, is professor emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
For the first 13 years of his career, Dr. Garrison worked in non-profit health policy at Battelle and then the Project HOPE Center for Health Affairs, where he was the Director from 1989-1992. Following this, he worked as an economist in the pharmaceutical industry for 12 years. From 2002-2004, he was vice president and head of Health Economics & Strategic Pricing in Roche Pharmaceuticals, based in Basel, Switzerland.
Dr. Garrison received a BA in Economics from Indiana University, and a PhD in Economics from Stanford University. He has more than 150 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies.
Dr. Garrison was elected as ISPOR President for July 2016-June 2017, following other leadership roles since 2005. He recently co-chaired the ISPOR Special Task Force on US Value Frameworks. He was selected in 2017 by PharmaVOICE as being among “100 of the Most Inspiring People” in the industry. He recently received the PhRMA Foundation and Personalized Medicine Coalition 2018 Value Assessment Challenge First-Prize Award as lead author on a paper on “A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine.”
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Marlene Gyldmark, PhD
Basel, Switzerland
In her current role, Marlene leads the EU HTA organizational readiness at BeiGene in the Global Value, Access, and Pricing group.
Marlene’s prior life science industry experience includes vice president global head Access Evidence at Idorsia, Switzerland; global head Health Policy and Outcomes Research at Roche Diabetes Care, Switzerland; global head Modelling, Outcomes Research, Statistics and Epidemiology, Roche Pharma, Switzerland; health economist at Pfizer Denmark, and Pricing and Economic Analyst at Novo Nordisk, Denmark. Before joining the life science industry, she worked as a researcher in the Danish Hospital Institute, Denmark and at University of Copenhagen, Denmark.
Since 1996 Marlene has been an external lecturer at University of Copenhagen, Denmark.
Other work experiences include serving as a member of the board of directors (2000-2012) at EASE Consulting, Denmark and member of the board of the Institute of Neurodiversity (2021- 2025). She has been a long-term member of ISPOR and served as member of the Board of Directors between 2021-2024. Currently, Marlene also acts as a Copenhagen Goodwill ambassador.
She holds a master’s in economics and policy sciences from University of Copenhagen, Denmark, and a MPhil in health economics from York University, UK.
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Afschin Gandjour
Frankfurt am Main, Germany
Afschin Gandjour is a medical doctor, health economist, and philosopher. His research focuses on cost-effectiveness analysis, decision modeling, and value-based pricing of pharmaceuticals.
Gandjour received an MD from Hannover Medical School in Germany, an MBA from Duke University, a PhD in health economics from the University of Cologne in Germany, and an MA in philosophy from the University of Düsseldorf in Germany. He held faculty positions at the University of Cologne Medical School, Baylor College of Medicine, and Louisiana State University Pennington. In terms of research productivity, he ranks among the top professors in business administration in Germany (#20 in 2022 based on Wirtschaftswoche magazine).
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Mark J Nuijten, MBA, PhD, MD
A2M - Minerva, bergen op zoom, 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.
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
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Uwe Siebert, MPH, MSc, ScD, MD
UMIT TIROL - University for Health Sciences and Technology; Harvard Chan School of Public Health, Hall in Tirol, Austria
Uwe Siebert, MD, MPH, MSc, ScD, is a professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL-University for Health Sciences and Technology in Austria and director of the Division for HTA in the ONCOTYROL–Center for Personalized Cancer Medicine in Austria. He is also adjunct professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health and Affiliated Researcher in the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston.
After medical school, he worked for several years as a physician in international public health projects in West Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in decision sciences at the Harvard School of Public Health.
His research interests include applying real-world evidence-based quantitative, causal and translational methods from public health, epidemiology, artificial intelligence, comparative effectiveness research, health services and outcomes research, economic evaluation, modeling, and health data a d decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, clinical guideline development, public health policies and patient guidance. His research focuses on cancer, infectious disease, cardiovascular disease, neurological disorders, and others.
He has been leading projects/work packages in several EU FP7, H2020 and Horizon Europe projects (eg, ELSA-GEN, BiomarCaRE, MedTecHTA, DEXHELPP, EUthyroid, FORECEE, MDS-RIGHT, RECETAS, CORE-MD, EUREGIO-EFH, CIDS, OnCoVID, 4D PICTURE, CATALYSE). He teaches HTA, health economics, modeling, epidemiology, causal inference and target trial emulation, and data and decision science for academia, industry, and health authorities in Europe, North and South America, and Asia. He directs the Continuing Education Program on Health Technology Assessment & Decision Sciences (htads.org).
He has served as member of the ISPOR Directors Board and as president of the Society for Medical Decision Making (SMDM). He is a leadership member of the ISPOR Personalized/Precision Medicine SIG, a member of the Latin America Consortium Advisory Committee of ISPOR, and co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force. He is a member of the Oncology Advisory Council and the National Committee for Cancer Screening of the Austrian Federal Ministry of Health.
He has authored more than 400 publications (> 30,000 citations, H index > 80), and is editor of the European Journal of Epidemiology. Further information Internet: http://htads.org, umit-tirol.at/dph, hsph.harvard.edu/uwe-siebert, Twitter: @UweSiebert9, LinkedIn: uwe-siebert9.
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Felicitas Kuehne, PhD
Hall in Tirol, Austria
Felicitas Kühne is a senior scientist and deputy coordinator at the Institute of Public Health, Medical Decision Making and Health Technology Assessment at the Department of Public Health, Medical Decision Making and Health Technology Assessment, UMIT TIROL - University for Health Sciences and Health Technology in Hall in Tirol, Austria. She is co-leading the Program on Causal Inference in Science and is the director of the HTADS course “Causal Inference for Assessing Effectiveness in Real-World Data and Clinical Trials: A Practical Hands-on Workshop”. Further, Felicitas Kühne is an outcomes research manager at Pfizer Pharma GmbH, Germany.
Felicitas Kühne holds a doctoral degree in Health Technology Assessment from UMIT TIROL as well as a master’s degree in health policy and management from the Harvard TH Chan School of Public Health, Boston, USA. She received her state approval as physiotherapist from the Georg-August-University of Göttingen, Germany and participated in Health-Economic Program of the University of Cologne, Germany. Before she started her position at UMIT TIROL in 2011, she worked as a consultant for pharmaceutical companies and healthcare providers, conducting several decision-analytic, real-world evidence, epidemiologic, and costing studies in a variety of disease areas.
Her research interests include evaluating public health interventions by applying advanced evidence-based quantitative methods from epidemiology, comparative effectiveness research, health services and outcomes research, economic evaluation, machine learning, and decision sciences. Her current substantive research focuses on identifying synergies of causal inference and decision science. The main disease areas are cardiovascular diseases, cancer, and infectious diseases including HIV/AIDS, hepatitis C, and pneumococcal disease. She teaches courses in decision-analytic modeling, economic evaluation, analysis of big data, and advanced causal epidemiologic methods at several universities and for industry in Europe and the USA.
She has authored several publications including textbook chapters and scientific articles and disseminated her finding at several conferences. She received financial support for her studies and research from several national and international organizations.
Felicitas Kühne is an active advisory board member for a NIMHD K01 award as well as a member of the editorial board of the journal of Medical Decision Making (MDM) and the journal of MDM Policy & Practice (MDM P&P). She is a member of the Working Group "Medical Decision Making" of the German Society for Medical Informatics, Biometry and Epidemiology (GMDS). She is also a member of the Professional Society for Health Economics and Outcomes Research (ISPOR), and the Society for Medical Decision Making (SMDM).
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Nicholas Latimer, MSc, PhD
SCHARR, University of Sheffield, Sheffield, 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 2019, Nick began a Senior Research Fellowship funded by Yorkshire Cancer Research in which he is investigating 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.
https://www.sheffield.ac.uk/scharr/sections/heds/staff/latimer_n
15:30 - 16:30
First-Time Attendee Orientation
Session Type: General Meeting
Mon 10 Nov
7:00 - 17:00
Registration Hours
Session Type: General Meeting
7:30 - 8:30
Morning Coffee Service
Session Type: General Meeting
8:30 - 9:45
Plenary Session: Welcome Remarks and Keynote—Celebrating 30 Years of ISPOR
Session Type: Plenary
Available On-Demand: Digital Conference Pass
Join us as we kick off ISPOR Europe 2025. This year, as ISPOR celebrates its 30th Anniversary, the conference theme is, "Powering Value and Access through Patient-Centered Collaboration." Hear from ISPOR leadership and key stakeholders as they reflect on 3 decades of advancing health economics and outcomes research (HEOR) excellence and explore the power of global collaboration in shaping the future of healthcare decision making. This session will also include an inspiring Keynote Address from Dave Snowden, CEO of The Cynefin Company.
Immediately following the opening presentations, the scientific plenary panel will take the stage.
Speakers
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Rob Abbott
ISPOR, Lawrenceville, NJ, United States
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Dave Snowden, PhD
The Cynefin Company, Brecon, Powys, Wales, United Kingdom
Plenary Session: Patient Engagement in Healthcare Investments—A Promise or a Practice?
Session Type: Plenary
Topics: Patient-Centered Research, Health Policy & Regulatory, Economic Evaluation
Level: Intermediate
Available On-Demand: Digital Conference Pass
Patient engagement is a key topic in many industry settings and interactions, but has it led to tangible results? How can we enhance the impact of patient engagement? In this panel, key stakeholders, including payers, investors, industry, regulators, and patients, will have an open discussion about how patient engagement can be improved to lead to tangible results. A key aim of this discussion is to set actionable deliverables for the next 5 years to improve access, relevance and efficiency based on patient input and involvement.
Moderator
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Joep Muijrers
Gilde Healthcare Partners, Utrecht, Netherlands
Joep joined Gilde Healthcare in 2021. He focuses on investing in late-stage opportunities including publicly traded companies that deliver better care at lower cost. He is actively engaged in multi-stakeholder interaction and is responsible for coordinating Gilde’s Impact Council.
Before joining Gilde, he served as CFO and Chief of Portfolio Strategy at Boston-based biotech company PureTech Health (PRTC), developing highly differentiated medicines for devastating diseases. Prior to PureTech, Joep worked as Partner and Portfolio Manager at LSP for 12 years and gained experience in life sciences investment banking in his role as Director of Corporate Finance and Capital Markets at Fortis Bank.
Joep holds a PhD in Molecular Biology from the European Molecular Biology Laboratory (EMBL) in Heidelberg, Germany, and a Master’s degree in Biochemistry from the University of Nijmegen, the Netherlands.
Speakers
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Carole Longson, PhD
Manchester, United Kingdom
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Steffen Thirstrup, PhD, MD
Amsterdam, Netherlands
Steffen Thirstrup is a medical doctor and board-certified specialist in clinical pharmacology and therapeutics. He holds a PhD in pharmacology and has a long background in clinical internal medicine with special emphasis on adult respiratory medicine. Additionally, Dr. Thirstrup was appointed adjunct professor in pharmacotherapy at the Faculty of Health Sciences, University of Copenhagen, in 2012.From 2004-09 Steffen Thirstrup worked at Danish Medicines Agency first as the Danish member of CHMP at the European Medicines Agency (EMA) for five years including 10 months as joint CHMP- and CAT-member, followed by a short period as head of Danish Institute for Rational Pharmacotherapy dealing with HTA and best practice guidelines for primary care. In 2011 Prof. Thirstrup rejoined the licensing division at the Danish Medicines Agency acting as Head of Division for Medicines Assessment and Clinical Trials. During this period Prof Thirstrup co-chaired the European Commission’s working group on market access for biosimilars medicinal products and acted as key scientific contact for the managing entity of the IMI beneficiaries for the PROTECT collaboration (Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium).
In March 2013, Prof Thirstrup joined the pharmaceutical consultancy company NDA Group AB as a full-time medical advisor on NDA’s regulatory advisory board. In April 2014 Prof Thirstrup was appointed as director for the Regulatory Advisory Board at NDA Regulatory Services Ltd.
Since June 2022 Prof Thirstrup has been the Chief Medical Officer at the European Medicines Agency, Amsterdam, The Netherlands
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David H.-U. Haerry
Positivrat, Zürich, Switzerland
David Haerry has been writing about scientific research and from medical conferences in lay language since 1996.
He has been involved in projects to educate healthcare professionals and doctors for over 30 years and became Secretary General of the Swiss Academic Foundation on Education in Infectious Diseases SAFE-ID in 2015. He has been involved with the European Medicines Agency EMA and Swissmedic for many years, and he played a key role in the EUPATI IMI project. He is a member of the Executive Committee of PFMD and a consultant on patient-centred clinical research and medicine throughout Europe.
He is co-author of numerous publications in the fields of infectious diseases, doctor-patient communication, research ethics and pharmacovigilance.
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Luc Truyen, MD, PhD
Argenx, Boston, MA, United States
9:30 - 19:00
Exhibit Hall Open
Session Type: General Meeting
9:45 - 10:15
Morning Break: Coffee and Connect (Exhibit Hall)
Session Type: General Meeting
10:15 - 11:15
Measuring What Matters: Practical Pathways for Personalized Endpoints and Patient-Centric Approaches in HTA Evaluation
Session Type: Workshop
Topics: Patient-Centered Research, Health Technology Assessment, Clinical Outcomes
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
Purpose: This workshop aims to discuss how personalized endpoints and patient-centric approaches can be effectively designed, implemented, and integrated into clinical trials, and explore their incorporation into health technology assessments (HTA). The session will offer practical tools, concrete examples, and expert insight to support broader adoption of patient-centered evidence in value assessment.
Description: As healthcare systems move toward more patient-centric approaches, it is crucial to reflect individual patient experiences and treatment goals in evidence generation. Traditional clinical endpoints may fail to capture what matters most to specific patient subgroups, particularly in rare diseases, neurodevelopmental and chronic conditions, due to heterogeneity in disease burden and treatment response. HTA bodies face the task of balancing the need for robust, population-level evidence with the growing demand for patient relevance in value assessment. While the importance of incorporating the patient voice is widely recognized, practical implementation of personalized endpoints in clinical trials and HTA evaluations remains limited.
This workshop will discuss both conceptual and practical aspects of personalized endpoints and patient-centric approaches. The panel will share qualitative and quantitative examples, including the Goal Attainment Scale (GAS), which captures individualized treatment goals, quantifies efficacy, and reflects patient priorities. Other examples will include adapting generic measures to specific diseases and patient populations. We will also discuss how such endpoints could be integrated into HTA frameworks.
•Danny Yeh: Moderator, sharing an overview of personalized endpoints in value assessment.
•Gunes Sevinc: Introduction to GAS as a structured, patient-centered endpoint used in drug development and beyond.
•Hannah Staunton: Industry perspective on opportunities and challenges in implementation.
•Dalia Dawoud: HTA methodologist stance on the use of personalized endpoints in current and future value frameworks.
The session will include polling and live Q&A to engage the audience and advance patient-relevant HTA.
Moderator
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Danny Yeh, PhD
Aesara, Burlingame, CA, United States
Speakers
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Gunes Sevinc, BSc, MSc, PhD
Ardea Outcomes, Vancouver, BC, Canada
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Hannah Staunton, MSc
Roche Products Ltd, Welwyn Garden City, United Kingdom
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Dalia Dawoud, BSc, MSc, PhD
Cairo University, Cairo, Egypt
Dalia Dawoud, PhD, is the director of PEHTA Consulting Ltd. She is also a professor 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, IMI EHDEN 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.
Patient Preference Evidence in Health Technology Assessment: What Do We Really Know?
Session Type: Issue Panel
Topics: Health Technology Assessment, Patient-Centered Research, Economic Evaluation
Track: Patient-Centered Evidence
Level: Introductory
Available On-Demand: Digital Conference Pass
ISSUE: This session will evaluate what works in patient preference evidence (PPE) submissions for health technology assessment (HTA) and identify where current approaches are lacking.
Overview: Despite continued proposals for incorporating PPE in HTA demonstrable impact remains limited. A 2020 ISPOR SIG report documented minimal use of health preference research in European reimbursement decisions. The session will start with an introduction to existing proposals for PPE in HTA (Hauber – 8 minutes). The session will then set out practical opportunities for PPE in HTA submissions focusing on key areas including evidence on the benefit-risk or benefit-burden trade offs that matter to patients; highlighting the value patients place on process of therapy; and settings in which existing methods fail to capture meaningful benefits that alternative preference methods might address (Meads – 14 minutes). The current evidence landscape on the use of PPE in HTA will be examined, contrasting successful PPE applications with problematic implementations. Notable successes will be discussed including the 2017 NICE daratumumab submission for multiple myeloma and PBAC consumer surplus demonstrations. These will be contrasted with applications that were poorly received and the reasons for this (Watson – 14 minutes). The session will then consider future directions for potential evidence integration, drawing on a series of collaborative workshops with HTA body representatives and their evaluations of hypothetical PPE case studies to identify practical pathways for meaningful implementation (Germeni – 14 minutes). The session will conclude with an interactive discussion featuring audience participation on potential barriers to developing fit-for-purpose PPE studies and exploring questions around different use cases where PPE might genuinely enhance HTA decision-making. This collaborative approach will help identify actionable solutions for bridging the gap between PPE theory and practice (10 minutes)
Moderator
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Brett Hauber, PhD
Pfizer, New York, NY, United States
Speakers
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Verity Watson, BA, MS, PhD
RTI Health Solutions, Manchester, United Kingdom
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David Meads
University of Leeds, Leeds, United Kingdom
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Evi Germeni, PhD
University of Glasgow, Glasgow, United Kingdom
Integrating Lifecycle Modeling and Public Perspectives into Evidence Frameworks
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session examines how lifecycle modeling and public perspectives can enhance evidence for health decision-making. Presentations cover life-cycle economic modeling of a tumor-agnostic precision oncology therapy with conditional authorization, public views on modifiers of cost-effectiveness thresholds, and applying a total system value framework to quantify the burden of metabolic liver disease in the US, and assessing the lifecycle value of highly specialized technologies and advanced therapy medicinal products in England.
Assessing the Full Life-Cycle Value of Highly Specialized Technologies and Advanced Therapy Medicinal Products in England
OBJECTIVES: Highly specialised technologies (HST) and advanced therapy medicinal products (ATMPs) are gaining market prominence. However, current value assessments often overlook their full lifecycle value. This study evaluate the lifecycle value of HST and ATMPs approved by the National Institute for Health Care and Excellence (NICE)—across both patent-protected and post-patent phases.
METHODS: We reviewed NICE technology appraisals of HST and ATMPs—including gene therapies—published between January 2021 and June 2024. Key data included product characteristics, eligible populations, quality-adjusted life years (QALYs), incremental cost-effectiveness ratios (ICERs), health system costs/savings and opportunity costs (£15,000/QALY threshold). We used data from the literature to inform expected post-patent market dynamics and to address gaps/redactions in NICE appraisal reporting. Costs and QALYs across the product lifecycle were simulated using a multi-cohort modelling approach.
RESULTS: Of 340 appraisals, 13 HSTs and 6 gene therapies met inclusion criteria. Median QALY gains (6.9) exceeded historical averages (2000-2020) for non-ATMPs (0.49). The median ICER (£100,000/QALY) was higher than that for non-ATMPs (£28,555). Despite small eligible populations, HSTs/ATMPs yield lifecycle value comparable to non-ATMPs via substantial QALY gains or cost savings. The median share of value accruing to manufacturers was 150% while median net population health effects of approval was a loss of 8,374 QALYs. Shares of value accruing to manufacturers varied widely across products reflecting differences in cost-effectiveness thresholds applied, health system savings and expected post-patent market dynamics. Findings were sensitive to the time until off-patent availability and to the cost of producing the HST/ATMP.
CONCLUSIONS: NICE approved HSTs/ATMPs often displaced more health than they delivered over the product lifecycle. This underscores critical trade-offs between incentivising the development of, and access to, these products and system-wide opportunity costs. This research also emphasises the importance ensuring that health systems can access affordable versions of these technologies at patent expiry.
The Burden of Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD) and Metabolic Dysfunction-Associated Steatohepatitis (MASH) in the US: A Total System Value Evidence Framework
OBJECTIVES: Metabolic dysfunction-associated steatotic liver disease (MASLD), where lipids accumulate in the liver, may progress to metabolic dysfunction-associated steatohepatitis (MASH), a disease stage characterised by liver inflammation and progressive damage. We aimed to develop an evidence framework to characterise MASLD/MASH from a total system perspective in the US, encompassing clinical, societal and economic consequences.
METHODS: Ovid/PubMed searches were conducted to identify evidence across the MASLD/MASH spectrum, including epidemiology, pathophysiology, cardiometabolic comorbidities, outcomes (clinical, societal and economic) and healthcare resource use. Evidence was categorised and connected to visually represent how MASLD/MASH impacts the wider system. A qualitative assessment of the strength of the evidence was conducted to identify uncertainty and evidence gaps. The framework was validated by clinical experts.
RESULTS: In our novel framework, disease prevalence, staging and progression (measured invasively [biopsy-determined ‘F-stage’] and non-invasively [e.g. FIB-4 scoring]), risk factors (e.g., metabolic syndrome, age, sex), hepatic (hepatocellular carcinoma, liver transplant, cardiovascular events) and non-hepatic outcomes, were connected to form a system. Clinical features were linked to outcomes to enable value-mapping and the illustration of disease burden- from the perspectives of multiple stakeholders: patients (quality of life, mortality), society (indirect costs, including informal care, absenteeism/presenteeism), healthcare providers (direct costs, resource use), and the environment. The quality of evidence was heterogenous, ranging from meta-analyses to single, small studies. Underdiagnosis of MASH creates uncertainty in terms of total prevalence, disease stage prevalence and burden. High-quality evidence related to risk of some clinical outcomes (e.g., transplant, complications), but limited evidence was available to correlate progression with other outcomes (e.g., quality of life, productivity).
CONCLUSIONS: A qualitative framework of disease elements and outcomes can be constructed, notwithstanding data limitations. The MASLD/MASH qualitative evidence framework is a useful tool to visualise the complex connections between disease features and outcomes, identify value drivers, support economic modelling and to inform evidence generation needs.
Potential Modifiers of Cost-Effectiveness Thresholds: Public Perspectives on Prioritization in Healthcare Spending
OBJECTIVES: Governments use modifiers - specific characteristics that adjust the cost-effectiveness threshold - to prioritize healthcare spending. This study explores public views on such modifiers to assess which could justify potential higher spending on new treatments in Europe.
METHODS: A literature review identified currently used and potential modifiers, including ISPOR value flower elements, resulting in 14 possible modifiers: purpose of treatment, degree of effectiveness, uncertainty, budget impact, age, severity of disease, rarity of disease, unmet need, equity, additional healthcare benefits, staff workload, environmental impact, value of hope, and value of knowing. Three focus groups were held in the Netherlands to explore public perspectives on national healthcare spending and to prioritize the 14 possible modifiers. Additionally, a questionnaire was conducted in five European countries (Bulgaria, Netherlands, Norway, Slovakia, Spain), where participants (n=100 per country) prioritized these modifiers to assess and compare perspectives across Europe.
RESULTS: Focus group participants (N=23) found trade-offs in healthcare spending complex, involving ethical, financial, and fairness considerations. Participants ranked degree of effectiveness, purpose of treatment, and severity of disease highest, while unmet need, value of hope, and environmental impact were ranked lowest. The questionnaire (N=529) confirmed severity of disease, degree of effectiveness, and purpose of treatment as the top three factors. While severity of disease was consistently ranked highest across European countries, environmental impact was consistently ranked lowest. The greatest cross-country variation was observed for additional healthcare benefits and unmet need.
CONCLUSIONS: Our study shows that public views on the prioritization of new treatments are largely consistent with existing policies, particularly related to the use of disease severity as a modifier. Although environmental impact is becoming more prominent in policy discussions, the public seems to consider it less relevant in healthcare spending decisions. Further research is planned to examine public preferences regarding the independent impact of each modifier on healthcare spending.
Life-Cycle Economic Modeling to Determine the Evolving Value of a Tumouragnostic Precision Oncology Therapy With Conditional Marketing Authorization
OBJECTIVES: Precision oncology challenges health technology assessment (HTA) with small benefiting populations and non-randomized comparators. Life-cycle assessment integrating real-world evidence (RWE) can address uncertainty in evolving value for reimbursement decisions. We determined the value of a tumour-agnostic targeted therapy for NTRK fusion-positive advanced cancer patients compared to second-line therapies using RWE-informed, patient-centric modeling. We compared cost-effectiveness against the initial Canadian HTA evaluation.
METHODS: Our individual-level semi-Markov model consisted of six health states, reflecting variations in tumour-specific care. We simulated 5,000 patients representing seven tumour groups using monthly transitions. We derived clinical inputs by combining regulatory data for entrectinib (STARTRK/ALKA-372-001) with cross-jurisdictional real-world data from the United States (Flatiron Health) and British Columbia, Canada. We derived utility weights using Bayesian hierarchal modeling combining weights from regulatory EQ-5D-3L responses and estimates from EQ-5D instruments from systematic reviews. We estimated costs (CAD2024, public payer perspective) and quality-adjusted life years (QALYs) over 10 years (1.5% discounting), and calculated incremental net monetary benefit (INMB) at $100,000/QALYs. To compare life-cycle value, we replicated the initial HTA evaluation, conducted cross-model validation, and used a real-world clinical case mix for patient-centric modeling.
RESULTS: With patient-centric modeling, incremental costs and QALYs were $30,810 (95%CI: -$8,130, $70,857) and 0.01 (-0.36, 0.38). INMB was -$29,997 (95%CI: -$65,944, $9,629), and 41.1% simulations had lower QALYs and higher costs. Heterogeneity across tumours was substantial: lung had the largest QALY gains (0.18), and colorectal the highest cost-effectiveness probability (31.4%). Relative to the initial evaluation, patient-centric modeling resulted in 55.0% lower incremental costs and 92.1% lower QALY gains, with a higher probability of being cost-effective (10.2% v. 0.0%).
CONCLUSIONS: Life-cycle evaluation with RWE and patient-centric modelling reshapes our understanding of value for therapies targeting rare genetic alterations. Case mix is a critical determinant in assessing the impact of precision oncology interventions on health system resources.
Unlocking the Untapped Potential of Comprehensive Genomic Profiling: Improving Patient Access and Outcomes
Session Type: Issue Panel
Topics: Health Policy & Regulatory
Level: Introductory
Available On-Demand: Digital Conference Pass
ISSUE: Addressing the untapped potential of Comprehensive Genomic Profiling (CGP) in tumour characterisation and treatment decision-making for cancer patients. The European Coalition for Access to Comprehensive Genomic Profiling (ECGP) recently published a report on this topic. CGP is revolutionizing tumour diagnostics by consolidating multiple tests into a single, comprehensive next-generation sequencing (NGS) assay, uncovering a wide range of genomic alterations/signatures that drive cancer growth. Despite ESMO’s recommendations, access to broad molecular diagnostics, including CGP, remains inconsistent and limited across Europe, highlighting a critical gap in equitable cancer care. This session will discuss how best to facilitate patient access to CGP, via case studies that show potential impact; leading to the development of clear recommendations following consensus-building discussion.
OVERVIEW: Opening and introduction panellists by Matias Olsen (5 minutes). Overview provided by Prof. Lawler for (10 minutes). Moderated discussion by panellist (30 minutes) on the preconditions for patient access. Discussion is brought to life with a series of case studies (at European or national level). These case studies underscore the commitment of policymakers to expand patient access to diagnostic technologies. However, there is room for improvement. Focus of this discussion will be on key enablers regarding: 1) Funding, assessment and reimbursement; and 2) Data and infrastructure needs to implement requirements. During this discussion, the panellists will provide insights from diverse perspectives; representing patients, HCPs and the health economic dividend. Q&A with the audience and key actions by Antonella Cardone and Prof Lawler (15 minutes).
Moderator
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Matias Olsen, MSc
European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), Brussels, Belgium
Speakers
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Antonella M Cardone
Cancer Patients Europe, Brussels, Belgium
Antonella Cardone is the CEO of Cancer Patients Europe, a pan-European all-cancer patient association. She is a Patient Advocacy Expert and Advisor to the Board of Pancreatic Cancer Europe (PCE), uniting physicians, patients, politicians, journalists, academia, and industry, and she is a member of Inspire2Live.
Antonella led the European Cancer Patient Coalition (ECPC) and served as Executive Director of the Fit for Work Global Alliance.
With 25 years of nonprofit experience in health, social, and employment sectors, she also directed the Global Smoke-free Partnership of the American Cancer Society.
Antonella holds Master's degrees in Science and Business Administration. She has been on the Boards of All.Can and Pancreatic Cancer Europe, where she was vice-chair.
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Mark Lawler, PhD
Queen's University,, Belfast, United Kingdom
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Claudio Jommi, MSc
Universita del Piemonte Orientale, Milano, Italy
Managed Entry Agreements: Can CEE Countries Mutualize Lessons Learned?
Session Type: Issue Panel
Topics: Health Technology Assessment, Health Policy & Regulatory
Level: Introductory
Available On-Demand: Digital Conference Pass
Issue:
Managed Entry Agreements (MEAs) in CEE are often negotiated individually, leading to inconsistent access and varying financial outcomes. This panel will debate whether MEAs can be mutualized across CEE to offer harmonized frameworks and shared learnings, or if national heterogeneity renders mutualization impractical.
Overview:
CEE countries face common challenges: limited negotiating power with manufacturers, scarce data on real-world outcomes, and budget constraints. While a collective MEA approach could streamline negotiations, increase transparency, and improve patient access, it may falter due to divergent regulatory standards, reimbursement timelines, and economic capacities.
This 90-minute session (15 minutes per speaker, 30-minute moderated debate, 15-minute audience Q&A) is structured to provide comprehensive timing:
15 min – Rok Hren, PhD (University of Ljubljana) advocates regionally aligned risk-sharing frameworks to optimize payer collaboration.
15 min – Guenka Petrova, PhD (Medical University of Sofia) highlights benefits of data-sharing for biologics but cautions about achieving consensus on value metrics.
15 min – Anna Kowalczuk, PhD, MSc (Polish HTA Agency) emphasizes legal and procedural disparities that may impede mutualization.
30 min – Moderated debate led by Izabela Pieniazek (Certara), exploring governance models, data infrastructure, and financial implications.
15 min – Audience Q&A and interactive discussion on next steps.
Attendees—including HTA bodies, payers, patient advocates, and industry representatives—will explore whether a phased or sub-regional approach could overcome obstacles. By session end, participants should have a clearer view of actionable steps toward or against MEA mutualization in CEE.
Moderator
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Izabela Pieniazek, MSc
Certara, KRAKOW, Poland
Speakers
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Rok Hren, MSc, PhD
Hren & Partner d.o.o., Ljubljana, Slovenia
Rok Hren has more than 15 years of commercial experience in pharmaceutical industry and more than 12 years on a board level in both (1) line management, which has included full P&L responsibility for operations in Slovenia and Romania, and (2) leadership regional functions in Central and Eastern Europe. He regularly presents on the topic of pharmaceutical economics and policies at conferences in Europe and is well experienced in healthcare media business.
He received his PhD in Physiology and Biophysics from Dalhousie University, Canada and MSc in International Health Policy (Health Economics) with Distinction from London School of Economics and Political Science, UK while he was a post-Doctoral Fellow at Nora Eccles Harrison Cardiovascular Research and Training Institute, University of Utah Medical School, USA. He is also an assistant professor at the University of Ljubljana and past president of the ISPOR Slovenia Regional Chapter. In total, his publications gathered 300/394 citations (excluding self-citations) in WoS/Scopus (as of September 10, 2016).
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Anna Kowalczuk, PhD, MSc
Agency for Health Technology Assessment and Tariff System, Warszawa, Poland
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Guenka I Petrova, ScD, PharmD, PhD
Medical University of Sofia, Faculty of Pharmacy, Sofia, Bulgaria
Lines of Therapy in Oncology Real World Data: Navigating Complexity for Robust Evidence
Session Type: Other Breakout Session
Topics: Real World Data & Information Systems, Methodological & Statistical Research, Epidemiology & Public Health
Level: Introductory
Available On-Demand: Digital Conference Pass
Purpose: The objectives of this session are to introduce the complexity and importance of lines of therapy (LOT) when using real world data (RWD), to summarize insights from ongoing international collaborations reviewing and developing frameworks for oncology LOT, and to engage members in a collective discussion on best practices frameworks and best practices for distinguishing LOT in RWD.
Description: Each systemic anticancer treatment line has implications for disease progression, safety, cost, and prognosis. Given the clinical significance, accurately characterizing LOTs is essential for research evaluating treatment use, outcomes, and costs. However, RWD sources, especially large population-based databases, often lack specific or complete information, requiring researchers to make assumptions. Further complicating this, there is no standardized approach among oncologists for defining LOTs in clinical practice, especially across health systems and oncology societies. This symposium brings together RWD and oncology experts to discuss ongoing projects and frameworks aimed at improving LOT the enumeration in cancer RWD.
This session will begin with an introduction to LOT concepts, accompanied by a digital handout summarizing session topics and links to online content. The clinical relevance, complexity, and diversity of LOT and RWD in oncology research will be reviewed (15 minutes, Bates). This will be followed by an overview of LOT frameworks developed by the European Society for Medical Oncology (15 minutes, Koopman). A regulatory perspective on using RWD to support real world evidence and the importance of high-quality LOT data will then be shared (15 minutes, Morales). The session will conclude with an audience-panel discussion (15 minutes, Mahmoudpour). Throughout, live polls will engage attendees on their experiences with LOT, perceptions of proposed frameworks, suggestions for next steps, and questions for the panel.
Moderator
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Hamid Mahmoudpour, PharmD, PhD
Merck Healthcare KGaA, Darmstadt, Germany
Speakers
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Benjamin Bates
Rutgers, New Brunswick, NJ, United States
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Miriam Koopman, MD, PhD
Utrecht University, Utrecht, Netherlands
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Denise Umuhire
European Medicines Agency, Amsterdam, Netherlands
Value Assessment from a Whole Health Perspective: Opportunities and Challenges
Session Type: Other Breakout Session
Topics: Methodological & Statistical Research
Level: Advanced
Available On-Demand: Digital Conference Pass
ISPOR’s Strategic Plan 2030 includes an objective to expand the definition of value to consider whole health. Given increasing government stakeholder interest in the value of healthcare investments, burgeoning real world data sources, and advancement in scientific understanding about how social, environmental, and economic factors contribute to the health of individuals and society, “whole health economics” is a critical HEOR frontier. This session will define whole health, articulate its relevance to HEOR, and showcase case examples from major ongoing projects on the topic. Eberechukwu Onukwugha will outline the whole health component of ISPOR’s strategic plan and will moderate the session; Laura Pizzi will provide an example from the Caring for Older Persons in their Environment (COPE) US dementia study where expanded caregiver evidence was used to successfully achieve Medicaid payment for a community-delivered supportive intervention; Andy Briggs will present research aimed at paving the way for transdisciplinary health economic and environmental economic methods; Malina Mueller will present her work on using input-output models to capture the societal values of medicines; and Oriana Ciani will share recommendations from the HI-PRIX project, funded through Horizon Europe, on broadening the scope of innovative payment and pricing schemes for health technologies. The group will have a moderated discussion on the main opportunities and challenges for HEOR’s whole health frontier, exploring issues related to study design (study perspective, time horizon), data sources (e.g., administrative, survey), measures (e.g., effect, cost) and partnerships (e.g., patients, researchers, payers).
Attendees will develop an understanding of whole health, assess its relevance for HEOR, and identify opportunities and challenges in applying broader value measures across case studies and policy contexts.
The session will include interactive elements such as live audience polling and a Q&A discussion to engage participants and gather diverse perspectives on advancing whole health in HEOR.
Moderator
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Eberechukwu Onukwugha, MSc, PhD
University of Maryland, School of Pharmacy, Baltimore, MD, United States
Eberechukwu Onukwugha, PhD is a Professor in the Department of Practice, Sciences, and Health Outcomes Research and Executive Director of Pharmaceutical Research Computing at the University of Maryland School of Pharmacy. She received a Doctor of Philosophy in economics (concentration: econometrics) from Virginia Polytechnic Institute and State University (Virginia Tech). Dr. Onukwugha completed a two-year postdoctoral fellowship in pharmacoeconomics and health outcomes research at the University of Maryland School of Pharmacy. She was a recipient of the PhRMA Foundation’s Post-Doctoral Fellowship in health economics and outcomes research. Dr. Onukwugha’s research interests are in cost analysis, health disparities, and medical decision-making by individuals and institutions. She has approximately 20 years of experience conducting health economics and outcomes research using administrative medical and pharmacy claims, hospital discharge, and prospectively-collected data. Dr. Onukwugha has authored or co-authored over 140 peer-reviewed articles in health economics and outcomes research. She is an Editorial Board member for PharmacoEconomics and an Associate Editor for Ethnicity & Disease. Dr. Onukwugha serves as President, ISPOR Board of Directors, 2024-2025, and serves on the Maryland Prescription Drug Affordability Board.
Speakers
Integrating AI into Market Access Workflows
Session Type: Issue Panel
Topics: Health Technology Assessment, Medical Technologies, Real World Data & Information Systems
Level: Intermediate
Available On-Demand: Digital Conference Pass
Description
AI is no longer a future concept—it is becoming embedded in the daily operations of pharmaceutical Market Access teams. This panel brings together experts from across the industry to explore how AI is starting to reshape core workflows, from developing value dossiers and pricing strategies to managing stakeholder insights and health economic evidence.
Rather than focusing on specific tools or products, this discussion aims to provide a general, cross-functional view on the current and near-future use of AI in Market Access. We will explore practical examples, organizational hurdles, regulatory considerations, and how teams are adapting skill sets and mindsets. The panel will share lessons learned, best practices, and predictions for how AI will continue to shape the field.
The session is ideal for professionals working in Market Access, Health Economics, and related functions who want to understand how AI is being used beyond theory—and how to prepare their teams and organizations for this transformation.
Summary
A cross-industry panel exploring how AI is being integrated into the daily workflows of Market Access teams. The session covers real-world applications, organizational change, and the evolving role of AI in shaping access strategies and value communication.
Learning objectives
Understand current AI use cases in Market Access teams
Identify key opportunities and challenges in adopting AI for daily workflows
Learn how to prepare teams and skill sets for an AI-driven future
Gain insight into the regulatory and ethical considerations around AI in access-related functions
Moderator
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Jessica Federer, Yale IRB
Advisory Board Member, New York, NY, United States
Speakers
Artificial Intelligence and Advanced Analytics in HTA
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session explores the transformative role of artificial intelligence and advanced analytical methods in optimizing health technology assessment. Topics include the application of generative AI for simulating complex HTA discussions, leveraging AI for efficient data extraction from HTA reports, comparing machine learning and traditional statistical approaches for predicting health outcomes like EQ-5D scores, and refining health economic models to capture critical clinical nuances. The session highlights how cutting-edge computational techniques can enhance the efficiency, accuracy, and depth of health economic and HTA research.
Simulating Sponsor-HTA Conversations: A Multi-Agent GenAI Framework for Reimbursement Processes
OBJECTIVES: Health technology assessments (HTA) agencies like National Institute for Health and Care Excellence (NICE) evaluate sponsor-submitted evidence to inform reimbursement decisions, with a key focus on identifying evidence gaps through clarification questions. This study aimed to develop and validate a multi-agent Generative artificial intelligence (GenAI) framework that simulate sponsor HTA conversations by generating clarification questions using sponsor-submitted data.
METHODS: A role-based, multi-agent large language models (LLMs) framework was developed to simulate sponsor-HTA interactions, featuring specialized AI agents representing HTA secretariat (lead), clinical, economic, PRO and patient/public involvement reviewers. The HTA secretariat coordinated the process and compiled clarification questions based on HTA guidelines. Using sponsor-submitted data for hepatocellular carcinoma (HCC), the tool generated questions across clinical, economic, and textual domains. Subject matter experts (SME) validated the outputs for relevance, accuracy, traceability, and alignment with HTA expectations.
RESULTS: Two NICE HTA submissions in first-line advanced/metastatic HCC were selected for sponsor-HTA simulation. The multi-agent GenAI system successfully simulated interactions by generating structured clarification questions based on sponsor-submitted evidence. A total of 104 questions were generated including 29 clinical, 62 economic, and 13 textual. The majority of questions focused on data-related gaps, while a smaller proportion sought clarification on methodological aspects. The AI-generated output was validated by a SME, demonstrating 80-85% agreement with actual clarification questions previously raised by NICE in the technology appraisals. The remaining 15-20% of questions, although not identical to historical NICE examples, were still relevant and evidence-based, reflecting potential gaps that would warrant further clarification within the HTA process.
CONCLUSIONS: The multi-agent GenAI framework provides a scalable, regulatory-aligned method for simulating NICE HTA reviews by generating structured clarification questions. It enhances sponsor preparedness and may streamline assessments, with future updates to include budget impact and real-world evidence.
Can AI-Assisted Data Extraction From HTA Reports Improve Comparative HTA Research: A Case Study on NICE Assessment Reports
OBJECTIVES: Data used in comparative health technology assessment (HTA) research is often manually extracted from HTA reports. This hinders the scope, reproducibility, updateability, and credibility of this research. This study examines the application of automated data extraction methods to extract research-relevant attributes from publicly available HTA reports. This study analyzes and compares the performance of various text-mining techniques, aiming to demonstrate the relevance and opportunities of these extraction methods.
METHODS: To analyze the performance of different text-mining approaches, 14 research-relevant attributes were extracted from National Institute for Health and Care Excellence (NICE) HTA reports using two natural language processing techniques (rule-based (NLP-R), classification models (NLP-CM)) and a generative AI technique (large language model-based (LLM), Claude 3 Opus). To analyze the performance of the extraction methods, accuracy and other method-specific measures were calculated and compared. Additionally, data extracted using the LLM-based extraction was analyzed for policy insights.
RESULTS: Extraction accuracies depended on the extraction method and attribute. Overall, the LLM-based approach performed best (88-98% accuracy for 12/14 attributes). Extraction of the outcome of the relative effectiveness assessment (REA) and the comparator was most challenging and had the lowest accuracies (~70% for the LLM-based approach). NLP-based methods required more development work and were unable to extract attributes at the medicine-indication combination level; however, they were independent of commercial software and free from reproducibility issues, which were the most significant limitations of the LLM-based approach. Graphs created using the LLM-extracted data give important policy insights that are updateable and reproducible and would have been difficult to obtain with manual data extraction.
CONCLUSIONS: Automatic data extraction for research-relevant attributes from HTA reports is possible and can provide important insights for comparative HTA research. Room for improvement remains, and future research should focus on expanding the system to different HTA organizations and refining the LLM-based approach.
Predicting EQ-5D Index Scores: A Comparison Study of Machine Learning and Statistical Methods on Health Survey for England Data
OBJECTIVES: The EQ-5D is widely used to measure health-related quality of life (HRQoL), especially in economic evaluations. When EQ-5D data are partially or entirely missing, index scores may be estimated using alternative data sources either directly by predicting index values or indirectly by predicting dimension responses and computing the index. This study compared statistical and machine learning (ML) approaches to identify the most accurate and efficient model for predicting EQ-5D-3L scores using a large, representative dataset from England. A secondary aim was to identify key predictors to inform research and policy.
METHODS: Data were drawn from seven waves of the Health Survey for England (2003-2006, 2008, 2011, and 2012) for individuals aged 16+. Six models were evaluated: two statistical methods (Ordinary Least Squares and logistic regression), two tree-based algorithms (XGBoost classification and regression), and two neural networks (NNs). Both direct (predicting index scores) and indirect (predicting dimension responses) approaches were tested. A holdout validation strategy trained models on 2003-2008 data, tuned on 2011, and tested on 2012. Performance was assessed using Mean Absolute Error (MAE) and Health Severity Group (HSG) accuracy. Feature importance was assessed using permutation analysis and SHAP.
RESULTS: NNs outperformed all models, with the best direct NN (one hidden layer) achieving the lowest MAE (0.088) and highest HSG accuracy (73.34%). XGBoost models were also competitive (MAE as low as 0.092; HSG accuracy up to 72.01%) and computationally efficient. Statistical models showed similar MAE but lower HSG accuracy, especially for pain and anxiety. Indirect prediction improved most models, though NNs performed well in both approaches. Key predictors: employment, chronic conditions, self-rated health, and recent illness highlighted the dominance of health-related over demographic factors.
CONCLUSIONS: NNs best captured complex EQ-5D-3L patterns and show promise when direct HRQoL data are unavailable. Findings highlight key health-related predictors and inform targeted interventions and policy.
Zoom in: Uncovering Clinical Nuance in Simplified Health Economic Model Structure Through Artificial Intelligence
OBJECTIVES: For health economic (HE) models to achieve clinical face validity, it is essential that each health state accurately reflects meaningful stages of disease to capture the full spectrum of data and natural history. In practice, however, many models are simplified for tractability and data availability. While simplification can aid technical implementation, it may limit meaningful engagement with clinicians and obscure key assumptions during health technology assessment (HTA). This study explores whether artificial intelligence (AI), in a hybrid intelligence framework, can help “re-expand” such models - starting from simplified structures and reconstructing the underlying clinical story - thereby clarifying what simplification omits and enabling better communication with stakeholders.
METHODS: We applied an AI-supported workflow incorporating large language model (LLM) prompt engineering, graph-based retrieval-augmented generation (GraphRAG), and structured reasoning. Starting from common simplified HE model templates, the framework deconstructed model elements, identified potential gaps in clinical representation, and proposed enriched structures. Expert reviewers iteratively assessed AI-suggested refinements to ensure alignment with clinical and economic reasoning. The goal was to expand model fidelity and visually and conceptually clarify the clinical meaning behind each health state.
RESULTS: The AI-guided expansions revealed several overlooked aspects of disease logic, such as treatment sequencing, complication pathways, and quality-of-life dynamics. These additions helped improve communication with clinical stakeholders and created more transparent structures for internal discussion and external submission. Importantly, the process also highlighted what was deliberately excluded in the original simplified models - making simplification itself a more conscious and explainable decision.
CONCLUSIONS: This work demonstrates the strategic value of hybrid intelligence in refining HE models - not just for technical accuracy, but for more effective communication, faster alignment with clinical experts, and better preparedness for payer engagement. It offers a scalable approach for organizations seeking to bridge the gap between operational simplicity and clinical credibility.
External Control Studies - What Does it Take to Get Real?
Session Type: Issue Panel
Topics: Study Approaches, Health Technology Assessment, Methodological & Statistical Research
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue: Although randomised controlled trials (RCTs) are the gold standard for assessing treatment efficacy and safety, certain conditions make it difficult to conduct adequately powered trials. In these cases, single-arm trials (SATs) and non-randomised studies are often pursued. However, their lack of a control group limits causal inference. External control (EC) groups are increasingly used to generate comparative evidence using real-world data (RWD) when RCTs are impractical or unethical. However, it is unclear to what extent these EC studies are fit for purpose.
Overview: The GetReal Institute developed an External Comparator Best Practices Framework to help decision-makers determine when to consider an EC study, what requirements need to be met and how to design and execute such a study. This panel will discuss whether the framework can support regulatory decision-making and health technology assessment (HTA) by providing structured guidance on data selection, study design, statistical analysis, and regulatory engagement. Each panelist will offer a critical perspective on the framework over the course of 10 minutes each, considering what is required to ensure broad adoption across stakeholder groups. The session will close with a Q&A for 15-20 mins. People from Industry, Regulatory, HTA and RWD research backgrounds would benefit from attending this session.
Moderator
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Mariam Bibi, BSc, PhD
GetReal Institute, Utrecht, Netherlands
Speakers
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Anke van Engen
IQVIA, Amsterdam, Netherlands
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Wim Goettsch, MSc, PhD
Utrecht University; Zorginstituut Nederland, Utrecht; Diemen, Netherlands
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Seamus Kent, MSc, PhD
Erasmus University Rotterdam, Amsterdam, Netherlands
10:30 - 13:30
Poster Session 1
Session Type: General Meeting
Poster Tours will be from 11:30–12:15 | Presenters will be with their posters from 12:30–13:30
Poster Session 1
Session Type: Research Posters
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
11:30 - 12:15
Oncology Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 1, Poster will be hung from 10:30 - 13:30.
HEOR Impact Cases Poster Tour
Session Type: HEOR Impact Cases
This tour will take place during Poster Session 1, Poster will be hung from 10:30 - 13:30.
Evaluating the cost of telemonitoring in cardiology from a service provider’s perspective: a case study from Northern Italy...
Problem Statement: The adoption of digital health solutions, such as telemonitoring in cardiology, holds significant promise for enhancing patient care and system efficiency. However, implementation is highly context-dependent and demands considerable adaptation, including dedicated human and financial resources. Transitioning from traditional in-person monitoring to a fully remote telemonitoring model presents operational and organisational challenges that differ across healthcare settings. Despite growing interest, real-world evidence on the costs associated with such transitions remains limited. Generating this evidence is essential to support informed investment decisions and reduce uncertainty for service providers and policymakers considering the shift to digitally enabled models of care.
Description: This study presents the results of field-based research conducted at a hospital in Rovereto (Northern Italy). Organisational and service-level data were collected through semi-structured interviews and analysis of archival documents, in collaboration with the clinical and administrative teams, to inform the estimation of an appropriate tariff level for the telemonitoring service in cardiology.
Lessons Learned: The study shows that the incremental cost of telemonitoring in cardiology is relatively marginal—around €48 per patient per year (assuming a five-year horizon and 1,000 patients). However, key challenges remain, including the apportioning of indirect costs, defining the scope of evaluation, and capturing the organisational context in which the service is embedded. To improve generalisability, it is essential to describe how the provider operates within the wider health system. Rapid technological change and intangible costs complicate estimation, making results time-sensitive. Scenario analysis and forecasting are vital to assess feasibility and guide future digital health investments.
Stakeholder Perspective: A service provider (local hospital) perspective was taken to evaluate the cost of implementing the digital health service.
Implementing a Value-Based Contract for Hereditary Angioedema Prophylaxis in Colombia: A Real-World Case Study...
Problem Statement: In resource-constrained systems like Colombia’s, uncertainty around newer therapies can delay access and strain payer budgets. Regarding Hereditary Angioedema (HAE), a rare and serious condition, long-term prophylaxis (LTP) with innovative treatments was difficult to implement consistently across payers. Health-maintenance organizations (HMOs) required a payment model that could support access while aligning with real-world treatment patterns and financial responsibility.
Description: A value-based agreement (VBA) was established between Takeda and two HMOs to enable LTP access for HAE patients. It incorporated reconciliations supported by a pre-structured tool tracking vial-use, dosing-frequency, and cohort dynamics. Lanadelumab trials showed 44.4% of patients being attack-free after 6 months of treatment. With clinical stability, dose-interval extension—from every two weeks (Q2W) to longer intervals (>Q2W)—becomes viable.; this was reflected in the VBA. By April 2025, of 178 LTP patients, 71 (39.9%) received treatment within the VBA. Physician-led dose-interval extension based on clinical stability was achieved in 63.4% (n=45) of patients receiving >Q2W dosing, while 36.6% (n=26) continued Q2W dosing. Efficiency was measured comparing actual to expected monthly-drug use: of 142 expected doses (April-2025), 97 were administered: representing a reduction of -35.5% (HMO#1), -16.9% (HMO#2), and a system-wide efficiency of -31.7%. Monthly validation reports ensured real-time monitoring and transparency.
Lessons Learned: This case illustrates that real-world VBAs can align clinical and economic objectives. Addressing rare disease therapies requires shared accountability between the pharmaceutical industry and healthcare systems. Collaboration ensures equitable access to innovative treatments despite limited resources. VBAs provide sustainable financing approaches, emphasizing transparency, resource prioritization, and impactful interventions. Innovation must balance therapeutic efficacy and economic sustainability, prioritizing patients’ access to treatments that improve quality of life.
Stakeholder Perspective: This case reflects a joint payer-industry perspective demonstrating how structured real-world data, co-managed by HMOs and a pharmaceutical partner, can guide treatment strategies improving access in complex, high-cost conditions.
Challenges in modelling the long-term cost-effectiveness of pharmacotherapies for managing overweight and obesity from the perspective of the National Institute for Health and Care Excellence
Problem Statement: 16% of adults live with obesity globally, including 6.5 million in the UK. Obesity is estimated to reduce life expectancy by 3-10 years. Randomized clinical trials demonstrated that obesity pharmacotherapies statistically significantly reduced the body mass index (BMI) of adults with obesity over 68-72 weeks compared to diet and exercise interventions alone, but economic modelling was required to understand the lifetime cost-effectiveness. Stakeholders needed to understand the impact of introducing new technologies for a large population into a healthcare system, and whether they could be delivered within existing infrastructure. This included defining the comparator and lifestyle support adjunct to pharmacological treatment and the associated costs, consideration of duration of treatment (lifetime, or any proposed stopping rules), estimating long-term BMI trajectories, and predicting long-term costs and effectiveness of reducing obesity-related complications. Due to the population size and potential for lifetime treatment, the decision risk and budget impact were large, and so consideration of uncertainty was particularly important.
Description: In economic models, risk equations linked patient characteristics and surrogate markers (principally BMI) to clinical events including diabetes and cardiovascular conditions. Scenario analyses were used to consider rate of weight regain after stopping pharmacotherapy, and relative differences in BMI between people remaining on pharmacotherapy (for longer than data were available) and people only receiving diet and exercise support. The residual impact of having previously lived with obesity was considered a key uncertainty. Scenario analyses were also used to address uncertainty regarding delivery and costs of the comparator and support provided alongside pharmacotherapy, and whether these could be delivered within primary care or required specialist weight management services. A phased introduction was required for one technology due to the considerable effort required to set up delivery of an obesity treatment in primary care, with a plan to re-evaluate this funding variation.
Lessons Learned: The appraisals highlighted the challenges of predicting lifetime cost-effectiveness from short-term clinical trial data, the role of economic models in analysing different scenarios, and the importance of real-world evidence for estimating long-term outcomes. The HEOR evidence was essential in determining that obesity pharmacotherapies are reimbursed, and highlighted evidence gaps that real world evidence can address.
Stakeholder Perspective: The perspective is that of the National Institute for Health and Care Excellence (NICE), with input from members of the Technology Appraisal Committee who evaluated the technologies, NICE technical staff who wrote the guidance, and the independent assessment group who reviewed the HEOR evidence.
Transforming Health Economics Through Patient-Centered Pathway Modelling - A Case Study in HIV Prevention...
Problem Statement: Traditional health economic models often fail to capture the complex, individualised patient journeys critical for demonstrating the full value of healthcare interventions. Globally, this limitation is faced by healthcare planners involved in chronic conditions requiring long-term management. As a case study, health planners in sub-Saharan Africa needed to evaluate the impact of scaling up HIV pre-exposure and post-exposure prophylaxis (PrEP/PEP) programmes. Traditional health economic models (e.g., cohort Markov models) were not capturing the full continuum of care or the individualised pathways of patients, making it difficult for stakeholders to see the true value and system-wide effects of expanding prevention services. A new approach was required to inform this decision-making problem with more granular and comprehensive evidence.
Description: A first-of-its-kind web-based patient pathway simulation platform that is accessible on any device—addresses this gap by providing unprecedented detail in modelling individual patients' healthcare journeys, resource utilisation, and outcomes. It employs simulation to model individual patient pathways through complete care continuums. Unlike conventional population-based approaches (e.g., Markov models), this new approach captures patient-specific characteristics, dynamic healthcare system constraints, and complex service interdependencies. We applied it to HIV prevention, modelling a 5-year scenario increasing PrEP and PEP provision from a 20%/80% split to a 50%/50% split. The model incorporated comprehensive resource utilisation data, quantifying impacts across the entire care pathway.
Lessons Learned: The decision support tool (DST) demonstrated 44 primary HIV infections averted over 5 years (versus a baseline of 7), providing clear cost-effectiveness evidence. Key lessons learnt include web-based delivery enhanced stakeholder engagement, enabling non-technical decision-makers to explore scenarios directly. Most importantly, modelling individual patient pathways rather than population averages revealed critical intervention points and more accurate cost projections. This patient-centric approach fundamentally changed how HEOR evidence supports decision-making, demonstrating that pathway-based modelling provides superior insights for healthcare policy and resource allocation.
Stakeholder Perspective: This web-based DST represents a paradigm shift in HEOR methodology, delivering value across all healthcare stakeholders. By simulating individual patient journeys, it enables patients to receive more personalised care, providers to access sophisticated economic evidence without technical barriers, payers to make informed formulary decisions with transparent cost-effectiveness data, and pharmaceutical companies to articulate value propositions with unprecedented granularity for market access. From a multi-stakeholder perspective, the platform's web-based delivery eliminates traditional modelling barriers—no specialised software, significant cost savings, and an intuitive interface—making sophisticated simulation accessible across the healthcare ecosystem. Its rapid customisation for diverse therapeutic areas supports evidence-based decision-making from clinical practice to health policy.
A Value-Based payment model for TAVI: The Next Chapter in Funding of High-Cost treatments
Problem Statement: Aortic stenosis (AS) is a common heart valve disease in Europe. Prognosis is poor if a severely stenotic valve is not replaced with a prosthetic valve. Transcatheter Aortic Valve Implantation (TAVI) is expensive, and traditional healthcare fee-for-service billing ignores the health benefits achieved, which creates pressure on funding. Prevalence of AS is increasing with age[i]. With the demographic transition toward an older population, focus on cost-efficient treatment for these patients is needed. In Finland, the share of those aged 65+ has risen from 22% to 37% since 2000 and is expected to reach 45% by 2040. The 85+ population will more than double, pushing healthcare spending (6.8% to 7.7% of GDP by 2070)[ii],[iii]. In response, Finland’s 2023 reform transferred health and social service responsibilities from municipalities to 21 Well-being Service Counties (WbSC) to improve efficiency. The reform replaced activity-based reimbursement with a fixed funding model to promote equity, efficiency, and outcomes. Finnish government strategies increasingly emphasize collaboration between healthcare providers and the life-sciences sector to address healthcare budget pressures[iv]. Heart Hospital in Tampere, Finland is a cardiac-center managing ~140,000 patient-events annually. Heart Hospital has adopted a value-based healthcare (VBHC) strategy to improve outcomes, efficiency, and sustainability through strategic partnerships that align economic and patient goals.
i https://www.escardio.org/Journals/E-Journal-of-Cardiology-Practice/Volume-18/epidemiology-of-aortic-valve-stenosis-as-and-of-aortic-valve-incompetence-aiii https://www.scoperatings.com/ratings-and-research/research/EN/172529iii https://www.bofbulletin.fi/en/2021/5/long-term-sustainability-of-the
-public-finances/?utm; https://link.springer.com/article/10.1007/s10433-022-00699-x?utmiii https://www.sitra.fi/en/publications/the-finnish-health-sector-growth-
and-competitiveness-vision-2030/#preface;iv https://www.businessfinland.fi/en/for-finnish-customers/services/programs/
health-360-program#:~:text=About%20program&text=The%20Health%20360%
20program%20supports,with%20investors%20and%20funding%20operators
Description: In 2024, Heart Hospital launched a TAVI-lifecycle payment model incorporating value-based risk-sharing with guarantee and patient-reported outcome bonus/sanction payments for WbSC. Heart Hospital announced a tender with a similar risk sharing and payment model towards the industry, allowing physicians to choose optimal products while ensuring economic stability over time. Anonymized outcome data was shared pre-tender to allow industry partners to calculate outcome probabilities and outcome-driven prices. Medtronic offered a long-term subscription-based payment model with risk sharing on relevant clinical outcomes and is currently supplying TAVI valves under this VBHC contract. Medtronic also supports PROM data collection in this partnership to track patient value.
Lessons Learned: By engaging in data-driven, risk-sharing partnerships, industry and healthcare providers can streamline workflows, ensure guideline-based treatment whilst alleviating healthcare budget pressure. The innovative TAVI lifecycle model offers a sustainable way to finance high-cost care by linking payments to treatment outcomes and was awarded the “efficacy impact of the year” award in Finland in 2024: “⋯The act is an example of a progressive building of risk sharing, it is scalable across patient groups and has national economic significance”[v].
vhttps://vaikuttavuusseura.wordpress.com/2024/12/05/vuoden-2024-
vaikuttavuusteot-on-valittu/
Stakeholder Perspective: Provider/industry perspective.
11:45 - 12:45
AI in CEE Health System Decision Making: Can Payers, Patients, and Policy Align for Smarter Access?
Session Type: Issue Panel
Topics: Health Technology Assessment, Study Approaches, Organizational Practices
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
"Issue"Central & Eastern European (CEE) payers face a paradox: single-payer systems give them rich RWD streams yet reimbursement dossiers still require long timelines for both creation and adjudication, driven by capacity limitations. "Overview"Overview provided by three expert speakers with a combined presentation time of approximately 36 minutes, followed by a 20-minute moderated discussion. The session will begin with a representative of the Polish HTA agency, who will provide an overview of the CEE HTA landscape, using Poland as a case study. This presentation will examine payer structures, highlight resource limitations, and outline major barriers to implementing AI in HTA, including data standardization challenges, regulatory uncertainty, methodological concerns such as algorithm validation and transparency, and ethical issues related to patient privacy, algorithmic bias, and informed consent.The second speaker, an HTA methodologist, will explore how AI is currently applied in literature reviews. This presentation will cover the benefits of AI integration, such as increased efficiency and quality control, while also addressing potential pitfalls including reproducibility issues, unclear regulatory frameworks, and the risk of data manipulation or falsification.The third speaker, an expert in AI and data science, will present a technical deep-dive into how AI tools can support accelerated evidence synthesis and dossier preparation, enable analysis of linked real-world data and synthesis of patient-reported outcomes, cost-effectiveness data, and enhance interpretation of complex data through traceable and transparent processes.The session will conclude with a 20-minute moderated discussion, including two live polls to assess audience perspectives on AI readiness in HTA and acceptable standards of evidence. The moderator will guide a dialogue focused on setting minimum validation criteria and exploring the potential for cross-border data-sharing.This session will be of particular value to national payers, HTA bodies, policymakers, pharmaceutical market access and HEOR teams, as well as AI technology vendors.
Moderator
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Vladimir Guzvic
Health Insurance Fund of Republic of Srpska, Banja Luka, Bosnia And Herzegovina
Speakers
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Anna Kowalczuk, PhD, MSc
Agency for Health Technology Assessment and Tariff System, Warszawa, Poland
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Maciej Grys
Certara, Cracow, Poland
Maciej Grys is a Consultant at Certara in Poland, with a Ph.D. in Cell Biology from Jagiellonian University. He has over seven years of experience in the clinical components of Health Technology Assessment, including systematic reviews, evidence synthesis, benefit-risk assessments, and outcome analyses. His expertise spans multiple therapeutic areas such as oncology, cardiology, HIV, rare diseases (orphan drugs), and reproductive medicine, with projects aligned to Polish Agency for Health Technology Assessment (AOTMiT) guidelines.
He has served as a project manager and reviewer in numerous international systematic and targeted literature reviews. He is a co-author of several peer-reviewed publications, including three original research papers on novel oncology therapies and two on medical devices in cardiology and cardiosurgery.
He played a key role in evaluating and implementing AI-powered tools for literature reviews at Certara, influencing company-wide adoption through rigorous platform testing and strategic collaboration with developers. His contributions earned him the “Spotlight 2024” award and recognition from senior leadership.
He actively promotes AI innovation across the organization—conducting trainings, establishing internal knowledge-sharing platforms, and leading cross-departmental onboarding of AI tools. His work has been presented at prominent scientific conferences, including PTFE 2024, ISPOR 2025 (Canada), and the HTA Kraków Symposium.
His ongoing collaborations with AI developers focus on advancing applications such as automated critical appraisal and results summarization, aiming to improve efficiency and maintain high-quality standards in HTA research.
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Kevin Kallmes, BS, MA, JD
Nested Knowledge, St. Paul, MN, United States
Kevin is the CEO and co-founder of Nested Knowledge, a leading software for AI-assisted systematic literature review. As a serial entrepreneur in medical research, therapies, and software, Kevin contributes to best review practices, provides training, resources, and research, and explores the frontier of responsible, human-in-the-loop AI solutions. This has driven widespread adoption of Nested Knowledge from academia to HTA bodies to life sciences companies as the single, central provider of living evidence synthesis in health economics and outcomes research.
Survival at the Margins: Navigating HTA Challenges in Modern Oncology Trials
Session Type: Forums
Topics: Economic Evaluation, Health Technology Assessment, Methodological & Statistical Research
Level: Intermediate
Available On-Demand: Digital Conference Pass
While the oncology therapeutic landscape continues to evolve to address unmet clinical needs, the increasing complexity of treatment pathways—marked by uncertainty around appropriate comparators, the need for rapid drug development, and novel mechanisms of action—has led to the use of unconventional trial designs such as basket or multicohort studies. These designs, although necessary to accelerate patient access and account for the unknowns of emerging therapies (e.g., optimal dosing, combination vs. monotherapy, treatment schedules), often lack clear or widely accepted comparators and generate limited long-term data. Such constraints can pose significant challenges for health technology assessment (HTA) bodies, particularly when trial data are immature, affected by treatment switching, or unable to demonstrate curative potential—offering instead only delayed progression, recurrence, or relapse. Together, these issues complicate survival extrapolation, a key component of economic evaluations used to inform reimbursement decisions.
This panel will explore methodological challenges and opportunities in survival extrapolation within the HTA context, focusing on three interrelated themes: (i) basket trials, (ii) treatment switching, and (iii) use of external evidence.
Hugo Pedder will open the session with an HTA example illustrating real-world challenges. Arthur Allignol will explore survival extrapolation in basket trials, focusing on small, diverse subgroups and lack of comparators, and will discuss methods for borrowing strength and addressing exchangeability. Min-Hua Jen will present a case study using external evidence—historical or real-world data—to improve extrapolation amid treatment switching, applying time-acceleration in a Bayesian framework without relying on covariate matching. Ash Bullement will share findings from a neutral comparison of external evidence-based methods, highlighting trade-offs, justification, and the importance of context in HTA submissions.
The session will conclude with a panel discussion and audience Q&A, offering practical insights for HTA stakeholders, statisticians, health economists, and regulatory affairs professionals navigating today’s oncology evidence landscape.
Moderator
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Hugo Pedder
University of Bristol, Bristol, United Kingdom
Speakers
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Arthur Allignol, PhD
Munich, Germany
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Min-Hua Jen, PhD
Eli Lilly, Uxbridge, United Kingdom
Dr Min-Hua Jen is currently Senior Director - Real-World & Access analytics at Eli Lilly, leading the International Business team on Market Access/HEOR/Medical affairs statistical support. She has extensive experience applying statistics to clinical research, epidemiology and health economics and outcomes research in academia and industry settings. She is an active member in the PSI/EFSPI HTA Special Interest Group (SIG) and the chair-elect of the ISPOR Oncology SIG. She was trained in Epidemiology and Statistics and obtained her PhD at University of Bristol. Her research interests including indirect treatment comparisons and network meta-analysis; particularly incorporate external data for time to event outcomes, surrogacy analyses, survival extrapolation, multilevel modelling and health economic modelling.
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Ash Bullement, BSc, MSc
Petauri, Nottingham, United Kingdom
The Potential Impact of AI in Healthcare: MENA Region Perspectives
Session Type: Forums
Topics: Organizational Practices, Economic Evaluation, Health Policy & Regulatory
Level: Intermediate
Available On-Demand: Digital Conference Pass
AI is revolutionizing Healthcare as we know it in many ways, it provides valuable opportunities and numerous applications which can support practitioners and patients, with time it’s impact on how we practice medicine is growing and so opportunities for implementation across different geographies. In this journey, we would like to walk you through a journey in the MENA Region where we would focus on how far AI applications in healthcare has progressed recently. We would aim to provide a perspective from three selected MENA countries. MENA is a diverse region in terms of demographics, healthcare trends and spending, and ultimately when it comes to AI impact on healthcare in MENA, we see a similar diversity. Dr. Mahmoud Elmahdawy will introduce the topic and present the speakers. Dr. Gihan Elsisi will provide insights on how AI can play a role in Egypt’s healthcare system, particularly in Egypt’s healthcare transformation towards universal healthcare coverage. Dr. Marie-Thérèse Sawaya will discuss AI implementation in Lebanon’s healthcare system, challenges and opportunities created in addition to health data governance considerations. Dr. Nadia Rashed Al Mazrouei will present the role of AI in transforming healthcare in UAE, AI based applications and opportunities for scalability in addition to regulations and legal framework.
Moderator
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Mahmoud Elmahdawy, PharmD
Cairo, Egypt
Speakers
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Gihan H Elsisi, BSc, MSc, PhD
The American University in Cairo, cairo, Egypt
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MARIE THERESE SAWAYA, PhD
BEIRUT, Lebanon
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Nadia Rashed Al Mazrouei, PhD
Sharjah, United Arab Emirates
Partnering for Progress: Advancing Patient-Centered Evidence in HEOR
Session Type: Forums
Topics: Patient-Centered Research
Level: Introductory
Available On-Demand: Digital Conference Pass
This forum is a dedicated session that brings together ISPOR’s key efforts in patient engagement- from foundational milestones to its latest initiatives – to examine how meaningful patient engagement is evolving in health economics and outcomes research (HEOR). The session offers a unique open opportunity for interested stakeholders to reflect on the continuing development of ISPOR’s Patient Engagement in HEOR Initiative, recognizing current achievements, and sharing insights on the future of the field.
As patient and public involvement moves from aspiration to expectation across global health systems, this session will spotlight how ISPOR has helped institutionalize the role of patients in HEOR research and decision-making from the establishment of regional roundtables and the Patient Council to convening the ISPOR Patient-Centered Research Summit 2024 and the inaugural ISPOR Patient-Centered Evidence track at ISPOR Europe 2025. Together, these milestones demonstrate how ISPOR has played a leading role in embedding patient perspectives into the science and methods of HEOR.
Speakers will explore emerging trends that are reshaping how we define and apply evidence—such as the integration of lived experience into value frameworks, the co-creation of research with patients as true partners, and novel methods to ensure evidence reflects what matters most to patients.
With insights from patient representatives, leaders, and researchers, the session will challenge traditional hierarchies of evidence and spotlight how shared decision-making, real-world lived experience, and public involvement are actively shaping regulatory, HTA, and reimbursement landscapes in both high- and low-resource settings.
Join us for this important conversation as we work to shape a more inclusive, equitable, and impactful future for patient engagement in HEOR.
Moderator
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Alan Balch, MS, PhD
Patient Advocate Foundation and National Patient Advocate Foundation, Hampton, VA, United States
Dr. Balch has more than 20 years of executive leadership in the non-profit sector spanning multiple advocacy areas including access and affordability, health equity, prevention and early detection, and cancer research, He became the CEO of both PAF and NPAF in 2013 and has served as a member of both Boards of Directors since 2007. From 2006-2013, he served as Vice President of the Preventive Health Partnership -- a national health promotion collaboration between the American Cancer Society, American Diabetes Association, and American Heart Association. Prior to his work with the Preventive Health Partnership, Dr. Balch was the Executive Director of Friends of Cancer Research from 2003 to 2006.
Dr. Balch currently serves or recently served on dozens of selective boards, steering committees, and councils for an array of institutions to include the National Academies of Medicine, the National Quality Forum (NQF), National Committee for Quality Assurance (NCQA), the Institute for Clinical and Economic Review (ICER), the Clinical Pathways Congress, the Council for Affordable Health Coverage (CAHC), the Innovation and Value Initiative (IVI), Core Quality Measure Collaborative (CQMC), the Foundation for the Accreditation of Cellular Therapy (FACT), the Hutchinson Institute for Cancer Outcomes Research (HICOR), the Duke-Margolis Value-Based Payment Consortium, the Specialty Pharmacy Certification Board (SPCB), and the Pharmacy Quality Alliance (PQA). Most recently, Dr. Balch was selected as the Chair of the Global Patient Council for the American Patient Representatives Roundtable for the Professional Society for Health Economics and Outcomes Research (ISPOR) after serving as the Co-Chair of the North American Patient Representatives Roundtable.
Dr. Balch also serves on the editorial board and as a contributing editor for the Journal of Clinical Pathways and on the advisory board for the Journal of Oncology Navigation and Survivorship. He is frequently invited to peer review article submissions to various publications including the Journal of Health Care for the Poor and Underserved, Journal of Clinical Oncology, American Journal of Preventive Medicine, and the American Journal of Public Health.
He earned his PhD in 2003 from the University of California Santa Cruz, his master’s degree in 1997 from the University of Texas San Antonio; and his bachelor’s degree in 1994 from Trinity University in San Antonio.
Speakers
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Maria Dutarte, MSc
European Patients’ Academy on Therapeutic Innovation (EUPATI), Utrecht, Netherlands
Maria Dutarte is the Executive Director of the European Patients’ Academy on Therapeutic Innovation (EUPATI). Maria has previously worked for the European AIDS Treatment Group (EATG) coordinating patient involvement in a number of training initiatives and scientific projects. She has also worked for different scientific and international organisations, e.g. French National Research Center for Scientific Research (CNRS), International AIDS Society (IAS), The Global Fund to Fight AIDS, Tuberculosis and Malaria and The International Foundation for Science (IFS). Her educational background is in International Education and Communications.
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Angie Botto-van Bemden, PhD
Musculoskeletal Research International, Holiday, FL, United States
12:45 - 13:15
Elevating HEOR Leadership: From Data Owner to Strategic Business Partner
Session Type: Fast Facts
Topics: Organizational Practices
Join us in this empowering and inspiring conversation about your leadership development in these unprecedented times!
In today’s rapidly evolving healthcare environment, the HEOR function is being called to move beyond its traditional role as a data supplier and become a true strategic partner in driving business outcomes.
This keynote explores the leadership shift required to enable this transformation, the risks of remaining in outdated mindsets, and the capabilities essential for success in this new paradigm, including business acumen, storytelling, and negotiation.
Through practical pathways and pharma industry best practices, we’ll discuss how HEOR leaders can move from transactional data delivery to value-driven, strategic dialogue, and position their function as a vital contributor to business and patient impact.
We will also touch upon how these necessary skills can be developed in the earlier career phases.
Let’s have an interactive discussion!
Speakers
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Karina Helene Hansen
Novartis A.G., Basel, Switzerland
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Natalia Scherff
Frankfurt, Germany
Natalia is an independent leadership development coach (ICF PCC, INSEAD), working predominantly in the Pharmaceutical Industry across the Globe.
She has 20 years of experience in corporate talent management, top executive search consulting, leadership assessment, and career development coaching.
For the last 4 years, while working for Novartis, Natalia has been talent partner and executive recruiter for top commercial roles like Country Presidents, and, functionally, for Value & Access Heads on Global & Countries level.
Natalia has two Business Master‘s Degrees obtained in Moscow and Frankfurt, and she is fluent in English, Russian, and German.
13:45 - 14:45
The Evidence Was There. So Why Didn’t It Matter?
Session Type: Issue Panel
Topics: Health Technology Assessment, Methodological & Statistical Research, Patient-Centered Research
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE:
Not all rigorous evidence is influential. Despite advances in methods like target trial emulation, quantitative bias analysis, and synthetic control arms, many high-quality real-world evidence (RWE) submissions still fail to sway decision-makers.
Why? Because methodological rigor, while essential, is not sufficient. In many cases, the underlying data are not “fit for purpose” and no amount of statistical sophistication can overcome missing clinical nuance, misclassification rooted in data capture, or unmeasured confounding beyond the reach of adjustment. Meanwhile, subtle but powerful dynamics (e.g., institutional familiarity, the perceived credibility, and the comfort of precedent) often shape how evidence is interpreted and used.
This panel explores the disconnect between scientific quality and decision impact, asking whether technical excellence is being filtered through institutional and human systems in ways that are rarely made explicit.
OVERVIEW:
This session will unpack both technical and institutional factors that affect the impact of RWE in regulatory and HTA decision-making. After a short framing by the moderator, speakers will discuss and debate the following:
• Why statistically sound approaches fail when the underlying data lack clinical validity or completeness.
• Whether there is a credibility penalty for unfamiliar methods, even when transparent and well-documented.
• If early engagement with regulators/HTAs, co-development with academic partners, or third-party validation can bridge the trust gap.
• What is needed to address the “below-the-surface” data quality issues that continue to limit the RWE enterprise.
Drawing on real case studies across Europe and North America, the panel will examine where even rigorous methods have fallen short and what lessons can be learned.
Moderator
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Luis Prieto
Novartis, Switzerland
Luis Prieto Rodriguez, PhD, MPH, MBA
Luis is Global Director of Health Data Partnerships at Novartis, based in Basel, Switzerland. Since joining the company in 2020 as Global RWE Director, he has supported assets across multiple therapeutic areas and now leads strategic partnerships to advance the use of real-world data across the Evidence Generation team in Global Medical Affairs. His work focuses on enabling data acquisition and external collaborations to support the execution of integrated evidence plans that address strategic needs across the product lifecycle—from development to clinical adoption and stakeholder engagement.
He began his career in 1990 at the Institut Hospital del Mar d’Investigacions Mèdiques in Barcelona, conducting research on patient-reported outcomes. He has held roles at the World Health Organization, Eli Lilly & Co., and the European Medicines Agency, and served as Vice President of Outcomes Research and Development at the International Consortium for Health Outcomes Measurement (ICHOM). Luis has been affiliated with the Faculty of Public Health and Policy at the London School of Hygiene and Tropical Medicine since 2007 and serves on the editorial boards of several peer-reviewed journals. He holds a PhD in Epidemiology and a Master’s in Public Health from the Universitat Autònoma de Barcelona, an MBA from ESADE Business School, and a Postgraduate Diploma in Health Economics from the University of York.
Speakers
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Mona Khalid
Dublin, Ireland
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Clark Paramore, MSPH
Biogen, Cambridge, MA, United States
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Miguel Hernán
Harvard T.H. Chan School of Public Health, Boston, MA, United States
How Could Patient-Centric HTA Evolve in the Changing Global Pricing Landscape?
Session Type: Spotlight
Topics: Health Technology Assessment, Economic Evaluation, Patient-Centered Research
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE: This panel will explore how Health Technology Assessment (HTA) will evolve over the next decade and what HTA may look like for innovative medicines entering early clinical trials today, undergoing HTA in 10 years’ time. Discussion topics include macroeconomic changes impacting the pharma business model for HTA, evolution of methods of assessment and appraisal, and opportunities for greater inclusion of the patient voice.
OVERVIEW: The complexity of HTA is growing, within the varying assessment disciplines of comparative clinical benefit, cost-effectiveness, and budget impact with increasing interest in other elements of the ISPOR value flower such as value of innovation, societal perspective, hope and equity. This complexity has fostered increasing collaboration between HTA agencies with formation of groupings such as EU HTA and HEMA, to develop more consistent and innovative methodologies.External environmental factors will also be considered such as how HTA deals with evolving cross-border collaborations, instability of global pricing landscape and the changing relationship between health authorities and HTAs.To remain fit for purpose, HTA will need to evolve while continuing to accommodate the diverse needs of patients, providers, industry and taxpayers, while accommodating a growing patient voice.An overview with be provided by Dr. Sullivan, approx. 5 minutes. He will outline the changing flow of influence between regulators and HTA bodies, and the potential impact of changes to the global pharma pricing model.Prof. Wu will offer perspectives on the evolution of HTA methods. Lynda Doward will outline how HTA can enable a more enriched and consistent embodiment of the patient voice. Nicholas Adlard will provide a manufacturer’s perspective. In conclusion, HTA must consider macroeconomic changes, while incorporating a consistent patient voice and reflecting national cultural identities and differing healthcare systems. Can HTA resolve these conflicts to offer systematic value assessment on a global level in the future?
Moderator
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Sean D Sullivan, PhD
University of Washington, Seattle, WA, United States
Sean D. Sullivan, BScPharm, MSc, PhD, is Professor and Dean, School of Pharmacy. He holds a joint appointment as Professor of Health Services in the School of Public Health. He holds adjunct appointments in the School of Medicine, the Public Health Sciences Division at the Fred Hutchinson Cancer Research Center, and at the Kaiser Permanente Washington Health Research Institute. He completed training in pharmacy at Oregon State University in 1983, obtained a master’s degree at the University of Texas in 1986 and a PhD in health economics and policy at the University of California, Berkeley in 1992. Dr. Sullivan has authored more than 400 journal articles, book chapters, task force reports and organizational and governmental publications. In many of these writings, he has assessed the evidence and applications of medical technology in relation to coverage and reimbursement decisions. His research interests include technology assessment, medical decision-making, and economic evaluation of medical technology. He is past president of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and past chair of the Academy of Managed Care Pharmacy (AMCP) Executive Committee of the Format for Formulary Submissions – the United States evidence-based guidelines for formulary decision making. Dr. Sullivan served as a past member of the Medicare Evidence Development and Coverage Advisory Committee, a past member of the Regence Blue Shield and Premera Blue Cross P/T Committee, and the Chair of the Premera Blue Cross Value Assessment Committee. He is also on the editorial boards of Value in Health, PharmacoEconomics, Journal of Medical Economics, and the European Journal of Health Economics. He was awarded the 2014 Stephen G. Avey Lifetime Achievement Award from the Academy of Managed Care Pharmacy (AMCP) and the 2015 APhA Academy of Pharmaceutical Research Sciences (APRS) Research Achievement Award.
Speakers
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Olivia Wu, MSc, PhD
University of Glasgow, Glasgow, United Kingdom
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Lynda Doward, MSc
RTI Health Solutions, Manchester, United Kingdom
Ms. Doward has over 30 years of experience conducting patient-centered outcomes research including the provision of strategic advice to pharmaceutical companies in the incorporation of the patient voice into drug development programs. Ms. Doward is an expert in the development of clinical outcome assessment (COA) strategies including the development of patient-centered clinical trial endpoints, the implementation of patient-reported and other COA outcome measures in clinical trial programs, and the inclusion of PRO and other COA value messages at key drug development hurdles. Ms. Doward has extensive experience in supporting pharmaceutical clients in their COA-related submissions to regulatory agencies in Europe and the US and advises on health-utility measurement strategies for reimbursement agencies in Europe. Ms. Doward has led the development of over 40 COA questionnaires that have been adapted and validated for use in over 60 languages worldwide.
Ms. Doward currently serves on the ISPOR COA Special Interest Group (leadership committee) and the ISPOR Patient Council (member) and was a member of the leadership committee of the completed ISPOR Good Research Practices Task Force for the measurement of health state utilities in clinical trials. Ms. Doward has acted as a consultant to the World Health Organization and has served as a Research Advisor to the UK Department of Health, and medical charities in the United Kingdom.
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Nicholas Adlard, MA, MBA, MSc
Novartis Pharma, Busserach, Switzerland
Modelling Methods and Practice
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session presents diverse topics in economic modelling: a real-world cost-effectiveness analysis of newer antidepressants in Black U.S. Medicaid beneficiaries with implications for evidence-based policy reform, methods to recover pre- and post-progression survival from published survival curves, and an evaluation of the complexity in cost-effectiveness modelling for test-and-treat strategies.
Real-World Cost-Effectiveness Analysis of Newer Antidepressants in Black Medicaid Beneficiaries: Evidence-Based Policy Reform to Address Treatment Disparities
OBJECTIVES: Johns Hopkins Medicine (JHM) identified significant treatment gaps in major depressive disorder (MDD) management among Black residents in underresourced Baltimore communities. Restrictive prior authorization policies in Maryland Medicaid limited Black patients' access to newer antidepressants with improved tolerability profiles, contributing to treatment disparities in major depressive disorder. This study evaluated the clinical and economic impact of evidence-based formulary policy changes to address these disparities.
METHODS: We conducted a retrospective cohort study using electronic health records from 8,200 Black patients with MDD (January 2019-December 2022). Propensity score matching using demographics, comorbidities, baseline depression severity, and socioeconomic indicators compared outcomes between newer antidepressants (vortioxetine, vilazodone, levomilnacipran) versus standard generic options over 24-month follow-up. Primary endpoints included medication adherence (proportion of days covered ≥80%), healthcare utilization, and total cost of care. Cost-effectiveness analysis incorporated quality-adjusted life years using EQ-5D utility weights. Model validation used bootstrap resampling with 1,000 iterations.
RESULTS: Among propensity-matched cohorts (n=4,100 each), newer antidepressants demonstrated superior medication adherence (68% vs 45%; OR 2.6, 95% CI 2.3-2.9), reduced emergency department utilization (31% decrease; IRR 0.69, 95% CI 0.62-0.77), and lower psychiatric hospitalization rates (18% decrease; IRR 0.82, 95% CI 0.71-0.95). The incremental cost-effectiveness ratio was $15,200 per quality-adjusted life year gained, with projected annual healthcare savings of $2,850 per patient. Following policy implementation, appropriate prescribing increased 340% over 18 months, generating $1.2 million in avoided acute care costs system-wide.
CONCLUSIONS: Real-world evidence demonstrating favorable cost-effectiveness of newer antidepressants enabled successful policy advocacy, resulting in modified prior authorization criteria across Maryland Medicaid managed care organizations. This approach provides a replicable framework for using HEOR evidence to address treatment disparities while achieving positive return on investment for healthcare stakeholders.
Beyond PFS and OS: Recovering Pre- and Post-Progression Survival From Survival Curves
OBJECTIVES: Progression-free survival (PFS) and overall survival (OS) are standard endpoints for assessing cancer treatment efficacy and are widely used in both clinical trials and health economic models. However, survival before and after progression—pre-progression survival (PrePS) and post-progression survival (PPS)—are rarely reported, despite their critical role in modelling disease progression and informing patients flows into subsequent treatment lines. As innovative drugs available at various disease stages may drive the assessment outcomes, precise information regarding treatment pathway is important. This study introduces a method to estimate PrePS and PPS from available PFS and OS data, enhancing model accuracy when direct estimates are unavailable.
METHODS: We developed a differential equations-based model to estimate PrePS and PPS by simulating patient transitions between health states over time. A two-step numerical optimization process was used to derive pre- and post-progression mortality rates that align model outputs with observed PFS and OS curves. To ensure identifiability, proportional hazards between PrePS and PPS were assumed. The model was validated using synthetic datasets under varied assumptions, including cases that violated proportional hazards, and further tested with real-world oncology data.
RESULTS: The method consistently generated accurate estimates across a range of PFS and OS curve shapes. In 72% of tested scenarios, the estimated curves differed from the originals by less than 10%, and in all cases, the difference remained under 20%. These results demonstrate the method’s robustness, flexibility, and applicability across diverse scenarios.
CONCLUSIONS: This approach provides a practical and reliable solution for estimating PrePS and PPS when not directly reported, enabling more accurate modelling of patient trajectories in semi-Markov models. While developed for oncology, the method is adaptable to other disease areas where similar survival modelling frameworks apply.
Cost-Effectiveness Modelling of Test and Treat Strategies: A Diagnosis of Over Complexity
OBJECTIVES: Modelling cost effectiveness (CE) of test and treat strategies is complex and challenging, requiring the incorporation of all data inputs relevant to the characteristics of the diagnostic test such as cost, biomarker prevalence and diagnostic accuracy, together with assumptions on survival estimates for true and false positive (TP and FP) and true and false negative (TN and FN) patients. This research explores if model complexity can be reduced to a more simplified structure, minimising data requirements and additional assumptions.
METHODS: We analysed the equations that estimate the probability, costs and Quality-Adjusted Life Years (QALYs) of each outcome node of a typical decision tree (DT) model that estimates the CE of a ‘biomarker test’ strategy versus a ‘no test’ strategy. Through algebraic reduction we derive a simplified yet equivalent expression for the incremental cost effectiveness ratio (ICER). In a hypothetical example we compare the ICER obtained using this approach versus one using the more complex model.
RESULTS: Based on the inherent symmetry within the model structure, the equation to calculate the ICER of a test vs no test strategy reduces to a simple mathematical expression. Only two parameters of the diagnostic test are required: test positive rate and cost. In the hypothetical example, assuming the weighted survival outcome of TP+FP patients is the same as that observed in the study on which the CE analysis is based, the ICER was estimated to be £71k/QALY using both methods.
CONCLUSIONS: The data requirements to model the CE of a diagnostic test and treat strategy can be significantly reduced and the model structure simplified, further obviating the need to make additional assumptions around survival and costs for FP, TP, FN and TN patients. This may have implications for countries that develop guidelines for methods of health economic evaluation specifically for co-dependent technologies.
Autonomous Construction of Excel-Based Health Economic Models by a Large Language Model: A Novel Agentic Method
OBJECTIVES: The use of Large Language Models (LLMs) to automate health economic modelling is an area of growing interest. The majority of research has focused on programmatic models. However, most health-economic analysis is performed in Microsoft Excel due to its familiarity among stakeholders. The objective of this study was to develop and validate a method that enables LLMs to autonomously construct well-formatted health economic models in Excel.
METHODS: An agentic method was developed to automate the construction of Markov models in Excel using an LLM. The process constructs models in segments (small regions of cells called ‘modules’). The agent maintains overall context through tools that provide: a summary of existing modules, the ability to inspect modules, and the ability to inspect named ranges. The method addresses two primary challenges in LLM-driven spreadsheet modeling: (1) translating spreadsheet content into text that an LLM can interpret, and (2) generating accurate, well-formatted spreadsheet content from LLM-produced text. Both challenges are reduced when limiting the scope of interpretation and construction to small regions of cells. To validate the method, we autonomously constructed a simple, exercise-book Markov model in Excel using claude-sonnet-3.5-v2 (without human intervention), starting from a natural language description of the input data and model structure.
RESULTS: Claude autonomously built the simple Markov model in Excel with a high degree of accuracy, and clear and consistent formatting. For example, using informative named ranges for all input values, colour-coding cells, and adding descriptive notes for calculation cells.
CONCLUSIONS: This study provides a framework for automating construction of Excel models using an LLM, which is a novel contribution. The study suggests that automation has the potential to increase consistency and efficiency in spreadsheet modelling. Further research could build upon this approach and validate accuracy and reliability on complex, real-world models.
Implementing a Program for Early Feasibility Studies in Europe: Goldmine or Fool’s Gold?
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Medical Technologies, Study Approaches
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE: The Medical Device Regulation (MDR) has raised evidence standards for regulatory approval in the EU, likely increasing the need for clinical trials to support CE marking. Traditional feasibility or pivotal trials often fail due to unforeseen issues in device design or implantation, leading to patient harm and higher development costs. Early Feasibility Studies (EFS), conducted at an early development stage, help assess initial safety and performance—especially when further non-clinical testing is limited—and support device refinement before larger trials. In the U.S., the FDA launched its EFS Program to streamline development, accelerate market access, and strengthen early-stage innovation. Whether Europe should implement a similar programme remains under discussion. This session will explore the potential value of a European EFS programme, aligned with MDR, and how it could improve regulatory and HTA processes. Key topics include the potential of EFS to enhance device safety, inform pivotal trial design, and support a life-cycle approach to evaluation.
OVERVIEW: An overview will be provided by Mr. Andrea Rappagliosi (Edwards Lifesciences, HEU-EFS private consortium coordinator) during the first 5 minutes. Each panelist will then share their perspective on the challenges of current clinical investigations in Europe and present recommendations from the HEU-EFS project in 15 minutes. In the last 10 minutes, Prof. Carlo Federici (SDA Bocconi School of Management, HEU-EFS leader) will collect questions from the audience to be discussed with panelists in a roundtable. Regulators, HTA agencies, notified bodies, patients associations, ethical and legal experts, the industry will particularly benefit from attending.
Moderator
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CARLO FEDERICI, PhD
SDA Bocconi School of Management and Bocconi University, Milan, Italy
Speakers
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Yasemin Zeisl, MSc
European Patients' Forum, Brussels, Belgium
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Tom Melvin, MB, BCh, BAO, LRCPI & SI, BCL LLM (NUI)
University of Galway, Galway, Ireland
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Marta Kerstan, MSc, EMBA
Johnson & Johnson MedTech, Zuchwil, Switzerland
Measuring the Broader Impact of Health on Quality of Life and Productivity: Advances in Patient-Reported Outcomes and Economic Evaluation
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session addresses the broader human and economic impacts of health conditions in health economics. Presentations examine expanding our understanding of the disease burden of dementia on family members and partners, variability in EuroQol Visual Analogue Scale (EQ-VAS) score across population health studies, and work productivity loss from respiratory infections in Denmark. The session concludes with priorities for advancing lost productivity measurement in economic evaluations, informed by a targeted literature review and multi-stakeholder workshop.
Work Productivity Loss in Individuals Due to Respiratory Infections: A Questionnaire Survey of Employees in Denmark
OBJECTIVES: Limited data exists on work productivity loss among employees due to respiratory infections, such as influenza, Covid-19, respiratory syncytial virus (RSV), and pneumonia. The objective of this study was to assess productivity loss (presenteeism and absenteeism) among individuals with respiratory infections in Denmark.
METHODS: We used survey data from 3,000 respondents employed in Danish private mid-size companies with 50+ employees. Data was collected from January to March 2025 via online interviews. Information about socioeconomic background, work environment, and number of sick days due to respiratory infections, defined as either partial or full sick days (absenteeism) or days at work while feeling sick (presenteeism) was included.
RESULTS: In the population, 32% of respondents were women, with a mean age of 44.5 years (range: 18-70). The majority of participants were from the Capital Region, and 31% of respondents reported having children at home. 95% were employed full time, with 61% working in organizations with 250+ employees. The survey encompassed more than 10 different industries and 7 distinct job types. On average, respondents worked from home 1.79 days per week. Notably, 30% reported being sick due to respiratory diseases during the past month, whereof 54% reported 1-3 sick days, 32% reported 4-7 sick days, and 14% reported 8+ sick days. 67% indicated they had not received any vaccinations. Among the 33% who were vaccinated, 97% were vaccinated for the flu, primarily provided by the employer, while 49% were vaccinated for Covid-19, primarily provided through the Danish national immunisation programmes. 6% were vaccinated for RSV and 6% were vaccinated for pneumonia.
CONCLUSIONS: Our research underscores that respiratory diseases during the winter months are associated with absenteeism and presenteeism leading to substantial productivity loss. More research is needed in this field to quantify the impact of this for the employees, employers, and society.
The Hidden Impact of Dementia on Family Members and Partners: A Large UK Cross-Sectional Study Using FROM-16
OBJECTIVES: A person's dementia can adversely affect the quality of life (QoL) of family members and partners. Measuring this often-neglected burden is critical to planning and providing appropriate support services. This study measures the impact of dementia on family members/partners using the Family Reported Outcome Measure (FROM-16).
METHODS: A large UK online cross-sectional study recruited family members and partners of people with dementia through Join Dementia Research and Healthwise Wales. The family members/partners completed the FROM-16 and basic demographic information.
RESULTS: 711 family members/partners (mean age=58.7 years, SD=12.5; females=81.3%) of patients (mean age=81.6, SD=9.6; females=66.9) with dementia completed the FROM-16. The FROM-16 mean total score was 17.48 (SD=6.8), meaning "a very large effect" on the QoL of family members, with females being more adversely impacted. The most commonly reported emotional impacts included feeling sad (97.2%), feeling worried (96.9%), and experiencing difficulty caring (94.2%). The most frequently reported personal and social impacts included family activities (88.1%), sleep (81.4%), and time for oneself (80.3%). Over 50% of family members/partners had a FROM-16 score≥17, indicating a 'very' or 'extremely large' effect on their QoL, with females and spouses/partners being impacted significantly more than males or other family members.
CONCLUSIONS: Dementia has a profound impact on the QoL of family members and partners. Routine use of FROM-16 could facilitate the provision of care support, preventing burnout among family members. Such routine data could be used in economic analysis of the burden as well as contributing to prediction of institutionalisation.
Exploring Variability in EQ VAS Scores: A Systematic Review and Meta-Regression of Population Health Studies
OBJECTIVES: The EQ Visual Analogue Scale (VAS), included in all EQ-5D instruments, is widely used to measure overall health status in population surveys. While its psychometric properties are well established in general populations, qualitative studies suggest substantial interpretative variability. This systematic review aimed to identify factors contributing to global variation in mean EQ VAS scores.
METHODS: We searched eight databases for English-language observational studies reporting EQ VAS scores in general population surveys, from inception to 24 January 2025. From each study, we extracted ten pre-specified variables: (1) comorbidity prevalence, (2) mean age, (3) gender ratio, (4) publication year, (5) EQ-5D version (3L/5L), (6) region (Europe, Americas, Asia, Oceania, Middle East), (7) sampling method, (8) administration mode, (9) survey venue, and (10) language. Regions followed UN Statistics Division classifications. We conducted random-effects meta-analysis, subgroup analysis, and meta-regression using the metafor package in R.
RESULTS: Of 16,525 records screened, 55 estimates from 51 studies were included, covering 24 language versions and 496,531 adults. Most data came from Europe (45.5%), South-East/East Asia (23.6%), and the Americas (18.2%). The average age was 46.3 years (SD 6.4). Surveys commonly used probabilistic sampling (72.7%) and interviewer administration (65.5%), especially household-based (87.3%). Univariate analyses identified comorbidity prevalence, age, region, administration mode, and language as significant contributors. In adjusted models, only comorbidity prevalence (β = -10.0, 95% CI -18.1 to -1.9) and the South-East/East/South Asia region (β = 7.6, 95% CI 1.4 to 13.9) remained significant, explaining 51.6% of variability.
CONCLUSIONS: Region and comorbidity prevalence explained most of the variation in EQ VAS scores. The difference between Asian and European populations exceeded the 5-point minimally important difference, suggesting possible ceiling effects. These findings highlight the EQ VAS’s potential for cross-regional comparisons and the need for more data from underrepresented regions to improve generalisability.
Advancing Lost Productivity Measurement in Economic Evaluations: Evidence Priorities From a Targeted Literature Review and Multi-Stakeholder Workshop
OBJECTIVES: Health technology assessment (HTA) agencies vary in their integration of economic impacts from lost productivity due to morbidity and mortality. Lost productivity is infrequently measured and, when it is measured, inconsistent methods are used, and the total impact of lost productivity is often underestimated. We aimed to identify priority areas to improve measurement and valuation of productivity loss through stakeholder-driven recommendations.
METHODS: We conducted a targeted literature review (TLR) of economic evaluations and HTA reports from the previous five years to understand the measurement and valuation of lost productivity. Our findings informed a workshop with eight health economists and health policy experts. Through structured discussions and prioritization exercises, we identified evidence priorities, valuation strategies, and recommendations for comprehensive data collection.
RESULTS: The TLR identified a strong reliance on wage-based valuations focused on absenteeism and a limited valuation of non-work productivity and presenteeism. Workshop participants (N=8) highlighted inconsistencies in productivity measurement and provided methodological recommendations. Participants broadly agreed on several priorities: (1) collecting longitudinal data to capture real-world productivity trends, (2) evaluating non-work productivity (e.g., caregiving), and (3) improving and standardizing methods to measure presenteeism. Beyond the three main priorities, participants proposed including fringe benefits in work-related valuations, disaggregating productivity components when presenting results to improve transparency, avoiding methods that may perpetuate inequities, and developing fit-for-purpose tools to measure real-world productivity losses across work and disease contexts. While participants strongly supported advancing evidence generation and consistent methods, questions remained regarding how to best address enduring methodological challenges and their ultimate impact on economic evaluation.
CONCLUSIONS: Our stakeholder-driven findings emphasize the need for comprehensive, longitudinal data collection that captures absenteeism, presenteeism, and non-work productivity for consistent applications in HTA. Future research should examine how these recommendations can be implemented in clinical trial and real-world settings to inform HTAs and policymaking.
Transparent, Traceable and Reliable Generative AI in HEOR: A Hands-On Workshop on Retrieval-Augmented Generation (RAG) For Evidence Generation
Session Type: Workshop
Topics: Methodological & Statistical Research
Level: Intermediate
Available On-Demand: Digital Conference Pass
Purpose
The growing adoption of Generative AI (GenAI) presents new opportunities and challenges for evidence generation in HEOR. As these tools evolve, stakeholders including HTA agencies, regulatory bodies, and internal teams increasingly require confidence that AI-generated outputs meet standards of transparency, traceability, and reliability.
This workshop will focus on RAG as a practical framework to meet these expectations in real-world HEOR workflows. Presenters will introduce key components of the RAG pipeline including chunking, embeddings, and retrieval using semantic search and explain how these design approaches may impact the accuracy, reproducibility, and transparency of GenAI outputs.
Using real-world examples, the session will demonstrate how RAG-based systems can be applied to common HEOR tasks such as literature reviews, evidence synthesis, and dossier development. The workshop will also explore how complex data formats including tables, graphs, and mixed media require tailored preprocessing to be integrated effectively into GenAI workflows.
Although technical in nature, this session is designed for a wide HEOR audience. Participants will gain essential insight into how specific technical decisions shape the trustworthiness of GenAI outputs, equipping them to make informed judgments when evaluating or implementing GenAI tools.
Description
(10 min) Bill Malcolm will first introduce Generative AI with RAG in HEOR and the importance of transparency, traceability and reliability.
(15 min) Rajdeep Kaur will discuss ongoing developments in RAG relevant to HEOR applications and will explain how to build a strong RAG pipeline for complex HEOR data.
(15 min) Sven L Klijn will walk through key steps of RAG pipeline to explain how the approach supports transparent and traceable evidence generation.
(10 min) Pall Jonsson will share on how RAG based approaches align with GenAI guidance from NICE, CDA, and ELEVATE-AI for HEOR and HTA use.
(10 min) Attendees will have the opportunity to engage with the panellists and ask questions.
Moderator
Speakers
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Rajdeep Kaur
Pharmacoevidence Pvt. Ltd., Mohali, India
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Sven L Klijn, MSc
Bristol Myers Squibb, Princeton, NJ, United States
Sven Klijn is director at Bristol Myers Squibb in the Global HEOR Economic & Predictive Modeling group, where he leads the innovative modeling agenda in hematology and cell therapy. In addition, Sven has an active role in providing modeling education and masterclasses at international congresses. He has widely published on innovative methods, especially in the field of survival extrapolation and Generative AI. Sven has training in public health and health economics and previously had various roles in CROs related to health economic modeling.
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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.
Patient Experience Data for Evidence Generation to Support Regulatory and Access Decision Making
Session Type: Issue Panel
Topics: Patient-Centered Research, Epidemiology & Public Health, Real World Data & Information Systems
Track: Patient-Centered Evidence
Level: Introductory
Available On-Demand: Digital Conference Pass
ISSUE: Patient Experience Data (PED) are considered in the EU as data that directly reflect the experience of a patient or carer, without input or interpretation by a healthcare professional, third party, or (AI-based) device.
Recent years have seen initiatives by regulators internationally to encourage the collection and use of PED in medicine development and throughout the whole lifecycle of medicines, to not only meet the requirements for quality, safety and efficacy, but also to incorporate patients’ and carers’ broader perspectives . This can allow a more informed assessment and decision-making by medicine regulators, health technology assessment (HTA) bodies and healthcare professionals and patients themselves.
However, the current lack of up-to-date and consolidated guidance on PED creates several uncertainties, both for stakeholders collecting or generating these data to support health related claims as well as for those on the receiving end of these data in their assessment for decision making.
EMA is developing a reflection paper to describe general principles on the use of PED across the lifecycle of medicinal products to encourage systematic consideration of PED in medicines development programs and regulatory submissions.
This session will discuss the key aspects of the EMA’s upcoming reflection paper on PED , preliminary perspectives from different stakeholders and possible next steps to further strengthen the use of PED in regulatory and access decision making.
OVERVIEW: To set the scene, an overview of the key aspects of the EMA’s reflection paper on PED will be provided (15 min), followed by a short presentation on ongoing EMA’s work to describe how PED is being incorporated in applications for marketing authorisation and extension of indication (15 min). A panel discussion and a Q&A will follow, to reflect different perspectives (see above list of panellists) and propose some possible next steps.
Moderator
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Steffen Thirstrup, PhD, MD
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
Speakers
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Denise Umuhire
European Medicines Agency, Amsterdam, Netherlands
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Carla Torre, PhD
University of Lisbon, Lisbon, Portugal
Does Environmental Sustainability Warrant a Spot on ISPOR’s Value Flower: A Multi-Stakeholder Debate
Session Type: Issue Panel
Topics: Health Technology Assessment, Health Policy & Regulatory, Health Service Delivery & Process of Care
Level: Introductory
Available On-Demand: Digital Conference Pass
Background: While traditional evaluations focus on clinical efficacy and cost-effectiveness, the potential environmental impact of medicines is gaining recognition as an important element that could influence decision-making and prioritization.
Objective: This panel will debate whether and how environmental impact of health interventions could be incorporated into value frameworks. As societies, healthcare systems and industry increasingly confront challenges posed by climate change, it is essential to evaluate how environmental sustainability (henceforth: sustainability) may be factored into the societal value of health interventions.
Discussion: Using polls & Q&A, we will explore critical questions such as:
- Should sustainability be included alongside traditional value criteria like clinical efficacy and cost?
- How can we effectively and holistically quantify the environmental impact of medicines?
- What frameworks can serve as models for incorporating sustainability into decision-making?
Conclusion: By examining the role of sustainability in value frameworks, participants will be equipped with diverse perspectives on how healthcare systems can best navigate the intersection of efficacy, cost, and environmental responsibility.
Moderator
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Amr Makady, PharmD, PhD
Janssen-Cilag B.V., Breda, Netherlands
Speakers
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Martin J Price, PhD
Johnson & Johnson, High wycombe, United Kingdom
Martin has a 30 year career in Market Access within the Pharmaceutical Industry, following experience working in Clinical Pharmacy. He is currently Vice President of Health Economics, Market Access and Reimbursement at Johnson & Johnson Innovative Medicine, Europe, Middle East and Africa.
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Melissa Pegg, BSc, MSc
York Health Economics Consortium, York, United Kingdom
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Jesper Juel-Helwig, MSc, MPH
Rigshospitalet, Copenhagen, Denmark
Is Transferring Evidence Across Europe a Herculean Task? The Opportunities and Challenges of Transportability Analyses in the Context of European Union Joint Clinical Assessments
Session Type: Workshop
Topics: Methodological & Statistical Research, Health Technology Assessment, Real World Data & Information Systems
Level: Intermediate
Available On-Demand: Digital Conference Pass
PURPOSE: The de-prioritisation of real-world evidence (RWE) in methodological guidelines for the newly European Union (EU) Health Technology Assessment (HTA) Regulation and the challenges in harmonising clinical evidence across diverse EU healthcare systems have been widely shared. Transportability analyses evaluating whether evidence from one context (e.g., population, setting, country) can be applied to another to answer external validity questions is not formally integrated in EU Joint Clinical Assessment (JCA), likely due to unclear RWE evidentiary standards and acceptance thresholds by Member States. Recent EU regulatory developments (e.g., European Healthcare Data Space) provide platforms for data-sharing and offer the opportunity for transportability analyses to be explored in EU JCAs.
DESCRIPTION: This workshop will involve interactive, hypothetical EU JCA evidence generation, role play and submission planning. Dr Sarri will present a transportability framework developed by the International Society for Pharmacoepidemiology special interest group and describe its potential uses in EU JCAs (6minutes). Dr Adamson will present a hypothetical EU JCA case study, outlining how transportability analyses may be used alongside trial data to address gaps for a technology with complex evidence generation needs, borrowing learnings from non-EU HTA transportability analyses. Dr Remiro-Azócar will explore opportunities and challenges of using estimands and doubly robust covariate adjustment methodologies in JCAs. Dr Duffield will discuss the role of international data during National Institute for Health and Care Excellence assessments and where adjustments may add value in the context of decision-making. (13minutes each). Through audience participation, various analytical approaches will be explored to address evidence gaps using transportability analyses and their roadblocks. An HTA Coordination Group discussion will be simulated with emphasis on validating transported comparative effects. The workshop will conclude with an interactive discussion including live polling questions (15minutes). Statisticians, payers, industry and patient representatives will benefit.
Moderator
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Grammati Sarri, MSc, PhD
Cytel, London, United Kingdom
Speakers
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Blythe Adamson, MPH, PhD
Flatiron Health, New York, NY, United States
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Stephen Duffield, PhD, MD
NICE, Liverpool, United Kingdom
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Antonio Remiro Azócar, PhD
Novo Nordisk, Madrid, Spain
COAs and Patient Preference Information: Building on a Promising but Challenging Relationship
Session Type: Workshop
Topics: Patient-Centered Research, Clinical Outcomes, Methodological & Statistical Research
Track: Patient-Centered Evidence
Level: Advanced
Available On-Demand: Digital Conference Pass
Integrating clinical outcome assessment (COA) and patient preference information (PPI) has the potential to improve the insights obtained from patient experience data. This workshop will explore opportunities, challenges, and recommendations for bridging gaps between COA and PPI research.
While it has been acknowledged that PPI and COAs can be complementary, debates on the exact nature of these synergies are limited. This is surprising, given the wide range of overlaps in applications. Examples of potential synergies include the use of concept elicitation data for COA development to inform the design of patient-preference studies, preference-weighting of COA data to inform regulatory benefit-risk assessment, and the use of PPI to elicit meaningful change thresholds. However, several methodological barriers remain to be resolved to fully realize the synergies between COAs and PPI. This includes questions on how to incorporate complex COAs in equally complex preference elicitation instruments and potential endogeneity bias from jointly analyzing COA data and PPI. Addressing methodological challenges could advance the science of both fields and improve the quality of patient experience data.
Ms. Bush will define COA and PPI, describe how COA development can inform PP study design and discuss how COAs enhance the interpretation of preference estimates. Dr. Groothuis-Oudshoorn will discuss statistical issues of jointly analyzing both types of data and provide some guidance on potential solutions. Dr. Heidenreich will discuss challenges incorporating COAs as attributes into preference elicitation instruments, outlining different approaches and validation needs. Dr. Tervonen will build on previous speakers by discussing how PPI methods can be used to derive meaningful change thresholds and aid interpretation of COA scores.
Moderator
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Sebastian Heidenreich, BSc, MSc, PhD
Thermo Fisher Scientific, London, United Kingdom
Speakers
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Elizabeth (Nicki) Bush, MS
OPEN Health, Zionsville, IN, United States
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Catharina Groothuis-Oudshoorn, PhD
University of Twente, Enschede, Netherlands
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Tommi Tervonen, PhD
Kielo Research, Zug, Switzerland
14:45 - 15:15
Afternoon Break: Coffee and Connect (Exhibit Hall)
Session Type: General Meeting
14:45 - 15:45
Meditation Session: Meditation for Mental Clarity
Session Type: General Meeting
Level: Introductory
This workshop is open to both novice and advanced practitioners where we integrate meditation with scientific insights, tailored for attendees of our scientific conference. Participants will acquire practical, evidence-based strategies for stress management and the enhancement of mental clarity. This session will highlight the fascinating connection between the breath and emotions and how we can leverage the former for managing the latter. Attendees will also experience guided meditation, specifically designed to induce mental focus. The workshop will conclude with a Q&A session, encouraging an exchange of questions and personal reflections on the meditative practice.
Leaders: The session will be led by Dr Jag Chhatwal and Dr. Rachael Fleurence. Dr. Chhatwal is an associate professor at Harvard Medical School and director of the Institute for Technology Assessment at Massachusetts General Hospital. He has been practicing meditation for two decades and is a certified meditation instructor with the Art of Living with 1000+ hours of training. Dr Fleurence is former senior advisor at the National Institutes of Health. She previously served at the White House on the launch of a national elimination plan for Hepatitis C. She has been practicing meditation for over 15 years.
15:00 - 16:00
HEOR Impact Cases in Affordability and Access
Session Type: HEOR Impact Cases
Impact of NHIS Reimbursement Caps on Epilepsy Care Equity and Outcomes in Ghana
Problem Statement: Ghana’s National Health Insurance Scheme (NHIS) seeks equitable healthcare, but fixed tariff caps on epilepsy medications cause supply shortages, increasing seizures, healthcare costs, and rural inequities. This challenges policymakers to design sustainable reimbursement policies for chronic illness care.
Description: NHIS reimburses epilepsy medications below market prices, leading pharmacies to limit stock due to losses. NHIS data (2016-2020) show medication shortages affected 60% of rural versus 30% of urban epilepsy patients, driven by reimbursement delays (up to 10 months rurally). This correlates with a 25% rise in seizure-related readmissions. Disability-adjusted life years (DALYs) averted analysis indicates consistent access prevents 15% more DALYs for epilepsy than other neurological conditions. The six-visit annual limit for chronic care restricts monitoring, worsening adherence. Cost-effectiveness models, using real-world evidence, project a 20% readmission cost reduction with policy reform, highlighting inequities.
Lessons Learned: NHIS tariff caps undermine epilepsy care equity, disproportionately impacting rural patients. Health Economics and Outcomes Research (HEOR) evidence reveals fixed reimbursement rates increase long-term costs and disparities. Stakeholder dialogues, informed by this case, advocate for dynamic pricing and Health Technology Assessment (HTA) to align reimbursements with market and clinical needs, projecting a 30% adherence improvement. These reforms offer scalable solutions for low- and middle-income countries (LMICs), underscoring HEOR’s role in balancing cost-effectiveness and equity in policy design.
Stakeholder Perspective: This case reflects the government perspective, as NHIS is government-funded. Policymakers grapple with fiscal sustainability versus equitable access. HEOR evidence empowers budget reallocation and reform decisions, enhancing chronic illness care delivery in Ghana and informing global LMIC reimbursement strategies.
From Evidence to Equity: Leveraging Real-World HEOR Analysis to Transform Antidepressant Access Policy for Black Medicaid Beneficiaries in Maryland
Problem Statement: Johns Hopkins Medicine identified treatment disparities in major depressive disorder management among Black Medicaid beneficiaries in Baltimore, where restrictive prior authorization policies limited access to newer antidepressants with improved tolerability profiles. Maryland Medicaid medical directors and managed care organizations required evidence-based recommendations to inform formulary policy decisions affecting 45,000 predominantly Black beneficiaries while containing costs.
Description: Three major Medicaid managed care organizations and Maryland Medicaid leadership faced pressure to expand antidepressant access while demonstrating fiscal responsibility. JHM population health administrators needed data to support value-based contract negotiations and care pathway modifications. Community health advocates demanded evidence to support policy arguments addressing treatment disparities. Researchers conducted comprehensive real-world evidence analysis using electronic health records from 8,200 Black patients with major depressive disorder (2019-2022). Propensity score matching compared clinical and economic outcomes between newer-generation antidepressants versus standard generic options over 24-month follow-up. Multivariable regression models adjusted for demographics, comorbidities, and socioeconomic factors.
Lessons Learned: Newer antidepressants demonstrated superior medication adherence (68% vs 45%; OR 2.6, 95% CI 2.3-2.9), reduced emergency department utilization (31% decrease), and lower psychiatric hospitalization rates (18% decrease). Cost-effectiveness analysis yielded $15,200 per quality-adjusted life year gained with projected annual healthcare savings of $2,850 per patient. Policy implementation resulted in 340% increase in appropriate prescribing over 18 months, generating $1.2 million in avoided acute care costs system-wide. Real-world HEOR evidence proved essential for overcoming payer resistance by demonstrating clear return on investment. Success required sustained collaboration between academic medical centers, payers, and community advocates to translate rigorous economic evidence into actionable policy changes addressing treatment disparities. This case demonstrates how population-level HEOR analysis can drive meaningful healthcare policy reform while simultaneously improving outcomes and containing costs for vulnerable populations.
Stakeholder Perspective: Armed with this evidence, stakeholders successfully negotiated modified prior authorization criteria across all three managed care organizations, eliminating barriers for patients with documented treatment failures. Maryland Medicaid incorporated findings into statewide formulary policy updates, expanding access criteria specifically for populations with documented health disparities.
The Longitudinal Population Health Impact of Opioid Settlement Funding: A Case Study from Pennsylvania in the United States
Problem Statement: Worldwide, about 60 million people used opioids at least once in 2021. In the United States (US), there has been a 7.5-fold increase in overdose deaths involving synthetic opioids from 2015 to 2022, making up 68% of national drug overdose deaths reported in 2022. To address their role in the US opioid epidemic, a $26 billion USD national settlement agreement with major pharmaceutical manufacturers and distributors was reached. Pennsylvania is to receive nearly $2 billion in opioid settlement funds over a 15-year period, raising important questions about how decisions are made regarding settlement fund resource allocation, including the ultimate public health effectiveness of improving the harm caused by the opioid epidemic.
Description: We collect information on interventions funded from settlement dollars from August 2022 to systematically measure the heterogeneity of funding uses, and the impact of these interventions on key population health outcomes of interest, such as trends in overdose deaths, trends in diagnosis of opioid use disorder, etc.
Lessons Learned: This study uses empirical data to assess the effectiveness of a strategy to allow local autonomy over the decision on interventions to fund from settlement funds to reduce the harm caused by opiates. To date over $83M USD has been spent and committed on approved remediation programs by all 67 counties in Pennsylvania. Our research characterizes the variation in spending by type of intervention (e.g., prevention, treatment, recovery support, etc.) and the populations targeted by the spending (e.g., justice-involved individuals, individuals seeking treatment, adolescents, etc.). We use statistical techniques to associate changes in key population health measures to the interventions, with the goal of assessing real world effectiveness.
Stakeholder Perspective: This research is conducted from the perspective of county governments in Pennsylvania. This study explores how to effectively structure and allocate damage awards to address important population and public health outcomes.
15:15 - 16:15
Modernizing Medical Device Evaluation: Aligning Lifecycle Evidence, HTA Methodologies, and Digital Support Tools
Session Type: Forums
Topics: Medical Technologies, Health Policy & Regulatory, Real World Data & Information Systems
Level: Intermediate
Available On-Demand: Digital Conference Pass
Medical devices (including diagnostics, implants, and therapeutic technologies) play a critical role in modern healthcare but present unique challenges for evaluation. Iterative design changes, operator-dependent performance, and broad clinical applications often limit the generalizability of pre-market trial data and complicate conventional health technology assessment (HTA) methods.
This forum will explore how evolving regulatory and HTA frameworks are adopting a lifecycle approach to evidence generation, integrating pre-market studies with structured post-market surveillance, real-world evidence (RWE), and registries. This model supports adaptive, ongoing assessment of device safety, effectiveness, and value. While focused on medical devices, the discussion will extend to the growing digital health ecosystem. Technologies such as software as a medical device (SaMD), digital therapeutics, and remote monitoring tools increasingly blur the lines between software and devices. These innovations require ongoing outcomes-based evaluation and increased methodological rigor, prompting regulatory and HTA systems to evolve accordingly. Digital tools, such as including AI-enabled diagnostics and connected monitoring platforms, can enhance evidence generation by enabling improved data capture, real-time monitoring, and continuous evaluation. However, their utility depends on integration into robust, transparent frameworks that uphold established regulatory and methodological standards.
This session will convene experts from regulatory agencies, HTA bodies, and industry to discuss how evaluation systems can adapt to a dynamic technological landscape. Panelists will share strategies for aligning regulatory, HTA, and payer expectations to foster a policy environment that promotes timely access to high-value medical and digital health technologies.
Moderator
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Lisa Weiss, MPH, PhD
MDIC, NEW YORK, NY, United States
Speakers
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Artem T Boltyenkov, MBA, PhD
Siemens Healthcare Diagnostics Inc., Lexington, SC, United States
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Anastasia Chalkidou
London, United Kingdom
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Rossella Di Bidino, MSc, PhD
Gemelli Teaching Hospital, Roma, Italy
<em>Value in Health</em> Showcase: Medicine Pricing in Theory and in Practice
Session Type: Forums
Topics: Organizational Practices
Level: Intermediate
Available On-Demand: Digital Conference Pass
The pricing of medicines is a key policy issue affecting efficiency, access, and investment in innovation. In this Forum, we showcase 3 new contributions to theory and evidence on pricing published in Value in Health. We begin with an author presentation of Value in Health 2025 Paper of the Year, "Patient Welfare Implications of Indication-Specific Value-Based Pricing (ISVBP) of Multi-Indication Drugs". ISVBP varies drug prices across users. The authors discuss the patient and social welfare implications of ISVBP and present their theoretical analysis. Results show that ISVBP is not necessarily patient welfare improving, suggesting ISVBP should be carefully evaluated and implemented. Next, we consider Europe's experience with international reference pricing (IRP), and its implications for the proposals to introduce IRP in the United States: "Referencing Drug Prices of Other Countries May Not Sustainably Lower Prices in the United States: Lessons From Europe". The authors note that IRP in Europe has resulted in delayed or no access in many countries, without sustainably reducing price levels. While lowering US drug prices is important, doing so through IRP is ill-advised. Finally, we consider evidence presented in a new Value in Health paper reporting "The Cost-Effectiveness of the Next 15 Drugs Selected for Medicare Drug Price Negotiations." This forum will be introduced by Value in Health Editors-in-Chief, C. Daniel Mullins and Nancy Devlin, who will moderate a panel discussion among the presenting authors of each paper, including audience Q&A. The Forum will aim to identify remaining research priorities and evidence gaps concerning pricing.
Speakers
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C. Daniel Mullins, PhD
University of Maryland School of Medicine, Baltimore, MD, United States
C. Daniel Mullins is a Professor at the University of Maryland School of Pharmacy. He is Founder and Executive Director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, a community-academic partnership for patient-driven research. Dr. Mullins has received approximately $25 million in funding as a Principal Investigator from AHRQ, FDA, NCI, NHLBI, NIA, NIMHD, the Patient-Centered Outcomes Research Institute (PCORI) and various patient advocacy organizations and pharmaceutical companies. At the University of Maryland Baltimore (UMB), he received the Dr. Patricia Sokolove Outstanding Mentor Award and the Dr. Martin Luther King Jr. Faculty Diversity Award. He was named Researcher of the Year at UMB and was awarded a University System of Maryland Wilson H. Elkins Professorship. At ISPOR, he has served as Editor-in-Chief of Value in Health since 2010 and received the Marilyn Dix Smith Leadership Award in 2017 and the Avedis Donabedian Lifetime Achievement Award in 2024.
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Nancy J Devlin, PhD
University of Melbourne, Melbourne, Australia
Building HTA Capacity in CEE: Is it Possible to be Made Within a Common Roadmap - From Pilot Projects to Sustainable Systems
Session Type: Issue Panel
Topics: Health Technology Assessment, Health Policy & Regulatory
Level: Introductory
Available On-Demand: Digital Conference Pass
Issue:
This panel will debate whether it is feasible to develop a comprehensive regional roadmap guiding Central and Eastern European (CEE) nations from fragmented capacity-building activities toward fully integrated, sustainable HTA infrastructures. It will identify core components—governance, financing, and shared data platforms—tailored to CEE contexts.
Overview:
HTA capacity in CEE is uneven: some countries rely on ad hoc, donor-driven pilots, while others have nascent or partially institutionalized agencies. Consequently, regional harmonization is limited, evidence standards vary, and resource constraints impede sustainability. The diversity of health systems, legal frameworks, and EU integration levels across CEE highlights the need for a context-sensitive roadmap. A common CEE roadmap could address shared challenges: establishing standardized training curricula, aligning legal mandates, defining governance structures, and creating interoperable data systems suited to CEE regulations.
This 90-minute session is structured as follows: three 15-minute presentations will outline distinct capacity-building experiences (organizational, institutional, and early-stage perspectives), followed by a 30-minute moderated debate to synthesize lessons, determine actionable steps, and propose a draft roadmap with timelines and collaborative mechanisms. The remaining 15 minutes are reserved for audience Q&A and interactive discussion. Attendees will include national HTA agency representatives, Ministry of Health officials, regional network coordinators, academic researchers, patient advocates, and industry stakeholders. They will gain practical insights into barriers to scaling pilots into permanent HTA bodies, strategies for harmonizing training and mentorship across borders, legal and governance prerequisites for institutionalization, funding mechanisms, and stakeholder engagement models.
By session’s end, participants should understand how to collaborate on a phased, region-wide roadmap—from initial pilot design to embedding HTA in national decision-making—and which policy levers and capacity-building tools are essential to sustain a CEE-wide HTA framework.
Moderator
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Malwina Holownia-Voloskova, MSc, PharmD
Certara, Cracow, Poland
Speakers
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Oleksandra Oleshchuk, PhD, MD
Horbachevsky Ternopil National Medical University, Ternopil, Ukraine
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Eva Turk, PhD, MSc
Nationa Institute of Public Health, Ljubljana, Slovenia
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Kelly Lenahan, BS, MPH
ISPOR, Lawrenceville, NJ, United States
Kelly Lenahan is the Director of Global HTA Initiatives at ISPOR – the professional society for health economics and outcomes research. She has been responsible for all the global health technology assessment activities at ISPOR since 2015. She collaborates with local experts to hold HTA Roundtables in 5 major regions of the globe. Kelly has a passion for sharing information and helping to make connections globally, especially in low- and middle-income countries, to improve healthcare systems and health technology assessment processes. She received her Master’s in Public Health with a concentration in Epidemiology from Rutgers University. Kelly has several publications about coverage restrictions for specialty drugs in the United States and received a Rising Star award from the Healthcare Businesswomen’s Association
Navigating Complexity in Population Health Decision-Making: Evidence, Values, and Real-World Constraints
Session Type: Forums
Topics: Epidemiology & Public Health, Health Technology Assessment, Economic Evaluation
Level: Intermediate
Available On-Demand: Digital Conference Pass
In an era of limited resources and increasing complexity, policy decision-making for population-level interventions, such as immunization programs, screening initiatives and linkage to care in oncology require careful balancing of clinical evidence, cost effectiveness, social values, equity, patient and public values and system-level priorities.
This panel brings together experiences from Austria, Canada and Jordan to explore how different frameworks and institutional contexts shape decisions for population-level programs.
Speakers will highlight how health technology assessment (HTA), and specifically economic evaluation, are integrated, or constrained, within broader decision processes. The Canadian experience will be used to illustrate Canada’s National Advisory Committee on Immunization’s (NACI) application of a values-informed framework to complex vaccine decisions in the context of an expanded mandate. In Austria, new funding prompted rapid modeling to support decisions for over twenty vaccines and multiple population groups to inform vaccine prioritization within the additional funding envelope. In Jordan, evidence generation to support breast cancer screening initiatives highlight the challenges of designing programs across diverse implementation pathways, and patient-centered collaboration local priorities in the context of a recent adopter of HTA.
Our discussion will draw attention to how domains within HTA decision frameworks and contextual factors, including trust in institutions, deliberative processes, system maturity, and political will, influence of real-world decision-making.
We will start with a question to the audience “What factors do you think should weigh most in deciding whether a population health intervention is funded?”
Following brief presentations, panelists will invite the audience to engage with provocative questions, share country experiences, and identify key research gaps. We will conclude with a collective discussion on how to better link evidence generation to actionable and equitable policies across diverse health systems, and provide an outlook on broader future societal impacts, including One Health.
Moderator
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Beate Jahn, PhD
UMIT – University for Health Sciences, Medical Informatics and Technology, Hall i.T., Austria
Speakers
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Günther Zauner, MSc, PhD
dwh GmbH, Vienna, Austria
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Abeer A Al Rabayah, MBA, MSc, DrPH
King Hussein Cancer Center, Amman, Jordan
Dr. Abeer Ahmad Al Rabayah leads the Center for Drug Policy and Technology Assessment (CDPTA) at the King Hussein Cancer Center (KHCC) in Amman, Jordan. In addition to her role at KHCC, Dr. Abeer holds a research associate position at the Institute of Public Health, Medical Decision Making, and Health Technology Assessment at UMIT TIROL – University for Health Sciences and Technology, Hall i.T., Austria.
As the director of the Hospital-Based Health Technology Assessment (HB-HTA) program and the founder of the Health Technology Assessment (HTA) fellowship program at KHCC, In 2024, the KHCC HB-HTA program received the King Hussein Cancer Research Award for Academic Program Excellence. Dr. Abeer has significantly contributed to the field. She has authored over 20 publications in HTA and Health Economics research, covering areas such as decision analytical modeling, HTA processes and methods, valuation of health outcomes, and Health-Related Quality of Life research.
Dr. Abeer is influential at the national, regional, and international levels in advancing the implementation and development of HTA. She is an active member of numerous HTA steering committees. She is the chair of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) HTA round table for the Middle East and Africa (2024-2026). Currently, Dr. Abeer is appointed by the World Health Organization to serve as a member of the Technical Advisory Group on Pricing Policies for Medicines (TAG-PPM), providing evidence-based technical advice to improve affordable access to essential and priority medicines.
Dr. Abeer holds a BSc in Pharmacy, a Master of Business Administration (MBA) from Jordan University, and a Master of Science (MSc) in International Health Technology Assessment from the University of Sheffield, United Kingdom. She is a PMP-certified project manager and a final-year Ph.D. candidate at UMIT TIROL – University for Health Sciences and Technology, Hall i.T., Austria.
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Beate Sander, MBA, RN, PhD
University Health Network / University of Toronto, Toronto, ON, Canada
Beate Sander, RN MBA MEcDev PhD, holds a Tier 1 Canada Research Chair in Economics of Infectious Diseases, is a Senior Scientist at the University Health Network (UHN), where she chairs the Health Systems and Policy Research Collaborative Centre, and Professor at the Institute of Health Policy, Management and Evaluation (IHPME), University of Toronto. She holds appointments as Adjunct Senior Scientist at ICES and Adjunct Scientist at Public Health Ontario (PHO).
Dr. Sander is an internationally recognized leader in infectious disease economics with extensive expertise in health economics and simulation modeling. Her research focuses on assessing infectious disease interventions using simulation modeling and estimating the burden of infectious diseases using population-based data. She leads methodological research to integrate equity considerations into economic evaluations, including recent work on inequality aversion.
Dr. Sander serves as an expert to national and international advisory bodies, including Canada’s National Advisory Committee on Immunization (NACI), where she chaired the Economics Task Group and co-chaired the Economic Guidelines Task Group. She held several leadership roles during the COVID-19 pandemic, advancing evidence generation and knowledge mobilization. Dr. Sander is Past President of the international Society for Medical Decision Making.
16:00 - 16:45
International Reference Pricing Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 2. Posters will be hung from 16:00 - 19:00.
Patient Engagement Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 2. Posters will be hung from 16:00 - 19:00.
16:00 - 19:00
Poster Session 2
Session Type: Research Posters
Poster Tours will be from 16:00–16:45 | Presenters will be with their posters from 18:00–19:00
Poster Session 2
Session Type: General Meeting
Poster Tours will be from 16:00–16:45 | Presenters will be with their posters from 18:00–19:00
16:20 - 16:50
Need. Method. Match. Explore the SUSTAIN-HTA Tools
Session Type: Fast Facts
Topics: Health Technology Assessment, Organizational Practices
Level: Intermediate
PURPOSE:
Despite the growing number of methodological innovations available to support HTA, many HTA bodies face challenges in identifying methods that are relevant, ready for use, and responsive to real-world needs. The SUSTAIN-HTA Methods Observatory Tool was developed to address this challenge by systematically identifying, cataloguing, and classifying methodological innovations that can support HTA. This workshop introduces participants to the Observatory and its companion Horizon Scanning Tool, offering hands-on experience to explore their functionalities and assess practical applicability. Participants will have the opportunity to provide feedback on how these tools can support methodological alignment and uptake across jurisdictions.
DESCRIPTION:
The workshop will begin with a 10-minute presentation by Saif Elayan, outlining the rationale, development, and intended applications of the SUSTAIN-HTA Methods Observatory and Horizon Scanning tools, including a demonstration of key features. Participants will access the tools and engage in interactive tasks simulating real-world use cases, including exploring unmet methodological needs, identifying matched methods, and testing search and filter functions (10 minutes).
Next, a panel of representatives from European HTA bodies will provide real-time reflections on their experience using the tools, offering live commentary on usability, structure, institutional value, and potential refinements to enhance the tools’ relevance and impact (15 minutes).
Participants will then engage with the panel in a 15-minute interactive segment, including guided questions, experience-sharing, and discussion of practical applications. Real-time polling and online feedback will be used to collect input on usability, relevance, and future improvements.
The workshop will conclude with a 10-minute discussion on future directions, focusing on how the tools can facilitate methodological alignment, foster adoption of innovative methods in HTA, and support knowledge exchange across Europe’s HTA ecosystem. The workshop is relevant to HTA professionals, academics, researchers working on methodological development, and policymakers.
Moderator
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Saif Elayan
Utrecht University, Utrecht, Netherlands
Speakers
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Ed Clifton, MSc
Glasgow, United Kingdom
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Rosa M Vivanco-Hidalgo, MSPH, PhD
AQuAS, Barcelona, Spain
Rosa Maria Vivanco Hidalgo, MD PhD MPH, is Neurologist, doctor of medicine (Universitat Autònoma de Barcelona), Master in Public Health (Universitat Pompeu Fabra) and is currently the director of health technology assessment area at the Agency for Health Quality and Assessment of Catalonia (AQuAS). She is a member of the Spanish Network of Health Technology Assessment Agencies (RedETS) and is part of Board of Directors of INAHTA. She is associate professor in masters and postgraduate courses about digital Health and HTA. She is membre of the mehtodological and procedures subgroup of the EU-HTAR.
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Marta Slomka, Dr n. med.
Warsaw, Poland
17:00 - 18:00
Operationalizing Life-Cycle Health Technology Assessment For Precision Medicine
Session Type: Other Breakout Session
Topics: Health Policy & Regulatory, Economic Evaluation, Methodological & Statistical Research
Level: Intermediate
Available On-Demand: Digital Conference Pass
Purpose
This session seeks to critically engage an international audience on operationalizing life-cycle health technology assessment (LC-HTA) and producing decision-grade real-world evidence (RWE) for precision medicine.
Our panel will: introduce a novel LC-HTA framework for highly uncertain precision medicine technologies; present learnings from framework implementation within a Canadian cancer care system; and discuss transportability of RWE generated for informing international regulatory and reimbursement decisions.
Description
Advances in precision medicine challenge conventional regulatory and reimbursement processes. These advances are rapid, involve small patient groups, and are frequently evaluated without a randomized comparison group. Life-cycle HTA alongside patient engagement and continuous RWE generation can resolve evidentiary uncertainty on comparative effectiveness and value.
This session will begin by introducing an LC-HTA framework operationalized within British Columbia, Canada’s cancer care system through the PRecision oncology Evidence Development in Cancer Treatment (PREDiCT) program (10’ Regier).
Centred on a case study of entrectinib, a conditionally authorized treatment targeting advanced NTRK-gene fusion-positive cancers, presenters will then: establish comparative effectiveness via target trial emulation and fusing single-arm Phase I/II entrectinib trial data with real-world data from British Columbia and the United States (13’ Weymann); determine life-cycle cost-effectiveness of entrectinib versus standard care, demonstrating how value-for-money evolves as RWE emerges (13’ Krebs); and discuss evidence transportability across borders for international decision-making (13’ Adamson).
The session will conclude with a facilitated discussion on advancing data fusion, LC-HTA operationalization, and RWE reliability across healthcare systems, engaging with the audience through a real-time prioritization polling exercise (10’).
Moderator
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Dean Regier, BA, MA, PhD
BC Cancer - ARCC - UBC, Burnaby, BC, Canada
Speakers
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Deirdre Weymann, MA
Simon Fraser University, Burnaby, BC, Canada
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Emanuel Krebs, MA
Cancer Control Research, BC Cancer, Vancouver, BC, Canada
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Blythe Adamson, MPH, PhD
Flatiron Health, New York, NY, United States
Diving into the Intended and Unintended Consequences of how the Societal Perspective is Captured in Cost-Effectiveness Analyses
Session Type: Issue Panel
Topics: Economic Evaluation, Health Technology Assessment, Health Policy & Regulatory
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue: Building on the conversation from ISPOR Montreal 2025, this panel will feature European, UK and US perspectives to take a deep dive into how to undertake a societal perspective cost-effectiveness analysis. The panel will discuss a set of case studies across different disease areas that have cost-effectiveness analysis results that have shown to move in both expected and unexpected directions. Methods for capturing societal perspective inputs will be compared and results illustrated. The issue panel will go beyond what should or should not be included in a societal perspective analysis, to thinking about how it is included and what the implications could be – sometimes unintended. The opportunities and challenges of alternative methods will be discussed. The use of a consistent cost-effectiveness threshold regardless of which outcomes are included will be debated and the additional consequences of taking a societal perspective will be highlighted. For example, what might a societal perspective analysis mean for cross-sector collaboration and are we prepared?
Overview: Dan Ollendorf (10 minutes) will introduce the discussion by outlining what a societal perspective cost-effectiveness analysis is intended to inform, the case studies that will be covered, and the societal domains that will be highlighted. Saskia Knies (10 minutes) will share ZIN’s approach to societal perspective analysis and share examples of how societal domains were captured in the focused case studies. Simone Sutherland (10 minutes) will highlight considerations from the same case studies and share a technology developers’ perspective on the importance of the societal perspective. Praveen Thokala (10 minutes) will reflect on the case studies presented and emphasize the implications of broader perspective on the opportunity costs considered. The audience will be engaged with interactive polling and Q&A to collectively prioritize how societal perspective domains should be captured in analyses and discuss priorities for future work.
Moderator
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Dan Ollendorf, MPH, PhD
Institute for Clinical & Economic Review, Boston, MA, United States
Speakers
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Saskia Knies, PhD
Zorginstituut Nederland, Diemen, Netherlands
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C Simone Sutherland, BSc, MSc, PhD
F. Hoffmann - La Roche AG, BASEL, Switzerland
C. Simone Sutherland is Global Access Evidence Lead and HTA Evidence Partner (HEP) at F. Hoffmann-LaRoche. She is responsible for contributing, planning and developing a robust evidence-base within the CVRM and Neurology (Neuromuscular Disease) therapeutic areas on a daily basis. In addition, she is the Lead of the Societal ICER Working Group, which was recently established to consider broader value elements into economic evaluation. Simone has been working in healthcare for over 20 years with experiences and expertise in clinical research, health economics and decision modelling. She holds a PhD in Epidemiology and Public Health from the University of Basel, where she is currently based.
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Praveen Thokala, MASc, PhD
SCHARR, University of Sheffield, Sheffield, United Kingdom
Leveraging Real-World Data in HTA
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session explores the methodological considerations of utilizing real-world data in healthcare. Presentations will cover topics such as validating real-world data sources for economic and resource use analysis, innovative methodologies for making indirect comparisons using real-world survival data, and frameworks for integrating real-world external control arms to accelerate clinical development.
Optimal Hemoglobin Thresholds for ICU Transfusion: A Target Trial Emulation in a Large Electronic Medical Recordbased Real-World Database
OBJECTIVES: To investigate the optimal hemoglobin threshold for initiating red‑blood‑cell transfusion in adult intensive care unit (ICU) patients.
METHODS: We emulated a three‑arm target trial within OneICU, an electronic medical record database with minute‑level physiologic data from Japanese ICUs (2013 - 2024). Adults whose hemoglobin ever fell below 9 g/dL were enrolled and followed in 6‑hour intervals from ICU admission to discharge. We compared three dynamic strategies: transfuse only when hemoglobin dropped below 7, 8, or 9 g/dL. The parametric g‑formula estimated causal effects, adjusting for static variables (age, sex, admission diagnosis, and hospital) and time-varying variables (body temperature, heart and respiratory rates, mean arterial pressure, oxygen saturation, pH, lactate, hemoglobin, and vasopressor use). Patients discharged from the ICU within 7 days were considered censored.
RESULTS: Among 16 233 admissions, 7 300 (45 %) received at least one transfusion. The mean age was 68.5 years, and 6855 (42.2%) were female. Estimated 7‑day ICU mortality was 7.9 % under the 7 g/dL strategy, 7.4 % under 8 g/dL (risk difference [RD] -0.5 %; 95 % CI -0.7 % to -0.4 %), and 6.7 % under 9 g/dL (RD -1.2 %; 95 % CI -1.6 % to -0.8 %). Subgroup analyses in patients ≥70 years, those with sepsis, and those with acute coronary syndrome showed a consistent benefit of a higher hemoglobin threshold.
CONCLUSIONS: A liberal transfusion strategy triggered at ≤9 g/dL resulted in clinically meaningful reductions in early ICU mortality compared with a restrictive 7 g/dL strategy. The use of a large real-world database with minute-level EMR records enabled estimation of intervention effects while accounting for treatment-confounder feedback.
Validation of Primary Care Resource Use and Costs in OMOP-mapped vs. Source CPRD Aurum Data
OBJECTIVES: To validate estimates of primary care resource use and costs from OMOP-mapped Clinical Practice Research Datalink (CPRD) Aurum data against those from the source CPRD Aurum, using a UK cohort of postmenopausal women with fragility fractures.
METHODS: We conducted a retrospective study (2010-2018) of women aged >50 with a first recorded fragility fracture (index). Clinically-relevant primary care encounters and associated costs were estimated over two years post-index date (or until censoring) using both OMOP-mapped and source CPRD Aurum datasets. Multiple encounters with the same healthcare specialty, on the same day, and for the same patient were considered duplicates and only one such record kept. Outcomes included encounter counts and cost estimates (2024 GBP). Differences were assessed using means and 95% confidence intervals from 1,000 bootstrap samples.
RESULTS: The OMOP-mapped cohort included 22,900 women (mean follow-up: 578 days), and the CPRD cohort 23,106 (577 days), with 22,571 appearing in both. At least one encounter was recorded for 97.3% (OMOP) and 97.0% (CPRD). OMOP data showed 9.2% more encounters (537,756 vs. 488,449), with significant variation across specialties. In OMOP, GPs accounted for 73.9% of encounters, nurses 7.9%, other specialties 12.5%, and 5.8% had “No matching concept” (GP Registrar, Seasonal and Locum GP, and Consultant in CPRD). In CPRD, these proportions were 82.9%, 8.3%, and 8.8%, respectively.Mean encounters per patient were 24.1 (CI: 23.8-24.5) in OMOP vs. 21.8 (21.5-22.1) in CPRD. Mean costs were £939.1 (CI: £927.2-£951.6) in OMOP vs. £858.9 (CI: £847.8-£869.1) in CPRD.
CONCLUSIONS: Primary care resource use and costs were slightly higher in the OMOP-mapped dataset. Differences likely stem from specialty mapping, which can be refined. OMOP-mapped analyses can yield results comparable to source CPRD, supporting the conduct of health economics research in single and federated approaches leveraging OMOP-mapped datasets.
A Framework for Accelerating Clinical Development with Real-World External Control Arms (ECA) in Phase 2 trials
OBJECTIVES: External control arms (ECAs) are an established tool to provide valuable supplementary evidence in Phase 3 trials but their potential to offer critical context for in the Phase 2 trial setting is underexplored. We therefore sought to develop a framework for generating an ECA for the Phase 2 trial setting that addresses the inherent challenges of these trials, including their relatively low sample sizes.
METHODS: A literature review of best practices in ECA design for Phase 3 trials was conducted and used to develop a protocol framework for an ECA for a Phase 2 single arm trial. To address the challenge of limited trial patient data for matching, we implemented a simulation approach to facilitate the matching process with the RWD ECA. This framework was applied to a pilot RWD ECA for a newly launched Phase 2 trial of Tucatinib and Doxil in HER2+ metastatic breast cancer (mBC).
RESULTS: This framework establishes key principles for ECA development in the Phase 2 setting, including (1) systematic RWD source identification and curation, leveraging both structured data and supplemental unstructured data; (2) rigorous emulation and application of trial eligibility criteria to define a comparable real-world cohort; and (3) implementation of adaptive matching strategies to address limited trial patient data. To navigate the challenge of small trial cohorts, the framework's adaptive matching component involved creating a simulated dataset via multiple imputation based on enrolled patients.
CONCLUSIONS: Successful implementation of an ECA in a Phase 2 trial setting necessitates a robust understanding of RWD availability and proactive strategies, such as data simulation, to address the complexities of patient matching in small, slow-accruing trials. Further exploration into adaptive ECA methodologies, including advanced simulation techniques, for such trials is warranted.
Real-World Evidence of Healthcare Resource Utilization in Cystic Fibrosis (CF) Patients Treated With Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in France Using Long-term Data From the SNDS Database Linked to the French CF Registry: Findings From...
OBJECTIVES: To describe real-world healthcare resource utilization (HCRU) in people with Cystic Fibrosis (pwCF) before and after ELX/TEZ/IVA initiation in France.
METHODS: This non-interventional, observational, retrospective cohort-study was based on data linkage between the French claims database (SNDS) and demographic and clinical characteristics from the French CF Registry. Inclusion criteria were age ≥12 years and ELX/TEZ/IVA initiation between 12/01/2019, and 12/31/2021. Data censoring was upon death, loss of follow-up or switch to another CFTRm. Assessment of HCRU notably included records of health care professional (HCP) consultations and unique identification codes of reimbursed prescription medications available in the SNDS; medical therapies include, inhaled, oral (tablets), and intravenous (vials) medications. HCP consultations include nurse, physiotherapists and physician visits and can take place during the same visit to a medical facility but are counted as unique events. Differences across HCRU between 12-months pre (baseline period) and 12-months post ELX/TEZ/IVA initiation (follow-up period) were assessed.
RESULTS: 2,316 pwCF met the inclusion criteria. At index date, mean age was 25.9 (SD: 10.3) years; 54.7% male; 59.9% were homozygous for F508del mutation; 46.6% previously received a CFTRm and mean ppFEV1 was 76.5% (SD: 25.0). Mean annual use of medical therapies reduced by 40% following ELX/TEZ/IVA initiation, from 269.4 (SD: 235.3) unique prescriptions of reimbursed medication during baseline to 161.7 (SD: 139.2) during follow-up. Mean annual IV antibiotics use reduced by 85.6% after ELX/TEZ/IVA initiation from 55.4 (SD: 121.0) prescriptions during baseline to 8.0 (SD: 39.3) during follow-up, consistent with the decrease in the proportion of patients receiving IV antibiotics [33.0% (baseline) vs. 8.2% (follow-up)]. Mean annual HCP consultations decreased by 38.1% after treatment initiation, from 135.0 (SD: 167.6) during baseline to 83.6 (SD: 165.8) during follow-up.
CONCLUSIONS: The findings demonstrated the substantial positive impact of ELX/TEZ/IVA reducing HCRU and were consistent with research from other countries.
Real World Evidence – From the Clinician’s Perspective
Session Type: Workshop
Topics: Health Service Delivery & Process of Care
Level: Introductory
Available On-Demand: Digital Conference Pass
Purpose:
• The goal of this workshop will be to provide an overview of the value of real world studies (RWS) for clinicians while identifying gaps in RWE knowledge and opportunities for improvement.
Description:
The following topics and questions will be covered during the session:
• What is the role of RWE in clinical practice:?
o Transition from ‘ideal” RCT study subject to real world patient:
- Understanding factors that provide discrepant results when comparing RCTs to RWS :
•Demographics
•Comorbidities
•Concomitant medications
•Access to care
•Patient concerns and preferences
•Compliance with treatment
•Adherence to management regimen (example diet, habits etc.)
- Distinguish between modifiable and non – modifiable factors that affect patient outcomes:
•Focus on modifiable factors that can yield maximum benefits to each patient
o Knowledge gaps addressed by RWS:
- Patient subgroups
- Factors affecting response to treatment in clinical practice
- Safety and rare adverse events
- Comparative effectiveness
- Patient satisfaction and preferences
The speakers will present examples of RWS from specific therapeutic areas: oncology, autoimmune diseases (dermatology, gastrointestinal and rheumatology), hypertension and diabetes.
Interactive Component:
•Audience will be presented with hypothetical situations where clinical decision making can be influenced by RWE to supplement results of RCTs.
o Audience will be polled on the clinical practice decision and will participate in a discussion about how RWE may have affected the decision.
o Audience will participate in a discussion on the barriers (hesitations) to incorporating RWE in clinical decision making.
•Audience will be polled about the value of RWE for:
o Individual patient management
o Modification of clinical practice patterns
o Health care policies
o Reimbursement decisions
Moderator
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Susan A Oliveria, MPH, ScD
Thermo Fisher Scientific, New York, NY, United States
Speakers
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Eleftherios samartzis
University Hospital Zürich, Switzerland
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John S Sampalis, PhD
PPD, Laval, QC, Canada
Dr. John Sampalis, MSc, PhD, is Vice President, Epidemiology and Scientific Affairs at PPD™, part of Thermo Fisher Scientific. Dr. Sampalis completed his MSc and PhD in epidemiology and biostatistics at McGill University and is a Fellow of the American College of Epidemiology and the American Heart Association. He is a Professor of Medicine at the Faculties of Medicine at McGill University and the Université de Montréal. Dr. Sampalis has more than 40 years experience in clinical research, with a focus on observational studies. He has published more than 200 peer-reviewed publications and more than 500 conference presentations on clinical studies and real-world evidence generation based on observational data, including epidemiological studies, registries, “big data” analytics, and health economics. He is an academic clinical epidemiologist and the founder of JSS Medical Research with extensive expertise in the design and execution of a wide range of global observational and interventional studies.
Embedding Effective Patient Involvement in EU Joint Clinical Assessments
Session Type: Issue Panel
Topics: Patient-Centered Research, Health Technology Assessment, Health Policy & Regulatory
Track: Patient-Centered Evidence
Level: Introductory
Available On-Demand: Digital Conference Pass
EU Joint Clinical Assessments (JCAs) for oncology went live in 2025, yet patient input remains confined to brief reactions to a near-final scope and a draft report. This “back-loaded” model risks tokenism by overlooking essential patient insights on outcomes, comparators and context.
This Issue Panel convenes the Coordination Group, a national HTA agency, and regional patient advocates to debate a no-delay, high-value roadmap. A real-time poll will gauge audience confidence in today’s model before panellists outline the following:
• Map the current regulatory touch-points for patient involvement and highlight clear opportunities for effective and timely patient engagement
• Showcase specific oncology and rare-disease cases where early patient insight has already reshaped regulatory and national HTA.
Stress-test four upgrade options:
1. Standing EU patient-led PICO survey to capture priority outcomes and feed scoping consolidation before the draft scope is written.
2. Compulsory lay summaries of both the manufacturer dossier and the JCA report—produced in plain language and all EU languages—to widen meaningful participation.
3. Patient co-authorship of contextual domains (unmet need, treatment acceptability, lived experience) so interpretations reflect real-world priorities.
4. NICE-style Patient/Carer involvement policy: NICE involves patients and carers through various channels and guided by comprehensive strategy.
Panellists will rank each option against timelines and legal boundaries, then invite the audience to vote on priorities for the Regulation’s 2026 review clause.
Moderator
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Antonella M Cardone
Cancer Patients Europe, Brussels, Belgium
Antonella Cardone is the CEO of Cancer Patients Europe, a pan-European all-cancer patient association. She is a Patient Advocacy Expert and Advisor to the Board of Pancreatic Cancer Europe (PCE), uniting physicians, patients, politicians, journalists, academia, and industry, and she is a member of Inspire2Live.
Antonella led the European Cancer Patient Coalition (ECPC) and served as Executive Director of the Fit for Work Global Alliance.
With 25 years of nonprofit experience in health, social, and employment sectors, she also directed the Global Smoke-free Partnership of the American Cancer Society.
Antonella holds Master's degrees in Science and Business Administration. She has been on the Boards of All.Can and Pancreatic Cancer Europe, where she was vice-chair.
Speakers
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Julie Spony, MSc
European Commission, B, Belgium
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Anne Willemsen, MSc
Zorginstituut Nederland, Diemen, Netherlands
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Alessandra Loscalzo, MSc - MPhil
AGENAS - Agenzia Nazionale per i Servizi Sanitari Regionali, ROME, Italy
Barriers and enablers to the adoption of innovative methods for health technology assessment (HTA) decision-making
Session Type: Issue Panel
Topics: Methodological & Statistical Research, Health Technology Assessment, Economic Evaluation
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE: Over the last few decades a number of new and innovative methods have become established in Health Technology Assessment (HTA) – for example Network Meta-Analysis (NMA) for evidence synthesis, and Rank Preserving Structural Failure Time Models (RPSFTM) for treatment switching. However, their acceptance and use has been incremental, especially in comparison to their development. A number of reasons have been suggested for the slow implementation of new methods in HTA, including; lack of understanding, fear of lack of acceptance by HTA agencies, lack of appreciation of the relative merits of new methods, time/personnel/resource constraints, and lack of software development. This panel will explore with the audience barriers and enablers to the adoption of new methods in HTA, drawing on real-world experiences across academic, policy and industry perspectives. OVERVIEW: Keith Abrams will introduce and provide an overview on the use and acceptance of new and novel methods in HTA (10 minutes – including audience participation via the ISPOR app), and the three panellists will then consider three different aspects of methods translation in HTA using illustrative examples (10 minutes each) – Nick Latimer will discuss what evidence is needed to persuade both analysts and HTA decision-makers of the relative merits of new methods; Rhiannon Owen will discuss, from a methodologist and HTA decision-maker’s perspective what drives methodological conservatism and barriers/enablers to adoption, including software; Ursula Becker will finally discuss, from an industry perspective, how use of a unified modelling approach may facilitate the adoption and use of new methods. Keith Abrams will then moderate a discussion involving the audience and panellists on the benefits and barriers of using new and novel methods in HTA, before eliciting the audience’s final views via the ISPOR app.
Moderator
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Keith R Abrams, BSc, MSc, PhD
University of Warwick, Coventry, United Kingdom
Speakers
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Nicholas Latimer, MSc, PhD
SCHARR, University of Sheffield, Sheffield, 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 2019, Nick began a Senior Research Fellowship funded by Yorkshire Cancer Research in which he is investigating 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.
https://www.sheffield.ac.uk/scharr/sections/heds/staff/latimer_n
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Rhiannon Owen, BSc, MSc, PhD
Swansea University, Swansea, United Kingdom
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Ursula Becker, MSc
Roche, Basel, Switzerland
As Global Access Evidence Enabler at F. Hoffmann-La Roche AG, Ursula Becker is an accomplished health economist and statistician with over two decades of experience in drug development, health technology assessment (HTA), and evidence generation.
She leads a team at Roche focused on HTA Evidence. With expertise spanning neuroscience, oncology, immunology, ophthalmology, and other therapeutic areas, Ursula has driven impactful projects on payer-oriented evidence generation, economic modeling, and statistical analyses.
Beyond Regulatory Approval: Making Patient Experience Central to HTA Decision-Making
Session Type: Issue Panel
Topics: Patient-Centered Research, Health Technology Assessment
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue:
As European healthcare systems shift toward more patient-centric, value-based care, the traditional evidence hierarchy - centered on randomized controlled trials - is under increasing scrutiny. This panel will examine the transition from "evidence of efficacy" to a broader "evidence of value" framework, emphasizing the evolving role of patient experience and preference data in Health Technology Assessment (HTA), pricing, and access decisions.
A significant disconnect exists between how regulatory and HTA bodies value patient experience data (PED). Regulatory agencies like the FDA and EMA have made notable progress, incorporating PROs into labeling and using patient input to inform approvals. In contrast, HTA bodies often may lag behind in systematically integrating PED into evaluations. This inconsistency presents challenges for aligning evidence with both regulatory and HTA expectations, particularly given the diverse methodologies used by different HTA agencies.
Overview:
Building on our ISPOR Montreal panel on value evidence archetypes, we found that PED - especially PRO data - is typically viewed as “supportive” rather than essential in access decisions. This highlights a misalignment in how stakeholders interpret and apply patient-centered evidence, despite its increasing importance.
Through real-world case studies, we’ll explore examples where strong PRO data supported regulatory approval but failed to influence HTA outcomes. We’ll identify strategies to better align PED with HTA decision-making by examining current uses of clinical, economic, real-world, and patient-generated evidence. Special attention will be paid to oncology, rare diseases, and chronic care.
Panelists from both pharmaceutical and HTA backgrounds will offer practical insights into optimizing evidence generation plans, aligning with payer expectations, and strengthening the patient voice in value assessment frameworks.
Attendees will leave with actionable strategies to enhance the impact of PED for decision making.
Moderator
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Martin Rost, PhD
AESARA, Boca Raton, FL, United States
Speakers
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Robyn Carson, MPH
AbbVie, Morris Plains, NJ, United States
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Patrick K Hopkinson, MBA, DPhil
PHTA Consulting, Wokingham, United Kingdom
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Indranil Bagchi, MS, PhD
GSK US, Collegeville, PA, United States
Dr. Indranil Bagchi is the Global Head of Pricing & Market Access at GSK. In this role, Indranil drives the overall strategy on value demonstration and market access across all product areas in the GSK portfolio, to secure access to medicines for our patients and to maximize the value of our portfolio.
Indranil has more than two decades of experience in market access across several major companies in the pharmaceutical industry. In 2014, Indranil received the ‘Outstanding 50 Asian Americans in Business’ award and in 2010, Indranil was recognized in Pharmaceutical Executive magazine’s annual roster of Emerging Leaders, “The New Breed of Leadership.” Indranil is a frequent speaker and contributor to forums, articles and conferences addressing issues related to access to medicines.
Prior to GSK, Indranil was Senior Vice President and Worldwide Head of Value and Access at Novartis Oncology. Previously at Pfizer, he was Vice President and Global Head of Payer Insights and Access and prior to that, he was with GlaxoSmithKline in Health Economics and Outcomes Research. Dr. Bagchi has an undergraduate degree in Pharmacy, a master’s degree in Pharmacy and Healthcare Administration and a doctoral degree in Pharmaceutical Socioeconomics.
Incorporating Patients' Perspectives in HTA
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session explores different approaches to capturing and applying patient perspectives in health research and decision-making. Presentations examine use of a visual analog scale for measuring health, social care, and carer-related quality of life; integration of patient experience data into clinical trial design; treatment burden and quality of life in Colombian patients with nAMD and DME; and the frequency and impact of patient input in HTA decisions for cell and gene therapies in the UK, Canada, and Australia.
Mixed-Method Exploration of Visual Analogue Scale (VAS) Variations to Measure Health, Social Care, and Carer Related Quality of Life (Qol)
OBJECTIVES: Visual analog scales (VAS) can serve as brief yet informative patient reported outcome measures. This mixed methods study aimed to assess perceptions of a VAS with variations in the construct and recall period for measuring health, social care, and carer related QoL.
METHODS: US adults who were: 1) diagnosed with chronic illness; 2) unpaid caregivers; 3) social care users (e.g., received caregiving, living with disability) were recruited from ResearchMatch. In an online survey, three VAS variations were presented in a random order. Variations included no recall period with different constructs (‘quality of life’, ‘health’, and ‘health and well-being’) on a 0-100 scale. In an interview, participants discussed construct interpretation, appropriateness of recall periods (none, today, past 7 days), and whether these influenced their responses. Interviews were analyzed qualitatively through thematic analysis.
RESULTS: Thirty-four participants completed the survey and interview. Mean responses for QoL, health, and health and well-being (HWB) were 66.4 (SD=21.5), 61.9 (SD=21.3), and 60.9 (SD=21.8), respectively. Compared to health, effect sizes for QoL and HWB were 0.25 (small) and 0.05 (negligible). All three VAS items were strongly correlated (r=0.84-0.96).Participants interpreted health as clinical diagnoses, emphasizing physical health over mental/emotional health.Interpretations of HWB varied: complementary to health, indicating mental/emotional health, or redundant with health.QoL evoked broader assessments encompassing economic or social-relationship status. Most participants preferred having no specified recall period, allowing consideration of timeframes (e.g., 1-24 months) relevant to their experience.
CONCLUSIONS: Mixed-methods analyses offered insights into participants’ perceptions when interpreting VAS versions for health, social care, and carer related QoL. Qualitatively, HWB was perceived differently from health, yet quantitatively, resultswere similar. Further research in larger samples and other countries can help to inform the extent to which constructs and recall periods affect responses.
The Frequency of Mentions and Impact of Patient Input in HTA Decision Making in Cell and Gene Therapies Across the UK Canada and Australia
OBJECTIVES: Patient input is considered in health technology assessments (HTAs) by the UK’s National Institute for Health and Care Excellence (NICE), Canada’s Drug Agency (CDA), and Australia’s Medical Services and Pharmaceutical Benefits Advisory Committees (MSAC and PBAC respectively). This research explored the frequency of mentions and impact of patient input, including both patient-reported outcomes (PROs) and supplementary patient inputs (SPIs), in HTAs of cell and gene therapies (CGTs).
METHODS: HTA reports of CGTs assessed by NICE, CDA and PBAC or MSAC since 2020 were identified. Natural language processing techniques, including the use of an OpenAI o4-mini language model, were used to gather relevant quotes and generate ratings of direct, indirect or no impact of PROs and SPIs on the HTA outcome, which were subsequently validated via human input. Counts of predefined relevant keywords were also extracted and analysed using a negative binomial generalized linear model, to explore how wordcount varied by market.
RESULTS: Across the 3 markets, 4 CGTs were identified which have undergone an assessment for at least 1 indication. A total of 21 HTA reports were analysed (7 per market). The variation in the total wordcounts across markets was not statistically significant (NS) (Australia: 99; Canada: 92; UK: 66). Where PRO outcomes were available, robust, and significant, they directly influenced the HTA outcome through their incorporation in economic analyses across markets. Where available, SPIs were acknowledged and had an indirect impact across all HTA reports, however none had a direct impact.
CONCLUSIONS: The variation in frequency of mentions of patient input in HTA reports across markets was NS. PROs were consistently found to have a direct impact on HTA outcomes. While SPIs only had an indirect impact, it is promising to observe their consistent consideration in CGT assessments across markets over the last five years.
Implementation of Patient Experience Data in Clinical Trial Design: A Literature Review and Semi-Structured Interviews
OBJECTIVES: Patient-centred healthcare is essential for aligning research and development with patients’ needs and preferences. Integrating Patient Experience Data (PED) into clinical trial design can improve endpoint selection, recruitment, retention, and ensure trials are relevant to what matters to patients. However, evidence on how PED is currently used in clinical trial design is limited. This study aimed to investigate current trial design practices and how patients’ perspectives are integrated into these processes.
METHODS: A literature review was conducted in PubMed and Embase, identifying 231 articles related to PED use in clinical trials. To enrich these findings, 28 semi-structured interviews were conducted with diverse stakeholders, including patient organizations, physicians, researchers, developers, regulators, pricing and reimbursement agencies, and policymakers. Interviews were transcribed and analyzed using thematic framework analysis.
RESULTS: Literature and interviews indicate increasing interest in PED, particularly in using tools like patient-reported outcome measures and patient-reported experience measures. However, their application remains inconsistent across disease areas and trial phases, often due to a lack of standardized methods and validated tools. More research on clearly defining patient-relevant endpoints and core outcome sets were highlighted as opportunities in clinical trial design. Stakeholders also emphasized the need for earlier patient involvement in clinical trial design, regulatory support for effective PED integration, and stronger cross-sector collaborations to define guidelines for implementing PED in clinical trials.
CONCLUSIONS: Despite a clear growing trend, PED integration in clinical trial design remains fragmented. Advancing toward patient-centered research requires early and meaningful patient involvement, standardized outcome measures, and coordinated multi-stakeholder efforts. Strengthening partnerships across healthcare decision-making can ensure clinical trial designs reflect patients’ real needs and preferences, thereby powering value, access, and impact through patient-centered collaboration.
Treatment Burden and Quality of Life in Patients With Namd and DME in Colombia: A Mixed Methods Study
OBJECTIVES: To describe the treatment burden and quality of life (QoL) of Colombian patients with neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME) treated with anti-VEGF therapies, and to identify adherence barriers, treatment satisfaction, patient and caregiver preferences.
METHODS: A mixed-method explanatory study evaluated through a quantitative survey quality of life in 62 nAMD and 46 DME adult patients using the NEI-VFQ-25 questionnaire. Treatment satisfaction was assessed using the MacTSQ for nAMD patients and RetTSQ for DME patients. Semi-structured interviews with 15 patients and 4 caregivers explored adherence barriers and treatment preferences. Findings were integrated using thematic analysis and data triangulation. Patients included had received anti-VEGF treatment within the last 6 months.
RESULTS: The study sample comprised 62% females. Bilateral eye involvement was reported in 54.6% of cases, with Aflibercept being the primary anti-VEGF agent (86.7%). The average NEI-VFQ-25 composite score was 59.2 with domains showing impairment in mental health, driving, and dependence in both diseases. Treatment satisfaction was lowest among patients with DME when they had bilateral vs unilateral disease (RetTSQ 70.6 vs 58.5) and with >6 injections, compared to 3-6 and <3 (59, 71.3, 73). Qualitative findings highlighted that patients faced significant financial burdens, long waiting times, and disruptions to daily routines due to their disease. Patients appreciated the treatment for improved vision despite challenges, preferring regimens that reduced visit frequency. The main reason for treatment adherence among patients with nAMD/DME is the desire to preserve their vision, supported by family or caregiver involvement and clear communication and trust in the doctor-patient relationship.
CONCLUSIONS: Anti-VEGF therapies improve perceived vision in nAMD and DME patients, but high treatment burden and logistical barriers hinder adherence and QoL. Addressing these factors through tailored strategies, enhanced doctor-patient communication, and caregiver inclusion is critical to optimize outcomes in real-world settings.
Integrating Capacity-Enhancing Innovations in Healthcare Systems: Are Current Methods Adequate to Demonstrate the Impact on Efficiency and Capacity?
Session Type: Issue Panel
Topics: Medical Technologies, Health Technology Assessment, Economic Evaluation
Level: Introductory
Available On-Demand: Digital Conference Pass
ISSUE:
Stakeholders considering new innovations do not typically focus on the innovation’s potential to increase efficiencies and release capacity into the healthcare system. Panel speakers will discuss and debate the relevance and importance of these value elements and whether current measures are appropriate to fully assess this value element.
OVERVIEW:
Healthcare systems are under pressure from an expanding aging population, an overloaded workforce, and constrained budgets. The demand for patient care is not being met, leading to long wait times and delayed patient care. Capacity-enhancing innovations (CEIs), which release capacity in the form of resources, have the potential to relieve overburdened healthcare systems and increase access to care.
This issue panel will define the ‘capacity-enhancing’ value dimension, identify appropriate measures, and debate the importance of these value elements. The panel will discuss how their organizations consider the potential for medical technologies to address capacity constraints, whether there are specific technologies that are better suited to enhance capacity within a healthcare system, and whether current methods for economic evaluation and tech assessments incorporate capacity measures appropriately.
Each speaker will present their perspective for 10 minutes with an additional 10 minutes reserved for debate facilitated by the moderator. The moderator will open the discussion for 10 minutes to allow the panel to address audience questions.
Individuals interested in the medical devices, diagnostics, and the digital health space would benefit from attending.
Moderator
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Michela Canobbio, MS
Becton Dickinson, Milano, Italy
Speakers
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Neil Hawkins, MBA, MSc, PhD
University of Glasgow, Oxford, United Kingdom
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Laura Sampietro-Colom, PhD, MD
Hospital Clinic de Barcelona, SABADELL, Spain
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Lotte Steuten, MSc, PhD
Office of Health Economics, London, United Kingdom
18:00 - 19:00
Welcome Reception (Exhibit Hall)
Session Type: General Meeting
Tue 11 Nov
7:00 - 17:00
Registration Hours
Session Type: General Meeting
7:30 - 8:30
Morning Coffee Service
Session Type: General Meeting
8:30 - 9:45
Plenary Session: Pragmatic Trials—Bridging Research and Real-World Care
Session Type: Plenary
Topics: Study Approaches, Health Technology Assessment, Patient-Centered Research
Level: Intermediate
Available On-Demand: Digital Conference Pass
While explanatory clinical trials, which focus on evaluating therapeutic efficacy under ideal circumstances, are crucial for learning about new therapeutic interventions, they often include non-representative patients due to stringent eligibility criteria. Additionally, they often use intermediate endpoints, which can lead to uncertainty about the applicability of study results to patients in the real world. Therefore, they may not offer the best setup to address clinically meaningful daily clinical questions based on patient-relevant endpoints, highlighting the need for optimizing therapeutic strategies. This can be partly achieved by incorporating more pragmatic elements into cancer clinical trials conducted in real-life settings to enhance patient-centricity, efficiency, and the generalizability of evidence. This approach could also consider the interests and needs of stakeholders in healthcare, HTA agencies, clinicians, payers, and most importantly, patients and caregivers. However, these more pragmatic trials, especially those addressing the overutilization of therapeutic interventions, are not without their challenges. Patient acceptability, trial designs and feasibility, as well as fitness for regulatory frameworks and, finally, financial resources to conduct such trials, represent some of the most concerning challenges. The panel will attempt to disentangle the role, benefits, and challenges of pragmatic trials. How pragmatic trials could help re-engineer the process of development into access. Can pragmatic clinical trials be part of the solution to addressing disparities and inequalities in access to clinical trials, thereby bringing faster therapeutic progress to all?
Moderator
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Denis Lacombe, MD
Brussels, Belgium
Denis Lacombe graduated with his MD from the University of Marseilles (France) in 1988 and obtained a Master Post-Doctoral Fellowship at The Roswell Park Cancer Institute (Buffalo, NY USA) for research in pharmacology and pharmacokinetics from 1989 to 1991. From 1991 to 1993, he worked as a Clinical Research Advisor in charge of the development of a new drug in oncology in the pharmaceutical industry.
Dr Lacombe joined the EORTC in 1993 as a research fellow and quickly became a very active and productive Clinical Research Physician involved in the conduct of clinical research from protocol development through publication for a number of oncology indications from phase I to phase III. Dr Lacombe contributed to the strategic evolution of the EORTC pan-European clinical and translational research infrastructure as well as developing partnerships with multiple stakeholders.
Dr Lacombe rose to the position of Director EORTC Headquarters in 2010, was appointed Director General in April 2015 and Chief Executive Officer in June 2021. In his current position, Denis Lacombe leads all activities promoting the EORTC as a major European organization in Cancer Clinical and Translational Research, including scientific activities, public relations and medium-term strategies as defined by the EORTC Board as well as internal and external communications.
Speakers
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Antonella M Cardone
Cancer Patients Europe, Brussels, Belgium
Antonella Cardone is the CEO of Cancer Patients Europe, a pan-European all-cancer patient association. She is a Patient Advocacy Expert and Advisor to the Board of Pancreatic Cancer Europe (PCE), uniting physicians, patients, politicians, journalists, academia, and industry, and she is a member of Inspire2Live.
Antonella led the European Cancer Patient Coalition (ECPC) and served as Executive Director of the Fit for Work Global Alliance.
With 25 years of nonprofit experience in health, social, and employment sectors, she also directed the Global Smoke-free Partnership of the American Cancer Society.
Antonella holds Master's degrees in Science and Business Administration. She has been on the Boards of All.Can and Pancreatic Cancer Europe, where she was vice-chair.
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Michael Zaiac, MD, PhD
Daiichi Sankyo Europe GmbH, Zürich, Switzerland
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Beate Wieseler
Cologne, Germany
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Richard Sullivan, MD, PhD
Institute of Cancer Policy, Centre for Conflict & Health Research, King's College London, London, United Kingdom
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Natasha Azzopardi-Muscat, MD, PhD
WHO Europe, Copenhagen, Denmark
Dr Natasha Azzopardi-Muscat, Director of the Division of Health Systems, WHO Regional Office for Europe.
Dr Natasha Azzopardi-Muscat is a medical doctor and a specialist in public health medicine.
Following her qualification as a medical doctor from the University of Malta in 1995, Dr Azzopardi-Muscat worked in various clinical specialties, including maternal and child health, mental health, and primary care. Having successfully completed a Master’s Degree in Health Services Management at the London School of Hygiene and Tropical Medicine and the London School of Economics and Political Science she returned to work at the Ministry of Health in Malta.
Between 2001 and 2013, she occupied various senior positions in the Ministry including Director for EU and International Affairs, Director General for Strategy and Chief Medical Officer.
In 2016 she obtained her PhD from the University of Maastricht having successfully defended her transdisciplinary research on “The Europeanisation of health systems: a small state perspective”. She is the author of several publications in public health and European health policy.
Between 2016 and 2020 she served as the President of the European Public Health Association (EUPHA) where she was actively involved in health advocacy at the European level.
In 2020, Dr Azzopardi-Muscat joined WHO/Europe and was appointed Director of the Division of Country Health Policies and Systems She served continuously in that position until her re-appointment as Director Health Systems in June 2025.
She is an associate professor at the University of Malta and a visiting scholar at the University of Maastricht.
In 2024 she was awarded the Andrija Stampar medal by the Association for Public Health Schools of the European Region (ASPHER) and in 2025 she was awarded an Honorary Fellowship by the Faculty of Public Health of the United Kingdom in recognition of her important contributions to public health.
Dr Azzopardi-Muscat is married to Dr Conrad Azzopardi and has three children Aidan, Sereena and Nathan.
Plenary Session: Welcome Remarks, Presidential Address, and Awards
Session Type: Plenary
Available On-Demand: Digital Conference Pass
Start Day 2 of ISPOR Europe 2025 with inspiring insights from the Society’s leadership. This session will feature welcome remarks, followed by addresses from the ISPOR President and WHO Europe, and an acknowledgement of prestigious ISPOR awards.
Immediately following the opening presentations, the scientific plenary panel will take the stage.
Speakers
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Uwe Siebert, MPH, MSc, ScD, MD
UMIT TIROL - University for Health Sciences and Technology; Harvard Chan School of Public Health, Hall in Tirol, Austria
Uwe Siebert, MD, MPH, MSc, ScD, is a professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL-University for Health Sciences and Technology in Austria and director of the Division for HTA in the ONCOTYROL–Center for Personalized Cancer Medicine in Austria. He is also adjunct professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health and Affiliated Researcher in the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston.
After medical school, he worked for several years as a physician in international public health projects in West Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in decision sciences at the Harvard School of Public Health.
His research interests include applying real-world evidence-based quantitative, causal and translational methods from public health, epidemiology, artificial intelligence, comparative effectiveness research, health services and outcomes research, economic evaluation, modeling, and health data a d decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, clinical guideline development, public health policies and patient guidance. His research focuses on cancer, infectious disease, cardiovascular disease, neurological disorders, and others.
He has been leading projects/work packages in several EU FP7, H2020 and Horizon Europe projects (eg, ELSA-GEN, BiomarCaRE, MedTecHTA, DEXHELPP, EUthyroid, FORECEE, MDS-RIGHT, RECETAS, CORE-MD, EUREGIO-EFH, CIDS, OnCoVID, 4D PICTURE, CATALYSE). He teaches HTA, health economics, modeling, epidemiology, causal inference and target trial emulation, and data and decision science for academia, industry, and health authorities in Europe, North and South America, and Asia. He directs the Continuing Education Program on Health Technology Assessment & Decision Sciences (htads.org).
He has served as member of the ISPOR Directors Board and as president of the Society for Medical Decision Making (SMDM). He is a leadership member of the ISPOR Personalized/Precision Medicine SIG, a member of the Latin America Consortium Advisory Committee of ISPOR, and co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force. He is a member of the Oncology Advisory Council and the National Committee for Cancer Screening of the Austrian Federal Ministry of Health.
He has authored more than 400 publications (> 30,000 citations, H index > 80), and is editor of the European Journal of Epidemiology. Further information Internet: http://htads.org, umit-tirol.at/dph, hsph.harvard.edu/uwe-siebert, Twitter: @UweSiebert9, LinkedIn: uwe-siebert9.
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Natasha Azzopardi-Muscat, MD, PhD
WHO Europe, Copenhagen, Denmark
Dr Natasha Azzopardi-Muscat, Director of the Division of Health Systems, WHO Regional Office for Europe.
Dr Natasha Azzopardi-Muscat is a medical doctor and a specialist in public health medicine.
Following her qualification as a medical doctor from the University of Malta in 1995, Dr Azzopardi-Muscat worked in various clinical specialties, including maternal and child health, mental health, and primary care. Having successfully completed a Master’s Degree in Health Services Management at the London School of Hygiene and Tropical Medicine and the London School of Economics and Political Science she returned to work at the Ministry of Health in Malta.
Between 2001 and 2013, she occupied various senior positions in the Ministry including Director for EU and International Affairs, Director General for Strategy and Chief Medical Officer.
In 2016 she obtained her PhD from the University of Maastricht having successfully defended her transdisciplinary research on “The Europeanisation of health systems: a small state perspective”. She is the author of several publications in public health and European health policy.
Between 2016 and 2020 she served as the President of the European Public Health Association (EUPHA) where she was actively involved in health advocacy at the European level.
In 2020, Dr Azzopardi-Muscat joined WHO/Europe and was appointed Director of the Division of Country Health Policies and Systems She served continuously in that position until her re-appointment as Director Health Systems in June 2025.
She is an associate professor at the University of Malta and a visiting scholar at the University of Maastricht.
In 2024 she was awarded the Andrija Stampar medal by the Association for Public Health Schools of the European Region (ASPHER) and in 2025 she was awarded an Honorary Fellowship by the Faculty of Public Health of the United Kingdom in recognition of her important contributions to public health.
Dr Azzopardi-Muscat is married to Dr Conrad Azzopardi and has three children Aidan, Sereena and Nathan.
9:30 - 19:00
Exhibit Hall Open
Session Type: General Meeting
9:45 - 10:15
Morning Break: Coffee and Connect (Exhibit Hall)
Session Type: General Meeting
10:15 - 11:15
Beyond the Health Sector: Embedding Labour Market Effects and Productivity in HTA
Session Type: Issue Panel
Topics: Economic Evaluation, Health Technology Assessment, Study Approaches
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue: Although the impact of poor health on labour force participation and macroeconomic outcomes is increasingly recognised, labour market and productivity impacts remain limited in HTA frameworks. While countries like the Netherlands incorporate broader economic impacts selectively, NICE in England continues to focus on health outcomes and health system costs. Yet with increasing policy interest in reducing labour market pressures and supporting return-to-work efforts, there is increasing impetus to account for labour market participation and productivity in HTA. Including these will highlight the broader value of some types of interventions, but raises questions around how to balance these benefits against health outcomes and address equity concerns—especially for patient groups outside working age. A range of methods issues about estimating these effects will also be considered.
Overview: This panel will explore not only whether labour market and productivity effects should be included in HTA, but how to do so in a consistent, transparent, and methodologically rigorous manner. Systematically including macroeconomic outcomes requires clear guidance on modelling techniques, data standards, and institutional governance. It also prompts debate about trade-offs: how to balance one patient group’s health improvement against another group’s productivity gains; how to manage the dissonance between those funding health and those benefiting from improved productivity; and how to avoid selective application that benefits certain technologies or stakeholders disproportionately. After the introduction (10min) the speakers will present and discuss their different viewpoints (30min). Subsequently, the presenters will be interviewed regarding their key messages and will engage in critical discussion on the challenges associated (10min). We close with an interactive debate with the audience (10min). This panel provides valuable insights for advanced researchers, decision makers and pharmaceutical industry stakeholders on how labour market and productivity dimensions could be utilized in HTA frameworks.
Moderator
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Mark Sculpher, PhD
University of York, York, United Kingdom
Speakers
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Malina Müller, BA, MA, PhD
WifOR Institute, Darmstadt, Germany
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Simon Walker
University of York, York, United Kingdom
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Dan Ollendorf, MPH, PhD
Institute for Clinical & Economic Review, Boston, MA, United States
Data from Early Access Programs: Essential HTA Evidence or Misaligned Value Tool?
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment, Real World Data & Information Systems
Track: Patient-Centered Evidence
Level: Introductory
Available On-Demand: Digital Conference Pass
ISSUE: While Early Access Programs (EAPs) remain focused on providing compassionate access for patients with serious conditions lacking therapeutic alternatives, price and reimbursement stakeholders increasingly debate whether EAPs should generate real-world evidence for HTA evaluations. This dual-purpose expectation creates fundamental tensions: programs designed for urgent medical access lack the rigorous data collection infrastructure of clinical trials, potentially yielding evidence that HTA bodies may consider insufficient for reimbursement decisions. As EU-HTA standardizes evidence requirements, the question becomes whether EAPs can legitimately support pricing and reimbursement decisions without compromising their primary humanitarian mission.
OVERVIEW: Kieron Lewis will provide a 10-minute overview examining how different EAP implementation models impact pricing and reimbursement strategies. While EAP data may inform EU-HTA PICO determinations and contextualize patient populations, manufacturers risk significant financial exposure when funding programs that regulators may later restrict. Varying reimbursement models across European jurisdictions create further complexity and may lead to inequitable access patterns. Mandatory safety monitoring adds complexity to programs already struggling to balance compassionate access with data quality. The panel will explore contrasting perspectives: whether EAPs should maintain their primary focus on compassionate access with minimal data requirements beyond safety monitoring, or evolve to incorporate structured data collection that could support HTA evaluations. Discussion will address the practical challenges manufacturers face in balancing patient access objectives with increasing pressure to generate reimbursement-grade evidence, while managing associated financial risks. Following 35 minutes of panel debate, 15 minutes of audience discussion will explore implementation challenges and ethical considerations. Key stakeholders include regulatory agencies, HTA bodies, pharmaceutical companies, ethics committees, and policymakers navigating patient access versus evidence requirements.
Moderator
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Kieron D Lewis, MASc
Clinigen Healthcare Limited, Bedford, United Kingdom
Kieron joined the Clinigen Group in 2014 and is responsible for delivering consulting services to Biotech and Pharmaceutical companies. Kieron has a vast amount of experience in planning and setting up Pre-Approval/Managed/Expanded Access, International Pharmacy and Named Patient Supply Programs. Kieron specializes in providing strategic and operational advice to companies on pre-launch development, Real World Data collection, market access strategies, and the implementation of specialist access programs for unlicensed medicines. Before joining Clinigen, Kieron spent over 20 years within the pharmaceutical industry fulfilling a range of senior executive roles contributing and leading both global and European launch plans. Within this time, Kieron has led multiple global pre-launches and launch activities for treatments addressing areas of high unmet medical need like oncology, rare disease and neurological treatments. Kieron now focuses his energies on working alongside pharmaceutical and biotechnology companies around the world to consider their strategy for pre-approval access and the development of global programs that contribute to strategic plans and allow patients to gain access to treatments that would otherwise be unavailable within their respective countries.
Speakers
The ECOnomiC Valuation of the Health Impacts of Climate Action (or inaction): The ECO-CHICA Guidelines
Session Type: Other Breakout Session
Topics: Methodological & Statistical Research, Economic Evaluation
Level: Intermediate
Available On-Demand: Digital Conference Pass
PURPOSE. To report on a major guideline initiative to harmonize methods across economic evaluation subdisciplines of environmental and health economics.
DESCRIPTION. The ECO-CHICA project team is a multi-institutional and trans-disciplinary research consortium that set out to develop guidelines for the valuation of health co-benefits of climate actions(/inactions) and associated economic evaluation methods that would be consistent across health and environmental economics sub-disciplines. Nearly 100 economists contributed to the Delphi Panel mediated guideline development process that concludes in October 2025.
This break-out session would be the first major international conference where the results would be reported. The session would cover the methods of guideline development, the agreed guidelines and a discussion of the remaining contentious issues.
Moderator
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Andrew Briggs, DPhil
London School of Hygiene & Tropical Medicine, London, United Kingdom
Speakers
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Mireia Jofre-Bonet, PhD
Office of Health Economics, London, United Kingdom
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Francis Ruiz
London School of Hygiene and Tropical Medicine (LSHTM), London, United Kingdom
I am a Senior Policy Advisor at the London School of Hygiene and Tropical Medicine. Since 2001, my work has focused on health economics and Health Technology Assessment (and related disciplines), with over 10 years experience working at a national government agency in the UK (NICE) responsible for coverage decisions and providing guidance to the British health care system.
My work since 2010 has focused on supporting the institutionalization of evidence informed priority setting in low-to-middle income countries (LMICs), mostly as part of the International Decision Support Initiative (iDSI) which has received funding from the Bill and Melinda Gates Foundation (BMGF) since 2013. My activities have centred on the coordination and delivery of technical assistance activities with government partners in LMIC settings. While I have most recently been supporting capacity strengthening and strategic development of HTA in Sub-Saharan Africa, particularly in Ghana, Rwanda and Nigeria, I have also worked in South-East Asia (China and Philippines) in helping build institutional structures for evidence-informed priority setting. I have also supported the development of global public goods (knowledge products) drawing on these experiences.
My ongoing research work includes the appropriate consideration of environmental impacts and climate change within an evidence informed priority setting process such as HTA, including the identification of relevant methods for the conduct of economic evaluation and the valuation of health co-benefits. I am also a co-investigator on a project related to the design and implementation of health benefit package design processes in Rwanda and Kenya.
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Shouro Dasgupta, PhD
CMCC, Venice, Italy
Patient Centric Approaches when Using Digital Health Technology in Drug Development
Session Type: Issue Panel
Topics: Clinical Outcomes, Medical Technologies, Patient-Centered Research
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
Digital health Technology (DHTs) are being used more frequently to generate patient experience data and measure outcomes in drug development. Applying patient focused approaches when using DHT derived Clinical Outcome Assessments (COA) requires additional considerations to ensure these measures reflect the symptoms and treatment impacts that matter to patients. In addition, participant input into the DHT selection process is essential and there is likely to be a requirement to demonstrate the useability of the DHT in the given context of use should the digital measures be used as part of health authority engagements. Both are essential when it comes to ensuring the correct anchors are selected, and that sufficient data is collected to support the determination of meaningful change thresholds and the endpoint analysis plan. Considering the perspective of patient, industry and health authorities, this panel will review the challenges when designing patient centric trials that utilize DHT’s, and share experiences in incorporating patient insight to ensure that both the endpoints and DHT selected adequately reflect the voice and experience of the patient.
Moderator
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Sarah Ernst, PhD
Sanofi, Boston, United States
Speakers
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Marie Mc Carthy, MBA
Novartis, Dublin, Ireland
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Christine Guo, PhD
Ametris, Pensacola, FL, United States
Preferences in Frail and Older Patient Populations
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session examines health assessment, preferences, and outcomes in older populations. Presentations include a preliminary psychometric evaluation of the Healthcare Improvement Scotland “Think Frailty” tool, analysis of mortality and COPD exacerbations in GOLD E patients in the Netherlands, and mixed-methods evidence from France on whether older adults consistently prefer avoiding nursing homes. The session concludes with a five-country comparison of EQ-5D-5L and PROMIS-10 performance in elderly populations from the EQ-DAPHNIE Project.
Comparative Performance of the EQ-5D-5L and PROMIS-10 in the Elderly: Data From the EQ-DAPHNIE Project in Five Countries
OBJECTIVES: This study compared the performance of the EQ-5D-5L and PROMIS-10 in measuring health-related quality of life (HRQL) among older adults in five English-speaking countries.
METHODS: Using EQ-DAPHNIE project data, 5403 respondents age ≥65 years were included from Canada (n=1312), United States (US) (n=1069), United Kingdom (UK) (n=970), Australia (n=1224), and New Zealand (n=828), with overall and age-subgroup analyses conducted for each country. Spearman correlations assessed convergent and divergent validity, and known-groups analysis examined discriminative validity of EQ-5D-5L index (Canadian value set), and PROMIS-10 Global Physical Health (GPH) and mental health (GMH) T-scores.
RESULTS: Respondents averaged 72.6 years and 48.2% were female across the five countries. Canada reported the lowest EQ-5D-5L index score (mean 0.817; SD 0.145) and PROMIS-10 GPH (47.0; 4.5) and GMH (49.3; 4.9) T-scores, while UK reported the highest scores (EQ-5D-5L 0.839; 0.154; PROMIS-10 GPH 48.5; 4.6). New Zealand reported the highest PROMIS-10 GMH T-score (51.5; 4.0). Convergent and divergent validity were demonstrated across all five countries: EQ-5D-5L mobility and pain/discomfort dimensions had moderate/strong correlations (0.45-0.82) with PROMIS-10 physical health items and weak correlations (0.15-0.39) with PROMIS-10 mental health items; while EQ-5D-5L anxiety/depression dimension had strong correlations (0.55-0.70) with PROMIS-10 mental health items and weak correlations (0.23-0.45) with PROMIS-10 physical health items. Discriminative ability of the EQ-5D-5L index, PROMIS-10 GPH and GMH T-scores were strongest amongst those with sleep and mental health disorders, including anxiety and depression (effect sizes 0.52-1.33), and weakest in diabetes, hypertension, and skin disease (0.06-0.51). The index and GPH-T also had strong discriminative ability for musculoskeletal disorders. Both measures’ performance varied across the five countries for heart disease, obesity, and osteoporosis.
CONCLUSIONS: Both EQ-5D-5L and PROMIS-10 effectively capture HRQL in older adults across five English-speaking countries. Despite their acceptable discriminative ability in some conditions, performance was relatively poor in common diseases including diabetes, hypertension, and skin disease.
Assessment of Mortality and COPD Exacerbations in GOLD E COPD Patients in the Netherlands
OBJECTIVES: To estimate the incidence of mortality in the overall COPD population and in those with Global Initiative for Chronic Obstructive Lung Disease Category E (GOLD-E) COPD within real-world settings in the Netherlands.
METHODS: In this longitudinal observational study, COPD patients (≥40 years) with primary care, outpatient pharmacy, hospital and ambulatory visits data were identified from the PHARMO Data Network (2017-2022; index event was defined as first FEV1 measurement at/after COPD diagnosis). Patients were required to have ≥1 COPD encounter and ≥12 months of continuous data during pre-index. GOLD-E patients were defined as those with ≥2 moderate exacerbations/≥1 COPD-related hospitalisation during ≥12 months pre-index. The cumulative incidence of mortality was estimated from index until end of follow-up using Kaplan-Meier analysis across three cohorts. Cohort 1: overall COPD patients; Cohort 2: GOLD-E patients who were former smokers on dual (ICS/LABA or LABA/LAMA) or triple (ICS/LABA/LAMA) therapy; and Cohort 3: GOLD-E patients with moderate-to-severe disease (GOLD 2/3).
RESULTS: The study identified 12,446 COPD patients (cohort 1), of which, 260 and 625 were included in cohort 2 and cohort 3, respectively. COPD patients in cohort 1 were elderly (median age, 69 years; 55% male), with 36.6% currently smoking and 39.3% having a cardiovascular co-morbidity. Patients in cohort 2 and 3 were slightly older (median age: 73 and 71 years, respectively), with a higher proportion having cardiovascular comorbidities (46.9% and 45.9%, respectively). The cumulative incidence of mortality at 5 years was 16.0% (95%CI: 15.2%-16.8%), 23.9% (95%CI: 17.6%-29.4%) and 23.5% (95%CI: 19.7%-27.8%) for cohort 1, 2 and 3, respectively.
CONCLUSIONS: Our findings indicate that COPD patients in the Netherlands had high cardiovascular comorbidity burden and mortality risk, particularly among those meeting GOLD-E. We further estimated mortality in approximately one-fourth of patients in 5 years in GOLD-E patients who were former smokers or had moderate-to-severe disease.
Do Older People Always Prefer Avoiding Nursing Homes? Evidence in France From a Mixed-Methods Approach
OBJECTIVES: As populations age, long-term care policies are increasingly challenged to balance individual preferences with budget constraints. The prevailing “aging in place” policy in France has prioritized home care, assuming that older people prefer avoiding nursing home stays in most circumstances. However, little is known about older people’s preferences towards long-term care options. This study investigates, for the first time in France, the preferences of elderly individuals when choosing between home care and nursing home care.
METHODS: Using a mixed-method approach that combines qualitative interviews and a discrete choice experiment (DCE), we explore how individual socioeconomic characteristics, care-related experiences, and specific features of nursing homes shape these preferences. The DCE employed a two-stage methodology: first, respondents evaluated hypothetical nursing home packages based on four attributes (quality of equipment and facilities, quality of professional care, geographical proximity, and out-of-pocket expenses); then, they chose between their preferred nursing home option and remaining at home. Our analysis, based on responses from 2,886 individuals aged over 60, reveals substantial heterogeneity in preferences.
RESULTS: While a majority consistently favored staying at home, in a hypothetical situation of heavy dependence, a significant proportion shifted their choice in response to improved nursing home characteristics, particularly enhancements in the quality of care and facility atmosphere. Additional factors, such as higher income, caregiving experience, and expectations of longevity, were associated with a greater propensity to choose institutional care.
CONCLUSIONS: These findings suggest that targeted investments in nursing home quality and affordability could better align long-term care services with the diverse needs of the elderly, complementing existing home care policies.
The Healthcare Improvement Scotland Think Frailty Tool: A Preliminary Psychometric Evaluation
OBJECTIVES: Identification of frailty is crucial to appropriately tailor patient care. Most frailty screening tools are difficult to use in unscheduled hospital settings. The Healthcare Improvement Scotland (HIS) ‘think frailty’ tool was developed to easily screen for frailty in frontline healthcare. We evaluated the psychometric properties of the HIS ‘think frailty’ tool.
METHODS: We conducted a prospective observational cohort study. Consecutive adults aged ≥65 who presented as unscheduled admissions to hospital were recruited. Recruitment occurred in 3 phases between 2017-2023. Participants completed the HIS ‘think frailty’ tool and the Rockwood Clinical Frailty Scale (CFS). We assessed HIS 'think frailty' tool's concurrent validity (Pearson’s correlations), overall discrimination (area-under-curve; AUC), intra-rater reliability (adjusted Kappa), inter-rater reliability between specialist and non-specialist assessors (adjusted Kappa), feasibility (percentage of missing data within each HIS ‘think frailty’ domain), and predictive validity for 30-day mortality and duration of hospital stay (Regression). A Maximum likelihood factor analysis was conducted to investigate the underlying factor structure.
RESULTS: A total of 2447 older adults were recruited. HIS ‘think frailty’ scores were significantly correlated with CFS (0.78,p<0.001) and discrimination was excellent (AUC:0.934,95%CI=0.921-0.946). Intra-rater agreement was substantial (Kappa=0.74,95%:0.61-0.86), as was Interrater agreement between a specialist and non-specialist assessor Kappa=(0.83,95%CI:0.68-0.97). All domains of HIS ‘think frailty’ had <1.5% missing data. HIS ‘think frailty’ was significantly associated with an increased risk of mortality up to 30 days (OR:1.49,95%CI=1.32-1.69) and likelihood of being in hospital at 30 days (OR:1.727,95%CI=1.52-1.96). Factor analysis suggested correlations between the items in the HIS ‘think frailty’ were within an acceptable range. The The Kaiser-Meyer-Olkin (KMO) test was acceptable (0.69) and Bartletts test of Sphericity was highly significant (<0.001). The scree plot indicated a single underlying factor accounting for 43.4% of the variance.
CONCLUSIONS: The HIS ‘Think Frailty’ tool has good psychometric properties for ‘font door’ frailty screening and can be used by non-specialists.
Advancing Statistical Methods in Survival Analysis
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session explores cutting-edge statistical approaches for analyzing time-to-event data in clinical research. Topics include handling competing risks, methods for censored data, optimizing survival models, and predicting long-term treatment effects in specific disease contexts.
Optimizing the Use of Excess Hazard Models in Survival Analysis: Considering Disease Severity and Follow-up Maturity
OBJECTIVES: Survival extrapolation is a critical component of Health Technology Assessment (HTA) and often conducted using standard parametric models, which do not account for background population mortality. This can lead to implausible hazard projections falling below age- and sex-matched mortality rates. In economic models, this is sometimes corrected by using artificial “clamping”, which introduces artificial discontinuities in the hazard. Excess hazard (EH) models offer an alternative approach by explicitly incorporating background mortality into survival projections. This study explores when EH models are most appropriate and how much follow-up is needed for reliable results.
METHODS: Several case studies were selected to represent diseases of varying severity, defined by differences in expected overall survival (OS). Standard parametric and additive EH models were applied to each. Published Kaplan-Meier OS curves were digitised, and individual patient data (IPD) were reconstructed. The impact of limited follow-up was assessed by artificially truncating survival data to simulate shorter observation periods.
RESULTS: In high-severity diseases with short survival, EH models produced results comparable to standard parametric models, as disease-specific hazards dominated background mortality. In contrast, EH models often failed to converge or yielded unstable estimates in low-severity settings where disease-related hazards were low. In cases where background mortality contributed notably to the overall hazards, EH models generated narrower and more stable extrapolations than standard models, particularly when at least 24 months of follow-up data were available. However, when follow-up was limited to 6 and 12 months, model variability and uncertainty increased markedly.
CONCLUSIONS: EH models are most valuable in settings where background mortality meaningfully contributes to the overall hazard. However, its use requires sufficient follow-up data to ensure model stability and accuracy. This work highlights specific contexts where EH modelling may provide benefits within HTA, despite limitations associated with reconstructed IPD.
Survival Time Adjusted Indirect Comparisons: A New Methodology for Comparing Between Phases of Treatment in Early Cancer
OBJECTIVES: In early cancer, new treatments may be administered prior to surgery (neoadjuvant), after surgery (adjuvant) or across both settings (perioperative). Without head-to-head studies, the comparative efficacy of these strategies must be estimated via indirect treatment comparison (ITC). Using a case study, we highlight the challenges of using standard ITC methodologies in comparing perioperative with adjuvant strategies and propose an alternative methodology.
METHODS: Following guidelines, we consider the feasibility of performing anchored or unanchored ITCs in a case study involving a (study 1) ‘perioperative’ study of drug A plus neoadjuvant therapy versus neoadjuvant therapy followed by best supportive care (BSC) and (study 2) an ‘adjuvant’ study of drug B versus BSC. An alternative survival-time adjusted approach using a counterfactual survival time (CST) methodology is proposed and its benefits and limitations are described.
RESULTS: Differences in randomisation times, trial eligibility, and control treatments are expected to invalidate the exchangeability assumption of standard anchored ITCs. Unanchored ITCs typically require overlap in populations limiting comparisons to the adjuvant phase and excluding relevant neoadjuvant outcomes. Using CST methodology, the ‘adjuvant’ survival times of eligible patients in the control arm of the ‘perioperative’ study are adjusted for the effects of adjuvant drug B using acceleration factors derived from the ‘adjuvant’ study. This yields a counterfactual arm representing outcomes for neoadjuvant therapy followed by ‘adjuvant’ drug B. The effects of perioperative drug A versus neoadjuvant therapy followed by drug B are estimated by comparing the ‘perioperative’ and counterfactual arms. Randomisation in the ‘perioperative’ study supports baseline comparability between arms, whilst randomisation in the adjuvant study supports estimation of the drug B effect. Key limitations include the assumption of exchangeable treatment effects across studies.
CONCLUSIONS: CST methods provide a promising approach to comparing between phases of treatment, where the assumptions underpinning standard ITC methods are unlikely to hold.
Approximating Competing Risk Events From Published Kaplan-Meier Curves Using Simultaneity-Based Event Assignment Methods
OBJECTIVES: State-transition modelling (STM) offers an alternative to partitioned-survival modelling, employing structural relationships between endpoints rather than assuming independence. However, STM typically requires individual-patient-data (IPD), which are often unavailable. When relying on published aggregate data (e.g., progression-free survival [PFS] and overall survival [OS]), existing literature-based methods for approximating competing-risk transitions, involve detecting event simultaneity from published Kaplan-Meier (KM) survival curves. This study evaluates the performance of simultaneity-based event assignment methods to estimate pre-progression death rates using simulated trial data.
METHODS: Censored IPD for progression and death events were simulated for a hypothetical oncology trial. KM curves and at-risk tables were generated at 3-month intervals. Pseudo-IPD for PFS and OS were derived from hand-digitized XY coordinates of the KM curves, using the Guyot algorithm. Pre-progression deaths were assigned by applying predefined error margins, which defined the time window within which PFS and OS events were considered simultaneous. Estimated pre-progression death counts, and cumulative hazards were compared to the simulated data across different error margins ranging up to 0.1 months.
RESULTS: In the simulated trial data (N=430, median follow-up ~33 months), 5.6% of the patients experienced pre-progression death and 53% experienced disease progression. Narrow error margins (e.g., 0.01 months) misclassified ~75% of pre-progression deaths as progression events. Wider margins (e.g., 0.1 months) overestimated pre-progression deaths by more than 100%. A similar trend of event-misclassification was observed in other simulated datasets with differing error-margins.
CONCLUSIONS: Simultaneity-based event assignment methods appear to have notable limitations when approximating competing-risk events from published KM survival curves. Their performance is highly sensitive to competing-event prevalence, data-extraction precision, and chosen simultaneity error-margins. Misclassification of competing events can potentially distort health-economic analyses employing STM, leading to inaccurate projections and unreliable decision-making in policy or clinical practice. Further research is required to assess the performance of alternative multi-state modelling approaches for approximating competing-risk events.
Predicting Treatment Effect on the Loss of Ability to Rise From Floor in Duchenne Muscular Dystrophy Using Individual Trajectories: An Application of Joint Modelling in FOR-DMD
OBJECTIVES: This analysis aimed to develop a model connecting longitudinal time to rise from floor (TTR) trajectories with time-to-event (TTE) data for loss of ability to rise from the floor (LoR) to characterize Duchenne Muscular Dystrophy (DMD) progression and predict future treatment effects. The FOR-DMD trial (NCT01603407) included 196 boys with DMD aged 4-7, randomized to daily prednisone, daily deflazacort, or intermittent prednisone. TTR was collected at baseline, 3, 6, and every 6 months until 60 months.
METHODS: A Bayesian joint model was developed, combining a longitudinal sub-model for TTR trajectories and a TTE sub-model for LoR. The longitudinal sub-model, a linear mixed-effects model, incorporated corticosteroid regimen-specific slopes and a quadratic function for log-transformed TTR over time. It included random effects for intercept and slope and was adjusted for baseline age, North Star Ambulatory Assessment (NSAA) total score, and corticosteroid regimen. The TTE sub-model was a parametric proportional hazards regression model that modeled time from randomization assuming a Weibull baseline hazard. It was adjusted for the same covariates, and longitudinal trajectories were incorporated using a current value association structure. The joint model was estimated using the rstanarm R package and JAGS.
RESULTS: The joint model estimated the association between TTR trajectory and LoR (4.7; 95% CrI: 3.4-6.4). A 16% annual decrease in TTR for daily vs. intermittent corticosteroids resulted in a LoR hazard ratio of 0.43 (95% CrI: 0.32, 0.55). These results were robust to various model adjustments, including removing covariates, adding interactions, using cubic splines for baseline hazard, and altering priors.
CONCLUSIONS: A joint model, developed using the FOR-DMD dataset, provides a robust framework for predicting treatment effects on the LoR in DMD patients, utilizing individual TTR trajectories and other covariates. This approach can predict the effect of new treatments on LoR based on observed TTR trajectories.
Outcome Assessment in Rare Diseases: Is JCA Breaking the Silos or Perpetuating the Chaos?
Session Type: Issue Panel
Topics: Patient-Centered Research, Health Technology Assessment, Health Policy & Regulatory
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE:
With the Joint Clinical Assessment (JCA) set to include orphan drugs from 2028, rare disease therapies will soon face a new evidence evaluation landscape. This poses particular challenges for rare diseases, where limited data and symptom heterogeneity make it difficult to select and measure outcomes that truly reflect patient experience. A lack of alignment among regulators, HTA bodies, and patient organisations risks creating further barriers to access. With differing priorities and evidence requirements, developers must plan separate outcome strategies, creating inefficiencies and potentially delaying patient access to new therapies. These challenges are especially acute for smaller biotech and non-profit developers with limited capacity to respond quickly to multiple requirements across member states. This session will debate whether harmonising outcome assessment strategies are feasible or appropriate for rare diseases across decision-makers with fundamentally different goals, and if mechanisms like parallel scientific advice or JCA guidance can realistically deliver better alignment.
OVERVIEW:
Laura Sawyer will introduce the topic and key issues. Milad Karimi will argue for better alignment to reduce inefficiencies and share challenges sponsors face in creating integrated plans for patient-experience data across stakeholders (10 minutes). Steffen Thirstrup will argue that full alignment may be unrealistic given differing remits, drawing on EMA and joint advice experience to explore where collaboration and convergence is possible (10 minutes). Olivier Chassany will support harmonisation and challenge inconsistencies of outcomes requirements (e.g. preference for generic vs disease-specific tools, global scores vs important subscales, and rigid endpoint hierarchies) across European HTA bodies, including within JCA (10 minutes). The final 20 minutes will be dedicated to panel discussion and invite audience input on improving outcomes alignment and anticipating downstream challenges (e.g. national HTA use and reimbursement). This session will benefit stakeholders involved in defining or assessing outcomes that support access decisions in rare diseases.
Moderator
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Laura Sawyer, BA, MSc
Symmetron, London, United Kingdom
Speakers
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Milad Karimi
BioMarin, London, United Kingdom
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Steffen Thirstrup, PhD, MD
Amsterdam, Netherlands
Steffen Thirstrup is a medical doctor and board-certified specialist in clinical pharmacology and therapeutics. He holds a PhD in pharmacology and has a long background in clinical internal medicine with special emphasis on adult respiratory medicine. Additionally, Dr. Thirstrup was appointed adjunct professor in pharmacotherapy at the Faculty of Health Sciences, University of Copenhagen, in 2012.From 2004-09 Steffen Thirstrup worked at Danish Medicines Agency first as the Danish member of CHMP at the European Medicines Agency (EMA) for five years including 10 months as joint CHMP- and CAT-member, followed by a short period as head of Danish Institute for Rational Pharmacotherapy dealing with HTA and best practice guidelines for primary care. In 2011 Prof. Thirstrup rejoined the licensing division at the Danish Medicines Agency acting as Head of Division for Medicines Assessment and Clinical Trials. During this period Prof Thirstrup co-chaired the European Commission’s working group on market access for biosimilars medicinal products and acted as key scientific contact for the managing entity of the IMI beneficiaries for the PROTECT collaboration (Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium).
In March 2013, Prof Thirstrup joined the pharmaceutical consultancy company NDA Group AB as a full-time medical advisor on NDA’s regulatory advisory board. In April 2014 Prof Thirstrup was appointed as director for the Regulatory Advisory Board at NDA Regulatory Services Ltd.
Since June 2022 Prof Thirstrup has been the Chief Medical Officer at the European Medicines Agency, Amsterdam, The Netherlands
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Olivier Chassany, PhD, MD
Assistance Publique–Hôpitaux de Paris (AP-HP), Paris, France
Professor of Therapeutics (Health Economics Clinical Trial Unit, AP-HP Paris hospitals, France), clinician, specialist in gastroenterology, with a long experience in developing Patient-Reported Outcomes (PRO) questionnaires. Involved for more than 20 years in dossiers expertise for EMA and French Drug Agency and over 32 years in Ethics Committees. Co-author of the EMA Reflection Paper on Health-Related Quality of Life. Involved in HTA dossiers expertise for pharma/CRO. Teaching Therapeutics and Methodology of clinical trials at Université Paris Cité. Past chair of the ISPOR SIG Clinical Outcomes Assessment (COA).
Navigating the Global Pricing Policy Landscape and Leveraging AI for Strategic Insights
Session Type: Workshop
Topics: Health Policy & Regulatory
Level: Intermediate
Available On-Demand: Digital Conference Pass
PURPOSE:
Navigating the complexities of global pharmaceutical pricing requires a robust understanding of evolving policies and innovative approaches. This workshop aims to provide participants with actionable insights into global pricing challenges, with an emphasis on European and cross-border implications. Attendees will explore how to leverage early scenario modeling and AI-driven tools to strengthen preparedness for global launches, mitigate risks, and optimize pricing strategies. The session also fosters collaboration through a hands-on simulation, allowing participants to apply key learnings to real-world global pricing and policy scenarios.
DESCRIPTION:
This workshop will provide participants with practical knowledge and strategies to address the challenges of global pricing policy shifts. Dr. David Ringger will begin the session with an overview of the global pricing policy landscape, focusing on the interplay between Europe, the US, and other regions, as well as the cross-border ripple effects of policy changes.
Neil Grubert will provide an overview of how current and proposed policy changes in Europe and the US are influencing international reference pricing and cross-border strategies (10 minutes).
Casper Paardekooper will discuss how early scenario modeling strengthens preparedness for global product launches, identifies risks, and ensures alignment across global teams (10 minutes).
The workshop will then explore the possibilities of AI in global pricing strategy. Dr. Leanna Baker Williams will demonstrate how AI-driven insights can anticipate policy-driven shifts, optimize pricing benchmarks, and enhance decision-making across regions (15 minutes).
The session concludes with an interactive global simulation. Participants will work collaboratively on a real-world-inspired case study to address cross-border pricing challenges, navigate policy changes, and apply AI-driven insights.
This interactive and informative workshop is ideal for professionals involved in pricing, market access, health policy, and strategy, particularly those navigating cross-border challenges in a globalized pharmaceutical industry.
Moderator
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David Ringger, PhD
Cencora, Bern, Switzerland
Speakers
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Neil Grubert
Neil Grubert Consulting, Basildon, United Kingdom
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Casper Paardekooper, MSc
Vintura, Utrecht, Netherlands
Casper Paardekooper is Partner and Board Member at strategy consultancy Vintura, part of Cencora, which focuses on transforming healthcare, making it more accessible and beneficial for all. In Vintura he is head of the Life Sciences practice, and he also leads Vintura's Value, Access & Policy Center of Excellence. In that capacity, he focuses on access policy, market access strategy, value demonstration and value-based healthcare. He is also part of Cencora’s EU HTA Center of Excellence, where he particularly focuses on organizational readiness of his clients. Casper is a trusted advisor to many large biopharma companies and brings more than 15 years of strategy consultancy in life sciences and healthcare.
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Leanna Baker Williams, PharmD
Cencora, Fort Johnson, LA, United States
Navigating a Maze of Indirect Treatment Comparisons: How to Ensure we Have Selected the Ideal Method for a Decision-Problem?
Session Type: Issue Panel
Topics: Health Technology Assessment, Methodological & Statistical Research, Clinical Outcomes
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue
The proliferation of complex therapies, numerous comparators and evolving evidence requirements have made indirect treatment comparisons (ITCs) indispensable in HTA. Since ISPOR’s 2014 Task Force guidance, various new statistical techniques have been developed to help deal with reconciling heterogeneous data to HTA evidentiary standards. However, methodological choices remain contentious, and their implementation can sometimes introduce almost as much uncertainty as it resolves.
Overview
This panel will debate methodological rigor, transparency, and decision-making frameworks for ITCs in HTA.
Dr. Hugo Pedder, University of Bristol, will present a pragmatic flow diagram to guide model selection in ITC, based on data availability and decision context, with clear references to alignment with different HTA body requirements.
Dr Lara Wolfson, MSD, will present the complexity and challenges of balancing methodological ideals as statistical leaders in pharmaceutical industry versus diverse and rigorous requirements from HTA agencies globally to ensure transparent, consistent communication of the relative value of the health technology being assessed to multiple stakeholders.
Dr Nicole Mittman, CDA-AMC, will present the HTA perspective on the evidence and methodology expectations to support comparison to other competing health technologies and for robust HTA decision-making, elucidating key trigger points when/how HTA agencies may update their guidance with new methods requirements.
The panel will be moderated by Professor Kate Ren, University of Sheffield.
The session will allocate 15 minutes per speaker, followed by 20 minutes of moderated debate on the following topics:
1. Whether current guidance adequately addresses emerging methods in ITC
2. The importance validating novel ITC approaches and assessing their robustness
3. The urgent need for updated guidance to harmonize standards across stakeholders
Moderator
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Kate Ren, PhD
ConnectHEOR|University of Sheffield, London|Sheffield, United Kingdom
Speakers
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Hugo Pedder
University of Bristol, Bristol, United Kingdom
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Lara Wolfson, MS, PhD
MSD, Zurich Flughafen, Switzerland
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Nicole Mittmann, MSc, PhD
Canada's Drug Agency, Toronto, ON, Canada
In her Chief Scientist role, Dr. Mittmann is responsible for ensuring that CDA (a.ka. CADTH) actively learns, ensures rigour and quality, mobilizes evidence, and links science to strategy. In her Scientific Evidence, Methodologies and Resources role, Nicole leads CDA’s shared science groups, including the Science and Methods, Health Economics, Research Information Services, Publishing, Early Scientific Advice and Real-World Evidence teams.
In her academic capacity, Dr. Mittmann holds an MSc and PhD in pharmacology from the University of Toronto. She holds a faculty position as an assistant professor at the University of Toronto in the Department of Pharmacology & Toxicology; and is cross-appointed to the Institute for Health Policy, Management and Evaluation. She is also an associate scientist at Sunnybrook Health Sciences Centre in Toronto, Canada. Dr. Mittmann has conducted and collaborated on notable research in the areas of economic evaluations, outcomes research, and drug/patient safety. Research methodologies include the examination of large databases, economic methodologies, and decision analysis.
She likes to link, leverage and liberate data and evidence.
Building Multi-Agent AI Systems to Reduce Subject Matter Expert Burden in Health Economics Research
Session Type: Workshop
Topics: Methodological & Statistical Research
Level: Intermediate
Available On-Demand: Digital Conference Pass
PURPOSE: This workshop will teach participants when and where to consider developing multi-agent AI systems in health economics and outcomes research (HEOR), what types of large language model (LLM) shortcomings these systems can address, and how to design multi-agent system architectures with clear understanding of objectives and design features. Participants will gain practical skills in transforming single-agent applications into multi-agent frameworks to improve quality while reducing subject matter expert burden, enabling immediate application to their own HEOR projects.
DESCRIPTION: Single-agent AI systems in HEOR often require extensive human oversight due to quality and accuracy concerns, limiting practical adoption despite their promise. Multi-agent systems offer a solution by enabling AI-to-AI validation and correction before human review, significantly improving output reliability while reducing review time and costs. Dr. Caro will introduce the rapid integration of AI systems in HEOR activities and current quality challenges, establishing the rationale for the need to maintain research standards while reducing expert oversight burden (10 min). Mr. Deniz will present multi-agent system fundamentals, including when these systems make sense, key design considerations, and implementation strategies for HEOR applications (15 min). Dr. Reddy will demonstrate a real-world HEOR application currently using a single-agent structure, explaining its objectives, workflow, and limitations (15 min). The workshop will conclude with an interactive group exercise where participants will collectively transform the presented single-agent system into a multi-agent architecture, applying the design principles discussed earlier. As a group, attendees will discuss specific validation checkpoints, define agent roles and responsibilities, interaction protocols, and develop quality assurance mechanisms (20 min). This hands-on approach ensures participants leave with practical experience in multi-agent system design and actionable frameworks they can implement in their own HEOR applications.
Moderator
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J. Jaime Caro, MD
Thermo Fisher Scientific, Lincoln, MA, United States
Speakers
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Brian Reddy, BA, MSc, PhD
Pfizer, Dublin, Ireland
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Baris Deniz, MSc
AIde Solutions, Chapel Hill, NC, United States
Baris Deniz is a seasoned expert in Health Economics and Outcomes Research (HEOR) and market access, with over 20 years of experience. His expertise spans integrated evidence strategies, health economic evaluations, evidence synthesis, and technology assessments. Baris has a deep understanding of the strategic role HEOR plays in bridging regulatory approvals with patient access, while generating robust evidence to demonstrate the value of medical interventions. Recently, his work has been in exploring innovative technologies and their applications in the HEOR domain to drive more effective outcomes and data-driven decision-making. He is the founder of AIde Solutions LLC, which focuses on leveraging GenAI in HEOR and scientific research.
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
Poster Session 3
Session Type: Research Posters
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
11:30 - 12:00
EU Pharmaceutical Package: Where are we, and Where are we Going?
Session Type: Fast Facts
Topics: Health Policy & Regulatory
Level: Introductory
As trilogue negotiations on the EU pharmaceutical package progress, stakeholders across the healthcare ecosystem are watching closely. This revision aims to reshape incentives, regulatory pathways, and access mechanisms across Europe—with direct implications for payers, HTA bodies, industry, and patients. In this fast facts session, we’ll unpack where the legislative process currently stands and the key issues shaping the final compromise. Areas of focus include: 1) progress and timelines in the trilogue phase; 2) potential impacts on data and market protection; 3) revised incentives for orphan and pediatric medicines; 4) the interplay with EU industrial and competitiveness goals; and 5) potential implications for national-level pricing, reimbursement, and access. With critical decisions ahead, this session offers a concise policy snapshot for understanding where the EU pharma package is headed and why it matters for health economists and payers alike.
Speakers
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Katja Murray
United States
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Tiago Beck
FTI Consulting, Brussels, Belgium
11:30 - 12:15
Research and Modelling Methods Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 3. Posters will be hung from 10:30 - 13:30.
Implementing the European HTA Regulation Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 3. Posters will be hung from 10:30 - 13:30.
11:45 - 12:45
Effectively Advocating for HEOR: Cross-Organization Experiences (A Women in HEOR Forum)
Session Type: Forums
Topics: Organizational Practices
Level: Introductory
Available On-Demand: Digital Conference Pass
Effective communication about the value of HEOR across functional groups within biopharmaceutical or medical device companies is essential to access adequate resources to support high-quality studies. The objective of the forum is to endow attendees with guidance from experienced industry leaders to identify opportunities for high-value HEOR projects, to determine who the key internal stakeholders are, to effectively sell the value proposition, and to measure and document its impact. Dr. Slejko will start the session, providing a high-level overview of the Women in HEOR Initiative and empirical studies documenting the value of HEOR. Dr. Reed will moderate a discussion with two to three ISPOR member panelists who will share positive and negative lessons learned based on their personal experience in the biopharma-device industry. The goal is for attendees to take away strategies to become more effective advocates for HEOR within their organizations to ensure that adequate funding and other resources are allocated toward high-value research. Attendees will participate in live polling about perceived barriers and facilitators to obtaining buy-in from key internal stakeholders for HEOR studies. Attendees will have the opportunity to ask questions of the panelists.
Moderator
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Shelby Reed, RPh, PhD
Duke Clinical Research Institute, Durham, NC, United States
Speakers
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Julia F. Slejko, PhD
University of Maryland School of Pharmacy, Baltimore, MD, United States
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Olivia Wu, MSc, PhD
University of Glasgow, Glasgow, United Kingdom
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Clarissa Cooblall
ISPOR, Lawrenceville, NJ, United States
Competencies That Matter: Building Effective HEOR Capabilities for Medical Devices and Diagnostics
Session Type: Forums
Topics: Medical Technologies, Real World Data & Information Systems
Level: Intermediate
Available On-Demand: Digital Conference Pass
As medical device and diagnostic companies face growing expectations to demonstrate the value of their technologies to regulators, payers, and providers, the role of Health Economics and Outcomes Research (HEOR) has become more critical—and more multifaceted—than ever. In the MedTech space, HEOR must move beyond traditional cost-effectiveness analyses to encompass a broader set of capabilities, including measuring operational impact, quantifying human factors, and innovating methods to generate patient-centered evidence. These evolving demands also call for effective cross-functional collaboration with regulatory, clinical, and market access teams.
This forum will explore how HEOR professionals can build and apply the capabilities needed to succeed in the MedTech sector, using the ISPOR HEOR Competencies Framework as a foundation. Two complementary approaches will guide the discussion: a conceptual review of the framework itself and a practical perspective grounded in project-based experience and organizational needs.
Mitch Higashi (ISPOR) will open the session by reviewing the Competencies Framework and its 13 domains, drawing from his own experience as an HEOR professional and leader to reflect on how these competencies align with—and may need to evolve for—the realities of MedTech. Dr. Wherry, an HEOR leader from a MedTech company, will share examples of how real-world evidence (RWE) has been used to quantify value—such as avoiding prospective studies when RWE supports regulatory or reimbursement decisions, and accelerating patient access through evidence-driven strategies. The moderator will provide concluding remarks and open the floor for questions during the final 10 minutes of the session.
Moderator
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Arturo Cabra, BS, MSc
GE HealthCare, Miami, FL, United States
Speakers
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Mitch Higashi, PhD
ISPOR, Villanova, PA, United States
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Kael Wherry, MS, PhD
Medtronic, Inc., New Brighton, MN, United States
Swimming in the Sea of RWE Guidance: Debating a Path Forward
Session Type: Forums
Topics: Real World Data & Information Systems
Level: Intermediate
Available On-Demand: Digital Conference Pass
Since 2016, there has been a substantial increase in the development of guidance for the generation, reporting, and use of real-world data/evidence (RWD/E) in healthcare decision-making. Several organizations (e.g., IDERHA, Duke Margolis, etc) have undertaken efforts to compile and compare guidances. However, analyzes of guidance are often quickly rendered outdated due to the continual publication of new documents—and gaps in guidance persist despite proliferation on certain topics like transparency and data quality. This session will identify areas of guidance redundancy and debate whether there is a need for additional guidance or if the current landscape is saturated.The session will begin with a brief overview (5 minutes) from the moderator, C. Daniel Mullins of current real-world data and real-world evidence (RWD/E) guidance issued by global stakeholders, presented by the moderator. This will be followed by a structured debate on the necessity of additional RWE guidance. Ashley Jaksa will present the position that further guidance is not required, advocating instead for the harmonization of existing guidance (15 minutes). In contrast, Sandipan Bhattacharjee will argue that the development of additional guidance is essential to support manufacturers in meeting the evolving evidence-generation requirements of regulatory and health technology assessment (HTA) bodies (15 minutes). Laura Pizzi will offer a critical commentary on both perspectives using an ISPOR lens, and engage the audience through a live poll to capture their views on the topic (15 minutes). Dr. Mullins will moderate a Q&A session with the audience (10 minutes).
Moderator
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C. Daniel Mullins, PhD
University of Maryland School of Medicine, Baltimore, MD, United States
C. Daniel Mullins is a Professor at the University of Maryland School of Pharmacy. He is Founder and Executive Director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, a community-academic partnership for patient-driven research. Dr. Mullins has received approximately $25 million in funding as a Principal Investigator from AHRQ, FDA, NCI, NHLBI, NIA, NIMHD, the Patient-Centered Outcomes Research Institute (PCORI) and various patient advocacy organizations and pharmaceutical companies. At the University of Maryland Baltimore (UMB), he received the Dr. Patricia Sokolove Outstanding Mentor Award and the Dr. Martin Luther King Jr. Faculty Diversity Award. He was named Researcher of the Year at UMB and was awarded a University System of Maryland Wilson H. Elkins Professorship. At ISPOR, he has served as Editor-in-Chief of Value in Health since 2010 and received the Marilyn Dix Smith Leadership Award in 2017 and the Avedis Donabedian Lifetime Achievement Award in 2024.
Speakers
Joint Clinical Assessment (JCA) in Practice: Strengthening EU HTA Through CEE Expertise and Patient Perspectives
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment, Organizational Practices
Level: Intermediate
Available On-Demand: Digital Conference Pass
The implementation of the Joint Clinical Assessment (JCA) under the EU HTA Regulation marks a pivotal shift toward more harmonized, transparent, and effective HTA across Europe. This panel brings together national HTA agencies and patient representatives—particularly from Central and Eastern Europe (CEE)—to share lessons learned from early JCA experiences, and to highlight the unique contributions that CEE countries and patient voices can bring to the European HTA landscape. With decades of experience navigating constrained healthcare budgets, CEE HTA bodies have developed pragmatic cost-effectiveness, affordability, and healthcare budget sustainability arrangements. Their insights, coupled with patient advocates' lived experiences of access barriers, can significantly enrich the JCA process—supporting more equitable, inclusive, and implementable assessments across Member States.
Panel Objectives and Themes:
1. First-Hand Implementation Experiences Across Europe
This session will feature perspectives from both larger and smaller HTA agencies in the CEE, including Poland (co-assessor in the first JCA process, and others, focusing on:
? Coordination and submission timelines in early JCA pilots
? Challenges in aligning national and EU-level perspectives
? Capacity building and competence development
? Communication effectiveness with MAHs
? Use of JCA findings in national pricing/reimbursement decisions
2. CEE Contributions and the Value of Diverse Perspectives
This part will explore:
? CEE HTA strengths: cost-effectiveness, practical decision-making, and resource use
? Patient perspectives from CEE: how systemic access barriers shape needs and expectations
? Strategic integration of regional expertise and patient input to enhance methodological consistency and legitimacy of EU-wide assessments
Expected Outcomes:
? Practical insights for optimizing national contributions to the JCA
? Recommendations for enhancing patient engagement across the JCA lifecycle
? Understanding how the inclusion of CEE expertise can support more robust, fair, and actionable EU-level HTA
Moderator
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Maciej Niewada, PhD
ISPOR Poland Chapter, Warszawa, Poland
Speakers
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Anna Kowalczuk, PhD, MSc
Agency for Health Technology Assessment and Tariff System, Warszawa, Poland
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Martin Visnansky, MBA, MSc, PharmD, PhD
University of Veterinary Medicine and Human Pharmacy, Bratislava, Slovakia
12:45 - 13:15
Core Outcome Sets - a Key Enabler of Patient-Centred, System-Wide Efficiencies Linking Clinical Trials, Regulatory Processes, and Reimbursement Decisions?
Session Type: Fast Facts
Topics: Clinical Outcomes, Patient-Centered Research, Health Technology Assessment
Track: Patient-Centered Evidence
Level: Intermediate
Moderator
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Alice M Biggane, BSc, MPH, PhD
Pfizer, Tadworth, United Kingdom
Alice is an outcomes research and innovation lead at Pfizer, where she identifies and bridges HRQoL evidence gaps for HTA. Alice holds a PhD in Applied Health Research, which explored methods for including patients and members of the public in outcome prioritisation in the context of core outcome sets for effectiveness trials and clinical guideline development.
Speakers
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Paula Williamson, BSc, PhD
Liverpool, United Kingdom
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Justin Doan, MPH, MSc, DrPH
Pfizer, Lake Mary, FL, United States
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Nichole Taske, PhD
London, United Kingdom
13:45 - 14:45
Expanding the Evidence Base in Network Meta-Analysis: Leveraging Surrogacy Meta-Analysis and Matching Techniques
Session Type: Workshop
Topics: Methodological & Statistical Research, Health Technology Assessment, Real World Data & Information Systems
Level: Introductory
Available On-Demand: Digital Conference Pass
PURPOSE: This workshop aims to provide participants with a practical understanding of how surrogate endpoint meta-analysis and matching techniques can be used to expand the evidence base in network meta-analyses (NMA). At the end of the session, attendees will learn how these approaches enhance the relevance and robustness of indirect comparisons in health technology assessment (HTA), with direct implications for reimbursement and regulatory decision-making.
DESCRIPTION: Traditional NMA often relies solely on randomised controlled trials (RCTs), which may not fully represent real-world treatment pathways or populations. There are also cases where there may be very limited RCTs evidence. To address evidence gaps, innovative methods like surrogate endpoint meta-analysis and matching of treatment arms techniques can help integrate data from diverse sources, including observational studies and single-arm studies. This workshop explores how these methods improve decision-making in the absence of comprehensive head-to-head evidence.
Raju will chair the session and introduce the topic, outlining the challenges in current evidence synthesis and setting the stage for methodological innovation (5 minutes). Janharpreet will present a summary and application of surrogate endpoint meta-analysis and matching of treatment arms techniques (15 minutes). Madhu will present a real-world case study demonstrating the application of these methods in an HTA setting from industry perspective (15 minutes). Kate will provide an overview of the acceptance of these methodologies from the perspective of a NICE External Assessment Group member (10 minutes).
Audience engagement will include live polling, interactive scenario discussions, and Q&A (15 minutes). Participants will evaluate example data scenarios, vote on methodological choices, and engage in discussion with panellists, promoting practical understanding and application of the concepts presented. This session will benefit HTA professionals and industry stakeholders involved in generating or evaluating comparative effectiveness evidence for decision-making in healthcare.
Moderator
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Raju Gautam, PhD
ConnectHEOR, London, United Kingdom
Speakers
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Janharpreet Singh, PhD
University of Leicester, Leicester, United Kingdom
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Madhusudan Kabra
United Kingdom
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Kate Ren, PhD
ConnectHEOR|University of Sheffield, London|Sheffield, United Kingdom
Can We Quantify the Public Health Impact of Vaccines in Mitigating Antimicrobial Resistance?
Session Type: Issue Panel
Topics: Epidemiology & Public Health, Study Approaches, Health Technology Assessment
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue: Antimicrobial resistance (AMR) poses a significant global public health threat, complicating infectious disease treatment, increasing morbidity and mortality, and escalating healthcare costs. Its emergence is largely driven by inappropriate antimicrobial use. Vaccination plays a critical role in combating AMR by preventing infectious diseases and thereby reducing antimicrobial usage. However, traditional economic evaluations often overlook these substantial public health benefits, primarily due to data gaps and technical challenges in quantifying them.Overview: This panel will explore the evaluation of the broader value of vaccines in combatting the global public health challenge of AMR, debating the need and approaches for evidence generation and methodological advancement. Dr. Huang will moderate the session and provide an overview of the global burden of AMR, summarize recent research on projecting AMR health economic burden of AMR, and highlight existing challenges and gaps (10 mins). Dr. Grijalva will examine key clinical sectors driving AMR development and transmission and discuss the public health implications of integrating vaccine strategies into AMR prevention efforts (15 mins). Dr. Elbasha will present a novel vaccine model, showcasing methodologies in quantifying population benefits of vaccines in AMR prevention and addressing uncertainties in resistance evolution. The case study will illustrate projected public health impacts in both high-income countries where antimicrobial overuse is prevalent, and low- and middle-income countries facing double challenges of antimicrobial shortage and rising resistance (15 mins). Dr. Drummond will discuss uncertainty assessment and model validations when demonstrating broader value of vaccines to decision makers and offer recommendations on future research (10 mins). The session will conclude with a moderated audience discussion, inviting attendees to share their views and insights.
Moderator
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Min Huang, PhD
Merck & Co. Inc, North Wales, PA, United States
Speakers
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Elamin Elbasha, BSc, MA, PhD
Merck, West Point, United States
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Michael Drummond, MCom, DPhil
University of York, York, United Kingdom
Michael Drummond is Professor Emeritus and former Director of the Centre for Health Economics at the University of York in the United Kingdom. His main field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 750 scientific papers. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. He served for 14 years as Co-Editor-in-Chief of Value in Health and was made Editor Emeritus in May 2024. He has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
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Carlos Grijalva, MPH, MD
Vanderbilt University, Nashville, TN, United States
What Lessons Can the US Learn from Europe’s Experience with International Reference Pricing?
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment
Level: Intermediate
Available On-Demand: Digital Conference Pass
The Trump administration has proposed international reference pricing (IRP) through a Most Favored Nations (MFN) mechanism to lower drug costs in the US. EU member countries have substantial experience with IRP, including very real unintended consequences. What lessons can the US learn from Europe’s substantial experience with pharmaceutical reference pricing?
Dr. Sullivan will provide an overview (10 mins) of the proposed Most Favored Nations (MFN) policy of pharmaceutical reference pricing, the countries to be included in the basket and the proposed referencing mechanisms. He also will introduce the speakers and manage the discussion. Dr. Kanavos will describe how IRP is currently implemented in Europe, explain the benefits, limitations and unintended consequences of IRP. He will also describe how countries have developed ways to work around referencing through delayed entry and differential access, value assessment through clinical and/or cost-effectiveness, and confidential pricing agreements (10 mins). Dr. Acha will discuss how the pharmaceutical industry experience with IRP and how the industry has responded to IRP in Europe. (10 mins). Finally, Dr. Spiesser will discuss how EU countries may or may not be able and willing to raise pharmaceutical prices in the face of lower US prices, including any legal or trade policy implications (10 mins). The panelists will engage with the audience (25 mins) to address questions related to the proposed MFN policy and possible unintended consequences in international markets of a US international price referencing policy.
Moderator
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Sean D Sullivan, PhD
University of Washington, Seattle, WA, United States
Sean D. Sullivan, BScPharm, MSc, PhD, is Professor and Dean, School of Pharmacy. He holds a joint appointment as Professor of Health Services in the School of Public Health. He holds adjunct appointments in the School of Medicine, the Public Health Sciences Division at the Fred Hutchinson Cancer Research Center, and at the Kaiser Permanente Washington Health Research Institute. He completed training in pharmacy at Oregon State University in 1983, obtained a master’s degree at the University of Texas in 1986 and a PhD in health economics and policy at the University of California, Berkeley in 1992. Dr. Sullivan has authored more than 400 journal articles, book chapters, task force reports and organizational and governmental publications. In many of these writings, he has assessed the evidence and applications of medical technology in relation to coverage and reimbursement decisions. His research interests include technology assessment, medical decision-making, and economic evaluation of medical technology. He is past president of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and past chair of the Academy of Managed Care Pharmacy (AMCP) Executive Committee of the Format for Formulary Submissions – the United States evidence-based guidelines for formulary decision making. Dr. Sullivan served as a past member of the Medicare Evidence Development and Coverage Advisory Committee, a past member of the Regence Blue Shield and Premera Blue Cross P/T Committee, and the Chair of the Premera Blue Cross Value Assessment Committee. He is also on the editorial boards of Value in Health, PharmacoEconomics, Journal of Medical Economics, and the European Journal of Health Economics. He was awarded the 2014 Stephen G. Avey Lifetime Achievement Award from the Academy of Managed Care Pharmacy (AMCP) and the 2015 APhA Academy of Pharmaceutical Research Sciences (APRS) Research Achievement Award.
Speakers
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Panos Kanavos, BSc, MSc, PhD
London School of Economics and Political Science, London, United Kingdom
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Virginia Lee Acha, BA, MPhil, DPhil
Merck, London, United Kingdom
Virginia (Ginny) has worked in industry and academia throughout her career, combining interests in science policy research and innovation performance within and across organizations. She joined MSD in 2017 to lead regulatory policy ex-US for innovation that leads to better treatment for patients globally. Since 2020 this scope has expanded, and Ginny now leads the talented and experienced Science and Regulatory Policy team comprising Global Regulatory Policy, Clinical Research Policy, Data and Advanced Digital Technologies Policy and Evidence Policy. Her work has regularly focused on policy shocks that challenge innovation and access to novel therapies for patients, including global and industry-wide policy work on BREXIT, COVID-19 and the R&D impacts of the Inflation Reduction Act.
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Julie Spiesser, MSc
TAKEDA France, Zollikon, Switzerland
Maximizing Patients' Voices in HTA: Innovations in Elicitation Practices
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session explores innovative approaches to eliciting patient and public preferences in healthcare research, with a focus on methodological development and evaluation. Presentations cover the comparison of narrative versus tabular formats in discrete choice experiments, methods for involving patients and the public in defining clinically important differences, and the potential of the Analytical Hierarchy Process as an alternative for health state valuation. The session also examines emerging preference elicitation techniques suitable for small-sample studies, highlighting their advantages, limitations, and implications for robust decision-making in health research. Together, these studies offer practical insights for improving the design, feasibility, and inclusivity of preference-based research.
Advancing Preference Elicitation Techniques for Rare Diseases and Limited Sample Populations
OBJECTIVES: Patient preference studies are increasingly used to inform healthcare decision making, with discrete choice experiments (DCEs) being the most widely applied method. However, DCEs may be less suitable for small-sample settings due to their sample size requirements, cognitive burden, and analytical complexity. Without appropriate methodological alternatives, researchers risk generating biased or non-robust evidence. This study aims to provide an evaluation of four different stated preference methods [DCE, Best-Worst Scaling (BWS), Multidimensional Thresholding (MDT), and Online Personal Utility Functions (OPUF)] in small sample contexts.
METHODS: We conducted a purposive review of the literature to examine four methods, focusing on: methodological features, robustness, and relevance to small-sample preference studies. Articles were identified through targeted searches of PubMed/Medline, Scopus and Google Scholar. Abstracts were screened to select relevant articles to the review objective. Drawing on prior literature and emerging empirical evidence, we provide a critical comparison of each method and their capacity to yield reliable and interpretable results in preference studies with limited sample sizes.
RESULTS: There is no one-size-fits-all preference elicitation method in small sample contexts. Each approach offers unique advantages and limitations depending on the research context. Method selection should be guided by study design, sample size, participant characteristics, cognitive burden, and the need for exploration of preference heterogeneity. While DCE and BWS are well-established with extensive precedent in the literature, newer methods such as MDT and OPUF show promise, particularly in small samples. However, their use is still emerging, and further research is needed to strengthen the validity and robustness of these innovative approaches.
CONCLUSIONS: This study recommends that the choice of method adopted should be tailored to the specific research objectives and study characteristics. Research suggests that BWS is a robust alternative method of eliciting preferences in small samples, while OPUF and MDT appear promising but require further validation.
Seeking Patient and Public’s Opinions to Determine Target Differences in Clinical Trials: A Scoping Review of Elicitation Methods
OBJECTIVES: Target differences are the difference in the outcome of interest that a trial is designed to detect. Target differences can be based on important or realistic differences. There are no agreed methods to directly involve patients and the public in determining target differences. We aimed to conduct a scoping review to identify elicitation methods and assess applied tools to seek opinions from patients or the public on target differences.
METHODS: We conducted a snowballing scoping review. Included studies asked patient or public’s opinions directly about important or realistic treatment differences to inform the design or interpretation of trials (i.e. excluded anchor or distribution-based methods). Abstracts and full texts were screened, and data was extracted by one researcher with an independent check done by a second researcher. Validity, feasibility and patient and public involvement (PPI) were assessed based on self-report.
RESULTS: We identified 61 full texts. From these, around 80% were published after 2010. Opinion seeking tools were applied most in neoplasms (20%) and musculoskeletal diseases (20%). Four categories of elicitation methods were identified: trade-off (69%), direct questioning (20%), educational (7%) and reflective (5%) methods. The most common concept elicited by opinion seeking was the minimally important difference (25%). Around half of the studies reported validity (48%), less than a third assessed feasibility (30%). Less than half of the studies reported PPI (41%).
CONCLUSIONS: We identified elicitation methods to directly involve patients and the public in determining important differences in clinical trials. This is a first step in ensuring important differences are robustly defined, and involve patients or the public, to avoid research waste. Their quality and acceptability, and what works in what context, needs to be assessed.
‘tell Me a Story’: A Randomized Study Comparing Tabulated and Narrative Presentations of a Discrete Choice Experiment
OBJECTIVES: Discrete choice experiments (DCEs) are frequently used to evaluate the preferences of the public, patients, and healthcare professionals. Choice alternatives in DCEs are usually presented in table format. However, our pilot work suggested some respondents might prefer a narrative format, presented as a patient story. Given limited information on respondent preferences for narrative formats, we randomised respondents to see either table or narrative formats of a DCE on antibiotic prescriptions, among the UK general public.
METHODS: The online DCE used 12 choice questions, described by seven attributes such as symptom severity and risk of complications. The narrative version provided the same information as the table, but structured as a short paragraph. We assessed respondent experience of the two formats by examining completion time, drop-out rate, self-reported difficulty, and use of short-cuts (speeding, same choice for all questions, nonsense free-text responses). Respondents’ choices were modelled using heteroskedastic logistic regression to identify any differences in variability of responses, and mixed-effect logistic regression with an interaction term for format.
RESULTS: 1608 adults completed the survey (802 table, 806 narrative). We saw no significant differences in survey experience measures (all p>0.05). Models for the subsamples were consistent with the pooled model, but were a better fit than the pooled model after allowing for differences of variability (likelihood ratio test p<0.001), indicating differences in preferences and/or variability. Three of the attributes showed a small effect of format (interaction p-values<0.05). When shown both, 58% preferred the table format.
CONCLUSIONS: Although some differences in preferences were observed, these were minor, leaving the main DCE conclusions unchanged. These are reassuring findings, suggesting either format can be used. However, there may be stronger effects in groups typically under-represented in online DCEs (e.g. lower educational attainment), for whom the tabular format may even be a deterrent, leaving their voices missing in preference measures.
Eliciting Health State Preferences With Analytical Hierarchy Process: An Illustration With EQ-5D-5L
OBJECTIVES: In the current EQ-VT protocol, discrete choice experiment (DCE) heavily depends on participants imagining hypothetical health states with five health-related quality of life (HRQoL) dimensions combined. Alternatively, the analytical hierarchy process (AHP) allows participants to compare dimensions in a pairwise manner, and may potentially impose lower cognitive burden on the respondents. The study aims to explore the applicability of AHP in EQ-5D-5L health state preference elicitation.
METHODS: A cross-sectional survey (n=504) was conducted in 2022-2023 based on the gender and age group composition of the Hong Kong SAR, China community. The respondents compared the EQ-5D five dimensions in pairs based on a scale of 1 (equal importance) to 9 (extreme importance), followed by five absolute scoring tasks to assign a 0 (worst HRQoL) to 100 (perfect HRQoL) value to each level of the five dimensions. The AHP attribute matrix was derived with Saaty’s principal right eigenvector method, and the attribute weightings were aggregated with arithmetic means. AHP health state values were computed by combining the pairwise comparisons and absolute scoring tasks. Predictive ability of the AHP was assessed by evaluating the preference agreement between the AHP health state values against health state values derived from the EQ-5D-5L(Hong Kong) value sets among the EQ-VT 196 health state pairs.
RESULTS: In terms of the EQ-VT health state pairs, AHP showcased high predictive ability in matching the preferred health states exhibited by the EQ-5D-5L-HK value set (92.9%). Similar findings were also revealed in the sub-group comparisons between gender (female: 91.84%; male: 94.9%), age group (<50: 94.4%; 50: 90.8%), and education attained (tertiary education: 92.9%; secondary education or below: 91.8%).
CONCLUSIONS: Findings uncovered may hint AHP as an useful alternative in health state preference elicitation. Further research may explore its full potential in settings with limited resources and biased samples.
Accelerating Patient Access to Innovations in Europe: Is it Possible to Integrate the JCA into Diverse HTA systems?
Session Type: Spotlight
Topics: Health Technology Assessment
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE: The Joint Clinical Assessment (JCA) offers a pivotal opportunity to streamline evidence generation across European markets, but integrating its clinically focused outcomes into diverse health technology assessment (HTA) systems presents both challenges and opportunities. Despite implementation in early 2025, there remain key outstanding questions, such as: How can JCA outputs be better aligned with the diverse evidence expectations of national HTA bodies? To what extent can Member State-specific PICOs be harmonised to avoid duplication of work while respecting local decision-making processes? How can the JCA report align to cost-effectiveness frameworks in economic-driven markets? How can we balance the patient-centric focus of patient-reported outcomes (PROs) with the technical requirements of economic modeling?
OVERVIEW: Overview provided by Ruairi O’Donnell (moderator), introducing the panel and challenges associated with the JCA procedure (10min).Anja Schiel will present a national HTA body perspective, highlighting how Norwegian authorities have begun adapting to the introduction of JCA (12min). James Ryan will share findings of the health technology developer (HTD) experience (12min), exploring how preparation for JCA can inform preparation for a range of market requirements, with implications for timing of key activities and cross-functional collaboration.Professor Taylor will share an academic insight on the implications of JCA for HTA science, exploring how state-of-art methods may evolve with respect to comparative evidence analysis and patient-relevant outcomes (12min). Time is allowed for audience questions and panel discussion. This session will benefit those with involvement in national HTA procedures, either from the HTD or assessor perspective.
Moderator
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Ruairi O'Donnell
Cencora, London, United Kingdom
Speakers
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Anja E Schiel, PhD
Norwegian Medicines Agency, Oslo, Norway
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James Ryan, BA, MSc
AstraZeneca, Cambridge, United Kingdom
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Rod Taylor
Glasgow, United Kingdom
Integrating Environmental, Well-Being, and Public Health in Health Economic Assessment
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session explores value metrics related to nutrition and the environment. Topics include quantifying the health and wellbeing benefits of outdoor spaces at healthcare facilities, assessing changes in the carbon footprint of severe asthma care before and after benralizumab initiation, and examining links between undernutrition, healthcare utilization, and costs. The session concludes with a case study on incorporating environmental impacts into budget impact models for CAR-T therapies in France.
The Value of the Health and Wellbeing Benefits of Healthcare Facilities Outdoor Spaces
OBJECTIVES: The health and wellbeing benefits of exposure to open space are well known. The use of open spaces has been shown to improve blood pressure, all-cause and cardiovascular mortality, mental health, premature deaths, and sleep quality. The National Health Service (NHS) in Scotland has 825 hectares of open space (half of which is greenspace) accessible to patients, caregivers, public and staff. This space offers unique opportunities to realise health and wellbeing benefits. This potential is unexplored, and no studies have estimated the health and wellbeing value and potential of these, or similar, health-based spaces.
METHODS: This study uses two survey-based non-market valuation methods to value NHS open spaces. First, the Office for National Statistics value of the health benefits of outdoor exposure based in Quality Adjusted Life Years (QALYs). This approach may however not capture the full range of benefits these spaces provide. Second, a stated preference contingent valuation method to elicit willingness to pay (WTP) for open spaces. The online survey was administered to a representative sample of 2,449 adults in Scotland in late 2024.
RESULTS: We estimate that there are 128 million annual visits to NHS open spaces. Most visits last less than 30 minutes and are linked to visits to access NHS services. Visits typically involve low-impact activities suggesting most of the health and wellbeing benefits are linked to mental wellbeing. Based on the ONS exposure method, the direct annual health benefits from visits to NHS open spaces are estimated as £86.1 million (£65.6 million - £129.5 million). Most of this value comes from primary care sites. Based on contingent valuation WTP estimates the value of local hospital sites is £653.5 million and local primary care sites is £560.4 million.
CONCLUSIONS: The findings will provide insights into the social and economic value generated by investment in NHS open spaces.
Carbon Footprint Associated With Severe Asthma Care Before and After Benralizumab Initiation: BENEFIT Study
OBJECTIVES: Benralizumab efficacy in patients (pts) with severe asthma (SA) had been described but its ecological impact on healthcare management is unknown.The aim is to describe and compare the carbon impact of SA care 12mo before and after benralizumab initiation.
METHODS: This French health claims database study included SA adult pts with at least 1 benralizumab dispensing between 01/01/2019 and 12/31/2023. For pts with at least 12 months of follow-up, the carbon footprint associated with SA management was compared using a Wilcoxon test for the 12mo before and after the initiation of benralizumab. Carbon emission values from the literature were assigned to asthma therapies, consultations, asthma-related hospital stays, emergency room visits, medical procedures, biological acts and transport.
RESULTS: This primary analysis included 8,270 pts (59.0% female, mean age 58.7 ± 15.2 years). The mean difference [95%CI] in carbon footprint (kgCO2e) between the 12mo before and after the initiation of benralizumab, was -19.9 [-22.7; -17.2] for medications, -25.5 [-28.8; -22.3] for asthma-related hospitalizations, -4.1 [-5.0; -3.2] for emergency room visits, and -20.5 [-21.8; -19.2] for medical procedures but consultations and visits increased (mean difference [95%CI]: 22.8 [16.7; 28.9]), mainly due to an increase in nursing visits (mean difference [95%CI]: 24.2 [19.4; 28.9]). The total carbon footprint was 595.7 ± 562.6 before vs 544.0 ± 587.0 after benralizumab initiation, resulting in a mean difference of -51.6 [-59.8; -43.4]. All p<0.0001.
CONCLUSIONS: In pts treated with benralizumab there was a reduction in the ecological impact of SA care by significantly reducing the overall carbon footprint.
Incorporating Environmental Impacts Into Budget Impact Models: A Case Study of CAR-T Therapies in France
OBJECTIVES: Explore and illustrate how environmental impacts can be identified, quantified, and integrated into budget impact models (BIMs), regarding recent French policy developments (including Haute Autorité de Santé 2022-2024 health environment roadmap and 2024 Social Security Financing Bill). We conducted a case study comparing CAR-T therapy with conventional chemotherapy in the French healthcare context.
METHODS: A targeted literature review was conducted to identify key frameworks for integrating environmental impacts into health technology assessments (NICE typology, Williams et al. (2023), ECOVAMED). A BIM was adapted to incorporate patient transport-related emissions, using official data on travel patterns and standard carbon intensity factors. Emissions were expressed in kgCO₂-equivalent and valued using the official national carbon price (€83.56/ton).
RESULTS: Carbon footprint was the most common environmental metric used in the literature, often derived through life cycle assessment. Thus, environmental impact was integrated via transport-related carbon emissions in the case study. This information was also easily available compared to other impacts (e.g., Hospitalization-related emissions in France). CAR-T therapy requires fewer patient visits (4 round trips, average distance for chemotherapy in France: 25 km/journey, i.e., 400 km total) compared to chemotherapy (13 round trips, 650 km total annually). Transport-related carbon emissions for CAR-T were estimated at 76,000kgCO₂e versus 123,500kgCO₂e for chemotherapy, translating into €6,351 and €10,320 in environmental costs, respectively. Including caregiver travel added further differentiation (19,000kgCO₂e, €1,588).
CONCLUSIONS: Incorporating environmental externalities altered the net budget impact and highlighted organizational and societal costs not captured in standard models. It can influence health-economic outcomes and decision-making. This approach should extend to other life cycle stages (e.g., production, distribution, disposal) to derive relevant information. Broader integration into BIMs could support more sustainable health decision-making, especially as French institutions are reflecting to move toward environmental inclusion in HTA and pricing. Standardized guidance will be essential to ensure comparability and avoid methodological variability.
Associations Between Undernutrition and Healthcare Utilization and Costs in Community-Dwelling Adults: A Longitudinal Observational Study
OBJECTIVES: The impact of undernutrition on healthcare utilisation and costs is underexplored in Asian populations. This study examined the longitudinal association between undernutrition and healthcare use across various settings among community-dwelling adults in Singapore.
METHODS: We analysed data from 1,703 participants in the Population Health Index study who consented to data linkage. Nutritional status was assessed annually using the Mini Nutritional Assessment and classified as undernourished if total score <24 (out of 30) or screening score <12 (out of 14). One-year and five-year healthcare utilisation and cost data were extracted from a centralised institutional repository, spanning five settings: primary care, specialist outpatient clinics (SOCs), emergency departments (EDs), day surgeries (DSs), and inpatient admissions. Two-part Hurdle models (logistic regression for any use; negative binomial or generalised linear models for intensity/cost) assessed associations with undernutrition, adjusting for baseline covariates.
RESULTS: Undernutrition was associated with higher odds of SOC visits (odds ratio [OR]: 1.66, 95% confidence interval [CI]: 1.03, 2.68), ED visits (OR: 1.85, 95% CI: 1.18, 2.92), and inpatient admissions (OR: 2.47, 95% CI: 1.42, 4.29) in one year, and with admissions in five years (OR: 1.62 , 95% CI: 1.07, 2.47). Among users, undernutrition was not linked to greater utilisation or costs in one year. Over five years, it was associated with fewer visits (Incidence Rate Ratio [IRR]: 0.75, 95% CI: 0.61, 0.94) and lower expenditure in primary care settings (β: -0.33, 95% CI: -0.58, -0.09), lower expenditure in SOCs (β: -0.41, 95% CI: -0.73, -0.09) but more admissions (IRR: 1.31, 95% CI: 1.04, 1.64), and higher ED costs (β: 0.27, 95% CI: 0.02, 0.53).
CONCLUSIONS: Undernutrition is independently associated with increased emergency and inpatient utilisation over time. Early detection and intervention may reduce preventable, high-cost healthcare utilisation, especially in ageing populations.
Trusting a Machine? Opportunities and Challenges to using Artificial Intelligence in Qualitative Patient Research
Session Type: Issue Panel
Topics: Patient-Centered Research, Methodological & Statistical Research, Study Approaches
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue: Qualitative patient research is at the center of patient-focused drug development, contributing to the selection and development of endpoints that evaluate new therapies on outcomes that matter to patients. However, robust qualitative research takes time and requires the use of highly experienced staff. AI has the potential to contribute to efficient and scaled delivery of qualitative research to ensure patient voices can be reflected in a wide range of decisions across the product life cycle.
Overview: This session will delve into the practical and ethical issues raised when introducing the use of AI into qualitative data analysis from the perspectives of study sponsor and researcher, drawing on practical experiences. The debate will be guided by the practical experiences of the panel members, including a pilot study that compared human and AI tasks in the analysis of qualitative data for regulatory submissions.
Christina Silver will moderate the session and provide a brief history of the use of AI in qualitative analysis leading to the present-day. Karen Bailey and Paul Cordero will discuss a case study of AI use-case with data from a hybrid concept elicitation and cognitive interview study involving non-Hodgkin’s lymphoma patients in order to understand how the use of AI can support the quality and efficiency of outputs. The speakers will reflect on the value of AI with human interaction in the development and use of the codebook, qualitative analysis plan, coding, content analysis, table outputs, and report writing. They will present the key scientific and potential regulatory challenges and preliminary guidance for use-cases for AI analysis of patient qualitative data. All speakers will provide practical insights from real applications but will also debate unsolved issues that require consensus among the sponsors, regulators, payers, scientific community and wider society.
Moderator
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Christina Silver, PhD, Other
University of Surrey, Guildford, United Kingdom
Speakers
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Karen Bailey, PhD
Evidera, London, United Kingdom
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Paul Cordero, PharmD, PhD
Sanofi, London, United Kingdom
Can We Have it “Whole”? Barriers and Opportunities for Embracing a Whole Health Perspective in HEOR
Session Type: Issue Panel
Topics: Economic Evaluation, Health Technology Assessment, Health Policy & Regulatory
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue
ISPOR’s strategic focus on Whole Health challenges the traditional boundaries of health economics and outcomes research (HEOR), calling for a holistic approach to healthcare to incorporate physical, mental, social, environmental, and spiritual dimensions of wellbeing. Whole Health may thus move economic evaluations beyond the healthcare sector to include societal outcomes such as productivity, education, social care, and broader economic resilience. As a result, the choice of perspective as well as analytic approach, e.g. cost-effectiveness/utility analysis (CEA/CUA), societal cost-benefit analysis (CBA), or the more recently proposed social decision-making analysis (SDMA), is an increasingly important decision that will shape the value narrative and influence policy making.
Overview
Through an illustrative case study based on findings from a recent analysis estimating the societal return on investment (ROI) of vaccines we will show the impact of using different valuation approaches. We then will discuss the pros and cons of embracing a Whole Health approach and what this would mean for decision making on a country level.
The moderator (Lotte Steuten) will provide a brief overview of key definitions and challenges (4 min). Each panellist will then provide their perspective with a special focus on key challenges and potential solutions in the context of the broader questions above (12 min). 20 minutes will be reserved for audience discussion.
Attendees will gain a deeper understanding of how normative and analytic choices affect value assessment and decision-making and how HEOR can evolve to support more holistic, inclusive health policy aligned with ISPOR’s Whole Health vision.
Key questions to be discussed will include how a Whole Health Approach could be implemented and sustainably operationalised, whether this will be realistically achievable given the current political context, and what other solutions might exist to improve the status quo in the meantime.
Moderator
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Lotte Steuten, MSc, PhD
Office of Health Economics, London, United Kingdom
Speakers
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Simon Brassel, BSc, MSc
Office of Health Economics, London, United Kingdom
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Nancy J Devlin, PhD
University of Melbourne, Melbourne, Australia
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Marcin Czech, MBA, PhD, MD
Institute of Mother and Child, Warsaw, Poland
Marcin Czech is a full professor and head of the Department of Pharmacoeconomics at the Institute of Mother and Child in Warsaw, postgraduate courses director at Warsaw University of Technology, Business School, Past President of ISPOR, Poland Chapter, former Undersecretary of State/ Vice Minister at the Ministry of Health.
He is the author of over 300 articles, books and reports in the field of management, health economics, pharmacoeconomics and quality of life.
A medical doctor by education, specialist in epidemiology and public health, specialist in psychiatry, holding PhD degrees in medicine and management, MBA; completed postgraduate studies in Health Economics, Leadership Academy, university trainings at the University of York, University of St. Andrews, Mc. Master University.
The Issue No One Talks About: Standardizing Adverse Event Inclusion in Economic Evaluations
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment, Real World Data & Information Systems
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE: The inclusion of adverse events (AEs) in economic evaluations (EE) is a critical yet often overlooked component of health technology assessment (HTA). While AEs can significantly impact both costs and quality of life, their integration into cost-effectiveness models is inconsistent, methodologically variable, and frequently absent. This omission risks underestimating the true burden of interventions—particularly those with substantial safety concerns-and may distort value assessments used to inform pricing, reimbursement, and access decisions. Currently, there is no standardised approach for incorporating AEs into economic evaluations. Some models include only severe or treatment-related AEs, while others rely on limited trial data or exclude AEs entirely due to data gaps or methodological uncertainty. This inconsistency reflects broader challenges: the lack of harmonised guidance, limited access to real-world safety data (RWSD), and insufficient use of patient registries that could provide richer, more representative safety profiles.
The issue, then, is whether the absence of standardised AE inclusion undermines the credibility, transparency, and patient-centeredness of EE. Addressing this gap could improve alignment between regulatory and HTA perspectives, enhance the use of RWSD, and ultimately lead to more informed and equitable healthcare decisions.
OVERVIEW: This panel will explore the scientific, methodological, and policy implications of standardising AE inclusion in EE. Moderator Tanja Fens will open with a brief reflection (5 min) and guide discussion through key questions (15 min): Should HTA bodies adopt standardised methods for AE inclusion to improve consistency and transparency?; What standards are needed to align regulatory and HTA use of safety data?; How can patient registries and RWSD be leveraged to better capture safety outcomes?; What are the practical implications for industry, policymakers, and patients regarding evidence and decision-making?
Panellists Peter Mol, Dalia Dawoud and Mondher Toumi will each present their perspectives (10 min each), followed by audience engagement through live polls and open Q&A (10 min).
Moderator
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Tanja Fens, PharmD, PhD
RUG/University of Groningen, Groningen, Netherlands
Speakers
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Peter Mol, PhD
College ter Beoordeling van Geneesmiddelen - Dutch Medicines Evaluation Board/ University Medical Ce, Groningen, Netherlands
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Dalia Dawoud, BSc, MSc, PhD
Cairo University, Cairo, Egypt
Dalia Dawoud, PhD, is the director of PEHTA Consulting Ltd. She is also a professor 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, IMI EHDEN 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.
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Mondher Toumi, PhD
Inovintell, Luxembourg, Luxembourg
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.
Is It Really Time to Abandon Excel or Can R Generate Excel Models for HTA Submissions?
Session Type: Issue Panel
Topics: Health Technology Assessment, Methodological & Statistical Research, Economic Evaluation
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE:
While R-based modeling has gained traction in the HEOR community for its flexibility, reproducibility, and computational efficiency, HTA agencies continue to favor Excel-based models due to their transparency and auditability. This divergence creates tension between innovation and regulatory tradition. A practical question arises: Can R serve both worlds—developing robust models while outputting transparent Excel workbooks for HTA review? Can such hybrid models be truly accepted by HTA agencies? How do we ensure traceability and audit trails in generated Excel files? And what are the best practices for implementing this dual approach?
OVERVIEW:
This panel will explore whether R can serve as the engine to generate Excel models that are both technically advanced and regulatorily compliant.
(5 min) Deepika Thakur will introduce the session and set the context for the ongoing debate between R-based and Excel-based modeling practices in HTA
(12 min) Karam Diaby will explain the foundational mechanics of developing HE models in R that programmatically produce Excel models, highlighting real-world applications and benefits
(12 min) Shubhram Pandey will deep-dive into workflows involving R Shiny and how they can be used to deliver interactive Excel-compatible outputs suitable for HTA submissions
(12 min) Prof. Howard Thom will reflect on HTA bodies’ expectations and whether R-generated Excel models could be seen as transparent, traceable, and submission-worthy
Following these presentations, there will be a 15-minute audience Q&A and discussion period, offering attendees actionable insights on balancing the analytical power of R with the regulatory demand for transparency and traceability in Excel-based HTA submissions.
Moderator
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Deepika Thakur, PhD
Hoffman La Roche AG, Courtice, ON, Canada
Speakers
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Shubhram Pandey, MSc
Pharmacoevidence Pvt. Ltd., SAS Nagar, Mohali, India
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Karam Diaby, BSc, MSc, PhD
Otsuka, Princeton, NJ, United States
Karam Diaby, PhD
Director, Health Economics & Value Evidence Partnership, Otsuka Pharmaceutical
Dr. Karam Diaby is a leading health economist with over 13 years of experience across academia
and industry. As Director of Health Economics & Value Evidence at Otsuka Pharmaceutical, he
leads global modeling and AI-enabled evidence strategies supporting HTA submissions in
neuroscience, oncology, immunology, and digital health.
His recent work pioneers the integration of Input-Output and macroeconomic modeling into
HTA to capture broader societal, productivity, and equity impacts. He has published over 70
peer-reviewed articles and received multiple honors, including the Innovation and Value
Initiative’s “Valuing Innovation” award.
At ISPOR Europe 2025, Dr. Diaby will share insights on how R-based modeling can reconcile
analytical innovation with the transparency demands of HTA, charting a path toward future-
ready evidence generation.
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Howard Thom, BA, MSc, PhD
Bristol Medical School : Population Health Sciences, Bristol, United Kingdom
Howard Thom has worked for over 10 years developing health economic models in R. These have been in many disease areas, including heart disease, stroke, physiotherapy, mental health, rheumatology, dermatology, and oncology. His methodological interests are structural uncertainty, value of information analysis, and the use of R for efficient modelling. He founded and co-chairs the R for Health Technology Assessment (HTA) scientific committee, organizing annual workshops on the use of R in HTA. He currently works at both the University of Bristol and at Clifton Insight, giving him the perspective of both commercial consulting and academia.
14:45 - 15:15
Afternoon Break: Coffee and Connect (Exhibit Hall)
Session Type: General Meeting
15:00 - 16:00
HEOR Impact Cases in Oncology and Rare Disease
Session Type: HEOR Impact Cases
Biosimilar Trastuzumab's Public Reimbursement inOncologyusing Cost-Effectiveness Evidence
Problem Statement: One of the primary causes of cancer-related mortality and morbidity among Indian women is HER2-positive breast cancer. Guidelines prescribe trastuzumab as a treatment for this population, but historically, public oncology clinics in India have not been able to provide extensive access to it due to its high purchasing cost. Whether biosimilar trastuzumab might be practically incorporated into government-funded chemotherapy pathways without jeopardizing financial sustainability was the main concern for state health authorities.
Description: To determine the cost-effectiveness and economic impacts of adding biosimilar trastuzumab to a major Indian state's essential medicines list (EML), a health economic review was conducted. The study compared biosimilar trastuzumab with chemotherapy against chemotherapy alone to patients with early-stage HER2-positive breast cancer using a decision-analytic method and a government payer perspective. Real-world data retrieved from Indian cancer registries was used to explain the clinical efficacy inputs, which were taken from published randomized trials. Estimates of utilized resources from tertiary cancer institutions and public procurement costs were evidence of cost inputs. The incremental cost-effectiveness ratio (ICER) produced by the model was ₹92,000 every quality-adjusted life year (QALY) gained, which is significantly smaller than the common willingness to pay threshold for India. A five-year budget impact analysis projected that expected reduction in hospitalization and disease recurrence rates would compensate for the additional expenses of ₹125 crore. The outcomes were examined in cooperation with national health technology assessment body (HTAIn) and given to the State Health Authority as a verification report.
Lessons Learned: Incorporating localized HEOR data positively influenced governmental purchasing practices and made it easier to include biosimilar trastuzumab in the state's EML based on research. Academic-government cooperation, early and ongoing interaction with individuals making decisions, and the utilization of data were key elements of success. A significant downstream influence on population-level cancer care equity was indicated by following the implementation evaluation, which showed a 60% rise in trastuzumab availability across public tertiary care facilities in just one year.
Stakeholder Perspective: This example is given from the perspective of the HTAIn body and the government payer, who worked together to overcome major accessibility and reimbursement challenges in oncology care by utilizing health economic research. The study demonstrates the direct application of HEOR to influence government health care allocation of resources in low- and middle-income countries.
Integrating Quantitative Patient and Carer Insights into HTA: The Impact of a Patient Organisation Survey on a NICE Appraisal
Problem Statement: X-linked hypophosphataemia (XLH) is a rare, lifelong, and progressively debilitating genetic disorder that significantly impacts patients' physical function, emotional wellbeing, and quality of life. Economic assessments often inadequately capture the full burden of disease. During the UK National Institute for Health and Care Excellence (NICE) assessment of a novel treatment for XLH, there was a need for real-world evidence to contextualize the impact of an effective treatment on the number of carers patients need and the extent to which caring for people with XLH affects quality of life.
Description: During NICE’s Technology Appraisal (TA993) key uncertainties were raised in the draft guidance around impact on carers. The company assessment assumed a spillover utility benefit for carers or family members. XLH being a genetic disorder, the committee noted the importance of quantifying the impact on family members, especially if they themselves were affected by XLH. Due to the scarcity of evidence on utility benefit for carers, XLH UK—a patient organisation—conducted a national survey following publication of the interim guidance. The survey collected quantitative data on caring responsibilities, including the number of carers an adult with XLH would have as well as qualitative insights on how treatment may impact family members’ quality of life. Insights from the survey were submitted as part of the patient organisation’s evidence and were referenced in multiple parts of NICE’s appraisal documentation, including the committee papers and the final appraisal determination. The evidence helped NICE understand the extent of the spillover effect and how an effective treatment may alter disease management and quality of life. This case demonstrates how patient-generated data was pivotal in shaping a positive reimbursement decision for a rare disease therapy.
Lessons Learned: This case underscores the importance and feasibility of integrating quantitative patient-reported evidence into HTA processes, particularly where standard clinical studies fall short. The XLH UK survey complemented trial data by offering a broader view of the disease burden. Challenges included ensuring methodological rigor in survey design given time limits, mitigated through collaboration with clinicians and patient experts. The success of this approach suggests a model for future patient-driven HEOR contributions, especially in the appraisal of orphan therapies.
Stakeholder Perspective: The primary stakeholders impacted were NICE (health technology assessor), the NHS (payer), and patients and carers. This case illustrates how structured patient and carer input can shape national-level reimbursement decisions and highlights the evolving role of patient organisations as contributors of impactful HEOR evidence.
Exploring Value Creation through Risk-Sharing Agreements in Oncology: Case Studies in Lung Cancer and Hepatocellular Carcinoma
Problem Statement: Beneficiaries of the Brazilian supplementary (private) healthcare system face significant barriers to access high-cost therapies, especially in complex conditions like cancer. The risk-sharing agreements (RSA) were designed to generate value across multiple stakeholders in the healthcare system while assessing mechanisms for value-based remuneration to overcome such barriers for access, with a main focus in overcoming financial barriers for access.
Description: Two RSAs were implemented to address financial barriers associated with high-cost oncology treatments. The first RSA focused on Extensive-stage Small Cell Lung Cancer (ES-SCLC) patients, a condition characterized by rapid disease progression and limited treatment options. The second RSA targeted patients with Hepatocellular Carcinoma (HCC), a common type of liver cancer with significant morbidity rates. The HEOR evidence used in these agreements included a comprehensive analysis of patient-reported outcome and treatment costs. Data collection involved tracking patient progress and financial reimbursements for unmet clinical goals. The approach assessed the cost-effectiveness and overall efficacy of the oncology therapies, as well as the impact of RSA on financial and clinical outcomes across stakeholder groups.
Lessons Learned: The implementation of RSAs demonstrated significant effectiveness in optimizing treatment costs and improving resource allocation. Approximately 85% of treated patients achieved the predefined clinical outcomes. Additionally, the reimbursement for unmet clinical goals resulted in a substantial 14% reduction in overall treatment costs. These findings highlight the value of adopting RSA as a strategic approach to performance and outcomes-based healthcare management. Key lessons include the relevance of incorporating RSA to mitigate financial barriers and enhance therapy access for patients.
Stakeholder Perspective: The perspective addressed in this analysis is primarily that of healthcare payers, who are responsible for managing the financial aspects of patient care. However, the RSAs also provide insights from the perspectives of patients, providers, and industry stakeholders. By facilitating more equitable access to costly therapies, the agreements benefited patients who otherwise would've faced significant financial hurdles. Providers gained from streamlined resource allocation, allowing them to focus on clinical excellence. The industry, meanwhile, has seen opportunities to demonstrate the cost-effectiveness of their therapies, supporting broader adoption in the healthcare system. Therefore, these RSAs collectively provided a comprehensive framework for improving access, optimizing costs, and advancing the quality of care within the healthcare system.
15:15 - 16:15
Harmonizing and Enabling Cross-Border Real-World Evidence Studies in Europe
Session Type: Forums
Topics: Real World Data & Information Systems, Health Policy & Regulatory, Methodological & Statistical Research
Level: Intermediate
Available On-Demand: Digital Conference Pass
As real-world evidence (RWE) becomes increasingly integral to regulatory, HTA, and payer decision-making in Europe, the ability to generate robust evidence from real-world data (RWD) across EU member states has emerged as a strategic priority. However, this remains challenging due to heterogeneity in healthcare data systems, variations in clinical coding, language and contextual differences, and uneven data infrastructure maturity. Recent reports from a consortium (Real4Reg) involving ten EU institutes, highlighted the constraints of availability and heterogeneity of RWD and the challenges in analyzing RWD from different settings and sources in the pre-authorization phase. Similarly, the TEHDAS report on Secondary Use of Health Data Through European Case Studies identified underdeveloped technical services and fragmented privacy regulations as significant barriers to cross-border RWD use (TEHDAS, 2022).
To highlight these challenges and to discuss potential future solutions, this panel brings together industry experts with hands-on experience leading multi-country RWE initiatives across Europe. Panelists will share specific challenges they encountered including those related to data interoperability, multilingual unstructured data, and compliance with country-specific privacy frameworks and present viable, real-world solutions that enabled successful study execution. The discussion will cover data harmonization methodologies used to align heterogeneous clinical variables across countries, along with AI-enabled technologies such as natural language processing and machine learning to support multilingual data integration and automated data quality assessment, and the use of federated networks and synthetic data to enable privacy-preserving analytics. The panel will address challenges around data access, governance, and the complexities of navigating varying national data privacy policies, alongside operational frameworks that support consistent, transparent, and auditable study designs across jurisdictions.
Moderator
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Ashwin Kumar Rai, MS
Thermo Fisher Scientific, Overland Park, KS, United States
Speakers
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Miren Taberna Saiz, PhD
Madrid, Spain
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Sandrine Bourguignon, MSc, PhD
RWEality, BONDOUFLE, France
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Michael Hurst, BSc, MSc
Bristol Myers Squibb, Uxbridge, United Kingdom
Director of Global HEOR, Cardiovascular, HTA and Early Assets at Bristol Myers Squibb with 12 years experience in the pharmaceutical industry. Specific research interests include advanced modelling methodology, real-world evidence and machine learning.
Making Health Equity Actionable in HTA: From Frameworks to Implementation
Session Type: Forums
Topics: Health Technology Assessment, Health Policy & Regulatory, Economic Evaluation
Level: Intermediate
Available On-Demand: Digital Conference Pass
Addressing health inequities is crucial to building fair and equitable healthcare systems. While several frameworks have emerged to guide the integration of equity into health technology assessment (HTA), implementation in practice remains limited. HTA agencies often face challenges such as lack of operational guidance, limited access to disaggregated equity - relevant data, and institutional or contextual barriers. Understanding these barriers and identifying facilitators can inform actionable strategies to reduce disparities and the equity implementation gap.
This forum will explore practical pathways to implement health equity in HTA decision making processes and present interim findings from the key project conducted by the Health Equity Research Special Interest Group (HER SIG). The forum will bring together global perspectives and stakeholder experiences across regions to share lessons learned and highlight promising practices.
The discussion will focus on the following key areas:
1. Overview of current approaches to incorporating health equity in HTA, including preliminary findings from the HER SIG’s key project exploring stakeholder perspectives and barriers and facilitators to implementation across countries.
2. Perspectives obtained through methodology development and implementation of health equity in HTA organizations across different regions, such as Europe and Asia-Pacific and across disease areas, including rare diseases.
3. Practical examples of advancing the implementation of health equity methods such as distributional cost-effectiveness analysis into decision-making processes.
4. Strategies to support integration of equity considerations into local HTA and align methodological approaches with policy needs.
The forum will adhere strictly to ISPOR’s Code of Conduct and Antitrust Guidelines. It will be an educational discussion without any promotional intent, ensuring an open and unbiased exchange of ideas.
As part of this forum, we welcome perspectives from researchers, HTA assessors, HEOR analysts, and industry stakeholders to collectively explore current state of and pathways to implement health equity in HTA decision making processes.
Moderator
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Rajshree Pandey, MPH, PhD
Alexion AstraZeneca Rare Disease, Boston, MA, United States
Speakers
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Antonio Ahumada-Canale, PhD
CHERE UTS, Sydney, Australia
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Paula Lorgelly, BSc, PhD
University of Auckland, Auckland, New Zealand
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Richard Cookson
Vaccine Value Assessment in CEE Countries: Challenges and Opportunities
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Epidemiology & Public Health, Health Technology Assessment
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
Vaccinations are considered efficient interventions in healthcare systems, both in terms of health and economy. This is also true for Central and Eastern European (CEE) countries with highly limited resources compared to Western European countries. The vaccination landscape in CEE countries is complex shaped by vaccine hesitancy, misinformation about the value of vaccination, structural and access barriers and suboptimal vaccination coverage. Romania and Poland have relatively low vaccination rates in the EU (measles, polio, flu), which poses a significant public health risk. Lengthy approval processes and insufficient value recognition for new vaccines can delay access in Poland & Hungary, economic constraints & limited healthcare budget impact vaccine availability and distribution in Bulgaria and Serbia. Some CEE countries and most of Balkan countries have a huge problem with vaccination coverage because of the misinformation about the safety of the vaccine and their efficacy. Therefore, efforts are needed to build public trust in vaccines and combat misinformation to showing the full value of vaccines in all CEE countries.
This panel will explore and discuss above challenges and opportunities in vaccination landscape across CEE countries and how to deal with this and improve access to vaccines in the region with special focus on i) the roles of different national bodies in making decisions on vaccinations, ii) the timeliness of assessments and decisions, ii) the methodologies used by national immunization advisory groups and HTA bodies to assess vaccines, iv) the transparency of recommendations and decision-making processes and v) payment models for vaccines.
The EU joint clinical assessment will become mandatory for vaccines in 2030. Until then it would be critical to explore specific components of the value assessment for vaccines ( e.g. herd immunity, reduced absenteeism from work, long-term perspective of cost-benefit analysis etc.), which have limited transferability across CEE countries.
Moderator
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Joanna A Lis, MBA, MSc, PhD
Sanofi, Warsaw, Poland
Speakers
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Marcin Czech, MBA, PhD, MD
Institute of Mother and Child, Warsaw, Poland
Marcin Czech is a full professor and head of the Department of Pharmacoeconomics at the Institute of Mother and Child in Warsaw, postgraduate courses director at Warsaw University of Technology, Business School, Past President of ISPOR, Poland Chapter, former Undersecretary of State/ Vice Minister at the Ministry of Health.
He is the author of over 300 articles, books and reports in the field of management, health economics, pharmacoeconomics and quality of life.
A medical doctor by education, specialist in epidemiology and public health, specialist in psychiatry, holding PhD degrees in medicine and management, MBA; completed postgraduate studies in Health Economics, Leadership Academy, university trainings at the University of York, University of St. Andrews, Mc. Master University.
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Zoltan Kalo, PhD
Center for Health Technology Assessment, Semmelweis University & Syreon Research Institute, Budapest, Hungary
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Jovan Mihajlovic, MSc, PhD
Jovan Mihajlovic, Novi Sad, Serbia
Final Recommendations from the ISPOR Taskforce on Statistical Evaluation of Surrogacy Endpoints for Value Demonstration and Health Economic Modelling
Session Type: Forums
Topics: Clinical Outcomes, Health Technology Assessment, Methodological & Statistical Research
Level: Advanced
Available On-Demand: Digital Conference Pass
In this Forum we will discuss the final recommendations from the ISPOR Statistical Evaluation of Surrogate Endpoints for HTA Decision Making Good Practices Task Force, which will be linked to the recent international HTA recommendations published by NICE on surrogate endpoints in cost-effectiveness analysis. The audience will gain a deeper understanding of statistical methods for clinical and economic value demonstration when surrogate endpoints are employed for licensing purposes. Despite the existence of guidelines on surrogate endpoints published by many HTA agencies, few HTA submissions explicitly present evidence supporting the translation of the treatment effect on the surrogate endpoints to the treatment effect on the target outcomes for clinical value and economic value demonstration. Dr Ciani will introduce the session (6 min). Dr. Bujkiewicz will discuss final recommendations related to statistical approaches available for surrogate endpoint evaluation, including considerations for predictions, associated uncertainty and emerging novel tools to tackle less optimal data scenarios to support decision making (12 minutes). Dr. Ouwens will discuss final recommendations around surrogacy and health economic modelling distinguishing partitioned survival analysis, state transition modeling and patient level simulations (12 min). Ms. Garrett will discuss implementation of the recommendations and compare the recommendations of the ISPOR taskforce to the recommendations of the HTA agencies report (12 minutes).Dr Ciani will open the Q&A session on these recommendations. The workshop ends with a poll on topics that deserve further exploration in future guidance (10 min).This informative workshop is designed for researchers, payers, regulators, and technology assessors, as well as for those working in the pharmaceutical industry or those interested in HTA and market access of health technologies.
Moderator
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Oriana Ciani, BSc, MSc, PhD
SDA Bocconi, Milan, Italy
Speakers
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Mario Ouwens, PhD
Astrazeneca, Mölndal, Sweden
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Sylwia Bujkiewicz, MSc, PhD
University of Leicester, Leicester, United Kingdom
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Zoe Garrett
NICE, Manchester, United Kingdom
16:00 - 16:45
Student Research Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 4. Posters will be hung from 16:00 - 19:00.
Pricing & Reimbursement Poster Tour
Session Type: Research Posters
This tour will take place during Poster Session 4. Posters will be hung from 16:00 - 19:00.
16:00 - 19:00
Poster Session 4
Session Type: Research Posters
Poster Tours will be from 16:00–16:45 | Presenters will be with their posters from 18:00–19:00
Poster Session 4
Session Type: General Meeting
Poster Tours will be from 16:00–16:45 | Presenters will be with their posters from 18:00–19:00
16:20 - 16:50
Defining Appropriate Benefits for Economic Evaluation: Do Recommendations From HEMA Help?
Session Type: Fast Facts
Topics: Economic Evaluation, Health Technology Assessment, Methodological & Statistical Research
Track: Patient-Centered Evidence
Level: Intermediate
Moderator
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Mark Sculpher, PhD
University of York, York, United Kingdom
Speakers
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Robert B McQueen, BA, MA, PhD
University of Colorado Skaggs School of Pharmacy and Pharmaceutical Science, Denver, CO, United States
R. Brett McQueen is the director for the Center for Pharmaceutical Value (pValue) at the University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences, where he is an associate professor in the Department of Clinical Pharmacy. Brett’s work includes comparative effectiveness research, cost-effectiveness applications and methods development, multi-criteria decision analysis, outcomes-based contracting, and patient preferences research. He is active in ISPOR through contributions to short courses, workshops, issue panels, and research presentations.
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Saskia Knies, PhD
Zorginstituut Nederland, Diemen, Netherlands
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Lou Garrison, PhD
The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, Seattle, WA, United States
Lou Garrison, PhD, is professor emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
For the first 13 years of his career, Dr. Garrison worked in non-profit health policy at Battelle and then the Project HOPE Center for Health Affairs, where he was the Director from 1989-1992. Following this, he worked as an economist in the pharmaceutical industry for 12 years. From 2002-2004, he was vice president and head of Health Economics & Strategic Pricing in Roche Pharmaceuticals, based in Basel, Switzerland.
Dr. Garrison received a BA in Economics from Indiana University, and a PhD in Economics from Stanford University. He has more than 150 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies.
Dr. Garrison was elected as ISPOR President for July 2016-June 2017, following other leadership roles since 2005. He recently co-chaired the ISPOR Special Task Force on US Value Frameworks. He was selected in 2017 by PharmaVOICE as being among “100 of the Most Inspiring People” in the industry. He recently received the PhRMA Foundation and Personalized Medicine Coalition 2018 Value Assessment Challenge First-Prize Award as lead author on a paper on “A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine.”
17:00 - 18:00
Cost-Effectiveness Modelling of Gene Therapies: Challenges and Solutions
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session focuses on advancing economic modelling approaches for high-cost, complex therapies, particularly in rare diseases and advanced treatments. Presentations include a systematic review of modelling challenges and solutions for rare disease technologies, early economic modelling to estimate the cost-effectiveness and value-based price of gene therapy for urofacial syndrome, and insights from NICE and CADTH-AMC appraisals of CAR-T cell therapies. The session concludes with an evaluation of the cost-effectiveness and budget impact of gene therapy for sickle cell disease in the Netherlands.
Cost-Effectiveness and Budget Impact of Gene Therapy for Eligible Patients With Sickle Cell Disease in the Netherlands
OBJECTIVES: Gene therapy offers promising benefits for patients with sickle cell disease (SCD) but also raises questions regarding affordability. This study aimed to evaluate the cost-effectiveness and budget impact of gene therapy compared to comprehensive care in patients with severe SCD genotypes (HbSS or HbSβ⁰/β⁺) aged ≥12 years with severe disease-related symptoms.
METHODS: We conducted a cost-effectiveness analysis (CEA) using a Markov state-transition model from a Dutch societal perspective over a lifetime horizon. Efficacy and safety data for gene therapy were sourced from the HGB-206 (NCT02140554) and HGB-210 (NCT03499818) clinical trials. Additional data on clinical outcomes, quality of life (QoL), and costs were obtained from the HGB-206 and HGB-210 trial, and scientific literature. Budget impact analysis (BIA) was performed from a healthcare perspective, covering a five-year time horizon. Uncertainty in the CEA and BIA were assessed through probabilistic sensitivity and scenario analysis.
RESULTS: After applying discounting and half-cycle correction, gene therapy yielded 11.70 additional life years, and 13.29 additional quality-adjusted life years (QALYs) compared to comprehensive care. The deterministic incremental cost-effectiveness ratio (ICER) was €123,384 per QALY gained; the probabilistic ICER was €124,972 per QALY gained. Results were sensitive to drug acquisition costs, survival rates, and utility of patients with no or mild vaso-occlusive events in the gene therapy group, and the method used to calculate productivity losses (human capital versus friction period). The budget impact of treating eligible patients with gene therapy over 5 years was projected to be approximately €71 million.
CONCLUSIONS: Gene therapy substantially improves survival and QoL for patients with severe SCD compared to comprehensive care but at a high cost. At the current cost-effectiveness threshold of €80,000 per QALY in the Netherlands, it is not considered cost-effective. Reducing the market price of gene therapy is essential to make it more economically viable and accessible.
A Systematic Review of Economic Modelling Challenges and Proposed Solutions for Rare Disease Technologies: An HTAi RDIG Initiative
OBJECTIVES: Rare diseases present distinct challenges for health technology assessment (HTA) due to limited evidence, small patient populations, and substantial population heterogeneity. The Health Technology Assessment international (HTAi) society’s Rare Diseases Interest Group (RDIG) is conducting a systematic literature review to identify novel approaches or adaptations in economic evaluation methods to address these challenges. This work presents initial findings from that review.
METHODS: We searched Ovid (MEDLINE, Embase), the Cost-Effectiveness Analysis Registry, and the Pediatric Economic Database Evaluation for studies published from 2018 onward. We also reviewed HTA websites (NICE, ICER, PBAC, CADTH, SMC, IQWiG). Two reviewers independently screened titles/abstracts and full texts, and a third reviewer resolved disagreements. A predefined template for data extraction was used. The review was registered in PROSPERO (CRD42024522132), and we followed the PRISMA 2020 and CADTH PRESS guidelines.
RESULTS: The initial search yielded 1314 records. After screening, 124 full-text articles were assessed for eligibility, and 23 studies were included in the final synthesis. Among the methodological challenges related to input and outcome parameters were limited natural history data, small sample sizes, lack of head-to-head trial data, reliance on single-arm designs, and heterogeneity or imbalance in trial populations, affecting representativeness and comparability. Additional challenges in modeling rare pediatric diseases included age restrictions on utility measures, difficulty collecting quality-of-life data outside trials, limited published health related quality-of-life data, and uncertainty from sparse data and poorly understood disease trajectories. While a few studies applied approaches such as customized matching, utility and hazard mapping, survival curve reconstruction, and expert elicitation, the application of novel methods remains limited.
CONCLUSIONS: While the methodological challenges of economic evaluation for rare disease technologies are widely acknowledged, our review highlights a gap between awareness and implementation of methodological innovation.
Early Economic Modelling to Estimate Cost-Effectiveness and Value-Based Price of a Gene Therapy for Urofacial Syndrome
OBJECTIVES: Many rare conditions are caused by genetic variants, and gene therapy offers a potential one-time curative treatment. Urofacial syndrome is a rare but devastating disorder featuring an inability to empty the urinary bladder, leading to recurrent urinary infections and kidney failure. It is usually caused by biallelic HPSE2 variants. A recent preclinical gene therapy model shows promise. Understanding its economic viability is essential to support further research and development. This study estimated the value-based price (VBP) of a hypothetical gene therapy for urofacial syndrome, and its cost-effectiveness compared with standard of care from a UK healthcare system perspective.
METHODS: An early economic evaluation used a Markov model to estimate lifetime costs(£ price-year 2024) and quality-adjusted life years (QALYs, EQ-5D-3L UK tariff) for gene therapy versus standard care. Gene therapy was modelled as a one-time curative intervention. VBP was calculated using a £100,000 per QALY threshold, following the NICE Highly Specialised Technologies decision-making criteria. One-way sensitivity analysis identified key drivers of cost-effectiveness. Scenario analyses were performed to examine the impact of varying assumptions around treatment effectiveness on the estimated VBP.
RESULTS: Gene therapy gained a mean lifetime QALYs of 21.26 (19.02-22.57) versus 16.67 (13.63-18.62) for standard care, with an incremental gain of 4.59 QALYs (3.16-7.26). Standard care costs were £336,166 (£248,176-£402,039), while VBP of gene therapy was estimated to be £795,008 (£597,816-£1,076,718). Scenario analyses indicated that under assumptions regarding the duration of treatment effectiveness, the VBP reduced between £262,902 and £750,549. For the base case VBP, probabilistic analysis indicated that the probability of a gene therapy being cost-effective was 42%.
CONCLUSIONS: This early health economic evaluation estimated the cost-effectiveness and value-based price of a gene therapy under development for urofacial syndrome. The sensitivity analyses highlight critical parameters that influence economic viability and identify priority areas for future research to reduce decision uncertainty.
Cost-Effectiveness Modelling of CAR-T Cell Therapies in Hematologic Malignancies: Insights From NICE and CDA-AMC Appraisals
OBJECTIVES: Chimeric antigen receptor T-cell therapies (CAR-Ts) have emerged as promising treatments for relapsed or refractory hematologic malignancies. They are usually associated with a high up-front cost and a complex manufacturing and administration process. This study aims to explore the approaches taken to model the cost effectiveness (CE) of these therapies in drug submissions to UK’s National Institute for Health and Care Excellence (NICE) and Canada’s Drug Agency (CDA-AMC).
METHODS: All publicly available NICE and CDA-AMC model appraisal reports up to June 2024 were identified from which information on CE model structures, inputs, underlying assumptions, model outcomes and evidence review group critiques were extracted and assessed.
RESULTS: Seventeen appraisals were identified (NICE: 7, CDA: 10). All models were 3-health-states Partitioned Survival Models (PSMs), six with a precedent decision-tree, and twelve included a mixture-cure component to extrapolate the overall survival (OS) to reflect potential cure for a patient subset. The submitted prices ranged from £282,000 to £297,000, and from C$485,021 to C$632,455 per administration, resulting in incremental cost-effectiveness ratios (ICERs) in the range of £20,000 to £60,000, and from C$58,178 to C$216,606 per quality-adjusted life year (QALY), for UK and Canada respectively. For England a single tariff to capture the costs of delivering CAR-Ts of £58,964 has now been established. Uncertainty in the OS long term survival benefit was the key limitation discussed across all appraisals. Despite the methodological challenges, most appraisals (15 of 17) led to positive reimbursement decisions, conditional on price reductions, collection of additional data and/or restricted to administration at specialized centres.
CONCLUSIONS: Standard economic modelling approaches in oncology (PSMs) were deemed adequate by NICE and CDA-AMC to inform reimbursement decisions for CAR-Ts. Further research into the long-term benefits of CAR-Ts is needed to determine whether new, non-conventional modelling approaches are warranted to accurately capture their value.
How Can We Make Innovative Payment And Pricing Schemes a ‘Win’ From All Perspectives?
Session Type: Issue Panel
Topics: Health Technology Assessment, Real World Data & Information Systems, Economic Evaluation
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue: Innovative payment and pricing schemes have been proposed as a solution for allowing patient access in situations where payers have concerns about uncertainty in the costs and outcomes of new health technologies, or their affordability. However, these schemes are difficult to implement in practice, in part due to the challenges in aligning the interests of the various stakeholders. Potentially these schemes are a ‘win’ for all parties. The financial risk to payers is reduced, manufacturers can sell their products, and patients gain earlier access to therapies. Yet the main stakeholders must have concerns or else it would be easier to implement schemes. The purpose of this panel is to explore in depth the barriers and enablers of innovative schemes, with a view to identifying key actions that can be taken to increase their likely success. The discussion will be informed by the findings of the recent EU Horizon Europe research project on Health Innovation Next Generation Payment and Pricing Models (HI-PRIX).
Overview: Michael Drummond (5) minutes) will moderate the session and give a brief overview of the current landscape surrounding innovative payment and pricing schemes. Then Mikel Berdud (15 minutes) will report the main findings from stakeholder surveys and a nominal group exercise exploring barriers and enablers, undertaken as part of the HI-PRIX project. This research covered the perspectives of manufacturers, HTA bodies, payers, providers, academics, patients and policy makers. Maria Cavaller (10 minutes) will then discuss in more detail the issues and concerns from the perspective of patients, especially those with rare diseases. Then, Ilaria Villa (10 minutes) will outline the issues and concerns from a manufacturer perspective, particularly smaller companies with large stakes in the products concerned. Finally, following a general discussion (15 minutes), involving the audience, some key actions to improve the likely success of schemes will be identified (5 minutes)
Moderator
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Michael Drummond, MCom, DPhil
University of York, York, United Kingdom
Michael Drummond is Professor Emeritus and former Director of the Centre for Health Economics at the University of York in the United Kingdom. His main field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 750 scientific papers. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. He served for 14 years as Co-Editor-in-Chief of Value in Health and was made Editor Emeritus in May 2024. He has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
Speakers
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Mikel Berdud, PhD
Office of Health Economics, London, United Kingdom
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Ilaria Villa, MSc
Fondazione Telethon, Milan, Italy
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Kate Morgan, MSc
Brussles, Belgium
Optimal Strategies for Predicting and Consolidating PICOs: One Best Method or a Blended Approach?
Session Type: Issue Panel
Topics: Health Technology Assessment
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE:
The PICO (Population, Intervention, Comparator, Outcome) framework, essential for structuring clinical research questions in systematic literature reviews (SLRs), plays a central role in the EU Joint Clinical Assessment (JCA) under the EU Health Technology Assessment (HTA) Regulation. However, scoping PICOs in this context is complex, as it requires incorporating input from all 27 Member States, leading to numerous PICOs, and an overwhelming amount of literature to review. This resource-intensive process highlights the challenge of predicting and consolidating PICOs reflecting both shared and local priorities.
To address these challenges, alternative approaches including country surveys and AI-driven PICO prediction using retrospective HTA and clinical data are being explored. While SLRs remain foundational, they often lack efficiency. This has sparked debate over whether one optimal method or a combination of tools tailored by therapeutic area or timing is more effective. The goal is to streamline evidence gathering, reduce duplication, and support coordinated decision-making across Member States within the evolving EU HTA framework.
OVERVIEW:
This panel addresses the challenges of PICO prediction within the EU-level JCA framework. Dr. Vo will provide a 5-minute overview of the issue, followed by three panelists representing an HTA country, academia, and industry, each sharing their perspectives (5 minutes each) on the importance and approach to tackling the PICO prediction dilemma. The debate (21 minutes) will be guided by three key questions (5 minutes each). Interactive polling will capture audience viewpoints.
Panelists will explore three methods to streamline PICO development: SLRs (offer evidence-based structure but are time-consuming); country surveys (bring national specificity but may lack standardization); and AI tools enhanced with expert input (promise speed and efficiency but require reliable data). The discussion will weigh the strengths and limitations of each, offering insights for JCA subgroups, HTA decision-makers, and other stakeholders seeking to optimize PICO formulation across diverse healthcare systems.
Moderator
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Pamela Vo, MS, PharmD
EVERSANA, Basel, Switzerland
Speakers
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Michal Stanák, MA, DPhil
National Institute for Value and Technologies in Health Care, Bratislava, Slovakia
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Zsombor Zrubka, MBA, PhD, MD
Óbuda University, Budapest, Hungary
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Michael Happich, DrPH, PhD
Eli Lilly, Bad Homburg, Germany
A New Era for EU HTA: What Can the EU HTAR Learn from the EMA’s Path to Harmonization?
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue
The EU HTA Regulation (EU HTAR) marks a significant step in harmonizing HTA across Europe. While aiming to reduce duplication and improve timely access, its implementation raises questions about feasibility, stakeholder alignment, and national uptake. This panel explores what the development of the European Medicines Agency (EMA), a comparable effort in EU-level harmonization three decades earlier, can teach us about the opportunities and challenges facing the EU HTAR.
Overview
The session begins with a presentation (±10min) of a comparative analysis by the researchers (Francine Brinkhuis/Wim Goettsch), examining the development of the EU HTAR alongside the EMA’s historical trajectory. Both initiatives were driven by a need for greater EU-level coordination but differ in mandate, legal authority, and integration into national systems. Nevertheless, both began with informal collaboration and faced similar challenges in aligning diverse national systems, offering a valuable basis for learning.
Drawing on this analysis, the panel will explore which elements of the EMA’s experience might inform EU HTAR implementation, which pitfalls to avoid, and where HTA may require a fundamentally different approach. Each panelist will briefly present their perspective (policy, regulatory, and industry, ±20min total). The panel will then engage in a structured debate and interactive audience discussion (±30min), moderated by Wim Goettsch. Polling tools will be used to gather audience input and stimulate real-time discussion between audience and panelists: How do today’s political and technological contexts shape HTA harmonization differently from the EMA’s path? Should JCAs take on a more binding role?
The aim is to foster open exchange, highlight areas of consensus and disagreement, and reflect on trade-offs shaping the EU HTAR’s long-term success. The session will interest regulators, policymakers, HTA bodies, industry, and academics seeking insight into how past institutional experiences can inform the ongoing implementation of the EU HTAR.
Moderator
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Wim Goettsch, MSc, PhD
Utrecht University; Zorginstituut Nederland, Utrecht; Diemen, Netherlands
Speakers
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Neil McAuslane, BSc, MSc, PhD
Centre for Innovation in Regulatory Science, Glasgow, United Kingdom
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Anja E Schiel, PhD
Norwegian Medicines Agency, Oslo, Norway
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Ansgar Hebborn, PhD
Roche Products Ltd, Basel, Switzerland
Measuring Health-Related Quality of Life Across the Lifespan
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session explores innovative approaches to measuring quality of life, well-being, and preferences across diverse populations and health contexts. Presentations include an empirical comparison of subjective well-being and health-related quality of life measures in Australian adolescents, quality of life and utility values across the Alzheimer’s disease spectrum, and a discrete choice experiment eliciting preferences for combination respiratory virus vaccines among US adults aged 50+. The session concludes with an evaluation of EQ-5D-5L and EQ-HWB for loneliness research.
Measuring Outcomes for Loneliness Research: Examining the Appropriateness of the EQ-5D-5L and EQ-HWB
OBJECTIVES: Loneliness is a global public health concern. This study aimed to (i) estimate utility values associated with different levels of loneliness using the EQ-5D-5L and EQ-HWB measures, (ii) evaluate their ability to differentiate between loneliness levels, and (iii) examine conceptual overlap between these measures and a validated loneliness scale.
METHODS: Data from an online survey comprising a nationally representative cross-sectional survey of Australian adults was used. The survey included information on demographics, the 20-item Revised UCLA Loneliness Scale (R-UCLA-LS), EQ-5D-5L, and EQ-HWB. The Kruskal-Wallis test was used to assess the statistical differences in utility scores from the EQ-5D-5L and EQ-HWB-Short, and also the EQ-HWB sum score, across three levels of loneliness (Low/Medium/High) as measured by the R-UCLA-LS. Exploratory factor analysis (EFA) was used to explore the conceptual overlap between the EQ-5D-5L and EQ-HWB with the R-UCLA-LS item measure. Additionally, ordinary least squares (OLS) regression was conducted to identify predictors of loneliness from the items that overlapped with the R-UCLA-LS.
RESULTS: 2072 respondents completed the survey. Both the EQ-5D-5L and EQ-HWB measures significantly differentiated between loneliness levels (p < 0.001). The mean EQ-5D-5L utility scores were 0.89 for low loneliness, 0.81 for moderate loneliness, and 0.72 for moderate to high loneliness. The EQ-HWB-S utility scores were 0.88, 0.75, and 0.58, respectively, showing greater sensitivity to the burden of loneliness. EFA revealed no overlap between EQ-5D-5L items and the R-UCLA-LS, while five EQ-HWB items overlapped with the R-UCLA-LS, including the “feel lonely” item, which is also part of the EQ-HWB-S. Regression analysis explained 53.58% of the variance in loneliness scores (F(10, 1463) = 168.87, p < 0.001), with “feeling lonely” and “feeling unsupported” contributing the most to predicting loneliness.
CONCLUSIONS: The EQ-HWB-S, in particular, shows promise as a brief yet sensitive tool for capturing loneliness-related burden and may be especially useful in interventions targeting loneliness.
Quality of Life and Utility Values Across the Alzheimer’s Disease Spectrum: Results From the What Matters Most Survey
OBJECTIVES: The What Matters Most (WMM) in Alzheimer’s disease (AD) research program has explored treatment-related needs, preferences, and priorities of people at risk or living with Alzheimer’s disease (PLWAD) and their care partners. To address an important data gap, estimates of health-related quality of life (HRQOL) and health utility were assessed across the full spectrum of disease in AD.
METHODS: As part of a prospective, cross-sectional survey, the EQ-5D-5L and Quality of Life in AD (QOL-AD) instruments were administered to participants in the United States (US) spanning 5 severity groups, including individuals at risk of or with preclinical AD, with mild cognitive impairment (MCI), or with mild AD, and care partners of individuals with moderate or severe AD. Recruitment targeted equal participation across groups and ≥50% people of color within each group. EQ-5D (domains, health utility) and QOL-AD (domains, total) scores were analyzed descriptively by group, and least squares analysis tested the significance of EQ-5D utility score differences across groups.
RESULTS: A total of 640 participants (375 PLWAD through mild AD; 265 care partners reporting on behalf of PLWAD with moderate or severe AD) completed the survey. Results consistently indicated that HRQOL impacts measured by EQ-5D and QOL-AD domains increased with AD severity. EQ-5D utility scores (mean [standard deviation]) for those at risk (0.75 [0.24]), with MCI (0.69 [0.19]), and with mild AD (0.67 [0.22]) were significantly higher (reflecting better HRQOL) than for those with moderate AD (0.53 [0.28]) and severe AD (0.40 [0.34]).
CONCLUSIONS: This study contributes to the existing literature on HRQOL in AD by providing evidence across the full AD spectrum that HRQOL consistently worsens on generic and AD-specific domains as severity increases. These results provide evidence of fundamental differences in the lived experience of AD across groups and can be used in future economic evaluations of new AD treatments.
Eliciting Preferences for Combination Vaccines Protecting Against Multiple Respiratory Viruses: A Cross-Sectional Discrete Choice Experiment of Adults Aged 50 in the US
OBJECTIVES: Respiratory viruses (RV) including influenza (flu), respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and parainfluenza virus type 3 (PIV3), are major contributors to respiratory diseases in older populations. Older individuals face complex RV vaccination decisions, choosing between specific viral coverage and broader respiratory illness (RI) protection). This study aimed to understand preferences for RV vaccines in a landscape reflecting both standalone and combination RV vaccines.
METHODS: An online discrete choice experiment (DCE) was fielded with adults aged ≥50 years in the United States. The DCE included seven attributes, identified through a targeted literature review and validated using qualitative interviews, including protection against varying severities of different combinations of RIs. Choice data were analyzed using a mixed logit model; outputs included relative attribute importance (RAI), predicted vaccine choice, and attribute trade-offs.
RESULTS: A sample of 803 participants with a mean age of 63.8 years (SD: 8.0) participated. Protection against severe RI had the largest impact on vaccine decision-making (RAI=22.9%), followed by risk of systemic adverse events (AEs; RAI=21.2%), protection against mild-moderate RI (RAI=21.0%), and risk of local AEs (RAI=14.9%). Viruses protected against (RAI=7.5%), number of shots required (RAI=6.6%), and duration of protection (RAI=6.0%) were less important. An RSV/hMPV combination vaccine was preferred over an RSV standalone vaccine (65% vs. 35%), given efficacy and safety assumptions for each and provided alongside a standalone flu vaccine. Participants were willing to accept two-shot vaccines versus one-shot vaccines in exchange for an increase in protection against RI (+9.3% mild-moderate; +8.9% severe); or a decrease in the risk of side effects (-15.0% local; -10.7% systemic).
CONCLUSIONS: Combination respiratory vaccines, which offer higher overall protection against RI, are valuable to adults aged ≥50 years, underscoring their potential to simplify vaccination schedules and improve coverage, ultimately reducing the burden of RI in this vulnerable demographic.
Empirical Comparison of Subjective Well-Being and Health-Related Quality of Life Measures in Australian Adolescents
OBJECTIVES: The distinction between subjective well-being (SWB) and health-related quality of life (HRQoL) remains conceptually ambiguous in the literature. While HRQoL predominantly focuses on health functioning, SWB encompasses a broader evaluation of life, extending beyond health-specific domains. Despite some conceptual overlap, these constructs are not interchangeable. This study aims to explore the empirical relationships between SWB, measured using a new preference-based Disability Wellbeing Index (DWI), and HRQoL, measured by the Child Health Utility 9D (CHU9D) and EQ-5D-5L (with psychosocial bolt-ons), among Australian adolescents aged 15 to 19 years.
METHODS: A nationwide, quota-based online survey was administered to Australian adolescents aged 15 to 19 years. The survey included the DWI, CHU9D, EQ-5D-5L with psychosocial bolt-ons, and a series of sociodemographic questions. Psychometric analyses, including exploratory factor analyses (EFA), were conducted to examine the underlying structures and interrelationships among these instruments.
RESULTS: Self-reported data were collected from 1,133 adolescents (45% female). Among them, 582 completed the CHU9D, and 585 completed the EQ-5D-5L and psychosocial bolt-ons; all adolescents completed the DWI. Only one CHU9D dimension (“sad”) and one EQ-5D-5L dimension (“anxiety”) showed strong correlations (Spearman’s ρ > 0.5) with the “mental health” domain of the DWI. EFAs further revealed limited overlap in latent constructs between the DWI and HRQoL measures, revealing that SWB and HRQoL measures capture distinct yet complementary aspects of adolescent health and well-being.
CONCLUSIONS: SWB and HRQoL instruments are not substitutes but provide complementary insights. Incorporating both types of measures in health economic evaluations may offer a more comprehensive understanding of adolescents’ overall well-being.
Increasing Transparency of Public Funding in Pharmaceutical Research and Development Reporting
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Methodological & Statistical Research, Epidemiology & Public Health
Level: Introductory
Available On-Demand: Digital Conference Pass
ISSUE: Escalating prices of innovative pharmaceuticals present challenges to healthcare systems worldwide. Meanwhile, current industry practices—including extensive share buybacks, strategic M&As, and reliance on public institutions to populate early R&D pipelines—raise questions about the true economics of innovation. Public contributions to pharmaceutical innovation remain systematically undervalued in pricing and reimbursement discussions.
A lack of transparency and under- or overreporting of R&D expenditures lead to issues in decision making. Both the WHO and the proposed revision of the European pharmaceutical legislation call for more transparency.
This session will explore practical recommendations for stakeholders to advance a more transparent, balanced approach to pharmaceutical innovation that serves both industry sustainability and public health needs.
OVERVIEW: Transparency in pharmaceutical research and development reporting is a complex issue that can be addressed from different angles. This panel will enrich the debate bringing to the same table speakers coming from: a national payer organisation, a national HTA body and a European Pharmaceutical Industry umbrella organisation.
The panel will begin with an overview provided by Daniel Fabian in approximately 10 minutes on a framework of analysis of public contributions. This research was funded by EC-grant agreement number 101095593 “HI-PRIX” to establish a framework of analysis for public contribution. A comprehensive taxonomy of these contributions includes direct funding mechanisms, infrastructure and capability development, and substantial academic and research institution support. The framework has been piloted on individual, and groups of products and is now being used in the Austrian appraisal board for pharmaceuticals.
Policy discussions this panel will address include the role of fair pricing models to acknowledge public contributions to pharmaceutical R&D, the potential impact of increased transparency in facilitating informed negotiations, and how that all could contribute to building a more predictable and plane level field for innovators and decision makers across Europe alike.
Moderator
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Jaime Espin, MSc, PhD
EASP, Granada, Spain
Speakers
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Daniel Fabian, MSc
Vienna, Austria
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Americo Cicchetti, BSc, MASc, DPhil
UNIVERSITA’ CATTOLICA DEL SACRO CUORE, Rome, Italy
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François Bouvy, -
EFPIA, Brussels, Belgium
Survival Analysis in the Era of Generative AI
Session Type: Other Breakout Session
Topics: Methodological & Statistical Research, Clinical Outcomes
Level: Intermediate
Available On-Demand: Digital Conference Pass
Purpose
Survival analysis, and parametric survival curve selection in particular, is a critical yet challenging component in health economic modeling, often introducing significant uncertainty in cost-effectiveness analyses. This panel discusses the application of Generative AI (GenAI) to support human decision making, by comprehensively assessing both trial and external data sources. The session aims to bridge the technical innovation in GenAI with practical applications in health economics, presenting evidence-based insights and exploring the implications for HTA and industry practices.
Description
The session begins with an introduction by moderator Sven Klijn (10’) to the challenges of parametric survival curve selection and the potential of GenAI to address these limitations. Will Rawlinson from Estima Scientific will discuss the technical innovation in GenAI (15’), covering architecture and methodology of the GenAI system, including how it processes complex trial data alongside external sources to recommend optimal curve selections. He will also discuss the role of the human and implementation aspects of the Human/AI feedback loop. Prof. Nick Latimer (University of Sheffield and Petauri Evidence), a world-leading expert in survival analysis, will critically evaluate the results that can be achieved through this approach (15’), highlighting comparative advantages over conventional human-only methods while addressing potential limitations. Siguroli Teitsson (BMS) will conduct a broader examination of implications for survival analysis for HTA, practical implementation considerations, and future directions for this technology (10’). Following these presentations, the session concludes with an interactive audience discussion, addressing key concerns regarding transparency, reliability, and clinical plausibility of AI-assisted curve selection methodologies.
This session will be particularly valuable for health economists, outcomes researchers, HTA bodies, and industry professionals seeking to understand how GenAI can be responsibly integrated into established methodological frameworks to enhance decision-making quality.
Moderator
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Sven L Klijn, MSc
Bristol Myers Squibb, Princeton, NJ, United States
Sven Klijn is director at Bristol Myers Squibb in the Global HEOR Economic & Predictive Modeling group, where he leads the innovative modeling agenda in hematology and cell therapy. In addition, Sven has an active role in providing modeling education and masterclasses at international congresses. He has widely published on innovative methods, especially in the field of survival extrapolation and Generative AI. Sven has training in public health and health economics and previously had various roles in CROs related to health economic modeling.
Speakers
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William Rawlinson
Estima Scientific, London, United Kingdom
Will is a senior health economist at Estima Scientific holding a degree in Physics and Philosophy from the University of Oxford. Will has 4 years’ experience developing cost-utility models and has specialized in applications of generative AI to health economic modelling. Will has published on the automation of R modelling using large language models (LLMs), and more recently has focused on applications of LLMs to Excel modelling and model reporting.
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Nicholas Latimer, MSc, PhD
SCHARR, University of Sheffield, Sheffield, 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 2019, Nick began a Senior Research Fellowship funded by Yorkshire Cancer Research in which he is investigating 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.
https://www.sheffield.ac.uk/scharr/sections/heds/staff/latimer_n
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Siguroli Teitsson, BSc, MSc
Bristol Myers Squibb, Denham, United Kingdom
Siguroli Teitsson is a Director in Global HEOR Economic & Predictive Modeling at Bristol Myers Squibb. In his role, Siguroli leads the advancement of innovative modeling and analytics in oncology, and drives the integration of cutting-edge AI automations in HEOR and market access, streamlining workflows to accelerate patient access to medicines. With a background in engineering and health economics, he has previously held senior roles in CROs and has extensive publication record in innovative analytics within the field of HEOR, contributing to advancements in methodology and practice.
Mind the Gaps: Managing Missing PRO Data in the Era of JCA for HTA Submissions
Session Type: Issue Panel
Topics: Patient-Centered Research, Methodological & Statistical Research, Health Technology Assessment
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue: A fundamental precept of medical practice is that treatments should align with patients’ needs, supported by adequate information to guide decision-making. Patient-reported outcome (PRO) data collected during clinical trials provides critical insights into patients’ experiences, allowing them a voice in the evaluation of treatments. Given the effort required to collect and report PROs—particularly as illness progresses—it is important to recognise this burden, which may lead to missing data and potential bias. It is consequently incumbent on analysts and assessors to use PRO data effectively while accounting for missingness.
Overview: Taking account of the importance of PRO data, this issue panel will discuss how to appropriately analyse PRO data accounting for missing data and the need for guidelines. Although analytic methods for handling missing data have advanced, guidance on their application in health technology assessments (HTAs)—including the EU Joint Clinical Assessment (JCA)—remains limited. It will address technical and practical challenges, draw on lessons from previous HTA assessments, and consider implications for the JCA. The audience will be invited to share perspectives on their experience of analysing PRO data and the necessity and scope for further guidelines around the appropriate analysis of missing data.
Miranda Cooper will outline challenges in handling missing PRO data from a health technology developer’s perspective (10-minutes). Professor Neil Hawkins will review recent NICE oncology appraisals, focusing on approaches used to handle missing PRO data, HTA agency feedback, and outcomes from reviewers and committees (15-minutes). Professor Olivier Chassany will highlight varying evidence requirements for PROs across major European HTA bodies in oncology, underscoring the need for harmonisation for HTA agencies and applicants (15-minutes). Professor Gianluca Baio will present methodological approaches to handle situations where substantial amount of missingness is present, with particular relevance to PRO data accounting for the longitudinal nature of the data (15-minutes).
Moderator
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Miranda Cooper, BSc, MSc
AstraZeneca, Barcelona, Spain
Speakers
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Neil Hawkins, MBA, MSc, PhD
University of Glasgow, Oxford, United Kingdom
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Olivier Chassany, PhD, MD
Assistance Publique–Hôpitaux de Paris (AP-HP), Paris, France
Professor of Therapeutics (Health Economics Clinical Trial Unit, AP-HP Paris hospitals, France), clinician, specialist in gastroenterology, with a long experience in developing Patient-Reported Outcomes (PRO) questionnaires. Involved for more than 20 years in dossiers expertise for EMA and French Drug Agency and over 32 years in Ethics Committees. Co-author of the EMA Reflection Paper on Health-Related Quality of Life. Involved in HTA dossiers expertise for pharma/CRO. Teaching Therapeutics and Methodology of clinical trials at Université Paris Cité. Past chair of the ISPOR SIG Clinical Outcomes Assessment (COA).
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Gianluca Baio, PhD
University College London, London, United Kingdom
Gianluca is professor of Statistics and Health Economics in the Department of Statistical Science at University College London. He graduated in Statistics and Economics from the University of Florence, Italy and then completed a PhD program in Applied Statistics again at the University of Florence, after a period at the Program on the Pharmaceutical Industry at the MIT Sloan School of Management, Cambridge (USA). He worked as a research fellow and then Lecturer in the Department of Statistical Sciences at University College London, UK. His main interests are in Bayesian statistical modelling for cost effectiveness analysis and decision-making problems in the health systems, hierarchical/multilevel models and causal inference using the decision-theoretic approach. Gianluca leads the Statistics for Health Economic Evaluation research group within the department of Statistical Science, whose activity revolves around the development and application of Bayesian statistical methodology for health economic evaluation, eg, cost-effectiveness or cost-utility analysis. He also collaborates with the UK National Institute for Health and Care Excellence (NICE) as a scientific advisor on Health Technology Appraisal projects. He has developed and maintains several R packages, many specifically devoted to health economic evaluation, including BCEA, survHE and missingHE.
Adding Nature’s Petal to the Value Flower: Can Inclusion of Environmental Impact in HTA Aid the Race to Net Zero?
Session Type: Issue Panel
Topics: Health Technology Assessment, Real World Data & Information Systems
Level: Introductory
Available On-Demand: Digital Conference Pass
Issue
Emissions from healthcare systems contribute heavily to climate change, which in turn has the potential to undo decades of progress in health. In recognition of this vicious cycle, many healthcare systems have entered the race to net zero, with HTA agencies around the world debating their role in supporting these efforts. These considerations are relevant in the context of our increasing understanding of ‘whole health’, as ISPOR leads dialogue around the definition of health, and how this may influence the inputs, outcomes and methods of conducting HTA.
Deliberations on how to evaluate environmental impacts of pharmaceuticals are made against a rapidly changing policy landscape, with introductions such as mandatory Environmental Risk Assessment (ERA) impacting launch of new medicines from 2024, and the EU procurement directive which sets the foundation for integrating sustainability in public procurement decisions.
Challenges are also inherent within any potential HTA process. There remains a lack of harmonised methods for collection and analysis of environmental impact data within HTA, as well as a lack of consensus on how this data can or should practically be used within reimbursement decision making.
Several questions must be explored: What role can HTA play in supporting efforts to reduce the environmental impacts of health systems? How will it interact with other incentives, such as regulation and policy? Can data collection and analysis be standardised? If so, how? Are there cases in which environmental impact should be explicitly deprioritised?
Overview
Following an introduction to the issues by the moderator (4 mins), the panel will debate the questions above. Panellists will each speak for 12 minutes, providing their perspectives on how use of environmental impact data will aid the pursuit to net zero, and debating cases in which it will/will not be important for decision makers. 20 minutes will be reserved for audience discussion.
Moderator
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Grace Hampson, MSc
Office of Health Economics, London, United Kingdom
Speakers
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Hector Tejero, PhD
Gabinete de la Ministra de Sanidad, Madrid, Spain
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Andrew Briggs, DPhil
London School of Hygiene & Tropical Medicine, London, United Kingdom
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Isabell Carmela Crasto De Stefano, MA
CHIESI FARMACEUTICI, Parma, Italy
A Match Made in Heaven? Optimal Selection of Preference Elicitation Methods to Address Study Purpose and Objectives
Session Type: Workshop
Topics: Patient-Centered Research, Methodological & Statistical Research, Study Approaches
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
Selection of the most appropriate method to capture patient treatment preferences is fundamental to support the consideration of patient priorities in decisions across the medical product lifecycle (MPLC). The discrete choice experiment (DCE) is the most commonly applied method in health but its application is often limited by the comparatively large sample size required. However, whilst extensive guidance and many training resources are available on DCEs, there are few resources to support the selection of non-DCE methods alongside DCEs.
This workshop aims to provide insights on the selection of preference elicitation methods to inform decisions along the MPLC, the extent to which different methods address different research objectives, and when to consider the use of non-DCE methods.
Jennifer Whitty will provide an introduction to preference elicitation method selection, poll the audience on their knowledge of DCE and non-DCE methods and moderate the workshop.
Michael Bui will present evidence from a systematic review suggesting suboptimal selection of preference elicitation methods across some studies.
Jorien Veldwijk will present methods identified as promising to elicit preferences for MPLC decision making, highlighting their application and gaps in existing guidance. She will discuss the outcomes of a study among MPLC stakeholders about importance of different method’s characteristics for decision-making.
Francesco Pignatti will focus on method selection for research questions to inform regulatory decisions, drawing from experience with submission of preference studies to support the evaluation of new drug applications to the European Medicines Agency.
Audience feedback will be sought (via poll and interactive discussion) on method selection across the MPLC to inform public consultation around the forthcoming ICH E22 guidance on Considerations for Patient Preference Studies.
Moderator
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Jennifer Whitty, PhD
Evidera, London, United Kingdom
Speakers
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Michael Bui, MSc
University of Twente, Enschede, Netherlands
Michael Bui is a PhD candidate at the Health Technology and Services Research section. He has a BSc degree in Creative Technology (cum laude), and two MSc degrees in Interaction Technology (cum laude) and Electrical Engineering (biomedical specialisation). His master's assignment received the Graduation Award for the best thesis of the Faculty of Electrical Engineering, Mathematics and Computer Science.
In his current work, he develops (statistical) methods to transfer findings from past patient preference studies to applications beyond their original scope. He examines how existing evidence can inform patient-relevant endpoint selection for clinical trial design, particularly when time or budget constraints prohibit de novo collection of preference data. His goal is to incorporate the patient perspective into medical product development, while contributing to sustainability of past research efforts.
He is a board member of the Dutch-Flemish Health Economics Association (VGE), and a member of the special interest group for health preference research of the Professional Society for Health Economics and Outcomes Research (ISPOR).
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Jorien Veldwijk, BSc, MSc, PhD
Erasmus University, Rotterdam, Netherlands
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Francesco Pignatti, MD
European Medicines Agency, Amsterdam, Netherlands
18:00 - 19:00
Networking Reception (Exhibit Hall)
Session Type: General Meeting
Wed 12 Nov
7:00 - 8:00
Morning Coffee Service
Session Type: General Meeting
7:00 - 13:00
Registration Hours
Session Type: General Meeting
8:00 - 9:00
Rethinking Access Frameworks for the Next Generation of Multi-Indications/I&I Therapies
Session Type: Issue Panel
Topics: Health Technology Assessment, Economic Evaluation
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE:
The Inflammation & Immunology (I&I) therapeutic area is a rapidly evolving landscape. Driven by innovation and advances in the understanding of immune responses, development efforts are leading to a broad range of new treatments. These include biologics such as monoclonal and bispecific antibodies targeting interleukins, orally administered small molecules that inhibit key proteins like Janus Kinases, and next-generation cytokine modulators.
These innovative therapies will allow for more precise targeting, improved clinical outcomes and, ultimately, better patient health. However, they also raise complex questions for both pharmaceutical companies and payers in defining appropriate access conditions. These include for example the management of multi-indication assets, combination therapies, the recognition of novel benefits (e.g., remission), the validation of new endpoints, the segmentation of target populations, and the emergence of new treatment lines.
Finding the right balance to ensure that the most promising innovations reach the appropriate patients will require a thorough understanding of the evolving I&I landscape, along with the constraints and priorities of all stakeholders.
OVERVIEW:
The session will open with an introductory overview by Stéphane Bazoche or Jessica Weddle (first 15 minutes). While much has been written about scientific and commercial trends in I&I, the impact of innovation on market access remains underexplored—despite the significant challenges it raises for both industry and payers.
This panel will examine the evolving value framework for I&I therapies, highlighting the key dimensions of value demonstration that will require attention in the years ahead to ensure appropriate and sustainable patient access. Topics will include the implications of clinical differentiation, the assessment of novel outcomes, and the policy and HTA shifts needed to keep pace with scientific progress.
The session will be particularly relevant to Market Access and HEOR professionals, especially those engaged in the I&I space, who seek to better anticipate and navigate the access environment for next-generation therapies.
Moderator
Speakers
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nazanin MEHIN, Pharm D
Sanofi, Boston, MA, France
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Dao Tu Xuan, PharmD, MSc
Takeda Pharmaceuticals U.S.A., Cambridge, MA, United States
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Oriol Solà-Morales, MSc, PhD, MD
Fundacio HITT, Barcelona, Spain
Oriol has built a career in the planning, policy and decision-making environment (Regional HTA Agency director (2007-2011); Innovation and Strategy Director in several hospitals, member of the Regional Research Advisory Council and policy advisor to 2 Health Ministers). In 2011, he founded HITT, which transformed in 2019 into the HiTT Foundation to strive for innovation in sustainable healthcare.
He is an assistant professor in Pharmacology and Health Economics at Universitat Internacional de Catalunya, teaching several courses nationally and internationally and a Research Fellow at the OHE (office for Health Economics). Oriol is an MD specialized in Internal Medicine, has earned an MSc from the London School of Economics and Political Science (LSE) and the London School of Hygiene and Tropical Medicine (LSHTM), and a PhD from Universitat Rovira i Virgili (URV).
Traditional And Emerging Methods for Including Caregiver’s Health-Related Quality of Life in Economic Evaluation: Can We Reconcile Modelling Assumptions With Caregivers’ Experiences?
Session Type: Issue Panel
Topics: Patient-Centered Research, Health Technology Assessment, Methodological & Statistical Research
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue: Economic evaluation for health technology assessment (HTA) may include, as an outcome of relevance, health-related quality of life (HRQoL) for caregivers as well as patients, but there is a lack of consensus on how to do this. Popular methods to model caregiver’s HRQoL are fraught with difficulty and may result in the “caregiver quality-adjusted life year (QALY) trap”, whereby extended patient survival has a negative impact on the incremental cost-effectiveness ratio (ICER) due to losses in caregiver utilities (stemming from the prolonged provision of informal care). Methods are emerging which are designed to overcome some of these limitations, but their assumptions and implications require examination to understand whether they can be reconciled with caregivers’ experiences and the aims of HTA agencies.
Overview: Ron Akehurst will introduce the topic (5 minutes). Fleur Chandler will present a caregiver’s perspective, describing her own experience of caregiving and the views of the caregiving community on how HTA includes caregivers’ HRQoL (10 minutes), informed by her background as a senior HTA practitioner. Becky Pennington will describe traditional methods for modelling caregiver’s HRQoL (caregiver utilities, disutilities, and increments) and emerging methods (multipliers, disentangling “caring for” and “caring about”) designed to overcome some of the limitations of traditional methods (15 minutes). Becky will speak from the perspective of HTA decision-makers using these approaches and also as an academic researcher developing new methods. Erik Landfeldt will present an industry perspective, describing how incorporating bereavement could overcome the caregiver QALY trap (8 minutes). The impacts of new and traditional methods of modelling caregivers’ HRQoL will be compared in a series of case studies (5 minutes). The moderator will welcome questions from the audience and oversee a debate between panellists on the extent to which current and emerging methods can be reconciled with caregiver’s experiences and HTA decision making (15 minutes).
Moderator
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Ron Akehurst, PhD
Lumanity, Sheffield, United Kingdom
Speakers
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Fleur Chandler, MSc
Independent, Middlesex, United Kingdom
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Becky Pennington
University of Sheffield, Sheffield, United Kingdom
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Erik Landfeldt, MSc, PhD
IQVIA, Täby, Sweden
Methodological Developments in Indirect Treatment Comparisons and Network-Meta-Analyses
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session explores early indirect treatment comparison assessments to inform evidence generation planning for joint clinical assessment, using generative AI and retrieval-augmented generation (RAG) to query NICE technology assessments, and simulation-based evaluation of population-adjusted indirect comparison methods. The session concludes with an exploration of generative AI’s ability to interpret and automate reporting of comprehensive network meta-analysis results.
An Early Start: Preliminary Indirect Treatment Comparison Assessments Informing Evidence Generation Planning for Joint Clinical Assessment
OBJECTIVES: Planned head-to-head trials are unlikely to address all PICOs in EU Joint Clinical Assessment (JCA), necessitating indirect treatment comparisons (ITCs). Pivotal trials are often designed primarily to address regulatory requirements, and ad hoc ITCs for HTA are regularly constrained by pivotal trial limitations or data gaps for comparators. Uncertainties in comparative effectiveness can lead to challenges in achieving reimbursement. To mitigate challenges within the context of JCA, we designed a roadmap for preliminary ITC assessments that manufacturers should initiate before Phase 3 to enable JCA-ready ITCs across PICOs.
METHODS: We reviewed HTA Coordination Group methodological and practical guidelines and designed a roadmap of preliminary investigations that manufacturers should conduct prior to and during the pivotal trial(s). The roadmap ensures evidence generation captures the unique value of an intervention while facilitating fit-for-purpose comparative evidence for JCA.
RESULTS: •The roadmap constitutes five key steps: 1. Targeted searches and PICO simulations to characterise treatment pathways and identify ongoing/planned comparator trials 2. Identification of potential treatment effect modifiers and prognostic factors via literature searches and clinical expert input 3. Formal comparisons of trial design, patient populations and reported outcomes, based on available data, to inform the validity of similarity and homogeneity assumptions in future ITCs 4. Recommendations for pivotal trial key design elements to facilitate consistent comparisons across PICOs concerning comparator arm selection, eligibility criteria, concomitant medications, stratification factors for randomisation, outcome variables and timepoints 5. Recommendations for supplementary evidence generation as needed, including real-world evidence-based external comparators •Feasibility assessments and ITCs should be living and adaptable to incorporate emerging comparator data and Phase 3 results to meet the 100-day timeframe between PICO finalisation and JCA submission
CONCLUSIONS: Our roadmap offers a structured approach for manufacturers to develop a stronger data package for JCA, with ITCs that are carefully planned, methodologically sound and suitable across PICOs.
Choosing Appropriate Population-adjusted Indirect Comparison Methods for Health Technology Assessment: A Simulation Study on the Impact of Effect Modifiers Baseline Imbalances and Sample Size
OBJECTIVES: In Health Technology Assessment (HTA), comparative clinical evidence is required for a new intervention. In the EU Joint Clinical Assessment, a large number of comparators of interest and limited availability of head-to-head trials necessitate the submission of indirect treatment comparisons (ITCs). In the case of dissimilar study populations, population-adjusted indirect comparisons (PAICs) adjust for differences in effect modifiers, aiming to improve the validity of the comparison. When individual patient data is available for at least one study in a connected network, EU methodological guidelines recommend various PAIC methods to estimate treatment effects — matching-adjusted indirect comparison (MAIC), simulated treatment comparison (STC), and multilevel network meta-regression (ML-NMR). However, the question remains of which method to choose in a given data situation.
METHODS: We conducted a simulation study to evaluate the performance of PAIC methods in scenarios relevant to HTA submissions. Each simulation represented an ITC between two studies, with the unadjusted Bucher method included as a reference case. Across the simulation scenarios, we varied the degree of baseline differences between populations, the strength of treatment effect modification, the true relative treatment effect, and the study sample size. Methods were evaluated in terms of bias, empirical coverage of 95% confidence intervals, and statistical power.
RESULTS: The simulations provide a structured comparison of PAIC methods relative to the unadjusted ITC, highlighting the impact of baseline imbalances, effect modification, and sample size on performance. Our results indicate a substantial reduction in bias with any method, while statistical power is below typical standards in many conditions. Detailed findings on the trade-offs between bias reduction, coverage, and power across scenarios are presented.
CONCLUSIONS: No single PAIC suits every situation. This study offers practical guidance for the use of PAICs in HTA applications, helping to inform methodological choices in the presence of imbalanced effect modifiers across studies.
Exploring the Ability of Generative AI to Interpret and Report Comprehensive NMA Results: A Step Towards the Automation of NMA Reports
OBJECTIVES: A previous study developed a system to automate network meta-analyses (NMAs) using large language models (LLMs). To maximize the benefits of automation, it is essential to automate the production of NMA reports, which include assessment and interpretation of results. This study aims to determine whether LLMs can assess and interpret comprehensive NMA outputs and accurately report these.
METHODS: This study used Claude 3.5 Sonnet (v2) to generate two NMA reports, including introduction, methods, results, and discussion sections. The reports were based on replicated results for overall survival (OS) and progression-free survival (PFS) from a previously published NMA in non-small cell lung cancer (Aggarwal, 2023) and change from baseline in HbA1c, and proportion of patients achieving HbA1c < 7% in diabetes (Witkowski, 2018). The results were provided as csv files and image files containing e.g., forest plots, rank-probability plots. The LLM was prompted to generate text interpreting comparative effectiveness results and associated statistical diagnostics. Prompting strategies included few-shot, pdf-interaction, and leveraging a “hybrid” approach that sent both figures and data to the LLM to maximise understanding of the evidence. The reports were qualitatively assessed by three expert NMA statisticians.
RESULTS: The three experts concurred that the LLM accurately interpreted the results, assessed rank-probability plots and provided an appropriate ordering of efficacy. The evaluation of heterogeneity, inconsistency, and convergence by the LLM was precise, providing a thorough analysis of the statistical evidence across all segments of the network.
CONCLUSIONS: The results show LLMs can correctly assess and interpret NMA results and the standardised associated statistical diagnostics and also automatically generate reports evaluating multiple outcomes. Scaling the approach to report results from alternative PICO selections, as required for health technology assessment (HTA) ready NMA reports and the Joint Clinical Assessment (JCA), could lead to substantial efficiency gains.
Thus SPOKE the TA: Querying NICE Technology Assessments Using Generative AI and RAG
OBJECTIVES: This project aimed to assess the accuracy of several generative AI (GenAI) approaches in extracting and analysing key information from published NICE technology appraisals (TAs) across disease areas.
METHODS: Two different AI tools were used to extract key data from NICE TAs, Pfizer’s VOX utilising Open AI’s GPT-4o, and Microsoft Copilot also utilising GPT-4o but with a different context window. Data extracted included: incidence, prevalence, decision problem, comparator information, and reimbursement outcomes from previously published NICE TA documents. This was done using retrieval augmented generation (RAG)- recent relevant TAs were uploaded to the tools before asking questions. Analysis was ran for four different disease areas: obesity, breast cancer, prostate cancer and lung cancer. For these preliminary results each response was rated between 1 (low accuracy) and 3 (high accuracy). The key scenarios used five TAs to compare tools (VOX 5 TA RAG and Copilot 5 TA RAG), as additional validation of optimal TA throughput another scenario used one TA (VOX 1 TA RAG) and then one scenario did not use any TAs (VOX non-RAG).
RESULTS: The most accurate results were seen when using Microsoft Copilot with five TAs, giving an accurate response (3) for 54% of responses, non-hallucination result (2) for 14% and an inaccurate response (1) for 32%. The least accurate results were seen in VOX five TA, with corresponding results of most accurate (3) 11%, non-hallucination (2) 46% and inaccurate (1) of 43%. It is plausible that this is due to a lower context window in VOX.
CONCLUSIONS: Generative AI tools can extract and analyse key parameters from NICE TAs, however this is sensitive to the type of tool used and the number of documents provided. As demonstrated, GenAI still hallucinates and at this time a human in the loop (HITL) approach is still recommended.
Lost in a Labyrinth: EMA and HTA Create Confusion around Patient-Centered Evidence Requirements in Europe
Session Type: Workshop
Topics: Patient-Centered Research, Clinical Outcomes, Health Technology Assessment
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
Purpose: In Europe, Regulatory and HTA guidelines are misaligned regarding requirements of Patient-Reported Outcomes and Health-Related Quality of Life (HRQoL) data. This has led to uncertainty for Sponsors in determining what data is best submitted. The purpose of this workshop is to review potential approaches for alignment and submission.
Description: EMA CHMP Guideline (2005) uses ‘optional’ terminology in the advice regarding HRQoL data and requires the majority of domains to be significantly improved and adjusted multiplicity to grant an HRQoL label. On the contrary, Theidel and Van Schulenburg (2016) described that trials including HRQoL instruments had percentage-wise higher chances of receiving added benefit ratings in Germany during HTA review, even if based on 1-2 symptoms or symptom impacts, e.g. in oncology. Other national EU agencies may also be receptive towards other PRO instruments to obtain a favourable approval and preferential reimbursement. Most recently, new Joint Clinical Assessment webinars from May 2025 were missing some standard evidence guardrails, suggesting that anything Patient-Centred may go — creating opportunities as well as confusion for PRO experts.
Dr Katja Rudell will introduce relevant guidelines and outline potential risks of growing patient centred evidence in the EU space (10 minutes) - and conduct a pulse check which guidelines are favoured by the ISPOR audience members (5 minutes), Dr Bryan Bennett will provide an industry perspective on the myriad of guidelines on product PRO/COA strategy in his discussions (10 minutes). Prof. Olivier Chassany will close the workshop presentation with an elaborated checklist approach that aims to reduce confusion on simplified processes of presenting patient-centred evidence for HTA from his experience as a reviewer of such data (15 minutes). Audience participation for discussions during each presentation and Q&A session at the end (15 minutes) will foster engaging talks on the problems and solutions of European submissions to elevate the importance and value of PRO, and HRQL data.
Moderator
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Katja Rudell, BSc, MSc, PhD, Other
Kielo Research, Cambridge, United Kingdom
Speakers
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Katja Rudell, BSc, MSc, PhD, Other
Kielo Research, Cambridge, United Kingdom
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Bryan Bennett, BSc, PhD
Jazz Pharma, Berwick-upon-Tweed, United Kingdom
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Olivier Chassany, PhD, MD
Assistance Publique–Hôpitaux de Paris (AP-HP), Paris, France
Professor of Therapeutics (Health Economics Clinical Trial Unit, AP-HP Paris hospitals, France), clinician, specialist in gastroenterology, with a long experience in developing Patient-Reported Outcomes (PRO) questionnaires. Involved for more than 20 years in dossiers expertise for EMA and French Drug Agency and over 32 years in Ethics Committees. Co-author of the EMA Reflection Paper on Health-Related Quality of Life. Involved in HTA dossiers expertise for pharma/CRO. Teaching Therapeutics and Methodology of clinical trials at Université Paris Cité. Past chair of the ISPOR SIG Clinical Outcomes Assessment (COA).
The Variable Thresholds Issue: A Necessary Tool or a Misguided Obsession?
Session Type: Issue Panel
Topics: Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE:
The willingness-to-pay (WTP) threshold is often key parameter in assessing whether a health intervention offers value for money, typically via an incremental cost-effectiveness ratio (ICER). Despite its theoretical appeal, WTP thresholds vary widely across health systems. Some countries link them to GDP, others adjust based on diverse modifiers, and some do not use explicit thresholds at all.
This divergence has led to two models: fixed/universal thresholds, applied uniformly, and variable thresholds, which adapt to context. For example, NICE (UK) applies modifiers to QALYs which in turn impacts the ICER; ICER (US) uses multi-criteria decision analysis; ZiNL (Netherlands) adjusts based on disease burden; and ICER (US) uses multi-criteria decision analysis. While universal thresholds are often promoted in theory, they are inconsistently applied in practice.
The core issue is that variable thresholds challenge the foundational assumption that a QALY is always worth the same, raising concerns not only about fairness and contextual relevance, but also about the theoretical coherence of cost-effectiveness analysis.
OVERVIEW:
This panel will explore whether variable thresholds better reflect diverse patient needs and system priorities. Moderator Cornelis Boersma will open with reflection (5 min) and guide discussion with provocative questions (15 min): Should HTA frameworks institutionalize variable thresholds—or abandon them altogether?; How are thresholds used to justify “value-based prices”?; What are the implications for transparency, consistency, and efficiency?
Industry stakeholders may view variable thresholds as a way to enable fairer access to high-cost innovations. Academics may scrutinize their implications for methodological rigor and consistency in health economic evaluations. Policy makers must balance the flexibility of variable thresholds with the need for transparent, predictable decision-making.
These perspectives will be explored by panellists Josephine Mauskopf, Jaime Caro, and Thea van Asselt, each offering a 10-minute presentation. The session concludes with audience engagement through live polls and open Q&A (10 minutes).
Moderator
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Cornelis Boersma, PhD
Health-Ecore, Zeist, Netherlands
Speakers
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Josephine Mauskopf, PhD
RTI Health Solutions, DURHAM, NC, United States
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J. Jaime Caro, MD
Thermo Fisher Scientific, Lincoln, MA, United States
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Thea van Asselt, PhD
University of Groningen, Groningen, Netherlands
Europe at Crossroads: Can it Compete With Other Regions For Healthcare Innovation?
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment, Economic Evaluation
Level: Intermediate
Available On-Demand: Digital Conference Pass
Pharmaceutical companies base R&D and launch strategies on expected return on investment. In the past decade, the US has significantly outpaced Europe in pharmaceutical R&D spending, while China is rapidly gaining ground. The US leads Europe on every investor metric from capital availability, intellectual property, speed of approval to rewards for innovation. For small biotech firms, Europe remains a particularly difficult environment, with fewer than half U.S. approved therapies available.
Inequalities in access within Europe are also significant. The 2024 WAIT indicator shows that nearly half (48%) of medicines are not available to all EU patients and it can take up to 3 yrs for medicines to become reimbursed in some countries post marketing authorization. The EU JCA initiative aims to attract investment and reduce access inequalities. However, there are multiple complexities and burdensome hurdles in the implementation process. Meanwhile, shifting geopolitical risks further challenge Europe’s global competitiveness.
How can Europe become a more attractive region for pharmaceutical R&D and timely launches? Are pricing and reimbursement strategies the primary barrier, or are HTA processes and regulatory inefficiencies equally responsible? What reforms are needed to incentivize R&D investment and equitable patient access across Europe?
Overview:
This panel will explore actionable solutions to boost Europe’s competitiveness in pharmaceutical R&D and launch strategies. Moderated by Cristina Masseria, the session brings together diverse perspectives across the healthcare ecosystem. Gergana Zlateva will bring extensive expertise in access to medicines, Brian O’Rourke will offer insights drawn from his deep experience in HTA decision-making processes, and Neal Masia will provide a financial and investment-oriented viewpoint.
The panel will actively engage the audience to capture their perspectives. The discussion will focus on identifying concrete steps to make Europe a more attractive and competitive environment for pharmaceutical innovation and patient access.
Moderator
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Cristina Masseria
Aesara, Madrid, Spain
Speakers
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Gergana Zlateva, PhD
Pfizer Inc., New York, NY, United States
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Brian O'Rourke, BSc, PharmD
Brian O'Rourke Health Care Consulting Inc., Ottawa, ON, Canada
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Neal Masia, PhD
EntityRisk, Inc., Princeton, NJ, United States
Addressing the Socioeconomic Burden and Policy Barriers in Rare Diseases
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session addresses challenges and innovations in rare disease research, policy, and care. Presentations cover the social and economic burden of inherited retinal disease in Sweden, the evolution of risk-sharing agreements for orphan drugs over the past decade, and a catalogue of actions to overcome policy barriers to drug repurposing. The session concludes with a decision-analytic framework for evaluating personalized, patient-centered care solutions in multiple system atrophy.
Catalogue of Actions to Address Policy Barriers of Drug Repurposing
OBJECTIVES: Exploring new therapeutic areas for established medicines has been gaining recognition, as it offers potentially faster, and more affordable alternatives to de novo drug development. Previous research in the REMEDi4ALL Horizon Europe project had identified policy-related barriers hindering the success of repurposed medicines (RMs). This study aims to identify push and pull mechanisms that could incentivize drug repurposing (DR) by addressing these barriers.
METHODS: Facilitating mechanisms were retrieved i) from the scientific literature, ii) prior good practices of R&D incentives e.g. in rare, neglected and tropical diseases, and iii) successful reference cases of DR. Mechanisms were compiled into a catalogue of actions (CoA) until saturation was reached. Identified mechanisms were divided into two categories. Push incentives aim to reduce the cost, time, and risk of R&D, while pull incentives aim to improve the financial and societal return for investors.
RESULTS: We identified 10 types of push incentives, including specific regulatory pathways for RMs, increased regulatory know-how and accelerated timelines and regulatory fee reductions or waived fees, enabling label extension initiated by third parties, exploring complementary evidence generation options for RMs, providing education of supply-side stakeholders, increase provision of different funding methods- and types of funders for R&D of RMs. We also identified 9 types of pull incentives, including exclusivity-type regulatory incentives for RMs, monitoring off-label use and bringing off-label use to on-label where possible, developing appropriate health technology assessment frameworks for RMs, differentiating pricing of RMs from pricing rules of on-patent and generic medicines, reducing time and cost of post authorization processes for RMs, providing education of demand-side stakeholders, compensation for successful R&D of RMs and increasing demand for RMs.
CONCLUSIONS: As a next step, the CoA will be validated by different stakeholder groups, aligned with previously identified policy barriers, and transformed into actionable policy recommendations.
Social and Economic Burden Among Patients With Inherited Retinal Disease: A Nationwide Matched Cohort Study in Sweden 2001-2021
OBJECTIVES: Inherited retinal diseases (IRDs) are often early-onset visual impairments associated with long-term social and economic challenges. Evidence on their population-level impact remains limited. This study evaluated associations between IRD and key social outcomes, including unemployment, education, income, absence from work due to sickness and marital status.
METHODS: A retrospective matched cohort study was conducted using Swedish national registers from 2001 to 2021. Adults with IRD (n = 6,058) were matched 1:10 with population controls (N = 66,527) by age, sex, and region. Incidence rates per 1,000 person-years were calculated. Poisson and ordered logistic regression models estimated adjusted incidence rate ratios (IRRs) and odds ratios (ORs). A parallel analysis examined educational outcomes among children with IRD (n = 10,597).
RESULTS: Among adults, IRD was associated with increased risks of unemployment due to health reasons (IRR, 1.25; 95% CI, 1.11-1.42), sickness absence (IRR, 1.04; 95% CI, 1.00-1.08), and long-term sickness absence (>14 days) (IRR, 1.28; 95% CI, 1.23-1.34). Higher risks were also observed for completing only primary education (IRR, 1.25; 95% CI, 1.03-1.51), low income (IRR, 1.17; 95% CI, 1.14-1.21), and illness compensation (IRR, 1.62; 95% CI, 1.44-1.82). Adults with IRD were less likely to be married (IRR, 0.97; 95% CI, 0.96-0.99) or reach higher income levels (IRR, 0.67; 95% CI, 0.64-0.70). No differences were found for overall unemployment or secondary education. Among children, IRD was linked to increased risk of completing only primary education (IRR, 1.63; 95% CI, 1.03-2.55).
CONCLUSIONS: IRD was consistently associated with adverse social outcomes, including greater risk of health-related unemployment, prolonged sickness absence from work, low income, and lower educational attainment. These disparities were evident in both adults and children, underscoring the need for early targeted interventions to address the long-term socioeconomic burden linked to IRD.
Evolution of Risksharing Agreements for Orphan Drugs 20152024
OBJECTIVES: Risk-sharing agreements (RSAs) are being used globally to help facilitate the reimbursement of high-cost drugs, particularly orphan drugs. This analysis considers the evolution of RSAs for orphan drugs around the world over the last decade based on the number of agreements made, geographic distribution, and agreement type.
METHODS: All available RSAs between 2015 and 2024 were identified from GlobalData’s Risk Sharing Database. To evaluate the five-year growth rate by market for orphan drug related RSAs, the dataset was divided into two timeframes: arrangements made from 2015-2018 and 2019-2024.
RESULTS: Between 2015-2024, the highest RSA activity was in the UK (812), followed by Australia (145). Orphan drug RSAs accounted for 62% and 59% of the arrangements in these two markets, respectively. Across all drug types, 90% of agreements made were finance-based, with only 10% performance-based. For orphan drugs specifically, performance-based RSAs were higher at 13%, while 87% of RSAs were finance-based. In 2019-2024, orphan drug related RSAs surged in Ireland (450%), followed by Australia (205%) and the UK (44%), compared to 2015-2018. In contrast, Sweden and the Netherlands experienced declines of 83% and 63%, respectively. Asian markets, in particular China and South Korea, demonstrated increased interest in RSAs and have signed their first orphan drug related RSAs in the last decade.
CONCLUSIONS: RSA activity remains high in established markets such as the UK and Australia. The higher proportion of performance-based RSAs for orphan drugs reflects the need for outcome-based models to address the clinical and economic uncertainty. Ireland demonstrated growth in the last decade, establishing itself as an evolving market. The entry of Asian markets suggests a shift in their stance towards RSAs, particularly for orphan drugs, thereby improving access. However, declines in some European markets may reflect concerns around the financial sustainability of RSAs.
Evaluating Personalized Care for Rare Disease: A Decision-Analytic Framework for Patient-Centered Solutions in Multiple System Atrophy
OBJECTIVES: Multiple system atrophy (MSA) is a rare neurodegenerative movement disorder with no effective treatment. Personalized care protocols tailored to individual needs can mitigate the decline in quality of life (QoL). We aim to develop a decision-analytic modelling framework for future implementation to assess long-term effectiveness, cost-effectiveness, and cost-utility of personalized best medical care (PBMC) with or without telemedicine (TM) compared to usual care in patients with MSA, to inform decision makers.
METHODS: We developed a decision-analytic framework to guide model design and data implementation, informed by literature and input from an interdisciplinary expert panel including clinicians, QoL and end-of-life care specialists, epidemiologists, ethicists, HTA experts, and decision scientists. The framework defines research questions, population, comparators, time horizon, health states, model type, and analytical methods. We followed ISPOR-SMDM and causal modeling guidelines.
RESULTS: The framework includes three interventions: 1) PBMC, 2) PBMC with TM, and 3) usual care (based on the European MSA cohort and MeDeMSA trial, NCT06072105). Health states were defined using the Unified Multiple System Atrophy Rating Scale Part IV, ranging from 1 (independent) to 5 (bedridden), incorporating background and MSA-specific mortality. Given the manageable number of states, a Markov state-transition cohort model was chosen. The model simulates a cohort starting at age 61, with 6-month cycles and a lifelong horizon. Outcomes include life expectancy, quality-adjusted life expectancy (QALE), harms, costs, and incremental cost-effectiveness ratios from both societal and healthcare perspectives. Natural history and transitions are informed by the European MSA cohort. Utility values, based on EQ-5D scores, inform QALE estimation. Resource use data are drawn from the trial, with additional inputs from literature and public datasets.
CONCLUSIONS: We present an evidence-based decision-analytic framework to evaluate long-term outcomes of best medical care for MSA. Future analyses will support clinical and policy decisions to improve care for patients with MSA.
Beyond P-Values and Sample Size Calculations: Probability of Success for Indirect Comparative Efficacy Forecasting and its Role in Industry Decision Making
Session Type: Workshop
Topics: Methodological & Statistical Research, Health Technology Assessment, Clinical Outcomes
Level: Intermediate
Available On-Demand: Digital Conference Pass
Purpose
Probability of success (PoS) is frequently used in clinical trials as a metric to assess predictions of eventual trial success through defining significance thresholds of hazard ratio or effect estimates between trial arms. It enables a better understanding of risk-benefit within clinical trial strategy and is useful specifically in resource optimisation.
Alongside clinical trials, PoS has the potential to be a useful tool when assessing indirect treatment comparison (ITC) outputs. This approach can aid in forecasting comparative efficacy alongside therapeutic product profiles and support strategy planning and decision making.
Participants will be introduced to practical frameworks for generating estimates of anticipated effects (and uncertainty) for comparators within the network for various definitions of success. This educational workshop will be valuable to researchers, health economists, and industry analysts who are interested in new and innovative ways to inform prioritisation and decision-making.
Description
Workshop attendees will obtain a working knowledge of the application and benefits of PoS analyses in an ITC context. The workshop will cover A) introduction to PoS in its original context, B) extension of PoS to ITC and comparative efficacy forecasting, C) results of an example case study including a live interactive simulation, and D) discussion of the utility of the approach for industry.
CS will moderate the session and introduce PoS as it is currently applied in clinical trials (10 min). ES will show how this method can be extended to comparative efficacy forecasting, through a case study demonstration via Shiny app (20 min). MH will discuss the role of PoS in industry decision making, including insight into its value in cross-disciplinary stakeholder discussions (15 min). Audience participation will include polling for expected outcomes during the live simulation portion, as well as elicitation of inputs to test in the Shiny app and Q+A (15 min).
Moderator
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Claire L Simons
OPEN Health, Rotterdam, Netherlands
Speakers
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Elizabeth Sigworth, PhD
OPEN Health Group, Rotterdam, Netherlands
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Michael Hurst, BSc, MSc
Bristol Myers Squibb, Uxbridge, United Kingdom
Director of Global HEOR, Cardiovascular, HTA and Early Assets at Bristol Myers Squibb with 12 years experience in the pharmaceutical industry. Specific research interests include advanced modelling methodology, real-world evidence and machine learning.
Taking a Time-out to Understand Innovations in Survival Modeling: A Practical and Open Discussion of Time-Varying Network Meta-Analyses (NMA) Informing Cost-Effectiveness Models (CEM) and Health Technology Assessments (HTA)
Session Type: Workshop
Topics: Methodological & Statistical Research, Economic Evaluation, Health Technology Assessment
Level: Intermediate
Available On-Demand: Digital Conference Pass
PURPOSE: Both the National Institute for Health and Care Excellence (NICE) HTA reviewers and European Union Joint Clinical Assessment (JCA) guidelines require time-varying NMA methods to more precisely compare survival benefits when the proportional hazards assumption is violated. However, detailed guidance including implementation into a CEM is limited. This workshop demonstrates the advantages, limitations and sensitivity of the CEM results when using different NMA methods. A recent NICE oncology technical appraisal (TA858) serves as the motivating example.
DESCRIPTION: Workshop attendees will acquire practical knowledge of several time-varying NMA methods, considering multivariate parametric, fractional polynomial, restricted cubic spline, M-splines and piecewise modelling within the context of building a CEM for HTA review. Topics include a) technical critique of different time-varying NMAs from the user perspective, b) application and interpretation of subsequent NMA results within the CEM and c) the differential impact of these advanced methods against the need for pragmatism with implications for HTA bodies.
Ms. Westley will chair the session, providing a brief introduction (5 min.). Dr. Liu will explain nuances of data preparation and how to compare different time-varying NMA approaches (10 min.). Dr. Kearns will demonstrate applications and limitations for the CEM including results uncertainty (15 min.). Dr. Pedder will discuss considerations and challenges encountered when employing these methods within HTA systems such as NICE and the JCA (15 min.). Through structured voting plus open discussion led by Ms. Westley (15 min.), audience participation will include choosing what they think is the most appropriate NMA model, observing consequences of their method selection during CEM implementation and discussing the broader informativeness towards HTA submission. This interactive workshop will be valuable to research analysts, clinicians and industry leaders interested in the call for robust but interpretable modelling approaches to inform HTA decision-making for both NICE and the JCA.
Moderator
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Tracy Westley, MSPH
Lumanity, Dundas, NB, Canada
Speakers
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Yu-Heng Liu, PharmD, MSc
Lumanity, Sheffield, United Kingdom
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Benjamin C Kearns, BSc, MSc
The University of Sheffield, Sheffield, United Kingdom
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Hugo Pedder
University of Bristol, Bristol, United Kingdom
Beyond the QALY: Alternatives in the US and Implications for EU
Session Type: Workshop
Topics: Study Approaches, Methodological & Statistical Research, Patient-Centered Research
Track: Patient-Centered Evidence
Level: Advanced
Available On-Demand: Digital Conference Pass
Purpose: Due to restrictions on QALYs in U.S. federal health decisions, alternative cost-effectiveness metrics have emerged, including EVLYG, HYT, and GRACE. These tools differ in how they value life extension, quality-of-life improvement, or both—especially for patients with poor baseline health. This session helps researchers, payers, and industry stakeholders understand the underlying assumptions of these non-QALY approaches, assess where they may be better or worse suited for evaluating the value of a given healthcare intervention, and understand the implications for HTA bodies.
Description: Dr. Devine (5 mins) will introduce speakers, briefly describe the non-QALY evolution in the US, and moderate the Q&A.
Dr. Basu (15 mins) will present an overview of these topics, laying out different assumptions for each metric and show new evidence on links between EQ-5D weights and GRACE methodology.
Mr. Ektare (15 mins) will demonstrate the implementation of these alternative metrics in three anonymized product profiles (late-stage haem-onc therapy, chronic pain palliative, pediatric SMA gene therapy).
Dr. Banks (15 mins) illustrates the challenges of using non-QALY measures in the EU context and the implications for EU HTA if the US continues to rely on these measures and also pursues most-favored-nation prices.
We will engage the audience with real-time polling at the beginning and end of the panel, and 10 minutes of Q&A to discuss the strengths and limitations of each of the metrics.
Moderator
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Beth Devine, MBA, PharmD, PhD
University of Washington, Seattle, WA, United States
Speakers
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Anirban Basu, PhD
University of Washington, Seattle, WA, United States
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Varun Ektare, MPH
Indence Research Private Limited, Thane West, India
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Jordan T Banks, BS, MPP, PhD
Avalere Health, Canoga Park, CA, United States
8:30 - 10:00
Networking Breakfast Bites (Exhibit Hall)
Session Type: General Meeting
8:30 - 11:30
Exhibit Hall Open
Session Type: General Meeting
9:00 - 11:30
Poster Session 5
Session Type: Research Posters
Presenters will be with their posters from 09:00–10:00
Poster Session 5
Session Type: General Meeting
Presenters will be with their posters from 09:00–10:00
10:00 - 11:00
Science at PACE? A Multi-Stakeholder Developed Framework for Accelerated Patient Access to Cancer Care
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment, Real World Data & Information Systems
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue:
The European Medicines Agency (EMA) has introduced accelerated regulatory pathways (APs) to enable earlier access to promising therapies, particularly in oncology, where there is high unmet medical need but comprehensive clinical data is not yet available. However, the corresponding processes for Health Technology Assessment (HTA) and pricing & reimbursement (P&R) differ across European countries. Some countries have introduced HTA APs and access funds, but these approaches vary substantially and face challenges, which is reflected in variable HTA and P&R decisions and, ultimately, unequal patient access. The introduction of the Joint Clinical Assessment (JCA) across European countries will bring significant change to HTA, though how APs will interact with JCA is uncertain. There is broad agreement that in areas with high unmet medical need timely patient access is particularly important. Hence, there is a need to develop a pan-European and stakeholder-endorsed framework for an AP that fits into existing HTA processes, including the JCA, and can be implemented consistently across health systems.
Several components must be agreed upon between stakeholders and countries if APs are to be implemented consistently, including treatment eligibility, evidential requirements, pricing models, and enforcement mechanisms.
Overview:
The session will explore the feasibility of a pan-European AP for oncology. The moderator will introduce the issue and outline an AP framework (10 minutes). Panellists (10 minutes each) will present their perspective on the framework, including its promises, pitfalls, and opportunities for practical implementation. The aim is to share perspectives and identify common ground to progress the debate for APs. The panel will be particularly useful to HTA representatives and payers considering the implementation of accelerated pathways.
The session will include interactive components, such as Q&A and polls, to ensure audience engagement.
This session is based on work funded by AstraZeneca, GSK and MSD.
Moderator
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Martina Garau, BA, MSc
Office of Health Economics, London, United Kingdom
Speakers
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Bettina Ryll, PhD, MD
Melanoma Patient Network Europe, Uppsala, Sweden
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Niklas Hedberg, MSc
TLV, Stockholm, Sweden
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Alexander Roediger, MSc
Merck, Jersey City, NJ, United States
Delay in Patients’ Access is the Price of Misalignment: Why Evidence Must Speak Also the Language of Payers and Patients
Session Type: Issue Panel
Topics: Patient-Centered Research, Real World Data & Information Systems, Clinical Outcomes
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
Biotech companies, regulators, payers, physicians, and patients all seek to understand the value new medicines may bring to current treatment pathways. However, each stakeholder defines "value" differently. For regulators, value typically centers around efficacy, safety, and manufacturing quality. Physicians consider treatment’s place in therapy and implementation feasibility. Budget holders focus on comparative clinical effectiveness, population size, affordability, and value for money. Meanwhile, patients prioritize also out-of-pocket costs, convenience, impact on daily functioning and independence, and implications for families.
However, clinical trials are often designed primarily to meet regulatory requirements with new medicines unable to articulate a compelling value story to the diverse stakeholders, resulting in delayed or even denied access and patient uptake.
Increasingly, RWE, advisory boards, and stakeholder engagement strategies are used to bridge this gap. Yet critical questions remain: Are these engagements occurring early enough in the product lifecycle? Do they meaningfully and equally incorporate the insights of all relevant stakeholders? Are feedback loops structured to enable tangible influence on evidence generation plans? And are opportunities for collaborative research being explored, resourced adequately, and successfully implemented?
This panel, moderated by Cristina Masseria, will bring together diverse perspectives from across the health ecosystem -with Adrian Cassidy from industry, Entela Xoxi as payer, and a patient advocate- to explore how companies can better integrate the voices and needs of different stakeholders into evidence generation strategies. Panelists will reflect on current practices, share examples, and debate what changes are needed to more effectively align evidence planning with the broader definitions of value held across the system also in consideration of EU-JCA.
Audience engagement will play a key role, with time for Q&A and experience sharing. The goal is to identify actionable strategies to align evidence generation with broader definitions of value, supporting timely and equitable access to innovation.
Moderator
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Cristina Masseria
Aesara, Madrid, Spain
Speakers
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Adrian Cassidy, PhD
Novartis, Basel, Switzerland
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Entela Xoxi, MSc, PharmD, PhD
Independent Consultant, Roma, Italy
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Maria Dutarte, MSc
European Patients’ Academy on Therapeutic Innovation (EUPATI), Utrecht, Netherlands
Maria Dutarte is the Executive Director of the European Patients’ Academy on Therapeutic Innovation (EUPATI). Maria has previously worked for the European AIDS Treatment Group (EATG) coordinating patient involvement in a number of training initiatives and scientific projects. She has also worked for different scientific and international organisations, e.g. French National Research Center for Scientific Research (CNRS), International AIDS Society (IAS), The Global Fund to Fight AIDS, Tuberculosis and Malaria and The International Foundation for Science (IFS). Her educational background is in International Education and Communications.
U.S.-Centric Drug Policies: How National Interests Are Reshaping Global Innovation and Access
Session Type: Workshop
Topics: Health Policy & Regulatory, Methodological & Statistical Research, Health Technology Assessment
Level: Intermediate
Available On-Demand: Digital Conference Pass
Purpose
Medicines have no national borders and can benefit citizens worldwide. However, in the global endeavor to fight diseases, the US has long cross-subsidized other countries in pharmaceutical R&D efforts. In response to domestic pressure to “make others pay”, the US administration has proposed a series of “America-First” policies to get other countries to pay their “fair” share.
Currently, patients outside of the US, even in advanced OECD countries, suffer from significant delay and lack of access to innovative medicines. The European Commission has introduced new Health Technology Assessment Regulations (effective from Jan 2025) as part of a broader proposal to narrow the access gap. The broader proposal also included important considerations on financing.
Creative cross-country solutions to finance global innovations are needed to better sustain long-term innovations and improve patient welfare.
This panel convenes a multistakeholder panel to discuss the impacts of America-First policies on global access to innovative medicines and incentives to innovate, and outline promising global differential pricing mechanisms to better balance access and innovation.
Description:
Meng Li will first discuss the existing cross-country disparities in access to innovative medicines, and review the spectrum of “America-First” policies proposed by the Trump administration.
Gunnar Esiason, a patient advocate with cystic fibrosis, will ground the discussion in patient reality, highlighting how delayed or foregone access will impact patients worldwide.
Richard Xie will use the net present value modeling to show how the proposed policies will alter launch strategies for innovators, resulting in more unequal access and less overall innovation.
Mikkel Oestergaard will synthesize the discussion, and propose creative solutions based on the ongoing work at the Global Access to Medical Innovation ISPOR SIG.
The panel will conclude with Q&A with the audience. Polling questions and handouts will be used to engage the audience throughout the session.
Moderator
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Meng Li, MS, PhD
Tufts Medical Center, The Center for the Evaluation of Value and Risk in Health, Boston, MA, United States
Speakers
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Gunnar Esiason, MBA
Boomer Esiason Foundation, New Canaan, CT, United States
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Richard Xie, PhD
RA Capital Management, Newton, MA, United States
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Mikkel Oestergaard, PhD
MSD Innovation & Development GmbH, Zurich, Switzerland
Handling Transitions between Child and Adult HRQoL in Economic Evaluation
Session Type: Issue Panel
Topics: Economic Evaluation, Patient-Centered Research, Methodological & Statistical Research
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
Issue: A number of PRO instruments, designed to generate health state utilities, are now available for children as well as for adults. In principle, these allow HRQoL to be measured in age-appropriate ways 'from cradle to grave'. But can the estimates of QALYs produced from child and adult versions of these instruments be interpreted in the same way? Do transitions between age-specific measures of HRQoL require special attention in cost effectiveness models?
Overview: Methods, instruments and evidence on child HRQoL have improved considerably in the last five years. There are now a number of generic measures of HRQoL developed to be age-appropriate, that are accompanied by utilities that allow estimation of QALYs. An ISPOR Taskforce on utilities for child HRQoL has recently concluded. However, a remaining challenge is understanding how the estimates of QALYs produced from child and adult HRQoL instruments compare. This is important, because many healthcare interventions in childhood generate survival gains and improvements in HRQoL that need to be modelled from childhood into adulthood. Systematic differences in how HRQoL is measured and valued in each age group could potentially cause discontinuities in HRQoL and QALYs, even when underlying health remains unchanged. In this issues panel, Nancy Devlin will present new evidence on how the measurement properties of the EQ-5D-5L and EQ-5D-Y-5L compare in data collected from adolescents who completed both; and how the characteristics of utilities for child and adult HRQoL instruments differ. Janine Vertsraete will present results showing how these differences in measurement and valuation between child and adult HRQoL instruments interact when analysing preference-weighted health state descriptions. Finally, Donna Rowen will consider how potential discontinuities in the measurement and valuation of HRQoL could, in principle, be addressed and what methods could be developed for use to support the transition between age-specific instruments in cost effectiveness modelling. The session will allow time for Q&A, led by moderator Louis Matza.
Moderator
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Louis Matza, PhD
Evidera, Bethesda, MD, United States
Speakers
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Nancy J Devlin, PhD
University of Melbourne, Melbourne, Australia
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Janine Verstraete, PhD
University of Cape Town, Cape Town, South Africa
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Donna Rowen, BA, MSc, PhD
SCHARR, University of Sheffield, Sheffield, United Kingdom
Patient Preferences in Clinical Trials and HTA
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session explores approaches for capturing the patient voice, featuring AI methods, patient engagement, preference data use, and methodological innovations. Topics span large language models, novel instruments, engagement frameworks, and economic modelling.
Patient Engagement in Economic Evaluations of Cancer Care: Mapping the Landscape and Advancing Practice
OBJECTIVES: While patient engagement is increasingly recognized in health technology assessment, its integration into economic evaluations remains rare and underdeveloped. This study aimed to (1) examine how patients are currently engaged in economic evaluations of cancer care, (2) identify key barriers and facilitators, and (3) develop practical recommendations to support more meaningful engagement.
METHODS: We conducted a multi-method study, which included a scoping review, a survey, and qualitative interviews. The review covered economic evaluations of cancer care published between 2013-2022 in 32 leading journals spanning oncology, health economics, and patient engagement. Eligible studies involved individuals affected by cancer at any stage of the evaluation. Authors of included studies were invited to complete a survey and participate in interviews. Thematic analysis was used to synthesize findings.
RESULTS: The review identified few studies reporting patient engagement, and involvement was typically limited to reviewing lay summaries or manuscripts. Only a small number engaged patients in shaping model structure or identifying relevant outcomes. Among 21 survey respondents, key barriers included uncertainty about the value of engagement, lack of training, and perceived lack of relevance. Early qualitative interviews showed widespread interest in engagement but highlighted three main challenges: uncertainty about how engagement influences analysis, limited practical guidance, and lack of financial support. Patient partners co-led all phases of this study, from design through interpretation.
CONCLUSIONS: Although interest in engaging patients in economic evaluations is growing, activity remains limited and under-supported. Advancing this work will require co-developed guidance, targeted training for researchers and patients, and sustained funding. Insights from this study are informing a Delphi consensus process to develop practical recommendations for integrating patient engagement across the economic evaluation process.
EQ-5D Analysis in a Rare Disease: Utilities Across Disease Stages in Multiple System Atrophy
OBJECTIVES: Multiple system atrophy (MSA) is a rare, rapidly progressive neurodegenerative disorder. In the European MSA Study Group (EMSA) natural history cohort, average health-related quality of life (HRQoL) was lower than in advanced cancers, yet MSA severity stages were not considered. This study aims to derive HRQoL by calculating EQ-5D-3L-based utility values stratified by MSA severity to inform health technology assessment of best medical care proposed in MeDeMSA clinical trial (NCT06072105).
METHODS: We retrospectively analyzed EMSA data. EQ-5D-3L health states were mapped to severity stages defined by the Unified MSA Rating Scale Part IV (UMSARS IV). EQ-5D-3L responses were converted into utility values using the reverse crosswalk method with the German time trade-off value set, enabling comparison with EQ-5D-5L-based findings. Plausibility was assessed using ordinal logit, linear mixed-effects, and generalized additive models. To address extreme values, sensitivity analyses applied symmetric trimming and winsorizing data, retaining the central 99%, 95%, 90%, and 80% of values. Missing data were evaluated, and appropriate imputation applied.
RESULTS: A total of 283 observations from 85 individuals were analyzed. Mean utility values declined from 0.732 (95% confidence interval (CI): 0.587-0.876) in UMSARS IV stage 1 to 0.202 (95% CI: 0.138-0.266) in stage 5. Winsorizing and trimming narrowed confidence intervals but did not substantially affect mean estimates. In linear mixed-effects models with random intercepts for individuals, disease severity explained 36.0% of utility variance, followed by pain (32.7%) and depression (16.9%). Three UMSARS IV entries and eight utility values were flagged as implausible based on model predictions assuming consistent health states and reasonable utility ranges. Of seven missing UMSARS IV values, one was imputed no EQ-5D data were imputed.
CONCLUSIONS: Our study provides the first utility estimates in MSA stratified by disease severity. Next, we will generate robust utility values to support health technology assessment of best medical care in MSA.
No Strong Preference? Acceptance of the Quantitative Incorporation of Patient Preference in Economic Modelling in Past NICE Technology Appraisals
OBJECTIVES: With stakeholders such as payers and patient organisations increasingly seeking to integrate the patient voice into reimbursement decisions, interest in patient preference (PP) is growing. Previous research has suggested that the inclusion of PP studies in NICE technology appraisals (TAs) frequently led to Committee consideration of intervention attributes in final appraisal documents, but few TAs incorporated PP quantitively in their modelling. This research investigated the incorporation of utility adjustments to reflect PP in economic modelling in past NICE TAs and the extent to which such adjustments were accepted by Committees.
METHODS: The NICE website was searched on 02 June 2025 for completed TAs. Information regarding the approach to utility adjustment for PP and subsequent critique was extracted from the 10 most recent appraisals in which PP had been quantitatively included in the economic model.
RESULTS: Ten appraisals explicitly incorporating utility adjustment for PP were identified after searching the 148 most recent NICE appraisals. All 10 appraisals incorporated a utility increment or decrement based on administration route, all favouring oral administration. Utility adjustment was included in the base case in 9/10 of the appraisals. Incorporation of utility adjustment for PP was accepted with no critique from the Committee or assessors in four appraisals, accepted with critique in three appraisals, and rejected in three appraisals. Lack of evidence supporting inclusion and limitations of the sources informing utility adjustment (e.g. vignette studies) were cited as key areas of uncertainty.
CONCLUSIONS: PP regarding treatment administration route appears to be increasingly incorporated into economic models via utility adjustment in NICE appraisals. Despite a lack of guidance and uncertainty about methodological approaches to quantitively incorporating PP, Committees have accepted the incorporation of adjustment for PP in the final decision-making modelling approach.
Utilizing LLMs to Enhance Patient-Reported Outcome Measures: Application to the EQ-5D and Bolt-ons
OBJECTIVES: Large language models (LLMs) have shown promising applications in healthcare, yet little is known about their potential to improve the measurement of patient-reported outcomes, which are central to inform decision-making across health system levels. We explored the use of LLMs to develop or extend patient-reported outcome measures (PROMs) based on information from patient-reported free-text data.
METHODS: The GPT-4o model was used to analyze data from 1,977 members of the Dutch Celiac Association who completed the EQ-5D-5L and narratively described the impact of celiac disease on their lives. Prompts to the LLM were designed to identify possible additional dimensions (i.e., ‘bolt-on’ dimensions) to the EQ-5D-5L, and to produce preliminary bolt-on item wordings for selected dimensions. Evaluation of the approach comprised: comparisons of dimensions identified by two alternative approaches (i.e., qualitative analysis and topic modelling); text-entry level agreement (i.e., Cohen’s Kappa) on identified dimensions; suitability of LLM-generated bolt-on wordings assessed against existing criteria using Likert scales; and a critical appraisal consisting of face validity assessments and a SWOT analysis.
RESULTS: The LLM identified 12 potential bolt-on dimensions to the EQ-5D-5L, of which 9 were also identified using qualitative analysis, and 5 using topic modelling. Text-entry level agreement between the LLM and qualitative approaches was ‘substantial’ or ‘almost perfect’, with two exceptions of poor/fair agreement (median Kappa=0.70, IQR=0.44-0.89). The LLM-generated potential bolt-on wordings for the 4 most common dimensions (i.e., ‘Dietary restrictions’, ‘Fatigue’, ‘Social participation’, and ‘Gastrointestinal symptoms’) scored 4/5, 4.4/5, 4.3/5, and 4.2/5 respectively when assessed against existing criteria.
CONCLUSIONS: This study demonstrates the promising potential of LLMs to inform the development or modification of PROMs based on patient-reported text data. A limitation to generalizability and reliability is the approach’s dependency on prompt quality. Further research should assess the approach’s transferability across disease areas and different data sources (e.g. social media, EHRs).
Measuring the Social and Environmental Impacts of Innovative Treatments
Session Type: Research Podiums
Available On-Demand: Digital Conference Pass
This session examines frameworks and methodologies to assess the social and environmental impact of innovative treatments. Presentations cover the Social Impact of Health (SIoH) framework, an evaluation framework for digital health and AI technologies, a triangulated approach to defining value in digital health interventions, and the challenges of assessing efficiency–equity trade-offs in digital health applications for stroke care. Presenters will discuss these novel value elements and the requirements for their evaluation.
Challenges in Assessing Trade-offs Between Efficiency and Equity in the Use of Digital Health Applications for Stroke Care
OBJECTIVES: Stroke is one of the leading causes of death and disability across the world. Digital health technologies, such as telemedicine, mobile health (mHealth) applications, and wearable devices, have potential to deliver better health outcomes from stroke care. However, there are concerns that these technologies may increase the health inequality. Distributional cost-effectiveness analysis (DCEA) is a framework to assess the trade-off between efficiency and health equity.With the aim of informing decision-making about the efficiency-equity trade-off from digital health, this study 1) identifies factors that are relevant for the evaluation of digital health technologies in stroke within the DCEA framework, 2) reviews how metrics for such factors can be specified, and 3) identifies potential sources for such metrics by different socioeconomic group.
METHODS: To demonstrate the efficiency-equity trade-off, the DCEA framework synthesise information about the effectiveness of different health technologies and the health outcomes for different social groups.
RESULTS: The effectiveness of digital health technologies depends on the acceptability, affordability, compliance, cost and effect. Each of these factors may be different for socioeconomic groups defined by location, sex, age, marital status, education, employment, and income. This would imply that different levels of gains in health outcomes (e.g. quality-adjusted life years) might arise for different social groups and thus impose additional health inequality. The data required for the application of the DCEA framework will be assessed, and strategies for specification of relevant metrics and potential sources for empirical evidence is illustrated and discussed.
CONCLUSIONS: Digital Health interventions have huge potential for transforming stroke care and introduce innovation across different parts of the stroke trajectory including prevention, acute care, and rehabilitation. These interventions may also increase health disparities. Therefore, the health economic analyses of digital health for stroke should consider both the clinical and socioeconomic dimensions, so the benefits of digital health innovations can be equitably distributed.
An Evaluation Framework for Healthcare Professionals’ Digital Health and AI Technologies: Evidence-Based Policy Recommendations
OBJECTIVES: Health systems continue to face mounting challenges for which digital health and AI technologies (DHAITs) can lend a helping hand. However, the lack of consensus on a taxonomy for digital health technologies along with the absence of appropriate value assessment frameworks, particularly for professional-facing solutions, inhibit their value to tackling health system challenges. In this study, we propose a comprehensive evidence-based taxonomy for professional-facing DHAITs, review existing evidence frameworks, highlight their shortcomings and present robust recommendations to evaluate these technologies.
METHODS: The study draws on scoping reviews and thematic analyses to develop a structured taxonomy that reflects the key characteristics of professional-facing DHAITs. It also examines evaluation frameworks put forward by six countries—UK, France, Germany, USA, South Korea, and Canada—to assess their current classification and evaluation proposals. A group of 9 experts were consulted to fine-tune the overall results of the study.
RESULTS: The proposed taxonomy includes seven core dimensions: interoperability, access platform, driving technology, data inputs, intended impact, intended use case, and intended beneficiary. The consolidated review results in eight policy recommendations stressing the need to align classification with evidence standards, expand HTA frameworks to include system-level impacts, and foster international regulatory cooperation. These recommendations target HTA agencies, notified bodies, international regulatory networks, payers, health ministries and developers, to facilitate remedies required to effectively evaluate these technologies and improve their impact in health systems.
CONCLUSIONS: The proposed taxonomy and review of existing evaluation frameworks contribute to the existing evidence gaps and ongoing work to define the best approach to evaluating DHAITs. Ultimately, properly evaluating DHAITs would ensure that they deliver on their promise to help tackle health system challenges.
The Social Impact of Health (SIoH) Framework: Capturing How Health-Driven Productivity Shapes National Development
OBJECTIVES: Health is a fundamental driver of national welfare. When individuals cannot reach their full productive potential due to illness, both economic output and societal well-being suffer. However, health investments are often viewed by policymakers as costs rather than drivers of long-term, cross-sector economic growth. This misperception is largely due to the difficulty of quantifying health-related productivity improvements compared to more tangible sectors like infrastructure.
METHODS: To address this gap, we developed the Social Impact of Health (SIoH) Framework, a methodology that redefines healthcare costs as economic investments, The framework measures the social and economic impact of health interventions by evaluating changes in a country's economic capacity, defined as total productivity from paid and unpaid work. It quantifies how the burden of disease translates into productivity losses from premature mortality and morbidity and measures the productivity potential when comparing a new intervention to an appropriate comparator. The framework uses a socioeconomic perspective (which captures the dual focus on social and macroeconomic dimensions), integrating real-world labor dynamics and demographic factors to reflect the broader societal value of interventions. Productivity is value using Gross Value Added, offering a broader, more accurate measure of illness-related economic impact.
RESULTS: The framework has been applied in several studies. For example, in Germany, the use of erenumab for patients with migraine was projected to save 13.1 billion Euros in productivity losses between 2020 and 2027. Likewise, adopting obinutuzumab for follicular lymphoma from 2017-2030 could save 188 million Euros in paid and 536 million Euros in unpaid productivity losses.
CONCLUSIONS: Although the framework has been applied in prior analyses, it has not yet been fully published. This is the first formal presentation of the complete SIoH Framework, a structured, evidence-based tool for assessing the broader economic and social value of health interventions and supporting more informed health policy decisions.
Defining ‘Value’ in Digital Health Interventions: A Triangulated Approach to Model Development
OBJECTIVES: Digital Health Interventions (DHIs) are reshaping healthcare, yet their evaluation remains overly focused on clinical outcomes. Broader dimensions, such as user experience, system integration, and societal relevance, are often overlooked. This study introduces the Concentric Value Model, a multidimensional framework designed to reflect the full value of DHIs across subject-, interaction-, system-, and society-level perspectives. The objective was to triangulate conceptual, empirical, and practice-based insights to develop a robust and transferable evaluation model.
METHODS: A triangulated design combined three components: (1) a systematic review of existing value assessment frameworks to identify dimensions, criteria, and indicators; (2) a meta-review of 147 systematic reviews to examine how DHIs are evaluated in practice, focusing on operationalized indicators; and (3) semi-structured interviews (N = 10) with digitally disengaged individuals and healthcare leaders to validate the relevance and comprehensiveness of the identified dimensions. All reviews followed PRISMA guidelines; qualitative analysis adhered to thematic and saturation standards.
RESULTS: The synthesis of 97 frameworks and 147 reviews revealed four interdependent value dimensions: (1) Subject (e.g., clinical outcomes, behavior), (2) Interaction (e.g., usability, access, engagement), (3) System (e.g., interoperability, efficiency), and (4) Society (e.g., sustainability, equity, legitimacy). Existing evaluations prioritize individual outcomes, while other dimensions remain fragmented. Interview data confirmed the relevance of all dimensions across diverse user groups, indicating the model’s broad applicability.
CONCLUSIONS: The Concentric Value Model offers a conceptually sound and empirically supported framework for evaluating DHIs. It addresses key gaps in current practice by integrating clinical, experiential, organizational, and societal perspectives. The model enhances transparency, supports strategic decision-making, and provides a scalable foundation for assessing digital health innovation beyond individual interventions.
Embedding the Patient Voice within EU HTA: How Can We Balance Inclusivity, Representativeness and Scientific Rigour?
Session Type: Issue Panel
Topics: Patient-Centered Research, Health Technology Assessment, Health Policy & Regulatory
Track: Patient-Centered Evidence
Level: Intermediate
Available On-Demand: Digital Conference Pass
ISSUE: The implementation of the HTA Regulation in Europe introduces new opportunities and challenges for integrating patient perspectives into Joint Scientific Consultations (JSC) and Joint Clinical Assessments (JCAs). Despite implementation in early 2025, there remain key outstanding questions: How do we establish clear and consistent frameworks for patient involvement? How do we balance inclusivity with representativeness by engaging patient organisations and carers? How do we manage conflicts of interest without excluding valuable expertise? How to ensure patient confidentiality of health status while allowing for open dialogue? How can we ensure access to understandable, timely information through plain language summaries? How should patient relevant outcomes be captured and interpreted?
OVERVIEW: Per Regulation 2021/2282, patients contribute as external experts in both the JSC and JCA procedures. They also engage via patient organisations in a stakeholder network maintaining dialogue with the HTA Coordination Group.
Panelists will explore practical solutions to the challenges, aiming to strengthen the legitimacy, transparency, and relevance of HTA processes across Europe.
Ruairi O’Donnell (moderator) will introduce the panel and the broader context of the discussion (10min).
Matteo Scarabelli will explore the policy shifts that have changed how the patient perspective is incorporated into decision-making, while identifying key challenges and opportunities inherent within the new HTA Regulation (12min).
Antonella Cardone will share insights from the patient organisation perspective, highlighting key initiatives undertaken to effectively integrate patient voice and perspective within HTA procedures (12min).
Justin Doan will share findings of the health technology developer (HTD) experience, exploring how companies can pragmatically integrate patient perspectives and outcomes into their development programmes and prepare for evidence-based procedures such as the JSC and JCA (12min).
Time (14min) allowed for questions/ panel discussion. Session will benefit those with involvement in national HTA procedures, from patient, assessor or HTD perspectives.
Moderator
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Ruairi O'Donnell
Cencora, London, United Kingdom
Speakers
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Matteo SCARABELLI
EFPIA, Brussels, Belgium
After a Phd in Philosophy, Matteo started building his expertise in the pharmaceutical market as responsible for the involvement of patients in Regulatory approval and access decisions for orphan drugs. He has then contributed designing the strategy of rare disease organisations across Europe for the new EU HTA, also participating in the genesis and in the inter-institutional negotiations of the HTA Regulation.
For three years now, he has been working at the heart of the pharmaceutical industry's efforts to prepare for the new European HTA setting and its implementation into national pricing and reimbursement systems.
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Antonella M Cardone
Cancer Patients Europe, Brussels, Belgium
Antonella Cardone is the CEO of Cancer Patients Europe, a pan-European all-cancer patient association. She is a Patient Advocacy Expert and Advisor to the Board of Pancreatic Cancer Europe (PCE), uniting physicians, patients, politicians, journalists, academia, and industry, and she is a member of Inspire2Live.
Antonella led the European Cancer Patient Coalition (ECPC) and served as Executive Director of the Fit for Work Global Alliance.
With 25 years of nonprofit experience in health, social, and employment sectors, she also directed the Global Smoke-free Partnership of the American Cancer Society.
Antonella holds Master's degrees in Science and Business Administration. She has been on the Boards of All.Can and Pancreatic Cancer Europe, where she was vice-chair.
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Justin Doan, MPH, MSc, DrPH
Pfizer, Lake Mary, FL, United States
Battle of the Bots: Navigating the Landscape of AI-Enabled Systematic Literature Review Platforms
Session Type: Issue Panel
Topics: Methodological & Statistical Research, Study Approaches, Organizational Practices
Level: Intermediate
As AI-enabled tools for systematic literature reviews (SLRs) rapidly multiply, HEOR professionals face growing uncertainty about how to critically assess and differentiate platforms. While many vendors promise faster, cheaper, and more accurate reviews, recent comparative research highlights significant variability in performance, particularly beyond abstract screening. This session brings together leading vendors for a moderated, non-promotional discussion focused on transparency, methodological rigor, and real-world utility. Panelists will each respond to a common set of structured questions, covering core capabilities, evidence traceability, human-AI integration, and regulatory readiness. Drawing on a recent multi-tool evaluation study, the session will highlight gaps in standardization and the importance of stakeholder-informed procurement. Attendees will leave with a practical framework to evaluate AI SLR tools and understand which features best align with their research, compliance, or access needs. Each presenter is expected to briefly describe their tool’s unique contribution, share specific use cases, and respond to comparative prompts in a roundtable format. No product demos or sales pitches—just evidence-based insights to cut through the noise.
Moderator
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Ramiro E Gilardino, MSc, MD
Independent, Dubendorf, Switzerland
Impact-driven healthcare executive with 15+ years of leadership in Global Market Access, Health Policy, and HEOR. Successfully led strategies that improved patient access, drove reimbursement success, and shaped health policy in pharma, biotech, and global organizations. Expert in advancing HTA frameworks and forging stakeholder partnerships to promote health equity and deliver value-based healthcare solutions globally.
Speakers
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Angeline Dhas, BS
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Ranita M Tarchand, BA, MS
Nested Knowledge, St Paul, MN, United States
Ranita is trained as a health economist and epidemiologist. In her role as Vice President of Scientific Strategy and Client Success at Nested Knowledge, she works with private and public institutions on implementing AI methodology and digital transformation at all stages of the product lifecycle. Her research focuses on living HTA methods and artificial intelligence in evidence synthesis and RWE.
Innovation and Environmental Sustainability in Healthcare: A Multi-Stakeholder Responsibility
Session Type: Issue Panel
Topics: Health Policy & Regulatory, Health Technology Assessment
Level: Introductory
Available On-Demand: Digital Conference Pass
Issue: Health systems world-wide are under increasing burden due to the impact of climate change on peoples’ health. At the same time, increasing healthcare resource use across the world results in emissions that accelerate climate change and negatively impact the health of current and future generations. In this panel session, we will show-case two/three initiatives that are currently underway to address aspects of environmental sustainability in healthcare: The Sustainable Markets Initiative Health System Task Force (SMI-HSTF), the Sustainable Health Coalition (SHC) and the Environmental Sustainability in Health Technology Assessment Working Group (ESHTA) of Health Technology Assessment international. The discussion will focus on how multi-stakeholder and cross-organisational collaboration can contribute to develop and adopt effective approaches, measures and controls across the innovation life cycle of health technologies for enhancing population health and environmental sustainability.
Overview:
After a brief introduction of the issue, the moderator will invite the representatives of the three initiatives for an eight min overview from their initiative’s perspective. The moderated discussion will then evolve around the opportunities and challenges in each of the multi-stakeholder approaches, how the approaches taken by the initiatives differ or overlap, and what will help to achieve effective complementary pathways and methods that serve the overall common goal. The discussion will also address better integration of environmental sustainability in healthcare and better health of current and future generations. These discussions will include aspects such as feasibility, usefulness, cost, responsibilities, equity, ethics, and learning curves. The audience will be encouraged to join the discussion and in addition, engage through integration of interactive questions.
Moderator
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Anke-Peggy Holtorf, MBA, PhD
Health Outcomes Strategies, GmbH, Basel, Switzerland
Speakers
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Priscille de La Tour, MSc
Sanofi, Lyon, France
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Melissa Pegg, BSc, MSc
York Health Economics Consortium, York, United Kingdom
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Fiona Adshead, MSc
Sustainable Healthcare Coalition, London, United Kingdom
Fiona chairs the Sustainable Healthcare Coalition that inspires partnerships and action on sustainable healthcare and is an independent expert advisor and board member for national and global organisations on wellbeing and sustainability.
A high-profile public health leader with a track record of reframing thinking and developing innovative strategy and programmes at the heart of business and government, her previous roles include Deputy Chief Medical Officer and Director General in the UK Government, Director of Chronic Disease and Health Promotion at the World Health Organisation and Bupa’s Chief Wellbeing Officer.
Fiona is a visiting Professor at UCL and is a Senior Associate at Cambridge Institute for Sustainability Leadership.
What Is Your Estimand? Navigating Conditional and Marginal Effects in Cost-Effectiveness Modeling
Session Type: Issue Panel
Topics: Methodological & Statistical Research, Economic Evaluation, Study Approaches
Level: Advanced
Available On-Demand: Digital Conference Pass
ISSUE: There has been increased recognition of the formulation of research questions through an estimand, and of the importance of appreciating the differences between marginal and conditional summary measures. When effect modification is present, conditional and marginal effect estimates can yield conflicting conclusions, creating challenges for HTA decision-making. These conflicts arise in both effectiveness-only and cost-effectiveness evaluations. For economic evaluation, a critical methodological question emerges: does the structure of the economic model and the nature of the net health benefit function align with the type of baseline risk and treatment effect estimates (i.e. conditional or marginal) being used as inputs? Furthermore, different model types have varying capabilities to handle patient heterogeneity. Understanding when and why these conflicts occur, and how to resolve them through appropriate model-estimand alignment, is essential for valid HTA conclusions.
OVERVIEW: Ms. Cope will provide a session overview and outline the scope of decision-making challenges. Dr. Remiro-Azócar will provide an overview of conditional and marginal estimands, when they differ, and their implications for transportability to different target populations. Dr. Phillippo will focus on network meta-analyses and population-adjusted indirect treatment comparison methods for deriving conditional or marginal treatment effects. Dr. Jansen will address the alignment between baseline risk and treatment effect estimand types for use in model-based cost-effectiveness evaluations, how the net health benefit function determines appropriate inputs, and the capabilities and limitations of different model types to obtain the cost-effectiveness estimates of interest. The session concludes with 15 minutes of debate lead by Ms. Cope on these questions: When should treatment decisions be based on conditional versus marginal effects? How should economic models be structured to properly handle the type of baseline risk and treatment effect estimates? Where should any marginalization occur in the modeling process? This session will benefit modelers, statisticians, and decision-makers.
Moderator
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Shannon Cope, MSc
Precision AQ, VANCOUVER, Canada
Shannon Cope is a leader in evidence generation and synthesis supporting the comparative effectiveness of new treatments required for regulatory and health technology assessments. Based in Vancouver, Canada, she serves as a Senior Advisor to Precision AQ, where she leads health economics and outcomes (HEOR) studies and supports Joint Clinical Assessments in Europe.
She has expertise in network meta-analysis, external control analyses, population-adjusted analyses, trial and individual-level surrogacy analyses, multi-level network meta-regressions that integrate data from randomized controlled trials with patient and study-level evidence, expert elicitation for time-to-event outcomes, and AI for evidence synthesis. Her work has contributed to numerous peer-reviewed publications and conference presentations, particularly in oncology and chronic disease management.
She holds a Master of Science degree in Health Administration from the University of Toronto and has been recognized for her contributions to methodological innovation in HEOR.
Speakers
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Antonio Remiro Azócar, PhD
Novo Nordisk, Madrid, Spain
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David Phillippo, BSc, MSc, PhD
University of Bristol, Bristol, United Kingdom
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Jeroen P Jansen, PhD
Precision AQ, San Francisco, CA, United States
Jeroen P. Jansen, PhD, is a methodologist working at the intersection of evidence synthesis, biostatistics, and health economics. He is an associate professor in the Department of Clinical Pharmacy in the School of Pharmacy at the University of California, San Francisco, and chief scientist, Health Economics & Outcomes Research at the Precision Medicine Group.
For the past 15 years, Dr. Jansen has worked on research to understand the clinical and economic value of healthcare interventions. His research has frequently been conducted in the context of health technology assessment (HTA) with a focus on comparative effectiveness and cost-effectiveness. Prompted by the challenges encountered in applied research projects, he has performed methodological research. Notable contributions are the development of novel statistical methods to overcome the typical challenges in model-based cost-effectiveness evaluations characterized by gaps in the evidence base and complex evidence structures. Furthermore, Dr. Jansen led initiatives to develop guidance for consumers and producers of network meta-analysis studies. He has promoted a more transparent and credible approach to model-based health economic evaluations and led the development of open-source simulation models to illustrate its feasibility.
Dr. Jansen has been involved in the ongoing development of an R software package to develop simulation models for health economic evaluations. His current research interests are the clinical and economic value of precision medicine, incorporating health disparities in health economic modeling studies, and statistical methods for evidence synthesis. He has published extensively in his areas of expertise and is widely cited. He is co-author of a textbook on network meta-analysis for decision-making and was associate editor for the Journal for Research Synthesis Methods. Dr. Jansen has a PhD in epidemiology from the Erasmus University in the Netherlands
11:00 - 11:30
Break (Exhibit Hall)
Session Type: General Meeting
11:30 - 12:45
Plenary Session: RWE in European Healthcare Decision Making—What's in it for Patients?
Session Type: Plenary
Topics: Real World Data & Information Systems, Health Policy & Regulatory, Patient-Centered Research
Level: Intermediate
Available On-Demand: Digital Conference Pass
Real-World Evidence (RWE) is increasingly shaping healthcare decision-making across Europe, not just for systems and stakeholders, but for the patients they serve. By capturing how treatments perform in everyday settings, RWE offers critical insights into patient outcomes, experiences, and unmet needs. At the heart of European efforts like DARWIN EU, collaborative networks are laying the groundwork for more connected, consistent, and patient-centered evidence generation. These initiatives aim to translate complex real-world data (RWD) into actionable knowledge that improves safety, access, and effectiveness of treatments across diverse patient populations. This plenary session will discuss the opportunities and challenges of RWE from a multi-stakeholder perspective and will explore how strategic cross-border partnerships and technological advancements—including AI—are helping to realize RWE’s full potential for patients. From regulators to researchers, all stakeholders have a role to play in ensuring that RWE efforts deliver meaningful, measurable value for patients.
Moderator
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Karen Facey, BSc, PhD
University of Edinburgh, Utrecht, Netherlands
Speakers
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Maria Dutarte, MSc
European Patients’ Academy on Therapeutic Innovation (EUPATI), Utrecht, Netherlands
Maria Dutarte is the Executive Director of the European Patients’ Academy on Therapeutic Innovation (EUPATI). Maria has previously worked for the European AIDS Treatment Group (EATG) coordinating patient involvement in a number of training initiatives and scientific projects. She has also worked for different scientific and international organisations, e.g. French National Research Center for Scientific Research (CNRS), International AIDS Society (IAS), The Global Fund to Fight AIDS, Tuberculosis and Malaria and The International Foundation for Science (IFS). Her educational background is in International Education and Communications.
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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.
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Sofie Gustafsson, PhD
Pfizer, Stockholm, Sweden
Dr. Sofie Gustafsson serves as the Global Director of RWE Partnerships and Innovations for International Developed Markets at Pfizer and is an affiliated researcher in Global Public Health at the Karolinska Institute. She holds a PharmD from Uppsala University, complemented by experience in both community and hospital pharmacy settings—an early foundation that inspired her commitment to optimizing drug utilization. Driven by this interest, Dr. Gustafsson pursued a second degree and subsequently earned a PhD in economics from Lund University, specializing in health economics.
Throughout her career, Dr. Gustafsson has been a driving force in integrating real-world evidence (RWE) into healthcare decision-making, with a particular emphasis on health technology assessment (HTA). While working at Sweden’s HTA agency, TLV, she was instrumental in developing and implementing the use of RWE for health technology assessment, including managed entry agreements and value-based pricing models for rare disease therapies. Her contributions have influenced patient access and advanced HTA practices.
Dr. Gustafsson has also contributed to national healthcare prioritization as a board member at Prioriteringscentrum, Linköping University. Internationally, she has been actively engaged with the Nordic Pharmaceutical Forum, the International Horizon Scanning Initiative (IHSI), and several IMI/IHI consortiums—Europe’s largest health-focused public-private partnership. She currently serves on the EHDEN Foundation Industry Advisory Board and sits on the steering committee of the IHI GREG Consortium, collaborating with stakeholders across fifteen countries to advance the use of RWE in regulatory and HTA decision-making.
At Pfizer, Dr. Gustafsson leads the establishment of strategic data partnerships that enable RWE studies across all therapeutic areas and stages of development for International Developed Markets. She also acts as the principal liaison between Pfizer Inc. and the Karolinska Institute.
With over fifteen years of experience spanning the pharmaceutical industry, academia, and public agencies, Dr. Gustafsson has successfully led complex, cross-functional projects and built strategic alliances to promote the effective use of RWE. Her commitment remains focused on fostering multi-sector collaboration and maximizing the impact of real-world data to accelerate patient access to innovative therapies.
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Renske Los, PhD
Erasmus MC, Rotterdam, Netherlands
Plenary Session: Special Remarks and Leadership Reports
Session Type: Plenary
Available On-Demand: Digital Conference Pass
As ISPOR Europe 2025 draws to a close, join us for final reflections from this year’s conference. ISPOR’s Chief Executive Officer will share key takeaways from the conference, and our Chief Science Officer will share important updates on ISPOR’s scientific direction and how members can engage.
Immediately following the opening presentations, the scientific plenary panel will take the stage.
Speakers
