Program

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

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.
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.
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.
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

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.
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

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.
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.
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.
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

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.
William Rawlinson, MSc
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.
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

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.
Rok Hren, MSc, PhD
Hren, 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).
Katarzyna Kolasa, PhD
Kozminski University, Warsaw, Poland
Driven with passion to improve healthcare, Katarzyna has focused her academic and business career on health economics.

She has been working with multiple pricing and reimbursement challenges worldwide for the last 25 years, while holding various regional and global leadership positions at Astra Zeneca, BMS, Biogen Idec, Lundbeck, GE Healthcare, Straub Medical, BD, and the Swedish County Council of Kalmar. Katarzyna is mentor and consultant to start ups involved in the development of innovative medical devices and digital health solutions from both Holland and Poland.

Since 2000, she has been an academic teacher and supervisor for over 30 MBA and PhD students. In partnership with the Polish Medical Research Agency, Deloitte Digital and the Polish Central Hospital of Ministry of Interior Affairs, she founded the first Digital Health 6 months educational program designed for digital transformation leaders working in the healthcare sector. Katarzyna developed an innovative Master Program Health Economics & Big Data (HEBDA) with the first edition being financed by EU Power Grant 2018 as well.

She is the founder of the Global Special Interest Group Digital Health and short courses “The Role of Digital Endpoints in the Value Generation for Health Technologies”, “Risk-Sharing/Performance-Based Arrangements in Developing Countries” for ISPOR, The Professional Society for Health Economics and Outcomes Research. She is currently a member of the ISPOR Education Council and a previous member of the ISPOR Health Science Policy Council as well.

Katarzyna has dedicated her academic research towards methodological advancements into the value assessment of pharmaceuticals, medical devices, and digital health solutions. Passionate about Big Data, she led the first project of machine learning adaptation for the optimal utilization of CT scanners granted by the Polish Ministry of Health. Since January 2022, she is the leader of AI special interest group at the Polish Chamber of Physicians. With the patronage of the Polish Parliamentary Commission for Innovation & Digitalization, she organized the first dialog about the societal preferences towards the adoption of AI in the healthcare in Poland.

Being a coauthor of more than 50 IF publications, she has presented her research at more than 60 international scientific conferences. As of 2022, Google Scholar reports over 730 citations to her work.

8:00 - 17:00

Reimbursement Systems for Pharmaceuticals in Europe

Session Type: Short Course

Topics: Health Policy & Regulatory

Level: Intermediate

Separate registration required.

Pharmaceutical reimbursement systems in Europe are complex, diverse, and heterogeneous, shaped by national policies, healthcare priorities, regulatory frameworks and underlying epistemological choices. This short course offers an in-depth exploration of these systems, focusing on the decision-making processes that determine whether and how new medicines are reimbursed or accessible across key European markets.

Unlike marketing authorization for pharmaceuticals (mainly regulated at the European level by EMA), pricing and reimbursement decisions in Europe are managed by individual member states. Health care services are generally covered by a single public health insurer operating under the Ministry of Health supervision. As a monopoly buyer (monopsony), this situation provides a leading position for the public health insurer to set reimbursement conditions. On the other side, pharmaceutical companies may be in a monopoly situation with a single provider or very few for the same medicinal class. Therefore, based on each country’s set of regulations, processes, and values, wide variations exist in pricing and reimbursement decisions of pharmaceuticals driven by power positions and desirability of new products. This course is essential for professionals involved in market access, health economics, regulatory affairs, and policymaking, providing the tools and knowledge needed to navigate the evolving landscape of pharmaceutical reimbursement in Europe.

Speakers

Mondher Toumi, 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.
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.
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.
Keith H Tolley, BA, MPhil, MPP
Tolley Limited, Buxton, United Kingdom
Keith has over 35 years’ experience in health economics across academia (University of York - Centre for Health Economics and University of Nottingham, UK 1987-1997), for several pharmaceutical companies, including GSK, Pfizer, and Ortho Biotech (1997-2005), and in consultancy as a Director at Mapi (Adelphi) Values and now Tolley. He has direct experience of HTA as performed by NICE and SMC, and reimbursement and pricing issues around Europe. Keith has managed and strategically contributed to company submissions to NICE and SMC across a range of disease areas. He has also reviewed and been involved in the development of health economic models for NICE and SMC and other HTA bodies and has reviewed economic models for their suitability (eg, structure, data inputs) for drug reimbursement purposes.

Keith is also a health economics assessor with the SMC, a position he has held since 2005, having previously been an industry representative on the NDC. In 2013, Keith also became an assessor for the All Wales Medicine Strategy Group (AWMSG) and has provided expert advice as part of the NICE Early Scientific Advice Program.
Krzysztof Kloc
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

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.”
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.”
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.”
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

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).
Jag Chhatwal, PhD
Harvard Medical School / Massachusetts General Hospital, Boston, MA, United States
Jag Chhatwal, PhD, is the director of the Institute for Technology Assessment at Massachusetts General Hospital and an associate professor at Harvard Medical School. He also serves as core faculty at the Center for Health Decision Science, Harvard T.H. Chan School of Public Health. Dr. Chhatwal has co-authored more than 125 original research articles and editorials in leading peer-reviewed journals. His research has informed health policy decisions at prominent organizations including the White House, the World Health Organization, and the CDC, and has been featured in major media outlets such as CNN, Forbes, National Public Radio, The New York Times, and The Wall Street Journal. Dr. Chhatwal serves as an associate editor of Value in Health and as guest editor for its special issue on artificial intelligence. He is also a member of the ISPOR Generative AI Working Group.
Dalia Dawoud, BSc, MSc, PhD
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.
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

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.
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.
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

Lou Garrison, PhD
The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, Seattle, WA, United States
Lou Garrison, PhD, is professor emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.

For the first 13 years of his career, Dr. Garrison worked in non-profit health policy at Battelle and then the Project HOPE Center for Health Affairs, where he was the Director from 1989-1992. Following this, he worked as an economist in the pharmaceutical industry for 12 years. From 2002-2004, he was vice president and head of Health Economics & Strategic Pricing in Roche Pharmaceuticals, based in Basel, Switzerland.

Dr. Garrison received a BA in Economics from Indiana University, and a PhD in Economics from Stanford University. He has more than 150 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies.

Dr. Garrison was elected as ISPOR President for July 2016-June 2017, following other leadership roles since 2005. He recently co-chaired the ISPOR Special Task Force on US Value Frameworks. He was selected in 2017 by PharmaVOICE as being among “100 of the Most Inspiring People” in the industry. He recently received the PhRMA Foundation and Personalized Medicine Coalition 2018 Value Assessment Challenge First-Prize Award as lead author on a paper on “A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine.”
Marlene Gyldmark, 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.
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).
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

Uwe Siebert, MPH, MSc, ScD, MD
UMIT TIROL - University for Health Sciences and Technology; Harvard Chan School of Public Health, Hall in Tirol, Austria
Uwe Siebert, MD, MPH, MSc, ScD, is a professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL-University for Health Sciences and Technology in Austria and director of the Division for HTA in the ONCOTYROL–Center for Personalized Cancer Medicine in Austria. He is also adjunct professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health and Affiliated Researcher in the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston.

After medical school, he worked for several years as a physician in international public health projects in West Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in decision sciences at the Harvard School of Public Health.

His research interests include applying real-world evidence-based quantitative, causal and translational methods from public health, epidemiology, artificial intelligence, comparative effectiveness research, health services and outcomes research, economic evaluation, modeling, and health data a d decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, clinical guideline development, public health policies and patient guidance. His research focuses on cancer, infectious disease, cardiovascular disease, neurological disorders, and others.

He has been leading projects/work packages in several EU FP7, H2020 and Horizon Europe projects (eg, ELSA-GEN, BiomarCaRE, MedTecHTA, DEXHELPP, EUthyroid, FORECEE, MDS-RIGHT, RECETAS, CORE-MD, EUREGIO-EFH, CIDS, OnCoVID, 4D PICTURE, CATALYSE). He teaches HTA, health economics, modeling, epidemiology, causal inference and target trial emulation, and data and decision science for academia, industry, and health authorities in Europe, North and South America, and Asia. He directs the Continuing Education Program on Health Technology Assessment & Decision Sciences (htads.org).

He has served as member of the ISPOR Directors Board and as president of the Society for Medical Decision Making (SMDM). He is a leadership member of the ISPOR Personalized/Precision Medicine SIG, a member of the Latin America Consortium Advisory Committee of ISPOR, and co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force. He is a member of the Oncology Advisory Council and the National Committee for Cancer Screening of the Austrian Federal Ministry of Health.
He has authored more than 400 publications (> 30,000 citations, H index > 80), and is editor of the European Journal of Epidemiology. Further information Internet: http://htads.org, umit-tirol.at/dph, hsph.harvard.edu/uwe-siebert, Twitter: @UweSiebert9, LinkedIn: uwe-siebert9.
Felicitas 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).
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

Rob Abbott
ISPOR, Lawrenceville, NJ, United States
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

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

Carole Longson, PhD
Manchester, United Kingdom
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
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.
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

Danny Yeh, PhD
Aesara, Burlingame, CA, United States

Speakers

Gunes Sevinc, BSc, MSc, PhD
Ardea Outcomes, Vancouver, BC, Canada
Hannah Staunton, MSc
Roche Products Ltd, Welwyn Garden City, United Kingdom
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

Brett Hauber, PhD
Pfizer, New York, NY, United States

Speakers

Verity Watson, BA, MS, PhD
RTI Health Solutions, Manchester, United Kingdom
David Meads
University of Leeds, Leeds, United Kingdom
Evi Germeni, PhD
University of Glasgow, Glasgow, United Kingdom

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

Matias Olsen, MSc
European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), Brussels, Belgium

Speakers

Antonella Cardone, MSs, MBA
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.
Mark Lawler, PhD
Queen's University,, Belfast, United Kingdom
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

Izabela Pieniazek, MSc
Certara, KRAKOW, Poland

Speakers

Rok Hren, MSc, PhD
Hren, 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).
Anna Kowalczuk, PhD, MSc
Agency for Health Technology Assessment and Tariff System, Warszawa, Poland
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

Hamid Mahmoudpour, PharmD, PhD
Merck Healthcare KGaA, Darmstadt, Germany

Speakers

Benjamin Bates
Rutgers, New Brunswick, NJ, United States
Miriam Koopman, MD, PhD
Utrecht University, Utrecht, Netherlands
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

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

Laura Pizzi, MPH, PharmD
ISPOR, Lawrenceville, NJ, United States
Laura Pizzi is Chief Science Officer at ISPOR.
Andrew Briggs, DPhil
London School of Hygiene & Tropical Medicine, London, United Kingdom
Malina Müller, BA, MA, PhD
WifOR Institute, Darmstadt, Germany
Oriana Ciani, BSc, MSc, PhD
SDA Bocconi, Milan, Italy

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

Jessica Federer, Yale IRB
Advisory Board Member, New York, NY, United States

Speakers

Philippe Habets
Netherlands
MD PhD in Computational Neuroscience. Automating evidence synthesis with EvidenceHunt.
Eelko den Breejen, MSc.
Pfizer, Capelle a.d. IJssel, Netherlands
Andreas Altemark, Masters
Roche, Basel, Switzerland

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

Mariam Bibi, BSc, PhD
GetReal Institute, Utrecht, Netherlands

Speakers

Anke van Engen
IQVIA, Amsterdam, Netherlands
Wim Goettsch, MSc, PhD
Utrecht University; Zorginstituut Nederland, Utrecht; Diemen, Netherlands
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

11:15 - 13:00

Lunch Service (Exhibit Hall)

Session Type: General Meeting

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

Vladimir Guzvic
Health Insurance Fund of Republic of Srpska, Banja Luka, Bosnia And Herzegovina

Speakers

Anna Kowalczuk, PhD, MSc
Agency for Health Technology Assessment and Tariff System, Warszawa, Poland
Maciej Grys
Certara, Cracow, Poland
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.

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

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

Mona Khalid
Dublin, Ireland
Clark Paramore, MSPH
Biogen, Cambridge, MA, United States
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

Moderator

Sean D Sullivan, PhD
University of Washington, Seattle, WA, United States
Sean D. Sullivan, BScPharm, MSc, PhD, is Professor and Dean, School of Pharmacy. He holds a joint appointment as Professor of Health Services in the School of Public Health. He holds adjunct appointments in the School of Medicine, the Public Health Sciences Division at the Fred Hutchinson Cancer Research Center, and at the Kaiser Permanente Washington Health Research Institute. He completed training in pharmacy at Oregon State University in 1983, obtained a master’s degree at the University of Texas in 1986 and a PhD in health economics and policy at the University of California, Berkeley in 1992. Dr. Sullivan has authored more than 400 journal articles, book chapters, task force reports and organizational and governmental publications. In many of these writings, he has assessed the evidence and applications of medical technology in relation to coverage and reimbursement decisions. His research interests include technology assessment, medical decision-making, and economic evaluation of medical technology. He is past president of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and past chair of the Academy of Managed Care Pharmacy (AMCP) Executive Committee of the Format for Formulary Submissions – the United States evidence-based guidelines for formulary decision making. Dr. Sullivan served as a past member of the Medicare Evidence Development and Coverage Advisory Committee, a past member of the Regence Blue Shield and Premera Blue Cross P/T Committee, and the Chair of the Premera Blue Cross Value Assessment Committee. He is also on the editorial boards of Value in Health, PharmacoEconomics, Journal of Medical Economics, and the European Journal of Health Economics. He was awarded the 2014 Stephen G. Avey Lifetime Achievement Award from the Academy of Managed Care Pharmacy (AMCP) and the 2015 APhA Academy of Pharmaceutical Research Sciences (APRS) Research Achievement Award.

Speakers

Olivia Wu, MSc, PhD
University of Glasgow, Glasgow, United Kingdom
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.
Nicholas Adlard, MA, MBA, MSc
Novartis Pharma, Busserach, Switzerland

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

CARLO FEDERICI, PhD
SDA Bocconi School of Management and Bocconi University, Milan, Italy

Speakers

Yasemin Zeisl, MSc
European Patients' Forum, Brussels, Belgium
Tom Melvin, MB, BCh, BAO, LRCPI & SI, BCL LLM (NUI)
University of Galway, Galway, Ireland
Marta Kerstan, MSc, EMBA
Johnson & Johnson MedTech, Zuchwil, Switzerland

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

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

Speakers

Rajdeep Kaur
Pharmacoevidence Pvt. Ltd., Mohali, India
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.
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

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

Denise Umuhire
European Medicines Agency, Amsterdam, Netherlands
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

Amr Makady, PharmD, PhD
Janssen-Cilag B.V., Breda, Netherlands

Speakers

Martin Price, MSc
Janssen-Cilag, High Wycombe, United Kingdom
Melissa Pegg, BSc, MSc
York Health Economics Consortium, York, United Kingdom
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

Grammati Sarri, MSc, PhD
Cytel, London, United Kingdom

Speakers

Blythe Adamson, MPH, PhD
Flatiron Health, New York, NY, United States
Stephen Duffield, PhD, MD
NICE, Liverpool, United Kingdom
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

Sebastian Heidenreich, BSc, MSc, PhD
Thermo Fisher Scientific, London, United Kingdom

Speakers

Elizabeth (Nicki) Bush, MS
OPEN Health, Zionsville, IN, United States
Catharina Groothuis-Oudshoorn, PhD
University of Twente, Enschede, Netherlands
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.

15:15 - 16:15

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

Malwina Holownia-Voloskova, MSc, PharmD
Certara, Cracow, Poland

Speakers

Oleksandra Oleshchuk, PhD, MD
Horbachevsky Ternopil National Medical University, Ternopil, Ukraine
Eva Turk, PhD, MSc
Nationa Institute of Public Health, Ljubljana, Slovenia
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

16:00 - 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

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

Dean Regier, BA, MA, PhD
BC Cancer - ARCC - UBC, Burnaby, BC, Canada

Speakers

Deirdre Weymann, MA
Simon Fraser University, Burnaby, BC, Canada
Emanuel Krebs, MA
Cancer Control Research, BC Cancer, Vancouver, BC, Canada
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

Dan Ollendorf, MPH, PhD
Institute for Clinical & Economic Review, Boston, MA, United States

Speakers

Saskia Knies, PhD
Zorginstituut Nederland, Diemen, Netherlands
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.
Praveen Thokala, MASc, PhD
SCHARR, University of Sheffield, Sheffield, United Kingdom

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

Susan A Oliveria, MPH, ScD
Thermo Fisher Scientific, New York, NY, United States

Speakers

Eleftherios samartzis, Dr. Med
University Hospital Zürich, Zürich, Switzerland
John S Sampalis, PhD
PPD, Laval, QC, Canada

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

Antonella Cardone, MSs, MBA
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

Julie Spony, MSc
European Commission, B, Belgium
Anne Willemsen, MSc
Zorginstituut Nederland, Diemen, Netherlands
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

Keith R Abrams, BSc, MSc, PhD
University of Warwick, Coventry, United Kingdom

Speakers

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
Rhiannon Owen, BSc, MSc, PhD
Swansea University, Swansea, United Kingdom
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

Martin Rost, PhD
AESARA, Boca Raton, FL, United States

Speakers

Robyn Carson, MPH
AbbVie, Morris Plains, NJ, United States
Patrick K Hopkinson, MBA, DPhil
PHTA Consulting, Wokingham, United Kingdom
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.

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

Michela Canobbio, MS
Becton Dickinson, Milano, Italy

Speakers

Neil Hawkins, MBA, MSc, PhD
University of Glasgow, Oxford, United Kingdom
Laura Sampietro-Colom, PhD, MD
Hospital Clinic de Barcelona, SABADELL, Spain
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

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

Antonella Cardone, MSs, MBA
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.
Michael Zaiac, MD, PhD
Daiichi Sankyo Europe GmbH, Zürich, Switzerland
Beate Wieseler
Cologne, Germany
Richard Sullivan, MD, PhD
Institute of Cancer Policy, Centre for Conflict & Health Research, King's College London, London, United Kingdom
Natasha Azzopardi-Muscat, MD, PhD
WHO Europe, Copenhagen, Denmark

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

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.
Natasha Azzopardi-Muscat, MD, PhD
WHO Europe, Copenhagen, Denmark

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

Mark Sculpher, PhD
University of York, York, United Kingdom

Speakers

Malina Müller, BA, MA, PhD
WifOR Institute, Darmstadt, Germany
Simon Walker
University of York, York, United Kingdom
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

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

Stephen Maddocks, BS
Medicines and Healthcare products Regulatory Agency, London, United Kingdom
Susanne S Michel, MD
Ascenian, Berlin, Germany
Formerly Department of Health Berlin and Department of Health England. Led European Health Research Institute at PricewaterhouseCoopers. Former Head of European Market Access at Evidera. Current Vice President Market Access and HEOR Europe at Clinigen.
Em Jennings, BS
Novartis, Cambridge, United Kingdom

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

Andrew Briggs, DPhil
London School of Hygiene & Tropical Medicine, London, United Kingdom

Speakers

Mireia Jofre-Bonet, PhD
Office of Health Economics, London, United Kingdom
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.
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

Sarah Ernst, PhD
Sanofi, Boston, United States

Speakers

Marie Mc Carthy, MBA
Novartis, Dublin, Ireland
Christine Guo, PhD
Actigraph, Pensacola, FL, United States

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

Laura Sawyer, BA, MSc
Symmetron, London, United Kingdom

Speakers

Milad Karimi
BioMarin, London, United Kingdom
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
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

David Ringger, PhD
Cencora, Bern, Switzerland

Speakers

Neil Grubert
Neil Grubert Consulting, Basildon, United Kingdom
Casper Paardekooper, MSc
Vintura, Utrecht, Netherlands
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

Kate Ren, PhD
ConnectHEOR|University of Sheffield, London|Sheffield, United Kingdom

Speakers

Hugo Pedder
University of Bristol, Bristol, United Kingdom
Lara Wolfson, MS, PhD
MSD, Zurich Flughafen, Switzerland
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

J. Jaime Caro, MD
Thermo Fisher Scientific, Lincoln, MA, United States

Speakers

Brian Reddy, BA, MSc, PhD
Pfizer, Dublin, Ireland
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

11:15 - 13:00

Lunch Service (Exhibit Hall)

Session Type: General Meeting

11:45 - 12:45

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

Maciej Niewada, PhD
ISPOR Poland Chapter, Warszawa, Poland

Speakers

Anna Kowalczuk, PhD, MSc
Agency for Health Technology Assessment and Tariff System, Warszawa, Poland
Martin Visnansky, MBA, MSc, PharmD, PhD
University of Veterinary Medicine and Human Pharmacy, Bratislava, Slovakia

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

Raju Gautam, PhD
ConnectHEOR, London, United Kingdom

Speakers

Janharpreet Singh, PhD
University of Leicester, Leicester, United Kingdom
Madhusudan Kabra
United Kingdom
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. Kelly 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

Min Huang, PhD
Merck & Co. Inc, North Wales, PA, United States

Speakers

Matthew Kelly, MD, MPH
University of Arkansas for Medical Sciences, Little Rock, AR, United States
Elamin Elbasha, BSc, MA, PhD
Merck, West Point, United States
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.

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

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

Panos Kanavos, BSc, MSc, PhD
London School of Economics and Political Science, London, United Kingdom
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.
Julie Spiesser, MSc
TAKEDA France, Zollikon, Switzerland

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

Moderator

Ruairi O'Donnell, PhD.
Cencora, London, United Kingdom

Speakers

Anja Schiel, PhD
Norwegian Medicines Agency, Oslo, Norway
James Ryan, BA, MSc
AstraZeneca, Cambridge, United Kingdom
Rod Taylor
Glasgow, United Kingdom

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

Christina Silver, PhD, Other
University of Surrey, Guildford, United Kingdom

Speakers

Karen Bailey, PhD
Evidera, London, United Kingdom
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

Lotte Steuten, MSc, PhD
Office of Health Economics, London, United Kingdom

Speakers

Simon Brassel, BSc, MSc
Office of Health Economics, London, United Kingdom
Nancy J Devlin, PhD
University of Melbourne, Melbourne, Australia
Marcin Czech, MBA, PhD, MD
Institute of Mother and Child, Warsaw, Poland

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

Tanja Fens, PharmD, PhD
RUG/University of Groningen, Groningen, Netherlands

Speakers

Peter Mol, PhD
College ter Beoordeling van Geneesmiddelen - Dutch Medicines Evaluation Board/ University Medical Ce, Groningen, Netherlands
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.
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

Deepika Thakur, PhD
Hoffman La Roche AG, Courtice, ON, Canada

Speakers

Shubhram Pandey, MSc
Pharmacoevidence Pvt. Ltd., SAS Nagar, Mohali, India
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.
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

15:15 - 16:15

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

Joanna A Lis, MBA, MSc, PhD
Sanofi, Warsaw, Poland

Speakers

Marcin Czech, MBA, PhD, MD
Institute of Mother and Child, Warsaw, Poland
Zoltan Kalo, PhD
Center for Health Technology Assessment, Semmelweis University & Syreon Research Institute, Budapest, Hungary
Jovan Mihajlovic, MSc, PhD
Jovan Mihajlovic, Novi Sad, Serbia

16:00 - 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

17:00 - 18:00

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

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

Mikel Berdud, PhD
Office of Health Economics, London, United Kingdom
Ilaria Villa, MSc
Fondazione Telethon, Milan, Italy
Kate Morgan, BA, MSc
Brussels, 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

Pamela Vo, MS, PharmD
EVERSANA, Basel, Switzerland

Speakers

Michal Stanák, MA, DPhil
National Institute for Value and Technologies in Health Care, Bratislava, Slovakia
Zsombor Zrubka, MBA, PhD, MD
Óbuda University, Budapest, Hungary
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

Wim Goettsch, MSc, PhD
Utrecht University; Zorginstituut Nederland, Utrecht; Diemen, Netherlands

Speakers

Neil McAuslane, BSc, MSc, PhD
Centre for Innovation in Regulatory Science, Glasgow, United Kingdom
Anja Schiel, PhD
Norwegian Medicines Agency, Oslo, Norway
Ansgar Hebborn, PhD
Roche Products Ltd, Basel, Switzerland

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

Track: Patient-Centered Evidence

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

Jaime Espin, MSc, PhD
EASP, Granada, Spain

Speakers

Daniel Fabian, MSc
Vienna, Austria
Americo Cicchetti, BSc, MASc, DPhil
UNIVERSITA’ CATTOLICA DEL SACRO CUORE, Rome, Italy
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

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

William Rawlinson, MSc
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.
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
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

Miranda Cooper, BSc, MSc
AstraZeneca, Barcelona, Spain

Speakers

Neil Hawkins, MBA, MSc, PhD
University of Glasgow, Oxford, United Kingdom
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).
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

Grace Hampson, MSc
Office of Health Economics, London, United Kingdom

Speakers

Hector Tejero, PhD
Gabinete de la Ministra de Sanidad, Madrid, Spain
Andrew Briggs, DPhil
London School of Hygiene & Tropical Medicine, London, United Kingdom
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

Jennifer Whitty, PhD
Evidera, London, United Kingdom

Speakers

Michael Bui, MSc
University of Twente, Enschede, Netherlands
Jorien Veldwijk, BSc, MSc, PhD
Erasmus University, Rotterdam, Netherlands
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 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

Jessica Weddle
France

Speakers

nazanin MEHIN, Pharm D
Sanofi, Boston, MA, France
Dao Tu Xuan, PharmD, MSc
Takeda Pharmaceuticals U.S.A., Cambridge, MA, United States
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

Ron Akehurst, PhD
Lumanity, Sheffield, United Kingdom

Speakers

Fleur Chandler, MSc
Independent, Middlesex, United Kingdom
Becky Pennington
University of Sheffield, Sheffield, United Kingdom
Erik Landfeldt, MSc, PhD
IQVIA, Täby, Sweden

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

Katja Rudell, BSc, MSc, PhD, Other
Kielo Research, Cambridge, United Kingdom

Speakers

Katja Rudell, BSc, MSc, PhD, Other
Kielo Research, Cambridge, United Kingdom
Bryan Bennett, BSc, PhD
Jazz Pharma, Berwick-upon-Tweed, United Kingdom
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

Cornelis Boersma, PhD
Health-Ecore, Zeist, Netherlands

Speakers

Josephine Mauskopf, PhD
RTI Health Solutions, DURHAM, NC, United States
J. Jaime Caro, MD
Thermo Fisher Scientific, Lincoln, MA, United States
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

Cristina Masseria
Aesara, Madrid, Spain

Speakers

Gergana Zlateva, PhD
Pfizer Inc., New York, NY, United States
Brian O'Rourke, BSc, PharmD
Brian O'Rourke Health Care Consulting Inc., Ottawa, ON, Canada
Neal Masia, PhD
EntityRisk, Inc., Princeton, NJ, United States

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

Claire L Simons
OPEN Health, Rotterdam, Netherlands

Speakers

Elizabeth Sigworth, PhD
OPEN Health Group, Rotterdam, Netherlands
Michael Hurst, BSc, MSc
Bristol Myers Squibb, Uxbridge, United Kingdom

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

Tracy Westley, MSPH
Lumanity, Dundas, NB, Canada

Speakers

Yu-Heng Liu, PharmD, MSc
Lumanity, Sheffield, United Kingdom
Benjamin C Kearns, BSc, MSc
The University of Sheffield, Sheffield, United Kingdom
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

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

Speakers

Anirban Basu, PhD
University of Washington, Seattle, WA, United States
Varun Ektare, MPH
Indence Research Private Limited, Thane West, India
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: 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

Martina Garau, BA, MSc
Office of Health Economics, London, United Kingdom

Speakers

Bettina Ryll, PhD, MD
Melanoma Patient Network Europe, Uppsala, Sweden
Niklas Hedberg, MSc
TLV, Stockholm, Sweden
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

Cristina Masseria
Aesara, Madrid, Spain

Speakers

Adrian Cassidy, PhD
Novartis, Basel, Switzerland
Entela Xoxi, MSc, PharmD, PhD
Independent Consultant, Roma, Italy
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

Meng Li, MS, PhD
Tufts Medical Center, The Center for the Evaluation of Value and Risk in Health, Boston, MA, United States

Speakers

Gunnar Esiason, MBA
Boomer Esiason Foundation, New Canaan, CT, United States
Richard Xie, PhD
RA Capital Management, Newton, MA, United States
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

Louis Matza, PhD
Evidera, Bethesda, MD, United States

Speakers

Nancy J Devlin, PhD
University of Melbourne, Melbourne, Australia
Janine Verstraete, PhD
University of Cape Town, Cape Town, South Africa
Donna Rowen, BA, MSc, PhD
SCHARR, University of Sheffield, Sheffield, United Kingdom

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

Ruairi O'Donnell, PhD.
Cencora, London, United Kingdom

Speakers

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.
Antonella Cardone, MSs, MBA
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.
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 forum brings together four leading vendors (DistillerSR, Nested Knowledge, CapeStart, and PICO Portal) 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. In the end, the audience will draw their conclusions and assign a winner of this scientific pitch! Session speakers to be determined.

Moderator

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.

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

Anke-Peggy Holtorf, MBA, PhD
Health Outcomes Strategies, GmbH, Basel, Switzerland

Speakers

Priscille de La Tour, MSc
Sanofi, Lyon, France
Melissa Pegg, BSc, MSc
York Health Economics Consortium, York, United Kingdom
Fiona Adshead, PhD
Sustainable Healthcare Coalition, Newton Abbot, United Kingdom

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

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

Antonio Remiro Azócar, PhD
Novo Nordisk, Madrid, Spain
David Phillippo, BSc, MSc, PhD
University of Bristol, Bristol, United Kingdom
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

Karen Facey, BSc, PhD
University of Edinburgh, Utrecht, Netherlands

Speakers

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.
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.
Sofie Gustafsson, PhD
Pfizer, Stockholm, Sweden

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

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

ISPOR Europe 2025

9 - 12 November

Program

Sunday, 9 November

7:00 - 18:00

Registration Hours

8:00 - 12:00

Advanced Patient-Reported Outcomes

Developing Decision-Grade Real-World Evidence

Health Economic Modeling in R: A Hands-on Introduction

Introduction to Applied Generative AI for HEOR

Risk-Sharing/Performance-Based Arrangements in Developing Countries

8:00 - 17:00

Reimbursement Systems for Pharmaceuticals in Europe

12:00 - 13:00

Break (Coffee Service, Lunch on Own)

13:00 - 17:00

Budget Impact Analysis II: Practical Steps to Building a BIA and Enhanced Applications

Prompt Engineering for HEOR: Practical Skills and Use Cases for HEOR Professionals

Using LLMs to Simplify Real-World Evidence Research

Valuation of Innovative Drugs

Causal Inference and Causal Estimands from Target Trial Emulations Using Evidence from Real-World Observational Studies and Clinical Trials

15:30 - 16:30

First-Time Attendee Orientation

Monday, 10 November

7:00 - 17:00

Registration Hours

7:30 - 8:30

Morning Coffee Service

8:30 - 9:45

Plenary Session: Welcome Remarks and Keynote—Celebrating 30 Years of ISPOR

Plenary Session: Patient Engagement in Healthcare Investments—A Promise or a Practice?

9:30 - 19:00

Exhibit Hall Open

9:45 - 10:15

Morning Break: Coffee and Connect (Exhibit Hall)

10:15 - 11:15

Measuring What Matters: Practical Pathways for Personalized Endpoints and Patient-Centric Approaches in HTA Evaluation

Patient Preference Evidence in Health Technology Assessment: What Do We Really Know?

Unlocking the Untapped Potential of Comprehensive Genomic Profiling: Improving Patient Access and Outcomes

Managed Entry Agreements: Can CEE Countries Mutualize Lessons Learned?

Lines of Therapy in Oncology Real World Data: Navigating Complexity for Robust Evidence

Value Assessment from a Whole Health Perspective: Opportunities and Challenges

Integrating AI into Market Access Workflows

External Control Studies - What Does it Take to Get Real?

10:30 - 13:30

Poster Session 1

11:15 - 13:00

Lunch Service (Exhibit Hall)

11:45 - 12:45

AI in CEE Health System Decision Making: Can Payers, Patients, and Policy Align for Smarter Access?

13:45 - 14:45

The Evidence Was There. So Why Didn’t It Matter?

How Could Patient-Centric HTA Evolve in the Changing Global Pricing Landscape?

Implementing a Program for Early Feasibility Studies in Europe: Goldmine or Fool’s Gold?

Transparent, Traceable and Reliable Generative AI in HEOR: A Hands-On Workshop on Retrieval-Augmented Generation (RAG) For Evidence Generation

Patient Experience Data for Evidence Generation to Support Regulatory and Access Decision Making

Does Environmental Sustainability Warrant a Spot on ISPOR’s Value Flower: A Multi-Stakeholder Debate

Is Transferring Evidence Across Europe a Herculean Task? The Opportunities and Challenges of Transportability Analyses in the Context of European Union Joint Clinical Assessments

COAs and Patient Preference Information: Building on a Promising but Challenging Relationship

14:45 - 15:15

Afternoon Break: Coffee and Connect (Exhibit Hall)

14:45 - 15:45

Meditation Session: Meditation for Mental Clarity

15:15 - 16:15

Building HTA Capacity in CEE: Is it Possible to be Made Within a Common Roadmap - From Pilot Projects to Sustainable Systems

16:00 - 19:00

Poster Session 2

17:00 - 18:00

Operationalizing Life-Cycle Health Technology Assessment For Precision Medicine

Diving into the Intended and Unintended Consequences of how the Societal Perspective is Captured in Cost-Effectiveness Analyses

Real World Evidence – From the Clinician’s Perspective

Embedding Effective Patient Involvement in EU Joint Clinical Assessments

Barriers and enablers to the adoption of innovative methods for health technology assessment (HTA) decision-making

Beyond Regulatory Approval: Making Patient Experience Central to HTA Decision-Making

Integrating Capacity-Enhancing Innovations in Healthcare Systems: Are Current Methods Adequate to Demonstrate the Impact on Efficiency and Capacity?

18:00 - 19:00

Welcome Reception (Exhibit Hall)

Tuesday, 11 November

7:00 - 17:00