Program

Sun May 17

7:00 AM - 6:00 PM

Registration Hours

Session Type: General Meeting

8:00 AM - 12:00 PM

Applied Generative AI for HEOR: Introduction

Session Type: Short Course

Topics: Methodological & Statistical Research

Level: Introductory

Separate registration required.

The rapid advancement in generative artificial intelligence (GenAI) presents an opportunity for transformative potential in the field of health economics and outcomes research (HEOR). This course provides an introductory understanding of generative AI models with a particular focus on large language models (LLMs), which are transforming the field of HEOR. Participants will be provided with an overview of the most appropriate ways to access LLMs, going beyond the use of chatbots. Further, they will be given insights into how to use prompt engineering, retrieval-augmented generation (RAG) and agents to conduct scientific research and gain an understanding on issues pertaining to privacy and security when using GenAI for HEOR. Participants will further explore specific applications of these models for conducting robust scientific HEOR research in, for example, systematic literature reviews (SLR) and economic evaluation. The course aims to equip participants with the knowledge to begin to use generative AI techniques for specific HEOR contexts and to appreciate how these innovative approaches can enhance HEOR activities. Practical exercises using Python and relevant AI frameworks will be incorporated for participants to follow along.

PREREQUISITES: Students should have a general understanding of common HEOR concepts such as SLRs and cost-effectiveness models. Knowledge of Python or similar programming languages such as R is considered a benefit but not required.

Speakers

William Rawlinson
Estima Scientific, London, United Kingdom
Will is a senior health economist at Estima Scientific holding a degree in Physics and Philosophy from the University of Oxford. Will has 4 years’ experience developing cost-utility models and has specialized in applications of generative AI to health economic modelling. Will has published on the automation of R modelling using large language models (LLMs), and more recently has focused on applications of LLMs to Excel modelling and model reporting.
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.
Sven L Klijn, MSc
Bristol Myers Squibb, Utrecht, Netherlands
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.

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
Apodeixis Strategies, LLC, 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
Cytel Inc., London, United Kingdom
Dalia Dawoud, PhD, is Research Principal, HTA Policy and Strategy. She is also the Director and CEO of PEHTA Consulting Ltd. and holds a professor position at the Faculty of Pharmacy, Cairo University. She has over 15 years experience as a health economist and researcher. Her work is largely focused on the application of HEOR in HTA and clinical guideline development. She worked at leading organizations including NICE, where she led a portfolio of HORIZON Europe projects such as HTx, EDiHTA and SUSTAIN HTA, and the Royal College of Physicians, London. She is widely published in the areas of health economics and outcomes research and serves as associate editor for Value in Health and as director on ISPOR Board of Directors (2023-2026). She is also a member of the ISPOR AI Working Group and ISPOR Living HTA Working Group.
Turgay Ayer, PhD
Value Analytics Labs, Boston, MA, United States
Turgay Ayer, PhD, holds the Virginia C. and Joseph C. Mello Chair and serves as the research director for Healthcare Analytics and Business Intelligence at the Center for Health & Humanitarian Systems at Georgia Tech. He is also the chief technology officer at Value Analytics Labs. Dr. Ayer holds a courtesy appointment at Emory Medical School where he teaches Big Data Analytics courses and serves as a Senior Scientist at the Centers for Disease Control and Prevention (CDC). Dr. Ayer’s research focuses on health economics modeling (HEOR), real-world evidence, data science, machine learning, econometric modeling, and healthcare analytics. He has published over 80 peer-reviewed journal papers and more than 300 conference abstracts, with his work featured in top-tier business, engineering, medical, and health policy journals. His research has attracted substantial attention from major media outlets, including The Wall Street Journal, The Washington Post, US News, and NPR. A recognized expert in HEOR, Dr. Ayer has been at the forefront of applying generative AI to navigate healthcare systems and support better decision-making. He has contributed significantly to the development of advanced models for predicting healthcare outcomes and designing innovative cost-effectiveness analysis frameworks. Under his leadership, Value Analytics Labs has focused on the development of cutting-edge technologies, including ValueGen.AI, to enhance healthcare analytics and improve the efficiency of healthcare decision-making processes.

Leveraging Real-World Data Throughout the Medical Devices and Diagnostics Product Lifecycle

Session Type: Short Course

Topics: Real World Data & Information Systems

Level: Intermediate

Separate registration required.

This course will focus on the opportunities and practical applications of conducting real-world data (RWD) studies and generating real-world evidence (RWE) for medical devices and diagnostics (MDD). RWD is increasingly being leveraged to support a variety of purposes in the MDD space, including regulatory, reimbursement, health technology assessment (HTA), and business needs. Leveraging RWD, especially secondary data sources, for MDD poses unique challenges, such as the difficulty in identifying devices in RWD sources, device operator characteristics potentially influencing outcomes, and the need to consider continuous device iterations in RWE generation. Thus, high-quality data and carefully designed studies are critical to increase the credibility and acceptance of RWD/E for MDD.

This course will provide an overview of the best practices, processes, and methods to design and execute studies to gather market insights and generate high-quality evidence for multiple stakeholders. The course will review different types of secondary data sources and methods to conduct descriptive analyses and comparative effectiveness research along the MDD product lifecycle. Specific topics will include the common questions that are answered with secondary data and the challenges and potential solutions that are unique to MDD products. Case studies will focus on a variety of technologies, from new technologies to follower products, and the strategies that are used to increase the chances of acceptance to gain and expand market access.

Speakers

Belinda A Mohr, PhD
Medtronic, Phoenix, AZ, United States
Arthi Chandran, MPH, MS, DrPH
ABBOTT, Santa Clara, CA, United States
Bijan J Borah, MSc, PhD
Mayo Clinic College of Medicine, Edina, MN, United States

Causal Inference and Causal Diagrams in Big, Real-World Observational Data and Pragmatic Trials

Session Type: Short Course

Topics: Real World Data & Information Systems

Level: Advanced

Separate registration required.

Innovative causal inference and target trial emulation methods are needed for the design and analysis of big real-world observational data and pragmatic trials. This course will introduce the principles of causation in comparative effectiveness research, the use of causal diagrams (directed acyclic graphs; DAGs) and focus on causal inference methods for time-independent confounding (multivariate regression, propensity scores) and time-dependent confounding (g-formula, marginal structural models with inverse probability of treatment weighting, and structural nested models with g-estimation). The “target trial” concept and a counterfactual approach with “replicates” will be used to apply causal methods to big real-world datasets with case examples from oncology, cardiovascular disease, HIV, nutrition and obstetrics. The course will consist of lectures, case examples drawn from the published literature and interactive discussion. The intended audience includes researchers from all substance matter fields, statisticians, epidemiologists, outcome researchers, health economists and health policy decision makers interested either in methods of causal analysis or causal interpretation of results based on the underlying method.

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.
Douglas Faries, PhD
Consultant, Alma, AR, United States
Doug Faries has a PhD in Statistics from Oklahoma State University. He spent 34 years as a statistician in the pharmaceutical industry, retiring as a vice president of Real-World Access and Analytics at Eli Lilly and Company where he led the development of real-world analytical capabilities for the business. Doug has extensive experience with the design and analysis of observational research including comparative effectiveness analyses and sensitivity analysis for unmeasured confounding. He remains active in the statistical community with over 150 peer-reviewed manuscripts, authoring books and book chapters on analysis of observational data, and teaching short courses on causal inference at national meetings.

Designing a Patient-Centered Strategy for Drug Development and Value

Session Type: Short Course

Topics: Patient-Centered Research

Level: Intermediate

Separate registration required.

This course focuses on how to design and govern a fit-for-purpose patient-centered strategy before instruments, endpoints, or implementation approaches are locked into a clinical program. Rather than teaching how to execute a specific measurement system, the course equips participants to make informed, defensible decisions about whether, when, and how tools such as patient-reported outcomes should be used to support patient-centered evidence generation across development, regulatory review, reimbursement, and value communication. The course will provide an overview of where and how patient-centered evidence can provide value to decision makers across the drug development lifecycle.

Participants will learn structured frameworks for identifying what aspects matter most to patients and other decision makers, identifying available measures and other ways to gather patient-centered information, assessing the strengths and limitations of existing instruments, determining when adaptation or new development may be warranted, and how to develop a measurement strategy to inform multiple decision makers. The course emphasizes critical judgment over mechanics, helping participants understand how qualitative and quantitative evidence expectations vary by decision maker (patients, clinicians, regulators, HTA bodies, payers) and how these differences influence early strategy choices.

Through interactive discussion and real-world examples, the course explores common strategic missteps—such as misaligned endpoints, overambitious claims, or inappropriate instrument selection—that can undermine downstream clinical, regulatory, or access objectives. Success stories are used to illustrate how strong upfront strategy development enables smoother execution later. By the end of the course, participants will be prepared to lead patient-centered strategy conversations, challenge assumptions, align cross-functional decision makers, and set realistic objectives—whether the next step is selecting an existing measurement system, adapting a legacy instrument, or deciding not to pursue patient-reported outcome endpoints at all.

PREREQUISITE: This course assumes that participants will have a basic knowledge of key PRO-related concepts (eg, health-related quality of life, symptoms, impacts, a general knowledge of the PRO development steps, and a working knowledge of PRO measurement within clinical programs)

Speakers

Ari Gnanasakthy, MBA, MSc
RTI- Health Solutions, RESEARCH TRIANGLE PARK, NC, United States
Ari Gnanasakthy is head of Patient-Reported Outcomes at RTI-HS. Prior to RTI-HS.

Mr. Gnanasakthy was the executive director and head of the Patient-Reported Outcomes Center of Excellence at Novartis Pharmaceuticals. He has almost 25 years of experience in the pharmaceutical industry. At Novartis, he worked in several departments, including Biostatistics, Health Economics, Pricing, and Outcomes Research. After receiving his bachelor's degree in mathematics, statistics, and computing, Mr. Gnanasakthy joined Rothamsted Experimental Station (UK), where he was responsible for the statistical analysis of survey data of agricultural soil in England and Wales. He then joined the Milk Marketing Board (UK), where he was a part of the team responsible for modeling lactation curves of dairy cows. Mr. Gnanasakthy's extensive experience in the field of statistics and outcome research has resulted in numerous abstracts and almost 40 publications. Throughout his career, Mr. Gnanasakthy has developed and validated over a dozen patient-reported outcomes instruments and currently serves in the editorial board of Cancer Clinical Trials and a reviewer for many professional journals, including Value in Health.
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, Research Triangle Park, NC, United States
Shanshan Qin, PhD, received her training on Qualitative Methodology (including statistic inference and estimation, traditional and modern testing theories, structural equation modeling, and mixed and mixture modeling) at University of Georgia. She has 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.
Diane Whalley
RTI- Health Solutions, Manchester, United Kingdom

8:00 AM - 3:30 PM

Budget Impact Analysis in Practice: A Hands-On Course on Strategic Conceptual Design, Model Building, and Communication

Session Type: Short Course

Topics: Economic Evaluation

Level: Intermediate

Separate registration required.

This hands-on, interactive course equips participants with the conceptual and practical tools needed to develop, adapt, and communicate budget impact analyses (BIA) for real-world decision making.

The course begins with a brief review of BIA concepts and a 6-step approach to strategically design these analyses. We will discuss practical applications, including customization to accommodate payer-specific data; balance of structural simplicity, accuracy, and face validity; interpretation of results; and development and communication of compelling value messages. Technical topics will include static versus dynamic budget impact models, good Excel model-building practices, considerations for device and diagnostic technologies, and incorporation of realistic features such as patient copayments and availability of generics.

Instructors will walk through 2 budget impact models programmed in Excel (1 static and 1 dynamic) and will work with participants during hands-on exercises to customize and adapt these models to specific real-world circumstances. These Excel-based models will be provided to participants in advance, both for use during the session and for later reference. Throughout the course, participants will have opportunities to network and to work in small groups to discuss key concepts, plan and implement model adaptations, and develop and communicate value narratives.

This course is designed for participants seeking to deepen their understanding of BIA, gain practical exposure to Excel-based models, and strengthen their ability to communicate BIA in ways that influence decision making. Registrants who wish to participate in the interactive portions of the course will need to bring materials for handwrtten activities.

PREREQUISITE: Participants are expected to have familiarity with basic budget-impact analysis concepts and working knowledge of Microsoft Excel. Individuals who need foundational knowledge of budget impact models are encouraged to register for the introductory level virtual short course “Primer on a 6-Step Approach to Budget Impact Analysis” on March 25, 2026. A microcourse on the basics of Budget Impact Analysis is available in the ISPOR Education Center.

Speakers

Stephanie Earnshaw, PhD
Access Strategy Consulting, 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 (eg, 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
AESARA, San Diego, CA, United States
Anita Brogan is vice President of Value Evidence at AESARA, Inc. In her role, she uses analytical techniques to assess and communicate the health and economic impact of emerging pharmaceutical and biotechnology interventions. She leads development of user-friendly and transparent cost-effectiveness, budget-impact, optimization, population, and other models programmed in Microsoft Excel and other platforms. She has expertise in modeling methodologies such as Markov and other stochastic models, infectious disease dynamic transmission 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, norovirus, Ebola, influenza, cystic fibrosis, bone health, mental health, women’s health, oncology, chronic pain, age-related macular degeneration, hospital-acquired infection, financial portfolio optimization, and vehicle routing. Her research has been presented at various professional conferences and published in peer-reviewed journals.

Dr. Brogan holds a PhD in Operations Research from the University of North Carolina at Chapel Hill. She serves on editorial board of Pharmacoeconomics and the ISPOR education council. 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). She is co-author of the book “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
Thor-Henrik Brodtkorb, PhD
RTI Health Solutions, Ljungskile, Sweden
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.

11:00 AM - 12:00 PM

First-Time Attendee Orientation

Session Type: General Meeting

New to ISPOR? Join us for this engaging and informative session designed to help first-time attendees make the most of their experience at ISPOR 2026. You’ll gain insights into the conference structure, key sessions, and networking opportunities while connecting with fellow newcomers and ISPOR leaders. Whether you’re looking to navigate the agenda, maximize learning, or build professional relationships, this session will set you up for success. Don’t miss this chance to start your ISPOR journey with confidence!

12:00 PM - 1:00 PM

Break (Lunch on Own)

Session Type: General Meeting

1:00 PM - 5:00 PM

Applied Generative AI for HEOR: Advanced Architectures

Session Type: Short Course

Topics: Methodological & Statistical Research

Level: Intermediate

Separate registration required.

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

This intermediate-level course builds upon basic concepts and is designed for HEOR professionals, data scientists, and decision makers seeking to understand not only how GenAI works, but how to implement and evaluate it effectively within regulated and evidence-driven environments. The course provides a practical framework for moving “from prototype to practice,” describing the lifecycle of GenAI implementation—from early sprints and pilot projects to production deployment. Participants will explore both technical and organizational perspectives, including workflow orchestration, modularization, scaling, and change management.

Retrieval-Augmented Generation (RAG) is a cornerstone architecture that integrates external knowledge bases into LLM workflows. Faculty will discuss why RAG is particularly relevant for HEOR, demonstrating how external information (eg, clinical data, published evidence, HTA guidance) can be incorporated in GenAI workflows according to best practice standards and used to improve factual accuracy and traceability. A guided practical session is included so participants become familiar with how to implement a simple RAG pipeline, learning how to chunk data, generate embeddings, and augment prompts for domain-specific use. The course will also provide an extensive overview of Agentic AI, a fast-evolving frontier in AI automation. Participants will examine how autonomous AI “agents” can coordinate multi-step HEOR processes—such as literature updates, model maintenance, or simulated committee reviews—while maintaining control and accountability. A second practical session will demonstrate an agentic workflow in action, showcasing task orchestration, monitoring, and boundary setting. Beyond technical topics, there will be a focus on evaluation and validation of GenAI solutions for HEOR, where participants will learn how to critically assess GenAI tools in terms of reliability, reproducibility, and regulatory alignment. This will also be discussed in the context of potential ethical concerns around the application of AI. Using frameworks such as ELEVATE-GenAI, and referencing NICE and FDA guidance, participants will learn how to ensure that AI-driven outputs meet HEOR’s high standards for quality and transparency. By the end of this course, participants will understand how to bridge the gap between exploratory AI use and operational excellence. They will leave with actionable frameworks and hands-on knowledge to evaluate, implement, and govern GenAI tools that enhance productivity, transparency, and scientific integrity across HEOR activities.

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

Speakers

Sven L Klijn, MSc
Bristol Myers Squibb, Utrecht, Netherlands
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.
Rajdeep Kaur, PhD
Pharmacoevidence Pvt. Ltd., Mohali, India
Dr. Rajdeep Kaur is the Lead of AI Sciences at Pharmacoevidence, with a Ph.D. in Computer Science and Engineering and 17+ years of expertise in advanced technologies. Her work focuses on Generative AI, machine learning, and cloud-enabled data systems, with a strong emphasis on real-world healthcare applications. She has successfully led multiple GenAI projects, combining deep technical expertise to deliver impactful AI-driven solutions.
Ghayath Janoudi, MSc, PhD
Loon, Cantley, QC, Canada
Dr. Ghayath Janoudi, MBBS, MSc, PhD, is the Founder and CEO of Loon, an AI-driven clinical research and market access company developing scientifically validated AI agents for Health Economics and Outcomes Research (HEOR), Health Technology Assessment (HTA), and reimbursement strategy. A medical doctor and health outcomes researcher by training, Dr. Janoudi holds a PhD in Clinical Epidemiology with a specialization in artificial intelligence for clinical research. He previously held senior leadership roles at Canada’s Drug Agency (formerly CADTH) and at clinical research organizations, where he led work on HTA, drug reimbursement policy, and value evidence evaluation. A recognized thought leader in AI for clinical discovery, Dr. Janoudi is a well-published author in AI-enabled evidence synthesis, and was named Canada’s 2024 Emerging Healthcare Leader for his contributions to accelerating timely and equitable access to innovative therapies.
Siguroli Teitsson, BSc, MSc
Bristol Myers Squibb, Denham, United Kingdom
Siguroli Teitsson is a Director in Global HEOR Economic & Predictive Modeling at Bristol Myers Squibb. In his role, Siguroli leads the advancement of innovative modeling and analytics in oncology, and drives the integration of cutting-edge AI automations in HEOR and market access, streamlining workflows to accelerate patient access to medicines. With a background in engineering and health economics, he has previously held senior roles in CROs and has extensive publication record in innovative analytics within the field of HEOR, contributing to advancements in methodology and practice.

Developing Decision-Grade Real-World Evidence

Session Type: Short Course

Topics: Real World Data & Information Systems

Level: Intermediate

Separate registration required.

In this course, participants will be introduced to the principles of what makes real-world evidence (RWE) decision-grade, including an extended example. In the first half of the course, we will review the most recent RWE frameworks and guidelines and examine case studies in which RWE was used in regulatory and HTA approval. The second half of the course is an extended example in which participants will examine a study that could support an indication expansion and interactively discuss how choices made in the design and implementation may affect the meaning and interpretability of results.

PREREQUISITE: Students are expected to be familiar with relevant concepts and methodologies for analyzing real-world data.

Speakers

Sebastian Schneeweiss, ScD, MD
Brigham and Women's Hospital and Harvard Medical School, Boston, MA, United States
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.

Using RWE to Inform the Value and Affordability Assessment of Cell and Gene Therapies

Session Type: Short Course

Topics: Real World Data & Information Systems

Level: Intermediate

Separate registration required.

This short course explores the role of real-world evidence (RWE) in supporting the economic evaluation of cell and gene therapies (CGTs). Many CGTs are one-time therapies that have the potential to offer transformative sustained benefits for patients with severe conditions. In many situations, the ‘do-nothing’ alternative is the norm, so the need for a control arm may be futile yet reducing the options for patients to be included in allegedly active arms. However, at launch there is often resistance from payers to reimburse these potentially transformative therapies due to the limited validity of the supporting evidence (small, single arm trials, etc) which leads to uncertainty regarding: the size and heterogeneity of the patient population eligible for CGTs; the definition of standard of care and natural disease progression, given CGTs may unlock treatment possibilities of previously deemed untreatable and rare diseases; the novel therapy’s duration of benefit; and the relative effectiveness of the novel therapy compared to the current standard of care, particularly, as cell and gene therapies are often only supported by a single arm trial.

Payer concerns with these uncertainties in the evidence are heightened by the typically high up-front costs associated with cell and gene therapies and the consequence for their affordability. While there is generally not a good understanding of the effect sizes and costs of standard of care. This course will provide an overview of the potential contribution, planning and use of RWE to help address these concerns. We will assume payer archetypes that are focused on one or more of relative effectiveness and cost-effectiveness (value), and budget impact (affordability). Within these archetypes, we will discuss the role and acceptability of RWE to inform payment and policy with emphasis on eligibility, appropriate comparators, durability of benefit, spending for patients that meet criteria for eligibility and the development of appropriate outcome-based agreements. A strong focus will be brought on validity (internal and external) of the existing data. We will further provide participants with real-world examples of using RWE to inform policy making.

This course will target a wide range of participants, from medical operations to HEOR, interested in understanding the depth of issues to consider when balancing access with payment for CGTs.

PREREQUISITE: This course requires familiarity with basic economic evaluation and HTA concepts and methodologies of pharmaceuticals.

Speakers

Daniel Gladwell, BA, MSc, PhD
Lumanity, Sheffield, United Kingdom
Dan Gladwell is the chief scientific officer for Lumanity HE&HTA. A health economist by background, he has a particular passion for demonstrating the value of highly innovative therapies that make a transformative difference to patient outcomes. Dan supported one of the first CAR T-cell therapies to be assessed by NICE. Since that appraisal Dan has been continuously engaged in supporting patient access to cell and gene therapies through engaging in HEOR evidence generation planning efforts at the Global, Regional and National levels; and informing efforts to shape the HTA policy context for cell and gene therapies.
Antal T Zemplenyi, MSc, PhD
University of Colorado Skaggs School of Pharmacy and Pharmaceutical Science, Denver, CO, United States
Oriol de Sola-Morales, MSc, PhD, MD
Fundacio HiTT, Barcelona, Spain
Renske MT ten Ham, PhD
Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Amsterdam, Netherlands

PROs in Clinical Trials: Endpoint Selection, Regulatory Strategy and Label Claims

Session Type: Short Course

Topics: Patient-Centered Research

Level: Advanced

Separate registration required.

This short course shows clinical development teams how to implement PROMIS® (Patient-Reported Outcomes Measurement Information System) in registration trials and regulatory submissions, using FDA Patient-Focused Drug Development guidance. Participants learn regulator-aligned methods applicable to other PRO measures, from endpoint selection to labeling claims. PROMIS provides comprehensive health assessment across physical, mental, and social domains using IRT-based scoring, computer-adaptive testing, digital capabilities, and validation across 80+ languages. The course covers domain/measure selection, protocol specification, estimands, meaningful change determination, psychometric evaluation, digital ePRO implementation, global translations, and regulatory review preparation. Interactive case-based learning uses real disease examples to guide participants through endpoint selection, protocol development, and regulatory defense. Faculty analyze successful label claims and challenging submissions to identify critical success factors and common pitfalls applicable across PRO instruments, including real-world evidence and HTA applications. A laptop is recommended for interactive exercises and accessing online resources.

PREREQUISITES: Participants should have basic familiarity with clinical trials and patient-reported outcomes, clinical trial design and regulatory processes. Prior PROMIS® knowledge is not required.

Speaker

David Cella, PhD
Northwestern University Feinberg School of Medicine, Chicago, IL, United States

Societal Valuation of Innovative Medicines: Emphasis on Investment Perspective and Orphan Disease

Session Type: Short Course

Topics: Health Policy & Regulatory

Level: Intermediate

Separate registration required.

Explore the value assessment of innovative drugs from the perspectives of relevant stakeholders, their respective data requirements, and their methods and processes. Gain a better understanding of the value assessment from the investor perspective, with a focus on orphan drugs and advanced therapy medical products (ATMPs). The value of medical innovation depends on a stakeholder's perspective in different decision contexts. Regulatory authorities (EMA, FDA) mainly consider the clinical value of medical innovation. In the context of coverage decisions, national health authorities may adopt a broader perspective by including clinical, economic criteria, and sometimes even other criteria such as equity and social values. For pricing and reimbursement, ""value-based pricing"" is the most widely accepted approach across countries, but it can vary from a narrow concept based on the incremental cost-effectiveness ratio (ICER) threshold to broader societal or holistic approaches.

Value-based pricing determines the maximum price from the national payer perspective. In the context of the investment decision, this price should exceed the minimum price for the investor acting in the international financial market to make a financial valuation. Furthermore, there are numerous other stakeholders, eg, patients, physicians, healthcare insurers, and employers--with their specific assessment of the value of medical innovation including, for example, patient and family quality of life, real-world effectiveness, budget impact, and the costs of lost productivity. Familiarity with health economic evaluation is desirable, but the course assumes little or no familiarity with economic valuation theory.

Speakers

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

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

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

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

5:00 PM - 7:30 PM

ISPOR College Bowl

Session Type: General Meeting

Join us for a competition of HEOR knowledge where student chapter teams go head-to-head for bragging rights and prizes! Formerly known as the Student Research Competition, this bracket-style tournament is an exciting and rapidly growing event.

Mon May 18

7:00 AM - 8:30 AM

Morning Coffee Service

Session Type: General Meeting

Don't miss the start of the day with the Plenary Session. Enjoy your morning coffee as you listen to dynamic presentations intended to inspire and empower.

7:00 AM - 5:00 PM

Registration Hours

Session Type: General Meeting

8:30 AM - 10:00 AM

US Pharmaceutical Policy: Leading or Following?

Session Type: Plenary

Topics: Health Policy & Regulatory, Economic Evaluation, Health Technology Assessment

Track: Access and Drug Pricing

Level: Advanced

Americans have run out of patience with paying three times more for innovative medicine than other developed countries. President Trump is negotiating ways to bring US drug prices in line with other countries through a most-favored-nation (MFN) approach. This strategy builds on efforts of the Biden Administration's Inflation Reduction Act (IRA) to directly negotiate drug prices in Medicare. In a wide-ranging discussion with Trump Administration officials, the biopharmaceutical industry, and payers, we will discuss MFN, IRA, and other efforts to lower drug prices in the United States -- with a view toward the economic and regulatory consequences and long-term consequences for the US and abroad.

Moderator

Dana P. Goldman, PhD
University of Southern California Schaeffer Institute for Public Policy & Government Service, Los Angeles, CA, United States

Speaker

Courtney Piron
Novartis, Washington, DC, United States

9:30 AM - 7:00 PM

Exhibit Hall Hours

Session Type: General Meeting

9:45 AM - 10:15 AM

Coffee and Connect

Session Type: General Meeting

Head to the exhibit hall to connect with fellow attendees and exhibitors over a steaming cup of coffee.

10:30 AM - 11:30 AM

Operationalizing Artificial Intelligence Guidance to Create Best in Class Abstraction and Curation Approaches

Session Type: Issue Panel

Topics: Health Policy & Regulatory, Real World Data & Information Systems, Study Approaches

Track: AI

Level: Intermediate

The panel will discuss global regulatory guidance on the use of artificial intelligence (AI) to support regulatory decision-making. The increasing prevalence of AI and its intersection with real-world data (RWD) is fundamentally changing how evidence is generated and assessed. AI’s ultimate value relies on extracting meaningful insights from complex RWD sources. A vast amount of this RWD exists in unstructured formats such as clinician notes and pathology reports. There is a pressing need to curate this data using natural language processing and other AI techniques. These techniques must be of sufficient quality so that the resulting analytic dataset can inform regulatory decision-making. The moderator will provide an overview of global guidances from authorities such as EMA, NICE, and CDA and they compare. Following the introduction, panelists will debate how best to operationalize the guidance, what is still needed from a regulatory perspective, and what biopharma needs in terms of assurances from data providers to meet regulatory expectations. The panelists will provide two differing company approaches to dissecting and implementing FDA’s 7-step process for establishing and assessing the credibility of an AI model for a specific context of use and the challenges they have encountered. A key piece of the discussion will focus on assessing data quality for data generated through this approach. Panelists will also discuss the need for more information from regulatory authorities on how to best operationalize certain aspects of the guidances, how this scheme may conflict with existing regulatory schemes such as 21 CFR Part 11, and what is needed from an international convergence or harmonization perspective. This session will equip HEOR professionals with multiple perspectives on how to navigate the technical and regulatory landscape of AI abstraction and curation approaches, ensuring that evidence generation strategies and HTA submissions create the best possible body of evidence.

Moderator

Nicholaas Honig, JD
Highlander Health, Duxbury, MA, United States

Speakers

Nate Nussbaum, MD
Target RWE, Durham, NC, United States
Dan Riskin, MBA, MD
Verantos, Inc., Menlo Park, CA, United States
Colin Anderson-Smits
Gilead, Boston, MA, United States

Is There a Consensus on the Framework for Evaluating Artificial Intelligence (AI)-Assisted Systematic Review Tools in HEOR?

Session Type: Issue Panel

Topics: Methodological & Statistical Research, Health Technology Assessment

Track: AI

Level: Intermediate

Issue: AI-assisted systematic literature reviews (AI-SLRs) have rapidly expanded, with dozens of commercial and open-source tools now available. However, the HEOR and HTA community lacks shared standards for evaluating tool performance, ensuring reproducibility, documenting risk, and benchmarking AI-SLR output against human-conducted reviews. As evidence synthesis teams increasingly adopt AI-SLRs for HEOR and HTA projects, a possible solution is to establish an evaluation framework from the perspectives of multiple stakeholders. A case competition (a.k.a. challenge) using AI-SLR tools could be used to test the common evaluation framework. This session will discuss the need for a common evaluation framework and a process to conduct the HEOR/HTA community-driven benchmarking challenge. Overview: This panel will bring together perspectives from ISPOR leadership, academia, AI methodology, and the pharmaceutical industry to co-create a path forward. An overview provided by Dr. Mullins will outline the current landscape and the role of ISPOR in shaping good-practice guidance for AI in evidence synthesis (5 min). A senior evidence generation lead (Dr. Zhang) will share real-world insights on the challenges and opportunities of AI-SLR adoption, highlighting vendor assessment, validation requirements, compliance considerations, quality control, and the balance between efficiency gains and decision-grade reliability (15 min). Dr. Shi will introduce the concept of case competitions, discussing the feasibility of shared reference datasets, “gold-standard” systematic reviews, and open evaluation protocols to support innovation and methodological rigor (10 min). Dr. Yang will then present a structured framework for evaluating AI-SLR tools, covering reference standard construction, accuracy metrics, reproducibility, workload reduction, hallucination/error types, and governance considerations aligned with GenAI reporting expectations (15 min). The session will conclude with interactive polling and collaborative consensus to identify priority actions, including whether HEOR/HTA communities should form a working group, issue guidance, or sponsor an AI-SLR challenge.

Moderator

C. Daniel Mullins, PhD
University of Maryland School of Medicine, Baltimore, MD, United States
C. Daniel Mullins is a Professor at the University of Maryland School of Pharmacy. He is Founder and Executive Director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, a community-academic partnership for patient-driven research. Dr. Mullins has received approximately $25 million in funding as a Principal Investigator from AHRQ, FDA, NCI, NHLBI, NIA, NIMHD, the Patient-Centered Outcomes Research Institute (PCORI) and various patient advocacy organizations and pharmaceutical companies. At the University of Maryland Baltimore (UMB), he received the Dr. Patricia Sokolove Outstanding Mentor Award and the Dr. Martin Luther King Jr. Faculty Diversity Award. He was named Researcher of the Year at UMB and was awarded a University System of Maryland Wilson H. Elkins Professorship. At ISPOR, he has served as Editor-in-Chief of Value in Health since 2010 and received the Marilyn Dix Smith Leadership Award in 2017 and the Avedis Donabedian Lifetime Achievement Award in 2024.

Speakers

Lizheng Shi, PhD
Tulane University School of Public Health and Tropical Medicine, New Orleans, LA, United States
Lizheng Shi, PhD, MsPharm, MA, is the Neal A. and Mary Vanselow Endowed Chair in the Department of Health Policy and Management at the School of Public Health and Tropical Medicine of Tulane University. He is the founding director of Tulane’s Health Systems Analytics Research Center (HSARC). Dr. Shi’s current health services research interest focuses on innovative health technologies to improve healthcare quality, access, and cost of patient-centered care from the equity perspective, using pharmaco-economics, health technology assessment, health analytics, and policy evaluation. Dr. Shi is dedicated to disseminating and translating population health knowledge at the local, national, and international levels. He is the associate editor of Value In Health and co-editor in chief for Pharmacoeconomics and Policy.
Yiduo Zhang, BA, MA, PhD
AstraZeneca, Gaithersburg, MD, United States
Mei Yang, PhD
EVYD Technology Limited, Short Hills, NJ, United States

Patient Experience Data as a Non-Negotiable: Can US Payers Afford to Stay Behind?

Session Type: Issue Panel

Topics: Patient-Centered Research, Health Technology Assessment, Economic Evaluation

Track: Access and Drug Pricing

Level: Intermediate

Issue While patient experience data (PED) - including PROs, quality-of-life measures, treatment preferences, and patient-generated data - are central to evidence expectations in U.S. regulatory decisions, their use in payer decision-making remains inconsistent. The FDA’s Patient-Focused Drug Development (PFDD) Guidance Series and the European Medicines Agency draft Reflection Paper on PED signal that patient experience data are considered part of the totality of evidence required for regulatory and access decisions. European HTA bodies increasingly treat QoL and patient-relevant outcomes as core determinants of added benefit and price negotiation. In contrast, the use of PED in formulary and access decisions by payers remains variable, limited to PRO data and coverage exceptions. Overview The panel will explore 3 questions: 1. Why does PED matter now? Panelists will discuss how PED strengthens clinical credibility, captures health outcomes beyond survival, and reflects elements of total cost of care such as functioning, productivity, and caregiver burden. Drawing on ISPOR Europe 2025 themes, the FDA PFDD Guidance Series, and the EMA PED Reflection Paper, the session will highlight expectations that PED be proactively incorporated into regulatory and reimbursement submissions. 2. Why hasn’t PED meaningfully influenced payer decisions? Despite increasing availability of PROs and PED, analyses show limited uptake in coverage and utilization management. Panelists will address methodological uncertainty, lack of standardization, perceived operational burden, and the belief that PED does not materially influence comparative effectiveness. 3. What would it take to change the dynamic? The discussion will identify practical actions for key stakeholders: • Industry: embed fit-for-purpose PED in pivotal trials and real-world studies; develop concise, payer-ready evidence packages. • Regulators/HTA bodies: clarify evidentiary standards; signal PED relevance beyond labeling; align approaches with EMA and FDA initiatives. • Payers: define how PED could influence access thresholds and contracting; increase transparency when PED informs decisions.

Moderator

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

Speakers

Brian O'Rourke, BSc, PharmD
Brian O'Rourke Health Care Consulting Inc., Ottawa, ON, Canada
Robyn Carson, MPH
AbbVie, Morris Plains, NJ, United States
Robyn T. Carson, MPH, is Vice President & Head of Patient-Centered Outcomes Research and HEOR-Strategy Aesthetics where she leads teams focused on integrating the patient voice through generation of patient experience data (PED) and ensuring deployment of best practices for clinical outcome assessment (COA) development, validation, and implementation across therapeutic areas at AbbVie. Robyn has driven patient-centered research in the pharmaceutical industry for 19 years where she has held roles in of increasing responsibility at Pfizer, Forest Labs, Actavis, Allergan and AbbVie. During her career, Robyn has made significant contributions to major product approvals and launches, as well as the development of multiple novel patient-reported outcome (PRO) instruments and innovative real-world research platforms. In addition, Robyn has led key departmental and enterprise-wide initiatives related to patient-focused drug development (PFDD) and patient-centricity. Robyn has also been a leader within the cross-industry Critical Path Institute PRO Consortium since 2008, currently serving as the Industry Co-Director. Prior to joining the pharmaceutical industry, Robyn conducted research at Columbia University, NYC Department of Health & Mental Hygiene, and served as a Research Fellow at the National Cancer Institute. Robyn holds a MPH in Epidemiology from Columbia University.
Denise Sánchez Palomo, JD, MS, MA
Opus Regulatory, New York City, NY, United States

Evolving Approaches to Measuring Benefits in Economic Evaluation: Do the Health Economics Methods Advisory (HEMA) Recommendations Go Far Enough for Global HTA Practice?

Session Type: Issue Panel

Topics: Health Technology Assessment, Economic Evaluation, Methodological & Statistical Research

Track: Expanded Value Measures

Level: Intermediate

Purpose: The Health Economics Methods Advisory (HEMA) group recently released recommendations proposing updated approaches to defining and measuring benefits in economic evaluation. These recommendations respond to growing interest in broader value elements, alternative benefit metrics, and approaches that better capture patient and societal outcomes. However, questions remain about whether HEMA's recommendations sufficiently reflect the field’s expanding methodological frontier and the needs of HTA agencies operating in diverse policy environments. Overview: This issue panel will examine the opportunities and limitations of the HEMA recommendations through a structured, multi-perspective debate. Dr. Padula will open the session with a brief overview (10 minutes) of the HEMA initiative and the international context for expanded value frameworks. Dr. Lakdawalla will assess the conceptual foundations of the proposals and their relationship to emerging economic methods. Dr. Ollendorf will discuss implementation challenges from an HTA practitioner’s perspective, while Dr. Steuten will evaluate the recommendations’ alignment with global HTA practice and consider whether they go far enough to accommodate novel value concepts. Each panelist will present for approximately 10 minutes, followed by a moderated exchange and audience discussion (20 minutes). The session will benefit HTA researchers, policymakers, payers, and methodologists seeking clarity on how evolving benefit-measurement approaches can be responsibly adopted in practice.

Moderator

William V Padula, PhD
University of Southern California, Rancho Palos Verdes, CA, United States

Speakers

Darius Lakdawalla, PhD
University of Southern California, Los Angeles, CA, United States
Darius Lakdawalla is a widely published, award-winning researcher and a leading authority on health economics and health policy. He holds the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation at the University of Southern California, where he sits on the faculties of the School of Pharmacy, the Sol Price School of Public Policy, and the Leonard D. Schaeffer Center for Health Policy and Economics, one of the nation’s premier health policy research centers.

His academic research has focused primarily on the economics of risks to health, the value and determinants of medical innovation, the economics of health insurance markets, and the industrial organization of healthcare markets. Dr. Lakdawalla serves as associate editor at the Journal of Health Economics and has previously served in this role at the American Journal of Health Economics and the Review of Economics and Statistics. His academic work has appeared in leading peer-reviewed journals of economics, health policy, and medicine, including the American Economic Review, Quarterly Journal of Economics, Health Affairs, the Journal of Health Economics, and the New England Journal of Medicine. In addition, his work has been featured by prominent popular press outlets, such as the Wall Street Journal, National Public Radio, Forbes, and the New York Times. Dr. Lakdawalla has also received the PhRMA Foundation Value Assessment Challenge Award, designed to encourage innovative approaches to defining and measuring value in health care, in 2019 (third place) and 2020 (first place), along with the ISPOR Excellence in Research Methodology Award, the Garfield Prize, and the Milken Institute Award for Distinguished Economic Research.
Dan Ollendorf, MPH, PhD
Institute for Clinical & Economic Review, Boston, MA, United States
Lotte Steuten, MSc, PhD
Office of Health Economics, London, United Kingdom

Integrating Equity Into HTA - Are Population Preferences Enough?

Session Type: Issue Panel

Topics: Health Technology Assessment, Economic Evaluation, Health Policy & Regulatory

Track: Expanded Value Measures

Level: Intermediate

Issue: Reducing health inequities requires prioritizing the health of those who are systematically disadvantaged. Equity-informative approaches to cost-effectiveness analysis (CEA), such as distributional CEA, assess equity impacts by weighting outcomes and opportunity costs of interventions across equity-relevant characteristics. These weights have been elicited through population surveys, often fielded online, under the assumption that in a tax-funded or insurance-based system, “the public” should decide the value of reducing health inequities. Yet, this seemingly democratic solution raises questions about whether these approaches are best suited to inform real-world decision making. In this issue panel we will discuss three challenges: representativeness, heterogeneity, and context. The use of online surveys may under-represent the preferences of those most disadvantaged and requires high literacy and numeracy. Results are heterogenous and sensitive to the allocation scenario, framing effects, survey design considerations, and individual characteristics. Finally, equity is context specific, and stakeholder priorities and political dynamics can shift across countries, health conditions, and equity dimensions. As decision contexts shift, the value ascribed to improving health equity may also shift, raising concerns that societal preferences elicited via online choice experiments misalign with real-world decisions. Taken together, these challenges highlight the need to clarify whose preferences should guide equity weighting in practice, and whether population-level surveys can meaningfully represent the priorities of those who experience the greatest health disadvantages. Overview: Following an introduction to equity preference research (Cadham), panelists will briefly present on the current state of elicitations in Canada (Iragorri) and the US (Slejko) with an eye towards the challenges outlined above. Kowal will then outline future directions for gathering preferences, focusing on subgroup definitions, sampling strategies, and ways to manage data gaps. Presentations will be followed by a 15-minute moderated discussion and 15-minute audience discussion.

Moderator

Christopher Cadham
Fred Hutchinson Cancer Center, Seattle, WA, United States

Speakers

Julia F. Slejko, PhD
University of Maryland Baltimore, Baltimore, MD, United States
Nicolas Iragorri, MS
University of Toronto, Toronto, ON, Canada
Stacey Kowal, BS, MSc
Genentech, Alameda, CA, United States

The Hows and Whys of Estimating Return on Investment (ROI) of HEOR to the Biopharmaceutical Industry

Session Type: Workshop

Topics: Economic Evaluation, Study Approaches

Level: Intermediate

Purpose: Health economics & outcomes research (HEOR) has become integral in the biopharmaceutical industry, yet its contributions to product success have rarely been quantified. This contrasts sharply with other components of biopharma spending, such as R&D and commercialization campaigns, in which measurement of financial impact and calculation of return on investment (ROI) are routine. The objective of this workshop is to describe the hows and whys of estimating the ROI of HEOR. Description: Participants of this workshop will learn the opportunities and challenges associated with ROI estimation to inform decision making on projects in the HEOR, RWE, and market access domains. Chris Blanchette (moderator) will set the stage by providing an overview of the issues and showcasing non-financial metrics historically adopted by industry to demonstrate impact (10 minutes). David Thompson will describe the ROI estimation framework, which combines decision-analytic modeling techniques with expected Net Present Value (eNPV) calculations of project costs and product revenues accruing over time to assess the potential value of an HEOR project (16 minutes). Montserrat Vera-Llonch will describe criteria that can be used to select HEOR projects for ROI estimation, based on high degrees of: (1) topicality, those projects at the forefront of current methodology and policy discussions; (2) quantifiability, those projects for which impact is clearcut and estimable; and (3) applicability, those projects that comprise a significant share of internal HEOR budgets (12 minutes). Craig Roberts will describe why biopharma HEOR functions may or may not want to routinely assess project value and how doing so could impact budget allocation decisions (12 minutes). The workshop will culminate with an interactive session in which attendees will be asked to suggest and prioritize HEOR projects for ROI assessment based on the criteria introduced in the session (10 minutes).

Moderator

Christopher M. Blanchette, MA, MBA, MSc, PhD
Novo Nordisk, Doylestown, PA, United States

Speakers

David Thompson, PhD
Rubidoux Research LLC, Manchester, MA, United States
Montserrat Vera-Llonch, MD MPH
Ionis Pharmaceuticals, Inc, Carlsbad, CA, United States
Craig Roberts, MBA, PharmD
Merck & Co. Inc, North Wales, PA, United States

Evidence Synthesis Across Multiple Outcomes: Unlocking the Value of Multivariate Methods

Session Type: Workshop

Topics: Methodological & Statistical Research, Clinical Outcomes, Patient-Centered Research

Track: Expanded Value Measures

Level: Intermediate

PURPOSE: Traditional network meta-analyses (NMA) typically focus on single outcomes like progression-free survival, yet clinical decision-making inherently requires balancing multiple endpoints simultaneously. This workshop brings together diverse perspectives to discuss innovative methods and the promising potential of multivariate evidence synthesis. How can multivariate approaches bridge otherwise disconnected treatment networks allowing for broader treatment comparisons? Can we jointly synthesize progression-free and overall survival through multi-state modeling for improved economic modelling? Can multivariate methods enable a more patient-centered treatment ranking by considering efficacy and safety related outcomes simultaneously? There are challenges to overcome and we address the complexities of between-outcome and between-comparison correlations, how to leverage these correlations to improve the precision of treatment effect estimates, and how to provide more comprehensive evidence that better reflects the multifaceted nature of clinical decision-making. DESCRIPTION: Attendees will gain practical insights into when and how to implement multivariate methods, understand their advantages over univariate analyses, and learn about emerging tools for the visualization of complex multi-outcome evidence. Dr. Williams will chair the session and begin by summarizing current practice (8 min). Dr. Campbell will then review how different multivariate NMA models can be used to bridge otherwise disconnected networks (15 min). Dr. Jansen will explain multi-state NMA of progression-free- and overall survival that is based on a Markov state-transition model where time-varying transition rates and relative treatment effects are modeled using aggregate-level data (15min). Finally, Dr. Chen will consider a new framework for PAtient-centered treatment ranking via Large-scale Multivariate NMA, termed as PALM (15 min). This workshop will engage attendees with real-time polling and structured discussion, leveraging the different perspectives of the panel to probe how multivariate methods can ultimately improve clinical decision-making.

Moderator

Harlan Campbell
Precision AQ, Rossland, BC, Canada

Speakers

Harlan Campbell
Precision AQ, Rossland, BC, Canada
Jeroen P Jansen, PhD
UCSF, 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
Yong Chen
University of Pennsylvania, Philadelphia, PA, United States

Value-Based Pricing for CMS Drug Price Negotiation: Mission Impossible?

Session Type: Issue Panel

Topics: Health Policy & Regulatory

Track: Access and Drug Pricing

Level: Intermediate

Issue/Overview: The CMS Medicare Drug Price Negotiation Program has now completed two negotiation cycles. Although the statute directs CMS to consider comparative clinical effectiveness, unmet clinical need, R&D and production costs, and other factors, the translation of these considerations into final negotiated prices remains opaque. With recent methodological work by Li et al. (2025) Xie et al. (2025), both in Value in Health, and Neumann et al. (2025) in Health Affairs Forefront, there is an interest in whether value-based pricing could serve as a transparent and analytically rigorous foundation for CMS. This issue panel will explore how negotiated prices might differ under value-based versus non–value-based approaches and examine the opportunities and challenges of integrating value assessment into CMS pricing decisions. The panel will discuss the practical, methodological, and policy implications of using value-based pricing to inform CMS negotiations, including transparency, feasibility, data requirements, stakeholder acceptance, and potential impacts on innovation and access.

Moderator

Peter Neumann, ScD
Tufts Medical Center, Boston, MA, United States

Speakers

Feng Xie, PhD
McMaster University, Hamilton, ON, ON, Canada
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.
Joshua Cohen, PhD
Tufts Medical Center, Boston, MA, United States

10:30 AM - 1:30 PM

Poster Session 1

Session Type: General Meeting

Poster Tours 11:45AM–12:30PM | Presenters will be with their posters from 12:30PM–1:30PM

11:15 AM - 1:15 PM

Lunch Service (Exhibit Hall)

Session Type: General Meeting

As you enjoy your lunch in the Poster and Exhibit Hall, seize the opportunity to engage in meaningful conversations with fellow attendees. Take this time to exchange ideas, forge new partnerships, or simply enjoy casual conversations.

1:45 PM - 2:45 PM

Use of Agentic AI to Create Health Economic Models in Both R and Excel

Session Type: Workshop

Topics: Methodological & Statistical Research

Track: AI

Level: Intermediate

Purpose Health economic models are designed to forecast health and cost outcomes associated with a specific treatment in a particular disease area. Technical specifications for such a model must be sufficiently detailed to serve as a blueprint for model programming. From the blueprint, the model can be implemented in Excel or coded in R. We will demonstrate how to leverage agentic AI to go from model concept to full implementation in both R and Excel. Description Workshop attendees will get working knowledge on the use of agentic AI to generate both the blueprint and the implemented economic model. The workshop will show how AI, with human in the loop, is used for the blueprint development and then for implementation. A detailed example in autoimmune disease will be used throughout. Participants will also receive a link to a site from where they can download the Excel engine, templates and the full example. [Jaime] will moderate the session, introducing the problem and guiding the active participation from the audience. [Brian] will describe the humans’ role in conceptualizing the model and introduce the autoimmune disease example. [Baris] will explain how a multi-agentic AI system with structured prompting can be used to develop, review and edit the specifications based on a modelling paper to generate the blueprint and then how a complementary agentic system can generate the corresponding R code, run analysis and develop the technical report. Then [Apoorva] will show how the same blueprint can be used with a general Excel template to create the spreadsheet version of the same model. [Jaime] will then lead the interaction with the participants, aided by probing questions and polling. This workshop will be valuable to researchers interested in leveraging agentic AI to create health economic models. The methodology of structured prompting with automated templates also can help with many other research tasks in the HEOR space.

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.
Apoorva Ambavane, MPH
Thermo Fisher Scientific, Dubai, United Arab Emirates

Behavioral Phenotyping for Value Assessment: Trajectory and AI Approaches to Rethink Medication Adherence Measurement

Session Type: Issue Panel

Topics: Patient-Centered Research, Methodological & Statistical Research, Health Policy & Regulatory

Track: AI

Level: Intermediate

ISSUE: Value assessments of health technologies are increasingly incorporating medication adherence data. Yet these analyses often rely on static metrics, such as the proportion of days covered (PDC) or the medication possession ratio (MPR), typically applying an arbitrary 80% cutoff to classify patients as “adherent” or “nonadherent”. While simple and widely used, these metrics mask substantial heterogeneity in real-world medication taking behavior, including intermittent use, early discontinuation, or overuse. Relying on static measures risks can misestimate the true clinical and economic impact of nonadherence and undervalue adherence enhancing interventions. Newer approaches, such as group-based trajectory modeling (GBTM) and latent class mixed models, combined with artificial intelligence (AI) and machine-learning (ML) models offer a way to move from static thresholds to dynamic, pattern-based classifications of nonadherence. The purpose of this workshop is to examine how advanced trajectory- and AI/ML-based adherence measures can be appropriately defined, validated, and incorporated into value assessment, and to identify the conditions under which they add real decision value beyond traditional metrics and can be practically embedded into economic evaluations and broader value frameworks. OVERVIEW: This session explores why measuring adherence is a critical structural input in in the value assessment of health technologies. Dweeti Nayak will introduce the importance of adherence measurement in these contexts (5 minutes). Bijan Borah will compare the traditional static measures with emerging trajectory-based approaches and illustrate how they capture dynamic adherence patterns (15 minutes). Chao Cai will introduce latent mixed models and compare their performance with GBTM and unsupervised learning methods for classifying adherence behaviors (15 minutes). Tamas Agh will discuss the integration of AI/ML methods into adherence trajectory modeling and present a real-world case demonstration (15 minutes). The final 10 minutes will be dedicated to audience discussion and Q&A, facilitated through ISPOR’s polling tools to encourage active engagement.

Moderator

Dweeti Nayak, MS
Precision Medicine Group, Jersey City, NJ, United States

Speakers

Bijan J Borah, MSc, PhD
Mayo Clinic College of Medicine, Edina, MN, United States
Chao Cai, PhD
University of South Carolina, Columbia, SC, United States
Chao Cai, Ph.D. is an assistant professor in the Department of Clinical Pharmacy and Outcomes Sciences at the University of South Carolina College of Pharmacy in Columbia, South Carolina.
Tamas Agh, MSc, PhD, MD
Center for HTA and Pharmacoeconomic Research, University of Pecs & Syreon Research Institute, Budapest, Hungary

Global Readiness for the Next Wave of Biotech Innovation: How Development Economics and Regulatory Context Predict Whether North America, Europe, or Asia Will Lead

Session Type: Issue Panel

Topics: Health Policy & Regulatory, Health Technology Assessment, Medical Technologies

Level: Introductory

Purpose: Concerns about where biotech innovation will originate, scale, and mature are intensifying as global R&D activity expands. North America continues to dominate investment dollars and capital markets, but Asia has rapidly increased clinical trial activity and developer participation at a fraction of the cost, suggesting gains in development efficiency. Europe occupies an intermediate position shaped by regulatory harmonization, public financing, and evolving innovation incentives. These divergent trajectories raise important questions for HEOR and policy. From an HEOR perspective, it remains unclear whether lower-cost development environments translate into comparable regulatory success and clinical value. Specifically, to what extent do development economics and regulatory context predict a region’s ability to efficiently generate evidence, sustain innovative pipelines, and deliver long-term value to health systems? Addressing these questions is critical as governments, investors, and manufacturers seek to allocate resources efficiently while balancing innovation, quality, and access. Overview: This multi-perspective panel will debate global readiness for the next wave of biotech innovation, grounded in HEOR principles. Padula will moderate the session and open with an overview of global biotech development trends, highlighting differences in investment intensity, evidence generation, and regulatory pathways across North America, Europe, and Asia (10 minutes). Panelists will each present for 10 minutes, offering data-driven, industry perspectives on regional strengths and limitations, and discuss whether lower-cost development environments translate into durable innovation and value creation, how cross-regional partnerships shape efficiency and risk, and whether these ecosystems retain advantages in regulatory success, market access, and reimbursement. The session will conclude with moderated exchange and audience discussion (20 minutes). This panel will benefit HEOR researchers, industry leaders, investors, and policymakers seeking to understand how development economics and regulatory environments shape the evolving geography of biotech innovation.

Moderator

William V Padula, PhD
University of Southern California, Rancho Palos Verdes, CA, United States

Speakers

Natalie Reid, MBA, MPH, PhD
Stage Analytics, Severna Park, MD, United States
Andrew Lee, PhD
BridgeBio, San Francisco, CA, United States
Michael Duffy, PhD
OrgaNova, Philadelphia, PA, United States

Whose Preferences Should Govern Health Technology Adoption? Debating the Foundations and the Future of Value Assessment

Session Type: Issue Panel

Topics: Health Technology Assessment, Economic Evaluation, Methodological & Statistical Research

Track: Expanded Value Measures

Level: Intermediate

Issue: At the center of health technology assessment (HTA) lies the question of whose preferences should drive decisions about health technology adoption. Should value assessment reflect the preferences of patients, premium- and tax-paying consumers, regulators, third-party payers, healthcare providers or some combination of these stakeholders? This question arises in a number of timely and difficult resource-allocation decisions, like how to manage soaring patient demand for anti-obesity medicines. It also underlies debates over whether to use so-called “novel” elements of value from the ISPOR “value flower,” the salience of patient-centered economic theories like Generalized Risk-Adjusted Cost-Effectiveness (GRACE), proper approaches for measuring health-related quality of life and equity, and the role of risk preferences in value assessment. Focusing on the preferences of patients and consumers supports the adoption of novel value elements and the incorporation of risk preferences into value assessment. In contrast, aligning HTA with the preferences of payers or regulators, or a notion of aggregate societal preferences, supports QALY-based decision making. Overview: Goodman will introduce the debate over whose preferences matter most, and its implications for a variety of current debates over the ISPOR value flower, QALYs, GRACE, and other methodological questions (5 minutes). Lakdawalla will present the case for incorporating individual-level risk preferences into value assessment and argue why such approaches may better align with welfare economics and decision-making (10 minutes). Briggs will argue that healthcare decision making should be anchored in aggregate societal preferences, while critically examining where, and under what conditions, departures from this approach may be justified (10 minutes). Whittington will provide an inclusive perspective, outlining strengths and weaknesses of both approaches, and practical solutions for the consideration within HTA (10 minutes). The panel will then “negotiate” a preference realignment for technology adoption, followed by questions and critique from the audience (25 minutes).

Moderator

Clifford Goodman, MS, PhD
Clifford Goodman LLC, Bethesda, MD, Canada

Speakers

Darius Lakdawalla, PhD
University of Southern California, Los Angeles, CA, United States
Darius Lakdawalla is a widely published, award-winning researcher and a leading authority on health economics and health policy. He holds the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation at the University of Southern California, where he sits on the faculties of the School of Pharmacy, the Sol Price School of Public Policy, and the Leonard D. Schaeffer Center for Health Policy and Economics, one of the nation’s premier health policy research centers.

His academic research has focused primarily on the economics of risks to health, the value and determinants of medical innovation, the economics of health insurance markets, and the industrial organization of healthcare markets. Dr. Lakdawalla serves as associate editor at the Journal of Health Economics and has previously served in this role at the American Journal of Health Economics and the Review of Economics and Statistics. His academic work has appeared in leading peer-reviewed journals of economics, health policy, and medicine, including the American Economic Review, Quarterly Journal of Economics, Health Affairs, the Journal of Health Economics, and the New England Journal of Medicine. In addition, his work has been featured by prominent popular press outlets, such as the Wall Street Journal, National Public Radio, Forbes, and the New York Times. Dr. Lakdawalla has also received the PhRMA Foundation Value Assessment Challenge Award, designed to encourage innovative approaches to defining and measuring value in health care, in 2019 (third place) and 2020 (first place), along with the ISPOR Excellence in Research Methodology Award, the Garfield Prize, and the Milken Institute Award for Distinguished Economic Research.
Andrew Briggs, DPhil
London School of Hygiene & Tropical Medicine, London, United Kingdom
Melanie D Whittington, MS, PhD
Leerink Center for Pharmacoeconomics, Boston, MA, United States
Melanie Whittington is the Managing Director and Head of the Leerink Center for Pharmacoeconomics where she leads pharmacoeconomic evaluations of in-development and recently approved pharmaceuticals and studies incentives for innovation. She is also a Senior Fellow at the Center for the Evaluation of Value and Risk in Health (CEVR) where she advises on CEVR projects related to value assessment, economic modeling, and CEVR databases.

Resolved That HEOR and RWE Scientists Should Actively Architect RWD vs. Be An End User of RWD: A Debate

Session Type: Issue Panel

Topics: Real World Data & Information Systems

Track: Real-World Evidence (RWE)

Level: Intermediate

PURPOSE: The growing interest in incorporating real-world data (RWD) into regulatory and payer decision-making combined with expanding technical capabilities for generating RWD creates a dynamic data ecosystem. Against this backdrop, users of RWD must decide: are we practical consumers of the data or active architects? Based on the intended real world evidence (RWE) generation purpose, practical consumers of RWD curate, manipulate, and link available RWD sources while active architects shape RWD infrastructure and specifications. Although Health Economics and Outcomes Research (HEOR) and RWE professionals extensively use RWD, there is little discussion of which role to play, limiting our ability to navigate the changing ecosystem with intention. Using a formal debate structure, speakers will explore implications for research, policy, industry, and patient outcomes, and identify actionable steps as we consider the strategic future of HEOR and RWE generation: Will we develop evidence using RWD as they are available to us or embrace the opportunity to actively architect RWD? DESCRIPTION: During this session, the speakers will present positions for and against the resolution that “HEOR and RWE scientists should actively architect RWD vs be an end user of RWD”. The active architect team (Aaron Kamauu and Mary Beth Ritchey) and practical consumer team (Sandipan Bhattacharjee and Ravinder Dhawan) will each deliver opening and rebuttal statements on two topics (40 mins). The session will include audience polls and an audience Q&A session (10 min). A final poll will determine the debate winner. Eberechukwu Onukwugha will introduce the session in context of the ISPOR-ISPE Data Specifications initiative, provide ground rules, facilitate the polls and Q&A, and summarize the discussion and next steps (10 min total). Attendees will gain a better understanding of the tradeoffs associated with each role and the implications for navigating the RWD ecosystem.

Moderator

Eberechukwu Onukwugha, MSc, PhD
University of Maryland, Baltimore 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

Aaron Kamauu, MPH, MS, MD
Navidence, Inc., Bountiful, UT, United States
Mary Beth Ritchey, MSPH, PhD
Med Tech Epi, LLC, Philadelphia, PA, United States
Sandipan Bhattacharjee, MS, PhD
Bayer U.S. LLC, Belle Mead, NJ, United States

Advancing Real-World Evidence in FDA Regulatory Decision-Making

Session Type: Spotlight

Topics: Real World Data & Information Systems, Health Policy & Regulatory, Epidemiology & Public Health

Track: Real-World Evidence (RWE)

Level: Intermediate

Purpose: The FDA Center for Drug Evaluation and Research regulates drug products and has a program for the evaluation of real-world data (RWD) to generate real-world evidence (RWE) for regulatory decision-making. This workshop offers a forum for sharing key activities and accomplishments of FDA’s RWE program with ISPOR attendees. The session will showcase topics of broad interest to ISPOR membership, including recent approvals that involved RWE, FDA funded research projects, new Agency-level RWE initiatives, and ongoing efforts to advance RWE policy to support regulatory decision-making. This aligns with ISPOR’s interest in using RWE to inform healthcare decisions. Description: Workshop participants will learn how FDA determines whether RWD are fit-for-use, study designs provide adequate scientific evidence, and study conduct meets regulatory requirements. They will be able to summarize key messages from FDA RWE guidance documents and explain how FDA-supported RWE research projects have addressed gaps in knowledge to support medical product development. Dr Bradley will chair the session and provide a brief overview of RWE-related activities of the FDA RWE program. Drs. Rahman and Weissfeld will cover notable RWE-based product approvals, draft and final RWE guidance documents, and selected RWE-related demonstration projects. Prof. Shirley Wang will provide perspective as an FDA funded collaborator on efforts in advancing the use of RWE in regulatory decision making.

Moderator

Marie Bradley
FDA, Silver Spring, MD, United States

Speakers

Motiur Rahman, MS, PhD
Food and Drug Administration (FDA), Silver Spring, MD, United States
Joel Weissfeld, MD, MPH
Food and Drug Administration (FDA), Silver Spring, MD, United States
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.

Policy Evidence: Data-Driven Decision-Making for Policymaking

Session Type: Issue Panel

Topics: Health Policy & Regulatory, Study Approaches, Organizational Practices

Track: Real-World Evidence (RWE)

Level: Introductory

ISSUE: Healthcare systems around the world are experiencing unprecedented pressures, balancing budget and economic, capacity and infrastructure, and workforce pressures. Every decision made has downstream effects and tradeoffs on patient outcomes, short-term budgets, and/or longer term fiscal impact. The strategic use of real world data (RWD) and real world evidence (RWE) is essential for informed evidence-based policymaking. Industry and government collaboration is necessary to generate timely, credible evidence policy using RWD to improve health outcomes, enhance system sustainability, and benefit society. OVERVIEW: This session will discuss the types of data policymakers are looking for, generating evidence targeting stakeholders who are contemplating policy change, and how policy evidence and complements traditional medical and HEOR studies, and the potential to advance the field of policy evidence.

Moderator

Lisa Feng
Alexion AstraZeneca Rare Disease, Washington, DC, United States

Speakers

Josh Trent, MA
Leavitt Partners, Washington, DC, United States
Liz Fowler, JD, PhD
Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, United States
Erin Trish, PhD
USC, Los Angeles, CA, United States

Real-World Patient Voice at Scale: Can Social Media Listening Inform US Payer and Access Decisions?

Session Type: Issue Panel

Topics: Patient-Centered Research, Study Approaches

Track: Real-World Evidence (RWE)

Level: Introductory

Health plans and value assessment bodies increasingly recognise the importance of patient experience, yet conventional real-world data (claims, EHRs, registries) rarely capture treatment burden, stigma, or cost-related non-adherence in sufficient depth. In parallel, patients and caregivers openly discuss these topics in online communities and social media. This panel will debate whether, when, and how social media listening (SML) can move beyond “insight” to become decision-relevant real-world evidence for US payer and access strategy. Panelists will explore methodological standards for SML (sampling, bias mitigation, validation), and how insights on discontinuation, switching, financial toxicity, and daily-life impact can complement traditional RWD and PRO data. A health plan or PBM perspective will consider what type of digital patient-voice evidence is credible enough to influence formulary or utilization-management discussions. An HEOR/RWE lead from industry will discuss practical use cases where SML shaped evidence generation plans, value messages, or payer engagements. A patient-community or digital-ethnography expert will highlight ethical considerations, representation of vulnerable groups, and how to avoid over-interpreting “loud voices.” The panel will use short opening statements followed by moderated debate around three questions: - What is the “minimum bar” for SML to be considered robust enough for payer dialogue? - How should SML be integrated with PROs and RWD in economic models and value dossiers? - In which therapeutic areas (e.g., oncology, rare diseases, chronic conditions) is SML most likely to change access strategy? Real-time polling and audience questions will be used to surface areas of consensus vs controversy.

Moderator

Dimple Dang, MA
MarksMan Healthcare Communications Private Limited, Hyderabad, India

Speakers

Reina Natero, PharmD
CVS Health, Portland, OR, United States
Shreekant Parasuraman, PharmD, PhD
Incyte, Wilmington, DE, United States
Mukul Singhal, PhD
Janssen Pharmaceuticals/Johnson and Johnson, Horsham, PA, United States

2:45 PM - 3:15 PM

Coffee and Connect

Session Type: General Meeting

Head to the exhibit hall to connect with fellow attendees and exhibitors over a steaming cup of coffee.

4:00 PM - 7:00 PM

Poster Session 2

Session Type: General Meeting

Poster Tours 4:00PM–4:45PM | Presenters will be with their posters from 6:00PM–7:00PM

4:45 PM - 5:45 PM

The Metrics Behind the Money: Case Studies and Insights From Bridging Health Economics and Investment

Session Type: Workshop

Topics: Economic Evaluation, Health Technology Assessment, Organizational Practices

Track: Access and Drug Pricing

Level: Introductory

Purpose: Despite sharing a similar goal to use resources efficiently to maximize returns for patients and society, health economics and health investment have historically operated as two different sectors. As the scrutiny over market-based mechanisms for drug pricing intensifies and as we enter one of the largest patent cliffs on record, the need for coordination of health economic expertise and health investment expertise is obvious. This workshop will share real-world case studies and lessons learned from integrating health economic approaches within health investment strategy. Participants will learn how health economic approaches can be integrated into healthcare investment strategies and how health economic studies can better reflect real-world investment decisions. Description: The workshop will be moderated by Dr. Whittington who will summarize the different objectives of health economics and health investment (5 mins). Dr. Li, health economist at an investment bank, will provide examples of how she has incorporated a health economic lens into equity research (5 mins). Dr. Xie, a health economist at a multi-stage investment management company, will describe using generalized cost-effectiveness analysis to communicate the societal value for a portfolio company prior to their acquisition (5 mins). Dr. VanSickle, a health economist at a healthcare investment firm, will explain how health economic principles are considered as part of due diligence and investment strategy (5 mins). Panelists will then participate in a fireside chat (20 mins) to discuss 1) the impacts of their efforts, 2) adaptations to health economic methods to align with investor strategy, 3) how and when the need for a health economic lens arises, 4) and opportunities for the future. The fireside chat will conclude with audience polling to assess the feasibility of the adaptations to health economic methods to align with investor strategy. Twenty minutes will be reserved for audience Q&A.

Moderator

Melanie D Whittington, MS, PhD
Leerink Center for Pharmacoeconomics, Boston, MA, United States
Melanie Whittington is the Managing Director and Head of the Leerink Center for Pharmacoeconomics where she leads pharmacoeconomic evaluations of in-development and recently approved pharmaceuticals and studies incentives for innovation. She is also a Senior Fellow at the Center for the Evaluation of Value and Risk in Health (CEVR) where she advises on CEVR projects related to value assessment, economic modeling, and CEVR databases.

Speakers

Meng Li, MS, PhD
Tufts Medical Center, The Center for the Evaluation of Value and Risk in Health, Boston, MA, United States
Richard Xie, PhD
RA Capital Management, Newton, MA, United States
Sorochi Van Sickle, PhD
Patient Square Capital, Menlo Park, CA, United States

Getting Closer to the Target: Practical Strategies to Hit the Mark in Trial Emulation

Session Type: Workshop

Topics: Methodological & Statistical Research, Study Approaches, Real World Data & Information Systems

Track: Real-World Evidence (RWE)

Level: Intermediate

Purpose As target trial emulation (TTE) frameworks gain prominence, there is increasing demand to engage with methodologically complex domains. Researchers must carefully define eligibility criteria, construct appropriate index dates, align follow-up time, control for confounding, maintain outcome masking, and ensure rigorous data quality—all while meeting rapidly evolving regulatory and HTA expectations. Even minor misalignment of design elements can introduce substantial bias and undermine the credibility of the evidence generated. Despite growing interest, practical guidance on how to operationalize TTE principles and choose between competing analytic approaches remains limited. This workshop aims to translate the conceptual framework into actionable strategies for rigorous design and evaluation of external control arm (ECA) and hybrid trials. Description This workshop will be chaired by Dr. Opper, who will introduce core TTE concepts (5 min). • Dr. Van Le (academia) will synthesize real-world lessons from ECAs submitted to regulatory/HTA bodies, covering data landscaping best practices to justify data source selection, and provide a framework for applying trial eligibility criteria to a real-world population (15 min). • Dr. Aggarwal (CRO) will compare established and emerging strategies for index date assignment, follow-up alignment, and approaches to minimize immortal time bias (10 min). • Dr. Debray (academia) will present key design concepts of hybrid designs, comparing to single arm trials and highlighting variation in statistical methods including Bayesian borrowing, using examples from FDA/EMA approvals (10 min). A 20-minute interactive, hands-on case exercise will demonstrate how design changes such as comparator eligibility, varying time zero, and comparator weighting can substantially affect results. Through facilitated discussion and live polling, participants will evaluate tradeoffs and apply bias-mitigation principles. This workshop will equip researchers and decision-makers with practical tools to operationalize TTE principles and choose credible, defensible analytic strategies to hit the target for trial emulation.

Moderator

Neisha Opper, MPH, PhD
Landmark Science, La Crescenta, CA, United States

Speakers

Hoa Le, MD, PhD
The University of North Carolina at Chapel Hill, Chapel Hill, NC, United States
Shivani Aggarwal
Landmark Science, Inc, Los Angeles, CA, United States
Thomas Debray, BSc, MSc, PhD
University Medical Center Göttingen, Utrecht, Netherlands

Agentic AI in Evidence Submissions: Rigor, Trust, Traceability & Compliance

Session Type: Workshop

Topics: Health Policy & Regulatory, Methodological & Statistical Research, Health Technology Assessment

Track: AI

Level: Intermediate

Purpose Generative and agentic AI can substantially accelerate evidence generation and synthesis. Yet the development and adoption of Agentic AI-driven workflows for evidence submissions raises critical questions: what are the methodological and implementation considerations when automating documentation and evidence packages using agentic AI frameworks? How should industry redesign workflows to preserve trust, traceability, and compliance when AI becomes an active contributor to evidence development? How can organizations ensure transparency and methodological rigor? And what documentation pathways will payers and regulators expect as AI-generated evidence becomes more prevalent? This workshop brings together leaders from biopharma, regulatory science, and AI research to offer a practical, multidisciplinary view of this rapidly evolving landscape. Description The session will open with an overview of the evolving role of agentic AI in HEOR. Dr. Wang (Tulane/NouStarX) will present an implementation framework for agentic AI in evidence submissions, focusing on knowledge-base generation, documentation automation, and quality controls. Ms. Ozer-Stillman (Takeda) will share real-world experience applying AI-enabled processes to GVD, adaptations for local HTA submissions and value communications tools, highlighting practical considerations for auditability, traceability, and cross-functional alignment. Dr. Huo (Johnson & Johnson) will discuss opportunities and constraints for AI-supported FDA engagements, including experiences related to programs such as Breakthrough Therapy Designation and Post-Marketing Requirements/Commitments, and will address the evidential standards and validation requirements necessary for AI-assisted evidence to be credible in regulatory interactions. Finally, Dr. Innes (FDA) will offer a forward-looking regulatory perspective on the transparency, robustness, and provenance expectations that may guide future acceptance of AI-generated evidence as organizations adopt agentic AI–driven workflows. The session will be followed by a moderated panel discussion focused on practical adoption guidance, governance considerations, and an open Q&A.

Moderator

Xiaoyan Wang, PhD
Tulane University, New Orleans, LA, United States

Speakers

Ipek Ozer Stillman, MBA, MSc
Takeda, Cambridge, MA, United States
Jinghai Stephen Huo, MD PhD
Janssen Pharmaceuticals (Johnson and Johnson), Raritan, NJ, United States
Gabriel Innes, VMD PhD
Food and Drug Administration (FDA), Silver Spring, MD, United States

Beyond Claims and EHRs: Social Media as Real-World Evidence to Uncover Patient Experiences and Unmet Needs

Session Type: Workshop

Topics: Real World Data & Information Systems, Health Technology Assessment, Health Service Delivery & Process of Care

Track: Real-World Evidence (RWE)

Level: Introductory

PURPOSE: Patients increasingly share experiences, treatment feedback, and concerns on social media and online health communities, creating a rich, real-time data source now recognized by the FDA in its Patient-Focused Drug Development: Methods to Identify What Is Important to Patients (2022). Advances in natural language processing (NLP), particularly large language models (LLMs) such as ChatGPT, enable scalable analysis of this unstructured content, uncovering insights into patient experiences and unmet needs often missed by traditional methods. This 60-minute workshop aims to build awareness and practical competence in social media listening (SML) and equip participants with strategies to leverage digital patient narratives. By the end of the session, attendees will understand the strengths and limitations of SML, and what state-of-the-art NLP and LLM technologies offer. Participants will also learn how and when to apply these approaches to transform patient voices into actionable insights that inform drug discovery, development, and value assessment. DESCRIPTION: Grounded in the FDA’s 2022 guidance, which explicitly recognizes social media as a valid source of qualitative and quantitative patient experience data, this session will provide an overview of SML methods and applications, addressing key considerations such as study design, data quality, and privacy through real-world examples. Dr. Jen will introduce the topic in the context of patient-centered drug development and traditional evidence sources (5 min). Dr. Song will discuss how SML addresses evidence gaps in rare diseases from a HEOR perspective (10 min). Dr. Sarker will present state-of-the-art NLP/LLM techniques for SML (15 min). Dr. Yao will share two real-world applications involving vaccines and chronic autoimmune disease (15 min). The session concludes with an interactive panel, enabling audience engagement through QR code-based polling and discussion (15 min). By combining regulatory context, technological innovation, and practical case studies, this workshop offers a roadmap for integrating SML into patient-focused drug development programs.

Moderator

Min-Hua Jen, PhD
Eli Lilly, Uxbridge, United Kingdom
Dr Min-Hua Jen is currently Executive Director - Advanced Analytics and Access Capabilities at Eli Lilly, leading the International Business team on Market Access/HEOR/Medical affairs statistical support. She has extensive experience applying statistics to clinical research, epidemiology and health economics and outcomes research in academia and industry settings. She is an active member in the PSI/EFSPI HTA Special Interest Group (SIG) and the chair-elect of the ISPOR Oncology SIG. She was trained in Epidemiology and Statistics and obtained her PhD at University of Bristol. Her research interests including indirect treatment comparisons and network meta-analysis; particularly incorporate external data for time to event outcomes, surrogacy analyses, survival extrapolation, multilevel modelling and health economic modelling.

Speakers

Chao Song
UCB, Duluth, GA, United States
Abeed Sarker, PhD
Emory University, Atlanta, GA, United States
Lixia Yao, PhD
Temple University, Chalfont, PA, United States

The NextGen of Clinical Trials: Patient-Driven or Tech-Driven?

Session Type: Workshop

Topics: Patient-Centered Research, Medical Technologies, Clinical Outcomes

Track: AI

Level: Intermediate

Purpose: With new advances in technology, how does the future of patient-centricity look? Emerging technologies like artificial intelligence, virtual reality, and digital platforms are integrating with traditional methods, such as qualitative interviews, surveys, and clinical outcome assessments (COAs), to personalize and improve patient care. These innovations create new opportunities to evolve care models (e.g., trials catered to individual needs, decentralized trials) and to inform decisions by regulators and payers. This session will examine how these tools and technologies can be used to systematically collect and/or generate high quality, data-driven, insightful patient experience data (PED) for decision-making and integration into the design and delivery of care. Description: Danny Yeh (AESARA) will open the session with a brief presentation that introduces core concepts, emerging technologies, and current use cases. He will then moderate the discussion on the integration of these technologies in clinical trials, drawing on insights from payers and HTA decision-makers. In response to this, the panel will provide perspectives from patient advocacy (Tracey Sikora; NORD), industry (Ebony Dashiell Aje; BioMarin), and regulatory (Selena Daniels; FDA). The panel will explore: • Why are we entering a new era in how we approach patient partnership and empowerment in clinical trials? • How can technology be integrated with traditional methods to ensure clinical trials are shaped by what matters most to each individual patient? • How does the rise of artificial intelligence and machine learning may influence the collection and use of PED? • How is the integration of PED collected in routine care (real world evidence) with traditional clinical trial data in decision-making? What standards need to be in place? • What to expect in the coming years and what issues warrant close attention The session will include interactive polling questions and time for audience questions and open dialogue.

Moderator

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

Speakers

Tracey Sikora, BSc
National Organization for Rare Disorders, Washington, DC, United States
Ebony Dashiell-Aje, PhD
BioMarin Pharmaceutical Inc., Washington, DC, United States
Ebony Dashiell-Aje, PhD is a leading expert in patient-centered outcomes research, including clinical outcome assessment (COA) and digital health technology development and optimization, measurement science strategy, and patient engagement across the medical product lifecycle. Currently, she serves as the Executive Director & Head of Patient-Centered Outcomes Science at BioMarin. Dr. Dashiell-Aje and has also served within the Division of Clinical Outcome Assessment (DCOA) in the Office of New Drugs at FDA as a scientific lead and consultant to CDER, CBER, and CDRH; she was a key player in shaping patient-focused drug development (PFDD) policy efforts. In addition, she has served as expert consultant to both pharmaceutical industry and federal government clients. As a health outcomes researcher and methodologist, Dr. Dashiell-Aje is driven by her passion for public health and promoting patient-centered medical product development, commercialization, and clinical care. Her research expertise has significantly contributed to evidence-based solutions and has helped shape health policy in multi-stakeholder environments.
Selena Daniels, BS, MS, PharmD
Food and Drug Administration, Silver Spring, MD, United States

Forks in the Road: Critical Decision Points and FDA Insights from Six Years of Physical Function COA Development

Session Type: Workshop

Topics: Patient-Centered Research, Clinical Outcomes, Health Policy & Regulatory

Level: Intermediate

Purpose To equip attendees with tools to engage FDA in developing and validating clinical outcome assessments (COAs). Drawing on a six-year FDA collaboration, we share insights and strategies for sponsors navigating the regulatory landscape of patient-reported and performance outcome measures. Description Developing COAs for regulatory purposes requires strategic decision-making throughout the research lifecycle. Disease-specific COA development demands substantial investment and multi-year timelines, creating barriers for rare diseases and small populations. While FDA guidance provides frameworks, sponsors face practical questions: What evidence demonstrates cross-disease utility? How should rigor be balanced with regulatory pragmatism? How can sponsors optimize limited FDA interactions? This interactive workshop draws on NUCOAT's (Northwestern University Clinical Outcome Assessment Team) position as an FDA-funded collaborative grant (PI: David Cella, PhD), enabling six years of near-monthly FDA consultation while developing physical function measures across rare diseases and sarcopenia of aging. This offered opportunities to test assumptions, refine methods, and understand which evidence most influenced FDA discussions. Attendees will navigate key "forks in the road"—decision points where methodological or strategic choices shaped NUCOAT's trajectory and FDA input proved pivotal, spanning: concept elicitation and content validity, measurement properties and validation, meaningful change determination, and qualification pathway planning. For each fork, speakers present real-world challenges. Attendees vote on preferred approaches via live polling, discuss with neighbors, then learn what pathway NUCOAT selected and how FDA engagement informed the decision. Each section concludes with practical "pearls" on anticipating regulatory concerns, prioritizing evidence, and preparing Drug Development Tool submissions. The workshop features study leadership and an FDA representative offering complementary perspectives to help attendees prepare for their own FDA engagements.

Moderator

Maja Kuharic, PhD
Northwestern University Feinberg School of Medicine, CHICAGO, IL, United States
Maja Kuharic, MPharm, MSc, PhD, is an Assistant Professor in the Department of Medical Social Sciences at Northwestern University Feinberg School of Medicine. Her research focuses on patient-reported outcomes (PROs), health-related quality of life, and economic evaluation. She specializes in the development, validation, and application of PROMIS measures and preference-based instruments such as the EQ-5D and EQ-HWB.

Speakers

David Cella, PhD
Northwestern University Feinberg School of Medicine, Chicago, IL, United States
Robyn Bent, MS, RN
Food and Drug Administration (FDA), Silver Spring, MD, United States
Courtney Hurt, MSW
Northwestern University, Chicago, IL, United States

The Alphabet Soup of Drug Pricing: Understanding Interactions of ASP, MFP, MFN, 340B, and MDRP

Session Type: Workshop

Topics: Health Policy & Regulatory, Health Service Delivery & Process of Care, Organizational Practices

Track: Access and Drug Pricing

Level: Intermediate

Purpose: This year marks the launch of the negotiated prices in the Medicare Drug Price Negotiation Program and introduces another pricing benchmark, the Maximum Fair Price (MFP). There are several other programs in the drug pricing landscape with significant policy changes – such as the 340B rebate model, the most favored nation (MFN) pricing models, and the Medicaid best price guarantee – setting the stage for a complex interplay between these programs and the MFP. These policy changes have the potential to bring more confusion to an already complex and opaque pricing system for manufacturers, health plans, healthcare providers, policymakers, patients, and researchers. Workshop participants will learn the fundamentals of the programmatic updates in the federal drug pricing landscape, identify critical points of interaction between programs, and learn how best to monitor these policies as they unfold. Description: Ramachandran will provide basics of the drug pricing programs with a brief history of the issue in Medicare, Medicaid, and commercial insurance (10 mins). Martin will summarize the most recent legislative and regulatory updates to these programs – effectuation of MFP, MFN models, 340B rebate model, and the Medicaid best price guarantee (15 mins). Coster will identify points of interactions between programs and identify challenges facing patients, providers, and manufacturers in navigating the new landscape and preview the next year of drug pricing policy including identifying areas for potential reform. (15 mins). Participants will engage with panelists in small group discussions for a series of three case-based simulations in oncology, injectable medication, and a long-acting oral medication. In each case, participants will be make pricing and access decisions using real-time polling as if they were a manufacturer, payer, or provider navigating current policy. (15 mins) Panelists will engage with audience to address questions on the impacts of these policies for patients, providers, and payers. (5 mins)

Moderator

Sujith Ramachandran, PhD
University of Mississippi, University, MS, United States

Speakers

Sujith Ramachandran, PhD
University of Mississippi, University, MS, United States
Kristi Martin
Camber Collective, Washington, DC, United States
Kristi Martin provides policy expertise and strategic counsel to advance meaningful health policy that focuses on improving the health and wellbeing of people while delivering practical policy solutions. She draws on more than 20 years of experience working in the public sector, with private sector clients, and in philanthropy. Most recently, Kristi served as the chief of staff and senior advisor to the deputy administrator in the Center for Medicare at the Centers for Medicare & Medicaid Services (CMS). While serving at CMS, she played a significant role advancing regulatory policy in Medicare and implementing the Inflation Reduction Act -- the most significant changes to Medicare Part D since its inception. Kristi facilitated and coordinated the implementation of the Medicare Drug Price Negotiation Program, which successfully negotiated the first set of drug prices under the Medicare Drug Price Negotiation Program that will save people with Medicare and the program billions of dollars on prescription drug costs as well as contributed to a wide range of other policy and operational initiatives that have made Medicare better than ever. Kristi previously was the Vice President for Health Care at Arnold Ventures where she led the philanthropy's prescription drug pricing portfolio and was the Managing Director of Waxman Strategies' health practice where she worked alongside Congressman Henry Waxman to drive outcomes in health policy. She has previously served several years in the U.S. Department of Health and Human Services, Office of Personnel Management, and Government Accountability Office. As a senior advisor in the Obama administration's Office of Health Reform, she had primary oversight responsibility for the coordinated and timely implementation of cross-cutting departmental public health and prevention initiatives under the Affordable Care Act, including addressing the rising cost of drugs and setting up the women's preventive services initiative. She received her bachelor's and master's degrees in health communication from the University of Kentucky and a Master of Public Administration from George Washington University.
John Coster, PhD
Center for Medicaid and CHIP Services, Washington, DC, United States

Live, Interactive Workshop of Generative AI for Real-World Market Access Challenges

Session Type: Workshop

Topics: Health Technology Assessment, Health Policy & Regulatory

Track: Access and Drug Pricing

Level: Introductory

PURPOSE: This workshop aims to provide a highly practical, scenario-driven exploration of generative AI (GenAI’s) capabilities and limitations for addressing real-world market access challenges—including evidence generation, payer communication, HTA submission development, and value story creation. Participants will learn how GenAI tools can be used responsibly, where human expertise remains indispensable, and how to evaluate readiness for adoption within their own organizations. DESCRIPTION: The workshop uses a problem-first, interactive format. Dr. Chhatwal will introduce the session and outline the increasing operational pressures facing market access teams. Market access expert Narin Yasar will present a series of realistic, high-impact scenarios drawn from global product launches, payer negotiations, and HTA submissions—such as generating rapid evidence summaries, developing alternative value messages, preparing country-specific HTA dossiers, addressing last-minute payer queries, and aligning cross-functional teams around a value narrative. GenAI expert Dr. Turgay Ayer will evaluate the feasibility and risks of applying GenAI to each scenario, emphasizing methodological guardrails, data-quality considerations, and governance best practices. He will also provide live demonstrations of GenAI applications that perform tasks such as summarizing clinical evidence, drafting payer-facing language, generating full landscape assessment reports in hours, and adapting global value materials for local contexts. The final segment consists of audience-driven scenario testing: participants will propose real challenges they face, and the panel will collaboratively assess how GenAI could—or could not—address them. This workshop provides actionable insights for HEOR and market access professionals seeking responsible, real-world integration of GenAI into their daily workflows.

Moderator

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.

Speakers

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

6:00 PM - 7:00 PM

Welcome Reception

Session Type: General Meeting

Join us for the Welcome Reception, supported by Corporate Partners. A perfect kick-off to connect, unwind, and gear up for what's ahead.

Tue May 19

7:00 AM - 8:30 AM

Morning Coffee Service

Session Type: General Meeting

Don't miss the start of the day with the Plenary Session. Enjoy your morning coffee as you listen to dynamic presentations intended to inspire and empower.

7:00 AM - 5:00 PM

Registration Hours

Session Type: General Meeting

8:30 AM - 10:00 AM

The True Cost of Cancer: Aligning Innovation, Access, and Affordability

Session Type: Plenary

Topics: Economic Evaluation, Health Service Delivery & Process of Care, Patient-Centered Research

Track: Expanded Value Measures

Level: Advanced

Cancer imposes a profound economic burden on patients, families, health systems, and insurers—often extending well beyond clinical outcomes. This plenary session will examine strategies to reduce the financial impact of cancer while preserving high-quality, equitable care. Speakers will explore the concept of financial toxicity as a critical and measurable outcome of cancer treatment, highlighting its effects on treatment adherence, quality of life, and long-term survivorship.

This session will spotlight advances in cancer care delivery research that identify cost drivers across the care continuum and evaluate interventions that improve efficiency, coordination, and patient-centered value. Panelists will also examine private sector solutions, including innovative benefit design, value-based payment models, data-driven care management, and partnerships between payers, providers, and life sciences companies.

A final focus will be the role of comprehensive cancer centers as engines of innovation—leveraging research, multidisciplinary care models, community outreach, and policy leadership to reduce costs and disparities at scale. Together, these perspectives will frame a forward-looking discussion on aligning incentives, accelerating evidence-based solutions, and building sustainable cancer care systems that minimize economic harm while maximizing outcomes for all stakeholders.

Moderator

Scott Ramsey, PhD, MD
Fred Hutchinson Cancer Research Center, Lake Forest Park, WA, United States
Dr. Ramsey is a general internist and health economist. He is a professor and director of the Hutchinson Institute for Cancer Outcomes Research, a multidisciplinary team devoted to cancer outcomes research. Dr. Ramsey is also a professor in the Schools of Medicine and Pharmacy at the University of Washington.

Trained in Medicine and economics, Dr. Ramsey’s research focuses on outcomes research and cancer care delivery. His studies on financial toxicity issues faced by cancer patients are widely cited. He leads the Value in Cancer Care initiative, a statewide quality and cost reporting program aimed at improving oncology care. His other research interest includes cancer care delivery research, pragmatic trial design, cost-effectiveness analysis, and stakeholder engagement.

Dr. Ramsey is co-chair of the National Cancer Institute’s Cancer Care Delivery Research Steering Committee and a co-chair of SWOG’s Cancer Care Delivery Committee. He is past president of the Professional Society for Health Economics and Outcomes Research (ISPOR) and has served on the National Academy of Science’s Cancer Policy Forum. He is co-principal Investigator for the Coordination and Communications Center of the National Cancer Institute’s Cancer Screening Research Network.

Speakers

Dawn Hershman, MD, MS, FASCO
Columbia University Medical Center, New York, NY, United States
Stacie Dusetzina, PhD
Vanderbilt University Medical Center, Nashville, TN, United States
Bobby Green, MD
ThymeCare, Nashville, TN, United States

9:30 AM - 7:00 PM

Exhibit Hall Hours

Session Type: General Meeting

9:45 AM - 10:15 AM

Coffee and Connect

Session Type: General Meeting

Head to the exhibit hall to connect with fellow attendees and exhibitors over a steaming cup of coffee.

10:30 AM - 11:30 AM

Bridging the Gap or Respecting the Divide? Discussing the Integration of Clinical Outcome Assessments and Patient Preference Data

Session Type: Issue Panel

Topics: Patient-Centered Research, Clinical Outcomes

Track: Expanded Value Measures

Level: Intermediate

There is growing recognition that integrating COAs with PPI can enrich our understanding of patient well-being. However, meaningful integration remains challenging due to differences in assumptions, measurement paradigms, and intended uses. Although these approaches stem from distinct traditions, combining them offers unique insights that neither can provide alone. Opportunities span clinical care, regulatory review, and HTA. For instance, clinicians can align treatment decisions with patient concerns by comparing expected outcomes and stated concerns. PPI also can help identify relevant domains for COA development and guide COA selection when multiple options exist. In regulatory reviews, COA-based benefits can be interpreted alongside patient tradeoffs, informing what constitutes a meaningful benefit. In HTA, incorporating preference heterogeneity into COA interpretations can inform anticipated treatment uptake, usage intensity, and real coverage costs. Despite these opportunities, substantial challenges persist. Key barriers include limited cross-disciplinary understanding, differing objectives for COA and PPI instruments, and lack of guidance on how COA and PPI data could be combined to maximize their impact. A candid discussion about when full integration adds value, and when coordinated but parallel use is more practical, is critical for advancing patient-centered evidence generation. Panelists will reflect on methodological considerations, potential value, and practical strategies for leveraging these complementary approaches to support more holistic and equitable decision making in clinical, regulatory, and coverage contexts. The panel will also consider when such integration provides insights that justify added complexity and investments, and which areas of complementarity, such as defining clinical care, contextualizing benefit-risk assessments, or supporting health technology assessments, should be prioritized. Each panelist will offer a 10-minute perspective, followed by moderated discussion and audience engagement.

Moderator

Shelby Reed, RPh, PhD
Duke Clinical Research Institute, Durham, NC, United States

Speakers

Karen V. MacDonald, BSc, MPH
IQVIA Canada, Calgary, AB
Juan M Gonzalez, PhD
Duke Clinical Research Institute, Cary, NC, United States
Mo Zhou, PhD
Novartis, East Hanover, NJ, United States

Rare Hope for Even Rarer Diseases: Innovation, Evidence, and Regulatory Perspectives

Session Type: Workshop

Topics: Methodological & Statistical Research, Health Policy & Regulatory, Clinical Outcomes

Track: AI

Level: Intermediate

Purpose Recent advances in natural language processing (NLP) and large language models (LLMs) offer promising pathways to accelerate research, enhance phenotyping, and strengthen evidence packages for regulatory and HTA submissions. Yet the rapid adoption of AI technologies raises critical methodological, operational, and regulatory questions for rare disease drug development. This workshop aims to (1) clarify how emerging AI capabilities can support small-population evidence generation; (2) showcase real-world applications across academia, industry, and regulatory science; and (3) identify transparency, quality, and compliance considerations essential for integrating AI-generated evidence into regulatory and HTA decision-making. Description Rare diseases face persistent evidence gaps due to small patient populations, heterogeneous clinical presentations, and fragmented data sources. Advances in NLP and generative/agentic AI offer new opportunities to uncover real-world insights, automate evidence synthesis, and support more agile development pathways. The session will begin with context on the current AI landscape and the evolving methodological needs specific to small-population research. Dr. Wang (Tulane/NouStarX) will present her research on how NLP and LLMs support underserved and small population research, highlighting how large-scale clinical text analysis and federated learning can strengthen cohort identification and study readiness. Dr. Weng (Columbia) will present the use of fine-tuned LLMs for early diagnosis of rare diseases and genetic testing recommendations using RAG models and human phenotype ontology. Mr. Su (Jazz Pharmaceuticals) will provide an industry perspective, including opportunities and constraints when integrating AI enabled workflows into value and market access strategies. Finally, Dr. Liu (FDA) will share a regulatory viewpoint on expectations for transparency, robustness, and methodological rigor when AI contributes to evidence packages in rare disease programs. The session concludes with a Q&A and digital polling designed to encourage audience interaction.

Moderator

Xiaoyan Wang, PhD
Tulane University, New Orleans, LA, United States

Speakers

Chunhua Weng, Ph. D
Columbia University, New York, NY, United States
Wayne Su, MSc
Jazz Pharmaceuticals, Philadelphia, PA, United States
Qi Liu, PhD, MStat, FCP
Food and Drug Administration (FDA), Silver Spring, MD, United States

Beyond the Bots: How AI-Enabled Literature Reviews Are Maturing, and What HEOR Needs Next

Session Type: Issue Panel

Topics: Study Approaches, Methodological & Statistical Research, Health Technology Assessment

Track: AI

Level: Intermediate

Issue AI-enabled literature review platforms have rapidly expanded across HEOR, regulatory, and medical affairs workflows. The first wave of pilots demonstrated substantial gains in screening speed and reviewer efficiency, but also revealed variability in accuracy, transparency, and reproducibility across review types and organizational settings. Now, as multiple teams have completed successive reviews with the same platforms, the field is entering a second phase, one where outputs can be compared across projects, procurement expectations are more structured, and human, AI workflows are stabilizing. Overview The panel will not compare product features. Instead, it will synthesize cross-platform learnings and debate what the HEOR community should expect—and require—from AI-enabled evidence synthesis in its next phase of adoption. Attendees will leave with: (1) a data-informed understanding of AI SLR maturity, (2) practical evaluation considerations for real-world implementation, and (3) a clearer view of how automation and human judgment must align to preserve scientific integrity. Panelists will examine how platform capabilities have evolved since 2025 and where divergence persists in real practice. Discussion topics will include: • How human–AI integration has changed across repeated projects • Which elements of transparency, version control, and auditability have proven essential • What patterns are emerging in validation, error tolerance, and sensitivity trade-offs • Where AI-enabled tools still face methodological gaps • What “good” should look like as the field moves toward standardization

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.

Speakers

Angeline B Dhas
MadeAi, Littleton, MA, United States
Angeline Babitha Dhas is the Product Manager for MadeAi Solutions, with over 12 years of experience at the intersection of healthcare, technology, and evidence generation. She specializes in identifying time- and resource-intensive tasks within HEOR processes and designing AI-driven solutions to improve efficiency and scalability. Angeline led the development and launch of MadeAi-LR, an AI-aided systematic literature review platform and is now expanding its capabilities to address challenges in Joint Clinical Assessments (JCA), Global Value Dossiers (GVD), drug repurposing, and social media analysis for patient-reported outcomes. Her work focuses on enabling smarter, faster evidence strategies through AI innovation.
Ranita M Tarchand, BA, MS
Nested Knowledge, St Paul, MN, United States
Ranita is trained as a health economist and epidemiologist. In her role as Vice President of Scientific Strategy and Client Success at Nested Knowledge, she works with private and public institutions on implementing AI methodology and digital transformation at all stages of the product lifecycle. Her research focuses on living HTA methods and artificial intelligence in evidence synthesis and RWE.
Mark Priatel
DistillerSR, Ottawa, ON, Canada

Data and Approaches to Measuring Affordability and Access to Healthcare Services

Session Type: Issue Panel

Topics: Health Service Delivery & Process of Care, Health Policy & Regulatory, Methodological & Statistical Research

Track: Access and Drug Pricing

Level: Intermediate

ISSUE: ISPOR’s Strategic Plan 2030 identified affordability and its impact on the long-term sustainability of healthcare systems as a central challenge. Growing patient cost sharing, evolving health plan options, and complex benefit design underscore a need to improve our understanding of affordability from a patient’s perspective. There is limited information about domains of cost-related management strategies (CrMS) and their association with satisfaction and health status among Medicare beneficiaries. Relatedly, beneficiaries may experience substantial changes in their out-of-pocket cost burden and access to providers following selection into Medicare Advantage compared to Traditional Medicare. These perspectives on affordability have different policy implications. OVERVIEW: This session will address affordability, access, and policy through evaluations and critical discussions of data sources, measurement, and methodological implications on estimates of the effect of affordability on access. Specifically, the session will focus on the Medicare population in the US using two survey data sources – The Medicare Current Beneficiary Survey (MCBS) and Medical Expenditure Panel Survey (MEPS). Using the MCBS, the associations of the domains of CrMS with satisfaction and health status among beneficiaries will be quantified using fixed effects logistic regression. The effect of changes in out-of-pocket burdens and cost barriers associated with plan enrollment choices will be assessed using a fuzzy regression discontinuity design with MEPS data. Following each presentation, another panelist will critically discuss the assumptions, data, methods, and what it implies for their conclusions. Presenters will introduce the MCBS and MEPS studies (15 minutes each). Discussants will critique the studies with time for presenters to respond (10 minutes each). The session will conclude with audience Q&A (10 min). Session attendees, e.g., researchers, will gain a better understanding of the tradeoffs among frameworks, datasets, and modeling strategies needed to study patient affordability and generate policy-relevant findings.

Moderator

Eric Barrette, MA, PhD
Medtronic, Minneapolis, MN, United States
Eric Barrette, M.A., Ph.D. is Vice President, HEOR & Market Access Analytics at Medtronic. In this role he supports the development of evidence needed for coverage, payment, and access to Medtronic's therapies. Prior to joining Medtronic he was Director of Research at the Health Care Cost Institute and he has worked as an economic consultant focused on health care and life sciences topics. He is also an instructor in the University of Minnesota, Carlson School of Management Medical Industry MBA program. Dr. Barrette research interests include medical technology outcomes research, Medicare payment policy, geographic variation in medical resource use and prices. He received his M.A. in Economics from the University of Arizona and a Ph.D. in Health Services Research, Policy, and Administration from the University of Minnesota.

Speakers

Eberechukwu Onukwugha, MSc, PhD
University of Maryland, Baltimore 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.
Wendy Xu, PhD
The Ohio State University, Columbus, OH, United States
Junling Wang, PhD
University of Tennessee College of Pharmacy, Memphis, TN, United States

Can the Estimand Framework Strengthen Patient-Centered Cancer Research Throughout the Evidence Generation Lifecycle Beyond Approval: Opportunities and Challenges Ahead?

Session Type: Issue Panel

Topics: Patient-Centered Research, Clinical Outcomes, Study Approaches

Track: Expanded Value Measures

Level: Intermediate

Issue: Despite adoption of the ICH E9(R1) addendum and the estimand framework by the FDA and other regulatory bodies, its role in shaping patient-centered objectives in cancer research remains unclear throughout the continuous evidence lifecycle from clinical development through post-approval research. Overview: This session will reinforce how the estimand framework can facilitate generation of meaningful patient-centered evidence and will identify current opportunities and challenges for cancer research. Dr. Park will provide an overview of the estimand framework and discuss the role of the estimand framework for both clinical trial and real-world evidence (RWE) studies (8-minutes). Dr. Sharaz will discuss how regulators and health technology assessment (HTA) bodies view patient-reported outcomes (PROs) with a focus on the role of data collection post-treatment and post-progression to supplement traditional survival and progression endpoints (10-minutes). Dr. Skaltsa will discuss the methodological challenges as well as the current advancements with regard to analyzing PROs collected in oncology trials in line with the estimand framework, during the treatment period and beyond (10-minutes). Dr. Roydhouse will discuss considerations for comparing patient-reported tolerability of different therapies, and how comparative tolerability endpoints can supplement other PRO and non-PRO endpoints in oncology (10-minutes). The session will reflect on future directions of methodological research based on the estimand framework for patient-centered research including the communication of estimands to patients, regulators, HTA bodies, and other diverse stakeholders (5-minutes). The session will conclude with an interactive discussion with audience participation to explore current challenges and opportunities ahead for patient-centered research and the estimand framework (17-minutes).

Moderator

Jay J Park, PhD
Core Clinical Sciences, Vancouver, BC, Canada

Speakers

SAEID SHAHRAZ
Gilead Sciences, Mountain View, CA, United States
Saeid Shahraz, MD, PhD, is a physician and measurement scientist with over 20 years of experience in health outcomes research. He leads clinical outcome assessment strategy and analytics as Director of Health Economics and Outcomes Research. With expertise in psychometrics, COA validation, and real-world data analysis, Dr. Shahraz supports evidence generation for clinical trials and regulatory submissions. His work bridges scientific rigor and patient-centered measurement to enhance the value and reliability of health outcomes data.
Konstantina Skaltsa, BSc, PhD
IQVIA, Barcelona, Spain
Jessica Roydhouse, PhD
Menzies Institute for Medical Research, University of Tasmania, Hobart, Australia
Dr Jessica Roydhouse is a Senior Research Fellow at the Menzies Institute for Medical Research, the Director of the Tasmanian Cancer Registry and the Academic Lead for the Prostate Cancer Outcomes Registry - Tasmania. Her research focuses on patient-reported outcomes, proxy reporting, cancer clinical trials and treatment tolerability. She was recently awarded the Prostate Cancer Foundation of Australia Priority Impact Research Award (2024). Jessica was recently Past Chair of the ISPOR Patient-Centered Special Interest Group and is an Editor-in-Chief of Quality of Life Research.

Including Equity in Health Technology Assessment: DCEA, Social Welfare Functions, and Inequality-Aversion Approaches

Session Type: Workshop

Topics: Economic Evaluation, Health Technology Assessment, Health Policy & Regulatory

Track: Expanded Value Measures

Level: Introductory

PURPOSE: Health technology assessments (HTAs) continue to grapple with methodologically-based methods to incorporate equity considerations. Current research (e.g., distributional cost-effectiveness analysis (DCEA)) provides clear descriptions of how interventions’ benefits and costs affect identified sub-populations. Social welfare functions (SWFs) provide formal and generalized ways to combine benefits accruing to various population members into a single weighted index of societal well-being. Atkinson’s inequality-aversion measure has been separately applied to consumption and health, but not to both simultaneously. This workshop will review methods at the frontiers of each of these approaches. DESCRIPTION: Workshop will provide state-of-the-art reviews of various approaches to introducing equity considerations into Health Technology Assessments (HTAs). Professor Griffin, a leading researcher of and advocate for DCEA, will begin with an overview of current DCEA methods as a basis for subsequent discussion. Professor Phelps will provide the foundational basis for valid social welfare functions, showing how the GRACE method for valuing healthcare gains enters such functions. This will include approaches to use these methods in fixed-budget settings. An application to understanding programs to accelerate treatment of rare diseases follows. Mr. Davis will then introduce, based on his in-progress doctoral dissertation, a new and important way to combine Atkinson’s (1970) Inequality Aversion both in the domains of wealth (consumption) and health. An example shows how introduction of new weight-loss drugs does not always compress inequality due to incomplete access by lower-income persons, and provides (for the first time) a monetized measure of the cost of reducing inequality. Audience participation will include an extensive audience Q&A session led by Professor Garrison (20 minutes). This workshop will provide attendees with a cutting-edge view into modern methods for incorporating equity considerations into HTAs.

Moderator

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

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

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

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

Speakers

Susan Griffin, PhD
University of York, York, United Kingdom
Charles E Phelps, MBA, PhD
University of Rochester, Pittsford, NY, United States
Charles Phelps, PhD is professor and provost emeritus at the University of Rochester. He previously held appointments in the departments of economics and political science and served as the director of the Public Policy Analysis Program and chair of the Department of Community and Preventive Medicine in the School of Medicine and Dentistry. Earlier, Dr. Phelps served as a senior staff economist and the director of the Program on Regulatory Policies and Institutions at the RAND Corporation. Dr. Phelps’s research cuts across the fields of health economics, health policy, health technology assessment, and related topics, and he is the author of Health Economics (now in its sixth edition), among other books. He has testified before US congressional committees on health policy and intellectual property issues. He is a fellow of the National Bureau of Economic Research and serves on the board of directors of the Health Care Cost Institute. He has served as the chair of the Board of Directors of VirtualScopics, Inc., and as a consultant to Gilead Sciences, Inc., CardioDx, and Kaiser Permanente of Northern California. He received his BA in mathematics from Pomona College, an MBA in hospital administration, and a PhD in business economics from the University of Chicago. He is a member of the National Academy of Medicine.
Ian J Davis, MA
University of Southern California, Los Angeles, CA, United States

Are Drug Prices Rising Too Fast? Rethinking Inflation Benchmarks Through the Lens of Value, Access, and Innovation

Session Type: Issue Panel

Topics: Health Technology Assessment, Economic Evaluation, Medical Technologies

Track: Access and Drug Pricing

Level: Introductory

Purpose: Concerns about rising drug prices routinely dominate headlines, with many reports concluding that launch prices outpace inflation. These findings have fueled recent government interventions, including Prescription Drug Affordability Boards (PDABs), that aim to curb price growth and protect affordability for patients and payers. Yet inflation-based benchmarking may overlook a more fundamental question: How does price growth compare to the growth in value delivered by new therapies? Overview: This issue panel will examine the appropriateness of inflation-based drug price benchmarks versus value-based assessment approaches through a structured, multi-perspective debate. Dr. William Padula (University of Southern California) will moderate the session and open with a brief overview (10 minutes) framing the economic tension between affordability, innovation, and value, drawing on recent analyses comparing drug price trajectories with corresponding health gains. Dr. Jonathan Campbell (National Pharmaceutical Council) will argue that inflation benchmarks obscure value creation and risk undermining incentives for high-impact innovation, emphasizing the role of value-based frameworks in contextualizing price growth. Dr. Laura Pizzi (ISPOR) will examine inflation-based benchmarks as a pragmatic policy tool for addressing affordability, budget predictability, and patient financial exposure, which align with the ISPOR mission. Dr. Andrew York (Maryland Prescription Drug Affordability Board) will focus on policy implementation, discussing how inflation- and value-based approaches are operationalized in real-world settings, including PDAB processes and payer decision-making. Each panelist will have 10 minutes to establish their position, and the presentations will be followed by a moderated exchange and audience discussion (20 minutes). This session will benefit HTA researchers, policymakers, payers, manufacturers, and patient advocates seeking to understand how price, value, and access considerations can be better aligned in pharmaceutical policy debates.

Moderator

William V Padula, PhD
University of Southern California, Rancho Palos Verdes, CA, United States

Speakers

Jon Campbell, PhD
National Pharmaceutical Council, Washington, DC, United States
Laura Pizzi, MPH, PharmD
ISPOR, Lawrenceville, NJ, United States
Laura Pizzi is Chief Science Officer at ISPOR.
Andrew York, JD, PharmD
Maryland Prescription Drug Affordability Board, Bowie, MD, United States

Unlocking the Power of Real-World Evidence: The CFL-SASS Expert Consensus

Session Type: Issue Panel

Topics: Health Policy & Regulatory, Study Approaches, Methodological & Statistical Research

Track: Real-World Evidence (RWE)

Level: Intermediate

ISSUE: Real-world evidence (RWE) is now central to demonstrating the value of medicines, yet one of the field’s greatest challenges remains: how to communicate RWE compliantly, transparently, and impactfully to the decision makers who rely on it. FDA’s Consistent with FDA-Required Labeling (CFL) pathway offers a mechanism to do this, but uncertainty around what constitutes “scientifically appropriate and statistically sound” (SASS) evidence has limited its use. OVERVIEW: This panel session will introduce the CFL-SASS Principles by Expert Consensus for RWE, the first multi-sponsor, expert-driven effort to clarify methodological and regulatory expectations for generating and communicating RWE under the CFL pathway. Speakers will each take 10 minutes to present the perspectives noted above, followed by questions for discussion and audience interaction. Attendees will learn about: a) Why dissemination—not generation—is now the critical bottleneck for RWE impact b) The opportunities and challenges of applying CFL-SASS evidence standards for RWE c) The expert consensus recommendations addressing methodological and regulatory issues d) How this work can strengthen internal review, improve evidence transparency, and elevate HEOR’s role in informing access and reimbursement decisions The session will feature leading RWE, regulatory, and HEOR experts in a lively, interactive discussion on how this initiative can help the field unlock the full value of RWE

Moderator

Paul Stang, PhD, MS
AESARA, Chapel Hill, NC, United States

Speakers

Sissi Pham, PharmD
AESARA, Chapel Hill, NC, United States
Richard Willke, PhD
Scintegral Health Economics, Lawrenceville, NJ, United States
Denise Sánchez Palomo, JD, MS, MA
Opus Regulatory, New York City, NY, United States

10:30 AM - 1:30 PM

Poster Session 3

Session Type: General Meeting

Poster Tours 11:45AM–12:30PM | Presenters will be with their posters from 12:30PM–1:30PM

11:15 AM - 1:15 PM

Lunch Service (Exhibit Hall)

Session Type: General Meeting

1:45 PM - 2:45 PM

Making Caregiver Impact Count: Bridging the Evidence-to-Policy Gap in Value Assessment and Decision-Making

Session Type: Workshop

Topics: Health Technology Assessment, Patient-Centered Research, Real World Data & Information Systems

Track: Expanded Value Measures

Level: Intermediate

Despite contributing an estimated $600 billion in unpaid labor annually, caregivers remain underrepresented in health technology assessments (HTAs) and health policy frameworks. This workshop aims to describe key gaps and propose actionable solutions to systematically integrate caregiver impacts into HTA and policy decisions, based on results from a landscape review and the outputs from a recent, iterative, multi-stakeholder deliberation process. R. Brett McQueen will summarize the outputs from the multi-stakeholder deliberation, revealing stakeholder-prioritized caregiving data and voting results, highlighting economic and longitudinal evidence most useful for current decision-making contexts (10 mins). Stacey Kowal will present how validated tools, such as the Zarit Burden Interview, Caregiver Reaction Assessment, Work Limitations Questionnaire, and Short-Form Health Survey, can produce relevant evidence across disease severity stages that is fit for purpose for health policy and HTA (10 mins). Yadira Montoya will outline the opportunities and challenges for collecting caregiver impact data through patient advocacy organizations along with feasible options for broadening collection of these data (10 mins). Audience participation will include live polling to understand experiences with collecting caregiver impact data and priorities for future evidence generation. This workshop will be valuable for a broad coalition of stakeholders interested or currently using caregiver impact data including but not limited to: policymakers strengthening caregiver support and guiding funding; Prescription Drug Affordability Boards and HTA bodies incorporating caregiver evidence into deliberations; employers and advocacy groups enhancing workplace policies and protections in support of caregivers; and researchers embedding caregiver outcomes in economic models.

Moderator

Robert B McQueen, BA, MA, PhD
University of Colorado Skaggs School of Pharmacy and Pharmaceutical Science, Aurora, CO, United States
R. Brett McQueen is the director for the Center for Pharmaceutical Value (pValue) at the University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences, where he is an associate professor in the Department of Clinical Pharmacy. Brett’s work includes comparative effectiveness research, cost-effectiveness applications and methods development, multi-criteria decision analysis, outcomes-based contracting, and patient preferences research. He is active in ISPOR through contributions to short courses, workshops, issue panels, and research presentations.

Speakers

Stacey Kowal, BS, MSc
Genentech, Alameda, CA, United States
Yadira Montoya, MS
National Alliance for Caregiving, Washington, DC, United States

The GenAI Paradox for Qualitative Evidence Summarization: Exploring Real-World Use Cases and Validation Frameworks for Understudied but Impactful Use Cases

Session Type: Workshop

Topics: Patient-Centered Research, Real World Data & Information Systems, Study Approaches

Track: AI

Level: Intermediate

PURPOSE: As HEOR embraces generative AI (GenAI) to improve efficiency, an important paradox has emerged. GenAI adoption overwhelmingly targets structured computational tasks (modeling, network meta-analysis, systematic literature reviews), with supporting ISPOR guidance: ELEVATE-AI and CHEERS-AI. However, tasks using largely unstructured qualitative data remain underexplored despite GenAI's potential to excel in critical HEOR workflows: synthesizing evidence repositories, analyzing patient experience data (PED), including patient interviews or social media data sources, and informing study design. This workshop shares validation approaches for GenAI applications in qualitative evidence generation and summarization. DESCRIPTION: Katelyn Keyloun (Arysana) will provide a 5-minute overview describing emerging validation approaches for probabilistic Large Language Models (LLMs) using largely qualitative unstructured data. The audience will explore new approaches from Panelists representing multidisciplinary perspectives, who will present real-world applications (30 minutes total) describing how AI processes, analyzes, and summarizes unstructured qualitative data, emphasizing validation approaches: 1. Conversational AI for Data Collection (Bill Byrom, Signant Health): LLM-powered chatbots conducting qualitative in-trial patient interviews, including validation of interview technique quality, transcript generation, and reporting capabilities versus traditional human approaches. 2. Generate Early Patient Insights from Social Media (Catherine Foley, AbbVie): Leveraging GenAI to extract and synthesize patient perspectives from digital communities, addressing validation challenges for patient-generated content and early signal detection. 3. LLMs for Study Design (Justyna Amelio, AbbVie): Applying GenAI to inform study design decisions and protocol development by synthesizing evidence across multiple sources, and validating AI-generated recommendations. Interactive audience participation (20 minutes) includes polls and moderated discussion exploring opportunities in adopting GenAI, including validation, implementation barriers, and organizational readiness.

Moderator

Katelyn Keyloun, BS, MS, PharmD
Arysana, Carson City, NV, United States

Speakers

Bill Byrom, PhD
Signant Health, Nottingham, United Kingdom
Catherine Foley, MA, MPH
AbbVie, Milton, MA, United States
Justyna Amelio, PhD
AbbVie, Maidenhead, United Kingdom

Incorporating Patient, Family, and Public Perspectives to Advance Novel and Societal Elements of Value in Value Assessment

Session Type: Workshop

Topics: Methodological & Statistical Research, Patient-Centered Research, Economic Evaluation

Track: Expanded Value Measures

Level: Intermediate

PURPOSE: Interest in expanding value and health technology assessments (V/HTAs) to incorporate novel elements of value in the broader societal perspective (e.g., option value, family/caregiver spillover) has grown rapidly. However, theoretical and methodological progress has outpaced the generation of patient-, family-, and stakeholder-informed evidence to support the integration of these elements of value in V/HTAs. This workshop will feature proof-of-concept applications to illustrate how incorporating diverse stakeholder perspectives can strengthen the development, prioritization, and application of novel value elements in V/HTAs. DESCRIPTION: Workshop attendees will gain practical insight into approaches for integrating diverse stakeholder perspectives in operationalizing broader value elements. Dr. Yang will present how stakeholder inputs—spanning families, school personnel, and community members —informed the development and implementation of a societal value framework for pediatric vaccination, using COVID and flu as examples (10 minutes). Dr. Li will present results from a national survey of cancer patients and caregivers assessing preferences related to preserving access to future innovations, demonstrating how stakeholder-derived evidence can inform the use of option value in V/HTAs (10 minutes). Dr. Slejko will present research on U.S. public preferences related to equity and findings from a multistakeholder eDelphi study that can guide recommendations on methods and needs for patient-centered V/HTAs (15 minutes). Finally, Dr. Xie will synthesize and discuss the practical strategies of incorporating stakeholder input in operationalizing novel elements of value in V/HTAs, and potential implications for patient welfare. Audience participation will include live polling, Q&A, and facilitated brainstorming on where patient and stakeholder input is most valuable and how these perspectives can be incorporated into the development and quantification of broader value elements (20 minutes).

Moderator

Richard Xie, PhD
RA Capital Management, Newton, MA, United States

Speakers

Meng Li, MS, PhD
Tufts Medical Center, The Center for the Evaluation of Value and Risk in Health, Boston, MA, United States
Julia F. Slejko, PhD
University of Maryland Baltimore, Baltimore, MD, United States
Jingyan Yang, DrPH
Pfizer Inc., New York, NY, United States

Coordinating the Quagmire: Real World Evidence in Value Assessment in Later Stages of the Product Lifecycle

Session Type: Issue Panel

Topics: Real World Data & Information Systems, Health Policy & Regulatory, Health Technology Assessment

Track: Real-World Evidence (RWE)

Level: Intermediate

ISSUE: Value assessment of a medicine at the middle or end of its lifecycle could benefit from much richer evidence base compared to launch and should be heavily informed by real-world evidence (RWE) on the patients who actually used the treatment in practice. Yet, there remains significant variability in how RWE is used to inform the value of pharmaceuticals post-launch. In the US, IRA negotiations involve submission of comparative RWE, with the extent of its use in CMS pricing decisions remaining unclear. In ex-U.S. markets, use of RWE in HTA reassessments and “living HTA” are increasingly of interest, with experience so far revealing the potential for these extensions of HTA to significantly change from value assessment at launch. This panel will propose a roadmap outlining how innovators can strategically plan post-launch RWE generation to meet long term evidence needs across the product lifecycle in US and key ex-US markets. OVERVIEW: Moderator will begin with presentation on the overview of the Issue. Panelist 1 will discuss US IRA experience on comparative RWE evaluation within the context of Price Negotiations. Panelist 2 will discuss role of RWE in HTA reassessments and as a component of living HTA. Panelist 3 will provide ISPOR and HEOR community perspective in the convergence of RWE and HTA activities. All Panelists will present their perspectives on the critical elements of a roadmap to anticipate post-launch RWE needs and how to streamline this process.

Moderator

Ambarish J. Ambegaonkar, PhD
APPERTURE LLC, Marlboro, NJ, United States

Speakers

Gergana Zlateva, PhD
Pfizer Inc., New York, NY, United States
Shanthy krishnarajah
Johnson and Johnson, New Hope, PA, United States
Laura Pizzi, MPH, PharmD
ISPOR, Lawrenceville, NJ, United States
Laura Pizzi is Chief Science Officer at ISPOR.

Beyond Cronbach’s Alpha: Modern Reliability Assessment for COA Research and Regulatory Decision-Making

Session Type: Other Breakout Session

Topics: Patient-Centered Research, Methodological & Statistical Research, Study Approaches

Track: Real-World Evidence (RWE)

Level: Intermediate

Purpose: This session is to advance COA measurement practice by addressing whether Cronbach’s alpha is fit for purpose and by equipping researchers with modern methods for evaluating reliability. As regulators increasingly request evidence beyond alpha to support an outcome measure’s fit-for-purpose, COA teams need clear guidance on when alpha is appropriate, when it is misleading, and how alternatives such as omega, ICC, split-half reliability, and IRT-based conditional reliability can strengthen COA submissions. This session will provide foundational understanding, practical analytic demonstrations, and regulatory context to help attendees improve reliability evaluation in clinical trials, qualification programs, and labeling claims. Real-time polling on current use of methods will have facilitate discussion. Description: Cronbach’s alpha remains the dominant measure of reliability in COA research, yet it is often applied under conditions that may violate its central assumptions or is used to support incorrect inferences. The session begins by clarifying the assumptions and properties of alpha, such as tau-equivalence, including unidimensionality and equal item contributions, and scale length dependence. The second presentation introduces contemporary alternatives, including McDonald’s omega, ICCs, split-half permutation methods, and IRT-based conditional reliability, explaining how each aligns with COA conceptual frameworks and different instrument designs. A third presentation compares alpha against these modern indices, using empirical examples, to demonstrate how reliability conclusions may change when multidimensionality, item information, or temporal stability are considered. The final presentation connects these methodological insights to regulatory expectations, highlighting how FDA and EMA increasingly critically evaluate internal consistency evidence, request domain-level reliability, caution against inflated alpha values, and rely on structural validity assessments.

Moderator

R. J. Wirth, PhD
Vector Psychometric Group, Chapel Hill, NC, United States

Speakers

Stephen Maher, PhD
Sharon, MA, United States
Danielle Rodriguez, PhD
Thermo Fisher Scientific, Snohomish, WA, United States
Patrick Daniele, MSc
Thermo Fisher Scientific, Vancouver, BC, Canada

Rural-Urban Disparities in Cancer Care: Risk Factors, Treatment Access, and Survival Outcomes

Session Type: Issue Panel

Topics: Health Service Delivery & Process of Care, Health Policy & Regulatory, Clinical Outcomes

Track: Expanded Value Measures

Level: Introductory

Disparities in cancer survival outcomes between urban and rural populations are particularly pertinent in Canada, the world’s second largest country by land area. Rural populations consistently experience poorer cancer survival outcomes that could be influenced by differences in risk factors for cancer, socioeconomic status, and access to public healthcare services associated with diagnosis, treatment and palliative care. We conducted a targeted literature review using MEDLINE and Embase to identify real-world evidence studies published in Canada, Australia and the US between 2010 and 2025. We identified studies that report on differences between urban and rural populations in the prevalence of various risk factors (such as obesity and smoking), disease stage at diagnosis, access to treatment (e.g., time from diagnosis to start of treatment), socioeconomic status, and overall survival from diagnosis. Inclusion criteria included lung, breast, prostate and colorectal cancer types (which represent the four most prevalent cancers in Canada), adult age, and English language. Understanding the extent to which rural populations are underserved in cancer care is a critical public health priority. We are proposing to bring together a panel of multidisciplinary experts with perspectives of the patient, clinician, researcher to discuss both the findings of this research and engage with policy makers to develop targeted strategies to promote health equity based on geographical location in cancer care across Canada.

Moderator

Cal Shephard, MSc
AstraZeneca, Toronto, ON, Canada

Speakers

Julie Stakiw, MD
Saskatchewan Cancer Agency, Saskatoon, SK, Canada
Robert Bick, BSc.
CanCertainty Coalition, Markham, ON, Canada
Soo Jin Seung, BSc
Sunnybrook Health Sciences Centre, Toronto, ON, Canada

HEOR as a Compass, Not a Roadblock: How and When Development-Stage Biotech Companies Should Leverage HEOR

Session Type: Workshop

Topics: Health Technology Assessment, Health Policy & Regulatory, Economic Evaluation

Track: Access and Drug Pricing

Level: Introductory

Purpose: Development-stage biotechnology companies are primarily focused on advancing a safe, effective drug and securing the capital necessary by fundraising through private capital investments to sustain innovation. As health policy uncertainty accelerates and price scrutiny intensifies, development-stage companies can no longer focus solely on evidence for regulatory approval; they also need to consider evidence to demonstrate potential commercial success. Evidence and approaches central to health economics and outcomes research can be leveraged to address this need. This workshop will highlight how and when health economic and outcomes research (HEOR) evidence and methodologies can be leveraged by development-stage biotechnology companies and when each approach could be implemented to support their fundraising, acquisition, and/or commercialization success. Participants will learn which health economic and outcomes research methodologies are most aligned with the goals of development-stage biotechnology companies and when each could be implemented. Description: The workshop will be introduced by Dr. Canestaro, who will briefly summarize the different phases of pre-clinical and clinical research, the goals for drug development, and the rapidly evolving policy landscape that impacts drug development (5 mins). Then, Dr. Canestaro will moderate a fireside chat with three panelists (Fine, Hepp, Masia) with experience implementing HEOR within the operations of development-stage biotechnology companies. Each panelist will provide real-world examples of how they have applied specific HEOR approaches within the development stage and will discuss 1) when the need for HEOR arises, 2) the impacts of their efforts, 3) adaptations to align with the goals of development-stage companies, and 4) missed opportunities. The session will conclude with audience polling to assess the relative strength of various health economic and outcomes research methods (e.g., budget impact, cost-effectiveness analysis, evidence synthesis, patient preference data) for development-stage companies. The session will reserve twenty minutes for audience Q&A.

Moderator

William J Canestaro, MS, PhD
University of Washington, Seattle, WA, United States

Speakers

Jen Fine, MS, ScD
Abivax, San Francisco, CA, United States
Zsolt Hepp, MS, PharmD
SEATTLE GENETICS, Kirkland, WA, United States
Neal Masia, PhD
EntityRisk, Inc., Princeton, NJ, United States

Pursuing Affordability Decision Rules: Towards Transparent, Evidence-Informed Budget Impact Thresholds

Session Type: Issue Panel

Topics: Health Technology Assessment, Economic Evaluation, Health Policy & Regulatory

Track: Access and Drug Pricing

Level: Intermediate

Issue Description Health systems worldwide increasingly face technologies that are clinically valuable yet financially burdensome. While cost-effectiveness thresholds are well studied, affordability—how much health systems can realistically pay, over what time horizon, and under what constraints—remains one of the most unresolved and contested issues in Health Techonology Assessment (HTA). Countries and organizations apply divergent approaches: some rely on implicit political judgements, others use explicit budget-impact triggers, and global agencies emphasize fiscal space and opportunity costs. This Issue Panel brings together three different perspectives to debate how affordability should be defined, measured, and incorporated into evidence-based decision making. The debate will explore tensions between value for money and short-term budgets, the relationship between affordability and equity, and the feasibility of global or standardized frameworks. We will include polling and audience interaction. Relevance & Importance Affordability is central to pricing and access decisions for oncology therapies, gene and cell therapies, vaccines, and high-cost chronic treatments. Yet payers, HTA agencies, and global health institutions lack consistent rules or guidance. This panel addresses a crucial gap by contrasting US, European, and Canadian perspectives, highlighting practical, economic, and political challenges in operationalizing affordability.

Moderator

Federico Augustovski, MSc, PhD, MD
Institute for Clinical Effectiveness and Health Policy, Buenos Aires, Argentina
Federico Augustovski is the current director of Health Economic Evaluations and Technology Assessment at the Institute for Clinical Effectiveness and Health Policy (IECS), an independent non-profit organization affiliated to the University of Buenos Aires, a CONICET (National Scientific and Technical Research Council) center, and one of the few INAHTA Health Technology Assessments agencies in Latin America. He is the director of the WHO Collaborating Centre in Health Technology Assessment and Economic Evaluations at IECS. He is a professor of Public Health at the School of Public Health of the University of Buenos Aires, where he teaches courses for graduate and postgraduate students in Decision Sciences; Patient Reported Outcomes (PRO) Development in Health as well as Health Economic Evaluations.

He was elected president 2017-2020 of ISPOR (The Professional Society for Health Economics and Outcomes Research). He is also the founding editor-in-chief for Latin America of Value in Health Regional Issues (ViHRI), the ISPOR peer-reviewed journal for Latin America, Asia, and Central & Eastern Europe and Africa. He is the director of the PAHO affiliated PROVAC Center of Excellence for decision making in vaccines. He leads a multidisciplinary team devoted to clinical and economic evaluations of new and existing preventive, diagnostic and therapeutic technologies that works doing research, education, and technical support with public and private health decision makers in Latin America.

He got his medical degree with honors at the University of Buenos Aires (1986-1991), and he is a specialist in family medicine. He practiced family medicine and was a staff physician for more than 20 years at the Family and Community Medicine Division of the Hospital Italiano de Buenos Aires (the leading academic non-profit hospital in Argentina). He got his MSc in Epidemiology (Harvard School of Public Health, 1997-1999). He was an Alban Scholar of the European Union in Health Economics (2003-2004) getting research and training experience at the Centre for Health Economics at York University in the UK.

His research production and publication in international peer-reviewed journals (with more than 160 PubMed indexed papers), concentrates on health technology assessments, health economic evaluations (ie, multi-country studies in Latin America, both model-based and individual patient level piggyback studies, PRO and preference status measures and validation (ie, derivation of Argentine, Uruguay and Peruvian weights for the EQ-5D, Argentine SF-36 validation, and discrete choice experiments).

Speakers

Marina Richardson
ICER, Boston, MA, United States
Marina Richardson is an associate director, Health Technology Methods and Health Economics with the Institute for Clinical and Economic Review (ICER). In her role, Marina leads and oversees the development of economic models to inform pricing and reimbursement decision-making and identifies and executes opportunities to enhance ICER's methodology and processes within Health Technology Assessment (HTA) and Health Economics. Prior to joining ICER, Marina led and contributed to reimbursement review reports and recommendations at Canada's Drug Agency (CDA-AMC), formerly CADTH. Marina has a PhD in Health Services Research from the University of Toronto and is an active contributor to the field including as a deputy editor for the International Journal of Technology Assessment in Health Care (IJTAHC), a member of the Ontario Immunization Advisory Committee (OIAC) in Ontario, Canada, and as a co-chair of the International Scientific Program Committee for Health Technology Assessment International (HTAi) 2025.
Lotte Steuten, MSc, PhD
Office of Health Economics, London, United Kingdom
Bernice Tsoi
pan-Canadian Pharmaceutical Alliance, Ottawa, ON, Canada

2:45 PM - 3:15 PM

Coffee and Connect

Session Type: General Meeting

Head to the exhibit hall to connect with fellow attendees and exhibitors over a steaming cup of coffee.

4:00 PM - 7:00 PM

Poster Session 4

Session Type: General Meeting

Poster Tours 4:00PM–4:45PM | Presenters will be with their posters from 6:00PM–7:00PM

4:45 PM - 5:45 PM

The Time Has Come for Patient-Centric Composite Endpoints in Comparative Studies

Session Type: Workshop

Topics: Study Approaches, Methodological & Statistical Research, Clinical Outcomes

Level: Intermediate

Purpose: Traditional analyses of randomized clinical trials (RCTs) or other comparative effectiveness studies do not routinely incorporate patient preferences. This workshop will provide participants with a step-by-step approach to eliciting preference weights and applying them to data from RCTs using novel methodologies—Win Statistics and Weighted Composite Endpoints (WCE)—to enable patient-centered comparative effectiveness research. Description: Dr. Gouda will review newer analytic approaches, including win statistics and weighted-composite endpoints (WCE), designed to address limitations of conventional analytic methods for treatment comparisons in RCTs. Dr. Reed will review alternative stated-preference methods that can be used to quantify the relative importance of various medical events for incorporation in these analytic methods. The audience will participate in a Case-1 best-worst scaling exercise used to generate weights for medical events relevant to benefit-risk evaluations of anticoagulants in a survey of 1028 patients with atrial fibrillation. Dr. Shoji will demonstrate how these patient-preference weights can be applied in win statistics and WCE approaches using a worked example from a patient-level analysis of 58,541 individuals across four RCTs of direct oral anticoagulants versus warfarin. The audience answer polling questions about their interpretation of findings from different approaches.

Moderator

Pishoy Gouda, MD, MSc
University of Calgary, Calgary, AB, Canada

Speakers

Shelby Reed, RPh, PhD
Duke Clinical Research Institute, Durham, NC, United States
Satoshi Shoji, MD, PhD
Duke Clinical Research Institute, Durham, NC, United States

Advancing Real-World Evidence for Pediatric Drug Safety: Methods, Off-Label Use, and Regulatory Applications

Session Type: Other Breakout Session

Topics: Real World Data & Information Systems, Methodological & Statistical Research, Health Policy & Regulatory

Track: Real-World Evidence (RWE)

Level: Intermediate

Purpose Children are often not included in randomized trials, leaving major evidence gaps for many medications used in pediatric care. Off-label prescribing is common across pediatric specialties, which increases the need for reliable real-world evidence (RWE) to inform safety and benefit–risk decisions. However, generating high-quality pediatric RWE is challenging because of low event rates, changing treatment patterns, and limited pediatric-specific clinical information in many datasets. This session aims to provide practical strategies to improve the design, analysis, and interpretation of pediatric RWE studies and to discuss how these approaches can support regulatory and clinical decision-making. Description Panelists will outline how to evaluate whether a real-world data source is fit for use in pediatric studies, including assessing completeness of variables, follow-up, capture of off-label use, and the ability to define exposures, covariates, and outcomes relevant to children. The session will describe approaches for addressing confounding and bias when sample sizes are small or covariates are imbalanced, such as overlap weighting. Examples from pediatric studies of biologics and other widely used therapies will illustrate how these methods work in practice, especially when studying rare safety outcomes such as serious infections. The session will also consider how strengthened RWE can inform safety assessment for off-label pediatric use and complement existing regulatory frameworks for benefit–risk evaluation. Panelists will highlight the importance of transparency, reproducibility, and clear communication of uncertainty when estimates are imprecise. Attendees will gain practical guidance for evaluating data fitness, selecting appropriate analytical methods, and interpreting findings in settings where traditional trial evidence is limited or unavailable. The discussion will emphasize actionable steps to support pediatric RWE generation.

Moderator

Ning Lyu, MS, PhD
University of Houston College of Pharmacy, Sugar Land, TX, United States

Speakers

Sebastian Schneeweiss, ScD, MD
Brigham and Women's Hospital and Harvard Medical School, Boston, MA, United States
Timothy Savage, MD, MPH, MSc
Brigham and Women's Hospital and Harvard Medical School, Boston, MA, United States

Can New Approaches to Estimating Dynamic Efficiency Help to Maintain Both Access and Innovation in Pharmaceutical Markets?

Session Type: Issue Panel

Topics: Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment

Track: Access and Drug Pricing

Level: Intermediate

Pharmaceutical firms and payers face dual challenges of ensuring patient access to medicines while maintaining economic incentives for development of innovative treatments. This balance - in economic terms, dynamic efficiency - is crucial. The global nature of pharmaceuticals adds complexity, as preferences and resources very across countries. Recently, novel frameworks have been proposed to estimate optimal producer surplus (PS), each with unique assumptions and relevance to different markets. This panel will discuss the core challenge of dynamic efficiency in pharmaceutical markets. Panelists will provide diverse perspectives on approaches to estimating dynamic efficiency at the industry and product levels. The issue that will be debated is whether dynamic efficiency and estimates of PS are helpful for characterizing the value of a medicine retrospectively, prospectively, or not at all. Attendees will gain insights into the complexities of dynamic efficiency and potential pathways to maintaining and demonstrating it as more patient-centered aspects of value are incorporated into value assessments. With real-time polling and 15-20 minutes of Q&A, the audience will be able to provide their perspectives on the importance of achieving dynamic efficiency and implications for value assessment.

Moderator

Robert J Nordyke, BS, MS, PhD
Beta6 Consulting, Topanga, CA, United States

Speakers

Kimberly Westrich, MA
National Pharmaceutical Council, Herndon, VA, United States
Kimberly Westrich, MA, is the Chief Strategy Officer of the National Pharmaceutical Council (NPC), which sponsors and conducts research on health policy issues related to the development and use of innovative biopharmaceuticals to improve the health of patients. NPC’s research contributes to the body of evidence that supports discussions and decisions about patient access to treatments, appropriate use, and the value innovative treatments provide to both patients and the healthcare system. Ms. Westrich provides strategic guidance to NPC’s policy research and communications activities. She leads several research initiatives across NPC’s portfolio, including employer-sponsored insurance. She has published extensively on issues related to value assessment frameworks, patient-centered formulary and benefit design, value-based contracts, quality performance measurement, and accountable care organizations. Additionally, Ms. Westrich leads NPC’s workplace culture initiative — most notably demonstrated by our company's recognition as a Certified Great Place To Work®. Ms. Westrich began her NPC career in 2000. She has also served as Director of Research at the Pharmaceutical Research and Manufacturers of America (PhRMA), worked as a healthcare consultant at The Lewin Group and Xcenda, and as a health economics and outcomes researcher at Johnson & Johnson. Ms. Westrich is a certified yoga teacher and life coach with a passion for helping others learn and thrive. She received her master’s degree in economics from Northwestern University and her undergraduate degree in economics and mathematics from the College of William and Mary.
Robert B McQueen, BA, MA, PhD
University of Colorado Skaggs School of Pharmacy and Pharmaceutical Science, Aurora, CO, United States
R. Brett McQueen is the director for the Center for Pharmaceutical Value (pValue) at the University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences, where he is an associate professor in the Department of Clinical Pharmacy. Brett’s work includes comparative effectiveness research, cost-effectiveness applications and methods development, multi-criteria decision analysis, outcomes-based contracting, and patient preferences research. He is active in ISPOR through contributions to short courses, workshops, issue panels, and research presentations.
Anirban Basu, PhD
University of Washington, Seattle, WA, United States

Following the Trail: A Journey of Individual Patient Preference Data from Response to Analysis to Clinical Encounter

Session Type: Workshop

Topics: Patient-Centered Research, Methodological & Statistical Research, Study Approaches

Track: Expanded Value Measures

Level: Introductory

PURPOSE: This workshop will guide participants along the trail of individual patient preference data from the moment a patient responds to a survey, through analytic methods that transform those responses into actionable insights, and finally to the clinical encounter where these insights inform shared decision-making. DESCRIPTION: Participants will begin their journey with a threshold exercise, experiencing firsthand the cognitive process patients navigate when expressing risk tolerance using this approach. Next, discussants will compare approaches for estimating patient-level preferences and risk-tolerance (probablistic threshold technique, the multidimensional threshold technique, respondent-level estimates from mixed logit models, and segmentation of individuals across latent classes that vary by risk-tolerance). Discussants will highlight practical considerations across methods including sample-size requirements, participant burden, and within-person consistency. We will conclude with a demonstration of how latent-class findings can be utilized to design predictive questions for decision-support tools, enabling clinicians to personalize benefit-risk discussions for individual patients. Attendees will conclude their journey with a greater understanding of the patient experience (i.e. what it feels like to contribute individual level preference data) and the researcher experience (i.e. weighing the methodological differences and practical applications across methods). Agenda: • Welcome and introduction (5 minutes) • Participate in threshold exercise (10 minutes) • Share/reflect break-out (5 minutes) • Probablistic Threshold and DCE Case Study (10 minutes) • Multidimensional Threshold Case Study (10 minutes) • Method Compare Q&A (5 minutes) • Individual Latent-class Predictions (10 minutes) • Questions/Group Discussion (5 minutes)

Moderator

Marco Boeri, BSc, MSc, PhD
OPEN Health, London, United Kingdom

Speakers

Jessie Sutphin, MA
Duke Clinical Research Institute, Mooresville, NC, United States
Jennifer Whitty, PhD
Thermo Fisher Scientific, London, United Kingdom
Juan M Gonzalez, PhD
Duke Clinical Research Institute, Cary, NC, United States

Advancing HEOR With Next-Generation AI: Multimodal LLMs, Digital Twins, and Reinforcement Learning for Personalized Therapy

Session Type: Workshop

Topics: Methodological & Statistical Research, Real World Data & Information Systems, Patient-Centered Research

Track: AI

Level: Intermediate

Purpose:This workshop will introduce AI and ML approaches—including unsupervised and supervised ML, LLMs, digital twins, and RL—to address unmet needs in HEOR. The session will help attendees understand when next-generation AI adds value beyond traditional analytics; how these approaches can strengthen personalized therapy in RWE studies; and review methodological, ethical, and transparency considerations for responsible HEOR application. Description:AI innovations—including generative and multimodal LLMs, digital twins, and RL—are reshaping HEOR by enabling deeper insights from real-world data, identifying meaningful patient subgroups, and informing individualized treatment strategies. This workshop provides an accessible overview of these emerging methods, their applications, and considerations for ethical implementation. The session will feature presentations: •Dr. Marcum (moderator): Overview of the evolution of ML/AI in HEOR, highlighting unmet needs. •Dr. Tian: Case study showing how integrating unsupervised and supervised ML uncovers meaningful patient subgroups in schizophrenia and inform personalized treatment strategies. •Dr. Wu: Application of digital twins and multimodal LLMs to predict treatment responses in oncology and mental health disorders, emphasizing potential to enhance individualized decision support. •Dr. Lo-Ciganic: Use of advanced ML to characterize complex treatment pathways using sunburst plots to reveal real-world prescribing patterns. RL Illustrations show support for providers tailoring treatment strategies for patients with opioid use disorder and co-occurring mental health conditions, along with key ethical considerations like fairness, bias detection, and transparency. Interactive components include live polling and scenario-based questions to engage attendees in evaluating strengths, limitations, and implementation challenges. The workshop will conclude with a moderated discussion on best practices for integrating next-generation AI/ML into HEOR to support personalized therapy and evidence-informed decision making.

Moderator

Zachary Marcum
Medicus Economics, Hamilton, OH, United States

Speakers

Weihsuan J Lo-Ciganic, MS, PhD
University of Pittsburgh School of Medicine, Pittsburgh, PA, United States
Marc Tian, PhD
Teva Pharmaceuticals, Skillman, NJ, United States
Yonghui Wu, PhD
University of Florida, Gainesville, FL, United States

Beyond the Usual First Launch: Making Differential Pricing Work for Innovative NCD Medicines in Middle-Income Countries

Session Type: Issue Panel

Topics: Health Policy & Regulatory, Economic Evaluation, Health Technology Assessment

Track: Access and Drug Pricing

Level: Intermediate

ISSUE: Shifts in US and EU pricing and reimbursement policies are reshaping launch incentives and global market access strategies. As these changes take hold, companies may look beyond traditional first-to-access countries. Yet middle-income countries (MICs)—home to 75% of the world’s population and 75% of the non-communicable disease (NCD) burden—still face delayed or absent access to patented innovations. This panel will explore how sustainable global differential pricing can enable timely, value-based, and affordable access to innovative NCD therapies in MICs. We will focus on system-level enablers and pragmatic solutions aligned with evolving market dynamics and commercial viability. Overview: A practical model for global differential pricing — anchored in countries’ health opportunity costs and local value-based assessment (Value-based Tiered Pricing, VBTP) — has been proposed to expand access in low- and middle-income settings. The panel will examine bottlenecks to implementation and highlight actionable pathways for payers, policymakers, and manufacturers to move from concept to execution in MICs. What the audience will learn • How US/EU policy shifts alter commercial opportunities for biopharmaceutical firms that prioritize MIC. • Why MICs are central to access for NCD innovations—and what delays timely uptake. • How to operationalize VBTP that is feasible, impactful and sustainable. • Concrete steps stakeholders can take within 12–24 months. Speaker contributions • Richard Xie: Commercial opportunities in MICs—assessing growth potential under differential pricing, with a case example from China. • Kalipso Chalkidou: Health system readiness—closing gaps in data, HTA capacity, and governance to enable credible local value assessment, transparent pricing, and accountable implementation. • Prashant Yadav: Safeguarding the model—assessing arbitrage risks and proposing supply chain and traceability solutions to prevent leakage while sustaining differential prices. Format: Moderator scene-setting: 5 minutes; Speaker presentations: 3 × 12 minutes; Audience discussion: ~20 minutes focused on implementation challenges and near-term actions.

Moderator

Mikkel Oestergaard, PhD
Merck Sharp & Dohme International Service B.V., Zurich, Switzerland

Speakers

Richard Xie, PhD
RA Capital Management, Newton, MA, United States
Kalipso Chalkidou, PhD
The Global Fund to Fight AIDS, TB and Malaria, Geneva, Switzerland
Prashant Yadav, PhD
Council on Foreign Relations, Washington, DC, United States

Measuring What Matters: A New Blueprint for HEOR Impact Metrics

Session Type: Issue Panel

Topics: Organizational Practices

Track: Expanded Value Measures

Level: Intermediate

Issue: HEOR provides the evidence needed to support affordability, value-based decision-making, and policy development, yet its strategic importance is still not consistently recognized. Traditional productivity metrics, such as publication counts or timelines, do not capture HEOR’s influence on payer strategy, evidence prioritization, policy engagement, or enterprise value. As policymakers and payers demand clearer demonstrations of value, HEOR’s inability to quantify its contributions has become a barrier to its credibility and relevance. The issue is understanding why HEOR’s impact remains difficult to measure and identifying practical approaches to clarify its role in policy, access, and value decisions. Overview: This 60-minute issue panel will debate the real-world challenges of demonstrating HEOR’s impact and explore contrasting viewpoints on how the field can strengthen its visibility with internal and external decision-makers. Three panelists representing biotech, midsize, and large HEOR organizations will present concise, distinct positions (10 minutes each). John will argue that HEOR undersells its influence and fails to measure the impact cross-functional partners already acknowledge. Kinga will contend that impact metrics should be grounded in how evidence is used in payer and policymaker interactions, supported by rapid feedback from integrated field teams. Jan will caution that global complexity, regional variation, and inconsistent data flows make unified HEOR impact metrics difficult to implement and potentially misleading. Moderated by AESARA—drawing on insights from its 2024 industry HEOR benchmarking initiative and its consulting experience in organizational transformation and evidence strategy—the session will begin by framing measurement gaps and opportunities for defining credible HEOR impact metrics. A 30-minute moderated debate and audience Q&A will challenge assumptions and surface practical solutions. HEOR stakeholders will leave with strategies to elevate HEOR’s visibility and better demonstrate its impact on policy, access, and enterprise decisions.

Moderator

Yamini Misra
AESARA, Houston, TX, United States

Speakers

Jan E. Hansen, PhD
Sanofi, Scottsdale, AZ, United States
Dr. Jan Hansen is Global Head of Health Economics and Value Assessment (HEVA), which is a function in the Global Market Access organization in Sanofi's Specialty Care business. In this role, Jan oversees global health economics and outcomes research (HEOR) strategy development and tactical project execution. Her team’s work leads to impactful dissemination and application of HEOR across global stakeholders and health care systems.

Prior to joining Sanofi, Jan had an impressive career spanning over three decades in life sciences. Jan has held distinguished leadership positions at Genentech and Allergan, where she demonstrated her talent for building and guiding high-performing HEOR and access teams. Her expertise in integrating health economics and HTA considerations into development strategies has consistently led to successful product positioning and enhanced patient reach.

As a past President of ISPOR and active contributor to prestigious organizations like the National Pharmaceutical Council, AMCP Foundation, and PhRMA Foundation, Jan has established herself as a thought leader in our industry. Jan holds a Ph.D. in Pharmacy Administration from the University of South Carolina and a B.S. in Pharmacy from the University of Iowa, where she was honored with the Distinguished Alumni Award for Science.
Kinga Borsos, MASc, MBA, PharmD
Boehringer Ingelheim, Randolph, NJ, United States
John O'Donnell, MPP, PhD
Madrigal Pharmaceuticals, Thornton, PA, United States

6:00 PM - 7:00 PM

Philadelphia Street Festival

Session Type: General Meeting

Wed May 20

7:00 AM - 8:00 AM

Coffee and Connect

Session Type: General Meeting

Head to the exhibit hall to connect with fellow attendees and exhibitors over a steaming cup of coffee.

7:00 AM - 1:00 PM

Registration Hours

Session Type: General Meeting

8:00 AM - 9:00 AM

From Engagement to Strategy: An Evidence Based Framework for Evaluating Patient-Centricity in the Pharmaceutical Industry

Session Type: Issue Panel

Topics: Organizational Practices, Patient-Centered Research, Health Policy & Regulatory

Track: Expanded Value Measures

Level: Introductory

ISSUE. Pharmaceutical companies increasingly articulate commitments to patient-centricity, yet the concept remains vague, variably defined, and inconsistently operationalized across the industry. In contrast, patient-centeredness in medicine is a well-established construct, coined by Enid Balint in 1969, expanded by Mead and Bower’s 2000 framework for doctor–patient relationships, and reinforced by the Institute of Medicine’s designation of patient-centeredness as a core dimension of healthcare quality in 2001. By contrast, the term is much newer for the pharmaceutical sector, surfacing in 1993 in the context of drug promotion and marketing and gaining traction in 2012, alongside regulatory initiatives such as the FDA’s Patient-Focused Drug Development program. In this session, panelists will propose robust frameworks with definitions, outcomes, and methods for evaluating patient centricity in the pharmaceutical industry, that can help sponsors as they develop a comprehensive patient centered corporate strategy and move beyond patient engagement in clinical research. OVERVIEW: In this session, the moderator will provide background and characterize current practices across companies. Industry panelists will discuss case examples, frameworks and performance indicators being developed to support patient centricity and along with challenges in addressing tensions between patient and corporate goals. Patients will provide experiences and perceptions of patient centricity within the sector and recommend considerations for improving practices. An academic and ethicist will offer ethical considerations and a framework to guide pharmaceutical company-patient relationships, informed by a literature review and focus groups with patients and clinicians (in oncology and infectious disease). The outcome of this session will include proposal of comprehensive frameworks to guide pharmaceutical company-patient relationships and translate commitments into coherent organizational strategy and accountable practice.

Moderator

Jennifer Miller, PhD
Yale School of Medicine, Westport, CT, United States

Speakers

Pamela Gavin, BA, MBA
National Organization for Rare Disorders, Danbury, CT, United States
Alan Balch, MS, PhD
Patient Advocate Foundation and National Patient Advocate Foundation, Hampton, VA, United States
Dr. Balch has more than 20 years of executive leadership in the non-profit sector spanning multiple advocacy areas including access and affordability, health equity, prevention and early detection, and cancer research, He became the CEO of both PAF and NPAF in 2013 and has served as a member of both Boards of Directors since 2007. From 2006-2013, he served as Vice President of the Preventive Health Partnership -- a national health promotion collaboration between the American Cancer Society, American Diabetes Association, and American Heart Association. Prior to his work with the Preventive Health Partnership, Dr. Balch was the Executive Director of Friends of Cancer Research from 2003 to 2006. Dr. Balch currently serves or recently served on dozens of selective boards, steering committees, and councils for an array of institutions to include the National Academies of Medicine, the National Quality Forum (NQF), National Committee for Quality Assurance (NCQA), the Institute for Clinical and Economic Review (ICER), the Clinical Pathways Congress, the Council for Affordable Health Coverage (CAHC), the Innovation and Value Initiative (IVI), Core Quality Measure Collaborative (CQMC), the Foundation for the Accreditation of Cellular Therapy (FACT), the Hutchinson Institute for Cancer Outcomes Research (HICOR), the Duke-Margolis Value-Based Payment Consortium, the Specialty Pharmacy Certification Board (SPCB), and the Pharmacy Quality Alliance (PQA). Most recently, Dr. Balch was selected as the Chair of the Global Patient Council for the American Patient Representatives Roundtable for the Professional Society for Health Economics and Outcomes Research (ISPOR) after serving as the Co-Chair of the North American Patient Representatives Roundtable. Dr. Balch also serves on the editorial board and as a contributing editor for the Journal of Clinical Pathways and on the advisory board for the Journal of Oncology Navigation and Survivorship. He is frequently invited to peer review article submissions to various publications including the Journal of Health Care for the Poor and Underserved, Journal of Clinical Oncology, American Journal of Preventive Medicine, and the American Journal of Public Health. He earned his PhD in 2003 from the University of California Santa Cruz, his master’s degree in 1997 from the University of Texas San Antonio; and his bachelor’s degree in 1994 from Trinity University in San Antonio.
Cosmina Hogea, MS, PhD
Gilead, Foster City, CA, United States

Real-World Data and Evidence Supporting Value Assessment for Access and Reimbursement Decisions: Advancements and Experiences in US and China

Session Type: Other Breakout Session

Topics: Health Policy & Regulatory, Real World Data & Information Systems, Health Technology Assessment

Track: Real-World Evidence (RWE)

Level: Intermediate

PURPOSE: The objective of this session is to explore, with series of new national initiatives, how real-world data (RWD) and real-world evidence (RWE) can better support comprehensive value assessment of innovative drugs for access and reimbursement decisions in the US and China. It offers a multi-stakeholder perspective on policy progress, international experience, and industry implications. DESCRIPTION: In recent years, the US and China have increasingly embraced RWD/RWE in access and reimbursement decisions, such as Medicare Part D and National Reimbursement Drug List (NRDL) negotiations, respectively. However, despite this progress, gaps and barriers remain. The session will begin with an introduction of the context and current status (5 minutes, Xie). The next speaker will then discuss China’s evolving policy environment and key initiatives (e.g., real-world research pilot for comprehensive value assessment), along with the administration’s vision and expected outcomes. Challenges in integrating RWE into value assessment and reimbursement decisions in China will also be addressed (15 minutes, He). The session will continue with a comparative analysis of RWE’s role in Medicare Part D vs. China’s NRDL reimbursement pathway. Specifically, the role of RWE in the first round of US Medicare Part D negotiations and recent price reduction in weight management products will be examined, offering insights for China’s evolving system (15 minutes, Shi). The last speaker will then provide a global pharmaceutical industry perspective. The impact of recent RWE-related policy shifts on market access strategy, the operational and technical barriers, and examples of RWE successfully supporting value assessment will be shared (15 minutes, Tang). The session concludes with a 10-minute panel discussion among presenters and the audience, addressing open questions and practical recommendations to strengthen the RWE ecosystem for drug value assessment, especially in evolving markets like China. This session will benefit all HTA professionals, policymakers, pharmaceutical manufacturers, and RWE researchers.

Moderator

Yang Xie, MPH, PhD
IQVIA, Shanghai, China

Speakers

Xiaoning He, PhD
Tianjin University, Tianjin, China
Associate Professor, Major in Health and Pharmacy Administration, School of Pharmaceutical Science and Technology, Tianjin University. Visiting Scholar during Sep, 2019 to Mar, 2020 in the University of Sheffield in the UK. Research directions: Pharmacoeconomics, Real-World Research, Health Equity, and Health Policy Evaluation. PI for 1 General Program and 1 Youth Program of the National Natural Science Foundation of China, 1 Youth Program of the Natural Science Foundation of Tianjin. Published more than 50 journal papers, including over 30 SCI & SSCI papers in journals such as Value Health and Pharmacoeconomics. Participated in compiling Pharmacoeconomic Evaluation Guidelines in China 2020. Member and Secretary of the Pharmacoeconomics Professional Committee of the Chinese Pharmaceutical Association, Member of the Pharmacoeconomics Professional Committee of the Chinese Research Hospital Association, Member of the Health Technology Economic Evaluation Professional Committee of the Chinese Health Economics Association, Member of the Real-World Research Professional Committee of the Chinese Association of Chinese Materia Medica, Member and Secretary of the Pharmacoeconomics Professional Committee of the Tianjin Pharmaceutical Association.
Zhiliu Tang, Dr
GSK, Shanghai, China
Lizheng Shi, PhD
Tulane University School of Public Health and Tropical Medicine, New Orleans, LA, United States
Lizheng Shi, PhD, MsPharm, MA, is the Neal A. and Mary Vanselow Endowed Chair in the Department of Health Policy and Management at the School of Public Health and Tropical Medicine of Tulane University. He is the founding director of Tulane’s Health Systems Analytics Research Center (HSARC). Dr. Shi’s current health services research interest focuses on innovative health technologies to improve healthcare quality, access, and cost of patient-centered care from the equity perspective, using pharmaco-economics, health technology assessment, health analytics, and policy evaluation. Dr. Shi is dedicated to disseminating and translating population health knowledge at the local, national, and international levels. He is the associate editor of Value In Health and co-editor in chief for Pharmacoeconomics and Policy.

A Rare Dilemma: How to Fit an Unfittable Into Current HTA Paradigm

Session Type: Issue Panel

Topics: Health Policy & Regulatory, Health Technology Assessment, Real World Data & Information Systems

Track: Access and Drug Pricing

Level: Intermediate

ISSUE: Rare diseases (RDs) expose critical limitations in traditional frameworks. Small, heterogeneous populations, surrogate endpoints, single-arm trials, and lack of natural history data make it difficult to apply conventional evaluation methods. These challenges result in significant delays to patient access, as payers and manufacturers struggle to reconcile clinical uncertainty with pricing and value. Dominant QALY-based frameworks—used by 90% of OECD countries—may fall short in capturing the nuanced value of rare disease treatments, further compounding access barriers. This panel will explore how select HTA systems have adapted their frameworks to better accommodate RDs specificities, and which best practices should be implemented more broadly. OVERVIEW: According to the EU WAIT indicator, time to reimbursement for RDs therapies remains significantly longer than for non-RD across Europe and currently stands at an average of 607 days without significant improvement over the last 5 years. Diana Sinkevich will first clearly state the issue and present how some EU HTA bodies have begun adapting their HTA frameworks due to growing recognition of the need for flexibility (e.g. UK, Germany, Scotland, Sweden, Netherlands). Dr. Santos will present the CONITEC approach to RDs HTA evaluations and discuss that in the absence of specific pathway for RDs in Brazil, CONITEC takes a pragmatic approach and rely on evidence-collection post-approval. Dr. Alyami will discuss the broader evolution of HTA in Saudi Arabia, and highlight how international best practices that support the assessment of RD evidence packages are being incorporated into evolving HTA. Lastly, Dr. Wong-Rieger will discuss the Canadian experience as well as the patient expectation from the HTA bodies and the role patients play in supporting HTA bodies in minimizing uncertainties. Stakeholders who will benefit include HTA agencies, payers, industry leaders, patient advocates, and policy makers seeking to improve equity and timeliness in rare disease access.

Moderator

Dziyana Sinkevich, BSc, MSc
Chiesi, parma, Italy

Speakers

Marcos Santos, MSc, PhD
Brasilia University, Sao Paulo, Brazil
Fatimah Alyami
Center of Health Technology Assessment, Riyadh, Saudi Arabia
Dr. Fatimah Alyami is a senior health economist and licensed senior pharmacist with over a decade of multidisciplinary experience spanning health economics, health technology assessment (HTA), pharmaceutical policy, and clinical pharmacy. She currently serves as Health Economic Assessment Senior Specialist at the Center of Health Technology Assessment within the Center for National Health Insurance (CNHI), where she leads the design and appraisal of economic evaluations, value-based healthcare models, and evidence-based policy strategies tailored to the Saudi healthcare system. Dr. Alyami holds a PhD in Pharmaceutical Science (Health Outcomes) from the University of Cincinnati, along with dual master’s degrees in Pharmaceutical Economics & Policy and Pharmaceutical Outcomes & Policy. She is an active member of ISPOR, ISPE, and the Saudi Pharmaceutical Society, with research presented at leading international conferences and published in peer-reviewed journals. Recognized for her leadership and scholarly contributions, Dr. Alyami brings a strong commitment to advancing value-driven, evidence-based decision-making to improve healthcare efficiency, sustainability, and patient outcomes in Saudi Arabia.
Durhane Wong-Rieger, PhD
Canadian Organization for Rare Disorders, Toronto, ON, Canada
Dr. Durhane Wong-Rieger is the president and CEO of the Canadian Organization for Rare Disorders. She holds a Ph.D. in psychology from McGill University and previously served as a professor at the University of Windsor. Internationally, she is president of the Asia Pacific Alliance of Rare Disease Organisations, treasurer of the United Nations Nongovernmental Committee for Rare Diseases, patient advisor to the APEC Rare Disease Network, member of the International Research Consortium on Rare Diseases and RDI-Lancet Commission on Rare Diseases as well as board member of Testing to Targeted Treatments (FT3). She is Co-Founder and Immediate Past Chair of Rare Diseases International. In Canada, she is chair of the Consumer Advocare Network, president and CEO of the Institute for Optimizing Health Outcomes, and chair of the Canadian Heart Patient Alliance. She is currently Co-Chair of the Implementation Advisory Group of Canada’s National Strategy on Drugs for Rare Diseases. A certified health coach, trainer, and frequent lecturer, Dr. Wong-Rieger has authored three books and multiple articles.

Productivity Gains From Generative AI Across the HEOR Workflow: Successful Case Studies

Session Type: Other Breakout Session

Topics: Economic Evaluation, Health Technology Assessment, Health Policy & Regulatory

Track: AI

Level: Introductory

Purpose Generative AI (GenAI) is increasingly being adopted for systematic literature reviews, yet its broader potential to accelerate additional components of the HEOR workflow—such as health economic model development and verification, and HTA submissions—remains under-recognized. The objective of this session is to present real-world applications that provide concrete evidence of GenAI’s ability to enhance efficiency, improve productivity, and maintain methodological rigor across diverse HEOR activities. Description This session will feature practitioner-led case studies illustrating how GenAI is being successfully deployed to gain productivity. The session will be moderated by Uwe Siebert, who will introduce recent advances in GenAI technologies and discuss their emerging role in reshaping HEOR workflows, quality standards, and evidence generation practices. Jag Chhatwal will present a case study on the use of GenAI to automate components of health economic model verification, including a project conducted in collaboration with NICE. He will describe how GenAI-enabled workflows applied standard verification checklists with significant efficiency gains while preserving transparency and methodological rigor. Ipek Stillman will share insights from a GenAI-supported health economic model replication project performed in industry. This presentation will highlight both the benefits and the challenges encountered when using GenAI into complex modeling tasks. Turgay Ayer will demonstrate how 1000+ agentic GenAI systems were used to generate comprehensive landscape assessment reports within 48 hours. Their case study will illustrate how multi-agent architectures can coordinate evidence gathering, synthesis, and narrative development across large information spaces to inform HEOR work. The session will conclude with a moderated discussion on best practices, limitations, risks, and key considerations for responsibly and effectively incorporating GenAI into HEOR workflows, along with implications for long-term productivity, methodological standards, and expectations of regulatory and HTA bodies.

Moderator

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

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

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

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

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

Speakers

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

Patient-Centered Clinical Trial Design: Using Patient Experience Research to Improve the Patient-Centeredness of Patient-Reported Outcomes Measures and Patient Preference Research to Prioritize Patient-Centered Endpoints

Session Type: Other Breakout Session

Topics: Patient-Centered Research, Methodological & Statistical Research, Study Approaches

Track: Expanded Value Measures

Level: Introductory

Purpose: Discuss challenges and considerations in using patient experience and patient preference research to: 1. assess the patient-centeredness of patient reported outcome measures (PROMs) used in clinical trials and 2. understand how patients prioritize among meaningful aspects of health (MAH). Description: The FDA’s patient focused drug development (PFDD) program encourages use of patient reported outcomes (PRO) and patient preference information (PPI) throughout the product development lifecycle. The identification of MAH can inform endpoint selection in patient-centered clinical trial design. In an FDA-sponsored study, we assessed symptoms and function and PROMs used for genitourinary syndrome of menopause (GSM). Qualitative interviews followed by a best-worst scaling (BWS) survey were conducted to examine MAH in GSM. The findings describe patient priorities among MAH, and can inform selection of patient-centered trial endpoints and future adaptations of existing PROMs. The GSM study will highlight challenges and lessons learned. The session will start with an overview of, and practical considerations for, use of patient experience evidence in endpoint selection (Poulos, 8 minutes). Dr. Peay will describe qualitative research to identify gaps in PROs used for GSM and identify ways to improve patient-centeredness (14 minutes). Dr. Poulos will describe the application of Case 1 BWS to prioritize MAH and the conceptual and methodological challenges in this application of PPI (14 minutes). Dr. Roberts will describe the FDA’s perspective on the value of this type of patient-centered research and how it may be used in FDA decision making and advice (14 minutes). Speakers will attend to challenges including a common but heterogeneous syndrome, underinformed patients, poorly defined symptoms and symptom impacts, and lack of specific PROMs. Interactive polling will be used to engage participants. The session will conclude with an interactive discussion on pragmatic approaches to ensuring that the potential for patient-centered clinical trial design is realized (10 minutes).

Moderator

Christine Poulos, PhD
RTI- Health Solutions, Research Triangle Park, NC, United States

Speakers

Christine Poulos, PhD
RTI- Health Solutions, Research Triangle Park, NC, United States
Holly Peay, MS, PhD
Faegre Drinker Biddle & Reath, Washington, DC, United States
Jason Roberts, PhD
Food and Drug Administration (FDA), Silver Spring, MD, United States

Rewriting the Global Evidence Playbook: China’s Emergence as a Strategic Hub for Evidence Generation

Session Type: Issue Panel

Topics: Real World Data & Information Systems, Health Policy & Regulatory, Health Technology Assessment

Track: Access and Drug Pricing

Level: Intermediate

ISSUE: China’s biopharmaceutical ecosystem is moving from “in China for China” to a bidirectional evidence model linking China and ex-China decisions. China’s stronger science, faster R&D, and growing pipeline are driving multinationals to source Phase 1/2 assets from Chinese innovators, increasing demand for China-generated clinical data to support global development (“in China for ex-China”). When trial design, execution, and quality align with global standards, these data are increasingly portable, enabling earlier development, indication expansion, and post-approval commitments. China real-world evidence (RWE), however, is not yet broadly transferable for ex-China payer/HTA use. Gaps in data provenance, coding, care pathways, representativeness, and governance limit comparability. This clinical-versus-RWE split raises practical questions about what China evidence can support globally today, and what must change for RWE to contribute tomorrow. Rare disease is a key proving ground. Diagnostic uncertainty, thin epidemiology, and heterogeneous care are major access and value barriers worldwide. In China, uneven diagnosis and limited natural-history data can deepen uncertainty; yet lower-cost, high-speed study infrastructure may enable faster trials and natural-history cohorts that strengthen both China filings and ex-China indication expansion. The landscape demands a new evidence playbook. OVERVIEW: Presentations (~15 minutes each) will cover: • Why China clinical evidence is becoming globally acceptable and how to make trials exportable. • Why China RWE remains limited for ex-China use and what advances could narrow the gap. • Bidirectional frameworks for using ex-China evidence in China and China evidence ex-China, including hybrid designs. • Rare disease methods to reduce diagnostic/epidemiologic uncertainty and enable indication expansion. The panel will conclude with audience debate to identify principles and guardrails for integrating China and global evidence streams to improve patient access.

Moderator

Lung-I Cheng, PhD
AESARA, Somerville, MA, United States

Speakers

Sheng Feng
LinkDoc Technology, Beijing, China
Kasey Fu, DrPh
Vertex, Boston, MA, United States
Hui Huang, PhD
CSD Partners, Boston, MA, United States

Multi-Agent AI and Interoperability: Advancing Reliable, Transparent Evidence Generation in HEOR

Session Type: Issue Panel

Topics: Methodological & Statistical Research

Track: AI

Level: Intermediate

Issue: Retrieval-Augmented Generation (RAG) is rapidly becoming a cornerstone for grounding evidence in HEOR workflows. By anchoring outputs in sourced literature and real-world data, RAG ensures that evidence generation is both transparent and traceable. Multi-agent architectures build on this foundation by orchestrating specialized agents to collaboratively process complex HEOR tasks, in which each agent has distinct roles such as document retrieval, synthesis, validation, and communication. This modular approach allows for consistency and reliability across the entire evidence generation pipeline, as each agent can leverage RAG to verify information and maintain a clear chain of reasoning. As HEOR processes scale, interoperability emerges as a critical enabler. Seamless communication among diverse agents and platforms ensures that data, context, and reasoning can be exchanged without loss of fidelity or meaning. The Model Context Protocol (MCP) addresses this challenge by offering a standardized framework for sharing contextual information, evidence, and logical reasoning among systems. MCP supports reproducibility, facilitates integration with regulatory workflows. This panel will present a comprehensive overview of how multi-agent architectures, RAG, and MCP have the potential to reshape the landscape of evidence generation in HEOR. System designers will share practical insights on interoperable, modular AI-driven solutions, while an HTA representative will discuss expectations around transparency, governance, validation, and responsible AI use. Attendees will gain an understanding of the opportunities and governance perspectives for deploying these advanced architectures. Overview: (5 min) Bill Malcolm: Introduction to multi-agent AI, interoperability, and MCP in HEOR/HTA. (10 min) Rajdeep Kaur: Agentic AI and RAG: how agentic systems interact with RAG for evidence retrieval, grounding, and workflow integration. (15 min) Sven L Klijn: Multi-agent architectures: designs, coordination, and interoperability challenges. (15 min) Daniel Ollendorf : HTA perspective: expectations for interoperable, agentic systems. (15 min) Audience Q&A

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, PhD
Pharmacoevidence Pvt. Ltd., Mohali, India
Dr. Rajdeep Kaur is the Lead of AI Sciences at Pharmacoevidence, with a Ph.D. in Computer Science and Engineering and 17+ years of expertise in advanced technologies. Her work focuses on Generative AI, machine learning, and cloud-enabled data systems, with a strong emphasis on real-world healthcare applications. She has successfully led multiple GenAI projects, combining deep technical expertise to deliver impactful AI-driven solutions.
Sven L Klijn, MSc
Bristol Myers Squibb, Utrecht, Netherlands
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.
Dan Ollendorf, MPH, PhD
Institute for Clinical & Economic Review, Boston, MA, United States

Advancing Patient-Centered Health Technology Assessment to Strengthen Equity and Transparency in Latin American Health Systems

Session Type: Issue Panel

Topics: Health Technology Assessment, Patient-Centered Research, Real World Data & Information Systems

Track: Real-World Evidence (RWE)

Level: Introductory

ISSUE: Over the past four decades, Latin America (LATAM) health systems have increasingly adopted participatory governance, reflecting broader democratic transition. In parallel, patient-centricity has become a core principle in research, value assessment and health technology assessment (HTA). Several HTA bodies in LATAM now formally acknowledge patient and citizen involvement (PCI) as essential to transparent, equitable, and legitimate decision-making. Yet meaningful implementation remains inconsistent and often disconnected from internationally used evidence-informed frameworks. The session will explore main successes and challenges in LATAM, how the region can learn from international good practices and frameworks, including those used in Canada and Europe, adapting to its local context considering governance capacity, patient heterogeneity, and structural inequities. We will also discuss how the global community can learn from LATAM experiences and successes. OVERVIEW: Ramiro Gilardino (5 min) will contextualize the global evolution of patient involvement in HTA, highlighting how lived experience, real-world evidence, and co-creation increasingly influence value frameworks and reimbursement decisions. Aline Silva (15 min) will present the current state of PCI in LATAM HTA, including including successes, persistent gaps, sustainability challenges, and recommendations based on global good practices/frameworks and learnings from a five-year implementation of a framework to improve PCI in Brazil’s HTA system. Helaine Capucho (15 min) will offer an industry perspective, reflecting on opportunities and limitations for integrating PCI across public and private systems in Brazil, and identifying enablers such as capacity-building, predictable processes, and transparent governance. Veronica Gousset (15 min) will share methodological approaches to planning, evaluating, and reporting the impact of patient involvement, focusing on how HTA agencies and stakeholders can measure value, reduce burden, and ensure equitable participation. A 10-minute Q&A will engage multiple stakeholders interested in improving patient-centricity and HTA.

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.

Speakers

Aline Silva Silveira, PhD
Brasila, Brazil
Veronica Lopez Gousset, MPH
Harvard T.H. Chan School of Public Health, Paris, France
Helaine Capucho, PhD
Interfarma, São Paulo, Brazil

8:30 AM - 10:00 AM

Networking Breakfast Bites (Exhibit Hall)

Session Type: General Meeting

8:30 AM - 11:30 AM

Exhibit Hall Hours

Session Type: General Meeting

9:00 AM - 11:30 AM

Poster Session 5

Session Type: General Meeting

Presenters will be with their posters from 9:00AM–10:00AM

10:00 AM - 11:00 AM

From Debate to Dialogue: Building Consensus on Prior Authorization Reform

Session Type: Issue Panel

Topics: Health Policy & Regulatory, Health Service Delivery & Process of Care

Track: Access and Drug Pricing

Level: Introductory

ISSUE: Prior authorization (PA) requires a care provider to obtain a health plan’s approval for a treatment or service before the plan will provide coverage. PA has become a prevalent component of healthcare benefit design, used to contain costs and manage prescription drug use. However, as PA has expanded, it has become a source of frustration across healthcare stakeholders. Policy reform activity has recently accelerated, but may be missing the mark. Payers acknowledge PA limitations but say it is an essential tool for ensuring clinically appropriate care and maintaining affordability. Patients report delays and disruptions in access, while clinicians experience administrative burden in navigating complex rules and appeals processes. Researchers have analyzed the implementation and impact of PA, including instances where restrictions are inconsistent with clinical guidelines. As PA reform activity intensifies, there is ongoing debate over how it should be scoped and executed. Payers seek to preserve mechanisms that promote appropriate care and contain costs, while patients and clinicians underscore the access barriers and harms created by current practices. Researchers seek to provide evidence to inform the discussions. This session will frame the current debate by examining trends in PA implementation over time and its impact on healthcare delivery and patient care from multiple stakeholder perspectives. It will highlight PA technology opportunities, ongoing reform efforts and aim to identify areas of common ground among stakeholders to support the development of more transparent and standardized approaches. OVERVIEW: Westrich will review the evolution of UM/PA and recent policy reform efforts (10 min) Chambers, Tsiao, and Hyde will share their perspectives on the application of PA in benefit design and patient care and how it can be improved (10 min each; see panelist perspective section for details). Moderated Q&A will explore common ground for meaningful reform and address audience questions (20 mins).

Moderator

Kimberly Westrich, MA
National Pharmaceutical Council, Herndon, VA, United States
Kimberly Westrich, MA, is the Chief Strategy Officer of the National Pharmaceutical Council (NPC), which sponsors and conducts research on health policy issues related to the development and use of innovative biopharmaceuticals to improve the health of patients. NPC’s research contributes to the body of evidence that supports discussions and decisions about patient access to treatments, appropriate use, and the value innovative treatments provide to both patients and the healthcare system. Ms. Westrich provides strategic guidance to NPC’s policy research and communications activities. She leads several research initiatives across NPC’s portfolio, including employer-sponsored insurance. She has published extensively on issues related to value assessment frameworks, patient-centered formulary and benefit design, value-based contracts, quality performance measurement, and accountable care organizations. Additionally, Ms. Westrich leads NPC’s workplace culture initiative — most notably demonstrated by our company's recognition as a Certified Great Place To Work®. Ms. Westrich began her NPC career in 2000. She has also served as Director of Research at the Pharmaceutical Research and Manufacturers of America (PhRMA), worked as a healthcare consultant at The Lewin Group and Xcenda, and as a health economics and outcomes researcher at Johnson & Johnson. Ms. Westrich is a certified yoga teacher and life coach with a passion for helping others learn and thrive. She received her master’s degree in economics from Northwestern University and her undergraduate degree in economics and mathematics from the College of William and Mary.

Speakers

James Chambers, MSc, PhD
Tufts Medical Center, Boston, MA, United States
Anna Hyde, MA
Arthritis Foundation, Silver Spring, MD, United States
Emily G Tsiao, PharmD
Premera Blue Cross, Federal Way, WA, United States

Beyond Black Boxes: Case Studies of Transparent, Validated LLM Workflows for Accelerating Global HTA Submissions and Decisions

Session Type: Spotlight

Topics: Health Technology Assessment, Methodological & Statistical Research, Real World Data & Information Systems

Track: AI

Level: Intermediate

Validation of AI/ML, LLM-enabled automation and model outputs cannot be one-size-fits-all; instead, it must be tailored to each application's evidentiary role and downstream decision-making impact. For clinical data extraction, automation workflows, and health economic model parameterization, this requires transparent, auditable frameworks with clearly defined gold standards, robust sampling strategies, and quality assurance pipelines. For higher-order applications like network meta-analyses and physician insight synthesis, validation must address both the accuracy of extracted source data and the reliability of model-synthesized conclusions. Key issues include managing heterogeneity in model behavior across settings, ensuring privacy and clinical oversight at scale, and communicating uncertainty introduced by model choices. While scientific consensus on a validation framework may not have been achieved yet, we can review case studies of recent frameworks applied in practice. Strengthening these validation practices will be essential to unlock the efficiency gains of LLM-enabled evidence generation while maintaining scientific rigor and enabling equitable, evidence-based decision-making globally. Dr. Devine opens the session, providing an overview and setting the context (8 minutes). Dr. Adamson introduces the application of LLMs for extraction of unstructured EHR data and physician insights and the VALID framework for assessing accuracy and reliability for decision making, providing a case study in disease progression dates in multiple countries. Mr. Reason demonstrates how validated LLM-generated data can be integrated into AI workflows for network meta-analysis, highlighting implications for transparency, reproducibility and HTA use. Finally, Mr. Malcolm outlines how AI-enabled automation has reduced HTA submission burden, particularly in LMIC contexts, and how automated evidence assembly can therefore accelerate submissions while maintaining data quality standards (12 minutes). Dr. Devine will facilitate an engaging discussion with the audience involving audience participation (15 min).

Moderator

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

Speakers

Blythe Adamson, MPH, PhD
Flatiron Health, New York, NY, United States
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.
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.

Changing Policies in the United States and the Return of Pediatric Vaccine Preventable Diseases

Session Type: Issue Panel

Topics: Epidemiology & Public Health, Health Policy & Regulatory, Economic Evaluation

Track: Expanded Value Measures

Level: Intermediate

Issue: Despite the safety and effectiveness of childhood vaccines, vaccine uptake is under threat due to hesitancy, acceptance, and politics. With the recent changes in personnel on the Advisory Committee on Immunization Practices (ACIP) and their active discussions about changing the recommended pediatric dosing schedule, along with ongoing outbreaks of pertussis and measles, the United States is at a crossroads. In fact, measles ‘elimination status’ is at risk or has been lost in all North American countries, so preparing for and managing endemic circulation of these pathogens is vital. Historically, mathematical modeling of infectious diseases has been a vital tool in predicting and controlling the spread of these pathogens. Effective communication strategies of these modeling efforts to inform policy makers and medical professionals is critical. Overview: Managing ongoing outbreaks of vaccine preventable disease (VPDs) and communicating strategies to mitigate their effects will be of utmost importance. Healthcare has changed since these pathogens last circulated endemically in North America and strategies for control and elimination have changed in the interim. Since it has been decades since these VPDs were endemic in North America, most scientists here are inexperienced with understanding and managing ongoing VPD outbreaks. To address this potential gap in guidance, scientific input from researchers who are experienced in elimination efforts in countries where these pathogens have remained endemic in tandem with disease modeling may help in informing policy decisions and communicating these results to stakeholders.

Moderator

Ruthie Birger, MS, PhD
Merck Inc. and Co., Rahway, NJ, United States

Speakers

Kevin Bakker
Merck & Co., Inc. - North Wales, PA, North Wales, PA, United States
Matt Ferrari, MS, PhD
Pennsylvania State University, State College, PA, United States

Amplifying Patient Voice: AI-Driven Narrative Analysis in Clinical Trials

Session Type: Issue Panel

Topics: Patient-Centered Research, Methodological & Statistical Research, Clinical Outcomes

Track: AI

Level: Advanced

ISSUE: Traditional Patient-Reported Outcome (PRO) instruments often miss the nuance of lived patient experience. Free-text entries and direct patient voice offer richer insights but have been underused due to analytical complexity and scalability challenges. Advances in AI—particularly large language models (LLMs) and conversational AI—now enable scalable capture, interpretation, and communication of patient narratives. Conversational agents provide interactive dialogue that elicits deeper context, reduces missingness, and generates structured summaries for clinicians, regulators, and decision-makers. This panel will examine how AI amplifies patient voice across development phases, improves interpretability for diverse stakeholders, and addresses risks such as bias, hallucination control, transparency, and regulatory validation. OVERVIEW: AI-enabled free-text analysis and direct patient voice complement structured PRO data, enabling scalable extraction of symptom narratives, patient priorities, and quality-of-life context that traditional instruments may miss. Practical frameworks for operationalization, validation, and regulatory acceptance of LLM-assisted analysis are essential for integrating narrative responses within eCOA platforms. This session offers an integrated view from real-world use, methodological standards, and future applications. Talk 1: Case study of LLM-based extraction and coding of an open-ended patient preference measure, covering workflow, timelines, quality checks, and integration into evidence packages. Talk 2: Methodological rigor—model performance metrics, bias detection, construct validity, version control, human review, and reproducibility—using oncology symptom capture examples. Talk 3: Conversational AI for dynamic engagement, reduced missingness, and richer lived-experience capture, alongside safety, hallucination control, and privacy considerations. Each talk runs 12 minutes, followed by 20 minutes of audience interaction. Attendees will gain actionable frameworks, validated benchmarks, and practical guidance to responsibly integrate AI-driven narrative analysis in PRO/eCOA research.

Moderator

Denise Globe, PhD
Gilead Sciences, Foster City, CA, United States
Denise Globe PhD is currently the Head of the GHEOR and the Global Value and Access Center of Excellence at Gilead. She has 30 years of experience in health care with a focus on quantitative policy research and direct research experiences in health economics research including the outcomes, process, financing and delivery of care. She leads a team at Gilead that is accountable for global observational research, evidence for access strategy and execution for the oncology, virology, liver and inflammation portfolios to maximize reimbursement and access across the life cycle.

Speakers

SAEID SHAHRAZ
Gilead Sciences, Mountain View, CA, United States
Saeid Shahraz, MD, PhD, is a physician and measurement scientist with over 20 years of experience in health outcomes research. He leads clinical outcome assessment strategy and analytics as Director of Health Economics and Outcomes Research. With expertise in psychometrics, COA validation, and real-world data analysis, Dr. Shahraz supports evidence generation for clinical trials and regulatory submissions. His work bridges scientific rigor and patient-centered measurement to enhance the value and reliability of health outcomes data.
Yuelin Li, PhD
Memorial Sloan Kettering Cancer Center, New York, NY, United States
Bill Byrom, PhD
Signant Health, Nottingham, United Kingdom

Is It Worth Building Disease-Specific Patient Reported Outcomes (DSPRO) Value Propositions for Orphan and Innovative Therapies? Bridging Economic Models and Patient Lived Experience

Session Type: Other Breakout Session

Topics: Patient-Centered Research, Clinical Outcomes, Health Technology Assessment

Track: Expanded Value Measures

Level: Intermediate

Purpose: This breakout session will create a shared space for both US and global patients, caregivers, payers, regulators, clinicians, and industry perspectives to openly discuss when does investing in DSPROs Value Propositions actually change conversations, decisions, and experiences and when does it mostly add complexity and cost? By the end of the session, participants will be able to: • Compare the pros and cons of DSPROs vs generic measures for valuing orphan/innovative therapies. • Discuss ROI for companies and “return on burden/benefit” for patients and families. • Pinpoint when DSPROs are truly “must-have” versus “nice-to-have” in real-world decisions. Description: This session will begin with a brief framing presentation (10 minutes) to outline how orphan and innovative therapies are currently evaluated, highlight where generic measures seem to miss what matters most to patients and caregivers when developing a value proposition. Next, three brief talks (~10 minutes each) will examine DSPROs from different perspectives: • Steve brings 15+ years of experience in industry HEOR perspective currently representing Ionis Pharmaceutical talks on when DSPROs strengthen (or fail to strengthen) value and “return on investment” • Angie brings 30+ years of experience as global lead for patient-led musculoskeletal health advocacy patient advocacy and EUPATI Fellow, she talks about how DSPROs can amplify or miss lived experience, including “return on burden” • Nora brings 10+ years of conducting and disseminating research on child health outcomes at Queens University and gives an academic perspective on how DSPROs show up in real-world decision making, including what level of outcome evidence feels meaningful versus excessive in practice. The session will conclude with a moderated, interactive discussion and live polling (20 minutes), using case scenarios and a simple discussion guide to help attendees explore when investing in DSPROs adds meaningful value for stakeholders and when existing approaches may be sufficient in both US and global settings.

Moderator

Nathan T Jibat, MBA, MPH, MD
Johns Hopkins University, Baltimore, MD, United States

Speakers

Steven Kymes, MHA, PhD
Ionis Pharmaceuticals, Carlsbad, CA, United States
Nora Fayed, PhD
Queen's University, Kingston, ON, Canada
Angie Botto-van Bemden, PhD
Musculoskeletal Research International, Holiday, FL, United States

Beyond Commercially Available Real-World Data: Harnessing Public Data for HEOR Research in the US and Globally

Session Type: Workshop

Topics: Real World Data & Information Systems, Study Approaches, Economic Evaluation

Track: Real-World Evidence (RWE)

Level: Introductory

Purpose: Commercial datasets are widely used in health economics and outcomes research studies (HEOR); however, public data sources offer advantages in transparency, accessibility, and population representativeness. This session will highlight the value of public data for HEOR research, focusing on access mechanisms, data coverage, and depth of available information, and how these resources can complement or substitute for proprietary datasets. Description: This session will begin with an overview of major public data sources globally (10 minutes), including sources from the United States (e.g., Medical Expenditure Panel Survey [MEPS]), Europe (e.g., Clinical Practice Research Datalink [CPRD]), Asia (e.g., Taiwan’s National Health Insurance Research Database [NHIRD]), and South America (e.g., DADASUS). The presentation will address database selection including trade-offs between commercial and public data and will consider timeliness, access requirements, cost, representativeness, depth of information, and linkage potential. Next, workshop facilitators will present three real-world case studies (10 minutes each) that illustrate how public data can be applied to answer HEOR research questions. Lia Pizzicato will present a U.S. example using MEPS on obesity; Cameron Cook will describe a study on cardiovascular disease using CPRD in the United Kingdom; and Fang-Ju Lin will outline a use case in oncology leveraging Taiwan’s NHIRD, cancer registry, and mortality data. Each case study will highlight practical considerations, strengths, and limitations encountered when working with public datasets. The session will conclude with an interactive component (20 minutes), beginning with a 10-minute facilitated discussion on the strengths and limitations of public data sources. In the final 10 minutes, facilitators will pose hypothetical HEOR research questions (e.g., burden of illness, treatment pattern evaluation, outcome comparisons), inviting the audience to propose suitable data sources. Through this discussion, participants will apply key concepts from the workshop and practice selecting datasets that are fit for purpose across various research scenarios.

Moderator

C. Daniel Mullins, PhD
University of Maryland School of Medicine, Baltimore, MD, United States
C. Daniel Mullins is a Professor at the University of Maryland School of Pharmacy. He is Founder and Executive Director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, a community-academic partnership for patient-driven research. Dr. Mullins has received approximately $25 million in funding as a Principal Investigator from AHRQ, FDA, NCI, NHLBI, NIA, NIMHD, the Patient-Centered Outcomes Research Institute (PCORI) and various patient advocacy organizations and pharmaceutical companies. At the University of Maryland Baltimore (UMB), he received the Dr. Patricia Sokolove Outstanding Mentor Award and the Dr. Martin Luther King Jr. Faculty Diversity Award. He was named Researcher of the Year at UMB and was awarded a University System of Maryland Wilson H. Elkins Professorship. At ISPOR, he has served as Editor-in-Chief of Value in Health since 2010 and received the Marilyn Dix Smith Leadership Award in 2017 and the Avedis Donabedian Lifetime Achievement Award in 2024.

Speakers

Cameron Cook, BA, MA, MS, PhD
AMGEN, Durham, NC, United States
Fang-Ju (Irene) Lin, RPh, PhD
National Taiwan University, Taipei City, Taiwan
Lia N Pizzicato, MPH
IQVIA, Rochester, NY, United States

From Engagement to Influence: What Should Patient-Centered Economic Models Actually Look Like?

Session Type: Issue Panel

Topics: Economic Evaluation, Patient-Centered Research, Health Technology Assessment

Track: Expanded Value Measures

Level: Intermediate

Patient engagement in value assessment has advanced considerably, yet its integration into economic modeling remains the exception rather than the rule. Most economic models continue to rely on clinician-driven assumptions, generic utility instruments, and representations of disease that may not accurately reflect patients lived experience and perspectives. While some examples demonstrate that engagement can shape model structure, outcome selection, and interpretation, the field lacks agreement on what meaningful patient-centered modeling should look like, and whether this evolution strengthens or threatens methodological rigor. This panel will debate the central issue: Should the lived experience of patients play a greater role in shaping, driving and influencing the analytical components of economic models, or should them remain advisory? If patient involvement is to extend beyond reviewing lay summaries, fundamental questions must be addressed. These include: How should patient-identified outcomes be incorporated when they fall outside the traditional cost per quality adjusted life year (QALY) framework? When does lived experience justify revising structural assumptions? How might patient-informed lived experiences and perspectives change cost-effectiveness ratios, equity considerations, and uncertainty analyses? And what safeguards are needed to avoid introducing bias, double-counting, or scope creep? Building on recent research, panelists will offer contrasting perspectives on the promise and pitfalls of deeper engagement: the modeler’s need for parsimony and internal validity, the patient partner’s demand for relevance and representation, and the decision-maker’s need for credible and transparent evidence. The session aims to surface tensions, clarify boundaries, and highlight practical, scalable ways to embed patient insights throughout the modeling lifecycle. Ultimately, this panel asks: if future economic models are co-created with patients, how should this be operationalized in practice? What principles, safeguards, and methodological approaches are needed to integrate patient-generated inputs while maintaining transparency, rigor, and fairness?

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.

Speakers

Marina Richardson
ICER, Boston, MA, United States
Marina Richardson is an associate director, Health Technology Methods and Health Economics with the Institute for Clinical and Economic Review (ICER). In her role, Marina leads and oversees the development of economic models to inform pricing and reimbursement decision-making and identifies and executes opportunities to enhance ICER's methodology and processes within Health Technology Assessment (HTA) and Health Economics. Prior to joining ICER, Marina led and contributed to reimbursement review reports and recommendations at Canada's Drug Agency (CDA-AMC), formerly CADTH. Marina has a PhD in Health Services Research from the University of Toronto and is an active contributor to the field including as a deputy editor for the International Journal of Technology Assessment in Health Care (IJTAHC), a member of the Ontario Immunization Advisory Committee (OIAC) in Ontario, Canada, and as a co-chair of the International Scientific Program Committee for Health Technology Assessment International (HTAi) 2025.
Eric Low
Eric Low Consulting, Haddington, United Kingdom
Julia F. Slejko, PhD
University of Maryland Baltimore, Baltimore, MD, United States

Strategic Integration of Generative AI in Health Economic Modeling: Emerging Methods, Implementation, Evaluation, and HTA Implications

Session Type: Workshop

Topics: Methodological & Statistical Research, Economic Evaluation, Health Technology Assessment

Track: AI

Level: Intermediate

PURPOSE: Advances in genAI are reshaping health economic analysis workflows, spanning academic methodological research through HTA submissions. As we increasingly engage with genAI-assisted modeling, clearer methodological guidance, performance evaluation standards, and implementation frameworks are needed. This workshop will present cross-stakeholder perspectives on: (1) recent genAI advancements and proposed frameworks for integrating agentic intelligence into economic modeling; (2) operational and methodological barriers to using genAI in industry and academic modeling workflows and the need for performance-based evaluation criteria; and (3) how HTA bodies may assess performance, transparency, and verification of genAI-assisted models. DESCRIPTION: Dr. Wang will contextualize recent advancements in genAI within HEOR and present her collaborative research on an implementation framework for integrating agentic intelligence into economic modeling (15 min). Mr. Feng will present the industry user’s perspective on integrating genAI into internal workflows, including how genAI-assisted health economic models should be evaluated on their performance rather than on their ability to replicate human-built models. He will discuss key barriers that limit modelers’ adoption of genAI tools in practice and will call for an initiative to establish a formal evaluation framework for genAI-assisted economic modeling (10 min). Dr. Wright will discuss the HTA perspective, highlighting stages where genAI-assisted approaches are likely to be most useful in evidence synthesis and modeling. She will also address the importance of transparency and validation in these approaches (10 min). Dr. Li will synthesize the discussion and present the academic user’s perspective, including practical concerns about the cost and access of specialized genAI tools, and methodological considerations related to ensuring comprehensive literature capture, transparent workflows, and reproducible modeling results. (10 min). Audience engagement will include polling questions and Q&A (15 min).

Moderator

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

Speakers

Xiaoyan Wang, PhD
Tulane University, New Orleans, LA, United States
Haidong Feng, MPH, MS
Merck, Boston, MA, United States
Abigail Wright
Institute for Clinical and Economic Review, Boston, MA, United States

11:00 AM - 11:30 AM

Break (Exhibit Hall)

Session Type: General Meeting

11:30 AM - 1:00 PM

Innovation Under Pressure: How Will U.S. Drug Policy Reshape Innovation, Evidence, and Access Globally?

Session Type: Plenary

Topics: Health Policy & Regulatory, Economic Evaluation, Health Technology Assessment

Track: Access and Drug Pricing

Level: Advanced

To say that we currently live in a VUCA (Volatile, Uncertain, Chaotic, Ambiguous) world when it comes to trade and policy would be an understatement. Global biopharmaceutical innovation is entering a pivotal decade, driven in large part by sweeping U.S. policy reforms that extend far beyond domestic borders. Building off the first plenary on U.S. Most Favored Nation and Medicare Drug Price Negotiation policies, this plenary will focus on the implications outside of the US.

With constrained budgets, trade policy influencing cost effectiveness thresholds, and reference pricing shaping launch decisions, the “global access ecosystem” is undergoing significant disruption. Drawing on perspectives from global HEOR, payer policy, and industry experts, this session will address the following questions: What will be the impact on development decisions, evidence generation, pricing corridors, launch strategies, and long run incentives to invest in innovation?

The session will conclude with a forward-looking discussion on how health systems can sustain meaningful rewards for innovation and adaptations that are necessary for innovators and the evidence generation community to remain resilient amidst change.

Moderator

Indranil Bagchi, MS, PhD
GSK US, Collegeville, PA, United States
Dr. Indranil Bagchi is the Global Head of Pricing & Market Access at GSK. In this role, Indranil drives the overall strategy on value demonstration and market access across all product areas in the GSK portfolio, to secure access to medicines for our patients and to maximize the value of our portfolio. Indranil has more than two decades of experience in market access across several major companies in the pharmaceutical industry. In 2014, Indranil received the ‘Outstanding 50 Asian Americans in Business’ award and in 2010, Indranil was recognized in Pharmaceutical Executive magazine’s annual roster of Emerging Leaders, “The New Breed of Leadership.” Indranil is a frequent speaker and contributor to forums, articles and conferences addressing issues related to access to medicines. Prior to GSK, Indranil was Senior Vice President and Worldwide Head of Value and Access at Novartis Oncology. Previously at Pfizer, he was Vice President and Global Head of Payer Insights and Access and prior to that, he was with GlaxoSmithKline in Health Economics and Outcomes Research. Dr. Bagchi has an undergraduate degree in Pharmacy, a master’s degree in Pharmacy and Healthcare Administration and a doctoral degree in Pharmaceutical Socioeconomics.

Speakers

Lotte Steuten, MSc, PhD
Office of Health Economics, London, United Kingdom
Darius Lakdawalla, PhD
University of Southern California, Los Angeles, CA, United States
Darius Lakdawalla is a widely published, award-winning researcher and a leading authority on health economics and health policy. He holds the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation at the University of Southern California, where he sits on the faculties of the School of Pharmacy, the Sol Price School of Public Policy, and the Leonard D. Schaeffer Center for Health Policy and Economics, one of the nation’s premier health policy research centers.

His academic research has focused primarily on the economics of risks to health, the value and determinants of medical innovation, the economics of health insurance markets, and the industrial organization of healthcare markets. Dr. Lakdawalla serves as associate editor at the Journal of Health Economics and has previously served in this role at the American Journal of Health Economics and the Review of Economics and Statistics. His academic work has appeared in leading peer-reviewed journals of economics, health policy, and medicine, including the American Economic Review, Quarterly Journal of Economics, Health Affairs, the Journal of Health Economics, and the New England Journal of Medicine. In addition, his work has been featured by prominent popular press outlets, such as the Wall Street Journal, National Public Radio, Forbes, and the New York Times. Dr. Lakdawalla has also received the PhRMA Foundation Value Assessment Challenge Award, designed to encourage innovative approaches to defining and measuring value in health care, in 2019 (third place) and 2020 (first place), along with the ISPOR Excellence in Research Methodology Award, the Garfield Prize, and the Milken Institute Award for Distinguished Economic Research.
Jens Grueger, PhD
Boston Consulting Group, Freiburg, Germany
Jens Grueger is a Senior Advisor at the Boston Consulting Group advising life sciences companies on pricing and market access, and a Senior Advisor at Curta, focusing on evidence, value, access and pricing. He is an Affiliate Professor of Health Economics at the CHOICE Institute, University of Washington School of Pharmacy and ISPOR’s 2020-2021 President. Previously, Jens had a distinguished career in the pharmaceutical industry. He was Senior Vice President and Head of Global Access for F. Hoffmann-La Roche (2011-2019). Jens was Vice President and Head of Global Market Access Primary Care at Pfizer (2009-2011), Head of Global Pricing & Health Economics at Novartis (1999-2009), and Director of Health Economics at the German affiliate of SmithKline Beecham Pharma (1994-1997). He founded Diversified Health Systems, an internet-based disease management services start-up (1997-1999).

ISPOR 2026

Sunday, May 17 - Wednesday, May 20

Program

ISPOR–The Professional Society for
Health Economics and Outcomes Research