ISPOR 2025: Exploring sessions on health technology assessment

With the EU HTA Regulation now in effect and the Inflation Reduction Act (IRA) reshaping US drug pricing, ISPOR 2025, the leading conference from ISPOR—The Professional Society for Health Economics and Outcomes Research, arrives at a critical time for global health policy and evidence generation. Across the 3 days of the conference, taking place May 13–16, in Montreal, Canada , experts will explore how evolving health technology assessment (HTA) methodologies and practices are influencing value assessment and market access strategies.

Selected sessions

From the introduction of joint clinical assessments (JCAs) in Europe to the growing recognition of caregiver burden in value frameworks, and the methodological complexities of indirect treatment comparisons, ISPOR 2025 will cover both foundational shifts and emerging challenges in HTA.

Global Impact of JCA: Evaluating the Implications of the New EU HTA Regulation

Date and time: May 14, 10:15am – 11:15am
Moderator: Danny Yeh (Aesara)
Speakers: Anouchka Vidal (Roche Products Ltd), Brian O'Rourke (Brian O'Rourke Health Care Consulting, Inc.), Federico Augustovski (Institute for Clinical Effectiveness and Health Policy)

The European Union's Health Technology Assessment Regulation (EU HTAR), effective from January 2025, introduces a harmonized framework for joint clinical assessments (JCAs) of new medicines and selected high-risk devices, aiming to standardize evaluations and improve patient access. This workshop will explore how the JCA is influencing HTA practices beyond Europe, drawing on insights from industry, the HTA community, and international public health agencies to discuss emerging challenges, opportunities, and cross-border collaboration in established and developing non-EU markets.

Lost in Translation: Disseminating Family Spillover Effects on Value and Affordability Assessment Decision Makers

Date and time: May 14, 10:15am – 11:15am
Moderator: Robert B McQueen (University of Colorado Skaggs School of Pharmacy and Pharmaceutical Science)
Speakers: Jason Resendez (National Alliance for Caregiving), Stacey Kowal (Genentech), Andrew York (Maryland Prescription Drug Affordability Board)

With a growing recognition of the impact of diseases and treatments on family members and informal caregivers is reshaping value and affordability assessments, this workshop will explore approaches for collecting, analyzing, and communicating evidence on family spillover effects to policymakers and HTA bodies. Speakers will share examples from Institute for Clinical and Economic Review (ICER), prescription drug affordability board (PDABs), advocacy efforts, and disease-specific research, and will engage attendees in a discussion on how best to integrate family spillover effects into policy and value frameworks.

What Causal Inference Teaches Us About the Limitations of Indirect Treatment Comparisons for Health Technology Assessment

Date and time: May 14, 10:15am – 11:15am
Moderator: Uwe Siebert (UMIT TIROL - University for Health Sciences and Technology; Harvard Chan School of Public Health)
Speakers: Arthur Chatton (Université Laval), Michael Webster-Clark (McGill University), Harlan Campbell (University of British Columbia)

This workshop will introduce key principles of causal inference and treatment effect heterogeneity to help attendees critically assess indirect treatment comparisons (ITCs) in HTA. Participants will explore why ITCs are considered, "essentially observational findings across trials," and learn strategies to better understand and evaluate the limitations and validity of ITCs when direct evidence is lacking.

Enhancing HTA: Surrogate Endpoints, Statistical Standards, and Their Impact on Patients

Date and time: May 14, 11:45am - 12:45pm
Moderator: Bart Heeg (Cytel)
Speakers: Dawn Lee (University of Exeter Medical School), Kristian Thorlund (McMaster University), Stephen D Stefani (UNIMED/RS)

As HTA systems seek faster yet reliable evidence, this session will explore emerging statistical methods for evaluating surrogate endpoints and their impact on decision-making. Attendees will gain early insights from the ISPOR Statistical Evaluation of Surrogate Endpoints for Health Technology Assessment (HTA) Decision-Making Task Force, including strategies to mitigate uncertainty and improve modeling, ultimately aiming to accelerate patient access while maintaining scientific rigor in regulatory and reimbursement evaluations.

Collecting Actionable Evidence on the Full Range of Economic Impacts From Patients and Caregivers: Advancing “Whole Person” HTA

Date and time: May 14, 1:45pm – 2:45pm
Moderator: Ushma Patel (Center for Innovation & Value Research)
Speakers: Elisabeth Oehrlein (Applied Patient Experience), Tina Aswani Omprakash (South Asian IBD Alliance), Stacey Kowal (Genentech)

This interactive session will introduce HEOR professionals to the Patient-Centered Economic Impacts Research Framework, emphasizing methods like patient experience mapping to capture the full financial impact of disease on patients and caregivers. Attendees will explore how to integrate these insights into holistic HTA, advancing more inclusive and meaningful value assessments that better reflect real-world patient and caregiver experiences.

Bringing Us Together or Pushing Us Apart: Will JCA, HEMA, and Other Cross-Border Collaboration Initiatives Improve Patient Access?

Date and time: May 14, 3:15pm – 4:15pm
Moderator: Erika Wissinger (Cencora)
Speakers: Michael Drummond (University of York), Eldon Spackman (O'Brien Institute for Public Health, University of Calgary), Jon Campbell (National Pharmaceutical Council)

With the EU HTAR now in force, coordinated HTA is entering a new era. This session will explore how cross-border initiatives, such as JCA, Joint Nordic HTA Bodies (JNHB) from Denmark, Finland, Iceland, Norway, and Sweden (formerly the FINOSE collaboration), and the Health Economics Methods Advisory (HEMA), are shaping global collaboration, improving efficiency, and potentially accelerating patient access. Panelists will assess the feasibility of deeper international HTA alignment, discuss its implications for health technology developers, and offer practical guidance on navigating diverse country needs while maximizing opportunities in an increasingly interconnected HTA landscape.

Challenges in the Implementation of Generalized Cost-Effectiveness Analysis (GCEA): Debating a Path Forward

Date and time: May 14, 5:00pm – 6:00pm
Moderator: Meng Li (Tufts Medical Center, The Center for the Evaluation of Value and Risk in Health)
Speakers: Jason Shafrin (FTI Consulting), Darius Lakdawalla (University of Southern California), Melanie D Whittington (Leerink Center for Pharmacoeconomics)

A newly published user guide on generalized cost-effectiveness analysis (GCEA) offers methods to quantify broader societal value elements in HTA, but debate continues around their feasibility, fairness, and policy implications. This session will explore how GCEA could reshape US pricing and coverage decisions, weighing its potential benefits against challenges like double counting, threshold ambiguity, and implementation complexity. Panelists will debate whether incorporating societal values through GCEA is a practical next step or an aspirational ideal requiring further refinement.

Introducing HEMA: An International Working Group for Principled Assessment of Novel HTA Methods and Processes

Date and time: May 14, 5:00pm – 6:00pm
Moderator: Dan Ollendorf (Institute for Clinical & Economic Review)
Speakers: Zal Press (Patient Commando), Lotte Steuten (Office of Health Economics), Nicole Mittmann (Canada's Drug Agency)

Introduced to ISPOR 2025 attendees in an earlier session, the international Health Economics Methods Advisory (HEMA) is a new global initiative convened by HTA bodies to critically assess emerging health-economic methods. This workshop will offer a deeper look into HEMA’s structure, priorities, and stakeholder engagement model. Participants will gain insights into how HEMA evaluates novel approaches (such as new value elements and analytic frameworks) and how the broader HTA community can engage in shaping evidence standards moving forward.

Plenary Session: Evolution of Evidence—Innovating for the Future of HTA

Date and time: May 14, 8:30am – 9:45am
Moderator: Nicole Mittmann (Canada's Drug Agency)
Speakers: Sudha Kutty (Canada's Drug Agency), Pascale Lehoux (INESSS), Donald Husereau (University of Ottawa), Connie Cote (Health Charities Coalition of Canada)

During the second plenary of the conference, the conversation moves beyond traditional clinical and economic metrics to reflect the growing influence of broader considerations – such as patient perspectives, health equity, environmental impact, and ethical values. This session will examine what that evolution looks like in practice, both globally and within the Canadian context. Experts from HTA bodies, academia, patient groups, and payers will explore how to integrate real-world data, redefine value, and adopt a whole health approach to build more resilient and responsive HTA systems.

Is IRA Becoming America’s HTA?

Date and time: May 15, 11:45am – 12:15pm
Speakers: Priti Jhingran (Genesis Research Group), Adam Weston (Genesis Research Group)

Building on other discussions of IRA policy shifts at the ISPOR 2025, this session takes a closer look at how the US Medicare Drug Price Negotiation (MDPN) program under the IRA mirrors global HTA practices. Speakers will compare the MFP process to established HTAs, explore its impact on branded product lifecycles, and outline strategic, evidence-based approaches manufacturers must adopt to navigate this evolving US pricing landscape.

Clinical Trial Design, Beyond the Regulatory

Date and time: May 15, 3:15pm – 4:15pm
Moderator: Kati Copley-Merriman (RTI Health Solutions)
Speakers: Lynda Doward (RTI Health Solutions), Emma Hawe (LCP Health Analytics), Shannon Cope (PRECISIONheor)

This session will explore how clinical trial design can better support reimbursement, patient-centered outcomes, and economic evaluations. Speakers will discuss integrating stakeholder needs early, designing for comparative effectiveness, and incorporating patient-reported outcomes to capture real-world impact. The panel will also cover the growing use of single-arm trials, external control arms, and planning for key data inputs such as utility and efficacy estimates. Attendees will gain practical insights into designing trials that generate robust evidence for future value demonstration and reimbursement decisions.

Plenary Session: Balancing Speed and Scientific Rigor—Patient-Centered Methodologies for Surrogate Endpoints in Accelerated Access

Date and time: May 16, 11:30am – 12:45pm
Moderator: Elisabeth Oehrlein (Applied Patient Experience, LLC)
Speakers: Ebony Dashiell-Aje (BioMarin Pharmaceutical, Inc.); Chester Good (UPMC Health Plan); Yvette A Venable (AstraZeneca), Durhane Wong-Rieger (Canadian Organization for Rare Disorders)

Deepening the exploration of surrogate endpoints introduced in an earlier session, the third and final plenary will explore how these intermediate measures can be made more meaningful for regulators and HTA bodies alike. As surrogate endpoints become more common, particularly in expedited approval pathways, this session will examine methods for validating them, incorporating patient input into endpoint selection, and striking the right balance between faster access and scientific rigor in both clinical development and real-world decision-making.

Latest research in HTA

Alongside these discussions, attendees can explore a broad collection of research posters at ISPOR 2025. Selected posters being presented include:

• Evolving HTA and Potential EU HTAR Ripple Effects in the US: Insights From a Stakeholder Survey – Anouchka Vidal (Hoffmann-La Roche)
• The Institute for Clinical and Economic Review and Joint Clinical Assessment: Insights for Manufacturers in an Evolving Health Technology Assessment Landscape – Colin Song (Wickenstones)
• Real-World Evidence (RWE) in Health Technology Assessment (HTA): Global Guidelines on the Use of RWE in Evidence Generation for HTA Evaluation – Coby Martin (Axtria)
• A Standardized Framework for the Acceptance of Non-Randomized Evidence From Real-World Data in HTA: Insights From EU Guidelines and National Practices – Kathrin Bogner (AMS Advanced Medical Services GmbH)
• Are Patients With Major Depressive Disorder (MDD) Getting Access to Innovation in HTA Markets? Review of Reimbursement Decisions of Pharmacotherapies for MDD in Europe, Australia, and Canada From 2001 to 2021 – Elio Asano (Johnson & Johnson)
• Health Equity and HTA: Is Equity Data Shaping Recommendations – Derek C Xu (Trinity Life Sciences)
• Assessability of Single-Arm Studies (SAT) Under Joint Clinical Assessment (JCA): Whether Drugs Assessed With SAT in the Last Years Would Fit Under the New EU HTA Framework? – Mondher Toumi (Clever-Access)
• Reimbursement Outcomes for EMA Conditionally Authorized Orphan Drugs (2022-2023): Implications for Joint Clinical Assessment Requirements – Michael Epstein (Clinigen)
• Analysis of NICE Assessments for Orphan Treatments and Key Drivers for Decision-Making – Stephen Ralston (Coronado Research)
• Too Many PICOS for Joint Clinical Assessments (JCA)? How To Adapt The Systematic Literature Reviews for Achieving Desired Outcomes – Sangeeta Budhia (Parexel International)
• Insights and Implications: An Analysis of the Studies Undertaken by HTAIn (Health Technology Assessment in India – Varshini Neethi Mohan (IIT Madras)
• Timing of Health Technology Assessment in the United States: An Evaluation of ICER Reviews Over Seven Years – Belen Herce-Hagiwara (Institute for Clinical and Economic Review)
• An Overview of Pricing and Reimbursement Frameworks for Medicinal Products in Central and Eastern European (CEE) Countries: Mechanisms, Rules, And Managed Entry Agreements (MEAs) – Andrew Mumford (Initiate Consultancy)
• Strategic Implications of EU HTAR (JCA and JSC) for Global Biopharma and Biotechs: Navigating Evolving Paradigms in Market Access – Clement Francois (Putnam)
• Use of Real-World Evidence in HTA Submissions and Cost-Effectiveness Evaluations in Asia – Tanja Babic (Stratenym)
• Enhancing Health Technology Assessment With Data Transportability Analyses: Addressing Bias and Generalizability Challenges In Non-Local Real-World Evidence – Cal Shephard (AstraZeneca)
• How HTA Should Consider the Global Value of Health Products to Support Public Decision? – Sandrine Bourguignon (RWEality)
• Mapping of Health Technology Assessment in China: Progress and Challenges from a Nationwide Repeated Cross-Sectional Survey – Yue Suo (Fudan University)

Coverage by The Evidence Base

The Evidence Base will be providing exclusive coverage of ISPOR 2025 (May 13–15, Montreal, Canada), including daily session highlights and in-depth features on all three plenaries. Register on The Evidence Base and follow us on LinkedIn to stay informed and up to date with the latest insights and developments from the conference.