ISPOR 2025: Exploring sessions on real-world data and real-world evidence

As healthcare decision-making and evidence generation becomes more complex, the demand for real-world data (RWD) and real-world evidence (RWE) continues to grow, driven by the need to complement clinical trial results with insights from routine care and diverse populations. At ISPOR 2025 (May 13–16, Montreal, Canada), sessions will explore the evolving role of RWE across drug development, commercialization, regulatory decision-making, and health equity. Discussions will highlight practical approaches, innovative methodologies, and ongoing challenges in generating reliable, fit-for-purpose evidence for a rapidly changing global landscape.

Selected sessions

RWE Generation: Blueprint for Drug Development and Commercialization

Date and time: May 14, 11:45am – 12:15pm
Moderator: Ashley Jaksa (Aetion)
Speakers: Katherine Mues (Aetion)

In this Exhibit Hall Theater, the speakers will highlight the often-overlooked value of robust descriptive RWE in informing clinical development and commercialization. They will outline a systematic, phased approach to RWE investment, emphasizing its importance for decision-making and the challenges that arise when critical RWE studies are delayed, fragmented, or neglected in favor of more complex but less frequently accepted use cases.

ISPOR Good Practices Task Force on Patient-Reported Outcomes (PROs) in Prospective Real-World Studies: Preliminary Recommendations

Date and time: May 14, 11:45am – 12:45pm
Moderator: Konrad Maruszczyk (University of Birmingham)
Speakers: Onyeka Illoh (FDA), Jessica Roydhouse (Menzies Institute for Medical Research, University of Tasmania), Tom Keeley (GSK)

This session will share preliminary recommendations from the patient-reported outcomes PROs in Prospective Real-World Study Design Task Force on best practices for collecting PROs in real-world studies. Speakers will address key considerations including study populations, data quality, patient partnership, and analytical methods, supported by case examples. The discussion will highlight methodological and logistical challenges from research, regulatory, and industry perspectives. Attendees will be asked to provide feedback to help shape ISPOR’s expert consensus on optimizing PRO use in RWE generation.

Bridging the Evidence Gap: Integrating Real-World Data With Randomized Controlled Trials

Date and time: May 14, 1:45pm – 2:45pm
Moderator: Mary Beth Ritchey (CERobs Consulting, LLC)

Speakers: Emilie Toresson Grip (Quantify Research), Fei Fei F Liu (BMS), Kirk Geale (Quantify Research)

Bringing together global perspectives, this workshop will explore how integrating RWD with randomized trial results can strengthen evidence generation and inform regulatory decisions. Speakers will share case studies showing how this approach extends findings to broader populations and addresses gaps in traditional trials. Practical insights on external control arms, study design, and implementation will be discussed, with interactive polling inviting audience input to shape the discussion and refine recommendations.

Harnessing AI and RWE: Developing Integrated Evidence Strategies for Market Access in the IRA Era

Date and time: May 14, 3:00pm – 3:30pm
Speakers: Jacqueline Vanderpuye-Orgle (Parexel), Matthew Gordon (Parexel)

This session aims to demonstrate how integrated evidence planning (IEP), supported by RWE and AI-enabled tools, can optimize clinical development and market access—especially in light of the Inflation Reduction Act. Through case studies, attendees will explore how RWE and AI can be embedded across the product lifecycle to enhance strategy, execution, and outcomes.

What’s Really Happening with GLP-1s? Insights From AI-Enabled RWD

Date and time: May 14, 4:00pm – 4:30pm
Speaker: Ryan Ahern (Truveta)

Highlighting the power of electronic health record (EHR)-linked data, this session explores how Truveta Data is transforming drug and device research. Speakers will share findings on GLP-1 use, treatment patterns, and access barriers, while showcasing how advanced AI, claims linkage, and genomic integration enable scalable, real-world insights. Attendees will learn how this comprehensive dataset supports faster, more representative studies and opens new opportunities for precision medicine and regulatory-grade research.

Real-World Life-Cycle Evaluation for Precision Medicine: From Conceptualization to Successful Implementation

Date and time: May 14, 5:00pm – 6:00pm
Moderator: Dean Regier (BC Cancer - ARCC – UBC)
Speakers: Deirdre Weymann (BC Cancer), Emanuel Krebs (Cancer Control Research), Blythe Adamson (Flatiron Health)

This session will examine how life-cycle health technology assessment (LC-HTA) and RWE can help address uncertainties in precision medicine during regulatory and reimbursement processes. Using a Canadian case study, speakers will present methods for combining single-arm trial data with RWD to assess comparative and cost-effectiveness. The discussion will also explore the relevance and transportability of RWE for international decision-making and consider practical challenges and opportunities for integrating LC-HTA approaches into healthcare systems through audience engagement.

Cloud-First Insights Generation: How Research Teams and RWD Sources Are Collaborating for Faster, More Secure Data Discovery and Assessment

Date and time: May 15, 10:15am – 10:45am
Speakers: Arnaub Chatterjee (Datavant), Courtney Vehling (Eli Lilly & Co)

As demand for high-quality RWE grows, this session explores how cloud-first strategies are advancing RWD discovery, access, and collaboration. The speakers will demonstrate how digital solutions enhance health economics and outcomes research (HEOR) by streamlining data use, protecting privacy, and enabling deeper insights into patient journeys. Attendees will learn how cloud platforms are transforming RWD-driven research to accelerate evidence generation, support market access, and inform health policy decisions.

Unlocking the Potential of Routine Patient Health Records for RWE

Date and time: May 15, 1:00pm – 1:30pm
Speakers: Bruce Feinberg (Cardinal Health), Harlen Hays (Cardinal Health)

This Exhibit Hall Theater session will look at how to leverage routine patient health records for regulatory-compliant RWE studies. Speakers will address differences from clinical trial data, selection bias, patient inclusion, and data comparability, and will share practical guidance on applying analytics and best practices to ensure RWE quality, reliability, and regulatory readiness.

Identifying Gaps and Establishing a Development Plan for Consensus Real-World Data Standards

Date and time: May 15, 1:45pm – 2:45pm
Moderator: Mitch Higashi (ISPOR)
Speakers: Mary Beth Ritchey (CERobs Consulting, LLC), Eberechukwu Onukwugha (University of Maryland)

As the use of RWD grows in HEOR, establishing data standards has become increasingly important to ensure credibility, consistency, and regulatory acceptance. This panel will examine existing standards, highlight key gaps, and explore the complexities of applying standards across diverse data types, including claims, EHR, wearables, and AI/ML. Through discussion and polling, participants will help identify priority areas and consider collaborative approaches to advance consensus standards for RWD in healthcare research and decision-making.

How Do We Generate RWE in Rare Diseases or Targeted Subgroups? Use of Multimodal Data and Methodologies

Date and time: May 15, 5:00pm – 6:00pm
Moderator: Phani Veeranki (Optum Life Sciences)
Speakers: Amy M Duhig (Takeda), Arpita Nag (AstraZeneca), Lucinda Orsini (COMPASS Pathways)

Generating RWE for rare and targeted diseases presents unique challenges, particularly in assembling appropriate patient cohorts. This Issue Panel will explore solutions using diverse data sources such as claims, EHRs, registries, and clinicogenomics. Panelists will discuss multimodal methodologies, including AI/ML and qualitative approaches, and the role of database linkages and tokenization. Practical examples will illustrate current practices, and the session will conclude with recommended best practices for designing observational studies to advance insights into real-world management of rare conditions.

Personalizing Race and Ethnicity Data to Improve Real-World Evidence Relevance and Reliability

Date and time: May 15, 5:00pm – 6:00pm
Moderator: Lysel Brignoli (Oracle Life Sciences France)

Speakers: Martina Furegato (Oracle Life Science), Carla Rodriguez-Watson (Reagan Udall Foundation for the FDA), Rachele Hendricks-Sturrup (Duke-Margolis Center for Health Policy, Washington)

This workshop will address the challenges of capturing accurate race and ethnicity data in RWD, essential for advancing inclusive and equitable healthcare. Speakers will explore methods to improve data quality, share case studies and national frameworks, and discuss integrating real-world patient-generated data. The session will also examine regulatory, research, and policy implications, offering strategies to strengthen data infrastructure and generate robust, representative RWE to support decision-making and health equity goals.

Addressing Information Bias in Electronic Health Records and Claims Data: What Can the Literature Tell Us and How Should We Respond?

Date and time: May 16, 8:00am – 9:00am
Moderator: Patrick Arena (Aetion, Inc.)
Speakers: Yezhou Sun (Merck & Co., Inc.), Mina Tadrous (University of Toronto), Daina B Esposito (Moderna)

As RWD plays a growing role in regulatory and HTA decision-making, addressing information bias is essential to ensure valid RWE. This session will explore how issues such as measurement error and misclassification impact studies using EHR and claims data. Speakers will share strategies, case studies, and mitigation approaches, while audience input will help shape a proposed tool aimed at strengthening best practices and improving study reliability across real-world research settings.

Are Social Determinants of Health (SDoH) Data Ready for Primetime?

Date and time: May 16, 8:00am – 9:00am
Moderator: Smita Kothari (Merck Sharp & Dohme International Service BV)
Speakers: Amy K O'Sullivan (Ontada), C Daniel Mullins (University of Maryland School of Pharmacy)

The role of SDoH in shaping health outcomes and disparities is widely recognized, but questions remain about the readiness of SDoH data captured in EHRs to inform research and guide interventions. This session will debate whether current data are robust enough to address disparities and improve clinical trial diversity. Drawing on contrasting viewpoints, speakers will examine progress in SDoH data collection, persistent gaps, and future directions to enhance its utility in advancing health equity.

Ensuring the Validity of Real-World Evidence Studies: How Much Can You Check the Data Before You Start?

Date and time: May 16, 10:00am – 11:00am
Moderator: Melvin Skip Olson (Olson Strategies GmbH)
Speakers: Nicole Mittmann (Canada's Drug Agency), Jennifer Christian (Target RWE), Helene Karcher (Novartis AG)

Balancing pre-specification and flexibility in RWE studies is challenging, especially when aiming for causal inference. This workshop will explore how much data can be examined before finalizing analysis plans without compromising scientific rigor. Speakers will present practical examples, propose boundaries for acceptable data checks, and discuss regulatory considerations. Audience interaction will help assess feasible approaches to data exploration prior to protocol registration, offering valuable insights for researchers, statisticians, and decision-makers navigating evolving RWE standards.

Fit-for-Purpose Real-World Data for Medical Device Decision Making: Hype or Hope?

Date and time: May 16, 10:00am – 11:00am
Moderator: Eric Barrette (Medtronic)
Speakers: Katherine Mues (Aetion), Farah Husein (CDA-AMC), Ami Buikema (Optum)

As RWE plays a growing role in device evaluation, defining "fit-for-purpose" RWD remains complex. This session will examine differences between devices and pharmaceuticals in RWD, challenges in data capture and validation, and implications for regulatory and payer decisions. Panelists will share perspectives from regulators, HTAs, and payers, and discuss innovative methods to improve device RWE acceptance, including advanced analytics, data linking, and study design strategies.

Latest research in real-world data and real-world evidence

Healthcare Resource Utilization in Real-World Data

Date and time: May 14, 10:15am – 11:15am
Moderator: Xiao Xu (Columbia University Irving Medical Center)

In this Podium Session, leading research abstracts, chosen by the Research Review Committee, will be presented orally by their authors. This session presents real-world studies on resource use and costs of care across oncology, cardiovascular, and cognitive health. Research includes:

• Economic Burden of Recurrence among Patients with High-Risk Non-Muscle-Invasive Bladder Cancer who Received Bacillus Calmette-Guérin in the United States: A SEER-Medicare Study – Wei Gao (Analysis Group, Inc.)
• Healthcare Resource Utilization (HCRU) and Cost among Patients with Metastatic Melanoma Receiving Nivolumab + Relatlimab (NIVO+RELA) or Nivolumab + Ipilimumab (NIVO+IPI) in the Optum Database – Justin Moser (HonorHealth Research and Innovation Institute)
• Long Term Healthcare Costs and Utilization Among Patients with Non Obstructive Hypertrophic Cardiomyopathy – Paulos Gebrehiwet (Cytokinetics, Inc.)

Real-World Evidence to Inform Decisions: Focus in Oncology

Date and time: May 15, 10:15am – 11:15am
Moderator: Maja Kuharic (Northwestern University)

Focusing on oncology, this Podium Session will present new research demonstrating how RWD and advanced analytics uncover treatment patterns, patient outcomes, and the impact of novel therapies, offering valuable insights to inform care and better understand patient journeys. This includes presentations on:

• Real-World Treatment Patterns of Advanced Melanoma in the United States – Katrine L. Wallace (Replimune)
• Navigating Real-World Data (RWD) Complexities: Operational Assessment Strategy to Identify Fit-For-Purpose Data Sources for Real-World Evidence (RWE) Studies With Regulatory Purpose (OASIS) – Summer Drummond (IQVIA)
• Oncology Trial Emulation Using Real-World Electronic Health Record Data: Results of the Coalition to Advance Real-World Evidence Through Randomized Controlled Trial Emulation (CARE) Initiative – Natalie Levy (Aetion)

Real-World Evidence Poster Tour

Date and time: May 15, 4:00pm – 4:45pm
In this Poster Tour, attendees can experience a guided walk-through of a series of posters presented during the ISPOR conference. Featured posters include:

• Operationalizing an Outcomes-Based Market Access Agreement Using Real-World Data From the Canadian Neuromuscular Disease Registry – Allison Wills (20Sense)
• Best Practices and Standards to Enhance the Quality of Rare Disease Registries in Canada – Jean-Eric Tarride (McMaster University)
• Structured Evaluation of Oncology Real-World Data Quality for Practical Applications – Vivek Verma (AstraZeneca Canada)

Other notable posters being presented include:

• Real-World Effectiveness of Tirzepatide vs. Semaglutide on HbA1c and Weight in GLP-1 RA Naïve Patients with T2D – Kendra Terrell (Eli Lilly and Company)
• Assessing the Comorbidity Burden of Ehlers-Danlos Syndromes (EDS): An Analysis Using US Real-World Data – Janna Manjelievskaia (Veradigm)
• Optimizing EHR Data Completeness: A Conceptual Framework for Bringing Real-World Data into Clinical Research through Relevant Completeness – Dan Drozd (PicnicHealth)
• The Role of Real-World Evidence in Breast Cancer Drug Submissions to NICE: Insights and Challenges – Telma Peixoto (Cytel International BV)
• When Secondary RWD Are Not Enough: A Process for Making an Early Decision to Invest in Primary Data Collection – Ulka Campbell (Aetion)
• Leveraging CMS Medicare Data for Oncology RWE: Benefits, Limitations, and Opportunities, and Insights into Enrollment Patterns – Keri Calkins (Mathematica)
• Advancing the Development of Real-World Data for Cancer Care in China: Challenges and Opportunities – Zheng Yin (IQVIA Greater China Real World Evidence)
• Use of Patient Support Program Data for Real-World Evidence Generation: Opportunities and Pitfalls Illustrated in a Case Study Assessing Trastuzumab Deruxtecan Among Patients With Breast Cancer – Rana Qadeer (AstraZeneca Canada)
• Youth Mental Health Crisis: Economic Impacts and HEOR Opportunities – Camille V Cook (LexisNexis Risk Solutions)
• Moving the Needle: Value and Use of Real-World Evidence in Oncology Clinical Pathways Decision-Making – Phoenix J Riley (AESARA)
• US Physician Characteristics Associated With Real-World Evidence Implementation in Oncology Practice – Thomas Porter (George Washington University School of Medicine)
• The Evolving Role of Real-World Evidence in US Reimbursement Approvals: Trends and Case Studies (2020-2024) – Jaideep K Kaneria (Tata Consultancy Services)
• Linkage of Clinical Trial Data to Real-World Data (RWD) Sources: A Scoping Review – Mehdi NajafZadeh (Medidata Solutions)
• Suicidal Ideation in Patients Treated With Glucagon-like Peptide 1 Receptor (GLP1) Agonists: A Retrospective Real-World Analysis – Jessica Probst (OM1)
• Operational Definitions in Practice: Finding and Evaluating Algorithms for Identifying Patients with Cervical and Uterine Cancer for Real World Data Studies – Evelyn J Rizzo (Mobility HEOR)
• Development and Application of a Novel Framework for Clinician Stakeholder Engagement in Real-world Data (RWD) Studies – Jessica Paulus (Ontada)
• Concomitant Usage of Contra-Indicated Medications in Patients With Atrial Fibrillation: A Comparison of Real-World Data Sources in the United States – Mike Sicilia (Forian)
• Real-World Evidence in FDA and EMA Regulatory Reviews: Insights on Its Role in Submission Packages and Approvals – Jean-Paul Collet (Evidinno Outcomes Research Inc)
• Development of Robust Comprehensive Code Lists to Ensure Consistency Across Global Real-World Evidence Studies – Aaron Kamauu (Navidence)
• Analysis and Management of Computable Operational Definition (CODef) Concept Variation Based on a Large Real-World Studies Library – Michael D Buck (Navidence)
• No Claim Left Behind: Viability of Assessing Early Real-World Vaccine Effectiveness in Open and Closed Administrative Healthcare Claims – Amanda Wilson (Aetion)
• Framework for Real-World Data Used in Regulatory Submissions – Shivani Aggarwal (Landmark Science)
• Use of Real-World Evidence in HTA Submissions and Cost-Effectiveness Evaluations in Asia – Tanja Babic (Stratenym)
• Linkage Of Patient Services and Real-world Healthcare Data: A Unique Approach to Gain Insights into Factors that Impact Medication Adherence – Samuel Igweokpala (United BioSource LLC)
• Advancing Breast Cancer Research in Japan Using EHR-Derived Real-World Data – Harlan Pittell (Flatiron Health)
• Race, Social Determinants of Health (SDoH), and Stage at Breast Cancer Diagnosis in Electronic Health Records (EHR) Data – Zuzanna Drebert (TriNetX)
• Enhancing Health Technology Assessment With Data Transportability Analyses: Addressing Bias and Generalizability Challenges In Non-Local Real-World Evidence – Cal Shephard (AstraZeneca)
• A Comprehensive Review of Real-World Evidence (RWE) Use in Submissions to Canada's Drug Agency (CDA) – Eon Ting (AstraZeneca)

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.