Leveraging Real-World Evidence to Assess Benefits and Risks of GLP-1-Based Therapies

Event Details

Purpose

To identify gaps in our knowledge of the benefits and risks of GLP-1-based therapies and how they could be addressed using real-world evidence (RWE) to inform policy and clinical practice decisions.

Meeting Objectives

At the end of the workshop, participants will identify gaps and opportunities for future research on:

1. Best practices for study designs of GLP-1-based therapies using RWE.
2. Identifying and developing data sources for RWD studies, including strengths and limitations.
3. Best practices for analytic methods to address issues specific to GLP-1-based therapies in studies using real-world data (RWD).
4. Use of RWE in clinical decision-making and clinical guidelines development.
5. Use of RWE for regulatory and healthcare policy decision-making

Background

GLP-1-based therapies have transformed the treatment landscape for both obesity and diabetes and offer new options for managing related complications such as major cardiovascular events and obstructive sleep apnea. Growing evidence from clinical trials and observational studies also suggests their potential for the prevention or treatment of multiple other chronic diseases ranging from liver, kidney, and heart disease to addiction, neurodegenerative disorders, and long COVID. With the rapidly increasing use of the relatively new class of GLP-1-based therapies, there is a critical need to identify rare but serious adverse effects, such as cancers and suicidal ideation, as well as health outcomes that may take years to develop and may not be captured in randomized clinical trials or detected through spontaneous reporting systems like the FDA Adverse Event Reporting System. Many questions remain about the use of GLP-1-based therapies in real-world settings, such as optimal treatment pathways for sustainable care, safety and effectiveness in specific subpopulations, heterogeneity of treatment response, and use in a broader range of clinical settings and presentations than those studied in clinical trials. Understanding the impact of GLP-1-based therapies on healthcare costs and benefits helps inform healthcare policy development. Real-world evidence (RWE) using real-world data (RWD) sources, including electronic health records (EHR) and claims data, offer the potential to answer many of these questions but also have significant limitations. In addition, challenges unique to GLP-1-based therapies, such as inadequate insurance coverage, medication shortages, compounded medications obtained outside of the healthcare system, and off-label uses require special consideration. This workshop aims to identify knowledge gaps in the optimal use of GLP-1-based therapies and how they could be addressed using real-world evidence (RWE).

Planning Committee

Craig Hales; National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sue Yanovski; NIDDK
Voula Osganian; NIDDK
Kevin Abbott; NIDDK
Debbie Gipson; NIDDK
Maren Laughlin; NIDDK
Jean Lawrence; NIDDK
Chris Lynch; NIDDK
Ravi Ravichandran; NIDDK
Aynur Unalp-Arida; NIDDK
Kenneth Wilkins; NIDDK
Laurie Donze; National Heart, Lung, and Blood Institute (NHLBI)
Lindsay Pool; NHLBI
Candice Price; NHLBI
Mike Wolz; NHLBI
Aaron Pawlyk; National Institute of Child Health and Human Development (NICHD)
Ed Sauter; National Cancer Institute (NCI)
Mir Ali; National Institute on Aging (NIA)
Mehdi Farokhnia; National Institute on Drug Abuse (NIDA)
Matt Rudorfer; National Institute of Mental Health (NIMH)
Kristy Nicks; National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Josh Fessel; National Center for Advancing Translational Sciences (NCATS)
Deborah Young-Hyman; NIH Office of Behavioral & Social Sciences Research (OBSSR)
Jeremy Green; NIH Office of Disease Prevention (ODP)
Edwina Wambogo; NIH Office of Dietary Supplements (ODS)
Drew Bremer; NIH Office of Nutrition Research (ONR)
Andrea Brandau; Patient-Centered Outcomes Research Institute (PCORI)
Alison Goodman; CDC, Division of Nutrition, Physical Activity, and Obesity
Robert Montierth; CDC, Division of Diabetes Translation 
Rui Li; CDC, Division of Diabetes Translation 
Osato Idubor; CDC, Division of Diabetes Translation 
Marie Bradley; FDA, Real World Evidence Program

Registration Deadline

May 1, 2025

Agenda

Wednesday, May 7, 2024

8:30 a.m. – 9:00 a.m.

Registration

Welcome

9:00 a.m. – 9:10 a.m.

Welcoming Remarks
Griffin P. Rodgers, M.D., MACP, Director, National Institute of Diabetes and Digestive and Kidney Diseases

9:10 a.m. – 9:25 a.m.

Opening Remarks from Workshop Co-Chairs
David Arterburn, M.D., M.P.H., Kaiser Permanente Washington Health Research Institute
Lesley Curtis, Ph.D., Duke University School of Medicine
Darren Toh, Sc.D., Harvard T.H. Chan School of Public Health

Keynote Presentations
Moderator: Kimberly Narain, M.D., Ph.D., M.P.H., University of California, Los Angeles

9:25 a.m. – 10:05 a.m.

Keynote Presentation 1: What Do We Still Need to Know about the Safety, Effectiveness, and Optimal Use of GLP-1-based Therapies?
David D’Alessio, M.D., Duke University School of Medicine

10:05 a.m. – 10:45 a.m.

Keynote Presentation 2: The Promise and Challenges of Real-world Evidence
Matthew Maciejewski, Ph.D., Duke University School of Medicine

10:45 a.m. – 11:00 a.m.

Break

Session 1: Real-world Evidence to Inform Regulatory Decisions and Clinical Practice

11:00 a.m. – 11:20 a.m.

Use of Real-world Evidence for Regulatory Decision-making
Marie Bradley, Ph.D., Food and Drug Administration

11:20 a.m. – 11:40 a.m.

Assessing Strength of Real-world Evidence for Clinical Practice and Guidelines Development
John Wong, M.D., Tufts University School of Medicine
Sarah Armstrong, M.D., Duke University School of Medicine

11:40 a.m. – 12:10 p.m.

Moderated Discussion
Moderator: Christina Wee, M.D., M.P.H., Harvard Medical School

12:10 p.m. – 1:10 p.m.

Lunch

Session 2: Real-world Evidence for Public and Private Payer Coverage Decisions

1:10 p.m. – 1:30 p.m.

Public Payer Perspective
(speaker TBD)

1:30 p.m. – 1:50 p.m.

Private Payer Perspective
David Hines, Nashville Public Schools

1:50 p.m. – 2:10 p.m.

Challenges with Assumptions for Economic Analyses and How Real-world Evidence May Inform Them
Noelia Duchovny, Ph.D., Congressional Budget Office

2:10 p.m. – 2:40 p.m.

Moderated Discussion
Moderator: Inmaculada Hernandez, Ph.D., University of California, San Diego

2:40 p.m. – 2:55 p.m.

Break

Session 3: Methodologic Approaches to Real-world Evidence Generation for GLP-1-based Therapies

2:55 p.m. – 3:15 p.m.

Pragmatic Trials for GLP-1-based Therapies Using Real-world Data
Lesley Curtis, Ph.D., Duke University School of Medicine
Ania Jastreboff, M.D., Ph.D., Yale School of Medicine

3:15 p.m. – 3:45 p.m.

Estimating the Effects of GLP-1-based Therapies Using Observational Data
Miguel Hernan, M.D., Dr.P.H., Harvard T.H. Chan School of Public Health

3:45 p.m. – 4:15 p.m.

Moderated Discussion
Darren Toh, Sc.D., Harvard Medical School

4:15 p.m. – 4:35 p.m.

Recap Day 1 by Workshop Co-Chairs

4:35 p.m.

Day 1 Meeting Adjournment

Thursday, May 8, 2024

9:00 a.m. – 9:10 a.m.

Welcome and Goals for Day 2
David Arterburn, M.D., M.P.H., Kaiser Permanente Washington Health Research Institute

Session 1: Best Practices for Studying GLP-1-based Therapies—Defining Exposure and Outcomes

9:10 a.m. – 9:40 a.m.

Defining Exposure to GLP-1-based Therapies and Comparison Groups Using Real-world Data
Kristina Lewis, M.D., M.P.H., S.M., Wake Forest University School of Medicine

9:40 a.m. – 10:40 a.m.

Real-world Effectiveness and Safety of GLP-1-based Therapies

1. Cardiovascular Kidney Metabolic Disease
   Rozalina McCoy, M.D., M.S., University of Maryland School of Medicine
2. Psychiatric Conditions (Addiction, Eating Disorders, Suicidality)
   Esti Iturralde, Ph.D., Kaiser Permanente Northern California
3. Neurodegenerative Diseases
   Serena Jingchuan Guo, M.D., Ph.D., University of Florida College of Pharmacy
4. Cancer
   Jennifer Lund, Ph.D., The University of North Carolina Gillings School of Global Public Health

10:40 a.m. – 10:55 a.m.

Break

10:55 a.m. – 11:25 a.m.

Moderated Discussion
Moderator: Kathleen McTigue, M.D., M.P.H., M.S., University of Pittsburgh School of Public Health

Session 2: Summary of Research Gaps and Opportunities

11:25 a.m. – 11:55 a.m.

Summary of Research Gaps and Opportunities

11:55 a.m. – 12:15 p.m.

Moderated Discussion
David Arterburn, M.D., M.P.H., Kaiser Permanente Washington Health Research Institute
Lesley Curtis, Ph.D., Duke University School of Medicine
Darren Toh, Sc.D., Harvard Medical School

12:15 p.m.

Meeting Adjournment

Travel

Hotel Accommodations

The Bethesdan Hotel Tapestry Collection by Hilton
8120 Wisconsin Avenue
Bethesda, MD 20814
Telephone: (301) 652-2000
Reserve a Room

Government Room Rate

A limited block of sleeping rooms for meeting participants has been reserved at The Bethesdan Hotel. The rate is the prevailing government rate of $276 per night for single occupancy, plus tax (13%). The room block will be in effect at the Government rate only until Sunday, April 6, 2025, or until full, whichever comes first. Any room reservations received after this date will be accepted on a space- and rate-availability basis. Please be certain that the hotel provides you with a confirmation number for your reservation.

Reservation Dates

Book your arrival date as Tuesday, May 6, 2025, and departure as Thursday, May 8, 2025. If you require alternate dates, please contact Danielle Johnikin of The Scientific Consulting Group, Inc. (SCG).

Check-in

When making a reservation, please provide your room and bedding preferences. The hotel will assign specific room types at check-in, based on availability. Please be advised that requests are not guaranteed. Check-in time is 3:00 p.m., and checkout time is 12:00 p.m.

Cancellations

If you need to cancel your reservation, please do so 48 hours prior to your arrival date, or you will be charged a no-show fee for 1 night on your credit card.

NIH Visitor Information

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Travel Resources

• NIH Info (DOCX, 14.12 KB)