Using Real-World Evidence to Support the FDA Regulatory Approval: Methodological Considerations

Friday, November 4, 2022

8:00 AM – 9:30 AM ET

Location: 236 CC

CE: 1.5

Chair(s)

Kevin Lu, PhD, FISPE, BPharm

Associate Professor
Clinical Pharmacy and Outcomes Sciences
University of South Carolina
Columbia, South Carolina, United States

Slides

Discussant(s)

Patricia Slattum, PharmD, PhD

Professor Emeritus
Pharmacotherapy and Outcomes Science
Virginia Commonwealth University
Richmond, Virginia, United States

Individual Symposium Abstract First Author(s)

Kevin Lu, PhD, FISPE, BPharm

Associate Professor
Clinical Pharmacy and Outcomes Sciences
University of South Carolina
Columbia, South Carolina, United States

Slides
Jie (Jenni) Li, PhD

Associate Director for RWE
Office of Suiveillance and Epidemiology
FDA CDER Office of Surveillance and Epidemiology
Silver Spring, Maryland, United States
Julia DiBello, PhD

Sr. Principal Scientist
Epidemiology
Merck & Co., Inc.
West Point, Pennsylvania, United States
Minghui Li, PhD

Assistant Professor
Clinical Pharmacy and Translational Science
University of Tennessee Health Science Center
Memphis, Tennessee, United States

Evidence from clinical trials has traditionally been used to support regulatory drug approval, whereas real-world evidence (RWE) has been used for post-marketing surveillance studies. With the enaction of the 21st Century Cures Act, the Food and Drug Administration (FDA) is evaluating the potential use of RWE to support new indications for an approved product or to satisfy post-approval study requirements. Yet the substantial evidence standard remains unchanged for FDA approval. To date, the FDA has issued guidelines or frameworks for using RWE for regulatory decisions. However, many methodological challenges remain unanswered when using RWE in gerontology research. This Pharmaceutical Care and Outcomes Research Interest Group-sponsored symposium consists of leading experts in the country to address these problems. The four papers in this symposium will address methodological challenges that are unique in gerontology research. Specifically, The first paper will introduce the FDA drug evaluation process, focusing on the key considerations for evaluating RWE in regulatory decision making; the second paper will discuss possible challenges related to data sources from which RWE are generated in aging research; the third paper will provide a case example of some of the challenges in using RWD sources to generate RWE in the Alzheimer’s Disease setting; and the fourth paper will provide recommendations for improving gerontology research based on RWE going forward.

Learning Objectives:

After attending this session, participants will be able to describe key considerations for study design and conduct to support the FDA regulatory approval of drugs for use in older adults using real-world evidence.
After attending this session, participants will be able to understand the strengths and limitations of studies using real-world evidence for assessing medication efficacy or safety concerns in older adults.

Presentations:

8:00 AM – 9:30 AM ET

Challenges Related to Data Sources for RWE in Aging Research
Individual Symposium Abstract First Author: Kevin Lu, PhD, FISPE, BPharm – University of South Carolina
8:00 AM – 9:30 AM ET

Key Considerations for Evaluating Real-World Evidence in Support of FDA Regulatory Decision-Making
Individual Symposium Abstract First Author: Jie (Jenni) Li, PhD – FDA CDER Office of Surveillance and Epidemiology
8:00 AM – 9:30 AM ET

Use of RWD to Generate RWE in the Alzheimer's Disease Setting: Challenges and Future Directions
Individual Symposium Abstract First Author: Julia R. DiBello, PhD – Merck & Co., Inc.
8:00 AM – 9:30 AM ET

Use RWE to Improve Drug Information for Geriatric Patients
Individual Symposium Abstract First Author: Minghui Li, PhD – University of Tennessee Health Science Center