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Leveraging Real-World Evidence and External Controls in Clinical Trials of Rare Outcomes/Diseases: Statistical Innovation and Novel Applications
Session Chair(s)
Huan Wang, PHD
Mathematical Statistician, CDER
FDA, United States
Learn how the Real-World Evidence (RWE) can be utilized in studies of rare outcomes and diseases. Speakers from industry, academia, and regulators share insights on external controls, causal inference, and evidence synthesis to inform regulatory submissions.
Learning Objective : Identify when to use hybrid design and or external controls for rare outcomes and diseases using RWE sources; Design RWE-based studies and address confounding, missing data, and intercurrent events; Evaluate robustness of study results with sensitivity analyses.
Speaker(s)
Comparison of an External Control to a Randomized Control: a Validation Exercise
Ruthie Davi, PHD, MS
Medidata, a Dassault Systèmes Company, United States
Senior Vice President
Real World Evidence for Post-Marketing Drug Safety Outcomes: FDA’s Sentinel System
Jennifer Lyons, PHD, MPH
Harvard Pilgrim Health Care Institute, United States
Research Scientist
Single Arm, External-Controlled Studies – Pitfalls and Pathways
Van Tran, PHD
FDA, United States
Mathematical Statistician, OTS, CDER
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