Alternatives to Placebo Controls and RCTs in Rare Disease Trials

Placebo-controlled trials are often impractical or ethically challenging in rare disease settings. This session will explore approaches that use alternative comparators, such as within-patient comparisons or external data sources, and discuss how these strategies can be used to generate interpretable evidence while addressing feasibility, ethical considerations, and regulatory expectations.

Moderator: Amy Comstock Rick, JD, Director of Strategic Coalitions, FDA Rare Disease Innovation Hub

Panelists:

• Oxana V. Crysler, MD, MHS, Clinical Associate Professor of Internal Medicine, University of Michigan, Rogel Cancer Center, a NORD Rare Disease Center of Excellence 
• Wonyul Lee, PhD, Senior Statistical Reviewer, CDER, FDA
• David Margolin, MD, PhD, Vice President of Clinical Development, uniQure, Inc.
• Susana Zaph, PhD, Senior Director, Head of Quantitative Systems Pharmacology -US, Sanofi

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