LEADER 3D: Challenges, Strategies & Regulatory Considerations for Rare Disease Clinical Trial Design

What are the regulatory considerations for designing a rare disease clinical trial? This LEADER 3D video provides an overview of FDA’s regulatory requirements and strategies for clinical trial design in rare disease drug development.
FDA acknowledges the unique challenges in rare disease drug development, such as small, heterogenous populations, varying time courses and manifestations of disease, and natural history that is not well understood. This video illustrates different clinical trial designs suitable for rare diseases, aiming to maximize clinical trial efficiency and the likelihood of interpretable results.
Rare disease drug development is not “one size fits all.” Learn how to navigate these scientific challenges and design a rare disease clinical trial that meets FDA’s regulatory requirements.
Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) is part of CDER’s Accelerating Rare disease Cures (ARC) program. Learn more about CDER’s ARC Program: www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/accelerating-rare-disease-cures-arc-program