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FDA Rare Disease Town Hall
Session Chair(s)
James Valentine, JD, MHS
Director
Hyman, Phelps & McNamara, PC, United States
Senior FDA leaders will discuss evolving regulatory trends in rare disease development, including accelerated approval, innovative trial designs, patient engagement, and real-world evidence, followed by open Q&A with attendees.
Learning Objective : Identify current FDA regulatory trends and scientific approaches shaping rare disease drug development; Apply FDA expectations around accelerated approval, surrogate endpoints, and innovative trial designs; Integrate patient voice and real-world evidence into development strategies to support benefit-risk assessment.
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