FDA & Rare Disease Drugs: Why Policy and Politics Are Heating Up

Health Affairs' Jeff Byers welcomes Deputy Editor Leslie Erdelack back to the pod to break down recent turbulence at the FDA following the departure of Vinay Prasad, whose decisions around rare‑disease gene therapies courted controversy. They explore the fast‑growing rare disease therapeutics market, why traditional clinical trials often don’t work for ultra‑rare genetic conditions, and the new FDA draft guidance for rare disease drug development.

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Related Articles:

• FDA vaccines chief who ran afoul of pharma to depart (Politico)
• Rare Disease Therapeutics Market to Surpass US$ 495.27 Billion by 2033 as Gene Therapy, RNA-based Drugs, and Biologics Transform Patient Care (PR Newswire)
• FDA NEWS RELEASE: FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases 
• FDA illuminates new approval pathway for bespoke gene editing therapies (Fierce Biotech)
• One Pivotal Trial, the New Default Option for FDA Approval — Ending the Two-Trial Dogma (The New England Journal of Medicine)