FDA & Rare Disease Drugs: Why Policy and Politics Are Heating Up
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Show Notes
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market, why traditional clinical trials often don’t work for ultra‑rare genetic conditions, and the new FDA draft guidance for rare disease drug development.
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- FDA NEWS RELEASE: FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases
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