WSJ Opinion: Potomac Watch The FDA's Gene-Therapy Reversal and the Exit of Vinay Prasad
Jul 30, 2025
The FDA's reversal on a gene therapy for Duchenne muscular dystrophy sparks debate over drug regulation and innovation. A significant halt on Elevitis, following patient deaths, raises questions about the future of gene therapy. The resignation of a key FDA official highlights ongoing challenges in balancing safety with patient access. Meanwhile, Robert F. Kennedy Jr. proposes reforms to the National Vaccine Injury Compensation Program, addressing its inefficiencies and possible corruption in claims processing.
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Cost vs. Access Conflict at FDA
- FDA officials like Vinay Prasad advocated cost-effectiveness and rigid trial designs, pushing to ration drugs based on price and marginal benefit.
- This contradicts FDA's core responsibility of approving drugs for safety and efficacy, creating tension within the agency.
Need for Flexible Regulation in Rare Diseases
- Rare disease treatments require regulatory flexibility as traditional large randomized trials may be impractical or unethical.
- FDA must balance the need for safety review with allowing patient access to promising but uncertain therapies.
FDA Leadership Turnover and Impact
- FDA leadership changes signal tension between accelerating approvals and skepticism about novel treatments.
- Conflicts over COVID vaccine policies contributed to turnover, influencing agency direction and public trust.