FDA Moves to Tighten Rare-Disease Drug Reviews as New Leadership Seeks Consistency
The U.S. Food and Drug Administration is preparing a broad reset of how rare-disease medicines are evaluated, with new leadership signaling a push for a more consistent, fit-for-purpose framework that could reshape development standards across the sector. The initiative is likely to affect trial design, evidence expectations and manufacturing oversight, with implications for biotech valuations and the pace of future approvals.
