FDA drafts guidance to speed approvals of individualized gene therapies, hailed as win for rare disease patients

The U.S. Food and Drug Administration released draft guidance aimed at streamlining the approval process for certain individualized gene therapies. The proposal would allow sponsors to avoid filing separate marketing applications for each patient‑specific product, a practice that has slowed access to these treatments.

By consolidating data requirements, the agency hopes to reduce administrative burdens while maintaining safety standards. The guidance targets therapies that are customized for a single patient but share a common manufacturing platform.

Rare disease advocacy groups welcomed the move, calling it a long‑overdue step toward faster patient access. They argue that the current approach has created delays and higher costs for treatments that address unmet medical needs.

Separately, the Missouri attorney general warned Eli Lilly to stop requiring hospitals to submit claims data as a condition for receiving discounted medicines under the 340B program, adding another regulatory focus for the industry.