The US Food and Drug Administration (FDA) has released a draft guidance outlining a new regulatory framework to support development and approval of personalised therapies designed for patients with rare genetic conditions where traditional large clinical trials may not be feasible. These therapies include genome editing and RNA based treatments that directly target a disease's underlying genetic or molecular cause.
The draft guidance describes how developers can generate substantial evidence of safety and effectiveness by demonstrating a clear biological rationale and using well characterised natural history data in lieu of traditional large randomised trials.
The FDA is accepting public comments on the draft guidance through late April 2026, after which it may be revised before being finalised.
Submit comments here using Docket No. FDA‑2026‑D‑1256.
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