The FDA said it plans to “reimagine” rare-disease therapy regulation through a Rare Disease Framework Initiative aimed at improving consistency and seeking external input. The agency’s approach looks to replace one-size-fits-all policy with “fit-for-purpose” standards by condition, covering everything from early development engagement to post-marketing requirements. The plan comes as the agency continues a broader “reset,” with former and current leaders facing criticism over perceived flexibility and uncertainty. Analysts said the initiative is not intended to lower evidentiary bars, but to reduce guesswork for sponsors developing therapies in small populations where endpoints and biomarker plans are harder to nail down. For biotech companies and rare-disease specialists, the immediate operational impact is likely to be tighter expectations on documentation and interaction timing—especially around trial design, surrogate endpoints, and the evidence plan that justifies benefit in ultra-rare settings. The FDA will solicit feedback at a meeting on Oct. 28, with industry reporting that early recommendations are targeted for February.