Individualised gene therapies for rare diseases: FDA draft guidance
October 28, 2026
The US Food and Drug Administration has issued draft guidance to help sponsors develop and seek approval for individualised gene therapies for rare diseases. In the same release, the Centers for Medicare & Medicaid Services (CMS) said it will broaden a voluntary model that negotiates access to these treatments for Medicaid patients.
Individualised gene therapies for rare diseases: FDA and CMS steps
The draft guidance, Individualized Human Genome Editing Products: Considerations for BLA Submission and Postapproval Management, covers therapies tailored to each patient’s specific genetic variant. Its central concept is the Individualized Feature Space (IFS): pre-defined boundaries for how a product may vary between patients within one BLA. A new patient-specific variant that falls inside those boundaries, and whose assays show adequate safety and activity, can be added without new clinical trials. It puts into practice elements of FDA’s Rare Disease Framework. Comments are invited on docket FDA-2026-D-11783 within 30 days of Federal Register publication.
On the payment side, CMS will broaden its Cell and Gene Therapy Access Model beyond sickle cell disease and invite manufacturers of therapies for other conditions to apply. The model is voluntary: CMS negotiates outcomes-based agreements with manufacturers on behalf of state Medicaid programmes. CMS will release a Request for Applications in the coming months, aiming to start negotiations sooner after FDA approval or licensure.
Why it matters
The release answers a question sponsors have raised: how to get approval for a therapy that may look slightly different for each patient. It does not say how payers would judge a variant added without its own trial. The CMS model is a separate track, covering negotiations for state Medicaid programmes rather than FDA review. Both steps remain open. The FDA text is a draft, and CMS has not yet released its Request for Applications. Our earlier piece on individualised genetic therapies covers the regulation, payment and HTA questions in more depth.
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