The US Food and Drug Administration (FDA) has proposed a regulatory framework for individualized human genome editing therapies, while the Centers for Medicare & Medicaid Services (CMS) plans to expand its Cell and Gene Therapy (CGT) Access Model beyond sickle cell disease. Together, these separate initiatives aim to support the development of therapies for rare genetic diseases and improve patient access to cell and gene therapies through Medicaid.

Announced by the US Department of Health and Human Services (HHS) on 27 October 2026, the initiatives address two challenges in rare disease treatment: regulatory requirements for therapies tailored to individual genetic variants and access to potentially life-changing treatments through Medicaid.

FDA proposes a framework for individualized genome editing products

FDA has issued draft guidance titled Individualized Human Genome Editing Products: Considerations for BLA Submission and Postapproval Management. The document describes how a single Biologics License Application (BLA) could cover multiple product variants tailored to different pathogenic gene variants associated with a monogenic disease.

The proposed framework centres on the Individualized Feature Space (IFS), which defines the permitted range of variation in genome editing components within a single BLA. Under the proposed approach, developers could add new variants within an established IFS through a BLA supplement supported by laboratory (in vitro) activity and safety data. Where the available clinical evidence and laboratory data adequately support the new variant, additional clinical trials may not be necessary. Further clinical evidence may be required if relevant uncertainties remain.

The guidance also addresses manufacturing process validation, potency assurance, unintended genetic changes, product labelling and identification. It is a draft for public comment and contains non-binding recommendations; it is not yet final and is not intended for implementation.

CMS plans to broaden its cell and gene therapy access model

Separately, CMS plans to expand its voluntary Cell and Gene Therapy Access Model beyond sickle cell disease to include therapies for other conditions. Under the model, CMS negotiates outcomes-based agreements with manufacturers on behalf of participating state Medicaid programmes.

According to HHS, the initial evaluation found that CMS-led negotiations for sickle cell disease therapies reduced administrative burdens for states and strengthened their negotiating leverage by pooling beneficiaries. CMS plans to issue a Request for Applications (RFA) inviting manufacturers of therapies for additional conditions to participate.

The next iteration is intended to allow negotiations to begin sooner after FDA approval or licensure and to accept applications for therapies that have already been approved or licensed. The expansion has been announced, but the new application process has not yet opened.

Public consultation

FDA is accepting comments on the draft guidance under docket FDA-2026-D-11783. The deadline for comments is 27 November 2026. The draft provides recommendations on BLA submissions and the postapproval management of individualized genome editing products, including the addition of new product variants. It should be considered alongside other relevant FDA guidance on genome editing and gene therapy development.

The CMS access model is a separate initiative focused on affordability and access through Medicaid; it does not form part of the FDA’s BLA guidance.