The U.S. Food and Drug Administration (FDA) has issued draft guidance intended to simplify the regulatory process for personalized gene therapies targeting rare genetic diseases, while the Centers for Medicare & Medicaid Services (CMS) plans to expand a separate initiative aimed at improving Medicaid patients’ access to cell and gene therapies.

The FDA guidance outlines how developers can submit and manage biologics license applications (BLAs) for individualized human genome editing products. The framework is designed to address the challenges of developing treatments for genetic conditions in which mutations can vary from one patient to another.

Under the proposed approach, developers could seek approval for multiple patient-specific versions of a therapy under a single BLA, rather than pursuing an entirely separate application for each variant. The guidance also describes how additional versions could be incorporated after approval, potentially reducing development timelines for treatments intended for patients with ultra-rare conditions.

A central component is the proposed Individualized Feature Space (IFS), which would establish predefined limits for acceptable variations in a gene-editing product. If a new patient-specific version falls within those parameters and laboratory testing demonstrates adequate safety and activity, sponsors could seek to add it to an existing BLA without conducting additional clinical trials. The review would draw on clinical data supporting the original approval alongside in vitro data for the new variant.

The draft guidance also addresses manufacturing requirements, safety testing for unintended genetic changes, and product naming and labeling. FDA officials said the framework is intended to provide greater regulatory clarity while maintaining standards for safety and effectiveness. The agency is accepting public comments on the draft for 30 days following its publication in the Federal Register under docket FDA-2026-D-11783.

In parallel, CMS is preparing to expand its Cell and Gene Therapy (CGT) Access Model beyond sickle cell disease to include treatments for other conditions. The voluntary program allows CMS to negotiate outcomes-based agreements with manufacturers on behalf of participating state Medicaid programs.

CMS said the model’s initial evaluation found that centralized negotiations for sickle cell disease therapies helped reduce administrative work for states and strengthened their negotiating position by combining purchasing leverage across beneficiaries.

The agency plans to issue a new Request for Applications in the coming months, inviting manufacturers of therapies for additional conditions to participate. The next phase is intended to allow negotiations to begin sooner after FDA approval or licensure while also permitting applications for treatments that are already approved.

Together, the FDA’s proposed regulatory framework and CMS’s planned expansion address two challenges in rare disease treatment: developing therapies for highly individualized genetic conditions and improving patients’ ability to access costly cell and gene therapies. Both initiatives are intended to support broader treatment options, although the FDA guidance remains in draft form and the CMS model’s expansion will depend on the forthcoming application process.

Visit www.fda.gov for more information.