FDA and CMS Announce New Steps to Advance Gene Therapies and Expand Access for Patients with Rare Diseases

WASHINGTON, D.C.-OCTOBER 27, 2026-The U.S. Department of Health and Human Services (HHS), through the Food and Drug Administration, today issued a draft guidance for industry to help sponsors develop and seek approval for personalized gene therapies designed to treat patients with rare genetic diseases. The guidance, Individualized Human Genome Editing Products: Considerations for BLA Submission and Postapproval Management, provides a clear regulatory framework for submitting and managing Biologics License Applications (BLAs) for individualized genome editing (GE) products. The Trump Administration is committed to modernizing approaches to safely get more cures and meaningful treatments to all Americans, regardless of the size of the patient population.

"Families confronting rare genetic diseases deserve more treatment options, no matter how few patients share their diagnosis," said HHS Secretary Robert F. Kennedy, Jr. "Under President Trump's leadership, we are clearing a path for scientists to develop personalized therapies and for patients to access them. We will advance innovation, uphold rigorous safety and effectiveness standards, and put patients with even the rarest diseases at the center of our efforts."

"For patients with rare genetic diseases, a one size fits all approach to treatment simply does not work and neither should a one size fits all approach to regulation," said Acting Commissioner of Food and Drugs Kyle Diamantas, J.D. "This guidance gives sponsors the regulatory clarity they need to develop therapies tailored to each patient's unique genetic makeup, while maintaining the gold standard science that Americans deserve. Under President Trump's leadership, FDA is committed to ensuring that even the rarest conditions have a pathway to innovative, life changing treatment."

"A medical breakthrough only matters to patients if they can get it," said CMS Administrator Dr. Mehmet Oz. "Under President Trump and Secretary Kennedy's leadership, CMS is working to give Americans with rare diseases, particularly children, a chance at healthier, fuller lives without cost standing in the way. CMS is bringing state Medicaid programs together to negotiate with drug manufacturers to help put life-changing therapies within reach."

Some rare diseases are caused by genetic mutations that differ from patient to patient, meaning a single treatment cannot work for everyone with the same condition. Individualized GE therapies are tailored to target each patient's specific genetic variant (mutation). Sponsors have sought clear FDA guidance on how to obtain approval for a therapy that may look slightly different for each patient it treats. This guidance provides that clarity, addressing how to structure a BLA that can accommodate multiple patient-specific product variants, and how to efficiently add new variants after approval.

"This guidance represents a critical step forward in FDA's efforts to support the development of individualized therapies for patients with rare genetic diseases," said Karim Mikhail, B. Pharm., M.S., Director of the Center for Biologics Evaluation and Research. "By providing a clear and scientifically rigorous framework for BLA submission and postapproval management, we are helping to ensure that patients - including those with ultra-rare conditions for whom no treatment currently exists - can more efficiently access therapies that may be life-changing."

Central to the framework is the concept of the Individualized Feature Space (IFS), a set of pre-defined boundaries describing how a GE product may vary from patient to patient while remaining within the scope of a single BLA. When a new patient-specific variant of the therapy falls within those boundaries and assays show that the variant has adequate safety and activity, sponsors can submit it to the BLA without needing to perform additional clinical trials. This guidance puts into practice key elements of FDA's Rare Disease Framework, including championing cutting-edge science and novel methods in rare disease drug development.

In addition to the IFS framework, the guidance also addresses manufacturing, safety testing, including risks of unintended genetic changes, and product naming and labeling. It describes how sponsors can add new patient-specific variants of an approved therapy through a streamlined review process, without requiring new clinical trials, by relying on clinical trials that supported approval of the BLA together with laboratory (in vitro) data for the new patient-specific variant. This approach will provide more patients with rare genetic diseases quicker access to more therapeutic options, all while maintaining rigorous safety and effectiveness standards. A recorded webinar, Individualized Human Genome Editing Products: Considerations for BLA and Postapproval Management, summarizing the key elements of the guidance is available on the FDA's YouTube channel.

The FDA is inviting public comment on the draft guidance. Comments may be submitted to the docket FDA-2026-D-11783 at regulations.gov within 30 days of publication in the Federal Register.

The FDA remains committed to advancing regulatory science so that patients with the rarest and most underserved conditions have a pathway to safe, effective, and innovative therapies.

The Centers for Medicare & Medicaid Services (CMS) is taking steps to broaden its Cell and Gene Therapy (CGT) Access Model beyond sickle cell disease to help more Medicaid patients access potentially life-changing treatments. Building on findings from the model's first evaluation report, CMS will invite manufacturers of therapies for other conditions to apply to participate.

Through the voluntary model, CMS negotiates outcomes-based agreements with manufacturers on behalf of state Medicaid programs. The initial evaluation found that CMS-led negotiations for sickle cell disease therapies eased states' administrative burden and strengthened their negotiating leverage by pooling beneficiaries.

In the coming months, CMS will release a Request for Applications (RFA) for manufacturers designed to broaden the model's scope and enable negotiations to begin sooner after a therapy receives FDA approval or licensure, while also welcoming applications for therapies already approved or licensed. This next iteration aims to make treatments more affordable and accessible.