FDA Issues Draft Guidance for Individualized Gene-Editing Therapies; CMS to Expand Cell and Gene Therapy Access Model
The U.S. Department of Health and Human Services (HHS) announced on October 27 that the Food and Drug Administration (FDA) has issued a draft guidance giving sponsors a clear framework for developing and winning approval of personalized gene therapies for patients with rare genetic diseases. The Centers for Medicare & Medicaid Services (CMS) said the same day it will expand its Cell and Gene Therapy (CGT) Access Model beyond sickle cell disease, to help more Medicaid patients reach such treatments.
The guidance, titled "Individualized Human Genome Editing Products: Considerations for BLA Submission and Postapproval Management," addresses therapies tailored to each patient's specific genetic variant. Some rare diseases are caused by mutations that differ from patient to patient, so a single treatment cannot work for everyone with the same condition, and sponsors had sought clear FDA guidance on how to obtain approval for a therapy that may look slightly different for each patient it treats.
Central to the framework is the "Individualized Feature Space" (IFS): a set of pre-defined boundaries describing how a gene-editing product may vary from patient to patient while remaining within the scope of a single Biologics License Application (BLA). When a new patient-specific variant falls within those boundaries and laboratory assays show adequate safety and activity, sponsors can add it to the BLA without performing additional clinical trials. The guidance also addresses manufacturing, safety testing including risks of unintended genetic changes, and product naming and labeling. FDA is inviting public comment on the draft, which may be submitted to docket FDA-2026-D-11783 within 30 days of publication in the Federal Register.
"Families confronting rare genetic diseases deserve more treatment options, no matter how few patients share their diagnosis," said HHS Secretary Robert F. Kennedy, Jr. Acting FDA Commissioner Kyle Diamantas said a one-size-fits-all approach to treatment does not work for rare diseases and neither should a one-size-fits-all approach to regulation. Karim Mikhail, director of FDA's Center for Biologics Evaluation and Research, called the guidance a critical step that can help patients, including those with ultra-rare conditions for which no treatment exists, access life-changing therapies more efficiently.
At CMS, the voluntary CGT Access Model has CMS negotiate outcomes-based agreements with manufacturers on behalf of state Medicaid programs. Its first evaluation found that CMS-led negotiations for sickle cell disease therapies eased states' administrative burden and strengthened their leverage by pooling beneficiaries. In the coming months CMS will issue a new Request for Applications to broaden the model's scope, enable negotiations to begin sooner after a therapy is approved, and welcome therapies already approved or licensed. "A medical breakthrough only matters to patients if they can get it," CMS Administrator Dr. Mehmet Oz said.
Source: GSM · USA (original Chinese edition)
Photo: 美国卫生与公众服务部（HHS）总部Hubert H. Humphrey大楼，位于华盛顿。照片：Matthew Bisanz，CC BY-SA 3.0，经Wikimedia Commons。
