FDA issues draft guidance to streamline approval of personalized gene therapies and expands CMS access model

The Food and Drug Administration released a draft guidance that lets a single biologics license application cover multiple variants of a gene‑therapy product. The guidance introduces an Individualized Feature Space, a set of predefined criteria that define how a therapy can differ between patients.

The agency says the framework is intended to be clear and scientifically rigorous, helping developers submit applications and manage post‑approval requirements for treatments targeting rare and ultra‑rare diseases.

At the same time, the Centers for Medicare & Medicaid Services announced plans to broaden its Cell and Gene Therapy Access Model, aiming to make these advanced therapies more readily available to patients.

CBER Director Karim Mikhail emphasized that the effort is designed to ensure patients with conditions that have no existing treatments can more efficiently access potentially life‑changing medicines.