FDA's Regenerative Medicine Advanced Therapy (RMAT) Program

By Isaiah Manoogian, PharmD

The rapidly expanding field of regenerative medicine drives innovative therapies to address unmet needs for patients with serious or life-threatening conditions. This shift aims to transition healthcare from simply "care" to "cure," repairing and restoring tissues and organs. The FDA established the Regenerative Medicine Advanced Therapy (RMAT) designation through the 21st Century Cures Act to expedite the development and review of these promising therapies.

To qualify for RMAT, an investigational product must fit the definition of a regenerative medicine therapy, be intended to treat a serious condition, and show preliminary clinical evidence of its potential to address an unmet medical need. This evidence can come from various clinical investigations, including well-designed retrospective studies or case series, and is evaluated by the Center for Biologics Evaluation and Research (CBER) on a case-by-case basis. RMAT designation offers significant benefits, such as intensive guidance, involvement of senior FDA managers, and the potential for a Rolling Review or Priority Review, all designed to accelerate the timeline for getting novel treatments to patients. Learn the criteria and the process for preparing a successful RMAT request.