RMAT Program Raises a Few Questions
Gene and cell therapies hold the potential to transform medicine and create a seismic shift in our ability to treat, and possibly cure, many diseases once considered untreatable. Exciting improvements have been observed in recent years. But this requires updating the regulatory structure as existing rules have not been designed for these kinds of therapies. To expedite the development and review of these innovative products, the 21st Century Cures Act includes the regenerative medicine advanced therapy (RMAT) designation. Prior to adding RMAT, only four possible designations existed: Fast Track, Breakthrough Therapy, Priority Review and Accelerated Approval.
In November 2017, the U.S. FDA issued a draft guidance document to describe the expedited programs available to sponsors of regenerative medicine therapies for serious conditions, including products designated as RMATs. According to the guidance, “regenerative medicine therapies to treat, modify, reverse, or cure serious conditions are eligible for FDA’s expedited programs, including Fast Track designation, Breakthrough Therapy designation, RMAT designation, Priority Review designation, and Accelerated Approval, if they meet the criteria for such programs”. To qualify for this designation, a product must be 1) defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or a combination product using such therapies or products; 2) intended to treat, modify, reverse or cure a serious or life-threatening disease or condition; and 3) preliminary clinical evidence must indicate the product has the potential to address unmet medical needs for such a disease or condition.