PDA Letter Article

RMAT Program Raises a Few Questions

by Austin Caudle, IQVIA


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 (1).