Gottlieb Briefs Lawmakers on Cures Act Developments

FDA Commissioner Scott Gottlieb discussed the agency’s progress in its implementation of the 21st Century Cures Act in testimony before the House & Energy Commerce Committee on Wednesday, where he highlighted the Regenerative Medicine Advanced Therapy (RMAT) designation and the agency’s use of real-world evidence (RWE).

Gottlieb reported that the RMAT designation, which was established by the Cures Act to give expedited reviews to innovative therapies, has already been put to use. As of June 30, CDER had granted 24 RMAT designations, he said.

Gottlieb also said the agency is actively working to incorporate RWE into its regulatory decision making, including information like billing data, registries, claims and electronic health records, in order to address the questions of broader patient populations. He said that the agency plans to release RWE guidance for drugs and biologics.