Cures Act Implementation Updates: RWE Framework, RMAT and Breakthrough Devices

| 25 July 2018 |  By 

US Food and Drug Administration (FDA) Commissioner Scott Gottlieb on Wednesday discussed before the House Energy & Commerce Committee the progress FDA has made in implementing the 21st Century Cures Act, with notable updates on the agency’s work on the use of real-world evidence (RWE), the breakthrough designation for medical devices and the Regenerative Medicine Advanced Therapy (RMAT) designation.

Gottlieb focused his comments in the hearing on drug pricing, with discussion of competition lag for non-orphan products, drug shortages and modernizing clinical trials, noting the use of master protocols, seamless trial designs (draft guidance coming soon), adaptive trials, modeling and simulations to allow for the evaluation of a product’s safety and effectiveness.

“The complexity of conducting trials is delaying to market the second and third in class drugs,” he said, noting that that means prices are not coming down.

On the RWE front, Gottlieb said in his written testimony that FDA is actively working to integrate electronic health records, registries, and claims and billing data into regulatory decision making and to answer questions relevant to broader populations of patients.

He noted that FDA is developing a framework to evaluate the use of RWE to support the approval of new indications of approved medical products, or to help satisfy post-approval study requirements for marketed products.

“RWE may go beyond current postmarketing surveillance capacities, eventually becoming applicable across all phases of medical product development,” he said.

For medical devices, Gottlieb notes FDA has designated 72 devices as breakthrough and authorized the marketing of six.

“Among those products was a brain implant for patients with blindness caused by damage to the optic nerve. The product mimics the perception of light through a miniature video camera worn by a patient that transmits signals to an implant in their visual cortex,” he explained.

The breakthrough designation facilitated early interactions between FDA and the sponsor and brought together intra-agency specialists to pose questions, solve problems and evaluate the benefits and risks of the device for which no standard existed, he noted.

In addition, as of June 30, FDA’s Center for Biologics Evaluation and Research (CBER) has granted 24 RMAT designations since the program’s inception.

Gottlieb noted that Chimeric Antigen Receptor T-cell (CAR-T) products have been considered a form of gene therapy, so the RMAT designation is available to CAR-T products that meet the other criteria for designation.



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