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Regenerative Medicine Designation More Useful 'Tool' Than CBER’s Marks Initially Anticipated

21 May 2021

Biologics center director was skeptical that RMAT would offer much beyond US FDA's existing breakthrough therapy pathway, but he has since come to appreciate the flexibility offered by less stringent designation criteria and leeway in the types of potential confirmatory evidence.

Tool belt — RMAT is a nice tool to have in the regulatory tool belt, CBER's Peter Marks said. • Source: Alamy

The US Food and Drug Administration’s Regenerative Medicine Advanced Therapy (RMAT) expedited pathway has evolved into a more useful “tool” than Center for Biologics Evaluation and Research Director Peter Marks originally expected when the program was established in December 2016 through the 21st Century Cures Act.

Speaking at the Food and Drug Law Institute annual meeting on 20 May, Marks said he initially was skeptical that...