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How FDA’s regenerative medicine framework puts the U.S., Japan and Europe on the same page

FDA’s regenerative medicines framework could level the global regulatory playing field for products like cell and gene therapies. It describes approaches to implementing expedited development and review pathways that make them comparable, and in some ways more attractive than those available in Japan and Europe.

The framework, released in November, reflects commitments from FDA Commissioner Scott Gottlieb and the agency’s senior career staff to promote technologies they believe can transform the treatment of many intractable diseases and conditions.

At the same time, the framework reflects the agency’s determination to protect patients and the emerging field of regenerative medicine from hucksters peddling phony cures and from organizations and individuals seeking to take dangerous shortcuts around regulation.

The framework builds on provisions of the 21st Century Cures Act, especially those that created the regenerative medicine advanced therapy (RMAT) designation. RMAT commits FDA to collaborate early in the product development cycle with sponsors to design clinical trials, address quality and manufacturing issues, and delineate a streamlined path to an approval decision. It is similar to, but easier to obtain than breakthrough designation.

FDA’s framework stretches 21st Century Cures by making technologies that are not explicitly specified in the law eligible for RMAT. These include chimeric antigen receptor (CAR) T, as well as certain gene therapies and gene editing products.

The framework links RMAT to expedited development and review pathways, especially accelerated approval. And it describes how FDA will implement provisions in 21st Century Cures that permit the agency to consider allowing sponsors to use real-world evidence to confirm clinical efficacy for RMAT-designated products following accelerated approval.

Taken together, the expansive criteria for obtaining RMAT and the flexible approaches to obtaining accelerated approval and confirming clinical efficacy could create a system in the U.S. that gets products to patients as efficiently as Japan’s conditional approval system for regenerative medicines.

FDA also has signaled its intention to take the global lead in defining innovative clinical trial strategies for regenerative medicines. These strategies could take the sting out of the agency’s decision to regulate procedures such as therapies based on autologous adipose tissue, which some physicians believe should be unregulated.


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