4:52 PM
 | 
Dec 15, 2017
 |  BioCentury  |  Regulation

Growth medium

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.

Guiding and expediting

FDA’s regenerative medicine framework is spelled out in two final and two draft guidance documents (see “Guiding Regeneration”).

Regenerative therapy developers were as cheered by the comments FDA released with the guidances as by they were by the contents of the documents.

In a statement, Gottlieb said that cell-based therapies “hold transformative promise for patients.” He added: “I believe that with the ability to facilitate the regeneration of parts of the human body, we’re bearing witness to the beginning of a paradigm shift in the practice of medicine.”

“We’re bearing witness to the beginning of a paradigm shift in the practice of medicine.”

Scott Gottlieb, FDA

FDA broke the most new ground in its draft guidance on “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions.”

The document puts in black and white the agency’s decision to extend RMAT beyond the specific technologies Congress included in the 21st Century Cures Act.

The law makes RMAT available to “cell therapies, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products.”

FDA is interpreting that language to include “gene therapies, including genetically modified cells, that lead to a durable modification of cells or tissues.” It is also willing to give RMAT designation to products that combine an RMAT-eligible biologic with a drug or device if the “biological product component provides the greatest contribution to the overall intended therapeutic effects of the combination product.”

FDA’s decision to include gene therapies and gene editing products “was a real interpretation of the statute, it was not required,” Kate Cook, EVP for drug and biological products at regulatory consultancy Greenleaf Health Inc., told BioCentury.

“By expressly including gene therapies within the category of products eligible for RMAT -- and not just ex vivo treatments but also in vivo treatments that use gene editing -- FDA is clearly making sure that its expedited approval programs are available to the most innovative and cutting edge biotechnologies that are being developed,” Michael Werner, executive director of the Alliance for Regenerative Medicine (ARM), told BioCentury.

The framework “demonstrates...

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