February 15, 2019
“We’re at an inflection point when it comes to cell and gene therapies. These treatments have the potential to address hundreds of vexing human diseases and conditions. When we first issued our comprehensive regenerative medicine policy framework in November 2017, our goal was to achieve a balanced and risk-based approach to support product development in cell-based therapies, while clarifying the FDA’s authorities and enforcement priorities to make sure we were protecting patients,” said FDA Commissioner Scott Gottlieb, M.D. “Providing a clear and predictable pathway to approval is a critical part of removing uncertainties for companies and fostering development of innovative, safe and effective products. The final guidances issued today demonstrate our continued commitment to fulfilling this promise. We’re providing information to product developers regarding how the agency intends to evaluate devices used with regenerative medicine advanced therapies, as well as providing information on the expedited development programs that may be available to regenerative medicine therapies targeted to serious conditions. Helping stakeholders understand our regulatory framework may help to more efficiently advance access to safe and effective regenerative medicine therapies. We know that cell-based therapies and gene therapies may hold transformative promise for patients. This is why the FDA is so committed to encouraging and supporting safe and effective innovation in this field.”
Today, the FDA finalized two guidance documents regarding regenerative medicine therapies. Both documents were first issued in draft in November 2017 as part of a suite of guidances that comprised the FDA’s comprehensive regenerative medicine policy framework.
The first final guidance, Evaluation of Devices Used with Regenerative Medicine Advanced Therapies, which is required by the 21st Century Cures Act, clarifies how the FDA will evaluate devices used in the recovery, isolation or delivery of regenerative medicine advanced therapies (RMATs). The guidance specifies that devices intended for use with a specific RMAT may, together with the RMAT, be considered to comprise a combination product.
The second final guidance, Expedited Programs for Regenerative Medicine Therapies for Serious Conditions, describes the expedited programs that may be available to sponsors of regenerative medicine therapies for serious conditions, including the new Regenerative Medicine Advanced Therapy (RMAT) designation created by the 21st Century Cures Act, Priority Review designation and Accelerated Approval. In addition, the guidance describes the regenerative medicine therapies that may be eligible for RMAT designation.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.