Insights

Cures Corner: FDA Launches Regenerative Medicine Advanced Therapy Designation Program

In late January 2017, FDA took the first steps to implement certain provisions of the 21st Century Cures Act ("Act"), signed into law on December 13, 2016, by launching a new designation program for regenerative medicine therapies. Implemented pursuant to Section 3033 of the Act, the Regenerative Medicine Advanced Therapy ("RMAT") Designation program aims to help foster the development and approval of regenerative medicine products. Despite the program's newness, on March 20, 2017, FDA issued one of the first RMAT designations to Humacyte's investigational human acellular vessel, "Humacyl."

Under the new program, a drug is eligible for RMAT designation if: (i) the drug is a regenerative medicine therapy; (ii) the drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and (iii) preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition. A drug constitutes a "regenerative medicine therapy" if it is a cell therapy, therapeutic tissue, engineering product, human cell and tissue product, or any combination product using such therapies or products. The term does not include certain human cells, tissues, and cellular or tissue-based products regulated solely by Section 361 of the Public Health Service Act and Part 1271 of Title 21 of the Code of Federal Regulations. To determine whether a disease or condition is serious or life-threatening, or whether a drug is intended to treat such disease or condition, FDA refers to its existing guidance on Expedited Programs for Serious Conditions—Drugs and Biologics.

The RMAT designation offers a few important benefits to drug sponsors. First, sponsors of RMAT-designated products are eligible for increased and earlier interactions with FDA to expedite development and review of the therapy, similar to those interactions currently available to sponsors of products with a breakthrough therapy designation. In addition, RMAT-designated products may be eligible for priority review and accelerated approval by FDA. Finally, once approved, sponsors of RMAT-designated products can satisfy post-approval requirements through the submission of real-world clinical evidence, such as through clinical studies, patient registries, and electronic health records, the collection of larger confirmatory data sets, or post-approval monitoring of all patients treated with the therapy prior to its approval.

Sponsors of regenerative medicine therapies can request RMAT designation from FDA's Office of Tissues and Advanced Therapies either concurrently with, or at any time after, the submission of an Investigational New Drug ("IND") application or an amendment to an existing IND application. The FDA will not grant RMAT designation if an IND is on hold or is placed on hold during the designation review. In addition, although FDA does not expect sponsors to submit primary data sets, the request for RMAT designation should describe the preliminary clinical evidence and include a brief description of any available therapies for the disease or condition, the study design, the population studied, and the endpoint(s) used, and a description of the study results and statistical analysis. Once an RMAT request has been received, the FDA has 60 days to determine whether a drug meets the criteria.

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