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Regenerative medicine advanced therapy designation - supporting expedition in drug development

In addition to the pathways such as Fast Track designation, Accelerated Approval, Priority Review designation and Breakthrough Therapy designation programs, FDA has an additional pathway to assist industry in expediting drug development, called Regenerative Medicine Advanced Therapy designation (RMAT, formerly known as RAT).

The RMAT designation was implemented as a provision of the 21st Century Cures Act. This act was enacted into law on December 13, 2016 and is described in Title III, Subtitle D – Patient Access to Therapies and Information, Section 3033, entitled “Accelerated Approval for Regenerative Advanced Therapies”.  

A drug is eligible for regenerative medicine advanced therapy (RMAT) designation if it meets the following criteria:

  • Cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products
  • Intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition
  • Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.

Benefits to drug developers

According to Section 3033 of the 21St Century Cures Act, RMAT provides the designee with:

  • Early interactions to discuss any potential surrogate or intermediate endpoint to be used to support the accelerated approval of an application for the product Eligibility for priority review
  • Eligibility for accelerated approval including surrogate or intermediate endpoints reasonably likely to predict long-term clinical benefit  and reliance upon data obtained from a meaningful number of sites

Application process

The request for RMAT designation must be made either concurrently with submission of an Investigational New Drug application (IND) or as an amendment to an existing IND. FDA will not grant a RMAT designation if an IND is on hold or is placed on hold during the designation review.

FDA does not expect submission of primary data (data sets), but the request for regenerative advanced therapy designation should describe the preliminary clinical evidence. The designation request should 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 analyses (e.g., subgroup analyses).

The Office of Tissues and Advanced Therapies (OTAT) will notify the sponsor no later than 60 calendar days after receipt of the designation request,  as to whether RMAT designation has been granted. If OTAT determines that the RMAT designation request was incomplete or that the drug development program does not meet the criteria for RMAT designation, OTAT will include a written description of the rationale for such determination.

Post approval requirements

The sponsor of a regenerative advanced therapy that is granted accelerated approval is subject to post approval requirements, such as:

  • Submission of clinical evidence, clinical studies, patient registries, or other sources of real world evidence, such as electronic health records
  • Collection of larger confirmatory data sets,
  • Post approval monitoring of all patients treated with such therapy prior to approval of the therapy

Need more information or support to apply?

ICON provides Regulatory Affairs services to draft and submit the Regenerative Medicine Advance Therapy designation request on your behalf. Please contact Enquiries@iconplc.com to discuss your needs and learn how we can help you navigate the process.

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