A New Expedited Review Designation: Regenerative Advanced Therapy

By Michelle Villasmil, Ph.D., RAC (U.S.), Regulatory Scientist

One of the many changes brought by the 21st Century Cures Act is the new expedited review designation entitled Regenerative Advanced Therapy (with the unfortunate acronym of RAT). FDA provides brief guidance for a RAT designation on their website (provided below). A drug is eligible for RAT designation if:

  • “the drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations;
  • the drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
  • preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.”1,2

While the benefits of RAT designation are less clearly defined than its more established expedited review designation counterparts (i.e., breakthrough therapy, fast track, accelerated approval, priority review), FDA does outline the following benefits of RAT designation:

  • early interactions to discuss potential surrogate or intermediate endpoints to support accelerated approval of an application;
  • eligibility for priority review; and
  • eligibility for accelerated approval.1

Primary data is not required to apply for RAT designation, but the RAT designation request should at least describe preliminary clinical evidence. The RAT designation request can be submitted with an Investigational New Drug application (IND) or as an amendment to an existing, cleared IND. The submission process for RAT designation requests is detailed on the FDA website.2

Guidance on RAT designation:

  1. 21st Century Cures Act, Section 3033
  2. http://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ucm537670.htm
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