The US Food and Drug Administration (FDA) on Thursday unveiled a new designation category created as part of the 21st Century Cures Act to allow drugs to be eligible for designation as a regenerative advanced therapy (RAT).
The stipulations for such a RAT designation include:
- “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.”
In terms of how to know if a company might qualify for such a designation, FDA points to its guidance on Expedited Programs for Serious Conditions – Drugs and Biologics, section III.A.
The guidance notes four other expedited designations: “Fast Track,” which includes actions to speed up development and review, “Breakthrough Therapy,” which features intensive guidance on efficient drug development, “Accelerated Approval,” which is based on “an effect on a surrogate endpoint or an intermediate clinical endpoint that is reasonably likely to predict a drug’s clinical benefit,” and “Priority Review,” which shortens the review clock from 10 months to six months.
The request for RAT designation must be made either concurrently with submission of an Investigational New Drug application (IND) or as an amendment to an existing IND, FDA says.
“We generally do not expect you to submit primary data (data sets), but your request for designation as a RAT should describe the preliminary clinical evidence,” FDA adds. “Please 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).”
In terms of preparing the submission, FDA notes: “If the RAT designation request is submitted to your IND as an amendment, the cover letter should specify that the submission contains a REQUEST FOR REGENERATIVE ADVANCED THERAPY DESIGNATION in bold, uppercase letters. If the request is submitted with an initial IND, the cover letter should specify that the submission contains both an INITIAL INVESTIGATIONAL NEW DRUG SUBMISSION and REQUEST FOR REGENERATIVE ADVANCED THERAPY DESIGNATION in bold, upper case letters.”
No later than 60 calendar days after receiving the designation request, FDA’s newly created Office of Tissues and Advanced Therapies (OTAT) will notify the sponsor if the designation has been granted. If the RAT designation request is incomplete or the drug development program does not meet the criteria for designation as a regenerative advanced therapy, OTAT says it will include a written description of the rationale for such determination.
Regenerative Advanced Therapy Designation