Regenerative Advanced Therapy Designation

As described in Section 3033 of the 21st Century Cures Act, a drug is eligible for designation as a regenerative advanced therapy (RAT) 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

We refer you to the Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics, section III.A., for FDA’s interpretation of whether a disease or condition is serious or life-threatening and whether a drug is intended to treat a serious disease or condition.

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. We will not grant a RAT designation if an IND is on hold or is placed on hold during the RAT designation review.

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. 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).

You may submit a request for designation as a regenerative advanced therapy to:

Food and Drug Administration
Center for Biologics Evaluation and Research
Office of Tissues and Advanced Therapies
Document Control Center
10903 New Hampshire Avenue
WO71, G112
Silver Spring, MD 20993-0002

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 receipt of the designation request, the Office of Tissues and Advanced Therapies (OTAT) will notify the sponsor as to whether RAT designation has been granted. If OTAT determines that the RAT designation request was incomplete or that the drug development program does not meet the criteria for designation as a regenerative advanced therapy, OTAT will include a written description of the rationale for such determination.