US Stem Cell Inc (USRM)

[Havoc23]

RAT Designation Process

Requests for RAT designation can be made concurrently or at any time after submission of an IND. FDA is required to respond within 60 days. If CBER’s Office of Tissues and Advanced Therapies (“OTAT”) determines that a RAT designation request is incomplete or that the drug development program does not meet the criteria for designation, it will inform the sponsor and include a written description of the rationale for its determination. FDA’s RAT website also provides that:

Sponsors should refer to FDA’s Expedited Programs Guidance 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.”
Consistent with fast track and breakthrough therapy designations, FDA does not expect that sponsors submit primary data. Instead, FDA requests that sponsors describe the preliminary clinical evidence.
Sponsors should provide 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).
Requests for RAT designation should be submitted to OTAT.
The cover letter for a request should clearly specify that the submission contains a “REQUEST FOR REGENERATIVE ADVANCED THERAPY DESIGNATION” in bold, upper case letters.