An investigational drug is eligible for RMAT designation if:
It meets the definition of regenerative medicine therapy
It is intended to treat, modify, reverse, or cure a serious condition; and
Preliminary clinical evidence indicates that the regenerative medicine therapy has the potential to address unmet medical needs for such condition. [13]
Regarding the preliminary clinical evidence to demonstrate the potential of a regenerative medicine therapy to address unmet medical needs, the FDA generally expects that such evidence would be obtained from clinical investigations specifically conducted to assess the effects of the therapy on a serious condition. When determining whether the preliminary clinical evidence is sufficient to support RMAT designation, CBER considers factors including but not limited to:
The rigor of data collection
The consistency and persuasiveness of the outcomes
The number of patients or subjects
The number of sites contributing to the data
The severity, rarity, or prevalence of the condition
As opposed to Breakthrough Therapy designation, the RMAT designation does not require evidence to indicate that the drug may offer a substantial improvement over available therapies. In order to apply for RMAT designation, a sponsor should submit a request to CBER either with an IND or in an IND amendment.
Pro tip
CBER will not accept requests for RMAT designation for INDs that are inactive or on clinical hold. Additionally, the FDA will not further process a pending RMAT designation request for an IND that is placed on inactive or hold status while the designation request is under review.