FDA Regenerative Advanced Therapy Designation Rules Are Up in Response to Cures Act

The 21st Century Cures Act specifies some new FDA rules about investigational stem cell-based regenerative medicine therapies and perhaps most importantly defined a new class of product called a “Regenerative Advanced Therapy” or RAT, which is entitled to an expedited review.

The FDA already has a new page up on RATs, which specifies the specifics of RAT designation and how an entity may submit an application for RAT designation. This was remarkably fast of the FDA.fda-rat-designation

Some things on the RATs page stand out including that a request for a RAT depends on an IND. The IND must either be existing or submitted along with the RAT request. This is a major point as obtaining an IND requires rigorous, controlled data.

Stem cell businesses out to make a quick buck off of patients and potentially hypothetically interested in get RATs are going to be out of luck unless they change their world view to be data-centric and also go for INDs.

Also, the FDA is emphasizing that the condition in question must be “serious of life-threatening” and the FDA decides on what meets this criteria.

Oddly, the FDA will not require data be part of the RAT itself. I don’t quite get this, although perhaps their thinking is that the data requirement of the IND will suffice.

1 Comment


  1. Thanks for posting this. It’s still a little vague, but it appears to keep the bar high for stem cell treatments. Do you think that this will affect the bogus clinics at all? They will still claim that they don’t need FDA regulation.

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