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.