The 21st Century Cures Act specifies some new FOOD AND DRUG ADMINISTRATION rules about investigational stem cell-based regenerative medicine treatments and perhaps most importantly defined a new class of product known as a “ Regenerative Advanced Therapy” or RAT, that is entitled to an expedited review.
The FOOD AND DRUG ADMINISTRATION already has a new page up on RATs, which specifies the specifics of VERWEIS designation and how an entity may submit an application for VERWEIS designation. This was remarkably fast of the FDA.
Some things on the RATs page stand out including that will a request for a RAT depends on an IND. The particular IND must either be existing or submitted combined with the RAT request. This is a major point as obtaining a good IND requires rigorous, controlled data.
Come cell businesses out to make a quick buck off of patients and possibly hypothetically interested in get RATs are going to be out of luck unless of course they change their world view to be data-centric as well as go for INDs.
Also, the FDA is certainly emphasizing that the condition in question must be “ serious associated with life-threatening” and the FDA decides on what meets this requirements.
Oddly, the FDA will not require information be part of the RAT itself. I don’ t very get this, although perhaps their thinking is that the data dependence on the IND will suffice.