The RMAT Designation Seems to Be Tougher than the BTD
(Thursday, July 7, 2022)
When the Regenerative Medicine Advance Therapy (RMAT) designation was proposed in 2016, it was assumed to be an easier expedited approval pathway specific for stem cells and other regenerative therapies, compared to the Breakthrough Therapy (BTD) designation. But it with about 40% approval rates and only 3 approvals, it may be turning out to be tougher than the BTD to secure and get approval for. In the last 5 years, FDA has received 194 RMAT requests and granted 76 of them (~40%), and so far, there have been three RMAT designated products approved by the FDA. To be fair, it is still early to make broad assumptions regarding the pathway, but by comparison, BTDs have been very successful with 229 approvals of which 7 were approved within a year of the BTD designation. Overall, BTDs have a track record of approval within 2-4 years of the designation. By contrast, with 3 approvals in 5+ years of enactment, RMATs seems to be tougher to get approved. The three RMAT product approvals were for rare diseases with non-stem cell products. FDA has not publicly released a list of the RMAT designations, so it is hard to know the kind of products that received these. There are some privately created lists based on news releases by the successful applicants which tell a limited story. The RMAT’s success would be interesting to follow and compare to the BTDs. Unlike the BTD, RMAT seems to be almost entirely controlled by one division within FDA’s Center of Biologics (CBER), while the BTDs are designated by almost all the divisions in the Center for Drug (CDER). It will be all speculations till we get more data from the FDA about the fate of the RMATs. We will wait and report.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC