At the end of 2016, the United States Congress passed the 21st Century Cures Act. Addressing some of the provisions included in the Act, the FDA followed through, on January 17, 2017, by introducing a new designation process for regenerative advanced therapies, called Regenerative Medicine Advanced Therapies (RMAT).
On a fundamental level, this new designation provides an easier path of approval which sees the FDA lower the bar for unmet medical need for serious or life-threatening diseases.
On one hand, advocates hope that the new law will make approval for new therapies less burdensome than the process required by the BLA application stream.
But just what does the new law entail? For a start, a “regenerative advanced therapy” is defined as:
“[…]The term ‘regenerative medicine therapy’ includes cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products, except for those regulated solely under section 361 of the Public Health Service Act and part 1271 8 of title 21, Code of Federal Regulations.”
The FDA further defines such drugs with the following criteria:
- The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
- Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition
The request for RMAT designation must be made either concurrently with submission of an Investigational New Drug (IND) application or as an amendment to an existing IND. The FDA does not require that applicants submit primary data with their RMAT applications.
How this will impact manufacturers of autologous and allogeneic cell therapies remains to be seen, but the early sentiment appears to be a sigh of relief.
The law requires the FDA to issue a guidance on the RMAT process within 1 year, while ongoing reports on the number of therapies approved each year is expected to be given by the Department of Health and Safety each year.
More information about RMATS and the application process can be found on the FDA website.