WIKIPEDIA, RYDDRAGYN The 21st Century Cures Act, approved by the US Congress plus signed into law by former President Barack Obama last December, includes a provision to speed up the advancement and approval of cell- and tissue-based therapies, tissues engineering products such as scaffolds, and combination treatments. Around this month, four firms’ products have earned this alleged regenerative medicine advanced therapy (RMAT) designation.
“ It really is very important…. You now have [a priority designation] that is very clearly for tissue and cell [therapies], ” Beth Roxland, an associate and a senior expert on law, ethics, and policy at the NYU Langone Medical School, told The Scientist. “ Overall, this piece of 21st Century Cures has been very much a step in the right direction for prioritizing possibly life-saving therapies. ”
The RMAT path (formerly called regenerative advanced therapy, or RAT) is definitely analogous to the breakthrough designation designed for traditional drug applicants and medical devices. Specifically, it will allow companies building regenerative medicine therapies to interact with the US Food and Medication Administration (FDA) earlier in the clinical testing process and much more frequently, and RMAT-designated products may be eligible for priority evaluation and accelerated approval.
“ The FDA’ s Center for Biologics Evaluation and Research is devoted to helping make regenerative medicine advanced therapies that are proved to be safe and effective available as soon as possible, particularly for patients with severe or life-threatening diseases or conditions lacking other treatment plans, ” Peter Marks, the center’ s director, published in an FDA blog post announcing the new process in Mar.
See “ Picking Up the Pace, ” January 2016
To qualify for the RMAT designation, the therapy must be intended to treat a serious or life-threatening disease and must have shown promising results in preclinical tests. To date, the FDA has received 17 applications for that RMAT designation, four of which have been granted, an agency spokesperson told The Scientist .
“ The fact that they’ ve already issued four of the in such a short time… it really seems like they’ re embracing this, ” said Paul Knoepfler, a stem cell biologist at the University of California, Davis.
Within March, for example , North Carolina– based Humacyte announced that this had received RMAT designation for its product HUMACYL, which usually uses smooth muscle cells from the aortic tissue associated with heart transplant donors to create a scaffold of extracellular matrix proteins. The material will then be transplanted into dialysis patients as a vascular prosthetic. “ In just recent several weeks, we’ ve had very, very good response from our [FDA] review team, ” said Bill Tente, vice president of quality, compliance, and regulatory matters at Humacyte. While HUMACYL received Fast Track Designation within 2014 and has had increased communication with the agency considering that, “ my sense is that some of that is coming from the RMAT designation. ”
In addition to the increased communication using the agency, Tente said he looks forward to having the ability to submit the company’ s biologic license application on the rolling basis, another perk of the designation that would permit researchers to get feedback on portions of a complete software. “ If you finish one section and you’ lso are able to send that off and get it reviewed, this is a very nice thing, ” he said. “ It possibly means your product would get to patients sooner, that is the whole thing behind the 21st Century Cures Act. ”
Of course , it will take some time to see how the new RMAT designation plays out in terms of new therapy approvals. “ If some of the RMAT therapies are ultimately proven to be effective and safe, and via RMAT they were able to get to patients plus help them more quickly, with few failures amongst the RMAT group, then the RMAT regulatory experiment will be proven a huge success, ” Knoepfler wrote on his lab’ s weblog, The Niche. “ We’ ll find out in a few years one method or another more clearly. ”
In the meantime, Roxland additional, “ there’ s still a whole bucket of issues over stem cells that this doesn’ t attempt to obtain into” — namely, the complex issue of how these items should be regulated overall and the continued existence of deceptive stem cell clinics in the U. S. and overseas. “ That the FDA is still very much wrestling with, ” she said.
Although the 21st Century Cures Respond doesn’ t touch on this regulatory issue, Roxland believed that the RMAT designation, in the long term, “ could have a positive, useful effect, ” she said. “ If you start obtaining more therapies on the market quicker or just more genuine remedies on the market via the RMAT designation or otherwise, then hopefully sufferers would have less of an impetus to go to the charlatans. ”
On the other hand, Knoepfler noted, the act includes vocabulary that allows companies to apply for RMAT designation on the basis of “ real-life evidence, ” as opposed to data from a preclinical study. “ A lot of [stem cell] clinics see a lot of individuals, ” he noted, and could potentially use those information to apply for RMAT. “ I would argue in some cases in the process associated with treating those hundreds of patients, they may have been in violation associated with FDA regulations…. We never quite know what the FOOD AND DRUG ADMINISTRATION is thinking, but they might find themselves in some interesting circumstances. ”
“ I think the general feeling amongst researchers is that if [the RMAT designation] benefits their particular study, it’ s good, but if it opens the doorway to charlatans, it’ s bad, ” Jeanne Loring, director of the Center for Regenerative Medicine at the Scripps Research Institute, told The Scientist in an email. “ Both will happen, I think. ”