Will the New Law Boost Stem Cell Products and Other Regenerative Therapies?   
​[Posted on: Thursday, December 8, 2016]

Regenerative therapies are set to receive renewed attention and attractive regulatory incentives thanks to the 21st Century Cures Act (Cures Act) which is all but certain to become the law this week. The new law defines “regenerative medicine therapy” to be cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products. Regulations for products which are currently subject to PHS section 361 and 21 CFR 1271 as HCT/Ps will stay unchanged. The law directs FDA to create a new designation for products called the “regenerative advanced therapy” and grant them accelerated approval. A product designated as regenerative advanced therapy would be eligible for expedited development program, accelerated approval, and priority review. Only regenerative products that intend to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and for which preliminary clinical evidence indicates that the product has the potential to address unmet medical needs for such a disease or condition, will be eligible for this new designation. So, in effect it is similar to the breakthrough therapy designation for drugs and biologics. Only products that are being investigated under an IND with the FDA will be eligible for this incentive, so HCT/P products that are marketed without a formal FDA approval under 21 CFR 1271, and therapies in pre-IND stage will not be eligible. Also, preliminary clinical data will be critical for the advanced regenerative therapy designation. FDA will get to make the final decision on the specific conditions a product must meet to be designated a regenerative advanced therapy just the way it does for the breakthrough designation. The new law seems quite attractive on its face, but the fine print should be carefully considered. The new law does not address the controversial topic of the definition of an HCT/P under 1271. Recently, there has been extensive debate over FDA’s narrow definition of products regulated under 1271, and those requiring IND and BLA/NDA. FDA has indicated that it considers practically all autologous cell therapies to be subject to the IND/BLA rules. The law is designed to help conventional cell therapy products being investigated under INDs, however, since very few such therapies have so far shown significant promise in clinical trials, it may not have a significant impact on the field of regenerative therapies where reports indicate that most promising products come from autologous cell therapy. However, FDA does not agree. In an article published last week, FDA senior officials re-emphasized the often-challenged assertion that the risk of autologous therapies is similar to that of conventional stem cell therapies. Recently FDA created a special office to regulate cellular therapies including regenerative therapies possibly in preparation for the new provisions of the Cures Act, but without a clear direction on autologous cellular therapies with significant clinical experience, this development only creates more hurdles for discussions to resolve this controversial issue at hand. This law is not going to help the numerous doctors and companies providing autologous cell based therapy options. The law also does not include the critical provision of the Regrow Act that would have created a conditional approval pathway for regenerative products that have been shown to be safe, and allowed marketing of products while the clinical trials under an IND are ongoing. The regenerative therapy provisions of the Cures Act failed to address the contentious aspects of regulations governing regenerative medicine, thereby limiting its impact on regenerative therapy products, in our opinion.