Yesterday, the U.S. Food and Drug Administration (FDA) announced a comprehensive framework for development and oversight of regenerative medicine products, including innovative cell-based therapies. This initiative builds on the agency’s existing framework to set forth more clearly which products are subject to regulatory enforcement. In September, as we previously discussed here and here, FDA’s Commissioner, Dr. Scott Gottlieb, prescribed a commitment for stepped-up enforcement against unscrupulous companies taking advantage of vulnerable patients. In yesterday’s press release, Dr. Gottlieb reaffirmed this commitment, and also stated that the agency supports the innovation being pursued by “responsible product developers” who represent the vast majority in the field. He emphasized the goal of providing greater clarity to facilitate understanding as to where the regulatory lines are drawn. FDA’s announcement included four guidance documents, two draft and two final, which defined a risk-based framework to drive advances in regenerative medicine and set forth how the agency intends to focus its enforcement actions against products that raise potentially significant safety concerns.
Despite the FDA’s strong language establishing this framework, the agency also stated in the final guidance relating to “minimal manipulation” and “homologous use” that the agency intends to exercise enforcement discretion, for violations of 21 CFR 1271, for a 3-year period, except for products that pose a potential significant safety concern. The apparent justification for the enforcement discretion is to provide time for product developers to obtain FDA approval. Unlike the agency’s September announcement on stem cell clinics, this one was not packaged with any new enforcement action announcements. We will be closely watching as FDA continues to implement the revised framework and will keep you posted should FDA determine that there are potential significant safety concerns that warrant enforcement.