A new publication from FDA Commissioner Scott Gottlieb and director of the FDA's Center for Biologics Evaluation and Research Peter Marks describes the ‘balancing act’ required to prevent major negative effects whilst promoting innovation. Titled “Balancing Safety and Innovation for Cell-Based Regenerative Medicine”, the article provides clarity on terms, and updates on Regenerative Medicine Advanced Therapy (RMAT) designations and how the FDA could deal with unregulated clinics in the future.
Whether or not a cell therapy has undergone “minimal manipulation” and is for “homologous” are key determinants in how the FDA decides to regulate it. In the article, Marks and Gottlieb state that products that will be regulated by the FDA do not include products that “do not undergo substantial processing (minimal manipulation), are used in a manner in the recipient that is similar to that in the donor (homologous use), are not combined with another drug or biologic product, and do not have a systemic effect, unless they are designed for autologous transplantation, first- or second-degree–related allogeneic transplantation, or reproductive use”. This could clarify the area of uncertainty over instances where cells are minimally manipulated but are not used in a homologous way; for example, bone marrow stem cells for COPD.
Learn more about the regulation of cell therapy and regenerative medicine:
Another interesting topic touched upon was the awarding of RMAT designations and the compliance of clinics. The article indicated that up to the end of last year, 43 requests for RMAT designation were granted of which 35 were acted upon and 13 were granted. Marks and Gottlieb also indicated in the article that tools could be provided to “encourage individual or small groups of physicians to collaborate in support of the development of a stem-cell or other regenerative medicine product, which will ultimately lead to the receipt of a biologics license by each of the physicians or groups”.
Could this be a path for unapproved clinics to legitimize their treatments? The number of RMAT designations is set to rise as more and more potential therapies come up the pipeline and with them are likely to be more unscrupulous clinics touting unapproved and unregulated treatments. With just two warning letters under their belt already, this article may be a sign that the FDA will be cracking down in the future.
Source: Marks P, Gottlieb S. Balancing Safety and Innovation for Cell-Based Regenerative Medicine. N. Engl. J. Med. 378:954-959 (2018)