Government
Expanding on a regenerative medicine framework first introduced in 2005, the US FDA launched a new policy initiative aimed at efficiently providing cell, stem cell and tissue products to patients. Earlier this month, the FDA released two draft guidance documents detailing the methods in which approvals for regenerative medicines can be accelerated for patients with serious diseases, and for using medical devices with regenerative medicines. Two final guidance documents were also released that clarified the cases in which cell- and tissue-based products would be excluded from the regulations, as well as provided regulatory definitions of “homologous use” and “minimal manipulation.” With these guidelines, the FDA aims to support innovation in drug development, and to protect patients from unsafe and unproven therapies.
In one of the draft guidelines, the FDA Center for Biologics Evaluation and Research (CBER) detailed the Regenerative Medicine Advanced Therapy (RMAT) designation that was established in the 21st Century Cures Act. The RMAT is used to hasten the review process for cell- and tissue- products. The CBER has received 34 requests for RMAT designation, of which it has acted on 31 requests and granted 11 RMAT designations. However, an RMAT designation does not guarantee that the product will be approved, nor does it exempt the manufacturer from demonstrating the product’s efficacy and safety for approval. According to the draft guidance, the CBER plans to evaluate “the rigor of data collection; the nature and meaningfulness of the outcomes; the number of patients or subjects, and the number of sites, contributing to the data; and the severity, rarity, or prevalence of the condition” when it comes to determining products to be considered for RMAT designation.
