The FDA has combined the benefits of fast track and breakthrough designaton in a new policy framework for the regenrative medicine industry. Named the Regenerative Medicine Advanced Therapy (RMAT) designation, it will expedite the process for selected cell therapies to reach the market.
Four documents pertaining to the new framework have been published with three building on existing texts under the 21st Century Cures Act. The RMAT draft device guidance is a new text and responses to it will be accepted for 90 days. Topics covered include clarifications on when cell and tissue-based products are exempt from established regulations; and the use of the terms “minimal manipulation” and “homologous use.”
Investigational regenerative medicine therapies that treat, modify, reverse or cure serious conditions may apply for the new designation. To do so they must have produced preliminary evidence of efficacy with a single, open-label study of a skin burn cell therapy cited by the FDA as an acceptable example.
FDA commissioner Scott Gottlieb outlined that another intention of the new designation is to inhibit the rise of non-legitimate cell therapies: “Alongside all the promise, we’ve also seen products marketed that are dangerous and have harmed people. With the policy framework the FDA is announcing today, we’re adopting a risk-based and science-based approach that builds upon existing regulations to support innovative product development while clarifying the FDA’s authorities and enforcement priorities.”
Source: Building on Cures Act, FDA lays out cell therapy fast track in regenerative medicine framework Press Release