The FDA has created a policy framework for cellular therapies and other regenerative medicines by releasing two draft and two final guidance documents. Publication of the texts builds on the 21st Century Cures Act by setting criteria for the new Regenerative Medicine Advanced Therapy (RMAT) designation and outlining the benefits of the regulatory status.
Given two of the documents are finalized versions of existing texts and a third seeks to simplify the regulation of devices that enable regenerative treatments, the RMAT guidance (PDF) is the novel part of the framework for drug developers. In the guidance, the FDA takes the RMAT status created by the 21st Century Cures Act, contextualizes it against other regulatory designations and explains how and why companies may want to apply.
RMAT is open to investigational regenerative medicine therapies that treat, modify, reverse or cure serious conditions and have generated promising preliminary clinical evidence of efficacy. The FDA cites a single-arm, open-label study of a skin burn cell therapy and a phase 2, dose-finding trial of a heart failure regenerative medicine as examples of the types of experiments that could provide preliminary evidence of efficacy.
Candidates that receive RMAT status will receive all the benefits of the FDA’s fast track and breakthrough designation. The law that created RMAT also covers accelerated approvals and how to satisfy the subsequent post-approval requirements.
The FDA is accepting comments on the RMAT text and draft device guidance for 90 days.
While aspects of the framework are intended to ensure effective regenerative medicines reach patients as soon as possible, FDA commissioner Scott Gottlieb, M.D., also framed it as an attempt to curb the rise of cellular therapies from unscrupulous providers.
“Alongside all the promise, we’ve also seen products marketed that are dangerous and have harmed people,” Gottlieb said in a statement. “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.”
The final guidance documents clarify when cell and tissue-based products are exempt from the established regulations and how the FDA interprets “minimal manipulation” and “homologous use.”