Regenerative Medicine Therapies – Final Guidances

By:  Kinsey S. Reagan

On February 15, 2019, FDA issued two final guidance documents for regenerative medicine advanced therapy (RMAT) products (Evaluation of Devices Used with Regenerative Medicine Advanced Therapies and Expedited Programs for Regenerative Medicine Therapies for Serious Conditions).  Having been proposed as draft guidance in November 2017, the relatively quick finalization of the guidances illustrates the importance of this growing field of medicine to FDA.

The first final guidance, as required by Section 3034 of the 21st Century Cures Act, addresses how FDA will evaluate devices that are used in the recovery, isolation, or delivery of RMATs.  The guidance covers a range of topics including:

  • The classification of devices that are not reviewed solely under a BLA as a constituent part of a biologic-led combination product;
  • The available premarket clearance pathways for clearance of such devices;
  • The application of “least burdensome” principles of regulation;
  • The appropriate procedure for submitting combination products for review; and
  • Factors to consider in determining whether a device may be used with only one particular type of cell-based RMAT or with many types, or conversely, whether a particular RMAT can be used with only a particular device or with a general class of devices.

Although the guidance is written in general terms and does not refer to specific RMATs or specific intended uses or attributes of RMATs, it nevertheless is a useful reminder of how these existing FDA policies will apply in the context of RMATs.

The second final guidance, issued on February 15, 2019, summarizes the expedited review programs that are potentially available for regenerative medicine therapies for serious conditions, including those meeting the criteria for designation as RMATs.  These include the accelerated approval pathway established for RMATs by the 21st Century Cures Act, as well as the applicability of FDA’s established expedited pathways (e.g., fast track, breakthrough therapy, priority review).  In addition to the general discussion, the guidance provides details on the procedures and data needed to obtain RMAT designation.

The second guidance also discusses considerations for clinical trial design in the context of regenerative medicine products and FDA’s recommendations for interactions between sponsors and the Office of Tissues and Advanced Therapies review staff.

We would be happy to assist affected companies, institutions, and other entities in evaluating the final guidances and their potential impact on the premarket clearance process for specific regenerative medicine-related products.