HHS Seeks Input on Regulating Deceased-Donor Islet Cell Therapies

The U.S. Department of Health and Human Services (HHS) is requesting information on how to regulate deceased-donor islet cells and islet cell products for treating type 1 diabetes. FDA and the Health Resources and Services Administration (HRSA) issued the request jointly, and it is scheduled for publication in the Federal Register on Thursday.

HHS is asking whether enough publicly available data exist to set standards for developing and transplanting these therapies and to support their safe and effective use. It also asks whether regulation should differ between isolated, otherwise unmodified islet cells and islet cell products.

The notice also seeks input on defining unmodified deceased-donor islet cells as organs. Under that approach, HRSA would regulate their procurement, allocation and transplantation through the Organ Procurement and Transplantation Network (OPTN), rather than FDA regulating them through a biologics license application (BLA). The OPTN would then need standards for safety, effectiveness, processing quality and consistency, communicable disease control and surveillance.

Currently, sponsors must submit a BLA to market an unmodified deceased-donor islet cell product. HHS cited FDA's 1998 request for cord blood outcome data as an example of a standards-based approach. That request led to guidance setting licensure standards for certain cord blood products.

The notice also includes questions on source material quality, manufacturing controls, real-world evidence and postmarketing requirements and asks whether reclassifying islets as organs could set a precedent for other donor-derived cellular products. An additional question is whether the OPTN framework, built for vascularized organs, suits processed cellular therapies.

Comments are due Nov. 8.