The demise of islet allotransplantation in the United States: A call for an urgent regulatory update

Am J Transplant. 2021 Apr;21(4):1365-1375. doi: 10.1111/ajt.16397. Epub 2021 Feb 10.

Abstract

Islet allotransplantation in the United States (US) is facing an imminent demise. Despite nearly three decades of progress in the field, an archaic regulatory framework has stymied US clinical practice. Current regulations do not reflect the state-of-the-art in clinical or technical practices. In the US, islets are considered biologic drugs and "more than minimally manipulated" human cell and tissue products (HCT/Ps). In contrast, across the world, human islets are appropriately defined as "minimally manipulated tissue" and not regulated as a drug, which has led to islet allotransplantation (allo-ITx) becoming a standard-of-care procedure for selected patients with type 1 diabetes mellitus. This regulatory distinction impedes patient access to islets for transplantation in the US. As a result only 11 patients underwent allo-ITx in the US between 2016 and 2019, and all as investigational procedures in the settings of a clinical trials. Herein, we describe the current regulations pertaining to islet transplantation in the United States. We explore the progress which has been made in the field and demonstrate why the regulatory framework must be updated to both better reflect our current clinical practice and to deal with upcoming challenges. We propose specific updates to current regulations which are required for the renaissance of ethical, safe, effective, and affordable allo-ITx in the United States.

Keywords: clinical research/practice; ethics and public policy; islet transplantation; islets of Langerhans; law/legislation; quality of care/care delivery.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Biological Products*
  • Costs and Cost Analysis
  • Diabetes Mellitus, Type 1* / surgery
  • Humans
  • Islets of Langerhans Transplantation*
  • Transplantation, Heterologous
  • United States

Substances

  • Biological Products