Abstract
The Food and Drug Administration (FDA) has been regulating human islets for allotransplantation as a biologic drug in the US. Consequently, the requirement of a biological license application (BLA) approval before clinical use of islet transplantation as a standard of care procedure has stalled the development of the field for the last 20 years. Herein, we provide our commentary to the multiple FDA’s position papers and guidance for industry arguing that BLA requirement has been inappropriately applied to allogeneic islets, which was delivered to the FDA Cellular, Tissue and Gene Therapies Advisory Committee on 15 April 2021. We provided evidence that BLA requirement and drug related regulations are inadequate in reassuring islet product quality and potency as well as patient safety and clinical outcomes. As leaders in the field of transplantation and endocrinology under the “Islets for US Collaborative” designation, we examined the current regulatory status of islet transplantation in the US and identified several anticipated negative consequences of the BLA approval. In our commentary we also offer an alternative pathway for islet transplantation under the regulatory framework for organ transplantation, which would address deficiencies of in current system.
Highlights
The Food and Drug Administration (FDA) decided to regulate human islets for transplantation as biological drugs in the United States (US) and published several position papers and a guidance for industry [1,2,3,4]
In Canada, Australia, Japan, and throughout Europe [9], islets are regulated as organs or tissue for transplantation, and these regions have already adapted clinical islet transplantation as a standard of care procedure for patients
We foresee the following downstream consequences: Transplant centers will have no alternative source of islets for clinical use; Transplant centers will have no control over the quality of islets for their patients; Access to islet transplantation will be limited due to increased costs and decreased availability of islets compared to a situation where islets are regulated as organs and are exempt from biological license application (BLA)
Summary
The Food and Drug Administration (FDA) decided to regulate human islets for transplantation as biological drugs in the United States (US) and published several position papers and a guidance for industry [1,2,3,4]. The FDA will effectively allow the commercialization of human islets that will be used for transplantation This is unprecedented in the field of organ transplantation, where the sale of kidneys, for example, is at least unethical and at most, a criminal offense. The Islets for US Collaborative comprises more than 50 medical experts and leaders in the fields of transplantation and diabetes from leading US academic institutions who have longstanding concerns about the regulatory status of islet transplantation in the US (www.isletsforus.org). Our intention is to share our concerns and recommendations with the broad medical community, government, and regulatory officials as well as our patients, and hopefully stimulate considerations of appropriate regulatory adjustments. Our intention is to share our concerns and recommendatio the broad medical community, government, and regulatory officials as well as tients, and hopefully stimulate considerations of appropriate regulatory adjustme
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