Regulatory updates are needed to prevent the commercialization of islet transplantation in the United States

Islet transplantation in the United States – Quo Vadis? An interview with Camilo Ricordi (CR), Ali Naji (AN), Peter Stock (PS), Piotr Witkowski (PW)

“…Based on this rationale, human organs are, and human islets should also, be regulated under the aegis of the HRSA (Health Resources and Services Administration) and submitted to OPTN and UNOS (United Network for Organ Sharing) regulatorynotably outside the drug regulations of the FDA. This proposed framework was specially designed and developed to ensure human organ quality and potency, as well as safety and effectiveness of transplantation [1,11] How does this system ensure islet quality, safety, and effectiveness? AN: OPTN and UNOS set comprehensive policies and bylaws to regulate every element involved in the therapy, including the organizational structure, personnel, quality and safety measurements, and postprocedure monitoring.…”

“…Based on this rationale, human organs are, and human islets should also, be regulated under the aegis of the HRSA (Health Resources and Services Administration) and submitted to OPTN and UNOS (United Network for Organ Sharing) regulatory – notably outside the drug regulations of the FDA. This proposed framework was specially designed and developed to ensure human organ quality and potency, as well as safety and effectiveness of transplantation [1,11]…”

“…The current law – the National Organ Transplantation Act (NOTA) – was amended in 1988 to also include subparts of organs in the definition of human organ. However, the definition of human organ under the OPTN Final Rule has not been subsequently amended to include human islets, which are subparts of human pancreas [11]. Consequently, for the past 20 years, the FDA has taken the position that islets are a biological drug requiring BLA.…”

“…Moreover, there is precedence for this. In 2007, human blood vessels for organ transplantation and again, in 2013, human vascularized composite allografts were removed from FDA oversight and placed under OPTN/UNOS organ regulation [11‐13]. The latter was done for patient safety concerns, as we now advocate for islets.…”

Arguments against the Requirement of a Biological License Application for Human Pancreatic Islets: The Position Statement of the Islets for US Collaborative Presented during the FDA Advisory Committee Meeting

“…As a group of experts and leaders in the field of transplantation and endocrinology (Islets for US Collaborative) we strongly advise against the BLA approval and have warned of the negative consequences this would have for patient safety, as well as the field of islet transplantation. We also propose a regulatory path for human islets similar to the one that already exists for other human organs in the US where oversight would be provided by the Health Resources and Services Administration (HRSA) along with the Organ Procurement and Transplantation Network (OPTN) and the United Network for Organ Sharing (UNOS) [5,6]. However, the FDA recently rejected this proposal, and the BLA approval is imminent [7,8].…”