Witkowski Piotr, Odorico Jon, Pyda Jordan, Anteby Roi, Stratta Robert J, Schrope Beth A, Hardy Mark A, Buse John, Leventhal Joseph R, Cui Wanxing, Hussein Shakir, Niederhaus Silke, Gaglia Jason, Desai Chirag S, Wijkstrom Martin, Kandeel Fouad, Bachul Piotr J, Becker Yolanda Tai, Wang Ling-Jia, Robertson R Paul, Olaitan Oyedolamu K, Kozlowski Tomasz, Abrams Peter L, Josephson Michelle A, Andreoni Kenneth A, Harland Robert C, Kandaswamy Raja, Posselt Andrew M, Szot Gregory L, Ricordi Camillo
Transplantation Institute, Department of Surgery, University of Chicago, Chicago, IL 60637, USA.
Division of Transplantation, Department of Surgery, University of Wisconsin, School of Medicine and Public Health, Madison, WI 53792, USA.
J Clin Med. 2021 Jun 29;10(13):2878. doi: 10.3390/jcm10132878.
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.
在美国，食品药品监督管理局（FDA）一直将用于同种异体移植的人类胰岛作为生物药物进行监管。因此，在将胰岛移植作为一种标准治疗程序临床使用之前，需要获得生物制品许可申请（BLA）批准这一要求在过去20年里阻碍了该领域的发展。在此，我们针对FDA的多篇立场文件以及给业界的指南发表评论，认为BLA要求不适用于异体胰岛，该评论于2021年4月15日提交给了FDA细胞、组织和基因疗法咨询委员会。我们提供的证据表明，BLA要求和药品相关法规在确保胰岛产品质量和效力以及患者安全和临床结果方面存在不足。作为“美国胰岛协作组织”指定的移植和内分泌领域的领导者，我们研究了美国胰岛移植的当前监管状况，并确定了BLA批准的几个预期负面后果。在我们的评论中，我们还在器官移植监管框架下为胰岛移植提供了一条替代途径，这将解决当前系统的缺陷。
