欧洲先进治疗药物的监管：我们是否走在正确的轨道上？

Regulation of advanced therapies in Europe: Are we on the right track?

机构信息

Donostia University Hospital and Biodonostia Health Research Institute, Advanced Therapies Unit, Paseo Dr. Begiristain s/n, 20014, Donostia-San Sebastian, Spain.

Andalusian Transplant Coordination, Servicio Andaluz de Salud, Avda. de la Constitución 18, 41071, Seville, Spain.

出版信息

Cell Stem Cell. 2023 Aug 3;30(8):1013-1016. doi: 10.1016/j.stem.2023.07.004.

DOI:10.1016/j.stem.2023.07.004

PMID:37541207

Abstract

Sixteen years after regulating advanced therapy medicinal products (ATMPs) in the European Union, few ATMPs have gained marketing authorization. Additionally, market withdrawals for commercial reasons and a lack of reimbursement are de facto blocking patient access. Here, we pinpoint the major factors underlying this roadblock and how to circumvent it.

摘要

在欧盟对先进治疗药物产品（ATMPs）进行监管 16 年后，获得营销授权的 ATMP 寥寥无几。此外，出于商业原因的市场撤出和缺乏报销实际上阻碍了患者的获得途径。在这里，我们指出了造成这一障碍的主要因素以及如何规避这些因素。