Global Regulatory and R&D (GRR&D) Policy, Amgen, Inc., 601 13th Street NW Suite 1100 North, Washington, DC, 20005, USA.
Ther Innov Regul Sci. 2022 Sep;56(5):698-703. doi: 10.1007/s43441-022-00430-z. Epub 2022 Jul 28.
The accelerated approval pathway has been criticized recently for employing lower regulatory standards than traditional drug approval, undue delays in withdrawing approvals of drugs for which studies have not confirmed clinical benefit, and confirmatory trials not being pursued with due diligence. This article examines the status of confirmatory studies of drugs approved under the US Food and Drug Administration's (FDA's) accelerated approval program between December 1992 and December 2021. It includes background on the program and provides broader context about the program's performance to date over its 30-year history. Our analysis demonstrates that the accelerated approval program has been largely successful, with half of accelerated approvals converted to traditional approval in a median time of 3.2 years. Furthermore, recent FDA actions show that the agency is appropriately managing the program when a drug approved under accelerated approval fails to confirm a clinical benefit. Any proposed changes to the program should be based on cumulative experience with the program, rather than outliers.
加速审批途径最近受到批评，因为它采用的监管标准低于传统药物审批，对研究未能证实临床益处的药物的批准撤回不当延迟，以及没有勤勉地进行确证性试验。本文检查了 1992 年 12 月至 2021 年 12 月期间美国食品和药物管理局（FDA）加速审批计划批准的药物的确证性研究的现状。它包括该计划的背景，并提供了更广泛的背景，说明该计划在 30 年的历史中迄今为止的表现。我们的分析表明，加速审批计划在很大程度上是成功的，有一半的加速批准在中位数为 3.2 年的时间内转化为传统批准。此外，最近 FDA 的行动表明，当根据加速审批批准的药物未能确认临床益处时，该机构正在适当地管理该计划。对该计划的任何拟议变更都应基于该计划的累积经验，而不是异常值。
