Ballreich Jeromie, Socal Mariana, Bennett Charles L, Xuan Andrew, Trujillo Antonio, Anderson Gerard
Department of Health Policy & Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland, USA.
Center for Drug Safety and Effectiveness, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland, USA.
Pharmacotherapy. 2023 Apr;43(4):300-304. doi: 10.1002/phar.2789. Epub 2023 Mar 16.
We evaluated US Food and Drug Administration labels for drugs approved under the accelerated approval pathway and whether these labels contained in sufficient information regarding their accelerated approval.
Retrospective, observational, cohort study.
Label information for drugs with an accelerated approved indication were ascertained from two online platforms: Drugs@FDA and FDA Drug Label Repository.
Drugs with indications receiving accelerated approval after January 1, 1992, but had not received full approval by December 31, 2020.
Outcomes include whether the drug label indicated the use of the accelerated approval pathway, identified the specific surrogate marker(s) that supported it, or described the clinical outcomes being evaluated in post-approval commitment trials.
253 clinical indications corresponding to 146 drugs received accelerated approval. We identified a total of 110 accelerated approval indications across 62 drugs that had not received full approval by December 31, 2020. A total of 13% of labels for accelerated approved indications lacked sufficient information that approval was via the accelerated approval or based on surrogate outcome measures: 7% did not mention accelerated approval but described surrogate markers, 4% did not mention accelerated approval nor describe surrogate markers, and 2% mentioned accelerated approval but did not describe surrogate markers. No label described the clinical outcomes being evaluated in post-approval commitment trials.
Labels for accelerated approved clinical indications that do not yet have full approval should be revised to include the information required in the FDA guidance to help guide clinical decision-making.
我们评估了美国食品药品监督管理局（US Food and Drug Administration）对通过加速批准途径获批药物的标签，以及这些标签是否包含有关其加速批准的充分信息。
回顾性观察队列研究。
具有加速批准适应症的药物的标签信息从两个在线平台确定：Drugs@FDA和FDA药品标签库。
1992年1月1日之后获得加速批准适应症，但在2020年12月31日之前尚未获得完全批准的药物。
结果包括药物标签是否表明使用了加速批准途径、确定支持该途径的具体替代指标，或描述在批准后承诺试验中正在评估的临床结果。
对应146种药物的253个临床适应症获得了加速批准。我们确定了截至2020年12月31日尚未获得完全批准的62种药物中的总共110个加速批准适应症。加速批准适应症的标签中，共有13%缺乏关于批准是通过加速批准或基于替代结果指标的充分信息：7%未提及加速批准但描述了替代指标，4%既未提及加速批准也未描述替代指标，2%提及了加速批准但未描述替代指标。没有标签描述在批准后承诺试验中正在评估的临床结果。
对于尚未获得完全批准的加速批准临床适应症的标签，应进行修订，以纳入FDA指南中要求的信息，以帮助指导临床决策。
