支持 FDA 加速批准的单臂临床试验具有适度的效应量，且存在较高的偏倚风险。
Single-arm clinical trials that supported FDA accelerated approvals have modest effect sizes and were at high risk of bias.
机构信息
Department of Epidemiology. School of Public Health. University of Sao Paulo, Brazil.
Department of Clinical Medicine, Universidad de Guadalajara, Guadalajara Jalisco, México.
出版信息
J Clin Epidemiol. 2022 Aug;148:193-195. doi: 10.1016/j.jclinepi.2022.01.018. Epub 2022 Jan 31.
OBJECTIVE
To describe effect sizes of single-arm clinical trials that supported AA approvals.
STUDY DESIGN AND SETTING
We reviewed all the single-arm approvals granted by the FDA-AA pathway between June1992 to December2020. Two independent reviewers identified single-arm studies and extracted data from FDA Full-Medical Reviews. We performed a meta-analysis to estimate the effect sizes and compared it between studies that met post-approval FDA requirements for RCTs with those that did not.
RESULTS
From the total of 254 approvals, single arm clinical trials describing effects of 54 drugs for 72 clinical indications were evaluated. The effect size estimated was OR:2.22(CI95%:1.76-2.81) [relative risk (RR) = 1.63(95CI% 1.38-1.92)]; 53% of treatments had a lower 95% CI bound crossing the null effect. Effect size did not differ between the treatments that met the FDA requirement for conducting post-approval RCTs.
CONCLUSIONS AND RELEVANCE
Treatment effects observed in the FDA AA single-arm studies was modest and can be to ascribed to bias.
目的
描述支持 AA 批准的单臂临床试验的效应量。
研究设计与设置
我们回顾了 1992 年 6 月至 2020 年 12 月期间通过 FDA-AA 途径获得的所有单臂批准。两位独立的审查员确定了单臂研究，并从 FDA 完整医疗审查中提取数据。我们进行了荟萃分析来估计效应量，并比较了符合 FDA 对 RCT 后批准要求的研究与不符合要求的研究之间的效应量。
结果
在总共 254 项批准中，评估了描述 72 种临床适应症的 54 种药物疗效的单臂临床试验。估计的效应量为 OR：2.22（CI95%：1.76-2.81）[相对风险（RR）=1.63（95CI% 1.38-1.92)]；53%的治疗方法的 95%置信区间下限低于零，表明有疗效。符合 FDA 进行后批准 RCT 要求的治疗方法之间的效应量没有差异。
结论和相关性
在 FDA AA 单臂研究中观察到的治疗效果适中，可归因于偏倚。
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