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Characteristics of pre-approval and post-approval studies for drugs granted accelerated approval by the US food and drug administration

机译：美国食品和药物管理局加速批准的药品批准前和批准后研究的特征

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Importance: Drugs treating serious conditions can receive US Food and Drug Administration (FDA) Accelerated Approval based on showing an effect in surrogate measures that are only reasonably likely to predict clinical benefit. Confirmatory trials are then required to determine whether these effects translate to clinical improvements. \udObjective: To characterize pre-approval and confirmatory clinical trials of drugs granted Accelerated Approval. \udDesign and Setting: Publicly available FDA documents were surveyed to evaluate the pre-approval trials leading to Accelerated Approval between 2009 and 2013. Information on the status and findings of required confirmatory studies was extracted from the FDA’s database of postmarketing requirements and commitments, ClinicalTrials.gov, and matched publications. End date of follow up was 7 April 2017.\udExposure: Granting of Accelerated Approval. \udMain Outcomes and Measures: Characteristics of pre-approval and confirmatory studies were compared in terms of study design features (randomization, blinding, comparator, primary endpoint) and indications. Subsequent regulatory decisions and estimated time between Accelerated Approval and fulfillment of regulatory requirements were reviewed. \udResults: FDA granted Accelerated Approval to 22 drugs for 24 indications in the study period. At a minimum 3 years of follow-up, 19 of 38 required confirmatory studies were completed (50%). The proportion of studies with randomized designs did not differ before and after Accelerated Approval (16%, 95% confidence interval [CI]: -15%-46%; P=0.31). Post-approval requirements were completed and demonstrated efficacy in 10 indications (42%) on the basis of trials that evaluated surrogate measures alone. Among the 14 indications (58%) that had not yet completed requirements, confirmatory studies failed to demonstrate clinical benefit in 2 (8%) indications; were terminated in 2 (8%); and were delayed by more than one year in 3 (13%) with no regulatory action. Studies were progressing according to target timelines for the remaining 7 indications (29%). Clinical benefit had not yet been confirmed for 7 indications that had been initially approved 5 or more years prior. \udConclusions and Relevance: Many drugs recently granted Accelerated Approval had had their efficacy confirmed in post-approval trials, although confirmatory trials have similar design elements to pre-approval trials, including reliance on surrogate measures as outcomes. Delays in completing post-approval confirmatory trials persisted for a minority of drugs.
机译：重要性：治疗严重疾病的药物可以获得美国食品和药物管理局（FDA）的加速批准，因为它显示出替代措施的效果，而该替代措施只能合理地预测临床获益。然后需要进行验证性试验以确定这些作用是否转化为临床改善。 \ ud目的：表征获得加速批准的药物的批准前和验证性临床试验。 \ ud设计与设置：对FDA公开文件进行了调查，以评估导致2009年至2013年间加速批准的批准前试验。所需验证性研究的状态和结果信息摘自FDA上市后要求和承诺数据库，ClinicalTrials。 .gov和相匹配的出版物。随访的结束日期是2017年4月7日。\ udExposure：授予加速批准。主要结果和措施：根据研究设计特征（随机化，盲法，比较者，主要终点）和适应症比较批准前和确证研究的特征。审查了随后的监管决定以及加速批准与达到监管要求之间的估计时间。 \ ud结果：在研究期间，FDA批准了22种药物的24种适应症的加速批准。在至少3年的随访中，完成了38项需要确认的研究中的19项（50％）。在加速批准前后，采用随机设计的研究比例没有差异（16％，95％置信区间[CI]：-15％-46％； P = 0.31）。在仅评估替代指标的试验的基础上，完成了批准后要求，并在10个适应症中证明了疗效（42％）。在尚未完成要求的14项适应症中（58％），验证性研究未能证明2项适应症（8％）的临床获益。被2（8％）终止；并且有3个（13％）延迟了一年多，没有采取任何监管措施。其余7项适应症（29％）的研究均按照目标时间表进行。尚未确认5年前或更早批准的7种适应症的临床获益。 \ ud结论和相关性：尽管确认性试验的设计要素与批准前的试验相似，包括依赖替代措施作为结果，但最近获得加速批准的许多药物的功效已在批准后的试验中得到证实。少数药物持续延迟完成批准后确认试验。

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