1012 来自SCI天天读 00:00 01:49
SCI
12 October 2019
US Food and Drug Administration review of statistical analysis of patient-reported outcomes in lung cancer clinical trials approved between January, 2008, and December, 2017
Fiero Mallorie H,Roydhouse Jessica K,Vallejo Jonathon et al. US Food and Drug Administration review of statistical analysis of patient-reported outcomes in lung cancer clinical trials approved between January, 2008, and December, 2017.[J] .Lancet Oncol., 2019, 20: e582-e589.
With the advent of patient-focused drug development, the US Food and Drug Administration (FDA) has redoubled its efforts to review patient-reported outcome (PRO) data in cancer trials submitted as part of a drug’s marketing application. This Review aims to characterise the statistical analysis of PRO data from pivotal lung cancer trials submitted to support FDA drug approval between January, 2008, and December, 2017. For each trial and PRO instrument identified, we evaluated prespecified PRO concepts, statistical analysis, missing data and sensitivity analysis, instrument completion, and clinical relevance. Of the 37 pivotal lung cancer trials used to support FDA drug approval, 25 (68%) trials included PRO measures. The most common prespecified PRO concepts were cough, dyspnoea, and chest pain. At the trial level, the most common statistical analyses were descriptive (24 trials [96%]), followed by time-to-event analyses (19 trials [76%]), longitudinal analyses (12 trials [48%]), and basic inferential tests or general linear models (10 trials [40%]). Our findings indicate a wide variation in the analytic techniques and data presentation methods used, with very few trials reporting clear PRO research objectives and sensitivity analyses for PRO results. Our work further supports the need for focused research objectives to justify and to guide the analytic strategy of PROs to facilitate the interpretation of patient experience.
随着以患者为中心的药物开发的出现，美国食品和药物管理局(FDA)加倍努力审查癌症试验中患者报告的结果(PRO)数据，这些数据是药物营销申请的一部分。本综述旨在对2008年1月至2017年12月期间为支持FDA药物批准而提交的关键肺癌临床试验PRO数据进行统计分析。对于每个确定的试验和PRO仪器，我们评估了预先指定的PRO概念、统计分析、缺失数据和敏感性分析、仪器完成情况和临床相关性。在37个用于支持FDA药物批准的关键肺癌试验中，有25个(68%)试验包括PRO措施。最常见的预先指定的专业概念是咳嗽、呼吸困难和胸痛。在试验层面，最常见的统计分析是描述性的(24个试验[96%])，其次是时间事件分析(19个试验[76%])、纵向分析(12个试验[48%])和基本推断试验或一般线性模型(10个试验[40%])。我们的研究结果表明，在分析技术和数据表示方法上存在很大的差异，很少有试验报告明确的PRO研究目标和PRO结果的敏感性分析。我们的工作进一步支持有针对性的研究目标的需要，以证明和指导专业人员的分析策略，以方便解释病人的经验。
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