Progression criteria in trials with an internal pilot: an audit of publicly funded randomised controlled trials
Progression criteria in trials with an internal pilot: an audit of publicly funded randomised controlled trials
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DOI：
10.1186/s13063-019-3578-y
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发表时间：
2019-08-09
期刊：
影响因子：
2.5
通讯作者：
Goodacre, Steve
中科院分区：
文献类型：
--
作者：
Herbert, Esther;Julious, Steven A.;Goodacre, Steve
英国每年花费数百万英镑用于医学研究，因此明智地使用研究资金非常重要。内部试点提供了提前停止试验的机会，如果很明显，该研究将无法招募足够的患者来证明干预措施是否具有临床效果。本研究旨在评估由卫生技术评估（HTA）计划资助的个体随机对照试验中内部试点的使用情况，并总结这些试验中选择的进展标准。方法从2012年至2016年卫生技术评估委员会资助决定的报告中确定研究。总共确定了242项试验，其中134项符合纳入稽查的条件。合格研究的方案位于NIHR期刊网站上，如果方案无法在线获得，则联系研究经理提供信息。结果超过三分之二（72.4%）的研究在其方案中表示，他们将包括一个内部试点阶段的研究和37.8%的研究没有一个内部试点进行了外部试点研究，以评估整个研究的可行性。一项典型的内部试点研究的目标样本量为510人，为期24个月，目标是在招募时间的前三分之一内招募总目标样本量的五分之一。近年来，采用三层结构进行进展规则的研究有所增加，2016年61.5%（16/26）的研究使用该系统，而2015年仅为11.8%（2/17）。在进展标准中给出目标招募率的研究数量也有所增加：2016年为42.3%（11/26），而2015年为35.3%（6/17）。结论内部试点的进展标准通常是明确的，但目标差异很大。对于实际标准，近年来红/黄/绿色系统越来越受欢迎。试验应证明其设定的目标是正确的，特别是在目标较低的情况下。
Background With millions of pounds spent annually on medical research in the UK, it is important that studies are spending funds wisely. Internal pilots offer the chance to stop a trial early if it becomes apparent that the study will not be able to recruit enough patients to show whether an intervention is clinically effective. This study aims to assess the use of internal pilots in individually randomised controlled trials funded by the Health Technology Assessment (HTA) programme and to summarise the progression criteria chosen in these trials. Methods Studies were identified from reports of the HTA committees' funding decisions from 2012 to 2016. In total, 242 trials were identified of which 134 were eligible to be included in the audit. Protocols for the eligible studies were located on the NIHR Journals website, and if protocols were not available online then study managers were contacted to provide information. Results Over two-thirds (72.4%) of studies said in their protocol that they would include an internal pilot phase for their study and 37.8% of studies without an internal pilot had done an external pilot study to assess the feasibility of the full study. A typical study with an internal pilot has a target sample size of 510 over 24 months and aims to recruit one-fifth of their total target sample size within the first one-third of their recruitment time. There has been an increase in studies adopting a three-tiered structure for their progression rules in recent years, with 61.5% (16/26) of studies using the system in 2016 compared to just 11.8% (2/17) in 2015. There was also a rise in the number of studies giving a target recruitment rate in their progression criteria: 42.3% (11/26) in 2016 compared to 35.3% (6/17) in 2015. Conclusions Progression criteria for an internal pilot are usually well specified but targets vary widely. For the actual criteria, red/amber/green systems have increased in popularity in recent years. Trials should justify the targets they have set, especially where targets are low.