Informing efficient randomised controlled trials: exploration of challenges in developing progression criteria for internal pilot studies.
Informing efficient randomised controlled trials: exploration of challenges in developing progression criteria for internal pilot studies.
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DOI：
10.1136/bmjopen-2016-013537
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发表时间：
2017-02-17
期刊：
影响因子：
2.9
通讯作者：
members of the Internal Pilot Trials Workshop supported by the Hubs for Trials Methodology Research
中科院分区：
文献类型：
--
作者：
Avery KN;Williamson PR;Gamble C;O'Connell Francischetto E;Metcalfe C;Davidson P;Williams H;Blazeby JM;members of the Internal Pilot Trials Workshop supported by the Hubs for Trials Methodology Research
设计具有内部试点阶段的研究可以优化试点工作的使用，为更有效的随机对照试验（RCT）提供信息。在内部试点和主要试验阶段之间仔细选择预先商定的决定或“进展”标准，为评估主要试验的可能成功和优化其设计，或在必要时做出不继续进行主要试验的决定提供了宝贵的机会。然而，缺乏关于适当选择和应用进展标准的指导。本文概述了在内部试点阶段随机对照试验的操作进展标准的优化制定和审查中需要考虑的关键问题。在医学研究委员会 (MRC) 试验方法研究中心研讨会上对利益相关者的意见进行结构化文献回顾和探讨。主要利益相关者包括试验参与者、方法学家、统计学家和资助者。尽管有 3 个常见问题占主导地位：试验招募、方案遵守情况和结果数据，但内部试验阶段的随机对照试验进展标准的使用存在相当大的差异。关于停止、修改或进行主要试验的决策过程的详细和系统的报告并不常见，这可能会妨碍研究界对内部试验阶段随机对照试验的适当和最佳使用的理解。提出了制定、使用和报告内部试点研究进展标准的 10 个重要技巧。应鼓励在文献中系统、透明地报告内部试点试验的设计、结果和评估，以促进研究界的理解并为未来的试验提供信息。
Designing studies with an internal pilot phase may optimise the use of pilot work to inform more efficient randomised controlled trials (RCTs). Careful selection of preagreed decision or ‘progression’ criteria at the juncture between the internal pilot and main trial phases provides a valuable opportunity to evaluate the likely success of the main trial and optimise its design or, if necessary, to make the decision not to proceed with the main trial. Guidance on the appropriate selection and application of progression criteria is, however, lacking. This paper outlines the key issues to consider in the optimal development and review of operational progression criteria for RCTs with an internal pilot phase. A structured literature review and exploration of stakeholders' opinions at a Medical Research Council (MRC) Hubs for Trials Methodology Research workshop. Key stakeholders included triallists, methodologists, statisticians and funders. There is considerable variation in the use of progression criteria for RCTs with an internal pilot phase, although 3 common issues predominate: trial recruitment, protocol adherence and outcome data. Detailed and systematic reporting around the decision-making process for stopping, amending or proceeding to a main trial is uncommon, which may hamper understanding in the research community about the appropriate and optimal use of RCTs with an internal pilot phase. 10 top tips for the development, use and reporting of progression criteria for internal pilot studies are presented. Systematic and transparent reporting of the design, results and evaluation of internal pilot trials in the literature should be encouraged in order to facilitate understanding in the research community and to inform future trials.