可行性研究中的样本量论证:超越已发表的指导意见。

IF 1.5 Q3 MEDICINE, RESEARCH & EXPERIMENTAL
Robert Montgomery
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引用次数: 0

摘要

试点和可行性研究对于确定是否应进行后续试验至关重要。为了有效,这些研究需要适当的设计,并有足够大的样本,以做出正确的决定,以高速率进行未来的试验。然而,与通常由功效分析证明的大型随机对照试验相比,关于样本量证明的最佳实践的清晰度明显较低。这导致许多可行性研究根据实际原因(例如,完成的能力)或公布的指南和各种经验法则来证明样本量的合理性。我们认为,可行性研究的设计应考虑或至少报告相关的操作特征,特别是确定未来试验可行时的可能性,以及确定试验不可行时的可能性,并且样本量的论证应尽可能基于这些操作特征。如果一项研究的相关操作特征没有报告,就很难知道这些试验的证据是否可以用来宣称未来的试验是可行的。在本文中,我们回顾了可行性研究中确定样本量的常用方法,报告了最近发表的可行性研究的叙述性回顾,并使用两个案例研究来突出我们认为在这些研究的当前设计和报告中存在的核心问题。最后,我们提供了一些建议,我们相信这些建议将增强这些重要试验的样本量合理性和整体有效性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Sample size justification in feasibility studies: moving beyond published guidance.

Pilot and feasibility studies are crucial for determining whether follow-up trials should be conducted. To be effective, these studies need to be properly designed and have large enough samples to make correct decisions about proceeding to a future trial at a high rate. However, there is significantly less clarity about best practices concerning sample size justifications compared to larger randomized controlled trials which are usually justified by power analyses. This has led to many feasibility studies justifying sample sizes based on pragmatic reasons (e.g., the ability to complete) or on published guidance and various rules of thumb. We argue that feasibility studies should be designed for, or at least report relevant operating characteristics, specifically, the probability of determining a future trial will be feasible when it is and the probability of determining a trial will be feasible when it is not and that sample size justifications should be based on these operating characteristics whenever possible. If relevant operating characteristics for a study are not reported, it is difficult to know whether the evidence from these trials can be used to claim a future trial would be feasible. In this paper, we review common methods for determining samples sizes in feasibility studies, report a narrative review of recently published feasibility studies and use two case-studies to highlight core issues we believe exist in the current design and reporting of these studies. Finally, we provide some suggestions that we believe will enhance the sample size justifications and overall effectiveness of these important trials.

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来源期刊
Pilot and Feasibility Studies
Pilot and Feasibility Studies Medicine-Medicine (miscellaneous)
CiteScore
2.70
自引率
5.90%
发文量
241
审稿时长
9 weeks
期刊介绍: Pilot and Feasibility Studies encompasses all aspects of the design, conduct and reporting of pilot and feasibility studies in biomedicine. The journal publishes research articles that are intended to directly influence future clinical trials or large scale observational studies, as well as protocols, commentaries and methodology articles. The journal also ensures that the results of all well-conducted, peer-reviewed, pilot and feasibility studies are published, regardless of outcome or significance of findings. Pilot and feasibility studies are increasingly conducted prior to a full randomized controlled trial. However, these studies often lack clear objectives, many remain unpublished, and there is confusion over the meanings of the words “pilot” and “feasibility”. Pilot and Feasibility Studies provides a forum for discussion around this key aspect of the scientific process, and seeks to ensure that these studies are published, so as to complete the publication thread for clinical research.
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