Choosing the target difference ('effect size') for a randomised controlled trial - DELTA(2) guidance protocol.
Choosing the target difference ('effect size') for a randomised controlled trial - DELTA(2) guidance protocol.
复制标题
DOI：
10.1186/s13063-017-1969-5
复制
发表时间：
2017-06-12
期刊：
影响因子：
2.5
通讯作者：
Vale LD
中科院分区：
文献类型：
--
作者：
Cook JA;Julious SA;Sones W;Rothwell JC;Ramsay CR;Hampson LV;Emsley R;Walters SJ;Hewitt C;Bland M;Fergusson DA;Berlin JA;Altman D;Vale LD
随机对照试验（RCT）设计的一个关键步骤是估计所需的受试者人数。到目前为止，最常见的方法是指定目标差异，然后估计相应的样本量;选择该样本量是为了保证试验将具有较高的统计学把握度来检测随机分组之间的差异（在计划的统计学显著性水平）。样本量对研究的进行有许多影响，以及对其选择进行科学和伦理方面的影响。尽管在随机对照试验的设计中，主要结局的目标差异起着关键作用，但其确定方式却很少受到关注。本文报告了TriAls差异提取（DELTA 2）项目的方案，该方案将为RCT样本量计算中主要结局的目标差异的规范和报告提供指导。DELTA 2项目有五个组成部分：对最近的方法学发展（第一阶段）和现有资助者指南（第二阶段）进行系统的文献综述;一项德尔菲研究（第三阶段）;一次为期两天的共识会议，将研究人员、资助者和患者代表聚集在一起，以及在相关利益相关者会议上举行一次性的参与会议（第四阶段）;以及编写和分发指导文件（第5阶段）。主要结局的目标差异说明是RCT设计的关键组成部分。有必要为研究人员和资助者提供更好的指导，以规范和报告试验设计的这一方面。该项目的目的是为研究人员和资助者提供基于共识的指导。本文的在线版本（doi：10.1186/s13063-017-1969-5）包含补充材料，可供授权用户使用。
A key step in the design of a randomised controlled trial (RCT) is the estimation of the number of participants needed. By far the most common approach is to specify a target difference and then estimate the corresponding sample size; this sample size is chosen to provide reassurance that the trial will have high statistical power to detect such a difference between the randomised groups (at the planned statistical significance level). The sample size has many implications for the conduct of the study, as well as carrying scientific and ethical aspects to its choice. Despite the critical role of the target difference for the primary outcome in the design of an RCT, the manner in which it is determined has received little attention. This article reports the protocol of the Difference ELicitation in TriAls (DELTA2) project, which will produce guidance on the specification and reporting of the target difference for the primary outcome in a sample size calculation for RCTs. The DELTA2 project has five components: systematic literature reviews of recent methodological developments (stage 1) and existing funder guidance (stage 2); a Delphi study (stage 3); a 2-day consensus meeting bringing together researchers, funders and patient representatives, as well as one-off engagement sessions at relevant stakeholder meetings (stage 4); and the preparation and dissemination of a guidance document (stage 5). Specification of the target difference for the primary outcome is a key component of the design of an RCT. There is a need for better guidance for researchers and funders regarding specification and reporting of this aspect of trial design. The aim of this project is to produce consensus based guidance for researchers and funders. The online version of this article (doi:10.1186/s13063-017-1969-5) contains supplementary material, which is available to authorized users.