Methodology State-of-the-art workshop: Choosing the target difference (“effect size”) for a randomised controlled trial

最先进的方法论研讨会：为随机对照试验选择目标差异（“效应大小”）

• 批准号: MC_PC_16101
• 负责人: Jonathan Cook
• 金额: $ 3.15万
• 依托单位: University of Oxford
• 依托单位国家: 英国
• 项目类别: Intramural
• 财政年份: 2016
• 资助国家: 英国
• 起止时间: 2016 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=MC_PC_16101
• 关键词: Methodology; State; art; workshop; Choosing

Randomised controlled trials (RCT) are considered the optimal study design to assess comparative clinical efficacy and effectiveness. A sample size calculation ensures the study has a reasonable chance to achieve its pre-specified objectives: A compromise is required between the possibility of being misled by chance (there is no true difference between treatments) and the risk of not identifying a difference even when one of the treatments is truly superior. The “target difference” is the size of the difference the RCT is designed to reliably investigate. It is also commonly referred to as the trial “effect size”. The critical question is whether a particular difference detected statistically, is of any practical interest. From a scientific and ethical standpoint, selecting an appropriate targeted difference is of crucial importance - specifying too small a target difference could be a wasteful (and perhaps unethical) use of resources, and too large could lead to an important difference being overlooked. Furthermore, the resultant undersized study will not produce reliable evidence.Despite there being many different approaches available to choose the target difference, few are used regularly in practice. The project will: 1. Review the existing guidance; 2. Identify key methodological developments or changes in practice; 3. Determine the scope of the guidance required; 4. Produce structured guidance for choosing and reporting the target difference (effect size)

随机对照试验（RCT）被认为是评估比较临床疗效和有效性的最佳研究设计。样本量计算确保研究有合理的机会实现其预先规定的目标：需要在被偶然误导的可能性（治疗之间没有真正的差异）和即使其中一种治疗确实具有上级优势也无法识别差异的风险之间进行折衷。“目标差异”是RCT旨在可靠研究的差异大小。它通常也被称为试验“效应量”。关键的问题是，统计学上检测到的特定差异是否具有任何实际意义。从科学和伦理的角度来看，选择一个适当的目标差异至关重要-指定太小的目标差异可能是对资源的浪费（也许是不道德的），而太大的目标差异可能导致一个重要的差异被忽视。此外，由此产生的小规模研究将不会产生可靠的证据。尽管有许多不同的方法可用于选择目标差异，但很少在实践中经常使用。该项目将：1。审查现有指南; 2.确定实践中的关键方法发展或变化; 3.确定所需指导的范围; 4.为选择和报告目标差异（效应量）提供结构化指导