在确定样本量计算中的目标差异时，临床重要性的重要性。
The importance of clinical importance when determining the target difference in sample size calculations.
机构信息
Edinburgh Clinical Trials Unit, Usher Institute, University of Edinburgh, Edinburgh, UK.
Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
出版信息
Trials. 2023 Aug 4;24(1):495. doi: 10.1186/s13063-023-07532-5.
Abstract
Recently, it was argued that clinically important differences should play no role in sample size calculations. Instead, it was proposed that sample size calculations should focus on setting realistic estimates of treatment benefit. We disagree, and argue in this article that considering the importance of a target difference is necessary in the context of randomised controlled trials of effectiveness, particularly definitive phase III trials. Ignoring clinical importance could have serious ethical and practical consequences.
摘要
最近有人认为，临床重要差异不应在样本量计算中发挥作用。相反，有人提议样本量计算应侧重于设定治疗效果的现实估计值。我们不同意这种观点，并在本文中认为，在有效性随机对照试验（特别是确定性 III 期试验）的背景下，考虑目标差异的重要性是必要的。忽视临床重要性可能会产生严重的伦理和实际后果。
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