The importance of clinical importance when determining the target difference in sample size calculations

Trials

Commentary

Jonathan A. Cook¹  Richard A. Parker² 

[1] Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK;Edinburgh Clinical Trials Unit, Usher Institute, University of Edinburgh, Edinburgh, UK;

关键词: Clinical trial;    RCT;    Power;    Effect size;    Assumed benefit;    Clinically relevant difference;    Minimum important difference;    Target difference;   

DOI  :  10.1186/s13063-023-07532-5

 received in 2023-05-25, accepted in 2023-07-20,  发布年份 2023

来源: Springer

【 摘 要 】

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.

【 授权许可】

CC BY   
© BioMed Central Ltd., part of Springer Nature 2023