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Lookup NU author(s): Jenni Hislop, Professor Luke ValeORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
Background Randomised controlled trials (RCTs) are widely accepted as the preferred study design for evaluating healthcare interventions. When the sample size is determined, a (target) difference is typically specified that the RCT is designed to detect. This provides reassurance that the study will be informative, i.e., should such a difference exist, it is likely to be detected with the required statistical precision. The aim of this review was to identify potential methods for specifying the target difference in an RCT sample size calculation. Methods and Findings A comprehensive systematic review of medical and non-medical literature was carried out for methods that could be used to specify the target difference for an RCT sample size calculation. The databases searched were MEDLINE, MEDLINE In-Process, EMBASE, the Cochrane Central Register of Controlled Trials, the Cochrane Methodology Register, PsycINFO, Science Citation Index, EconLit, the Education Resources Information Center (ERIC), and Scopus (for in-press publications); the search period was from 1966 or the earliest date covered, to between November 2010 and January 2011. Additionally, textbooks addressing the methodology of clinical trials and International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) tripartite guidelines for clinical trials were also consulted. A narrative synthesis of methods was produced. Studies that described a method that could be used for specifying an important and/or realistic difference were included. The search identified 11,485 potentially relevant articles from the databases searched. Of these, 1,434 were selected for full-text assessment, and a further nine were identified from other sources. Fifteen clinical trial textbooks and the ICH tripartite guidelines were also reviewed. In total, 777 studies were included, and within them, seven methods were identified—anchor, distribution, health economic, opinion-seeking, pilot study, review of the evidence base, and standardised effect size. Conclusions A variety of methods are available that researchers can use for specifying the target difference in an RCT sample size calculation. Appropriate methods may vary depending on the aim (e.g., specifying an important difference versus a realistic difference), context (e.g., research question and availability of data), and underlying framework adopted (e.g., Bayesian versus conventional statistical approach). Guidance on the use of each method is given. No single method provides a perfect solution for all contexts
Author(s): Hislop J, Adewuyi TE, Vale LD, Harrild K, Fraser C, Gurung T, Altman DG, Briggs AH, Fayers P, Ramsay CR, Norrie JD, Harvey IM, Buckley B, Cook JA
Publication type: Article
Publication status: Published
Journal: PLoS Medicine
Year: 2014
Volume: 11
Issue: 5
Pages: e1001645
Print publication date: 13/05/2014
Acceptance date: 04/04/2014
Date deposited: 12/08/2014
ISSN (electronic): 1549-1676
Publisher: Public Library of Science
URL: http://www.plosmedicine.org/article/info%3Adoi%2F10.1371%2Fjournal.pmed.1001645
DOI: 10.1371/journal.pmed.1001645
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