Rothwell, J.C. orcid.org/0000-0001-6275-3807, Julious, S.A. and Cooper, C.L. (2018) A study of target effect sizes in randomised controlled trials published in the Health Technology Assessment journal. Trials, 19. 544.
Abstract
BACKGROUND: When designing a randomised controlled trial (RCT), an important consideration is the sample size required. This is calculated from several components; one of which is the target difference. This study aims to review the currently reported methods of elicitation of the target difference as well as to quantify the target differences used in Health Technology Assessment (HTA)-funded trials. METHODS: Trials were identified from the National Institute of Health Research Health Technology Assessment journal. A total of 177 RCTs published between 2006 and 2016 were assessed for eligibility. Eligibility was established by the design of the trial and the quality of data available. The trial designs were parallel-group, superiority RCTs with a continuous primary endpoint. Data were extracted and the standardised anticipated and observed effect size estimates were calculated. Exclusion criteria was based on trials not providing enough detail in the sample size calculation and results, and trials not being of parallel-group, superiority design. RESULTS: A total of 107 RCTs were included in the study from 102 reports. The most commonly reported method for effect size derivation was a review of evidence and use of previous research (52.3%). This was common across all clinical areas. The median standardised target effect size was 0.30 (interquartile range: 0.20-0.38), with the median standardised observed effect size 0.11 (IQR 0.05-0.29). The maximum anticipated and observed effect sizes were 0.76 and 1.18, respectively. Only two trials had anticipated target values above 0.60. CONCLUSION: The most commonly reported method of elicitation of the target effect size is previous published research. The average target effect size was 0.3. A clear distinction between the target difference and the minimum clinically important difference is recommended when designing a trial. Transparent explanation of target difference elicitation is advised, with multiple methods including a review of evidence and opinion-seeking advised as the more optimal methods for effect size quantification.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated. |
Keywords: | Effect size; HTA; Health technology assessment; Randomised controlled trial; Target difference |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > School of Health and Related Research (Sheffield) > ScHARR - Sheffield Centre for Health and Related Research |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 16 Oct 2018 15:41 |
Last Modified: | 16 Oct 2018 15:41 |
Published Version: | https://doi.org/10.1186/s13063-018-2886-y |
Status: | Published |
Publisher: | BMC |
Refereed: | Yes |
Identification Number: | 10.1186/s13063-018-2886-y |
Related URLs: | |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:137234 |