Wilson, DT, Walwyn, REA, Brown, J et al. (2 more authors) (2016) Statistical challenges in assessing potential efficacy of complex interventions in pilot or feasibility studies. Statistical Methods in Medical Research, 25 (3). pp. 997-1009. ISSN 0962-2802
Abstract
Early phase trials of complex interventions currently focus on assessing the feasibility of a large RCT and on conducting pilot work. Assessing the efficacy of the proposed intervention is generally discouraged, due to concerns of underpowered hypothesis testing. In contrast, early assessment of efficacy is common for drug therapies, where phase II trials are often used as a screening mechanism to identify promising treatments. In this paper we outline the challenges encountered in extending ideas developed in the phase II drug trial literature to the complex intervention setting. The prevalence of multiple endpoints and clustering of outcome data are identified as important considerations, having implications for timely and robust determination of optimal trial design parameters. The potential for Bayesian methods to help to identify robust trial designs and optimal decision rules is also explored.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © The Author(s) 2015. This is an author produced version of a paper published in Statistical Methods in Medical Research. Uploaded in accordance with the publisher's self-archiving policy. |
Keywords: | complex interventions; pilot; feasibility; phase II; sample size |
Dates: |
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Institution: | The University of Leeds |
Academic Units: | The University of Leeds > Faculty of Medicine and Health (Leeds) > School of Medicine (Leeds) > Inst of Clinical Trials Research (LICTR) (Leeds) |
Depositing User: | Symplectic Publications |
Date Deposited: | 12 May 2015 15:53 |
Last Modified: | 22 Jan 2018 15:14 |
Published Version: | https://dx.doi.org/10.1177/0962280215589507 |
Status: | Published |
Publisher: | SAGE Publications |
Identification Number: | 10.1177/0962280215589507 |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:84371 |