Sully, B.G.O., Julious, S.A. and Nicholl, J. (2014) An investigation of the impact of futility analysis in publicly funded trials. Trials, 15. ARTN 61. ISSN 1745-6215
Abstract
Background
Publicly funded trials regularly fail to recruit their target sample size or find a significant positive result. Adaptive clinical trials which may partly mediate against the problems are not often applied. In this paper we investigate the potential of a form of adaption in a clinical trial - a futility analysis - to see if it has potential to improve publicly funded trials.
Methods
Outcome data from trials funded by two UK bodies, the Health Technology Assessment (HTA) programme and the UK Medical Research Council (MRC), were collected. These data were then used to simulate each trial with a single futility analysis using conditional power, undertaken after 50% to 90% of the patients had been recruited. Thirty-three trials recruiting between 2002 and 2008 met the inclusion criteria. Stopping boundaries of conditional powers of 20%, 30% and 40% were considered and outcomes included the number of trials successfully stopped and number of patients saved.
Results
Inclusion of a futility analysis after 75% of the patients had been recruited would have potentially resulted in 10 trials, which went on to have negative results, correctly stopping for futility using a stopping boundary of 30%. A total of 807 patients across all the trials would potentially have been saved using these futility parameters. The proportion of studies successfully recruiting would also have increased from 45% to 64%.
Conclusions
A futility assessment has the potential to increase efficiency, save patients and decrease costs in publicly funded trials. While there are logistical issues in undertaking futility assessments we recommend that investigators should aim to include a futility analysis in their trial design wherever possible.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © Sully et al.; licensee BioMed Central Ltd. 2014. This article is published under license to BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly credited. 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: | Adaptive designs; Futility; Recruitment; Efficiency; Health technology assessment; Medical research council |
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: | 29 Jul 2016 12:57 |
Last Modified: | 29 Mar 2018 13:03 |
Published Version: | http://dx.doi.org/10.1186/1745-6215-15-61 |
Status: | Published |
Publisher: | BioMed Central |
Refereed: | Yes |
Identification Number: | 10.1186/1745-6215-15-61 |
Related URLs: | |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:83446 |