Campbell, MJ, Lancaster, GA and Eldridge, SM (2018) A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint. Pilot and Feasibility Studies, 4. 130 - ?. ISSN 2055-5784

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Abstract

Background: It has been argued that true endpoints (or 'hard' endpoints) for clinical trials, which are meaningful to clinicians, researchers and patients alike, are limited to those that measure health status, survival and cost. Other endpoints are termed 'surrogate' endpoints and are intended to substitute and predict the true endpoint.  A number of trials that describe using surrogate endpoints use the term 'pilot' in the title of the paper but the reason for this, as related by the authors, is the use of these surrogate endpoints in the trial. The conduct and reporting of such a trial may follow the traditional pattern for a conventional randomised controlled trial (RCT) as defined by the original CONSORT statement, with power-based sample size calculations, and significance tests of the results. However, this is contrary to the guidelines of the CONSORT extension for the reporting of pilot trials. Main body: We review the definition of a surrogate endpoint and the use of surrogate endpoints in clinical trials. We consider to what extent a trial could be considered a pilot trial if it uses a surrogate endpoint and discuss two examples that illustrate current practice. Conclusion: Trials which use surrogate endpoints should only be described as 'pilot' when a definitive trial is a distinct possibility and the authors consider conditions which would indicate whether the definitive main trial was worthwhile and feasible. Simply because a trial uses a surrogate endpoint is not justification for calling it a pilot trial.

Item Type: Article
Additional Information: © The Author(s). 2018 Open Access 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.
Uncontrolled Keywords: Pilot trials; Surrogate endpoints; Randomised controlled trials; CONSORT
Subjects: R Medicine > RA Public aspects of medicine
Divisions: Faculty of Medicine and Health Sciences > Primary Care Health Sciences
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Depositing User: Symplectic
Date Deposited: 19 Sep 2018 14:12
Last Modified: 19 Sep 2018 14:12
URI: http://eprints.keele.ac.uk/id/eprint/5347

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