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Informing efficient randomised controlled trials: Exploration of challenges in developing progression criteria for internal pilot studies

Avery, KNL, Williamson, PR, Gamble, C, Francischetto, EOC, Metcalfe, C, Davidson, P, Williams, H, Blazeby, JM, Blencowe, N, Bugge, C, Campbell, M, Collinson, M, Cooper, C, Darbyshire, J, Dimairo, M, Dore, C, Eldridge, S, Farrin, A, Foster, N, Gilbody, S, Goodacre, S, Hampson, L, Kolias, AG, Lamb, S, Lane, A, Maguire, L, Norrie, J, Pickering, R, Shorter, GW and Treweek, S (2017) Informing efficient randomised controlled trials: Exploration of challenges in developing progression criteria for internal pilot studies. BMJ Open, 7 (2). e013537. [Journal article]

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URL: http://bmjopen.bmj.com/content/7/2/e013537

DOI: 10.1136/bmjopen-2016-013537

Abstract

Objectives: Designing studies with an internal pilotphase may optimise the use of pilot work to informmore efficient randomised controlled trials (RCTs).Careful selection of preagreed decision or ‘progression’criteria at the juncture between the internal pilot andmain trial phases provides a valuable opportunity toevaluate the likely success of the main trial andoptimise its design or, if necessary, to make thedecision not to proceed with the main trial. Guidanceon the appropriate selection and application ofprogression criteria is, however, lacking. This paperoutlines the key issues to consider in the optimaldevelopment and review of operational progressioncriteria for RCTs with an internal pilot phase.Design: A structured literature review and explorationof stakeholders’ opinions at a Medical ResearchCouncil (MRC) Hubs for Trials Methodology Researchworkshop. Key stakeholders included triallists,methodologists, statisticians and funders.Results: There is considerable variation in the use ofprogression criteria for RCTs with an internal pilotphase, although 3 common issues predominate: trialrecruitment, protocol adherence and outcome data.Detailed and systematic reporting around the decisionmakingprocess for stopping, amending or proceedingto a main trial is uncommon, which may hamperunderstanding in the research community about theappropriate and optimal use of RCTs with an internalpilot phase. 10 top tips for the development, use andreporting of progression criteria for internal pilotstudies are presented.Conclusions: Systematic and transparent reporting ofthe design, results and evaluation of internal pilot trialsin the literature should be encouraged in order tofacilitate understanding in the research community andto inform future trials.

Item Type:Journal article
Keywords:randomised control trials, randomized control trials, pilot studies, guidance
Faculties and Schools:Faculty of Life and Health Sciences
Faculty of Life and Health Sciences > School of Psychology
Research Institutes and Groups:Psychology Research Institute > Psychotraumatology, Mental Health & Suicidal Behaviour
Psychology Research Institute
ID Code:38745
Deposited By: Dr Gillian Shorter
Deposited On:11 Oct 2017 08:33
Last Modified:17 Oct 2017 16:31

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