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

KNL Avery, PR Williamson, C Gamble, EOC Francischetto, C Metcalfe, P Davidson, H Williams, JM Blazeby, N Blencowe, C Bugge, M Campbell, M Collinson, C Cooper, J Darbyshire, M Dimairo, C Dore, S Eldridge, A Farrin, N Foster, S GilbodyS Goodacre, L Hampson, AG Kolias, S Lamb, A Lane, L Maguire, J Norrie, R Pickering, GW Shorter, S Treweek

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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.
Original languageEnglish
Pages (from-to)e013537
JournalBMJ Open
Issue number2
Publication statusAccepted/In press - 19 Jan 2017


  • randomised control trials
  • randomized control trials
  • pilot studies
  • guidance


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