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

Kerry N L Avery*, Paula R. Williamson, Carrol Gamble, Elaine O Connell Francischetto, Chris Metcalfe, Peter Davidson, Hywel Williams, Jane M. Blazeby, Natalie Blencowe, Carol Bugge, Michael Campbell, Michelle Collinson, Cindy Cooper, Janet Darbyshire, Munya Dimairo, Caroline Doré, Sandra Eldridge, Amanda Farrin, Nadine Foster, Simon GilbodySteve Goodacre, Lisa Hampson, Angelos G. Kolias, Sallie Lamb, Athene Lane, Lisa Maguire, John Norrie, Ruth Pickering, Gillian Shorter, Shaun Treweek

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

66 Citations (Scopus)
320 Downloads (Pure)

Abstract

Objectives: Designing studies with an internal pilot phase may optimise the use of pilot work to inform more efficient randomised controlled trials (RCTs). Careful selection of preagreed decision or 'progression' criteria at the juncture between the internal pilot and main trial phases provides a valuable opportunity to evaluate the likely success of the main trial and optimise its design or, if necessary, to make the decision not to proceed with the main trial. Guidance on the appropriate selection and application of progression criteria is, however, lacking. This paper outlines the key issues to consider in the optimal development and review of operational progression criteria for RCTs with an internal pilot phase. Design: A structured literature review and exploration of stakeholders' opinions at a Medical Research Council (MRC) Hubs for Trials Methodology Research workshop. Key stakeholders included triallists, methodologists, statisticians and funders. Results: There is considerable variation in the use of progression criteria for RCTs with an internal pilot phase, although 3 common issues predominate: trial recruitment, protocol adherence and outcome data. Detailed and systematic reporting around the decisionmaking process for stopping, amending or proceeding to a main trial is uncommon, which may hamper understanding in the research community about the appropriate and optimal use of RCTs with an internal pilot phase. 10 top tips for the development, use and reporting of progression criteria for internal pilot studies are presented. Conclusions: Systematic and transparent reporting of the design, results and evaluation of internal pilot trials in the literature should be encouraged in order to facilitate understanding in the research community and to inform future trials.

Original languageEnglish
Article numbere013537
JournalBMJ Open
Volume7
Issue number2
DOIs
Publication statusPublished - 01 Feb 2017

ASJC Scopus subject areas

  • Medicine(all)

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