Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2017 Feb 17;7(2):e013537.
doi: 10.1136/bmjopen-2016-013537.

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

Kerry N L Avery  1 , Paula R Williamson  2 , Carrol Gamble  2 , Elaine O'Connell Francischetto  1 , Chris Metcalfe  1 , Peter Davidson  3 , Hywel Williams  4 , Jane M Blazeby  1   5 ; members of the Internal Pilot Trials Workshop supported by the Hubs for Trials Methodology Research
Collaborators, Affiliations
Review

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

Kerry N L Avery et al. BMJ Open. .

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 decision-making 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.

Keywords: STATISTICS & RESEARCH METHODS.

PubMed Disclaimer

Conflict of interest statement

Competing interests: All authors have completed the ICMJE uniform disclosure form at http://www.icmje.org/coi_disclosure.pdf and declare: SL is Chair of the National Institute for Health Research Health Technology Assessment Programme Clinical Evaluation and Trials Board. HW is Chair of the National Institute for Health Research Health Technology Assessment Programme Commissioning Board and Programme Director for the NIHR HTA Programme.

References

    1. Pocock SJ. Clinical trials. A practical approach. Chichester: John Wiley & Sons, 1983.
    1. Craig P, Dieppe P, Macintyre S et al. . Developing and evaluating complex interventions: the new Medical Research Council Guidance. Int J Nurs Stud 2013;50:587–92. 10.1016/j.ijnurstu.2012.09.010 - DOI - PubMed
    1. Lancaster GA, Dodd S, Williamson PR. Design and analysis of pilot studies: recommendations for good practice. J Eval Clin Pract 2004;10:307–12. 10.1111/j..2002.384.doc.x - DOI - PubMed
    1. Bower P, Brueton V, Gamble C et al. . Interventions to improve recruitment and retention in clinical trials: a survey and workshop to assess current practice and future priorities. Trials 2014;15:399–407. 10.1186/1745-6215-15-399 - DOI - PMC - PubMed
    1. McCulloch P, Altman DG, Campbell WB et al. . No surgical innovation without evaluation: the IDEAL recommendations. Lancet 2009;374:1105–12. 10.1016/S0140-6736(09)61116-8 - DOI - PubMed