Guidance for using pilot studies to inform the design of intervention trials with continuous outcomes
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CLEP-146397-guidance-for-using ...
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Final Published Version
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Univ Arizona, Mel & Enid Zuckerman Coll Publ Hlth, Dept Epidemiol & BiostatIssue Date
2018-01
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DOVE MEDICAL PRESS LTDCitation
Guidance for using pilot studies to inform the design of intervention trials with continuous outcomes 2018, Volume 10:153 Clinical EpidemiologyJournal
Clinical EpidemiologyRights
© 2018 Bell et al. This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms. php and incorporate the Creative Commons Attribution – Non Commercial (unported, v3.0) License.Collection Information
This item from the UA Faculty Publications collection is made available by the University of Arizona with support from the University of Arizona Libraries. If you have questions, please contact us at repository@u.library.arizona.edu.Abstract
Background: A pilot study can be an important step in the assessment of an intervention by providing information to design the future definitive trial. Pilot studies can be used to estimate the recruitment and retention rates and population variance and to provide preliminary evidence of efficacy potential. However, estimation is poor because pilot studies are small, so sensitivity analyses for the main trial's sample size calculations should be undertaken. Methods: We demonstrate how to carry out easy-to-perform sensitivity analysis for designing trials based on pilot data using an example. Furthermore, we introduce rules of thumb for the size of the pilot study so that the overall sample size, for both pilot and main trials, is minimized. Results: The example illustrates how sample size estimates for the main trial can alter dramatically by plausibly varying assumptions. Required sample size for 90% power varied from 392 to 692 depending on assumptions. Some scenarios were not feasible based on the pilot study recruitment and retention rates. Conclusion: Pilot studies can be used to help design the main trial, but caution should be exercised. We recommend the use of sensitivity analyses to assess the robustness of the design assumptions for a main trial.Note
Open access journal.UA Open Access Publishing Fund.
ISSN
1179-1349PubMed ID
29403314Version
Final published versionSponsors
NCI NIH HHS [P30 CA023074]Additional Links
https://www.dovepress.com/guidance-for-using-pilot-studies-to-inform-the-design-of-intervention--peer-reviewed-article-CLEPae974a485f413a2113503eed53cd6c53
10.2147/CLEP.S146397
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Except where otherwise noted, this item's license is described as © 2018 Bell et al. This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms. php and incorporate the Creative Commons Attribution – Non Commercial (unported, v3.0) License.
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