The Role and Interpretation of Pilot Studies in Clinical Research

By A. Leon, L. Davis, H. Kraemer. Journal of Psychiatric Research, 2010

1,870 citations recorded by the source corpus.

Pilot studies represent a fundamental phase of the research process. The purpose of conducting a pilot study is to examine the feasibility of an approach that is intended to be used in a larger scale study. The roles and limitations of pilot studies are described here using a clinical trial as an example. A pilot study can be used to evaluate the feasibility of recruitment, randomization, retention, assessment procedures, new methods, and implementation of the novel intervention. A pilot study is not a hypothesis testing study. Safety, efficacy and effectiveness are not evaluated in a pilot. Contrary to tradition, a pilot study does not provide a meaningful effect size estimate for planning subsequent studies due to the imprecision inherent in data from small samples. Feasibility results do not necessarily generalize beyond the inclusion and exclusion criteria of the pilot design. A pilot study is a requisite initial step in exploring a novel intervention or an innovative application of an intervention. Pilot results can inform feasibility and identify modifications needed in the design of a larger, ensuing hypothesis testing study. Investigators should be forthright in stating these objectives of a pilot study. Grant reviewers and other stakeholders should expect no more.

Subfields: Economics and Econometrics, Sociology and Political Science, Statistics and Probability

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Citations in the corpus

When power analyses based on pilot data are biased: Inaccurate effect size estimators and follow-up bias (2018, background): “Pilot studies can be performed because they have other uses, such as determining the feasibility of performing the designed study in practice, which is especially useful when trying out new methods or procedures (Leon et al., 2011).”
Designing a Pilot Sequential Multiple Assignment Randomized Trial for Developing an Adaptive Treatment Strategy (2012, positive): “Scientists preparing to execute a randomized trial should also refer to the literature on executing external pilot studies for pointers on more general uses of pilot studies [34–36].”
Smartphones for Smarter Delivery of Mental Health Programs: A Systematic Review (2013, background): “Furthermore, small sample sizes hamper the precision and accuracy of the statistical results and therefore limit our interpretations [45].”
Senolytics in idiopathic pulmonary fibrosis: Results from a first-in-human, open-label, pilot study (2019, background): “However, the primary purpose of this pilot study was to evaluate study feasibility rather than drug efficacy [39,40].”
The significance filter, the winner's curse and the need to shrink (2020, methodology): “are often used to inform the sample size calculation of a larger trial [13],[7].”
Pilot Testing for Feasibility in a Study of Student Retention and Attrition in Online Undergraduate Programs. (2018, background): “Nevertheless, as described above, properly conducted pilot studies can greatly strengthen outcomes of the main study regardless of the field (van Teijlingen & Hundley, 2002; Cohen, Manion, & Morrison, 2007; Gudmundsdottir & Brock-Utne, 2010; Leon et al., 2011; Doody & Doody, 2015).”