A tutorial on pilot studies: The what, why and How

Department of Clinical Epidemiology and Biostatistics, McMaster University, Hamilton ON, Canada.
BMC Medical Research Methodology (Impact Factor: 2.27). 01/2010; 10(1):1. DOI: 10.1186/1471-2288-10-1
Source: PubMed


Pilot studies for phase III trials - which are comparative randomized trials designed to provide preliminary evidence on the clinical efficacy of a drug or intervention - are routinely performed in many clinical areas. Also commonly know as "feasibility" or "vanguard" studies, they are designed to assess the safety of treatment or interventions; to assess recruitment potential; to assess the feasibility of international collaboration or coordination for multicentre trials; to increase clinical experience with the study medication or intervention for the phase III trials. They are the best way to assess feasibility of a large, expensive full-scale study, and in fact are an almost essential pre-requisite. Conducting a pilot prior to the main study can enhance the likelihood of success of the main study and potentially help to avoid doomed main studies. The objective of this paper is to provide a detailed examination of the key aspects of pilot studies for phase III trials including: 1) the general reasons for conducting a pilot study; 2) the relationships between pilot studies, proof-of-concept studies, and adaptive designs; 3) the challenges of and misconceptions about pilot studies; 4) the criteria for evaluating the success of a pilot study; 5) frequently asked questions about pilot studies; 7) some ethical aspects related to pilot studies; and 8) some suggestions on how to report the results of pilot investigations using the CONSORT format.

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    • "(p. 7) Thabane et al. (2010) indicated that a pilot study is " a small single-centre study " (p. 3) and " a small study that is similar in size to someone else's published study " (p. 3). "
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    • "In this research, a pilot study was conducted with 20 international nurses who are not from the participating institutions. This helped to determine the potential recruitment rates, retention rates and enhanced the likelihood of success of the main study (Thabane et al., 2010). The questionnaires were also assessed on the clarity and A C C E P T E D M A N U S C R I P T "
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    • "The piloting of the RCT procedures showed excellent process and management (e.g., few missing data, no failures in assessment or intervention administration, no equipment failures, excellent collaboration from referring clinicians). The procedures' assessment was consistent with a successful pilot study with the condition of a minor protocol modification in terms of resources (Thabane et al., 2010). More specifically, the procedures' analysis indicated that recruitment rates could be optimized both in terms of numbers and overall duration of recruitment by increasing the resources (e.g., doubling the time devoted to screening and recruitment from 0.3 to 0.6 FTE). "
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