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Title Fundamentals of a Good Pilot Study Essentials for CTSA Investigators
Year 2010
Contributor/Contact Charity Moore, Rickey Carter, Paul Nietert, Paul Stewart
CTSA Mayo, MUSC, UNC, U Pittsburgh
Success Topic Collaboration
Description of Success
We often underestimate the importance of pilot studies in clinical research and do not appreciate that the majority of clinical research studies that take place in academic medical centers are pilot studies, or preparatory studies designed to test the designs, measures, procedures, recruitment criteria, and operational strategies that are under consideration for use in a subsequent, often larger, study. Our junior clinical and translational researchers, as well as our residents, complete a clinical research or pilot project. Many of these can be characterized by a very non-productive cycle of choosing a new research topic, testing the topic in a pilot framework, obtaining no significant findings, abandoning the research topic, and then choosing a new research topic. BERD biostatisticians from four of our CTSA sites (Charity Moore, University of Pittsburgh; Rickey Carter, Mayo Clinic; Paul Nietert, Medical University of South Carolina; and Paul Steward, University of North Carolina) collaborated to provide a set of very useful recommendations for investigators considering a pilot study. First and foremost, a master protocol document that is updated throughout the research is highly recommended. This document tracks all of the particulars of the study, with sections specifying objectives, study design, enrollment and withdrawal, schedule, procedures and evaluations, safety and clinical monitoring, biostatistical considerations, database management plans, and quality assurance, and appendices of data collection; and provides documentation for grant applications instruments

Keeping in mind the next study is the first principle guiding the use of pilot studies. A well-planned pilot study can not only help in subsequent clinical studies where preliminary efforts to find an optimal dose but also in determining the feasibility or acceptance of a subsequent study of an intervention in a community. It is important that the researcher understand whether the main purpose of their pilot study is to investigate a process (e.g., recruitment feasibility, protocol refinement, willingness of physician to randomize or patient to be randomized) or an outcome (e.g., complication rates or efficacy measures). The aims and methods of a pilot study must be aligned with the goals of the subsequent study.

It is essential to do good study design planning for the pilot study. Pilot study aims should serve to identify and address issues that could occur with respect to future study conceptualization, study design, sample size, sample selection, data collection, data management, and data analysis. Once the aims are clearly defined, the specification of the primary outcome variables should follow. In some cases, the investigators will seek to generate preliminary data on an established outcome of interest. The following set of questions should be answered as a part of the design of the pilot study: (1) What will be the study design of the subsequent study? (2) What statistical strategies and methods will be used in the subsequent study? (3) What novel or untested information is needed to plan the subsequent study, and when will this information be needed? (4) Is the proposed design of the pilot study adequate to obtain the information? (5) What information cannot be obtained, and how will the lack of this information be handled? (6) What generalizable knowledge can be used to plan the pilot study or subsequent study? Regardless of the type of study, the hallmark of a well-developed analysis plan is a carefully constructed strategy for achieving each aim.

Justification for the sample size in a pilot study provides an investigator with a degree of confidence in whether the finding will be meaningful and applicable to a larger study. Some pilot studies will require hypothesis testing to guide decisions about whether further research developments are needed before the larger subsequent study can be undertaken. Whether or not the study includes a hypothesis test, what is always a valid consideration for sample size is the anticipated level of precision of statistical estimators: estimates, conference intervals, and anticipated levels of precision to name a few. Confidence intervals (CI) represent a plausible range for the true population value by accounting for distributional assumptions. Most new investigators are quire anxious about the size of their study and at least 12 participants for pilot studies with primary focus of estimating average values and variability for planning larger subsequent studies is a feasible recommendation. This size is quite practical for most early stage investigators to conduct within single centers while still providing valuable preliminary information.

Finally, pilot studies are truly a critical component in doing good clinical research. These studies are intended to allow investigators to obtain preliminary data and refine procedures and hypotheses to develop protocols for more definitive subsequent studies and to generate grant proposals for funding from national agencies. For junior and developing investigators, the benefits of pilot-funded studies are numerous. A pilot study can provide an ideal opportunity to gain experience with new procedures, new collaborations, protocol writing, funding applications, and institutional review board applications.

Moore CG, Carter R, Nietert PJ, Stewart PW (2011). Recommendations for planning pilot studies in clinical and translational research. Clinical and Translational Science, 4(5): 332-337.
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Topic revision: r1 - 21 Nov 2011 - 15:30:43 - MaryBanach

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