Friday, January 13, 2012

Checklist for reviewing pilot studies

Pilot Study Checklist

At least one of the following reasons to conduct the study applies
·      Study administration
·      Data management
·      Scientific

Aims and objectives are clearly stated

Collected data are consistent with goals

No statistical hypothesis is tested

Sample size is justified (not necessarily in a statistical sense)

The way in which collected will be used in the design of a larger study has been addressed

This study will answer the question on whether a full scale trial/experiment is worth pursuing

Criteria that will determine continuation of a larger study are specified



Other recommendations and thoughts

Distinguish between
·      Toy problems for learning experience
·      Identifying feasibility answers
·      Getting “preliminary data” (why?)

Always identify
·      What the goal of the study is
·      How it will be determined whether the goal is met
·      What steps need to be taken to meet the goal

What is innovative or novel.

A poorly designed study can be used as an example to train investigators in designing experiments.

Example of a badly designed study

Prior to a larger clinical trial, a pilot study involving 10 patients is proposed. The goal of the project is to investigate how well patients will tolerate wearing a new ambulatory heart monitor while receiving an experimental medication. Data will be downloaded from the monitors and will be analyzed using a t-test procedure for comparison of post-treatment heart rate to pre-treatment heart rate.

·      The main goal was to assess tolerability but there is no measure defined for it. No data to address the main goal is collected.
·      There is an implicit hypothesis tested.
·      Sample size is not justified and may be driven by the desire to detect a difference.


If the goal is to estimate tolerability, where tolerability is defined as a dichotomous variable. Then the investigator needs to describe a criterion for unacceptable tolerability. The closer the intolerability is to zero, the larger the sample size needs to be.

Suggest a two tiered review. Statistician need only review protocols that have scientific merit.


References

http://www.ctspedia.org/do/view/CTSpedia/SuccessStory041

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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