The size of a pilot study for a clinical trial should be calculated in relation to considerations of precision and efficiency

Objective: To investigate methods to determine the size of a pilot study to inform a power calculation for a randomized controlled trial (RCT) using an interval/ratio outcome measure.

Study design: Calculations based on confidence intervals (CIs) for the sample standard deviation (SD).

Results: Based on CIs for the sample SD, methods are demonstrated whereby (1) the observed SD can be adjusted to secure the desired level of statistical power in the main study with a specified level of confidence; (2) the sample for the main study, if calculated using the observed SD, can be adjusted, again to obtain the desired level of statistical power in the main study; (3) the power of the main study can be calculated for the situation in which the SD in the pilot study proves to be an underestimate of the true SD; and (4) an "efficient" pilot size can be determined to minimize the combined size of the pilot and main RCT.

Conclusion: Trialists should calculate the appropriate size of a pilot study, just as they should the size of the main RCT, taking into account the twin needs to demonstrate efficiency in terms of recruitment and to produce precise estimates of treatment effect.