This research was motivated by a clinical trial design for a cognitive study. The pilot study was a matched-pairs design where some data are missing, specifically the missing data coming at the end of the study. Existing approaches to determine sample size are all based on asymptotic approaches (e.g., the generalized estimating equation (GEE) approach). When the sample size in a clinical trial is small to medium, these asymptotic approaches may not be appropriate for use due to the unsatisfactory Type I and II error rates. For this reason, we consider the exact unconditional approach to compute the sample size for a matched-pairs study with incomplete data. Recommendations are made for each possible missingness pattern by comparing the exact sample sizes based on three commonly used test statistics, with the existing sample size calculation based on the GEE approach. An example from a real surgeon-reviewers study is used to illustrate the application of the exact sample size calculation in study designs.
Keywords: cognitive study; exact approach; incomplete data; missing completely at random; paired data; sample size.
© 2017 The British Psychological Society.