Sample size variation in single-time post-dose assessment vs multi-time post-dose assessment.

Abstract

Background: Many randomized trials measure a continuous outcome simultaneously at baseline and after taking the drug. For a single continuous post-treatment outcome, the sample size calculation is simple, but if there are assessments at multiple time point post-treatment then this longitudinal data may give more insights by analyzing the data using the repeated measures method. Also, if the sample size is calculated using the single time-point method for longitudinal data, it may lead to a larger than required sample size, increasing the cost and time.

Methods: In this research, an effort is made to determine the size of the sample for repeated measures case and then compare with the single post-baseline case. The sample sizes were examined under different scenarios for the continuous type of response variable. Under 'Mean contrast' and 'Diff contrast' the sample sizes were calculated with different correlations. These two scenarios were again examined under compound symmetry as well as discrete Auto regressive of order 1 type of correlation structure in longitudinal data. The graphical presentation is given for better visualization of the scenarios.

Results: The sample size required for highly correlated longitudinal data using the multi-timepoint sample size derivation method led to a smaller sample size requirement compared to the single timepoint sample size calculation method.

Conclusions: This study will help researchers to make better decisions in choosing the right method for sample size determination which may reduce the time and cost of conducting the experiment. Additionally, it is crucial to carefully evaluate and choose the appropriate method when the correlation is weak, as this can significantly impact the accuracy of the results. More complex correlation structures are not studied in this article but can be studied in the same fashion.