Statistical inference for clinical trials with binary responses when there is a shift in patient population.

In clinical research, it is not uncommon to modify a trial procedure and/or statistical methods of ongoing clinical trials through protocol amendments. A major modification to the study protocol could result in a shift in target patient population. In addition, frequent and significant modifications could lead to a totally different study that is unable to address the medical questions that the original study intended to answer. In this article, we propose a logistic regression model for statistical inference based on a binary study endpoint for trials with protocol amendments. Under the proposed method, sample size adjustment is also derived.

Duke Scholars

Author Shein-Chung Chow Biostatistics & Bioinformatics, Division of Biostatistics