Modern Approaches to Clinical Trials Using SAS: Classical, Adaptive, and Bayesian Methods

8.4 Analysis Example for a Binary Endpoint

Planning for a phase IIb study in the indication of systemic lupus erythematosus (SLE) has begun. This study will use the systemic lupus erythematosus responder index (SRI) as the primary endpoint. The SRI is a composite binary endpoint. In order to be considered a responder, a subject must show a 4-point reduction on the primary symptom score (SLEDAI-2K), have no worsening symptoms based on a physician’s assessment, and no new organ domain scores in the two highest categories of a secondary symptom score (BILAG). All three criteria must be met in order to be considered a responder. It has been decided that three active dose groups and one placebo group will be included in this study, with 60 subjects ...