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

11.3 Efficiency of Classical Enrichment Designs

Simon and Maitournam [24] and Maitournam and Simon [25] considered a phase III randomized trial setting to evaluate molecular targeted treatment using a binary endpoint or continuous endpoint. The evaluations were done under the assumption that the assay was predictive of a subset of patients who benefited from the targeted therapy. They compared the all-comers design where all the patients are allowed to participate in the trial with the enrichment design with restricted subset population. They compared the operating characteristics based on average screened and randomized sample size.

For a binary endpoint, the relative efficiency of the targeted design versus the untargeted design can be expressed ...