The effects of heterogeneity on Simon Phase II clinical trial design operating characteristics

• Main Authors: Christopher N Barnes, Shesh N Rai
• Format: Article
• Language: English
• Published: Dove Medical Press 2010-07-01
• Series: Open Access Journal of Clinical Trials
• Online Access: http://www.dovepress.com/the-effects-of-heterogeneity-on-simon-phase-ii-clinical-trial-design-o-a4750

Christopher N Barnes1, Shesh N Rai21Department of Bioinformatics and Biostatistics, University of Louisville, 2Biostatistics Shared Facility, James Graham Brown Cancer Center, Louisville, KentuckyAbstract: The homogeneity assumption of a Simon Phase II clinical trial is commonly violated due to excess variation in the response known as response heterogeneity. Using a general framework to model heterogeneity, we investigate its effects on the operating characteristics of the Simon trial design using the standard practice of averaging responses. We show that, under heterogeneity and averaging, the Simon designs have higher than expected errors which may result in false negative and false positive Phase II outcomes.Keywords: Simon Trial, Phase II, heterogeneity