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Activity Number: 130
Type: Contributed
Date/Time: Monday, August 5, 2013 : 8:30 AM to 10:20 AM
Sponsor: Biopharmaceutical Section
Abstract - #310284
Title: Trial Duration and Sample Size Trade-Off When There Are Subgroups with Different Expected Treatment Effects
Author(s): Kyle D. Rudser*+ and Edward Bendert and Joseph S. Koopmeiners
Companies: University of Minnesota and Statistics Collaborative and University of Minnesota
Keywords: Clinical trial design ; Biomarker ; Enrichment ; Sample size ; Heterogeneous effects ; Operating characteristics
Abstract:

Statistical study design considerations typically focus on sample size, power, and a single population treatment effect given a fixed significance level (generally 0.05). Eligibility criteria are formulated to select the patient population of interest to be studied for which the magnitude of the treatment effect is expected to hold. In some instances we may expect a larger treatment effect in one group while others exhibit an attenuated effect. Identification of these subgroups can be based on a clinical decision rule, e.g., biomarker cutoff, but may not be precise, i.e., sensitivity and specificity are not simultaneously at 100%. In these settings there can be a trade-off between a smaller average treatment effect with broader enrollment criteria and a larger effect with restricted criteria but longer enrollment duration. We evaluate the impact of including attenuated subgroups on design operating characteristics and illustrate scenarios where overall trial enrollment duration may be shorter by not being restrictive.


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