title: Treatment selection in a randomized clinical trial via covariate-specific treatment effect curves.
abstract: For time-to-event data in a randomized clinical trial, we proposed two new methods for selecting an optimal treatment for a patient based on the covariate-specific treatment effect curve, which is used to represent the clinical utility of a predictive biomarker. To select an optimal treatment for a patient with a specific biomarker value, we proposed pointwise confidence intervals for each covariate-specific treatment effect curve and the difference between covariate-specific treatment effect curves of two treatments. Furthermore, to select an optimal treatment for a future biomarker-defined subpopulation of patients, we proposed confidence bands for each covariate-specific treatment effect curve and the difference between each pair of covariate-specific treatment effect curve over a fixed interval of biomarker values. We constructed the confidence bands based on a resampling technique. We also conducted simulation studies to evaluate finite-sample properties of the proposed estimation methods. Finally, we illustrated the application of the proposed method in a real-world data set.
authors.affiliation: 1 School of Statistics, Southwestern University of Finance and Economics, Chengdu, Sichuan, China.
authors.forename: Yunbei
authors.initials: Y
authors.lastname: Ma
authors.affiliation: 2 HSR&D Center of Excellence, VA Puget Sound Health Care System, Department of Biostatistics, University of Washington, Seattle, USA.|3 Beijing International Center for Mathematical Research, Peking University, Beijing, China.
authors.forename: Xiao-Hua
authors.initials: XH
authors.lastname: Zhou
mesh_terms: D015415:Biomarkers; D003110:Colonic Neoplasms / Q000188:drug therapy / Q000235:genetics; D016001:Confidence Intervals; D016259:Genes, myc / Q000235:genetics; D006801:Humans; D016032:Randomized Controlled Trials as Topic / Q000379:methods*
chemical_list: D015415:Biomarkers
keywords: covariate-specific treatment effect curve; pointwise confidence interval; predictive biomarker; simultaneous confidence interval; time-to-event outcome; varying coefficient