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Erschienen in:
2016 | OriginalPaper | Buchkapitel
Personalized Effective Dose Selection in Dose Ranging Studies
verfasst von : Xiwen Ma, Wei Zheng, Yuefeng Lu
Erschienen in: Statistical Applications from Clinical Trials and Personalized Medicine to Finance and Business Analytics
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Abstract
We consider the problem of predicting the personalized minimum effective dose and estimating the dose-dependent optimal subgroups in dose-ranging studies. Our research is motivated by a real randomized, double-blind, placebo-controlled phase II dose-ranging study with genetic markers. One goal of the analysis is to identify subgroups with enhanced benefit/risk profiles with approriate doses and inform the study design of future phase III trials. To the best of our knowledge, this problem has not been systematically studied before. We proposed a novel framework to nonparametrically model the dose-dependent biomarker-outcome relationship and to estimate the personalized effective dose and dose-dependent optimal subgroups. Our proposed method will be useful for identifying the respondent subgroups and their accompanying doses for the future study design. We illustrate the proposed method with simulation studies. Our method compares favorably to two ad-hoc approaches.