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Subgroup analysis with semiparametric models toward precision medicine.

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In analyzing clinical trials, one important objective is to classify the patients into treatment-favorable and nonfavorable subgroups. Existing parametric methods are not robust, and the commonly used classification rules ignore… Click to show full abstract

In analyzing clinical trials, one important objective is to classify the patients into treatment-favorable and nonfavorable subgroups. Existing parametric methods are not robust, and the commonly used classification rules ignore the fact that the implications of treatment-favorable and nonfavorable subgroups can be different. To address these issues, we propose a semiparametric model, incorporating both our knowledge and uncertainty about the true model. The Wald statistics is used to test the existence of subgroups, while the Neyman-Pearson rule to classify each subject. Asymptotic properties are derived, simulation studies are conducted to evaluate the performance of the method, and then method is used to analyze a real-world trial data.

Keywords: medicine; medicine subgroup; subgroup analysis; semiparametric models; models toward; analysis semiparametric

Journal Title: Statistics in medicine
Year Published: 2018

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