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Subgroup detection and sample size calculation with proportional hazards regression for survival data
Author(s) -
Kang Suhyun,
Lu Wenbin,
Song Rui
Publication year - 2017
Publication title -
statistics in medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.996
H-Index - 183
eISSN - 1097-0258
pISSN - 0277-6715
DOI - 10.1002/sim.7441
Subject(s) - covariate , mathematics , statistics , censoring (clinical trials) , test statistic , sample size determination , nonparametric statistics , subgroup analysis , proportional hazards model , statistic , statistical hypothesis testing , econometrics , confidence interval
In this paper, we propose a testing procedure for detecting and estimating the subgroup with an enhanced treatment effect in survival data analysis. Here, we consider a new proportional hazard model that includes a nonparametric component for the covariate effect in the control group and a subgroup‐treatment–interaction effect defined by a change plane. We develop a score‐type test for detecting the existence of the subgroup, which is doubly robust against misspecification of the baseline effect model or the propensity score but not both under mild assumptions for censoring. When the null hypothesis of no subgroup is rejected, the change‐plane parameters that define the subgroup can be estimated on the basis of supremum of the normalized score statistic. The asymptotic distributions of the proposed test statistic under the null and local alternative hypotheses are established. On the basis of established asymptotic distributions, we further propose a sample size calculation formula for detecting a given subgroup effect and derive a numerical algorithm for implementing the sample size calculation in clinical trial designs. The performance of the proposed approach is evaluated by simulation studies. An application to an AIDS clinical trial data is also given for illustration.