Change-Plane Analysis for Subgroup Detection and Sample Size Calculation.

TitleChange-Plane Analysis for Subgroup Detection and Sample Size Calculation.
Publication TypeJournal Article
Year of Publication2017
AuthorsFan, Ailin, Rui Song, and Wenbin Lu
JournalJ Am Stat Assoc
Date Published2017

We propose a systematic method for testing and identifying a subgroup with an enhanced treatment effect. We adopts a change-plane technique to first test the existence of a subgroup, and then identify the subgroup if the null hypothesis on non-existence of such a subgroup is rejected. A semiparametric model is considered for the response with an unspecified baseline function and an interaction between a subgroup indicator and treatment. A doubly-robust test statistic is constructed based on this model, and asymptotic distributions of the test statistic under both null and local alternative hypotheses are derived. Moreover, a sample size calculation method for subgroup detection is developed based on the proposed statistic. The finite sample performance of the proposed test is evaluated via simulations. Finally, the proposed methods for subgroup identification and sample size calculation are applied to a data from an AIDS study.

Alternate JournalJ Am Stat Assoc
Original PublicationChange-plane analysis for subgroup detection and sample size calculation.
PubMed ID28804182
PubMed Central IDPMC5553128
Grant ListP01 CA142538 / CA / NCI NIH HHS / United States