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Two-stage adaptive enrichment design for testing an active factor.


ABSTRACT: We propose an adaptive enrichment approach to test an active factor, which is a factor whose effect is non-zero in at least one subpopulation. We implement a two-stage play-the-winner design where all subjects in the second stage are enrolled from the subpopulation that has the highest observed effect in the first stage. We recommend a weighted Fisher's combination of the most powerful test for each stage, respectively: the first stage Hotelling's test and the second stage noncentral chi-square test. The test is further extended to cover binary outcomes and time-to-event outcomes.

SUBMITTER: Ding AA 

PROVIDER: S-EPMC6881517 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature

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Two-stage adaptive enrichment design for testing an active factor.

Ding A Adam AA   Wu Samuel S SS   Dean Natalie E NE   Zahigian Rachel S RS  

Journal of biopharmaceutical statistics 20190528 1


We propose an adaptive enrichment approach to test an active factor, which is a factor whose effect is non-zero in at least one subpopulation. We implement a two-stage play-the-winner design where all subjects in the second stage are enrolled from the subpopulation that has the highest observed effect in the first stage. We recommend a weighted Fisher's combination of the most powerful test for each stage, respectively: the first stage Hotelling's test and the second stage noncentral chi-square  ...[more]

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