Abstract:
:We propose a new adaptive threshold detection and enrichment design in which the biomarker threshold is adaptively estimated and updated by optimizing a trade-off between the size of the biomarker positive population and the magnitude of the treatment effect in that population. Enrichment is based on an enrollment criterion that accounts for the uncertainty in estimation of the threshold. Early termination for futility is allowed based on predictive success probability. Valid testing and estimation techniques for the treatment effect overall and inpatient subgroups are studied. Simulations and an example demonstrate advantages of the proposed design over existing designs.
journal_name
J Biopharm Statjournal_title
Journal of biopharmaceutical statisticsauthors
Wang T,Wang X,George SL,Zhou Hdoi
10.1080/10543406.2020.1832110subject
Has Abstractpub_date
2020-11-11 00:00:00pages
1-17eissn
1054-3406issn
1520-5711pub_type
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