A review of phase II trial designs for initial marker validation.

ABSTRACT: Phase II clinical trials aim to identify promising experimental regimens for further testing in phase III trials. In this review article, we focus on phase II designs for initial predictive biomarker validation to determine if a drug should be developed for an unselected patient population or for a biomarker-defined patient subset only. Several prospective designs for biomarker-directed therapy have been proposed, differing primarily in the study population, or randomization scheme, or both. The design choice is driven by scientific rationale, marker prevalence, strength of preliminary evidence, assay performance, and turn-around times for marker assessment. The enrichment design is most appropriate when compelling preliminary evidence suggests treatment benefit in only certain marker-defi

SUBMITTER: Mandrekar SJ 

PROVIDER: S-EPMC3779487 | biostudies-literature | 2013 Nov

REPOSITORIES: biostudies-literature