title: Group sequential designs for developing and testing biomarker-guided personalized therapies in comparative effectiveness research.
abstract: Biomarker-guided personalized therapies offer great promise to improve drug development and improve patient care, but also pose difficult challenges in designing clinical trials for the development and validation of these therapies. We first give a review of the existing approaches, briefly for clinical trials in new drug development and in more detail for comparative effectiveness trials involving approved treatments. We then introduce new group sequential designs to develop and test personalized treatment strategies involving approved treatments.
authors.affiliation: Department of Statistics, Stanford University, Stanford, CA, USA.
authors.forename: Tze Leung
authors.initials: TL
authors.lastname: Lai
authors.forename: Olivia Yueh-Wen
authors.initials: OY
authors.lastname: Liao
authors.forename: Dong Woo
authors.initials: DW
authors.lastname: Kim
mesh_terms: D001499:Bayes Theorem; D015415:Biomarkers / Q000378:metabolism*; D002985:Clinical Protocols; D057186:Comparative Effectiveness Research / Q000379:methods* / Q000592:standards; D004358:Drug Therapy; D006801:Humans; D015233:Models, Statistical; D057285:Precision Medicine / Q000379:methods* / Q000592:standards; D016032:Randomized Controlled Trials as Topic / Q000379:methods / Q000592:standards
chemical_list: D015415:Biomarkers
keywords: Adaptive randomization; Biomarker classifiers; Generalized likelihood ratio statistics; Group sequential design; Multiple testing; Targeted therapies