title: A Bayesian multi-stage cost-effectiveness design for animal studies in stroke research.
abstract: Much progress has been made in the area of adaptive designs for clinical trials. However, little has been done regarding adaptive designs to identify optimal treatment strategies in animal studies. Motivated by an animal study of a novel strategy for treating strokes, we propose a Bayesian multi-stage cost-effectiveness design to simultaneously identify the optimal dose and determine the therapeutic treatment window for administrating the experimental agent. We consider a non-monotonic pattern for the dose-schedule-efficacy relationship and develop an adaptive shrinkage algorithm to assign more cohorts to admissible strategies. We conduct simulation studies to evaluate the performance of the proposed design by comparing it with two standard designs. These simulation studies show that the proposed design yields a significantly higher probability of selecting the optimal strategy, while it is generally more efficient and practical in terms of resource usage.
authors.affiliation: 1 Division of Clinical and Translational Sciences, Department of Internal Medicine, McGovern Medical School, The University of Texas Health Science Center at Houston, USA.|2 Biostatistics/Epidemiology/Research Design Core, Center for Clinical and Translational Sciences, The University of Texas Health Science Center at Houston, USA.
authors.forename: Chunyan
authors.initials: C
authors.lastname: Cai
authors.affiliation: 3 Department of Biostatistics, The University of Texas, MD Anderson Cancer Center, USA.
authors.forename: Jing
authors.initials: J
authors.lastname: Ning
authors.affiliation: 3 Department of Biostatistics, The University of Texas, MD Anderson Cancer Center, USA.
authors.forename: Xuelin
authors.initials: X
authors.lastname: Huang
mesh_terms: D032761:Animal Experimentation; D000818:Animals; D001499:Bayes Theorem; D035843:Biomedical Research; D003362:Cost-Benefit Analysis; D004305:Dose-Response Relationship, Drug; D012107:Research Design / Q000706:statistics & numerical data; D020521:Stroke / Q000188:drug therapy
keywords: Admissible set; Bayesian approach; animal study; cost-effectiveness design; multi-stage design