title: An adaptive two-stage dose-response design method for establishing proof of concept.
abstract: We propose an adaptive two-stage dose-response design where a prespecified adaptation rule is used to add and/or drop treatment arms between the stages. We extend the multiple comparison procedures-modeling (MCP-Mod) approach into a two-stage design. In each stage, we use the same set of candidate dose-response models and test for a dose-response relationship or proof of concept (PoC) via model-associated statistics. The stage-wise test results are then combined to establish "global" PoC using a conditional error function. Our simulation studies showed good and more robust power in our design method compared to conventional and fixed designs.
authors: Department of Biostatistics and Computational Biology, Dana-Faber Cancer Institute and Harvard School of Public Health, Boston, Massachusetts 02215, USA. yoko.tnk@gmail.com
authors: Yoko
authors: Y
authors: Franchetti
authors: Stewart J
authors: SJ
authors: Anderson
authors: Allan R
authors: AR
authors: Sampson
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods* / Q000706:statistics & numerical data; D003198:Computer Simulation; D004305:Dose-Response Relationship, Drug; D015195:Drug Design; D015233:Models, Statistical; D012107:Research Design / Q000592:standards / Q000706:statistics & numerical data*; D018401:Sample Size
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