Using the partitioning principle to adaptively design dose-response studies.
An adaptive two-stage design is proposed for dose-response studies to find the minimum effective dose. The procedure is conducted in a stepwise fashion based on the partition testing principle with familywise error rate controlled strongly. We examine a wide dose range vs. a placebo in the first stage. Then an interim analysis is conducted with potential modification of design features of the experiment. Ineffective and/or unsafe dose treatments are terminated, and selected doses are further investigated in the second stage. Inference is based on a pre-chosen conditional error function. Several conditional error functions are discussed and compared.
Ling
Xiang
X
Amgen Inc, Thousand Oaks, California, USA. xiangl@amgen.com
Hsu
Jason
J
D000465:Algorithms; D017322:Clinical Trials, Phase II as Topic / Q000379:methods / Q000706:statistics & numerical data*; D004305:Dose-Response Relationship, Drug; D006801:Humans; D015233:Models, Statistical; D010919:Placebos / Q000008:administration & dosage; D016032:Randomized Controlled Trials as Topic / Q000379:methods / Q000706:statistics & numerical data; D012107:Research Design; D016896:Treatment Outcome
D010919:Placebos