title: Point and Interval Estimators of the Target Dose in Clinical Dose-Finding Studies with Active Control.
abstract: In a clinical dose finding study with active control a new drug with several dose levels is compared with an active comparator drug. The main focus of such studies often lies on the estimation of a target dose that leads to the same efficacy as the control. This article investigates the finite sample properties of the maximum likelihood estimation of the target dose and compares several approaches for constructing corresponding confidence intervals under the assumption of a linear dose-response curve and normal error terms. Furthermore, the impact of deviations from the model assumptions regarding the error distribution is explored.
authors.affiliation: a Department of Medical Statistics , University Medical Center Göttingen , Humboldtallee , Göttingen , Germany.
authors.forename: H-J
authors.initials: HJ
authors.lastname: Helms
authors.forename: N
authors.initials: N
authors.lastname: Benda
authors.forename: T
authors.initials: T
authors.lastname: Friede
mesh_terms: D017322:Clinical Trials, Phase II as Topic / Q000379:methods* / Q000706:statistics & numerical data; D016001:Confidence Intervals; D003627:Data Interpretation, Statistical; D004305:Dose-Response Relationship, Drug; D054796:Drug Dosage Calculations; D006801:Humans; D016013:Likelihood Functions; D016014:Linear Models; D004364:Pharmaceutical Preparations / Q000008:administration & dosage*; D012107:Research Design / Q000706:statistics & numerical data; D016896:Treatment Outcome
chemical_list: D004364:Pharmaceutical Preparations
keywords: Active control; Dose-finding study; Inverse regression; Linear model