title: A note on partial covariate-adjustment and design considerations in noninferiority trials when patient-level data are not available.
abstract: The traditional fixed margin approach to evaluating an experimental treatment through an active-controlled noninferiority trial is simple and straightforward. However, its utility relies heavily on the constancy assumption of the experimental data. The recently developed covariate-adjustment method permits more flexibility and improved discriminatory capacity compared to the fixed margin approach. However, one major limitation of this covariate-adjustment methodology is its adherence on the patient-level data, which may not be accessible to investigators in practice. In this article, under some assumptions, we examine the feasibility of a partial covariate-adjustment approach based on data typically available from journal publications or other public data when the patient-level data are unavailable. We illustrate the usefulness of this approach through two real examples. We also provide design considerations on the efficiency of the partial covariate-adjustment approach.
authors: U.S. Food and Drug Administration (US FDA), Division of Biometrics IV, Office of Biometrics/CDER/OTS/FDA, Silver Spring, Maryland 20993, USA. lei.nie@fda.hhs.gov
authors: Lei
authors: L
authors: Nie
authors: Guoxing Greg
authors: GG
authors: Soon
authors: Karen
authors: K
authors: Qi
authors: Yong
authors: Y
authors: Chen
authors: Haitao
authors: H
authors: Chu
mesh_terms: D000704:Analysis of Variance; D000998:Antiviral Agents / Q000493:pharmacokinetics / Q000627:therapeutic use; D016001:Confidence Intervals; D018849:Controlled Clinical Trials as Topic / Q000379:methods / Q000706:statistics & numerical data*; D015658:HIV Infections / Q000188:drug therapy / Q000276:immunology; D006526:Hepatitis C / Q000188:drug therapy / Q000276:immunology; D006801:Humans; D015233:Models, Statistical; D012107:Research Design / Q000592:standards / Q000706:statistics & numerical data*; D018401:Sample Size; D016896:Treatment Outcome
chemical_list: D000998:Antiviral Agents