title: Inference for Surrogate Endpoint Validation in the Binary Case.
abstract: Surrogate endpoint validation for a binary surrogate endpoint and a binary true endpoint is investigated using the criteria of proportion explained (PE) and the relative effect (RE). The concepts of generalized confidence intervals and fiducial intervals are used for computing confidence intervals for PE and RE. The numerical results indicate that the proposed confidence intervals are satisfactory in terms of coverage probability, whereas the intervals based on Fieller's theorem and the delta method fall short in this regard. Our methodology can also be applied to interval estimation problems in a causal inference-based approach to surrogate endpoint validation.
authors.affiliation: a The Biostatistics Center, Department of Epidemiology and Biostatistics , The George Washington University , Rockville , Maryland , USA.
authors.forename: Ionut
authors.initials: I
authors.lastname: Bebu
authors.affiliation: b Department of Mathematics and Statistics , University of Maryland Baltimore County , Baltimore , Maryland , USA.
authors.forename: Thomas
authors.initials: T
authors.lastname: Mathew
authors.affiliation: c Infectious Disease Clinical Research Program, Department of Preventive Medicine and Biometrics , Uniformed Services University of the Health Sciences , Bethesda , Maryland , USA.
authors.forename: Brian
authors.initials: B
authors.lastname: Agan
mesh_terms: D000465:Algorithms; D015984:Causality; D003198:Computer Simulation; D016001:Confidence Intervals; D023381:Endpoint Determination / Q000379:methods* / Q000706:statistics & numerical data*; D015658:HIV Infections / Q000401:mortality / Q000628:therapy; D006801:Humans; D016015:Logistic Models; D008268:Macular Degeneration / Q000188:drug therapy; D011379:Prognosis; D016032:Randomized Controlled Trials as Topic / Q000706:statistics & numerical data; D015203:Reproducibility of Results
keywords: Causal effects; Fiducial interval; Generalized confidence interval; Proportion explained; Relative effect