title: Bayesian approach to non-inferiority trials for normal means.
abstract: Regulatory framework recommends that novel statistical methodology for analyzing trial results parallels the frequentist strategy, e.g. the new method must protect type-I error and arrive at a similar conclusion. Keeping these in mind, we construct a Bayesian approach for non-inferiority trials with normal response. A non-informative prior is assumed for the mean response of the experimental treatment and Jeffrey's prior for its corresponding variance when it is unknown. The posteriors of the mean response and variance of the treatment in historical trials are then assumed as priors for its corresponding parameters in the current trial, where that treatment serves as the active control. From these priors, a Bayesian decision criterion is derived to determine whether the experimental treatment is non-inferior to the active control. This criterion is evaluated and compared with the frequentist method using simulation studies. Results show that both Bayesian and frequentist approaches perform alike, but the Bayesian approach has a higher power when the variances are unknown. Both methods also arrive at the same conclusion of non-inferiority when applied on two real datasets. A major advantage of the proposed Bayesian approach lies in its ability to provide posterior probabilities for varying effect sizes of the experimental treatment over the active control.
authors.affiliation: Office of Biostatistics, Food and Drug Administration, USA Mark.Gamalo@fda.hhs.gov.
authors.forename: M Amper
authors.initials: MA
authors.lastname: Gamalo
authors.affiliation: Department of Statistics, University of Connecticut, USA.
authors.forename: Rui
authors.initials: R
authors.lastname: Wu
authors.affiliation: Statistical Science and Policy, Office of Biostatistics, Food and Drug Administration, USA.
authors.forename: Ram C
authors.initials: RC
authors.lastname: Tiwari
mesh_terms: D000704:Analysis of Variance; D018798:Anemia, Iron-Deficiency / Q000097:blood / Q000188:drug therapy; D001499:Bayes Theorem; D056808:Biostatistics; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data*; D003198:Computer Simulation; D006801:Humans; D015233:Models, Statistical; D009798:Ocular Hypertension / Q000188:drug therapy / Q000503:physiopathology; D035501:Uncertainty