title: Active-control trials with binary data: a comparison of methods for testing superiority or non-inferiority using the odds ratio.
abstract: This paper considers methods for testing for superiority or non-inferiority in active-control trials with binary data, when the relative treatment effect is expressed as an odds ratio. Three asymptotic tests for the log-odds ratio based on the unconditional binary likelihood are presented, namely the likelihood ratio, Wald and score tests. All three tests can be implemented straightforwardly in standard statistical software packages, as can the corresponding confidence intervals. Simulations indicate that the three alternatives are similar in terms of the Type I error, with values close to the nominal level. However, when the non-inferiority margin becomes large, the score test slightly exceeds the nominal level. In general, the highest power is obtained from the score test, although all three tests are similar and the observed differences in power are not of practical importance.
authors.affiliation: Departamento de Estatística, Universidade Federal de Minas Gerais, Belo Horizonte, Brazil.
authors.forename: Arminda Lucia
authors.initials: AL
authors.lastname: Siqueira
authors.forename: Anne
authors.initials: A
authors.lastname: Whitehead
authors.forename: Susan
authors.initials: S
authors.lastname: Todd
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods* / Q000706:statistics & numerical data*; D016001:Confidence Intervals; D035061:Control Groups; D006801:Humans; D016013:Likelihood Functions; D016017:Odds Ratio; D010919:Placebos; D016896:Treatment Outcome
chemical_list: D010919:Placebos