title: Non-inferiority of new procedure to standard procedure in stratified matched-pair design.
abstract: We consider the statistical testing for non-inferiority of a new treatment compared with the standard one under matched-pair setting in a stratified study or in several trials. A non-inferiority test based on the efficient scores and a Mantel-Haenszel (M-H) like procedure with restricted maximum likelihood estimators (RMLEs) of nuisance parameters and their corresponding sample size formulae are presented. We evaluate the above tests and the M-H type Wald test in level and power. The stratified score test is conservative and provides the best power. The M-H like procedure with RMLEs gives an accurate level. However, the Wald test is anti-conservative and we suggest caution when it is used. The unstratified score test is not biased but it is less powerful than the stratified score test when base-line probabilities related to strata are not the same. This investigation shows that the stratified score test possesses optimum statistical properties in testing non-inferiority. A common difference between two proportions across strata is the basic assumption of the stratified tests, we present appropriate tests to validate the assumption and related remarks.
authors.affiliation: Biostatistics Branch, Division of Cancer Epidemiology and Genetics, NCI, NIH, Department of Health & Human Services, Executive Plaza South, Room 8028, 6120 Executive Boulevard, MSC 7240, Rockville, Maryland 20892-7240, USA. namj@mail.nih.gov
authors.forename: Jun-mo
authors.initials: JM
authors.lastname: Nam
mesh_terms: D000465:Algorithms; D001699:Biometry / Q000379:methods*; D003198:Computer Simulation; D018849:Controlled Clinical Trials as Topic / Q000379:methods*; D003627:Data Interpretation, Statistical; D016015:Logistic Models; D015233:Models, Statistical; D017063:Outcome Assessment, Health Care / Q000379:methods*; D018401:Sample Size; D016896:Treatment Outcome