Sample size determination for matched-pair equivalence trials using rate ratio.
In this article, we compare Wald-type, logarithmic transformation, and Fieller-type statistics for the classical 2-sided equivalence testing of the rate ratio under matched-pair designs with a binary end point. These statistics can be implemented through sample-based, constrained least squares estimation and constrained maximum likelihood (CML) estimation methods. Sample size formulae based on the CML estimation method are developed. We consider formulae that control a prespecified power or confidence width. Our simulation studies show that statistics based on the CML estimation method generally outperform other statistics and methods with respect to actual type I error rate and average width of confidence intervals. Also, the corresponding sample size formulae are valid asymptotically in the sense that the exact power and actual coverage probability for the estimated sample size are generally close to their prespecified values. The methods are illustrated with a real example from a clinical laboratory study.
Tang
Nian-Sheng
NS
Department of Statistics, Yunnan University, Kunming 650091, China.
Tang
Man-Lai
ML
Wang
Shun-Fang
SF
D001699:Biometry; D016001:Confidence Intervals; D004342:Drug Hypersensitivity / Q000175:diagnosis / Q000276:immunology; D006801:Humans; D016018:Least-Squares Analysis; D016013:Likelihood Functions; D010406:Penicillins / Q000276:immunology; D011852:Radioallergosorbent Test / Q000379:methods / Q000706:statistics & numerical data; D018401:Sample Size
D010406:Penicillins