Biostatistics Advance Access first published online on October 27, 2006
This version published online on May 14, 2007
Biostatistics, doi:10.1093/biostatistics/kxl034
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Sample size determination for matched-pair equivalence trials using rate ratio
Department of Statistics, Yunnan University, Kunming 650091, China
Department of Mathematics, Hong Kong Baptist University, Kowloon, Hong Kong mltang{at}math.hkbu.edu.hk
Department of Statistics, Yunnan University, Kunming 650091, China
* To whom correspondence should be addressed.
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.
Keywords: Constrained maximum likelihood estimation method; Equivalence study; Sample size formula; Score test statistic
Received June 21, 2005; revised December 8, 2005; revised March 25, 2006; revised April 28, 2006; revised July 10, 2006; revised September 17, 2006; accepted for publication October 20, 2006.