Sample size determination for matched-pair equivalence trials using rate ratio

Nian Sheng Tang, Man Lai TANG*, Shun Fang Wang

*Corresponding author for this work

Research output: Contribution to journalJournal articlepeer-review

13 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)625-631
Number of pages7
Issue number3
Publication statusPublished - Jul 2007

Scopus Subject Areas

  • Statistics and Probability
  • Statistics, Probability and Uncertainty

User-Defined Keywords

  • Constrained maximum likelihood estimation method
  • Equivalence study
  • Sample size formula
  • Score test statistic


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