title: Sample size determination for a three-arm equivalence trial of Poisson and negative binomial responses.
abstract: Assessing equivalence or similarity has drawn much attention recently as many drug products have lost or will lose their patents in the next few years, especially certain best-selling biologics. To claim equivalence between the test treatment and the reference treatment when assay sensitivity is well established from historical data, one has to demonstrate both superiority of the test treatment over placebo and equivalence between the test treatment and the reference treatment. Thus, there is urgency for practitioners to derive a practical way to calculate sample size for a three-arm equivalence trial. The primary endpoints of a clinical trial may not always be continuous, but may be discrete. In this paper, the authors derive power function and discuss sample size requirement for a three-arm equivalence trial with Poisson and negative binomial clinical endpoints. In addition, the authors examine the effect of the dispersion parameter on the power and the sample size by varying its coefficient from small to large. In extensive numerical studies, the authors demonstrate that required sample size heavily depends on the dispersion parameter. Therefore, misusing a Poisson model for negative binomial data may easily lose power up to 20%, depending on the value of the dispersion parameter.
authors.affiliation: a Biostatistics and Data Science , Boehringer Ingelheim Pharmaceuticals, Inc. , Ridgefield , Connecticut , USA.
authors.forename: Yu-Wei
authors.initials: YW
authors.lastname: Chang
authors.affiliation: b Center for Drug Evaluation and Research , US Food and Drug Administration , Silver Spring , Maryland , USA.
authors.forename: Yi
authors.initials: Y
authors.lastname: Tsong
authors.affiliation: c Department of Statistical Science , Temple University , Philadelphia , Pennsylvania , USA.
authors.forename: Zhigen
authors.initials: Z
authors.lastname: Zhao
mesh_terms: D000074099:Equivalence Trials as Topic; D006801:Humans; D015233:Models, Statistical; D018401:Sample Size
keywords: Equivalence trial; Poisson; negative binomial; power; sample size; superiority test