Power analysis for clustered non-continuous responses in multicenter trials

T. Chen, K. Knox, J. Arora, W. Tang, J. Kowalski, X. M. Tu

Research output: Contribution to journalArticlepeer-review

Abstract

Power analysis for multi-center randomized control trials is quite difficult to perform for non-continuous responses when site differences are modeled by random effects using the generalized linear mixed-effects model (GLMM). First, it is not possible to construct power functions analytically, because of the extreme complexity of the sampling distribution of parameter estimates. Second, Monte Carlo (MC) simulation, a popular option for estimating power for complex models, does not work within the current context because of a lack of methods and software packages that would provide reliable estimates for fitting such GLMMs. For example, even statistical packages from software giants like SAS do not provide reliable estimates at the time of writing. Another major limitation of MC simulation is the lengthy running time, especially for complex models such as GLMM, especially when estimating power for multiple scenarios of interest. We present a new approach to address such limitations. The proposed approach defines a marginal model to approximate the GLMM and estimates power without relying on MC simulation. The approach is illustrated with both real and simulated data, with the simulation study demonstrating good performance of the method.

Original languageEnglish (US)
Pages (from-to)979-995
Number of pages17
JournalJournal of Applied Statistics
Volume43
Issue number6
DOIs
StatePublished - Apr 25 2016

Keywords

  • GEE
  • GLIMMIX
  • NLMIXED
  • intraclass correlation
  • marginal models

ASJC Scopus subject areas

  • Statistics and Probability
  • Statistics, Probability and Uncertainty

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