On the use of stochastic ordering to test for trend with clustered binary data

We introduce the use of stochastic ordering for defining treatment-related trend in clustered exchangeable binary data for both when cluster sizes are fixed and when they vary randomly. In the latter case, there is a well-documented tendency for such data to be sparse, a problem we address by making an assumption of interpretability or, equivalently, marginal compatibility. Our procedures are based on a representation of the joint distribution of binary exchangeable random variables by a saturated model, and may hence be considered nonparametric. The definition of trend by stochastic ordering is proposed to ensure a flexibility that allows for various forms of monotone increases in response to the cluster as a whole to be included in the evaluation of the trend. We obtain maximum likelihood estimates of probability functions under stochastic ordering using mixture-likelihood-based algorithms. Since the data are sparse, we avoid the use of asymptotic results and obtain p-values of the likelihood ratio procedures by permutation resampling. We demonstrate how the proposed framework can be used in risk assessment, and provide comparisons with existing procedures. Copyright 2010, Oxford University Press.