Covariate-adjusted generalized pairwise comparisons in small samples

Abstract

Semiparametric Probabilistic Index Models allow for the comparison of two groups of observations, whilst adjusting for covariates, thereby fitting nicely within the framework of Generalized Pairwise Comparisons. As with most regression approaches in this setting, the limited amount of data results in invalid inference as the asymptotic normality assumption is not met. In addition, separation issues might arise when considering small samples. In this paper, we show that the parameters of the Probabilistic Index Model can be estimated using Generalized Estimating Equations, for which adjustments exist that lead to estimators of the sandwich variance-covariance matrix with improved finite sample properties and that can deal with bias due to separation. In this way, appropriate inference can be performed as is shown through extensive simulation studies. The known relationships between the probabilistic index and other GPC statistics allow to also provide valid inference for e.g. the net treatment benefit or the success odds.