Proving Efficiency of NPMLE and Identities

A method is given for proving efficiency of NPMLE directly linked to empirical process theory. The conditions in general are appropriate consistency of the NPMLE, differentiability of the model, differentiability of the parameter of interest, local convexity of the parameter space, and a Donsker class condition for the class of efficient influence functions obtained by varying the parameters. For the case that the model is linear in the parameter and the parameter space is convex, as with most nonparametric missing data models, we show that the method leads to an identity for the NPMLE which almost says that the NPMLE is efficient and provides us straightforwardly with a consistency and efficiency proof. This identify is extended to an almost linear class of models which contain biased sampling models. To illustrate, the method is applied to the univariate censoring model, random truncation models, interval censoring case I model, the class of parametric models and to a class of semiparametric models.

MULTIPLE MODEL EVALUATION ABSENT THE GOLD STANDARD VIA MODEL COMBINATION

We describe a method for evaluating an ensemble of predictive models given a sample of observations comprising the model predictions and the outcome event measured with error. Our formulation allows us to simultaneously estimate measurement error parameters, true outcome — aka the gold standard — and a relative weighting of the predictive scores. We describe conditions necessary to estimate the gold standard and for these estimates to be calibrated and detail how our approach is related to, but distinct from, standard model combination techniques. We apply our approach to data from a study to evaluate a collection of BRCA1/BRCA2 gene mutation prediction scores. In this example, genotype is measured with error by one or more genetic assays. We estimate true genotype for each individual in the dataset, operating characteristics of the commonly used genotyping procedures and a relative weighting of the scores. Finally, we compare the scores against the gold standard genotype and find that Mendelian scores are, on average, the more refined and better calibrated of those considered and that the comparison is sensitive to measurement error in the gold standard.