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Nicholas Hartman

Publications and source records attributed to Nicholas Hartman.

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Smooth Concordance Metrics for Survival Models

Concordance indices are widely popular metrics for assessing the ability of predictive survival models to discriminate underlying risk levels. However, these statistics have also been criticized for using only the rank orderings of the model's predicted risk scores and being insensitive to important model features, such as the addition of strong predictor variables into the model. In this paper, we address these limitations by developing smooth concordance metrics that model the underlying risk discrimination probabilities as continuous functions of the predicted risk score differences, where the shapes of these functions are estimated from the observed data. As a result, these smooth concordance metrics assess model performance across the entire range of possible risk score differences, allowing one to identify specific scenarios where the candidate model performs especially well or better than other models. Simulations show that the proposed smooth concordance metrics provide more detailed information about risk discrimination performance and are much more sensitive to the addition of meaningful predictors. We apply these methods to compare predictive survival models for cancer recurrence.

stat.ME

Targeted Parameter Estimation for Robust Empirical Bayes Ranking

Ordering the expected outcomes across a collection of clusters after performing a covariate adjustment commonly arises in many applied settings, such as healthcare provider evaluation. Regression parameters in such covariate adjustment models are frequently estimated by maximum likelihood or through other criteria that do not directly evaluate the quality of the rankings resulting from using a particular set of parameter estimates. In this article, we propose both a novel empirical Bayes ranking procedure and an associated estimation approach for finding the regression parameters of the covariate adjustment model. By building our ranking approach around estimating approximate percentiles of the covariate-adjusted cluster-level means, we are able to develop manageable expressions for the expected ranking squared-error loss associated with any choice of the covariate-adjustment model parameters, and we harness this to generate a novel unbiased estimator for this expected loss. Minimization of this unbiased estimator directly leads to a novel ranking procedure that is often more robust than conventional empirical Bayes ranking methods. Through a series of simulation studies, we show that our approach consistently delivers improved ranking squared-error performance relative to competing methods, such as posterior expected ranks and ranking the components of the best linear unbiased predictor. Estimating rankings using our method is illustrated with an example from a longitudinal study evaluating test scores across a large group of schools.

stat.ME

Optimal Survival Analyses With Prevalent and Incident Patients

Period-prevalent cohorts are often used for their cost-saving potential in epidemiological studies of survival outcomes. Under this design, prevalent patients allow for evaluations of long-term survival outcomes without the need for long follow-up, whereas incident patients allow for evaluations of short-term survival outcomes without the issue of left-truncation. In most period-prevalent survival analyses from the existing literature, patients have been recruited to achieve an overall sample size, with little attention given to the relative frequencies of prevalent and incident patients and their statistical implications. Furthermore, there are no existing methods available to rigorously quantify the impact of these relative frequencies on estimation and inference and incorporate this information into study design strategies. To address these gaps, we develop an approach to identify the optimal mix of prevalent and incident patients that maximizes precision over the entire estimated survival curve, subject to a flexible weighting scheme. In addition, we prove that inference based on the weighted log-rank test or Cox proportional hazards model is most powerful with an entirely prevalent or incident cohort, and we derive theoretical formulas to determine the optimal choice. Simulations confirm the validity of the proposed optimization criteria and show that substantial efficiency gains can be achieved by recruiting the optimal mix of prevalent and incident patients. The proposed methods are applied to assess waitlist outcomes among kidney transplant candidates.

stat.ME

Robust Privacy-Preserving Models for Cluster-Level Confounding: Recognizing Disparities in Access to Transplantation

In applications where the study data are collected within cluster units (e.g., patients within transplant centers), it is often of interest to estimate and perform inference on the treatment effects of the cluster units. However, it is well-established that cluster-level confounding variables can bias these assessments, and many of these confounding factors may be unobservable. In healthcare settings, data sharing restrictions often make it impossible to directly fit conventional risk-adjustment models on patient-level data, and existing privacy-preserving approaches cannot adequately adjust for both observed and unobserved cluster-level confounding factors. In this paper, we propose a privacy-preserving model for cluster-level confounding that only depends on publicly-available summary statistics, can be fit using a single optimization routine, and is robust to outlying cluster unit effects. In addition, we develop a Pseudo-Bayesian inference procedure that accounts for the estimated cluster-level confounding effects and corrects for the impact of unobservable factors. Simulations show that our estimates are robust and accurate, and the proposed inference approach has better Frequentist properties than existing methods. Motivated by efforts to improve equity in transplant care, we apply these methods to evaluate transplant centers while adjusting for observed geographic disparities in donor organ availability and unobservable confounders.

stat.AP

Composite Scores for Transplant Center Evaluation: A New Individualized Empirical Null Method

Risk-adjusted quality measures are used to evaluate healthcare providers while controlling for factors beyond their control. Existing healthcare provider profiling approaches typically assume that the risk adjustment is perfect and the between-provider variation in quality measures is entirely due to the quality of care. However, in practice, even with very good models for risk adjustment, some between-provider variation will be due to incomplete risk adjustment, which should be recognized in assessing and monitoring providers. Otherwise, conventional methods disproportionately identify larger providers as outliers, even though their provider effects need not be "extreme.'' Motivated by efforts to evaluate the quality of care provided by transplant centers, we develop a composite evaluation score based on a novel individualized empirical null method, which robustly accounts for overdispersion due to unobserved risk factors, models the marginal variance of standardized scores as a function of the effective center size, and only requires the use of publicly-available center-level statistics. The evaluations of United States kidney transplant centers based on the proposed composite score are substantially different from those based on conventional methods. Simulations show that the proposed empirical null approach more accurately classifies centers in terms of quality of care, compared to existing methods.

stat.AP