arXiv subjects

Thomas P. A. Debray

Publications and source records attributed to Thomas P. A. Debray.

5 recordsLinked to original sources

arXiv2026

Non-parametric assessment of the calibration of individualized treatment effects

An important aspect of the performance of algorithms that predict individualized treatment effects (ITE) is moderate calibration, i.e., the average treatment effect among individuals with predicted treatment effect of z being equal to z. The assessment of moderate calibration is challenging on two fronts: counterfactual responses are unobserved, and quantifying the conditional response function for models that generate continuous predicted values requires regularization. Perhaps because of these challenges, there is currently no inferential method for the null hypothesis that an ITE model is moderately calibrated in a population. In this work, we propose non-parametric methods for the assessment of moderate calibration of ITE models for binary outcomes using data from a randomized trial. These methods simultaneously resolve both challenges, resulting in novel graphical, numerical, and inferential methods for the assessment of moderate calibration. The key idea is to formulate a stochastic process for the cumulative prediction errors that obeys a functional central limit theorem, enabling the use of the properties of Brownian motion for asymptotic inference. We propose two approaches to construct this process from a sample: a conditional approach that relies on predicted risks (often an output of ITE models), and a marginal approach based on replacing the cumulative conditional moments with their marginal counterparts. Numerical simulations confirm the desirable properties of both approaches and their ability to detect miscalibration of different forms. We use a case study to provide suggestions on graphical presentation and the interpretation of results. Moderate calibration of predicted ITEs can be assessed without requiring regularization techniques or making assumptions about the functional form of treatment response. The accompanying cumulcalib R package implements this method.

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arXiv2021

Adjusting for misclassification of an exposure in an individual participant data meta-analysis

A common problem in the analysis of multiple data sources, including individual participant data meta-analysis (IPD-MA), is the misclassification of binary variables. Misclassification may lead to biased estimates of model parameters, even when the misclassification is entirely random. We aimed to develop statistical methods that facilitate unbiased estimation of adjusted and unadjusted exposure-outcome associations and between-study heterogeneity in IPD-MA, where the extent and nature of exposure misclassification may vary across studies. We present Bayesian methods that allow misclassification of binary exposure variables to depend on study- and participant-level characteristics. In an example of the differential diagnosis of dengue using two variables, where the gold standard measurement for the exposure variable was unavailable for some studies which only measured a surrogate prone to misclassification, our methods yielded more accurate estimates than analyses naive with regard to misclassification or based on gold standard measurements alone. In a simulation study, the evaluated misclassification model yielded valid estimates of the exposure-outcome association, and was more accurate than analyses restricted to gold standard measurements. Our proposed framework can appropriately account for the presence of binary exposure misclassification in IPD-MA. It requires that some studies supply IPD for the surrogate and gold standard exposure and misclassification is exchangeable across studies conditional on observed covariates (and outcome). The proposed methods are most beneficial when few large studies that measured the gold standard are available, and when misclassification is frequent.

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arXiv2021

Quality control, data cleaning, imputation

This chapter addresses important steps during the quality assurance and control of RWD, with particular emphasis on the identification and handling of missing values. A gentle introduction is provided on common statistical and machine learning methods for imputation. We discuss the main strengths and weaknesses of each method, and compare their performance in a literature review. We motivate why the imputation of RWD may require additional efforts to avoid bias, and highlight recent advances that account for informative missingness and repeated observations. Finally, we introduce alternative methods to address incomplete data without the need for imputation.

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arXiv2021

Measurement Error in Meta-Analysis (MEMA) -- a Bayesian framework for continuous outcome data

Ideally, a meta-analysis will summarize data from several unbiased studies. Here we consider the less than ideal situation in which contributing studies may be compromised by measurement error. Measurement error affects every study design, from randomized controlled trials to retrospective observational studies. We outline a flexible Bayesian framework for continuous outcome data which allows one to obtain appropriate point and interval estimates with varying degrees of prior knowledge about the magnitude of the measurement error. We also demonstrate how, if individual-participant data (IPD) are available, the Bayesian meta-analysis model can adjust for multiple participant-level covariates, measured with or without measurement error.

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arXiv2017

Multiple imputation for multilevel data with continuous and binary variables

We present and compare multiple imputation methods for multilevel continuous and binary data where variables are systematically and sporadically missing. The methods are compared from a theoretical point of view and through an extensive simulation study motivated by a real dataset comprising multiple studies. Simulations are reproducible. The comparisons show why these multiple imputation methods are the most appropriate to handle missing values in a multilevel setting and why their relative performances can vary according to the missing data pattern, the multilevel structure and the type of missing variables. This study shows that valid inferences can only be obtained if the dataset gathers a large number of clusters. In addition, it highlights that heteroscedastic MI methods provide more accurate inferences than homoscedastic methods, which should be reserved for data with few individuals per cluster. Finally, the method of Quartagno and Carpenter (2016a) appears generally accurate for binary variables, the method of Resche-Rigon and White (2016) with large clusters, and the approach of Jolani et al. (2015) with small clusters.

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