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Propensity score methods are increasingly being used to reduce estimation bias of treatment effects for observational studies. Previous research has shown that propensity score methods consistently estimate the marginal hazard ratio for…

Methodology · Statistics 2019-11-19 Haodi Liang , Cecilia Cotton

Methods to handle missing data have been extensively explored in the context of estimation and descriptive studies, with multiple imputation being the most widely used method in clinical research. However, in the context of clinical risk…

Methodology · Statistics 2024-11-25 Junhui Mi , Rahul D. Tendulkar , Sarah M. C. Sittenfeld , Sujata Patil , Emily C. Zabor

Principal component analysis (PCA) is recognised as a quintessential data analysis technique when it comes to describing linear relationships between the features of a dataset. However, the well-known sensitivity of PCA to non-Gaussian…

Machine Learning · Statistics 2019-10-28 Jean P. Chereau , Bruno Scalzo Dees , Danilo P. Mandic

The use of flexible machine-learning (ML) models to generate imputations of missing data within the framework of Multiple Imputation (MI) has recently gained traction, particularly in observational settings. For randomised controlled trials…

Methodology · Statistics 2025-10-07 Mia S. Tackney , Jonathan W. Bartlett , Elizabeth Williamson , Kim May Lee

Epidemiologic studies and clinical trials with a survival outcome are often challenged by immortal time (IMT), a period of follow-up during which the survival outcome cannot occur because of the observed later treatment initiation. It has…

Applications · Statistics 2022-02-08 Jiping Wang , Peter Peduzzi , Michael Wininger , Shuangge Ma

Practical problems with missing data are common, and statistical methods have been developed concerning the validity and/or efficiency of statistical procedures. On a central focus, there have been longstanding interests on the mechanism…

Methodology · Statistics 2020-03-26 Rui Duan , C. Jason Liang , Pamela Shaw , Cheng Yong Tang , Yong Chen

Inverse probability weighting (IPW) methods are commonly used to analyze non-ignorable missing data under the assumption of a logistic model for the missingness probability. However, solving IPW equations numerically may involve…

Methodology · Statistics 2025-07-24 Pengfei Li , Jing Qin , Yukun Liu

A simple and common type of medical research involves the comparison of one treatment against another. The logical aim should be both to establish which treatment is superior and the strength of evidence supporting this conclusion, a task…

Methodology · Statistics 2022-12-08 Nicholas Adams

Data mining and machine learning techniques such as classification and regression trees (CART) represent a promising alternative to conventional logistic regression for propensity score estimation. Whereas incomplete data preclude the…

Machine Learning · Statistics 2018-07-26 Bas B. L. Penning de Vries , Maarten van Smeden , Rolf H. H. Groenwold

Standard methods for estimating average causal effects require complete observations of the exposure and confounders. In observational studies, however, missing data are ubiquitous. Motivated by a study on the effect of prescription opioids…

Methodology · Statistics 2025-06-30 Lan Wen , Glen McGee

Count endpoints are common in clinical trials, particularly for recurrent events such as hypoglycemia. When interest centers on comparing overall event rates between treatment groups, negative binomial (NB) regression is widely used because…

Methodology · Statistics 2026-01-27 Jiren Sun , Linda Amoafo , Yongming Qu

External information, such as prior information or expert opinions, can play an important role in the design, analysis and interpretation of clinical trials. However, little attention has been devoted thus far to incorporating external…

Applications · Statistics 2013-04-24 Minge Xie , Regina Y. Liu , C. V. Damaraju , William H. Olson

The naive importance sampling (IS) estimator generally does not work well in examples involving simultaneous inference on several targets, as the importance weights can take arbitrarily large values, making the estimator highly unstable. In…

Methodology · Statistics 2022-04-20 Vivekananda Roy , Evangelos Evangelou

Exclusion and exogeneity are core assumptions in instrumental variable (IV) analyses, but their empirical validity is often debated. This paper develops new sensitivity analyses for these assumptions. Our results accommodate arbitrary…

Econometrics · Economics 2026-04-10 Paul Diegert , Matthew A. Masten , Alexandre Poirier

In most medical research, the average treatment effect is used to evaluate a treatment's performance. However, precision medicine requires knowledge of individual treatment effects: What is the difference between a unit's measurement under…

Computation · Statistics 2022-08-30 Mingyang Cai , Stef van Buuren , Gerko Vink

Missing values are a major challenge in most data science projects working on real data. To avoid losing valuable information, imputation methods are used to fill in missing values with estimates, allowing the preservation of samples or…

Machine Learning · Computer Science 2024-07-17 Pedro Pons-Suñer , Laura Arnal , J. Ramón Navarro-Cerdán , François Signol

Missing data is a common issue in real-world datasets. This paper studies the performance of impute-then-regress pipelines by contrasting theoretical and empirical evidence. We establish the asymptotic consistency of such pipelines for a…

Machine Learning · Statistics 2025-01-08 Dimitris Bertsimas , Arthur Delarue , Jean Pauphilet

While observational data are routinely used to estimate causal effects of biomedical treatments, doing so requires special methods to adjust for observed confounding. These methods invariably rely on untestable statistical and causal…

Methodology · Statistics 2026-03-02 Arman Oganisian

This paper introduces a novel iterative method for missing data imputation that sequentially reduces the mutual information between data and the corresponding missingness mask. Inspired by GAN-based approaches that train generators to…

Machine Learning · Statistics 2025-11-26 Jiahao Yu , Qizhen Ying , Leyang Wang , Ziyue Jiang , Song Liu

Missing values in electronic health record (EHR) data pose a significant challenge for epidemiologic research. Traditional methods for handling missing data, like mean imputation, may introduce bias. Multiple imputation (MI) offers a…