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Patients often discontinue treatment in a clinical trial because their health condition is not improving. Consequently, the patients still in the study at the end of the trial have better health outcomes on average than the initial patient…

Methodology · Statistics 2022-06-06 Alex Ocampo , Heinz Schmidli , Peter Quarg , Francesca Callegari , Marcello Pagano

Missing data is a common problem in medical research, and is commonly addressed using multiple imputation. Although traditional imputation methods allow for valid statistical inference when data are missing at random (MAR), their…

In many clinical trials treatments need to be repeatedly applied as diseases relapse frequently after remission over a long period of time (e.g., 35 weeks). Most research in statistics focuses on the overall trial design, such as sample…

Methodology · Statistics 2014-04-01 Yanxun Xu , Yuan Ji

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

In many clinical trials, outcomes of interest include binary-valued endpoints. It is not uncommon that a binary-valued outcome is dichotomized from a continuous outcome at a threshold of clinical interest. To reach the objective, common…

Applications · Statistics 2021-08-05 Chenchen Ma , Xin Shen , Yongming Qu , Yu Du

Missing data is a common concern in health datasets, and its impact on good decision-making processes is well documented. Our study's contribution is a methodology for tackling missing data problems using a combination of synthetic dataset…

Machine Learning · Computer Science 2022-11-08 Gift Khangamwa , Terence L. van Zyl , Clint J. van Alten

In some causal inference scenarios, the treatment variable is measured inaccurately, for instance in epidemiology or econometrics. Failure to correct for the effect of this measurement error can lead to biased causal effect estimates.…

Machine Learning · Computer Science 2024-09-13 Antti Pöllänen , Pekka Marttinen

Linear quantile regression models aim at providing a detailed and robust picture of the (conditional) response distribution as function of a set of observed covariates. Longitudinal data represent an interesting field of application of such…

Methodology · Statistics 2015-07-30 Maria Francesca Marino , Nikos Tzavidis , Marco Alfo'

Understanding whether and how treatment effects vary across subgroups is crucial to inform clinical practice and recommendations. Accordingly, the assessment of heterogeneous treatment effects (HTE) based on pre-specified potential effect…

Methodology · Statistics 2023-12-04 Bryan S. Blette , Scott D. Halpern , Fan Li , Michael O. Harhay

Constant (naive) imputation is still widely used in practice as this is a first easy-to-use technique to deal with missing data. Yet, this simple method could be expected to induce a large bias for prediction purposes, as the imputed input…

Statistics Theory · Mathematics 2024-02-07 Alexis Ayme , Claire Boyer , Aymeric Dieuleveut , Erwan Scornet

Widely used methods for analyzing missing data can be biased in small samples. To understand these biases, we evaluate in detail the situation where a small univariate normal sample, with values missing at random, is analyzed using either…

Statistics Theory · Mathematics 2017-03-27 Paul T. von Hippel

The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed…

Applications · Statistics 2022-08-03 Lingjing Jiang , Michael Rosenblum , Yu Du

In modern large-scale observational studies, data collection constraints often result in partially labeled datasets, posing challenges for reliable causal inference, especially due to potential labeling bias and relatively small size of the…

Methodology · Statistics 2025-04-22 Yuqian Zhang , Abhishek Chakrabortty , Jelena Bradic

The analysis of randomized trials is often complicated by the occurrence of intercurrent events and missing values. Even though there are different strategies to address missing values it is still common to require missing values…

Methodology · Statistics 2025-11-11 A. Ruiz de Villa , Ll. Badiella

Missing data is a pervasive problem in epidemiology, with multiple imputation (MI) a commonly used analysis method. MI is valid when data are missing at random (MAR). However, definitions of MAR with multiple incomplete variables are not…

Methodology · Statistics 2025-04-14 Paul Madley-Dowd , Rachael A. Hughes , Maya B. Mathur , Jon Heron , Kate Tilling

Missing data occur frequently in empirical studies in health and social sciences, often compromising our ability to make accurate inferences. An outcome is said to be missing not at random (MNAR) if, conditional on the observed variables,…

Methodology · Statistics 2019-01-23 BaoLuo Sun , Lan Liu , Wang Miao , Kathleen Wirth , James Robins , Eric Tchetgen Tchetgen

Dynamic Bayesian networks (DBNs) are increasingly used in healthcare due to their ability to model complex temporal relationships in patient data while maintaining interpretability, an essential feature for clinical decision-making.…

Machine Learning · Computer Science 2026-04-30 Federico Pirola , Fabio Stella , Marco Grzegorczyk

Analysis of competing risks data is often complicated by the incomplete or selectively missing information on the cause of failure. Standard approaches typically assume that the cause of failure is missing at random (MAR), an assumption…

Dynamic prediction of causal effects under different treatment regimes conditional on an individual's characteristics and longitudinal history is an essential problem in precision medicine. This is challenging in practice because outcomes…

Methodology · Statistics 2023-03-07 Yizhen Xu , Jisoo Kim , Laura K. Hummers , Ami A. Shah , Scott Zeger

Finite mixture distributions arise in sampling a heterogeneous population. Data drawn from such a population will exhibit extra variability relative to any single subpopulation. Statistical models based on finite mixtures can assist in the…

Methodology · Statistics 2024-01-19 Andrew M. Raim , Nagaraj K. Neerchal , Jorge G. Morel