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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…

统计方法学 · 统计学 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…

统计方法学 · 统计学 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…

统计方法学 · 统计学 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…

应用统计 · 统计学 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…

机器学习 · 计算机科学 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.…

机器学习 · 计算机科学 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…

统计方法学 · 统计学 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…

统计方法学 · 统计学 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…

统计理论 · 数学 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…

统计理论 · 数学 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…

应用统计 · 统计学 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…

统计方法学 · 统计学 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…

统计方法学 · 统计学 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…

统计方法学 · 统计学 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,…

统计方法学 · 统计学 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.…

机器学习 · 计算机科学 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…

统计方法学 · 统计学 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…

统计方法学 · 统计学 2024-01-19 Andrew M. Raim , Nagaraj K. Neerchal , Jorge G. Morel