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Case-cohort studies are conducted within cohort studies, wherein collection of exposure data is limited to a subset of the cohort, leading to a large proportion of missing data by design. Standard analysis uses inverse probability weighting…

Varying coefficient models are widely used to characterize dynamic associations between longitudinal outcomes and covariates. Existing work on varying coefficient models, however, all assumes that observation times are independent of the…

Methodology · Statistics 2026-01-27 Yu Gu , Yangjianchen Xu , Peijun Sang

In pharmacoepidemiology, safety and effectiveness are frequently evaluated using readily available administrative and electronic health records data. In these settings, detailed confounder data are often not available in all data sources…

Selection bias can hinder accurate estimation of association parameters in binary disease risk models using non-probability samples like electronic health records (EHRs). The issue is compounded when participants are recruited from multiple…

Micro-randomized trials (MRTs) are increasingly used to evaluate mobile health interventions with binary proximal outcomes. Standard inverse probability weighting (IPW) estimators are unbiased but unstable in small samples or under extreme…

Methodology · Statistics 2025-10-10 Jinho Cha , Eunchan Cha

Win measures, including the win ratio (WR), win odds (WO), net benefit (NB), and desirability of outcome ranking (DOOR), are increasingly used in randomized clinical trials with multiple hierarchical ordinal endpoints. In practice, however,…

Methodology · Statistics 2026-05-27 Yi Liu , Huiman Barnhart , Sean O'Brien , Yuliya Lokhnygina , Roland A. Matsouaka

Trial-based economic evaluations are typically performed on cross-sectional variables, derived from the responses for only the completers in the study, using methods that ignore the complexities of utility and cost data (e.g. skewness and…

Methodology · Statistics 2018-05-21 Andrea Gabrio , Michael J. Daniels , Gianluca Baio

The inverse probability weighting (IPW) is broadly utilized to address missing data problems including causal inference but may suffer from large variances and biases due to propensity score model misspecification. To solve these problems,…

Methodology · Statistics 2020-08-05 Hiroto Katsumata

Rheumatoid arthritis clinical trials are strategically designed to collect the disease activity score of each patient over multiple clinical visits, meanwhile a patient may drop out before their intended completion due to various reasons.…

Applications · Statistics 2018-01-29 Violeta G. Hennessey , Luis G. Leon-Novelo , Juan Li , Li Zhu , Eric Chi , Joseph G. Ibrahim

Causal inference requires evaluating models on balanced distributions between treatment and control groups, while training data often exhibits imbalance due to historical decision-making policies. Most conventional statistical methods…

Machine Learning · Statistics 2025-11-21 Akira Tanimoto

In observational studies, the propensity score plays a central role in estimating causal effects of interest. The inverse probability weighting (IPW) estimator is commonly used for this purpose. However, if the propensity score model is…

Methodology · Statistics 2025-03-21 Shunichiro Orihara , Tomotaka Momozaki , Tomoyuki Nakagawa

Generalized estimating equation (GEE) is widely adopted for regression modeling for longitudinal data, taking account of potential correlations within the same subjects. Although the standard GEE assumes common regression coefficients among…

Methodology · Statistics 2022-07-11 Tsubasa Ito , Shonosuke Sugasawa

We consider estimation of average treatment effects given observational data with high-dimensional pretreatment variables. Existing methods for this problem typically assume some form of sparsity for the regression functions. In this work,…

Methodology · Statistics 2024-04-12 Yuhao Wang , Rajen D. Shah

Longitudinal datasets measured repeatedly over time from individual subjects, arise in many biomedical, psychological, social, and other studies. A common approach to analyse high-dimensional data that contains missing values is to learn a…

Machine Learning · Statistics 2021-04-21 Siddharth Ramchandran , Gleb Tikhonov , Kalle Kujanpää , Miika Koskinen , Harri Lähdesmäki

We develop a novel doubly-robust (DR) imputation framework for longitudinal studies with monotone dropout, motivated by the informative dropout that is common in FDA-regulated trials for Alzheimer's disease. In this approach, the missing…

Methodology · Statistics 2023-05-05 Yuqi Qiu , Karen Messer

In longitudinal studies using routinely collected data, such as electronic health records (EHRs), patients tend to have more measurements when they are unwell; this informative observation pattern may lead to bias. While semi-parametric…

Methodology · Statistics 2024-10-02 Rose Garrett , Brian Feldman , Eleanor Pullenayegum

Causal inference methods for treatment effect estimation usually assume independent units. However, this assumption is often questionable because units may interact, resulting in spillover effects between them. We develop augmented inverse…

Methodology · Statistics 2025-04-08 Corinne Emmenegger , Meta-Lina Spohn , Timon Elmer , Peter Bühlmann

While the inverse probability of treatment weighting (IPTW) is a commonly used approach for treatment comparisons in observational data, the resulting estimates may be subject to bias and excessively large variance when there is lack of…

Methodology · Statistics 2024-02-13 Zhiqiang Cao , Lama Ghazi , Claudia Mastrogiacomo , Laura Forastiere , F. Perry Wilson , Fan Li

Longitudinal data are common in clinical trials and observational studies, where missing outcomes due to dropouts are always encountered. Under such context with the assumption of missing at random, the weighted generalized estimating…

Methodology · Statistics 2019-04-30 Chixiang Chen , Biyi Shen , Lijun Zhang , Yuan Xue , Ming Wang

Extending generalized estimating equations (GEE) to ordinal response data requires a conversion of the ordinal response to a vector of binary category indicators. That leads to a rather complicated association structure, and the…

Methodology · Statistics 2017-05-23 Aristidis K. Nikoloulopoulos