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Adjustment for prognostic baseline variables can reduce bias due to covariate imbalance and increase efficiency in randomized trials. While the use of covariate adjustment in late-phase trials is justified by favorable large-sample…

Methodology · Statistics 2025-04-08 Ethan Ashby , Bo Zhang , Genevieve G Fouda , Youyi Fong , Holly Janes

We consider the problem of indirect comparison, where a treatment arm of interest is absent by design in one randomized controlled trial but available in the other. The former is the target trial, and the latter is the source trial. The…

Methodology · Statistics 2025-06-06 Zehao Su , Helene C. W. Rytgaard , Henrik Ravn , Frank Eriksson

Proxy variables are commonly used in causal inference when unmeasured confounding exists. While most existing proximal methods assume a unidirectional causal relationship between two primary variables, many social and biological systems…

Methodology · Statistics 2025-07-21 Jiaqi Min , Xueyue Zhang , Shanshan Luo

The probability-scale residual (PSR) is well defined across a wide variety of variable types and models, making it useful for studies of HIV/AIDS. In this manuscript, we highlight some of the properties of the PSR and illustrate its…

Applications · Statistics 2018-03-02 Bryan E. Shepherd , Qi Liu , Valentine Wanga , Chun Li

Previous deep learning approaches for survival analysis have primarily relied on ranking losses to improve discrimination performance, which often comes at the expense of calibration performance. To address such an issue, we propose a novel…

Machine Learning · Computer Science 2024-11-22 Dongjoon Lee , Hyeryn Park , Changhee Lee

The distribution-free method of conformal prediction (Vovk et al, 2005) has gained considerable attention in computer science, machine learning, and statistics. Candes et al. (2023) extended this method to right-censored survival data,…

Methodology · Statistics 2025-06-04 Jing Qin , Jin Piao , Jing Ning , Yu Shen

Inferring causal effects from an observational study is challenging because participants are not randomized to treatment. Observational studies in infectious disease research present the additional challenge that one participant's treatment…

Methodology · Statistics 2020-12-25 Brian G. Barkley , Michael G. Hudgens , John D. Clemens , Mohammad Ali , Michael E. Emch

Causal mediation analysis has been extended to estimate path-specific effects with multiple intermediate variables, isolating treatment effects through a mediator of interest while excluding pathways through its ancestors. Such analyses…

Methodology · Statistics 2026-05-12 Yang Bai , Sihan Wu , Baoluo Sun , Yifan Cui

We present a new joint longitudinal and survival model aimed at estimating the association between the risk of an event and the change in and history of a biomarker that is repeatedly measured over time. We use cubic B-splines models for…

Applications · Statistics 2009-10-12 Elizabeth R. Brown

Modeling symptom progression to identify informative subjects for a new Huntington's disease clinical trial is problematic since time to diagnosis, a key covariate, can be heavily censored. Imputation is an appealing strategy where censored…

Methodology · Statistics 2025-02-11 Sarah C. Lotspeich , Tanya P. Garcia

Instrumental variable (IV) methods are central to causal inference from observational data, particularly when a randomized experiment is not feasible. However, of the three conventional core IV identification conditions, only one, IV…

Methodology · Statistics 2025-09-23 Zhonghua Liu , Baoluo Sun , Ting Ye , David Richardson , Eric Tchetgen Tchetgen

Learning causal relationships among a set of variables, as encoded by a directed acyclic graph, from observational data is complicated by the presence of unobserved confounders. Instrumental variables (IVs) are a popular remedy for this…

Methodology · Statistics 2025-04-17 Jing Zou , Wei Li , Wei Lin

Estimating the prevalence of a medical condition, or the proportion of the population in which it occurs, is a fundamental problem in healthcare and public health. Accurate estimates of the relative prevalence across groups -- capturing,…

Computers and Society · Computer Science 2023-12-13 Divya Shanmugam , Kaihua Hou , Emma Pierson

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

We study within-host HIV dynamics using a three--component nonlinear ordinary differential equation model for healthy CD4$^{+}$ T cells, infected CD4$^{+}$ T cells, and free virus. In addition to the baseline model without treatment, we…

Numerical Analysis · Mathematics 2025-12-16 Abdul Rab

Instrumental variable (IV) methods allow us the opportunity to address unmeasured confounding in causal inference. However, most IV methods are only applicable to discrete or continuous outcomes with very few IV methods for censored…

Methodology · Statistics 2020-09-30 Youjin Lee , Edward H. Kennedy , Nandita Mitra

Prevalent cohort sampling is commonly used to study the natural history of a disease when the disease is rare or it usually takes a long time to observe the failure event. It is known, however, that the collected sample in this situation is…

Methodology · Statistics 2022-09-05 Omidali Aghababaei Jazi

To infer the treatment effect for a single treated unit using panel data, synthetic control methods construct a linear combination of control units' outcomes that mimics the treated unit's pre-treatment outcome trajectory. This linear…

Methodology · Statistics 2024-05-07 Hongxiang Qiu , Xu Shi , Wang Miao , Edgar Dobriban , Eric Tchetgen Tchetgen

Background: Estimations of causal effects from observational data are subject to various sources of bias. One method of adjusting for the residual biases in the estimation of a treatment effect is through negative control outcomes, where…

Methodology · Statistics 2022-07-29 Hon Hwang , Juan C Quiroz , Blanca Gallego

Coarse structural nested mean models are used to estimate treatment effects from longitudinal observational data. Coarse structural nested mean models lead to a large class of estimators. It turns out that estimates and standard errors may…

Statistics Theory · Mathematics 2021-06-25 Judith J. Lok , Department of Mathematics , Statistics , Boston University