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RCTs sometimes test interventions that aim to improve existing services targeted to a subset of individuals identified after randomization. Accordingly, the treatment could affect the composition of service recipients and the offered…

Methodology · Statistics 2022-05-18 Peter Z. Schochet

Clustered observations are ubiquitous in controlled and observational studies and arise naturally in multi-centre trials or longitudinal surveys. We present a novel model for the analysis of clustered observations where the marginal…

Methodology · Statistics 2022-11-03 Luisa Barbanti , Torsten Hothorn

Many research questions in public health and medicine concern sustained interventions in populations defined by substantive priorities. Existing methods to answer such questions typically require a measured covariate set sufficient to…

Methodology · Statistics 2023-06-30 Audrey Renson , Michael Hudgens , Alexander Keil , Paul Zivich , Allison Aiello

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

Methods for the evaluation of the predictive accuracy of biomarkers with respect to survival outcomes subject to right censoring have been discussed extensively in the literature. In cancer and other diseases, survival outcomes are commonly…

Methodology · Statistics 2018-06-06 Yuan Wu , Xiaofei Wang , Jiaxing Lin , Beilin Jia , Kouros Owzar

The primary analysis of randomized screening trials for cancer typically adheres to the intention-to-screen principle, measuring cancer-specific mortality reductions between screening and control arms. These mortality reductions result from…

Methodology · Statistics 2021-07-30 Sudipta Saha , Zhihui Liu , Olli Saarela

In this work, we are interested in studying the causal effect of an endogenous binary treatment on a dependently censored duration outcome. By dependent censoring, it is meant that the duration time ($T$) and right censoring time ($C$) are…

Econometrics · Economics 2025-04-04 Gilles Crommen , Jad Beyhum , Ingrid Van Keilegom

Stepped wedge cluster randomized trials (SW-CRTs) with binary outcomes are increasingly used in prevention and implementation studies. Marginal models represent a flexible tool for analyzing SW-CRTs with population-averaged interpretations,…

Methodology · Statistics 2021-01-05 Fan Li , Hengshi Yu , Paul J. Rathouz , Elizabeth L. Turner , John S. Preisser

Individualized treatment rules can lead to better health outcomes when patients have heterogeneous responses to treatment. Very few individualized treatment rule estimation methods are compatible with a multi-treatment observational study…

The restricted mean survival time is a clinically easy-to-interpret measure that does not require any assumption of proportional hazards. We focus on two ways to directly model the survival time and adjust the covariates. One is to…

Methodology · Statistics 2022-11-03 Keisuke Hanada , Junji Moriya , Masahiro Kojima

Stepped wedge cluster randomized trials (SW-CRTs) have become increasingly popular and are used for a variety of interventions and outcomes, often chosen for their feasibility advantages. SW-CRTs must account for time trends in the outcome…

Methodology · Statistics 2024-07-16 Lee Kennedy-Shaffer , Victor De Gruttola , Marc Lipsitch

The heterogeneous treatment effect plays a crucial role in precision medicine.There is evidence that real-world data, even subject to biases, can be employed as supplementary evidence for randomized clinical trials to improve the…

Methodology · Statistics 2025-09-04 Guangcai Mao , Shu Yang , Xiaofei Wang

In Huntington's disease research, a current goal is to understand how symptoms change prior to a clinical diagnosis. Statistically, this entails modeling symptom severity as a function of the covariate 'time until diagnosis', which is often…

Methodology · Statistics 2024-09-19 Seong-ho Lee , Brian D. Richardson , Yanyuan Ma , Karen S. Marder , Tanya P. Garcia

Hazard ratios are ubiquitously used in time to event analysis to quantify treatment effects. Although hazard ratios are invaluable for hypothesis testing, other measures of association, both relative and absolute, may be used to fully…

Methodology · Statistics 2020-11-02 Federico Ambrogi , Simona Iacobelli , Per Kragh Andersen

Longitudinal cluster randomized trials (L-CRTs) are increasingly used to evaluate the cost-effectiveness of healthcare interventions across multiple assessment periods, yet design methods for powering these trials remain underdeveloped.…

Methodology · Statistics 2026-03-20 Hao Wang , Jingxia Liu , Drew B. Cameron , Jiaqi Tong , Donna Spiegelman , Daniella Meeker , Fan Li

Estimating prognosis conditional on surviving an initial high-risk period is crucial in clinical research. Yet, standard metrics such as hazard ratios are often difficult to interpret, while mean-based summaries are sensitive to outliers…

Methodology · Statistics 2026-02-16 Taekwon Hong , Woojung Bae , Sang Kyu Lee , Dongrak Choi , Jong-Hyeon Jeong

Censored quantile regression (CQR) has become a valuable tool to study the heterogeneous association between a possibly censored outcome and a set of covariates, yet computation and statistical inference for CQR have remained a challenge…

Statistics Theory · Mathematics 2022-10-25 Xuming He , Xiaoou Pan , Kean Ming Tan , Wen-Xin Zhou

Sequential nested trial (SNT) emulation is a powerful approach for maximizing precision and avoiding time-related biases. However, there exists little discussion about the implied causal estimands in comparison to a real-world single point…

Estimating a unit's responses to interventions with an associated dose, the "conditional average dose response" (CADR), is relevant in a variety of domains, from healthcare to business, economics, and beyond. Such a response typically needs…

Machine Learning · Computer Science 2024-07-29 Christopher Bockel-Rickermann , Toon Vanderschueren , Jeroen Berrevoets , Tim Verdonck , Wouter Verbeke

We investigate the estimation of the extreme value index when the data are subject to random censorship. We prove, in a unified way, detailed asymptotic normality results for various estimators of the extreme value index and use these…

Statistics Theory · Mathematics 2008-12-18 John H. J. Einmahl , Amélie Fils-Villetard , Armelle Guillou