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In comparative effectiveness research, treated and control patients might have a different start of follow-up as treatment is often started later in the disease trajectory. This typically occurs when data from treated and controls are not…

Methodology · Statistics 2024-06-10 Rik van Eekelen , Patrick M. M. Bossuyt , Nan van Geloven

The aim of clinical effectiveness research using repositories of electronic health records is to identify what health interventions 'work best' in real-world settings. Since there are several reasons why the net benefit of intervention may…

Methodology · Statistics 2020-06-19 Jie Zhu , Blanca Gallego

Many interventions are both beneficial to initiate and harmful to stop. Traditionally, to determine whether to deploy that intervention in a time-limited way depends on if, on average, the increase in the benefits of starting it outweigh…

Treatment policy estimands are frequently favored by regulators, as they assess the effect of treatment assignment regardless of post-randomization events. Despite best efforts, missing data due to study discontinuation cannot be fully…

Methodology · Statistics 2026-05-13 Ajmal Oodally , Craig Wang , Zheng Li , Tim Morris , Tobias Mütze , Arunava Chakravartty

With the increased availability of large health databases comes the opportunity of evaluating treatment effect on new data sources.Through these databases time-dependent outcomes can be analysed as events that can be measured using counting…

Methodology · Statistics 2022-04-08 Camille Nevoret , Sandrine Katsahian , Agathe Guilloux

In the statistical literature, a number of methods have been proposed to ensure valid inference about marginal effects of variables on a longitudinal outcome in settings with irregular monitoring times. However, the potential biases due to…

Methodology · Statistics 2021-12-23 Janie Coulombe , Erica E M Moodie , Robert W Platt

This paper considers the problem of inferring the causal effect of a variable $Z$ on a dependently censored survival time $T$. We allow for unobserved confounding variables, such that the error term of the regression model for $T$ is…

Statistics Theory · Mathematics 2024-10-02 Gilles Crommen , Jad Beyhum , Ingrid Van Keilegom

Estimation of treatment efficacy of real-world clinical interventions involves working with continuous outcomes such as time-to-death, re-hospitalization, or a composite event that may be subject to censoring. Counterfactual reasoning in…

Machine Learning · Computer Science 2022-08-11 Chirag Nagpal , Mononito Goswami , Keith Dufendach , Artur Dubrawski

This paper proposes a framework that incorporates the two-way fixed effects model as a special case to conduct causal inference with a continuous treatment. Treatments are allowed to change over time and potential outcomes are dependent on…

Methodology · Statistics 2025-07-01 Zhiguo Xiao , Peikai Wu

Marginal structural models (MSMs) are widely used in observational studies to estimate the causal effect of time-varying treatments. Despite its popularity, limited attention has been paid to summarizing the treatment history in the outcome…

Methodology · Statistics 2024-09-18 Jiewen Liu , Todd A. Miano , Stephen Griffiths , Michael G. S. Shashaty , Wei Yang

Many clinical questions involve estimating the effects of multiple treatments using observational data. When using longitudinal data, the interest is often in the effect of treatment strategies that involve sustaining treatment over time.…

Methodology · Statistics 2024-05-03 Emily Granger , Gwyneth Davies , Ruth H. Keogh

This paper addresses the problem of identifying and estimating the causal effect of a treatment in the presence of unmeasured confounding and various types of right-censoring. Examples of these censoring mechanisms are administrative…

Statistics Theory · Mathematics 2025-03-19 Ilias Willems , Sara Rutten , Gilles Crommen , Ingrid Van Keilegom

This paper studies the identification of causal effects of a continuous treatment using a new difference-in-difference strategy. Our approach allows for endogeneity of the treatment, and employs repeated cross-sections. It requires an…

Econometrics · Economics 2023-04-18 Xavier D'Haultfoeuille , Stefan Hoderlein , Yuya Sasaki

The target trial framework enables causal inference from longitudinal observational data by emulating randomized trials initiated at multiple time points. Precision is often improved by pooling information across trials, with standard…

Methodology · Statistics 2026-01-08 Edoardo Efrem Gervasoni , Liesbet De Bus , Stijn Vansteelandt , Oliver Dukes

In clinical trials, an experimental treatment is sometimes added on to a standard of care or control therapy in multiple treatment phases (e.g., concomitant and maintenance phases) to improve patient outcomes. When the new regimen provides…

Methodology · Statistics 2020-11-19 Sudipta Bhattacharya , Jyotirmoy Dey

It is often of interest in observational studies to measure the causal effect of a treatment on time-to-event outcomes. In a medical setting, observational studies commonly involve patients who initiate medication therapy and others who do…

Methodology · Statistics 2019-10-07 Reagan Mozer , Mark E. Glickman

Treatment switching in a randomized controlled trial is said to occur when a patient randomized to one treatment arm switches to another treatment arm during follow-up. This can occur at the point of disease progression, whereby patients in…

Methodology · Statistics 2021-03-24 Andrew Ying , Eric J. Tchetgen Tchetgen

Uncertainty quantification of causal effects is crucial for safety-critical applications such as personalized medicine. A powerful approach for this is conformal prediction, which has several practical benefits due to model-agnostic…

Machine Learning · Computer Science 2026-02-04 Maresa Schröder , Dennis Frauen , Jonas Schweisthal , Konstantin Heß , Valentyn Melnychuk , Stefan Feuerriegel

Observational studies of recurrent event rates are common in biomedical statistics. Broadly, the goal is to estimate differences in event rates under two treatments within a defined target population over a specified followup window.…

Methodology · Statistics 2024-11-13 Arman Oganisian , Anthony Girard , Jon A. Steingrimsson , Patience Moyo

The article is focused on studying how to predict the failure times of coherent systems from the early failure times of their components. Both the cases of independent and dependent components are considered by assuming that they are…

Applications · Statistics 2024-09-30 Jorge Navarro , Antonio Arriaza , Alfonso Suárez-Llorens