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In this paper, we outline a principled approach to estimate an individualized treatment rule that is appropriate for data from observational studies where, in addition to treatment assignment not being independent of individual…

Methodology · Statistics 2019-06-05 Jeremy Roth , Noah Simon

In this study, we develop consistent estimators for key parameters that govern the dynamics of tumor cell populations when subjected to pharmacological treatments. While these treatments often lead to an initial reduction in the abundance…

Applications · Statistics 2024-03-21 Kevin Leder , Ruping Sun , Zicheng Wang , Xuanming Zhang

Estimating causal effects on time-to-event outcomes from observational data is particularly challenging due to censoring, limited sample sizes, and non-random treatment assignment. The need for answering such "when-if" questions--how the…

Machine Learning · Computer Science 2025-11-19 Jessy Xinyi Han , Devavrat Shah

This paper considers the identification of dynamic treatment effects with panel data, in complex designs where the treatment may not be binary and may not be absorbing. We first show that under no-anticipation and parallel-trends…

Econometrics · Economics 2025-12-23 Clément de Chaisemartin , Xavier D'Haultfœuille

A treatment policy defines when and what treatments are applied to affect some outcome of interest. Data-driven decision-making requires the ability to predict what happens if a policy is changed. Existing methods that predict how the…

Machine Learning · Computer Science 2023-06-21 Çağlar Hızlı , ST John , Anne Juuti , Tuure Saarinen , Kirsi Pietiläinen , Pekka Marttinen

Stroke is a major cause of mortality and long--term disability in the world. Predictive outcome models in stroke are valuable for personalized treatment, rehabilitation planning and in controlled clinical trials. In this paper we design a…

Applications · Statistics 2016-02-24 Abhishek Sengupta , Vaibhav Rajan , Sakyajit Bhattacharya , G R K Sarma

With medical tests becoming increasingly available, concerns about over-testing and over-treatment dramatically increase. Hence, it is important to understand the influence of testing on treatment selection in general practice. Most…

Methodology · Statistics 2020-08-11 Yun Li , Irina Bondarenko , Michael R. Elliott , Timothy P. Hofer , Jeremy M. G. Taylor

Unmeasured confounding, unethical exposure, and ill-defined interventions pose significant challenges to evaluating policy-relevant mediation estimands in medicine and public health. In observational studies involving harmful exposures, the…

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

Quantifying a patient's health status provides clinicians with insight into patient risk, and the ability to better triage and manage resources. Early Warning Scores (EWS) are widely deployed to measure overall health status, and risk of…

Artificial Intelligence · Computer Science 2024-07-15 Thea Barnes , Enrico Werner , Jeffrey N. Clark , Raul Santos-Rodriguez

In the presence of competing events, many investigators are interested in a direct treatment effect on the event of interest that does not capture treatment effects on competing events. Classical survival analysis methods that treat…

Methodology · Statistics 2025-10-29 Takuya Kawahara , Sean McGrath , Jessica G Young

Measuring treatment efficacy in mixture of subgroups from a randomized clinical trial is a fundamental problem in personalized medicine development, in deciding whether to treat the entire patient population or to target a subgroup. We show…

Methodology · Statistics 2014-09-03 Ying Ding , Hui-Min Lin , Jason C. Hsu

This paper develops a nonparametric model that represents how sequences of outcomes and treatment choices influence one another in a dynamic manner. In this setting, we are interested in identifying the average outcome for individuals in…

Econometrics · Economics 2019-01-16 Sukjin Han

One size fits all approaches to medicine have become a thing of the past as the understanding of individual differences grows. The paper introduces a test for the presence of heterogeneity in treatment effects in a clinical trial.…

Randomized controlled trials are the gold standard for evaluating the efficacy of an intervention. However, there is often a trade-off between selecting the most scientifically relevant primary endpoint versus a less relevant, but more…

Methodology · Statistics 2026-01-12 Jack M. Wolf , Joseph S. Koopmeiners , David M. Vock

We consider continuous-time survival or more general event-history settings, where the aim is to infer the causal effect of a time-dependent treatment process. This is formalised as the effect on the outcome event of a (possibly…

Methodology · Statistics 2024-04-23 Kjetil Røysland , Pål Ryalen , Mari Nygård , Vanessa Didelez

In this work, we consider the problem of predicting the course of a progressive disease, such as cancer or Alzheimer's. Progressive diseases often start with mild symptoms that might precede a diagnosis, and each patient follows their own…

Machine Learning · Computer Science 2018-03-19 Yingying Zhu , Mert R. Sabuncu

This article studies the estimation of the causal effect of a time-varying treatment on time-to-an-event or on some other continuously distributed outcome. The paper applies to the situation where treatment is repeatedly adapted to…

Statistics Theory · Mathematics 2008-06-19 Judith J. Lok

Single-subject health data are becoming increasingly available thanks to advances in self-tracking technology (e.g., wearable devices, mobile apps, sensors, implants). Many users and health caregivers seek to use such observational time…

Applications · Statistics 2019-01-23 Eric Jay Daza

When the primary outcome is difficult to collect, surrogate endpoint is typically used as a substitute. It is possible that for every individual, treatment has a positive effect on surrogate, and surrogate has a positive effect on primary…

Methodology · Statistics 2017-12-27 Linquan Ma , Yunjian Yin , Lan Liu , Zhi Geng

Indirect experiments provide a valuable framework for estimating treatment effects in situations where conducting randomized control trials (RCTs) is impractical or unethical. Unlike RCTs, indirect experiments estimate treatment effects by…

Machine Learning · Computer Science 2023-12-06 Yash Chandak , Shiv Shankar , Vasilis Syrgkanis , Emma Brunskill
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