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Technological advancements in the field of mobile devices and wearable sensors have helped overcome obstacles in the delivery of care, making it possible to deliver behavioral treatments anytime and anywhere. Increasingly the delivery of…

Applications · Statistics 2017-11-13 Walter Dempsey , Peng Liao , Santosh Kumar , Susan A. Murphy

The progression-free survival ratio (PFSr) is a widely used measure in personalized oncology trials. It evaluates the effectiveness of treatment by comparing two consecutive event times - one under standard therapy and one under an…

Methodology · Statistics 2025-12-23 Merle Munko , Simon Mack , Marc Ditzhaus , Stefan Fröhling , Dennis Dobler , Dominic Edelmann

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

In observational studies of survival time featuring a binary time-dependent treatment, the hazard ratio (an instantaneous measure) is often used to represent the treatment effect. However, investigators are often more interested in the…

Methodology · Statistics 2020-05-21 Yun Li , Douglas E. Schaubel , Kevin He

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

We propose a novel personalized concept for the optimal treatment selection for a situation where the response is a multivariate vector, that could contain right-censored variables such as survival time. The proposed method can be applied…

Methodology · Statistics 2022-10-03 Chathura Siriwardhana , K. B. Kulasekera , Somnath Datta

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

We are interested in the estimation of average treatment effects based on right-censored data of an observational study. We focus on causal inference of differences between t-year absolute event risks in a situation with competing risks. We…

Factorial analyses offer a powerful nonparametric means to detect main or interaction effects among multiple treatments. For survival outcomes, e.g. from clinical trials, such techniques can be adopted for comparing reasonable…

Methodology · Statistics 2023-02-06 Takeshi Emura , Marc Ditzhaus , Dennis Dobler , Kenta Murotani

Studies involving both randomized experiments as well as observational data typically involve time-to-event outcomes such as time-to-failure, death or onset of an adverse condition. Such outcomes are typically subject to censoring due to…

Methodology · Statistics 2023-02-27 Chirag Nagpal , Vedant Sanil , Artur Dubrawski

Simulation studies are useful for evaluating and developing statistical methods for the analyses of complex problems. Performance of methods may be affected by multiple complexities present in real scenarios. Generating sufficiently…

Leveraging external controls -- relevant individual patient data under control from external trials or real-world data -- has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial…

Methodology · Statistics 2022-07-13 Yanyao Yi , Ying Zhang , Yu Du , Ting Ye

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the U.S. Food and Drug Administration recently issued guidance that emphasizes the importance of…

Methodology · Statistics 2023-06-12 Kelly Van Lancker , Frank Bretz , Oliver Dukes

Balanced representation learning methods have been applied successfully to counterfactual inference from observational data. However, approaches that account for survival outcomes are relatively limited. Survival data are frequently…

Machine Learning · Statistics 2021-03-04 Paidamoyo Chapfuwa , Serge Assaad , Shuxi Zeng , Michael J. Pencina , Lawrence Carin , Ricardo Henao

This article considers the joint modeling of longitudinal covariates and partly-interval censored time-to-event data. Longitudinal time-varying covariates play a crucial role in obtaining accurate clinically relevant predictions using a…

Methodology · Statistics 2024-12-05 Annabel Webb , Nan Zou , Serigne Lo , Jun Ma

We develop randomization-based tests for heterogeneous treatment effects in the presence of network interference. Leveraging the exposure mapping framework, we study a broad class of null hypotheses that represent various forms of constant…

Econometrics · Economics 2025-06-25 Julius Owusu

Treatment effect estimation is a fundamental problem in causal inference. We focus on designing efficient randomized controlled trials, to accurately estimate the effect of some treatment on a population of $n$ individuals. In particular,…

Machine Learning · Computer Science 2022-10-14 Raghavendra Addanki , David Arbour , Tung Mai , Cameron Musco , Anup Rao

In this paper we review an approach to estimating the causal effect of a time-varying treatment on time to some event of interest. This approach is designed for the situation where the treatment may have been repeatedly adapted to patient…

Statistics Theory · Mathematics 2007-06-13 J. J. Lok , R. D. Gill , A. W. van der Vaart , J. M. Robins

We consider a randomized controlled trial between two groups. The objective is to identify a population with characteristics such that the test therapy is more effective than the control therapy. Such a population is called a subgroup. This…

Methodology · Statistics 2021-12-06 Shintaro Yuki , Kensuke Tanioka , Hiroshi Yadohisa

Two-sample tests with censored outcomes are a classical topic in statistics with wide use even in cutting edge applications. There are at least two modes of inference used to justify two-sample tests. One is usual superpopulation inference…

Statistics Theory · Mathematics 2022-10-06 Xinran Li , Dylan S. Small