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The estimands framework outlined in ICH E9 (R1) describes the components needed to precisely define the effects to be estimated in clinical trials, which includes how post-baseline "intercurrent" events (IEs) are to be handled. In…

Methodology · Statistics 2023-08-28 Thomas Drury , Juan J Abellan , Nicky Best , Ian R. White

Meta-analysis is a statistical method used in evidence synthesis for combining, analyzing and summarizing studies that have the same target endpoint and aims to derive a pooled quantitative estimate using fixed and random effects models or…

Methodology · Statistics 2022-04-25 Ivette Raices Cruz , Matthias C. M. Troffaes , Johan Lindström , Ullrika Sahlin

In randomized experiments, interactions between units might generate a treatment diffusion process. This is common when the treatment of interest is an actual object or product that can be shared among peers (e.g., flyers, booklets,…

Methodology · Statistics 2021-09-17 Costanza Tortú , Irene Crimaldi , Fabrizia Mealli , Laura Forastiere

Causal inference for observational longitudinal studies often requires the accurate estimation of treatment effects on time-to-event outcomes in the presence of time-dependent patient history and time-dependent covariates. To tackle this…

Machine Learning · Statistics 2022-06-17 Jie Zhu , Blanca Gallego

The primary analysis in two-arm clinical trials usually involves inference on a scalar treatment effect parameter; e.g., depending on the outcome, the difference of treatment-specific means, risk difference, risk ratio, or odds ratio. Most…

Methodology · Statistics 2022-04-25 Anastasios A. Tsiatis , Marie Davidian

When drawing causal inferences about the effects of multiple treatments on clustered survival outcomes using observational data, we need to address implications of the multilevel data structure, multiple treatments, censoring and unmeasured…

Methodology · Statistics 2022-02-18 Liangyuan Hu , Jiayi Ji , Ronald D. Ennis , Joseph W. Hogan

In many scenarios, the observational data needed for causal inferences are spread over two data files. In particular, we consider scenarios where one file includes covariates and the treatment measured on one set of individuals, and a…

Methodology · Statistics 2020-09-22 Sharmistha Guha , Jerome P. Reiter , Andrea Mercatanti

Predicting the effect of interventions with many possible variations, e.g., therapeutic content that affects mental health outcomes or an earnings call transcript that drives movement in share price, is useful across several domains.…

Machine Learning · Computer Science 2026-05-27 Nikita Dhawan , Arnav Paruthi , Andrew Kim , Lovedeep Gondara , Jekaterina Novikova , Chris J. Maddison

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

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

Biases in observational data of treatments pose a major challenge to estimating expected treatment outcomes in different populations. An important technique that accounts for these biases is reweighting samples to minimize the discrepancy…

Machine Learning · Computer Science 2020-09-14 Michal Ozery-Flato , Pierre Thodoroff , Matan Ninio , Michal Rosen-Zvi , Tal El-Hay

Survival analysis, or time-to-event modelling, is a classical statistical problem that has garnered a lot of interest for its practical use in epidemiology, demographics or actuarial sciences. Recent advances on the subject from the point…

Machine Learning · Computer Science 2021-07-28 Guillaume Ausset , Tom Ciffreo , Francois Portier , Stephan Clémençon , Timothée Papin

This work bridges the gap between staggered adoption designs and survival analysis to estimate causal effects in settings with time-varying treatments, addressing a fundamental challenge in medical research exemplified by the Stanford Heart…

Methodology · Statistics 2025-03-04 Xiang Meng , Iavor Bojinov

Stochastic processes often exhibit sudden systematic changes in pattern a short time before certain failure events. Examples include increase in medical costs before death and decrease in CD4 counts before AIDS diagnosis. To study such…

Applications · Statistics 2010-11-17 Kwun Chuen Gary Chan , Mei-Cheng Wang

Digital monitoring studies collect real-time high frequency data via mobile sensors in the subjects' natural environment. This data can be used to model the impact of changes in physiology on recurrent event outcomes such as smoking, drug…

Methodology · Statistics 2022-04-15 Walter Dempsey

In this paper, we introduce new parametric and semiparametric regression techniques for a recurrent event process subject to random right censoring. We develop models for the cumula- tive mean function and provide asymptotically normal…

Statistics Theory · Mathematics 2015-03-17 Olivier Bouaziz , Ségolen Geffray , Olivier Lopez

Background Study individuals may face repeated events overtime. However, there is no consensus around learning approaches to use in a high-dimensional framework for survival data (when the number of variables exceeds the number of…

Methodology · Statistics 2022-03-30 Juliette Murris , Anais Charles-Nelson , Audrey Lavenu , Sandrine Katsahian

Conventional survival analysis approaches estimate risk scores or individualized time-to-event distributions conditioned on covariates. In practice, there is often great population-level phenotypic heterogeneity, resulting from (unknown)…

Machine Learning · Statistics 2020-03-03 Paidamoyo Chapfuwa , Chunyuan Li , Nikhil Mehta , Lawrence Carin , Ricardo Henao

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

Modern health data science applications leverage abundant molecular and electronic health data, providing opportunities for machine learning to build statistical models to support clinical practice. Time-to-event analysis, also called…