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Meta-analysis is a well-established method for integrating results from several independent studies to estimate a common quantity of interest. However, meta-analysis is prone to selection bias, notably when particular studies are…

Applications · Statistics 2024-11-20 Layna Charlie Dennett , Antony Overstall , Dankmar Boehning

For the analysis of a time-to-event endpoint in a single-arm or randomized clinical trial it is generally perceived that interpretation of a given estimate of the survival function, or the comparison between two groups, hinges on some…

Methodology · Statistics 2023-04-17 Kaspar Rufibach , Lynda Grinsted , Jiang Li , Hans-Jochen Weber , Cheng Zheng , Jiangxiu Zhou

Some units from a population receive the same treatment that is different from treatments available for other reservoir populations. The minimal sufficient statistic $s$ for the pre-treatment $x$-covariates's distributions in the…

Statistics Theory · Mathematics 2014-08-05 Yannis G. Yatracos

In this paper we present a data-adaptive estimation procedure for estimation of average treatment effects in a time-to-event setting based on generalized random forests. In these kinds of settings, the definition of causal effect parameters…

Methodology · Statistics 2021-04-28 Helene C. W. Rytgaard , Claus T. Ekstrøm , Lars V. Kessing , Thomas A. Gerds

The assumption that no subject's exposure affects another subject's outcome, known as the no-interference assumption, has long held a foundational position in the study of causal inference. However, this assumption may be violated in many…

Methodology · Statistics 2018-11-27 Caleb H. Miles , Maya Petersen , Mark J. van der Laan

In the quest to make defensible causal claims from observational data, it is sometimes possible to leverage information from "placebo treatments" and "placebo outcomes". Existing approaches employing such information focus largely on point…

Methodology · Statistics 2024-11-26 Adam Rohde , Chad Hazlett

Time-to-event outcomes are often subject to left truncation and right censoring. While many survival analysis methods have been developed to handle truncation and censoring, majority of the past works require strong independence…

Methodology · Statistics 2025-07-28 Yuyao Wang , Andrew Ying , Ronghui Xu

Dropout is common in clinical studies, with up to half of patients leaving early due to side effects or other reasons. When dropout is informative (i.e., dependent on survival time), it introduces censoring bias, because of which treatment…

Machine Learning · Computer Science 2026-05-12 Yuxin Wang , Dennis Frauen , Jonas Schweisthal , Maresa Schröder , Stefan Feuerriegel

The estimand framework provides guidance on handling intercurrent events, such as treatment discontinuation, in the analysis of clinical trial responses. Under ICH E9(R1), the treatment policy (TP) strategy incorporates post-discontinuation…

Methodology · Statistics 2026-04-07 Myeongjong Kang , Sangyoon Yi

For many rare diseases with no approved preventive interventions, promising interventions exist, yet it has been difficult to conduct a pivotal phase 3 trial that could provide direct evidence demonstrating a beneficial effect on the target…

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

In Survival Analysis, the observed lifetimes often correspond to individuals for which the event occurs within a specific calendar time interval. With such interval sampling, the lifetimes are doubly truncated at values determined by the…

Methodology · Statistics 2021-03-29 Carla Moreira , Jacobo de Uña-Álvarez , Ana Cristina Santos , Henrique Barros

In settings where units' outcomes are affected by others' treatments, there has been a proliferation of ways to quantify effects of treatments on outcomes, including via indirect exposure to other units' treatments. Here we consider two…

Methodology · Statistics 2026-03-10 Sahil Loomba , Dean Eckles

Most of the widely used estimators of the average treatment effect (ATE) in causal inference rely on the assumptions of unconfoundedness and overlap. Unconfoundedness requires that the observed covariates account for all correlations…

Statistics Theory · Mathematics 2025-07-01 Yang Cai , Alkis Kalavasis , Katerina Mamali , Anay Mehrotra , Manolis Zampetakis

Meta-analyses of survival studies aim to reveal the variation of an effect measure of interest over different studies and present a meaningful summary. They must address between study heterogeneity in several dimensions and eliminate…

We develop a Bayesian nonparametric (BNP) approach to evaluate the causal effect of treatment in a randomized trial where a nonterminal event may be censored by a terminal event, but not vice versa (i.e., semi-competing risks). Based on the…

Methodology · Statistics 2019-07-23 Yanxun Xu , Daniel Scharfstein , Peter Müller , Michael Daniels

A parameter-dependent model involving nonlinear diffusion for an age-structured population is studied. The parameter measures the intensity of the mortality. A bifurcation approach is used to establish existence of positive equilibrium…

Analysis of PDEs · Mathematics 2010-02-10 Christoph Walker

Standard methods for estimating average causal effects require complete observations of the exposure and confounders. In observational studies, however, missing data are ubiquitous. Motivated by a study on the effect of prescription opioids…

Methodology · Statistics 2025-06-30 Lan Wen , Glen McGee

The estimand framework is increasingly established to pose research questions in confirmatory clinical trials. In evidence synthesis, the uptake of estimands has been modest, and the PICO (Population, Intervention, Comparator, Outcome)…

Who should we prioritize for treatment when causal effects cannot be estimated? In practice, organizations often rely on predictive proxies: ads are targeted using purchase probabilities, and retention incentives are allocated using…

Machine Learning · Statistics 2025-10-15 Carlos Fernández-Loría , Jorge Loría