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Causal inference across multiple data sources offers a promising avenue to enhance the generalizability and replicability of scientific findings. However, data integration methods for time-to-event outcomes, common in biomedical research,…

Methodology · Statistics 2025-05-16 Yi Liu , Alexander W. Levis , Ke Zhu , Shu Yang , Peter B. Gilbert , Larry Han

Externally controlled trials are crucial in clinical development when randomized controlled trials are unethical or impractical. These trials consist of a full treatment arm with the experimental treatment and a full external control arm.…

Methodology · Statistics 2025-03-11 Chenyin Gao , Xiang Zhang , Shu Yang

The analysis of causal effects when the outcome of interest is possibly truncated by death has a long history in statistics and causal inference. The survivor average causal effect is commonly identified with more assumptions than those…

Methodology · Statistics 2020-03-24 Jaffer M. Zaidi , Eric J. Tchetgen Tchetgen , Tyler J. VanderWeele

Likelihood methods for measuring statistical evidence obey the likelihood principle while maintaining bounded and well-controlled frequency properties. These methods lend themselves to sequential study designs because they measure the…

Methodology · Statistics 2017-11-07 Jeffrey D Blume , Leena Choi

In cluster randomized trials, patients are typically recruited after clusters are randomized, and the recruiters and patients may not be blinded to the assignment. This often leads to differential recruitment and consequently systematic…

Methodology · Statistics 2021-10-05 Fan Li , Zizhong Tian , Jennifer Bobb , Georgia Papadogeorgou , Fan Li

Recently developed survival analysis methods improve upon existing approaches by predicting the probability of event occurrence in each of a number pre-specified (discrete) time intervals. By avoiding placing strong parametric assumptions…

Machine Learning · Statistics 2023-10-25 Jimmy Hickey , Ricardo Henao , Daniel Wojdyla , Michael Pencina , Matthew M. Engelhard

This work develops a flexible inferential framework for nonparametric causal inference in time-to-event settings, based on stochastic interventions defined through multiplicative scaling of the intensity governing an intermediate event…

Methodology · Statistics 2026-02-04 Helene Charlotte Wiese Rytgaard , Mark van der Laan

For obtaining causal inferences that are objective, and therefore have the best chance of revealing scientific truths, carefully designed and executed randomized experiments are generally considered to be the gold standard. Observational…

Applications · Statistics 2008-11-12 Donald B. Rubin

The onset of several silent, chronic diseases such as diabetes can be detected only through diagnostic tests. Due to cost considerations, self-reported outcomes are routinely collected in lieu of expensive diagnostic tests in large-scale…

Applications · Statistics 2015-09-15 Xiangdong Gu , Yunsheng Ma , Raji Balasubramanian

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)…

Often in follow-up studies intermediate events occur in some patients, such as reinterventions or adverse events. These intermediate events directly affect the shapes of their longitudinal profiles. Our work is motivated by two studies in…

Clinical trials often allow patients in the control arm to switch to the treatment arm if their physical conditions are worse than certain tolerance levels. For instance, treatment switching arises in the Concorde clinical trial, which aims…

Applications · Statistics 2023-09-06 Alessandra Mattei , Peng Ding , Veronica Ballerini , Fabrizia Mealli

In this paper, we propose a robust method to estimate the average treatment effects in observational studies when the number of potential confounders is possibly much greater than the sample size. We first use a class of penalized…

Methodology · Statistics 2018-12-21 Yang Ning , Sida Peng , Kosuke Imai

When data are right-censored, i.e. some outcomes are missing due to a limited period of observation, survival analysis can compute the "time to event". Multiple classes of outcomes lead to a classification variant: predicting the most…

Artificial Intelligence · Computer Science 2024-06-21 Julie Alberge , Vincent Maladière , Olivier Grisel , Judith Abécassis , Gaël Varoquaux

Survival Analysis (SA) constitutes the default method for time-to-event modeling due to its ability to estimate event probabilities of sparsely occurring events over time. In this work, we show how to improve the training and inference of…

Machine Learning · Computer Science 2023-12-12 Chris Solomou

Data from both a randomized trial and an observational study are sometimes simultaneously available for evaluating the effect of an intervention. The randomized data typically allows for reliable estimation of average treatment effects but…

Methodology · Statistics 2021-12-01 David Cheng , Tianxi Cai

In many medical studies, an ultimate failure event such as death is likely to be affected by the occurrence and timing of other intermediate clinical events. Both event times are subject to censoring by loss-to-follow-up but the nonterminal…

Methodology · Statistics 2021-01-18 Fei Gao , Fan Xia , Kwun Chuen Gary Chan

Researchers are frequently interested in understanding the causal effect of treatment interventions. However, in some cases, the treatment of interest--readily available in a randomized controlled trial (RCT)--is either not directly…

Methodology · Statistics 2025-09-29 Lan Wen , Aaron L Sarvet

In many causal studies, outcomes are censored by death, in the sense that they are neither observed nor defined for units who die. In such studies, the focus is usually on the stratum of always survivors up to a single fixed time s.…

Methodology · Statistics 2024-01-02 Giulio Grossi , Marco Mariani , Alessandra Mattei , Fabrizia Mealli

In many clinical contexts, estimating effects of treatment in time-to-event data is complicated not only by confounding, censoring, and heterogeneity, but also by the presence of a cured subpopulation in which the event of interest never…

Methodology · Statistics 2026-02-06 Yuqi Li , Quinn Lanners , Matthew M. Engelhard
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