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Efficient transport and generalization of survival treatment effects

生存治疗效应的有效迁移与推广

Randomized controlled trials provide internally valid estimates of treatment effects, but their results may not directly apply to broader target populations due to differences in baseline covariate distributions, adherence to treatment or variations in outcome mechanisms. Under standard transport and time-to-event identifiability assumptions, we develop nonparametric, debiased machine learning estimators for transporting and generalizing causal survival treatment effect…

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Causal Survival Analysis in Platform Trials with Non-Concurrent Controls

使用非同期对照的平台试验因果生存分析

Platform trials allow treatment arms to enter and exit over time while maintaining a shared control arm, yielding concurrent and non-concurrent controls (NCC). Pooling NCC is often motivated as a strategy to improve statistical efficiency, but it is unclear which estimand is targeted, what assumptions justify identification and estimation, and when precision gains are achievable; these questions are further complicated by time-to-event/survival data. Motivated by the…

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Fast Uncertainty Quantification for Kernel-Based Estimators in Large-Scale Causal Inference

大规模因果推断中核估计量的快速不确定性量化

Kernel methods are widely used in causal inference for tasks such as treatment effect estimation, policy evaluation, and policy learning. The bootstrap is a standard tool for uncertainty quantification because of its broad applicability. As increasingly large datasets become available, such as the 2023 U.S. Natality data from the National Vital Statistics System (NVSS), which includes 3,596,017 registered births, the computational demands of these methods increase…

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Modern Causal Inference Approaches to Improve Power for Subgroup Analysis in Randomized Controlled Trials

利用现代因果推断提高随机对照试验亚组分析的检验力

Randomized controlled trials (RCTs) often include subgroup analyses to assess whether treatment effects vary across pre-specified patient populations. However, these analyses frequently suffer from small sample sizes which limit the power to detect heterogeneous effects. Power can be improved by leveraging predictors of the outcome -- i.e., through covariate adjustment -- as well as by borrowing external data from similar RCTs or observational studies. The benefits of…

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Clarifying identification and estimation of treatment effects in the Sequential Parallel Comparison Design

厘清序贯平行比较设计中治疗效应的识别与估计

Sequential parallel comparison design (SPCD) clinical trials aim to adjust active treatment effect estimates for placebo response to minimize the impact of placebo responders on the estimates. This is potentially accomplished using a two stage design by measuring treatment effects among all participants during the first stage, then classifying some placebo arm participants as placebo non-responders who will be re-randomized in the second stage. In this paper, we use causal…

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Identification and estimation of causal effects using non-concurrent controls in platform trials

平台试验中利用非同期对照识别与估计因果效应

Platform trials are multi-arm designs that simultaneously evaluate multiple treatments for a single disease within the same overall trial structure. Unlike traditional randomized controlled trials, they allow treatment arms to enter and exit the trial at distinct times while maintaining a control arm throughout. This control arm comprises both concurrent controls, where participants are randomized concurrently to either the treatment or control arm, and non-concurrent…

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Non-parametric efficient estimation of marginal structural models with multi-valued time-varying treatments

多值时变处理下边际结构模型的非参数有效估计

Marginal structural models are a popular method for estimating causal effects in the presence of time-varying exposures. In spite of their popularity, no scalable non-parametric estimator exist for marginal structural models with multi-valued and time-varying treatments. In this paper, we use machine learning together with recent developments in semiparametric efficiency theory for longitudinal studies to propose such an estimator. The proposed estimator is based on a study…

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A Stable and Efficient Covariate-Balancing Estimator for Causal Survival Effects

用于生存因果效应的稳定高效协变量平衡估计量

We propose an empirically stable and asymptotically efficient covariate-balancing approach to the problem of estimating survival causal effects in data with conditionally-independent censoring. This addresses a challenge often encountered in state-of-the-art nonparametric methods: the use of inverses of small estimated probabilities and the resulting amplification of estimation error. We validate our theoretical results in experiments on synthetic and semi-synthetic data.

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A Fast Bootstrap Algorithm for Causal Inference with Large Data

大规模数据因果推断的快速自助法算法

Estimating causal effects from large experimental and observational data has become increasingly prevalent in both industry and research. The bootstrap is an intuitive and powerful technique used to construct standard errors and confidence intervals of estimators. Its application however can be prohibitively demanding in settings involving large data. In addition, modern causal inference estimators based on machine learning and optimization techniques exacerbate the…

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