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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.…

统计方法学 · 统计学 2025-03-11 Chenyin Gao , Xiang Zhang , Shu Yang

This chapter covers different approaches to policy evaluation for assessing the causal effect of a treatment or intervention on an outcome of interest. As an introduction to causal inference, the discussion starts with the experimental…

计量经济学 · 经济学 2019-10-03 Martin Huber

Important questions for impact evaluation require knowledge not only of average effects, but of the distribution of treatment effects. The inability to observe individual counterfactuals makes answering these empirical questions…

计量经济学 · 经济学 2026-05-25 Bruno Fava

The primary benefit of identifying a valid surrogate marker is the ability to use it in a future trial to test for a treatment effect with shorter follow-up time or less cost. However, previous work has demonstrated potential heterogeneity…

统计方法学 · 统计学 2022-09-20 Layla Parast , Tianxi Cai , Lu Tian

The LATE monotonicity assumption of Imbens and Angrist (1994) precludes "defiers," individuals whose treatment always runs counter to the instrument, in the terminology of Balke and Pearl (1993) and Angrist et al. (1996). I allow for…

统计方法学 · 统计学 2020-07-24 Amanda Kowalski

Alcohol misuse is a key target of public health strategies aimed at reducing cardiovascular risk. The effect of excessive alcohol consumption on blood pressure may vary systematically with individuals' unobserved propensity to engage in…

统计方法学 · 统计学 2026-03-11 Ashish Patel , Francis J DiTraglia , Stephen Burgess

We present a novel data-oriented statistical framework that assesses the presumed Gaussian dependence structure in a pairwise setting. This refers to both multivariate normality and normal copula goodness-of-fit testing. The proposed test…

统计方法学 · 统计学 2024-04-23 Jakub Woźny , Piotr Jaworski , Damian Jelito , Marcin Pitera , Agnieszka Wyłomańska

Evaluating the causal effect of an intervention on multivariate outcomes is challenging when the outcomes are interdependent and derived rather than directly observed. Effective connectivity, which summarizes the directional neural…

统计方法学 · 统计学 2026-04-02 Haiyue Song , Ani Eloyan , Youjin Lee

Recently, a group of experiments tested local realism with random choices prepared by humans. These various tests were subject to additional assumptions, which lead to loopholes in the interpretations of almost all of the experiments. Among…

量子物理 · 物理学 2020-09-08 Stanisław Sołtan , Dawid Dopierała , Adam Bednorz

Equivalence testing compares the hypothesis that an effect $\mu$ is large against the alternative that it is negligible. Here, `large' is classically expressed as being larger than some `equivalence margin' $\Delta$. A longstanding problem…

统计理论 · 数学 2026-03-18 Stan Koobs , Nick W. Koning

Causal mediation analysis aims at disentangling a treatment effect into an indirect mechanism operating through an intermediate outcome or mediator, as well as the direct effect of the treatment on the outcome of interest. However, the…

计量经济学 · 经济学 2020-05-05 Martin Huber , Lukáš Lafférs

Causal inference studies whether the presence of a variable influences an observed outcome. As measured by quantities such as the "average treatment effect," this paradigm is employed across numerous biological fields, from vaccine and drug…

In modeling multivariate time series for either forecast or policy analysis, it would be beneficial to have figured out the cause-effect relations within the data. Regression analysis, however, is generally for correlation relation, and…

机器学习 · 统计学 2021-11-23 Xingwei Hu

There has long been an apparent consensus in the literature on intra-household allocation and fertility that greater paternal involvement in childcare relaxes maternal time constraints, enabling mothers to increase their labor supply or…

综合经济学 · 经济学 2026-05-14 Jackie Dajin Young , Marwil J. Davila-Fernandez

Causal inference is a critical research area with multi-disciplinary origins and applications, ranging from statistics, computer science, economics, psychology to public health. In many scientific research, randomized experiments provide a…

统计方法学 · 统计学 2022-07-26 Jingying Zeng

Experiments deliver credible treatment-effect estimates but, because they are costly, are often restricted to specific sites, small populations, or particular mechanisms. A common practice across several fields is therefore to combine…

计量经济学 · 经济学 2025-12-30 Aristotelis Epanomeritakis , Davide Viviano

Comparative effectiveness evidence from randomized trials may not be directly generalizable to a target population of substantive interest when, as in most cases, trial participants are not randomly sampled from the target population.…

统计方法学 · 统计学 2022-04-12 Fan Li , Ashley L. Buchanan , Stephen R. Cole

Surrogate markers are often employed in clinical trials to replace primary outcomes that may be difficult, expensive, or time-consuming to measure directly. These markers can accelerate the evaluation of new treatments, provided they…

统计方法学 · 统计学 2026-03-17 Pietro Carlotti , Layla Parast

Utilizing randomized experiments to evaluate the effect of short-term treatments on the short-term outcomes has been well understood and become the golden standard in industrial practice. However, as service systems become increasingly…

统计方法学 · 统计学 2025-09-10 Shuze Chen , David Simchi-Levi , Chonghuan Wang

Classical randomized experiments, equipped with randomization-based inference, provide assumption-free inference for treatment effects. They have been the gold standard for drawing causal inference and provide excellent internal validity.…

统计方法学 · 统计学 2021-09-22 Zihao Yang , Tianyi Qu , Xinran Li