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Related papers: Study designs for extending causal inferences from…

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Applied researchers in biomedicine and related fields are often interested in estimating the causal effect of a treatment or intervention. Although randomized clinical trials are considered the gold standard for establishing causal effects,…

While probabilistic models describe the dependence structure between observed variables, causal models go one step further: they predict, for example, how cognitive functions are affected by external interventions that perturb neuronal…

Neurons and Cognition · Quantitative Biology 2021-04-12 Sebastian Weichwald , Jonas Peters

The causal effect of an intervention (treatment/exposure) on an outcome can be estimated by: i) specifying knowledge about the data-generating process; ii) assessing under what assumptions a target quantity, such as for example a causal…

Methodology · Statistics 2021-03-05 Michael Schomaker

Randomized saturation designs are a family of designs which assign a possibly different treatment proportion to each cluster of a population at random. As a result, they generalize the well-known (stratified) completely randomized designs…

Methodology · Statistics 2022-03-21 Chencheng Cai , Jean Pouget-Abadie , Edoardo M. Airoldi

One way to investigate the precision of estimates likely to result from planned experiments and planned epidemiological studies is to simulate a large number of possible outcomes and analyse the sets of possible results. This appears to be…

Computation · Statistics 2013-06-28 G. K. Robinson , L. M. Ryan

Randomized clinical trials are the gold standard for analyzing treatment effects, but high costs and ethical concerns can limit recruitment, potentially leading to invalid inferences. Incorporating external trial data with similar…

Methodology · Statistics 2024-09-09 Yujia Gu , Hanzhong Liu , Wei Ma

We extend Fisher's randomization test (FRT) to test conditional independence between observed outcomes and treatments given covariates in both randomized experiments and observational studies, with no restriction on the variable type of…

Methodology · Statistics 2025-06-12 Zhen Zhong

External controls from historical trials or observational data can augment randomized controlled trials when large-scale randomization is impractical or unethical, such as in drug evaluation for rare diseases. However, non-randomized…

Methodology · Statistics 2025-05-08 Ke Zhu , Shu Yang , Xiaofei Wang

Estimating the effects of interventions in networks is complicated when the units are interacting, such that the outcomes for one unit may depend on the treatment assignment and behavior of many or all other units (i.e., there is…

Methodology · Statistics 2014-08-15 Dean Eckles , Brian Karrer , Johan Ugander

Comparison and contrast are the basic means to unveil causation and learn which treatments work. To build good comparison groups, randomized experimentation is key, yet often infeasible. In such non-experimental settings, we illustrate and…

Methodology · Statistics 2024-01-30 Ambarish Chattopadhyay , Jose R. Zubizarreta

We study the problem of causal discovery through targeted interventions. Starting from few observational measurements, we follow a Bayesian active learning approach to perform those experiments which, in expectation with respect to the…

Machine Learning · Statistics 2019-10-10 Julius von Kügelgen , Paul K Rubenstein , Bernhard Schölkopf , Adrian Weller

In this paper, we discuss structure learning of causal networks from multiple data sets obtained by external intervention experiments where we do not know what variables are manipulated. For example, the conditions in these experiments are…

Machine Learning · Statistics 2016-10-28 Yango He , Zhi Geng

Causal inference from observational data provides strong evidence for the best action in decision-making without performing expensive randomized trials. The effect of an action is usually not identifiable under unobserved confounding, even…

Machine Learning · Computer Science 2026-02-02 Md Musfiqur Rahman , Ziwei Jiang , Hilaf Hasson , Murat Kocaoglu

Factorial designs are widely used due to their ability to accommodate multiple factors simultaneously. The factor-based regression with main effects and some interactions is the dominant strategy for downstream data analysis, delivering…

Methodology · Statistics 2021-12-09 Anqi Zhao , Peng Ding

This paper studies the evaluation of methods for targeting the allocation of limited resources to a high-risk subpopulation. We consider a randomized controlled trial to measure the difference in efficiency between two targeting methods and…

Applications · Statistics 2018-04-04 Eric Potash

With the ubiquitous availability of unstructured data, growing attention is paid as how to adjust for selection bias in such non-probability samples. The majority of the robust estimators proposed by prior literature are either fully or…

Methodology · Statistics 2022-04-08 Ali Rafei , Michael R. Elliott , Carol A. C. Flannagan

Composite endpoints are increasingly used in clinical trials to capture treatment effects across multiple or hierarchically ordered outcomes. Although inference procedures based on win statistics, such as the win ratio, win odds, and net…

Methodology · Statistics 2025-10-28 Xi Fang , Zhiqiang Cao , Fan Li

Rare variants are hypothesized to be largely responsible for heritability and susceptibility to disease in humans. So rare variants association studies hold promise for understanding disease. Conversely though, the rareness of the variants…

Methodology · Statistics 2021-12-06 Lorenzo Masoero , Joshua Schraiber , Tamara Broderick

In medical research, a scenario often entertained is randomized controlled $2^2$ factorial design with a binary outcome. By utilizing the concept of potential outcomes, Dasgupta et al. (2015) proposed a randomization-based causal inference…

Methodology · Statistics 2017-11-15 Jiannan Lu

As clinical decision-making increasingly moves toward individualized and context-specific treatment recommendations, reliance on any single evidence source, randomized or observational, may be insufficient. Principled integration of…