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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

Consider the problem of determining the effect of a compound on a specific cell type. To answer this question, researchers traditionally need to run an experiment applying the drug of interest to that cell type. This approach is not…

Methodology · Statistics 2023-06-13 Chandler Squires , Dennis Shen , Anish Agarwal , Devavrat Shah , Caroline Uhler

To make informed policy recommendations from observational data, we must be able to discern true treatment effects from random noise and effects due to confounding. Difference-in-Difference techniques which match treated units to control…

Methodology · Statistics 2019-09-12 Nicholas Illenberger , Dylan S. Small , Pamela A. Shaw

We argue that randomized controlled trials (RCTs) are special even among settings where average treatment effects are identified by a nonparametric unconfoundedness assumption. This claim follows from two results of Robins and Ritov (1997):…

Methodology · Statistics 2021-09-28 P. M. Aronow , James M. Robins , Theo Saarinen , Fredrik Sävje , Jasjeet Sekhon

Matching is a commonly used causal inference study design in observational studies. Through matching on measured confounders between different treatment groups, valid randomization inferences can be conducted under the no unmeasured…

Methodology · Statistics 2024-09-20 Jeffrey Zhang , Siyu Heng

We propose a novel resampling-based method to construct an asymptotically exact test for any subset of hypotheses on coefficients in high-dimensional linear regression. It can be embedded into any multiple testing procedure to make…

Methodology · Statistics 2022-05-26 Anna Vesely , Jelle J. Goeman , Livio Finos

In high-throughput screenings, it is common to estimate the effects of many treatments using a small number of independent trials of each. Because little is known about the distributional properties of the measurements from these trials, it…

Applications · Statistics 2025-03-17 Jackson Loper , Jeffrey Regier

In an observational study, it is common to leverage known null effect to detect bias. One such strategy is to set aside a placebo sample -- a subset of data immune from the hypothesized cause-and-effect relationship. Existence of an effect…

Methodology · Statistics 2022-07-05 Ting Ye , Shuxiao Chen , Bo Zhang

An artificial intelligence (AI) model can be viewed as a function that maps inputs to outputs in high-dimensional spaces. Once designed and well trained, the AI model is applied for inference. However, even optimized AI models can produce…

Artificial Intelligence · Computer Science 2026-02-27 Sha Hu

In this paper, we propose a novel method for causal inference within the framework of counterfactual and synthetic control. Matching forward the generalized synthetic control method, our instrumented principal component analysis method…

Econometrics · Economics 2024-09-17 Cong Wang

In randomized experiments with noncompliance, tests may focus on compliers rather than on the overall sample. Rubin (1998) put forth such a method, and argued that testing for the complier average causal effect and averaging permutation…

Methodology · Statistics 2016-02-23 Laura Forastiere , Fabrizia Mealli , Luke Miratrix

In healthcare, there is much interest in estimating policies, or mappings from covariates to treatment decisions. Recently, there is also interest in constraining these estimated policies to the standard of care, which generated the…

Methodology · Statistics 2026-02-17 Samuel J. Weisenthal , Sally W. Thurston , Ashkan Ertefaie

Study samples often differ from the target populations of inference and policy decisions in non-random ways. Researchers typically believe that such departures from random sampling -- due to changes in the population over time and space, or…

Methodology · Statistics 2023-07-20 Tamara Broderick , Ryan Giordano , Rachael Meager

Permutation tests are a powerful and flexible approach to inference via resampling. As computational methods become more ubiquitous in the statistics curriculum, use of permutation tests has become more tractable. At the heart of the…

Methodology · Statistics 2025-06-09 Johanna Hardin , Lauren Quesada , Julie Ye , Nicholas J. Horton

Recent observations, especially in cancer immunotherapy clinical trials with time-to-event outcomes, show that the commonly used proportial hazard assumption is often not justifiable, hampering an appropriate analyse of the data by hazard…

Methodology · Statistics 2021-02-23 Marc Ditzhaus , Menggang Yu , Jin Xu

Generalized linear models usually assume a common dispersion parameter, an assumption that is seldom true in practice. Consequently, standard parametric methods may suffer appreciable loss of type I error control. As an alternative, we…

Methodology · Statistics 2024-09-16 Riccardo De Santis , Jelle J. Goeman , Jesse Hemerik , Samuel Davenport , Livio Finos

Data integration methods aim to extract low-dimensional embeddings from high-dimensional outcomes to remove unwanted variations, such as batch effects and unmeasured covariates, across heterogeneous datasets. However, multiple hypothesis…

Methodology · Statistics 2025-12-15 Jin-Hong Du , Kathryn Roeder , Larry Wasserman

Statistical hypothesis testing and effect size measurement are routine parts of quantitative research. Advancements in computer processing power have greatly improved the capability of statistical inference through the availability of…

Methodology · Statistics 2024-01-18 Michael J. Crosse , John J. Foxe , Sophie Molholm

In observational causal inference, in order to emulate a randomized experiment, weights are used to render treatments independent of observed covariates. This property is known as balance; in its absence, estimated causal effects may be…

Methodology · Statistics 2020-07-16 David Arbour , Drew Dimmery , Arjun Sondhi

This paper presents a simple method for carrying out inference in a wide variety of possibly nonlinear IV models under weak assumptions. The method is non-asymptotic in the sense that it provides a finite sample bound on the difference…

Econometrics · Economics 2018-09-12 Joel L. Horowitz
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