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Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…

Methodology · Statistics 2025-08-05 Di Ran , Fanni Zhang , Sima Shahsavari , Kristine Broglio , Alasdair Henderson , Binbing Yu

In this paper, we address the issue of estimating and inferring distributional treatment effects in randomized experiments. The distributional treatment effect provides a more comprehensive understanding of treatment heterogeneity compared…

Econometrics · Economics 2025-01-15 Tatsushi Oka , Shota Yasui , Yuta Hayakawa , Undral Byambadalai

Randomized controlled trials (RCTs) are the gold standard for assessing drug safety and efficacy. However, RCTs have some drawbacks which have led to the use of single-arm studies to make certain internal drug development and regulatory…

Causal discovery traditionally relies on statistical methods applied to observational data, often requiring large datasets and assumptions about underlying causal structures. Recent advancements in Large Language Models (LLMs) have…

Machine Learning · Computer Science 2025-04-16 Yuni Susanti , Michael Färber

Functional data is a powerful tool for capturing and analyzing complex patterns and relationships in a variety of fields, allowing for more precise modeling, visualization, and decision-making. For example, in healthcare, functional data…

Methodology · Statistics 2023-04-26 Xiyuan Gao , Jiayi Wang , Guanyu Hu , Jianguo Sun

The rapid finding of effective therapeutics requires the efficient use of available resources in clinical trials. The use of covariate adjustment can yield statistical estimates with improved precision, resulting in a reduction in the…

Methodology · Statistics 2021-09-10 Nicholas Williams , Michael Rosenblum , Iván Díaz

Observational studies can play a useful role in assessing the comparative effectiveness of competing treatments. In a clinical trial the randomization of participants to treatment and control groups generally results in well-balanced groups…

Randomized control trials (RCTs) are the gold standard for estimating causal effects, but often use samples that are non-representative of the actual population of interest. We propose a reweighting method for estimating population average…

Methodology · Statistics 2022-02-09 Kellie Ottoboni , Jason Poulos

There is growing interest in exploring causal effects in target populations via data combination. However, most approaches are tailored to specific settings and lack comprehensive comparative analyses. In this article, we focus on a typical…

Methodology · Statistics 2024-09-17 Peng Wu , Shanshan Luo , Zhi Geng

Every design choice will have different effects on different units. However traditional A/B tests are often underpowered to identify these heterogeneous effects. This is especially true when the set of unit-level attributes is…

Artificial Intelligence · Computer Science 2016-11-09 Alexander Peysakhovich , Akos Lada

In prior work we have introduced an asymptotic threshold of sufficient randomness for causal inference from observational data. In this paper we extend that prior work in three main ways. First, we show how to empirically estimate a lower…

Methodology · Statistics 2023-09-07 Brian Knaeble , Braxton Osting , Placede Tshiaba

In randomized clinical trials (RCTs), the accurate estimation of marginal treatment effects is crucial for determining the efficacy of interventions. Enhancing the statistical power of these analyses is a key objective for statisticians.…

Methodology · Statistics 2026-01-30 Emilie Højbjerre-Frandsen , Mark J. van der Laan , Alejandro Schuler

Unobserved confounding is one of the main challenges when estimating causal effects. We propose a causal reduction method that, given a causal model, replaces an arbitrary number of possibly high-dimensional latent confounders with a single…

Machine Learning · Statistics 2023-02-24 Maximilian Ilse , Patrick Forré , Max Welling , Joris M. Mooij

Causal effect estimation from observational data is a challenging problem, especially with high dimensional data and in the presence of unobserved variables. The available data-driven methods for tackling the problem either provide an…

Methodology · Statistics 2022-07-25 Debo Cheng , Jiuyong Li , Lin Liu , Jiji Zhang , Jixue Liu , Thuc Duy Le

The purpose of this paper is to investigate and develop methods for analysis of multi-center randomized clinical trials which only rely on the randomization process as a basis of inference. Our motivation is prompted by the fact that most…

Applications · Statistics 2008-07-28 Lu Zheng , Marvin Zelen

Data augmentation has been widely applied as an effective methodology to improve generalization in particular when training deep neural networks. Recently, researchers proposed a few intensive data augmentation techniques, which indeed…

Machine Learning · Computer Science 2019-11-22 Zhuoxun He , Lingxi Xie , Xin Chen , Ya Zhang , Yanfeng Wang , Qi Tian

In this paper, we develop a multiply robust inference procedure of the average treatment effect (ATE) for data with high-dimensional covariates. We consider the case where it is difficult to correctly specify a single parametric model for…

Methodology · Statistics 2025-09-03 Xintao Xia , Yumou Qiu

Hybrid studies allow investigators to simultaneously study an intervention effectiveness outcome and an implementation research outcome. In particular, type 2 hybrid studies support research that places equal importance on both outcomes…

We study the problem of learning personalized decision policies from observational data while accounting for possible unobserved confounding. Previous approaches, which assume unconfoundedness, i.e., that no unobserved confounders affect…

Machine Learning · Computer Science 2019-11-05 Nathan Kallus , Angela Zhou

Empirical likelihood is a popular nonparametric or semi-parametric statistical method with many nice statistical properties. Yet when the sample size is small, or the dimension of the accompanying estimating function is high, the…

Statistics Theory · Mathematics 2010-10-05 Yukun Liu , Jiahua Chen