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Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform…

机器学习 · 统计学 2018-10-22 Onur Atan , William R. Zame , Mihaela van der Schaar

Randomized clinical trials (RCTs) are ideal for estimating causal effects, because the distributions of background covariates are similar in expectation across treatment groups. When estimating causal effects using observational data,…

统计方法学 · 统计学 2019-02-27 Anthony D. Scotina , Roee Gutman

We predict the average effect of Medicaid expansion on the non-elderly adult uninsurance rate among states that did not expand Medicaid in 2014 as if they had expanded their Medicaid eligibility requirements. Using American Community Survey…

应用统计 · 统计学 2022-05-24 Max Rubinstein , Amelia Haviland , David Choi

Randomized Controlled Trials (RCTs) may suffer from limited scope. In particular, samples may be unrepresentative: some RCTs over- or under- sample individuals with certain characteristics compared to the target population, for which one…

统计方法学 · 统计学 2024-03-15 Bénédicte Colnet , Julie Josse , Gaël Varoquaux , Erwan Scornet

Confounding is a significant obstacle to unbiased estimation of causal effects from observational data. For settings with high-dimensional covariates -- such as text data, genomics, or the behavioral social sciences -- researchers have…

人工智能 · 计算机科学 2024-02-01 Katherine A. Keith , Sergey Feldman , David Jurgens , Jonathan Bragg , Rohit Bhattacharya

Population-adjusted indirect comparisons estimate treatment effects when access to individual patient data is limited and there are cross-trial differences in effect modifiers. Popular methods include matching-adjusted indirect comparison…

应用统计 · 统计学 2021-11-05 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

We consider the task of evaluating policies of algorithmic resource allocation through randomized controlled trials (RCTs). Such policies are tasked with optimizing the utilization of limited intervention resources, with the goal of…

人工智能 · 计算机科学 2023-02-07 Aditya Mate , Bryan Wilder , Aparna Taneja , Milind Tambe

We derive new variance formulas for inference on a general class of estimands of causal average treatment effects in a Randomized Control Trial (RCT). We generalize Robins (1988) and show that when the estimand of interest is the Sample…

统计理论 · 数学 2017-10-19 Jasjeet S. Sekhon , Yotam Shem-Tov

Researchers are frequently interested in understanding the causal effect of treatment interventions. However, in some cases, the treatment of interest--readily available in a randomized controlled trial (RCT)--is either not directly…

统计方法学 · 统计学 2025-09-29 Lan Wen , Aaron L Sarvet

There is a growing literature on design-based methods to estimate average treatment effects (ATEs) for randomized controlled trials (RCTs) for full sample analyses. This article extends these methods to estimate ATEs for discrete subgroups…

统计方法学 · 统计学 2023-10-16 Peter Z. Schochet

A central obstacle in the objective assessment of treatment effect (TE) estimators in randomized control trials (RCTs) is the lack of ground truth (or validation set) to test their performance. In this paper, we propose a novel…

The restricted mean survival time (RMST) difference offers an interpretable causal contrast to estimate the treatment effect for time-to-event outcomes, yet a wide range of available estimators leaves limited guidance for practice. We…

统计方法学 · 统计学 2026-03-02 Charlotte Voinot , Clément Berenfeld , Imke Mayer , Bernard Sebastien , Julie Josse

In many practical situations, randomly assigning treatments to subjects is uncommon due to feasibility constraints. For example, economic aid programs and merit-based scholarships are often restricted to those meeting specific income or…

统计方法学 · 统计学 2025-04-25 Kevin Christian Wibisono , Debarghya Mukherjee , Moulinath Banerjee , Ya'acov Ritov

For learning about the causal effect of a treatment, a randomized controlled trial (RCT) is considered the gold standard. However, randomizing treatment is sometimes unethical or infeasible, and instead an observational study may be…

统计方法学 · 统计学 2024-04-01 Dylan S. Small

Randomized controlled trials (RCTs) often suffer from limited inferential efficiency in estimating treatment effects due to their small sample sizes. In recent years, incorporating external controls (ECs) has gained increasing attention as…

统计方法学 · 统计学 2026-04-16 Qinwei Yang , Jingyi Li , Peng Wu , Shu Yang

Multi-regional clinical trials (MRCTs) play an increasingly crucial role in global pharmaceutical development by expediting data gathering and regulatory approval across diverse patient populations. However, differences in recruitment…

统计方法学 · 统计学 2024-04-15 Kaiyuan Hua , Hwanhee Hong , Xiaofei Wang

Epidemiologists and applied statisticians often believe that relative effect measures conditional on covariates, such as risk ratios and mean ratios, are ``transportable'' across populations. Here, we examine the identification of causal…

统计方法学 · 统计学 2022-02-24 Issa J. Dahabreh , Sarah E. Robertson , Jon A. Steingrimsson

Participant noncompliance, in which participants do not follow their assigned treatment protocol, often obscures the causal relationship between treatment and treatment effect in randomized trials. In the longitudinal setting, the…

统计方法学 · 统计学 2023-02-09 Ross L Peterson , David M Vock , Joseph S Koopmeiners

Researchers often use linear regression to analyse randomized experiments to improve treatment effect estimation by adjusting for imbalances of covariates in the treatment and control groups. Our work offers a randomization-based inference…

统计理论 · 数学 2022-07-08 Hanzhong Liu , Yuehan Yang

The Average Treatment Effect (ATE) is a global measure of the effectiveness of an experimental treatment intervention. Classical methods of its estimation either ignore relevant covariates or do not fully exploit them. Moreover, past work…

统计方法学 · 统计学 2013-11-05 Emil Pitkin , Richard Berk , Lawrence Brown , Andreas Buja , Ed George , Kai Zhang , Linda Zhao