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In observational studies, treatments are typically not randomized and therefore estimated treatment effects may be subject to confounding bias. The instrumental variable (IV) design plays the role of a quasi-experimental handle since the IV…

Methodology · Statistics 2016-08-30 Lan Liu , Wang Miao , Baoluo Sun , James Robins , Eric Tchetgen Tchetgen

We provide a simple distribution regression estimator for treatment effects in the difference-in-differences (DiD) design. Our procedure is particularly useful when the treatment effect differs across the distribution of the outcome…

Econometrics · Economics 2026-05-20 Iván Fernández-Val , Jonas Meier , Aico van Vuuren , Francis Vella

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,…

The asymptotic efficiency, ARE_{p,2}, of the tests for multivariate means theta in \R^d based on the p-means relative to the standard 2-mean, (approximate) likelihood ratio test (LRT), is considered for large dimensions d. It turns out that…

Statistics Theory · Mathematics 2010-06-04 Iosif Pinelis

We are interested in the distribution of treatment effects for an experiment where units are randomized to a treatment but outcomes are measured for pairs of units. For example, we might measure risk sharing links between households…

Econometrics · Economics 2022-10-11 Eric Auerbach , Yong Cai

Measuring disease progression in clinical trials for testing novel treatments for multifaceted diseases as Progressive Supranuclear Palsy (PSP), remains challenging. In this study we assess a range of statistical approaches to compare…

Many major works in social science employ matching to make causal conclusions, but different matches on the same data may produce different treatment effect estimates, even when they achieve similar balance or minimize the same loss…

Applications · Statistics 2023-03-23 Marco Morucci , Cynthia Rudin

Doubly robust (DR) estimation is a crucial technique in causal inference and missing data problems. We propose a novel Propensity score Augmentved Doubly robust (PAD) estimator to enhance the commonly used DR estimator for average treatment…

Methodology · Statistics 2023-04-18 Liangbo Lyu , Molei Liu

In this paper, we propose a robust method to estimate the average treatment effects in observational studies when the number of potential confounders is possibly much greater than the sample size. We first use a class of penalized…

Methodology · Statistics 2018-12-21 Yang Ning , Sida Peng , Kosuke Imai

Difference-in-differences (DID) approaches are widely used for estimating causal effects with observational data before and after an intervention. DID traditionally estimates the average treatment effect among the treated after making a…

Methodology · Statistics 2025-06-24 Julia C. Thome , Andrew J. Spieker , Peter F. Rebeiro , Chun Li , Tong Li , Bryan E. Shepherd

Recurrent events are common and important clinical trial endpoints in many disease areas, e.g., cardiovascular hospitalizations in heart failure, relapses in multiple sclerosis, or exacerbations in asthma. During a trial, patients may…

Methodology · Statistics 2026-04-08 Jiren Sun , Tobias Mutze , Richard Cook , Tianmeng Lyu

The win ratio is increasingly used in randomized trials due to its intuitive clinical interpretation, ability to incorporate the relative importance of composite endpoints, and its capacity for combining different types of outcomes (e.g.…

Methodology · Statistics 2026-02-02 Tracy Bergemann , Tim Hanson

Complementary features of randomized controlled trials (RCTs) and observational studies (OSs) can be used jointly to estimate the average treatment effect of a target population. We propose a calibration weighting estimator that enforces…

Methodology · Statistics 2022-02-16 Dasom Lee , Shu Yang , Lin Dong , Xiaofei Wang , Donglin Zeng , Jianwen Cai

In randomized experiments, the actual treatments received by some experimental units may differ from their treatment assignments. This non-compliance issue often occurs in clinical trials, social experiments, and the applications of…

Methodology · Statistics 2022-04-19 Jiyang Ren

Human understanding of randomness and variation is shaped by a number of cognitive biases. Here we relate a lesser-known cognitive bias, the "outcome orientation", to medical questions and describe the harm that the outcome orientation can…

Physics Education · Physics 2017-02-20 Parris Taylor Humphrey , Joanna Masel

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

In the classic potential outcome framework, the local average treatment effect (LATE) and its identification via an instrumental variable are stated in a deterministic setting at the individual level: each individual has settled potential…

Methodology · Statistics 2026-05-14 Hanti Lin

Adjusting for (baseline) covariates with working regression models becomes standard practice in the analysis of randomized clinical trials (RCT). When the dimension $p$ of the covariates is large relative to the sample size $n$,…

Methodology · Statistics 2025-12-24 Yujia Gu , Lin Liu , Wei Ma

We present BEEP (Biomedical Evidence-Enhanced Predictions), a novel approach for clinical outcome prediction that retrieves patient-specific medical literature and incorporates it into predictive models. Based on each individual patient's…

Computation and Language · Computer Science 2022-11-17 Aakanksha Naik , Sravanthi Parasa , Sergey Feldman , Lucy Lu Wang , Tom Hope

In this paper, we introduce a new causal methodology that accounts for the rarity and frequency of events in observational studies based on their relevance to the underlying problem. Specifically, we propose a direct causal effect metric…

Artificial Intelligence · Computer Science 2025-02-28 Usef Faghihi , Amir Saki