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We study the estimation of distributional treatment effects in randomized experiments with imperfect compliance. When participants do not adhere to their assigned treatments, we leverage treatment assignment as an instrumental variable to…

统计方法学 · 统计学 2025-10-28 Undral Byambadalai , Tomu Hirata , Tatsushi Oka , Shota Yasui

Objective: Randomised controlled trials (RCTs) are widely considered as gold standard for assessing the effectiveness of new health interventions. When treatment non-compliance is present in RCTs, the treatment effect in the subgroup of…

应用统计 · 统计学 2025-03-25 Theodosios Papazoglou , Ed Waddingham , Alastair Young

Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…

统计方法学 · 统计学 2025-06-11 Ahmed Boughdiri , Julie Josse , Erwan Scornet

Randomized Controlled Trials (RCTs) represent a gold standard when developing policy guidelines. However, RCTs are often narrow, and lack data on broader populations of interest. Causal effects in these populations are often estimated using…

机器学习 · 计算机科学 2023-03-07 Zeshan Hussain , Michael Oberst , Ming-Chieh Shih , David Sontag

Randomized Controlled Trials (RCTs) are often considered the gold standard for estimating causal effect, but they may lack external validity when the population eligible to the RCT is substantially different from the target population.…

统计方法学 · 统计学 2023-01-11 Bénédicte Colnet , Julie Josse , Erwan Scornet , Gaël Varoquaux

In the presence of heterogeneity between the randomized controlled trial (RCT) participants and the target population, evaluating the treatment effect solely based on the RCT often leads to biased quantification of the real-world treatment…

统计方法学 · 统计学 2022-10-05 Dasom Lee , Shu Yang , Xiaofei Wang

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…

统计方法学 · 统计学 2022-02-16 Dasom Lee , Shu Yang , Lin Dong , Xiaofei Wang , Donglin Zeng , Jianwen Cai

There is a growing literature on design-based methods to estimate average treatment effects for randomized controlled trials (RCTs) using the underpinnings of experiments. In this article, we build on these methods to consider design-based…

统计方法学 · 统计学 2024-01-17 Peter Z Schochet

Health economic evaluations face the issues of non-compliance and missing data. Here, non-compliance is defined as non-adherence to a specific treatment, and occurs within randomised controlled trials (RCTs) when participants depart from…

应用统计 · 统计学 2019-02-26 Karla DiazOrdaz , Richard Grieve

Randomized controlled trials (RCTs) provide strong internal validity compared with observational studies. However, selection bias threatens the external validity of randomized trials. Thus, RCT results may not apply to either broad public…

统计方法学 · 统计学 2017-04-26 Ziyue Chen , Eloise Kaizar

Randomized experiments can provide unbiased estimates of sample average treatment effects. However, estimates of population treatment effects can be biased when the experimental sample and the target population differ. In this case, the…

统计方法学 · 统计学 2022-11-10 Wenqi Shi , Xi Lin

RCTs sometimes test interventions that aim to improve existing services targeted to a subset of individuals identified after randomization. Accordingly, the treatment could affect the composition of service recipients and the offered…

统计方法学 · 统计学 2022-05-18 Peter Z. Schochet

Subgroup analyses of randomized controlled trials (RCTs) constitute an important component of the drug development process in precision medicine. In particular, subgroup analyses of early-stage trials often influence the design and…

统计方法学 · 统计学 2025-02-12 Daniel Schwartz , Riddhiman Saha , Steffen Ventz , Lorenzo Trippa

A randomized controlled trial (RCT) is widely regarded as the gold standard for assessing the causal effect of a treatment or intervention, assuming perfect implementation. In practice, however, randomization can be compromised for various…

统计方法学 · 统计学 2026-04-21 Yin Tang , Yanyuan Ma , Jiwei Zhao

Randomized controlled trials (RCTs) frequently utilize covariate-adaptive randomization (CAR) (e.g., stratified block randomization) and commonly suffer from imperfect compliance. This paper studies the identification and inference for the…

计量经济学 · 经济学 2025-05-02 Federico A. Bugni , Mengsi Gao , Filip Obradovic , Amilcar Velez

Randomized controlled trials often enroll participants whose characteristics differ from those of a target population, which can limit the generalizability of the estimated treatment effects when effect modifiers differ across populations.…

统计方法学 · 统计学 2026-05-15 Lan Wen , Issa J. Dahabreh , Yu-Han Chiu

In a randomized control trial, the precision of an average treatment effect estimator can be improved either by collecting data on additional individuals, or by collecting additional covariates that predict the outcome variable. We propose…

统计方法学 · 统计学 2017-09-27 Pedro Carneiro , Sokbae Lee , Daniel Wilhelm

This study proposes a method to identify treatment effects without exclusion restrictions in randomized experiments with noncompliance. Exploiting a baseline survey commonly available in randomized experiments, I decompose the…

综合经济学 · 经济学 2021-06-03 Masayuki Sawada

Randomized controlled trials (RCTs) face inherent limitations, such as ethical or resource constraints, which lead to a limited number of study participants. To address these limitations, recent research endeavors have sought to incorporate…

统计方法学 · 统计学 2025-02-20 Peijin Wang , Hwanhee Hong , Kyungeun Jeon , Laine Elliott Thomas

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):…

统计方法学 · 统计学 2021-09-28 P. M. Aronow , James M. Robins , Theo Saarinen , Fredrik Sävje , Jasjeet Sekhon
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