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Estimating causal effects in a target population with unmeasured confounders is challenging, especially when instrumental variables (IVs) are unavailable. However, IVs from auxiliary populations with similar problems can help infer causal…

Methodology · Statistics 2025-08-06 Wei Li , Jiapeng Liu , Peng Ding , Zhi Geng

Non-adherence to assigned treatment is common in randomised controlled trials (RCTs). Recently, there has been an increased interest in estimating causal effects of treatment received, for example the so-called local average treatment…

Methodology · Statistics 2018-12-05 Karla DiazOrdaz , James Carpenter

Estimating conditional average treatment effects (CATEs) from observational data is relevant in many fields such as personalized medicine. However, in practice, the treatment assignment is usually confounded by unobserved variables and thus…

Methodology · Statistics 2023-01-24 Dennis Frauen , Stefan Feuerriegel

A major challenge in instrumental variables (IV) analysis is to find instruments that are valid, or have no direct effect on the outcome and are ignorable. Typically one is unsure whether all of the putative IVs are in fact valid. We…

Statistics Theory · Mathematics 2017-08-10 Zijian Guo , Hyunseung Kang , T. Tony Cai , Dylan S. Small

In this paper, we study nonparametric estimation of instrumental variable (IV) regressions. Recently, many flexible machine learning methods have been developed for instrumental variable estimation. However, these methods have at least one…

Machine Learning · Statistics 2023-02-13 Andrew Bennett , Nathan Kallus , Xiaojie Mao , Whitney Newey , Vasilis Syrgkanis , Masatoshi Uehara

The recently published ICH E9 addendum on estimands in clinical trials provides a framework for precisely defining the treatment effect that is to be estimated, but says little about estimation methods. Here we report analyses of a clinical…

Applications · Statistics 2023-09-25 Camila Olarte Parra , Rhian M. Daniel , David Wright , Jonathan W. Bartlett

Variable selection for optimal treatment regime in a clinical trial or an observational study is getting more attention. Most existing variable selection techniques focused on selecting variables that are important for prediction, therefore…

Methodology · Statistics 2014-05-22 Ailin Fan , Wenbin Lu , Rui Song

To improve precision of estimation and power of testing hypothesis for an unconditional treatment effect in randomized clinical trials with binary outcomes, researchers and regulatory agencies recommend using g-computation as a reliable…

Methodology · Statistics 2023-03-29 Ting Ye , Marlena Bannick , Yanyao Yi , Jun Shao

A common concern in non-inferiority (NI) trials is that non adherence due, for example, to poor study conduct can make treatment arms artificially similar. Because intention to treat analyses can be anti-conservative in this situation, per…

Methodology · Statistics 2023-12-04 Katy E Morgan , Ian R White , Clémence Leyrat , Simon Stanworth , Brennan C Kahan

Unobserved spatial confounding variables are prevalent in environmental and ecological applications where the system under study is complex and the data are often observational. Instrumental variables (IVs) are a common way to address…

Methodology · Statistics 2021-03-02 Andrew Giffin , Brian J. Reich , Shu Yang , Ana G. Rappold

Individualized treatment rule (ITR) recommends treatment on the basis of individual patient characteristics and the previous history of applied treatments and their outcomes. Despite the fact there are many ways to estimate ITR with binary…

Methodology · Statistics 2017-08-15 Pavel Shvechikov , Evgeniy Riabenko

This paper considers inference in a linear instrumental variable regression model with many potentially weak instruments, in the presence of heterogeneous treatment effects. I first show that existing test procedures, including those that…

Econometrics · Economics 2025-04-24 Luther Yap

We study categorical instrumental variable (IV) models with instrument, treatment, and outcome taking finitely many values. We derive a simple closed-form characterization of the set of joint distributions of potential outcomes that are…

Statistics Theory · Mathematics 2025-11-13 Yilin Song , F. Richard Guo , K. C. Gary Chan , Thomas S. Richardson

Instrumental variables regression is a tool that is commonly used in the analysis of observational data. The instrumental variables are used to make causal inference about the effect of a certain exposure in the presence of unmeasured…

Methodology · Statistics 2023-09-07 Valentin Vancak , Arvid Sjölander

A growing statistical literature focuses on causal inference in the context of experiments where the target of inference is the average treatment effect in a finite population and random assignment determines which subjects are allocated to…

Methodology · Statistics 2025-09-04 Jonas M. Mikhaeil , Donald P. Green

The treatment assignment mechanism in a randomized clinical trial can be optimized for statistical efficiency within a specified class of randomization mechanisms. Optimal designs of this type have been characterized in terms of the…

Methodology · Statistics 2025-09-03 Wei Zhang , Zhiwei Zhang , Aiyi Liu

Most previous studies of the causal relationship between malaria and stunting have been studies where potential confounders are controlled via regression-based methods, but these studies may have been biased by unobserved confounders.…

Applications · Statistics 2015-11-11 Hyunseung Kang , Benno Kreuels , Jürgen May , Dylan S. Small

Clinical trials with a hybrid control arm (a control arm constructed from a combination of randomized patients and real-world data on patients receiving usual care in standard clinical practice) have the potential to decrease the cost of…

Methodology · Statistics 2021-08-20 Joanna Harton , Brian Segal , Ronac Mamtani , Nandita Mitra , Rebecca Hubbard

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…

Methodology · Statistics 2017-04-26 Ziyue Chen , Eloise Kaizar

We propose a reinforcement learning method for estimating an optimal dynamic treatment regime for survival outcomes with dependent censoring. The estimator allows the failure time to be conditionally independent of censoring and dependent…

Methodology · Statistics 2022-05-13 Hunyong Cho , Shannon T. Holloway , David J. Couper , Michael R. Kosorok