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This paper provides clear and practical guidance on the specification of imputation models when multiple imputation is used in conjunction with doubly robust estimation methods for causal inference. Through theoretical arguments and…

Methodology · Statistics 2025-12-19 Lucy D'Agostino McGowan

A data science task can be deemed as making sense of the data or testing a hypothesis about it. The conclusions inferred from data can greatly guide us to make informative decisions. Big data has enabled us to carry out countless prediction…

Machine Learning · Computer Science 2022-01-12 Wenhao Zhang , Ramin Ramezani , Arash Naeim

We study causal inference under case-control and case-population sampling. Specifically, we focus on the binary-outcome and binary-treatment case, where the parameters of interest are causal relative and attributable risks defined via the…

Econometrics · Economics 2023-10-24 Sung Jae Jun , Sokbae Lee

Large scale disease screening is a complicated process in which high costs must be balanced against pressing public health needs. When the goal is screening for infectious disease, one approach is group testing in which samples are…

Applications · Statistics 2021-03-02 Gregory Haber , Yaakov Malinovsky , Paul S. Albert

Significant treatment effects are often emphasized when interpreting and summarizing empirical findings in studies that estimate multiple, possibly many, treatment effects. Under this kind of selective reporting, conventional treatment…

Econometrics · Economics 2025-12-11 Andreas Dzemski , Ryo Okui , Wenjie Wang

This paper introduces an overidentification test of two alternative assumptions to identify the average treatment effect on the treated in a two-period panel data setting: unconfoundedness and common trends. Under the unconfoundedness…

Econometrics · Economics 2024-06-25 Martin Huber , Eva-Maria Oeß

It is generally believed that bias is minimized in well-controlled randomized clinical trials. However, bias can arise in active controlled noninferiority trials because the inference relies on a previously estimated effect size obtained…

Applications · Statistics 2013-12-02 Lei Nie , Zhiwei Zhang , Daniel Rubin , Jianxiong Chu

We seek to understand the probability an individual benefits from treatment (PIBT), an inestimable quantity that must be bounded in practice. Given the innate uncertainty in the population-level bounds on PIBT, we seek to better understand…

Methodology · Statistics 2024-04-04 Gabriel Ruiz , Oscar Hernan Madrid Padilla

This paper develops a class of potential outcomes models characterized by three main features: (i) Unobserved heterogeneity can be represented by a vector of potential outcomes and a type describing the manner in which an instrument…

Econometrics · Economics 2023-10-10 Manu Navjeevan , Rodrigo Pinto , Andres Santos

The estimands framework outlined in ICH E9 (R1) describes the components needed to precisely define the effects to be estimated in clinical trials, which includes how post-baseline "intercurrent" events (IEs) are to be handled. In…

Methodology · Statistics 2023-08-28 Thomas Drury , Juan J Abellan , Nicky Best , Ian R. White

Machine learning models are often trained to predict the outcome resulting from a human decision. For example, if a doctor decides to test a patient for disease, will the patient test positive? A challenge is that historical decision-making…

Machine Learning · Computer Science 2024-04-23 Sidhika Balachandar , Nikhil Garg , Emma Pierson

When studying the association between treatment and a clinical outcome, a parametric multivariable model of the conditional outcome expectation is often used to adjust for covariates. The treatment coefficient of the outcome model targets a…

Methodology · Statistics 2026-05-07 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

Given the prominence of targeted therapy and immunotherapy in cancer treatment, it becomes imperative to consider heterogeneity in patients' responses to treatments, which contributes greatly to the widely used proportional hazard…

Methodology · Statistics 2024-09-01 Powei Chen , Siying Hu , Haojin Zhou

Inverse probability weights are commonly used in epidemiology to estimate causal effects in observational studies. Researchers can typically focus on either the average treatment effect or the average treatment effect on the treated with…

Methodology · Statistics 2022-10-05 Eli Ben-Michael , Luke Keele

Substudies of the Childhood Asthma Management Program [Control. Clin. Trials 20 (1999) 91-120; N. Engl. J. Med. 343 (2000) 1054-1063] seek to identify patient characteristics associated with asthma symptoms and lung function. To determine…

Motivated by a study about prompt coronary angiography in myocardial infarction, we propose a method to estimate the causal effect of a treatment in two-arm experimental studies with possible non-compliance in both treatment and control…

Statistics Theory · Mathematics 2012-10-26 F. Bartolucci , A. Farcomeni

In causal inference, it is common to estimate the causal effect of a single treatment variable on an outcome. However, practitioners may also be interested in the effect of simultaneous interventions on multiple covariates of a fixed target…

Methodology · Statistics 2022-11-24 Jaime Roquero Gimenez , Dominik Rothenhäusler

Although randomized experiments are widely regarded as the gold standard for estimating causal effects, missing data of the pretreatment covariates makes it challenging to estimate the subgroup causal effects. When the missing data…

Statistics Theory · Mathematics 2014-01-08 Peng Ding , Zhi Geng

Patient-reported outcomes (PROs) directly collected from cancer patients being treated with radiation therapy play a vital role in assisting clinicians in counseling patients regarding likely toxicities. Precise prediction and evaluation of…

Machine Learning · Computer Science 2024-11-19 Yang Yan , Zhong Chen , Cai Xu , Xinglei Shen , Jay Shiao , John Einck , Ronald C Chen , Hao Gao

Recently, there has been great interest in estimating the conditional average treatment effect using flexible machine learning methods. However, in practice, investigators often have working hypotheses about effect heterogeneity across…

Methodology · Statistics 2023-09-13 Chan Park , Hyunseung Kang