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Estimation of heterogeneous treatment effects is an active area of research. Most of the existing methods, however, focus on estimating the conditional average treatment effects of a single, binary treatment given a set of pre-treatment…

Methodology · Statistics 2025-05-30 Max Goplerud , Kosuke Imai , Nicole E. Pashley

It is crucial to design Phase II cancer clinical trials that balance the efficiency of treatment selection with clinical practicality. Sargent and Goldberg proposed a frequentist design that allow decision-making even when the primary…

Methodology · Statistics 2025-05-15 Moka Komaki , Satoru Shinoda , Haiyan Zheng , Kouji Yamamoto

Personalized medicine, a paradigm of medicine tailored to a patient's characteristics, is an increasingly attractive field in health care. An important goal of personalized medicine is to identify a subgroup of patients, based on baseline…

Machine Learning · Statistics 2023-01-31 Hengrui Cai , Wenbin Lu , Rachel Marceau West , Devan V. Mehrotra , Lingkang Huang

Individualized treatment regimes (ITRs) aim to improve clinical outcomes by assigning treatment based on patient-specific characteristics. However, existing methods often struggle with high-dimensional covariates, limiting accuracy,…

Machine Learning · Statistics 2026-01-13 Sungtaek Son , Eardi Lila , Kwun Chuen Gary Chan

In medical, social, and behavioral research we often encounter datasets with a multilevel structure and multiple correlated dependent variables. These data are frequently collected from a study population that distinguishes several…

Methodology · Statistics 2023-12-18 Xynthia Kavelaars , Joris Mulder , Maurits Kaptein

Randomized controlled trials typically analyze the effectiveness of treatments with the goal of making treatment recommendations for patient subgroups. With the advance of electronic health records, a great variety of data has been…

Machine Learning · Computer Science 2021-03-31 Zhiliang Wu , Yinchong Yang , Yunpu Ma , Yushan Liu , Rui Zhao , Michael Moor , Volker Tresp

Dynamic treatment regimes or policies are a sequence of decision functions over multiple stages that are tailored to individual features. One important class of treatment policies in practice, namely multi-stage stationary treatment…

Machine Learning · Statistics 2025-01-09 Daiqi Gao , Yufeng Liu , Donglin Zeng

Estimation of individual treatment effect in observational data is complicated due to the challenges of confounding and selection bias. A useful inferential framework to address this is the counterfactual (potential outcomes) model which…

Machine Learning · Statistics 2017-01-23 Min Lu , Saad Sadiq , Daniel J. Feaster , Hemant Ishwaran

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

The effects of treatments may differ between persons with different characteristics. Addressing such treatment heterogeneity is crucial to investigate whether patients with specific characteristics are likely to benefit from a new…

Methodology · Statistics 2024-03-29 Xynthia Kavelaars , Joris Mulder , Maurits Kaptein

Many policies involve dynamics in their treatment assignments, where individuals receive sequential interventions over multiple stages. We study estimation of an optimal dynamic treatment regime that guides the optimal treatment assignment…

Econometrics · Economics 2024-09-04 Shosei Sakaguchi

This paper estimates individual treatment effects in a triangular model with binary--valued endogenous treatments. Following the identification strategy established in Vuong and Xu (2014), we propose a two--stage estimation approach. First,…

Methodology · Statistics 2016-10-28 Qian Feng , Quang Vuong , Haiqing Xu

Estimating personalized treatment effects from high-dimensional observational data is essential in situations where experimental designs are infeasible, unethical, or expensive. Existing approaches rely on fitting deep models on outcomes…

Machine Learning · Computer Science 2022-02-02 Andrew Jesson , Panagiotis Tigas , Joost van Amersfoort , Andreas Kirsch , Uri Shalit , Yarin Gal

Case-mix heterogeneity across studies complicates meta-analyses. As a result of this, treatments that are equally effective on patient subgroups may appear to have different effectiveness on patient populations with different case mix. It…

Applications · Statistics 2021-08-25 Tat-Thang Vo , Raphael Porcher , Anna Chaimani , Stijn Vansteelandt

In randomized trials involving multiple treatments, bivariate survival outcomes present significant analytical challenges for making decisions. This paper addresses the problem of deriving optimal individualized treatment rules to maximize…

Machine Learning · Statistics 2026-05-29 Kun Ren , Yifan Cui , Wen Su

To estimate the causal effect of an endogenous treatment using clustered data, the canonical two-stage least squares (2sls) estimates a linear regression of the outcome on treatment status using an instrumental variable (IV) and conducts…

Methodology · Statistics 2026-04-03 Anqi Zhao , Peng Ding , Fan Li

Comparing outcomes across treatments is essential in medicine and public policy. To do so, researchers typically estimate a set of parameters, possibly counterfactual, with each targeting a different treatment. Treatment-specific means are…

Methodology · Statistics 2025-10-07 Alec McClean , Yiting Li , Sunjae Bae , Mara A. McAdams-DeMarco , Iván Díaz , Wenbo Wu

The treatment allocation mechanism in a randomized clinical trial can be optimized by maximizing the nonparametric efficiency bound for a specific measure of treatment effect. Optimal treatment allocations which may or may not depend on…

Methodology · Statistics 2025-05-23 Wei Zhang , Zhiwei Zhang , Aiyi Liu

Randomized trials typically estimate average relative treatment effects, but decisions on the benefit of a treatment are possibly better informed by more individualized predictions of the absolute treatment effect. In case of a binary…

Methodology · Statistics 2021-08-20 J Hoogland , J IntHout , M Belias , MM Rovers , RD Riley , FE Harrell , KGM Moons , TPA Debray , JB Reitsma

There has been significant attention given to developing data-driven methods for tailoring patient care based on individual patient characteristics. Dynamic treatment regimes formalize this through a sequence of decision rules that map…

Methodology · Statistics 2022-02-22 Eric J. Rose , Erica E. M. Moodie , Susan Shortreed