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Considerations regarding clinical effectiveness and cost are essential in comparing the overall value of two treatments. There has been growing interest in methodology to integrate cost and effectiveness measures in order to inform policy…

Methodology · Statistics 2019-12-04 Andrew J. Spieker , Nicholas Illenberger , Jason A. Roy , Nandita Mitra

This paper focuses on the Bayesian Network Propensity Score (BNPS), a novel approach for estimating treatment effects in observational studies characterized by unknown (and likely unbalanced) designs and complex dependency structures among…

In recent years, theoretical results and simulation evidence have shown Bayesian additive regression trees to be a highly-effective method for nonparametric regression. Motivated by cost-effectiveness analyses in health economics, where…

When data on treatment assignment, outcomes, and covariates from a randomized trial are available, a question of interest is to what extent covariates can be used to optimize treatment decisions. Statistical hypothesis testing of…

Applications · Statistics 2020-02-04 Mohsen Sadatsafavi , Mohammad Ali Mansournia , Paul Gustafson

Band selection has a great impact on the spectral recovery quality. To solve this ill-posed inverse problem, most band selection methods adopt hand-crafted priors or exploit clustering or sparse regularization constraints to find most…

Computer Vision and Pattern Recognition · Computer Science 2023-05-17 Hai-Miao Hu , Zhenbo Xu , Wenshuai Xu , You Song , YiTao Zhang , Liu Liu , Zhilin Han , Ajin Meng

One of the most common ways researchers compare survival outcomes across treatments when confounding is present is using Cox regression. This model is limited by its underlying assumption of proportional hazards; in some cases, substantial…

Applications · Statistics 2021-02-02 Elizabeth A. Handorf , Marc Smaldone , Sujana Movva , Nandita Mitra

In the fight against hard-to-treat diseases such as cancer, it is often difficult to discover new treatments that benefit all subjects. For regulatory agency approval, it is more practical to identify subgroups of subjects for whom the…

Methodology · Statistics 2014-10-09 Wei-Yin Loh , Xu He , Michael Man

The premise of independence among subjects in the same cluster/group often fails in practice, and models that rely on such untenable assumption can produce misleading results. To overcome this severe deficiency, we introduce a new…

Methodology · Statistics 2022-02-22 Jussiane Nader Gonçalves , Wagner Barreto-Souza , Hernando Ombao

Patient-level health economic data collected alongside clinical trials are an important component of the process of technology appraisal, with a view to informing resource allocation decisions. For end of life treatments, such as cancer…

Applications · Statistics 2020-11-24 Andrea Gabrio

Estimation and inference of treatment effects under unconfounded treatment assignments often suffer from bias and the `curse of dimensionality' due to the nonparametric estimation of nuisance parameters for high-dimensional confounders.…

Methodology · Statistics 2025-07-08 Zeqi Wu , Meilin Wang , Wei Huang , Zheng Zhang

Nonparametric estimators of the mean total cost have been proposed in a variety of settings. In clinical trials it is generally impractical to follow up patients until all have responded, and therefore censoring of patient outcomes and…

Applications · Statistics 2008-12-18 Joseph C. Gardiner , Lin Liu , Zhehui Luo

Propensity scores are commonly used to reduce the confounding bias in non-randomized observational studies for estimating the average treatment effect. An important assumption underlying this approach is that all confounders that are…

Methodology · Statistics 2022-08-02 Youfei Yu , Jiacong Du , Min Zhang , Zhenke Wu , Andrew M. Ryan , Bhramar Mukherjee

This paper proposes a new statistical approach for assessing treatment effect using Bayesian Networks (BNs). The goal is to draw causal inferences from observational data with a binary outcome and discrete covariates. The BNs are here used…

It is often of interest to estimate regression functions non-parametrically. Penalized regression (PR) is one statistically-effective, well-studied solution to this problem. Unfortunately, in many cases, finding exact solutions to PR…

Methodology · Statistics 2021-12-08 Brayan Ortiz , Noah Simon

Linear regression is arguably the most fundamental statistical model; however, the validity of its use in randomized clinical trials, despite being common practice, has never been crystal clear, particularly when stratified or…

Methodology · Statistics 2023-02-14 Wei Ma , Fuyi Tu , Hanzhong Liu

Risk prediction models are a crucial tool in healthcare. Risk prediction models with a binary outcome (i.e., binary classification models) are often constructed using methodology which assumes the costs of different classification errors…

Estimating the cure fraction in a diseased population, especially in the presence of competing mortality causes, is crucial for both patients and clinicians. It offers a valuable measure for monitoring and interpreting trends in disease…

Understanding how treatment effects vary on individual characteristics is critical in the contexts of personalized medicine, personalized advertising and policy design. When the characteristics are of practical interest are only a subset of…

Methodology · Statistics 2023-05-03 Peng Wu , ShaSha Han , Xingwei Tong , Runze Li

Estimates of the effects of treatment on cost from observational studies are subject to bias if there are unmeasured confounders. It is therefore advisable in practice to assess the potential magnitude of such biases. We derive a general…

Applications · Statistics 2014-01-09 Elizabeth A. Handorf , Justin E. Bekelman , Daniel F. Heitjan , Nandita Mitra

In this paper, we address the issue of estimating and inferring distributional treatment effects in randomized experiments. The distributional treatment effect provides a more comprehensive understanding of treatment heterogeneity compared…

Econometrics · Economics 2025-01-15 Tatsushi Oka , Shota Yasui , Yuta Hayakawa , Undral Byambadalai
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