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Related papers: Non-compliance and missing data in health economic…

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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…

General Economics · Economics 2021-06-03 Masayuki Sawada

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

When analyzing data from randomized clinical trials, covariate adjustment can be used to account for chance imbalance in baseline covariates and to increase precision of the treatment effect estimate. A practical barrier to covariate…

Methodology · Statistics 2023-07-04 Chia-Rui Chang , Yue Song , Fan Li , Rui Wang

Practical problems with missing data are common, and statistical methods have been developed concerning the validity and/or efficiency of statistical procedures. On a central focus, there have been longstanding interests on the mechanism…

Methodology · Statistics 2020-03-26 Rui Duan , C. Jason Liang , Pamela Shaw , Cheng Yong Tang , Yong Chen

Researchers are frequently interested in understanding the causal effect of treatment interventions. However, in some cases, the treatment of interest--readily available in a randomized controlled trial (RCT)--is either not directly…

Methodology · Statistics 2025-09-29 Lan Wen , Aaron L Sarvet

Missing data is prevalent in tabular machine learning (ML) models, and different missing data treatment methods can significantly affect ML model training results. However, little is known about how ML researchers and engineers choose…

Machine Learning · Computer Science 2025-03-24 Wanyi Chen , Mary Cummings

In 2016, the Dutch National Health Care Institute issued new guidelines that aggregated and updated previous recommendations on key elements for conducting economic evaluation. However, the impact on standard practice after the introduction…

Methodology · Statistics 2022-03-30 Andrea Gabrio

Missing data is a challenge when developing, validating and deploying clinical prediction models (CPMs). Traditionally, decisions concerning missing data handling during CPM development and validation havent accounted for whether…

Understanding whether and how treatment effects vary across subgroups is crucial to inform clinical practice and recommendations. Accordingly, the assessment of heterogeneous treatment effects (HTE) based on pre-specified potential effect…

Methodology · Statistics 2023-12-04 Bryan S. Blette , Scott D. Halpern , Fan Li , Michael O. Harhay

Most practical data science problems encounter missing data. A wide variety of solutions exist, each with strengths and weaknesses that depend upon the missingness-generating process. Here we develop a theoretical framework for training and…

Machine Learning · Computer Science 2022-11-15 Jahan C. Penny-Dimri , Christoph Bergmeir , Julian Smith

Individual-level effectiveness and healthcare resource use (HRU) data are routinely collected in trial-based economic evaluations. While effectiveness is often expressed in terms of utility scores derived from some health-related quality of…

Methodology · Statistics 2024-07-31 Andrea Gabrio

Machine learning is increasingly used to select which individuals receive limited-resource interventions in domains such as human services, education, development, and more. However, it is often not apparent what the right quantity is for…

Machine Learning · Computer Science 2025-03-20 Vibhhu Sharma , Bryan Wilder

Missing data arise in most applied settings and are ubiquitous in electronic health records (EHR). When data are missing not at random (MNAR) with respect to measured covariates, sensitivity analyses are often considered. These post-hoc…

Methodology · Statistics 2023-07-11 Alexander W. Levis , Rajarshi Mukherjee , Rui Wang , Heidi Fischer , Sebastien Haneuse

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…

Applications · Statistics 2025-03-25 Theodosios Papazoglou , Ed Waddingham , Alastair Young

Methods for addressing missing data have become much more accessible to applied researchers. However, little guidance exists to help researchers systematically identify plausible missing data mechanisms in order to ensure that these methods…

Applications · Statistics 2020-07-29 Adam Davey , Ting Dai

We provide sufficient conditions for the identification of the heterogeneous treatment effects, defined as the conditional expectation for the differences of potential outcomes given the untreated outcome, under the nonignorable treatment…

Methodology · Statistics 2019-01-15 Keisuke Takahata , Takahiro Hoshino

In Randomised Controlled Trials (RCT) with treatment non-compliance, instrumental variable approaches are used to estimate complier average causal effects. We extend these approaches to cost-effectiveness analyses, where methods need to…

Methodology · Statistics 2016-12-02 Karla DiazOrdaz , Angelo Franchini , Richard Grieve

Causally interpretable meta-analysis combines information from a collection of randomized controlled trials to estimate treatment effects in a target population in which experimentation may not be possible but covariate information can be…

Methodology · Statistics 2022-05-03 Jon A. Steingrimsson , David H. Barker , Ruofan Bie , Issa J. Dahabreh

Economic evaluations from individual-level data are an important component of the process of technology appraisal, with a view to informing resource allocation decisions. A critical problem in these analyses is that both effectiveness and…

Applications · Statistics 2018-02-01 Andrea Gabrio , Alexina J. Mason , Gianluca Baio

Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…

Methodology · Statistics 2025-08-05 Di Ran , Fanni Zhang , Sima Shahsavari , Kristine Broglio , Alasdair Henderson , Binbing Yu