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Micro-randomized trials (MRTs) are widely used to assess the marginal and moderated effect of mobile health (mHealth) treatments delivered via mobile devices. In many applications, the mHealth treatments are categorical with multiple levels…

Methodology · Statistics 2025-04-23 Jeremy Lin , Tianchen Qian

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

In clinical practice, decision-making relies heavily on established protocols, often formalised as rules. Concurrently, Machine Learning (ML) models, trained on clinical data, aspire to integrate into medical decision-making processes.…

Artificial Intelligence · Computer Science 2024-11-06 Christel Sirocchi , Muhammad Suffian , Federico Sabbatini , Alessandro Bogliolo , Sara Montagna

Development of new medications is a very lengthy and costly process. Finding novel indications for existing drugs, or drug repositioning, can serve as a useful strategy to shorten the development cycle. In this study, we present an approach…

Genomics · Quantitative Biology 2017-12-13 Kai Zhao , Hon-Cheong So

We investigate how to learn treatment effects away from the cutoff in multiple-cutoff regression discontinuity designs. Using a microeconomic model, we demonstrate that the parallel-trend type assumption proposed in the literature is…

Econometrics · Economics 2025-09-03 Yuta Okamoto , Yuuki Ozaki

In randomized trials, repeated measures of the outcome are routinely collected. The mixed model for repeated measures (MMRM) leverages the information from these repeated outcome measures, and is often used for the primary analysis to…

Methodology · Statistics 2023-07-20 Bingkai Wang , Yu Du

Risk adjustment has become an increasingly important tool in healthcare. It has been extensively applied to payment adjustment for health plans to reflect the expected cost of providing coverage for members. Risk adjustment models are…

Machine Learning · Computer Science 2019-07-16 Qiu-Yue Zhong , Andrew H. Fairless , Jasmine M. McCammon , Farbod Rahmanian

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

Standard objective functions used during the training of neural-network-based predictive models do not consider clinical criteria, leading to models that are not necessarily clinically acceptable. In this study, we look at this problem from…

Quantitative Methods · Quantitative Biology 2020-09-24 Maxime De Bois , Mounîm A. El Yacoubi , Mehdi Ammi

Here we introduce Partially Observed Structural Causal Models (POSCMs) that formalize causal systems where latent contexts co-determine both the interaction structure and downstream mechanisms on observed variables. POSCMs provide an…

Machine Learning · Computer Science 2026-05-06 Turan Orujlu , Jordan Matelsky , Martin V. Butz , Charley M. Wu , Konrad P. Kording

Due to unmeasured confounding, it is often not possible to identify causal effects from a postulated model. Nevertheless, we can ask for partial identification, which usually boils down to finding upper and lower bounds of a causal quantity…

Machine Learning · Statistics 2022-03-01 Jakob Zeitler , Ricardo Silva

Marginal structural models are a popular method for estimating causal effects in the presence of time-varying exposures. In spite of their popularity, no scalable non-parametric estimator exist for marginal structural models with…

Methodology · Statistics 2024-09-30 Axel Martin , Michele Santacatterina , Iván Díaz

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

Causal machine learning (ML) offers flexible, data-driven methods for predicting treatment outcomes including efficacy and toxicity, thereby supporting the assessment and safety of drugs. A key benefit of causal ML is that it allows for…

Given data on the choices made by consumers for different offer sets, a key challenge is to develop parsimonious models that describe and predict consumer choice behavior while being amenable to prescriptive tasks such as pricing and…

Machine Learning · Statistics 2025-04-15 Yanqiu Ruan , Xiaobo Li , Karthyek Murthy , Karthik Natarajan

Discovering subgroups with the maximum average treatment effect is crucial for targeted decision making in domains such as precision medicine, public policy, and education. While most prior work is formulated in the potential outcome…

Machine Learning · Computer Science 2025-11-26 Lincen Yang , Zhong Li , Matthijs van Leeuwen , Saber Salehkaleybar

Despite the common occurrence of interference in Difference-in-Differences (DiD) applications, standard DiD methods rely on an assumption that interference is absent, and comparatively little work has considered how to accommodate and learn…

Methodology · Statistics 2025-11-03 Zach Shahn , Paul Zivich , Audrey Renson

Model-based recursive partitioning (MOB) can be used to identify subgroups with differing treatment effects. The detection rate of treatment-by-covariate interactions and the accuracy of identified subgroups using MOB depend strongly on the…

Applications · Statistics 2022-09-07 Cynthia Huber , Norbert Benda , Tim Friede

Randomized clinical trials typically aim to estimate a marginal treatment effect. While covariate adjustment can improve precision, it may change the estimand in nonlinear models due to noncollapsibility, leading to conditional rather than…

Methodology · Statistics 2026-05-25 Leticia Wuethrich , Torsten Hothorn

In many practical applications of machine learning, a discrepancy often arises between a source distribution from which labeled training examples are drawn and a target distribution for which only unlabeled data is observed. Traditionally,…

Machine Learning · Statistics 2025-03-05 Paweł Teisseyre , Jan Mielniczuk