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Clinical trials are indispensable in developing new treatments, but they face obstacles in patient recruitment and retention, hindering the enrollment of necessary participants. To tackle these challenges, deep learning frameworks have been…

Machine Learning · Computer Science 2023-03-27 Chia-Yuan Chang , Jiayi Yuan , Sirui Ding , Qiaoyu Tan , Kai Zhang , Xiaoqian Jiang , Xia Hu , Na Zou

Multivalued treatment models have typically been studied under restrictive assumptions: ordered choice, and more recently unordered monotonicity. We show how treatment effects can be identified in a more general class of models that allows…

Econometrics · Economics 2018-05-02 Sokbae Lee , Bernard Salanié

A treatment policy defines when and what treatments are applied to affect some outcome of interest. Data-driven decision-making requires the ability to predict what happens if a policy is changed. Existing methods that predict how the…

Machine Learning · Computer Science 2023-06-21 Çağlar Hızlı , ST John , Anne Juuti , Tuure Saarinen , Kirsi Pietiläinen , Pekka Marttinen

Personalized treatment effect estimates are often of interest in high-stakes applications -- thus, before deploying a model estimating such effects in practice, one needs to be sure that the best candidate from the ever-growing machine…

Machine Learning · Statistics 2023-06-07 Alicia Curth , Mihaela van der Schaar

Controlled experiments are widely used in many applications to investigate the causal relationship between input factors and experimental outcomes. A completely randomized design is usually used to randomly assign treatment levels to…

Methodology · Statistics 2026-05-12 Yiou Li , Lulu Kang , Xiao Huang

The present communication addresses a set of observations, obeying both deterministic as well as statistical formal requirements, and serving to operate within the framework of the dynamical systems theory, with a certain emphasis placed on…

Physics and Society · Physics 2017-06-30 Adam Gadomski , Marcel Ausloos , Tahlia Casey

We investigate the optimal design of experimental studies that have pre-treatment outcome data available. The average treatment effect is estimated as the difference between the weighted average outcomes of the treated and control units. A…

At our behest or otherwise, while our software is being executed, a huge variety of design assumptions is continuously matched with the truth of the current condition. While standards and tools exist to express and verify some of these…

Software Engineering · Computer Science 2016-05-09 Vincenzo De Florio

Studies often report estimates of the average treatment effect. While the ATE summarizes the effect of a treatment on average, it does not provide any information about the effect of treatment within any individual. A treatment strategy…

Methodology · Statistics 2025-06-13 Nicholas T. Williams , Katherine L. Hoffman Iván Díaz , Kara E. Rudolph

Accurate models of clinical actions and their impacts on disease progression are critical for estimating personalized optimal dynamic treatment regimes (DTRs) in medical/health research, especially in managing chronic conditions.…

Methodology · Statistics 2021-02-19 William Hua , Hongyuan Mei , Sarah Zohar , Magali Giral , Yanxun Xu

In parametric Bayesian designs of early phase cancer clinical trials with drug combinations exploring a discrete set of partially ordered doses, several authors claimed that there is no added value in including an interaction term to model…

Methodology · Statistics 2022-08-12 Mourad Tighiouart , José L. Jiménez , Marcio A. Diniz , André Rogatko

While artificial-intelligence-based methods suffer from lack of transparency, rule-based methods dominate in safety-critical systems. Yet, the latter cannot compete with the first ones in robustness to multiple requirements, for instance,…

Artificial Intelligence · Computer Science 2022-02-01 Andrei Aksjonov , Ville Kyrki

The ongoing pandemic has highlighted the importance of reliable and efficient clinical trials in healthcare. Trial sites, where the trials are conducted, are chosen mainly based on feasibility in terms of medical expertise and access to a…

Machine Learning · Computer Science 2022-04-14 Rakshith S Srinivasa , Cheng Qian , Brandon Theodorou , Jeffrey Spaeder , Cao Xiao , Lucas Glass , Jimeng Sun

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

Methodology · Statistics 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

A dynamic treatment regimen (DTR) is a pre-specified sequence of decision rules which maps baseline or time-varying measurements on an individual to a recommended intervention or set of interventions. Sequential multiple assignment…

Methodology · Statistics 2019-10-23 Brook Luers , Min Qian , Inbal Nahum-Shani , Connie Kasari , Daniel Almirall

Clinical trials often evaluate multiple outcome variables to form a comprehensive picture of the effects of a new treatment. The resulting multidimensional insight contributes to clinically relevant and efficient decision-making about…

Methodology · Statistics 2023-08-14 X. M. Kavelaars , J. Mulder , M. C. Kaptein

A key aspect of patient-focused drug development is identifying and measuring outcomes that are important to patients in clinical trials. Many medical conditions affect multiple symptom domains, and a consensus approach to determine the…

Methodology · Statistics 2025-10-14 David S. Robertson , Thomas Jaki

Purpose: The 3+3 design has been shown to be less likely to achieve the objectives of phase I dose-finding trials when compared with more advanced model-based designs. One major criticism of the 3+3 design is that it is based on simple…

Methodology · Statistics 2019-04-30 Meizi Liu , Sue-Jane Wang , Yuan Ji

Medical research has evolved conventions for choosing sample size in randomized clinical trials that rest on the theory of hypothesis testing. Bayesians have argued that trials should be designed to maximize subjective expected utility in…

Methodology · Statistics 2018-12-11 Charles F. Manski , Aleksey Tetenov

We consider the optimal design problem for identifying effective dose combinations within drug combination studies where the effect of the combination of two drugs is investigated. Drug combination studies are becoming increasingly…