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We study the design of multi-armed parallel group clinical trials to estimate personalized treatment rules that identify the best treatment for a given patient with given covariates. Assuming that the outcomes in each treatment arm are…

统计理论 · 数学 2022-07-13 David Azriel , Yosef Rinott , Martin Posch

The use of patient-level information from previous studies, registries, and other external datasets can support the analysis of single-arm and randomized controlled trials to evaluate and test experimental treatments. However, the…

统计方法学 · 统计学 2025-10-23 Gopal Kotecha , Daniel E. Schwartz , Steffen Ventz , Lorenzo Trippa

Classical approaches to experimental design assume that intervening on one unit does not affect other units. There are many important settings, however, where this non-interference assumption does not hold, as when running experiments on…

最优化与控制 · 数学 2020-07-01 Stefan Wager , Kuang Xu

When making treatment selection decisions, it is essential to include a causal effect estimation analysis to compare potential outcomes under different treatments or controls, assisting in optimal selection. However, merely estimating…

机器学习 · 统计学 2024-10-08 Sherly Alfonso-Sánchez , Kristina P. Sendova , Cristián Bravo

One of the key challenges when developing a predictive model is the capability to describe the domain knowledge and the cause-effect relationships in a simple way. Decision rules are a useful and important methodology in this context,…

机器学习 · 计算机科学 2021-10-19 Francisco Valente , Jorge Henriques , Simão Paredes , Teresa Rocha , Paulo de Carvalho , João Morais

We consider optimal regimes for algorithm-assisted human decision-making. Such regimes are decision functions of measured pre-treatment variables and, by leveraging natural treatment values, enjoy a "superoptimality" property whereby they…

统计方法学 · 统计学 2024-02-23 Mats J. Stensrud , Julien Laurendeau , Aaron L. Sarvet

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.…

人工智能 · 计算机科学 2024-11-06 Christel Sirocchi , Muhammad Suffian , Federico Sabbatini , Alessandro Bogliolo , Sara Montagna

Dynamic treatment regimes are of growing interest across the clinical sciences as these regimes provide one way to operationalize and thus inform sequential personalized clinical decision making. A dynamic treatment regime is a sequence of…

统计方法学 · 统计学 2013-11-27 Eric B. Laber , Min Qian , Dan J. Lizotte , William E. Pelham , Susan A. Murphy

The sequential treatment decisions made by physicians to treat chronic diseases are formalized in the statistical literature as dynamic treatment regimes. To date, methods for dynamic treatment regimes have been developed under the…

统计方法学 · 统计学 2022-02-22 Janie Coulombe , Erica E. M. Moodie , Susan M. Shortreed , Christel Renoux

The keyboard design is a novel phase I dose-finding method that is simple and has good operating characteristics. This paper studies theoretical properties of the keyboard design, including the optimality of its decision rules, coherence in…

统计方法学 · 统计学 2017-12-20 Haitao Pan , Ruitao Lin , Ying Yuan

The optimal dynamic treatment rule (ODTR) framework offers an approach for understanding which kinds of patients respond best to specific treatments -- in other words, treatment effect heterogeneity. Recently, there has been a proliferation…

Pragmatic randomized trials are designed to provide evidence for clinical decision-making rather than regulatory approval. Common features of these trials include the inclusion of heterogeneous or diverse patient populations in a wide range…

统计方法学 · 统计学 2019-11-20 Eleanor J. Murray , Sonja A. Swanson , Miguel A. Hernán

An optimal dynamic treatment regime (DTR) is a sequence of decision rules aimed at providing the best course of treatments individualized to patients. While conventional DTR estimation uses longitudinal data, such data can also be…

统计方法学 · 统计学 2025-02-06 Larry Dong , Eleanor Pullenayegum , Rodolphe Thiébaut , Olli Saarela

Prediction algorithms that quantify the expected benefit of a given treatment conditional on patient characteristics can critically inform medical decisions. Quantifying the performance of treatment benefit prediction algorithms is an…

统计方法学 · 统计学 2023-05-17 Yuan Xia , Paul Gustafson , Mohsen Sadatsafavi

Research on decision support applications in healthcare, such as those related to diagnosis, prediction, treatment planning, etc., have seen enormously increased interest recently. This development is thanks to the increase in data…

机器学习 · 计算机科学 2021-03-25 Jussi Tohka , Mark van Gils

Trial engagement effects are effects of trial participation on the outcome that are not mediated by treatment assignment. Most work on extending (generalizing or transporting) causal inferences from a randomized trial to a target population…

统计方法学 · 统计学 2024-07-23 Lawson Ung , Tyler J. VanderWeele , Issa J. Dahabreh

Both cluster randomized trials and quasi-experimental designs are used to evaluate the impact of health and social policies and interventions. Stepped-wedge cluster randomized trials randomize a staggered adoption approach, while recent…

统计方法学 · 统计学 2026-04-15 Haidong Lu , Gregg S. Gonsalves , Fan Li , Guanyu Tong , Lee Kennedy-Shaffer

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…

统计方法学 · 统计学 2025-10-07 Alec McClean , Yiting Li , Sunjae Bae , Mara A. McAdams-DeMarco , Iván Díaz , Wenbo Wu

Data integration approaches are increasingly used to enhance the efficiency and generalizability of studies. However, a key limitation of these methods is the assumption that outcome measures are identical across datasets -- an assumption…

统计方法学 · 统计学 2025-05-19 Harsh Parikh , Trang Quynh Nguyen , Elizabeth A. Stuart , Kara E. Rudolph , Caleb H. Miles

Click-through rate (CTR) prediction is a critical task for many applications, as its accuracy has a direct impact on user experience and platform revenue. In recent years, CTR prediction has been widely studied in both academia and…

信息检索 · 计算机科学 2025-11-19 Jieming Zhu , Jinyang Liu , Shuai Yang , Qi Zhang , Xiuqiang He