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A common problem in Phase II clinical trials is the comparison of dose response curves corresponding to different treatment groups. If the effect of the dose level is described by parametric regression models and the treatments differ in…

Statistics Theory · Mathematics 2016-03-16 Chrystel Feller , Kirsten Schorning , Holger Dette , Georgina Bermann , Björn Bornkamp

Stroke is the second most common cause of death in developed countries, where rapid clinical intervention can have a major impact on a patient's life. To perform the revascularization procedure, the decision making of physicians considers…

Computer Vision and Pattern Recognition · Computer Science 2018-09-25 Adriano Pinto , Sergio Pereira , Raphael Meier , Victor Alves , Roland Wiest , Carlos A. Silva , Mauricio Reyes

To make informed health policy decisions regarding a treatment, we must consider both its cost and its clinical effectiveness. In past work, we introduced the net benefit separation (NBS) as a novel measure of cost-effectiveness. The NBS is…

Methodology · Statistics 2021-01-27 Nicholas Illenberger , Nandita Mitra , Andrew J. Spieker

Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…

Methodology · Statistics 2026-01-22 Xinyuan Chen , Fan Li

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

Clinical decision-making is a feedback system where risk estimates influence treatment, which in turn changes disease trajectories, and both shape clinicians' measurement practices. Static prediction often fails clinically: models trained…

Artificial Intelligence · Computer Science 2026-05-19 Pujun Feng , Xiaoyu Guo , Seyed Ehsan Saffari , Min Hun Lee , Siew-Kei Lam , Erik Cambria , Xibin Sun , Yangtao Zhou , Tong Yang , Xiaoyu Zhang , Tao Tan , Yue Sun , Bin Cui

Cancer is one of the most common diseases worldwide, posing a serious threat to human health and leading to the deaths of a large number of people. It was observed during the drug administration in chemotherapy that immune cells, cancer…

Quantitative Methods · Quantitative Biology 2020-08-20 SeyedMehdi Abtahi , Mojtaba Sharifi

Randomized trials are considered the gold standard for making informed decisions in medicine, yet they often lack generalizability to the patient populations in clinical practice. Observational studies, on the other hand, cover a broader…

Methodology · Statistics 2026-04-14 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

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

The difference-in-differences (DID) method identifies the average treatment effects on the treated (ATT) under mainly the so-called parallel trends (PT) assumption. The most common and widely used approach to justify the PT assumption is…

Econometrics · Economics 2023-08-23 Kyunghoon Ban , Désiré Kédagni

Prediction uncertainty estimation has clinical significance as it can potentially quantify prediction reliability. Clinicians may trust 'blackbox' models more if robust reliability information is available, which may lead to more models…

Machine Learning · Computer Science 2022-10-04 Michael Dohopolski , Kai Wang , Biling Wang , Ti Bai , Dan Nguyen , David Sher , Steve Jiang , Jing Wang

Markov Decision Processes and Dynamic Treatment Regimes have grown increasingly popular in the treatment of diseases, including cancer. However, cancer treatment often impacts quality of life drastically, and people often fail to take…

Applications · Statistics 2020-12-01 Navonil Deb , Abhinandan Dalal , Gopal Krishna Basak

Dynamic treatment regimes operationalize the clinical decision process as a sequence of functions, one for each clinical decision, where each function takes as input up-to-date patient information and gives as output a single recommended…

Methodology · Statistics 2012-08-08 Eric B. Laber , Daniel J. Lizotte , Bradley Ferguson

Meta-analyses of survival studies aim to reveal the variation of an effect measure of interest over different studies and present a meaningful summary. They must address between study heterogeneity in several dimensions and eliminate…

A dynamic treatment regime is a sequence of decision rules in which each decision rule recommends treatment based on features of patient medical history such as past treatments and outcomes. Existing methods for estimating optimal dynamic…

Methodology · Statistics 2015-05-22 Kristin A. Linn , Eric B. Laber , Leonard A. Stefanski

To enable personalized cancer treatment, machine learning models have been developed to predict drug response as a function of tumor and drug features. However, most algorithm development efforts have relied on cross validation within a…

The Predictive Approaches to Treatment Effect Heterogeneity statement focused on baseline risk as a robust predictor of treatment effect and provided guidance on risk-based assessment of treatment effect heterogeneity in the RCT setting.…

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

Multicriteria decision analysis (MCDA) is a widely used tool to support decisions in which a set of alternatives should be ranked or classified based on multiple criteria. Recent studies in MCDA have shown the relevance of considering not…

Machine Learning · Computer Science 2024-01-17 Betania Silva Carneiro Campello , Leonardo Tomazeli Duarte , João Marcos Travassos Romano

We consider a setting in which we have a treatment and a large number of covariates for a set of observations, and wish to model their relationship with an outcome of interest. We propose a simple method for modeling interactions between…

Methodology · Statistics 2012-12-14 Lu Tian , Ash Alizadeh , Andrew Gentles , Robert Tibshirani