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Observational longitudinal studies are a common means to study treatment efficacy and safety in chronic mental illness. In many such studies, treatment changes may be initiated by either the patient or by their clinician and can thus vary…

Methodology · Statistics 2020-06-12 Zekun Xu , Eric Laber , Ana-Maria Staicu , Emanuel Severus

In many clinical trials, individuals in different subgroups have experience differential treatment effects. This leads to individualized differences in treatment benefit. In this article, we introduce the general concept of predictive…

Methodology · Statistics 2018-07-11 Debashis Ghosh , Youngjoo Cho

Treatment effect estimation is essential for informed decision-making in many fields such as healthcare, economics, and public policy. While flexible machine learning models have been widely applied for estimating heterogeneous treatment…

Machine Learning · Computer Science 2025-09-29 Pascal Memmesheimer , Vincent Heuveline , Jürgen Hesser

Conventional treatment policies map patient covariates to a single recommended intervention in order to maximize expected clinical outcomes. Although a rich body of causal inference methods has been developed to estimate such policies,…

Machine Learning · Computer Science 2026-05-20 Laura Fuentes-Vicente , Mathieu Even , Gaëlle Dormion , Antoine Chambaz , Uri Shalit , Julie Josse

Identifying optimal medical treatments to improve survival has long been a critical goal of pharmacoepidemiology. Traditionally, we use an average treatment effect measure to compare outcomes between treatment plans. However, new methods…

Interim analyses for group-sequential decision making are prevalent in clinical trials. Methodology is well established and has been routinely implemented over the last decades. Still, confusions and uncertainties on aspects of how to…

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

Methodology · Statistics 2021-08-31 Elja Arjas , Dario Gasbarra

Randomized experiments have been used to assist decision-making in many areas. They help people select the optimal treatment for the test population with certain statistical guarantee. However, subjects can show significant heterogeneity in…

Artificial Intelligence · Computer Science 2017-05-25 Yan Zhao , Xiao Fang , David Simchi-Levi

Randomized experimentation (also known as A/B testing or bucket testing) is widely used in the internet industry to measure the metric impact obtained by different treatment variants. A/B tests identify the treatment variant showing the…

Tree ensembles are non-parametric methods widely recognized for their accuracy and ability to capture complex interactions. While these models excel at prediction, they are difficult to interpret and may fail to uncover useful relationships…

Machine Learning · Statistics 2026-04-01 Brian Liu , Rahul Mazumder , Peter Radchenko

In settings where units' outcomes are affected by others' treatments, there has been a proliferation of ways to quantify effects of treatments on outcomes, including via indirect exposure to other units' treatments. Here we consider two…

Methodology · Statistics 2026-03-10 Sahil Loomba , Dean Eckles

Sequence classification is the task of predicting a class label given a sequence of observations. In many applications such as healthcare monitoring or intrusion detection, early classification is crucial to prompt intervention. In this…

Machine Learning · Computer Science 2020-10-07 Maayan Shvo , Andrew C. Li , Rodrigo Toro Icarte , Sheila A. McIlraith

Systematic use of the published results of randomized clinical trials is increasingly important in evidence-based medicine. In order to collate and analyze the results from potentially numerous trials, evidence tables are used to represent…

Computation and Language · Computer Science 2015-09-18 Antonio Trenta , Anthony Hunter , Sebastian Riedel

Clinical trials are a systematic endeavor to assess the safety and efficacy of new drugs or treatments. Conducting such trials typically demands significant financial investment and meticulous planning, highlighting the need for accurate…

Machine Learning · Computer Science 2025-11-03 Tien Huu Do , Antoine Masquelier , Nae Eoun Lee , Jonathan Crowther

Digital health interventions (DHIs) and remote patient monitoring (RPM) have shown great potential in improving chronic disease management through personalized care. However, barriers like limited efficacy and workload concerns hinder…

Machine Learning · Computer Science 2025-12-23 Johannes O. Ferstad , Emily B. Fox , David Scheinker , Ramesh Johari

Prediction models developed before the introduction of a new treatment may be used to estimate treatment effects of newly introduced treatments. One approach, known as model-based clinical evaluation in radiotherapy, does this by comparing…

A master protocol trial uses a single overarching protocol to test multiple therapies, often across several diseases or subtypes. Although such trials offer considerable flexibility and efficiency, their constrained and non-uniform…

Methodology · Statistics 2026-04-29 Shiyu Wan , Yuhan Qian , Yanyao Yi , Nicole Mayer-Hamblett , Patrick J. Heagerty , Ting Ye

We consider a general difference-in-differences model in which the treatment variable of interest may be non-binary and its value may change in each period. It is generally difficult to estimate treatment parameters defined with the…

Econometrics · Economics 2023-05-30 Takahide Yanagi

This article discusses the application of stochastic intervention to find the optimal treatment distribution yielding a high value of expected potential outcome under the setting where the number of treatments is allowed to vary with $n$.…

Statistics Theory · Mathematics 2026-04-22 Rohit Chaudhuri

In order to identify important variables that are involved in making optimal treatment decision, Lu et al. (2013) proposed a penalized least squared regression framework for a fixed number of predictors, which is robust against the…

Machine Learning · Statistics 2015-10-16 Chengchun Shi , Rui Song , Wenbin Lu