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When a new treatment is considered for use, whether a pharmaceutical drug or a search engine ranking algorithm, a typical question that arises is, will its performance exceed that of the current treatment? The conventional way to answer…

Machine Learning · Computer Science 2016-10-27 Nir Rosenfeld , Yishay Mansour , Elad Yom-Tov

Integrating data from multiple heterogeneous sources has become increasingly popular to achieve a large sample size and diverse study population. This paper reviews development in causal inference methods that combines multiple datasets…

Methodology · Statistics 2021-10-05 Xu Shi , Ziyang Pan , Wang Miao

There are multiple cluster randomised trial designs that vary in when the clusters cross between control and intervention states, when observations are made within clusters, and how many observations are made at that time point. Identifying…

Methodology · Statistics 2023-07-20 Samuel I. Watson , Alan Girling , Karla Hemming

We develop a hybrid control approach for robot learning based on combining learned predictive models with experience-based state-action policy mappings to improve the learning capabilities of robotic systems. Predictive models provide an…

Robotics · Computer Science 2020-06-09 Ian Abraham , Alexander Broad , Allison Pinosky , Brenna Argall , Todd D. Murphey

Despite their cost, randomized controlled trials (RCTs) are widely regarded as gold-standard evidence in disciplines ranging from social science to medicine. In recent decades, researchers have increasingly sought to reduce the resource…

Methodology · Statistics 2026-02-24 Guilherme Duarte

External control arms can inform early clinical development of experimental drugs and provide efficacy evidence for regulatory approval. However, accessing sufficient real-world or historical clinical trials data is challenging. Indeed,…

Randomised controlled trials (RCTs) are regarded as the gold standard for estimating causal treatment effects on health outcomes. However, RCTs are not always feasible, because of time, budget or ethical constraints. Observational data such…

Methodology · Statistics 2024-02-20 Li Su , Roonak Rezvani , Shaun R. Seaman , Colin Starr , Isaac Gravestock

Participation incentives is a well-known issue inhibiting randomized controlled trials (RCTs) in medicine, as well as a potential cause of user dissatisfaction for RCTs in online platforms. We frame this issue as a non-standard…

Computer Science and Game Theory · Computer Science 2026-01-13 Yingkai Li , Aleksandrs Slivkins

Indirect experiments provide a valuable framework for estimating treatment effects in situations where conducting randomized control trials (RCTs) is impractical or unethical. Unlike RCTs, indirect experiments estimate treatment effects by…

Machine Learning · Computer Science 2023-12-06 Yash Chandak , Shiv Shankar , Vasilis Syrgkanis , Emma Brunskill

Augmenting randomized controlled trials (RCTs) with external real-world data (RWD) has the potential to improve the finite sample efficiency of treatment effect estimators. We describe using adaptive targeted maximum likelihood estimation…

Methodology · Statistics 2025-01-30 Sky Qiu , Jens Tarp , Andrew Mertens , Mark van der Laan

Machine learning models are susceptible to being misled by biases in training data that emphasize incidental correlations over the intended learning task. In this study, we demonstrate the impact of data bias on the performance of a machine…

Materials Science · Physics 2024-12-11 Ali Davariashtiyani , Busheng Wang , Samad Hajinazar , Eva Zurek , Sara Kadkhodaei

Conjoint analysis is a popular experimental design used to measure multidimensional preferences. Researchers examine how varying a factor of interest, while controlling for other relevant factors, influences decision-making. Currently,…

Methodology · Statistics 2024-11-20 Dae Woong Ham , Kosuke Imai , Lucas Janson

There is increasing interest in combining information from experimental studies, including randomized and single-group trials, with information from external experimental or observational data sources. Such efforts are usually motivated by…

Methodology · Statistics 2026-04-23 Lawson Ung , Guanbo Wang , Sebastien Haneuse , Miguel A. Hernán , Issa J. Dahabreh

Conditional independence (CI) testing is a fundamental and challenging task in modern statistics and machine learning. Many modern methods for CI testing rely on powerful supervised learning methods to learn regression functions or Bayes…

Machine Learning · Statistics 2023-10-31 Felipe Maia Polo , Yuekai Sun , Moulinath Banerjee

Heteroskedasticity poses several methodological challenges in designing valid and powerful procedures for simultaneous testing of composite null hypotheses. In particular, the conventional practice of standardizing or re-scaling…

Methodology · Statistics 2025-09-04 Bowen Gang , Trambak Banerjee

Most clinical trials involve the comparison of a new treatment to a control arm (e.g., the standard of care) and the estimation of a treatment effect. External data, including historical clinical trial data and real-world observational…

Methodology · Statistics 2021-03-17 Tianjian Zhou , Yuan Ji

Population-adjusted indirect comparisons estimate treatment effects when access to individual patient data is limited and there are cross-trial differences in effect modifiers. Popular methods include matching-adjusted indirect comparison…

Applications · Statistics 2021-11-05 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

This paper reports on the challenges and lessons we learned while running controlled experiments in crowdsourcing platforms. Crowdsourcing is becoming an attractive technique to engage a diverse and large pool of subjects in experimental…

Human-Computer Interaction · Computer Science 2020-11-06 Jorge Ramírez , Marcos Baez , Fabio Casati , Luca Cernuzzi , Boualem Benatallah

Randomized controlled trials (RCTs) are indispensable for establishing the clinical value of medical artificial-intelligence (AI) tools, yet their high cost and long timelines hinder timely validation as new models emerge rapidly. Here, we…

Machine Learning · Computer Science 2025-11-17 Michael Nercessian , Wenxin Zhang , Alexander Schubert , Daphne Yang , Maggie Chung , Ahmed Alaa , Adam Yala

In this study, we focus on estimating the heterogeneous treatment effect (HTE) for survival outcome. The outcome is subject to censoring and the number of covariates is high-dimensional. We utilize data from both the randomized controlled…

Methodology · Statistics 2025-03-21 Xin Ye , Shu Yang , Xiaofei Wang , Yanyan Liu