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Humans can be notoriously imperfect evaluators. They are often biased, unreliable, and unfit to define "ground truth." Yet, given the surging need to produce large amounts of training data in educational applications using AI, traditional…

Artificial Intelligence · Computer Science 2025-08-04 Danielle R. Thomas , Conrad Borchers , Kenneth R. Koedinger

Complementary features of randomized controlled trials (RCTs) and observational studies (OSs) can be used jointly to estimate the average treatment effect of a target population. We propose a calibration weighting estimator that enforces…

Methodology · Statistics 2022-02-16 Dasom Lee , Shu Yang , Lin Dong , Xiaofei Wang , Donglin Zeng , Jianwen Cai

AI models are often evaluated based on their ability to predict the outcome of interest. However, in many AI for social impact applications, the presence of an intervention that affects the outcome can bias the evaluation. Randomized…

Machine Learning · Computer Science 2025-11-18 Winston Chen , Michael W. Sjoding , Jenna Wiens

Randomized controlled trials (RCTs) often include subgroup analyses to assess whether treatment effects vary across pre-specified patient populations. However, these analyses frequently suffer from small sample sizes which limit the power…

Randomized trials are typically designed to detect average treatment effects but often lack the statistical power to uncover individual-level treatment effect heterogeneity, limiting their value for personalized decision-making. To address…

Machine Learning · Statistics 2026-03-19 Rickard Karlsson , Piersilvio De Bartolomeis , Issa J. Dahabreh , Jesse H. Krijthe

In this paper we present tools for applied researchers that re-purpose off-the-shelf methods from the computer-science field of machine learning to create a "discovery engine" for data from randomized controlled trials (RCTs). The applied…

Machine Learning · Statistics 2019-05-13 Jens Ludwig , Sendhil Mullainathan , Jann Spiess

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

Recent years have seen increasing use of supervised learning methods for segmentation tasks. However, the predictive performance of these algorithms depends on the quality of labels. This problem is particularly pertinent in the medical…

Computer Vision and Pattern Recognition · Computer Science 2020-10-26 Le Zhang , Ryutaro Tanno , Mou-Cheng Xu , Chen Jin , Joseph Jacob , Olga Ciccarelli , Frederik Barkhof , Daniel C. Alexander

Mean-based estimators of causal effects in randomized experiments may behave poorly if the potential outcomes have a heavy tail or contain outliers. An alternative estimator proposed by Rosenbaum (1993) estimates a constant additive…

Methodology · Statistics 2026-02-09 Aditya Ghosh , Nabarun Deb , Bikram Karmakar , Bodhisattva Sen

We propose a novel multi-task neural network approach for estimating distributional treatment effects (DTE) in randomized experiments. While DTE provides more granular insights into the experiment outcomes over conventional methods focusing…

Machine Learning · Computer Science 2025-07-11 Tomu Hirata , Undral Byambadalai , Tatsushi Oka , Shota Yasui , Shingo Uto

Platform trials are multi-arm designs that simultaneously evaluate multiple treatments for a single disease within the same overall trial structure. Unlike traditional randomized controlled trials, they allow treatment arms to enter and…

Methodology · Statistics 2024-12-16 Michele Santacatterina , Federico Macchiavelli Giron , Xinyi Zhang , Ivan Diaz

Randomized control trials (RCTs) have been the gold standard to evaluate the effectiveness of a program, policy, or treatment on an outcome of interest. However, many RCTs assume that study participants are willing to share their…

Applications · Statistics 2021-12-07 Manjusha Kancharla , Hyunseung Kang

In this paper we study the problems of estimating heterogeneity in causal effects in experimental or observational studies and conducting inference about the magnitude of the differences in treatment effects across subsets of the…

Machine Learning · Statistics 2022-06-08 Susan Athey , Guido Imbens

Subgroup analyses of randomized controlled trials (RCTs) constitute an important component of the drug development process in precision medicine. In particular, subgroup analyses of early-stage trials often influence the design and…

Methodology · Statistics 2025-02-12 Daniel Schwartz , Riddhiman Saha , Steffen Ventz , Lorenzo Trippa

Cost-Effectiveness Analyses (CEAs) alongside randomised controlled trials (RCTs) are increasingly often designed to collect resource use and preference-based health status data for the purpose of healthcare technology assessment. However,…

Applications · Statistics 2016-07-22 Andrea Gabrio , Alexina Mason , Gianluca Baio

The reliability of supervised machine learning systems depends on the accuracy and availability of ground truth labels. However, the process of human annotation, being prone to error, introduces the potential for noisy labels, which can…

Computer Vision and Pattern Recognition · Computer Science 2023-09-19 David Tschirschwitz , Christian Benz , Morris Florek , Henrik Norderhus , Benno Stein , Volker Rodehorst

[See paper for full abstract] Meta-analysis is a crucial tool for answering scientific questions. It is usually conducted on a relatively small amount of ``trusted'' data -- ideally from randomized, controlled trials -- which allow causal…

Machine Learning · Statistics 2024-07-15 Shiva Kaul , Geoffrey J. Gordon

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

While randomised controlled trials (RCTs) are the gold standard for estimating causal treatment effects, their limited sample sizes and restrictive criteria make it difficult to extrapolate to a broader population. Observational data, while…

Methodology · Statistics 2025-09-09 Stephanie Riley , Ricardo Silva , Matthew Sperrin

Randomized controlled trials (RCT's) allow researchers to estimate causal effects in an experimental sample with minimal identifying assumptions. However, to generalize or transport a causal effect from an RCT to a target population,…

Methodology · Statistics 2022-02-08 Melody Huang