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Hybrid randomized controlled trials (hybrid RCTs) integrate external control data, such as historical or concurrent data, with data from randomized trials. While numerous frequentist and Bayesian methods, such as the test-then-pool and…

Methodology · Statistics 2025-10-07 Han Chang Chiam , Franz König , Martin Posch

The importance of uncertainty quantification is increasingly recognized in the diverse field of machine learning. Accurately assessing model prediction uncertainty can help provide deeper understanding and confidence for researchers and…

Machine Learning · Computer Science 2024-12-03 Tianyi Chen , Yingzhou Lu , Nan Hao , Yuanyuan Zhang , Capucine Van Rechem , Jintai Chen , Tianfan Fu

Randomized controlled trials (RCTs) are considered as the gold standard for testing causal hypotheses in the clinical domain. However, the investigation of prognostic variables of patient outcome in a hypothesized cause-effect route is not…

Randomization inference (RI) is typically interpreted as testing Fisher's "sharp" null hypothesis that all unit-level effects are exactly zero. This hypothesis is often criticized as restrictive and implausible, making its rejection…

Methodology · Statistics 2023-08-29 Devin Caughey , Allan Dafoe , Xinran Li , Luke Miratrix

Technology companies are increasingly using randomized controlled trials (RCTs) as part of their development process. Despite having fine control over engineering systems and data instrumentation, these RCTs can still be imperfectly…

Software Engineering · Computer Science 2022-09-05 Jeffrey Wong , Jasmine Nettiksimmons , Jiannan Lu , Katherine Livins

Causal inference is vital for informed decision-making across fields such as biomedical research and social sciences. Randomized controlled trials (RCTs) are considered the gold standard for internal validity of inferences, whereas…

Methodology · Statistics 2025-12-02 Ruoqi Yu , Bikram Karmakar , Jessica Vandeleest , Eleanor Bimla Schwarz

Observational studies provide the only evidence on the effectiveness of interventions when randomized controlled trials (RCTs) are impractical due to cost, ethical concerns, or time constraints. While many methodologies aim to draw causal…

While deep learning models often achieve high predictive accuracy, their predictions typically do not come with any provable guarantees on risk or reliability, which are critical for deployment in high-stakes applications. The framework of…

Machine Learning · Computer Science 2025-10-13 Christopher Yeh , Nicolas Christianson , Adam Wierman , Yisong Yue

Observational healthcare data offer the potential to estimate causal effects of medical products on a large scale. However, the confidence intervals and p-values produced by observational studies only account for random error and fail to…

Applications · Statistics 2024-05-02 Jami J. Mulgrave , David Madigan , George Hripcsak

In a randomised clinical trial, when the result of the primary endpoint shows a significant benefit, the secondary endpoints are scrutinised to identify additional effects of the treatment. However, this approach entails a risk of…

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 work, we proposed a novel inferential procedure assisted by machine learning based adjustment for randomized control trials. The method was developed under the Rosenbaum's framework of exact tests in randomized experiments with…

Methodology · Statistics 2024-07-23 Han Yu , Alan D. Hutson , Xiaoyi Ma

We propose a restricted win probability estimand for comparing treatments in a randomized trial with a time-to-event outcome. We also propose Bayesian estimators for this summary measure as well as the unrestricted win probability. Bayesian…

Methodology · Statistics 2024-11-06 Michelle Leeberg , Xianghua Luo , Thomas A. Murray

Randomized control trials (RCTs) are the gold standard for estimating causal effects, but often use samples that are non-representative of the actual population of interest. We propose a reweighting method for estimating population average…

Methodology · Statistics 2022-02-09 Kellie Ottoboni , Jason Poulos

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…

Methodology · Statistics 2019-11-20 Eleanor J. Murray , Sonja A. Swanson , Miguel A. Hernán

Objectives To develop and validate a deep learning-based diagnostic model incorporating uncertainty estimation so as to facilitate radiologists in the preoperative differentiation of the pathological subtypes of renal cell carcinoma (RCC)…

Image and Video Processing · Electrical Eng. & Systems 2023-11-14 Ni Yao , Hang Hu , Kaicong Chen , Chen Zhao , Yuan Guo , Boya Li , Jiaofen Nan , Yanting Li , Chuang Han , Fubao Zhu , Weihua Zhou , Li Tian

Causal inferences from a randomized controlled trial (RCT) may not pertain to a target population where some effect modifiers have a different distribution. Prior work studies generalizing the results of a trial to a target population with…

Machine Learning · Statistics 2024-06-06 Ilker Demirel , Ahmed Alaa , Anthony Philippakis , David Sontag

Air Traffic Control (ATC) is a safety-critical domain in which incorrect interpretation of instructions may lead to severe operational consequences. While large language models (LLMs) demonstrate strong general performance, their…

Computation and Language · Computer Science 2026-05-13 Yujing Chang , Yash Guleria , Duc-Thinh Pham , Nhut-Huy Pham , Ningli Wang , Vu N. Duong , Sameer Alam

A C++ class was written for the calculation of frequentist confidence intervals using the profile likelihood method. Seven combinations of Binomial, Gaussian, Poissonian and Binomial uncertainties are implemented. The package provides…

Data Analysis, Statistics and Probability · Physics 2010-01-21 J. Lundberg , J. Conrad , W. Rolke , A. Lopez

Randomized controlled trials (RCTs) yield internally valid causal effect estimates, but generalizing these results to target populations with different characteristics requires an untestable selection ignorability assumption: conditional on…

Methodology · Statistics 2026-03-31 Amir Asiaee , Samhita Pal , Jared D. Huling
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