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The randomized controlled trial (RCT) is the gold standard for estimating the average treatment effect (ATE) of a medical intervention but requires 100s-1000s of subjects, making it expensive and difficult to implement. While a cross-over…

Signal Processing · Electrical Eng. & Systems 2023-05-10 Sayeri Lala , Niraj K. Jha

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

Amidst rising appreciation for privacy and data usage rights, researchers have increasingly acknowledged the principle of data minimization, which holds that the accessibility, collection, and retention of subjects' data should be kept to…

Cryptography and Security · Computer Science 2021-10-22 Winston Chou

In the rapidly changing healthcare landscape, the implementation of offline reinforcement learning (RL) in dynamic treatment regimes (DTRs) presents a mix of unprecedented opportunities and challenges. This position paper offers a critical…

Machine Learning · Computer Science 2024-06-05 Zhiyao Luo , Yangchen Pan , Peter Watkinson , Tingting Zhu

Estimating optimal dynamic treatment regimes (DTRs) using observational data is often challenged by nonignorable missing covariates arsing from informative monitoring of patients in clinical practice. To address nonignorable missingness of…

Methodology · Statistics 2025-07-01 Jian Sun , Bo Fu , Li Su

Clinical Decision Support Systems (CDSSs) provide reasoning and inquiry guidance for physicians, yet they face notable challenges, including high maintenance costs and low generalization capability. Recently, Large Language Models (LLMs)…

Computation and Language · Computer Science 2026-04-24 Yue Guo , Fanfu Wang , Jianwei Lv , Xincheng Shi , Yuchen Li , Youya Wang , Yunsheng Zeng , Yujing Liu , Yunhao Qiao , Gen Li , Junfeng Wang , Bo Yuan

In this work, we present our various contributions to the objective of building a decision support tool for the diagnosis of rare diseases. Our goal is to achieve a state of knowledge where the uncertainty about the patient's disease is…

Drawing causal inferences from observational studies (OS) requires unverifiable validity assumptions; however, one can falsify those assumptions by benchmarking the OS with experimental data from a randomized controlled trial (RCT). A major…

Randomized controlled trials (RCTs) are widely regarded as the gold standard for causal inference in biomedical research. For instance, when estimating the average treatment effect on the treated (ATT), a doubly robust estimation procedure…

Methodology · Statistics 2025-09-26 Chi-Shian Dai , Chao Ying , Yang Ning , Jiwei Zhao

Randomized Controlled Trials (RCTs) are the gold standard for evaluating the effect of new medical treatments. Treatments must pass stringent regulatory conditions in order to be approved for widespread use, yet even after the regulatory…

Machine Learning · Statistics 2025-03-13 Omer Noy Klein , Alihan Hüyük , Ron Shamir , Uri Shalit , Mihaela van der Schaar

The Difference-in-Differences (DiD) method is a fundamental tool for causal inference, yet its application is often complicated by missing data. Although recent work has developed robust DiD estimators for complex settings like staggered…

Methodology · Statistics 2026-01-27 Lorenzo Testa , Edward H. Kennedy , Matthew Reimherr

Radiologists highly desire fully automated AI for radiology report generation (R2G), yet existing approaches fall short in clinical utility. Reinforcement learning (RL) holds potential to address these shortcomings, but its adoption in this…

Computer Vision and Pattern Recognition · Computer Science 2026-03-05 Zilin Lu , Ruifeng Yuan , Weiwei Cao , Wanxing Chang , Zhongyu Wei , Sinuo Wang , Yong Xia , Ling Zhang , Jianpeng Zhang

Recently, diabetic retinopathy (DR) screening utilizing ultra-wide optical coherence tomography angiography (UW-OCTA) has been used in clinical practices to detect signs of early DR. However, developing a deep learning-based DR analysis…

Image and Video Processing · Electrical Eng. & Systems 2022-10-19 Gitaek Kwon , Eunjin Kim , Sunho Kim , Seongwon Bak , Minsung Kim , Jaeyoung Kim

Randomized controlled trials (RCTs) have been the cornerstone of clinical evidence; however, their cost, duration, and restrictive eligibility criteria limit power and external validity. Studies using real-world data (RWD), historically…

Methodology · Statistics 2025-11-26 Shu Yang , Margaret Gamalo , Haoda Fu

In precision medicine, Dynamic Treatment Regimes (DTRs) are treatment protocols that adapt over time in response to a patient's observed characteristics. A DTR is a set of decision functions that takes an individual patient's information as…

Methodology · Statistics 2022-03-17 Cong Jiang , Michael Wallace , Mary Thompson

Time-series data with missing values are commonly encountered in many fields, such as healthcare, meteorology, and robotics. The imputation aims to fill the missing values with valid values. Most imputation methods trained the models…

Machine Learning · Computer Science 2024-11-21 Tae-Min Choi , Ji-Su Kang , Jong-Hwan Kim

We propose a reinforcement learning (RL)-based system that would automatically prescribe a hypothetical patient medication that may help the patient with their mental health-related speech disfluency, and adjust the medication and the…

Tremor is a key diagnostic feature of Parkinson's Disease (PD), Essential Tremor (ET), and other central nervous system (CNS) disorders. Clinicians or trained raters assess tremor severity with TETRAS scores by observing patients. Lacking…

Computer Vision and Pattern Recognition · Computer Science 2021-05-05 Li Zhang , Vijay Yadav , Vidya Koesmahargyo , Anzar Abbas , Isaac Galatzer-Levy

We developed a study design for rare disease clinical trials (RDTs) that efficiently evaluate treatments, promotes access to new treatments during treatment development, and optimizes healthcare resource utilization for future treatment…

Applications · Statistics 2016-07-04 Jian Yong , Sohaib H. Mohammad , Yan Yuan

The imputation of missing values in multivariate time series (MTS) data is critical in ensuring data quality and producing reliable data-driven predictive models. Apart from many statistical approaches, a few recent studies have proposed…

Machine Learning · Computer Science 2023-05-17 Maksims Kazijevs , Manar D. Samad