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Developing drugs for rare diseases presents unique challenges from a statistical perspective. These challenges may include slowly progressive diseases with unmet medical needs, poorly understood natural history, small population size,…

Rare diseases affect a relatively small number of people, which limits investment in research for treatments and cures. Developing an efficient method for rare disease detection is a crucial first step towards subsequent clinical research.…

Machine Learning · Computer Science 2018-12-04 Wenyuan Li , Yunlong Wang , Yong Cai , Corey Arnold , Emily Zhao , Yilian Yuan

When developing clinical prediction models, it can be challenging to balance between global models that are valid for all patients and personalized models tailored to individuals or potentially unknown subgroups. To aid such decisions, we…

Pharmaceutical targeting is one of key inputs for making sales and marketing strategy planning. Targeting list is built on predicting physician's sales potential of certain type of patient. In this paper, we present a time-sensitive…

Machine Learning · Computer Science 2018-12-04 Weiyu Huang , Yunlong Wang , Li Zhou , Emily Zhao , Yilian Yuan , Alejandro Ribero

Accurate and explainable health event predictions are becoming crucial for healthcare providers to develop care plans for patients. The availability of electronic health records (EHR) has enabled machine learning advances in providing these…

Machine Learning · Computer Science 2021-05-18 Chang Lu , Chandan K. Reddy , Prithwish Chakraborty , Samantha Kleinberg , Yue Ning

Randomized controlled trials are not only the golden standard in medicine and vaccine trials but have spread to many other disciplines like behavioral economics, making it an important interdisciplinary tool for scientists. When designing…

Methodology · Statistics 2021-11-30 Tassilo Schwarz

Automated classification methods for disease diagnosis are currently in the limelight, especially for imaging data. Classification does not fully meet a clinician's needs, however: in order to combine the results of multiple tests and…

Quantitative Methods · Quantitative Biology 2018-05-07 PierGianLuca Porta Mana , Claudia Bachmann , Abigail Morrison

Benefiting from the powerful expressive capability of graphs, graph-based approaches have been popularly applied to handle multi-modal medical data and achieved impressive performance in various biomedical applications. For disease…

Machine Learning · Computer Science 2022-03-14 Shuai Zheng , Zhenfeng Zhu , Zhizhe Liu , Zhenyu Guo , Yang Liu , Yuchen Yang , Yao Zhao

Rare diseases affect hundreds of millions of people worldwide but are hard to detect since they have extremely low prevalence rates (varying from 1/1,000 to 1/200,000 patients) and are massively underdiagnosed. How do we reliably detect…

Machine Learning · Computer Science 2019-12-02 Limeng Cui , Siddharth Biswal , Lucas M. Glass , Greg Lever , Jimeng Sun , Cao Xiao

Few-shot graph classification aims at predicting classes for graphs, given limited labeled graphs for each class. To tackle the bottleneck of label scarcity, recent works propose to incorporate few-shot learning frameworks for fast…

Machine Learning · Computer Science 2022-05-10 Song Wang , Yushun Dong , Xiao Huang , Chen Chen , Jundong Li

A pharmacological effect of a drug on cells, organs and systems refers to the specific biochemical interaction produced by a drug substance, which is called its mechanism of action. Drug repositioning (or drug repurposing) is a fundamental…

Machine Learning · Computer Science 2020-05-19 Dehua Chen , Amir Jalilifard , Adriano Veloso , Nivio Ziviani

Healthcare artificial intelligence systems often degrade in performance when deployed across institutions, with documented performance drops and perpetuation of discriminatory patterns embedded in data. This brittleness comes, in part, from…

Machine Learning · Computer Science 2026-03-30 Munib Mesinovic , Max Buhlan , Tingting Zhu

Medication recommendation systems have gained significant attention in healthcare as a means of providing tailored and effective drug combinations based on patients' clinical information. However, existing approaches often suffer from…

Machine Learning · Computer Science 2024-08-13 Zihao Zhao , Yi Jing , Fuli Feng , Jiancan Wu , Chongming Gao , Xiangnan He

Precision medicine is an evolving area in the medical field and rely on biomarkers to make patient enrichment decisions, thereby providing drug development direction. A traditional statistical approach is to find the cut-off that leads to…

Methodology · Statistics 2025-04-14 Gina D'Angelo , Xiaowen Tian , Chuyu Deng , Xian Zhou

Recommender system research has oftentimes focused on approaches that operate on large-scale datasets containing millions of user interactions. However, many small businesses struggle to apply state-of-the-art models due to their very…

Outcome estimation of treatments for target individuals is an important foundation for decision making based on causal relations. Most existing outcome estimation methods deal with binary or multiple-choice treatments; however, in some…

Machine Learning · Computer Science 2021-09-14 Shonosuke Harada , Hisashi Kashima

Rare diseases are very difficult to identify among large number of other possible diagnoses. Better availability of patient data and improvement in machine learning algorithms empower us to tackle this problem computationally. In this…

Machine Learning · Computer Science 2016-09-07 Ravi Garg , Shu Dong , Sanjiv Shah , Siddhartha R Jonnalagadda

Rare diseases affecting 350 million individuals are commonly associated with delay in diagnosis or misdiagnosis. To improve those patients' outcome, rare disease detection is an important task for identifying patients with rare conditions…

Machine Learning · Computer Science 2019-07-03 Kezi Yu , Yunlong Wang , Yong Cai , Cao Xiao , Emily Zhao , Lucas Glass , Jimeng Sun

Benefiting from the powerful expressive capability of graphs, graph-based approaches have achieved impressive performance in various biomedical applications. Most existing methods tend to define the adjacency matrix among samples manually…

Machine Learning · Computer Science 2021-07-02 Shuai Zheng , Zhenfeng Zhu , Zhizhe Liu , Zhenyu Guo , Yang Liu , Yao Zhao

Rare diseases have extremely low-data regimes, unlike common diseases with large amount of available labeled data. Hence, to train a neural network to classify rare diseases with a few per-class data samples is very challenging, and so far,…

Computer Vision and Pattern Recognition · Computer Science 2020-07-16 Xiaomeng Li , Lequan Yu , Yueming Jin , Chi-Wing Fu , Lei Xing , Pheng-Ann Heng