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The COVID-19 pandemic has driven ever-greater demand for tools which enable efficient exploration of biomedical literature. Although semi-structured information resulting from concept recognition and detection of the defining elements of…

Computation and Language · Computer Science 2021-05-27 Simon Šuster , Karin Verspoor , Timothy Baldwin , Jey Han Lau , Antonio Jimeno Yepes , David Martinez , Yulia Otmakhova

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

In evidence-based medicine (EBM), defining a clinical question in terms of the specific patient problem aids the physicians to efficiently identify appropriate resources and search for the best available evidence for medical treatment. In…

Computation and Language · Computer Science 2019-11-12 Di Jin , Peter Szolovits

Data from observational studies (OSs) is widely available and readily obtainable yet frequently contains confounding biases. On the other hand, data derived from randomized controlled trials (RCTs) helps to reduce these biases; however, it…

Methodology · Statistics 2024-10-30 Dong Yao , Caizhi Tang , Qing Cui , Longfei Li

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

Objectives: With accurate estimates of expected safety results, clinical trials could be better designed and monitored. We evaluated methods for predicting serious adverse event (SAE) results in clinical trials using information only from…

Computation and Language · Computer Science 2025-08-12 Qixuan Hu , Xumou Zhang , Jinman Kim , Florence Bourgeois , Adam G. Dunn

We present a corpus of 5,000 richly annotated abstracts of medical articles describing clinical randomized controlled trials. Annotations include demarcations of text spans that describe the Patient population enrolled, the Interventions…

Computation and Language · Computer Science 2018-06-13 Benjamin Nye , Junyi Jessy Li , Roma Patel , Yinfei Yang , Iain J. Marshall , Ani Nenkova , Byron C. Wallace

Clinical trials offer a fundamental opportunity to discover new treatments and advance the medical knowledge. However, the uncertainty of the outcome of a trial can lead to unforeseen costs and setbacks. In this study, we propose a new…

Computation and Language · Computer Science 2022-04-04 Georgios Katsimpras , Georgios Paliouras

Covariate adjustment is widely recommended to improve statistical efficiency in randomized clinical trials (RCTs), yet empirical evidence comparing available strategies remains limited. This lack of real-world evaluation leaves unresolved…

Applications · Statistics 2026-02-03 Yulin Shao , Liangbo Lyu , Menggang Yu , Bingkai Wang

The efficacy of an intervention can be assessed by randomizing patients to different diagnostic tests instead of directly to an intervention and control. This principle is applied by allocating individuals to intervention if the test result…

Applications · Statistics 2022-01-11 Huw Llewelyn

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

Clinical trial outcome prediction seeks to estimate the likelihood that a clinical trial will successfully reach its intended endpoint. This process predominantly involves the development of machine learning models that utilize a variety of…

Biomolecules · Quantitative Biology 2024-05-14 Chufan Gao , Tianfan Fu , Jimeng Sun

Research is a continuous phenomenon. It is recursive in nature. Every research is based on some earlier research outcome. A general approach in reviewing the literature for a problem is to categorize earlier work for the same problem as…

Information Retrieval · Computer Science 2015-07-01 Bilal Hayat Butt , Muhammad Rafi , Arsal Jamal , Raja Sami Ur Rehman , Syed Muhammad Zubair Alam , Muhammad Bilal Alam

Randomized controlled trials (RCTs) often suffer from limited inferential efficiency in estimating treatment effects due to their small sample sizes. In recent years, incorporating external controls (ECs) has gained increasing attention as…

Methodology · Statistics 2026-04-16 Qinwei Yang , Jingyi Li , Peng Wu , Shu Yang

Covariate adjustment and methods of incorporating historical data in randomized clinical trials (RCTs) each provide opportunities to increase trial power. We unite these approaches for the analysis of RCTs with binary outcomes based on the…

Methodology · Statistics 2022-12-21 Alyssa M. Vanderbeek , Jessica L. Ross , David P. Miller , Alejandro Schuler

Background: The standard regulatory approach to assess replication success is the two-trials rule, requiring both the original and the replication study to be significant with effect estimates in the same direction. The sceptical p-value…

Methodology · Statistics 2025-05-01 Jeanette Köppe , Charlotte Micheloud , Stella Erdmann , Rachel Heyard , Leonhard Held

We present BEEP (Biomedical Evidence-Enhanced Predictions), a novel approach for clinical outcome prediction that retrieves patient-specific medical literature and incorporates it into predictive models. Based on each individual patient's…

Computation and Language · Computer Science 2022-11-17 Aakanksha Naik , Sravanthi Parasa , Sergey Feldman , Lucy Lu Wang , Tom Hope

Problem lists are intended to provide clinicians with a relevant summary of patient medical issues and are embedded in many electronic health record systems. Despite their importance, problem lists are often cluttered with resolved or…

Information Retrieval · Computer Science 2020-08-05 Justin Lovelace , Nathan C. Hurley , Adrian D. Haimovich , Bobak J. Mortazavi

The estimation of individual treatment effects (ITE) focuses on predicting the outcome changes that result from a change in treatment. A fundamental challenge in observational data is that while we need to infer outcome differences under…

Machine Learning · Computer Science 2025-12-23 Zichuan Lin , Xiaokai Huang , Jiate Liu , Yuxuan Han , Jia Chen , Xiapeng Wu , Deheng Ye

Electronic Health Record (EHR) data has been of tremendous utility in Artificial Intelligence (AI) for healthcare such as predicting future clinical events. These tasks, however, often come with many challenges when using classical machine…

Machine Learning · Computer Science 2021-04-08 Tingyi Wanyan , Jing Zhang , Ying Ding , Ariful Azad , Zhangyang Wang , Benjamin S Glicksberg