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The growing availability of large health databases has expanded the use of observational studies for comparative effectiveness research. Unlike randomized trials, observational studies must adjust for systematic differences in patient…

Methodology · Statistics 2026-01-21 Haidong Lu , Fan Li , Laine E. Thomas , Fan Li

In recent years, precision treatment strategy have gained significant attention in medical research, particularly for patient care. We propose a novel framework for estimating conditional average treatment effects (CATE) in time-to-event…

Methodology · Statistics 2024-07-29 Runjia Li , Victor B. Talisa , Chung-Chou H. Chang

The rapid advancement of large language models (LLMs) has accelerated their integration into clinical decision support, particularly in prescription review. To enable systematic and fine-grained evaluation, we developed RxBench, a…

Computation and Language · Computer Science 2025-12-03 Yan Yang , Mouxiao Bian , Peiling Li , Bingjian Wen , Ruiyao Chen , Kangkun Mao , Xiaojun Ye , Tianbin Li , Pengcheng Chen , Bing Han , Jie Xu , Kaifeng Qiu , Junyan Wu

Under what circumstances is it a threat to the parallel trends assumption required for Difference in Differences (DiD) studies if treatment decisions are based on past values of the outcome? We explore via simulation studies whether…

Methodology · Statistics 2022-08-02 Zach Shahn

Applied Difference-in-Differences studies often involve outcomes that are discrete, mixed, censored, or otherwise non-continuously distributed, while policy questions frequently concern distributional effects rather than mean effects alone.…

Econometrics · Economics 2026-05-22 Nelly K. Djuazon , Emmanuel Selorm Tsyawo

The dearth of prescribing guidelines for physicians is one key driver of the current opioid epidemic in the United States. In this work, we analyze medical and pharmaceutical claims data to draw insights on characteristics of patients who…

Machine Learning · Computer Science 2020-05-01 Chirag Nagpal , Dennis Wei , Bhanukiran Vinzamuri , Monica Shekhar , Sara E. Berger , Subhro Das , Kush R. Varshney

Bridging the gap between internal and external validity is crucial for heterogeneous treatment effect estimation. Randomised controlled trials (RCTs), favoured for their internal validity due to randomisation, often encounter challenges in…

The difference-in-differences (DID) design is widely used in observational studies to estimate the causal effect of a treatment when repeated observations over time are available. Yet, almost all existing methods assume linearity in the…

Applications · Statistics 2020-09-29 Soichiro Yamauchi

Biomarker measurements can be relatively easy and quick to obtain and they are useful to investigate whether a compound works as intended on a mechanistic, pharmacological level. In some situations, it is realistic to assume that patients,…

Methodology · Statistics 2018-06-26 Björn Bornkamp , Georgina Bermann

There is a growing interest in using a longitudinal observational databases to detect drug safety signal. In this paper we present a novel method, which we used online during the OMOP Cup. We consider homogeneous ensembling, which is based…

Machine Learning · Statistics 2011-10-05 Vladimir Nikulin

Marginal structural models are a popular tool for investigating the effects of time-varying treatments, but they require an assumption of no unobserved confounders between the treatment and outcome. With observational data, this assumption…

Methodology · Statistics 2021-06-10 Matthew Blackwell , Soichiro Yamauchi

Machine learning for data-driven diagnosis has been actively studied in medicine to provide better healthcare. Supporting analysis of a patient cohort similar to a patient under treatment is a key task for clinicians to make decisions with…

Medical Physics · Physics 2020-03-25 Rongchen Guo , Takanori Fujiwara , Yiran Li , Kelly M. Lima , Soman Sen , Nam K. Tran , Kwan-Liu Ma

We develop a method for assigning high-quality labels to unstructured text. This method is based on fine-tuning an efficient, open-source language model with data extracted from a large, proprietary language model. We apply this method to…

General Economics · Economics 2025-01-27 Maya M. Durvasula , Sabri Eyuboglu , David M. Ritzwoller

Qualitative interactions occur when a treatment effect or measure of association varies in sign by sub-population. Of particular interest in many biomedical settings are absence/presence qualitative interactions, which occur when an effect…

Methodology · Statistics 2020-10-20 Aaron Hudson , Ali Shojaie

The study of competition among brands in a common category is an interesting strategic issue for involved firms. Sales monitoring and prediction of competitors' performance represent relevant tools for management. In the pharmaceutical…

Applications · Statistics 2016-02-10 Renato Guseo , Cinzia Mortarino

Concerns over reproducibility in science extend to research using existing healthcare data; many observational studies investigating the same topic produce conflicting results, even when using the same data. To address this problem, we…

Applications · Statistics 2018-03-30 Martijn J. Schuemie , Patrick B. Ryan , George Hripcsak , David Madigan , Marc A. Suchard

Causal inference in a program evaluation setting faces the problem of external validity when the treatment effect in the target population is different from the treatment effect identified from the population of which the sample is…

Methodology · Statistics 2021-12-23 Kyungchul Song , Zhengfei Yu

Clinical trials assessing neurological treatment are challenging due to the diversity of brain function, and the difficulty in quantifying it. Traditional treatment studies in epilepsy use seizure frequency as the primary outcome measure,…

Applications · Statistics 2025-12-02 Ian Miller , Ann Hyslop , Colin Decker

Prescription opioids relieve moderate-to-severe pain after surgery, but overprescription can lead to misuse and overdose. Understanding factors associated with post-surgical opioid refills is crucial for improving pain management and…

Methodology · Statistics 2025-09-16 Eileen Yang , Donglin Zeng , Mark Bicket , Yi Li

There is a growing trend of applying machine learning methods to medical datasets in order to predict patients' future status. Although some of these methods achieve high performance, challenges still exist in comparing and evaluating…

Medical Physics · Physics 2020-03-25 Yiran Li , Takanori Fujiwara , Yong K. Choi , Katherine K. Kim , Kwan-Liu Ma
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