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The comparison of different medical treatments from observational studies or across different clinical studies is often biased by confounding factors such as systematic differences in patient demographics or in the inclusion criteria for…

Methodology · Statistics 2025-05-15 Ekkehard Glimm , Lillian Yau

While Large Language Models (LLMs) achieve superhuman performance on standardized medical licensing exams, these static benchmarks have become saturated and increasingly disconnected from the functional requirements of clinical workflows.…

The ability to predict individualized treatment effects (ITEs) based on a given patient's profile is essential for personalized medicine. We propose a hypothesis testing approach to choosing between two potential treatments for a given…

Methodology · Statistics 2020-08-11 Tianxi Cai , Tony Cai , Zijian Guo

Recent work has focused on nonparametric estimation of conditional treatment effects, but inference has remained relatively unexplored. We propose a class of nonparametric tests for both quantitative and qualitative treatment effect…

Methodology · Statistics 2026-04-07 Oliver Dukes , Mats J. Stensrud , Riccardo Brioschi , Aaron Hudson

Autoregressive models can predict clinical events, but generating patient-conditioned multi-step trajectories that respond to intervention tokens and testing whether those responses preserve known pharmacological associations has received…

Machine Learning · Computer Science 2026-05-07 Chris Sainsbury , Feng Dong , Andreas Karwath

This paper analyzes difference-in-differences designs with a continuous treatment. We show that treatment-on-the-treated-type parameters are identified under a parallel trends assumption analogous to the binary treatment case. However,…

Econometrics · Economics 2026-01-05 Brantly Callaway , Andrew Goodman-Bacon , Pedro H. C. Sant'Anna

Currently, approximately 30% of epileptic patients treated with antiepileptic drugs (AEDs) remain resistant to treatment (known as refractory patients). This project seeks to understand the underlying similarities in refractory patients vs.…

Machine Learning · Computer Science 2017-04-28 David Von Dollen

In comparative effectiveness research, treated and control patients might have a different start of follow-up as treatment is often started later in the disease trajectory. This typically occurs when data from treated and controls are not…

Methodology · Statistics 2024-06-10 Rik van Eekelen , Patrick M. M. Bossuyt , Nan van Geloven

This paper studies identification of average treatment effects in a panel data setting. It introduces a novel nonparametric factor model and proves identification of average treatment effects. The identification proof is based on the…

Econometrics · Economics 2025-03-26 Susan Athey , Guido Imbens

Difference-in-differences is based on a parallel trends assumption, which states that changes over time in average potential outcomes are independent of treatment assignment, possibly conditional on covariates. With time-varying treatments,…

Methodology · Statistics 2024-06-25 Nicholas Illenberger , Iván Díaz , Audrey Renson

The creation of the ICH E9 (R1) estimands framework has led to more precise specification of the treatment effects of interest in the design and statistical analysis of clinical trials. However, it is unclear how the new framework relates…

Methodology · Statistics 2024-12-20 Thomas Drury , Jonathan W. Bartlett , David Wright , Oliver N. Keene

Quantile and quantile effect functions are important tools for descriptive and causal analyses due to their natural and intuitive interpretation. Existing inference methods for these functions do not apply to discrete random variables. This…

Methodology · Statistics 2018-09-03 Victor Chernozhukov , Iván Fernández-Val , Blaise Melly , Kaspar Wüthrich

We introduce an expert curated, real-world benchmark for evaluating document-grounded question-answering (QA) motivated by generic drug assessment, using the U.S. Food and Drug Administration (FDA) drug label documents. Drug labels contain…

Computation and Language · Computer Science 2026-03-23 Betty Xiong , Jillian Fisher , Benjamin Newman , Meng Hu , Shivangi Gupta , Yejin Choi , Lanyan Fang , Russ B Altman

A common problem in clinical trials is to test whether the effect of an explanatory variable on a response of interest is similar between two groups, e.g. patient or treatment groups. In this regard, similarity is defined as equivalence up…

Methodology · Statistics 2024-01-12 Niklas Hagemann , Giampiero Marra , Frank Bretz , Kathrin Möllenhoff

Tech companies (e.g., Google or Facebook) often use randomized online experiments and/or A/B testing primarily based on the average treatment effects to compare their new product with an old one. However, it is also critically important to…

Methodology · Statistics 2021-11-09 Chengchun Shi , Shikai Luo , Hongtu Zhu , Rui Song

Specialized clinical AI assistants are rapidly entering medical practice, often framed as safer or more reliable than general-purpose large language models (LLMs). Yet, unlike frontier models, these clinical tools are rarely subjected to…

Computation and Language · Computer Science 2025-12-02 Krithik Vishwanath , Mrigayu Ghosh , Anton Alyakin , Daniel Alexander Alber , Yindalon Aphinyanaphongs , Eric Karl Oermann

Most causal inference methods consider counterfactual variables under interventions that set the treatment deterministically. With continuous or multi-valued treatments or exposures, such counterfactuals may be of little practical interest…

Methodology · Statistics 2021-07-07 Iván Díaz , Nicholas Williams , Katherine L. Hoffman , Edward J. Schenck

This technical report introduces a Named Clinical Entity Recognition Benchmark for evaluating language models in healthcare, addressing the crucial natural language processing (NLP) task of extracting structured information from clinical…

In many medical and business applications, researchers are interested in estimating individualized treatment effects using data from a randomized experiment. For example in medical applications, doctors learn the treatment effects from…

Methodology · Statistics 2022-03-01 Kevin Wu Han , Han Wu

This paper considers the inference of trends in multiple, nonstationary time series. To test whether trends are parallel to each other, we use a parallelism index based on the L2-distances between nonparametric trend estimators and their…

Methodology · Statistics 2015-03-17 David Degras , Zhiwei Xu , Ting Zhang , Wei Biao Wu