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In the context of individual-level causal inference, we study the problem of predicting whether someone will respond or not to a treatment based on their features and past examples of features, treatment indicator (e.g., drug/no drug), and…

Machine Learning · Statistics 2019-06-04 Nathan Kallus

Randomized trials are considered the gold standard for making informed decisions in medicine, yet they often lack generalizability to the patient populations in clinical practice. Observational studies, on the other hand, cover a broader…

Methodology · Statistics 2026-04-14 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

In observational studies with time-to-event outcomes, the g-formula can be used to estimate a treatment effect in the presence of confounding factors. However, the asymptotic distribution of the corresponding stochastic process is…

Statistics Theory · Mathematics 2024-04-26 Jasmin Rühl , Sarah Friedrich

The difference-in-differences (DID) research design is a key identification strategy which allows researchers to estimate causal effects under the parallel trends assumption. While the parallel trends assumption is counterfactual and cannot…

Methodology · Statistics 2026-05-12 Jonas M. Mikhaeil , Christopher Harshaw

This paper provides asymptotically valid tests for the null hypothesis of no treatment effect heterogeneity. Importantly, I consider the presence of heterogeneity that is not explained by observed characteristics, or so-called idiosyncratic…

Econometrics · Economics 2023-04-04 Jaime Ramirez-Cuellar

There are very different statistical methods for demonstrating a trend in pharmacological experiments. Here, the focus is on sparse models with only one parameter to be estimated and interpreted: the increase in the regression model and the…

Applications · Statistics 2020-07-21 Ludwig A. Hothorn

Medical image diagnosis is challenging because many diseases resemble normal anatomy and exhibit substantial interpatient variability. Clinicians routinely rely on comparative diagnosis, such as referencing cross-patient healthy control…

Computer Vision and Pattern Recognition · Computer Science 2026-02-24 Ruinan Jin , Gexin Huang , Xinwei Shen , Qiong Zhang , Yan Shuo Tan , Xiaoxiao Li

In an interlaboratory key comparison, a data analysis procedure for this comparison was proposed and recommended by CIPM [1, 2, 3], therein the degrees of equivalence of measurement standards of the laboratories participated in the…

Other Statistics · Statistics 2011-12-14 Thang H. Le , Nguyen D. Do

In this paper, we propose a new approach to causal inference with panel data. Instead of using panel data to adjust for differences in the distribution of unobserved heterogeneity between the treated and comparison groups, we instead use…

Econometrics · Economics 2025-12-01 Brantly Callaway , Derek Dyal , Pedro H. C. Sant'Anna , Emmanuel S. Tsyawo

Recently, there has been great interest in estimating the conditional average treatment effect using flexible machine learning methods. However, in practice, investigators often have working hypotheses about effect heterogeneity across…

Methodology · Statistics 2023-09-13 Chan Park , Hyunseung Kang

We discovered secular trend bias in a drug effectiveness study for a recently approved drug. We compared treatment outcomes between patients who received the newly approved drug and patients exposed to the standard treatment. All patients…

Machine Learning · Computer Science 2018-08-21 Sharon Hensley Alford , Piyush Madan , Shilpa Mahatma , Italo Buleje , Yanyan Han , Fang Lu

We consider a general difference-in-differences model in which the treatment variable of interest may be non-binary and its value may change in each period. It is generally difficult to estimate treatment parameters defined with the…

Econometrics · Economics 2023-05-30 Takahide Yanagi

In recent years there have been a lot of interest to test for similarity between biological drug products, commonly known as biologics. Biologics are large and complex molecule drugs that are produced by living cells and hence these are…

Methodology · Statistics 2019-02-21 Lin Dong , Sujit K. Ghosh

Drug repurposing has attracted increasing attention from both the pharmaceutical industry and the research community. Many existing computational drug repurposing methods rely on preclinical data (e.g., chemical structures, drug targets),…

Quantitative Methods · Quantitative Biology 2020-07-28 Qianlong Wen , Ruoqi Liu , Ping Zhang

This paper proposes a novel approach for estimating treatment effects in panel data settings, addressing key limitations of the standard difference-in-differences (DID) approach. The standard approach relies on the parallel trends…

Econometrics · Economics 2026-01-14 Shoya Ishimaru

We present MedSim, a novel semantic SIMilarity method based on public well-established bio-MEDical knowledge graphs (KGs) and large-scale corpus, to study the therapeutic substitution of antibiotics. Besides hierarchy and corpus of KGs,…

Computation and Language · Computer Science 2018-12-06 Kai Lei , Kaiqi Yuan , Qiang Zhang , Ying Shen

Comparison and contrast are the basic means to unveil causation and learn which treatments work. To build good comparison groups, randomized experimentation is key, yet often infeasible. In such non-experimental settings, we illustrate and…

Methodology · Statistics 2024-01-30 Ambarish Chattopadhyay , Jose R. Zubizarreta

Difference-in-differences (DiD) is the most popular observational causal inference method in health policy, employed to evaluate the real-world impact of policies and programs. To estimate treatment effects, DiD relies on the "parallel…

Applications · Statistics 2024-08-09 Shuo Feng , Ishani Ganguli , Youjin Lee , John Poe , Andrew Ryan , Alyssa Bilinski

The best evidence concerning comparative treatment effectiveness comes from clinical trials, the results of which are reported in unstructured articles. Medical experts must manually extract information from articles to inform…

Computation and Language · Computer Science 2022-01-11 Benjamin E. Nye , Jay DeYoung , Eric Lehman , Ani Nenkova , Iain J. Marshall , Byron C. Wallace

Researchers are increasingly turning to machine learning (ML) algorithms to investigate causal heterogeneity in randomized experiments. Despite their promise, ML algorithms may fail to accurately ascertain heterogeneous treatment effects…

Methodology · Statistics 2024-04-23 Kosuke Imai , Michael Lingzhi Li