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This paper presents methods to study the causal effect of a binary treatment on a functional outcome with observational data. We define a Functional Average Treatment Effect and develop an outcome regression estimator. We show how to obtain…

Methodology · Statistics 2025-09-08 Kreske Ecker , Xavier de Luna , Lina Schelin

Externally controlled trials (ECTs) are increasingly used when randomized controls are infeasible, unethical, or insufficient, including applications in rare diseases, oncology, pediatrics, and post-approval effectiveness research. Although…

A new method is proposed to explore sources of cross-site impact variance in multi-site trials of social interventions. With this approach, aggregate reports from participants in the treatment arm about the treatment experience are used to…

Applications · Statistics 2021-12-03 David R. Judkins , Gabriel Durham

Integrative analysis of data from multiple sources is critical to making generalizable discoveries. Associations that are consistently observed across multiple source populations are more likely to be generalized to target populations with…

Methodology · Statistics 2022-12-19 Zijian Guo

Estimating causal effects from observational data requires identifying valid adjustment sets. This task is especially challenging in realistic settings where latent confounding and feedback loops are present. Existing approaches typically…

Machine Learning · Computer Science 2026-05-08 Ana Leticia Garcez Vicente , Gijs van Seeventer , Saber Salehkaleybar

This paper introduces a framework for incorporating prior information into the design of sequential experiments. These sources may include past experiments, expert opinions, or the experimenter's intuition. We model the problem using a…

Econometrics · Economics 2024-10-01 Frederico Finan , Demian Pouzo

We extend Fisher's randomization test (FRT) to test conditional independence between observed outcomes and treatments given covariates in both randomized experiments and observational studies, with no restriction on the variable type of…

Methodology · Statistics 2025-06-12 Zhen Zhong

External validation is often recommended to ensure the generalizability of ML models. However, it neither guarantees generalizability nor equates to a model's clinical usefulness (the ultimate goal of any clinical decision-support tool).…

Machine Learning · Computer Science 2023-05-16 Alex Youssef , Michael Pencina , Anshul Thakur , Tingting Zhu , David Clifton , Nigam H. Shah

Sibling fixed effects (FE) models are useful for estimating causal treatment effects while offsetting unobserved sibling-invariant confounding. However, treatment estimates are biased if an individual's outcome affects their sibling's…

Methodology · Statistics 2021-11-09 David C. Mallinson

Identification of causal effects is one of the most fundamental tasks of causal inference. We consider an identifiability problem where some experimental and observational data are available but neither data alone is sufficient for the…

Artificial Intelligence · Computer Science 2019-03-13 Santtu Tikka , Juha Karvanen

This paper extends the work of Clarke [1] on the Bayesian foundations of the biomagnetic inverse problem. It derives expressions for the expectation and variance of the a posteriori source current probability distribution given a prior…

Medical Physics · Physics 2009-10-31 R. Hasson , S. J. Swithenby

Age-specific fertility rates (ASFRs) provide the most extensive record of reproductive change, but their aggregate nature obscures the individual-level behavioral mechanisms that drive fertility trends. To bridge this micro-macro divide, we…

Applications · Statistics 2025-07-25 Daniel Ciganda , Ignacio Campón , Iñaki Permanyer , Jakob H Macke

Enhancing the external validity of trial results is essential for their applicability to real-world populations. However, violations of the positivity assumption can limit both the generalizability and transportability of findings. To…

Methodology · Statistics 2025-04-22 Jun Lu , Sanjib Basu

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

We consider large-scale studies in which it is of interest to test a very large number of hypotheses, and then to estimate the effect sizes corresponding to the rejected hypotheses. For instance, this setting arises in the analysis of gene…

Methodology · Statistics 2015-03-31 Kean Ming Tan , Noah Simon , Daniela Witten

Generalizability and transportability methods have been proposed to address the external validity bias of randomized clinical trials that results from differences in the distribution of treatment effect modifiers between trial and target…

Large-scale replication studies like the Reproducibility Project: Psychology (RP:P) provide invaluable systematic data on scientific replicability, but most analyses and interpretations of the data fail to agree on the definition of…

Methodology · Statistics 2022-03-08 Kenneth Hung , William Fithian

Background: Animals from the same litter are often more alike compared with animals from different litters. This litter-to-litter variation, or "litter effects", can influence the results in addition to the experimental factors of interest.…

Quantitative Methods · Quantitative Biology 2013-03-25 Stanley E. Lazic , Laurent Essioux

We establish concentration rates for estimation of treatment effects in experiments that incorporate prior sources of information -- such as past pilots, related studies, or expert assessments -- whose external validity is uncertain. Each…

Econometrics · Economics 2026-03-24 Frederico Finan , Demian Pouzo

Despite their importance in supporting experimental conclusions, standard statistical tests are often inadequate for research areas, like the life sciences, where the typical sample size is small and the test assumptions difficult to…

Methodology · Statistics 2011-04-15 Pietro Berkes , Jozsef Fiser
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