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While much of the causal inference literature has focused on addressing internal validity biases, both internal and external validity are necessary for unbiased estimates in a target population of interest. However, few generalizability…

Methodology · Statistics 2023-04-07 Irina Degtiar , Tim Layton , Jacob Wallace , Sherri Rose

With the advent of generative modeling techniques, synthetic data and its use has penetrated across various domains from unstructured data such as image, text to structured dataset modeling healthcare outcome, risk decisioning in financial…

Machine Learning · Computer Science 2021-05-11 Aman Gupta , Deepak Bhatt , Anubha Pandey

Randomized experiments play a major role in data-driven decision making across many different fields and disciplines. In medicine, for example, randomized controlled trials (RCTs) are the backbone of clinical trial methodology for testing…

Applications · Statistics 2016-08-30 Andrew W. Correia

Randomized controlled trials (RCTs) yield internally valid causal effect estimates, but generalizing these results to target populations with different characteristics requires an untestable selection ignorability assumption: conditional on…

Methodology · Statistics 2026-03-31 Amir Asiaee , Samhita Pal , Jared D. Huling

Factor analysis is over a century old, but it is still problematic to choose the number of factors for a given data set. The scree test is popular but subjective. The best performing objective methods are recommended on the basis of…

Methodology · Statistics 2015-11-12 A. B. Owen , J. Wang

Causal inference is vital for informed decision-making across fields such as biomedical research and social sciences. Randomized controlled trials (RCTs) are considered the gold standard for internal validity of inferences, whereas…

Methodology · Statistics 2025-12-02 Ruoqi Yu , Bikram Karmakar , Jessica Vandeleest , Eleanor Bimla Schwarz

Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform…

Machine Learning · Statistics 2018-10-22 Onur Atan , William R. Zame , Mihaela van der Schaar

Interim analyses are vital in clinical trials for early decision-making. While frequentist implications are well-established, the consequences of repeated Bayesian interim monitoring for efficacy, specifically regarding multiplicity, remain…

Methodology · Statistics 2025-12-30 Suyu Liu , Beibei Guo , Laura Thompson , Lei Nie , Ying Yuan

An individualized treatment rule (ITR) tailors treatments to a patient's specific characteristics. However, randomized controlled trials (RCTs) are often underpowered to detect the treatment effect heterogeneity needed for reliable ITR…

Methodology · Statistics 2026-04-14 Yuan Bian , Donglin Zeng , Hyun-Joon Yang , Leanne M. Williams , Yuanjia Wang

Randomized clinical trials are the gold standard when estimating the average treatment effect. However, they are usually not a random sample from the real-world population because of the inclusion/exclusion rules. Meanwhile, observational…

Methodology · Statistics 2024-12-11 Kuan Jiang , Wenjie Hu , Shu Yang , Xinxing Lai , Xiaohua Zhou

Familiar statistical tests and estimates are obtained by the direct observation of cases of interest: a clinical trial of a new drug, for instance, will compare the drug's effects on a relevant set of patients and controls. Sometimes,…

Methodology · Statistics 2010-12-09 Bradley Efron

This study examines the application of Bayesian approach in the context of clinical trials, emphasizing their increasing importance in contemporary biomedical research. While conventional frequentist approach provides a foundational basis…

Methodology · Statistics 2026-01-16 Paramahansa Pramanik , Arnab Kumar Maity , Anjan Mandal , Haley Kate Robinson

Use of historical control data to augment a small internal control arm in a randomized control trial (RCT) can lead to significant improvement of the efficiency of the trial. It introduces the risk of potential bias, since the historical…

Methodology · Statistics 2022-10-05 Jixian Wang , Hongtao Zhang , Ram Tiwari

Many macroeconomic policy questions may be assessed in a case study framework, where the time series of a treated unit is compared to a counterfactual constructed from a large pool of control units. I provide a general framework for this…

Econometrics · Economics 2018-03-02 Daniel Kinn

Objective: Randomized controlled trial (RCT) results often inform clinical decision-making, but the highly curated populations of trials and the care provided during the trial are often not reflective of real-world practice. The objective…

Applications · Statistics 2024-02-26 Guanbo Wang , Ting-Wei Ernie Liao , David Furfaro , Leo Anthony Celi , Kevin Sheng-Kai Ma

Incorporating historical data or real-world evidence has a great potential to improve the efficiency of phase I clinical trials and to accelerate drug development. For model-based designs, such as the continuous reassessment method (CRM),…

Methodology · Statistics 2020-06-11 Yanhong Zhou , J. Jack Lee , Shunguang Wang , Stuart Bailey , Ying Yuan

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the U.S. Food and Drug Administration recently issued guidance that emphasizes the importance of…

Methodology · Statistics 2023-06-12 Kelly Van Lancker , Frank Bretz , Oliver Dukes

Similarity choice data occur when humans make choices among alternatives based on their similarity to a target, e.g., in the context of information retrieval and in embedding learning settings. Classical metric-based models of similarity…

Machine Learning · Computer Science 2025-08-21 Hugo Sales Corrêa , Suryanarayana Sankagiri , Daniel Ratton Figueiredo , Matthias Grossglauser

Background: The hazard ratio of the Cox proportional hazards model is widely used in randomized controlled trials to assess treatment effects. However, two properties of the hazard ratio including the non-collapsibility and built-in…

Methodology · Statistics 2024-01-17 Helen Bian , Menglan Pang , Guanbo Wang , Zihang Lu

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