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Sample size calculations can be challenging with skewed continuous outcomes in randomized controlled trials (RCTs). Standard t-test-based calculations may require data transformation, which may be difficult before data collection.…

External controls from historical trials or observational data can augment randomized controlled trials when large-scale randomization is impractical or unethical, such as in drug evaluation for rare diseases. However, non-randomized…

Methodology · Statistics 2025-05-08 Ke Zhu , Shu Yang , Xiaofei Wang

Two-phase sampling designs have been widely adopted in epidemiological studies to reduce costs when measuring certain biomarkers is prohibitively expensive. Under these designs, investigators commonly relate survival outcomes to risk…

Methodology · Statistics 2025-12-12 Jooho Kim , Yei Eun Shin

Partially rank-ordered set (PROS) sampling is a generalization of ranked set sampling in which rankers are not required to fully rank the sampling units in each set, hence having more flexibility to perform the necessary judgemental ranking…

Statistics Theory · Mathematics 2015-04-29 Armin Hatefi , Mohammad Jafari Jozani

Out-of-distribution (OOD) prediction is often approached by restricting models to causal or invariant covariates, avoiding non-causal spurious associations that may be unstable across environments. Despite its theoretical appeal, this…

Methodology · Statistics 2026-01-06 Shuozhi Zuo , Yixin Wang

Companies offering web services routinely run randomized online experiments to estimate the causal impact associated with the adoption of new features and policies on key performance metrics of interest. These experiments are used to…

Methodology · Statistics 2023-07-13 Lorenzo Masoero , Doug Hains , James McQueen

A crucial task for a randomized controlled trial (RCT) is to specify a statistical method that can yield an efficient estimator and powerful test for the treatment effect. A novel and effective strategy to obtain efficient and powerful…

Understanding how treatment effects vary across patient characteristics is essential for personalized medicine, yet randomized controlled trials (RCTs) are often underpowered to detect heterogeneous treatment effects (HTEs). We propose a…

Methodology · Statistics 2025-07-22 Amir Asiaee , Chiara Di Gravio , Cole Beck , Yuting Mei , Samhita Pal , Jared D. Huling

Data from observational studies (OSs) is widely available and readily obtainable yet frequently contains confounding biases. On the other hand, data derived from randomized controlled trials (RCTs) helps to reduce these biases; however, it…

Methodology · Statistics 2024-10-30 Dong Yao , Caizhi Tang , Qing Cui , Longfei Li

The increasing prevalence of rich sources of data and the availability of electronic medical record databases and electronic registries opens tremendous opportunities for enhancing medical research. For example, controlled trials are…

Methodology · Statistics 2015-09-23 Liwen Ouyang , Daniel W. Apley , Sanjay Mehrotra

Modern deep learning heavily relies on large labeled datasets, which often comse with high costs in terms of both manual labeling and computational resources. To mitigate these challenges, researchers have explored the use of informative…

Machine Learning · Statistics 2023-09-07 Yong Lin , Chen Liu , Chenlu Ye , Qing Lian , Yuan Yao , Tong Zhang

We study optimal variance reduction solutions for count and ratio metrics in online controlled experiments. Our methods leverage flexible machine learning tools to incorporate covariates that are independent from the treatment but have…

Methodology · Statistics 2022-09-05 Ying Jin , Shan Ba

Randomized controlled trials are the standard method for estimating causal effects, ensuring sufficient statistical power and confidence through adequate sample sizes. However, achieving such sample sizes is often challenging. This study…

Methodology · Statistics 2025-03-28 Keisuke Hanada , Masahiro Kojima

Risk for autism can be influenced by genetic mutations in hundreds of genes. Based on findings showing that genes with highly correlated gene expressions are functionally interrelated, "guilt by association" methods such as DAWN have been…

Methodology · Statistics 2020-03-10 Kevin Z. Lin , Han Liu , Kathryn Roeder

Observed events in recommendation are consequence of the decisions made by a policy, thus they are usually selectively labeled, namely the data are Missing Not At Random (MNAR), which often causes large bias to the estimate of true outcomes…

Machine Learning · Computer Science 2021-10-05 Zifeng Wang , Xi Chen , Rui Wen , Shao-Lun Huang

Covariate adjustment is an important tool in the analysis of randomized clinical trials and observational studies. It can be used to increase efficiency and thus power, and to reduce possible bias. While most statistical tests in randomized…

Methodology · Statistics 2011-08-03 Xiaoru Wu , Zhiliang Ying

In this paper we combine the Alias method with the concept of systematic sampling, a method commonly used in particle filters for efficient low-variance resampling. The proposed method allows very fast sampling from a discrete distribution:…

Data Structures and Algorithms · Computer Science 2025-09-30 Ilari Vallivaara , Katja Poikselkä , Pauli Rikula , Juha Röning

Class imbalanced datasets are common in real-world applications that range from credit card fraud detection to rare disease diagnostics. Several popular classification algorithms assume that classes are approximately balanced, and hence…

Machine Learning · Statistics 2018-09-10 Val Andrei Fajardo , David Findlay , Roshanak Houmanfar , Charu Jaiswal , Jiaxi Liang , Honglei Xie

This paper studies inference in randomized controlled trials with covariate-adaptive randomization when there are multiple treatments. More specifically, we study inference about the average effect of one or more treatments relative to…

Econometrics · Economics 2019-01-21 Federico A. Bugni , Ivan A. Canay , Azeem M. Shaikh

Remarkable progress has been made in difference-in-differences (DID) approaches to causal inference that estimate the average effect of a treatment on the treated (ATT). Of these, the semiparametric DID (SDID) approach incorporates a…

Methodology · Statistics 2026-03-09 Takamichi Baba , Yoshiyuki Ninomiya