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Clinical randomized controlled trials (RCTs) collect hundreds of measurements spanning various metric types (e.g., laboratory tests, cognitive/motor assessments, etc.) across 100s-1000s of subjects to evaluate the effect of a treatment, but…

机器学习 · 计算机科学 2024-06-25 Sayeri Lala , Niraj K. Jha

The integration of real-world data (RWD) and randomized controlled trials (RCT) is increasingly important for advancing causal inference in scientific research. This combination holds great promise for enhancing the efficiency of causal…

统计方法学 · 统计学 2024-07-02 Xi Lin , Jens Magelund Tarp , Robin J. Evans

Many social and environmental phenomena are associated with macroscopic changes in the built environment, captured by satellite imagery on a global scale and with daily temporal resolution. While widely used for prediction, these images and…

统计方法学 · 统计学 2024-07-25 Connor T. Jerzak , Ritwik Vashistha , Adel Daoud

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…

统计方法学 · 统计学 2025-07-22 Amir Asiaee , Chiara Di Gravio , Cole Beck , Yuting Mei , Samhita Pal , Jared D. Huling

When treating depression, clinicians are interested in determining the optimal treatment for a given patient, which is challenging given the amount of treatments available. To advance individualized treatment allocation, integrating data…

Commonly used methods to analyze incomplete longitudinal clinical trial data include complete case analysis (CC) and last observation carried forward (LOCF). However, such methods rest on strong assumptions, including missing completely at…

Randomized controlled trials (RCTs) are increasingly prevalent in education research, and are often regarded as a gold standard of causal inference. Two main virtues of randomized experiments are that they (1) do not suffer from…

Routinely collected nation-wide registers contain socio-economic and health-related information from a large number of individuals. However, important information on lifestyle, biological and other risk factors is available at most for…

统计方法学 · 统计学 2024-09-23 Tommi Härkänen , Sangita Kulathinal , Arya Panthalanickal Vijayakumar

Patients often discontinue treatment in a clinical trial because their health condition is not improving. Consequently, the patients still in the study at the end of the trial have better health outcomes on average than the initial patient…

统计方法学 · 统计学 2022-06-06 Alex Ocampo , Heinz Schmidli , Peter Quarg , Francesca Callegari , Marcello Pagano

Economic evaluations from individual-level data are an important component of the process of technology appraisal, with a view to informing resource allocation decisions. A critical problem in these analyses is that both effectiveness and…

应用统计 · 统计学 2018-02-01 Andrea Gabrio , Alexina J. Mason , Gianluca Baio

Missing outcomes are a commonly occurring problem for cluster randomised trials, which can lead to biased and inefficient inference if ignored or handled inappropriately. Two approaches for analysing such trials are cluster-level analysis…

统计方法学 · 统计学 2016-08-19 Anower Hossain , Karla Diaz-Ordaz , Jonathan W. Bartlett

Causally interpretable meta-analysis combines information from a collection of randomized controlled trials to estimate treatment effects in a target population in which experimentation may not be possible but covariate information can be…

统计方法学 · 统计学 2022-05-03 Jon A. Steingrimsson , David H. Barker , Ruofan Bie , Issa J. Dahabreh

Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…

统计方法学 · 统计学 2025-06-11 Ahmed Boughdiri , Julie Josse , Erwan Scornet

This paper is concerned with identification, estimation, and specification testing in causal evaluation problems when data is selective and/or missing. We leverage recent advances in the literature on graphical methods to provide a unifying…

计量经济学 · 经济学 2023-08-10 Simon Calmar Andersen , Louise Beuchert , Phillip Heiler , Helena Skyt Nielsen

Researchers often run resource-intensive randomized controlled trials (RCTs) to estimate the causal effects of interventions on outcomes of interest. Yet these outcomes are often noisy, and estimated overall effects can be small or…

计量经济学 · 经济学 2023-12-21 Jann Spiess , Vasilis Syrgkanis , Victor Yaneng Wang

AI models are often evaluated based on their ability to predict the outcome of interest. However, in many AI for social impact applications, the presence of an intervention that affects the outcome can bias the evaluation. Randomized…

机器学习 · 计算机科学 2025-11-18 Winston Chen , Michael W. Sjoding , Jenna Wiens

While randomized controlled trials (RCTs) are the gold standard for estimating treatment effects in medical research, there is increasing use of and interest in using real-world data for drug development. One such use case is the…

统计方法学 · 统计学 2021-10-11 Devin Incerti , Michael T Bretscher , Ray Lin , Chris Harbron

Data is a cornerstone of empirical software engineering (ESE) research and practice. Data underpin numerous process and project management activities, including the estimation of development effort and the prediction of the likely location…

软件工程 · 计算机科学 2020-12-22 Michael F. Bosu , Stephen G. MacDonell

Leveraging external controls -- relevant individual patient data under control from external trials or real-world data -- has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial…

统计方法学 · 统计学 2022-07-13 Yanyao Yi , Ying Zhang , Yu Du , Ting Ye

Missing data is a common concern in health datasets, and its impact on good decision-making processes is well documented. Our study's contribution is a methodology for tackling missing data problems using a combination of synthetic dataset…

机器学习 · 计算机科学 2022-11-08 Gift Khangamwa , Terence L. van Zyl , Clint J. van Alten