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In clinical settings, we often face the challenge of building prediction models based on small observational data sets. For example, such a data set might be from a medical center in a multi-center study. Differences between centers might…

Building trustworthy, effective, and responsible machine learning systems hinges on understanding how differences in training data and modeling decisions interact to impact predictive performance. In this work, we seek to better understand…

Machine Learning · Computer Science 2022-11-14 Esther Rolf , Ben Packer , Alex Beutel , Fernando Diaz

Learning about causal effects in target populations and their subsets may be facilitated by combining information from multiple sources. One major class of study designs that combine information involves appending an index study with data…

External validation is widely regarded as the gold standard for prognostic model evaluation. In this study, we challenge the assumption that successful external calibration guarantees model generalizability and propose two complementary…

It is increasingly common to augment randomized controlled trial with external controls from observational data, to evaluate the treatment effect of an intervention. Traditional approaches to treatment effect estimation involve ambiguous…

Methodology · Statistics 2025-03-28 Bo Liu , Laine Thomas , Rury R. Holman , Fan Li

We consider the estimation of measures of model performance in a target population when covariate and outcome data are available on a sample from some source population and covariate data, but not outcome data, are available on a simple…

Methodology · Statistics 2023-06-16 Jon A. Steingrimsson , Sarah E. Robertson , Issa J. Dahabreh

Utilizing established risk factors and prognostic models can often improve the construction of a newer risk model that uses novel biomarkers in a smaller, internal study. However, directly borrowing information from an established…

Methodology · Statistics 2026-03-12 Nicholas C. Henderson

The use of patient-level information from previous studies, registries, and other external datasets can support the analysis of single-arm and randomized controlled trials to evaluate and test experimental treatments. However, the…

Methodology · Statistics 2025-10-23 Gopal Kotecha , Daniel E. Schwartz , Steffen Ventz , Lorenzo Trippa

We present methods for estimating loss-based measures of the performance of a prediction model in a target population that differs from the source population in which the model was developed, in settings where outcome and covariate data are…

Estimation of individual treatment effects is commonly used as the basis for contextual decision making in fields such as healthcare, education, and economics. However, it is often sufficient for the decision maker to have estimates of…

Machine Learning · Computer Science 2020-08-13 Maggie Makar , Fredrik D. Johansson , John Guttag , David Sontag

There is a growing need for flexible general frameworks that integrate individual-level data with external summary information for improved statistical inference. External information relevant for a risk prediction model may come in…

Methodology · Statistics 2023-04-11 Tian Gu , Jeremy M. G. Taylor , Bhramar Mukherjee

Causal inference in a program evaluation setting faces the problem of external validity when the treatment effect in the target population is different from the treatment effect identified from the population of which the sample is…

Methodology · Statistics 2021-12-23 Kyungchul Song , Zhengfei Yu

As the use of machine learning in high impact domains becomes widespread, the importance of evaluating safety has increased. An important aspect of this is evaluating how robust a model is to changes in setting or population, which…

Machine Learning · Computer Science 2021-03-16 Adarsh Subbaswamy , Roy Adams , Suchi Saria

Suppose we are interested in the effect of a treatment in a clinical trial. The efficiency of inference may be limited due to small sample size. However, external control data are often available from historical studies. Motivated by an…

Methodology · Statistics 2021-12-10 Xinyu Li , Wang Miao , Fang Lu , Xiao-Hua Zhou

In a data-scarce field such as healthcare, where models often deliver predictions on patients with rare conditions, the ability to measure the uncertainty of a model's prediction could potentially lead to improved effectiveness of decision…

Machine Learning · Statistics 2020-05-26 Lotta Meijerink , Giovanni Cinà , Michele Tonutti

Conventional meta analysis of model performance conducted using datasources from different underlying populations often result in estimates that cannot be interpreted in the context of a well defined target population. In this manuscript we…

Methodology · Statistics 2024-09-23 Jon A. Steingrimsson , Lan Wen , Sarah Voter , Issa J. Dahabreh

Assessment of model fitness is a key part of machine learning. The standard paradigm is to learn models by minimizing a chosen loss function averaged over training data, with the aim of achieving small losses on future data. In this paper,…

Machine Learning · Statistics 2023-10-17 Ludvig Hult , Dave Zachariah , Petre Stoica

Machine Learning (ML) algorithms are vital for supporting clinical decision-making in biomedical informatics. However, their predictive performance can vary across demographic groups, often due to the underrepresentation of historically…

Machine Learning · Computer Science 2025-03-04 Ioannis Bilionis , Ricardo C. Berrios , Luis Fernandez-Luque , Carlos Castillo

With the development of biomedical science, researchers have increasing access to an abundance of studies focusing on similar research questions. There is a growing interest in the integration of summary information from those studies to…

Methodology · Statistics 2023-11-13 Jianxuan Zang , K. C. G. Chan , Fei Gao

In epidemiology, obtaining accurate individual exposure measurements can be costly and challenging. Thus, these measurements are often subject to error. Regression calibration with a validation study is widely employed as a study design and…

Methodology · Statistics 2026-02-24 Zexiang Li , Donna Spiegelman , Molin Wang , Zuoheng Wang , Xin Zhou
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