Propensity‐based standardization to enhance the validation and interpretation of prediction model discrimination for a target population

Author:

de Jong Valentijn M. T.12ORCID,Hoogland Jeroen13ORCID,Moons Karel G. M.1,Riley Richard D.4ORCID,Nguyen Tri‐Long5ORCID,Debray Thomas P. A.16ORCID

Affiliation:

1. Julius Center for Health Sciences and Primary Care University Medical Center Utrecht, Utrecht University Utrecht The Netherlands

2. Data Analytics and Methods Task Force European Medicines Agency Amsterdam The Netherlands

3. Department of Epidemiology and Data Science Amsterdam University Medical Centers Amsterdam The Netherlands

4. Institute of Applied Health Research, College of Medical and Dental Sciences University of Birmingham Birmingham UK

5. Section of Epidemiology, Department of Public Health, Faculty of Health and Medical Sciences University of Copenhagen Copenhagen Denmark

6. Smart Data Analysis and Statistics Utrecht The Netherlands

Abstract

External validation of the discriminative ability of prediction models is of key importance. However, the interpretation of such evaluations is challenging, as the ability to discriminate depends on both the sample characteristics (ie, case‐mix) and the generalizability of predictor coefficients, but most discrimination indices do not provide any insight into their respective contributions. To disentangle differences in discriminative ability across external validation samples due to a lack of model generalizability from differences in sample characteristics, we propose propensity‐weighted measures of discrimination. These weighted metrics, which are derived from propensity scores for sample membership, are standardized for case‐mix differences between the model development and validation samples, allowing for a fair comparison of discriminative ability in terms of model characteristics in a target population of interest. We illustrate our methods with the validation of eight prediction models for deep vein thrombosis in 12 external validation data sets and assess our methods in a simulation study. In the illustrative example, propensity score standardization reduced between‐study heterogeneity of discrimination, indicating that between‐study variability was partially attributable to case‐mix. The simulation study showed that only flexible propensity‐score methods (allowing for non‐linear effects) produced unbiased estimates of model discrimination in the target population, and only when the positivity assumption was met. Propensity score‐based standardization may facilitate the interpretation of (heterogeneity in) discriminative ability of a prediction model as observed across multiple studies, and may guide model updating strategies for a particular target population. Careful propensity score modeling with attention for non‐linear relations is recommended.

Funder

European Commission

Publisher

Wiley

Subject

Statistics and Probability,Epidemiology

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