Methodological guidance for the evaluation and updating of clinical prediction models: a systematic review

Abstract Background Clinical prediction models are often not evaluated properly in specific settings or updated, for instance, with information from new markers. These key steps are needed such that models are fit for purpose and remain relevant in the long-term. We aimed to present an overview of m...

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Main Authors: M. A. E. Binuya, E. G. Engelhardt, W. Schats, M. K. Schmidt, E. W. Steyerberg
Format: Article
Language:English
Published: BMC 2022-12-01
Series:BMC Medical Research Methodology
Subjects:
Online Access:https://doi.org/10.1186/s12874-022-01801-8
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author M. A. E. Binuya
E. G. Engelhardt
W. Schats
M. K. Schmidt
E. W. Steyerberg
author_facet M. A. E. Binuya
E. G. Engelhardt
W. Schats
M. K. Schmidt
E. W. Steyerberg
author_sort M. A. E. Binuya
collection DOAJ
description Abstract Background Clinical prediction models are often not evaluated properly in specific settings or updated, for instance, with information from new markers. These key steps are needed such that models are fit for purpose and remain relevant in the long-term. We aimed to present an overview of methodological guidance for the evaluation (i.e., validation and impact assessment) and updating of clinical prediction models. Methods We systematically searched nine databases from January 2000 to January 2022 for articles in English with methodological recommendations for the post-derivation stages of interest. Qualitative analysis was used to summarize the 70 selected guidance papers. Results Key aspects for validation are the assessment of statistical performance using measures for discrimination (e.g., C-statistic) and calibration (e.g., calibration-in-the-large and calibration slope). For assessing impact or usefulness in clinical decision-making, recent papers advise using decision-analytic measures (e.g., the Net Benefit) over simplistic classification measures that ignore clinical consequences (e.g., accuracy, overall Net Reclassification Index). Commonly recommended methods for model updating are recalibration (i.e., adjustment of intercept or baseline hazard and/or slope), revision (i.e., re-estimation of individual predictor effects), and extension (i.e., addition of new markers). Additional methodological guidance is needed for newer types of updating (e.g., meta-model and dynamic updating) and machine learning-based models. Conclusion Substantial guidance was found for model evaluation and more conventional updating of regression-based models. An important development in model evaluation is the introduction of a decision-analytic framework for assessing clinical usefulness. Consensus is emerging on methods for model updating.
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spelling doaj.art-e2d1d4e33e7a4a12b923e6b1df3ba73c2022-12-22T04:23:39ZengBMCBMC Medical Research Methodology1471-22882022-12-0122111410.1186/s12874-022-01801-8Methodological guidance for the evaluation and updating of clinical prediction models: a systematic reviewM. A. E. Binuya0E. G. Engelhardt1W. Schats2M. K. Schmidt3E. W. Steyerberg4Division of Molecular Pathology, the Netherlands Cancer Institute – Antoni van Leeuwenhoek HospitalDivision of Molecular Pathology, the Netherlands Cancer Institute – Antoni van Leeuwenhoek HospitalScientific Information Service, The Netherlands Cancer Institute – Antoni van Leeuwenhoek HospitalDivision of Molecular Pathology, the Netherlands Cancer Institute – Antoni van Leeuwenhoek HospitalDepartment of Biomedical Data Sciences, Leiden University Medical CenterAbstract Background Clinical prediction models are often not evaluated properly in specific settings or updated, for instance, with information from new markers. These key steps are needed such that models are fit for purpose and remain relevant in the long-term. We aimed to present an overview of methodological guidance for the evaluation (i.e., validation and impact assessment) and updating of clinical prediction models. Methods We systematically searched nine databases from January 2000 to January 2022 for articles in English with methodological recommendations for the post-derivation stages of interest. Qualitative analysis was used to summarize the 70 selected guidance papers. Results Key aspects for validation are the assessment of statistical performance using measures for discrimination (e.g., C-statistic) and calibration (e.g., calibration-in-the-large and calibration slope). For assessing impact or usefulness in clinical decision-making, recent papers advise using decision-analytic measures (e.g., the Net Benefit) over simplistic classification measures that ignore clinical consequences (e.g., accuracy, overall Net Reclassification Index). Commonly recommended methods for model updating are recalibration (i.e., adjustment of intercept or baseline hazard and/or slope), revision (i.e., re-estimation of individual predictor effects), and extension (i.e., addition of new markers). Additional methodological guidance is needed for newer types of updating (e.g., meta-model and dynamic updating) and machine learning-based models. Conclusion Substantial guidance was found for model evaluation and more conventional updating of regression-based models. An important development in model evaluation is the introduction of a decision-analytic framework for assessing clinical usefulness. Consensus is emerging on methods for model updating.https://doi.org/10.1186/s12874-022-01801-8Prediction modelModel evaluationValidationImpact assessmentDiscriminationCalibration
spellingShingle M. A. E. Binuya
E. G. Engelhardt
W. Schats
M. K. Schmidt
E. W. Steyerberg
Methodological guidance for the evaluation and updating of clinical prediction models: a systematic review
BMC Medical Research Methodology
Prediction model
Model evaluation
Validation
Impact assessment
Discrimination
Calibration
title Methodological guidance for the evaluation and updating of clinical prediction models: a systematic review
title_full Methodological guidance for the evaluation and updating of clinical prediction models: a systematic review
title_fullStr Methodological guidance for the evaluation and updating of clinical prediction models: a systematic review
title_full_unstemmed Methodological guidance for the evaluation and updating of clinical prediction models: a systematic review
title_short Methodological guidance for the evaluation and updating of clinical prediction models: a systematic review
title_sort methodological guidance for the evaluation and updating of clinical prediction models a systematic review
topic Prediction model
Model evaluation
Validation
Impact assessment
Discrimination
Calibration
url https://doi.org/10.1186/s12874-022-01801-8
work_keys_str_mv AT maebinuya methodologicalguidancefortheevaluationandupdatingofclinicalpredictionmodelsasystematicreview
AT egengelhardt methodologicalguidancefortheevaluationandupdatingofclinicalpredictionmodelsasystematicreview
AT wschats methodologicalguidancefortheevaluationandupdatingofclinicalpredictionmodelsasystematicreview
AT mkschmidt methodologicalguidancefortheevaluationandupdatingofclinicalpredictionmodelsasystematicreview
AT ewsteyerberg methodologicalguidancefortheevaluationandupdatingofclinicalpredictionmodelsasystematicreview