Assessing Discriminative Performance at External Validation of Clinical Prediction Models

PLoS One. 2016 Feb 16;11(2):e0148820. doi: 10.1371/journal.pone.0148820. eCollection 2016.

Abstract

Introduction: External validation studies are essential to study the generalizability of prediction models. Recently a permutation test, focusing on discrimination as quantified by the c-statistic, was proposed to judge whether a prediction model is transportable to a new setting. We aimed to evaluate this test and compare it to previously proposed procedures to judge any changes in c-statistic from development to external validation setting.

Methods: We compared the use of the permutation test to the use of benchmark values of the c-statistic following from a previously proposed framework to judge transportability of a prediction model. In a simulation study we developed a prediction model with logistic regression on a development set and validated them in the validation set. We concentrated on two scenarios: 1) the case-mix was more heterogeneous and predictor effects were weaker in the validation set compared to the development set, and 2) the case-mix was less heterogeneous in the validation set and predictor effects were identical in the validation and development set. Furthermore we illustrated the methods in a case study using 15 datasets of patients suffering from traumatic brain injury.

Results: The permutation test indicated that the validation and development set were homogenous in scenario 1 (in almost all simulated samples) and heterogeneous in scenario 2 (in 17%-39% of simulated samples). Previously proposed benchmark values of the c-statistic and the standard deviation of the linear predictors correctly pointed at the more heterogeneous case-mix in scenario 1 and the less heterogeneous case-mix in scenario 2.

Conclusion: The recently proposed permutation test may provide misleading results when externally validating prediction models in the presence of case-mix differences between the development and validation population. To correctly interpret the c-statistic found at external validation it is crucial to disentangle case-mix differences from incorrect regression coefficients.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Brain Injuries / physiopathology*
  • Humans
  • Logistic Models
  • Models, Theoretical*
  • Prognosis*

Grants and funding

This work was funded by The Netherlands Organization for Scientific Research (ZonMw 9120.8004 (TOP)). The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.