Development and Validation of Risk Prediction Models
There has been increased interest in the use of clinical risk prediction models for decision-making in medicine for patient care. This has been accelerated through the focus on precision medicine, the revolution in omics data, and increasing use of randomized controlled trial and electronic health record databases. These models are expected to assist diagnostic assessment, prognostication, and therapeutic decision-making. Randomized controlled trial data are highly relevant for modeling treatment benefit and treatment effect heterogeneity. The development and validation of prediction models requires careful methodology and reporting, and an evidence-based approach is needed to bring risk prediction models to clinical practice. This chapter provides an overview of the key steps and considerations to develop and validate risk prediction models. We comment on the role of clinical trials throughout the process. A risk prediction model for the occurrence of breast cancer is used as an example.
KeywordsPrediction models Diagnostic Prognostic Treatment effect Precision medicine Development Predictors Validation Calibration Discrimination Utility
Data collection and sharing was supported by the National Cancer Institute-funded Breast Cancer Surveillance Consortium (HHSN261201100031C). You can learn more about the BCSC at http://www.bcsc-research.org/. We thank the BCSC participants, investigators, mammography facilities, and radiologists for the data they have provided for this study.
This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 755394. (MyPeBS study), and from the Research Foundation – Flanders (FWO) grant G0B4716N; Internal Funds KU Leuven grant C24/15/037.
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