Clinical Prediction Models: A Practical Approach to Development, Validation, and Updating (Statistics for Biology and Health)

"The second edition of this volume provides insight and practical illustrations on how modern statistical concepts and regression methods can be applied in medical prediction problems, including diagnostic and prognostic outcomes. Many advances have been made in statistical approaches towards outcome prediction, but a sensible strategy is needed for model development, validation, and updating, such that prediction models can better support medical practice. There is an increasing need for personalized evidence-based medicine that uses an individualized approach to medical decision-making. In this Big Data era, there is expanded access to large volumes of routinely collected data and an increased number of applications for prediction models, such as targeted early detection of disease and individualized approaches to diagnostic testing and treatment. Clinical Prediction Models presents a practical checklist that needs to be considered for development of a valid prediction model. Steps include preliminary considerations such as dealing with missing values; coding of predictors; selection of main effects and interactions for a multivariable model; estimation of model parameters with shrinkage methods and incorporation of external data; evaluation of performance and usefulness; internal validation; and presentation formatting. The text also addresses common issues that make prediction models suboptimal, such as small sample sizes, exaggerated claims, and poor generalizability. The text is primarily intended for clinical epidemiologists and biostatisticians. Including many case studies and publicly available R code and data sets, the book is also appropriate as a textbook for a graduate course on predictive modeling in diagnosis and prognosis. While practical in nature, the book also provides a philosophical perspective on data analysis in medicine that goes beyond predictive modeling. Updates to this new and expanded edition include: · A discussion of Big Data and its implications for the design of prediction models · Machine learning issues · More simulations with missing 'y' values · Extended discussion on between-cohort heterogeneity · Description of ShinyApp · Updated LASSO illustration · New case studies ." -- prové de l'editor This book provides insight and practical illustrations on how modern statistical concepts and regression methods can be applied in medical prediction problems, including diagnostic and prognostic outcomes. Many advances have been made in statistical approaches towards outcome prediction, but these innovations are insufficiently applied in medical research. Old-fashioned, data hungry methods are often used in data sets of limited size, validation of predictions is not done or done simplistically, and updating of previously developed models is not considered. A sensible strategy is needed for model development, validation, and updating, such that prediction models can better support medical practice. Clinical prediction models presents a practical checklist with seven steps that need to be considered for development of a valid prediction model. These include preliminary considerations such as dealing with missing values; coding of predictors; selection of main effects and interactions for a multivariable model; estimation of model parameters with shrinkage methods and incorporation of external data; evaluation of performance and usefulness; internal validation; and presentation formats. The steps are illustrated with many small case-studies and R code, with data sets made available in the public domain. The book further focuses on generalizability of prediction models, including patterns of invalidity that may be encountered in new settings, approaches to updating of a model, and comparisons of centers after case-mix adjustment by a prediction model. The text is primarily intended for clinical epidemiologists and biostatisticians. It can be used as a textbook for a graduate course on predictive modeling in diagnosis and prognosis. It is beneficial if readers are familiar with common statistical models in medicine: linear regression, logistic regression, and Cox regression. The book is practical in nature. But it provides a philosophical perspective on data analysis in medicine that goes beyond predictive modeling. In this era of evidence-based medicine, randomized clinical trials are the basis for assessment of treatment efficacy. Prediction models are key to individualizing diagnostic and treatment decision making This book aims to provide insight and practical illustrations on how modern statistical concepts and regression methods can be applied in medical prediction problems, including diagnostic and prognostic outcomes. Many advances have been made in statistical approaches towards outcome prediction, but these innovations are insufficiently applied in medical research. Old-fashioned, data hungry methods are often used in data sets of limited size, validation of predictions is not done or only in a simplistic way, and updating of already available models is not considered. A sensible strategy is needed for model development, validation, and updating, such that prediction models can better support medical practice. The text is primarily intended for epidemiologists and applied biostatisticians. It can be used as a textbook for a graduate course on predictive modeling in diagnosis and prognosis. It is beneficial if readers are familiar with common statistical models in medicine: linea. Prediction models are important in various fields, including medicine, physics, meteorology, and finance. Prediction models will become more relevant in the medical field with the increase in knowledge on potential predictors of outcome, e.g. from genetics. Also, the number of applications will increase, e.g. with targeted early detection of disease, and individualized approaches to diagnostic testing and treatment. The current era of evidence-based medicine asks for an individualized approach to medical decision-making. Evidence-based medicine has a central place for meta-analysis to summarize results from randomized controlled trials; similarly prediction models may summarize the effects of predictors to provide individualized predictions of a diagnostic or prognostic outcome