Introduction * Setting the Scene * Regulatory Aspects in Using Surrogate Markers in Clinical Trials * Notation and Motivating Studies * The History of Surrogate Marker Validation * Validation Using Single-Trial Data: Mixed Binary and Continuous Outcomes * A Meta-Analytic Validation Framework for Contiuous Outcomes * The Choice of Units * Extensions of the Meta-Analytic Approach to Surrogate Endpoints * Meta-Analytic Validation with Binary Outcomes * Validation in the Case of Two Failure-Time Endpoints * An Ordinal Surrogate for a Survival True Endpoint * A Combination of Longitudinal and Survival Endpoints * Repeated Measures and Surrogate Marker Validation * Bayesian Evaluation of Surrogate Endpoints * Surrogate Marker Validation in Mental Health * The Evaluation of Surrogate Endpoints in Practice: Experience in HIV * An Alternative Measure for Meta-Analytic Surrogate Marker Validation * Discussion: Surrogate Endpoint Definition and Evaluation *The Promise and Peril of Surrogate End Points in Cancer Research
Covers the latest research on a sensitive and controversial topic in a professional and well researched manner.
Provides practical outlook as well as model guidelines and software tools that should be of interest to people who use the software tools described and those who do not.
Related title by Co-author Geert Molenbergh has sold more than 3500 copies world wide.
Provides dual viewpoints: from scientists in the industry as well as regulatory authorities.
Both humanitarian and commercial considerations have spurred intensive search for methods to reduce the time and cost required to develop new therapies. The identification and use of surrogate endpoints, i.e., measures that can replace or supplement other endpoints in evaluations of experimental treatments or other interventions, is a general strategy that has stimulated both enthusiasm and skepticism.
This book offers a balanced account on this controversial topic. The text presents major developments of the last couple of decades, together with a unified, meta-analytic framework within which surrogates can be evaluated from several angles. Methodological development is coupled with perspectives on various therapeutic areas. Academic views are juxtaposed with standpoints of scientists working in the biopharmaceutical industry as well as of colleagues from the regulatory authorities.