Handbook Of Statistical Methods For Precision Medicine
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Handbook of Statistical Methods for Precision Medicine
The statistical study and development of analytic methodology for individualization of treatments is no longer in its infancy. Many methods of study design, estimation, and inference exist, and the tools available to the analyst are ever growing. This handbook introduces the foundations of modern statistical approaches to precision medicine, bridging key ideas to active lines of current research in precision medicine. The contributions in this handbook vary in their level of assumed statistical knowledge; all contributions are accessible to a wide readership of statisticians and computer scientists including graduate students and new researchers in the area. Many contributions, particularly those that are more comprehensive reviews, are suitable for epidemiologists and clinical researchers with some statistical training. The handbook is split into three sections: Study Design for Precision Medicine, Estimation of Optimal Treatment Strategies, and Precision Medicine in High Dimensions. The first focuses on designed experiments, in many instances, building and extending on the notion of sequential multiple assignment randomized trials. Dose finding and simulation-based designs using agent-based modelling are also featured. The second section contains both introductory contributions and more advanced methods, suitable for estimating optimal adaptive treatment strategies from a variety of data sources including non-experimental (observational) studies. The final section turns to estimation in the many-covariate setting, providing approaches suitable to the challenges posed by electronic health records, wearable devices, or any other settings where the number of possible variables (whether confounders, tailoring variables, or other) is high. Together, these three sections bring together some of the foremost leaders in the field of precision medicine, offering new insights and ideas as this field moves towards its third decade.
Handbook of Generalized Pairwise Comparisons
In today's healthcare landscape, there is a pressing need for quantitative methodologies that include the patients' perspective in any treatment decision. Handbook of Generalized Pairwise Comparisons: Methods for Patient-Centric Analysis provides a comprehensive overview of an innovative and powerful statistical methodology that generalizes the traditional Wilcoxon-Mann-Whitney test by extending it to any number of outcomes of any type and including thresholds of clinical relevance into a single, multidimensional evaluation. The book covers the statistical foundations of generalized pairwise comparisons (GPC), applications in various disease areas, implications for regulatory approvals and benefit-risk analyses, and considerations for patient-centricity in clinical research. With contributions from leading experts in the field, this book stands as an essential resource for a more holistic and patient-centric assessment of treatment effects.
Handbook of Biomarkers and Precision Medicine
"The field of Biomarkers and Precision Medicine in drug development is rapidly evolving and this book presents a snapshot of exciting new approaches. By presenting a wide range of biomarker applications, discussed by knowledgeable and experienced scientists, readers will develop an appreciation of the scope and breadth of biomarker knowledge and find examples that will help them in their own work." -Maria Freire, Foundation for the National Institutes of Health Handbook of Biomarkers and Precision Medicine provides comprehensive insights into biomarker discovery and development which has driven the new era of Precision Medicine. A wide variety of renowned experts from government, academia, teaching hospitals, biotechnology and pharmaceutical companies share best practices, examples and exciting new developments. The handbook aims to provide in-depth knowledge to research scientists, students and decision makers engaged in Biomarker and Precision Medicine-centric drug development. Features: Detailed insights into biomarker discovery, validation and diagnostic development with implementation strategies Lessons-learned from successful Precision Medicine case studies A variety of exciting and emerging biomarker technologies The next frontiers and future challenges of biomarkers in Precision Medicine Claudio Carini, Mark Fidock and Alain van Gool are internationally recognized as scientific leaders in Biomarkers and Precision Medicine. They have worked for decades in academia and pharmaceutical industry in EU, USA and Asia. Currently, Dr. Carini is Honorary Faculty at Kings’s College School of Medicine, London, UK. Dr. Fidock is Vice President of Precision Medicine Laboratories at AstraZeneca, Cambridge, UK. Prof.dr. van Gool is Head Translational Metabolic Laboratory at Radboud university medical school, Nijmegen, NL.