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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.
1. Provides a comprehensive overview of meta-analysis methods and applications. 2. Divided into four major sub-topics, covering univariate meta-analysis, multivariate, applications and policy. 3. Designed to be suitable for graduate students and researchers new to the field. 4. Includes lots of real examples, with data and software code made available. 5. Chapters written by the leading researchers in the field.
Bringing together leading authorities, this unique handbook reviews the breadth of current approaches for studying how people think, feel, and behave in everyday environments, rather than in the laboratory. The volume thoroughly describes experience sampling methods, diary methods, physiological measures, and other self-report and non-self-report tools that allow for repeated, real-time measurement in natural settings. Practical guidance is provided to help the reader design a high-quality study, select and implement appropriate methods, and analyze the resulting data using cutting-edge statistical techniques. Applications across a wide range of psychological subfields and research areas are discussed in detail.
Handbook of Statistical Analysis and Data Mining Applications, Second Edition, is a comprehensive professional reference book that guides business analysts, scientists, engineers and researchers, both academic and industrial, through all stages of data analysis, model building and implementation. The handbook helps users discern technical and business problems, understand the strengths and weaknesses of modern data mining algorithms and employ the right statistical methods for practical application. This book is an ideal reference for users who want to address massive and complex datasets with novel statistical approaches and be able to objectively evaluate analyses and solutions. It has clear, intuitive explanations of the principles and tools for solving problems using modern analytic techniques and discusses their application to real problems in ways accessible and beneficial to practitioners across several areas—from science and engineering, to medicine, academia and commerce. - Includes input by practitioners for practitioners - Includes tutorials in numerous fields of study that provide step-by-step instruction on how to use supplied tools to build models - Contains practical advice from successful real-world implementations - Brings together, in a single resource, all the information a beginner needs to understand the tools and issues in data mining to build successful data mining solutions - Features clear, intuitive explanations of novel analytical tools and techniques, and their practical applications
A timely update of a highly popular handbook on statistical genomics This new, two-volume edition of a classic text provides a thorough introduction to statistical genomics, a vital resource for advanced graduate students, early-career researchers and new entrants to the field. It introduces new and updated information on developments that have occurred since the 3rd edition. Widely regarded as the reference work in the field, it features new chapters focusing on statistical aspects of data generated by new sequencing technologies, including sequence-based functional assays. It expands on previous coverage of the many processes between genotype and phenotype, including gene expression and epigenetics, as well as metabolomics. It also examines population genetics and evolutionary models and inference, with new chapters on the multi-species coalescent, admixture and ancient DNA, as well as genetic association studies including causal analyses and variant interpretation. The Handbook of Statistical Genomics focuses on explaining the main ideas, analysis methods and algorithms, citing key recent and historic literature for further details and references. It also includes a glossary of terms, acronyms and abbreviations, and features extensive cross-referencing between chapters, tying the different areas together. With heavy use of up-to-date examples and references to web-based resources, this continues to be a must-have reference in a vital area of research. Provides much-needed, timely coverage of new developments in this expanding area of study Numerous, brand new chapters, for example covering bacterial genomics, microbiome and metagenomics Detailed coverage of application areas, with chapters on plant breeding, conservation and forensic genetics Extensive coverage of human genetic epidemiology, including ethical aspects Edited by one of the leading experts in the field along with rising stars as his co-editors Chapter authors are world-renowned experts in the field, and newly emerging leaders. The Handbook of Statistical Genomics is an excellent introductory text for advanced graduate students and early-career researchers involved in statistical genetics.
In today’s global and highly competitive environment, continuous improvement in the processes and products of any field of engineering is essential for survival. This book gathers together the full range of statistical techniques required by engineers from all fields. It will assist them to gain sensible statistical feedback on how their processes or products are functioning and to give them realistic predictions of how these could be improved. The handbook will be essential reading for all engineers and engineering-connected managers who are serious about keeping their methods and products at the cutting edge of quality and competitiveness.
The statistics profession is at a unique point in history. The need for valid statistical tools is greater than ever; data sets are massive, often measuring hundreds of thousands of measurements for a single subject. The field is ready to move towards clear objective benchmarks under which tools can be evaluated. Targeted learning allows (1) the full generalization and utilization of cross-validation as an estimator selection tool so that the subjective choices made by humans are now made by the machine, and (2) targeting the fitting of the probability distribution of the data toward the target parameter representing the scientific question of interest. This book is aimed at both statisticians and applied researchers interested in causal inference and general effect estimation for observational and experimental data. Part I is an accessible introduction to super learning and the targeted maximum likelihood estimator, including related concepts necessary to understand and apply these methods. Parts II-IX handle complex data structures and topics applied researchers will immediately recognize from their own research, including time-to-event outcomes, direct and indirect effects, positivity violations, case-control studies, censored data, longitudinal data, and genomic studies.
Dynamic Treatment Regimes: Statistical Methods for Precision Medicine provides a comprehensive introduction to statistical methodology for the evaluation and discovery of dynamic treatment regimes from data. Researchers and graduate students in statistics, data science, and related quantitative disciplines with a background in probability and statistical inference and popular statistical modeling techniques will be prepared for further study of this rapidly evolving field. A dynamic treatment regime is a set of sequential decision rules, each corresponding to a key decision point in a disease or disorder process, where each rule takes as input patient information and returns the treatment option he or she should receive. Thus, a treatment regime formalizes how a clinician synthesizes patient information and selects treatments in practice. Treatment regimes are of obvious relevance to precision medicine, which involves tailoring treatment selection to patient characteristics in an evidence-based way. Of critical importance to precision medicine is estimation of an optimal treatment regime, one that, if used to select treatments for the patient population, would lead to the most beneficial outcome on average. Key methods for estimation of an optimal treatment regime from data are motivated and described in detail. A dedicated companion website presents full accounts of application of the methods using a comprehensive R package developed by the authors. The authors’ website www.dtr-book.com includes updates, corrections, new papers, and links to useful websites.
"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.
The majority of medical research involves quantitative methods and so it is essential to be able to understand and interpret statistics. This book shows readers how to develop the skills required to critically appraise research evidence effectively, and how to conduct research and communicate their findings.