Dynamic Treatment Regimes
Dynamic Treatment Regimes

Author: Anastasios A. Tsiatis

Publisher: CRC Press

Published: 2019-12-19

Total Pages: 602

ISBN-13: 1498769780

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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.

Dynamic Treatment Regimes
Dynamic Treatment Regimes

Author: Anastasios A. Tsiatis

Publisher: CRC Press

Published: 2019-12-10

Total Pages: 602

ISBN-13: 9781498769778

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"Precision medicine seeks to use data to construct principled, i.e., evidence-based, treatment strategies that dictate where, when, and to whom treatment should be applied. This book provides an accessible yet comprehensive introduction to statistical methodology for dynamic treatment regimes"--

Statistical Methods for Dynamic Treatment Regimes
Statistical Methods for Dynamic Treatment Regimes

Author: Bibhas Chakraborty

Publisher: Springer Science & Business Media

Published: 2013-07-23

Total Pages: 220

ISBN-13: 1461474280

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Statistical Methods for Dynamic Treatment Regimes shares state of the art of statistical methods developed to address questions of estimation and inference for dynamic treatment regimes, a branch of personalized medicine. This volume demonstrates these methods with their conceptual underpinnings and illustration through analysis of real and simulated data. These methods are immediately applicable to the practice of personalized medicine, which is a medical paradigm that emphasizes the systematic use of individual patient information to optimize patient health care. This is the first single source to provide an overview of methodology and results gathered from journals, proceedings, and technical reports with the goal of orienting researchers to the field. The first chapter establishes context for the statistical reader in the landscape of personalized medicine. Readers need only have familiarity with elementary calculus, linear algebra, and basic large-sample theory to use this text. Throughout the text, authors direct readers to available code or packages in different statistical languages to facilitate implementation. In cases where code does not already exist, the authors provide analytic approaches in sufficient detail that any researcher with knowledge of statistical programming could implement the methods from scratch. This will be an important volume for a wide range of researchers, including statisticians, epidemiologists, medical researchers, and machine learning researchers interested in medical applications. Advanced graduate students in statistics and biostatistics will also find material in Statistical Methods for Dynamic Treatment Regimes to be a critical part of their studies.

Clinical and Statistical Considerations in Personalized Medicine
Clinical and Statistical Considerations in Personalized Medicine

Author: Claudio Carini

Publisher: CRC Press

Published: 2014-03-27

Total Pages: 368

ISBN-13: 1466593873

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Clinical and Statistical Considerations in Personalized Medicine explores recent advances related to biomarkers and their translation into clinical development. Leading clinicians, biostatisticians, regulators, commercial professionals, and researchers address the opportunities and challenges in successfully applying biomarkers in drug discovery and preclinical and clinical development. Robust Biomarkers for Drug Development and Disease Treatment The first four chapters discuss biomarker development from a clinical perspective. Coverage ranges from an introduction to biomarkers to advances in RNAi screens, epigenetics, and rare diseases as targets for personalized medicine approaches. Subsequent chapters examine the statistical considerations in applying a personalized medicine approach, including multiplicity in pharmacogenomics. The last chapter assesses the regulatory issues involved in using biomarkers. Improve Patient Care and Reduce Costs and Side Effects Despite the vast amount of literature on biomarkers, there is no comprehensive book that integrates the clinical and statistical components. This book is one of the first to incorporate both the clinical and statistical aspects of biomarkers in the personalized medicine paradigm. Covering a wide spectrum of personalized medicine-related topics, it presents state-of-the-art techniques for advancing the application of biomarkers in drug discovery and development.

Handbook of Statistical Methods for Precision Medicine
Handbook of Statistical Methods for Precision Medicine

Author: Eric Laber

Publisher:

Published: 2024-10

Total Pages: 0

ISBN-13: 9781003216223

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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.

Quantitative Methods for Precision Medicine
Quantitative Methods for Precision Medicine

Author: Rongling Wu

Publisher: CRC Press

Published: 2022-12-26

Total Pages: 307

ISBN-13: 1482219468

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Modern medicine is undergoing a paradigm shift from a "one-size-fits-all" strategy to a more precise patient-customized therapy and medication plan. While the success of precision medicine relies on the level of pharmacogenomic knowledge, dissecting the genetic mechanisms of drug response in a sufficient detail requires powerful computational tools. Quantitative Methods for Precision Medicine: Pharmacogenomics in Action presents the advanced statistical methods for mapping pharmacogenetic control by integrating pharmacokinetic and pharmacodynamic principles of drug-body interactions. Beyond traditional reductionist-based statistical genetic approaches, statistical formulization in this book synthesizes elements of multiple disciplines to infer, visualize, and track how pharmacogenes interact together as an intricate but well-coordinated system to mediate patient-specific drug response. Features: Functional and systems mapping models to characterize the genetic architecture of multiple medication processes Statistical methods for analyzing informative missing data in pharmacogenetic association studies Functional graph theory of inferring genetic interaction networks from association data Leveraging the concept of epistasis to capture its bidirectional, signed and weighted properties Modeling gene-induced cell-cell crosstalk and its impact on drug response A graph model of drug-drug interactions in combination therapies Critical methodological issues to improve pharmacogenomic research as the cornerstone of precision medicine This book is suitable for graduate students and researchers in the fields of biology, medicine, bioinformatics and drug design and delivery who are interested in statistical and computational modelling of biological processes and systems. It may also serve as a major reference for applied mathematicians, computer scientists, and statisticians who attempt to develop algorithmic tools for genetic mapping, systems pharmacogenomics and systems biology. It can be used as both a textbook and research reference. Professionals in pharmaceutical sectors who design drugs and clinical doctors who deliver drugs will also find it useful.

Advanced Medical Statistics (2nd Edition)
Advanced Medical Statistics (2nd Edition)

Author: Ying Lu

Publisher: World Scientific

Published: 2015-06-29

Total Pages: 1471

ISBN-13: 9814583324

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The book aims to provide both comprehensive reviews of the classical methods and an introduction to new developments in medical statistics. The topics range from meta analysis, clinical trial design, causal inference, personalized medicine to machine learning and next generation sequence analysis. Since the publication of the first edition, there have been tremendous advances in biostatistics and bioinformatics. The new edition tries to cover as many important emerging areas and reflect as much progress as possible. Many distinguished scholars, who greatly advanced their research areas in statistical methodology as well as practical applications, also have revised several chapters with relevant updates and written new ones from scratch.The new edition has been divided into four sections, including, Statistical Methods in Medicine and Epidemiology, Statistical Methods in Clinical Trials, Statistical Genetics, and General Methods. To reflect the rise of modern statistical genetics as one of the most fertile research areas since the publication of the first edition, the brand new section on Statistical Genetics includes entirely new chapters reflecting the state of the art in the field.Although tightly related, all the book chapters are self-contained and can be read independently. The book chapters intend to provide a convenient launch pad for readers interested in learning a specific topic, applying the related statistical methods in their scientific research and seeking the newest references for in-depth research.