Statistical Methods for Dynamic Treatment Regimes

Author: Bibhas Chakraborty
Publisher: Springer Science & Business Media
ISBN: 1461474280
Format: PDF, ePub, Mobi
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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.

Methods in Comparative Effectiveness Research

Author: Constantine Gatsonis
Publisher: CRC Press
ISBN: 1466511974
Format: PDF, ePub, Docs
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Comparative effectiveness research (CER) is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care (IOM 2009). CER is conducted to develop evidence that will aid patients, clinicians, purchasers, and health policy makers in making informed decisions at both the individual and population levels. CER encompasses a very broad range of types of studies—experimental, observational, prospective, retrospective, and research synthesis. This volume covers the main areas of quantitative methodology for the design and analysis of CER studies. The volume has four major sections—causal inference; clinical trials; research synthesis; and specialized topics. The audience includes CER methodologists, quantitative-trained researchers interested in CER, and graduate students in statistics, epidemiology, and health services and outcomes research. The book assumes a masters-level course in regression analysis and familiarity with clinical research.

Adaptive Treatment Strategies in Practice Planning Trials and Analyzing Data for Personalized Medicine

Author: Michael R. Kosorok
Publisher: SIAM
ISBN: 1611974186
Format: PDF
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Personalized medicine is a medical paradigm that emphasizes systematic use of individual patient information to optimize that patient's health care, particularly in managing chronic conditions and treating cancer. In the statistical literature, sequential decision making is known as an adaptive treatment strategy (ATS) or a dynamic treatment regime (DTR). The field of DTRs emerges at the interface of statistics, machine learning, and biomedical science to provide a data-driven framework for precision medicine. The authors provide a learning-by-seeing approach to the development of ATSs, aimed at a broad audience of health researchers. All estimation procedures used are described in sufficient heuristic and technical detail so that less quantitative readers can understand the broad principles underlying the approaches. At the same time, more quantitative readers can implement these practices. This book provides the most up-to-date summary of the current state of the statistical research in personalized medicine; contains chapters by leaders in the area from both the statistics and computer sciences fields; and also contains a range of practical advice, introductory and expository materials, and case studies.

Handbook of Statistical Systems Biology

Author: Michael Stumpf
Publisher: John Wiley & Sons
ISBN: 1119952042
Format: PDF, ePub, Docs
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Systems Biology is now entering a mature phase in which the key issues are characterising uncertainty and stochastic effects in mathematical models of biological systems. The area is moving towards a full statistical analysis and probabilistic reasoning over the inferences that can be made from mathematical models. This handbook presents a comprehensive guide to the discipline for practitioners and educators, in providing a full and detailed treatment of these important and emerging subjects. Leading experts in systems biology and statistics have come together to provide insight in to the major ideas in the field, and in particular methods of specifying and fitting models, and estimating the unknown parameters. This book: Provides a comprehensive account of inference techniques in systems biology. Introduces classical and Bayesian statistical methods for complex systems. Explores networks and graphical modeling as well as a wide range of statistical models for dynamical systems. Discusses various applications for statistical systems biology, such as gene regulation and signal transduction. Features statistical data analysis on numerous technologies, including metabolic and transcriptomic technologies. Presents an in-depth presentation of reverse engineering approaches. Provides colour illustrations to explain key concepts. This handbook will be a key resource for researchers practising systems biology, and those requiring a comprehensive overview of this important field.

Understanding Machine Learning

Author: Shai Shalev-Shwartz
Publisher: Cambridge University Press
ISBN: 1107057132
Format: PDF, ePub, Docs
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Introduces machine learning and its algorithmic paradigms, explaining the principles behind automated learning approaches and the considerations underlying their usage.

Bioinformatics Computing

Author: Bryan P. Bergeron
Publisher: Prentice Hall Professional
ISBN: 9780131008250
Format: PDF, ePub, Docs
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Comprehensive and concise, this handbook has chapters on computing visualization, large database designs, advanced pattern matching and other key bioinformatics techniques. It is a practical guide to computing in the growing field of Bioinformatics--the study of how information is represented and transmitted in biological systems, starting at the molecular level.

Statistical Methods in Healthcare

Author: Frederick Faltin
Publisher: John Wiley & Sons
ISBN: 1119942047
Format: PDF, Docs
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In recent years the number of innovative medicinal products and devices submitted and approved by regulatory bodies has declined dramatically. The medical product development process is no longer able to keep pace with increasing technologies, science and innovations and the goal is to develop new scientific and technical tools and to make product development processes more efficient and effective. Statistical Methods in Healthcare focuses on the application of statistical methodologies to evaluate promising alternatives and to optimize the performance and demonstrate the effectiveness of those that warrant pursuit is critical to success. Statistical methods used in planning, delivering and monitoring health care, as well as selected statistical aspects of the development and/or production of pharmaceuticals and medical devices are also addressed. With a focus on finding solutions to these challenges, this book: Provides a comprehensive, in-depth treatment of statistical methods in healthcare, along with a reference source for practitioners and specialists in health care and drug development. Offers a broad coverage of standards and established methods through leading edge techniques. Uses an integrated, case-study based approach, with focus on applications. Looks at the use of analytical and monitoring schemes to evaluate therapeutic performance. Features the application of modern quality management systems to clinical practice, and to pharmaceutical development and production processes. Addresses the use of modern Statistical methods such as Adaptive Design, Seamless Design, Data Mining, Bayesian networks and Bootstrapping that can be applied to support the challenging new vision. Practitioners in healthcare-related professions, ranging from clinical trials to care delivery to medical device design, as well as statistical researchers in the field, will benefit from this book.

Design of Experiments in Nonlinear Models

Author: Luc Pronzato
Publisher: Springer Science & Business Media
ISBN: 1461463637
Format: PDF, Mobi
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Design of Experiments in Nonlinear Models: Asymptotic Normality, Optimality Criteria and Small-Sample Properties provides a comprehensive coverage of the various aspects of experimental design for nonlinear models. The book contains original contributions to the theory of optimal experiments that will interest students and researchers in the field. Practitionners motivated by applications will find valuable tools to help them designing their experiments. The first three chapters expose the connections between the asymptotic properties of estimators in parametric models and experimental design, with more emphasis than usual on some particular aspects like the estimation of a nonlinear function of the model parameters, models with heteroscedastic errors, etc. Classical optimality criteria based on those asymptotic properties are then presented thoroughly in a special chapter. Three chapters are dedicated to specific issues raised by nonlinear models. The construction of design criteria derived from non-asymptotic considerations (small-sample situation) is detailed. The connection between design and identifiability/estimability issues is investigated. Several approaches are presented to face the problem caused by the dependence of an optimal design on the value of the parameters to be estimated. A survey of algorithmic methods for the construction of optimal designs is provided.

Toward Precision Medicine

Author: Committee on a Framework for Development a New Taxonomy of Disease
Publisher: National Academies Press
ISBN: 0309222222
Format: PDF, ePub
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Motivated by the explosion of molecular data on humans-particularly data associated with individual patients-and the sense that there are large, as-yet-untapped opportunities to use this data to improve health outcomes, Toward Precision Medicine explores the feasibility and need for "a new taxonomy of human disease based on molecular biology" and develops a potential framework for creating one. The book says that a new data network that integrates emerging research on the molecular makeup of diseases with clinical data on individual patients could drive the development of a more accurate classification of diseases and ultimately enhance diagnosis and treatment. The "new taxonomy" that emerges would define diseases by their underlying molecular causes and other factors in addition to their traditional physical signs and symptoms. The book adds that the new data network could also improve biomedical research by enabling scientists to access patients' information during treatment while still protecting their rights. This would allow the marriage of molecular research and clinical data at the point of care, as opposed to research information continuing to reside primarily in academia. Toward Precision Medicine notes that moving toward individualized medicine requires that researchers and health care providers have access to very large sets of health- and disease-related data linked to individual patients. These data are also critical for developing the information commons, the knowledge network of disease, and ultimately the new taxonomy.

Statistical Causal Inferences and Their Applications in Public Health Research

Author: Hua He
Publisher: Springer
ISBN: 3319412590
Format: PDF, ePub, Mobi
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This book compiles and presents new developments in statistical causal inference. The accompanying data and computer programs are publicly available so readers may replicate the model development and data analysis presented in each chapter. In this way, methodology is taught so that readers may implement it directly. The book brings together experts engaged in causal inference research to present and discuss recent issues in causal inference methodological development. This is also a timely look at causal inference applied to scenarios that range from clinical trials to mediation and public health research more broadly. In an academic setting, this book will serve as a reference and guide to a course in causal inference at the graduate level (Master's or Doctorate). It is particularly relevant for students pursuing degrees in statistics, biostatistics, and computational biology. Researchers and data analysts in public health and biomedical research will also find this book to be an important reference.