Bayesian analyses have made important inroads in modern clinical research due, in part, to the incorporation of the traditional tools of noninformative priors as well as the modern innovations of adaptive randomization and predictive power. Presenting an introductory perspective to modern Bayesian procedures, Elementary Bayesian Biostatistics explores Bayesian principles and illustrates their application to healthcare research. Building on the basics of classic biostatistics and algebra, this easy-to-read book provides a clear overview of the subject. It focuses on the history and mathematical foundation of Bayesian procedures, before discussing their implementation in healthcare research from first principles. The author also elaborates on the current controversies between Bayesian and frequentist biostatisticians. The book concludes with recommendations for Bayesians to improve their standing in the clinical trials community. Calculus derivations are relegated to the appendices so as not to overly complicate the main text. As Bayesian methods gain more acceptance in healthcare, it is necessary for clinical scientists to understand Bayesian principles. Applying Bayesian analyses to modern healthcare research issues, this lucid introduction helps readers make the correct choices in the development of clinical research programs.
Emphasizing statistical concepts used in medicine, the interpretation of methods, and applications, Medical Biostatistics, Second Edition shows how biostatistical methods are important tools in managing uncertainties in medicine and the health sciences. With coverage ranging from elementary topics, such as mean and standard deviation, to advanced approaches, such as logistic regression and multivariate methods, this edition is even more far-reaching in scope than its predecessor. New to the Second Edition Full chapters on clinical trials, observational studies, laboratory experiments, survival analysis, and logistic regression A new chapter on clinimetrics and evidence-based medicine Expanded discussions on epistemic uncertainties, crossover designs, equivalence trials and studies, hazard functions, and the log-rank test The introduction of research evidence, multilevel regression, classification, and regression trees at an elementary level Sample size formulas for relative risk, odds ratio, and survival studies Numerous additional examples from contemporary medical literature Numbered steps for the many procedures and remarks Requiring only high school algebra, this text enables a solid understanding of the statistical concepts required to critically examine medical literature, scientifically plan and carry out medical investigations, and meaningfully analyze data.
Author: Emmanuel Lesaffre
Publisher: John Wiley & Sons
Release Date: 2012-06-18
The growth of biostatistics has been phenomenal in recent years and has been marked by considerable technical innovation in both methodology and computational practicality. One area that has experienced significant growth is Bayesian methods. The growing use of Bayesian methodology has taken place partly due to an increasing number of practitioners valuing the Bayesian paradigm as matching that of scientific discovery. In addition, computational advances have allowed for more complex models to be fitted routinely to realistic data sets. Through examples, exercises and a combination of introductory and more advanced chapters, this book provides an invaluable understanding of the complex world of biomedical statistics illustrated via a diverse range of applications taken from epidemiology, exploratory clinical studies, health promotion studies, image analysis and clinical trials. Key Features: Provides an authoritative account of Bayesian methodology, from its most basic elements to its practical implementation, with an emphasis on healthcare techniques. Contains introductory explanations of Bayesian principles common to all areas of application. Presents clear and concise examples in biostatistics applications such as clinical trials, longitudinal studies, bioassay, survival, image analysis and bioinformatics. Illustrated throughout with examples using software including WinBUGS, OpenBUGS, SAS and various dedicated R programs. Highlights the differences between the Bayesian and classical approaches. Supported by an accompanying website hosting free software and case study guides. Bayesian Biostatistics introduces the reader smoothly into the Bayesian statistical methods with chapters that gradually increase in level of complexity. Master students in biostatistics, applied statisticians and all researchers with a good background in classical statistics who have interest in Bayesian methods will find this book useful.
Get Up to Speed on Many Types of Adaptive Designs Since the publication of the first edition, there have been remarkable advances in the methodology and application of adaptive trials. Incorporating many of these new developments, Adaptive Design Theory and Implementation Using SAS and R, Second Edition offers a detailed framework to understand the use of various adaptive design methods in clinical trials. New to the Second Edition Twelve new chapters covering blinded and semi-blinded sample size reestimation design, pick-the-winners design, biomarker-informed adaptive design, Bayesian designs, adaptive multiregional trial design, SAS and R for group sequential design, and much more More analytical methods for K-stage adaptive designs, multiple-endpoint adaptive design, survival modeling, and adaptive treatment switching New material on sequential parallel designs with rerandomization and the skeleton approach in adaptive dose-escalation trials Twenty new SAS macros and R functions Enhanced end-of-chapter problems that give readers hands-on practice addressing issues encountered in designing real-life adaptive trials Covering even more adaptive designs, this book provides biostatisticians, clinical scientists, and regulatory reviewers with up-to-date details on this innovative area in pharmaceutical research and development. Practitioners will be able to improve the efficiency of their trial design, thereby reducing the time and cost of drug development.
Author: Robert G. Newcombe
Publisher: CRC Press
Release Date: 2012-08-25
Confidence Intervals for Proportions and Related Measures of Effect Size illustrates the use of effect size measures and corresponding confidence intervals as more informative alternatives to the most basic and widely used significance tests. The book provides you with a deep understanding of what happens when these statistical methods are applied in situations far removed from the familiar Gaussian case. Drawing on his extensive work as a statistician and professor at Cardiff University School of Medicine, the author brings together methods for calculating confidence intervals for proportions and several other important measures, including differences, ratios, and nonparametric effect size measures generalizing Mann-Whitney and Wilcoxon tests. He also explains three important approaches to obtaining intervals for related measures. Many examples illustrate the application of the methods in the health and social sciences. Requiring little computational skills, the book offers user-friendly Excel spreadsheets for download at www.crcpress.com, enabling you to easily apply the methods to your own empirical data.
Interval-Censored Time-to-Event Data: Methods and Applications collects the most recent techniques, models, and computational tools for interval-censored time-to-event data. Top biostatisticians from academia, biopharmaceutical industries, and government agencies discuss how these advances are impacting clinical trials and biomedical research. Divided into three parts, the book begins with an overview of interval-censored data modeling, including nonparametric estimation, survival functions, regression analysis, multivariate data analysis, competing risks analysis, and other models for interval-censored data. The next part presents interval-censored methods for current status data, Bayesian semiparametric regression analysis of interval-censored data with monotone splines, Bayesian inferential models for interval-censored data, an estimator for identifying causal effect of treatment, and consistent variance estimation for interval-censored data. In the final part, the contributors use Monte Carlo simulation to assess biases in progression-free survival analysis as well as correct bias in interval-censored time-to-event applications. They also present adaptive decision making methods to optimize the rapid treatment of stroke, explore practical issues in using weighted logrank tests, and describe how to use two R packages. A practical guide for biomedical researchers, clinicians, biostatisticians, and graduate students in biostatistics, this volume covers the latest developments in the analysis and modeling of interval-censored time-to-event data. It shows how up-to-date statistical methods are used in biopharmaceutical and public health applications.
Author: Carol K. Redmond
Publisher: John Wiley & Sons
Release Date: 2001-04-25
The second volume in the Wiley reference series inBiostatistics. Featuring articles from the prestigious Encyclopedia ofBiostatistics, many of which have been fully revised and updated toinclude recent developments, Biostatistics in Clinical Trials alsoincludes up to 25% newly commissioned material reflecting thelatest thinking in: Bayesian methods Benefit/risk assessment Cost-effectiveness Ethics Fraud With exceptional contributions from leading experts in academia,government and industry, Biostatistics in Clinical Trials has beendesigned to complement existing texts by providing extensive,up-to-date coverage and introducing the reader to the researchliterature. Offering comprehensive coverage of all aspects of clinical trialsBiostatistics in Clinical Trials: Includes concise definitions and introductions to numerousconcepts found in current literature Discusses the software and textbooks available Uses extensive cross-references helping to facilitate furtherresearch and enabling the reader to locate definitions and relatedconcepts Biostatistics in Clinical Trials offers both academics andpractitioners from various disciplines and settings, such asuniversities, the pharmaceutical industry and clinical researchorganisations, up-to-date information as well as references toassist professionals involved in the design and conduct of clinicaltrials.
Adaptive design has become an important tool in modern pharmaceutical research and development. Compared to a classic trial design with static features, an adaptive design allows for the modification of the characteristics of ongoing trials based on cumulative information. Adaptive designs increase the probability of success, reduce costs and the time to market, and promote accurate drug delivery to patients. Reflecting the state of the art in adaptive design approaches, Adaptive Design Theory and Implementation Using SAS and R provides a concise, unified presentation of adaptive design theories, uses SAS and R for the design and simulation of adaptive trials, and illustrates how to master different adaptive designs through real-world examples. The book focuses on simple two-stage adaptive designs with sample size re-estimation before moving on to explore more challenging designs and issues that include drop-loser, adaptive dose-funding, biomarker-adaptive, multiple-endpoint adaptive, response-adaptive randomization, and Bayesian adaptive designs. In many of the chapters, the author compares methods and provides practical examples of the designs, including those used in oncology, cardiovascular, and inflammation trials. Equipped with the knowledge of adaptive design presented in this book, you will be able to improve the efficiency of your trial design, thereby reducing the time and cost of drug development.
In clinical trial practice, controversial statistical issues inevitably occur regardless of the compliance with good statistical practice and good clinical practice. But by identifying the causes of the issues and correcting them, the study objectives of clinical trials can be better achieved. Controversial Statistical Issues in Clinical Trials covers commonly encountered controversial statistical issues in clinical trials and, whenever possible, makes recommendations to resolve these problems. The book focuses on issues occurring at various stages of clinical research and development, including early-phase clinical development (such as bioavailability/bioequivalence), bench-to-bedside translational research, and late-phase clinical development. Numerous examples illustrate the impact of these issues on the evaluation of the safety and efficacy of the test treatment under investigation. The author also offers recommendations regarding possible resolutions of the problems. Written by one of the preeminent experts in the field, this book provides a useful desk reference and state-of-the art examination of problematic issues in clinical trials for scientists in the pharmaceutical industry, medical/statistical reviewers in government regulatory agencies, and researchers and students in academia.
With new statistical and scientific issues arising in adaptive clinical trial design, including the U.S. FDA’s recent draft guidance, a new edition of one of the first books on the topic is needed. Adaptive Design Methods in Clinical Trials, Second Edition reflects recent developments and regulatory positions on the use of adaptive designs in clinical trials. It unifies the vast and continuously growing literature and research activities on regulatory requirements, scientific and practical issues, and statistical methodology. New to the Second Edition Along with revisions throughout the text, this edition significantly updates the chapters on protocol amendment and clinical trial simulation to incorporate the latest changes. It also includes five entirely new chapters on two-stage adaptive design, biomarker adaptive trials, target clinical trials, sample size and power estimation, and regulatory perspectives. Following in the tradition of its acclaimed predecessor, this second edition continues to offer an up-to-date resource for clinical scientists and researchers in academia, regulatory agencies, and the pharmaceutical industry. Written in an intuitive style at a basic mathematical and statistical level, the book maintains its practical approach with an emphasis on concepts via numerous examples and illustrations.
Author: Allen B. Downey
Publisher: O'Reilly Germany
Release Date: 2012-05-31
Wenn Sie programmieren können, beherrschen Sie bereits Techniken, um aus Daten Wissen zu extrahieren. Diese kompakte Einführung in die Statistik zeigt Ihnen, wie Sie rechnergestützt, anstatt auf mathematischem Weg Datenanalysen mit Python durchführen können. Praktischer Programmier-Workshop statt grauer Theorie: Das Buch führt Sie anhand eines durchgängigen Fallbeispiels durch eine vollständige Datenanalyse -- von der Datensammlung über die Berechnung statistischer Kennwerte und Identifikation von Mustern bis hin zum Testen statistischer Hypothesen. Gleichzeitig werden Sie mit statistischen Verteilungen, den Regeln der Wahrscheinlichkeitsrechnung, Visualisierungsmöglichkeiten und vielen anderen Arbeitstechniken und Konzepten vertraut gemacht. Statistik-Konzepte zum Ausprobieren: Entwickeln Sie über das Schreiben und Testen von Code ein Verständnis für die Grundlagen von Wahrscheinlichkeitsrechnung und Statistik: Überprüfen Sie das Verhalten statistischer Merkmale durch Zufallsexperimente, zum Beispiel indem Sie Stichproben aus unterschiedlichen Verteilungen ziehen. Nutzen Sie Simulationen, um Konzepte zu verstehen, die auf mathematischem Weg nur schwer zugänglich sind. Lernen Sie etwas über Themen, die in Einführungen üblicherweise nicht vermittelt werden, beispielsweise über die Bayessche Schätzung. Nutzen Sie Python zur Bereinigung und Aufbereitung von Rohdaten aus nahezu beliebigen Quellen. Beantworten Sie mit den Mitteln der Inferenzstatistik Fragestellungen zu realen Daten.
R ist eine objekt-orientierte und interpretierte Sprache und Programmierumgebung für Datenanalyse und Grafik - frei erhältlich unter der GPL. Ziel dieses Buches ist es, nicht nur ausführlich in die Grundlagen der Sprache R einzuführen, sondern auch ein Verständnis der Struktur der Sprache zu vermitteln. Leicht können so eigene Methoden umgesetzt, Objektklassen definiert und ganze Pakete aus Funktionen und zugehöriger Dokumentation zusammengestellt werden. Die enormen Grafikfähigkeiten von R werden detailliert beschrieben. Das Buch richtet sich an alle, die R als flexibles Werkzeug zur Datenenalyse und -visualisierung einsetzen möchten: Studierende, die Daten in Projekten oder für ihre Diplomarbeit analysieren möchten, Forschende, die neue Methoden ausprobieren möchten, und diejenigen, die in der Wirtschaft täglich Daten aufbereiten, analysieren und anderen in komprimierter Form präsentieren.