Elementary Bayesian Biostatistics

Author: Lemuel A. Moyé
Publisher: CRC Press
ISBN: 9781584887256
Release Date: 2016-04-19
Genre: Mathematics

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.

Bayesian Methods in Epidemiology

Author: Lyle D. Broemeling
Publisher: CRC Press
ISBN: 9781466564978
Release Date: 2013-08-13
Genre: Mathematics

Written by a biostatistics expert with over 20 years of experience in the field, Bayesian Methods in Epidemiology presents statistical methods used in epidemiology from a Bayesian viewpoint. It employs the software package WinBUGS to carry out the analyses and offers the code in the text and for download online. The book examines study designs that investigate the association between exposure to risk factors and the occurrence of disease. It covers introductory adjustment techniques to compare mortality between states and regression methods to study the association between various risk factors and disease, including logistic regression, simple and multiple linear regression, categorical/ordinal regression, and nonlinear models. The text also introduces a Bayesian approach for the estimation of survival by life tables and illustrates other approaches to estimate survival, including a parametric model based on the Weibull distribution and the Cox proportional hazards (nonparametric) model. Using Bayesian methods to estimate the lead time of the modality, the author explains how to screen for a disease among individuals that do not exhibit any symptoms of the disease. With many examples and end-of-chapter exercises, this book is the first to introduce epidemiology from a Bayesian perspective. It shows epidemiologists how these Bayesian models and techniques are useful in studying the association between disease and exposure to risk factors.

Statistical Evaluation of Diagnostic Performance

Author: Kelly H. Zou
Publisher: CRC Press
ISBN: 9781439812235
Release Date: 2016-04-19
Genre: Mathematics

Statistical evaluation of diagnostic performance in general and Receiver Operating Characteristic (ROC) analysis in particular are important for assessing the performance of medical tests and statistical classifiers, as well as for evaluating predictive models or algorithms. This book presents innovative approaches in ROC analysis, which are relevant to a wide variety of applications, including medical imaging, cancer research, epidemiology, and bioinformatics. Statistical Evaluation of Diagnostic Performance: Topics in ROC Analysis covers areas including monotone-transformation techniques in parametric ROC analysis, ROC methods for combined and pooled biomarkers, Bayesian hierarchical transformation models, sequential designs and inferences in the ROC setting, predictive modeling, multireader ROC analysis, and free-response ROC (FROC) methodology. The book is suitable for graduate-level students and researchers in statistics, biostatistics, epidemiology, public health, biomedical engineering, radiology, medical imaging, biomedical informatics, and other closely related fields. Additionally, clinical researchers and practicing statisticians in academia, industry, and government could benefit from the presentation of such important and yet frequently overlooked topics.

Interval Censored Time to Event Data

Author: Ding-Geng (Din) Chen
Publisher: CRC Press
ISBN: 9781466504257
Release Date: 2012-07-19
Genre: Mathematics

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.

Bayesian Analysis Made Simple

Author: Phil Woodward
Publisher: CRC Press
ISBN: 9781439839553
Release Date: 2016-04-19
Genre: Mathematics

Although the popularity of the Bayesian approach to statistics has been growing for years, many still think of it as somewhat esoteric, not focused on practical issues, or generally too difficult to understand. Bayesian Analysis Made Simple is aimed at those who wish to apply Bayesian methods but either are not experts or do not have the time to create WinBUGS code and ancillary files for every analysis they undertake. Accessible to even those who would not routinely use Excel, this book provides a custom-made Excel GUI, immediately useful to those users who want to be able to quickly apply Bayesian methods without being distracted by computing or mathematical issues. From simple NLMs to complex GLMMs and beyond, Bayesian Analysis Made Simple describes how to use Excel for a vast range of Bayesian models in an intuitive manner accessible to the statistically savvy user. Packed with relevant case studies, this book is for any data analyst wishing to apply Bayesian methods to analyze their data, from professional statisticians to statistically aware scientists.

Sample Size Calculations in Clinical Research

Author: Shein-Chung Chow
Publisher: CRC Press
ISBN: 9780824748234
Release Date: 2003-03-04
Genre: Mathematics

Sample size calculation plays an important role in clinical research. It is not uncommon, however, to observe discrepancies among study objectives (or hypotheses), study design, statistical analysis (or test statistic), and sample size calculation. Focusing on sample size calculation for studies conducted during the various phases of clinical research and development, Sample Size Calculation in Clinical Research explores the causes of discrepancies and how to avoid them. This volume provides formulas and procedures for determination of sample size required not only for testing equality, but also for testing non-inferiority/superiority, and equivalence (similarity) based on both untransformed (raw) data and log-transformed data under a parallel-group design or a crossover design with equal or unequal ratio of treatment allocations. It contains a comprehensive and unified presentation of statistical procedures for sample size calculation that are commonly employed at various phases of clinical development. Each chapter includes, whenever possible, real examples of clinical studies from therapeutic areas such as cardiovascular, central nervous system, anti-infective, oncology, and women's health to demonstrate the clinical and statistical concepts, interpretations, and their relationships and interactions. The book highlights statistical procedures for sample size calculation and justification that are commonly employed in clinical research and development. It provides clear, illustrated explanations of how the derived formulas and/or statistical procedures can be used.

Adaptive Design Theory and Implementation Using SAS and R Second Edition

Author: Mark Chang
Publisher: CRC Press
ISBN: 9781482256598
Release Date: 2014-12-01
Genre: Mathematics

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.

Bayesian Modeling in Bioinformatics

Author: Dipak K. Dey
Publisher: CRC Press
ISBN: 1420070185
Release Date: 2010-09-03
Genre: Mathematics

Bayesian Modeling in Bioinformatics discusses the development and application of Bayesian statistical methods for the analysis of high-throughput bioinformatics data arising from problems in molecular and structural biology and disease-related medical research, such as cancer. It presents a broad overview of statistical inference, clustering, and classification problems in two main high-throughput platforms: microarray gene expression and phylogenic analysis. The book explores Bayesian techniques and models for detecting differentially expressed genes, classifying differential gene expression, and identifying biomarkers. It develops novel Bayesian nonparametric approaches for bioinformatics problems, measurement error and survival models for cDNA microarrays, a Bayesian hidden Markov modeling approach for CGH array data, Bayesian approaches for phylogenic analysis, sparsity priors for protein-protein interaction predictions, and Bayesian networks for gene expression data. The text also describes applications of mode-oriented stochastic search algorithms, in vitro to in vivo factor profiling, proportional hazards regression using Bayesian kernel machines, and QTL mapping. Focusing on design, statistical inference, and data analysis from a Bayesian perspective, this volume explores statistical challenges in bioinformatics data analysis and modeling and offers solutions to these problems. It encourages readers to draw on the evolving technologies and promote statistical development in this area of bioinformatics.

Elementary Bayesian Biostatistics

Author: Lemuel A. Moyé
Publisher: CRC Press
ISBN: 9781584887256
Release Date: 2016-04-19
Genre: Mathematics

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.

Bayesian Biostatistics and Diagnostic Medicine

Author: Lyle D. Broemeling
Publisher: CRC Press
ISBN: 1584887680
Release Date: 2007-07-12
Genre: Mathematics

There are numerous advantages to using Bayesian methods in diagnostic medicine, which is why they are employed more and more today in clinical studies. Exploring Bayesian statistics at an introductory level, Bayesian Biostatistics and Diagnostic Medicine illustrates how to apply these methods to solve important problems in medicine and biology. After focusing on the wide range of areas where diagnostic medicine is used, the book introduces Bayesian statistics and the estimation of accuracy by sensitivity, specificity, and positive and negative predictive values for ordinal and continuous diagnostic measurements. The author then discusses patient covariate information and the statistical methods for estimating the agreement among observers. The book also explains the protocol review process for cancer clinical trials, how tumor responses are categorized, how to use WHO and RECIST criteria, and how Bayesian sequential methods are employed to monitor trials and estimate sample sizes. With many tables and figures, this book enables readers to conduct a Bayesian analysis for a large variety of interesting and practical biomedical problems.

Bayesian Adaptive Methods for Clinical Trials

Author: Scott M. Berry
Publisher: CRC Press
ISBN: 1439825513
Release Date: 2010-07-19
Genre: Mathematics

Already popular in the analysis of medical device trials, adaptive Bayesian designs are increasingly being used in drug development for a wide variety of diseases and conditions, from Alzheimer’s disease and multiple sclerosis to obesity, diabetes, hepatitis C, and HIV. Written by leading pioneers of Bayesian clinical trial designs, Bayesian Adaptive Methods for Clinical Trials explores the growing role of Bayesian thinking in the rapidly changing world of clinical trial analysis. The book first summarizes the current state of clinical trial design and analysis and introduces the main ideas and potential benefits of a Bayesian alternative. It then gives an overview of basic Bayesian methodological and computational tools needed for Bayesian clinical trials. With a focus on Bayesian designs that achieve good power and Type I error, the next chapters present Bayesian tools useful in early (Phase I) and middle (Phase II) clinical trials as well as two recent Bayesian adaptive Phase II studies: the BATTLE and ISPY-2 trials. In the following chapter on late (Phase III) studies, the authors emphasize modern adaptive methods and seamless Phase II–III trials for maximizing information usage and minimizing trial duration. They also describe a case study of a recently approved medical device to treat atrial fibrillation. The concluding chapter covers key special topics, such as the proper use of historical data, equivalence studies, and subgroup analysis. For readers involved in clinical trials research, this book significantly updates and expands their statistical toolkits. The authors provide many detailed examples drawing on real data sets. The R and WinBUGS codes used throughout are available on supporting websites. Scott Berry talks about the book on the CRC Press YouTube Channel.

Medical Product Safety Evaluation

Author: Jie Chen
Publisher: CRC Press
ISBN: 9781351021968
Release Date: 2018-08-31
Genre: Mathematics

Medical Product Safety Evaluation: Biological Models and Statistical Methods presents cutting-edge biological models and statistical methods that are tailored to specific objectives and data types for safety analysis and benefit-risk assessment. Some frequently encountered issues and challenges in the design and analysis of safety studies are discussed with illustrative applications and examples. Medical Product Safety Evaluation: Biological Models and Statistical Methods presents cutting-edge biological models and statistical methods that are tailored to specific objectives and data types for safety analysis and benefit-risk assessment. Some frequently encountered issues and challenges in the design and analysis of safety studies are discussed with illustrative applications and examples. The book is designed not only for biopharmaceutical professionals, such as statisticians, safety specialists, pharmacovigilance experts, and pharmacoepidemiologists, who can use the book as self-learning materials or in short courses or training programs, but also for graduate students in statistics and biomedical data science for a one-semester course. Each chapter provides supplements and problems as more readings and exercises.

Bayesian Designs for Phase I II Clinical Trials

Author: Ying Yuan
Publisher: CRC Press
ISBN: 9781498709569
Release Date: 2016-06-08
Genre: Mathematics

Reliably optimizing a new treatment in humans is a critical first step in clinical evaluation since choosing a suboptimal dose or schedule may lead to failure in later trials. At the same time, if promising preclinical results do not translate into a real treatment advance, it is important to determine this quickly and terminate the clinical evaluation process to avoid wasting resources. Bayesian Designs for Phase I–II Clinical Trials describes how phase I–II designs can serve as a bridge or protective barrier between preclinical studies and large confirmatory clinical trials. It illustrates many of the severe drawbacks with conventional methods used for early-phase clinical trials and presents numerous Bayesian designs for human clinical trials of new experimental treatment regimes. The first two chapters minimize the technical language to make them accessible to non-statisticians. These chapters discuss the severe drawbacks of the conventional paradigm used for early-phase clinical trials and explain the phase I–II paradigm for optimizing dose, or more general treatment regimes, based on both efficacy and toxicity. The remainder of the book covers a wide variety of clinical trial methodologies, including designs to optimize the dose pair of a two-drug combination, jointly optimize dose and schedule, identify optimal personalized doses, optimize novel molecularly targeted agents, and choose doses in two treatment cycles. Written by research leaders from the University of Texas MD Anderson Cancer Center, this book shows how Bayesian designs for early-phase clinical trials can explore, refine, and optimize new experimental treatments. It emphasizes the importance of basing decisions on both efficacy and toxicity.

Design and Analysis of Clinical Trials with Time to Event Endpoints

Author: Karl E. Peace
Publisher: CRC Press
ISBN: 9781420066401
Release Date: 2009-04-23
Genre: Mathematics

Using time-to-event analysis methodology requires careful definition of the event, censored observation, provision of adequate follow-up, number of events, and independence or "noninformativeness" of the censoring mechanisms relative to the event. Design and Analysis of Clinical Trials with Time-to-Event Endpoints provides a thorough presentation of the design, monitoring, analysis, and interpretation of clinical trials in which time-to-event is of critical interest. After reviewing time-to-event endpoint methodology, clinical trial issues, and the design and monitoring of clinical trials, the book focuses on inferential analysis methods, including parametric, semiparametric, categorical, and Bayesian methods; an alternative to the Cox model for small samples; and estimation and testing for change in hazard. It then presents descriptive and graphical methods useful in the analysis of time-to-event endpoints. The next several chapters explore a variety of clinical trials, from analgesic, antibiotic, and antiviral trials to cardiovascular and cancer prevention, prostate cancer, astrocytoma brain tumor, and chronic myelogonous leukemia trials. The book then covers areas of drug development, medical practice, and safety assessment. It concludes with the design and analysis of clinical trials of animals required by the FDA for new drug applications. Drawing on the expert contributors’ experiences working in biomedical research and clinical drug development, this comprehensive resource covers an array of time-to-event methods and explores an assortment of real-world applications.

Exercises and Solutions in Biostatistical Theory

Author: Lawrence Kupper
Publisher: CRC Press
ISBN: 9781439895023
Release Date: 2010-11-09
Genre: Mathematics

Drawn from nearly four decades of Lawrence L. Kupper’s teaching experiences as a distinguished professor in the Department of Biostatistics at the University of North Carolina, Exercises and Solutions in Biostatistical Theory presents theoretical statistical concepts, numerous exercises, and detailed solutions that span topics from basic probability to statistical inference. The text links theoretical biostatistical principles to real-world situations, including some of the authors’ own biostatistical work that has addressed complicated design and analysis issues in the health sciences. This classroom-tested material is arranged sequentially starting with a chapter on basic probability theory, followed by chapters on univariate distribution theory and multivariate distribution theory. The last two chapters on statistical inference cover estimation theory and hypothesis testing theory. Each chapter begins with an in-depth introduction that summarizes the biostatistical principles needed to help solve the exercises. Exercises range in level of difficulty from fairly basic to more challenging (identified with asterisks). By working through the exercises and detailed solutions in this book, students will develop a deep understanding of the principles of biostatistical theory. The text shows how the biostatistical theory is effectively used to address important biostatistical issues in a variety of real-world settings. Mastering the theoretical biostatistical principles described in the book will prepare students for successful study of higher-level statistical theory and will help them become better biostatisticians.