Bayesian Methods and Ethics in a Clinical Trial Design

A balanced treatment of the theories, methodologies, and design issues involved in clinical trials using statistical methods There has been enormous interest and development in Bayesian adaptive designs, especially for early phases of clinical trials. However, for phase III trials, frequentist methods still play a dominant role through controlling type I and type II errors in the hypothesis testing framework. From practical perspectives, Clinical Trial Design: Bayesian and Frequentist Adaptive Methods provides comprehensive coverage of both Bayesian and frequentist approaches to all phases of clinical trial design. Before underpinning various adaptive methods, the book establishes an overview of the fundamentals of clinical trials as well as a comparison of Bayesian and frequentist statistics. Recognizing that clinical trial design is one of the most important and useful skills in the pharmaceutical industry, this book provides detailed discussions on a variety of statistical designs, their properties, and operating characteristics for phase I, II, and III clinical trials as well as an introduction to phase IV trials. Many practical issues and challenges arising in clinical trials are addressed. Additional topics of coverage include: Risk and benefit analysis for toxicity and efficacy trade-offs Bayesian predictive probability trial monitoring Bayesian adaptive randomization Late onset toxicity and response Dose finding in drug combination trials Targeted therapy designs The author utilizes cutting-edge clinical trial designs and statistical methods that have been employed at the world's leading medical centers as well as in the pharmaceutical industry. The software used throughout the book is freely available on the book's related website, equipping readers with the necessary tools for designing clinical trials. Clinical Trial Design is an excellent book for courses on the topic at the graduate level. The book also serves as a valuable reference for statisticians and biostatisticians in the pharmaceutical industry as well as for researchers and practitioners who design, conduct, and monitor clinical trials in their everyday work.

A balanced treatment of the theories, methodologies, and design issues involved in clinical trials using statistical methods There has been enormous interest and development in Bayesian adaptive designs, especially for early phases of clinical trials. However, for phase III trials, frequentist methods still play a dominant role through controlling type I and type II errors in the hypothesis testing framework. From practical perspectives, Clinical Trial Design: Bayesian and Frequentist Adaptive Methods provides comprehensive coverage of both Bayesian and frequentist approaches to all phases of clinical trial design. Before underpinning various adaptive methods, the book establishes an overview of the fundamentals of clinical trials as well as a comparison of Bayesian and frequentist statistics. Recognizing that clinical trial design is one of the most important and useful skills in the pharmaceutical industry, this book provides detailed discussions on a variety of statistical designs, their properties, and operating characteristics for phase I, II, and III clinical trials as well as an introduction to phase IV trials. Many practical issues and challenges arising in clinical trials are addressed. Additional topics of coverage include: Risk and benefit analysis for toxicity and efficacy trade-offs Bayesian predictive probability trial monitoring Bayesian adaptive randomization Late onset toxicity and response Dose finding in drug combination trials Targeted therapy designs The author utilizes cutting-edge clinical trial designs and statistical methods that have been employed at the world's leading medical centers as well as in the pharmaceutical industry. The software used throughout the book is freely available on the book's related website, equipping readers with the necessary tools for designing clinical trials. Clinical Trial Design is an excellent book for courses on the topic at the graduate level. The book also serves as a valuable reference for statisticians and biostatisticians in the pharmaceutical industry as well as for researchers and practitioners who design, conduct, and monitor clinical trials in their everyday work.

HIGHLIGHTS THE USE OF BAYESIAN STATISTICS TO GAIN INSIGHTS FROM EMPIRICAL DATA Featuring an accessible approach, Bayesian Methods for Management and Business: Pragmatic Solutions for Real Problems demonstrates how Bayesian statistics can help to provide insights into important issues facing business and management. The book draws on multidisciplinary applications and examples and utilizes the freely available software WinBUGS and R to illustrate the integration of Bayesian statistics within data-rich environments. Computational issues are discussed and integrated with coverage of linear models, sensitivity analysis, Markov Chain Monte Carlo (MCMC), and model comparison. In addition, more advanced models including hierarchal models, generalized linear models, and latent variable models are presented to further bridge the theory and application in real-world usage. Bayesian Methods for Management and Business: Pragmatic Solutions for Real Problems also features: Numerous real-world examples drawn from multiple management disciplines such as strategy, international business, accounting, and information systems An incremental skill-building presentation based on analyzing data sets with widely applicable models of increasing complexity An accessible treatment of Bayesian statistics that is integrated with a broad range of business and management issues and problems A practical problem-solving approach to illustrate how Bayesian statistics can help to provide insight into important issues facing business and management Bayesian Methods for Management and Business: Pragmatic Solutions for Real Problems is an important textbook for Bayesian statistics courses at the advanced MBA-level and also for business and management PhD candidates as a first course in methodology. In addition, the book is a useful resource for management scholars and practitioners as well as business academics and practitioners who seek to broaden their methodological skill sets.

This book aims to fill the gap that exists between theoretical treatments of chromatography, and clinical chemistry and toxicology texts, which focus almost exclusively on clinical relevance and applications. Chromatography has a vast array of clinical applications, and though the chromatographic methods were first introduced decades ago, new applications of this technology are being used to explore previously inaccessible frontiers in clinical diagnostics and toxicological testing. An up-to-date book devoted to clinical and toxicological applications of chromatographic methods will serve as an instructional and reference text, useful to students, laboratory technicians, and researchers.

A complete guide to the key statistical concepts essential for the design and construction of clinical trials As the newest major resource in the field of medical research, Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs presents a timely and authoritative reviewof the central statistical concepts used to build clinical trials that obtain the best results. The referenceunveils modern approaches vital to understanding, creating, and evaluating data obtained throughoutthe various stages of clinical trial design and analysis. Accessible and comprehensive, the first volume in a two-part set includes newly-written articles as well as established literature from the Wiley Encyclopedia of Clinical Trials. Illustrating a variety of statistical concepts and principles such as longitudinal data, missing data, covariates, biased-coin randomization, repeated measurements, and simple randomization, the book also provides in-depth coverage of the various trial designs found within phase I-IV trials. Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs also features: Detailed chapters on the type of trial designs, such as adaptive, crossover, group-randomized, multicenter, non-inferiority, non-randomized, open-labeled, preference, prevention, and superiority trials Over 100 contributions from leading academics, researchers, and practitioners An exploration of ongoing, cutting-edge clinical trials on early cancer and heart disease, mother-to-child human immunodeficiency virus transmission trials, and the AIDS Clinical Trials Group Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs is an excellent reference for researchers, practitioners, and students in the fields of clinicaltrials, pharmaceutics, biostatistics, medical research design, biology, biomedicine, epidemiology,and public health.

Bayesian methods are a powerful tool in many areas of science and engineering, especially statistical physics, medical sciences, electrical engineering, and information sciences. They are also ideal for civil engineering applications, given the numerous types of modeling and parametric uncertainty in civil engineering problems. For example, earthquake ground motion cannot be predetermined at the structural design stage. Complete wind pressure profiles are difficult to measure under operating conditions. Material properties can be difficult to determine to a very precise level – especially concrete, rock, and soil. For air quality prediction, it is difficult to measure the hourly/daily pollutants generated by cars and factories within the area of concern. It is also difficult to obtain the updated air quality information of the surrounding cities. Furthermore, the meteorological conditions of the day for prediction are also uncertain. These are just some of the civil engineering examples to which Bayesian probabilistic methods are applicable. Familiarizes readers with the latest developments in the field Includes identification problems for both dynamic and static systems Addresses challenging civil engineering problems such as modal/model updating Presents methods applicable to mechanical and aerospace engineering Gives engineers and engineering students a concrete sense of implementation Covers real-world case studies in civil engineering and beyond, such as: structural health monitoring seismic attenuation finite-element model updating hydraulic jump artificial neural network for damage detection air quality prediction Includes other insightful daily-life examples Companion website with MATLAB code downloads for independent practice Written by a leading expert in the use of Bayesian methods for civil engineering problems This book is ideal for researchers and graduate students in civil and mechanical engineering or applied probability and statistics. Practicing engineers interested in the application of statistical methods to solve engineering problems will also find this to be a valuable text. MATLAB code and lecture materials for instructors available at http://www.wiley.com/go/yuen

The focus of this book is on «ill-posed inverse problems». These problems cannot be solved only on the basis of observed data. The building of solutions involves the recognition of other pieces of a priori information. These solutions are then specific to the pieces of information taken into account. Clarifying and taking these pieces of information into account is necessary for grasping the domain of validity and the field of application for the solutions built. For too long, the interest in these problems has remained very limited in the signal-image community. However, the community has since recognized that these matters are more interesting and they have become the subject of much greater enthusiasm. From the application field’s point of view, a significant part of the book is devoted to conventional subjects in the field of inversion: biological and medical imaging, astronomy, non-destructive evaluation, processing of video sequences, target tracking, sensor networks and digital communications. The variety of chapters is also clear, when we examine the acquisition modalities at stake: conventional modalities, such as tomography and NMR, visible or infrared optical imaging, or more recent modalities such as atomic force imaging and polarized light imaging.

Methods and Applications of Statistics in Clinical Trials, Volume 2: Planning, Analysis, and Inferential Methods includes updates of established literature from the Wiley Encyclopedia of Clinical Trials as well as original material based on the latest developments in clinical trials. Prepared by a leading expert, the second volume includes numerous contributions from current prominent experts in the field of medical research. In addition, the volume features: • Multiple new articles exploring emerging topics, such as evaluation methods with threshold, empirical likelihood methods, nonparametric ROC analysis, over- and under-dispersed models, and multi-armed bandit problems • Up-to-date research on the Cox proportional hazard model, frailty models, trial reports, intrarater reliability, conditional power, and the kappa index • Key qualitative issues including cost-effectiveness analysis, publication bias, and regulatory issues, which are crucial to the planning and data management of clinical trials

A practical «how to» guide for multiple methods in metabolism, with a critical and objective discussion of strengths, limitations, and appropriate applications of the described methods. Edited by the winner of the Oskar-Minkowski Prize of the EASD in 2006 Clinical trials in populations at risk of or with overt diabetes mellitus are being performed all around the world to test novel drugs and approaches to managing these diseases. During the last decade, new methods and techniques have been introduced – and are being developed further – that facilitate monitoring of metabolism and even diagnosis of certain defects in human metabolism. While some of these methods are restricted to research facilities, others are already used in clinical practice. The correct beneficial use of these methods requires knowledge of the nature of the techniques and their clinical relevance. Clinical Diabetes Research: Methods and Techniques provides summaries of established state-of-the art-techniques and introduces the new technologies, evaluating their potential in clinical diabetes research. One section compares the efficacy of all metabolic tests that measure insulin action. Another section describes methods that allow quantification of whole body and intracellular metabolic fluxes relevant to diabetes research. The final section focuses on the planning and pitfalls of clinical studies in the field of metabolism. Each chapter throughout the book combines the theoretical basis of the approach with practical recommendations. There are further chapters on the design of clinical trials and ethical considerations. The publication is timely in view of the impressive development of the methods and techniques with their application to understanding the pathophysiology of diabetes, insulin resistance syndromes, obesity, and the metabolic effects of treatment. Clinical Diabetes Research: Methods and Techniques is of great relevance for researchers (from students and post-docs to principal investigators) in the field of human metabolism, clinical endocrinologists and diabetologists who are involved in clinical studies.

The Tutorials in Biostatistics have become a very popular feature of the prestigious Wiley journal, Statistics in Medicine (SIM). The introductory style and practical focus make them accessible to a wide audience including medical practitioners with limited statistical knowledge. This book represents the first of two volumes presenting the best tutorials published in SIM, focusing on statistical methods in clinical studies. Topics include the design and analysis of clinical trials, epidemiology, survival analysis, and data monitoring. Each tutorial is focused on a medical problem, has been fully peer-reviewed and edited, and is authored by leading researchers in biostatistics. Many articles include an appendix on the latest developments since publication in the journal and additional references. This will appeal to statisticians working in medical research, as well as statistically-minded clinicians, biologists, epidemiologists and geneticists. It will also appeal to graduate students of biostatistics.