Praise for the Second Edition: “…a grand feast for biostatisticians. It stands ready to satisfy the appetite of any pharmaceutical scientist with a respectable statistical appetite.” —Journal of Clinical Research Best Practices The Third Edition of Design and Analysis of Clinical Trials provides complete, comprehensive, and expanded coverage of recent health treatments and interventions. Featuring a unified presentation, the book provides a well-balanced summary of current regulatory requirements and recently developed statistical methods as well as an overview of the various designs and analyses that are utilized at different stages of clinical research and development. Additional features of this Third Edition include: • New chapters on biomarker development and target clinical trials, adaptive design, trials for evaluating diagnostic devices, statistical methods for translational medicine, and traditional Chinese medicine • A balanced overview of current and emerging clinical issues as well as newly developed statistical methodologies • Practical examples of clinical trials that demonstrate everyday applicability, with illustrations and examples to explain key concepts • New sections on bridging studies and global trials, QT studies, multinational trials, comparative effectiveness trials, and the analysis of QT/QTc prolongation • A complete and balanced presentation of clinical and scientific issues, statistical concepts, and methodologies for bridging clinical and statistical disciplines • An update of each chapter that reflects changes in regulatory requirements for the drug review and approval process and recent developments in statistical design and methodology for clinical research and development Design and Analysis of Clinical Trials, Third Edition continues to be an ideal clinical research reference for academic, pharmaceutical, medical, and regulatory scientists/researchers, statisticians, and graduate-level students.
A systematic approach to all aspects of designing and conducting clinical trials The success or failure of clinical trials hinges on hundreds of details that need to be developed, often under less than ideal conditions. Written by one of the world's leading trialists, Clinical Trials Handbook: Design and Conduct provides clinicians with a complete guide to designing, conducting, and evaluating clinical trials—teaching them how to simplify the process and avoid costly mistakes. The author draws on his extensive clinical trials experience to outline all steps employed in setting up and running clinical trials, from budgeting and fundraising to publishing the results. Along the way, practical advice is offered while also addressing a mix of logistical, ethical, psychological, behavioral, and administrative issues inherent to clinical trials. Topics of coverage include: Protocols for drug masking, controls, and treatment randomization Consent, enrollment, eligibility, and follow-up procedures Different types of sample size design and data collection and processing Working with study centers, research staff, and various committees Monitoring treatment effects and performance, and ensuring quality control Data analysis and access policies for study data and documents Clinical Trials Handbook is invaluable for practicing clinicians and trialists who would like to learn more about or improve their understanding of the design and execution of clinical trials. The book is also an excellent supplement for courses on clinical trials at the graduate level.
Provides an introduction to the various statistical techniques involved in medical research and drug development with a focus on estimating the success probability of an experiment Success Probability Estimation with Applications to Clinical Trials details the use of success probability estimation in both the planning and analyzing of clinical trials and in widely used statistical tests. Devoted to both statisticians and non-statisticians who are involved in clinical trials, Part I of the book presents new concepts related to success probability estimation and their usefulness in clinical trials, and each section begins with a non-technical explanation of the presented concepts. Part II delves deeper into the techniques for success probability estimation and features applications to both reproducibility probability estimation and conservative sample size estimation. Success Probability Estimation with Applications to Clinical Trials: • Addresses the theoretical and practical aspects of the topic and introduces new and promising techniques in the statistical and pharmaceutical industries Features practical solutions for problems that are often encountered in clinical trials Includes success probability estimation for widely used statistical tests, such as parametric and nonparametric models Focuses on experimental planning, specifically the sample size of clinical trials using phase II results and data for planning phase III trials Introduces statistical concepts related to success probability estimation and their usefulness in clinical trials Success Probability Estimation with Applications to Clinical Trials is an ideal reference for statisticians and biostatisticians in the pharmaceutical industry as well as researchers and practitioners in medical centers who are actively involved in health policy, clinical research, and the design and evaluation of clinical trials.
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.
An accessible and practical approach to the design and analysis of experiments in the health sciences Design and Analysis of Experiments in the Health Sciences provides a balanced presentation of design and analysis issues relating to data in the health sciences and emphasizes new research areas, the crucial topic of clinical trials, and state-of-the- art applications. Advancing the idea that design drives analysis and analysis reveals the design, the book clearly explains how to apply design and analysis principles in animal, human, and laboratory experiments while illustrating topics with applications and examples from randomized clinical trials and the modern topic of microarrays. The authors outline the following five types of designs that form the basis of most experimental structures: Completely randomized designs Randomized block designs Factorial designs Multilevel experiments Repeated measures designs A related website features a wealth of data sets that are used throughout the book, allowing readers to work hands-on with the material. In addition, an extensive bibliography outlines additional resources for further study of the presented topics. Requiring only a basic background in statistics, Design and Analysis of Experiments in the Health Sciences is an excellent book for introductory courses on experimental design and analysis at the graduate level. The book also serves as a valuable resource for researchers in medicine, dentistry, nursing, epidemiology, statistical genetics, and public health.
A thoroughly updated new edition of the essential reference on the design, practice, and analysis of clinical trials Clinical Trials Dictionary: Terminology and Usage Recommendations, Second Edition presents clear, precise, meticulously detailed entries on all aspects of modern-day clinical trials. Written and compiled by one of the world’s leading clinical trialists, this comprehensive volume incorporates areas of medicine, statistics, epidemiology, computer science, and bioethics—providing a treasure trove of key terms and ideas. This new edition continues to supply readers with the A–Z terminology needed to design, conduct, and analyze trials, introducing a vocabulary for the characterization and description of related features and activities. More than 300 new entries are now included, reflecting the current usage practices and conventions in the field, along with usage notes with recommendations on when to use the term in question. Detailed biographical notes highlight prominent historical figures and institutions in the field, and an extensive bibliography has been updated to provide readers with additional resources for further study. The most up-to-date work of its kind, Clinical Trials Dictionary, Second Edition is an essential reference for anyone who needs to report on, index, analyze, or assess the scientific strength and validity of clinical trials.
Using examples and case studies from industry, academia and research literature, Randomized Clinical Trials provides a detailed overview of the key issues involved in designing, conducting, analysing and reporting randomized clinical trials. It examines the methodology for conducting Phase III clinical trials, developing the protocols, the practice for capturing, measuring, and analysing the resulting clinical data and their subsequent reporting. Randomized clinical trials are the principal method for determining the relative efficacy and safety of alternative treatments, interventions or medical devices. They are conducted by groups comprising one or more of pharmaceutical and allied health-care organisations, academic institutions, and charity supported research groups. In many cases such trials provide the key evidence necessary for the regulatory approval of a new product for future patient use. Randomized Clinical Trials provides comprehensive coverage of such trials, ranging from elementary to advanced level. Written by authors with considerable experience of clinical trials, Randomized Clinical Trials is an authoritative guide for clinicians, nurses, data managers and medical statisticians involved in clinical trials research and for health care professionals directly involved in patient care in a clinical trial context.
Clinical trials have revolutionized the way disease is prevented, detected and treated, and early death avoided, and they continue to be an expanding area of research. They are central to the work of pharmaceutical companies, and there are many academic and public sector organizations that conduct trials on a wide variety of interventions, including drugs, devices, surgical techniques, and changes in behaviour and lifestyle. A Concise Guide to Clinical Trials provides a comprehensive yet easy-to-read overview of the design, conduct and analysis of trials. It requires no prior knowledge on the subject as the important concepts are introduced throughout. There are chapters that distinguish between the different types of trials, and an introduction to systematic reviews, health-related quality of life and health economic evaluation. The book also covers the ethical and legal requirements in setting up a clinical trial due to an increase in governance responsibilities and regulations. This practical guidebook is ideal for busy clinicians and other health professionals who do not have enough time to attend courses or search through extensive textbooks. It will help anyone involved in undertaking clinical research, or those reading about trials. The book is aimed at: Those wishing to learn about clinical trials for the first time, or as a quick reference guide, for example as part of a taught course on clinical trials Health professionals who wish to conduct their own trials, or participate in other people’s studies People who work in pharmaceutical companies, grant funding organisations, or regulatory agencies
Clinical trials tasks and activities are widely diverse and require certain skill sets to both plan and execute. This book provides professionals in the field of clinical research with valuable information on the challenging issues of the design, execution, and management of clinical trials, and how to resolve these issues effectively. It discusses key obstacles such as challenges to patient recruitment, investigator and study site selection, and dealing with compliance issues. Through practical examples, professionals working with medical device clinical trials will discover the appropriate steps to take.
An essential introduction to conducting the various stages of medical device clinical trials Clinical research continues to be one of the most vital components of pharmaceutical, biostatistical, and medical studies. Design, Execution, and Management of Medical Device Clinical Trials provides a uniform methodology for conducting and managing clinical trials. Written in a style that is accessible to readers from diverse educational and professional backgrounds, this book provides an in-depth and broad overview for successfully performing clinical tasks and activities. Throughout the book, practical examples compiled from both the author's and other researchers' previous clinical trial experiences are discussed in a sequential manner as they occur in the study, starting from the development of the clinical protocol and the selection of clinical sites and ending with the completion of the final clinical study report. Next, readers are guided through the development of important clinical documents, including informed consent forms, case report forms, and study logs. A careful review of the Food and Drug Administration (FDA) and International Conference on Harmonisation (ICH) regulations applicable to medical devices is also featured. Additional coverage includes: Qualification and selection of investigators Study monitoring visits Definitions and reporting procedures for adverse events The use of biostatistical methodology in clinical research, including the use of biostatistics for sample size determination and study endpoints The roles and responsibilities of all members of a clinical research team The book concludes with an insightful discussion of special ethical conduct for human research and challenging issues to consider during the design of clinical studies. A glossary lists important clinical and statistical terms used in clinical research, and an extensive reference section provides additional resources for the most up-to-date literature on the topic. Design, Execution, and Management of Medical Device Clinical Trials is an excellent book for clinical research or epidemiology courses at the upper-undergraduate and graduate levels. It is also an indispensable reference for clinical research associates, clinical managers, clinical scientists, biostatisticians, pharmacologists, and any professional working in the field of clinical research who would like to better understand clinical research practices.
Best practices for conducting effective and safe clinical trials Clinical trials are arguably the most important steps in proving drug effectiveness and safety for public use. They require intensive planning and organization and involve a wide range of disciplines: data management, biostatistics, pharmacology, toxicology, modeling and simulation, regulatory monitoring, ethics, and particular issues for given disease areas. Clinical Trials Handbook provides a comprehensive and thorough reference on the basics and practices of clinical trials. With contributions from a range of international authors, the book takes the reader through each trial phase, technique, and issue. Chapters cover every key aspect of preparing and conducting clinical trials, including: Interdisciplinary topics that have to be coordinated for a successful clinical trialData management (and adverse event reporting systems) Biostatistics, pharmacology, and toxicology Modeling and simulation Regulatory monitoring and ethics Particular issues for given disease areas-cardiology, oncology, cognitive, dementia, dermatology, neuroscience, and more With unique information on such current issues as adverse event reporting (AER) systems, adaptive trial designs, and crossover trial designs, Clinical Trials Handbook will be a ready reference for pharmaceutical scientists, statisticians, researchers, and the many other professionals involved in drug development.
Provides timely applications, modifications, and extensions of experimental designs for a variety of disciplines Design and Analysis of Experiments, Volume 3: Special Designs and Applications continues building upon the philosophical foundations of experimental design by providing important, modern applications of experimental design to the many fields that utilize them. The book also presents optimal and efficient designs for practice and covers key topics in current statistical research. Featuring contributions from leading researchers and academics, the book demonstrates how the presented concepts are used across various fields from genetics and medicinal and pharmaceutical research to manufacturing, engineering, and national security. Each chapter includes an introduction followed by the historical background as well as in-depth procedures that aid in the construction and analysis of the discussed designs. Topical coverage includes: Genetic cross experiments, microarray experiments, and variety trials Clinical trials, group-sequential designs, and adaptive designs Fractional factorial and search, choice, and optimal designs for generalized linear models Computer experiments with applications to homeland security Robust parameter designs and split-plot type response surface designs Analysis of directional data experiments Throughout the book, illustrative and numerical examples utilize SAS®, JMP®, and R software programs to demonstrate the discussed techniques. Related data sets and software applications are available on the book's related FTP site. Design and Analysis of Experiments, Volume 3 is an ideal textbook for graduate courses in experimental design and also serves as a practical, hands-on reference for statisticians and researchers across a wide array of subject areas, including biological sciences, engineering, medicine, and business.
This book gives professionals in clinical research valuable information on the challenging issues of the design, execution, and management of clinical trials, and how to resolve these issues effectively. It also provides understanding and practical guidance on the application of contemporary statistical methods to contemporary issues in safety evaluation during medical product development. Each chapter provides sufficient detail to the reader to undertake the design and analysis of experiments at various stages of product development, including comprehensive references to the relevant literature. Provides a guide to statistical methods and application in medical product development Assists readers in undertaking design and analysis of experiments at various stages of product development Features case studies throughout the book, as well as, SAS and R code
Facial Aesthetics: Concepts and Clinical Diagnosis is a unique new illustrated resource for facial aesthetic surgery and dentistry, providing the comprehensive clinical textbook on the art and science of facial aesthetics for clinicians involved in the management of facial deformities, including orthodontists, oral and maxillofacial surgeons, plastic and reconstructive surgeons and aesthetic dentists. It aims to provide readers with a comprehensive examination of facial aesthetics in the context of dentofacial and craniofacial diagnosis and treatment planning. This aim is achieved through coupling meticulous research and practical clinical advice with beautifully drawn supporting illustrations and diagrams. Structured over 24 logically arranged and easy-to-follow chapters, Part I of Facial Aesthetics covers the historical evidence for facial aesthetic canons and concepts in depth. It incorporates all aspects relevant to the work of the clinician, including the philosophical and scientific theories of facial beauty, facial attractiveness research, facial expression and the psychosocial ramifications of facial deformities. Part II of the book then goes on to examine clinical evaluation and diagnosis in considerable detail under four sections, from the initial consultation interview and acquisition of diagnostic records (section 1), complete clinical examination and analysis of the craniofacial complex (section 2), in depth analysis of each individual facial region using a top-down approach (section 3) and finally focussing on smile and dentogingival aesthetic evaluation (section 4). An in-depth, thoughtful, practical and absorbing reference, Facial Aesthetics will find an enthusiastic reception among facial aesthetic surgeons and aesthetic dentists with an interest in refining their understanding and appreciation of the human face and applying practical protocols to their clinical diagnosis and treatment planning. Key features: Examines facial aesthetics in a clinical context Promotes an interdisciplinary approach to facial aesthetic analysis Detailed description of the systematic clinical evaluation of the facial soft tissues and craniodentoskeletal complex Detailed, step-by-step aesthetic analysis of each facial region In-depth analysis of 2D and 3D clinical diagnostic records Evidence-based approach, from antiquity to contemporary scientific evidence, to the guidelines employed in planning the correction of facial deformities Treatment planning from first principles highlighted Clinical notes are highlighted throughout Clearly organized and practical format Highly illustrated in full colour throughout