Data Monitoring Committees in Clinical Trials. A Practical Perspective

There has been substantial growth in the use of data monitoring committees in recent years, by both government agencies and the pharmaceutical industry. This growth has been brought about by increasing recognition of the value of such committees in safeguarding trial participants as well as protecting trial integrity and the validity of conclusions. This very timely book describes the operation of data monitoring committees, and provides an authoritative guide to their establishment, purpose and responsibilities. * Provides a practical overview of data monitoring in clinical trials. * Describes the purpose, responsibilities and operation of data monitoring committees. * Provides directly applicable advice for those managing and conducting clinical trials, and those serving on data monitoring committees. * Gives insight into clinical data monitoring to those sitting on regulatory and ethical committees. * Discusses issues pertinent to those working in clinical trials in both the US and Europe. The practical guidance provided by this book will be of use to professionals working in and/or managing clinical trials, in academic, government and industry settings, particularly medical statisticians, clinicians, trial co-ordinators, and those working in regulatory affairs and bioethics.

A valuable new edition of the trusted, practical guide to managing data in clinical trials Regardless of size, type, or complexity, accurate results for any clinical trial are ultimately determined by the quality of the collected data. Management of Data in Clinical Trials, Second Edition explores data management and trial organization as the keys to developing an accurate and reliable clinical trial. With a focus on the traditional aspects of data collection as well as recent advances in technology, this new edition provides a complete and accessible guide to the management structure of a clinical trial, from planning and development to design and analysis. Practical approaches that result in the collection of complete and timely data are also provided. While maintaining a comprehensive overview of the knowledge and tools that are essential for the organization of a modern clinical trial, the author has expanded the topical coverage in the Second Edition to reflect the possible uses of recent advances in technology in the data collection process. In addition, the Second Edition discusses the impact of international regulations governing the conduct of clinical trials and provides guidelines on ensuring compliance with national requirements. Newly featured topics include: The growing availability of «off-the-shelf» solutions for clinical trials Potential models for collaboration in the conduct of clinical trials between academia and the pharmaceutical industry The increasing use of the Internet in the collection of data and management of trials Regulatory requirements worldwide and compliance with the ICH Good Clinical Practice (GCP) Guidelines Development of Standard Operating Procedures for the conduct of clinical trials Complete with chapter summaries that reinforce key points as well as over one hundred examples, Management of Data in Clinical Trials, Second Edition is an ideal resource for practitioners in the clinical research community who are involved in the development of clinical trials, including data managers, research associates, data coordinators, physicians, and statisticians. This book also serves as an excellent supplemental text for courses in clinical trials at both the undergraduate and graduate levels.

This book provides practical guidance for statisticians, clinicians, and researchers involved in clinical trials in the biopharmaceutical industry, medical and public health organisations. Academics and students needing an introduction to handling missing data will also find this book invaluable. The authors describe how missing data can affect the outcome and credibility of a clinical trial, show by examples how a clinical team can work to prevent missing data, and present the reader with approaches to address missing data effectively. The book is illustrated throughout with realistic case studies and worked examples, and presents clear and concise guidelines to enable good planning for missing data. The authors show how to handle missing data in a way that is transparent and easy to understand for clinicians, regulators and patients. New developments are presented to improve the choice and implementation of primary and sensitivity analyses for missing data. Many SAS code examples are included – the reader is given a toolbox for implementing analyses under a variety of assumptions.

At last – a new edition of the highly acclaimed book Clinical Trials in Psychiatry This book provides a concise but thorough overview of clinical trials in psychiatry, invaluable to those seeking solutions to numerous problems relating to design, methodology and analysis of such trials. Practical examples and applications are used to ground theory whenever possible. The Second Edition includes new information regarding: Recent important psychiatric trials More specific discussion of psychiatry in the USA and the particular problems of trials in the USA, including comments about the FDA (U.S. Food and Drug Administration) An extended chapter on meta-analysis Further discussion of sub-group analysis Special features include appendices outlining how to design and report clinical trials, what websites and software programs are appropriate and an extensive reference section. From the reviews of the First Edition: “Everitt & Wessely are to be congratulated on producing an excellent guide to help overcome the snags in clinical trial research. Clearly written and in an engrossing style, the book is likely to become a classic textbook on clinical trials, and not just in psychiatry. The authors’ enthusiasm and grasp of clinical trial research make for a gripping and insightful read…it is one of the very best books that has been written on clinical trials.” THE BRITISH JOURNAL OF PSYCHIATRY "The experience of both authors in this area gives the book a very pragmatic approach grounded in reality, with theoretical overviews invariably being followed by practical examples and applications… an invaluable companion to anyone involved in, or contemplating undertaking, clinical trials research.” PSYCHOLOGICAL MEDICINE

A unique overview that melds the concepts of conditional probability and stochastic processes into real-life applications The role of randomization techniques in clinical trials has become increasingly important. This comprehensive guide combines both the applied aspects of randomization in clinical trials with a probabilistic treatment of properties of randomization. Taking an unabashedly non-Bayesian and nonparametric approach to inference, the book focuses on the linear rank test under a randomization model, with added discussion on likelihood-based inference as it relates to sufficiency and ancillarity. Developments in stochastic processes and applied probability are also given where appropriate. Intuition is stressed over mathematics, but not without a clear development of the latter in the context of the former. Providing a consolidated review of the field, the book includes relevant and practical discussions of: * The benefits of randomization in terms of reduction of bias * Randomization as a basis for inference * Covariate-adaptive and response-adaptive randomization * Current philosophies, controversies, and new developments With ample problem sets, theoretical exercises, and short computer simulations using SAS, Randomization in Clinical Trials: Theory and Practice is equally useful as a standard textbook in biostatistics graduate programs as well as a reliable reference for biostatisticians in practice.

Although clinical trials were virtually unheard of in psychiatry for many years, they are now the gold standard for judging whether drugs are safe and useful. But should they be? What is the true status of clinical trials? Even when they ostensibly demonstrate a benefit of a certain treatment, the strict patient selection criteria, poor compliance and high drop-out rate leave the conclusions open to question. Are the new treatments really better or more cost-effective than the old? Do they have fewer side effects? In this book the authors take a critical look at recent developments and present a series of trenchant and challenging observations. Section I examines the significant changes in law and the regulatory environment that have occurred during the past ten years. Has fossilization handicapped the US Food and Drug Administration in promoting treatment advances? How can the plethora of findings be regulated? This is particularly pertinent in genomic studies and there are two chapters addressing the impact of genomics on psychiatric research. This section also addresses the role of women in drug trials – a group long excluded but now demanding a part, for without testing how can optimal treatments be devised? The next two Sections highlight clinical trials in the major areas of psychiatric pharmacological treatment, including Mood Disorders, especially Bipolar, Anxiety Disorders, and addictions. Chapters on pharmacological treatments for Eating Disorders, Attention Deficit Disorder, Autism and Asperger’s Syndrome, and Impulse Control Disorder represent the latest thinking on these subjects. The final Section contains a consummate example of out-of-the [Western]– box thinking, namely consideration of herbal medicines – used by a large number of patients, with or without medical supervision. We conclude with a close look at the problem of side effects, then selected thoughts about methodology. Clearly written, the text provides immediate access to new developments across the spectrum of drug testing. Clinical Trials in Psychopharmacology: A Better Brain is provocative reading for psychiatrists, pharmacologists and all those interested in improved drug treatments for patients with mental illness. Raises questions about the conduct of trials and the credibility of their outcomes that are relevant not just in psychiatry but all areas of medicine Discusses the ethical problems in assessing outcomes in humans, including children

Clinical Trials in Older Adults is the first book to consider the methodological issues underlying the evaluation of new treatments in older people. Provides information on the methodology, monitoring and regulations for those planning to conduct a clinical trials involving older adults Contains examples of ongoing trials involving older adults, and presents the main characteristics of many recently published Depicts how the issues regarding older adults in clinical trials could be properly addressed with the appropriate study design and conduct Identifies key issues in performing clinical trials in older patients with common geriatric conditions, i.e. Alzheimer’s dementia, depression, low muscle mass, cancer

This comprehensive, unified text on the principles and practice of clinical trials presents a detailed account of how to conduct the trials. It describes the design, analysis, and interpretation of clinical trials in a non-technical manner and provides a general perspective on their historical development, current status, and future strategy. Features examples derived from the author's personal experience.

Presents elements of clinical trial methods that are essential in planning, designing, conducting, analyzing, and interpreting clinical trials with the goal of improving the evidence derived from these important studies This Third Edition builds on the text’s reputation as a straightforward, detailed, and authoritative presentation of quantitative methods for clinical trials. Readers will encounter the principles of design for various types of clinical trials, and are then skillfully guided through the complete process of planning the experiment, assembling a study cohort, assessing data, and reporting results. Throughout the process, the author alerts readers to problems that may arise during the course of the trial and provides common sense solutions. All stages of therapeutic development are discussed in detail, and the methods are not restricted to a single clinical application area. The authors bases current revisions and updates on his own experience, classroom instruction, and feedback from teachers and medical and statistical professionals involved in clinical trials. The Third Edition greatly expands its coverage, ranging from statistical principles to new and provocative topics, including alternative medicine and ethics, middle development, comparative studies, and adaptive designs. At the same time, it offers more pragmatic advice for issues such as selecting outcomes, sample size, analysis, reporting, and handling allegations of misconduct. Readers familiar with the First and Second Editions will discover revamped exercise sets; an updated and extensive reference section; new material on endpoints and the developmental pipeline, among others; and revisions of numerous sections. In addition, this book: • Features accessible and broad coverage of statistical design methods—the crucial building blocks of clinical trials and medical research – now complete with new chapters on overall development, middle development, comparative studies, and adaptive designs • Teaches readers to design clinical trials that produce valid qualitative results backed by rigorous statistical methods • Contains an introduction and summary in each chapter to reinforce key points • Includes discussion questions to stimulate critical thinking and help readers understand how they can apply their newfound knowledge • Provides extensive references to direct readers to the most recent literature, and there are numerous new or revised exercises throughout the book Clinical Trials: A Methodologic Perspective, Third Edition is a textbook accessible to advanced undergraduate students in the quantitative sciences, graduate students in public health and the life sciences, physicians training in clinical research methods, and biostatisticians and epidemiologists. Steven Piantadosi, MD, PhD, is the Phase One Foundation Distinguished Chair and Director of the Samuel Oschin Cancer Institute, and Professor of Medicine at Cedars-Sinai Medical Center in Los Angeles, California. Dr. Piantadosi is one of the world’s leading experts in the design and analysis of clinical trials for cancer research. He has taught clinical trials methods extensively in formal courses and short venues. He has advised numerous academic programs and collaborations nationally regarding clinical trial design and conduct, and has served on external advisory boards for the National Institutes of Health and other prominent cancer programs and centers. The author of more than 260 peer-reviewed scientific articles, Dr. Piantadosi has published extensively on research results, clinical applications, and trial methodology. While his papers have contributed to many areas of oncology, he has also collaborated on diverse studies outside oncology including lung disease and degenerative neurological disease.

Learn rigorous statistical methods to ensure valid clinical trials This Second Edition of the critically hailed Clinical Trials builds on the text's reputation as a straightforward and authoritative presentation of statistical methods for clinical trials. Readers are introduced to the fundamentals of design for various types of clinical trials and then skillfully guided through the complete process of planning the experiment, assembling a study cohort, assessing data, and reporting results. Throughout the process, the author alerts readers to problems that may arise during the course of the trial and provides commonsense solutions. The author bases the revisions and updates on his own classroom experience, as well as feedback from students, instructors, and medical and statistical professionals involved in clinical trials. The Second Edition greatly expands its coverage, ranging from statistical principles to controversial topics, including alternative medicine and ethics. At the same time, it offers more pragmatic advice for issues such as selecting outcomes, sample size, analysis, reporting, and handling allegations of misconduct. Readers familiar with the First Edition will discover completely new chapters, including: * Contexts for clinical trials * Statistical perspectives * Translational clinical trials * Dose-finding and dose-ranging designs Each chapter is accompanied by a summary to reinforce the key points. Revised discussion questions stimulate critical thinking and help readers understand how they can apply their newfound knowledge, and updated references are provided to direct readers to the most recent literature. This text distinguishes itself with its accessible and broad coverage of statistical design methods–the crucial building blocks of clinical trials and medical research. Readers learn to conduct clinical trials that produce valid qualitative results backed by rigorous statistical methods.