The definitive reference work on clinical trials, this book presents a wealth of detailed, practical information on the design, conduct, and analysis of both single center and multicenter trials. No other book on clinical trials offers as much detail as Meinert does on such issues as sample size calculation, stratification and randomization, data systems design, consent form development, publication policies, preparation of funding requests, and reporting procedures.
A Practical Guide to Design, Analysis, and Reporting
Author: Duolao Wang,Ameet Bakhai
This book aims to demystify clinical trials. It is divided into five sections: fundamentals of trial design, alternative trial designs, basics of statistical analysis, special trial issues in data analysis, and reporting of trials. Using simple language the book explains with illustrations of numerous trial examples, the conceptual and methodological issues that occur at all stages of clinical trial covering trial design, conduct, analysis and reporting. The book is an educational and approachable reference in a difficult area of medicine where clinicians often feel uncertain and this material helps them review, appraise and publish trials and clinical evidence.
Practical Handbook of Sample Size Guidelines for Clinical Trials is a concise guide and powerful software utility program that provides a valuable, non-technical blueprint for the design and analysis of survival clinical trials. This text and software allow clinical researchers to write more effective protocols or research grant proposals in a fraction of the time it would take them otherwise. Clinical researchers also gain insight into how biostatisticians analyze trial data and discover what "p-values" really tell them. If you are a biostatistician or student, this book and software will be an indispensable tool for study design. Furthermore, no other book provides justification for survival analysis results at such an introductory level. The program increases your flexibility because it allows you to browse through various planning parameter configurations by changing one parameter at a time, circumventing the need to re-enter the set of planning parameters. Practical Handbook of Sample Size Guidelines for Clinical Trials is ideal for biostatisticians, clinical oncologists, epidemiologists, public health specialists, hematologists, and other researchers who need a concise, easy-to-use tool for sample size determination.
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.
Lawrence M. Friedman,Curt D. Furberg,David L. DeMets,David M. Reboussin,Christopher B. Granger
Author: Lawrence M. Friedman,Curt D. Furberg,David L. DeMets,David M. Reboussin,Christopher B. Granger
This is the fifth edition of a very successful textbook on clinical trials methodology, written by recognized leaders who have long and extensive experience in all areas of clinical trials. The three authors of the first four editions have been joined by two others who add great expertise. A chapter on regulatory issues has been included and the chapter on data monitoring has been split into two and expanded. Many contemporary clinical trial examples have been added. There is much new material on adverse events, adherence, issues in analysis, electronic data, data sharing and international trials. This book is intended for the clinical researcher who is interested in designing a clinical trial and developing a protocol. It is also of value to researchers and practitioners who must critically evaluate the literature of published clinical trials and assess the merits of each trial and the implications for the care and treatment of patients. The authors use numerous examples of published clinical trials to illustrate the fundamentals. The text is organized sequentially from defining the question to trial closeout. One chapter is devoted to each of the critical areas to aid the clinical trial researcher. These areas include pre-specifying the scientific questions to be tested and appropriate outcome measures, determining the organizational structure, estimating an adequate sample size, specifying the randomization procedure, implementing the intervention and visit schedules for participant evaluation, establishing an interim data and safety monitoring plan, detailing the final analysis plan and reporting the trial results according to the pre-specified objectives. Although a basic introductory statistics course is helpful in maximizing the benefit of this book, a researcher or practitioner with limited statistical background would still find most if not all the chapters understandable and helpful. While the technical material has been kept to a minimum, the statistician may still find the principles and fundamentals presented in this text useful.
As a result of the expansion in the area of pharmaceutical medicine there is an ever-increasing need for educational resources. The Dictionary of Clinical Trials, Second Edition comprehensively explains the 3000 words and short phrases commonly used when designing, running, analysing and reporting clinical trials. This book is a quick, pocket reference tool to understand the common and less well-used terms within the discipline of clinical trials, and provides an alternative to the textbooks available. Terms are heavily cross-referenced, which helps the reader to understand how terms fit into the broad picture of clinical trials. Wide ranging, brief, pragmatic explanations of clinical trial terminology Scope includes medical, statistical, epidemiological, ethical, regulatory and data management terminology Thoroughly revised and expanded - increase of 280 terms from First Edition, reference to Cochrane included From the reviews of the First Edition: "This invaluable text explains the majority of clinical trial terms, in alphabetical order, that are likely to be found in clinical trial protocols, reports, regulatory guidelines, and published manuscripts... Fully comprehensive - provides definitions of clinical trial terms in one complete volume... Includes extensive use of graphs throughout." LA DOC STI "...covers a range of subject matter, with emphasis on medical, statistical, epidemiological and ethical terms... a useful adjunct to standard clinical trial texts... a reference source to keep within easy reach." TALANTA The Dictionary of Clinical Trials, Second Edition is a ‘must-have’ for all pharmaceutical companies who conduct a lot of clinical trials, in all or one therapeutic area. The book is also of interest for public health and health science workers, and for contract research organisations and departments of medicine, where medics are involved with clinical trials.
The aim of this unique volume is to help medical researchers design clinical trials to improve survival, remission duration, or time to recurrence of disease. Written in a user-friendly step-by-step format, this work enables the researcher-with no background in statistics-to determine sample size and write statistical considerations for their protocols. It provides critical language which can help with FDA submissions and/or research grants. It also provides the mathematical justification of the material at a level consistent with one year of undergraduate mathematical statistics. It presents survival analysis methods at a more elementary level than any known text. Filled with tables, figures, plus an extensive appendix, this one-of-a-kind reference is an absolute must for all clinical researchers and biostatisticians.
The second edition of Oncology Clinical Trials has been thoroughly revised and updated and now contains the latest designs and methods of conducting and analyzing cancer clinical trials in the era of precision medicine with biologic agents—including trials investigating the safety and efficacy of targeted therapies, immunotherapies, and combination therapies as well as novel radiation therapy modalities. Now divided into six sections this revamped book provides the necessary background and expert guidance from the principles governing oncology clinical trials to the innovative statistical design methods permeating the field; from conducting trials in a safe and effective manner, analyzing and interpreting the data, to a forward-looking assessment and discussion of regulatory issues impacting domestic, international, and global clinical trials. Considered by many as the gold standard reference on oncology clinical trials in the field, the second edition continues to provide examples of real-life flaws and real-world examples for how to successfully design, conduct and analyze quality clinical trials and interpret them. With chapters written by oncologists, researchers, biostatisticians, clinical research administrators, and industry and FDA representatives, this volume provides a comprehensive guide in the design, conduct, monitoring, analysis, and reporting of clinical trials in oncology. NEW TO THIS EDITION: Outlines how to design clinical trials with and without biomarker testing—including genomics-based “basket” trials, and adaptive trials for all phases during treatment and quality-of-life trials Includes new chapters on immunotherapy trials, radiation therapy trials, multi-arm trials, meta-analysis and adaptive design, use of genomics, dose modifications and use of ancillary treatments in investigational studies, establishing surrogate endpoints, practical issues with correlative studies, cost-effectiveness analysis, and more Comprehensively covers all regulatory aspects in the pursuit of global oncology trials Digital access to the ebook included
Study Design, Endpoints and Biomarkers, Drug Safety, and FDA and ICH Guidelines
Author: Tom Brody
Publisher: Academic Press
Clinical Trials, Second Edition, offers those engaged in clinical trial design a valuable and practical guide. This book takes an integrated approach to incorporate biomedical science, laboratory data of human study, endpoint specification, legal and regulatory aspects and much more with the fundamentals of clinical trial design. It provides an overview of the design options along with the specific details of trial design and offers guidance on how to make appropriate choices. Full of numerous examples and now containing actual decisions from FDA reviewers to better inform trial design, the 2nd edition of Clinical Trials is a must-have resource for early and mid-career researchers and clinicians who design and conduct clinical trials. Contains new and fully revised material on key topics such as biostatistics, biomarkers, orphan drugs, biosimilars, drug regulations in Europe, drug safety, regulatory approval and more Extensively covers the "study schema" and related features of study design Incorporates laboratory data from studies on human patients to provide a concrete tool for understanding the concepts in the design and conduct of clinical trials Includes decisions made by FDA reviewers when granting approval of a drug as real world learning examples for readers
Behavior is an aspect of clinical trials that has been somewhat neglected, and the problems associated with behavioral assessment and control in clinical trials are complex. These contributions deal with the selection and application of assessment techniques in different patient populations, the scr
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.
The second volume in the Wiley reference series in Biostatistics. Featuring articles from the prestigious Encyclopedia of Biostatistics, many of which have been fully revised and updated to include recent developments, Biostatistics in Clinical Trials also includes up to 25% newly commissioned material reflecting the latest 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 been designed to complement existing texts by providing extensive, up-to-date coverage and introducing the reader to the research literature. Offering comprehensive coverage of all aspects of clinical trials Biostatistics in Clinical Trials: Includes concise definitions and introductions to numerous concepts found in current literature Discusses the software and textbooks available Uses extensive cross-references helping to facilitate further research and enabling the reader to locate definitions and related concepts Biostatistics in Clinical Trials offers both academics and practitioners from various disciplines and settings, such as universities, the pharmaceutical industry and clinical research organisations, up-to-date information as well as references to assist professionals involved in the design and conduct of clinical trials.
Provides a practical approach to understanding the components of a clinical research trial as well as the tools to conduct a well-organized study. Designed for those interested in developing or enhancing skills to coordinate all aspects of clinical trials such as regulatory requirements, budgeting, contracts, patient recruitments and participation, and gathering and recording clear, invaluable data.
Over the last twenty years there has been a dramatic upsurge in theapplication of meta-analysis to medical research. This has mainlybeen due to greater emphasis on evidence-based medicine and theneed for reliable summaries of the vast and expanding volume ofclinical research. At the same time there have been great stridesin the development and refinement of the associated statisticalmethodology. This book describes the planning, conduct andreporting of a meta-analysis as applied to a series of randomizedcontrolled clinical trials. * The various approaches are presented within a general unifiedframework. * Meta-analysis techniques are described in detail, from theirtheoretical development through to practical implementation. * Each topic discussed is supported by detailed workedexamples. * A comparison of fixed and random effects approaches is included,as well as a discussion of Bayesian methods and cumulativemeta-analysis. * Fully documented programs using standard statistical proceduresin SAS are available on the Web. Ideally suited for practising statisticians andstatistically-minded medical professionals, the book will also beof use to graduate students of medical statistics. The book is aself-contained and comprehensive account of the subject and anessential purchase for anyone involved in clinical trials.
David J. Kerr,Kirstine Knox,Diane Robertson,Derek Stewart,Renee Watson
A Guide to Clinical Trials in the NHS for Healthcare Professionals
Author: David J. Kerr,Kirstine Knox,Diane Robertson,Derek Stewart,Renee Watson
Publisher: John Wiley & Sons
What will happen during and after a clinical trial? How will a trial affect my quality of life? What are the benefits and risks of a trial? What does giving consent mean and what will it involve? Will I incur costs during and because of the trial? These are the questions that should be raised every time a health care professional talks through with a patient the pros and cons of entering a clinical trial. Clinical Trials Explained has been designed in consultation with doctors and patients who have taken part in trials, providing an essential and detailed guide to the clinical trials process. It therefore helps both clinicians and patients make well-informed decisions on whether to take part in a clinical trial.
Clinical Trials in Osteoporosisis a practical handbook on clinical trials in the growing field of osteoporosis. Topics covered include study design, technical issues, data collection, quality assurance, data analysis and presentation. It aims to take the user through the process step-by-step from start to finish, also providing a background on regulatory guidelines, ethical implications, endpoints, current therapies and the ideal drug to use. There are no other books at present that specifically address the issue of clinical trials in osteoporosis. A number of issues dealt with in this book have been brought together in one publication for the first time. Clinical Trials in Osteoporosisis intended to serve as a practical manual for clinicians and scientists coming to the subject new and to provide a standard for existing centers to measure themselves against.
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. This book is accompanied by downloadable files available below under the DOWNLOADS tab. These files include: MATHEMATICA program – A set of downloadable files that tracks the chapters, containing code pertaining to each. SAS PROGRAMS and DATA FILES used in the book. The following software programs, included in the downloadables, were developed by the author, Steven Piantadosi, M.D., Ph.D: RANDOMIZATION – This program generates treatment assignments for a clinical trial using blocked stratified randomization. CRM – Implements the continual reassessment methods for dose finding clinical trials. OPTIMAL – Calculates two-stage optimal phase II designs using the Simon method. POWER – This is a power and sample size program for clinical trials. Executables for installing these programs can also be found at https://risccweb.csmc.edu/biostats/. 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.
David J. Spiegelhalter,Keith R. Abrams,Jonathan P. Myles
Author: David J. Spiegelhalter,Keith R. Abrams,Jonathan P. Myles
Publisher: John Wiley & Sons
The Bayesian approach involves collecting data from past experience in order to reach conclusions about future events. This introduction covers a broad array of topics. It is for statisticians in the pharmaceutical industry, as well as anyone involved in conducting clinical trials and HTA work.
A systematic approach to all aspects of designing andconducting clinical trials The success or failure of clinical trials hinges on hundreds ofdetails that need to be developed, often under less than idealconditions. Written by one of the world's leading trialists,Clinical Trials Handbook: Design and Conduct providesclinicians with a complete guide to designing, conducting, andevaluating clinical trials—teaching them how to simplify theprocess and avoid costly mistakes. The author draws on his extensive clinical trials experience tooutline all steps employed in setting up and running clinicaltrials, from budgeting and fundraising to publishing the results.Along the way, practical advice is offered while also addressing amix of logistical, ethical, psychological, behavioral, andadministrative issues inherent to clinical trials. Topics ofcoverage include: Protocols for drug masking, controls, and treatmentrandomization Consent, enrollment, eligibility, and follow-up procedures Different types of sample size design and data collection andprocessing Working with study centers, research staff, and variouscommittees Monitoring treatment effects and performance, and ensuringquality control Data analysis and access policies for study data anddocuments Clinical Trials Handbook is invaluable for practicingclinicians and trialists who would like to learn more about orimprove their understanding of the design and execution of clinicaltrials. The book is also an excellent supplement for courses onclinical trials at the graduate level.