Cancer Clinical Trials

Author: Stephen L. George
Publisher: CRC Press
ISBN: 1498706908
Format: PDF, Mobi
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Cancer Clinical Trials: Current and Controversial Issues in Design and Analysis provides statisticians with an understanding of the critical challenges currently encountered in oncology trials. Well-known statisticians from academic institutions, regulatory and government agencies (such as the U.S. FDA and National Cancer Institute), and the pharmaceutical industry share their extensive experiences in cancer clinical trials and present examples taken from actual trials. The book covers topics that are often perplexing and sometimes controversial in cancer clinical trials. Most of the issues addressed are also important for clinical trials in other settings. After discussing general topics, the book focuses on aspects of early and late phase clinical trials. It also explores personalized medicine, including biomarker-based clinical trials, adaptive clinical trial designs, and dynamic treatment regimes.

Design and Analysis of Clinical Trials for Predictive Medicine

Author: Shigeyuki Matsui
Publisher: CRC Press
ISBN: 1466558164
Format: PDF
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Design and Analysis of Clinical Trials for Predictive Medicine provides statistical guidance on conducting clinical trials for predictive medicine. It covers statistical topics relevant to the main clinical research phases for developing molecular diagnostics and therapeutics—from identifying molecular biomarkers using DNA microarrays to confirming their clinical utility in randomized clinical trials. The foundation of modern clinical trials was laid many years before modern developments in biotechnology and genomics. Drug development in many diseases is now shifting to molecularly targeted treatment. Confronted with such a major break in the evolution toward personalized or predictive medicine, the methodologies for design and analysis of clinical trials is now evolving. This book is one of the first attempts to contribute to this evolution by laying a foundation for the use of appropriate statistical designs and methods in future clinical trials for predictive medicine. It is a useful resource for clinical biostatisticians, researchers focusing on predictive medicine, clinical investigators, translational scientists, and graduate biostatistics students.

Statistical Design and Analysis of Clinical Trials

Author: Weichung Joe Shih
Publisher: CRC Press
ISBN: 1482250500
Format: PDF, ePub, Docs
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Statistical Design and Analysis of Clinical Trials: Principles and Methods concentrates on the biostatistics component of clinical trials. Developed from the authors’ courses taught to public health and medical students, residents, and fellows during the past 15 years, the text shows how biostatistics in clinical trials is an integration of many fundamental scientific principles and statistical methods. Teach Your Students How to Design, Monitor, and Analyze Clinical Trials The book begins with ethical and safety principles, core trial design concepts, the principles and methods of sample size and power calculation, and analysis of covariance and stratified analysis. It then focuses on sequential designs and methods for two-stage Phase II cancer trials to Phase III group sequential trials, covering monitoring safety, futility, and efficacy. The authors also discuss the development of sample size reestimation and adaptive group sequential procedures, explain the concept of different missing data processes, and describe how to analyze incomplete data by proper multiple imputations. Turn Your Students into Better Clinical Trial Investigators This text reflects the academic research, commercial development, and public health aspects of clinical trials. It gives students a multidisciplinary understanding of the concepts and techniques involved in designing and analyzing various types of trials. The book’s balanced set of homework assignments and in-class exercises are appropriate for students in (bio)statistics, epidemiology, medicine, pharmacy, and public health.

Clinical Trials in Oncology Third Edition

Author: Stephanie Green
Publisher: CRC Press
ISBN: 1439814481
Format: PDF, Kindle
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The third edition of the bestselling Clinical Trials in Oncology provides a concise, nontechnical, and thoroughly up-to-date review of methods and issues related to cancer clinical trials. The authors emphasize the importance of proper study design, analysis, and data management and identify the pitfalls inherent in these processes. In addition, the book has been restructured to have separate chapters and expanded discussions on general clinical trials issues, and issues specific to Phases I, II, and III. New sections cover innovations in Phase I designs, randomized Phase II designs, and overcoming the challenges of array data. Although this book focuses on cancer trials, the same issues and concepts are important in any clinical setting. As always, the authors use clear, lucid prose and a multitude of real-world examples to convey the principles of successful trials without the need for a strong statistics or mathematics background. Armed with Clinical Trials in Oncology, Third Edition, clinicians and statisticians can avoid the many hazards that can jeopardize the success of a trial.

Bayesian Designs for Phase I II Clinical Trials

Author: Ying Yuan
Publisher: CRC Press
ISBN: 1498709567
Format: PDF
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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.

Applied Meta Analysis with R

Author: Ding-Geng (Din) Chen
Publisher: CRC Press
ISBN: 1466505990
Format: PDF, Docs
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In biostatistical research and courses, practitioners and students often lack a thorough understanding of how to apply statistical methods to synthesize biomedical and clinical trial data. Filling this knowledge gap, Applied Meta-Analysis with R shows how to implement statistical meta-analysis methods to real data using R. Drawing on their extensive research and teaching experiences, the authors provide detailed, step-by-step explanations of the implementation of meta-analysis methods using R. Each chapter gives examples of real studies compiled from the literature. After presenting the data and necessary background for understanding the applications, various methods for analyzing meta-data are introduced. The authors then develop analysis code using the appropriate R packages and functions. This systematic approach helps readers thoroughly understand the analysis methods and R implementation, enabling them to use R and the methods to analyze their own meta-data. Suitable as a graduate-level text for a meta-data analysis course, the book is also a valuable reference for practitioners and biostatisticians (even those with little or no experience in using R) in public health, medical research, governmental agencies, and the pharmaceutical industry.

Modern Adaptive Randomized Clinical Trials

Author: Oleksandr Sverdlov
Publisher: CRC Press
ISBN: 1482239892
Format: PDF, ePub
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Is adaptive randomization always better than traditional fixed-schedule randomization? Which procedures should be used and under which circumstances? What special considerations are required for adaptive randomized trials? What kind of statistical inference should be used to achieve valid and unbiased treatment comparisons following adaptive randomization designs? Modern Adaptive Randomized Clinical Trials: Statistical and Practical Aspects answers these questions and more. From novel designs to cutting-edge applications, this book presents several new and key developments in adaptive randomization. It also offers a fresh and critical look at a number of already-classical topics. Featuring contributions from statisticians, clinical trialists, and subject-matter experts in academia and the pharmaceutical industry, the text: Clarifies the taxonomy of the concept of adaptive randomization Discusses restricted, covariate-adaptive, response-adaptive, and covariate-adjusted response-adaptive (CARA) randomization designs, as well as randomized designs with treatment selection Gives an exposition to many novel adaptive randomization techniques such as brick tunnel randomization, targeted least absolute shrinkage and selection operator (LASSO)-based CARA randomization, multi-arm multi-stage (MAMS) designs, to name a few Addresses the issues of statistical inference following covariate-adaptive and response-adaptive randomization designs Describes a successful implementation of a single pivotal phase II/III adaptive trial in infants with proliferating hemangioma Explores some practical aspects of phase II dose-ranging studies and examines statistical monitoring and interim analysis issues in response-adaptive randomized clinical trials Modern Adaptive Randomized Clinical Trials: Statistical and Practical Aspects covers a wide spectrum of topics related to adaptive randomization designs in contemporary clinical trials. The book provides a thorough exploration of the merits of adaptive randomization and aids in identifying when it is appropriate to apply such designs in practice.

Designing Clinical Research

Author: Stephen B. Hulley
Publisher: Lippincott Williams & Wilkins
ISBN: 1469840545
Format: PDF, Docs
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Designing Clinical Research sets the standard for providing a practical guide to planning, tabulating, formulating, and implementing clinical research, with an easy-to-read, uncomplicated presentation. This product incorporates current research methodology--including molecular and genetic clinical research--and offers an updated syllabus for conducting a clinical research workshop. Emphasis is on common sense as the main ingredient of good science. The book explains how to choose well-focused research questions and details the steps through all the elements of study design, data collection, quality assurance, and basic grant-writing.

Design and Analysis of Bridging Studies

Author: Jen-pei Liu
Publisher: CRC Press
ISBN: 1439846340
Format: PDF, ePub, Mobi
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As the development of medicines has become more globalized, the geographic variations in the efficacy and safety of pharmaceutical products need to be addressed. To accelerate the product development process and shorten approval time, researchers are beginning to design multiregional trials that incorporate subjects from many countries around the world under the same protocol. Design and Analysis of Bridging Studies addresses the issues arising from bridging studies and multiregional clinical trials. For bridging studies, the book explores ethnic sensitivity, the necessity of bridging studies, types of bridging studies, and the assessment of similarity between regions based on bridging evidence. For multiregional clinical trials, the text considers regional differences, assesses the consistency of treatment effect across regions, and discusses sample size determination for each region. Taking into account the International Conference Harmonisation (ICH) E5 framework for bridging studies, the book provides a unified summary of the growing literature and research activities in this area. It covers the regulatory requirements, scientific and practical issues, and statistical methodology for designing and evaluating bridging studies and multiregional clinical trials, with the goal of inspiring new research activities in the field.

Clinical Trial Design

Author: Guosheng Yin
Publisher: John Wiley & Sons
ISBN: 1118183320
Format: PDF, Docs
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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.