All the Essentials to Start Using Adaptive Designs in No TimeCompared to traditional clinical trial designs, adaptive designs often lead to increased success rates in drug development at reduced costs and time. Introductory Adaptive Trial Designs: A Practical Guide with R motivates newcomers to quickly and easily grasp the essence of adaptive desig
Platform trials test multiple therapies in one indication, one therapy for multiple indications, or both. These novel clinical trial designs can dramatically increase the cost-effectiveness of drug development, leading to life-altering medicines for people suffering from serious illnesses, possibly at lower cost. Currently, the cost of drug development is unsustainable. Furthermore, there are particular problems in rare diseases and small biomarker defined subsets in oncology, where the required sample sizes for traditional clinical trial designs may not be feasible. The editors recruited the key innovators in this domain. The 20 articles discuss trial designs from perspectives as diverse as quantum computing, patient’s rights to information, and international health. The book begins with an overview of platform trials from multiple perspectives. It then describes impacts of platform trials on the pharmaceutical industry’s key stakeholders: patients, regulators, and payers. Next it provides advanced statistical methods that address multiple aspects of platform trials, before concluding with a pharmaceutical executive’s perspective on platform trials. Except for the statistical methods section, only a basic qualitative knowledge of clinical trials is needed to appreciate the important concepts and novel ideas presented. Zoran Antonijevic is the founder of the consulting firm Z-Adaptive Designs. He held executive positions in Pharmaceutical Companies and CROs and has designed more than 100 clinical trials in numerous therapeutic areas, many of which included adaptive designs. He has authored numerous papers and scientific presentations and edited Optimization of Pharmaceutical R&D Programs and Portfolios. Robert A. Beckman is Professor of Oncology and of Biostatistics, Bioinformatics, and Biomathematics at the Lombardi Comprehensive Cancer Center and the Innovation Center of Biomedical Informatics, Georgetown University Medical Center. He has played significant leadership roles in developing new oncology clinical research groups at four pharmaceutical companies, while co-inventing several novel clinical and statistical development strategies. His versatile publication record comprises more than 250 contributions ranging from computational chemistry to clinical oncology. The editors are past and current chairs of the Drug Information Association Adaptive Design Scientific Working Group, an international group of over 200 statisticians focused on novel clinical study designs and their role in bringing new medicines to patients.
Author: David Robertson
Publisher: Academic Press
Release Date: 2009-03-02
Clinical or translational science is the field of study devoted to investigating human health and disease, interventions and outcomes for the purposes of developing new treatment approaches, devices, and modalities to improve health. New molecular tools and diagnostic technologies based on clinical and translational research have lead to a better understanding of human disease and the application of new therapeutics for enhanced health. Clinical and Translational Science is designed as the most authoritative and modern resource for the broad range of investigators in various medical specialties taking on the challenge of clinical research. Prepared with an international perspective, this resource begins with experimental design and investigative tools to set the scene for readers. It then moves on to human genetics and pharmacology with a focus on statistics, epidemiology, genomic information, drug discovery and development, and clinical trials. Finally, it turns to legal, social, and ethical issues of clinical research concluding with a discussion of future prospects to provide readers with a comprehensive view of the this developing area of science. Clinical research is one of the fastest growing fields in private practice and academic medicine with practical biological, physiological, cellular, and therapeutic applications Contributions from international leaders provide insight into background and future understanding for clinical and translational science Provides the structure for complete instruction and guidance on the subject from fundamental principles, approaches and infrastructure to human genetics, human pharmacology, research in special populations, the societal context of human research, and the future of human research
Author: J. Rick Turner
Publisher: Springer Science & Business Media
Release Date: 2010-07-16
New Drug Development: Second Edition provides an overview of the design concepts and statistical practices involved in therapeutic drug development. This wide spectrum of activities begins with identifying a potentially useful drug candidate that can perhaps be used in the treatment or prevention of a condition of clinical concern, and ends with marketing approval being granted by one or more regulatory agencies. In between, it includes drug molecule optimization, nonclinical and clinical evaluations of the drug’s safety and efficacy profiles, and manufacturing considerations. The more inclusive term lifecycle drug development can be used to encompass the postmarketing surveillance that is conducted all the time that a drug is on the market and being prescribed to patients with the relevant clinical condition. Information gathered during this time can be used to modify the drug (for example, dose prescribed, formulation, and mode of administration) in terms of its safety and its effectiveness. The central focus of the first edition of this book is captured by its subtitle, 'Design, Methodology, and Analysis'. Optimum quality study design and experimental research methodology must be employed if the data collected—numerical representations of biological information—are to be of optimum quality. Optimum quality data facilitate optimum quality statistical analysis and interpretation of the results obtained, which in turn permit optimum quality decisions to be made: Rational decision making is predicated on appropriate research questions and optimum quality numerical information. The book took a non-computational approach to statistics, presenting instead a conceptual framework and providing readers with a sound working knowledge of the importance of design, methodology, and analysis. Not everyone needs to be an expert in statistical analysis, but it is very helpful for work (or aspire to work) in the pharmaceutical and biologics industries to be aware of the fundamental importance of a sound scientific and clinical approach to the planning, conduct, and analysis of clinical trials.
Author: Lawrence M. Friedman
Publisher: Springer Science & Business Media
Release Date: 2010-09-09
The clinical trial is “the most definitive tool for evaluation of the applicability of clinical research.” It represents “a key research activity with the potential to improve the quality of health care and control costs through careful comparison of alternative treatments” . It has been called on many occasions, “the gold st- dard” against which all other clinical research is measured. Although many clinical trials are of high quality, a careful reader of the medical literature will notice that a large number have deficiencies in design, conduct, analysis, presentation, and/or interpretation of results. Improvements have occurred over the past few decades, but too many trials are still conducted without adequate attention to its fundamental principles. Certainly, numerous studies could have been upgraded if the authors had had a better understanding of the fundamentals. Since the publication of the first edition of this book, a large number of other texts on clinical trials have appeared, most of which are indicated here [2–21]. Several of them, however, discuss only specific issues involved in clinical trials. Additionally, many are no longer current. The purpose of this fourth edition is to update areas in which major progress has been made since the publication of the third edition. We have revised most chapters considerably and added one on ethical issues.
Author: Douglas G. Altman
Publisher: CRC Press
Release Date: 1990-11-22
Most medical researchers, whether clinical or non-clinical, receive some background in statistics as undergraduates. However, it is most often brief, a long time ago, and largely forgotten by the time it is needed. Furthermore, many introductory texts fall short of adequately explaining the underlying concepts of statistics, and often are divorced from the reality of conducting and assessing medical research. Practical Statistics for Medical Research is a problem-based text for medical researchers, medical students, and others in the medical arena who need to use statistics but have no specialized mathematics background. The author draws on twenty years of experience as a consulting medical statistician to provide clear explanations to key statistical concepts, with a firm emphasis on practical aspects of designing and analyzing medical research. The text gives special attention to the presentation and interpretation of results and the many real problems that arise in medical research.
Author: Leon Shargel
Publisher: McGraw Hill Professional
Release Date: 2015-11-22
The landmark textbook on the theoretical and practical applications of biopharmaceutics and pharmacokinetics—now fully updated. Explains how to detect clinical pharmacokinetic problems and apply basic pharmacokinetic principles to solve them Helps you critically evaluate biopharmaceutic studies involving drug product equivalency and unequivalency Chapters have been revised to reflect the latest clinical perspectives on drug performance, bioavailability, bioequivalence, pharmacokinetics, pharmacodynamics, and drug therapy The field’s leading text for more than three decades, Applied Biopharmaceutics & Pharmacokinetics gets you up to speed on the basics of the discipline like no other resource. Practical problems and clinical examples with discussions are integrated within each chapter to help you apply principles to patient care and drug consultation situations. In addition, outstanding pedagogy, including chapter objectives, chapter summaries, and FAQs, plus additional application questions, identify and focus on key concepts. Written by authors who have both academic and clinical experience, Applied Biopharmaceutics & Pharmacokinetics shows you how to use raw data and formulate the pharmacokinetic models and parameters that best describe the process of drug absorption, distribution, and elimination. The book also helps you work with pharmacokinetic and biopharmaceutic parameters to design and evaluate dosage regimens of drugs. In the seventh edition of this must-have interactive learning tool, most of the chapters are updated to reflect our current understanding of complex issues associated with safe and efficacious drug therapy.
Author: John G. Webster
Release Date: 2006
This set is an alphabetically organized compilation of about 300 articles that describe critical aspects of medical devices and instrumentation. The articles emphasize the contributions of engineering, physics, and computers to each of the general areas of anesthesiology, biomaterials, burns, cardiology, clinical chemistry, clinical engineering, communicative disorders, computers in medicine, critical care medicine, dermatology, dentistry, ear, nose, and throat, emergency medicine, endocrinology, gastroenterology, genetics, geriatrics, gynecology, hematology, heptology, internal medicine, medical physics, microbiology, nephrology, neurology, nutrition, obstetrics, oncology, ophthalmology, orthopedics, pain, pediatrics, peripheral vascular disease, pharmacology, physical therapy, psychiatry, pulmonary medicine, radiology, rehabilitation, surgery, tissue engineering, transducers, and urology.
Author: Gerald J. Hahn
Release Date: 2008-07-28
Genre: Business & Economics
An insightful guide to the use of statistics for solving key problems in modern-day business and industry This book has been awarded the Technometrics Ziegel Prize for the best book reviewed by the journal in 2010. Technometrics is a journal of statistics for the physical, chemical and engineering sciences, published jointly by the American Society for Quality and the American Statistical Association. Criteria for the award include that the book brings together in one volume a body of material previously only available in scattered research articles and having the potential to significantly improve practice in engineering and science. Highlighting the relevance of statistical methods in everyday applications, The Role of Statistics in Business and Industry bridges the gap between the tools of statistics and their use in today's business world. This one-of-a-kind resource encourages the proactive use of statistics in three well-organized and succinct parts: Setting the Stage provides an introduction to statistics, with a general overview of its uses in business and industry Manufactured Product Applications explains how statistical techniques assist in designing, building, improving, and ensuring the reliability of a wide variety of manufactured products such as appliances, plastic materials, aircraft engines, and locomotives Other Applications describe the role of statistics in pharmaceuticals, finance, and business services, as well as more specialized areas including the food, semiconductor, and communications industries This book is truly unique in that it first describes case studies and key business problems, and then shows how statistics is used to address them, while most literature on the topic does the reverse. This approach provides a comprehensive understanding of common issues and the most effective methods for their treatment. Each chapter concludes with general questions that allow the reader to test their understanding of the presented statistical concepts as well as technical questions that raise more complex issues. An extensive FTP site provides additional material, including solutions to some of the applications. With its accessible style and real-world examples, The Role of Statistics in Business and Industry is a valuable supplement for courses on applied statistics and statistical consulting at the upper-undergraduate and graduate levels. It is also an ideal resource for early-career statisticians and practitioners who would like to learn the value of applying statistics to their everyday work.