Clinical Trial Optimization Using R explores a unified and broadly applicable framework for optimizing decision making and strategy selection in clinical development, through a series of examples and case studies. It provides the clinical researcher with a powerful evaluation paradigm, as well as supportive R tools, to evaluate and select among simultaneous competing designs or analysis options. It is applicable broadly to statisticians and other quantitative clinical trialists, who have an interest in optimizing clinical trials, clinical trial programs, or associated analytics and decision making. This book presents in depth the Clinical Scenario Evaluation (CSE) framework, and discusses optimization strategies, including the quantitative assessment of tradeoffs. A variety of common development challenges are evaluated as case studies, and used to show how this framework both simplifies and optimizes strategy selection. Specific settings include optimizing adaptive designs, multiplicity and subgroup analysis strategies, and overall development decision-making criteria around Go/No-Go. After this book, the reader will be equipped to extend the CSE framework to their particular development challenges as well.
With Applications to Cancer Clinical Trials Using R
Author: Jianrong Wu
Publisher: CRC Press
Statistical Methods for Survival Trial Design: With Applications to Cancer Clinical Trials Using R provides a thorough presentation of the principles of designing and monitoring cancer clinical trials in which time-to-event is the primary endpoint. Traditional cancer trial designs with time-to-event endpoints are often limited to the exponential model or proportional hazards model. In practice, however, those model assumptions may not be satisfied for long-term survival trials. This book is the first to cover comprehensively the many newly developed methodologies for survival trial design, including trial design under the Weibull survival models; extensions of the sample size calculations under the proportional hazard models; and trial design under mixture cure models, complex survival models, Cox regression models, and competing-risk models. A general sequential procedure based on the sequential conditional probability ratio test is also implemented for survival trial monitoring. All methodologies are presented with sufficient detail for interested researchers or graduate students.
Demissie Alemayehu, PhD,Joseph C. Cappelleri, PhD,Birol Emir, PhD,Kelly H. Zou, PhD, Pstat
Author: Demissie Alemayehu, PhD,Joseph C. Cappelleri, PhD,Birol Emir, PhD,Kelly H. Zou, PhD, Pstat
Publisher: CRC Press
With ever-rising healthcare costs, evidence generation through Health Economics and Outcomes Research (HEOR) plays an increasingly important role in decision-making about the allocation of resources. Accordingly, it is now customary for health technology assessment and reimbursement agencies to request for HEOR evidence, in addition to data from clinical trials, to inform decisions about patient access to new treatment options. While there is a great deal of literature on HEOR, there is a need for a volume that presents a coherent and unified review of the major issues that arise in application, especially from a statistical perspective. Statistical Topics in Health Economics and Outcomes Research fulfils that need by presenting an overview of the key analytical issues and best practice. Special attention is paid to key assumptions and other salient features of statistical methods customarily used in the area, and appropriate and relatively comprehensive references are made to emerging trends. The content of the book is purposefully designed to be accessible to readers with basic quantitative backgrounds, while providing an in-depth coverage of relatively complex statistical issues. The book will make a very useful reference for researchers in the pharmaceutical industry, academia, and research institutions involved with HEOR studies. The targeted readers may include statisticians, data scientists, epidemiologists, outcomes researchers, health economists, and healthcare policy and decision-makers.
"The self-controlled case series has emerged as a key methodology for studying the effects of healthcare interventions. The overall literature around the self-controlled case series has exploded in recent years and this important and timely book pulls it all together in an effective and clear manner. It certainly belongs on the shelf (or beside the keyboard) of every analyst conducting observational studies in healthcare." —David Madigan, Columbia University Self-Controlled Case Series Studies: A Modelling Guide with R provides the first comprehensive account of the self-controlled case series (SCCS) method, a statistical technique for investigating associations between outcome events and time-varying exposures. The method only requires information from individuals who have experienced the event of interest, and automatically controls for multiplicative time-invariant confounders, even when these are unmeasured or unknown. It is increasingly being used in epidemiology, most frequently to study the safety of vaccines and pharmaceutical drugs. Key features of the book include: A thorough yet accessible description of the SCCS method, with mathematical details provided in separate starred sections. Comprehensive discussion of assumptions and how they may be verified. A detailed account of different SCCS models, extensions of the SCCS method, and the design of SCCS studies. Extensive practical illustrations and worked examples from epidemiology. Full computer code from the associated R package SCCS, which includes all the data sets used in the book. The book is aimed at a broad range of readers, including epidemiologists and medical statisticians who wish to use the SCCS method, and also researchers with an interest in statistical methodology. The three authors have been closely involved with the inception, development, popularisation and programming of the SCCS method.
Guest Editor Dane Nichols, MD, has assembled a panel of experts focusing on Hemodynamic Support in Septic Shock. Topics include: Oxygen Delivery and Consumption: A Macro-Circulatory Perspective; Mean Arterial Pressure: Therapeutic Goals and Pharmacologic Support,Mechanisms; Detection and Potential Management of Microcirculatory Disturbances; Detection of Hypoxia at the Cellular Level; Type A & B Lactic Acidosis: Recognition, Kinetics, and Associated Prognosis; Venous blood gases: What Can They Tell Us About the State of the Circulation; Noninvasive Assessment of Cardiac Preload and Performance through CO2 Rebreathing/ETCO2 Monitoring.
This issue of the Surgical Oncology Clinics will review the important clinical trials from the past eight years in following topics: breast cancer, soft tissue sarcoma, rectal and anal carcinoma, pancreatic adenocarcinoma, hepatocellular carcinoma, gastric cancer, esophageal cancer, colon cancer, advanced and metastatic colorectal carcinoma, gastrointestinal stromal tumors, and melanoma.
This issue provides fully updated information on respiratory infections, including healthcare associated pneumonia, new diagnostic tests for pneumonia, epidemic viral pneumonia and other emerging pathogens, biomarkers to optimize antibiotic therapy for pneumonia, pharmacokinetics and pharmacodynamics to improve management of penumonia. Nosocomial tracheobronchitis and bronchiectasis are also discussed. Articles on CAP and VAP, including an examination of the impact of guidelines on outcomes, de-escalation therapy, inhaled antibiotic therapy and prevention of VAP are also included.
Clinical trials are the engine of progress in the development of new drugs and devices for the detection, monitoring, prevention and treatment of cancer. A well conceived, carefully designed and efficiently conducted clinical trial can produce results that change clinical practice overnight, deliver new oncology drugs and diagnostics to the marketplace, and expand the horizon of contemporary thinking about cancer biology. A poorly done trial does little to advance the field or guide clinical practice, consumes precious clinical and financial resources and challenges the validity of the ethical contract between investigators and the volunteers who willingly give their time and effort to benefit future patients. With chapters written by oncologists, researchers, biostatisticians, clinical research administrators, and industry and FDA representatives, Oncology Clinical Trials, provides a comprehensive guide for both early-career and senior oncology investigators into the successful design, conduct and analysis of an oncology clinical trial. Oncology Clinical Trials covers how to formulate a study question, selecting a study population, study design of Phase I, II, and III trials, toxicity monitoring, data analysis and reporting, use of genomics, cost-effectiveness analysis, systemic review and meta-analysis, and many other issues. Many examples of real-life flaws in clinical trials that have been reported in the literature are included throughout. The book discusses clinical trials from start to finish focusing on real-life examples in the development, design and analysis of clinical trials. Oncology Clinical Trials features: A systematic guide to all aspects of the design, conduct, analysis, and reporting of clinical trials in oncology Contributions from oncologists, researchers, biostatisticians, clinical research administrators, and industry and FDA representatives Hot topics in oncology trials including multi-arm trials, meta-analysis and adaptive design, use of genomics, and cost-effectiveness analysis Real-life examples from reported clinical trials included throughout
Robert O. Williams,David R. Taft,Jason T. McConville
Author: Robert O. Williams,David R. Taft,Jason T. McConville
Publisher: CRC Press
This title demonstrates how advanced formulation designs and delivery technologies can be used to improve drug efficacy and treatment outcomes in particular therapeutic categories or disease states. It discusses nanoparticle systems for cancer treatments, and also presents cutting edge immono-regulation agents for transplantation and the local targeting of drugs, especially poorly water soluble drugs to provide enhanced therapeutic outcomes. In addition, this title highlights areas of therapy showing the most promise for improvement in clinical outcomes via advanced formulation design.
Elizabeth A. Eisenhauer,Christopher Twelves,Marc Buyse
Author: Elizabeth A. Eisenhauer,Christopher Twelves,Marc Buyse
Publisher: Oxford University Press
Phase I trials are a critical first step in the study of novel cancer therapeutic approaches. Their primary goals are to identify the recommended dose, schedule and pharmacologic behavior of new agents or new combinations of agents and to describe the adverse effects of treatment. In cancer therapeutics, such studies have particular challenges. Due to the nature of the effects of treatment, most such studies are conducted in patients with advanced malignancy, rather than in healthy volunteers. Further, the endpoints of these trials are usually measures adverse effects rather than molecular target or anti-tumor effects. These factors render the design, conduct, analysis and ethical aspects of phase I cancer trials unique. As the only comprehensive book on this topic, Phase I Cancer Clinical Trials is a useful resource for oncology trainees or specialists interested in understanding cancer drug development. New to this edition are chapters on Phase 0 Trials and Immunotherapeutics, and updated information on the process, pitfalls, and logistics of Phase I Trials
Preclinical Drug Development, Second Edition discusses the broad and complicated realm of preclinical drug development. Topics range from assessment of pharmacology and toxicology to industry trends and regulatory expectations to requirements that support clinical trials. Highlights of the Second Edition include: Pharmacokinetics Modeling and simulation Formulation and routes of administration Toxicity evaluations The assessment of drug absorption and metabolism Interspecies scaling Lead molecule selection and optimization via profiling Screening using in silico and in vitro toxicity evaluations The book also includes case studies on preclinical pharmacokinetic-pharmacodynamic modeling and simulation in drug development, a review of ICH preclinical guidelines, and experimental methods used to study membrane drug transport and metabolism. This guide is a fundamental resource for medicinal chemists, biologists, and other specialists in the drug development sciences.
Author: Stephan R. Targan,Fergus Shanahan,Loren C. Karp
Publisher: John Wiley & Sons
This is the state-of–the-art book on inflammatory bowel disease you have been waiting for Written and edited by international experts in gastroenterology this up-to-date volume provides a complete review of the basic science behind inflammatory bowel disease (IBD), as well as evidence-based clinical guidance on diagnosis, treatment and long-term management of IBD. In 50 chapters the authors cover the latest and most promising treatment modalities and the science behind them. There are chapters which cover the advances in the medical and surgical treatment of conditions such as Crohn's disease and ulcerative colitis, as well as chapters focusing on nutrition, imaging and complementary medicine. This is an invaluable information resource for all those in the medical team treating patients with IBD. Whether you are a gastroenterologist, gastrointestinal surgeon or GI nurse specialist this book deserves a place in your library.
Why Clinical Research Can’t Guarantee The Right Medical Answers.
Author: Ronald Gauch
Publisher: Springer Science & Business Media
The truth is, few people know the first thing about clinical research. The public reads about a medical research project that announces unbelievable results for a miraculous drug. Some years later, another investigation completely wipes out those initial favorable findings. Hormones Cut Women’s Risk of Heart Disease (San Francisco Chronicle, 1994) Hormones Don’t Protect Women from Heart Disease, Study Says (Washington Post, 2001) The people are confused because we do not understand the process behind these conflicting results. Our health, and in fact, our very lives are dependent on clinical trials, but we know little about them. This book explains the issues the public needs to be aware of when it comes to clinical research. It uncovers the problems in medical investigations that can not be overcome no matter how much care and diligence medical researchers bring to a research project. The basic premise that drives the writing is that it is impossible for medical researchers to guarantee that they can get all the right answers from a single study. No matter how good the investigators are, no matter how well a study is planned, no matter how carefully the plans are executed and no matter how conscientiously the results are analyzed and interpreted – the answer may still be wrong. The deck is stacked against medical researchers and the public – you – should be skeptical of the results no matter how impressive they seem on the surface.
"Pediatric Neurocritical Care is by far the most comprehensive book I have read dealing with this topic... This text will provide an excellent reference for those interested in the pediatric intensive care cases or pediatrics in general. It is written very well and is chock-full of information that Practioners in a pediatric ICU setting would use." --Donna Jacobs, R. EEG T., CNIM, The Neurodiagnostic Journal Caring for children with neurocritical illness often requires multi-specialty collaboration and the understanding of an ever-expanding body of data. This book gives clinicians the up-to-date, concise, and clinically relevant guidance they need to provide optimal care to children with these acute neurologic disorders. The chapters in Pediatric Neurocritical Care are authored by experts in each individual topic, and co-edited by a neurologist and intensivist to ensure that all topics have been fully addressed from both perspectives. The book covers the complete continuum of care from assessment, monitoring, and condition specific management through rehabilitation. Early chapters present differential diagnosis and management approaches to common overarching problems such as coma, headache, and elevated intracranial pressure, followed by chapters focusing on the evaluation and management of specific conditions including traumatic brain injury, stroke, seizures, central nervous system infections, and demyelinating disorders. The final chapters address important associated psychological, social, and ethical issues. To reflect the full spectrum of specialties involved in the burgeoning field of pediatric neurocritical care, the book brings together an international group of experts from multiple disciplines including critical care medicine, anesthesiology, neurology, neurosurgery, rehabilitation medicine, psychology, and pediatric subspecialties. Pediatric Neurocritical Care features: Every topic is addressed from a neurologic and critical care perspective. Comprehensive coverage includes the evaluation of common overarching problems and management of specific conditions, in addition to discussion of related psychological, ethical, and social issues. Chapters are written by internationally known experts from the fields of critical care medicine, anesthesiology, neurology, neurosurgery, rehabilitation medicine, and many affiliated specialties.
Written by noted experts with day-to-day experience in cardiac resynchronization therapy (CRT), this comprehensive, practical reference gives physicians a thorough knowledge of the indications, techniques for implantation, complications, programming, and follow-up of CRT devices in patients with heart failure and intra- and interventricular conduction delays. Each chapter has how-to and troubleshooting sections to help readers avoid or navigate the pitfalls encountered in day-to-day clinical practice. Each chapter also has a summary box capturing the key clinical pearls. This book will be a valuable aid in preparing for the Heart Rhythm Exam/International Board of Heart Rhythm Examiners (IBHRE) exam.
Panos M. Pardalos,Thomas F. Coleman,Petros Xanthopoulos
Author: Panos M. Pardalos,Thomas F. Coleman,Petros Xanthopoulos
Publisher: Springer Science & Business Media
This volume covers some of the topics that are related to the rapidly growing field of biomedical informatics. In June 11-12, 2010 a workshop entitled ‘Optimization and Data Analysis in Biomedical Informatics’ was organized at The Fields Institute. Following this event invited contributions were gathered based on the talks presented at the workshop, and additional invited chapters were chosen from world’s leading experts. In this publication, the authors share their expertise in the form of state-of-the-art research and review chapters, bringing together researchers from different disciplines and emphasizing the value of mathematical methods in the areas of clinical sciences. This work is targeted to applied mathematicians, computer scientists, industrial engineers, and clinical scientists who are interested in exploring emerging and fascinating interdisciplinary topics of research. It is designed to further stimulate and enhance fruitful collaborations between scientists from different disciplines.
Globulins: Advances in Research and Application: 2011 Edition is a ScholarlyBrief™ that delivers timely, authoritative, comprehensive, and specialized information about Globulins in a concise format. The editors have built Globulins: Advances in Research and Application: 2011 Edition on the vast information databases of ScholarlyNews.™ You can expect the information about Globulins in this eBook to be deeper than what you can access anywhere else, as well as consistently reliable, authoritative, informed, and relevant. The content of Globulins: Advances in Research and Application: 2011 Edition has been produced by the world’s leading scientists, engineers, analysts, research institutions, and companies. All of the content is from peer-reviewed sources, and all of it is written, assembled, and edited by the editors at ScholarlyEditions™ and available exclusively from us. You now have a source you can cite with authority, confidence, and credibility. More information is available at http://www.ScholarlyEditions.com/.
überraschende Antworten auf alltägliche Lebensfragen ; [warum wohnen Drogenhändler bei ihren Müttern? Führt mehr Polizei zu weniger Kriminalität? Sind Swimmingpools gefährlicher als Revolver? Macht gute Erziehung glücklich?]
Author: Steven D. Levitt,Stephen J. Dubner
Sind Swimmingpools gefährlicher als Revolver? Warum betrügen Lehrer? Der preisgekrönte Wirtschaftswissenschaftler Steven D. Levitt kombiniert Statistiken, deren Zusammenführung und Gegenüberstellung auf den ersten Blick absurd erscheint, durch seine Analysetechnik aber zu zahlreichen Aha-Effekten führt. Ein äußerst unterhaltsamer Streifzug durch die Mysterien des Alltags, der uns schmunzeln lässt und stets über eindimensionales Denken hinausführt.