A Non-statisticians Guide to Thinking, Designing, and Executing
Author: Joseph Tal
Publisher: Academic Press
Strategy and Statistics in Clinical Trials is for all individuals engaged in clinical research, including professors, physicians, researchers in corporate and government laboratories, nurses, members of the allied health professions, and post-doctoral and graduate students who are potentially less exposed to understanding the pivotal role of statistics. . Enables nonstatisticians to better understand research processes and statistics' role in these processes . Offers real-life case studies and provides a practical, "how to" guide to biomedical R&D . Delineates the statistical building blocks and concepts of clinical trials . Promotes effective cooperation between statisticians and important other parties.
The Tutorials in Biostatistics have become a very popular feature of the prestigious Wiley journal, Statistics in Medicine (SIM). The introductory style and practical focus make them accessible to a wide audience including medical practitioners with limited statistical knowledge. This book represents the first of two volumes presenting the best tutorials published in SIM, focusing on statistical methods in clinical studies. Topics include the design and analysis of clinical trials, epidemiology, survival analysis, and data monitoring. Each tutorial is focused on a medical problem, has been fully peer-reviewed and edited, and is authored by leading researchers in biostatistics. Many articles include an appendix on the latest developments since publication in the journal and additional references. This will appeal to statisticians working in medical research, as well as statistically-minded clinicians, biologists, epidemiologists and geneticists. It will also appeal to graduate students of biostatistics.
Personalized medicine is a medical paradigm that emphasizes systematic use of individual patient information to optimize that patient's health care, particularly in managing chronic conditions and treating cancer. In the statistical literature, sequential decision making is known as an adaptive treatment strategy (ATS) or a dynamic treatment regime (DTR). The field of DTRs emerges at the interface of statistics, machine learning, and biomedical science to provide a data-driven framework for precision medicine.÷ The authors provide a learning-by-seeing approach to the development of ATSs, aimed at a broad audience of health researchers. All estimation procedures used are described in sufficient heuristic and technical detail so that less quantitative readers can understand the broad principles underlying the approaches. At the same time, more quantitative readers can implement these practices. This book provides the most up-to-date summary of the current state of the statistical research in personalized medicine; contains chapters by leaders in the area from both the statistics and computer sciences fields; and also contains a range of practical advice, introductory and expository materials, and case studies.÷
A complete guide to the key statistical concepts essential for the design and construction of clinical trials As the newest major resource in the field of medical research, Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs presents a timely and authoritative reviewof the central statistical concepts used to build clinical trials that obtain the best results. The referenceunveils modern approaches vital to understanding, creating, and evaluating data obtained throughoutthe various stages of clinical trial design and analysis. Accessible and comprehensive, the first volume in a two-part set includes newly-written articles as well as established literature from the Wiley Encyclopedia of Clinical Trials. Illustrating a variety of statistical concepts and principles such as longitudinal data, missing data, covariates, biased-coin randomization, repeated measurements, and simple randomization, the book also provides in-depth coverage of the various trial designs found within phase I-IV trials. Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs also features: Detailed chapters on the type of trial designs, such as adaptive, crossover, group-randomized, multicenter, non-inferiority, non-randomized, open-labeled, preference, prevention, and superiority trials Over 100 contributions from leading academics, researchers, and practitioners An exploration of ongoing, cutting-edge clinical trials on early cancer and heart disease, mother-to-child human immunodeficiency virus transmission trials, and the AIDS Clinical Trials Group Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs is an excellent reference for researchers, practitioners, and students in the fields of clinicaltrials, pharmaceutics, biostatistics, medical research design, biology, biomedicine, epidemiology,and public health.
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
Drug development is the process of finding and producing therapeutically useful pharmaceuticals, turning them into safe and effective medicine, and producing reliable information regarding the appropriate dosage and dosing intervals. With regulatory authorities demanding increasingly higher standards in such developments, statistics has become an intrinsic and critical element in the design and conduct of drug development programmes. Statistical Issues in Drug Development presents an essential and thought provoking guide to the statistical issues and controversies involved in drug development. This highly readable second edition has been updated to include: Comprehensive coverage of the design and interpretation of clinical trials. Expanded sections on missing data, equivalence, meta-analysis and dose finding. An examination of both Bayesian and frequentist methods. A new chapter on pharmacogenomics and expanded coverage of pharmaco-epidemiology and pharmaco-economics. Coverage of the ICH guidelines, in particular ICH E9, Statistical Principles for Clinical Trials. It is hoped that the book will stimulate dialogue between statisticians and life scientists working within the pharmaceutical industry. The accessible and wide-ranging coverage make it essential reading for both statisticians and non-statisticians working in the pharmaceutical industry, regulatory bodies and medical research institutes. There is also much to benefit undergraduate and postgraduate students whose courses include a medical statistics component.
Methods and Applications of Statistics in Clinical Trials,Volume 2: Planning, Analysis, and Inferential Methods includesupdates of established literature from the Wiley Encyclopedia ofClinical Trials as well as original material based on the latestdevelopments in clinical trials. Prepared by a leading expert, thesecond volume includes numerous contributions from currentprominent experts in the field of medical research. In addition,the volume features: • Multiple new articles exploring emerging topics, such asevaluation methods with threshold, empirical likelihood methods,nonparametric ROC analysis, over- and under-dispersed models, andmulti-armed bandit problems • Up-to-date research on the Cox proportional hazardmodel, frailty models, trial reports, intrarater reliability,conditional power, and the kappa index • Key qualitative issues including cost-effectivenessanalysis, publication bias, and regulatory issues, which arecrucial to the planning and data management of clinical trials
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.
American Statistical Association. Section on Bayesian Statistical Science
Since 1945, "The Annual Deming Conference on Applied Statistics" has been an important event in the statistics profession. In Clinical Trial Biostatistics and Biopharmaceutical Applications, prominent speakers from past Deming conferences present novel biostatistical methodologies in clinical trials as well as up-to-date biostatistical applications from the pharmaceutical industry. Divided into five sections, the book begins with emerging issues in clinical trial design and analysis, including the roles of modeling and simulation, the pros and cons of randomization procedures, the design of Phase II dose-ranging trials, thorough QT/QTc clinical trials, and assay sensitivity and the constancy assumption in noninferiority trials. The second section examines adaptive designs in drug development, discusses the consequences of group-sequential and adaptive designs, and illustrates group sequential design in R. The third section focuses on oncology clinical trials, covering competing risks, escalation with overdose control (EWOC) dose finding, and interval-censored time-to-event data. In the fourth section, the book describes multiple test problems with applications to adaptive designs, graphical approaches to multiple testing, the estimation of simultaneous confidence intervals for multiple comparisons, and weighted parametric multiple testing methods. The final section discusses the statistical analysis of biomarkers from omics technologies, biomarker strategies applicable to clinical development, and the statistical evaluation of surrogate endpoints. This book clarifies important issues when designing and analyzing clinical trials, including several misunderstood and unresolved challenges. It will help readers choose the right method for their biostatistical application. Each chapter is self-contained with references.
In clinical trial practice, controversial statistical issues inevitably occur regardless of the compliance with good statistical practice and good clinical practice. But by identifying the causes of the issues and correcting them, the study objectives of clinical trials can be better achieved. Controversial Statistical Issues in Clinical Trials covers commonly encountered controversial statistical issues in clinical trials and, whenever possible, makes recommendations to resolve these problems. The book focuses on issues occurring at various stages of clinical research and development, including early-phase clinical development (such as bioavailability/bioequivalence), bench-to-bedside translational research, and late-phase clinical development. Numerous examples illustrate the impact of these issues on the evaluation of the safety and efficacy of the test treatment under investigation. The author also offers recommendations regarding possible resolutions of the problems. Written by one of the preeminent experts in the field, this book provides a useful desk reference and state-of-the art examination of problematic issues in clinical trials for scientists in the pharmaceutical industry, medical/statistical reviewers in government regulatory agencies, and researchers and students in academia.
Constantine Gatsonis,Alicia Carriquiry,Robert E. Kass,Andrew Gelman,David Higdon,Donna K. Pauler,Isabella Verdinelli
Author: Constantine Gatsonis,Alicia Carriquiry,Robert E. Kass,Andrew Gelman,David Higdon,Donna K. Pauler,Isabella Verdinelli
Publisher: Springer Science & Business Media
This volume contains invited case studies with the accompanying discussion as well as contributed papers selected by a refereeing process of 6th Workshop on Case Studies in Bayesian Statistics was held at the Carnegie Mellon University in October, 2001.
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.
This volume contains a selection of chapters base on papers presented at the Fourth Seattle Symposium in Biostatistics: Clinical Trials. The symposium was held in 2010 to celebrate the 40th anniversary of the University of Washington School of Public Health and Community Medicine. It featured keynote lectures by David DeMets and Susan Ellenberg and 16 invited presentations by other prominent researchers. The papers contained in this volume encompass recent methodological advances in several important clinical trials research, such as biomarkers, meta-analyses, sequential and adaptive clinical trials, and various genetic bioinformatic techniques. This volume will be a valuable reference for researchers and practitioners in the field of clinical trials.
This volume, representing a compilation of authoritative reviews on a multitude of uses of statistics in epidemiology and medical statistics written by internationally renowned experts, is addressed to statisticians working in biomedical and epidemiological fields who use statistical and quantitative methods in their work. While the use of statistics in these fields has a long and rich history, explosive growth of science in general and clinical and epidemiological sciences in particular have gone through a see of change, spawning the development of new methods and innovative adaptations of standard methods. Since the literature is highly scattered, the Editors have undertaken this humble exercise to document a representative collection of topics of broad interest to diverse users. The volume spans a cross section of standard topics oriented toward users in the current evolving field, as well as special topics in much need which have more recent origins. This volume was prepared especially keeping the applied statisticians in mind, emphasizing applications-oriented methods and techniques, including references to appropriate software when relevant. · Contributors are internationally renowned experts in their respective areas · Addresses emerging statistical challenges in epidemiological, biomedical, and pharmaceutical research · Methods for assessing Biomarkers, analysis of competing risks · Clinical trials including sequential and group sequential, crossover designs, cluster randomized, and adaptive designs · Structural equations modelling and longitudinal data analysis
Statistical Challenges in Oncology Clinical Development presents an overview of statistical considerations in oncology clinical trials, including both early and late phase of development. It illustrates how the novel statistical methods can enrich the design and analysis of modern oncology trials. The book covers wide spectrum of statistical methodology with real life examples. The book covers all aspects of cancer clinical trial starting from early phase development. The early part of the book covers novel phase I dose escalation design, exposure response analysis and innovative phase II design. This includes early development strategy for cancer immunotherapy trials. The authors also emphasized the role of biomarker and modern era of precision medicine. The second part focuses on the late stage development. This includes the application of adaptive design, safety analysis and quality of life data analysis. The final part discusses current regulatory perspective and challenges. Statisticians, key opinion leaders and strategy makers involved in oncology trials will find this book useful. This book provides researchers a solid overview of available methodologies. Along with describing the necessary statistical theory, the authors illustrate the practical application of the techniques. The authors include many relevant real life examples from pharmaceutical industry and academia based on their first-hand experience. Along with relevant references, the book highlights current regulatory views. Features: It covers a wide spectrum of topics related to real life statistical challenges in oncology clinical trials. This provides a comprehensive overview of novel statistical methods to improve trial design and statistical analysis. Detailed case studies illustrate the real-life applications. Authors are eminent members from pharmaceutical industry, FDA and academia who have several years of hands in experience in clinical trial.
John E. Niederhuber,James O. Armitage,James H Doroshow,Michael B. Kastan,Joel E. Tepper
Author: John E. Niederhuber,James O. Armitage,James H Doroshow,Michael B. Kastan,Joel E. Tepper
Publisher: Elsevier Health Sciences
Practical and clinically focused, Abeloff’s Clinical Oncology is a trusted medical reference book designed to capture the latest scientific discoveries and their implications for cancer diagnosis and management of cancer in the most accessible manner possible. Abeloff’s equips everyone involved - from radiologists and oncologists to surgeons and nurses - to collaborate effectively and provide the best possible cancer care. Consult this title on your favorite e-reader, conduct rapid searches, and adjust font sizes for optimal readability. Select the most appropriate tests and imaging studies for cancer diagnosis and staging of each type of cancer, and manage your patients in the most effective way possible by using all of the latest techniques and approaches in oncology. Enhance your understanding of complex concepts with a color art program that highlights key points and illustrates relevant scientific and clinical problems. Stay at the forefront of the latest developments in cancer pharmacology, oncology and healthcare policy, survivorship in cancer, and many other timely topics. See how the most recent cancer research applies to practice through an increased emphasis on the relevance of new scientific discoveries and modalities within disease chapters. Streamline clinical decision making with abundant new treatment and diagnostic algorithms as well as concrete management recommendations. Take advantage of the collective wisdom of preeminent multidisciplinary experts in the field of oncology, including previous Abeloff’s editors John E. Niederhuber, James O. Armitage, and Michael B. Kastan as well as new editors James H. Doroshow from the National Cancer Institute and Joel E. Tepper of Gunderson & Tepper: Clinical Radiation Oncology. Quickly and effortlessly access the key information you need with the help of an even more user-friendly, streamlined format. Access the complete contents anytime, anywhere at Expert Consult, and test your mastery of the latest knowledge with 500 online multiple-choice review questions.
Covering advanced massage therapy skills, this practical resource prepares you to work with medical professionals in a clinical setting, such as a hospital, hospice, long-term care, or other health-related practice. It discusses the many skills you need to succeed in this environment, helping you become a contributing member of an integrated team. Also covered are the essentials of clinical massage, such as indications and contraindications, review of massage methods, range of motion testing, SOAP note documentation, and a massage therapy general protocol. Case studies show how a multidisciplinary approach applies to real-world clients. By coordinating your work with other health professionals, you can enhance patient care in any clinical setting! Includes a DVD with: Two hours of video showing specific applications, featuring author Sandy Fritz. A complete general protocol for massage. State-of-the-art animations depicting biologic functions and medical procedures. 700 full-color illustrations accompany procedures, concepts, and techniques. An integrated healthcare approach covers the healthcare environment and the skills necessary to be a contributing member of an integrated healthcare team. A research-based focus emphasizes research, clinical reasoning, and outcome-based massage application — for effective massage application in conjunction with healthcare intervention. A complete general protocol provides a guide to treating disorders and maintaining wellness, with recommendations for positioning and interventions, using a step-by-step sequence that can easily be modified to meet a patient's specific needs. A palliative protocol helps you temporarily relieve a patient's symptoms of disorders or diseases. Case studies focus on outcome-based massage for individuals with multiple health issues, detailing assessment, medical intervention, justification for massage, and session documentation. Coverage of advanced massage therapy skills and decision-making skills includes specific themes for effective massage application, allowing you to consolidate massage treatment based on the main outcomes — useful when working with individuals with multiple pathologies or treatment needs. A discussion of aromatherapy provides safe recommendations for the use of essential oils in conjunction with massage, to promote healing of the body and mind. Descriptions of illness and injury include relevant anatomy/physiology/pathophysiology, as well as strategies and massage applications to use for pain management, immune support, stress management, chronic illness, and post-surgical needs. Coverage of insurance and reimbursement issues relates to you as a massage professional. Strategies for general conditions such as substance abuse, mental health, orthopedic injury, and cardiovascular disorders help you specialize in clinical massage. Expert authors provide knowledge in research, massage therapy in healthcare, and manual therapies. Learning resources include chapter outlines, chapter learning objectives, key terms, and workbook-style exercises. A companion Evolve website includes: PubMed links to research supporting best practices and justification for massage application. More information on topics such as insurance, pharmacology, and nutrition. More information on anatomy and physiology and other subjects. A comprehensive glossary with key terms and some audio pronunciations.