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.
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 second of two volumes presenting the best tutorials published in SIM, focusing on statistical modeling of complex data. Topics include clustered data, hierarchical models, mixed models, genetic modeling, and meta-analysis. 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.
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.
Frederick W. Faltin,Ron S. Kenett,Fabrizio Ruggeri
Author: Frederick W. Faltin,Ron S. Kenett,Fabrizio Ruggeri
Publisher: John Wiley & Sons
In recent years the number of innovative medicinal products and devices submitted and approved by regulatory bodies has declined dramatically. The medical product development process is no longer able to keep pace with increasing technologies, science and innovations and the goal is to develop new scientific and technical tools and to make product development processes more efficient and effective. Statistical Methods in Healthcare focuses on the application of statistical methodologies to evaluate promising alternatives and to optimize the performance and demonstrate the effectiveness of those that warrant pursuit is critical to success. Statistical methods used in planning, delivering and monitoring health care, as well as selected statistical aspects of the development and/or production of pharmaceuticals and medical devices are also addressed. With a focus on finding solutions to these challenges, this book: Provides a comprehensive, in-depth treatment of statistical methods in healthcare, along with a reference source for practitioners and specialists in health care and drug development. Offers a broad coverage of standards and established methods through leading edge techniques. Uses an integrated, case-study based approach, with focus on applications. Looks at the use of analytical and monitoring schemes to evaluate therapeutic performance. Features the application of modern quality management systems to clinical practice, and to pharmaceutical development and production processes. Addresses the use of modern Statistical methods such as Adaptive Design, Seamless Design, Data Mining, Bayesian networks and Bootstrapping that can be applied to support the challenging new vision. Practitioners in healthcare-related professions, ranging from clinical trials to care delivery to medical device design, as well as statistical researchers in the field, will benefit from this book.
Analysis of Clinical Trials Using SAS®: A Practical Guide, Second Edition bridges the gap between modern statistical methodology and real-world clinical trial applications. Tutorial material and step-by-step instructions illustrated with examples from actual trials serve to define relevant statistical approaches, describe their clinical trial applications, and implement the approaches rapidly and efficiently using the power of SAS. Topics reflect the International Conference on Harmonization (ICH) guidelines for the pharmaceutical industry and address important statistical problems encountered in clinical trials. Commonly used methods are covered, including dose-escalation and dose-finding methods that are applied in Phase I and Phase II clinical trials, as well as important trial designs and analysis strategies that are employed in Phase II and Phase III clinical trials, such as multiplicity adjustment, data monitoring, and methods for handling incomplete data. This book also features recommendations from clinical trial experts and a discussion of relevant regulatory guidelines. This new edition includes more examples and case studies, new approaches for addressing statistical problems, and the following new technological updates: SAS procedures used in group sequential trials (PROC SEQDESIGN and PROC SEQTEST) SAS procedures used in repeated measures analysis (PROC GLIMMIX and PROC GEE) macros for implementing a broad range of randomization-based methods in clinical trials, performing complex multiplicity adjustments, and investigating the design and analysis of early phase trials (Phase I dose-escalation trials and Phase II dose-finding trials) Clinical statisticians, research scientists, and graduate students in biostatistics will greatly benefit from the decades of clinical research experience and the ready-to-use SAS macros compiled in this book.
Essential Statistical Methods for Medical Statistics presents only key contributions which have been selected from the volume in the Handbook of Statistics: Medical Statistics, Volume 27 (2009). While the use of statistics in these fields has a long and rich history, the explosive growth of science in general, and of clinical and epidemiological sciences in particular, has led to the development of new methods and innovative adaptations of standard methods. This volume is appropriately focused for individuals working in these fields. Contributors are internationally renowned experts in their respective areas. · 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
Methodische Grundlagen der Planung, Durchführung und Auswertung
Author: Martin Schumacher,Gabriele Schulgen-Kristiansen
Praxisorientierte Einführung in die methodischen Grundlagen klinischer Studien in Forschung und Industrie. Prinzipien zur Planung und Durchführung sowie die wichtigsten statistischen Verfahren zur Auswertung sind verständlich dargestellt und erklärt: Methodik für Therapiestudien, spezielle Verfahren von Diagnose- und Prognosestudien. Anhand von Beispielen konkreter Studien - vorwiegend aus ihrem eigenen Arbeitsumfeld - illustrieren die Autoren anschaulich Prinzipien und Methoden, und zeigen exemplarisch in der Praxis auftretende Probleme. Plus: historische und neue Entwicklungen, internationale Qualitätsstandards, aktuelle Anforderungen.
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
Preeminent Experts Update a Well-Respected Book Taking into account the regulatory and scientific developments that have occurred since the second edition, Design and Analysis of Bioavailability and Bioequivalence Studies, Third Edition provides a complete presentation of the latest progress of activities and results in bioavailability and bioequivalence on regulatory requirements, scientific and practical issues, and statistical methodology. New to the Third Edition Four new chapters that present a thorough account of novel developments in the field New and updated sections that reflect recent advances in the statistical methodology in the design and analysis of bioavailability and bioequivalence studies Reorganization of the material into five parts, making it easier to access related information together Over 100 new references from the literature Like its bestselling predecessors, this edition covers all of the statistical problems that may occur in the various stages of design and data analysis. Keeping the mathematics and statistics at a fundamental level, it continues to focus on practical concepts rather than technical details.
This edition is a pretty complete textbook and tutorial for medical and health care students, as well as a recollection/update bench, and help desk for professionals. Novel approaches already applied in published clinical research will be addressed: matrix analyses, alpha spending, gate keeping, kriging, interval censored regressions, causality regressions, canonical regressions, quasi-likelihood regressions, novel non-parametric regressions. Each chapter can be studied as a stand-alone, and covers one field in the fast growing world of regression analyses. The authors, as professors in statistics and machine learning at European universities, are worried, that their students find regression-analyses harder than any other methodology in statistics. This is serious, because almost all of the novel methodologies in current data mining and data analysis include elements of regression-analysis. It is the main incentive for writing this 28 chapter edition, consistent of - 28 major fields of regression analysis, - their condensed maths, - their applications in medical and health research as published so far, - step by step analyses for self-assessment, - conclusion and reference sections. Traditional regression analysis is adequate for epidemiology, but lacks the precision required for clinical investigations. However, in the past two decades modern regression methods have proven to be much more precise. And so it is time, that a book described regression analyses for clinicians. The current edition is the first to do so. It is written for a non-mathematical readership. Self-assessment data-files are provided through Springer' s "Extras Online".
The US Food and Drug Administration's Report to the Nation in 2004 and 2005 indicated that one of the top reasons for drug recall was that stability data did not support existing expiration dates. Pharmaceutical companies conduct stability studies to characterize the degradation of drug products and to estimate drug shelf life. Illustrating how stability studies play an important role in drug safety and quality assurance, Statistical Design and Analysis of Stability Studies presents the principles and methodologies in the design and analysis of stability studies. After introducing the basic concepts of stability testing, the book focuses on short-term stability studies and reviews several methods for estimating drug expiration dating periods. It then compares some commonly employed study designs and discusses both fixed and random batch statistical analyses. Following a chapter on the statistical methods for stability analysis under a linear mixed effects model, the book examines stability analyses with discrete responses, multiple components, and frozen drug products. In addition, the author provides statistical methods for dissolution testing and explores current issues and recent developments in stability studies. To ensure the safety of consumers, professionals in the field must carry out stability studies to determine the reliability of drug products during their expiration period. This book provides the material necessary for you to perform stability designs and analyses in pharmaceutical research and development.
This book is comprised of the presentations delivered at the 25th ICSA Applied Statistics Symposium held at the Hyatt Regency Atlanta, on June 12-15, 2016. This symposium attracted more than 700 statisticians and data scientists working in academia, government, and industry from all over the world. The theme of this conference was the “Challenge of Big Data and Applications of Statistics,” in recognition of the advent of big data era, and the symposium offered opportunities for learning, receiving inspirations from old research ideas and for developing new ones, and for promoting further research collaborations in the data sciences. The invited contributions addressed rich topics closely related to big data analysis in the data sciences, reflecting recent advances and major challenges in statistics, business statistics, and biostatistics. Subsequently, the six editors selected 19 high-quality presentations and invited the speakers to prepare full chapters for this book, which showcases new methods in statistics and data sciences, emerging theories, and case applications from statistics, data science and interdisciplinary fields. The topics covered in the book are timely and have great impact on data sciences, identifying important directions for future research, promoting advanced statistical methods in big data science, and facilitating future collaborations across disciplines and between theory and practice.
State-of-the-Art Methods for Drug Safety Assessment Responding to the increased scrutiny of drug safety in recent years, Quantitative Evaluation of Safety in Drug Development: Design, Analysis and Reporting explains design, monitoring, analysis, and reporting issues for both clinical trials and observational studies in biopharmaceutical product development. It presents the latest statistical methods for drug safety assessment. The book’s three sections focus on study design, safety monitoring, and data evaluation/analysis. The book addresses key challenges across regulatory agencies, industry, and academia. It discusses quantitative approaches to safety evaluation and risk management in drug development, covering Bayesian methods, effective safety graphics, and risk-benefit evaluation. Written by a team of experienced leaders, this book brings the most advanced knowledge and statistical methods of drug safety to the statistical, clinical, and safety community. It shares best practices and stimulates further research and methodology development in the drug safety area.
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.
The core principles of statistical analysis are too easily forgotten in today’s world of powerful computers and time-saving algorithms. This step-by-step primer takes researchers who lack the confidence to conduct their own analyses right back to basics, allowing them to scrutinize their own data through a series of rapidly executed reckonings on a simple pocket calculator. A range of easily navigable tutorials facilitate the reader’s assimilation of the techniques, while a separate chapter on next generation Flash prepares them for future developments in the field. This practical volume also contains tips on how to deny hackers access to Flash internet sites. An ideal companion to the author’s co-authored works on statistical analysis for Springer such as Statistics Applied to Clinical Trials, this monograph will help researchers understand the processes involved in interpreting clinical data, as well as being a necessary prerequisite to mastering more advanced statistical techniques. The principles of statistical analysis are easily forgotten in today’s world of time-saving algorithms. This step-by-step primer takes researchers back to basics, enabling them to examine their own data through a series of sums on a simple pocket calculator.
Analyzing Longitudinal Clinical Trial Data: A Practical Guide provide practical and easy to implement approaches for bringing the latest theory on analysis of longitudinal clinical trial data into routine practice.?This book, with its example-oriented approach that includes numerous SAS and R code fragments, is an essential resource for statisticians and graduate students specializing in medical research. The authors provide clear descriptions of the relevant statistical theory and illustrate practical considerations for modeling longitudinal data. Topics covered include choice of endpoint and statistical test; modeling means and the correlations between repeated measurements; accounting for covariates; modeling categorical data; model verification; methods for incomplete (missing) data that includes the latest developments in sensitivity analyses, along with approaches for and issues in choosing estimands; and means for preventing missing data. Each chapter stands alone in its coverage of a topic. The concluding chapters provide detailed advice on how to integrate these independent topics into an over-arching study development process and statistical analysis plan.
Von A wie Ausreißer bis Z wie Z-Verteilung Entdecken Sie mit Statistik für Dummies Ihren Spaß an der Statistik und werfen Sie einen Blick hinter die Kulissen dieser komplizierten, aber hilfreichen Wissenschaft! Deborah Rumsey zeigt Ihnen das nötige statistische Handwerkszeug wie Stichprobe, Wahrscheinlichkeit, Bias, Median, Durchschnitt und Korrelation. Sie lernen die verschiedenen grafischen Darstellungsmöglichkeiten von statistischem Material kennen und werden über die unterschiedlichen Methoden der Auswertung erstaunt sein. Schärfen Sie mit diesem Buch Ihr Bewusstsein für Zahlen und deren Interpretation, sodass Ihnen keiner mehr etwas vormachen kann!
Succinct yet thorough, this updated edition offers the best knowledge on epidemiology, biostatistics, preventive medicine, and public health in one convenient source. This text also serves as an outstanding resource for preparing for the USMLE, and the American Board of Preventive Medicine, and includes STUDENT CONSULT online access.