Statistical Methods for Evaluating Safety in Medical Product Development

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.

Using a series of over 40 case studies, this valuable text illustrates the processes and pitfalls involved in evaluating psychological research. Invites students to consider whether the conclusion drawn at the end of each case is correct, or whether the results could have an alternative explanation. Cross-referencing between sections is made easy by page references that link the case studies and explanations. Cases reflect the range of research methods taught at undergraduate level and include qualitative research. Gives students an understanding of methodological problems. Equips students to critically evaluate published research. The author and publishers have made every effort to be fair to the authors of the research described in this book. Like all sciences, psychology is forged in the fire of criticism and refinement, and we hope that the original researchers will understand that any criticism is made with the utmost respect. If you are an author of one of the pieces of research mentioned in the book and would like to respond to the critique presented, please e-mail your comments to Andrew McAleer at Andrew.McAleer@oxon.blackwellpublishing.com.

Statistical data are not always precise numbers, or vectors, or categories. Real data are frequently what is called fuzzy. Examples where this fuzziness is obvious are quality of life data, environmental, biological, medical, sociological and economics data. Also the results of measurements can be best described by using fuzzy numbers and fuzzy vectors respectively. Statistical analysis methods have to be adapted for the analysis of fuzzy data. In this book, the foundations of the description of fuzzy data are explained, including methods on how to obtain the characterizing function of fuzzy measurement results. Furthermore, statistical methods are then generalized to the analysis of fuzzy data and fuzzy a-priori information. Key Features: Provides basic methods for the mathematical description of fuzzy data, as well as statistical methods that can be used to analyze fuzzy data. Describes methods of increasing importance with applications in areas such as environmental statistics and social science. Complements the theory with exercises and solutions and is illustrated throughout with diagrams and examples. Explores areas such quantitative description of data uncertainty and mathematical description of fuzzy data. This work is aimed at statisticians working with fuzzy logic, engineering statisticians, finance researchers, and environmental statisticians. It is written for readers who are familiar with elementary stochastic models and basic statistical methods.

Praise for the First Edition « . . . the book is a valuable addition to the literature in the field, serving as a much-needed guide for both clinicians and advanced students.»—Zentralblatt MATH A new edition of the cutting-edge guide to diagnostic tests in medical research In recent years, a considerable amount of research has focused on evolving methods for designing and analyzing diagnostic accuracy studies. Statistical Methods in Diagnostic Medicine, Second Edition continues to provide a comprehensive approach to the topic, guiding readers through the necessary practices for understanding these studies and generalizing the results to patient populations. Following a basic introduction to measuring test accuracy and study design, the authors successfully define various measures of diagnostic accuracy, describe strategies for designing diagnostic accuracy studies, and present key statistical methods for estimating and comparing test accuracy. Topics new to the Second Edition include: Methods for tests designed to detect and locate lesions Recommendations for covariate-adjustment Methods for estimating and comparing predictive values and sample size calculations Correcting techniques for verification and imperfect standard biases Sample size calculation for multiple reader studies when pilot data are available Updated meta-analysis methods, now incorporating random effects Three case studies thoroughly showcase some of the questions and statistical issues that arise in diagnostic medicine, with all associated data provided in detailed appendices. A related web site features Fortran, SAS®, and R software packages so that readers can conduct their own analyses. Statistical Methods in Diagnostic Medicine, Second Edition is an excellent supplement for biostatistics courses at the graduate level. It also serves as a valuable reference for clinicians and researchers working in the fields of medicine, epidemiology, and biostatistics.

Praise for the Second Edition «As a comprehensive statistics reference book for quality improvement, it certainly is one of the best books available.» —Technometrics This new edition continues to provide the most current, proven statistical methods for quality control and quality improvement The use of quantitative methods offers numerous benefits in the fields of industry and business, both through identifying existing trouble spots and alerting management and technical personnel to potential problems. Statistical Methods for Quality Improvement, Third Edition guides readers through a broad range of tools and techniques that make it possible to quickly identify and resolve both current and potential trouble spots within almost any manufacturing or nonmanufacturing process. The book provides detailed coverage of the application of control charts, while also exploring critical topics such as regression, design of experiments, and Taguchi methods. In this new edition, the author continues to explain how to combine the many statistical methods explored in the book in order to optimize quality control and improvement. The book has been thoroughly revised and updated to reflect the latest research and practices in statistical methods and quality control, and new features include: Updated coverage of control charts, with newly added tools The latest research on the monitoring of linear profiles and other types of profiles Sections on generalized likelihood ratio charts and the effects of parameter estimation on the properties of CUSUM and EWMA procedures New discussions on design of experiments that include conditional effects and fraction of design space plots New material on Lean Six Sigma and Six Sigma programs and training Incorporating the latest software applications, the author has added coverage on how to use Minitab software to obtain probability limits for attribute charts. new exercises have been added throughout the book, allowing readers to put the latest statistical methods into practice. Updated references are also provided, shedding light on the current literature and providing resources for further study of the topic. Statistical Methods for Quality Improvement, Third Edition is an excellent book for courses on quality control and design of experiments at the upper-undergraduate and graduate levels. the book also serves as a valuable reference for practicing statisticians, engineers, and physical scientists interested in statistical quality improvement.

A new edition of the most comprehensive overview of statistical methods for environmental monitoring applications Thoroughly updated to provide current research findings, Statistical Methods for Groundwater Monitoring, Second Edition continues to provide a comprehensive overview and accessible treatment of the statistical methods that are useful in the analysis of environmental data. This new edition expands focus on statistical comparison to regulatory standards that are a vital part of assessment, compliance, and corrective action monitoring in the environmental sciences. The book explores quantitative concepts useful for surface water monitoring as well as soil and air monitoring applications while also maintaining a focus on the analysis of groundwater monitoring data in order to detect environmental impacts from a variety of sources, such as industrial activity and waste disposal. The authors introduce the statistical properties of alternative approaches, such as false positive and false negative rates, that are associated with each test and the factors related to these error rates. The Second Edition also features: An introduction to Intra-laboratory Calibration Curves and random-effects regression models for non-constant measurement variability Coverage of statistical prediction limits for a gamma-distributed random variable, with a focus on estimation and testing of parameters in environmental monitoring applications A unified treatment of censored data with the computation of statistical prediction, tolerance, and control limits Expanded coverage of statistical issues related to laboratory practice, such as detection and quantitation limits An updated chapter on regulatory issues that outlines common mistakes to avoid in groundwater monitoring applications as well as an introduction to the newest regulations for both hazardous and municipal solid waste facilities Each chapter provides a general overview of a problem, followed by statistical derivation of the solution and a relevant example complete with computational details that allow readers to perform routine application of the statistical results. Relevant issues are highlighted throughout, and recommendations are also provided for specific problems based on characteristics such as number of monitoring wells, number of constituents, distributional form of measurements, and detection frequency. Statistical Methods for Groundwater Monitoring, Second Edition is an excellent supplement to courses on environmental statistics at the upper-undergraduate and graduate levels. It is also a valuable resource for researchers and practitioners in the fields of biostatistics, engineering, and the environmental sciences who work with statistical methods in their everyday work.

The recording and analysis of food data are becoming increasingly sophisticated. Consequently, the food scientist in industry or at study faces the task of using and understanding statistical methods. Statistics is often viewed as a difficult subject and is often avoided because of its complexity and a lack of specific application to the requirements of food science. This situation is changing – there is now much material on multivariate applications for the more advanced reader, but a case exists for a univariate approach aimed at the non-statistician. This second edition of Statistical Methods for Food Science provides a source text on accessible statistical procedures for the food scientist, and is aimed at professionals and students in food laboratories where analytical, instrumental and sensory data are gathered and require some form of summary and analysis before interpretation. It is suitable for the food analyst, the sensory scientist and the product developer, and others who work in food-related disciplines involving consumer survey investigations will also find many sections of use. There is an emphasis on a ‘hands-on’ approach, and worked examples using computer software packages and the minimum of mathematical formulae are included. The book is based on the experience and practice of a scientist engaged for many years in research and teaching of analytical and sensory food science at undergraduate and post-graduate level. This revised and updated second edition is accompanied by a new companion website giving the reader access to the datasets and Excel spreadsheets featured in the book. Check it out now by visiting www.wiley.com/go/bower/statistical or by scanning the QR code below.

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.

The definitive resource for survey questionnaire testing and evaluation Over the past two decades, methods for the development, evaluation, and testing of survey questionnaires have undergone radical change. Research has now begun to identify the strengths and weaknesses of various testing and evaluation methods, as well as to estimate the methods’ reliability and validity. Expanding and adding to the research presented at the International Conference on Questionnaire Development, Evaluation and Testing Methods, this title presents the most up-to-date knowledge in this burgeoning field. The only book dedicated to the evaluation and testing of survey questionnaires, this practical reference work brings together the expertise of over fifty leading, international researchers from a broad range of fields. The volume is divided into seven sections: Cognitive interviews Mode of administration Supplements to conventional pretests Special populations Experiments Multi-method applications Statistical modeling Comprehensive and carefully edited, this groundbreaking text offers researchers a solid foundation in the latest developments in testing and evaluating survey questionnaires, as well as a thorough introduction to emerging techniques and technologies.

Assessment Methods in Statistical Education: An International Perspective provides a modern, international perspective on assessing students of statistics in higher education. It is a collection of contributions written by some of the leading figures in statistical education from around the world, drawing on their personal teaching experience and educational research. The book reflects the wide variety of disciplines, such as business, psychology and the health sciences, which include statistics teaching and assessment. The authors acknowledge the increasingly important role of technology in assessment, whether it be using the internet for accessing information and data sources or using software to construct and manage individualised or online assessments. Key Features: Presents successful assessment strategies, striking a balance between formative and summative assessment, individual and group work, take-away assignments and supervised tests. Assesses statistical thinking by questioning students’ ability to interpret and communicate the results of their analysis. Relates assessment to the real world by basing it on real data in an appropriate context. Provides a range of individualised assessment methods, including those that deter plagiarism and collusion by providing each student with a unique problem to solve or dataset to analyse. This book is essential reading for anyone involved in teaching statistics at tertiary level or interested in statistical education research.