Introduces many of the practical adaptive statistical methods and provides a comprehensive approach to tests of significance and confidence intervals.

Statistical Methods in Healthcare 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.

Already popular in the analysis of medical device trials, adaptive Bayesian designs are increasingly being used in drug development for a wide variety of diseases and conditions, from Alzheimer's disease and multiple sclerosis to obesity, diabetes, hepatitis C, and HIV. Written by leading pioneers of Bayesian clinical trial designs, Bayesian Adapti

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.

In response to the US FDA's Critical Path Initiative, innovative adaptive designs are being used more and more in clinical trials due to their flexibility and efficiency, especially during early phase development. Handbook of Adaptive Designs in Pharmaceutical and Clinical Development provides a comprehensive and unified presentation of the princip

With new statistical and scientific issues arising in adaptive clinical trial design, including the U.S. FDA's recent draft guidance, a new edition of one of the first books on the topic is needed. Adaptive Design Methods in Clinical Trials, Second Edition reflects recent developments and regulatory positions on the use of adaptive designs in clini

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.?

Provides the tools needed to successfully perform adaptive tests across a broad range of datasets Adaptive Tests of Significance Using Permutations of Residuals with R and SAS illustrates the power of adaptive tests and showcases their ability to adjust the testing method to suit a particular set of data. The book utilizes state-of-the-art software to demonstrate the practicality and benefits for data analysis in various fields of study. Beginning with an introduction, the book moves on to explore the underlying concepts of adaptive tests, including: Smoothing methods and normalizing transformations Permutation tests with linear methods Applications of adaptive tests Multicenter and cross-over trials Analysis of repeated measures data Adaptive confidence intervals and estimates Throughout the book, numerous figures illustrate the key differences among traditional tests, nonparametric tests, and adaptive tests. R and SAS software packages are used to perform the discussed techniques, and the accompanying datasets are available on the book's related website. In addition, exercises at the end of most chapters enable readers to analyze the presented datasets by putting new concepts into practice. Adaptive Tests of Significance Using Permutations of Residuals with R and SAS is an insightful reference for professionals and researchers working with statistical methods across a variety of fields including the biosciences, pharmacology, and business. The book also serves as a valuable supplement for courses on regression analysis and adaptive analysis at the upper-undergraduate and graduate levels.

Modern statistics deals with large and complex data sets, and consequently with models containing a large number of parameters. This book presents a detailed account of recently developed approaches, including the Lasso and versions of it for various models, boosting methods, undirected graphical modeling, and procedures controlling false positive selections. A special characteristic of the book is that it contains comprehensive mathematical theory on high-dimensional statistics combined with methodology, algorithms and illustrations with real data examples. This in-depth approach highlights the methods’ great potential and practical applicability in a variety of settings. As such, it is a valuable resource for researchers, graduate students and experts in statistics, applied mathematics and computer science.

This Festschrift is dedicated to Götz Trenkler on the occasion of his 65th birthday. As can be seen from the long list of contributions, Götz has had and still has an enormous range of interests, and colleagues to share these interests with. He is a leading expert in linear models with a particular focus on matrix algebra in its relation to statistics. He has published in almost all major statistics and matrix theory journals. His research activities also include other areas (like nonparametrics, statistics and sports, combination of forecasts and magic squares, just to mention afew). Götz Trenkler was born in Dresden in 1943. After his school years in East G- many and West-Berlin, he obtained a Diploma in Mathematics from Free University of Berlin (1970), where he also discovered his interest in Mathematical Statistics. In 1973, he completed his Ph.D. with a thesis titled: On a distance-generating fu- tion of probability measures. He then moved on to the University of Hannover to become Lecturer and to write a habilitation-thesis (submitted 1979) on alternatives to the Ordinary Least Squares estimator in the Linear Regression Model, a topic that would become his predominant ?eld of research in the years to come.