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Value of Information for Healthcare Decision-Making introduces the concept of Value of Information (VOI) use in health policy decision-making to determine the sensitivity of decisions to assumptions, and to prioritise and design future research. These methods, and their use in cost-effectiveness analysis, are increasingly acknowledged by health technology assessment authorities as vital. Key Features: Provides a comprehensive overview of VOI Simplifies VOI Showcases state-of-the-art techniques for computing VOI Includes R statistical software package Provides results when using VOI methods Uses realistic decision model to illustrate key concepts The primary audience for this book is health economic modellers and researchers, in industry, government, or academia, who wish to perform VOI analysis in health economic evaluations. It is relevant for postgraduate researchers and students in health economics or medical statistics who are required to learn the principles of VOI or undertake VOI analyses in their projects. The overall goal is to improve the understanding of these methods and make them easier to use.
Disease screening and disease surveillance (DSDS) constitute two critical areas in public health, each presenting distinctive challenges primarily due to their sequential decision-making nature and complex data structures. Statistical Methods for Dynamic Disease Screening and Spatio-Temporal Disease Surveillance explores numerous recent analytic methodologies that enhance traditional techniques. The author, a prominent researcher specializing in innovative sequential decision-making techniques, demonstrates how these novel methods effectively address the challenges of DSDS. After a concise introduction that lays the groundwork for comprehending the challenges inherent in DSDS, the book delve...
Bayesian Precision Medicine presents modern Bayesian statistical models and methods for identifying treatments tailored to individual patients using their prognostic variables and predictive biomarkers. The process of evaluating and comparing treatments is explained and illustrated by practical examples, followed by a discussion of causal analysis and its relationship to statistical inference. A wide array of modern Bayesian clinical trial designs are presented, including applications to many oncology trials. The later chapters describe Bayesian nonparametric regression analyses of datasets arising from multistage chemotherapy for acute leukemia, allogeneic stem cell transplantation, and tar...
Association Models in Epidemiology: Study Designs, Modeling Strategies, and Analytic Methods is written by an epidemiologist for graduate students, researchers, and practitioners who will use regression techniques to analyze data. It focuses on association models rather than prediction models. The book targets students and working professionals who lack bona fide modeling experts but are committed to conducting appropriate regression analyses and generating valid findings from their projects. This book aims to offer detailed strategies to guide them in modeling epidemiologic data. Features Custom-Tailored Models: Discover association models specifically designed for epidemiologic study desig...
Causal Inference in Pharmaceutical Statistics introduces the basic concepts and fundamental methods of causal inference relevant to pharmaceutical statistics. This book covers causal thinking for different types of commonly used study designs in the pharmaceutical industry, including but not limited to randomized controlled clinical trials, longitudinal studies, singlearm clinical trials with external controls, and real-world evidence studies. The book starts with the central questions in drug development and licensing, takes the reader through the basic concepts and methods via different study types and through different stages, and concludes with a roadmap to conduct causal inference in cl...
Many conventional survival analysis methods, such as the Kaplan-Meier method for survival function estimation and the partial likelihood method for Cox model regression coefficients estimation, were developed under the assumption that survival times are subject to right censoring only. However, in practice, survival time observations may include interval-censored data, especially when the exact time of the event of interest cannot be observed. When interval-censored observations are present in a survival dataset, one generally needs to consider likelihood-based methods for inference. If the survival model under consideration is fully parametric, then likelihood-based methods impose neither t...
This book traces the history of German records captured by American and British troops in 1945 and the negotiations for their return into German custody.
Cell and gene therapies have become the third major drug modality in pharmaceutical medicine of the 21st century after low molecular weight and antibody drugs. The gene therapy (GTx) field is rapidly advancing, and yet there are still fundamental scientific questions that remain to be answered. Development of GTx products poses unique challenges and opportunities for drug developers. However, there is lack of a systematic exposition of the GTx product development and the pivotal role of the biostatistician in this process. Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, s...
Cancer screening has been carried out for six decades – however, there are many unsolved problems: how to estimate key parameters involved in screenings, such as sensitivity, the time duration in the preclinical state (i.e., sojourn time), and time duration in the disease-free state; how to estimate the distribution of lead time, the diagnosis time advanced by screening; how to evaluate the long-term outcomes of screening, including the probability of overdiagnosis among the screen-detected; when to schedule the first exam based on one’s current age and risk tolerance; and when to schedule the upcoming exam based on one’s screening history, age, and risk tolerance. These problems need ...
This unique book officially defines microbiome statistics as a specific new field of statistics and addresses the statistical analysis of correlation, association, interaction, and composition in microbiome research. It also defines the study of the microbiome as a hypothesis-driven experimental science and describes two microbiome research themes and six unique characteristics of microbiome data, as well as investigating challenges for statistical analysis of microbiome data using the standard statistical methods. This book is useful for researchers of biostatistics, ecology, and data analysts. Presents a thorough overview of statistical methods in microbiome statistics of parametric and no...