This book covers classic epidemiological designs that use a reference/control group, including case-control, case-cohort, nested case-control and variations of these designs, such as stratified and two-stage designs. It presents a unified view of these sampling designs as representations of an underlying cohort or target population of interest. This enables various extended designs to be introduced and analysed with a similar approach: extreme sampling on the outcome (extreme case-control design) or on the exposure (exposure-enriched, exposure-density, countermatched), designs that re-use prior controls and augmentation sampling designs. Further extensions exploit aggregate data for efficient cluster sampling, accommodate time-varying exposures and combine matched and unmatched controls. Self-controlled designs, including case-crossover, self-controlled case series and exposure-crossover, are also presented. The test-negative design for vaccine studies and the use of negative controls for bias assessment are introduced and discussed. This book is intended for graduate students in biostatistics, epidemiology and related disciplines, or for health researchers and data analysts interested in extending their knowledge of study design and data analysis skills. This book Bridges the gap between epidemiology and the more mathematically oriented biostatistics books. Assembles the wealth of epidemiological knowledge about observational study designs that is scattered over several decades of scientific publications. Illustrates the performance of methods in real research applications. Provides guidelines for implementation in standard software packages (Stata, R). Includes numerous exercises, covering simple mathematical proofs, consideration of proposed or published designs, and practical data analysis.

This remarkable text raises the analysis of data in health sciences and policy to new heights of refinement and applicability by introducing cutting-edge meta-analysis strategies while reviewing more commonly used techniques. Each chapter builds on sound principles, develops methodologies to solve statistical problems, and presents concrete applications used by experienced medical practitioners and health policymakers. Written by more than 30 celebrated international experts, Meta-Analysis in Medicine and Health Policy employs copious examples and pictorial presentations to teach and reinforce biostatistical techniques more effectively and poses numerous open questions of medical and health policy research.

Neglected Diseases: Extensive Space for Modern Drug Discovery provides in-depth reviews on the last progresses about neglected tropical diseases research. Topics covered in this volume include Leishmaniasis, Tripanosomiasis, Onchocerciasis and Ebolavirus infections, with insights on the future of the research on them. Part of the volume is devoted to recent contributions this field received from X-Ray crystallography.

This book begins with an introduction of pragmatic cluster randomized trials (PCTs) and reviews various pragmatic issues that need to be addressed by statisticians at the design stage. It discusses the advantages and disadvantages of each type of PCT, and provides sample size formulas, sensitivity analyses, and examples for sample size calculation. The generalized estimating equation (GEE) method will be employed to derive sample size formulas for various types of outcomes from the exponential family, including continuous, binary, and count variables. Experimental designs that have been frequently employed in PCTs will be discussed, including cluster randomized designs, matched-pair cluster randomized design, stratified cluster randomized design, stepped-wedge cluster randomized design, longitudinal cluster randomized design, and crossover cluster randomized design. It demonstrates that the GEE approach is flexible to accommodate pragmatic issues such as hierarchical correlation structures, different missing data patterns, randomly varying cluster sizes, etc. It has been reported that the GEE approach leads to under-estimated variance with limited numbers of clusters. The remedy for this limitation is investigated for the design of PCTs. This book can assist practitioners in the design of PCTs by providing a description of the advantages and disadvantages of various PCTs and sample size formulas that address various pragmatic issues, facilitating the proper implementation of PCTs to improve health care. It can also serve as a textbook for biostatistics students at the graduate level to enhance their knowledge or skill in clinical trial design. Key Features: Discuss the advantages and disadvantages of each type of PCTs, and provide sample size formulas, sensitivity analyses, and examples. Address an unmet need for guidance books on sample size calculations for PCTs; A wide variety of experimental designs adopted by PCTs are covered; The sample size solutions can be readily implemented due to the accommodation of common pragmatic issues encountered in real-world practice; Useful to both academic and industrial biostatisticians involved in clinical trial design; Can be used as a textbook for graduate students majoring in statistics and biostatistics.

An understanding of each of the critical components of the funding process is key to meeting the challenges posed by the increasingly intense competition for research funds. This book is a vital tool for those who want to build and maximize their grant support. Although many publications provide valuable information about proposal preparation, few cover the full spectrum of issues--from planning through execution--in the funding process. The book leads off with a discussion of the relationship between researchers and the funding environment, features of good short- and long-range funding plans, characteristics of funding organizations in terms of funding power, mission, and priorities, and the manner in which funding information is disseminated. Succeeding chapters focus on the actual development of the many different types of opportunities--research projects, multicomponent research programs, career development and training programs, and small business innovation research. These chapters emphasize conceptualizing an idea, optimizing the researcher-sponsor match, and testing the concept for competitiveness. Further chapters deliver strategies for translating research ideas into written proposals, preparing administrative sections and communicating with a sponsor. The final chapters are dedicated to the outcomes of the proposal process: reviews, rebuttals, and resubmissions; and to progress reports and future proposals for maintaining and building on funding. Flowcharts, examples, and summary tables are used throughout the text to highlight key points.

Counterfeit drugs continue to infiltrate the drug market in the United States, causing illness and death. This book addresses this issue and examines the recent trends in drug counterfeiting over the past 5-10 years. The text shows perspectives from crime lab and toxicology lab personnel and academic researchers, and includes topics such as a history of cases and issues with counterfeit drugs, trends observed in forensic labs, instrumental methods and approaches used in detecting counterfeit medicines, and policy approaches for controlling counterfeit drugs. There is a focus on ways to reduce counterfeit drugs in the market, to help improve the health and safety of people all over the world. Features : Focuses on recent (5-10) year trends in counterfeit drugs and analysis. Shows perspectives from crime lab and toxicology lab personnel and academic researchers. Focuses on drugs seized by law enforcement and approaches to reducing counterfeit medicine in the market. Discusses the detection and analysis of counterfeit drugs, and appropriate tools for combating this issue. Emphasizes the global impact of illegal medicines.

Sphingolipids in Cancer, Volume 140, the latest release in the Advances in Cancer Research series, provides invaluable information on the exciting and fast-moving field of cancer research. Topics discussed in this updated volume include Mechanisms of ceramide-dependent cancer cell death, Sphingolipids as regulators of autophagy and endocytic trafficking, The role and function of sphingomyelin biosynthesis in the development of cancer, Neutral sphingomyelinases in cancer: Friend or foe?, Sphingolipid rendezvous at the crossroad of NAFLD and senescence, Ceramide signaling and p53 pathways, Sphingolipid regulation of RNA Biology in cancer phenotypes, The role of ceramide-1-phosphate in tumor cell survival and dissemination, and more.

A guide through the maze of the pharmaceutical research and development process, Medical Writing in Drug Development fills a gap in the libraries of technical writers, college instructors, and corporate professionals associated with the pharmaceutical process. As it discusses critical information, such as strategies and techniques pivotal to crafting documents for drug development, it also overviews drug research, document types, the roles of professional writers, and information technology. In no time at all, you will be creating persuasive technical documents, building complex facts into coherent messages, and contributing to the effective marketing of new products with promotional pieces that meet legal and ethical standards.Medical Writing in Drug Development helps medical writers and scientific, regulatory, and marketing professionals develop a working knowledge of the technical documents crucial to successful drug research. New and seasoned professional writers alike will benefit from the book's detailed discussions of: using abstracts, slides, and posters to present up-to-the-minute research how patient-education materials, health-economic assessments, and electronic journals provide ongoing challenges in medical writing a dossier approach that expedites regulatory submissions for international drug development structural constraints and rhetorical approaches toward regulatory documents presenting intricate information in scientifically unbiased, yet technically convincing language the effects of electronic publishing, computer graphics, and related technology on the practice of medical writing within pharmaceutical research Practical as a foundation text for undergraduate, graduate, and certificate programs in pharmaceutical or medical technical writing, Medical Writing in Drug Development will help you develop practical strategies for handling journal manuscripts, conference materials, and promotional pieces. No other text will clarify the main aspects of the pharmaceutical research and development process while offering you insight on the key issues dominating the healthcare arena.

This volume describes important medical discoveries, from the introduction of the first antibiotic to the present, where serendipity, intuition, coincidence, or laboratory accident played an important role in bringing a discovery to light. Although chance is the principal determinant, the book emphasizes other factors, such as economic and political exigencies and being in the right place at the right time.

Key Features: 1. Describes the development of the randomized, controlled trial as the gold standard of proof. 2. Unravels the meaning of "randomized," "double-blind" and "p-values" in a simplified manner for students and clinicians. 3. Contains timeless information on how medical evidence can be understood.

This work provides descriptions, explanations and examples of the Bayesian approach to statistics, demonstrating the utility of Bayesian methods for analyzing real-world problems in the health sciences. The work considers the individual components of Bayesian analysis.;College or university bookstores may order five or more copies at a special student price, available on request from Marcel Dekker, Inc.

Presents the latest developments on the interaction of metal complexes with nucleic acids, the building blocks of life. Bioinorganic chemistry is a highly interdisciplinary area of research and is of great interest to scientists working in the fields of coordination chemistry, biochemistry, supramolecular chemistry, nanotechnology, computational chemistry, and inorganic chemistry in general. Includes the latest research in DNA recognition by supramolecular metal complexes. Describes the applications of this exciting area of research in metal-nucleic acid chemistry.

This book deals with pathophysiology and pharmacology of spontaneously hypertensive rats (SHR) and describes new trends in SHR research from hemodynamic characteristics to immunological views. It is devoted to the use of tissue culture studies to elaborate SHR characteristics.

The number of, and interest in, quality of life studies has grown dramatically in the last decade. On an ever increasing basis, patients, clinicians, researchers, and health policy regulators are considering quality of life in assessing treatment alternatives. Unfortunately, most discussions of quality of life are narrow in scope -- applying to only one disease group. This unique book represents the concerted effort of experts in academia, federal government health care regulators, and pharmaceutical industry representatives to define the promise and the problems associated with quality of life studies. The issues covered range from cross cutting ones to those that are specific to particular illnesses.
Because quality of life takes into consideration such domains as mood, vocation, family, sexual functioning, social participation, and costs, this book will serve as an invaluable companion to readers with an interest in behavioral medicine research.

The concepts of estimands, analyses (estimators), and sensitivity are interrelated. Therefore, great need exists for an integrated approach to these topics. This book acts as a practical guide to developing and implementing statistical analysis plans by explaining fundamental concepts using accessible language, providing technical details, real-world examples, and SAS and R code to implement analyses. The updated ICH guideline raises new analytic and cross-functional challenges for statisticians. Gaps between different communities have come to surface, such as between causal inference and clinical trialists, as well as among clinicians, statisticians, and regulators when it comes to communicating decision-making objectives, assumptions, and interpretations of evidence. This book lays out a path toward bridging some of these gaps. It offers A common language and unifying framework along with the technical details and practical guidance to help statisticians meet the challenges A thorough treatment of intercurrent events (ICEs), i.e., postrandomization events that confound interpretation of outcomes and five strategies for ICEs in ICH E9 (R1) Details on how estimands, integrated into a principled study development process, lay a foundation for coherent specification of trial design, conduct, and analysis needed to overcome the issues caused by ICEs: A perspective on the role of the intention-to-treat principle Examples and case studies from various areas Example code in SAS and R A connection with causal inference Implications and methods for analysis of longitudinal trials with missing data Together, the authors have offered the readers their ample expertise in clinical trial design and analysis, from an industrial and academic perspective.

The volume is comprehensively covers emerging technologies for health care, various aspects of biomedical research towards understanding of pathophysiology of the diseases, advances in improvement in diagnostic procedures and therapeutic tools, the fundamental role of biomedical research in the development of new medicinal products

This volume investigates the links between the incidence of diet-related cancers and dietary patterns within Europe. It presents current understanding of the major cancers thought to be caused by diet alongside detailed data on regional variations in dietary composition, and collates these sets of information to illustrate associations between foods and nutrients and the risk of cancer at specific sites. There is particular discussion of the role of fat, meat, fibre, cereals and fresh vegetables. The importance of the "Mediterranean diet", and regional variance in this diet within Europe, is examined. Japanese and US dietary evidence is also considered. This book highlights the debate on cancer and diet, and points the way ahead for important new research.

This book is a continuation of the book Medicine Researcher Biographical Sketches and Research Summaries which compiles biographical sketches of top professionals in the field of medicinal research, as well as research summaries from a number of different focuses in this important field.

Drug development is an iterative process. The recent publications of regulatory guidelines further entail a lifecycle approach. Blending data from disparate sources, the Bayesian approach provides a flexible framework for drug development. Despite its advantages, the uptake of Bayesian methodologies is lagging behind in the field of pharmaceutical development. Written specifically for pharmaceutical practitioners, Bayesian Analysis with R for Drug Development: Concepts, Algorithms, and Case Studies, describes a wide range of Bayesian applications to problems throughout pre-clinical, clinical, and Chemistry, Manufacturing, and Control (CMC) development. Authored by two seasoned statisticians in the pharmaceutical industry, the book provides detailed Bayesian solutions to a broad array of pharmaceutical problems. Features Provides a single source of information on Bayesian statistics for drug development Covers a wide spectrum of pre-clinical, clinical, and CMC topics Demonstrates proper Bayesian applications using real-life examples Includes easy-to-follow R code with Bayesian Markov Chain Monte Carlo performed in both JAGS and Stan Bayesian software platforms Offers sufficient background for each problem and detailed description of solutions suitable for practitioners with limited Bayesian knowledge Harry Yang, Ph.D., is Senior Director and Head of Statistical Sciences at AstraZeneca. He has 24 years of experience across all aspects of drug research and development and extensive global regulatory experiences. He has published 6 statistical books, 15 book chapters, and over 90 peer-reviewed papers on diverse scientific and statistical subjects, including 15 joint statistical works with Dr. Novick. He is a frequent invited speaker at national and international conferences. He also developed statistical courses and conducted training at the FDA and USP as well as Peking University. Steven Novick, Ph.D., is Director of Statistical Sciences at AstraZeneca. He has extensively contributed statistical methods to the biopharmaceutical literature. Novick is a skilled Bayesian computer programmer and is frequently invited to speak at conferences, having developed and taught courses in several areas, including drug-combination analysis and Bayesian methods in clinical areas. Novick served on IPAC-RS and has chaired several national statistical conferences.

Following many reports that were published in the last two decades on correlations of multiphase structures of the surface of materials with their antithrombogenicity or biocompatibility a research project ''Design of Multiphase Biomedical Materials'' was carried out in Japan between 1982 and 1986. The objective of this research project was to elucidate various aspects of biomedical behaviour of multiphase systems at the interface with living bodies at the molecular, cellular and tissue levels. Multiphase materials studied cover polymers having microphase-separated structures, hydrogels, immobilized enzymes (or cells), ceramics and metallic materials.
The research project was carried out by the following subgroups:
-- Multiphase biomedical materials with microdomain structures
-- Multiphase biomedical materials containing liquid components
-- Hybrid-type multiphase biomedical materials with biological components
-- Inorganic and metallic multiphase biomedical materials
-- Methods for analysis and evaluation of multiphase biomedical materials
This book contains the results of the research project in an edited form and aims to provoke a better understanding about various aspects of cell--material interactions in which the multiphase systems play a crucial role.

Combining the latest thinking in the field with practical, step-by-step guidance, the Third Edition of John W. Creswell and Vicki L. Plano Clark's Designing and Conducting Mixed Methods Research now covers seven mixed methods designs with accompanying journal articles illustrating each design. The authors walk readers through the entire research process, and present updated examples from published mixed methods studies drawn from multiple disciplines. In addition, this new edition includes information about the dynamic and evolving nature of the field of mixed methods research, four additional methodological approaches, and coverage of new directions in mixed methods.

Cancer is a major healthcare burden across the world and impacts not only the people diagnosed with various cancers but also their families, carers, and healthcare systems. With advances in the diagnosis and treatment, more people are diagnosed early and receive treatments for a disease where few treatments options were previously available. As a result, the survival of patients with cancer has steadily improved and, in most cases, patients who are not cured may receive multiple lines of treatment, often with financial consequences for the patients, insurers and healthcare systems. Although many books exist that address economic evaluation, Economic Evaluation of Cancer Drugs using Clinical Trial and Real World Data is the first unified text that specifically addresses the economic evaluation of cancer drugs. The authors discuss how to perform cost-effectiveness analyses while emphasising the strategic importance of designing cost-effectiveness into cancer trials and building robust economic evaluation models that have a higher chance of reimbursement if truly cost-effective. They cover the use of real-world data using cancer registries and discuss how such data can support or complement clinical trials with limited follow up. Lessons learned from failed reimbursement attempts, factors predictive of successful reimbursement and the different payer requirements across major countries including US, Australia, Canada, UK, Germany, France and Italy are also discussed. The book includes many detailed practical examples, case studies and thought-provoking exercises for use in classroom and seminar discussions. Iftekhar Khan is a medical statistician and health economist and a lead statistician at Oxford Unviersity's Center for Statistics in Medicine. Professor Khan is also a Senior Research Fellow in Health Economics at University of Warwick and is a Senior Statistical Assessor within the Licensing Division of the UK Medicine and Health Regulation Agency. Ralph Crott is a former professor in Pharmacoeconomics at the University of Montreal in Quebec, Canada and former head of the EORTC Health Economics Unit and former senior health economist at the Belgian HTA organization. Zahid Bashir has over twelve years experience working in the pharmaceutical industry in medical affairs and oncology drug development where he is involved in the design and execution of oncology clinical trials and development of reimbursement dossiers for HTA submission.

* Innovative clinical trial design * Regulatory approval * Real world evidence

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 clinical trials. It unifies the vast and continuously growing literature and research activities on regulatory requirements, scientific and practical issues, and statistical methodology. New to the Second EditionAlong with revisions throughout the text, this edition significantly updates the chapters on protocol amendment and clinical trial simulation to incorporate the latest changes. It also includes five entirely new chapters on two-stage adaptive design, biomarker adaptive trials, target clinical trials, sample size and power estimation, and regulatory perspectives. Following in the tradition of its acclaimed predecessor, this second edition continues to offer an up-to-date resource for clinical scientists and researchers in academia, regulatory agencies, and the pharmaceutical industry. Written in an intuitive style at a basic mathematical and statistical level, the book maintains its practical approach with an emphasis on concepts via numerous examples and illustrations.