Download or read book Rare Diseases and Orphan Products written by Institute of Medicine and published by National Academies Press. This book was released on 2011-04-03 with total page 442 pages. Available in PDF, EPUB and Kindle. Book excerpt: Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.
Download or read book Orphan Drugs and Rare Diseases written by David Pryde and published by Royal Society of Chemistry. This book was released on 2014-07-30 with total page 487 pages. Available in PDF, EPUB and Kindle. Book excerpt: Orphan drugs are designated drug substances that are intended to treat rare or ‘orphan’ diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world’s population; however, individually, any single, rare disease may only affect a handful of people making them commercially unattractive for the biopharmaceutical industry to target. Ground breaking legislation, starting with the Orphan Drug Act that was passed in the US in 1983 to provide financial incentives for companies to develop orphan drugs, has sparked ever increasing interest from biopharmaceutical companies to tackle rare diseases. These developments have made rare diseases, and the orphan drugs that treat them, sufficiently attractive to pharmaceutical development and many pharmaceutical companies now have research units dedicated to this area of research. It is therefore timely to review the area of orphan drugs and some of the basic science, drug discovery and regulatory factors that underpin this important, and growing, area of biomedical research. Written by a combination of academic and industry experts working in the field, this text brings together expert authors in the regulatory, drug development, genetics, biochemistry, patient advocacy group, medicinal chemistry and commercial domains to create a unique and timely reference for all biomedical researchers interested in finding out more about orphan drugs and the rare diseases they treat. Providing an up-to-date monograph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs and will appeal to medicinal and pharmaceutical chemists, biochemists and anyone working within the fields of rare disease research and drug development or pharmaceuticals in industry or academia.
Download or read book Innovative Methods for Rare Disease Drug Development written by Shein-Chung Chow and published by CRC Press. This book was released on 2020-11-11 with total page 286 pages. Available in PDF, EPUB and Kindle. Book excerpt: In the United States, a rare disease is defined by the Orphan Drug Act as a disorder or condition that affects fewer than 200,000 persons. For the approval of "orphan" drug products for rare diseases, the traditional approach of power analysis for sample size calculation is not feasible because there are only limited number of subjects available for clinical trials. In this case, innovative approaches are needed for providing substantial evidence meeting the same standards for statistical assurance as drugs used to treat common conditions. Innovative Methods for Rare Disease Drug Development focuses on biostatistical applications in terms of design and analysis in pharmaceutical research and development from both regulatory and scientific (statistical) perspectives. Key Features: Reviews critical issues (e.g., endpoint/margin selection, sample size requirements, and complex innovative design). Provides better understanding of statistical concepts and methods which may be used in regulatory review and approval. Clarifies controversial statistical issues in regulatory review and approval accurately and reliably. Makes recommendations to evaluate rare diseases regulatory submissions. Proposes innovative study designs and statistical methods for rare diseases drug development, including n-of-1 trial design, adaptive trial design, and master protocols like platform trials. Provides insight regarding current regulatory guidance on rare diseases drug development like gene therapy.
Download or read book The Waxman Report written by Henry Waxman and published by Twelve. This book was released on 2009-07-02 with total page 147 pages. Available in PDF, EPUB and Kindle. Book excerpt: At a time when some of the most sweeping national initiatives in decades are being debated, Congressman Henry Waxman offers a fascinating inside account of how Congress really works by describing the subtleties and complexities of the legislative process. For four decades, Waxman has taken visionary and principled positions on crucial issues and been a driving force for change. Because of legislation he helped champion, our air is cleaner, our food is safer, and our medical care better. Thanks to his work as a top watchdog in Congress, crucial steps have been taken to curb abuses on Wall Street, to halt wasteful spending in Iraq, and to ban steroids from Major League Baseball. Few legislators can match his accomplishments or his insights on how good work gets done in Washington. In this book, Waxman affords readers a rare glimpse into how this is achieved-the strategy, the maneuvering, the behind-the-scenes deals. He shows how the things we take for granted (clear information about tobacco's harmfulness, accurate nutritional labeling, important drugs that have saved countless lives) started out humbly-derided by big business interests as impossible or even destructive. Sometimes, the most dramatic breakthroughs occur through small twists of fate or the most narrow voting margin. Waxman's stories are surprising because they illustrate that while government's progress may seem glacial, much is happening, and small battles waged over years can yield great results. At a moment when so much has been written about what's wrong with Congress-the grid, the partisanship, the influence of interest groups-Henry Waxman offers sophisticated, concrete examples of how government can (and should) work.
Download or read book Orphan Drug Law Matures into Medical Mainstay written by and published by DIANE Publishing. This book was released on with total page 12 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Introduction to Basics of Pharmacology and Toxicology written by Gerard Marshall Raj and published by Springer Nature. This book was released on 2019-11-16 with total page 410 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book illustrates, in a comprehensive manner, the most crucial principles involved in pharmacology and allied sciences. The title begins by discussing the historical aspects of drug discovery, with up to date knowledge on Nobel Laureates in pharmacology and their significant discoveries. It then examines the general pharmacological principles - pharmacokinetics and pharmacodynamics, with in-depth information on drug transporters and interactions. In the remaining chapters, the book covers a definitive collection of topics containing essential information on the basic principles of pharmacology and how they are employed for the treatment of diseases. Readers will learn about special topics in pharmacology that are hard to find elsewhere, including issues related to environmental toxicology and the latest information on drug poisoning and treatment, analytical toxicology, toxicovigilance, and the use of molecular biology techniques in pharmacology. The book offers a valuable resource for researchers in the fields of pharmacology and toxicology, as well as students pursuing a degree in or with an interest in pharmacology.
Download or read book Rare Diseases Epidemiology Update and Overview written by Manuel Posada de la Paz and published by Springer. This book was released on 2017-12-06 with total page 675 pages. Available in PDF, EPUB and Kindle. Book excerpt: The fields of rare diseases research and orphan products development continue to expand with more products in research and development status. In recent years, the role of the patient advocacy groups has evolved into a research partner with the academic research community and the bio-pharmaceutical industry. Unique approaches to research and development require epidemiological data not previously available to assist in protocol study design and patient recruitment for clinical trials required by regulatory agencies prior to approval for access by patents and practicing physicians.
Download or read book Principles and Practice of Clinical Trials written by Steven Piantadosi and published by Springer Nature. This book was released on 2022-07-19 with total page 2573 pages. Available in PDF, EPUB and Kindle. Book excerpt: This is a comprehensive major reference work for our SpringerReference program covering clinical trials. Although the core of the Work will focus on the design, analysis, and interpretation of scientific data from clinical trials, a broad spectrum of clinical trial application areas will be covered in detail. This is an important time to develop such a Work, as drug safety and efficacy emphasizes the Clinical Trials process. Because of an immense and growing international disease burden, pharmaceutical and biotechnology companies continue to develop new drugs. Clinical trials have also become extremely globalized in the past 15 years, with over 225,000 international trials ongoing at this point in time. Principles in Practice of Clinical Trials is truly an interdisciplinary that will be divided into the following areas: 1) Clinical Trials Basic Perspectives 2) Regulation and Oversight 3) Basic Trial Designs 4) Advanced Trial Designs 5) Analysis 6) Trial Publication 7) Topics Related Specific Populations and Legal Aspects of Clinical Trials The Work is designed to be comprised of 175 chapters and approximately 2500 pages. The Work will be oriented like many of our SpringerReference Handbooks, presenting detailed and comprehensive expository chapters on broad subjects. The Editors are major figures in the field of clinical trials, and both have written textbooks on the topic. There will also be a slate of 7-8 renowned associate editors that will edit individual sections of the Reference.
Download or read book Orphan Drug Act written by United States. Congress. House. Committee on Energy and Commerce. Subcommittee on Health and the Environment and published by . This book was released on 1990 with total page 268 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Drug Repurposing written by Farid A. Badria and published by BoD – Books on Demand. This book was released on 2020-12-02 with total page 236 pages. Available in PDF, EPUB and Kindle. Book excerpt: Drug repurposing or drug repositioning is a new approach to presenting new indications for common commercial and clinically approved existing drugs. For example, chloroquine, an old antimalarial drug, showed promising results for treating COVID-19, interfering with MDR in several types of cancer, and chemosensitizing human leukemic cells.This book focuses on the hypothesis, risk/benefits, and economic impacts of drug repurposing on drug discovery in dermatology, infectious diseases, neurological disorders, cancer, and orphan diseases. It brings together up-to-date research to provide readers with an informative, illustrative, and easy-to-read book useful for students, clinicians, and the pharmaceutical industry.
Download or read book NORD Guide to Rare Disorders written by National Organization for Rare Disorders and published by Lippincott Williams & Wilkins. This book was released on 2003 with total page 982 pages. Available in PDF, EPUB and Kindle. Book excerpt: NORD Guide to Rare Disorders is a comprehensive, practical, authoritative guide to the diagnosis and management of more than 800 rare diseases. The diseases are discussed in a uniform, easy-to-follow format--a brief description, signs and symptoms, etiology, related disorders, epidemiology, standard treatment, investigational treatment, resources, and references.The book includes a complete directory of orphan drugs, a full-color atlas of visual diagnostic signs, and a Master Resource List of support groups and helpful organizations. An index of symptoms and key words offers physicians valuable assistance in finding the information they need quickly.
Download or read book Rare Disease Drug Development written by Raymond A. Huml and published by Springer Nature. This book was released on 2021-11-08 with total page 418 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case for real-world data and evidence generation in orphan medicinal drug development. Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers.
Download or read book Registries for Evaluating Patient Outcomes written by Agency for Healthcare Research and Quality/AHRQ and published by Government Printing Office. This book was released on 2014-04-01 with total page 396 pages. Available in PDF, EPUB and Kindle. Book excerpt: This User’s Guide is intended to support the design, implementation, analysis, interpretation, and quality evaluation of registries created to increase understanding of patient outcomes. For the purposes of this guide, a patient registry is an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy purposes. A registry database is a file (or files) derived from the registry. Although registries can serve many purposes, this guide focuses on registries created for one or more of the following purposes: to describe the natural history of disease, to determine clinical effectiveness or cost-effectiveness of health care products and services, to measure or monitor safety and harm, and/or to measure quality of care. Registries are classified according to how their populations are defined. For example, product registries include patients who have been exposed to biopharmaceutical products or medical devices. Health services registries consist of patients who have had a common procedure, clinical encounter, or hospitalization. Disease or condition registries are defined by patients having the same diagnosis, such as cystic fibrosis or heart failure. The User’s Guide was created by researchers affiliated with AHRQ’s Effective Health Care Program, particularly those who participated in AHRQ’s DEcIDE (Developing Evidence to Inform Decisions About Effectiveness) program. Chapters were subject to multiple internal and external independent reviews.
Download or read book Handbook of Neuroemergency Clinical Trials written by Brett E. Skolnick and published by Academic Press. This book was released on 2017-11-13 with total page 408 pages. Available in PDF, EPUB and Kindle. Book excerpt: Handbook of Neuroemergency Clinical Trials, Second Edition, focuses on the practice of clinical trials in acute neuroscience populations, or what have been called neuroemergencies. Neuroemergencies are complex, life-threatening diseases and disorders, often with devastating consequences, including death or disability. The overall costs are staggering in terms of annual incidence and costs associated with treatment and survival, yet despite their significance as public health issues, there are few drugs and devices available for definitive treatment. The book focuses on novel therapies and the unique challenges their intended targets pose for the design and analysis of clinical trials. This volume provides neurologists, neuroscientists, and drug developers with a more complete understanding of the scientific and medical issues of relevance in designing and initiating clinical development plans for novel drugs intended for acute neuroscience populations. The editors provide the best understanding of the pitfalls associated with acute CNS drug development and the best information on how to approach and solve issues that have plagued drug development. Presents a comprehensive overview on clinical trials and drug development challenges in acute neuroscience populations Provides neurologists, neuroscientists and drug developers with a complete understanding of scientific and medical issues related to designing clinical trials Edited by leaders in the field who have designed and managed over 50 neuroemergency clinical trials
Download or read book International Public Goods and Transfer of Technology Under a Globalized Intellectual Property Regime written by Keith E. Maskus and published by Cambridge University Press. This book was released on 2005-06-08 with total page 952 pages. Available in PDF, EPUB and Kindle. Book excerpt: Distinguished economists, political scientists, and legal experts discuss the implications of the increasingly globalized protection of intellectual property rights for the ability of countries to provide their citizens with such important public goods as basic research, education, public health, and environmental protection. Such items increasingly depend on the exercise of private rights over technical inputs and information goods, which could usher in a brave new world of accelerating technological innovation. However, higher and more harmonized levels of international intellectual property rights could also throw up high roadblocks in the path of follow-on innovation, competition and the attainment of social objectives. It is at best unclear who represents the public interest in negotiating forums dominated by powerful knowledge cartels. This is the first book to assess the public processes and inputs that an emerging transnational system of innovation will need to promote technical progress, economic growth and welfare for all participants.
Download or read book Rare Diseases Epidemiology written by Manuel Posada de la Paz and published by Springer. This book was released on 2012-11-07 with total page 542 pages. Available in PDF, EPUB and Kindle. Book excerpt: In our etiologic research, we epidemiologists need to leave behind the concepts of ‘cohort’ study and ‘case–control’ study and adopt that of the etiologic study as the singular substitute for these. With this sentence, the famous epidemiologist Professor Olli S. Miettinen began his personal re ection on the future of the epidemiology [1]. He sought to highlight the fact that the role of the epidemiologist should be mainly focused on aetiological research. Nevertheless, the widespread idea still exists that epidemiology is limited to purely providing gures and descriptive data on the frequency and distribution of disease. Indeed, it is more than likely that the precise aim of those rst classic epidemiological steps, i. e. , methods essentially based on describing the distri- tion of a given disease, is still not all that well understood by many scientists, let alone the general public. Such descriptions seek to generate hypotheses and afford explanations for key factors (be these risk factors or the presumable causes th- selves), which might justify differences in terms of persons, time or place and, in turn, ultimately serve to develop preventive measures and/or gain quality-adjusted life years. To restrict the goals of epidemiology to activities exclusively concerned with reporting gures or even complex statistical results is a great mistake, one that renders it dif cult to take full advantage of the epidemiologist’s true role, which is “to study disease determinants and to assess the actual impact of factors involved in their development, distribution and dissemination”.
Download or read book Health Technology Assessment in Japan written by Isao Kamae and published by Springer Nature. This book was released on 2019-09-03 with total page 265 pages. Available in PDF, EPUB and Kindle. Book excerpt: Representing the first book on the topic, this work offers the reader an introduction to the Japanese systems for health technology assessment (HTA) officially introduced by the Ministry of Health, Labour and Welfare (MHLW) in 2016. Policy and guidelines are discussed, with the relevant methods and conditions of cost-effectiveness analysis explained alongside. Numerous instructive examples and exercises, ranging from basic to advanced, impart valuable knowledge and insight on the quantitative methods for economic evaluation, which will appeal to both beginners and experts. This guidebook is authored by Japan’s foremost expert in HTA and pharmacoeconomics, with a view to strengthening the reader’s expertise in value-based healthcare and decision-making. The methods presented are essential to informing regulatory, local and patient decisions; as such, the book is equally recommended to industry and government, as well as academia, and anyone with an interest in Japanese HTA.
