Rare Diseases 2008 2016
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Rare Diseases 2008 2016
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Author :
language : en
Publisher:
Release Date : 2018
Rare Diseases 2008 2016 written by and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018 with categories.
Rare diseases are a threat to the health of EU citizens insofar as they are life-threatening, highly complex diseases, affecting millions of European citizens. Healthcare systems are not designed to address the problems raised by the scant knowledge, the rarity of the experts and the multidisciplinary dimension of these diseases. Furthermore, research is hampered by the lack of data. The patients' voice is difficult to hear as there are thousands of different diseases. Despite all these challenges, year after year, the European Union is building solutions to address the problems in an efficient way.
Nord Guide To Rare Disorders
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Author : National Organization for Rare Disorders
language : en
Publisher: Lippincott Williams & Wilkins
Release Date : 2003
Nord Guide To Rare Disorders written by National Organization for Rare Disorders and has been published by Lippincott Williams & Wilkins this book supported file pdf, txt, epub, kindle and other format this book has been release on 2003 with Medical categories.
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.
Experiences Of Rare Diseases
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Author : Lauren Limb
language : en
Publisher:
Release Date : 2010
Experiences Of Rare Diseases written by Lauren Limb and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2010 with Great Britain categories.
Rare Diseases
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Author :
language : en
Publisher:
Release Date : 2014
Rare Diseases written by and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014 with categories.
Rare diseases affect millions of Europeans but can be difficult to diagnose, treat and study because the patient population and disease experts are dispersed across Europe. Through the second EU health programme 2008-2013, Europe is supporting projects that share knowledge and expertise on rare diseases.
Rare Diseases And Orphan Products
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Author : Institute of Medicine
language : en
Publisher: National Academies Press
Release Date : 2011-04-03
Rare Diseases And Orphan Products written by Institute of Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2011-04-03 with Medical categories.
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.
Rare Diseases Epidemiology Update And Overview
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Author : Manuel Posada de la Paz
language : en
Publisher: Springer
Release Date : 2017-12-06
Rare Diseases Epidemiology Update And Overview written by Manuel Posada de la Paz and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2017-12-06 with Medical categories.
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.
Rare Disease Drug Development
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Author : Raymond A. Huml
language : en
Publisher: Springer Nature
Release Date : 2021-11-08
Rare Disease Drug Development written by Raymond A. Huml and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-11-08 with Medical categories.
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.
Orphan Drugs And Rare Diseases
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Author : David Pryde
language : en
Publisher: Royal Society of Chemistry
Release Date : 2014-07-30
Orphan Drugs And Rare Diseases written by David Pryde and has been published by Royal Society of Chemistry this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014-07-30 with Science categories.
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.
Rare Diseases Epidemiology
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Author : Manuel Posada de la Paz
language : en
Publisher: Springer
Release Date : 2012-11-07
Rare Diseases Epidemiology written by Manuel Posada de la Paz and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2012-11-07 with Medical categories.
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”.
Registries For Evaluating Patient Outcomes
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Author : Agency for Healthcare Research and Quality/AHRQ
language : en
Publisher: Government Printing Office
Release Date : 2014-04-01
Registries For Evaluating Patient Outcomes written by Agency for Healthcare Research and Quality/AHRQ and has been published by Government Printing Office this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014-04-01 with Medical categories.
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.