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Institute of Medicine,Board on Health Sciences Policy,Committee on Accelerating Rare Diseases Research and Orphan Product Development
Author : Institute of Medicine,Board on Health Sciences Policy,Committee on Accelerating Rare Diseases Research and Orphan Product Development
Publisher : National Academies Press
Release : 2011-04-03
Category : Medical
ISBN : 9780309158060
Book Rare Diseases and Orphan Products Description/Summary:
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.
Book Rare Diseases and Orphan Drugs Description/Summary:
Rare Diseases and Orphan Drugs shows that much of what we now know about common diseases has been achieved by studying rare diseases. It proposes that future advances in the prevention, diagnosis, and treatment of common diseases will come as a consequence of our accelerating progress in the field of rare diseases. Understanding the complex steps in the development of common diseases, such as cancer, cardiovascular disease, and metabolic diseases, has proven a difficult problem. Rare diseases, however, are often caused by aberrations of a single gene. In rare diseases, we may study how specific genetic defects can trigger a series of events that lead to the expression of a particular disease. Often, the disease process manifested in a certain rare disease is strikingly similar to the disease process observed in a common disease. This work ties the lessons learned about rare diseases to our understanding of common ones. Chapters covering the number of common diseases are minimized, while rare diseases are introduced as single diseases or as members of diseases classes. After reading this book, readers will appreciate how further research into the rare diseases may lead to new methods for preventing, diagnosing, and treating all diseases, rare or common. Makes rare diseases relevant to clinicians and researchers by tying lessons learned about the rare diseases to our understanding of the common diseases Stresses basic pathologic mechanisms that account for human disease (e.g., disorders of cell development, replication, maintenance, function and structure), that can be understood without prior training in pathology Discusses advanced concepts in molecular biology and genetics in a simple, functional context appropriate for medical trainees and new researchers Offers insights into how further research into rare diseases may lead to new methods for preventing, diagnosing, and treating all diseases.
This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups. The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey. After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs. The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs. A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patients A source of updated information, news and trends for those who are already active in this fast-evolving field Covers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe
Book Rare Diseases Epidemiology: Update and Overview Description/Summary:
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.
Orphan is about the struggle to save the lives of children who, because of an unlucky roll of the genetic dice, are born with any one of several thousand rare genetic disorders. Many are burdened with diseases that carry mysterious names, some of which you will read about for the first time in this book, along with compelling stories about the physicians, scientists, and parents who have taken them on. Orphan is more than a book about disease and research-it gives voice to thousands of people who, all too often, have endured terrible illnesses, bravely faced arduous clinical trials, and (sometimes) known victories, almost always in silence. It recounts extraordinary breakthroughs and hopes for the future. Many of the disorders that will end our lives are in some part genetically influenced. We really are all orphans, and this book is for all of us.
Book Registries for Evaluating Patient Outcomes Description/Summary:
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.
Book Communicating Rare Diseases and Disorders in the Digital Age Description/Summary:
A primary concern of rare disease diagnosis is the lack of accurate information that may lead to delayed interventions, administering inaccurate treatments, and social consequences. Health communication continues to be one-way and rely on the expertise from the health practitioner. In such a broad spectrum of rare diseases, patients may find it difficult to obtain timely information, accurate diagnosis, and appropriate treatments, surgeries, medications, or psychological counseling in their own countries. The use of information and communication technologies can create new communication channels that address this lack of knowledge. Communicating Rare Diseases and Disorders in the Digital Age is an essential reference source that uses computer-mediated communication to improve patient knowledge when afflicted or dealing with rare health conditions. Featuring research on topics such as support networking, eHealth management, and social computing, this book is ideally designed for health practitioners, physicians, patients, medical administrators, nurses, surgeons, infectious disease educators, hospital directors, world health organizations, academicians, students, and researchers seeking coverage on current advances in health communication, computer science, and epidemiology.
National Research Council,Institute of Medicine,Board on Health Sciences Policy,Division on Earth and Life Studies,Nuclear and Radiation Studies Board,Committee on State of the Science of Nuclear Medicine
Author : National Research Council,Institute of Medicine,Board on Health Sciences Policy,Division on Earth and Life Studies,Nuclear and Radiation Studies Board,Committee on State of the Science of Nuclear Medicine
Publisher : National Academies Press
Release : 2007-09-11
Category : Medical
ISBN : 0309134153
Book Advancing Nuclear Medicine Through Innovation Description/Summary:
Nearly 20 million nuclear medicine procedures are carried out each year in the United States alone to diagnose and treat cancers, cardiovascular disease, and certain neurological disorders. Many of the advancements in nuclear medicine have been the result of research investments made during the past 50 years where these procedures are now a routine part of clinical care. Although nuclear medicine plays an important role in biomedical research and disease management, its promise is only beginning to be realized. Advancing Nuclear Medicine Through Innovation highlights the exciting emerging opportunities in nuclear medicine, which include assessing the efficacy of new drugs in development, individualizing treatment to the patient, and understanding the biology of human diseases. Health care and pharmaceutical professionals will be most interested in this book's examination of the challenges the field faces and its recommendations for ways to reduce these impediments.
Book NORD Guide to Rare Disorders Description/Summary:
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.
Orphan lung diseases differ from the more common pulmonary disorders, due to the fact that the respiratory physician will only see a few of them each year or even during their career. However, as a specialist, it is necessary to identify and confirm such a diagnosis in a patient. This Monograph comprehensively covers the most common and/or complex of these orphan lung diseases. This Monograph should be seen as a solid companion for the respiratory specialist each time they need to consider a diagnosis of one of these orphan diseases.
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.
Book Office of Orphan Products Development (OOPD). Description/Summary:
Presents the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) in Rockville, Maryland, which promotes the development of products for the diagnosis and treatment of rare diseases. Posts contact information via mailing address, telephone and fax numbers, and e-mail. Contains information on orphan drug designation and incentives of the Orphan Drug Act, as well as a FAQ section. Includes a staff directory. Links to the FDA home page and other sources of rare disease information.
RARE CANCER AGENDA 2030 Ten Recommendations from the EU Joint Action on Rare Cancers 1. Rare cancers are the rare diseases of oncology 2. Rare cancers should be monitored 3. Health systems should exploit networking 4. Medical education should exploit and serve healthcare networking 5. Research should be fostered by networking and should take into account an expected higher degree of uncertainty 6. Patient-physician shared clinical decision-making should be especially valued 7. Appropriate state-of-the-art instruments should be developed in rare cancer 8. Regulation on rare cancers should tolerate a higher degree of uncertainty 9. Policy strategies on rare cancers and sustainability of interventions should be based on networking 10. Rare cancer patients should be engaged