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Report Of The National Commission On Orphan Diseases
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Book Synopsis Report of the National Commission on Orphan Diseases by : National Commission on Orphan Diseases (U.S.)
Download or read book Report of the National Commission on Orphan Diseases written by National Commission on Orphan Diseases (U.S.) and published by . This book was released on 1989 with total page 128 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Rare Diseases and Orphan Products by : Institute of Medicine
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.
Author :National Organization for Rare Disorders Publisher :Lippincott Williams & Wilkins ISBN 13 :9780781730631 Total Pages :982 pages Book Rating :4.7/5 (36 download)
Book Synopsis NORD Guide to Rare Disorders by : National Organization for Rare Disorders
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.
Book Synopsis Orphan Drugs and Rare Diseases by : David C Pryde
Download or read book Orphan Drugs and Rare Diseases written by David C Pryde and published by Royal Society of Chemistry. This book was released on 2014-07-30 with total page 350 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.
Book Synopsis Report of the National Commission on Orphan Diseases by : National Commission on Orphan Diseases (U.S.)
Download or read book Report of the National Commission on Orphan Diseases written by National Commission on Orphan Diseases (U.S.) and published by . This book was released on 1989 with total page 132 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Author :United States. National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research Publisher : ISBN 13 : Total Pages :614 pages Book Rating :4.:/5 (318 download)
Book Synopsis The Belmont Report by : United States. National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research
Download or read book The Belmont Report written by United States. National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research and published by . This book was released on 1978 with total page 614 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis The Financial Crisis Inquiry Report by : Financial Crisis Inquiry Commission
Download or read book The Financial Crisis Inquiry Report written by Financial Crisis Inquiry Commission and published by Cosimo, Inc.. This book was released on 2011-05-01 with total page 692 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Financial Crisis Inquiry Report, published by the U.S. Government and the Financial Crisis Inquiry Commission in early 2011, is the official government report on the United States financial collapse and the review of major financial institutions that bankrupted and failed, or would have without help from the government. The commission and the report were implemented after Congress passed an act in 2009 to review and prevent fraudulent activity. The report details, among other things, the periods before, during, and after the crisis, what led up to it, and analyses of subprime mortgage lending, credit expansion and banking policies, the collapse of companies like Fannie Mae and Freddie Mac, and the federal bailouts of Lehman and AIG. It also discusses the aftermath of the fallout and our current state. This report should be of interest to anyone concerned about the financial situation in the U.S. and around the world.THE FINANCIAL CRISIS INQUIRY COMMISSION is an independent, bi-partisan, government-appointed panel of 10 people that was created to "examine the causes, domestic and global, of the current financial and economic crisis in the United States." It was established as part of the Fraud Enforcement and Recovery Act of 2009. The commission consisted of private citizens with expertise in economics and finance, banking, housing, market regulation, and consumer protection. They examined and reported on "the collapse of major financial institutions that failed or would have failed if not for exceptional assistance from the government."News Dissector DANNY SCHECHTER is a journalist, blogger and filmmaker. He has been reporting on economic crises since the 1980's when he was with ABC News. His film In Debt We Trust warned of the economic meltdown in 2006. He has since written three books on the subject including Plunder: Investigating Our Economic Calamity (Cosimo Books, 2008), and The Crime Of Our Time: Why Wall Street Is Not Too Big to Jail (Disinfo Books, 2011), a companion to his latest film Plunder The Crime Of Our Time. He can be reached online at www.newsdissector.com.
Book Synopsis Rare Diseases Epidemiology: Update and Overview by : Manuel Posada de la Paz
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 667 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.
Book Synopsis Investing to Overcome the Global Impact of Neglected Tropical Diseases by : World Health Organization
Download or read book Investing to Overcome the Global Impact of Neglected Tropical Diseases written by World Health Organization and published by World Health Organization. This book was released on 2015-08-05 with total page 211 pages. Available in PDF, EPUB and Kindle. Book excerpt: "The presence, or absence, of neglected tropical diseases (NTDs) can be seen as a proxy for poverty and for the success of interventions aimed at reducing poverty. Today, coverage of the public-health interventions recommended by the World Health Organization (WHO) against NTDs may be interpreted as a proxy for universal health coverage and shared prosperity - in short, a proxy for coverage against neglect. As the world's focus shifts from development to sustainable development, from poverty eradication to shared prosperity, and from disease-specific goals to universal health coverage, control of NTDs will assume an important role towards the target of achieving universal health coverage, including individual financial risk protection. Success in overcoming NTDs is a "litmus test" for universal health coverage against NTDs in endemic countries. The first WHO report on NTDs (2010) set the scene by presenting the evidence for how these interventions had produced results. The second report (2013) assessed the progress made in deploying them and detailed the obstacles to their implementation. This third report analyses for the first time the investments needed to achieve the scale up of implementation required to achieve the targets of the WHO Roadmap on NTDs and universal coverage against NTDs. INVESTING TO OVERCOME THE GLOBAL IMPACT OF NEGLECTED TROPICAL DISEASES presents an investment strategy for NTDs and analyses the specific investment case for prevention, control, elimination and eradication of 12 of the 17 NTDs. Such an analysis is justified following the adoption by the Sixty-sixth World Health Assembly in 2013 of resolution WHA6612 on neglected tropical diseases, which called for sufficient and predictable funding to achieve the Roadmap's targets and sustain control efforts. The report cautions, however, that it is wise investment and not investment alone that will yield success. The report registers progress and challenges and signals those that lie ahead. Climate change is expected to increase the spread of several vector-borne NTDs, notably dengue, transmission of which is directly influenced by temperature, rainfall, relative humidity and climate variability primarily through their effects on the vector. Investments in vector-borne diseases will avoid the potentially catastrophic expenditures associated with their control. The presence of NTDs will thereby signal an early warning system for climate-sensitive diseases. The ultimate goal is to deliver enhanced and equitable interventions to the most marginalized populations in the context of a changing public-health and investment landscape to ensure that all peoples affected by NTDs have an opportunity to lead healthier and wealthier lives."--Publisher's description.
Book Synopsis Reducing Environmental Cancer Risk by : Suzanne H. Reuben
Download or read book Reducing Environmental Cancer Risk written by Suzanne H. Reuben and published by DIANE Publishing. This book was released on 2010-10 with total page 240 pages. Available in PDF, EPUB and Kindle. Book excerpt: Though overall cancer incidence and mortality have continued to decline in recent years, cancer continues to devastate the lives of far too many Americans. In 2009 alone, 1.5 million American men, women, and children were diagnosed with cancer, and 562,000 died from the disease. There is a growing body of evidence linking environmental exposures to cancer. The Pres. Cancer Panel dedicated its 2008¿2009 activities to examining the impact of environmental factors on cancer risk. The Panel considered industrial, occupational, and agricultural exposures as well as exposures related to medical practice, military activities, modern lifestyles, and natural sources. This report presents the Panel¿s recommend. to mitigate or eliminate these barriers. Illus.
Book Synopsis Rare Disease Drug Development by : Raymond A. Huml
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.
Book Synopsis A Nationwide Framework for Surveillance of Cardiovascular and Chronic Lung Diseases by : Institute of Medicine
Download or read book A Nationwide Framework for Surveillance of Cardiovascular and Chronic Lung Diseases written by Institute of Medicine and published by National Academies Press. This book was released on 2011-08-26 with total page 200 pages. Available in PDF, EPUB and Kindle. Book excerpt: Chronic diseases are common and costly, yet they are also among the most preventable health problems. Comprehensive and accurate disease surveillance systems are needed to implement successful efforts which will reduce the burden of chronic diseases on the U.S. population. A number of sources of surveillance data--including population surveys, cohort studies, disease registries, administrative health data, and vital statistics--contribute critical information about chronic disease. But no central surveillance system provides the information needed to analyze how chronic disease impacts the U.S. population, to identify public health priorities, or to track the progress of preventive efforts. A Nationwide Framework for Surveillance of Cardiovascular and Chronic Lung Diseases outlines a conceptual framework for building a national chronic disease surveillance system focused primarily on cardiovascular and chronic lung diseases. This system should be capable of providing data on disparities in incidence and prevalence of the diseases by race, ethnicity, socioeconomic status, and geographic region, along with data on disease risk factors, clinical care delivery, and functional health outcomes. This coordinated surveillance system is needed to integrate and expand existing information across the multiple levels of decision making in order to generate actionable, timely knowledge for a range of stakeholders at the local, state or regional, and national levels. The recommendations presented in A Nationwide Framework for Surveillance of Cardiovascular and Chronic Lung Diseases focus on data collection, resource allocation, monitoring activities, and implementation. The report also recommends that systems evolve along with new knowledge about emerging risk factors, advancing technologies, and new understanding of the basis for disease. This report will inform decision-making among federal health agencies, especially the Department of Health and Human Services; public health and clinical practitioners; non-governmental organizations; and policy makers, among others.
Book Synopsis Report of the National Advisory Commission on Civil Disorders by : United States. National Advisory Commission on Civil Disorders
Download or read book Report of the National Advisory Commission on Civil Disorders written by United States. National Advisory Commission on Civil Disorders and published by . This book was released on 1968 with total page 450 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Author :WHO Commission on Social Determinants of Health Publisher :World Health Organization ISBN 13 :9241563702 Total Pages :257 pages Book Rating :4.2/5 (415 download)
Book Synopsis Closing the Gap in a Generation by : WHO Commission on Social Determinants of Health
Download or read book Closing the Gap in a Generation written by WHO Commission on Social Determinants of Health and published by World Health Organization. This book was released on 2008 with total page 257 pages. Available in PDF, EPUB and Kindle. Book excerpt: Social justice is a matter of life and death. It affects the way people live, their consequent chance of illness, and their risk of premature death. We watch in wonder as life expectancy and good health continue to increase in parts of the world and in alarm as they fail to improve in others.
Author :National Academies of Sciences, Engineering, and Medicine Publisher :National Academies Press ISBN 13 :0309377722 Total Pages :473 pages Book Rating :4.3/5 (93 download)
Book Synopsis Improving Diagnosis in Health Care by : National Academies of Sciences, Engineering, and Medicine
Download or read book Improving Diagnosis in Health Care written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2015-12-29 with total page 473 pages. Available in PDF, EPUB and Kindle. Book excerpt: Getting the right diagnosis is a key aspect of health care - it provides an explanation of a patient's health problem and informs subsequent health care decisions. The diagnostic process is a complex, collaborative activity that involves clinical reasoning and information gathering to determine a patient's health problem. According to Improving Diagnosis in Health Care, diagnostic errors-inaccurate or delayed diagnoses-persist throughout all settings of care and continue to harm an unacceptable number of patients. It is likely that most people will experience at least one diagnostic error in their lifetime, sometimes with devastating consequences. Diagnostic errors may cause harm to patients by preventing or delaying appropriate treatment, providing unnecessary or harmful treatment, or resulting in psychological or financial repercussions. The committee concluded that improving the diagnostic process is not only possible, but also represents a moral, professional, and public health imperative. Improving Diagnosis in Health Care, a continuation of the landmark Institute of Medicine reports To Err Is Human (2000) and Crossing the Quality Chasm (2001), finds that diagnosis-and, in particular, the occurrence of diagnostic errorsâ€"has been largely unappreciated in efforts to improve the quality and safety of health care. Without a dedicated focus on improving diagnosis, diagnostic errors will likely worsen as the delivery of health care and the diagnostic process continue to increase in complexity. Just as the diagnostic process is a collaborative activity, improving diagnosis will require collaboration and a widespread commitment to change among health care professionals, health care organizations, patients and their families, researchers, and policy makers. The recommendations of Improving Diagnosis in Health Care contribute to the growing momentum for change in this crucial area of health care quality and safety.
Book Synopsis Orphan Drugs by : Elizabeth Hernberg-Ståhl
Download or read book Orphan Drugs written by Elizabeth Hernberg-Ståhl and published by Elsevier. This book was released on 2013-11-15 with total page 336 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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 Synopsis Drug Repurposing by : Farid A. Badria
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.
