Author: National Commission on Orphan Diseases (U.S.)
Publisher:
ISBN:
Category : Federal aid to medical research
Languages : en
Pages : 128

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Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309158060
Category : Medical
Languages : en
Pages : 442

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Book Description
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 Commission on Orphan Diseases (U.S.)
Publisher:
ISBN:
Category : Federal aid to medical research
Languages : en
Pages : 132

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Author: Manuel Posada de la Paz
Publisher: Springer
ISBN: 3319671448
Category : Medical
Languages : en
Pages : 675

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Book Description
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.

Author: David Pryde
Publisher: Royal Society of Chemistry
ISBN: 1782624201
Category : Science
Languages : en
Pages : 487

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Book Description
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.

Author: United States. National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research
Publisher:
ISBN:
Category : Ethics, Medical
Languages : en
Pages : 614

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Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309468086
Category : Medical
Languages : en
Pages : 235

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Book Description
Thanks to remarkable advances in modern health care attributable to science, engineering, and medicine, it is now possible to cure or manage illnesses that were long deemed untreatable. At the same time, however, the United States is facing the vexing challenge of a seemingly uncontrolled rise in the cost of health care. Total medical expenditures are rapidly approaching 20 percent of the gross domestic product and are crowding out other priorities of national importance. The use of increasingly expensive prescription drugs is a significant part of this problem, making the cost of biopharmaceuticals a serious national concern with broad political implications. Especially with the highly visible and very large price increases for prescription drugs that have occurred in recent years, finding a way to make prescription medicinesâ€"and health care at largeâ€"more affordable for everyone has become a socioeconomic imperative. Affordability is a complex function of factors, including not just the prices of the drugs themselves, but also the details of an individual's insurance coverage and the number of medical conditions that an individual or family confronts. Therefore, any solution to the affordability issue will require considering all of these factors together. The current high and increasing costs of prescription drugsâ€"coupled with the broader trends in overall health care costsâ€"is unsustainable to society as a whole. Making Medicines Affordable examines patient access to affordable and effective therapies, with emphasis on drug pricing, inflation in the cost of drugs, and insurance design. This report explores structural and policy factors influencing drug pricing, drug access programs, the emerging role of comparative effectiveness assessments in payment policies, changing finances of medical practice with regard to drug costs and reimbursement, and measures to prevent drug shortages and foster continued innovation in drug development. It makes recommendations for policy actions that could address drug price trends, improve patient access to affordable and effective treatments, and encourage innovations that address significant needs in health care.

Author: National Research Council
Publisher: National Academies Press
ISBN: 0309142393
Category : Law
Languages : en
Pages : 348

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Book Description
Scores of talented and dedicated people serve the forensic science community, performing vitally important work. However, they are often constrained by lack of adequate resources, sound policies, and national support. It is clear that change and advancements, both systematic and scientific, are needed in a number of forensic science disciplines to ensure the reliability of work, establish enforceable standards, and promote best practices with consistent application. Strengthening Forensic Science in the United States: A Path Forward provides a detailed plan for addressing these needs and suggests the creation of a new government entity, the National Institute of Forensic Science, to establish and enforce standards within the forensic science community. The benefits of improving and regulating the forensic science disciplines are clear: assisting law enforcement officials, enhancing homeland security, and reducing the risk of wrongful conviction and exoneration. Strengthening Forensic Science in the United States gives a full account of what is needed to advance the forensic science disciplines, including upgrading of systems and organizational structures, better training, widespread adoption of uniform and enforceable best practices, and mandatory certification and accreditation programs. While this book provides an essential call-to-action for congress and policy makers, it also serves as a vital tool for law enforcement agencies, criminal prosecutors and attorneys, and forensic science educators.

Author: Tarun Weeramanthri
Publisher: Frontiers Media SA
ISBN: 2889455017
Category :
Languages : en
Pages : 149

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Book Description
Precision Public Health is a new and rapidly evolving field, that examines the application of new technologies to public health policy and practice. It draws on a broad range of disciplines including genomics, spatial data, data linkage, epidemiology, health informatics, big data, predictive analytics and communications. The hope is that these new technologies will strengthen preventive health, improve access to health care, and reach disadvantaged populations in all areas of the world. But what are the downsides and what are the risks, and how can we ensure the benefits flow to those population groups most in need, rather than simply to those individuals who can afford to pay? This is the first collection of theoretical frameworks, analyses of empirical data, and case studies to be assembled on this topic, published to stimulate debate and promote collaborative work.

Author: Edward Livingston Trudeau
Publisher:
ISBN: 9781519051073
Category :
Languages : en
Pages : 201

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Book Description
Coming from a long line of doctors, it seemed natural that Edward Livingston Trudeau would follow in that path. Never did he dream that only shortly after returning from his honeymoon in 1871, he would be diagnosed with tuberculosis.Despite being devastated by the news, he spent the rest of his life in the research and treatment of tuberculosis, establishing the Adirondack Cottage Sanitarium at Saranac Lake. His laboratory there was the first in the United States dedicated to a better understanding of the disease.In this intensely interesting, inspiring, and often witty memoir, Dr. Trudeau relates the trials and victories of his professional and personal life. His long marriage was one of the bedrocks of his life.Among his most celebrated patients was author Robert Louis Stevenson, with whom he became friends. He also mentions an evening with Stevenson and Libby Custer (widow of General George Armstrong Custer) that was highly entertaining.As for living a life with illness, Trudeau says:"I have had ample opportunity in the past forty years to get used to illness and suffering; but it took me a long time to learn, imperfectly though it be, that acquiescence is the only way for the tuberculous invalid to conquer fate. To cease to rebel and struggle, and to learn to be content with part of a loaf when one cannot have a whole loaf, though a hard lesson to learn, is good philosophy for the tuberculous invalid, and to his astonishment he often finds that what he considers the half-loaf, when acquiesced in, proves most satisfying."