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Reforming the Orphan Drug Act for the 21st Century.
New England Journal of Medicine, 2019Reforming the Orphan Drug Act for the 21st Century The pharmaceutical market has undergone radical changes, including markedly increased prices for rare-disease drugs.
A. Sarpatwari, A. Kesselheim
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2018
Rare diseases represent a group of conditions affecting a very limited number of patients. Low profitability resulting from the small size of target population coupled with difficulties in conducting the research causes the lack of interest from the pharmaceutical industry.
Mondher Toumi +3 more
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Rare diseases represent a group of conditions affecting a very limited number of patients. Low profitability resulting from the small size of target population coupled with difficulties in conducting the research causes the lack of interest from the pharmaceutical industry.
Mondher Toumi +3 more
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Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU
European Journal of Clinical Pharmacology, 2012To assess the methodological quality of Orphan Medicinal Product (OMP) dossiers and discuss possible reasons for the small number of products licensed.Information about orphan drug designation, approval, refusal or withdrawal was obtained from the website of the European Medicines Agency and from the European Public Assessment Reports.From 2000 up to ...
Roberta Joppi +2 more
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The Orphan Drug Act at 35: Observations and an Outlook for the Twenty-First Century
Innovation Policy and the Economy, 2019On the thirty-fifth anniversary of the adoption of the Orphan Drug Act (ODA), we describe the enormous changes in the markets for therapies for rare diseases that have emerged over recent decades.
Nicholas Bagley +4 more
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Evaluating The Impact Of The Orphan Drug Act's Seven-Year Market Exclusivity Period.
Health Affairs, 2018For thirty-five years the Orphan Drug Act of 1983 has provided incentives for pharmaceutical manufacturers to develop drugs to treat rare diseases-conditions that affect fewer than 200,000 people in the US.
A. Sarpatwari +4 more
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Does an Orphan Drug Policy Make a Difference in Access? A Comparison of Canada and Australia
International Journal of Health Services, 2019Canada has been discussing whether to implement an orphan drug policy for more than 25 years. Recently, the federal government announced funding for orphan drugs starting in 2022, and the Canadian Senate has recommended that the country develop an orphan
J. Lexchin, N. Moroz
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Radiopharmaceuticals as orphan drugs
Seminars in Nuclear Medicine, 1996Rare diseases and conditions are defined as diseases or conditions that affect less than 200,000 individuals in the United States. Because of the limited population suffering from any given rare disorder, there is little economic motivation for the development of drug products for its diagnosis or treatment.
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Orphan drug policies in different countries
Journal of Pharmaceutical Health Services Research, 2019Due to the low prevalence of rare diseases (RDs) and the small number of individuals affected with a RD, challenges complicate the development of and accessibility to medications and medical devices to prevent, identify, treat or cure these RDs. The aims
Amar Abbas +3 more
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Pharmaceuticals, Policy and Law, 2010
Rare diseases are frequently genetically determined and caused by an inborn metabolic error, thus occurring early in life and affecting normal growth, and sexual and CNS maturation. The development of a paediatric orphan medicine presents a real hurdle for pharmaceutical companies because of the nature of the patient population where little knowledge ...
Laura Folgori, Mariana Catapano
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Rare diseases are frequently genetically determined and caused by an inborn metabolic error, thus occurring early in life and affecting normal growth, and sexual and CNS maturation. The development of a paediatric orphan medicine presents a real hurdle for pharmaceutical companies because of the nature of the patient population where little knowledge ...
Laura Folgori, Mariana Catapano
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Orphan drugs—or orphan diseases?
1988In the beginning, the question was that of how to provide drugs for rare diseases; and the beginning in this country was perhaps around 1972 when the ‘Pharmaceutical Working Party’ of the National Economic Development Office discussed the matter and agreed to keep it under review [1].
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