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Plant molecular farming: a promising frontier for orphan drug production
Biotechnology LettersOrphan diseases comprise a range of disorders that individually affect a small percentage of people, but collectively impact millions of people worldwide. Patients with this disorder often face significant challenges in diagnosis, treatment, and access to care due to their rare nature and limited understanding and treatment options.
Balamurugan Shanmugaraj +2 more
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2017
Over 30 years ago, the United States (US) Congress passed the Orphan Drug Act (ODA) to encourage the development of products for rare diseases or conditions ("orphan products"). The Act provided incentives to sponsors for developing products with orphan designation and established a grant program to fund studies of orphan products.
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Over 30 years ago, the United States (US) Congress passed the Orphan Drug Act (ODA) to encourage the development of products for rare diseases or conditions ("orphan products"). The Act provided incentives to sponsors for developing products with orphan designation and established a grant program to fund studies of orphan products.
openaire +2 more sources
Labeling Pediatric Products: BPCA Efforts and Orphan Drug Opportunities
The Journal of Pediatric Pharmacology and TherapeuticsOBJECTIVE Although the Orphan Drug Act (ODA) has substantially increased the number of therapies available for rare diseases, there are still unmet needs for drug development in pediatric patients. The current study represents an exploratory collaborative effort from the National Institutes of Health (NIH) and the US Food and Drug Administration (FDA)
Hui Wei +3 more
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Survey Indicates 133 Orphan Drug Products in Development for 96 Relatively Rare Disorders
JAMA: The Journal of the American Medical Association, 1989ABSTRACT EIGHTY-FIVE companies have 133 drugs in various stages of development for 96 rare diseases.This is the first comprehensive listing of orphan drugs since the Orphan Drug Act became law in 1983. The list was compiled by the Pharmaceutical Manufacturers Association in response to a request by the National Commission on Orphan Diseases (JAMA. 1989;
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Clinical Pharmacology & Therapeutics, 2010
Many neurological diseases or conditions are rare disorders. The Orphan Drug Act (ODA) of 1983 was promulgated to promote the development of products for such conditions. In this Opinion piece, we discuss how the ODA has affected neurological diseases, note how current and future sponsors (any person(s) or entity (i.e., academic, corporate body ...
K A, Burke +3 more
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Many neurological diseases or conditions are rare disorders. The Orphan Drug Act (ODA) of 1983 was promulgated to promote the development of products for such conditions. In this Opinion piece, we discuss how the ODA has affected neurological diseases, note how current and future sponsors (any person(s) or entity (i.e., academic, corporate body ...
K A, Burke +3 more
openaire +2 more sources
The Orphan Drug Act and the Federal Government's Orphan Products Development Program.
Public health reports (Washington, D.C. : 1974), 1984Through the combined efforts of agencies and organizations in the public and private sector, drugs have been made available that would not have been at hand without a specific focus on the orphan drug issue. It is anticipated that these cooperative efforts will continue beyond the first enthusiastic burst engendered by the inception of new and ...
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Orphan products: drugs and devices for rare diseases.
Nursing economic$, 1993The issues of orphan products include an inadequate definition of orphan status that hampers implementation of the legislation, difficulties in evaluating the products themselves in order to ensure safety and effectiveness, neglect in developing orphan devices, and conflict over creating unnecessary monopolies and excessive profits.
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Antibody–drug conjugates: Smart chemotherapy delivery across tumor histologies
Ca-A Cancer Journal for Clinicians, 2022Paolo Tarantino +2 more
exaly
The Orphan Drug Products—Another Solution?
Drug Intelligence & Clinical Pharmacy, 1983openaire +1 more source