The Current Environment in Orphan Drug Development

Pharmaceutical development over the past two decades has shown a substantial increase in the number of drugs available to treat rare (“orphan”) diseases, largely due to the passage of the Orphan Drug Act. This article provides an overview of orphan diseases and conditions; the Orphan Drug Act; the F...

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Bibliographic Details
Published in:Drug information journal 2003-01, Vol.37 (4), p.373-379
Main Author: Haffner, Marlene E.
Format: Article
Language:English
Subjects:
Clinical trials
Drug development
Drug overdose
Drugs
Interferon
Medical treatment
Orphan drugs
Pharmaceuticals
Pharmaceutics
Product development
Rare diseases
Regulation
US Food and Drug Administration
USA
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Summary:Pharmaceutical development over the past two decades has shown a substantial increase in the number of drugs available to treat rare (“orphan”) diseases, largely due to the passage of the Orphan Drug Act. This article provides an overview of orphan diseases and conditions; the Orphan Drug Act; the Food and Drug Administration's Office of Orphan Products Development, which administers the Orphan Drug Act; the changing nature of rare disease treatment; and designing clinical trials to investigate rare diseases. The success of the Orphan Drug Act is far greater than anticipated; to date 242 orphan drugs have been approved. The Orphan Drug Act continues to encourage manufacturers to develop treatments to help more than 11 million patients in the United States who suffer from rare diseases.
ISSN:2168-4790
0092-8615
2168-4804
2164-9200
DOI:10.1177/009286150303700404