Medicine Approvals for Rare Diseases

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Medicine Approvals for Rare Diseases
Drug Approvals for Rare Diseases Have Increased
from Fewer than 10 to More than 160
Rare diseases are those that affect 200,000 or
fewer people in the U.S. In total, 25 million
Americans have rare diseases.
The Orphan Drug Act (ODA) was passed in 1983 to
encourage the research and development of
medicines to treat rare diseases. Since then, the
FDA has approved more than 200 medicines for rare
diseases.
Drug Approvals
Note All medicines approved for rare diseases
include first-line approvals and subsequent
approvals for new disease areas Sources FDA,
Office of Orphan Product Development, List of All
Approved Orphan Products Through the Year 2005
and Orphan Products Development, at
http//www.fda.gov/orphan NIH, Office of Rare
Diseases, Biennial and Annual Report on the Rare
Diseases Research Activities at the National
Institutes of Health, FY 2004, 2005.