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
160 the U.S. In total, 25 million
160 Americans have rare diseases.
108 The Orphan Drug Act (ODA) was
passed in 1983 to encourage the
80 research and development of
medicines to treat rare diseases.
Since then, the FDA has approved
40 more than 200 medicines for rare
1970's 1984–1994 1995–2005
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.