Rare Diseases
• An “orphan” or rare disease affects fewer than 200,000 people in the United States.[i]
• There are approximately 6,000–7,000 rare diseases.[ii]
• Between 85 and 90 percent of orphan diseases are serious or life-threatening.[iii]
• Rare diseases affect 25 million Americans. One in every 10 individuals in this country has received a diagnosis of a rare disease.[iv]
• The Orphan Drug Act was enacted in 1983 and encourages companies to develop and manufacture drugs for rare conditions.
• In the decade after the Orphan Drug Act was passed, there were about 10 times as many drugs approved for rare diseases compared with the decade before.[v]
• More than 350 medicines have been approved for rare diseases since 1983. Over 160 orphan drugs were approved from 1995 to 200, compared with 108 from 1984 to 1994 and only 10 in the decade prior to the passage of the Orphan Drug Act in 1983.[vi]
• Since the Orphan Drug Act, over 2,300 drugs in development have been designated as orphan products.[vii]
• The number of medicines in development designated as orphan products more than doubled from 208 in 2000 to 2002 to 425 in 2006 to 2008.[viii]
• There are currently 460 medicines in development for rare diseases in late stage clinical trials or awaiting FDA approval.[ix]
• In the pipeline are potential treatments for anthrax, cystic fibrosis, and West Nile Virus, which have been granted orphan status by the FDA.[x]
• The number of orphan drugs is expected to rise in the coming years as more new medicines are developed that target specific genetic disorders.[xi]
[i]National Organization for Rare Disorders, http://www.rarediseases.org/info/about.html (accessed 1 March 2011).
[ii]National Institutes of Health. "Rare Diseases Clinical Research Network Fact Sheet" http://report.nih.gov/NIHfactsheets/ViewFactSheet.aspx?csid=126&key=R#R (Accessed 1 March 2011).
[iii]Food and Drug Administration, "Office of Orphan Products Development," Budget 2010, http://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/
BudgetReports/UCM153550.pdf (accessed 1 March 2011).
[iv]National Organization for Rare Disorders, http://www.rarediseases.org/info/about.html.
[v]Food and Drug Administration, Office of Orphan Products Development, http://www.fda.gov/orphan.
[vi]Food and Drug Administration, Orphan Drug Designations and Approvals Database, http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm , (accessed 2 February 2010); Food and Drug Administration, Office of Orphan Products Development, “List of All Approved Orphan Products Through the Year 2005,” 13 May 2005, http://www.fda.gov/orphan/designat/allap.rtf (accessed 7 September 2005).
[vii]Food and Drug Administration, Orphan Drug Designations and Approvals Database, http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm , (accessed 23 June 2010).
[viii]Tufts Center for the Study of Drug Development, “U.S. Orphan Product Designations More Than Doubled From 2000–02 to 2006–08,” Tufts CSDD Impact Report 12, no. 1 (2010).
[ix]Pharmaceutical Research and Manufacturers of America, “Orphan Drugs in Development for Rare Diseases,” 24 February 2011, http://www.phrma.org/sites/default/files/878/rarediseases2011.pdf (accessed 1 March 2011).
[x]Adis R&D Insight, 23 June 2010.
[xi]Food and Drug Administration, “Office of Orphan Products Development,” op. cit.
