Rare Diseases

An “orphan” or rare disease affects fewer than 200,000 people in the United States.¹

There are approximately 6,000–7,000 rare diseases.²

Between 85 and 90 percent of orphan diseases are serious or life-threatening.³

One in every 10 individuals in this country has received a diagnosis of a rare disease.⁴

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.⁵

In the last decade (1995–2005), over 160 orphan drugs were approved, compared with 108 from 1984 to 1994 and only 10 in the decade prior to the passage of the Orphan Drug Act in 1983.⁶

Since the Orphan Drug Act, over 1,450 drugs in development have been designated as orphan products.⁷

In the pipeline are potential treatments for anthrax, cystic fibrosis, and West Nile Virus, which have been granted orphan status by the FDA.⁸

The number of orphan drugs is expected to rise in the coming years as more new medicines are developed that target specific genetic disorders.⁹

1. National Organization for Rare Disorders, http://www.rarediseases.org/info/about.html.
2. National Institutes of Health, Office of Rare Diseases, Access to Quality Testing for Rare Diseases: A National Conference, Overview (Rockville, MD: NIH, 26 September 2005), http://rarediseases.info.nih.gov/QTRD/overview.html.
3. Food and Drug Administration, “Office of Orphan Products Development,” Budget 2006, http://www.fda.gov/oc/oms/ofm/budget/2006/PDFs/Summary/Pages194thru199.pdf (accessed 7 September 2005).
4. National Organization for Rare Disorders, http://www.rarediseases.org/info/about.html.
5. Food and Drug Administration, Office of Orphan Products Development, http://www.fda.gov/orphan.
6. 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).
7. Food and Drug Administration, “Office of Orphan Products Development,” op. cit.
8. Food and Drug Administration, “Office of Orphan Products Development,” op. cit.
9. Food and Drug Administration, “Office of Orphan Products Development,” op. cit.