Background
A rare disease or disorder is defined in the U.S. as one affecting fewer than 200,000 Americans. There are around 6,000 known rare diseases, and it is estimated that about 25 million Americans are affected by them. Over 220 treatments have been developed in the last two decades for rare diseases and disorders, but many more are needed.

Office of Rare Diseases
The Office of Rare Diseases at the National Institutes of Health (NIH) was established in 1993 to promote research and provide information. However, this office was not given any authority. Also, although Congress has substantially increased research funding for NIH over the years, funding for rare diseases and disorders has only increased slightly.

The Rare Diseases Act of 2002
This Act establishes the Office of Rare Diseases as a federal entity able to recommend a national research agenda, coordinate research, and provide educational activities for researchers.

Rare Diseases Orphan Product Development Act
This Act increase the U.S. government investment in the development of diagnostics and treatments for rare diseases and disorders by establishing regional centers of excellence for clinical research and doubling the funding for the Orphan Product Research Grant Program.

Significant legislation
These pieces of legislation are very important for the progress of research on treatments and cures for rare diseases and disorders. They were both signed into law by President Bush on November 7, 2002.

Information for this article was taken from:
- National Organization for Rare Disorders
- U.S. Senate: Summary and Findings of the Rare Diseases Act of 2001
- The Congressional Record: Rare Diseases Act of 2001

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