President Bush Signs Rare Diseases Legislation

New Laws Address Unmet Needs of 25 Million Americans

MEDIA RELEASE:

Contact: Diane Dorman
Office (202) 496-1296; Cell (202) 258-6457

Danbury, Connecticut, November 7, 2002 - The White House announced today that President Bush signed into law the Rare Diseases Act (H.R. 4013) and the Rare Diseases Orphan Product Development Act (H.R. 4014). The legislation addresses a longstanding unmet need to develop new treatments and cures for rare disorders.

Rare "orphan" diseases are defined as medical conditions affecting fewer than 200,000 Americans. Although the incidence of each disease is small, combined together approximately 25 million Americans suffer from at least one of the known 6,000 rare disorders.

Listed among the 6,000 rare medical conditions are sickle cell anemia, Tay-Sachs disease, hemophilia, Fanconi's anemia, Tourette syndrome, Lou Gehrig's disease (amyotrophic lateral sclerosis) and the Bubble Boy Disease (severe combined immune deficiency). The majority, however, are obscure diseases such as Landau Kleffner Syndrome, Wilson's disease, tyrosinemia, Canavan disease, or Creutzfeldt-Jakob disease. Some of these illnesses affect only a few hundred people worldwide.

"Not since the passage of the Orphan Drug Act in 1983 which created financial incentives and research grants, has legislation been enacted that has such a profound and lasting impact on the millions suffering with rare diseases," said Abbey Meyers, President of the National Organization for Rare Disorders (NORD). "The entire rare disease community has worked so hard for this momentous day".

Originally introduced in August 2001 by Senators Edward Kennedy (D-MA) and Orrin Hatch (R-UT) - the Rare Diseases Act of 2001 (S. 1379) was later split into two separate bills: H.R. 4013, sponsored by Representative John Shimkus (R-IL), nearly doubles the budget for the Office of Rare Diseases (ORD) at the National Institutes of Health (NIH), and authorizes the office to enter into cooperative agreements and to award grants for clinical research into rare diseases. H.R. 4014, sponsored by Representative Mark Foley (R-FL), doubles the funding for the Food and Drug Administration's Orphan Products Research Grant program. Congressman Henry Waxman (D-CA), a long-time supporter of the rare disease community and the chief sponsor of the Orphan Drug Act, co-sponsored both H.R. 4013 and H.R. 4014.

To read the complete text of this news release from NORD, go to www.rarediseases.org. To read the White House announcement of this news, go to www.whitehouse.gov.

19.Sept.'03