President Bush Signs Rare Diseases Legislation; New Laws Address Unmet Needs of 25 Million Americans

11/7/2002

From: Diane Dorman of the National Organization for Rare Disorders, 202-496-1296, or cell 202-258-6457

DANBURY, Conn., Nov, 7 -- 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," Meyers continued.

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.

The Office of Rare Diseases at the NIH was initially established in 1993 to promote research and collaboration on orphan diseases. Today that office serves as a link between basic and translational research, and stimulates new research on rare diseases. H.R. 4013 provides a statutory authorization for this important office and will allow the ORD to play an increased role in formulating the research agenda at the NIH.

In the nearly twenty years since the passage of the Orphan Drug Act, the FDA's orphan products research grant program has supported small clinical trials at academic institutions and small companies. The grants have enabled scientists to develop the preliminary scientific data necessary to prove that a new treatment warrants commercial development and FDA approval. With very limited funding, these grants helped 30 new drugs and medical devices to reach the American market. If not for this program, therapies for such rare diseases as sickle cell anemia and narcolepsy would not be on the market today. H.R. 4014 will enable the FDA to double the number of new research grants, thus propelling development of many new treatments for rare diseases in years to come.

Asked about the next steps, Diane Dorman, NORD's vice president for public policy said, "Our work is still cut out for us. The bills authorize increased funding for both the NIH and FDA research programs, but lawmakers must approve the funds to finance them. So we'll be turning to our friends in Washington, DC asking for their help in the 108th Congress to ensure that the programs are fully funded in accordance with Congressional intent."

NORD is a federation of approximately 125 voluntary health organizations and over 60,000 patients, families, healthcare providers, and individuals dedicated to helping people with rare "orphan" diseases. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service.

Note to Editors: Go to the White House web site at http://www.whitehouse.gov for complete details.