[tri-med] FYI - PRESIDENT BUSH SIGNS RARE DISEASES LEGISLATION

DATE:   November 7, 2002

RE:     PRESIDENT BUSH SIGNS RARE DISEASES LEGISLATION


MEDIA RELEASE:

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

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

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," 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
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