[Congressional Record Volume 147, Number 112 (Friday, August 3, 2001)]
[Senate]
[Page S8952]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]

      By Mr. KENNEDY (for himself and Mr. Hatch):
  S. 1379. A bill to amend the Public Health Service Act to establish 
an Office of Rare Diseases at the National Institutes of Health, and 
for other purposes; to the Committee on Health, Education, Labor, and 
Pensions.
  Mr. KENNEDY. Mr. President, I am pleased to introduce the Rare 
Diseases Act of 2001.
  This legislation, in conjunction with companion legislation 
introduced by Senator Hatch to amend the orphan drug tax credit, 
promises to greatly enhance the prospects for developing new treatments 
and diagnostics, and even cures for literally thousands of rare 
diseases and disorders.
  The Rare Diseases Act provides a statutory authorization for the 
existing Office of Rare Diseases at the National Institutes of Health, 
NIH, and authorizes regional centers of excellence for rare disease 
research and training. The Act also increases the funding for the Food 
and Drug Administration's, FDA, Orphan Product Research Grant program, 
which has provided vital support for clinical research on new 
treatments for rare diseases and disorders.
  I am encouraged that, consistent with our legislation, the President 
has proposed in fiscal year 2002 to create a network of centers of 
excellence for rare diseases. This proposal originated with the NIH, in 
recommendations of a Special Emphasis Panel convened to examine the 
state of rare disease research. Because the Panel itself was convened 
in response to a request of the Senate Appropriations Committee in 
1966, it is appropriate that we are today introducing legislation which 
represents the fruition of a long, deliberative process involving both 
the Congress and the NIH.
  It is important to note that Congress has had a longstanding interest 
in rare diseases. In 1983, Congress enacted the Orphan Drug Act to 
promote the development of treatments for rare diseases and disorders. 
Such diseases affect small patient populations, typically smaller than 
200,000 individuals in the United States, and include Huntington's 
disease, myoclonus, ALS, Lou Gehrig's disease, Tourette syndrome, and 
muscular dystrophy. Although each disease may be rare, there are, in 
sum, 25 million Americans today who suffer from the six thousand known 
rare diseases and disorders.
  As an original sponsor of the Orphan Drug Act, I am pleased it has 
been a great success, leading to the development of over 220 treatments 
for rare diseases and disorders. But the greatest share of credit is 
due to the original author of the Act, Congressman Henry Waxman of 
California, and to a woman named Abbey Meyers.
  During the 1970s, an organization called the National Organization 
for Rare Disorders, NORD, was founded by Abbey to provide services and 
to lobby on behalf of patients with rare diseases and disorders. It was 
Abbey and her organization which were instrumental in pressing Congress 
for enactment of legislation to encourage the development of orphan 
drugs.
  In light of this important history, I am very pleased that the Rare 
Diseases Act of 2001 is supported by NORD. And I am also pleased to 
join my colleague, Senator Hatch, a champion of research into rare 
diseases, in introducing this legislation.
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