[107th Congress Public Law 280]
[From the U.S. Government Printing Office]
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[DOCID: f:publ280.107]
[[Page 1987]]
RARE DISEASES ACT OF 2002
[[Page 116 STAT. 1988]]
Public Law 107-280
107th Congress
An Act
To amend the Public Health Service Act to establish an Office of Rare
Diseases at the National Institutes of Health, and for other
purposes. <<NOTE: Nov. 6, 2002 - [H.R. 4013]>>
Be it enacted by the Senate and House of Representatives of the
United States of America in Congress <<NOTE: Rare Diseases Act of
2002.>> assembled,
SECTION 1. <<NOTE: 42 USC 201 note.>> SHORT TITLE.
This Act may be cited as the ``Rare Diseases Act of 2002''.
SEC. 2. <<NOTE: 42 USC 283h note.>> FINDINGS AND PURPOSES.
(a) Findings.--Congress makes the following findings:
(1) Rare diseases and disorders are those which affect small
patient populations, typically populations smaller than 200,000
individuals in the United States. Such diseases and conditions
include Huntington's disease, amyotrophic lateral sclerosis (Lou
Gehrig's disease), Tourette syndrome, Crohn's disease, cystic
fibrosis, cystinosis, and Duchenne muscular dystrophy.
(2) For many years, the 25,000,000 Americans suffering from
the over 6,000 rare diseases and disorders were denied access to
effective medicines because prescription drug manufacturers
could rarely make a profit from marketing drugs for such small
groups of patients. The prescription drug industry did not
adequately fund research into such treatments. Despite the
urgent health need for these medicines, they came to be known as
``orphan drugs'' because no companies would commercialize them.
(3) During the 1970s, an organization called the National
Organization for Rare Disorders (NORD) was founded to provide
services and to lobby on behalf of patients with rare diseases
and disorders. NORD was instrumental in pressing Congress for
legislation to encourage the development of orphan drugs.
(4) The Orphan Drug Act created financial incentives for the
research and production of such orphan drugs. New Federal
programs at the National Institutes of Health and the Food and
Drug Administration encouraged clinical research and commercial
product development for products that target rare diseases. An
Orphan Products Board was established to promote the development
of drugs and devices for rare diseases or disorders.
(5) Before 1983, some 38 orphan drugs had been developed.
Since the enactment of the Orphan Drug Act, more than 220 new
orphan drugs have been approved and marketed in the
[[Page 116 STAT. 1989]]
United States and more than 800 additional drugs are in the
research pipeline.
(6) Despite the tremendous success of the Orphan Drug Act,
rare diseases and disorders deserve greater emphasis in the
national biomedical research enterprise. The Office of Rare
Diseases at the National Institutes of Health was created in
1993, but lacks a statutory authorization.
(7) The National Institutes of Health has received a
substantial increase in research funding from Congress for the
purpose of expanding the national investment of the United
States in behavioral and biomedical research.
(8) Notwithstanding such increases, funding for rare
diseases and disorders at the National Institutes of Health has
not increased appreciably.
(9) To redress this oversight, the Department of Health and
Human Services has proposed the establishment of a network of
regional centers of excellence for research on rare diseases.
(b) Purposes.--The purposes of this Act are to--
(1) amend the Public Health Service Act to establish an
Office of Rare Diseases at the National Institutes of Health;
and
(2) increase the national investment in the development of
diagnostics and treatments for patients with rare diseases and
disorders.
SEC. 3. NIH OFFICE OF RARE DISEASES AT NATIONAL INSTITUTES OF HEALTH.
Title IV of the Public Health Service Act (42 U.S.C. 281 et seq.),
as amended by Public Law 107-84, is amended by inserting after section
404E the following:
``office of rare diseases
``Sec. 404F. <<NOTE: 42 USC 283h.>> (a) Establishment.--There is
established within the Office of the Director of NIH an office to be
known as the Office of Rare Diseases (in this section referred to as the
`Office'), which shall be headed by a Director (in this section referred
to as the `Director'), appointed by the Director of NIH.
``(b) Duties.--
``(1) In general.--The Director of the Office shall carry
out the following:
``(A) The Director shall recommend an agenda for
conducting and supporting research on rare diseases
through the national research institutes and centers.
The agenda shall provide for a broad range of research
and education activities, including scientific workshops
and symposia to identify research opportunities for rare
diseases.
``(B) The Director shall, with respect to rare
diseases, promote coordination and cooperation among the
national research institutes and centers and entities
whose research is supported by such institutes.
``(C) The Director, in collaboration with the
directors of the other relevant institutes and centers
of the National Institutes of Health, may enter into
cooperative agreements with and make grants for regional
centers of excellence on rare diseases in accordance
with section 404G.
[[Page 116 STAT. 1990]]
``(D) The Director shall promote the sufficient
allocation of the resources of the National Institutes
of Health for conducting and supporting research on rare
diseases.
``(E) The Director shall promote and encourage the
establishment of a centralized clearinghouse for rare
and genetic disease information that will provide
understandable information about these diseases to the
public, medical professionals, patients and families.
``(F) <<NOTE: Reports.>> The Director shall
biennially prepare a report that describes the research
and education activities on rare diseases being
conducted or supported through the national research
institutes and centers, and that identifies particular
projects or types of projects that should in the future
be conducted or supported by the national research
institutes and centers or other entities in the field of
research on rare diseases.
``(G) <<NOTE: Reports.>> The Director shall prepare
the NIH Director's annual report to Congress on rare
disease research conducted by or supported through the
national research institutes and centers.
``(2) Principal advisor regarding orphan diseases.--With
respect to rare diseases, the Director shall serve as the
principal advisor to the Director of NIH and shall provide
advice to other relevant agencies. The Director shall provide
liaison with national and international patient, health and
scientific organizations concerned with rare diseases.
``(c) Definition.--For purposes of this section, the term `rare
disease' means any disease or condition that affects less than 200,000
persons in the United States.
``(d) Authorization of Appropriations.--For the purpose of carrying
out this section, there are authorized to be appropriated such sums as
already have been appropriated for fiscal year 2002, and $4,000,000 for
each of the fiscal years 2003 through 2006.''.
SEC. 4. RARE DISEASE REGIONAL CENTERS OF EXCELLENCE.
Title IV of the Public Health Service Act (42 U.S.C. 281 et seq.),
as amended by section 3, is further amended by inserting after section
404F the following:
``rare disease regional centers of excellence
``Sec. 404G. <<NOTE: 42 USC 283i.>> (a) Cooperative Agreements and
Grants.--
``(1) In general.--The Director of the Office of Rare
Diseases (in this section referred to as the `Director'), in
collaboration with the directors of the other relevant
institutes and centers of the National Institutes of Health, may
enter into cooperative agreements with and make grants to public
or private nonprofit entities to pay all or part of the cost of
planning, establishing, or strengthening, and providing basic
operating support for regional centers of excellence for
clinical research into, training in, and demonstration of
diagnostic, prevention, control, and treatment methods for rare
diseases.
``(2) Policies.--A cooperative agreement or grant under
paragraph (1) shall be entered into in accordance with policies
established by the Director of NIH.
``(b) Coordination With Other Institutes.--The Director shall
coordinate the activities under this section with similar activities
conducted by other national research institutes, centers and
[[Page 116 STAT. 1991]]
agencies of the National Institutes of Health and by the Food and Drug
Administration to the extent that such institutes, centers and agencies
have responsibilities that are related to rare diseases.
``(c) Uses for Federal Payments Under Cooperative Agreements or
Grants.--Federal payments made under a cooperative agreement or grant
under subsection (a) may be used for--
``(1) staffing, administrative, and other basic operating
costs, including such patient care costs as are required for
research;
``(2) clinical training, including training for allied
health professionals, continuing education for health
professionals and allied health professions personnel, and
information programs for the public with respect to rare
diseases; and
``(3) clinical research and demonstration programs.
``(d) Period of Support; Additional Periods.--Support of a center
under subsection (a) may be for a period of not to exceed 5 years. Such
period may be extended by the Director for additional periods of not
more than 5 years if the operations of such center have been reviewed by
an appropriate technical and scientific peer review group established by
the Director and if such group has recommended to the Director that such
period should be extended.
``(e) Authorization of Appropriations.--For the purpose of carrying
out this section, there are authorized to be appropriated such sums as
already have been appropriated for fiscal year 2002, and $20,000,000 for
each of the fiscal years 2003 through 2006.''.
Approved November 6, 2002.
LEGISLATIVE HISTORY--H.R. 4013 (S. 1379):
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HOUSE REPORTS: No. 107-543 (Comm. on Energy and Commerce).
SENATE REPORTS: No. 107-129 accompanyng S. 1379 (Comm. on Health,
Education, Labor, and Pensions).
CONGRESSIONAL RECORD, Vol. 148 (2002):
Oct. 1, considered and passed House.
Oct. 17, considered and passed Senate.
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