Sets forth the duties of such Office and regional centers, including research and educational duties.
Title II: Food and Drug Administration - Amends the Orphan Drug Act to authorize a specified amount of appropriations for FY 2002 and such sums as may be necessary for subsequent fiscal years for grants and contracts for the development of drugs for rare diseases and conditions.
[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[S. 1379 Introduced in Senate (IS)]
107th CONGRESS
1st Session
S. 1379
To amend the Public Health Service Act to establish an Office of Rare
Diseases at the National Institutes of Health, and for other purposes.
_______________________________________________________________________
IN THE SENATE OF THE UNITED STATES
August 3, 2001
Mr. Kennedy (for himself and Mr. Hatch) introduced the following bill;
which was read twice and referred to the Committee on Health,
Education, Labor, and Pensions
_______________________________________________________________________
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.
Be it enacted by the Senate and House of Representatives of the
United States of America in Congress assembled,
SECTION 1. SHORT TITLE.
This Act may be cited as the ``Rare Diseases Act of 2001''.
SEC. 2. 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 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.
(10) The Food and Drug Administration supports small
clinical trials through Orphan Products Research Grants. Such
grants embody successful partnerships of government and
industry, and have led to the development of at least 23 drugs
and four medical devices for rare diseases and disorders. Yet
the appropriations in Fiscal Year 2001 for such grants were
less than in Fiscal Year 1995.
(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.
TITLE I--NATIONAL INSTITUTES OF HEALTH
SEC. 101. NIH OFFICE OF RARE DISEASES.
Title IV of the Public Health Service Act (42 U.S.C. 281 et seq.)
is amended by inserting after section 404D the following:
``office of rare diseases
``Sec. 404E. (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 shall enter into cooperative
agreements with and make grants for regional centers of
excellence on rare diseases in accordance with section
404F.
``(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) 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) 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 $4,000,000
for fiscal year 2002, and such sums as may be necessary for each
subsequent fiscal year.''.
SEC. 102. 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 101, is further amended by inserting after
section 404E the following:
``rare disease regional centers of excellence
``Sec. 404F. (a) Cooperative Agreements and Grants.--
``(1) In general.--The Director of the Office of Rare
Diseases (in this section referred to as the `Director') shall
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 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 $20,000,000
for fiscal year 2002, and such sums as may be necessary for each
subsequent fiscal year.''.
TITLE II--FOOD AND DRUG ADMINISTRATION
SEC. 201. GRANTS AND CONTRACTS FOR THE DEVELOPMENT OF ORPHAN DRUGS.
Subsection (c) of section 5 of the Orphan Drug Act (21 U.S.C.
360ee(c)) is amended to read as follows:
``(c) For grants and contracts under subsection (a) there are
authorized to be appropriated $25,000,000 for fiscal year 2002, and
such sums as may be necessary for each subsequent fiscal year.''.
SEC. 202. TECHNICAL AMENDMENT.
Section 527(a) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C.360cc(a)) is amended in the matter following paragraph (2)--
(1) by striking ``, of such certification,''; and
(2) by striking ``, the issuance of the certification,''.
<all>
Introduced in Senate
Sponsor introductory remarks on measure. (CR S8952)
Read twice and referred to the Committee on Health, Education, Labor, and Pensions.
Committee on Health, Education, Labor, and Pensions. Ordered to be reported with an amendment in the nature of a substitute favorably.
Committee on Health, Education, Labor, and Pensions. Reported by Senator Kennedy with an amendment in the nature of a substitute. With written report No. 107-129.
Committee on Health, Education, Labor, and Pensions. Reported by Senator Kennedy with an amendment in the nature of a substitute. With written report No. 107-129.
Placed on Senate Legislative Calendar under General Orders. Calendar No. 298.
Llama 3.2 · runs locally in your browser
Ask anything about this bill. The AI reads the full text to answer.
Enter to send · Shift+Enter for new line