[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[H.R. 4013 Reported in House (RH)]
Union Calendar No. 324
107th CONGRESS
2d Session
H. R. 4013
[Report No. 107-543]
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 HOUSE OF REPRESENTATIVES
March 20, 2002
Mr. Shimkus (for himself, Mr. Waxman, Mr. Foley, Mr. Brown of Ohio,
Mrs. Roukema, Mr. Rush, Mr. King, Mr. Greenwood, and Mr. Dingell)
introduced the following bill; which was referred to the Committee on
Energy and Commerce
June 26, 2002
Additional sponsors: Mr. McGovern, Mr. Horn, Mr. Smith of New Jersey,
Ms. McKinney, Mrs. Morella, Mr. Wynn, Mr. Green of Texas, Mr. Towns,
Mr. Frank, Mr. Lynch, Mr. Pallone, Ms. Rivers, Mrs. Capps, Mr. George
Miller of California, Ms. DeGette, Mr. Weldon of Florida, Mr. Hoeffel,
Mr. Lantos, Mr. Stupak, Mrs. Mink of Hawaii, Mrs. Kelly, Ms. Slaughter,
Mr. McDermott, Ms. Woolsey, Mr. Platts, Ms. Velazquez, Mr. Kind, Mrs.
Johnson of Connecticut, Mr. Bonior, Mr. Wexler, Ms. Norton, Mr. Lucas
of Oklahoma, Mr. Moran of Virginia, Mr. Sununu, Mr. Isakson, Mrs.
Lowey, Mr. Olver, Ms. Schakowsky, Mr. Kennedy of Rhode Island, Mr. Tom
Davis of Virginia, Mr. Price of North Carolina, Mr. Wolf, Mr. Levin,
Mr. Deutsch, Ms. Carson of Indiana, and Mr. Sanders
June 26, 2002
Committed to the Committee of the Whole House on the State of the Union
and ordered to be printed
_______________________________________________________________________
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 2002''.
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.
(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. (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.
``(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 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. (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 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.''.
Union Calendar No. 324
107th CONGRESS
2d Session
H. R. 4013
[Report No. 107-543]
_______________________________________________________________________
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.
_______________________________________________________________________
June 26, 2002
Committed to the Committee of the Whole House on the State of the Union
and ordered to be printed