[House Report 107-543]
[From the U.S. Government Publishing Office]
107th Congress Report
HOUSE OF REPRESENTATIVES
2d Session 107-543
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RARE DISEASES ACT OF 2002
_______
June 26, 2002.--Committed to the Committee of the Whole House on the
State of the Union and ordered to be printed
_______
Mr. Tauzin, from the Committee on Energy and Commerce, submitted the
following
R E P O R T
[To accompany H.R. 4013]
The Committee on Energy and Commerce, to whom was referred
the bill (H.R. 4013) to amend the Public Health Service Act to
establish an Office of Rare Diseases at the National Institutes
of Health, and for other purposes, having considered the same,
report favorably thereon without amendment and recommend that
the bill do pass.
CONTENTS
Page
Purpose and Summary.............................................. 1
Background and Need for Legislation.............................. 2
Hearings......................................................... 2
Committee Consideration.......................................... 2
Committee Votes.................................................. 2
Committee Oversight Findings..................................... 3
Statement of General Performance Goals and Objectives............ 3
New Budget Authority, Entitlement Authority, and Tax Expenditures 3
Committee Cost Estimate.......................................... 3
Congressional Budget Office Estimate............................. 3
Federal Mandates Statement....................................... 3
Advisory Committee Statement..................................... 3
Constitutional Authority Statement............................... 3
Applicability to Legislative Branch.............................. 4
Section-by-Section Analysis of the Legislation................... 4
Changes in Existing Law Made by the Bill, as Reported............ 4
Purpose and Summary
The purpose of H.R. 4013, the Rare Diseases Act of 2002, is
to authorize in statute the Office of Rare Diseases at the
National Institutes of Health (``NIH''). Rare diseases are
diseases affecting fewer than 200,000 individuals in the United
States. The Director of the Office of Rare Diseases will serve
as the principal advisor to the Director of the NIH with
respect to rare diseases, and shall serve to promote sufficient
allocation of NIH resources to rare disease research, and
promote and encourage the establishment of a centralized
clearinghouse for rare disease information for the benefit of
the public, medical professionals, patients, and families.
Background and Need for Legislation
In 1993, the NIH created an Office of Rare Diseases within
the Office of the Director of NIH, to respond to the reporting
requirements of the Orphan Drug Act of 1983. The Office of Rare
Diseases implements the recommendations of the National
Commission on Orphan Diseases, and responds to requests for
information on rare diseases.
Rare, or Orphan, Diseases affect fewer than 200,000
individuals in America. Presently, more than 6,000 rare
diseases affecting more than 25 million Americans are known.
Prior to the enactment of the Orphan Disease Act of 1983, there
was not enough of an incentive for drug and biological
manufacturers to invest in treatments for these diseases. The
Orphan Drug Act created greater incentives through increased
exclusivity (7 years) for drugs and biologicals which treat
Orphan Diseases, among other things.
While the Orphan Drug Act has achieved dramatic increases
in research into, and treatments for, rare diseases, more still
needs to be done. One positive step includes authorizing in
statute the Office of Rare Diseases. This action sends the
strong signal of the Congress' commitment for both this Office
as well as for rare disease research generally. Also,
authorizing regional centers of excellence for rare disease
research is a positive step, as it will enable the NIH to
select sites to concentrate on finding cures and treatment
methods for rare diseases. Both of these proposals are
contained within H.R. 4013.
Hearings
The Subcommittee on Health held a hearing on ``The National
Institutes of Health: Investing in Research to Prevent and Cure
Disease'' on June 6, 2002. The Subcommittee received testimony
from Claude Lenfant, M.D., Director, National Heart, Lung, and
Blood Institute; Audrey S. Penn, M.D., Acting Director,
National Institute of Neurological Disorders and Stroke; Robert
O. Bonow, M.D., President-elect, American Heart Association;
Eric Hargis, President and CEO, The Epilepsy Foundation; Edward
Sanchez, M.D., M.P.H., Commissioner, Texas Department of
Health; Daniel Jones, M.D., Vice Chancellor, University Medical
Center, University of Mississippi.
Committee Consideration
On Wednesday, June 19, 2002, the Full Committee met in open
markup session and favorably ordered reported H.R. 4013, the
Rare Diseases Act of 2002, by voice vote, a quorum being
present.
Committee Votes
Clause 3(b) of rule XIII of the Rules of the House of
Representatives requires the Committee to list the record votes
on the motion to report legislation and amendments thereto.
There were no record votes taken in connection with ordering
H.R. 4013 reported. A motion by Mr. Tauzin to order H.R. 4013
reported to the House, without amendment, was agreed to by a
voice vote.
Committee Oversight Findings
Pursuant to clause 3(c)(1) of rule XIII of the Rules of the
House of Representatives, the Committee held an oversight
hearing and made findings that are reflected in this report.
Statement of General Performance Goals and Objectives
The objective of this legislation is to increase support
for rare disease research within and without the National
Institutes of Health through the statutory authorization of the
Office of Rare Diseases and by allowing the Secretary to
establish rare disease regional centers of excellence.
New Budget Authority, Entitlement Authority, and Tax Expenditures
In compliance with clause 3(c)(2) of rule XIII of the Rules
of the House of Representatives, the Committee finds that H.R.
4103, would result in no new or increased budget authority,
entitlement authority, or tax expenditures or revenues.
Committee Cost Estimate
The Committee adopts as its own the cost estimate prepared
by the Director of the Congressional Budget Office pursuant to
section 402 of the Congressional Budget Act of 1974, which is
included in the report to accompany H.R. 4984.
Congressional Budget Office Estimate
Pursuant to clause 3(c)(3) of rule XIII of the Rules of the
House of Representatives, the cost estimate provided by the
Congressional Budget Office pursuant to section 402 of the
Congressional Budget Act of 1974 is included in the report to
accompany H.R. 4984.
Federal Mandates Statement
The Committee adopts as its own the estimate of Federal
mandates prepared by the Director of the Congressional Budget
Office pursuant to section 423 of the Unfunded Mandates Reform
Act. The estimate is included in the report to accompany H.R.
4984.
Advisory Committee Statement
No advisory committees within the meaning of section 5(b)
of the Federal Advisory Committee Act were created by this
legislation.
Constitutional Authority Statement
Pursuant to clause 3(d)(1) of rule XIII of the Rules of the
House of Representatives, the Committee finds that the
Constitutional authority for this legislation is provided in
Article I, section 8, clause 3, which grants Congress the power
to regulate commerce with foreign nations, among the several
States, and with the Indian tribes.
Applicability to Legislative Branch
The Committee finds that the legislation does not relate to
the terms and conditions of employment or access to public
services or accommodations within the meaning of section
102(b)(3) of the Congressional Accountability Act.
Section-by-Section Analysis of the Legislation
Section 1. Short Title
Section 1 establishes that the short title for this
legislation is the Rare Diseases Act of 2002.
Section 2. Findings and Purposes
Section 2 contains Congressional findings.
Section 3. NIH Office of Rare Diseases at National Institutes of Health
Section 3 establishes within the Office of the Director of
the NIH an office to be known as the Office of Rare Diseases,
and ensures that the Director of the Office of Rare Diseases
will (1) recommend an agenda for conducting and supporting
research on rare diseases; (2) promote, with respect to rare
diseases, coordination and cooperation among NIH institutes and
centers; and, (3) promote the sufficient allocation of the
resources of the NIH for conducting and supporting research on
rare diseases. The section also authorizes $4 million in each
of Fiscal Years 2003 through 2006 for these purposes.
Section 4. Rare Disease Regional Centers of Excellence
Section 4 empowers the Director of the Office of Rare
Diseases, in collaboration with the directors of the other
relevant institutes and centers of the NIH, to enter into
cooperative agreements and make grants for the establishment of
regional centers of excellence for clinical research into,
training in, and demonstration of diagnostic, prevention,
control, and treatment methods for rare diseases. This section
authorizes $20 million in each of Fiscal Years 2003 through
2006 for these purposes.
Changes in Existing Law Made by the Bill, as Reported
In compliance with clause 3(e) of rule XIII of the Rules of
the House of Representatives, changes in existing law made by
the bill, as reported, are shown as follows (new matter is
printed in italic and existing law in which no change is
proposed is shown in roman):
PUBLIC HEALTH SERVICE ACT
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TITLE IV--NATIONAL RESEARCH INSTITUTES
Part A--National Institutes of Health
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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.
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.
* * * * * * *