[House Report 107-195]
[From the U.S. Government Publishing Office]
107th Congress Report
HOUSE OF REPRESENTATIVES
1st Session 107-195
======================================================================
MUSCULAR DYSTROPHY COMMUNITY ASSISTANCE, RESEARCH AND EDUCATION
AMENDMENTS OF 2001
_______
September 5, 2001.--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. 717]
[Including cost estimate of the Congressional Budget Office]
The Committee on Energy and Commerce, to whom was referred
the bill (H.R. 717) to amend the Public Health Service Act to
provide for research and services with respect to Duchenne
muscular dystrophy, having considered the same, report
favorably thereon with amendments and recommend that the bill
as amended do pass.
CONTENTS
Page
Amendment........................................................ 2
Purpose and Summary.............................................. 5
Background and Need for Legislation.............................. 6
Hearings......................................................... 6
Committee Consideration.......................................... 6
Committee Votes.................................................. 7
Committee Oversight Findings..................................... 7
Statement of General Performance Goals and Objectives............ 7
New Budget Authority, Entitlement Authority, and Tax Expenditures 7
Committee Cost Estimate.......................................... 7
Congressional Budget Office Estimate............................. 7
Federal Mandates Statement....................................... 9
Advisory Committee Statement..................................... 9
Constitutional Authority Statement............................... 9
Applicability to Legislative Branch.............................. 10
Section-by-Section Analysis of the Legislation................... 10
Changes in Existing Law Made by the Bill, as Reported............ 11
Amendment
The amendments are as follows:
Strike all after the enacting clause and insert the
following:
SECTION 1. SHORT TITLE.
This Act may be cited as the ``Muscular Dystrophy Community
Assistance, Research and Education Amendments of 2001'', or the ``MD-
CARE Act''.
SEC. 2. FINDINGS.
Congress makes the following findings:
(1) Of the childhood muscular dystrophies, Duchenne Muscular
Dystrophy (DMD) is the world's most common and catastrophic
form of genetic childhood disease, and is characterized by a
rapidly progressive muscle weakness that almost always results
in death, usually by 20 years of age.
(2) Duchenne muscular dystrophy is genetically inherited, and
mothers are the carriers in approximately 70 percent of all
cases.
(3) If a female is a carrier of the dystrophin gene, there is
a 50 percent chance per birth that her male offspring will have
Duchenne muscular dystrophy, and a 50 percent chance per birth
that her female offspring will be carriers.
(4) Duchenne is the most common lethal genetic disorder of
childhood worldwide, affecting approximately 1 in every 3,500
boys worldwide.
(5) Children with muscular dystrophy exhibit extreme symptoms
of weakness, delay in walking, waddling gait, difficulty in
climbing stairs, and progressive mobility problems often in
combination with muscle hypertrophy.
(6) Other forms of muscular dystrophy affecting children and
adults include Becker, limb girdle, congenital,
facioscapulohumeral, myotonic, oculopharyngeal, distal, and
Emery-Dreifuss muscular dystrophies.
(7) Myotonic muscular dystrophy (also known as Steinert's
disease and dystrophia myotonica) is the second most prominent
form of muscular dystrophy and the type most commonly found in
adults. Unlike any of the other muscular dystrophies, the
muscle weakness is accompanied by myotonia (delayed relaxation
of muscles after contraction) and by a variety of abnormalities
in addition to those of muscle.
(8) Facioscapulohumeral muscular dystrophy (referred to in
this section as ``FSHD'') is a neuromuscular disorder that is
inherited genetically and has an estimated frequency of 1 in
20,000. FSHD, affecting between 15,000 to 40,000 persons,
causes a progressive and sever loss of skeletal muscle
gradually bringing weakness and reduced mobility. Many persons
with FSHD become severely physically disabled and spend many
decades in a wheelchair.
(9) FSHD is regarded as a novel genetic phenomenon resulting
from a crossover of subtelomeric DNA and may be the only human
disease caused by a deletion-mutation.
(10) Each of the muscular dystrophies, though distinct in
progressivity and severity of symptoms, have a devastating
impact on tens of thousands of children and adults throughout
the United States and worldwide and impose severe physical and
economic burdens on those affected.
(11) Muscular dystrophies have a significant impact on
quality of life--not only for the individual who experiences
its painful symptoms and resulting disability, but also for
family members and caregivers.
(12) Development of therapies for these disorders, while
realistic with recent advances in research, is likely to
require costly investments and infrastructure to support gene
and other therapies.
(13) There is a shortage of qualified researchers in the
field of neuromuscular research.
(14) Many family physicians and health care professionals
lack the knowledge and resources to detect and properly
diagnose the disease as early as possible, thus exacerbating
the progressiveness of symptoms in cases that go undetected or
misdiagnosed.
(15) There is a need for efficient mechanisms to translate
clinically relevant findings in muscular dystrophy research
from basic science to applied work.
(16) Educating the public and health care community
throughout the country about this devastating disease is of
paramount importance and is in every respect in the public
interest and to the benefit of all communities.
SEC. 3. EXPANSION, INTENSIFICATION, AND COORDINATION OF ACTIVITIES OF
NATIONAL INSTITUTES OF HEALTH WITH RESPECT TO
RESEARCH ON MUSCULAR DYSTROPHY.
Part A of title IV of the Public Health Service Act (42 U.S.C. 281 et
seq.) is amended by adding at the end the following:
``SEC. 404E. MUSCULAR DYSTROPHY; INITIATIVE THROUGH DIRECTOR OF
NATIONAL INSTITUTES OF HEALTH.
``(a) Expansion, Intensification, and Coordination of Activities.--
``(1) In general.--The Director of NIH, in coordination with
the Directors of the National Institute of Neurological
Disorders and Stroke, the National Institute of Arthritis and
Muscoskeletal and Skin Diseases, the National Institute of
Child Health and Human Development, and the other national
research institutes as appropriate, shall expand and intensify
programs of such Institutes with respect to research and
related activities concerning various forms of muscular
dystrophy, including Duchenne, myotonic, facioscapulohumeral
muscular dystrophy (referred to in this section as `FSHD') and
other forms of muscular dystrophy.
``(2) Coordination.--The Directors referred to in paragraph
(1) shall jointly coordinate the programs referred to in such
paragraph and consult with the Muscular Dystrophy Interagency
Coordinating Committee established under section 6 of the MD-
CARE Act.
``(3) Allocations by director of nih.--The Director of NIH
shall allocate the amounts appropriated to carry out this
section for each fiscal year among the national research
institutes referred to in paragraph (1).
``(b) Centers of Excellence.--
``(1) In general.--The Director of NIH shall award grants and
contracts under subsection (a)(1) to public or nonprofit
private entities to pay all or part of the cost of planning,
establishing, improving, and providing basic operating support
for centers of excellence regarding research on various forms
of muscular dystrophy.
``(2) Research.--Each center under paragraph (1) shall
supplement but not replace the establishment of a comprehensive
research portfolio in all the muscular dystrophies. As a whole,
the centers shall conduct basic and clinical research in all
forms of muscular dystrophy including early detection,
diagnosis, prevention, and treatment, including the fields of
muscle biology, genetics, noninvasive imaging, genetics,
pharmacological and other therapies.
``(3) Coordination of centers; reports.--The Director of
NIH--
``(A) shall, as appropriate, provide for the
coordination of information among centers under
paragraph (1) and ensure regular communication between
such centers; and
``(B) shall require the periodic preparation of
reports on the activities of the centers and the
submission of the reports to the Director.
``(4) Organization of centers.--Each center under paragraph
(1) shall use the facilities of a single institution, or be
formed from a consortium of cooperating institutions, meeting
such requirements as may be prescribed by the Director of NIH.
``(5) Duration of support.--Support for a center established
under paragraph (1) may be provided under this section for a
period of not to exceed 5 years. Such period may be extended
for 1 or more additional periods not exceeding 5 years if the
operations of such center have been reviewed by an appropriate
technical and scientific peer review group established by the
Director of NIH and if such group has recommended to the
Director that such period should be extended.
``(c) Facilitation of Research.--The Director of NIH shall provide
for a program under subsection (a)(1) under which samples of tissues
and genetic materials that are of use in research on muscular dystrophy
are donated, collected, preserved, and made available for such
research. The program shall be carried out in accordance with accepted
scientific and medical standards for the donation, collection, and
preservation of such samples.
``(d) Coordinating Committee.--
``(1) In general.--The Secretary shall establish the Muscular
Dystrophy Coordinating Committee (referred to in this section
as the `Coordinating Committee') to coordinate activities
across the National Institutes and with other Federal health
programs and activities relating to the various forms of
muscular dystrophy.
``(2) Composition.--The Coordinating Committee shall consist
of not more than 15 members to be appointed by the Secretary,
of which--
``(A) \2/3\ of such members shall represent
governmental agencies, including the directors or their
designees of each of the national research institutes
involved in research with respect to muscular dystrophy
and representatives of all other Federal departments
and agencies whose programs involve health functions or
responsibilities relevant to such diseases, including
the Centers for Disease Control and Prevention, the
Health Resources and Services Administration and the
Food and Drug Administration and representatives of
other governmental agencies that serve children with
muscular dystrophy, such as the Department of
Education; and
``(B) \1/3\ of such members shall be public members,
including a broad cross section of persons affected
with muscular dystrophies including parents or legal
guardians, affected individuals, researchers, and
clinicians.
Members appointed under subparagraph (B) shall serve for a term
of 3 years, and may serve for an unlimited number of terms if
reappointed.
``(3) Chair.--
``(A) In general.--With respect to muscular
dystrophy, the Chair of the Coordinating Committee
shall serve as the principal advisor to the Secretary,
the Assistant Secretary for Health, and the Director of
NIH, and shall provide advice to the Director of the
Centers for Disease Control and Prevention, the
Commissioner of Food and Drugs, and to the heads of
other relevant agencies. The Coordinating Committee
shall select the Chair for a term not to exceed 2
years.
``(B) Appointment.--The Chair of the Committee shall
be appointed by and be directly responsible to the
Secretary.
``(4) Administrative support; terms of service; other
provisions.--The following shall apply with respect to the
Coordinating Committee:
``(A) The Coordinating Committee shall receive
necessary and appropriate administrative support from
the Department of Health and Human Services.
``(B) The Coordinating Committee shall meet as
appropriate as determined by the Secretary, in
consultation with the chair.
``(e) Plan for HHS Activities.--
``(1) In general.--Not later than 1 year after the date of
enactment of this section, the Coordinating Committee shall
develop a plan for conducting and supporting research and
education on muscular dystrophy through the national research
institutes and shall periodically review and revise the plan.
The plan shall--
``(A) provide for a broad range of research and
education activities relating to biomedical,
epidemiological, psychosocial, and rehabilitative
issues, including studies of the impact of such
diseases in rural and underserved communities;
``(B) identify priorities among the programs and
activities of the National Institutes of Health
regarding such diseases; and
``(C) reflect input from a broad range of scientists,
patients, and advocacy groups.
``(2) Certain elements of plan.--The plan under paragraph (1)
shall, with respect to each form of muscular dystrophy, provide
for the following as appropriate:
``(A) Research to determine the reasons underlying
the incidence and prevalence of various forms of
muscular dystrophy.
``(B) Basic research concerning the etiology and
genetic links of the disease and potential causes of
mutations.
``(C) The development of improved screening
techniques.
``(D) Basic and clinical research for the development
and evaluation of new treatments, including new
biological agents.
``(E) Information and education programs for health
care professionals and the public.
``(f) Reports to Congress.--The Coordinating Committee shall
biennially submit to the Committee on Energy and Commerce of the House
of Representatives, and the Committee on Health, Education, Labor, and
Pensions of the Senate, a report that describes the research,
education, and other activities on muscular dystrophy being conducted
or supported through the Department of Health and Human Services. Each
such report shall include the following:
``(1) The plan under subsection (e)(1) (or revisions to the
plan, as the case may be).
``(2) Provisions specifying the amounts expended by the
Department of Health and Human Services with respect to various
forms of muscular dystrophy, including Duchenne, myotonic, FSHD
and other forms of muscular dystrophy.
``(3) Provisions identifying particular projects or types of
projects that should in the future be considered by the
national research institutes or other entities in the field of
research on all muscular dystrophies.
``(g) Public Input.--The Secretary shall, under subsection (a)(1),
provide for a means through which the public can obtain information on
the existing and planned programs and activities of the Department of
Health and Human Services with respect to various forms of muscular
dystrophy and through which the Secretary can receive comments from the
public regarding such programs and activities.
``(h) Authorization of Appropriations.--For the purpose of carrying
out this section, there are authorized to be appropriated such sums as
may be necessary for each of fiscal years 2002 through 2006. The
authorization of appropriations established in the preceding sentence
is in addition to any other authorization of appropriations that is
available for conducting or supporting through the National Institutes
of Health research and other activities with respect to muscular
dystrophy.''.
SEC. 4. DEVELOPMENT AND EXPANSION OF ACTIVITIES OF CENTERS FOR DISEASE
CONTROL AND PREVENTION WITH RESPECT TO
EPIDEMIOLOGICAL RESEARCH ON MUSCULAR DYSTROPHY.
Part B of title III of the Public Health Service Act (42 U.S.C. 243
et seq.) is amended by inserting after section 317P the following:
``SEC. 317Q. SURVEILLANCE AND RESEARCH REGARDING MUSCULAR DYSTROPHY.
``(a) In General.--The Secretary, acting through the Director of the
Centers for Disease Control and Prevention, may award grants and
cooperative agreements to public or nonprofit private entities
(including health departments of States and political subdivisions of
States, and including universities and other educational entities) for
the collection, analysis, and reporting of data on Duchenne and other
forms of muscular dystrophy. In making such awards, the Secretary may
provide direct technical assistance in lieu of cash.
``(b) National Muscular Dystrophy Epidemiology Program.--The
Secretary, acting through the Director of the Centers for Disease
Control and Prevention, may award grants to public or nonprofit private
entities (including health departments of States and political
subdivisions of States, and including universities and other
educational entities) for the purpose of carrying out epidemiological
activities regarding Duchenne and other forms of muscular dystrophies,
including collecting and analyzing information on the number,
incidence, correlates, and symptoms of cases. In carrying out the
preceding sentence, the Secretary shall provide for a national
surveillance program. In making awards under this subsection, the
Secretary may provide direct technical assistance in lieu of cash.
``(c) Coordination With Centers of Excellence.--The Secretary shall
ensure that epidemiological information under subsections (a) and (b)
is made available to centers of excellence supported under section
404E(b) by the Director of the National Institutes of Health.
``(d) Authorization of Appropriations.--There are authorized to be
appropriated such sums as may be necessary to carry out this
section.''.
SEC. 5. INFORMATION AND EDUCATION.
(a) In General.--The Secretary of Health and Human Services (referred
to in this Act as the ``Secretary'') shall establish and implement a
program to provide information and education on muscular dystrophy to
health professionals and the general public, including information and
education on advances in the diagnosis and treatment of muscular
dystrophy and training and continuing education through programs for
scientists, physicians, medical students, and other health
professionals who provide care for patients with muscular dystrophy.
(b) Stipends.--The Secretary may use amounts made available under
this section provides stipends for health professionals who are
enrolled in training programs under this section.
(c) Authorization of Appropriations.--There are authorized to be
appropriated such sums as may be necessary to carry out this section.
SEC. 6. REPORT TO CONGRESS.
Not later than January 1, 2003, and each January 1 thereafter, the
Secretary shall prepare and submit to the appropriate committees of
Congress, a report concerning the implementation of this Act and the
amendments made by this Act.
Amend the title so as to read:
A bill to amend the Public Health Service Act to provide
for research with respect to various forms of muscular
dystrophy, including Duchenne, Becker, limb girdle, congenital,
facioscapulohumeral, myotonic, oculopharyngeal, distal, and
Emery-Dreifuss muscular dystrophies.
Purpose and Summary
H.R. 717, the Muscular Dystrophy Childhood Assistance,
Research and Education Amendments of 2001, allows the Director
of the National Institutes of Health (NIH), in coordination
with the Directors of the National Institute of Neurological
Disorders and Stroke, the National Institute of Arthritis, and
the National Institute of Child Health and Human Development to
expand programs with respect to activities concerning Duchenne.
The legislation also creates Centers of Excellence for
Duchenne, which shall conduct basic and clinical research into
Duchenne and various other muscular dystrophies.
Background and Need for Legislation
Duchenne Muscular Dystrophy (DMD) is the most lethal
genetic disorder of childhood worldwide, affecting
approximately one in every 3,500 boys worldwide. The disease
has a significant impact on quality of life, not only for the
individual who experiences its painful symptoms and resulting
disability, but also for family members and care givers.
Symptoms of DMD include loss of muscle tissue, inability to
walk, decreased lung capacity, and inability to move the major
joints of the body.
The course of DMD is fairly predictable. Children with the
disorder are often late in learning to walk. A preschooler with
DMD may seem clumsy and fall often. Soon, he has trouble
climbing stairs, getting up from the floor, or running. By
school age, the child may walk on his toes or the balls of his
feet, with a slightly rolling gait. He has a waddling and
unsteady gait, and can easily fall over. Nearly all children
with DMD lose the ability to walk sometime between ages 7 and
12. In the teen years, activities involving the arms, legs, or
trunk require assistance or mechanical support.
Thanks to advances in many areas of medicine, there are
very good therapies available to assist children with all the
effects of DMD and other muscular dystrophies. By using all
available therapies, patients can prolong their comfort,
function, and life expectancy. Despite these advances, however,
current treatment options for Duchenne are minimal in efficacy
and palliative, aimed at simply managing the symptoms in an
effort to optimize the quality of life.
Hearings
The Subcommittee on Health held a hearing on June 27, 2001
on H.R. 717 entitled, ``Advancing the Health of the American
People: Addressing Various Public Health Needs.'' The witnesses
testifying on the bill were Mr. Ed McMahon, National Vice
President, Muscular Dystrophy Association and Ms. Pat Furlong,
President, Parent Project Muscular Dystrophy.
Committee Consideration
On Wednesday, July 11, 2001, the Subcommittee on Health met
in an open markup session and approved H.R. 717 for Full
Committee consideration, as amended, by voice vote, a quorum
being present. On Wednesday, July 18, 2001, the Full Committee
met in open markup session and ordered H.R. 717 favorably
reported to the House, as amended, by unanimous consent, 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. 717 reported. A motion by Mr. Tauzin to order H.R. 717
reported to the House, as amended, was agreed to by unanimous
consent.
Committee Oversight Findings
Pursuant to clause 3(c)(1) of rule XIII of the Rules of the
House of Representatives, the Committee held a legislative
hearing and made findings that are reflected in this report.
Statement of General Performance Goals and Objectives
The goal of H.R. 717 is to allow the Director of the
National Institutes of Health to expand programs with respect
to activities concerning Duchenne, and create Centers of
Excellence for Duchenne to conduct basic and clinical research
into Duchenne and other muscular dystrophies.
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.
717, the Muscular Dystrophy Childhood Assistance, Research and
Education Amendments of 2001, 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.
Congressional Budget Office Estimate
Pursuant to clause 3(c)(3) of rule XIII of the Rules of the
House of Representatives, the following is the cost estimate
provided by the Congressional Budget Office pursuant to section
402 of the Congressional Budget Act of 1974:
U.S. Congress,
Congressional Budget Office,
Washington, DC, August 10, 2001.
Hon. W.J. ``Billy'' Tauzin,
Chairman, Committee on Energy and Commerce,
House of Representatives, Washington, DC.
Dear Mr. Chairman: The Congressional Budget Office has
prepared the enclosed estimate of H.R. 717, the MD-CARE Act, as
ordered reported by the Committee on Energy and Commerce on
July 18, 2001.
If you wish further details on this estimate, we will be
pleased to provide them. The CBO staff contact is Christopher
J. Topoleski.
Sincerely,
Robert A. Sunshine
(For Dan L. Crippen, Director).
Enclosure.
H.R. 717--Muscular Dystrophy Community Assistance, Research and
Education Amendments of 2001 (MD-CARE Act)
Summary: H.R. 717 would require the National Institutes of
Health (NIH) and the Centers for Disease Control and Prevention
(CDC) to provide grants and expand research on the health needs
of individuals with muscular dystrophy. Assuming the
appropriation of the necessary amounts, CBO estimates that
implementing H.R. 717 would cost $4 million in 2002 and $56
million over the 2002-2006 period. The legislation would not
affect direct spending or receipts; therefore, pay-as-you-go
procedures would not apply.
H.R. 717 contains no intergovernmental or private-sector
mandates as defined in the Unfunded Mandates Reform Act (UMRA).
State, local, and tribal governments would be eligible for
grants authorized by the bill for research activities
associated with muscular dystrophy.
Estimated Cost to the Federal Government: The estimated
budgetary impact of H.R. 717 is shown in the following table.
The costs of this legislation fall within budget function 550
(health).
----------------------------------------------------------------------------------------------------------------
By fiscal year, in millions of dollars--
-----------------------------------------------
2001 2002 2003 2004 2005 2006
----------------------------------------------------------------------------------------------------------------
SPENDING SUBJECT TO APPROPRIATION
Spending Under Current Law:
Estimated Authorization Level \1\........................... 6504 6664 6809 6959 7107 7265
Estimated Outlays........................................... 5326 6063 6474 6773 6992 7100
Proposed Changes:
Estimated Authorization Level............................... 0 11 14 15 15 15
Estimated Outlays........................................... 0 4 10 13 14 15
Spending Under H.R. 717:
Estimated Authorization Level............................... 6504 6675 6823 6974 7122 7280
Estimated Outlays........................................... 5326 6067 6484 6786 7006 7115
----------------------------------------------------------------------------------------------------------------
\1\ The 2001 level is the amount appropriated for that year for the agencies that would be affected by H.R. 717.
The 2002-2006 levels are CBO baseline projections, including adjustments for anticipated inflation.
Basis of estimate: The bill would authorize the NIH to
award grants to and contract with public and nonprofit private
entities known as ``Centers of Excellence'' to provide basic
and clinical research on muscular dystrophy, including
diagnosis, early detection, prevention, and treatment. The
centers would be awarded renewable contracts for up to five
years for each contract period. CBO assumes that NIH would
designate one center of excellance in 2002 and two additional
centers in 2003.
The NIH would also be required to establish a program under
which samples of tissue and other genetic materials used in
muscular dystrophy research would be collected, stored, and
made available for study.
The Secretary of Health and Human Services (HHS) would be
required to establish a coordinating committee to organize
muscular dystrophy research activities across the NIH and other
federal health programs. The committee would consist of 15
members appointed from both government agencies and from
members of the public affected by muscular dystrophy. The
committee would be required to produce a plan that identifies
opportunities for research and education on muscular dystrophy.
In addition, the committee would be required to submit a
biennial report to the Congress summarizing expenditures by HHS
and the current and future research agenda.
Based on amounts spent in the past for similar activities,
CBO estimates that the activities of the NIH and the
coordinating committee would cost $2 million in 2002 and $28
million over the 2002-2006 period if the necessary amounts are
appropriated.
The bill would authorize appropriations of such sums as
necessary for the Centers for Disease Control and Prevention to
award grants to public or nonprofit private entities to conduct
research, carry out epidemiological activities, and establish a
national muscular dystrophy surveillance program. Based on
Information provided by the CDC about amounts spent in the past
for similar activities and on the agency's historical spending
patterns, CBO estimates the agency would spend $2 million in
2002 and $28 million over the 2002-2006 period for those
purposes if the necessary amounts are appropriated.
This estimate assumes that the bill would be enacted and
initial appropriations provided by October 1, 2001.
Pay as you-go considerations: None.
Intergovernmental and private-sector impact: H.R. 717
contains no intergovernmental or private-sector mandates as
defined in UMRA. State, local, and tribal governments would be
eligible for grants authorized by the bill for research
activities associated with muscular dystrophy.
Estimate prepared by: Federal Costs: Christopher J.
Topoleski and Jeanne De Sa. Impact on State, Local, and Tribal
Governments: Leo Lex. Impact on the Private Sector: Amy
Fedigan.
Estimate approved by: Robert A. Sunshine, Assistant
Director for Budget Analysis.
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.
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
This Act may be cited as the ``Muscular Dystrophy Community
Assistance, Research, and Education Amendments of 2001,'' or
the ``MD-CARE Act.''
Section 2. Findings
Section 2 finds that: (1) Duchenne Muscular Dystrophy (DMD)
is the world's most common and catastrophic form of genetic
childhood disease; (2) DMD is genetically inherited, and
mothers are the carriers in approximately 70 percent of all
cases; (3) DMD affects 1 in 3,500 boys worldwide; (4) there are
other dystrophies which also have a devastating impact on the
quality of life for patients, including Myotonic muscular
dystrophy and Facioscapulohumeral muscular dystrophy; (5) there
is a shortage of qualified researchers in the field of
neuromuscular research; (6) development of therapies for these
disorders is costly; and, (7) educating the public and health
care community is in the public interest and to the benefit of
all communities.
Section 3. Expansion, intensification, and coordination of activities
of National Institutes of Health with respect to research on
muscular dystrophy
Section 3 directs the Director of the NIH to expand and
intensify activities with respect to various forms of muscular
dystrophy, and to allocate the amounts appropriated to carry
out this section.
In addition, this section directs the Director of NIH to
award grants and contracts to public or nonprofit private
entities to pay all, or part of, the costs of planning,
establishing, improving, and providing support to centers of
excellence regarding research on various forms of muscular
dystrophy.
The Secretary of Health and Human Services is directed to
establish the Muscular Dystrophy Coordinating Committee to
coordinate activities across other Federal health programs
relating to the various forms of muscular dystrophy.
Section 4. Development and expansion of activities of Centers for
Disease Control and Prevention with respect to epidemiological
research on muscular dystrophy
Section 4 allows the Secretary, acting through the Director
of the Centers for Disease Control and Prevention, to award
grants and cooperative agreements to public or nonprofit
private entities for the collection, analysis, and reporting of
data on Duchenne and other forms of muscular dystrophy. This
section also allows the Secretary to award grants to public or
nonprofit private entities for the purpose of carrying out
epidemiological activities regarding muscular dystrophy.
Section 5. Information and education
Section 5 requires the Secretary of Health and Human
Services to establish and implement a program to provide
information and education on muscular dystrophy, including
information and education on advances in the diagnosis and
treatment of muscular dystrophy.
Section 6. Report to Congress
Section 6 requires that not later than January 1, 2003, and
each January 1 thereafter, the Secretary shall prepare and
submit to the appropriate committees of Congress, a report
concerning the implementation of this Act and the amendments
made by this Act.
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 III--GENERAL POWERS AND DUTIES OF PUBLIC HEALTH SERVICE
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Part B--Federal-State Cooperation
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SEC. 317Q. SURVEILLANCE AND RESEARCH REGARDING MUSCULAR DYSTROPHY.
(a) In General.--The Secretary, acting through the Director
of the Centers for Disease Control and Prevention, may award
grants and cooperative agreements to public or nonprofit
private entities (including health departments of States and
political subdivisions of States, and including universities
and other educational entities) for the collection, analysis,
and reporting of data on Duchenne and other forms of muscular
dystrophy. In making such awards, the Secretary may provide
direct technical assistance in lieu of cash.
(b) National Muscular Dystrophy Epidemiology Program.--The
Secretary, acting through the Director of the Centers for
Disease Control and Prevention, may award grants to public or
nonprofit private entities (including health departments of
States and political subdivisions of States, and including
universities and other educational entities) for the purpose of
carrying out epidemiological activities regarding Duchenne and
other forms of muscular dystrophies, including collecting and
analyzing information on the number, incidence, correlates, and
symptoms of cases. In carrying out the preceding sentence, the
Secretary shall provide for a national surveillance program. In
making awards under this subsection, the Secretary may provide
direct technical assistance in lieu of cash.
(c) Coordination With Centers of Excellence.--The Secretary
shall ensure that epidemiological information under subsections
(a) and (b) is made available to centers of excellence
supported under section 404E(b) by the Director of the National
Institutes of Health.
(d) Authorization of Appropriations.--There are authorized to
be appropriated such sums as may be necessary to carry out this
section.
TITLE IV--NATIONAL RESEARCH INSTITUTES
Part A--National Institutes of Health
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SEC. 404E. MUSCULAR DYSTROPHY; INITIATIVE THROUGH DIRECTOR OF NATIONAL
INSTITUTES OF HEALTH.
(a) Expansion, Intensification, and Coordination of
Activities.--
(1) In general.--The Director of NIH, in coordination
with the Directors of the National Institute of
Neurological Disorders and Stroke, the National
Institute of Arthritis and Muscoskeletal and Skin
Diseases, the National Institute of Child Health and
Human Development, and the other national research
institutes as appropriate, shall expand and intensify
programs of such Institutes with respect to research
and related activities concerning various forms of
muscular dystrophy, including Duchenne, myotonic,
facioscapulohumeral muscular dystrophy (referred to in
this section as ``FSHD'') and other forms of muscular
dystrophy.
(2) Coordination.--The Directors referred to in
paragraph (1) shall jointly coordinate the programs
referred to in such paragraph and consult with the
Muscular Dystrophy Interagency Coordinating Committee
established under section 6 of the MD-CARE Act.
(3) Allocations by director of nih.--The Director of
NIH shall allocate the amounts appropriated to carry
out this section for each fiscal year among the
national research institutes referred to in paragraph
(1).
(b) Centers of Excellence.--
(1) In general.--The Director of NIH shall award
grants and contracts under subsection (a)(1) to public
or nonprofit private entities to pay all or part of the
cost of planning, establishing, improving, and
providing basic operating support for centers of
excellence regarding research on various forms of
muscular dystrophy.
(2) Research.--Each center under paragraph (1) shall
supplement but not replace the establishment of a
comprehensive research portfolio in all the muscular
dystrophies. As a whole, the centers shall conduct
basic and clinical research in all forms of muscular
dystrophy including early detection, diagnosis,
prevention, and treatment, including the fields of
muscle biology, genetics, noninvasive imaging,
genetics, pharmacological and other therapies.
(3) Coordination of centers; reports.--The Director
of NIH--
(A) shall, as appropriate, provide for the
coordination of information among centers under
paragraph (1) and ensure regular communication
between such centers; and
(B) shall require the periodic preparation of
reports on the activities of the centers and
the submission of the reports to the Director.
(4) Organization of centers.--Each center under
paragraph (1) shall use the facilities of a single
institution, or be formed from a consortium of
cooperating institutions, meeting such requirements as
may be prescribed by the Director of NIH.
(5) Duration of support.--Support for a center
established under paragraph (1) may be provided under
this section for a period of not to exceed 5 years.
Such period may be extended for 1 or more additional
periods not exceeding 5 years if the operations of such
center have been reviewed by an appropriate technical
and scientific peer review group established by the
Director of NIH and if such group has recommended to
the Director that such period should be extended.
(c) Facilitation of Research.--The Director of NIH shall
provide for a program under subsection (a)(1) under which
samples of tissues and genetic materials that are of use in
research on muscular dystrophy are donated, collected,
preserved, and made available for such research. The program
shall be carried out in accordance with accepted scientific and
medical standards for the donation, collection, and
preservation of such samples.
(d) Coordinating Committee.--
(1) In general.--The Secretary shall establish the
Muscular Dystrophy Coordinating Committee (referred to
in this section as the ``Coordinating Committee'') to
coordinate activities across the National Institutes
and with other Federal health programs and activities
relating to the various forms of muscular dystrophy.
(2) Composition.--The Coordinating Committee shall
consist of not more than 15 members to be appointed by
the Secretary, of which--
(A) \2/3\ of such members shall represent
governmental agencies, including the directors
or their designees of each of the national
research institutes involved in research with
respect to muscular dystrophy and
representatives of all other Federal
departments and agencies whose programs involve
health functions or responsibilities relevant
to such diseases, including the Centers for
Disease Control and Prevention, the Health
Resources and Services Administration and the
Food and Drug Administration and
representatives of other governmental agencies
that serve children with muscular dystrophy,
such as the Department of Education; and
(B) \1/3\ of such members shall be public
members, including a broad cross section of
persons affected with muscular dystrophies
including parents or legal guardians, affected
individuals, researchers, and clinicians.
Members appointed under subparagraph (B) shall serve
for a term of 3 years, and may serve for an unlimited
number of terms if reappointed.
(3) Chair.--
(A) In general.--With respect to muscular
dystrophy, the Chair of the Coordinating
Committee shall serve as the principal advisor
to the Secretary, the Assistant Secretary for
Health, and the Director of NIH, and shall
provide advice to the Director of the Centers
for Disease Control and Prevention, the
Commissioner of Food and Drugs, and to the
heads of other relevant agencies. The
Coordinating Committee shall select the Chair
for a term not to exceed 2 years.
(B) Appointment.--The Chair of the Committee
shall be appointed by and be directly
responsible to the Secretary.
(4) Administrative support; terms of service; other
provisions.--The following shall apply with respect to
the Coordinating Committee:
(A) The Coordinating Committee shall receive
necessary and appropriate administrative
support from the Department of Health and Human
Services.
(B) The Coordinating Committee shall meet as
appropriate as determined by the Secretary, in
consultation with the chair.
(e) Plan for HHS Activities.--
(1) In general.--Not later than 1 year after the date
of enactment of this section, the Coordinating
Committee shall develop a plan for conducting and
supporting research and education on muscular dystrophy
through the national research institutes and shall
periodically review and revise the plan. The plan
shall--
(A) provide for a broad range of research and
education activities relating to biomedical,
epidemiological, psychosocial, and
rehabilitative issues, including studies of the
impact of such diseases in rural and
underserved communities;
(B) identify priorities among the programs
and activities of the National Institutes of
Health regarding such diseases; and
(C) reflect input from a broad range of
scientists, patients, and advocacy groups.
(2) Certain elements of plan.--The plan under
paragraph (1) shall, with respect to each form of
muscular dystrophy, provide for the following as
appropriate:
(A) Research to determine the reasons
underlying the incidence and prevalence of
various forms of muscular dystrophy.
(B) Basic research concerning the etiology
and genetic links of the disease and potential
causes of mutations.
(C) The development of improved screening
techniques.
(D) Basic and clinical research for the
development and evaluation of new treatments,
including new biological agents.
(E) Information and education programs for
health care professionals and the public.
(f) Reports to Congress.--The Coordinating Committee shall
biennially submit to the Committee on Energy and Commerce of
the House of Representatives, and the Committee on Health,
Education, Labor, and Pensions of the Senate, a report that
describes the research, education, and other activities on
muscular dystrophy being conducted or supported through the
Department of Health and Human Services. Each such report shall
include the following:
(1) The plan under subsection (e)(1) (or revisions to
the plan, as the case may be).
(2) Provisions specifying the amounts expended by the
Department of Health and Human Services with respect to
various forms of muscular dystrophy, including
Duchenne, myotonic, FSHD and other forms of muscular
dystrophy.
(3) Provisions identifying particular projects or
types of projects that should in the future be
considered by the national research institutes or other
entities in the field of research on all muscular
dystrophies.
(g) Public Input.--The Secretary shall, under subsection
(a)(1), provide for a means through which the public can obtain
information on the existing and planned programs and activities
of the Department of Health and Human Services with respect to
various forms of muscular dystrophy and through which the
Secretary can receive comments from the public regarding such
programs and activities.
(h) Authorization of Appropriations.--For the purpose of
carrying out this section, there are authorized to be
appropriated such sums as may be necessary for each of fiscal
years 2002 through 2006. The authorization of appropriations
established in the preceding sentence is in addition to any
other authorization of appropriations that is available for
conducting or supporting through the National Institutes of
Health research and other activities with respect to muscular
dystrophy.
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