Requires the Director to: (1) award grants and contracts for establishing at least five related centers of excellence; and (2) provide for a related tissue donation program.
Requires the Secretary of Health and Human Services to establish the Muscular Dystrophy Coordinating Committee to coordinate NIH programs and other Federal programs.
Authorizes the Secretary, through the Centers for Disease Control and Prevention, to: (1) award grants and cooperative agreements for muscular dystrophy data collection and analysis; and (2) conduct a National Muscular Dystrophy Surveillance Program. Requires the Secretary, through the Centers, to establish at least three centers of excellence in muscular dystrophy epidemiology.
Requires the Secretary to establish a program to provide professionals and the public with muscular dystrophy information.
[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[S. 805 Introduced in Senate (IS)]
107th CONGRESS
1st Session
S. 805
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.
_______________________________________________________________________
IN THE SENATE OF THE UNITED STATES
May 1, 2001
Mr. Wellstone (for himself, Mr. Cochran, Ms. Collins, Mr. Bennett, Mr.
Breaux, Mr. Bunning, Mrs. Clinton, Mr. Corzine, Mr. Daschle, Mr.
Dayton, Mr. Dorgan, Mr. Hutchinson, Mr. Johnson, Mr. Kerry, Mr. Kohl,
Ms. Mikulski, Mr. Sarbanes, Mr. Schumer, Ms. Snowe, Ms. Stabenow, and
Mr. Voinovich) 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 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.
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 ``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 Institutes
of Health 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) Number of centers; duration of support.--
``(A) In general.--The Director of NIH shall
provide for the establishment of not less than 5
centers under paragraph (1).
``(B) Duration.--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 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 Surveillance 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 conduct of a National Muscular Dystrophy
Surveillance Program. In making such awards, the Secretary may provide
direct technical assistance in lieu of cash.
``(c) Centers of Excellence in Muscular Dystrophy Epidemiology.--
``(1) In general.--The Secretary, acting through the
Director of the Centers for Disease Control and Prevention,
shall establish not less than 3 regional centers of excellence
in muscular dystrophy epidemiology for the purpose of
collecting and analyzing information on the number,
incidence, correlates, and symptoms of Duchenne and other forms of
muscular dystrophies.
``(2) Recipients of awards for establishment of centers.--
Centers under paragraph (1) shall be established and operated
through the awarding of grants or 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) that
conduct research.
``(3) Certain requirements.--An award for a center under
paragraph (1) may be made only if the entity involved submits
to the Secretary an application containing such agreements and
information as the Secretary may require, including an
agreement that the center involved will operate in accordance
with the following:
``(A) The center will collect, analyze, and report
muscular dystrophy data according to guidelines
prescribed by the Director, after consultation with
relevant State and local public health officials,
private sector researchers, and advocates for those
with muscular dystrophy.
``(B) The center will assist with the development
and coordination of State and related muscular
dystrophy surveillance efforts within a region.
``(C) The center will identify eligible cases and
controls through its surveillance systems and conduct
research into factors which may cause muscular
dystrophy.
``(D) The center will develop or extend an area of
special research expertise (including genetics,
immunology, and other relevant research specialty
areas).
``(d) Definition.--In this title, the term `State' means each of
the several States, the District of Columbia, the Commonwealth of
Puerto Rico, American Samoa, Guam, the Commonwealth of the Northern
Mariana Islands, the United States Virgin Islands, and the Trust
Territory of the Pacific Islands.
``(e) 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 title and the
amendments made by this Act.
<all>
Introduced in Senate
Sponsor introductory remarks on measure. (CR S4115-4116)
Read twice and referred to the Committee on Health, Education, Labor, and Pensions.
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