[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[H.R. 717 Introduced in House (IH)]
107th CONGRESS
1st Session
H. R. 717
To amend the Public Health Service Act to provide for research and
services with respect to Duchenne muscular dystrophy.
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
February 14, 2001
Mr. Wicker (for himself, Mr. Peterson of Minnesota, Mr. Greenwood, Mr.
Tanner, Mr. Ehrlich, Mr. Cramer, Mr. Gordon, Mrs. Emerson, Mr. Riley,
Mr. Bryant, Mr. Ford, Mr. Foley, Ms. Hooley of Oregon, Mr. King, Mr.
Hobson, Mr. Pickering, Mr. Chambliss, Mr. Ehlers, Mr. Towns, Mr.
McGovern, Mr. LaTourette, Mr. Doolittle, Mr. Watts of Oklahoma, Ms.
Granger, Mr. Blumenauer, Mr. Murtha, Mr. Olver, Mr. Boehlert, Mr.
Goodlatte, Mr. Holden, Mr. Watkins, Mr. Coble, Mr. Isakson, Mr.
LoBiondo, Mr. McCrery, Mr. Kerns, Mr. Gilman, Mr. Rohrabacher, Mr.
Issa, Mr. Calvert, Mr. Langevin, Mrs. Meek of Florida, Mr. Hastings of
Florida, Ms. Brown of Florida, Mr. Miller of Florida, Mr. Otter, Mr.
Walden of Oregon, Mrs. Myrick, Mr. LaHood, Mr. Lipinski, Mr. Lewis of
Kentucky, Mr. Wolf, Mr. Hostettler, Mr. Kingston, Mr. Scarborough, Mr.
Upton, Mr. Leach, Mr. Gillmor, Mr. Walsh, Mr. Quinn, Mr. Ganske, Mr.
Jones of North Carolina, Mr. Bachus, Mr. Oxley, Mr. Tiahrt, Mr. Weller,
Mr. Matsui, Mr. Weldon of Florida, Mr. Reynolds, Mr. Gutknecht, Mr.
Chabot, Mr. Hunter, Mr. Goode, Mr. Fletcher, Mr. Skelton, Mr. Moran of
Virginia, Mr. Rodriguez, Mr. Turner, Mr. Bentsen, Mr. Abercrombie, Mr.
Gonzalez, Mr. Bilirakis, Mr. Armey, Mr. McHugh, Mr. Jenkins, Mr. Boyd,
Mr. Putnam, Mr. Rogers of Michigan, Mr. Keller, Mrs. Kelly, and Mr.
Manzullo) introduced the following bill; which was referred to the
Committee on Energy and Commerce
_______________________________________________________________________
A BILL
To amend the Public Health Service Act to provide for research and
services with respect to Duchenne muscular dystrophy.
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 ``Duchenne Muscular Dystrophy
Childhood Assistance, Research and Education Amendments of 2001'', or
the ``DMD CARE Act''.
SEC. 2. FINDINGS.
The Congress finds as follows:
(1) Duchenne is the most common form of muscular dystrophy,
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) Children with Duchenne 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.
(5) Duchenne is the most common lethal genetic disorder of
childhood worldwide, affecting approximately one in every 3,500
boys worldwide.
(6) Although the dystrophin gene that causes DMD was
successfully identified and isolated by medical researchers in
1987, Federal research devoted to potential treatment options
or a cure since this initial discovery has been minimal.
(7) Because of the limited Federal support for medical
research specific to this form of muscular dystrophy, current
treatment options are minimal in efficacy and palliative, aimed
at simply managing the symptoms in an effort to optimize the
quality of life.
(8) 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.
(9) This 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 caregivers.
(10) Currently there exists only a small quantity of public
information about Duchenne muscular dystrophy, and what little
information does exist remains inadequately disseminated and
insufficient in addressing the needs of specific diverse
populations and other underserved groups.
(11) 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
DUCHENNE MUSCULAR DYSTROPHY.
Subpart 4 of part C of title IV of the Public Health Service Act
(42 U.S.C. 285d et seq.) is amended by inserting after section 441A the
following section:
``duchenne muscular dystrophy; initiative through director of national
institutes of health
``Sec. 441B. (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, National Institute of Arthritis and
Muscoskeletal and Skin Diseases and the National Institute of
Child Health and Human Development, shall expand and intensify
programs of such Institutes with respect to research and related
activities concerning Duchenne muscular dystrophy.
``(2) Coordination.--The Directors referred to in
subsection (a) shall jointly coordinate the programs referred
to in such subsection and consult with the Muscular Dystrophy
Interagency Coordinating Committee.
``(3) Allocations by director of nih.--With respect to
amounts appropriated to carry out this section for a fiscal
year, the Director of NIH shall allocate the amounts among the
national research institutes that are referred to in subsection
(a).
``(b) Centers of Excellence.--
``(1) In general.--The Director shall under subsection
(a)(1) make awards of grants and contracts 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 Duchenne muscular dystrophy.
``(2) Research.--Each center under paragraph (1) shall
conduct basic and clinical research into Duchenne muscular
dystrophy. Such research should include investigations into the
diagnosis, early detection, prevention, control, and adequate
treatment of Duchenne muscular dystrophy. The centers, as a
group, shall conduct research including the fields of muscle
biology, genetics, non-invasive imaging, gene and
pharmaceutical therapies.
``(3) Coordination of centers; reports.--The Director
shall, as appropriate, provide for the coordination of
information among centers under paragraph (1) and ensure
regular communication between such centers, and may 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.
``(5) Number of centers; duration of support.--
``(A) In general.--The Director shall provide for
the establishment of not less than three 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 one 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 and if such group has recommended to the
Director that such period should be extended.
``(c) Facilitation of Research.--The Director shall under
subsection (a)(1) provide for a program under which samples of tissues
and genetic materials that are of use in research on Duchenne 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 such
diseases.
``(2) Composition.--The Coordinating Committee shall be
composed of the directors or their designees of each of the
national research institutes involved in research with respect
to Duchenne 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 Services and Resources Administration and the Food and
Drug Administration.
``(3) Chair.--
``(A) In general.--With respect to Duchenne
muscular dystrophy, the Chair of the 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 other relevant
agencies.
``(B) Director of nih.--The Chair of the Committee
shall be directly responsible to the Director of NIH.
``(e) Plan for NIH Activities.--
``(1) In general.--Not later than 1 year after the date of
the enactment of this section, the Coordinating Committee shall
develop a plan for conducting and supporting research and
education on Duchenne 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,
psychosocial, and rehabilitative issues, including
studies of the impact of such diseases on boys 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 Duchenne muscular dystrophy, provide
for the following as appropriate:
``(A) Research to determine the reasons underlying
the incidence and prevalence of Duchenne 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.
``(3) Implementation of plan.--The Director of NIH shall
ensure that programs and activities of the National Institutes
of Health regarding Duchenne muscular dystrophy are implemented
in accordance with the plan under paragraph (1).
``(f) Reports to Congress.--The Coordinating Committee under
subsection (b)(1) 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 Duchenne muscular
dystrophy being conducted or supported through the national research
institutes, and that in addition includes the following:
``(1) The plan under subsection (c)(1) (or revisions to the
plan, as the case may be).
``(2) Provisions specifying the amounts expended by the
National Institutes of Health with respect to Duchenne 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 Duchenne muscular dystrophy.
``(g) Public Input.--The Director shall under subsection (a)(1)
provide for means through which the public can obtain information on
the existing and planned programs and activities of the National
Institutes of Health with respect to Duchenne muscular dystrophy and
through which the Director 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 the fiscal years 2001 through 2005. 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 Duchenne
muscular dystrophy.''.
SEC. 4. DEVELOPMENT AND EXPANSION OF ACTIVITIES OF CENTERS FOR DISEASE
CONTROL AND PREVENTION WITH RESPECT TO EPIDEMIOLOGICAL
RESEARCH ON DUCHENNE 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
section:
``surveillance and research regarding duchenne muscular dystrophy
``Sec. 317Q. (a) In General.--The Secretary, acting through the
Director of the Centers for Disease Control and Prevention, may make
awards of grants and cooperative agreements for the collection,
analysis, and reporting of data on Duchenne muscular dystrophy. An
entity may receive such an award only if the entity is a public or
nonprofit private entity (including health departments of States and
subdivisions of States, and including universities and other
educational entities). In making such awards, the Secretary may provide
direct technical assistance in lieu of cash.
``(b) National Duchenne Muscular Dystrophy Surveillance Program.--
``(1) In general.--The Secretary of Health and Human
Services (in this section referred to as the `Secretary'),
acting through the Director of the Centers for Disease Control
and Prevention, may make awards of grants and cooperative
agreements for the collection, analysis, and reporting of data
on Duchenne muscular dystrophy. In making such awards, the
Secretary may provide direct technical assistance in lieu of
cash.
``(2) Eligibility.--To be eligible to receive an award
under paragraph (1) an entity shall be a public or nonprofit
private entity (including health departments of States and
political subdivisions of States, and including universities
and other educational entities).
``(c) Centers of Excellence in Duchenne and Related Muscular
Dystrophies Epidemiology.--
``(1) In general.--The Secretary, acting through the
Director of the Centers for Disease Control and Prevention,
shall establish not less than three regional centers of
excellence in Duchenne muscular dystrophy epidemiology for the
purpose of collecting and analyzing information on the number,
incidence, correlates, and symptoms of Duchenne and related
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 that conduct research,
including health departments of States and political
subdivisions of States, and including universities and other
educational entities.
``(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
Duchenne 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 Duchenne muscular dystrophy.
``(B) The center will assist with the development
and coordination of State Duchenne 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 Duchenne 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 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 shall establish and implement a
program to provide information and education on Duchenne muscular
dystrophy to health professionals and the general public, including
information and education on advances in the diagnosis and treatment of
Duchenne muscular dystrophy and training and continuing education
through programs for scientists, physicians, and other health
professionals who provide care for patients with Duchenne muscular
dystrophy.
(b) Stipends.--The Secretary may use amounts made available under
this section to provide 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. INTER-AGENCY MUSCULAR DYSTROPHY COORDINATING COMMITTEE.
(a) Establishment.--The Secretary shall establish a committee to be
known as the ``Muscular Dystrophy Coordinating Committee'' (in this
section referred to as the ``Committee'') to coordinate all efforts
within the Department of Health and Human Services concerning Duchenne
muscular dystrophy, including activities carried out through the
National Institutes of Health and the Centers for Disease Control and
Prevention under this Act (and the amendments made by this Act).
(b) Membership.--
(1) In general.--The Committee shall be composed of the
Directors of such national research institutes, of the Centers
for Disease Control and Prevention, and of such other agencies
and such other officials as the Secretary determines
appropriate.
(2) Additional members.--If determined appropriate by the
Secretary, the Secretary may appoint to the Committee--
(A) parents or legal guardians of individuals with
Duchenne muscular dystrophy; and
(B) representatives of other governmental agencies
that serve children with Duchenne muscular dystrophy
such as the Department of Education.
(c) Administrative Support; Terms of Service; Other Provisions.--
The following shall apply with respect to the Committee:
(1) The Committee shall receive necessary and appropriate
administrative support from the Department of Health and Human
Services.
(2) Members of the Committee appointed under subsection
(b)(2)(A) shall serve for a term of 3 years, and may serve for
an unlimited number of terms if reappointed.
(3) The Committee shall meet not less than two times each
year.
SEC. 7. 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 title.
SEC. 8. DEFINITION.
For purposes of this Act, the term ``Secretary'' means the
Secretary of Health and Human Services.
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