Text: H.R.717 — 107th Congress (2001-2002)All Information (Except Text)
Public Law No: 107-84 (12/18/2001)
[107th Congress Public Law 84]
[From the U.S. Government Printing Office]
[[Page 115 STAT. 823]]
Public Law 107-84
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. <<NOTE: Dec. 18, 2001 - [H.R. 717]>>
Be it enacted by the Senate and House of Representatives of the
United States of America in Congress assembled, <<NOTE: Muscular
Dystrophy Community Assistance, Research and Education Amendments of
SECTION 1. SHORT TITLE. <<NOTE: 42 USC 201 note.>>
This Act may be cited as the ``Muscular Dystrophy Community
Assistance, Research and Education Amendments of 2001'', or the ``MD-
SEC. 2. FINDINGS. <<NOTE: 42 USC 247b-18 note.>>
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
(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
(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.
[[Page 115 STAT. 824]]
(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
(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
(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
(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
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. <<NOTE: 42 USC 283g.>> 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
[[Page 115 STAT. 825]]
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-
``(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 <<NOTE: Grants. Contracts.>> 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
``(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
``(d) Coordinating Committee.--
[[Page 115 STAT. 826]]
``(1) In <<NOTE: Establishment.>> 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
``(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;
``(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
Members appointed under subparagraph (B) shall serve for a term
of 3 years, and may serve for an unlimited number of terms if
``(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
``(4) Administrative support; terms of service; other
provisions.--The <<NOTE: Applicability.>> 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 <<NOTE: Deadline.>> 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--
[[Page 115 STAT. 827]]
``(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
``(B) Basic research concerning the etiology and
genetic links of the disease and potential causes of
``(C) The development of improved screening
``(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
[[Page 115 STAT. 828]]
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. <<NOTE: 42 USC 247b-18.>> 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. <<NOTE: 42 USC 247b-19.>>
(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.
[[Page 115 STAT. 829]]
SEC. 6. REPORT TO CONGRESS. <<NOTE: 42 USC 247b-18 note.>>
Not <<NOTE: Deadline.>> 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.
SEC. 7. <<NOTE: 42 USC 281 note.>> STUDY ON THE USE OF CENTERS OF
EXCELLENCE AT THE NATIONAL INSTITUTES OF HEALTH.
(a) Review.--Not <<NOTE: Deadline. Contracts.>> later than 60 days
after the date of enactment of this Act, the Secretary of Health and
Human Services shall enter into a contract with the Institute of
Medicine for the purpose of conducting a study and making
recommendations on the impact of, need for, and other issues associated
with Centers of Excellence at the National Institutes of Health.
(b) Areas of Review.--In conducting the study under subsection (a),
the Institute of Medicine shall at a minimum consider the following:
(1) The current areas of research incorporating Centers of
Excellence (which shall include a description of such areas) and
the relationship of this form of funding mechanism to other
forms of funding for research grants, including investigator
initiated research, contracts and other types of research
(2) The distinctive aspects of Centers of Excellence,
including the additional knowledge that may be expected to be
gained through Centers of Excellence as compared to other forms
of grant or contract mechanisms.
(3) The costs associated with establishing and maintaining
Centers of Excellence, and the record of scholarship and
training resulting from such Centers. The research and training
contributions of Centers should be assessed on their own merits
and in comparison with other forms of research support.
(4) Specific areas of research in which Centers of
Excellence may be useful, needed, or underused, as well as areas
of research in which Centers of Excellence may not be helpful.
(5) Criteria that may be applied in determining when Centers
of Excellence are an appropriate and cost-effective research
investment and conditions that should be present in order to
consider the establishment of Centers of Excellence.
(6) Alternative research models that may accomplish results
similar to or greater than Centers of Excellence.
(c) Report.--Not <<NOTE: Deadline.>> later than 1 year after the
date on which the contract is entered into under subsection (a), the
Institute of Medicine shall complete the study under such subsection and
submit a report to the Secretary of Health and Human Services
[[Page 115 STAT. 830]]
and the appropriate committees of Congress that contains the results of
Approved December 18, 2001.
LEGISLATIVE HISTORY--H.R. 717:
HOUSE REPORTS: No. 107-195 (Comm. on Energy and Commerce).
CONGRESSIONAL RECORD, Vol. 147 (2001):
Sept. 24, considered and passed House.
Nov. 15, considered and passed Senate, amended.
Nov. 29, House concurred in Senate amendment.