[Congressional Bills 110th Congress]
[From the U.S. Government Publishing Office]
[H.R. 5265 Introduced in House (IH)]
110th CONGRESS
2d Session
H. R. 5265
To amend the Public Health Service Act to provide for research with
respect to various forms of muscular dystrophy, including Becker,
congenital, distal, Duchenne, Emery-Dreifuss facioscapulohumeral, limb-
girdle, myotonic, and oculopharyngeal, muscular dystrophies.
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
February 7, 2008
Mr. Engel (for himself and Mr. Burgess) 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 with
respect to various forms of muscular dystrophy, including Becker,
congenital, distal, Duchenne, Emery-Dreifuss facioscapulohumeral, limb-
girdle, myotonic, and oculopharyngeal, 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 ``Paul D. Wellstone Muscular Dystrophy
Community Assistance, Research, and Education Amendments of 2008''.
SEC. 2. FINDINGS.
The Congress finds as follows:
(1) The muscular dystrophies are devastating diseases that
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.
(2) DMD is the most common lethal genetic disorder of
childhood worldwide, affecting approximately 1 in every 3,500
boys born each year around the globe. It is characterized by a
rapidly progressive muscle weakness that almost always results
in death from respiratory or cardiac failure, typically in the
late teens or twenties.
(3) Myotonic muscular dystrophy is the second most
prominent form of muscular dystrophy and the type most commonly
found in adults affecting an estimated 1 in 8,000 people.
However, it can affect people of any age--from birth to old
age. Described as the most variable disease known in medicine,
it is multi-systemic and can cause not only muscle atrophy and
myotonia, but also serious cardiac, respiratory, endocrine,
gastrointestinal, skeletal and central nervous system
complications, as well as problems with the eyes, teeth and
hair. As it passes from one generation to the next, it
generally worsens with earlier onset. Congenital myotonic
muscular dystrophy is the most severe form of myotonic muscular
dystrophy affecting infants and causing severe cognitive
delays. It often causes sudden death; however, others can live
for many years with this slowly degenerative disorder.
(4) Facioscapulohumeral muscular dystrophy (referred to in
this section as ``FSHD'') is the second most prevalent adult
muscular dystrophy and the third most prevalent muscular
dystrophy of men, women and children. It is inherited
genetically and has an estimated incidence of 1 in 20,000
persons. Many leading FSHD scientists note that the prevalence
may be three times higher due to undiagnosed and misdiagnosed
cases. FSHD, affecting between 15,000 to 40,000 persons, causes
a lifelong progressive and severe loss of all skeletal muscles
gradually bringing weakness and reduced mobility. It is
genetically transmitted to children, can occur spontaneously,
and may affect entire families. Persons with FSHD may also
experience hearing loss, vision problems and respiratory
insufficiency; some may become severely physically disabled and
spend decades in a wheelchair and on a ventilator. FSHD is
caused by a novel epigenetic phenomenon not found in other
forms of muscular dystrophy and is caused by a contraction of
repetitive DNA previously thought to be ``junk DNA''. The
unique epigenetic structure of FSHD is unprecedented in other
muscular dystrophies and genetic disorders and demands novel
approaches and new research groups. Understanding this
mechanism will have great benefit to other areas of biomedical
research including cancer and other disease of epigenetic
origin.
(5) Congenital muscular dystrophies represent a group of
distinct diseases, which begin at birth, with varying severity
and involvement of both muscle strength and brain. These
diseases often lead to premature infant death, or severely
disabled young children who require 24-hour care given their
developmental delay compounded by muscle weakness. Other
children live to young adulthood and typically require the use
of a wheelchair for mobility.
(6) Forms of muscular dystrophy affecting children and
adults include Becker, congenital, distal, Duchenne, Emery-
Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and
oculopharyngeal muscular dystrophies. The limb-girdle muscular
dystrophies are of 15 known different types.
(7) Each of the muscular dystrophies, though distinct in
progressivity and severity of symptoms, has a devastating
impact on hundreds of thousands of children and adults
throughout the United States and worldwide, as well as imposes
severe physical and economic burdens on those affected. In many
of the muscular dystrophies, there are associated medical
problems arising from pulmonary issues, respiratory
insufficiency, cardiomyopathy, which in many cases is the cause
of death for persons with muscular dystrophy.
(8) In the 5 years since enactment of the Muscular
Dystrophy Community Assistance, Research and Education
Amendments of 2001 (MD-CARE Act) and due directly to the
momentum established by the MD-CARE Act, progress has been made
in the battle against the Muscular Dystrophies.
(9) Investments made by the Federal Government as a result
of the MD-CARE Act include the creation of the MD Coordinating
Committee (MDCC), the development of the MDCC Action Plan,
expansion of the NIH research portfolios, establishment of 6
Paul D. Wellstone Muscular Dystrophy Cooperative Research
Centers (co-funded, in part, by a national non-profit health
organization), development of the Muscular Dystrophy
Surveillance, Tracking and Research Network (MD STARnet), and
the launch of a comprehensive education and outreach
initiative.
(10) In the past few years, the NIH program in
translational research in muscular dystrophy has grown
significantly and funded a number of large-scale projects to
further the development of therapies for muscular dystrophy. As
part of this program, the National Institute of Neurological
Disorders and Stroke (NINDS) and the National Institute of
Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the
National Institutes of Health (NIH) awarded a $15.4 million,
five-year cooperative agreement to develop new small molecule
drugs for the treatment of Duchenne muscular dystrophy (DMD)
and potentially other forms of muscular dystrophy as well. The
project is a unique research collaboration between private,
public, and non-profit partners to build upon previous research
and discovery work originally initiated by non-profit partners
to identify new treatments for muscular dystrophy. Also through
the translational program, three other major cooperative
agreements have been awarded for highly targeted therapy
development projects in the muscular dystrophies.
(11) Due to the initiatives made possible through the MD-
CARE Act, national non-profit organizations have joined in
model strategic collaborations with academic research
institutions, public funders of MD research, and industry to
expand investments in MD research activities and to create new
platforms for translational research. These have led to the
development of the first potential therapies for DMD, myotonic,
facioscapulohumeral, limb-girdle and other conditions that are
proceeding through clinical trials.
(12) Advancements in care have helped prolong life and
quality of life for patients with muscular dystrophy.
(13) Notwithstanding these promising developments, the
majority of the directions envisioned by the Action Plan for
the Muscular Dystrophies, developed pursuant to the MD-CARE
Act, have not been realized. Where recent momentum has been
achieved, its sustainability is fragile and directly dependent
upon continued Federal support for the early phase planning and
programs created through the MD-CARE Act.
(14) There remains a shortage of qualified researchers in
the field of muscular dystrophy research. Many family
physicians and health care professionals still lack the
knowledge and resources to detect and properly diagnose
muscular dystrophy as early as possible, thus delaying
management of symptoms in cases that go undetected or
misdiagnosed.
(15) As new understandings of the genetic basis for disease
and potential treatment has emerged, the public and health care
communities are in urgent need of education and outreach to
ensure competent, informed engagement in genetic testing and
counseling and appropriate patient characterization so that
patients are able to participate in new avenues of research and
clinical trials.
(16) As basic research into the muscular dystrophies points
the way to new therapeutic targets, there is an urgent need to
support the clinical research infrastructure necessary to bring
these therapeutic leads to human trials; these infrastructure
needs include validated endpoints, current natural history
studies, biomarkers, clinical research networks, patient
registries and databases.
(17) In order to improve lives and develop effective
treatments for individuals with muscular dystrophy, there must
be improved communications and partnerships between patients,
patient advocacy, researchers, and clinical care providers. To
that end, renewed effort to work together by all parties is a
critical element for successful outcomes in the years to come.
(18) Continued focus and investment are required to build
on the current momentum, respond to public need, and ensure
that federally funded research and other innovation is
translated to therapeutic targets as quickly as possible.
SEC. 3. EXPANSION, INTENSIFICATION, AND COORDINATION OF ACTIVITIES OF
NIH WITH RESPECT TO RESEARCH ON MUSCULAR DYSTROPHY.
(a) Technical Correction.--Section 404E of the Public Health
Service Act (42 U.S.C. 283g) is amended by striking subsection (f)
(relating to reports to Congress) and redesignating subsection (g) as
subsection (f).
(b) Amendments.--Section 404E of the Public Health Service Act (42
U.S.C. 283g) is amended--
(1) in subsection (a)(1), by inserting ``the National
Heart, Lung, and Blood Institute,'' after ``the Eunice Kennedy
Shriver National Institute of Child Health and Human
Development,'';
(2) in subsection (b)(1), by adding at the end of the
following: ``Such centers of excellence shall be known as the
`Paul D. Wellstone Muscular Dystrophy Cooperative Research
Centers'.''; and
(3) by adding at the end the following:
``(g) Clinical Research.--The Coordinating Committee shall give
special consideration to the urgent need to enhance the clinical
research infrastructure required to test emerging therapies for the
various forms of muscular dystrophy by prioritizing the achievement of
the goals related to this topic in the plan under subsection (e)(1).
``(h) Authorization of Appropriations.--There are authorized to be
appropriated to carry out this section such sums as may be necessary
for each of fiscal years 2008 through 2012.''.
SEC. 4. DEVELOPMENT AND EXPANSION OF ACTIVITIES OF CDC WITH RESPECT TO
EPIDEMIOLOGICAL RESEARCH ON MUSCULAR DYSTROPHY.
Section 317Q of the Public Health Service Act (42 U.S.C. 247b-18)
is amended--
(1) by redesignating subsection (d) as subsection (f); and
(2) by inserting after subsection (c) the following:
``(d) Data.--In carrying out this section, the Secretary shall
ensure that any data on patients that is collected as part of the
Muscular Dystrophy STARnet (under a grant under this section) is
regularly updated to reflect changes in patient condition over time,
particularly with respect to any improvements realized through patient
adherence to care considerations or utilization of a treatment or
therapy.
``(e) Reports and Study.--
``(1) Annual report.--Not later than 18 months after the
date of the enactment of the Paul D. Wellstone Muscular
Dystrophy Community Assistance, Research, and Education
Amendments of 2008, and annually thereafter, the Director of
the Centers for Disease Control and Prevention shall submit to
the appropriate committees of the Congress a report--
``(A) concerning the activities carried out by MD
STARnet site funded under this section during the year
for which the report is prepared;
``(B) containing the data collected and findings
derived from the MD STARnet sites each fiscal year (as
funded under a grant under this section during fiscal
years 2008 through 2012); and
``(C) that every 2 years outlines prospective data
collection objectives and strategies.
``(2) Tracking health outcomes.--The Director of the
Centers for Disease Control and Prevention shall provide
prospective health outcome data on the health and survival of
people with muscular dystrophy.''.
SEC. 5. INFORMATION AND EDUCATION.
Section 5 of the Muscular Dystrophy Community Assistance, Research
and Education Amendments of 2001 (42 U.S.C. 247b-19) is amended--
(1) by redesignating subsection (c) as subsection (d); and
(2) by inserting after subsection (b) the following:
``(c) Requirements of CDC.--In carrying out this section, the
Director of the Centers for Disease Control and Prevention shall--
``(1) partner with leaders in the muscular dystrophy
patient community; and
``(2) widely disseminate the Duchenne-Becker muscular
dystrophy care considerations as broadly as possible, including
through partnership opportunities with the muscular dystrophy
patient community.''.
SEC. 6. STANDARDS OF CARE.
Part A of title IX of the Public Health Service Act (42 U.S.C. 299
et seq.) is amended by adding at the end the following:
``SEC. 904. STANDARDS OF CARE RELATING TO MUSCULAR DYSTROPHY.
``The Director shall--
``(1) evaluate the available scientific evidence for the
appropriate medical or patient organizations for purposes of
the development and issuance of an initial set of care
considerations for Duchenne-Becker muscular dystrophy and
provide ongoing review and updates where appropriate; and
``(2) replicate the same methodology used to develop the
Duchenne-Becker muscular dystrophy care considerations
developed under paragraph (1) as a model for other muscular
dystrophies.''.
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