[House Report 113-556]
[From the U.S. Government Publishing Office]
113th Congress Report
HOUSE OF REPRESENTATIVES
2d Session 113-556
======================================================================
PAUL D. WELLSTONE MUSCULAR DYSTROPHY COMMUNITY ASSISTANCE, RESEARCH AND
EDUCATION AMENDMENTS OF 2014
_______
July 24, 2014.--Committed to the Committee of the Whole House on the
State of the Union and ordered to be printed
_______
Mr. Upton, from the Committee on Energy and Commerce,
submitted the following
R E P O R T
[To accompany H.R. 594]
[Including cost estimate of the Congressional Budget Office]
The Committee on Energy and Commerce, to whom was referred
the bill (H.R. 594) to reauthorize and extend the Paul D.
Wellstone Muscular Dystrophy Community Assistance, Research,
and Education Amendments of 2008, having considered the same,
report favorably thereon with an amendment and recommend that
the bill as amended do pass.
CONTENTS
Page
Purpose and Summary.............................................. 3
Background and Need for Legislation.............................. 3
Hearings......................................................... 3
Committee Consideration.......................................... 4
Committee Votes.................................................. 4
Committee Oversight Findings..................................... 4
Statement of General Performance Goals and Objectives............ 4
New Budget Authority, Entitlement Authority, and Tax Expenditures 4
Earmark, Limited Tax Benefits, and Limited Tariff Benefits....... 4
Committee Cost Estimate.......................................... 4
Congressional Budget Office Estimate............................. 4
Federal Mandates Statement....................................... 6
Duplication of Federal Programs.................................. 6
Disclosure of Directed Rule Makings.............................. 6
Advisory Committee Statement..................................... 6
Applicability to Legislative Branch.............................. 6
Section-by-Section Analysis of the Legislation................... 7
Changes in Existing Law Made by the Bill, as Reported............ 7
The amendment is as follows:
Strike all after the enacting clause and insert the
following:
SECTION 1. SHORT TITLE.
This Act may be cited as the ``Paul D. Wellstone Muscular Dystrophy
Community Assistance, Research and Education Amendments of 2014''.
SEC. 2. INITIATIVE THROUGH THE DIRECTOR OF THE NATIONAL INSTITUTES OF
HEALTH.
Section 404E of the Public Health Service Act (42 U.S.C. 283g) is
amended--
(1) in subsection (a)(1)--
(A) by striking ``Muscoskeletal'' and inserting
``Musculoskeletal''; and
(B) by inserting ``Becker, congenital muscular
dystrophy, limb-girdle muscular dystrophy,'' after
``Duchenne,'';
(2) in subsection (b)--
(A) in paragraph (2)--
(i) by striking ``genetics,'' at the second
place it appears; and
(ii) by inserting ``cardiac and pulmonary
function, and'' after ``imaging,''; and
(B) in paragraph (3), by inserting ``and sharing of
data'' after ``regular communication'';
(3) in subsection (d)--
(A) in paragraph (2)--
(i) in the matter preceding subparagraph (A),
by striking ``15'' and inserting ``18''; and
(ii) in subparagraph (A)--
(I) by striking ``and the Food and
Drug Administration'' and inserting ``,
the Food and Drug Administration, and
the Administration for Community
Living'';
(II) by inserting ``and adults''
after ``children''; and
(III) by striking ``such as the
Department of Education'' and inserting
``including the Department of Education
and the Social Security
Administration''; and
(B) in paragraph (4)(B), by inserting ``, but shall
meet no fewer than two times per calendar year'' before
the period; and
(4) in subsection (e)--
(A) in paragraph (1)--
(i) in the matter preceding subparagraph (A),
by striking ``through the national research
institutes'' and inserting ``through the
agencies represented on the Coordinating
Committee pursuant to subsection (d)(2)(A)'';
and
(ii) in subparagraph (A)--
(I) by inserting ``public services,''
before ``and rehabilitative issues'';
and
(II) by inserting ``, studies to
demonstrate the cost-effectiveness of
providing independent living resources
and support to patients with various
forms of muscular dystrophy, and
studies to determine optimal clinical
care interventions for adults with
various forms of muscular dystrophy''
after ``including studies of the impact
of such diseases in rural and
underserved communities''; and
(B) in paragraph (2)(D), by inserting after
``including new biological agents'' the following:
``and new clinical interventions to improve the health
of those with muscular dystrophy''.
SEC. 3. SURVEILLANCE AND RESEARCH REGARDING MUSCULAR DYSTROPHY.
The second sentence of section 317Q(b) of the Public Health Service
Act (42 U.S.C. 247b-18(b)) is amended by inserting before the period
the following: ``and, to the extent possible, ensure that data be
representative of all affected populations and shared in a timely
manner''.
SEC. 4. INFORMATION AND EDUCATION.
Section 5(c) of the Muscular Dystrophy Community Assistance, Research
and Education Amendments of 2001 (42 U.S.C. 247b-19(c)) is amended--
(1) in paragraph (2)--
(A) by inserting ``for pediatric and adult patients,
including acute care considerations,'' after ``issuance
of care considerations'';
(B) by inserting ``various'' before ``other forms of
muscular dystrophy''; and
(C) by striking ``and'' at the end;
(2) by redesignating paragraph (3) as paragraph (4);
(3) by inserting after paragraph (2) the following:
``(3) in developing and updating care considerations under
paragraph (2), incorporate strategies specifically responding
to the findings of the national transitions survey of minority,
young adult, and adult communities of muscular dystrophy
patients; and''; and
(4) in paragraph (4), as redesignated, by inserting
``various'' before ``other forms of muscular dystrophy''.
Purpose and Summary
H.R. 594, the ``Paul D. Wellstone Muscular Dystrophy
Community Assistance, Research, and Education Amendments of
2014'' (MD CARE Amendments) was introduced on February 8, 2013,
by Rep. Burgess (R-TX) and Rep. Engel (D-NY) to update and
improve the Paul D. Wellstone Muscular Dystrophy Community
Assistance, Research, and Education Amendments of 2008.
Background and Need for Legislation
Muscular dystrophy (MD) is a group of more than 30 genetic
diseases characterized by progressive weakness and loss of
voluntary muscles that control movement. The muscular
dystrophies are a category of multi-systemic disorders that
range in age of onset, progression, and severity. Muscular
dystrophy affects hundreds of thousands of children and adults
throughout the U.S. and around the world. Some forms of
muscular dystrophy are seen in infancy or childhood, while
others may not appear until adulthood. Many of the muscular
dystrophies are characterized by cardiovascular, pulmonary, and
digestive issues. Of the nine types of muscular dystrophy,
Duchenne muscular dystrophy is the most common. There is no
cure for muscular dystrophy. The goal of treatment is a
reduction in symptoms and a delay in progress.\1\
---------------------------------------------------------------------------
\1\http://www.ninds.nih.gov/disorders/md/md.htm
---------------------------------------------------------------------------
The MD CARE Act was signed into law in December 2001,
reauthorized in October 2008, and has yielded great advances in
understanding the specific causes of the various forms of the
muscular dystrophies, the mechanisms of these diseases,
identification of therapeutic targets, and clinical trial
development. It supports programs that have benefited the lives
of muscular dystrophy patients and their families through
Federal biomedical research at National Institutes of Health
(NIH). It also supports surveillance, research, and outreach
activities related to muscular dystrophy at the Centers for
Disease Control and Prevention (CDC) that would improve
diagnosis, data collection, and care. As a result, there is a
growing population of men and women with pediatric-onset forms
of Muscular dystrophy now living into adulthood.
H.R. 594 improves upon current programs and updates them to
reflect the scientific developments in the field of muscular
dystrophy. It would revise the composition of the Muscular
Dystrophy Coordinating Committee (MDCC) to include the Social
Security Administration (SSA) and the Administration for
Community Living (ACL), and ensure that data collected by the
CDC captures data related to all affected populations.
Hearings
The Committee on Energy and Commerce has not held hearings
on the legislation.
Committee Consideration
On June 19, 2014, the Subcommittee on Health met in open
markup session and forwarded H.R. 594 to the full Committee, as
amended, by a voice vote.
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. 594 reported. A motion by Mr. Upton to order H.R. 594
reported to the House, as amended, was agreed to by a voice
vote.
Committee Oversight Findings
Pursuant to clause 3(c)(1) of rule XIII of the Rules of the
House of Representatives, the Committee has not held hearings
on this legislation.
Statement of General Performance Goals and Objectives
Pursuant to clause 3(c)(1) of rule XIII of the House of
Representatives, the goal of the legislation is to reauthorize
Federal programs related to muscular dystrophy.
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.
594 would result in no new or increased budget authority,
entitlement authority, or tax expenditures or revenues.
Earmark, Limited Tax Benefits, and Limited Tariff Benefits
In compliance with clause 9(e), 9(f), and 9(g) of rule XXI
of the Rules of the House of Representatives, the Committee
finds that H.R. 594 contains no earmarks, limited tax benefits,
or limited tariff benefits.
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, July 23, 2014.
Hon. Fred Upton,
Chairman, Committee on Energy and Commerce,
House of Representatives, Washington, DC.
Dear Mr. Chairman: The Congressional Budget Office has
prepared the enclosed cost estimate for H.R. 594, the Paul D.
Wellstone Muscular Dystrophy Community Assistance, Research,
and Education Amendments of 2014.
If you wish further details on this estimate, we will be
pleased to provide them. The CBO staff contacts are Jamease
Miles and Santiago Vallinas.
Sincerely,
Douglas W. Elmendorf.
Enclosure.
H.R. 594--Paul D. Wellstone Muscular Dystrophy Community Assistance,
Research, and Education Amendments of 2014
Summary: H.R. 594 would amend the Public Health Service Act
to reauthorize surveillance, research, and education activities
relating to muscular dystrophy. The bill would expand the
portfolios of the National Institutes of Health (NIH) and the
Centers for Disease Control and Prevention (CDC) to include
additional forms of muscular dystrophy. It also would direct
CDC to capture more representative data regarding muscular
dystrophy across populations.
CBO estimates that implementing H.R. 594 would cost $323
million over the 2015-2019 period, assuming appropriation of
the necessary amounts. Pay-as-you-go procedures do not apply to
this legislation because it would not affect direct spending or
revenues.
H.R. 594 contains no intergovernmental and private-sector
mandates as defined in the Unfunded Mandates Reform Act (UMRA).
Estimated cost to the Federal Government: The estimated
budgetary effect of H.R. 594 is shown in the following table.
The costs of this legislation fall within budget function 550
(health).
----------------------------------------------------------------------------------------------------------------
By fiscal year, in millions of dollars--
-------------------------------------------------------
2015 2016 2017 2018 2019 2015-2019
----------------------------------------------------------------------------------------------------------------
CHANGES IN SPENDING SUBJECT TO APPROPRIATION
Estimated Authorization Level........................... 78 80 81 83 85 407
Estimated Outlays....................................... 20 64 77 80 83 323
----------------------------------------------------------------------------------------------------------------
Note: Numbers may not add to totals because of rounding.
Basis of estimate: For this estimate, CBO assumes that H.R.
594 will be enacted near the end of fiscal year 2014, that the
necessary amounts will be appropriated each year, and that
outlays will follow historical spending patterns for the
affected programs.
The CDC and NIH administer activities that support
surveillance, research, and education activities for muscular
dystrophy of various forms. Authority to operate NIH-funded
programs expired at the end of fiscal year 2009. However, since
2009 the Congress has appropriated funds each year for NIH to
continue operating its research programs. The Congress
appropriated about $30 billion to the NIH for fiscal year 2014.
Of that total, NIH allocated about $78 million for activities
related to muscular dystrophy.
H.R. 594 would reauthorize NIH-funded initiatives for the
advancement of muscular dystrophy education, research, and
treatment. CBO estimates that implementing H.R. 594 would cost
$323 million for NIH activities over the 2015-2019 period,
assuming the availability of appropriated funds. The bill would
not increase CDC's current surveillance and research activities
regarding muscular dystrophy; thus, CBO expects that the
legislation would not affect spending by CDC to administer
those programs.
Pay-As-You-Go considerations: None.
Intergovernmental and private-sector impact: H.R. 594
contains no intergovernmental or private-sector mandates as
defined in UMRA and would impose no costs on state, local, or
tribal governments.
Estimate prepared by: Federal costs: Jamease Miles and
Santiago Vallinas; Impact on state, local, and tribal
governments: J'nell L. Blanco; Impact on the private sector:
Alexia Diorio.
Estimate approved by: Holly Harvey, Deputy 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.
Duplication of Federal Programs
No provision of H.R. 594 establishes or reauthorizes a
program of the Federal Government known to be duplicative of
another Federal program, a program that was included in any
report from the Government Accountability Office to Congress
pursuant to section 21 of Public Law 111-139, or a program
related to a program identified in the most recent Catalog of
Federal Domestic Assistance.
Disclosure of Directed Rule Makings
The Committee estimates that enacting H.R. 594 would not
specifically direct a rulemaking within the meaning of 5 U.S.C.
551.
Advisory Committee Statement
No advisory committees within the meaning of section 5(b)
of the Federal Advisory Committee Act were created by this
legislation.
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
Section 1 states the legislation may be cited as the ``Paul
D. Wellstone Muscular Dystrophy Community Assistance, Research,
and Education Amendments of 2014''.
Section 2. Initiative through the Director of the National Institutes
of Health
Section 2 would increase the membership MDCC) from 15 to 18
members to accommodate the inclusion of the other Federal
agencies, such as the SSA and the ACL. The plan developed by
the MDCC would include public services, various studies, and
the development of clinical interventions to improve the health
of those with muscular dystrophy.
The Committee encourages the MDCC to ensure that the needs
of a new population of adults living with muscular dystrophy
are addressed within the Action Plan and other initiatives.
These individuals have been well served by the Individuals with
Disabilities Education Act and the Americans with Disability
Act, but barriers exist for young people transitioning to
adulthood to live independently and maintain employment. The
Committee encourages the MDCC to evaluate the cost-
effectiveness of providing independent living resources and
promoting employment of this population, including the impact
of doing so on overall Federal spending.
Section 3. Surveillance and research regarding muscular dystrophy
Section 3 would ensure that the existing program captures
data from across the muscular dystrophies and that the data
would be shared in a timely manner.
The incidence and prevalence of the muscular dystrophies is
the same across all ethnic and racial groups. The Committee is
aware that there are gaps in those who are represented within
multi-disciplinary clinical care centers and clinical trials.
Therefore, the Committee encourages the CDC to continue
carrying out its national surveillance program and work to
capture and share, in a timely manner, data across all affected
populations.
Section 4. Information and education
Section 4 would include pediatric and adult patients and
acute care considerations in the development and issuance of
care considerations for muscular dystrophy. In addition, care
considerations should incorporate strategies from the findings
of the national transition survey of minority, young adult, and
adult communities of muscular dystrophy patients.
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 (existing law
proposed to be omitted is enclosed in black brackets, new
matter is printed in italic, existing law in which no change is
proposed is shown in roman):
PUBLIC HEALTH SERVICE ACT
* * * * * * *
TITLE III--GENERAL POWERS AND DUTIES OF PUBLIC HEALTH SERVICE
* * * * * * *
Part B--Federal-State Cooperation
* * * * * * *
SEC. 317Q. SURVEILLANCE AND RESEARCH REGARDING MUSCULAR DYSTROPHY.
(a) * * *
(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
and, to the extent possible, ensure that data be representative
of all affected populations and shared in a timely manner. In
making awards under this subsection, the Secretary may provide
direct technical assistance in lieu of cash.
* * * * * * *
TITLE IV--NATIONAL RESEARCH INSTITUTES
Part A--National Institutes of Health
* * * * * * *
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]
Musculoskeletal and Skin Diseases, the Eunice Kennedy
Shriver National Institute of Child Health and Human
Development, the National Heart, Lung, and Blood
Institute, 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, Becker, congenital
muscular dystrophy, limb-girdle muscular dystrophy,
myotonic, facioscapulohumeral muscular dystrophy
(referred to in this section as ``FSHD'') and other
forms of muscular dystrophy.
* * * * * * *
(b) Centers of Excellence.--
(1) * * *
(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, cardiac
and pulmonary function, and [genetics,] pharmacological
and other therapies.
(3) Coordination of centers.--The Director of NIH
shall, as appropriate, provide for the coordination of
information among centers under paragraph (1) and
ensure regular communication and sharing of data
between such centers.
* * * * * * *
(d) Coordinating Committee.--
(1) * * *
(2) Composition.--The Coordinating Committee shall
consist of not more than [15] 18 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], the Food and
Drug Administration, and the Administration for
Community Living and representatives of other
governmental agencies that serve children and
adults with muscular dystrophy, [such as the
Department of Education] including the
Department of Education and the Social Security
Administration; and
* * * * * * *
(4) Administrative support; terms of service; other
provisions.--The following shall apply with respect to
the Coordinating Committee:
(A) * * *
(B) The Coordinating Committee shall meet as
appropriate as determined by the Secretary, in
consultation with the chair, but shall meet no
fewer than two times per calendar year.
(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] through the
agencies represented on the Coordinating Committee
pursuant to subsection (d)(2)(A) 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, public services,
and rehabilitative issues, including studies of
the impact of such diseases in rural and
underserved communities, studies to demonstrate
the cost-effectiveness of providing independent
living resources and support to patients with
various forms of muscular dystrophy, and
studies to determine optimal clinical care
interventions for adults with various forms of
muscular dystrophy;
* * * * * * *
(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) * * *
* * * * * * *
(D) Basic and clinical research for the
development and evaluation of new treatments,
including new biological agents and new
clinical interventions to improve the health of
those with muscular dystrophy.
* * * * * * *
----------
MUSCULAR DYSTROPHY COMMUNITY ASSISTANCE, RESEARCH AND EDUCATION
AMENDMENTS OF 2001
* * * * * * *
SEC. 5. INFORMATION AND EDUCATION.
(a) * * *
* * * * * * *
(c) Requirements.--In carrying out this section, the
Secretary may--
(1) partner with leaders in the muscular dystrophy
patient community;
(2) cooperate with professional organizations and the
patient community in the development and issuance of
care considerations for pediatric and adult patients,
including acute care considerations, for Duchenne-
Becker muscular dystrophy, and various other forms of
muscular dystrophy, and in periodic review and updates,
as appropriate; [and]
(3) in developing and updating care considerations
under paragraph (2), incorporate strategies
specifically responding to the findings of the national
transitions survey of minority, young adult, and adult
communities of muscular dystrophy patients; and
[(3)] (4) widely disseminate the Duchenne-Becker
muscular dystrophy and various other forms of muscular
dystrophy care considerations as broadly as possible,
including through partnership opportunities with the
muscular dystrophy patient community.
* * * * * * *