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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.

           *       *       *       *       *       *       *