39–006 

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 


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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: 


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1http://www.ninds.nih.gov/disorders/md/md.htm 

‘‘(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

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. 


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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: 


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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 


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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. 


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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): 


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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 ACTIVI- TIES.— (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 

øMuscoskeletal¿ 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.— 


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(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 øgenetics,¿ 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 ø15¿ 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 øand 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, øsuch 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 øthrough 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 


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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; øand¿ 

(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 

ø(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. 

* * * * * * * 

Æ 


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