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107th Congress                                                   Report
                        HOUSE OF REPRESENTATIVES
 1st Session                                                    107-195

======================================================================



 
    MUSCULAR DYSTROPHY COMMUNITY ASSISTANCE, RESEARCH AND EDUCATION 
                           AMENDMENTS OF 2001

                                _______
                                

 September 5, 2001.--Committed to the Committee of the Whole House on 
            the State of the Union and ordered to be printed

                                _______
                                

 Mr. Tauzin, from the Committee on Energy and Commerce, submitted the 
                               following

                              R E P O R T

                        [To accompany H.R. 717]

      [Including cost estimate of the Congressional Budget Office]

  The Committee on Energy and Commerce, to whom was referred 
the bill (H.R. 717) to amend the Public Health Service Act to 
provide for research and services with respect to Duchenne 
muscular dystrophy, having considered the same, report 
favorably thereon with amendments and recommend that the bill 
as amended do pass.

                                CONTENTS

                                                                   Page
Amendment........................................................     2
Purpose and Summary..............................................     5
Background and Need for Legislation..............................     6
Hearings.........................................................     6
Committee Consideration..........................................     6
Committee Votes..................................................     7
Committee Oversight Findings.....................................     7
Statement of General Performance Goals and Objectives............     7
New Budget Authority, Entitlement Authority, and Tax Expenditures     7
Committee Cost Estimate..........................................     7
Congressional Budget Office Estimate.............................     7
Federal Mandates Statement.......................................     9
Advisory Committee Statement.....................................     9
Constitutional Authority Statement...............................     9
Applicability to Legislative Branch..............................    10
Section-by-Section Analysis of the Legislation...................    10
Changes in Existing Law Made by the Bill, as Reported............    11

                               Amendment

  The amendments are as follows:
  Strike all after the enacting clause and insert the 
following:

SECTION 1. SHORT TITLE.

  This Act may be cited as the ``Muscular Dystrophy Community 
Assistance, Research and Education Amendments of 2001'', or the ``MD-
CARE Act''.

SEC. 2. FINDINGS.

  Congress makes the following findings:
          (1) Of the childhood muscular dystrophies, Duchenne Muscular 
        Dystrophy (DMD) is the world's most common and catastrophic 
        form of genetic childhood disease, and is characterized by a 
        rapidly progressive muscle weakness that almost always results 
        in death, usually by 20 years of age.
          (2) Duchenne muscular dystrophy is genetically inherited, and 
        mothers are the carriers in approximately 70 percent of all 
        cases.
          (3) If a female is a carrier of the dystrophin gene, there is 
        a 50 percent chance per birth that her male offspring will have 
        Duchenne muscular dystrophy, and a 50 percent chance per birth 
        that her female offspring will be carriers.
          (4) Duchenne is the most common lethal genetic disorder of 
        childhood worldwide, affecting approximately 1 in every 3,500 
        boys worldwide.
          (5) Children with muscular dystrophy exhibit extreme symptoms 
        of weakness, delay in walking, waddling gait, difficulty in 
        climbing stairs, and progressive mobility problems often in 
        combination with muscle hypertrophy.
          (6) Other forms of muscular dystrophy affecting children and 
        adults include Becker, limb girdle, congenital, 
        facioscapulohumeral, myotonic, oculopharyngeal, distal, and 
        Emery-Dreifuss muscular dystrophies.
          (7) Myotonic muscular dystrophy (also known as Steinert's 
        disease and dystrophia myotonica) is the second most prominent 
        form of muscular dystrophy and the type most commonly found in 
        adults. Unlike any of the other muscular dystrophies, the 
        muscle weakness is accompanied by myotonia (delayed relaxation 
        of muscles after contraction) and by a variety of abnormalities 
        in addition to those of muscle.
          (8) Facioscapulohumeral muscular dystrophy (referred to in 
        this section as ``FSHD'') is a neuromuscular disorder that is 
        inherited genetically and has an estimated frequency of 1 in 
        20,000. FSHD, affecting between 15,000 to 40,000 persons, 
        causes a progressive and sever loss of skeletal muscle 
        gradually bringing weakness and reduced mobility. Many persons 
        with FSHD become severely physically disabled and spend many 
        decades in a wheelchair.
          (9) FSHD is regarded as a novel genetic phenomenon resulting 
        from a crossover of subtelomeric DNA and may be the only human 
        disease caused by a deletion-mutation.
          (10) Each of the muscular dystrophies, though distinct in 
        progressivity and severity of symptoms, have a devastating 
        impact on tens of thousands of children and adults throughout 
        the United States and worldwide and impose severe physical and 
        economic burdens on those affected.
          (11) Muscular dystrophies have a significant impact on 
        quality of life--not only for the individual who experiences 
        its painful symptoms and resulting disability, but also for 
        family members and caregivers.
          (12) Development of therapies for these disorders, while 
        realistic with recent advances in research, is likely to 
        require costly investments and infrastructure to support gene 
        and other therapies.
          (13) There is a shortage of qualified researchers in the 
        field of neuromuscular research.
          (14) Many family physicians and health care professionals 
        lack the knowledge and resources to detect and properly 
        diagnose the disease as early as possible, thus exacerbating 
        the progressiveness of symptoms in cases that go undetected or 
        misdiagnosed.
          (15) There is a need for efficient mechanisms to translate 
        clinically relevant findings in muscular dystrophy research 
        from basic science to applied work.
          (16) Educating the public and health care community 
        throughout the country about this devastating disease is of 
        paramount importance and is in every respect in the public 
        interest and to the benefit of all communities.

SEC. 3. EXPANSION, INTENSIFICATION, AND COORDINATION OF ACTIVITIES OF 
                    NATIONAL INSTITUTES OF HEALTH WITH RESPECT TO 
                    RESEARCH ON MUSCULAR DYSTROPHY.

  Part A of title IV of the Public Health Service Act (42 U.S.C. 281 et 
seq.) is amended by adding at the end the following:

``SEC. 404E. MUSCULAR DYSTROPHY; INITIATIVE THROUGH DIRECTOR OF 
                    NATIONAL INSTITUTES OF HEALTH.

  ``(a) Expansion, Intensification, and Coordination of Activities.--
          ``(1) In general.--The Director of NIH, in coordination with 
        the Directors of the National Institute of Neurological 
        Disorders and Stroke, the National Institute of Arthritis and 
        Muscoskeletal and Skin Diseases, the National Institute of 
        Child Health and Human Development, and the other national 
        research institutes as appropriate, shall expand and intensify 
        programs of such Institutes with respect to research and 
        related activities concerning various forms of muscular 
        dystrophy, including Duchenne, myotonic, facioscapulohumeral 
        muscular dystrophy (referred to in this section as `FSHD') and 
        other forms of muscular dystrophy.
          ``(2) Coordination.--The Directors referred to in paragraph 
        (1) shall jointly coordinate the programs referred to in such 
        paragraph and consult with the Muscular Dystrophy Interagency 
        Coordinating Committee established under section 6 of the MD-
        CARE Act.
          ``(3) Allocations by director of nih.--The Director of NIH 
        shall allocate the amounts appropriated to carry out this 
        section for each fiscal year among the national research 
        institutes referred to in paragraph (1).
  ``(b) Centers of Excellence.--
          ``(1) In general.--The Director of NIH shall award grants and 
        contracts under subsection (a)(1) to public or nonprofit 
        private entities to pay all or part of the cost of planning, 
        establishing, improving, and providing basic operating support 
        for centers of excellence regarding research on various forms 
        of muscular dystrophy.
          ``(2) Research.--Each center under paragraph (1) shall 
        supplement but not replace the establishment of a comprehensive 
        research portfolio in all the muscular dystrophies. As a whole, 
        the centers shall conduct basic and clinical research in all 
        forms of muscular dystrophy including early detection, 
        diagnosis, prevention, and treatment, including the fields of 
        muscle biology, genetics, noninvasive imaging, genetics, 
        pharmacological and other therapies.
          ``(3) Coordination of centers; reports.--The Director of 
        NIH--
                  ``(A) shall, as appropriate, provide for the 
                coordination of information among centers under 
                paragraph (1) and ensure regular communication between 
                such centers; and
                  ``(B) shall require the periodic preparation of 
                reports on the activities of the centers and the 
                submission of the reports to the Director.
          ``(4) Organization of centers.--Each center under paragraph 
        (1) shall use the facilities of a single institution, or be 
        formed from a consortium of cooperating institutions, meeting 
        such requirements as may be prescribed by the Director of NIH.
          ``(5) Duration of support.--Support for a center established 
        under paragraph (1) may be provided under this section for a 
        period of not to exceed 5 years. Such period may be extended 
        for 1 or more additional periods not exceeding 5 years if the 
        operations of such center have been reviewed by an appropriate 
        technical and scientific peer review group established by the 
        Director of NIH and if such group has recommended to the 
        Director that such period should be extended.
  ``(c) Facilitation of Research.--The Director of NIH shall provide 
for a program under subsection (a)(1) under which samples of tissues 
and genetic materials that are of use in research on muscular dystrophy 
are donated, collected, preserved, and made available for such 
research. The program shall be carried out in accordance with accepted 
scientific and medical standards for the donation, collection, and 
preservation of such samples.
  ``(d) Coordinating Committee.--
          ``(1) In general.--The Secretary shall establish the Muscular 
        Dystrophy Coordinating Committee (referred to in this section 
        as the `Coordinating Committee') to coordinate activities 
        across the National Institutes and with other Federal health 
        programs and activities relating to the various forms of 
        muscular dystrophy.
          ``(2) Composition.--The Coordinating Committee shall consist 
        of not more than 15 members to be appointed by the Secretary, 
        of 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 and representatives of 
                other governmental agencies that serve children with 
                muscular dystrophy, such as the Department of 
                Education; and
                  ``(B) \1/3\ of such members shall be public members, 
                including a broad cross section of persons affected 
                with muscular dystrophies including parents or legal 
                guardians, affected individuals, researchers, and 
                clinicians.
        Members appointed under subparagraph (B) shall serve for a term 
        of 3 years, and may serve for an unlimited number of terms if 
        reappointed.
          ``(3) Chair.--
                  ``(A) In general.--With respect to muscular 
                dystrophy, the Chair of the Coordinating Committee 
                shall serve as the principal advisor to the Secretary, 
                the Assistant Secretary for Health, and the Director of 
                NIH, and shall provide advice to the Director of the 
                Centers for Disease Control and Prevention, the 
                Commissioner of Food and Drugs, and to the heads of 
                other relevant agencies. The Coordinating Committee 
                shall select the Chair for a term not to exceed 2 
                years.
                  ``(B) Appointment.--The Chair of the Committee shall 
                be appointed by and be directly responsible to the 
                Secretary.
          ``(4) Administrative support; terms of service; other 
        provisions.--The following shall apply with respect to the 
        Coordinating Committee:
                  ``(A) The Coordinating Committee shall receive 
                necessary and appropriate administrative support from 
                the Department of Health and Human Services.
                  ``(B) The Coordinating Committee shall meet as 
                appropriate as determined by the Secretary, in 
                consultation with the chair.
  ``(e) Plan for HHS Activities.--
          ``(1) In general.--Not later than 1 year after the date of 
        enactment of this section, the Coordinating Committee shall 
        develop a plan for conducting and supporting research and 
        education on muscular dystrophy through the national research 
        institutes and shall periodically review and revise the plan. 
        The plan shall--
                  ``(A) provide for a broad range of research and 
                education activities relating to biomedical, 
                epidemiological, psychosocial, and rehabilitative 
                issues, including studies of the impact of such 
                diseases in rural and underserved communities;
                  ``(B) identify priorities among the programs and 
                activities of the National Institutes of Health 
                regarding such diseases; and
                  ``(C) reflect input from a broad range of scientists, 
                patients, and advocacy groups.
          ``(2) Certain elements of plan.--The plan under paragraph (1) 
        shall, with respect to each form of muscular dystrophy, provide 
        for the following as appropriate:
                  ``(A) Research to determine the reasons underlying 
                the incidence and prevalence of various forms of 
                muscular dystrophy.
                  ``(B) Basic research concerning the etiology and 
                genetic links of the disease and potential causes of 
                mutations.
                  ``(C) The development of improved screening 
                techniques.
                  ``(D) Basic and clinical research for the development 
                and evaluation of new treatments, including new 
                biological agents.
                  ``(E) Information and education programs for health 
                care professionals and the public.
  ``(f) Reports to Congress.--The Coordinating Committee shall 
biennially submit to the Committee on Energy and Commerce of the House 
of Representatives, and the Committee on Health, Education, Labor, and 
Pensions of the Senate, a report that describes the research, 
education, and other activities on muscular dystrophy being conducted 
or supported through the Department of Health and Human Services. Each 
such report shall include the following:
          ``(1) The plan under subsection (e)(1) (or revisions to the 
        plan, as the case may be).
          ``(2) Provisions specifying the amounts expended by the 
        Department of Health and Human Services with respect to various 
        forms of muscular dystrophy, including Duchenne, myotonic, FSHD 
        and other forms of muscular dystrophy.
          ``(3) Provisions identifying particular projects or types of 
        projects that should in the future be considered by the 
        national research institutes or other entities in the field of 
        research on all muscular dystrophies.
  ``(g) Public Input.--The Secretary shall, under subsection (a)(1), 
provide for a means through which the public can obtain information on 
the existing and planned programs and activities of the Department of 
Health and Human Services with respect to various forms of muscular 
dystrophy and through which the Secretary can receive comments from the 
public regarding such programs and activities.
  ``(h) Authorization of Appropriations.--For the purpose of carrying 
out this section, there are authorized to be appropriated such sums as 
may be necessary for each of fiscal years 2002 through 2006. The 
authorization of appropriations established in the preceding sentence 
is in addition to any other authorization of appropriations that is 
available for conducting or supporting through the National Institutes 
of Health research and other activities with respect to muscular 
dystrophy.''.

SEC. 4. DEVELOPMENT AND EXPANSION OF ACTIVITIES OF CENTERS FOR DISEASE 
                    CONTROL AND PREVENTION WITH RESPECT TO 
                    EPIDEMIOLOGICAL RESEARCH ON MUSCULAR DYSTROPHY.

  Part B of title III of the Public Health Service Act (42 U.S.C. 243 
et seq.) is amended by inserting after section 317P the following:

``SEC. 317Q. SURVEILLANCE AND RESEARCH REGARDING MUSCULAR DYSTROPHY.

  ``(a) In General.--The Secretary, acting through the Director of the 
Centers for Disease Control and Prevention, may award grants and 
cooperative agreements to public or nonprofit private entities 
(including health departments of States and political subdivisions of 
States, and including universities and other educational entities) for 
the collection, analysis, and reporting of data on Duchenne and other 
forms of muscular dystrophy. In making such awards, the Secretary may 
provide direct technical assistance in lieu of cash.
  ``(b) National Muscular Dystrophy Epidemiology Program.--The 
Secretary, acting through the Director of the Centers for Disease 
Control and Prevention, may award grants to public or nonprofit private 
entities (including health departments of States and political 
subdivisions of States, and including universities and other 
educational entities) for the purpose of carrying out epidemiological 
activities regarding Duchenne and other forms of muscular dystrophies, 
including collecting and analyzing information on the number, 
incidence, correlates, and symptoms of cases. In carrying out the 
preceding sentence, the Secretary shall provide for a national 
surveillance program. In making awards under this subsection, the 
Secretary may provide direct technical assistance in lieu of cash.
  ``(c) Coordination With Centers of Excellence.--The Secretary shall 
ensure that epidemiological information under subsections (a) and (b) 
is made available to centers of excellence supported under section 
404E(b) by the Director of the National Institutes of Health.
  ``(d) Authorization of Appropriations.--There are authorized to be 
appropriated such sums as may be necessary to carry out this 
section.''.

SEC. 5. INFORMATION AND EDUCATION.

  (a) In General.--The Secretary of Health and Human Services (referred 
to in this Act as the ``Secretary'') shall establish and implement a 
program to provide information and education on muscular dystrophy to 
health professionals and the general public, including information and 
education on advances in the diagnosis and treatment of muscular 
dystrophy and training and continuing education through programs for 
scientists, physicians, medical students, and other health 
professionals who provide care for patients with muscular dystrophy.
  (b) Stipends.--The Secretary may use amounts made available under 
this section provides stipends for health professionals who are 
enrolled in training programs under this section.
  (c) Authorization of Appropriations.--There are authorized to be 
appropriated such sums as may be necessary to carry out this section.

SEC. 6. REPORT TO CONGRESS.

  Not later than January 1, 2003, and each January 1 thereafter, the 
Secretary shall prepare and submit to the appropriate committees of 
Congress, a report concerning the implementation of this Act and the 
amendments made by this Act.

  Amend the title so as to read:

    A bill to amend the Public Health Service Act to provide 
for research with respect to various forms of muscular 
dystrophy, including Duchenne, Becker, limb girdle, congenital, 
facioscapulohumeral, myotonic, oculopharyngeal, distal, and 
Emery-Dreifuss muscular dystrophies.

                          Purpose and Summary

    H.R. 717, the Muscular Dystrophy Childhood Assistance, 
Research and Education Amendments of 2001, allows the Director 
of the National Institutes of Health (NIH), in coordination 
with the Directors of the National Institute of Neurological 
Disorders and Stroke, the National Institute of Arthritis, and 
the National Institute of Child Health and Human Development to 
expand programs with respect to activities concerning Duchenne. 
The legislation also creates Centers of Excellence for 
Duchenne, which shall conduct basic and clinical research into 
Duchenne and various other muscular dystrophies.

                  Background and Need for Legislation

    Duchenne Muscular Dystrophy (DMD) is the most lethal 
genetic disorder of childhood worldwide, affecting 
approximately one in every 3,500 boys worldwide. The disease 
has 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 care givers. 
Symptoms of DMD include loss of muscle tissue, inability to 
walk, decreased lung capacity, and inability to move the major 
joints of the body.
    The course of DMD is fairly predictable. Children with the 
disorder are often late in learning to walk. A preschooler with 
DMD may seem clumsy and fall often. Soon, he has trouble 
climbing stairs, getting up from the floor, or running. By 
school age, the child may walk on his toes or the balls of his 
feet, with a slightly rolling gait. He has a waddling and 
unsteady gait, and can easily fall over. Nearly all children 
with DMD lose the ability to walk sometime between ages 7 and 
12. In the teen years, activities involving the arms, legs, or 
trunk require assistance or mechanical support.
    Thanks to advances in many areas of medicine, there are 
very good therapies available to assist children with all the 
effects of DMD and other muscular dystrophies. By using all 
available therapies, patients can prolong their comfort, 
function, and life expectancy. Despite these advances, however, 
current treatment options for Duchenne are minimal in efficacy 
and palliative, aimed at simply managing the symptoms in an 
effort to optimize the quality of life.

                                Hearings

    The Subcommittee on Health held a hearing on June 27, 2001 
on H.R. 717 entitled, ``Advancing the Health of the American 
People: Addressing Various Public Health Needs.'' The witnesses 
testifying on the bill were Mr. Ed McMahon, National Vice 
President, Muscular Dystrophy Association and Ms. Pat Furlong, 
President, Parent Project Muscular Dystrophy.

                        Committee Consideration

    On Wednesday, July 11, 2001, the Subcommittee on Health met 
in an open markup session and approved H.R. 717 for Full 
Committee consideration, as amended, by voice vote, a quorum 
being present. On Wednesday, July 18, 2001, the Full Committee 
met in open markup session and ordered H.R. 717 favorably 
reported to the House, as amended, by unanimous consent, a 
quorum being present.

                            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. 717 reported. A motion by Mr. Tauzin to order H.R. 717 
reported to the House, as amended, was agreed to by unanimous 
consent.

                      Committee Oversight Findings

    Pursuant to clause 3(c)(1) of rule XIII of the Rules of the 
House of Representatives, the Committee held a legislative 
hearing and made findings that are reflected in this report.

         Statement of General Performance Goals and Objectives

    The goal of H.R. 717 is to allow the Director of the 
National Institutes of Health to expand programs with respect 
to activities concerning Duchenne, and create Centers of 
Excellence for Duchenne to conduct basic and clinical research 
into Duchenne and other muscular dystrophies.

   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. 
717, the Muscular Dystrophy Childhood Assistance, Research and 
Education Amendments of 2001, would result in no new or 
increased budget authority, entitlement authority, or tax 
expenditures or revenues.

                        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, August 10, 2001.
Hon.  W.J. ``Billy'' Tauzin,
Chairman, Committee on Energy and Commerce,
House of Representatives, Washington, DC.
    Dear Mr. Chairman: The Congressional Budget Office has 
prepared the enclosed estimate of H.R. 717, the MD-CARE Act, as 
ordered reported by the Committee on Energy and Commerce on 
July 18, 2001.
    If you wish further details on this estimate, we will be 
pleased to provide them. The CBO staff contact is Christopher 
J. Topoleski.
            Sincerely,
                                         Robert A. Sunshine
                                    (For Dan L. Crippen, Director).
    Enclosure.

H.R. 717--Muscular Dystrophy Community Assistance, Research and 
        Education Amendments of 2001 (MD-CARE Act)

    Summary: H.R. 717 would require the National Institutes of 
Health (NIH) and the Centers for Disease Control and Prevention 
(CDC) to provide grants and expand research on the health needs 
of individuals with muscular dystrophy. Assuming the 
appropriation of the necessary amounts, CBO estimates that 
implementing H.R. 717 would cost $4 million in 2002 and $56 
million over the 2002-2006 period. The legislation would not 
affect direct spending or receipts; therefore, pay-as-you-go 
procedures would not apply.
    H.R. 717 contains no intergovernmental or private-sector 
mandates as defined in the Unfunded Mandates Reform Act (UMRA). 
State, local, and tribal governments would be eligible for 
grants authorized by the bill for research activities 
associated with muscular dystrophy.
    Estimated Cost to the Federal Government: The estimated 
budgetary impact of H.R. 717 is shown in the following table. 
The costs of this legislation fall within budget function 550 
(health).

----------------------------------------------------------------------------------------------------------------
                                                                     By fiscal year, in millions of dollars--
                                                                 -----------------------------------------------
                                                                   2001    2002    2003    2004    2005    2006
----------------------------------------------------------------------------------------------------------------
                                        SPENDING SUBJECT TO APPROPRIATION

Spending Under Current Law:
    Estimated Authorization Level \1\...........................    6504    6664    6809    6959    7107    7265
    Estimated Outlays...........................................    5326    6063    6474    6773    6992    7100
Proposed Changes:
    Estimated Authorization Level...............................       0      11      14      15      15      15
    Estimated Outlays...........................................       0       4      10      13      14      15
Spending Under H.R. 717:
    Estimated Authorization Level...............................    6504    6675    6823    6974    7122    7280
    Estimated Outlays...........................................    5326    6067    6484    6786    7006    7115
----------------------------------------------------------------------------------------------------------------
\1\ The 2001 level is the amount appropriated for that year for the agencies that would be affected by H.R. 717.
  The 2002-2006 levels are CBO baseline projections, including adjustments for anticipated inflation.

    Basis of estimate: The bill would authorize the NIH to 
award grants to and contract with public and nonprofit private 
entities known as ``Centers of Excellence'' to provide basic 
and clinical research on muscular dystrophy, including 
diagnosis, early detection, prevention, and treatment. The 
centers would be awarded renewable contracts for up to five 
years for each contract period. CBO assumes that NIH would 
designate one center of excellance in 2002 and two additional 
centers in 2003.
    The NIH would also be required to establish a program under 
which samples of tissue and other genetic materials used in 
muscular dystrophy research would be collected, stored, and 
made available for study.
    The Secretary of Health and Human Services (HHS) would be 
required to establish a coordinating committee to organize 
muscular dystrophy research activities across the NIH and other 
federal health programs. The committee would consist of 15 
members appointed from both government agencies and from 
members of the public affected by muscular dystrophy. The 
committee would be required to produce a plan that identifies 
opportunities for research and education on muscular dystrophy. 
In addition, the committee would be required to submit a 
biennial report to the Congress summarizing expenditures by HHS 
and the current and future research agenda.
    Based on amounts spent in the past for similar activities, 
CBO estimates that the activities of the NIH and the 
coordinating committee would cost $2 million in 2002 and $28 
million over the 2002-2006 period if the necessary amounts are 
appropriated.
    The bill would authorize appropriations of such sums as 
necessary for the Centers for Disease Control and Prevention to 
award grants to public or nonprofit private entities to conduct 
research, carry out epidemiological activities, and establish a 
national muscular dystrophy surveillance program. Based on 
Information provided by the CDC about amounts spent in the past 
for similar activities and on the agency's historical spending 
patterns, CBO estimates the agency would spend $2 million in 
2002 and $28 million over the 2002-2006 period for those 
purposes if the necessary amounts are appropriated.
    This estimate assumes that the bill would be enacted and 
initial appropriations provided by October 1, 2001.
    Pay as you-go considerations: None.
    Intergovernmental and private-sector impact: H.R. 717 
contains no intergovernmental or private-sector mandates as 
defined in UMRA. State, local, and tribal governments would be 
eligible for grants authorized by the bill for research 
activities associated with muscular dystrophy.
    Estimate prepared by: Federal Costs: Christopher J. 
Topoleski and Jeanne De Sa. Impact on State, Local, and Tribal 
Governments: Leo Lex. Impact on the Private Sector: Amy 
Fedigan.
    Estimate approved by: Robert A. Sunshine, 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.

                      Advisory Committee Statement

    No advisory committees within the meaning of section 5(b) 
of the Federal Advisory Committee Act were created by this 
legislation.

                   Constitutional Authority Statement

    Pursuant to clause 3(d)(1) of rule XIII of the Rules of the 
House of Representatives, the Committee finds that the 
Constitutional authority for this legislation is provided in 
Article I, section 8, clause 3, which grants Congress the power 
to regulate commerce with foreign nations, among the several 
States, and with the Indian tribes.

                  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

    This Act may be cited as the ``Muscular Dystrophy Community 
Assistance, Research, and Education Amendments of 2001,'' or 
the ``MD-CARE Act.''

Section 2. Findings

    Section 2 finds that: (1) Duchenne Muscular Dystrophy (DMD) 
is the world's most common and catastrophic form of genetic 
childhood disease; (2) DMD is genetically inherited, and 
mothers are the carriers in approximately 70 percent of all 
cases; (3) DMD affects 1 in 3,500 boys worldwide; (4) there are 
other dystrophies which also have a devastating impact on the 
quality of life for patients, including Myotonic muscular 
dystrophy and Facioscapulohumeral muscular dystrophy; (5) there 
is a shortage of qualified researchers in the field of 
neuromuscular research; (6) development of therapies for these 
disorders is costly; and, (7) educating the public and health 
care community is in the public interest and to the benefit of 
all communities.

Section 3. Expansion, intensification, and coordination of activities 
        of National Institutes of Health with respect to research on 
        muscular dystrophy

    Section 3 directs the Director of the NIH to expand and 
intensify activities with respect to various forms of muscular 
dystrophy, and to allocate the amounts appropriated to carry 
out this section.
    In addition, this section directs the Director of NIH to 
award grants and contracts to public or nonprofit private 
entities to pay all, or part of, the costs of planning, 
establishing, improving, and providing support to centers of 
excellence regarding research on various forms of muscular 
dystrophy.
    The Secretary of Health and Human Services is directed to 
establish the Muscular Dystrophy Coordinating Committee to 
coordinate activities across other Federal health programs 
relating to the various forms of muscular dystrophy.

Section 4. Development and expansion of activities of Centers for 
        Disease Control and Prevention with respect to epidemiological 
        research on muscular dystrophy

    Section 4 allows the Secretary, acting through the Director 
of the Centers for Disease Control and Prevention, to award 
grants and cooperative agreements to public or nonprofit 
private entities for the collection, analysis, and reporting of 
data on Duchenne and other forms of muscular dystrophy. This 
section also allows the Secretary to award grants to public or 
nonprofit private entities for the purpose of carrying out 
epidemiological activities regarding muscular dystrophy.

Section 5. Information and education

    Section 5 requires the Secretary of Health and Human 
Services to establish and implement a program to provide 
information and education on muscular dystrophy, including 
information and education on advances in the diagnosis and 
treatment of muscular dystrophy.

Section 6. Report to Congress

    Section 6 requires that not later than January 1, 2003, and 
each January 1 thereafter, the Secretary shall prepare and 
submit to the appropriate committees of Congress, a report 
concerning the implementation of this Act and the amendments 
made by this Act.

         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 (new matter is 
printed in italic and existing law in which no change is 
proposed is shown in roman):

                       PUBLIC HEALTH SERVICE ACT




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TITLE III--GENERAL POWERS AND DUTIES OF PUBLIC HEALTH SERVICE

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Part B--Federal-State Cooperation

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SEC. 317Q. SURVEILLANCE AND RESEARCH REGARDING MUSCULAR DYSTROPHY.

  (a) In General.--The Secretary, acting through the Director 
of the Centers for Disease Control and Prevention, may award 
grants and cooperative agreements to public or nonprofit 
private entities (including health departments of States and 
political subdivisions of States, and including universities 
and other educational entities) for the collection, analysis, 
and reporting of data on Duchenne and other forms of muscular 
dystrophy. In making such awards, the Secretary may provide 
direct technical assistance in lieu of cash.
  (b) National Muscular Dystrophy Epidemiology Program.--The 
Secretary, acting through the Director of the Centers for 
Disease Control and Prevention, may award grants to public or 
nonprofit private entities (including health departments of 
States and political subdivisions of States, and including 
universities and other educational entities) for the purpose of 
carrying out epidemiological activities regarding Duchenne and 
other forms of muscular dystrophies, including collecting and 
analyzing information on the number, incidence, correlates, and 
symptoms of cases. In carrying out the preceding sentence, the 
Secretary shall provide for a national surveillance program. In 
making awards under this subsection, the Secretary may provide 
direct technical assistance in lieu of cash.
  (c) Coordination With Centers of Excellence.--The Secretary 
shall ensure that epidemiological information under subsections 
(a) and (b) is made available to centers of excellence 
supported under section 404E(b) by the Director of the National 
Institutes of Health.
  (d) Authorization of Appropriations.--There are authorized to 
be appropriated such sums as may be necessary to carry out this 
section.

                 TITLE IV--NATIONAL RESEARCH INSTITUTES

Part A--National Institutes of Health

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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 and Skin 
        Diseases, the National Institute of Child Health and 
        Human Development, and the other national research 
        institutes as appropriate, shall expand and intensify 
        programs of such Institutes with respect to research 
        and related activities concerning various forms of 
        muscular dystrophy, including Duchenne, myotonic, 
        facioscapulohumeral muscular dystrophy (referred to in 
        this section as ``FSHD'') and other forms of muscular 
        dystrophy.
          (2) Coordination.--The Directors referred to in 
        paragraph (1) shall jointly coordinate the programs 
        referred to in such paragraph and consult with the 
        Muscular Dystrophy Interagency Coordinating Committee 
        established under section 6 of the MD-CARE Act.
          (3) Allocations by director of nih.--The Director of 
        NIH shall allocate the amounts appropriated to carry 
        out this section for each fiscal year among the 
        national research institutes referred to in paragraph 
        (1).
  (b) Centers of Excellence.--
          (1) In general.--The Director of NIH shall award 
        grants and contracts under subsection (a)(1) to public 
        or nonprofit private entities to pay all or part of the 
        cost of planning, establishing, improving, and 
        providing basic operating support for centers of 
        excellence regarding research on various forms of 
        muscular dystrophy.
          (2) Research.--Each center under paragraph (1) shall 
        supplement but not replace the establishment of a 
        comprehensive research portfolio in all the muscular 
        dystrophies. As a whole, the centers shall conduct 
        basic and clinical research in all forms of muscular 
        dystrophy including early detection, diagnosis, 
        prevention, and treatment, including the fields of 
        muscle biology, genetics, noninvasive imaging, 
        genetics, pharmacological and other therapies.
          (3) Coordination of centers; reports.--The Director 
        of NIH--
                  (A) shall, as appropriate, provide for the 
                coordination of information among centers under 
                paragraph (1) and ensure regular communication 
                between such centers; and
                  (B) shall require the periodic preparation of 
                reports on the activities of the centers and 
                the submission of the reports to the Director.
          (4) Organization of centers.--Each center under 
        paragraph (1) shall use the facilities of a single 
        institution, or be formed from a consortium of 
        cooperating institutions, meeting such requirements as 
        may be prescribed by the Director of NIH.
          (5) Duration of support.--Support for a center 
        established under paragraph (1) may be provided under 
        this section for a period of not to exceed 5 years. 
        Such period may be extended for 1 or more additional 
        periods not exceeding 5 years if the operations of such 
        center have been reviewed by an appropriate technical 
        and scientific peer review group established by the 
        Director of NIH and if such group has recommended to 
        the Director that such period should be extended.
  (c) Facilitation of Research.--The Director of NIH shall 
provide for a program under subsection (a)(1) under which 
samples of tissues and genetic materials that are of use in 
research on muscular dystrophy are donated, collected, 
preserved, and made available for such research. The program 
shall be carried out in accordance with accepted scientific and 
medical standards for the donation, collection, and 
preservation of such samples.
  (d) Coordinating Committee.--
          (1) In general.--The Secretary shall establish the 
        Muscular Dystrophy Coordinating Committee (referred to 
        in this section as the ``Coordinating Committee'') to 
        coordinate activities across the National Institutes 
        and with other Federal health programs and activities 
        relating to the various forms of muscular dystrophy.
          (2) Composition.--The Coordinating Committee shall 
        consist of not more than 15 members to be appointed by 
        the Secretary, of 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 and 
                representatives of other governmental agencies 
                that serve children with muscular dystrophy, 
                such as the Department of Education; and
                  (B) \1/3\ of such members shall be public 
                members, including a broad cross section of 
                persons affected with muscular dystrophies 
                including parents or legal guardians, affected 
                individuals, researchers, and clinicians.
        Members appointed under subparagraph (B) shall serve 
        for a term of 3 years, and may serve for an unlimited 
        number of terms if reappointed.
          (3) Chair.--
                  (A) In general.--With respect to muscular 
                dystrophy, the Chair of the Coordinating 
                Committee shall serve as the principal advisor 
                to the Secretary, the Assistant Secretary for 
                Health, and the Director of NIH, and shall 
                provide advice to the Director of the Centers 
                for Disease Control and Prevention, the 
                Commissioner of Food and Drugs, and to the 
                heads of other relevant agencies. The 
                Coordinating Committee shall select the Chair 
                for a term not to exceed 2 years.
                  (B) Appointment.--The Chair of the Committee 
                shall be appointed by and be directly 
                responsible to the Secretary.
          (4) Administrative support; terms of service; other 
        provisions.--The following shall apply with respect to 
        the Coordinating Committee:
                  (A) The Coordinating Committee shall receive 
                necessary and appropriate administrative 
                support from the Department of Health and Human 
                Services.
                  (B) The Coordinating Committee shall meet as 
                appropriate as determined by the Secretary, in 
                consultation with the chair.
  (e) Plan for HHS Activities.--
          (1) In general.--Not later than 1 year after the date 
        of enactment of this section, the Coordinating 
        Committee shall develop a plan for conducting and 
        supporting research and education on muscular dystrophy 
        through the national research institutes and shall 
        periodically review and revise the plan. The plan 
        shall--
                  (A) provide for a broad range of research and 
                education activities relating to biomedical, 
                epidemiological, psychosocial, and 
                rehabilitative issues, including studies of the 
                impact of such diseases in rural and 
                underserved communities;
                  (B) identify priorities among the programs 
                and activities of the National Institutes of 
                Health regarding such diseases; and
                  (C) reflect input from a broad range of 
                scientists, patients, and advocacy groups.
          (2) Certain elements of plan.--The plan under 
        paragraph (1) shall, with respect to each form of 
        muscular dystrophy, provide for the following as 
        appropriate:
                  (A) Research to determine the reasons 
                underlying the incidence and prevalence of 
                various forms of muscular dystrophy.
                  (B) Basic research concerning the etiology 
                and genetic links of the disease and potential 
                causes of mutations.
                  (C) The development of improved screening 
                techniques.
                  (D) Basic and clinical research for the 
                development and evaluation of new treatments, 
                including new biological agents.
                  (E) Information and education programs for 
                health care professionals and the public.
  (f) Reports to Congress.--The Coordinating Committee shall 
biennially submit to the Committee on Energy and Commerce of 
the House of Representatives, and the Committee on Health, 
Education, Labor, and Pensions of the Senate, a report that 
describes the research, education, and other activities on 
muscular dystrophy being conducted or supported through the 
Department of Health and Human Services. Each such report shall 
include the following:
          (1) The plan under subsection (e)(1) (or revisions to 
        the plan, as the case may be).
          (2) Provisions specifying the amounts expended by the 
        Department of Health and Human Services with respect to 
        various forms of muscular dystrophy, including 
        Duchenne, myotonic, FSHD and other forms of muscular 
        dystrophy.
          (3) Provisions identifying particular projects or 
        types of projects that should in the future be 
        considered by the national research institutes or other 
        entities in the field of research on all muscular 
        dystrophies.
  (g) Public Input.--The Secretary shall, under subsection 
(a)(1), provide for a means through which the public can obtain 
information on the existing and planned programs and activities 
of the Department of Health and Human Services with respect to 
various forms of muscular dystrophy and through which the 
Secretary can receive comments from the public regarding such 
programs and activities.
  (h) Authorization of Appropriations.--For the purpose of 
carrying out this section, there are authorized to be 
appropriated such sums as may be necessary for each of fiscal 
years 2002 through 2006. The authorization of appropriations 
established in the preceding sentence is in addition to any 
other authorization of appropriations that is available for 
conducting or supporting through the National Institutes of 
Health research and other activities with respect to muscular 
dystrophy.

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