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        H.R.717

                      One Hundred Seventh Congress

                                 of the

                        United States of America


                          AT THE FIRST SESSION

         Begun and held at the City of Washington on Wednesday,
             the third day of January, two thousand and one


                                 An Act


 
  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.

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled,

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``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 
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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
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 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.

SEC. 7. STUDY ON THE USE OF CENTERS OF EXCELLENCE AT THE NATIONAL 
              INSTITUTES OF HEALTH.

    (a) Review.--Not later than 60 days after the date of enactment of 
this Act, the Secretary of Health and Human Services shall enter into a 
contract with the Institute of Medicine for the purpose of conducting a 
study and making recommendations on the impact of, need for, and other 
issues associated with Centers of Excellence at the National Institutes 
of Health.
    (b) Areas of Review.--In conducting the study under subsection (a), 
the Institute of Medicine shall at a minimum consider the following:
        (1) The current areas of research incorporating Centers of 
    Excellence (which shall include a description of such areas) and 
    the relationship of this form of funding mechanism to other forms 
    of funding for research grants, including investigator initiated 
    research, contracts and other types of research support awards.
        (2) The distinctive aspects of Centers of Excellence, including 
    the additional knowledge that may be expected to be gained through 
    Centers of Excellence as compared to other forms of grant or 
    contract mechanisms.
        (3) The costs associated with establishing and maintaining 
    Centers of Excellence, and the record of scholarship and training 
    resulting from such Centers. The research and training 
    contributions of Centers should be assessed on their own merits and 
    in comparison with other forms of research support.
        (4) Specific areas of research in which Centers of Excellence 
    may be useful, needed, or underused, as well as areas of research 
    in which Centers of Excellence may not be helpful.
        (5) Criteria that may be applied in determining when Centers of 
    Excellence are an appropriate and cost-effective research 
    investment and conditions that should be present in order to 
    consider the establishment of Centers of Excellence.
        (6) Alternative research models that may accomplish results 
    similar to or greater than Centers of Excellence.
    (c) Report.--Not later than 1 year after the date on which the 
contract is entered into under subsection (a), the Institute of 
Medicine shall complete the study under such subsection and submit a 
report to the Secretary of Health and Human Services and the 
appropriate committees of Congress that contains the results of such 
study.

                               Speaker of the House of Representatives.

                            Vice President of the United States and    
                                               President of the Senate.

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