[107th Congress Public Law 84]
[From the U.S. Government Printing Office]


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[DOCID: f:publ084.107]


[[Page 115 STAT. 823]]

Public Law 107-84
107th Congress

                                 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. <<NOTE: Dec. 18, 2001 -  [H.R. 717]>> 

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled, <<NOTE: Muscular 
Dystrophy Community Assistance, Research and Education Amendments of 
2001.>> 

SECTION 1. SHORT TITLE. <<NOTE: 42 USC 201 note.>> 

    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. <<NOTE: 42 USC 247b-18 note.>> 

    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.

[[Page 115 STAT. 824]]

            (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. <<NOTE: 42 USC 283g.>>  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

[[Page 115 STAT. 825]]

        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 <<NOTE: Grants. Contracts.>> 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.--

[[Page 115 STAT. 826]]

            ``(1) In <<NOTE: Establishment.>> 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 <<NOTE: Applicability.>> 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 <<NOTE: Deadline.>> 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--

[[Page 115 STAT. 827]]

                    ``(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.''.

[[Page 115 STAT. 828]]

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. <<NOTE: 42 USC 247b-18.>> 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. <<NOTE: 42 USC 247b-19.>> 

    (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.

[[Page 115 STAT. 829]]

SEC. 6. REPORT TO CONGRESS. <<NOTE: 42 USC 247b-18 note.>> 

    Not <<NOTE: Deadline.>> 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. <<NOTE: 42 USC 281 note.>> STUDY ON THE USE OF CENTERS OF 
            EXCELLENCE AT THE NATIONAL INSTITUTES OF HEALTH.

    (a) Review.--Not <<NOTE: Deadline. Contracts.>> 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 <<NOTE: Deadline.>> 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

[[Page 115 STAT. 830]]

and the appropriate committees of Congress that contains the results of 
such study.

    Approved December 18, 2001.

LEGISLATIVE HISTORY--H.R. 717:
---------------------------------------------------------------------------

HOUSE REPORTS: No. 107-195 (Comm. on Energy and Commerce).
CONGRESSIONAL RECORD, Vol. 147 (2001):
            Sept. 24, considered and passed House.
            Nov. 15, considered and passed Senate, amended.
            Nov. 29, House concurred in Senate amendment.

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