[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[H.R. 717 Reported in Senate (RS)]






                                                       Calendar No. 208
107th CONGRESS
  1st Session
                                H. R. 717


_______________________________________________________________________


                   IN THE SENATE OF THE UNITED STATES

                           September 25, 2001

     Received; read twice and referred to the Committee on Health, 
                     Education, Labor, and Pensions

                            October 30, 2001

               Reported by Mr. Kennedy, with an amendment
                  [Insert the part printed in italic]

_______________________________________________________________________

                                 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 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 providing 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.




                                                       Calendar No. 208

107th CONGRESS

  1st Session

                               H. R. 717

_______________________________________________________________________

                                 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.

_______________________________________________________________________

                            October 30, 2001

                       Reported with an amendment