[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[H.R. 717 Introduced in House (IH)]







107th CONGRESS
  1st Session
                                H. R. 717

  To amend the Public Health Service Act to provide for research and 
         services with respect to Duchenne muscular dystrophy.


_______________________________________________________________________


                    IN THE HOUSE OF REPRESENTATIVES

                           February 14, 2001

Mr. Wicker (for himself, Mr. Peterson of Minnesota, Mr. Greenwood, Mr. 
 Tanner, Mr. Ehrlich, Mr. Cramer, Mr. Gordon, Mrs. Emerson, Mr. Riley, 
 Mr. Bryant, Mr. Ford, Mr. Foley, Ms. Hooley of Oregon, Mr. King, Mr. 
   Hobson, Mr. Pickering, Mr. Chambliss, Mr. Ehlers, Mr. Towns, Mr. 
  McGovern, Mr. LaTourette, Mr. Doolittle, Mr. Watts of Oklahoma, Ms. 
   Granger, Mr. Blumenauer, Mr. Murtha, Mr. Olver, Mr. Boehlert, Mr. 
    Goodlatte, Mr. Holden, Mr. Watkins, Mr. Coble, Mr. Isakson, Mr. 
  LoBiondo, Mr. McCrery, Mr. Kerns, Mr. Gilman, Mr. Rohrabacher, Mr. 
Issa, Mr. Calvert, Mr. Langevin, Mrs. Meek of Florida, Mr. Hastings of 
 Florida, Ms. Brown of Florida, Mr. Miller of Florida, Mr. Otter, Mr. 
 Walden of Oregon, Mrs. Myrick, Mr. LaHood, Mr. Lipinski, Mr. Lewis of 
Kentucky, Mr. Wolf, Mr. Hostettler, Mr. Kingston, Mr. Scarborough, Mr. 
 Upton, Mr. Leach, Mr. Gillmor, Mr. Walsh, Mr. Quinn, Mr. Ganske, Mr. 
Jones of North Carolina, Mr. Bachus, Mr. Oxley, Mr. Tiahrt, Mr. Weller, 
  Mr. Matsui, Mr. Weldon of Florida, Mr. Reynolds, Mr. Gutknecht, Mr. 
Chabot, Mr. Hunter, Mr. Goode, Mr. Fletcher, Mr. Skelton, Mr. Moran of 
Virginia, Mr. Rodriguez, Mr. Turner, Mr. Bentsen, Mr. Abercrombie, Mr. 
Gonzalez, Mr. Bilirakis, Mr. Armey, Mr. McHugh, Mr. Jenkins, Mr. Boyd, 
  Mr. Putnam, Mr. Rogers of Michigan, Mr. Keller, Mrs. Kelly, and Mr. 
  Manzullo) introduced the following bill; which was referred to the 
                    Committee on Energy and Commerce

_______________________________________________________________________

                                 A BILL


 
  To amend the Public Health Service Act to provide for research and 
         services with respect to Duchenne muscular dystrophy.

    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 ``Duchenne Muscular Dystrophy 
Childhood Assistance, Research and Education Amendments of 2001'', or 
the ``DMD CARE Act''.

SEC. 2. FINDINGS.

    The Congress finds as follows:
            (1) Duchenne is the most common form of muscular dystrophy, 
        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) Children with Duchenne 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.
            (5) Duchenne is the most common lethal genetic disorder of 
        childhood worldwide, affecting approximately one in every 3,500 
        boys worldwide.
            (6) Although the dystrophin gene that causes DMD was 
        successfully identified and isolated by medical researchers in 
        1987, Federal research devoted to potential treatment options 
        or a cure since this initial discovery has been minimal.
            (7) Because of the limited Federal support for medical 
        research specific to this form of muscular dystrophy, current 
        treatment options are minimal in efficacy and palliative, aimed 
        at simply managing the symptoms in an effort to optimize the 
        quality of life.
            (8) 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.
            (9) This 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 caregivers.
            (10) Currently there exists only a small quantity of public 
        information about Duchenne muscular dystrophy, and what little 
        information does exist remains inadequately disseminated and 
        insufficient in addressing the needs of specific diverse 
        populations and other underserved groups.
            (11) 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 
              DUCHENNE MUSCULAR DYSTROPHY.

    Subpart 4 of part C of title IV of the Public Health Service Act 
(42 U.S.C. 285d et seq.) is amended by inserting after section 441A the 
following section:

``duchenne muscular dystrophy; initiative through director of national 
                          institutes of health

    ``Sec. 441B. (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, National Institute of Arthritis and 
        Muscoskeletal and Skin Diseases and the National Institute of 
        Child Health and Human Development, shall expand and intensify 
programs of such Institutes with respect to research and related 
activities concerning Duchenne muscular dystrophy.
            ``(2) Coordination.--The Directors referred to in 
        subsection (a) shall jointly coordinate the programs referred 
        to in such subsection and consult with the Muscular Dystrophy 
        Interagency Coordinating Committee.
            ``(3) Allocations by director of nih.--With respect to 
        amounts appropriated to carry out this section for a fiscal 
        year, the Director of NIH shall allocate the amounts among the 
        national research institutes that are referred to in subsection 
        (a).
    ``(b) Centers of Excellence.--
            ``(1) In general.--The Director shall under subsection 
        (a)(1) make awards of grants and contracts 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 Duchenne muscular dystrophy.
            ``(2) Research.--Each center under paragraph (1) shall 
        conduct basic and clinical research into Duchenne muscular 
        dystrophy. Such research should include investigations into the 
        diagnosis, early detection, prevention, control, and adequate 
        treatment of Duchenne muscular dystrophy. The centers, as a 
        group, shall conduct research including the fields of muscle 
        biology, genetics, non-invasive imaging, gene and 
        pharmaceutical therapies.
            ``(3) Coordination of centers; reports.--The Director 
        shall, as appropriate, provide for the coordination of 
        information among centers under paragraph (1) and ensure 
        regular communication between such centers, and may 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.
            ``(5) Number of centers; duration of support.--
                    ``(A) In general.--The Director shall provide for 
                the establishment of not less than three centers under 
                paragraph (1).
                    ``(B) Duration.--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 one 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 and if such group has recommended to the 
                Director that such period should be extended.
    ``(c) Facilitation of Research.--The Director shall under 
subsection (a)(1) provide for a program under which samples of tissues 
and genetic materials that are of use in research on Duchenne 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 such 
        diseases.
            ``(2) Composition.--The Coordinating Committee shall be 
        composed of the directors or their designees of each of the 
        national research institutes involved in research with respect 
        to Duchenne 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 Services and Resources Administration and the Food and 
        Drug Administration.
            ``(3) Chair.--
                    ``(A) In general.--With respect to Duchenne 
                muscular dystrophy, the Chair of the 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 other relevant 
                agencies.
                    ``(B) Director of nih.--The Chair of the Committee 
                shall be directly responsible to the Director of NIH.
    ``(e) Plan for NIH Activities.--
            ``(1) In general.--Not later than 1 year after the date of 
        the enactment of this section, the Coordinating Committee shall 
        develop a plan for conducting and supporting research and 
        education on Duchenne 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, 
                psychosocial, and rehabilitative issues, including 
                studies of the impact of such diseases on boys 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 Duchenne muscular dystrophy, provide 
        for the following as appropriate:
                    ``(A) Research to determine the reasons underlying 
                the incidence and prevalence of Duchenne 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.
            ``(3) Implementation of plan.--The Director of NIH shall 
        ensure that programs and activities of the National Institutes 
        of Health regarding Duchenne muscular dystrophy are implemented 
        in accordance with the plan under paragraph (1).
    ``(f) Reports to Congress.--The Coordinating Committee under 
subsection (b)(1) shall biennially submit to the Committee on 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 Duchenne muscular 
dystrophy being conducted or supported through the national research 
institutes, and that in addition includes the following:
            ``(1) The plan under subsection (c)(1) (or revisions to the 
        plan, as the case may be).
            ``(2) Provisions specifying the amounts expended by the 
        National Institutes of Health with respect to Duchenne 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 Duchenne muscular dystrophy.
    ``(g) Public Input.--The Director shall under subsection (a)(1) 
provide for means through which the public can obtain information on 
the existing and planned programs and activities of the National 
Institutes of Health with respect to Duchenne muscular dystrophy and 
through which the Director 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 the fiscal years 2001 through 2005. 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 Duchenne 
muscular dystrophy.''.

SEC. 4. DEVELOPMENT AND EXPANSION OF ACTIVITIES OF CENTERS FOR DISEASE 
              CONTROL AND PREVENTION WITH RESPECT TO EPIDEMIOLOGICAL 
              RESEARCH ON DUCHENNE 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 
section:

   ``surveillance and research regarding duchenne muscular dystrophy

    ``Sec. 317Q. (a) In General.--The Secretary, acting through the 
Director of the Centers for Disease Control and Prevention, may make 
awards of grants and cooperative agreements for the collection, 
analysis, and reporting of data on Duchenne muscular dystrophy. An 
entity may receive such an award only if the entity is a public or 
nonprofit private entity (including health departments of States and 
subdivisions of States, and including universities and other 
educational entities). In making such awards, the Secretary may provide 
direct technical assistance in lieu of cash.
    ``(b) National Duchenne Muscular Dystrophy Surveillance Program.--
            ``(1) In general.--The Secretary of Health and Human 
        Services (in this section referred to as the `Secretary'), 
        acting through the Director of the Centers for Disease Control 
        and Prevention, may make awards of grants and cooperative 
        agreements for the collection, analysis, and reporting of data 
        on Duchenne muscular dystrophy. In making such awards, the 
        Secretary may provide direct technical assistance in lieu of 
        cash.
            ``(2) Eligibility.--To be eligible to receive an award 
        under paragraph (1) an entity shall be a public or nonprofit 
        private entity (including health departments of States and 
        political subdivisions of States, and including universities 
        and other educational entities).
    ``(c) Centers of Excellence in Duchenne and Related Muscular 
Dystrophies Epidemiology.--
            ``(1) In general.--The Secretary, acting through the 
        Director of the Centers for Disease Control and Prevention, 
        shall establish not less than three regional centers of 
        excellence in Duchenne muscular dystrophy epidemiology for the 
        purpose of collecting and analyzing information on the number, 
        incidence, correlates, and symptoms of Duchenne and related 
        muscular dystrophies.
            ``(2) Recipients of awards for establishment of centers.--
        Centers under paragraph (1) shall be established and operated 
        through the awarding of grants or cooperative agreements to 
        public or nonprofit private entities that conduct research, 
        including health departments of States and political 
        subdivisions of States, and including universities and other 
        educational entities.
            ``(3) Certain requirements.--An award for a center under 
        paragraph (1) may be made only if the entity involved submits 
        to the Secretary an application containing such agreements and 
        information as the Secretary may require, including an 
        agreement that the center involved will operate in accordance 
        with the following:
                    ``(A) The center will collect, analyze, and report 
                Duchenne muscular dystrophy data according to 
                guidelines prescribed by the Director, after 
                consultation with relevant State and local public 
                health officials, private sector researchers, and 
                advocates for those with Duchenne muscular dystrophy.
                    ``(B) The center will assist with the development 
                and coordination of State Duchenne and related muscular 
                dystrophy surveillance efforts within a region.
                    ``(C) The center will identify eligible cases and 
                controls through its surveillance systems and conduct 
                research into factors which may cause Duchenne muscular 
                dystrophy.
                    ``(D) The center will develop or extend an area of 
                special research expertise (including genetics, 
                immunology, and other relevant research specialty 
                areas).
    ``(d) Definition.--In this title, the term `State' means each of 
the several States, the District of Columbia, the Commonwealth of 
Puerto Rico, American Samoa, Guam, the Commonwealth of the Northern 
Mariana Islands, the Virgin Islands, and the Trust Territory of the 
Pacific Islands.
    ``(e) 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 shall establish and implement a 
program to provide information and education on Duchenne muscular 
dystrophy to health professionals and the general public, including 
information and education on advances in the diagnosis and treatment of 
Duchenne muscular dystrophy and training and continuing education 
through programs for scientists, physicians, and other health 
professionals who provide care for patients with Duchenne muscular 
dystrophy.
    (b) Stipends.--The Secretary may use amounts made available under 
this section to provide 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. INTER-AGENCY MUSCULAR DYSTROPHY COORDINATING COMMITTEE.

    (a) Establishment.--The Secretary shall establish a committee to be 
known as the ``Muscular Dystrophy Coordinating Committee'' (in this 
section referred to as the ``Committee'') to coordinate all efforts 
within the Department of Health and Human Services concerning Duchenne 
muscular dystrophy, including activities carried out through the 
National Institutes of Health and the Centers for Disease Control and 
Prevention under this Act (and the amendments made by this Act).
    (b) Membership.--
            (1) In general.--The Committee shall be composed of the 
        Directors of such national research institutes, of the Centers 
        for Disease Control and Prevention, and of such other agencies 
        and such other officials as the Secretary determines 
        appropriate.
            (2) Additional members.--If determined appropriate by the 
        Secretary, the Secretary may appoint to the Committee--
                    (A) parents or legal guardians of individuals with 
                Duchenne muscular dystrophy; and
                    (B) representatives of other governmental agencies 
                that serve children with Duchenne muscular dystrophy 
                such as the Department of Education.
    (c) Administrative Support; Terms of Service; Other Provisions.--
The following shall apply with respect to the Committee:
            (1) The Committee shall receive necessary and appropriate 
        administrative support from the Department of Health and Human 
        Services.
            (2) Members of the Committee appointed under subsection 
        (b)(2)(A) shall serve for a term of 3 years, and may serve for 
        an unlimited number of terms if reappointed.
            (3) The Committee shall meet not less than two times each 
        year.

SEC. 7. 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 title and the 
amendments made by this title.

SEC. 8. DEFINITION.

    For purposes of this Act, the term ``Secretary'' means the 
Secretary of Health and Human Services.
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