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

Summary: Paul D. Wellstone Muscular Dystrophy Community Assistance, Research, and Education Amendments of 2008 - Amends the Public Health Service Act to designate centers of excellence for research on various forms of muscular dystrophy as Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers.

Requires the Muscular Dystrophy Interagency Coordinating Committee to give special consideration to enhancing the clinical research infrastructure required to test emerging therapies for the various forms of muscular dystrophy.

Requires the Secretary of Health and Human Services to ensure that any data on patients that is collected as part of the Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet) is regularly updated to reflect changes in patient condition over time. Requires the Director of the Centers for Disease Control and Prevention (CDC) to: (1) report to the appropriate congressional committees on MD STARnet and data collection; and (2) make publicly available prospective health outcome data on the health and survival of people with muscular dystrophy.

Requires the Director of CDC, in carrying out a program to provide information and education on muscular dystrophy to health professionals and the general public, to: (1) partner with leaders in the muscular dystrophy patient community; and (2) widely disseminate the Duchenne-Becker muscular dystrophy care considerations.

Requires the Director of the Agency for Healthcare Research and Quality to: (1) evaluate the available scientific evidence to develop and issue an initial set of care considerations for Duchenne-Becker muscular dystrophy and provide ongoing review and updates where appropriate; and (2) replicate the same systematic review methodology used to develop such care considerations as a model for other muscular dystrophies.

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Paul D. Wellstone Muscular Dystrophy 
Community Assistance, Research, and Education Amendments Act of 2008''.

SEC. 2. FINDINGS.

    Congress makes the following findings:
            (1) The muscular dystrophies are devastating diseases that 
        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.
            (2) Duchenne muscular dystrophy (referred to in this 
        section as ``DMD'') is the most common lethal genetic disorder 
        of childhood worldwide, affecting approximately 1 in every 
        3,500 boys born each year around the globe. It is characterized 
        by a rapidly progressive muscle weakness that almost always 
        results in death from respiratory or cardiac failure, typically 
        in the late teens or twenties.
            (3) Myotonic muscular dystrophy is the second most 
        prominent form of muscular dystrophy and the type most commonly 
        found in adults, affecting an estimated 1 in 8,000 people. 
        However, it can affect people of any age, from birth to old 
        age. Described as the most variable disease known in medicine, 
        it is multisystemic and can cause not only muscle atrophy and 
        myotonia, but also serious cardiac, respiratory, endocrine, 
        gastrointestinal, skeletal and central nervous system 
        complications, as well as problems with the eyes, teeth, and 
        hair. As it passes from one generation to the next, it 
        generally worsens with earlier onset. Congenital myotonic 
        muscular dystrophy is the most severe form of myotonic muscular 
        dystrophy affecting infants and causing severe cognitive 
        delays. It often causes sudden death; however, others can live 
        for many years with this slowly degenerative disorder.
            (4) Facioscapulohumeral muscular dystrophy (referred to in 
        this section as ``FSHD'') is the second most prevalent adult 
        muscular dystrophy and the third most prevalent muscular 
        dystrophy of men, women and children. It is inherited 
        genetically and has an estimated incidence of 1 in 20,000 
        persons. Many leading FSHD scientists note that the prevalence 
        may be 3 times higher due to undiagnosed and misdiagnosed 
        cases. FSHD, affecting between 15,000 to 40,000 persons, causes 
        a lifelong progressive and severe loss of all skeletal muscles 
        gradually bringing weakness and reduced mobility. It is 
        genetically transmitted to children, can occur spontaneously, 
        and may affect entire families. Persons with FSHD may also 
        experience hearing loss, vision problems, and respiratory 
        insufficiency; some may become severely physically disabled and 
        spend decades in a wheelchair and on a ventilator. FSHD is 
        caused by a novel epigenetic phenomenon not found in other 
        forms of muscular dystrophy and is caused by a contraction of 
        repetitive DNA previously thought to be ``junk DNA''. The 
        unique epigenetic structure of FSHD is unprecedented in other 
        muscular dystrophies and genetic disorders and demands novel 
        approaches and new research groups. Understanding this 
        mechanism will have great benefit to other areas of biomedical 
        research including cancer and other disease of epigenetic 
        origin.
            (5) Congenital muscular dystrophies represent a group of 
        distinct diseases, which begin at birth, with varying severity 
        and involvement of both muscle strength and brain. These 
        diseases often lead to premature infant death, or severely 
        disabled young children who require 24-hour care given their 
        developmental delay compounded by muscle weakness. Other 
        children live to young adulthood and typically require the use 
        of a wheelchair for mobility.
            (6) Forms of muscular dystrophy affecting children and 
        adults include Becker, congenital, distal, Duchenne, Emery-
        Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and 
        oculopharyngeal muscular dystrophies. The limb-girdle muscular 
        dystrophies are of 15 known different types.
            (7) Each of the muscular dystrophies, though distinct in 
        progressivity and severity of symptoms, has a devastating 
        impact on hundreds of thousands of children and adults 
        throughout the United States and worldwide, as well as imposes 
        severe physical and economic burdens on those affected. In many 
        of the muscular dystrophies, there are associated medical 
        problems arising from pulmonary issues, respiratory 
        insufficiency, cardiomyopathy, which in many cases is the cause 
        of death for persons with muscular dystrophy.
            (8) In the 5 years since enactment of the Muscular 
        Dystrophy Community Assistance, Research and Education 
        Amendments of 2001 (the MD-CARE Act (Public Law 107-84)), and 
        due directly to the momentum established by such Act, progress 
        has been made in the battle against the muscular dystrophies.
            (9) Investments made by the Federal Government as a result 
        of the MD-CARE Act include the creation of the MD Coordinating 
        Committee (MDCC), the development of the MDCC Action Plan, 
        expansion of the National Institutes of Health (referred to in 
        this section as the ``NIH'') research portfolios, establishment 
        of 6 Paul D. Wellstone Muscular Dystrophy Cooperative Research 
        Centers, funding of a $15,400,000 National Institutes of Health 
        U54 grant and others focused on DMD, development of the 
        Muscular Dystrophy Surveillance, Tracking and Research Network 
        (MD STARnet), and the launch of a comprehensive education and 
        outreach initiative.
            (10) In the past few years, the NIH program in 
        translational research in muscular dystrophy has grown 
        significantly and funded a number of large-scale projects to 
        further the development of therapies for muscular dystrophy. As 
        part of this program, the National Institute of Neurological 
        Disorders and Stroke (NINDS) and the National Institute of 
        Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the 
        National Institutes of Health (NIH) awarded a $15,400,000, 5-
        year cooperative agreement to develop new small molecule drugs 
        for the treatment of DMD and potentially other forms of 
        muscular dystrophy as well. The project is a unique research 
        collaboration between private, public, and nonprofit partners 
        to build upon previous research and discovery work originally 
        initiated by non-profit partners to identify new treatments for 
        muscular dystrophy. Also through the translational program, 3 
        other major cooperative agreements have been awarded for highly 
        targeted therapy development projects in the muscular 
        dystrophies.
            (11) Due to the initiatives made possible through the MD-
        CARE Act, national nonprofit organizations have joined in model 
        strategic collaborations with academic research institutions, 
        public funders of muscular dystrophy research, and industry to 
        expand investments in muscular dystrophy research activities 
        and to create new platforms for translational research. These 
        have led to the development of the first potential therapies 
        for DMD, myotonic, facioscapulohumeral, limb-girdle, and other 
        conditions that are proceeding through clinical trials.
            (12) Advancements in care have helped prolong life and 
        quality of life for patients with muscular dystrophy.
            (13) Notwithstanding these promising developments, the 
        majority of the directions envisioned by the Action Plan for 
        the Muscular Dystrophies, developed pursuant to the MD-CARE 
        Act, have not been realized. Where recent momentum has been 
        achieved, its sustainability is fragile and directly dependent 
        upon continued Federal support for the early phase planning and 
        programs created through the MD-CARE Act.
            (14) There remains a shortage of qualified researchers in 
        the field of muscular dystrophy research. Many family 
        physicians and health care professionals still lack the 
        knowledge and resources to detect and properly diagnose 
        muscular dystrophy as early as possible, thus delaying 
        management of symptoms in cases that go undetected or 
        misdiagnosed.
            (15) As new understandings of the genetic basis for disease 
        and potential treatment has emerged, the public and health care 
        communities are in urgent need of education and outreach to 
        ensure competent, informed engagement in genetic testing and 
        counseling and appropriate patient characterization so that 
        patients are able to participate in new avenues of research and 
        clinical trials.
            (16) As basic research into the muscular dystrophies points 
        the way to new therapeutic targets, there is an urgent need to 
        support the clinical research infrastructure necessary to bring 
        these therapeutic leads to human trials; these infrastructure 
        needs include validated endpoints, current natural history 
        studies, biomarkers, clinical research networks, patient 
        registries, and databases.
            (17) In order to improve lives and develop effective 
        treatments for individuals with muscular dystrophy, there must 
        be improved communications and partnerships between patients, 
        patient advocacy, researchers, and clinical care providers. To 
        that end, renewed effort to work together by all parties is a 
        critical element for successful outcomes in the years to come.
            (18) Continued focus and investment are required to build 
        on the current momentum, respond to public need, and ensure 
        that federally funded research and other innovation is 
        translated to therapeutic targets as quickly as possible.

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

    Section 404E of the Public Health Service Act (42 U.S.C. 283g) is 
amended--
            (1) in subsection (a)(1), by inserting ``the National 
        Heart, Lung, and Blood Institute,'' after ``Child Health and 
        Human Development,'';
            (2) in subsection (b)(1), by adding at the end the 
        following: ``Such centers of excellence shall be known as the 
        `Paul D. Wellstone Muscular Dystrophy Cooperative Research 
        Centers'.''; and
            (3) by adding at the end the following:
    ``(h) Clinical Research.--The Coordinating Committee shall give 
special consideration to the urgent need to enhance the clinical 
research infrastructure required to test emerging therapies for the 
various forms of muscular dystrophy by prioritizing the achievement of 
those goals in the plan related to this topic.
    ``(i) Authorization of Appropriations.--There is authorized to be 
appropriated to carry out this section, such sums as may be necessary 
for each of fiscal years 2008 through 2012.''.

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

    Section 317Q of the Public Health Service Act (42 U.S.C. 247b-18) 
is amended--
            (1) by redesignating subsection (d) as subsection (f); and
            (2) by inserting after subsection (c) the following:
    ``(d) Data.--In carrying out this section, the Secretary shall 
ensure that any data on patients that is collected as part of the 
Muscular Dystrophy Surveillance, Tracking and Research Network 
(referred to in this section as the `MD STARnet') under a grant under 
this section is regularly updated to reflect changes in patient 
condition over time, particularly with respect to any improvements 
realized through patient adherence to care considerations or 
utilization of a treatment or therapy.
    ``(e) Reports and Tracking.--
            ``(1) Annual report.--Not later than 18 months after the 
        date of enactment of the Paul D. Wellstone Muscular Dystrophy 
        Community Assistance, Research, and Education Amendments Act of 
        2008, and annually thereafter, the Director of the Centers for 
        Disease Control and Prevention shall submit to the appropriate 
        committees of Congress a report--
                    ``(A) concerning the activities carried out by MD 
                STARnet sites funded under this section during the year 
                for which the report is prepared;
                    ``(B) containing the data collected and findings 
                derived from the MD STARnet sites each fiscal year (as 
                funded under a grant under this section during the 
                periods of fiscal years 2008 through 2012); and
                    ``(C) that every 2 years outlines prospective data 
                collection objectives and strategies.
            ``(2) Tracking health outcomes.--The Director of the 
        Centers for Disease Control and Prevention shall make publicly 
        available prospective health outcome data on the health and 
        survival of people with muscular dystrophy.''.

SEC. 5. INFORMATION AND EDUCATION.

    Section 5 of the Muscular Dystrophy Community Assistance, Research 
and Education Amendments of 2001 (42 U.S.C. 247b-19) is amended--
            (1) by redesignating subsection (c) as subsection (d); and
            (2) by inserting after subsection (b), the following:
    ``(c) Requirements of the Centers for Disease Control and 
Prevention.--In carrying out this section, the Director of the Centers 
for Disease Control and Prevention shall--
            ``(1) partner with leaders in the muscular dystrophy 
        patient community; and
            ``(2) widely disseminate the Duchenne-Becker Muscular 
        Dystrophy care considerations described in section 904 as 
        broadly as possible, including through partnership 
        opportunities with the muscular dystrophy patient community.''.

SEC. 6. STANDARDS OF CARE.

    Part A of title IX of the Public Health Service Act (42 U.S.C. 299 
et seq.) is amended by adding at the end the following:

``SEC. 904. STANDARDS OF CARE RELATING TO MUSCULAR DYSTROPHY.

    ``The Director shall--
            ``(1) evaluate the available scientific evidence for the 
        appropriate medical or patient organizations for purposes of 
        the development and issuance of an initial set of care 
        considerations for Duchenne-Becker Muscular Dystrophy and 
        provide ongoing review and updates where appropriate; and
            ``(2) replicate the same systematic review methodology used 
        to develop the Duchenne-Becker Muscular Dystrophy care 
        considerations developed under paragraph (1) as a model for 
        other muscular dystrophies.''.