SECTION 1. SHORT TITLE.

    This Act may be cited as the ``SMA Treatment Acceleration Act''.

SEC. 2. FINDINGS.

    The Congress makes the following findings:
            (1) Spinal muscular atrophy (SMA) is the number one genetic 
        killer of children under the age of 2.
            (2) SMA is an inherited and often fatal disease that 
        destroys the nerves controlling voluntary muscle movement, 
        which affects crawling, walking, head and neck control, and 
        even swallowing.
            (3) It is estimated that SMA occurs in nearly 1 of every 
        6,000 births and is therefore similar in incidence and severity 
        to other well-known genetic diseases such as cystic fibrosis 
        and Duchenne muscular dystrophy, both of which may also benefit 
        from additional focus and progress on SMA.
            (4) SMA is caused by the mutation of a single gene. This is 
        extremely advantageous for genetic screening and therapeutic 
        development. The gene mutation that causes SMA is carried by 
        one in every 40 people, or approximately 7,500,000 Americans. 
        Each child of 2 carriers of the mutant gene has a 1 in 4 chance 
        of developing SMA.
            (5) The discovery of the gene responsible for the disease, 
        SMN1, as well as a disease modifying ``back-up'' SMN2 gene has 
        opened the door to new SMA treatments. Modulating genes exist 
        not only for SMA but also for other genetic disorders, 
        including Duchenne Muscular Dystrophy, Parkinson's, and 
        Alzheimer's disease. The modulation of these genes might be 
        expected to impact these disorders. Success with SMN2 induction 
        for SMA will serve as an important proof of principle and 
        impetus for ongoing research in these other conditions.
            (6) Based on the advanced genetic understanding of SMA, the 
        disease was selected by the National Institutes of Health (NIH) 
        and the National Institute of Neurological Disorders and Stroke 
        (NINDS) as the prototype for the National Institutes of 
        Health's accelerated drug discovery effort, singling out SMA as 
        the disease closest to treatment of more than 600 neurological 
        disorders.
            (7) In 2003, the National Institute of Neurological 
        Disorders and Stroke (NINDS) established the Spinal Muscular 
        Atrophy Project: A Collaborative Program to Accelerate 
        Therapeutics Development for SMA. The SMA Project's unique 
        collaborative process between private, public, and non-profit 
        partners provides a model translational research program that 
        can be replicated to accelerate the development of safe and 
        effective treatments for a wide variety of disorders.
            (8) National non-profit organizations dedicated to finding 
        a treatment and cure for SMA continue to provide substantial 
        private funding and are collaborating with private 
        biotechnology companies, large pharmaceutical companies, and 
        clinical investigators to identify new drug compounds and 
        facilitate the rapid translation of promising new therapies to 
        individuals with SMA. The aforementioned investment by national 
        non-profit organizations towards finding a treatment and cure 
        for SMA is approximately equal, on an annual basis, to the 
        resources committed by the Federal Government.
            (9) A Food and Drug Administration-approved SMA animal 
        model exists that closely mimics the human disease. A number of 
        therapeutics have been identified which are effective in animal 
        models of spinal muscular atrophy.
            (10) There is an urgent need to provide Federal support 
        enabling investigators to mount national clinical trials to 
        demonstrate that these treatments are safe and effective for 
        SMA patients.
            (11) The establishment and support of a national clinical 
        trials network and a data coordinating center will promote 
        rigorous patient evaluation using common protocols and allow 
        investigators to study large numbers of patients to provide 
        answers more rapidly than individual sites acting alone.
            (12) There is a demonstrated need for greater interagency 
        coordination on SMA research and involvement by additional 
        government partners to support the ongoing work of NINDS on the 
        SMA Project as well the work of private SMA voluntary 
        organizations, including most notably the need for active 
        engagement by the National Institute of Child Health and Human 
        Development (NICHD), along with support from the National 
        Center for Research Resources, the Centers for Disease Control 
        and Prevention, the Food and Drug Administration, and the 
        Health Resources and Services Administration
            (13) Despite such landmark legislation as the Orphan Drug 
        Act and the Best Pharmaceuticals for Children Act, additional 
        incentives for industry to engage early in the drug development 
        process and through to drug approval are warranted for diseases 
        as severe and devastating in infant and children populations as 
        SMA.
            (14) 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. Furthermore, 
        greater awareness of SMA may lead to the identification of pre-
        symptomatic SMA-afflicted children, which has significant 
        benefits relative to clinical trials and the search for a 
        treatment and cure.

SEC. 3. CLINICAL TRIALS NETWORK FOR SPINAL MUSCULAR ATROPHY.

    (a) Clinical Trials Network.--The Director of NIH, in coordination 
with the Directors of the National Institute of Neurological Disorders 
and Stroke and the National Institute of Child Health and Human 
Development, shall provide for the upgrading and unification of 
existing SMA clinical trial sites to establish a national clinical 
trials network for SMA. The Director of NIH shall ensure that such 
network--
            (1) conducts coordinated, multisite, clinical trials of 
        pharmacological approaches to the treatment of SMA; and
            (2) rapidly and efficiently disseminates scientific 
        findings to the field.
    (b) Data Coordinating Center.--The Director of NIH, in coordination 
with the Directors of the National Institute of Neurological Disorders 
and Stroke and the National Institute of Child Health and Human 
Development, shall establish a data coordinating center with respect to 
SMA to--
            (1) provide expert assistance in the design, conduct, data 
        analysis, and data management of collaborative clinical and 
        descriptive research projects;
            (2) provide appropriate and capable leadership and 
        expertise in biostatistics, developmental study design, data 
        management, data analysis, and project management, including 
        staff and site training and quality assurance procedures;
            (3) provide research support activities in designing data 
        collection modules, operational and procedure manuals, quality 
        control systems, and a communications system for clinical site 
        principal investigators, research coordinators, and other 
        network staff;
            (4) organize and conduct multi-site monitoring activities; 
        and
            (5) provide regular reports to the National Institute of 
        Neurological Disorders and Stroke and the National Institute of 
        Child Health and Human Development on enrollment and the 
        allocation of resources.
    (c) Pre-Clinical Activities.--The Director of NIH, in coordination 
with the Directors of the National Institute of Neurological Disorders 
and Stroke and the National Institute of Child Health and Human 
Development, shall expand and intensify programs of such Institutes 
with respect to pre-clinical translation research and medicinal 
chemistry related to SMA.

SEC. 4. NATIONAL PATIENT REGISTRY.

    (a) In General.--The Secretary of Health and Human Services, acting 
through the Director of the Centers for Disease Control and Prevention, 
shall enhance and provide ongoing support to the existing SMA patient 
registry to provide for expanded research on the epidemiology of SMA.
    (b) Longitudinal Data.--In carrying out subsection (a), the 
Secretary shall ensure the collection and analysis of longitudinal data 
related to individuals of all ages with SMA, including infants, young 
children, adolescents, and adults of all ages.

SEC. 5. NIH COORDINATING COMMITTEE ON SMA.

    (a) Coordinating Committee.--
            (1) In general.--The Secretary shall establish the Spinal 
        Muscular Atrophy Coordinating Committee to coordinate 
        activities across the National Institutes of Health and with 
        other Federal health programs and activities relating to SMA.
            (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--
                            (i) the Directors (or their designees) of 
                        the National Institute of Neurological 
                        Disorders and Stroke, the National Institute of 
                        Child Health and Human Development, other 
                        national research institutes involved in 
                        research with respect to SMA, and the National 
                        Center for Research Resources;
                            (ii) representatives of all other Federal 
                        departments, agencies, and advisory committees 
                        whose programs involve health functions or 
                        responsibilities relevant to SMA, including the 
                        Centers for Disease Control and Prevention, the 
                        Health Resources and Services Administration, 
                        the Food and Drug Administration, and the 
                        Advisory Committee on Heritable Disorders and 
                        Genetic Diseases in Newborns and Children; and
                            (iii) representatives of other governmental 
                        agencies that serve children with SMA, 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 SMA, including parents or legal guardians, 
                affected individuals, researchers, and clinicians.
            (3) Term.--Members of the Coordinating Committee appointed 
        under paragraph (2)(B) shall be appointed for a term of 3 
        years, and may serve for an unlimited number of terms if 
        reappointed.
            (4) Chair.--
                    (A) In general.--With respect to SMA, 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.
                    (B) Appointment.--The Secretary shall appoint the 
                Chair of the Coordinating Committee from among 
                individuals nominated by the Coordinating Committee. 
                The Chair shall be appointed for a term not to exceed 2 
                years and may be reappointed for not more than 1 
                additional term.
            (5) Administrative support; terms of service; other 
        provisions.--The following shall apply with respect to the 
        Coordinating Committee:
                    (A) The Secretary shall provide the Coordinating 
                Committee with necessary and appropriate administrative 
                support.
                    (B) The Coordinating Committee shall meet as 
                determined appropriate by the Secretary, in 
                consultation with the Chair of the Coordinating 
                Committee, but no less than twice each year.
    (b) Study on Barriers to Drug Development.--
            (1) Study.--The Coordinating Committee shall conduct a 
        study to identify current and potential future barriers to the 
        development of drugs for treating SMA and other similar genetic 
        disorders. Such study shall--
                    (A) identify barriers related to the activities of 
                government, industry, and academic medicine;
                    (B) include substantial input from scientists and 
                organizations with direct involvement in SMA research 
                and drug development; and
                    (C) consider obstacles to drug development at all 
                points along the research continuum from preclinical 
                research to new drug approval.
            (2) Report to congress.--Not later than 1 year after the 
        date of the enactment of this Act, the Coordinating Committee 
        shall submit to the Congress a report on the results of the 
        study described in paragraph (1) together with such 
        recommendations for legislation or administrative action as the 
        Coordinating Committee determines appropriate.

SEC. 6. NIH TRANS-INSTITUTE COLLABORATION ON SMA RESEARCH.

    (a) In General.--To ensure the success of the SMA Project that was 
initiated and has been led by National Institute of Neurological 
Disorders and Stroke, the Director of NIH shall establish a trans-
National Institutes of Health cooperative research initiative on SMA.
    (b) Duties.--The cooperative research initiative established under 
subsection (a) shall consist of the following activities:
            (1) The Director of the National Institute of Neurological 
        Disorders and Stroke shall report to the Director of NIH on the 
        ongoing needs of the SMA Project and required next steps to 
        ensure the continued success of the Project.
            (2) Based on the needs of the SMA Project identified in the 
        report required by paragraph (1), the Director of the National 
        Institute of Child Health and Human Development shall provide 
        direct and ongoing support of SMA research and drug 
        development.
            (3) The Director of NIH shall identify and promote 
        opportunities for greater collaboration and involvement in SMA 
        research and drug development by other national research 
        institutes.

SEC. 7. DRUG DEVELOPMENT PROMOTION.

    Not later than 6 months after the date of the enactment of this 
Act, the Secretary, in direct consultation with the Commissioner of 
Food and Drugs and the Coordinating Committee, shall submit specific 
recommendations to the Congress to improve and expand on the incentives 
provided pursuant to the Orphan Drug Act (Public Law 97-414) and 
related statutes to directly and indirectly promote SMA drug 
development, such as through the creation of unique incentives for rare 
pediatric treatments.

SEC. 8. EDUCATION AND AWARENESS ON SMA FOR HEALTH CARE PROFESSIONALS.

    (a) In General.--The Secretary shall establish and implement a 
program to provide information and education on SMA to health 
professionals and the general public, including information and 
education on advances in the diagnosis and treatment of SMA and 
training and continuing education through programs for scientists, 
physicians, medical students, and other health professionals who 
provide care for patients with SMA.
    (b) Stipends.--The Secretary may award stipends to health 
professionals who are enrolled in training programs under this section.

SEC. 9. DEFINITIONS.

    In this Act:
            (1) The term ``Director of NIH'' means the Director of the 
        National Institutes of Health.
            (2) The term ``Coordinating Committee'' means the Spinal 
        Muscular Atrophy Coordinating Committee.
            (3) The term ``Secretary'' means the Secretary of Health 
        and Human Services.
            (4) The term ``SMA'' means spinal muscular atrophy.
SMA Treatment Acceleration Act - Requires the Director of the National Institutes of Health (NIH) to upgrade and unify existing spinal muscular atrophy (SMA) clinical trial sites to establish a national clinical trials network for SMA. Requires the Director of NIH to ensure that such network: (1) conducts coordinated, multisite, clinical trials of pharmacological approaches to the treatment of SMA; and (2) rapidly and effectively disseminates scientific findings to the field.

Requires the Director of NIH to: (1) establish an SMA data coordinating center; and (2) expand and intensify NIH programs with respect to preclinical translation research and medicinal chemistry related to SMA.

Requires the Secretary of Health and Human Services, acting through the Director of the Centers for Disease Control and Prevention (CDC), to enhance and provide ongoing support to the existing SMA patient registry to provide for expanded research on the epidemiology of SMA. Directs the Secretary to ensure the collection and analysis of longitudinal data related to individuals of all ages with SMA.

Requires the Secretary to establish the Spinal Muscular Atrophy Coordinating Committee to: (1) coordinate activities across NIH and with other federal health programs and activities relating to SMA; and (2) conduct a study to identify barriers to the development of drugs for treating SMA and similar genetic disorders.

Requires the Director of NIH to establish a trans-National Institutes of Health cooperative research initiative on SMA to ensure the success of the SMA projects led by the National Institute of Neurological Disorders and Stroke.

Requires the Secretary to submit recommendations to Congress to improve and expand on incentives provided under the Orphan Drug Act and related statutes to promote SMA drug development.
{"src": "billsum_test", "title": "To authorize the Secretary of Health and Human Services to conduct activities to rapidly advance treatments for spinal muscular atrophy, neuromuscular disease, and other pediatric diseases, and for other purposes."}