SMA Treatment Acceleration Act of 2009 - Amends the Public Health Service Act to require the Director of the National Institutes of Health (NIH) to establish a national clinical trials network for spinal muscular atrophy by upgrading and unifying spinal muscular atrophy clinical trial sites and recruiting new investigators and sites. Requires the Director to ensure that such network: (1) conducts coordinated, multisite, clinical trials of therapies and clinical approaches to the treatment of spinal muscular atrophy; and (2) rapidly and efficiently disseminates scientific findings to the field.
Requires the Director to: (1) establish a data coordinating center with respect to spinal muscular atrophy; and (2) expand and intensify NIH programs with respect to preclinical translation research related to spinal muscular atrophy.
Requires the Secretary of Health and Human Services, acting through the Director of the Centers for Disease Control and Prevention (CDC), to enhance and support a spinal muscular atrophy patient registry to provide for expanded epidemiological research towards improving awareness, management, treatment, and prevention of spinal muscular atrophy. Requires the Secretary to ensure the collection and analysis of longitudinal data related to individuals of all ages.
Directs the Secretary to establish the Interagency Spinal Muscular Atrophy Research Coordinating Committee. Sets forth the duties of the Committee, including to develop a comprehensive strategy related to spinal muscular atrophy research and other related neurological diseases and disorders.
Requires the Secretary to establish a program to provide information and education on spinal muscular atrophy to health professionals and the general public.
[Congressional Bills 111th Congress]
[From the U.S. Government Publishing Office]
[H.R. 2149 Introduced in House (IH)]
111th CONGRESS
1st Session
H. R. 2149
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.
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
April 28, 2009
Mr. Kennedy (for himself and Mr. Cantor) introduced the following bill;
which was referred to the Committee on Energy and Commerce
_______________________________________________________________________
A BILL
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.
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 ``SMA Treatment Acceleration Act of
2009''.
SEC. 2. CLINICAL TRIALS NETWORK FOR SPINAL MUSCULAR ATROPHY.
Part B of title IV of the Public Health Service Act is amended by
adding at the end the following new section:
``SEC. 409J. 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, the National Institute of Child
Health and Human Development, and such other Institutes and Centers as
specified by the Director shall provide for the upgrading and
unification of spinal muscular atrophy clinical trial sites and the
recruitment of new investigators and sites to establish a national
clinical trials network for spinal muscular atrophy. The Director of
NIH shall ensure that such network--
``(1) conducts coordinated, multi-site, clinical trials of
therapies and clinical approaches to the treatment of spinal
muscular atrophy; and
``(2) rapidly and efficiently disseminates scientific
findings to the field.
``(b) Data Coordinating Center.--The Director of NIH, in
coordination with the Commissioner of Food and Drugs and the Directors
of the National Institute of Neurological Disorders and Stroke, the
National Institute of Child Health and Human Development, and such
other Institutes and Centers as specified by the Director, shall
establish a data coordinating center with respect to spinal muscular
atrophy to--
``(1) provide expert assistance in the design, conduct,
data analysis, data management, and data warehousing of
collaborative clinical and descriptive research projects;
``(2) organize and conduct multi-site monitoring
activities;
``(3) provide regular reports to the National Institute of
Neurological Disorders and Stroke, the National Institute of
Child Health and Human Development, such other Institutes and
Centers as specified by the Director, and the Food and Drug
Administration on enrollment and the allocation of resources;
and
``(4) conduct such other activities as are deemed necessary
by the Secretary.
``(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
related to spinal muscular atrophy.''.
SEC. 3. NATIONAL PATIENT REGISTRY.
Part P of title III of the Public Health Service Act is amended by
adding at the end the following new section:
``SEC. 399S. NATIONAL SPINAL MUSCULAR ATROPHY PATIENT REGISTRY.
``(a) In General.--The Secretary, acting through the Director of
the Centers for Disease Control and Prevention and in coordination with
the Director of the NIH, shall enhance and provide ongoing support to
an existing or new spinal muscular atrophy patient registry to provide
for expanded epidemiological research towards improving awareness,
management, treatment, and prevention of spinal muscular atrophy.
``(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 spinal muscular atrophy,
including infants, young children, adolescents, and adults of all
ages.''.
SEC. 4. INTERAGENCY SPINAL MUSCULAR ATROPHY RESEARCH COORDINATING
COMMITTEE.
Part B of title IV of the Public Health Service Act, as amended by
section 2, is further amended by adding at the end the following new
section:
``SEC. 409K. INTERAGENCY SPINAL MUSCULAR ATROPHY RESEARCH COORDINATING
COMMITTEE.
``(a) Establishment.--Not later than 6 months after the date of the
enactment of this section, the Secretary shall establish a committee,
to be known as the Interagency Spinal Muscular Atrophy Research
Coordinating Committee (in this section referred to as the
`Committee').
``(b) Duties.--The Committee shall--
``(1) share and coordinate information on existing research
activities, and make recommendations to the National Institutes
of Health and other Federal agencies regarding how to improve
existing research programs, that are related to spinal muscular
atrophy research and other related neurological diseases and
disorders;
``(2) develop a comprehensive strategy related to spinal
muscular atrophy research and other related neurological
diseases and disorders and advise the National Institutes of
Health and other Federal agencies, expanding proposals for
collaborative, multidisciplinary research, including proposals
for Common Fund research described in section 402(b)(7) and
other proposals that involve collaboration between 2 or more
national research institutes or national centers;
``(3) provide annual reports to the Secretary regarding the
National Institutes of Health and other Federal agencies'
collaborative multidisciplinary research efforts to support
spinal muscular atrophy, including the Spinal Muscular Atrophy
Project at the National Institute of Neurological Disorders and
Stroke, the ongoing and future research needs to advance
therapies for spinal muscular atrophy, and recommendations on
how to strengthen the collaboration of research activities by
the institutes and agencies to improve the results;
``(4) develop a summary of advances in research related to
spinal muscular atrophy research and other related neurological
diseases and disorders research supported or conducted by
Federal agencies; and
``(5) not later than 1 year after the date of the
establishment of the Committee, make recommendations to the
Secretary--
``(A) regarding any appropriate changes to research
activities, including recommendations to improve the
research portfolio of the National Institutes of Health
to ensure that scientifically based strategic planning
is implemented in support of research priorities that
impact research activities related to spinal muscular
atrophy and other related neurological diseases and
disorders;
``(B) identifying barriers to the development of
new treatments and cures for spinal muscular atrophy
and other related neurological diseases and disorders;
``(C) regarding public participation in decisions
relating to spinal muscular atrophy research and other
related neurological diseases and disorders to increase
the involvement of patient advocacy and community
organizations representing a broad geographical area;
``(D) on how best to disseminate information on
spinal muscular atrophy progress; and
``(E) on how to expand partnerships between public
entities, including Federal agencies, and private
entities to expand collaborative, cross-cutting
research.
``(c) Rule of Construction.--In carrying out the duties described
in subsection (b) with respect to research on spinal muscular atrophy,
nothing in this section shall be construed to restrict the Secretary
from including other neurological or genetic disorders, as appropriate,
in such research if doing so may advance research in spinal muscular
atrophy or other related neurological diseases and disorders.
``(d) Membership.--
``(1) In general.--The Committee shall be composed of the
following voting members:
``(A) Not more than 11 voting Federal
representatives as follows:
``(i) The Director of the Centers for
Disease Control and Prevention.
``(ii) The Director of the National
Institutes of Health and the directors of such
national research institutes and national
centers (which shall include the National
Institute of Neurological Disorders and Stroke,
the National Institute of Child Health and
Human Development, and the National Center for
Research Resources) as the Secretary determines
appropriate.
``(iii) The Commissioner of Food and Drugs.
``(iv) The heads of such other agencies and
advisory committees as the Secretary determines
appropriate, including the Health Resources and
Services Administration, the Agency for
Healthcare Research and Quality, and the
Advisory Committee on Heritable Disorders and
Genetic Diseases in Newborns and Children.
``(v) Representatives of other Federal
agencies that conduct or support neurological
research, or provide support services and
resources for individuals with Spinal Muscular
Atrophy, such as the Department of Education
and the Social Security Administration.
``(B) 9 additional voting members appointed under
paragraph (2).
``(2) Additional members.--The Committee shall include
additional voting members appointed by the Secretary as
follows:
``(A) 6 members shall be appointed from among
scientists, physicians, and other health professionals,
who--
``(i) are not officers or employees of the
United States;
``(ii) represent multiple disciplines,
including clinical, basic, and public health
sciences;
``(iii) represent different geographical
regions of the United States;
``(iv) are from practice settings,
academia, or other research settings; and
``(v) are experienced in scientific peer
review process.
``(B) 3 members shall be appointed from members of
the general public, who represent individuals with
spinal muscular atrophy.
``(3) Nonvoting members.--The Committee shall include such
nonvoting members as the Secretary determines to be
appropriate.
``(e) Chairperson.--The voting members of the Committee shall
select a chairperson from among the Federal members of the Committee
described in subsection (d)(1)(A). The selection of a chairperson may
be subject to the approval of the Secretary.
``(f) Meetings.--The Committee shall meet at the call of the
chairperson of the Committee or upon the request of the Secretary, but
in no case less often than once each year.
``(g) Review.--In 2012, and biennially thereafter, the Secretary
shall review the necessity of the Committee.''.
SEC. 5. EDUCATION AND AWARENESS ON SMA FOR HEALTH CARE PROFESSIONALS.
Part P of title III of the Public Health Service Act, as amended by
section 3, is further amended by adding at the end the following new
section:
``SEC. 399T. INFORMATION AND EDUCATION ON SMA.
``The Secretary shall establish and implement a program to provide
information and education on spinal muscular atrophy to health
professionals and the general public, including information and
education on advances in the screening, diagnosis, and treatment of
spinal muscular atrophy and training and continuing education through
programs for scientists, physicians, medical students, and other health
professionals who provide care for patients with spinal muscular
atrophy.''.
<all>
Introduced in House
Introduced in House
Sponsor introductory remarks on measure. (CR E993)
Referred to the House Committee on Energy and Commerce.
Referred to the Subcommittee on Health.
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