I 
111TH CONGRESS 
1ST SESSION H. R. 2149 
To authorize the Secretary of Health and Human Services to conduct activi-
ties to rapidly advance treatments for spinal muscular atrophy, neuro-
muscular 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 Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘SMA Treatment Accel-
eration Act of 2009’’. 
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SEC. 2. CLINICAL TRIALS NETWORK FOR SPINAL MUS-
CULAR 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 MUS-
CULAR ATROPHY. 
‘‘(a) CLINICAL TRIALS NETWORK.—The Director of 
NIH, in coordination with the Directors of the National 
Institute of Neurological Disorders and Stroke, the Na-
tional 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 re-
cruitment of new investigators and sites to establish a na-
tional 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 sci-
entific 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 
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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 In-
stitute of Neurological Disorders and Stroke, the 
National Institute of Child Health and Human De-
velopment, 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 Develop-
ment, shall expand and intensify programs of such Insti-
tutes with respect to pre-clinical translation research re-
lated to spinal muscular atrophy.’’. 
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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 PA-
TIENT REGISTRY. 
‘‘(a) IN GENERAL.—The Secretary, acting through 
the Director of the Centers for Disease Control and Pre-
vention 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 spi-
nal muscular atrophy. 
‘‘(b) LONGITUDINAL DATA.—In carrying out sub-
section (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 RE-
SEARCH 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: 
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‘‘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 Sec-
retary shall establish a committee, to be known as the 
Interagency Spinal Muscular Atrophy Research Coordi-
nating Committee (in this section referred to as the ‘Com-
mittee’). 
‘‘(b) DUTIES.—The Committee shall— 
‘‘(1) share and coordinate information on exist-
ing research activities, and make recommendations 
to the National Institutes of Health and other Fed-
eral agencies regarding how to improve existing re-
search programs, that are related to spinal muscular 
atrophy research and other related neurological dis-
eases and disorders; 
‘‘(2) develop a comprehensive strategy related 
to spinal muscular atrophy research and other re-
lated neurological diseases and disorders and advise 
the National Institutes of Health and other Federal 
agencies, expanding proposals for collaborative, mul-
tidisciplinary research, including proposals for Com-
mon Fund research described in section 402(b)(7) 
and other proposals that involve collaboration be-
tween 2 or more national research institutes or na-
tional centers; 
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‘‘(3) provide annual reports to the Secretary re-
garding the National Institutes of Health and other 
Federal agencies’ collaborative multidisciplinary re-
search 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 ad-
vance therapies for spinal muscular atrophy, and 
recommendations on how to strengthen the collabo-
ration 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 re-
search supported or conducted by Federal agencies; 
and 
‘‘(5) not later than 1 year after the date of the 
establishment of the Committee, make recommenda-
tions to the Secretary— 
‘‘(A) regarding any appropriate changes to 
research activities, including recommendations 
to improve the research portfolio of the Na-
tional Institutes of Health to ensure that sci-
entifically based strategic planning is imple-
mented in support of research priorities that 
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impact research activities related to spinal mus-
cular atrophy and other related neurological 
diseases and disorders; 
‘‘(B) identifying barriers to the develop-
ment of new treatments and cures for spinal 
muscular atrophy and other related neurological 
diseases and disorders; 
‘‘(C) regarding public participation in deci-
sions relating to spinal muscular atrophy re-
search and other related neurological diseases 
and disorders to increase the involvement of pa-
tient advocacy and community organizations 
representing a broad geographical area; 
‘‘(D) on how best to disseminate informa-
tion on spinal muscular atrophy progress; and 
‘‘(E) on how to expand partnerships be-
tween public entities, including Federal agen-
cies, and private entities to expand collabo-
rative, 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 mus-
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cular 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 rep-
resentatives as follows: 
‘‘(i) The Director of the Centers for 
Disease Control and Prevention. 
‘‘(ii) The Director of the National In-
stitutes 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 Administra-
tion, the Agency for Healthcare Research 
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and Quality, and the Advisory Committee 
on Heritable Disorders and Genetic Dis-
eases in Newborns and Children. 
‘‘(v) Representatives of other Federal 
agencies that conduct or support neuro-
logical research, or provide support serv-
ices and resources for individuals with Spi-
nal Muscular Atrophy, such as the Depart-
ment of Education and the Social Security 
Administration. 
‘‘(B) 9 additional voting members ap-
pointed 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, in-
cluding clinical, basic, and public health 
sciences; 
‘‘(iii) represent different geographical 
regions of the United States; 
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‘‘(iv) are from practice settings, aca-
demia, 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 Sec-
retary determines to be appropriate. 
‘‘(e) CHAIRPERSON.—The voting members of the 
Committee shall select a chairperson from among the Fed-
eral 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 re-
quest 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 Com-
mittee.’’. 
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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 pro-
gram to provide information and education on spinal mus-
cular atrophy to health professionals and the general pub-
lic, 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.’’. 
Æ 
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