II 

111TH CONGRESS 

1ST SESSION S. 1158 

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 SENATE OF THE UNITED STATES 

MAY 21, 2009 

Ms. STABENOW (for herself, Mr. ISAKSON, and Mr. WHITEHOUSE) introduced 

the following bill; which was read twice and referred to the Committee 

on Health, Education, Labor, and Pensions 

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. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘SMA Treatment Accel5 eration Act of 2009’’. 


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1 SEC. 2. CLINICAL TRIALS NETWORK FOR SPINAL MUS2 CULAR ATROPHY. 

3 Part B of title IV of the Public Health Service Act 

4 is amended by adding at the end the following new section: 

5 ‘‘SEC. 409J. CLINICAL TRIALS NETWORK FOR SPINAL MUS6 CULAR ATROPHY. 

7 ‘‘(a) CLINICAL TRIALS NETWORK.—The Director of 

8 NIH, in coordination with the Directors of the National 

9 Institute of Neurological Disorders and Stroke, the Na10 tional Institute of Child Health and Human Development, 

11 and such other Institutes and Centers as specified by the 

12 Director shall provide for the upgrading and unification 

13 of spinal muscular atrophy clinical trial sites and the re14 cruitment of new investigators and sites to establish a na15 tional clinical trials network for spinal muscular atrophy. 

16 The Director of NIH shall ensure that such network— 

17 ‘‘(1) conducts coordinated, multisite, clinical 

18 trials of therapies and clinical approaches to the 

19 treatment of spinal muscular atrophy; and 

20 ‘‘(2) rapidly and efficiently disseminates sci21 entific findings to the field. 

22 ‘‘(b) DATA COORDINATING CENTER.—The Director 

23 of NIH, in coordination with the Commissioner of Food 

24 and Drugs and the Directors of the National Institute of 

25 Neurological Disorders and Stroke, the National Institute 

26 of Child Health and Human Development, and such other 


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1 Institutes and Centers as specified by the Director, shall 

2 establish a data coordinating center with respect to spinal 

3 muscular atrophy to— 

4 ‘‘(1) provide expert assistance in the design, 

5 conduct, data analysis, data management, and data 

6 warehousing of collaborative clinical and descriptive 

7 research projects; 

8 ‘‘(2) organize and conduct multi-site monitoring 

9 activities; 

10 ‘‘(3) provide regular reports to the National In11 stitute of Neurological Disorders and Stroke, the 

12 National Institute of Child Health and Human De13 velopment, such other Institutes and Centers as 

14 specified by the Director, and the Food and Drug 

15 Administration on enrollment and the allocation of 

16 resources; and 

17 ‘‘(4) conduct such other activities as are 

18 deemed necessary by the Secretary. 

19 ‘‘(c) PRE-CLINICAL ACTIVITIES.—The Director of 

20 NIH, in coordination with the Directors of the National 

21 Institute of Neurological Disorders and Stroke and the 

22 National Institute of Child Health and Human Develop23 ment, shall expand and intensify programs of such Insti24 tutes with respect to pre-clinical translation research re25 lated to spinal muscular atrophy.’’. 


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1 SEC. 3. NATIONAL PATIENT REGISTRY. 

2 Part P of title III of the Public Health Service Act 

3 is amended by adding at the end the following new section: 

4 ‘‘SEC. 399S. NATIONAL SPINAL MUSCULAR ATROPHY PA5 TIENT REGISTRY. 

6 ‘‘(a) IN GENERAL.—The Secretary, acting through 

7 the Director of the Centers for Disease Control and Pre8 vention and in coordination with the Director of the NIH, 

9 shall enhance and provide ongoing support to a spinal 

10 muscular atrophy patient registry to provide for expanded 

11 epidemiological research towards improving awareness, 

12 management, treatment, and prevention of spinal mus13 cular atrophy. 

14 ‘‘(b) LONGITUDINAL DATA.—In carrying out sub15 section (a), the Secretary shall ensure the collection and 

16 analysis of longitudinal data related to individuals of all 

17 ages with spinal muscular atrophy, including infants, 

18 young children, adolescents, and adults of all ages.’’. 

19 SEC. 4. INTERAGENCY SPINAL MUSCULAR ATROPHY RE20 SEARCH COORDINATING COMMITTEE. 

21 Part B of title IV of the Public Health Service Act, 

22 as amended by section 2, is further amended by adding 

23 at the end the following new section: 


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1 ‘‘SEC. 409K. INTERAGENCY SPINAL MUSCULAR ATROPHY 

2 RESEARCH COORDINATING COMMITTEE. 

3 ‘‘(a) ESTABLISHMENT.—Not later than 6 months 

4 after the date of the enactment of this section, the Sec5 retary shall establish a committee, to be known as the 

6 Interagency Spinal Muscular Atrophy Research Coordi7 nating Committee (in this section referred to as the ‘Com8 mittee’). 

9 ‘‘(b) DUTIES.—The Committee shall— 

10 ‘‘(1) share and coordinate information on exist11 ing research activities, and make recommendations 

12 to the National Institutes of Health and other Fed13 eral agencies regarding how to improve existing re14 search programs, that are related to spinal muscular 

15 atrophy research and other related neurological dis16 eases and disorders; 

17 ‘‘(2) develop a comprehensive strategy related 

18 to spinal muscular atrophy research and other re19 lated neurological diseases and disorders and advise 

20 the National Institutes of Health and other Federal 

21 agencies, expanding proposals for collaborative, mul22 tidisciplinary research, including proposals for Com23 mon Fund research described in section 402(b)(7) 

24 and other proposals that involve collaboration be25 tween 2 or more national research institutes or na26 tional centers; 


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1 ‘‘(3) provide annual reports to the Secretary re2 garding the National Institutes of Health and other 

3 Federal agencies’ collaborative multidisciplinary re4 search efforts to support spinal muscular atrophy, 

5 including the Spinal Muscular Atrophy Project at 

6 the National Institute of Neurological Disorders and 

7 Stroke, the ongoing and future research needs to ad8 vance therapies for spinal muscular atrophy, and 

9 recommendations on how to strengthen the collabo10 ration of research activities by the institutes and 

11 agencies to improve the results; 

12 ‘‘(4) develop a summary of advances in research 

13 related to spinal muscular atrophy research and 

14 other related neurological diseases and disorders re15 search supported or conducted by Federal agencies; 

16 and 

17 ‘‘(5) not later than 1 year after the date of the 

18 establishment of the Committee, make recommenda19 tions to the Secretary— 

20 ‘‘(A) regarding any appropriate changes to 

21 research activities, including recommendations 

22 to improve the research portfolio of the Na23 tional Institutes of Health to ensure that sci24 entifically-based strategic planning is imple25 mented in support of research priorities that 


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1 impact research activities related to spinal mus2 cular atrophy and other related neurological 

3 diseases and disorders; 

4 ‘‘(B) identifying barriers to the develop5 ment of new treatments and cures for spinal 

6 muscular atrophy and other related neurological 

7 diseases and disorders; 

8 ‘‘(C) regarding public participation in deci9 sions relating to spinal muscular atrophy re10 search and other related neurological diseases 

11 and disorders to increase the involvement of pa12 tient advocacy and community organizations 

13 representing a broad geographical area; 

14 ‘‘(D) on how best to disseminate informa15 tion on spinal muscular atrophy progress; and 

16 ‘‘(E) on how to expand partnerships be17 tween public entities, including Federal agen18 cies, and private entities to expand collabo19 rative, cross-cutting research. 

20 ‘‘(c) RULE OF CONSTRUCTION.—In carrying out the 

21 duties described in subsection (b) with respect to research 

22 on spinal muscular atrophy, nothing in this section shall 

23 be construed to restrict the Secretary from including other 

24 neurological or genetic disorders, as appropriate, in such 

25 research if doing so may advance research in spinal musVerDate Nov 24 2008 19:31 May 26, 2009 Jkt 079006 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\S1158.IS S1158

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1 cular atrophy or other related neurological diseases and 

2 disorders. 

3 ‘‘(d) MEMBERSHIP.— 

4 ‘‘(1) IN GENERAL.—The Committee shall be 

5 composed of the following voting members: 

6 ‘‘(A) Not more than 11 voting Federal rep7 resentatives as follows: 

8 ‘‘(i) The Director of the Centers for 

9 Disease Control and Prevention. 

10 ‘‘(ii) The Director of the National In11 stitutes of Health and the directors of such 

12 national research institutes and national 

13 centers (which shall include the National 

14 Institute of Neurological Disorders and 

15 Stroke, the National Institute of Child 

16 Health and Human Development, and the 

17 National Center for Research Resources) 

18 as the Secretary determines appropriate. 

19 ‘‘(iii) The Commissioner of Food and 

20 Drugs. 

21 ‘‘(iv) The heads of such other agencies 

22 and advisory committees as the Secretary 

23 determines appropriate, including the 

24 Health Resources and Services Administra25 tion, the Agency for Healthcare Research 


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1 and Quality, and the Advisory Committee 

2 on Heritable Disorders and Genetic Dis3 eases in Newborns and Children. 

4 ‘‘(v) Representatives of other Federal 

5 agencies that conduct or support neuro6 logical research, or provide support serv7 ices and resources for individuals with spi8 nal muscular atrophy, such as the Depart9 ment of Education and the Social Security 

10 Administration. 

11 ‘‘(B) 9 additional voting members ap12 pointed under paragraph (2). 

13 ‘‘(2) ADDITIONAL MEMBERS.—The Committee 

14 shall include additional voting members appointed by 

15 the Secretary as follows: 

16 ‘‘(A) 6 members shall be appointed from 

17 among scientists, physicians, and other health 

18 professionals, who— 

19 ‘‘(i) are not officers or employees of 

20 the United States; 

21 ‘‘(ii) represent multiple disciplines, in22 cluding clinical, basic, and public health 

23 sciences; 

24 ‘‘(iii) represent different geographical 

25 regions of the United States; 


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1 ‘‘(iv) are from practice settings, aca2 demia, or other research settings; and 

3 ‘‘(v) are experienced in scientific peer 

4 review process. 

5 ‘‘(B) 3 members shall be appointed from 

6 members of the general public, who represent 

7 individuals with spinal muscular atrophy. 

8 ‘‘(3) NONVOTING MEMBERS.—The Committee 

9 shall include such nonvoting members as the Sec10 retary determines to be appropriate. 

11 ‘‘(e) CHAIRPERSON.—The voting members of the 

12 Committee shall select a chairperson from among the Fed13 eral members of the Committee described in subsection 

14 (d)(1)(A). The selection of a chairperson may be subject 

15 to the approval of the Secretary. The chairperson shall 

16 serve for a term of not to exceed 2 years, but may be 

17 re-elected as provided for in the first sentence. 

18 ‘‘(f) MEETINGS.—The Committee shall meet at the 

19 call of the chairperson of the Committee or upon the re20 quest of the Secretary, but in no case less often than once 

21 each year. 

22 ‘‘(g) REVIEW.—In 2012, and biennially thereafter, 

23 the Secretary shall review the necessity of the Com24 mittee.’’. 


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1 SEC. 5. EDUCATION AND AWARENESS ON SMA FOR HEALTH 

2 CARE PROFESSIONALS. 

3 Part P of title III of the Public Health Service Act, 

4 as amended by section 3, is further amended by adding 

5 at the end the following new section: 

6 ‘‘SEC. 399T. INFORMATION AND EDUCATION ON SMA. 

7 ‘‘The Secretary shall establish and implement a pro8 gram to provide information and education on spinal mus9 cular atrophy to health professionals and the general pub10 lic, including information and education on advances in 

11 the screening, diagnosis, and treatment of spinal muscular 

12 atrophy and training and continuing education through 

13 programs for scientists, physicians, medical students, and 

14 other health professionals who provide care for patients 

15 with spinal muscular atrophy.’’. 

Æ 


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