ment, shall expand and intensify programs of such Insti-
tutes 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 Cen-
ters for Disease Control and Prevention, shall enhance 
and provide ongoing support to the existing SMA patient 
registry to provide for expanded research on the epidemi-
ology of SMA. 
(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 SMA, including infants, young children, adoles-
cents, 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 Com-
mittee to coordinate activities across the National 
Institutes of Health and with other Federal health 
programs and activities relating to SMA. 
(2) COMPOSITION.—The Coordinating Com-
mittee shall consist of not more than 15 members to 
be appointed by the Secretary, of which— 
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(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 Insti-
tute of Child Health and Human Develop-
ment, other national research institutes in-
volved in research with respect to SMA, 
and the National Center for Research Re-
sources; 
(ii) representatives of all other Fed-
eral 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 Re-
sources and Services Administration, the 
Food and Drug Administration, and the 
Advisory Committee on Heritable Dis-
orders and Genetic Diseases in Newborns 
and Children; and 
(iii) representatives of other govern-
mental agencies that serve children with 
SMA, such as the Department of Edu-
cation; and 
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(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, re-
searchers, and clinicians. 
(3) TERM.—Members of the Coordinating Com-
mittee 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 Di-
rector 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 agen-
cies. 
(B) APPOINTMENT.—The Secretary shall 
appoint the Chair of the Coordinating Com-
mittee from among individuals nominated by 
the Coordinating Committee. The Chair shall be 
appointed for a term not to exceed 2 years and 
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may be reappointed for not more than 1 addi-
tional 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 Co-
ordinating Committee with necessary and ap-
propriate administrative support. 
(B) The Coordinating Committee shall 
meet as determined appropriate by the Sec-
retary, in consultation with the Chair of the Co-
ordinating Committee, but no less than twice 
each year. 
(b) STUDY
ON BARRIERS
TO DRUG DEVELOP-
MENT.— 
(1) STUDY.—The Coordinating Committee shall 
conduct a study to identify current and potential fu-
ture barriers to the development of drugs for treat-
ing SMA and other similar genetic disorders. Such 
study shall— 
(A) identify barriers related to the activi-
ties of government, industry, and academic 
medicine; 
(B) include substantial input from sci-
entists and organizations with direct involve-
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ment 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 Con-
gress a report on the results of the study described 
in paragraph (1) together with such recommenda-
tions 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 Direc-
tor of NIH shall establish a trans-National Institutes of 
Health cooperative research initiative on SMA. 
(b) DUTIES.—The cooperative research initiative es-
tablished under subsection (a) shall consist of the fol-
lowing 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 
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