I 
112TH CONGRESS 
1ST SESSION 
H. R. 640 
To amend the Public Health Service Act to provide for the establishment 
of a National Acquired Bone Marrow Failure Disease Registry, to author-
ize research on acquired bone marrow failure diseases, and for other 
purposes. 
IN THE HOUSE OF REPRESENTATIVES 
FEBRUARY 10, 2011 
Ms. MATSUI introduced the following bill; which was referred to the 
Committee on Energy and Commerce 
A BILL 
To amend the Public Health Service Act to provide for 
the establishment of a National Acquired Bone Marrow 
Failure Disease Registry, to authorize research on ac-
quired bone marrow failure diseases, and for other pur-
poses. 
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 ‘‘Bone Marrow Failure 
Disease Research and Treatment Act of 2011’’. 
SEC. 2. FINDINGS. 
The Congress finds the following: 
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(1) Between 20,000 and 30,000 Americans are 
diagnosed each year with myelodysplastic syndromes, 
aplastic 
anemia, 
paroxysmal 
nocturnal 
hemo-
globinuria, and other acquired bone marrow failure 
diseases. 
(2) Acquired bone marrow failure diseases have 
a debilitating and often fatal impact on those diag-
nosed with these diseases. 
(3) While some treatments for acquired bone 
marrow failure diseases can prolong and improve the 
quality of patients’ lives, there is no single cure for 
these diseases. 
(4) The prevalence of acquired bone marrow 
failure diseases in the United States will continue to 
grow as the general public ages. 
(5) Evidence exists suggesting that acquired 
bone marrow failure diseases occur more often in 
minority populations, particularly in Asian-American 
and Hispanic/Latino populations. 
(6) The National Heart, Lung, and Blood Insti-
tute and the National Cancer Institute have con-
ducted important research into the causes of and 
treatments for acquired bone marrow failure dis-
eases. 
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(7) The National Marrow Donor Program Reg-
istry has made significant contributions to the fight 
against bone marrow failure diseases by connecting 
millions of potential marrow donors with individuals 
and families suffering from these conditions. 
(8) Despite these advances, a more comprehen-
sive Federal strategic effort among numerous Fed-
eral agencies is needed to discover a cure for ac-
quired bone marrow failure disorders. 
(9) Greater Federal surveillance of acquired 
bone marrow failure diseases is needed to gain a bet-
ter understanding of the causes of acquired bone 
marrow failure diseases. 
(10) The Federal Government should increase 
its research support for and engage with public and 
private organizations in developing a comprehensive 
approach to combat and cure acquired bone marrow 
failure diseases. 
SEC. 3. NATIONAL ACQUIRED BONE MARROW FAILURE DIS-
EASE REGISTRY. 
Part B of the Public Health Service Act (42 U.S.C. 
311 et seq.) is amended by inserting after section 317T 
the following: 
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‘‘SEC. 317U. NATIONAL ACQUIRED BONE MARROW FAILURE 
DISEASE REGISTRY. 
‘‘(a) ESTABLISHMENT OF REGISTRY.— 
‘‘(1) IN GENERAL.—Not later than 6 months 
after the date of the enactment of this section, the 
Secretary, acting through the Director of the Cen-
ters for Disease Control and Prevention, shall— 
‘‘(A) develop a system to collect data on 
acquired bone marrow failure diseases; and 
‘‘(B) establish and maintain a national and 
publicly available registry, to be known as the 
National Acquired Bone Marrow Failure Dis-
ease Registry, in accordance with paragraph 
(3). 
‘‘(2) RECOMMENDATIONS
OF
ADVISORY
COM-
MITTEE.—In carrying out this subsection, the Sec-
retary shall take into consideration the recommenda-
tions of the Advisory Committee on Acquired Bone 
Marrow Failure Diseases established under sub-
section (b). 
‘‘(3) PURPOSES OF REGISTRY.—The National 
Acquired Bone Marrow Failure Disease Registry— 
‘‘(A) shall identify the incidence and preva-
lence of acquired bone marrow failure diseases 
in the United States; 
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‘‘(B) shall be used to collect and store data 
on acquired bone marrow failure diseases, in-
cluding data concerning— 
‘‘(i) the age, race or ethnicity, general 
geographic location, sex, and family history 
of individuals who are diagnosed with ac-
quired bone marrow failure diseases, and 
any other characteristics of such individ-
uals determined appropriate by the Sec-
retary; 
‘‘(ii) the genetic and environmental 
factors that may be associated with devel-
oping acquired bone marrow failure dis-
eases; 
‘‘(iii) treatment approaches for deal-
ing with acquired bone marrow failure dis-
eases; 
‘‘(iv) outcomes for individuals treated 
for acquired bone marrow failure diseases, 
including outcomes for recipients of stem 
cell therapeutic products as contained in 
the database established pursuant to sec-
tion 379A; and 
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‘‘(v) any other factors pertaining to 
acquired bone marrow failure diseases de-
termined appropriate by the Secretary; and 
‘‘(C) shall be made available— 
‘‘(i) to the general public; and 
‘‘(ii) to researchers to facilitate fur-
ther research into the causes of, and treat-
ments for, acquired bone marrow failure 
diseases in accordance with standard prac-
tices of the Centers for Disease Control 
and Preventions. 
‘‘(b) ADVISORY COMMITTEE.— 
‘‘(1) ESTABLISHMENT.—Not later than 6 
months after the date of the enactment of this sec-
tion, the Secretary, acting through the Director of 
the Centers for Disease Control and Prevention, 
shall establish an advisory committee, to be known 
as the Advisory Committee on Acquired Bone Mar-
row Failure Diseases. 
‘‘(2) MEMBERS.—The members of the Advisory 
Committee on Acquired Bone Marrow Failure Dis-
eases shall be appointed by the Secretary, acting 
through the Director of the Centers for Disease 
Control and Prevention, and shall include at least 
one representative from each of the following: 
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‘‘(A) A national patient advocacy organiza-
tion with experience advocating on behalf of pa-
tients suffering from acquired bone marrow 
failure diseases. 
‘‘(B) The National Institutes of Health, in-
cluding at least one representative from each 
of— 
‘‘(i) the National Cancer Institute; 
‘‘(ii) the National Heart, Lung, and 
Blood Institute; and 
‘‘(iii) the Office of Rare Diseases. 
‘‘(C) The Centers for Disease Control and 
Prevention. 
‘‘(D) Clinicians with experience in— 
‘‘(i) diagnosing or treating acquired 
bone marrow failure diseases; and 
‘‘(ii) medical data registries. 
‘‘(E) Epidemiologists who have experience 
with data registries. 
‘‘(F) Publicly or privately funded research-
ers who have experience researching acquired 
bone marrow failure diseases. 
‘‘(G) The entity operating the C.W. Bill 
Young Cell Transplantation Program estab-
lished pursuant to section 379 and the entity 
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operating the C.W. Bill Young Cell Transplan-
tation Program Outcomes Database. 
‘‘(3) RESPONSIBILITIES.—The Advisory Com-
mittee on Acquired Bone Marrow Failure Diseases 
shall provide recommendations to the Secretary on 
the establishment and maintenance of the National 
Acquired Bone Marrow Failure Disease Registry, in-
cluding recommendations on the collection, mainte-
nance, and dissemination of data. 
‘‘(4) PUBLIC
AVAILABILITY.—The Secretary 
shall make the recommendations of the Advisory 
Committee on Acquired Bone Marrow Failure Dis-
ease publicly available. 
‘‘(c) GRANTS.—The Secretary, acting through the 
Director of the Centers for Disease Control and Preven-
tion, may award grants to, and enter into contracts and 
cooperative agreements with, public or private nonprofit 
entities for the management of, as well as the collection, 
analysis, and reporting of data to be included in, the Na-
tional Acquired Bone Marrow Failure Disease Registry. 
‘‘(d) DEFINITION.—In this section, the term ‘ac-
quired bone marrow failure disease’ means— 
‘‘(1) myelodysplastic syndromes (MDS); 
‘‘(2) aplastic anemia; 
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‘‘(3) 
paroxysmal 
nocturnal 
hemoglobinuria 
(PNH); 
‘‘(4) pure red cell aplasia; 
‘‘(5) acute myeloid leukemia that has pro-
gressed from myelodysplastic syndromes; or 
‘‘(6) large granular lymphocytic leukemia. 
‘‘(e) AUTHORIZATION OF APPROPRIATIONS.—There 
is authorized to be appropriated to carry out this section 
$3,000,000 for each of fiscal years 2012 through 2016.’’. 
SEC. 4. PILOT STUDIES THROUGH THE AGENCY FOR TOXIC 
SUBSTANCES AND DISEASE REGISTRY. 
(a) PILOT STUDIES.—The Secretary of Health and 
Human Services, acting through the Administrator of the 
Agency for Toxic Substances and Disease Registry, shall 
conduct pilot studies to determine which environmental 
factors, including exposure to toxins, may cause acquired 
bone marrow failure diseases. 
(b) COLLABORATION WITH THE RADIATION INJURY 
TREATMENT NETWORK.—In carrying out the directives of 
this section, the Secretary may collaborate with the Radi-
ation Injury Treatment Network of the C.W. Bill Young 
Cell Transplantation Program established pursuant to sec-
tion 379 of the Public Health Service Act (42 U.S.C. 
274j) to— 
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(1) augment data for the pilot studies author-
ized by this section; 
(2) access technical assistance that may be pro-
vided by the Radiation Injury Treatment Network; 
or 
(3) perform joint research projects. 
(c) AUTHORIZATION OF APPROPRIATIONS.—There is 
authorized to be appropriated to carry out this section 
$1,000,000 for each of fiscal years 2012 through 2016. 
SEC. 5. MINORITY-FOCUSED PROGRAMS ON ACQUIRED 
BONE MARROW FAILURE DISEASES. 
Title XVII of the Public Health Service Act (42 
U.S.C. 300u et seq.) is amended by inserting after section 
1707A the following: 
‘‘MINORITY-FOCUSED PROGRAMS ON ACQUIRED BONE 
MARROW FAILURE DISEASES 
‘‘SEC. 1707B. (a) INFORMATION
AND REFERRAL 
SERVICES.— 
‘‘(1) IN GENERAL.—Not later than 6 months 
after the date of the enactment of this section, the 
Secretary, acting through the Deputy Assistant Sec-
retary for Minority Health, shall establish and co-
ordinate outreach and informational programs tar-
geted to minority populations affected by acquired 
bone marrow failure diseases. 
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‘‘(2) PROGRAM
REQUIREMENTS.—Minority-fo-
cused outreach and informational programs author-
ized by this section— 
‘‘(A) shall make information about treat-
ment options and clinical trials for acquired 
bone marrow failure diseases publicly available, 
and 
‘‘(B) shall provide referral services for 
treatment options and clinical trials, 
at the national minority health resource center sup-
ported under section 1707(b)(8) (including by means 
of the center’s website, through appropriate loca-
tions such as the center’s knowledge center, and 
through appropriate programs such as the center’s 
resource persons network) and through minority 
health consultants located at each Department of 
Health and Human Services regional office. 
‘‘(b) HISPANIC AND ASIAN-AMERICAN AND PACIFIC 
ISLANDER OUTREACH.— 
‘‘(1) IN
GENERAL.—The Secretary, acting 
through the Deputy Assistant Secretary for Minority 
Health, shall undertake a coordinated outreach ef-
fort to connect Hispanic, Asian-American, and Pa-
cific Islander communities with comprehensive serv-
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ices focused on treatment of, and information about, 
acquired bone marrow failure diseases. 
‘‘(2) COLLABORATION.—In carrying out this 
subsection, the Secretary may collaborate with public 
health agencies, nonprofit organizations, community 
groups, and online entities to disseminate informa-
tion about treatment options and clinical trials for 
acquired bone marrow failure diseases. 
‘‘(c) GRANTS AND COOPERATIVE AGREEMENTS.— 
‘‘(1) IN GENERAL.—Not later than 6 months 
after the date of the enactment of this section, the 
Secretary, acting through the Deputy Assistant Sec-
retary for Minority Health, shall award grants to, or 
enter into cooperative agreements with, entities to 
perform research on acquired bone marrow failure 
diseases. 
‘‘(2) REQUIREMENT.—Grants and cooperative 
agreements authorized by this subsection shall be 
awarded or entered into on a competitive, peer-re-
viewed basis. 
‘‘(3) SCOPE OF RESEARCH.—Research funded 
under this section shall examine factors affecting the 
incidence of acquired bone marrow failure diseases 
in minority populations. 
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‘‘(d) DEFINITION.—In this section, the term ‘ac-
quired bone marrow failure disease’ has the meaning given 
to such term in section 317U(d). 
‘‘(e) AUTHORIZATION OF APPROPRIATIONS.—There 
is authorized to be appropriated to carry out this section 
$2,000,000 for each of fiscal years 2012 through 2016.’’. 
SEC. 6. DIAGNOSIS AND QUALITY OF CARE FOR ACQUIRED 
BONE MARROW FAILURE DISEASES. 
(a) GRANTS.—The Secretary of Health and Human 
Services, acting through the Director of the Agency for 
Healthcare Research and Quality, shall award grants to 
entities to improve diagnostic practices and quality of care 
with respect to patients with acquired bone marrow failure 
diseases. 
(b) AUTHORIZATION OF APPROPRIATIONS.—There is 
authorized to be appropriated to carry out this section 
$2,000,000 for each of fiscal years 2012 through 2016. 
SEC. 7. DEFINITION. 
In this Act, the term ‘‘acquired bone marrow failure 
disease’’ means— 
(1) myelodysplastic syndromes (MDS); 
(2) aplastic anemia; 
(3) 
paroxysmal 
nocturnal 
hemoglobinuria 
(PNH); 
(4) pure red cell aplasia; 
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(5) acute myeloid leukemia that progressed 
from myelodysplastic syndromes; or 
(6) large granular lymphocytic leukemia. 
Æ 
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