HR 640
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 authorize
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 acquired bone marrow failure 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 `Bone Marrow Failure Disease Research and Treatment
Act of 2011'.
SEC. 2. FINDINGS.
The Congress finds the following:
(1) Between 20,000 and 30,000 Americans are diagnosed each year with myelodysplastic
syndromes, aplastic anemia, paroxysmal nocturnal hemoglobinuria, and other
acquired bone marrow failure diseases.
(2) Acquired bone marrow failure diseases have a debilitating and often
fatal impact on those diagnosed 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 Institute and the National Cancer
Institute have conducted important research into the causes of and treatments
for acquired bone marrow failure diseases.
(7) The National Marrow Donor Program Registry 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 comprehensive Federal strategic effort
among numerous Federal agencies is needed to discover a cure for acquired
bone marrow failure disorders.
(9) Greater Federal surveillance of acquired bone marrow failure diseases
is needed to gain a better 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 DISEASE 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:
`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 Centers
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 Disease Registry,
in accordance with paragraph (3).
`(2) RECOMMENDATIONS OF ADVISORY COMMITTEE- In carrying out this subsection,
the Secretary shall take into consideration the recommendations of the Advisory
Committee on Acquired Bone Marrow Failure Diseases established under subsection
(b).
`(3) PURPOSES OF REGISTRY- The National Acquired Bone Marrow Failure Disease
Registry--
`(A) shall identify the incidence and prevalence of acquired bone marrow
failure diseases in the United States;
`(B) shall be used to collect and store data on acquired bone marrow failure
diseases, including data concerning--
`(i) the age, race or ethnicity, general geographic location, sex, and
family history of individuals who are diagnosed with acquired bone marrow
failure diseases, and any other characteristics of such individuals
determined appropriate by the Secretary;
`(ii) the genetic and environmental factors that may be associated with
developing acquired bone marrow failure diseases;
`(iii) treatment approaches for dealing with acquired bone marrow failure
diseases;
`(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 section
379A; and
`(v) any other factors pertaining to acquired bone marrow failure diseases
determined appropriate by the Secretary; and
`(C) shall be made available--
`(i) to the general public; and
`(ii) to researchers to facilitate further research into the causes
of, and treatments for, acquired bone marrow failure diseases in accordance
with standard practices of the Centers for Disease Control and Preventions.
`(1) ESTABLISHMENT- Not later than 6 months after the date of the enactment
of this section, 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 Marrow Failure Diseases.
`(2) MEMBERS- The members of the Advisory Committee on Acquired Bone Marrow
Failure Diseases 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:
`(A) A national patient advocacy organization with experience advocating
on behalf of patients suffering from acquired bone marrow failure diseases.
`(B) The National Institutes of Health, including 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 researchers who have experience researching
acquired bone marrow failure diseases.
`(G) The entity operating the C.W. Bill Young Cell Transplantation Program
established pursuant to section 379 and the entity operating the C.W.
Bill Young Cell Transplantation Program Outcomes Database.
`(3) RESPONSIBILITIES- The Advisory Committee 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,
including recommendations on the collection, maintenance, and dissemination
of data.
`(4) PUBLIC AVAILABILITY- The Secretary shall make the recommendations of
the Advisory Committee on Acquired Bone Marrow Failure Disease publicly
available.
`(c) Grants- The Secretary, acting through the Director of the Centers for
Disease Control and Prevention, 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 National Acquired Bone Marrow Failure Disease Registry.
`(d) Definition- In this section, the term `acquired bone marrow failure disease'
means--
`(1) myelodysplastic syndromes (MDS);
`(3) paroxysmal nocturnal hemoglobinuria (PNH);
`(4) pure red cell aplasia;
`(5) acute myeloid leukemia that has progressed 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
Radiation Injury Treatment Network of the C.W. Bill Young Cell Transplantation
Program established pursuant to section 379 of the Public Health Service Act
(42 U.S.C. 274j) to--
(1) augment data for the pilot studies authorized by this section;
(2) access technical assistance that may be provided 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 Secretary
for Minority Health, shall establish and coordinate outreach and informational
programs targeted to minority populations affected by acquired bone marrow
failure diseases.
`(2) PROGRAM REQUIREMENTS- Minority-focused outreach and informational programs
authorized by this section--
`(A) shall make information about treatment 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 supported under section
1707(b)(8) (including by means of the center's website, through appropriate
locations 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 effort to connect
Hispanic, Asian-American, and Pacific Islander communities with comprehensive
services 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 information 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 Secretary
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-reviewed 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.
`(d) Definition- In this section, the term `acquired 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);
(3) paroxysmal nocturnal hemoglobinuria (PNH);
(4) pure red cell aplasia;
(5) acute myeloid leukemia that progressed from myelodysplastic syndromes;
or
(6) large granular lymphocytic leukemia.
END
