1.This Act may be cited as the
Bone Marrow Failure Disease Research
and Treatment Act of 2011
.
2.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.
3.National Acquired
Bone Marrow Failure Disease RegistryPart B of the Public Health Service Act (42
U.S.C. 311 et seq.) is amended by inserting after section 317T the
following:
317U.National
Acquired Bone Marrow Failure Disease Registry
(a)Establishment of
registry
(1)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 committeeIn
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)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.
(b)
(1)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)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)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)The Secretary shall make the recommendations of the
Advisory Committee on Acquired Bone Marrow Failure Disease publicly
available.
(c)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)In
this section, 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;
(5)acute myeloid leukemia that has progressed
from myelodysplastic syndromes; or
(6)large granular
lymphocytic leukemia.
(e)Authorization of
appropriationsThere is authorized to be appropriated to carry
out this section $3,000,000 for each of fiscal years 2012 through
2016.
.
4.Pilot studies
through the agency for toxic substances and disease registry
(a)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 NetworkIn 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
appropriationsThere is authorized to be appropriated to carry
out this section $1,000,000 for each of fiscal years 2012 through 2016.
5.Minority-focused
programs on acquired bone marrow failure diseasesTitle XVII of the Public Health Service Act
(42 U.S.C. 300u et seq.) is amended by inserting after section 1707A the
following:
1707B.Minority-focused programs on acquired bone marrow failure
diseases(a)Information and
referral services
(1)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)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)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)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)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)Grants
and cooperative agreements authorized by this subsection shall be awarded or
entered into on a competitive, peer-reviewed basis.
(3)Research funded under this section shall examine factors
affecting the incidence of acquired bone marrow failure diseases in minority
populations.
(d)In this section, the term acquired
bone marrow failure disease has the meaning given to such term in
section 317U(d).
(e)Authorization of
appropriationsThere is authorized to be appropriated to carry
out this section $2,000,000 for each of fiscal years 2012 through
2016.
.
6.Diagnosis and
quality of care for acquired bone marrow failure diseases
(a)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
appropriationsThere is authorized to be appropriated to carry
out this section $2,000,000 for each of fiscal years 2012 through 2016.
7.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;
(5)acute myeloid leukemia that progressed from
myelodysplastic syndromes; or
(6)large granular
lymphocytic leukemia.