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C.W. Bill Young Cell Transplantation Program
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FY
2007 Actual |
FY
2008
Enacted |
FY
2009
Estimate |
FY
2009 +/-
FY 2008 |
BA |
$25,168,000
|
$23,517,000 |
$22,701,000 |
-$816,000 |
FTE |
5 |
5 |
5 |
5 |
Authorizing
Legislation: Section 379 of the Public Health Service
Act, as amended and Public Law 109-129.
FY
2009 Authorization |
$38,000,000
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Allocation
Method |
Contract
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Program Description and Accomplishments
The primary goal of the C.W. Bill Young Cell Transplantation
Program (Program) is to increase the number of transplants
for recipients suitably matched to biologically unrelated
donors of bone marrow and cord blood. The Program
works towards this goal by providing a national system
for recruiting potential bone marrow donors, tissue
typing potential donors, coordinating the procurement
of bone marrow and umbilical cord blood units for
transplantation, offering patient and donor advocacy
services, providing for public and professional education,
and collecting, analyzing, and reporting on transplant
outcomes data. Blood stem cell transplantation offers
the possibility of a cure for Americans suffering
from leukemia and other life-threatening blood and
genetic disorders. Each year approximately 38,000
people under the age of 55 are diagnosed with these
fatal illnesses, and about 16,000 of them cannot be
successfully treated with therapy other than a blood
stem cell transplant. When doctors have a patient
in need of transplantation, they initially try to
locate donors related to the patient. If none are
available, as is the case for approximately 70 percent
of patients, they need to search for a suitable unrelated
donor.
Per authorizing legislation signed on December 20,
2005 (The Stem Cell Therapeutic and Research Act of
2005, P.L. 109-129), the C.W. Bill Young Cell Transplantation
Program is the successor to the National Bone Marrow
Donor Registry. While the scope of activities required
of the Program is similar to that of the Registry,
the Program has expanded responsibility of collecting,
analyzing, and reporting on outcomes data for all
allogeneic transplants and on other therapeutic uses
of blood stem cells. In addition, unlike the Registry,
which was administered under a single contract, the
Program is made up of four contracts that require
close coordination. Also, unlike the Registry, P.L.
109-129 required the establishment of an Advisory
Council at the Department level to provide recommendations
to the Secretary on activities related to the Program.
In FY 2006, the Health Resources and Services Administration
(HRSA) awarded four contracts for: (1) Cord Blood
Coordinating Center which is responsible
for facilitating transplants with blood stem cells
from cord blood units and providing expectant mothers
with information on options regarding the use of umbilical
cord blood (i.e., public donation, private storage,
research and discard; (2) Bone Marrow Coordinating
Center which is responsible for recruiting
adult potential donors of blood stem cells, especially
from underrepresented ethnic and racial minority populations
and for facilitating transplants with blood stem cell
from adult donors; (3) Stem Cell Therapeutic
Outcomes Database which is responsible for
continuing and extending the collection of outcomes
data on unrelated donor blood stem cell transplants
using cells from adult donors and cord blood, developing
and implementing data collection for related donor
blood stem cell transplants, and developing and implementing
an approach to collect data on emerging therapeutic
uses of blood stem cells from a donor; and, (4) a
combined Office of Patient Advocacy and Single Point
of Access which provides a single point of
access for physicians, health care providers and patients
to search for a blood stem cell product from an adult
donor or cord blood unit, assist patients in need
of a blood stem cell transplant from diagnosis to
survivorship, and identify the gaps in services and
offer programs to help meet the needs of patients.
Contracts for all components of the Program were awarded
through a competitive contracting process that emphasized
technical merit. Contract opportunities were announced
nationally and proposals were then reviewed by objective
review committees, composed of individuals who are
qualified by training and experience in particular
fields related to the Program. Funding decisions were
made based on committee assessments of technical merit,
evaluation of past performance, and evaluation of
proposed costs. When exercising option years beyond
the original two-year base period of the contracts,
HRSA will consider current performance and compliance
with contract terms.
In Fiscal Year (FY) 2007, the most recent year for
which data are available, the Program demonstrated
continued improvement in meeting its three long-term
goals: (1) increasing the number of transplants facilitated
for minority patients; (2) increasing the number of
blood stem cell transplants facilitated; and (3) increasing
the rate of survival at one-year, post-transplant.
The Program goals are supported by three overarching
measures:
(1) increase the number of adult volunteer potential
donors of minority race and ethnicity on the Registry;
(2) increase the number of cord blood units listed
on the Registry; and (3) decrease the cost for human
leukocyte antigen (HLA) tissue typing needed to match
patients and donors. The Program continues to serve
a diverse patient population:
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In FY 2005, a total of 5,907,923 adult volunteers
were listed on the Registry, of whom 1,591,628 (or
26.9 percent) self-identified as belonging to a
racial/ethnic minority population group. In FY 2006,
a total of 6,317,827 adult volunteers were listed
on the Registry, of whom 1,698,616 (or 26.9 percent)
self-identified as belonging to a racial/ethnic
minority population group. In FY 2007, the program
slightly exceeded its goal of 1.83 million adult
volunteer potential donors of minority race and
ethnicity. In FY 2007, a total of 6,856,150 adult
volunteers were listed on the Registry, of whom
1,856,434 (or 27.1 percent) self-identified as belonging
to a racial/ethnic minority population group. The
program was able to achieve its target by establishing
additional initiatives to increase community awareness
and outreach in minority populations. To make further
improvements in recruitment and to continue to meet
future targets, the contractor has reorganized the
recruitment structure.
Although there will be a continued emphasis on recruiting
and retaining minority donors to increase minority
Americans' chances of finding an unrelated donor,
significant added responsibilities under the new
legislation may preclude sustaining the level of
recruitment called for in the Registry goals. Given
the more complex structure and broader Program responsibilities,
HRSA plans to assess the appropriateness of previously
established targets under the Registry.
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In FY 2005, the Registry listed 45,807 cord blood
units. In FY 2006, the Registry listed 51,693 units,
a 12.8 percent increase over FY 2005. In FY 2007,
the Program listed 69,081 cord blood units, a 34
percent increase over FY 2006. The targets established
for FY 2007-2009 have been met. New targets are
not being proposed because HRSA support for cord
blood collection and listing now is through the
related program, National Cord Blood Inventory,
for which goals are being developed. Therefore,
HRSA will propose deleting this specific goal from
the Program and establishing a cord blood collection
goal for new units for the National Cord Blood Inventory.
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The cost of Human Leukocyte Antigen (HLA) typing
strongly influences the number of potential volunteer
donors who can be recruited to join the Registry.
Reducing the unit cost of typing makes increases
in donor recruitment possible without funding increases.
In FY 2004, the National Marrow Donor Program successfully
negotiated a 2.7 percent reduction in cost with
its contracted laboratories for Human Leukocyte
Antigen (HLA) tissue typing. The cost of tissue
typing decreased from $65.00 in FY 2003 to $63.65
in FY 2004. The Registry contractor did not negotiate
new laboratory contracts during FY 2005. In FY 2006,
the Registry negotiated an 18 percent cost reduction
for tissue typing cost to $52.00 per person, which
is expected to remain in effect over the next three
years. This reduction in tissue typing cost far
exceeds the initial long term target ($56.42) established
for the Registry.
A Program Assessment Rating Tool (PART) review of
the National Bone Marrow Donor Registry (Registry),
predecessor to the C. W. Bill Young Cell Transplantation
Program, was conducted in 2004. The program received
a rating of Moderately Effective. The review found
that HRSA program staff provided effective oversight
over the program and that staff appropriately were
heavily engaged in all aspects of Registry operations,
particularly committee work (which is where program
policies are initially discussed). It also found that
performance standards already were in place within
the contract for the major Registry activities. During
the PART review, aggressive long-term goals and annual
measures to increase the number of transplants facilitated
and improve patient survival were established.
Since the PART assessment the Program has: (1) ensured
a seamless transition from the Registry structure
to the newly authorized and more complex C.W. Bill
Young Cell Transplantation Program structure, (2)
established a methodology for determining one-year
patient survival rates and established baselines and
targets for this performance measure, (3) updated
and implemented a comprehensive plan to increase transplants,
and (4) established an Outcomes Database Working Group
to receive recommendations regarding the best approach
to collecting outcomes data on unrelated donor transplants,
related transplants and emerging therapeutic uses
of blood stem cells from a donor.
HRSA has completed the transition to the new program
structure, and all parts of the complex new structure
are functioning fully. However, given the more complex
structure and broader Program responsibilities, HRSA
plans to assess the appropriateness of performance
targets previously established under the Registry.
Funding includes costs associated with grant reviews,
processing of grants through the Grants Administration
Tracking and Evaluation System (GATES) and HRSA’s
electronic handbook, and follow-up performance reviews.
Funding History
FY
2004 |
$22,662,000
|
FY
2005 |
$25,416,000
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FY
2006 |
$25,145,000
|
FY
2007 |
$25,168,000
|
FY
2008 |
$23,517,000 |
Budget
Request
The FY 2009 Budget request of $22,701,000 is a decrease
of $816,000 over the FY 2008 Enacted level. The FY
2009 Budget will enable continued recruitment of new
donors and continued facilitation of unrelated blood
stem cell transplantation.
The FY 2009 Budget request will support achieving
the Program’s ambitious performance targets
which include listing on the Registry 2,060,000 adult
volunteers from racially/ethnically diverse minority
population groups. These funds will also be used to
support an infrastructure comprised of multiple contracts
(Cord Blood Coordinating Center, a Bone Marrow Coordinating
Center a combined contract for the Office of Patient
Advocacy and Single Point of Access, and a Stem Cell
Therapeutic Outcomes Database). The majority of the
funds will be used to support recruitment of new donors
(including tissue typing costs). The Program will
continue its central goal of increasing the number
of transplants for recipients suitably matched to
biologically unrelated donors of bone marrow and cord
blood. Additionally, through the Stem Cell Therapeutic
Outcomes Database, the Program will continue efforts
to collect comprehensive outcomes data on related-donor
and unrelated-donor transplants, assess quality of
life for transplant recipients, work with foreign
transplant centers to obtain data on U.S. stem cell
products provided to them for transplant, and new
initiatives to collect data on emerging therapies
using cells derived from bone marrow and umbilical
cord blood.
Funding will also allow the Program to continue critical
planning activities to prepare to respond to a radiation
or chemical (mustard agent) emergency that would leave
some casualties with temporary or permanent marrow
failure, and to facilitate emergency transplants for
those casualties who would not otherwise recover marrow
function.
# |
Key
Outcomes |
FY
2004 Actual |
FY
2005 Actual |
FY
2006 |
FY
2007 |
FY
2008
Target |
FY
2009
Target |
Out
Year Target |
Target |
Actual |
Target |
Actual |
Long-Term Objective: Expand
the availability of health care, particularly
to underserved, vulnerable, and special needs
populations |
24.1 |
Increase by 95% the number of
blood stem cell transplants facilitated annually
by the Registry. (Baseline - 2003: 2,310) |
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2010: 4,500 |
24.2 |
Increase by 100% the number of
blood stem cell transplants facilitated annually
by the Registry for minority patients. (Baseline
- 2003: 318) |
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2010:
636 |
24.3 |
Increase the rate of patient
survival at one year, post transplant. (Baseline:
2003, 62%) |
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2010:
69% |
# |
Key
Outputs |
FY
2004 Actual |
FY
2005 Actual |
FY
2006 |
FY
2007 |
FY
2008
Target/ Est. |
FY
2009
Target/ Est. |
Out
Year Target/ Est. |
Target/
Est. |
Actual |
Target/
Est. |
Actual |
Long-Term
Objective: Expand the availability of health care,
particularly to underserved, vulnerable, and special
needs populations |
24.II.A.1 |
Increase the number
of cord blood units listed on the Registry. |
35,926 |
45,807 |
36,500 |
51,693 |
38,500 |
69,081 |
NA |
NA |
NA |
24.II.A.2. |
Increase the number
of adult volunteer potential donors of minority
race and ethnicity. |
1.47
M |
1.59
M |
1.71
M |
1.70
M |
1.83
M |
1.85M |
1.94
M |
2.06
M |
NA |
Efficiency
Measures |
24.E. |
Decrease the unit
cost of HLA-typing of potential donors by 2% each
year. |
$63.65
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$63.65
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$61.17
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$52.00
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$59.95
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$52.00
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$52.00
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$52.00
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$52.00
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Other Outputs |
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Contracts |
1 |
1 |
4 |
4 |
4 |
4 |
4 |
4 |
NA |
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Appropriated Amount
($ Million) |
22.652 |
25.416 |
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25.145 |
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25.168 |
23.517 |
22.701 |
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Notes:
The
FY 2008 and 2009 targets for 24.II.A.1. have been
changed to NA (not applicable) as a result of the
new program structure per P.L 109-129, under which
HRSA funding for cord blood collection occurs through
a related program, the National Cord Blood Inventory.
Appropriate measures and targets for the National
Cord Blood Inventory will be developed by summer 2008.
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