C.W. Bill Young Cell Transplantation Program

Authorizing Legislation: Section 379 of the Public Health Service Act, as amended and Public Law 109-129.

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:

• 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.
  
• 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.
  
• 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

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.

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.