Healthcare Systems

C.W. Bill Young Cell Transplantation Program

Authorizing Legislation - Title III, Section 379 of the Public Health Service Act.

FY 2008 Authorization..................................................................................................$38,000,000

Statement of the Budget Request - The FY 2008 Budget of $22,701,000 is a decrease of $2,461,000 below the FY 2007 Continuing Resolution (CR).

Program Description - The purpose 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. Blood stem cell transplants offer the possibility of a cure for Americans suffering from leukemia and other blood and genetic diseases. Each year approximately 38,000 people less than 55 years old 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.

The tissue types of marrow donors must be closely matched with those of their recipients in order for the transplant to be successful. Since tissue types are inherited, patients are more likely to find a closely matched donor within their own racial and ethnic group. However, due to the high rate of diversity in the tissue types of minorities, especially African Americans, the Program must continue to recruit donors from these groups to increase the possibility that patients will find a closely matched donor. For this reason, recruitment efforts under the Program emphasize increasing the number of minority volunteer donors.

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. The Health Resources and Services Administration (HRSA) awarded contracts for a Cord Blood Coordinating Center, a Bone Marrow Coordinating Center, an Outcomes Database, and a combined Office of Patient Advocacy/Single Point of Access in FY 2006. P.L. 109-129 also required the establishment of an Advisory Council at the Department level. During FY 2007, all the components of the Program will be in place. While the scope of activities required of the Program are 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 encompasses four contracts that require close coordination.

Rationale for the Budget Request The FY 2008 Budget request of $22,701,000 reflects a reduction of $2,461,000 below the FY 2007 CR. The FY 2008 Budget will enable continued recruitment of new donors and continued facilitation of unrelated blood stem cell transplantation.
A Program Assessment Rating Tool (PART) review of the National Bone Marrow Donor Registry program, predecessor to the C. W. Bill Young Cell Transplantation Program, was conducted in FY 2004 for the FY 2006 Budget. The program received a rating of Moderately Effective. After the new Program is fully implemented in FY 2007, HRSA plans to revisit its targets under the new structure, which is considerably more complex than the Registry program structure. Although there will be an emphasis on recruiting and retaining minority donors to increase minority Americans' chances of finding an unrelated donor, the Program may not be able to sustain the level of recruitment called for in the PART goals. HRSA will aggressively seek efficiencies in managing these contracts, but some reduction in recruitment may be unavoidable due to an increase in the infrastructure required under the new Program structure.

Finally, the Program will also be able to continue planning exercises to prepare to respond to a radiation or chemical (mustard) 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.

Funding for the C.W. Bill Young Cell Transplantation Program during the last five years has been as follows:

Outputs -

Per authorizing legislation signed on December 20, 2005, the successor to the National Bone Marrow Donor Registry is the C.W. “Bill” Young Cell Transplantation Program (Program). Implementation of this successor program began during FY 2006. During FY 2007, all components of this program will be in place. The performance results and targets discussed below are those of the previous National Bone Marrow Donor Registry Program.

Performance Analysis - In an effort to increase the likelihood of a patient finding a blood stem cell donor, the Registry has focused its efforts on increasing the donor pool, particularly on adding donors from racial and ethnic minority populations. The number of volunteer potential donors on the Registry has grown by at least 6 percent over each of the past three years.

In FY 2006, the Registry increased the number of potential donors by 6.9 percent over FY 2005. The Registry also significantly increased the number of adult volunteer donors, who self-identified as belonging to a racial/ethnic minority population group, from 1,591,628 in FY 2005 to 1,698,616 in FY 2006; slightly below the established target of 1,710,000. The Registry increased the number of cord blood units from 45,807 in FY 2005 to 51,693 in FY 2006, representing a 12.8 percent increase. (See “Details of Performance Analysis.”)

A Program Assessment Rating Tool (PART) review of the National Bone Marrow Donor Registry program was conducted for the FY 2006 Budget. The program received a rating of Moderately Effective. During this review, the program developed a new set of performance measures that replaced previous measures.

In an effort to continue performance improvements, the Registry will continue to strive to recruit and retain minority donors to increase minority Americans' chances of finding a donor among Registry volunteers. The Registry also will work with cord blood banks to increase the number of high quality, racially diverse cord blood units on the Registry. These efforts will assist in achieving the long-term goal of increasing the number of blood stem cell transplants facilitated by the Registry and improving transplant outcomes.

Note: After the new Program is fully implemented, HRSA plans to revisit its performance targets under the new structure, which is considerably more complex than the Registry program structure. Although there will be an emphasis on recruiting and retaining minority donors to increase minority Americans' chances of finding an unrelated donor, the Program may not be able to sustain the level of adult donor recruitment called for in the PART goals. The sustainability may be a challenge due to an increase in the infrastructure required under the Program structure. HRSA will continue to seek operational efficiencies to minimize the impact on adult donor recruitment.