| Original language | English (US) |
|---|---|
| Pages (from-to) | 1380-1392 |
| Number of pages | 13 |
| Journal | Biology of Blood and Marrow Transplantation |
| Volume | 13 |
| Issue number | 11 |
| DOIs | |
| State | Published - Nov 2007 |
Bibliographical note
Funding Information:Robert Baitty , representing the U.S. Department of Health and Human Services (HHS), Health Resources and Services Administration (HRSA), Division of Transplantation, Blood Stem Cell Transplantation Program, presented an update on the C.W. Bill Young Cell Transplantation Program (the Program) and the National Cord Blood Inventory. The aims of the Stem Cell Therapeutic and Research Act of 2005 are to increase the number of unrelated donor transplants, increase the public inventory of high quality CBUs from diverse populations and increase the number of CBUs available for research. The Act authorizes appropriations of , representing the U.S. Department of Health and Human Services (HHS), Health Resources and Services Administration (HRSA), Division of Transplantation, Blood Stem Cell Transplantation Program, presented an update on the C.W. Bill Young Cell Transplantation Program (the Program) and the National Cord Blood Inventory. The aims of the Stem Cell Therapeutic and Research Act of 2005 are to increase the number of unrelated donor transplants, increase the public inventory of high quality CBUs from diverse populations and increase the number of CBUs available for research. The Act authorizes appropriations of $15 million/year for FY 2007-2010; funding is temporary until 150,000 CBUs are obtained. HRSA’s implementation approach is guided by three principles: a single point of access for patients and physicians to all sources of blood stem cells, collection of high-quality diverse CBU expeditiously, and collection of complete data on clinical outcomes of transplants. The National Cord Blood Inventory (NCBI) will be funded through one-time, 10-year contracts awarded competitively with HRSA funds for ≤3 years; funded cord blood banks must participate in the Program for ≥10 years; and the NCBI CBUs must be available through the Program in perpetuity (or until they are no longer viable). Contracts for the first cohort of banks were awarded November 2, 2006, to six banks (New York Blood Center, Duke University, MD Anderson, Puget Sound Blood Center, University of Colorado, and StemCyte). About 10,500 CBU are to be collected with first year of funding for the first cohort and these are to be comprised of ∼63% racial and ethnic minorities. Request for Proposals for a second cohort of banks was published April 12 with responses due May 15, 2007. HRSA will hold annual competitions for new cohorts of banks, as funding permits. Infrastructure contracts include a contract for the stem cell therapeutic outcomes database, which was awarded September 15, 2006, to the Center for International Blood and Marrow Transplant Research; contracts for an Office of Patient Advocacy/Single Point of Access, Bone Marrow Coordinating Center, and a Cord Blood Coordinating Center, which were awarded September 25, 2006, to the National Marrow Donor Program. An Advisory Council on Blood Stem Cell Transplantation is being established whose purpose is to consider and make recommendations to the Secretary of HHS on matters related to the Program. Ongoing work includes: complete the transition from the former National Bone Marrow Donor Registry structure to C.W. Bill Young Cell Transplantation Program, launch a web site for the Program (mid-Summer 2007), begin collection of blood stem cell transplant outcomes data (mid-Summer 2007), complete establishment of the Advisory Council and convene its first meeting, and future cycles of funding for NCBI collections. Further, implement the Related Cord Blood Donor Demonstration Project, revisit interim program requirements with Advisory Council and public input, and define targets for composition of NCBI and the size and composition of adult donor registry in consultation with the Advisory Council. Finally, to encourage research in collaboration with other Federal agencies and the Advisory Council to improve transplant outcomes, refine approaches to CB transplants for adult patients, better define CBU characteristics required for good outcomes, improve reliability and comparability of measures used in CBU selection, and to collect and report data regarding new uses of stem cells from CBU and adult donors.5 million/year for FY 2007-2010; funding is temporary until 150,000 CBUs are obtained. HRSA’s implementation approach is guided by three principles: a single point of access for patients and physicians to all sources of blood stem cells, collection of high-quality diverse CBU expeditiously, and collection of complete data on clinical outcomes of transplants. The National Cord Blood Inventory (NCBI) will be funded through one-time, 10-year contracts awarded competitively with HRSA funds for ≤3 years; funded cord blood banks must participate in the Program for ≥10 years; and the NCBI CBUs must be available through the Program in perpetuity (or until they are no longer viable). Contracts for the first cohort of banks were awarded November 2, 2006, to six banks (New York Blood Center, Duke University, MD Anderson, Puget Sound Blood Center, University of Colorado, and StemCyte). About 10,500 CBU are to be collected with first year of funding for the first cohort and these are to be comprised of ∼63% racial and ethnic minorities. Request for Proposals for a second cohort of banks was published April 12 with responses due May 15, 2007. HRSA will hold annual competitions for new cohorts of banks, as funding permits. Infrastructure contracts include a contract for the stem cell therapeutic outcomes database, which was awarded September 15, 2006, to the Center for International Blood and Marrow Transplant Research; contracts for an Office of Patient Advocacy/Single Point of Access, Bone Marrow Coordinating Center, and a Cord Blood Coordinating Center, which were awarded September 25, 2006, to the National Marrow Donor Program. An Advisory Council on Blood Stem Cell Transplantation is being established whose purpose is to consider and make recommendations to the Secretary of HHS on matters related to the Program. Ongoing work includes: complete the transition from the former National Bone Marrow Donor Registry structure to C.W. Bill Young Cell Transplantation Program, launch a web site for the Program (mid-Summer 2007), begin collection of blood stem cell transplant outcomes data (mid-Summer 2007), complete establishment of the Advisory Council and convene its first meeting, and future cycles of funding for NCBI collections. Further, implement the Related Cord Blood Donor Demonstration Project, revisit interim program requirements with Advisory Council and public input, and define targets for composition of NCBI and the size and composition of adult donor registry in consultation with the Advisory Council. Finally, to encourage research in collaboration with other Federal agencies and the Advisory Council to improve transplant outcomes, refine approaches to CB transplants for adult patients, better define CBU characteristics required for good outcomes, improve reliability and comparability of measures used in CBU selection, and to collect and report data regarding new uses of stem cells from CBU and adult donors. Dr. Dennis Confer discussed the development of the National Cord Blood Center. The contracts for the Single Point of Access/Office of Patient Advocacy (OPA/SPA) and the Cord Blood Coordinating Center were awarded to the National Marrow Donor Program (NMDP). The contract for the Stem Cell Therapeutic Outcome Database was awarded to the CIBMTR. The OPA/SPA provides a uniform, consolidated search report (all searches are submitted here), provides advocacy services for patients, implements a plan to increase transplants, and facilitates access to transplantation services for patients. The Cord Blood Coordinating Center facilitates transplants of cord blood from NCBI banks and from other participating banks, collects standardized CBU information from the banks and provides this to the OPA/SPA, and coordinates search and distribution of CBUs. The Cord Blood Coordinating Center will also develop an inter-bank technical proficiency program, support public and professional education and recruitment activities, collaborate with CBBs to make CBUs available for research, improve operating efficiencies in collaboration with the network, support contingency planning for emergencies, and require transplant centers to submit outcomes data to the SCTOD. The Center for International Blood and Marrow Transplant Research (CIBMTR) represents an affiliation between the research programs of former IBMTR/ABMTR at the Medical College of Wisconsin and NMDP to support clinical research in hematopoietic cell transplantation and related fields. The CIBMTR as the SCTOD contractor must establish electronic data capture systems, collect data on all allogeneic transplants with a recipient or donor in the U.S, collect data on other cellular therapies, disseminate data within the program, make transplant data publicly available, perform analyses of optimal size for the adult donor registry and cord blood unit inventory, and conduct and support research. In summary, the C.W. Bill Young Transplantation Program provides a blueprint for organizing and managing of unrelated donor hematopoietic cell transplantation, and establishes a clear emphasis on Cord Blood as an important source of hematopoietic and non-hematopoietic cells for treatment of disease. Dr. Ellen Lazarus , representing the FDA, Office of Cellular, Tissue and Gene Therapies, Division of Human Tissues, presented an update on FDA Regulatory Activities for Cord Blood. A draft guidance was published January 16, 2007, and represents FDA’s current thinking but does not establish legally enforceable responsibilities. Its purpose is to recommend ways for cord blood banks to apply for licensure for specified indications, to explain applicable regulations in Title 21 of the Code of Federal Regulations, and to provide other information about the manufacture of human cord blood progenitor cells (HPC-C) and how to comply with the applicable regulatory requirements. The Guidance may be used when applying for a biologics license. The FDA will review the application, schedule a prelicense inspection as soon as possible after receiving the complete application, and if the application is not complete, will identify and advise the establishment of additional information that they will need to submit. The Draft Guidance Sections include factors of significance include HPC-C description and characterization, manufacturer information, methods of manufacturing, and other information. Applicable regulations and post-marketing activities include applicable CGTPs and CGMPs and clinical outcome data collection. An Advisory Committee meeting was held on 4/30/07, at which CB issues under consideration were addressed. The next steps area will be reviewing comments to the docket and recommendations of the Advisory Committee, and finalization of the Guidance. License applications will be accepted at any time.