Implementation of the Stem Cell Therapeutic and Research Act of 2005

Transcription

1 Implementation of the Stem Cell Therapeutic and Research Act of 2005 The 6 th Annual Somatic Cell Therapy Symposium Bethesda, MD September 26, 2006 James F. Burdick, M.D. U.S. Department of Health and Human Services (HHS), Health Resources and Services Administration (HRSA), Division of Transplantation

2 Presentation Outline Information about HRSA Stem Cell Therapeutic and Research Act of Background & Overview HRSA s Implementation Approach Focus on 3 Program Components: National Cord Blood Inventory Cord Blood Coordinating Center Advisory Council Future Work

3 Health Resources and Services Administration (HRSA) Mission: HRSA provides national leadership, program resources and services needed to improve access to culturally competent, quality health care HRSA focuses on uninsured, underserved, and special needs populations HRSA Goals Goal 1: Improve Access to Health Care Goal 2: Improve Health Outcomes Goal 3: Improve the Quality of Health Care Goal 4: Eliminate Health Disparities Goal 5: Improve the Public Health and Health Care Systems Goal 6: Enhance the Ability of the Health Care System to Respond to Public Health Emergencies Goal 7: Achieve Excellence in Management Practices

4 Health Resources and Services Administration, continued HRSA Programs include: Community Health Centers HIV services under the Ryan White CARE Act Maternal and Child Health Programs Health Professions Development Programs National systems to facilitate transplantation: Solid Organs Organ Procurement & Transplantation (UNOS) Scientific Registry of Transplant Recipients (URREA) Blood Stem Cells National Bone Marrow Donor Registry (NMDP) Stem Cell Therapeutic and Research Act of 2005

5 Stem Cell Therapeutic and Research Act of 2005 (P.L ) Authorizing law, preceded by efforts to: Enact authorizing legislation for collection and distribution of cord blood units (CBU) Re-authorize the National Bone Marrow Donor Registry Appropriations for a National Cord Blood Stem Cell Bank Program, FY Conference Report required an Institute of Medicine (IOM) study; report issued April 14, 2005 Appropriations totaled nearly $24 million (including $1 million specified for IOM study)

6 P.L , continued Signed December 20, 2005 Aims are to increase: Number of unrelated donor transplants Public inventory of high quality CBU from diverse populations Number of CBU available for research Very exciting opportunity to help more patients obtain transplants, other therapy

7 P.L , continued Four Sections Sec.1: Short Title Sec.2: National Cord Blood Inventory Sec.3: C.W. Bill Young Cell Transplantation Program (the Program) Sec.4: Report on status of FDA Licensure of CBU

8 P.L : Section 2 National Cord Blood Inventory (NCBI) HRSA funding for high quality, diverse CBU Target of 150,000 new units Authorizes appropriations of $15 million/year, FY Federal funding temporary until 150,000 CBU Funding structure: One-time contracts Federal funding limited to 3 years CBB must participate in the Program for 10 years NCBI CBU must be available through the Program in perpetuity Related CB Demonstration, FY Some CBU not appropriate for transplantation to be available for research

9 P.L : Section 3 C.W. Bill Young Cell Transplantation Program (successor to National Bone Marrow Donor Registry) Provides the infrastructure for the Program Facilitation of transplants and outcomes data collection Up to 5 components established by HRSA contracts Advisory Council at HHS level Provisions regarding accrediting organization(s) for CBB, informed consent for donating CBU Federal funding intended to be ongoing Authorizes $38 million/year, FY Approximately $25 million available in FY 2006

10 Program Components Department of Health and Human Services Advisory Council HRSA/Division of Transplantation Accrediting Organizations Cord Blood Banks Cord Blood Coordinating Center Outcomes Database Bone Marrow Coordinating Center Blood Stem Cell Single Point of Access Office of Patient Advocacy Possible New HRSA Contract Organizations Other New Organizations or Relationships Transplant Centers Patients Referring Physicians

11 HRSA s Implementation Approach Guided by three principles: Single Point of Access for patients and physicians to all sources of blood stem cells Collection of high-quality diverse CBU, expeditiously Complete data on clinical outcomes of transplants Recognizes field is evolving rapidly Informed by extensive consultation: Request for Information published August 15, 2005 Teleconferences with transplant physicians Consultation with reps. of ASBMT Teleconferences with public and private CBB

12 HRSA s Implementation Approach, continued Consultation, continued Site visits to public and private cord blood banks Teleconferences on informed consent Correspondence with accrediting organizations Teleconferences on accreditation (announced in Federal Register) Discussions with experts in CBU processing Consultation with other Federal agencies (e.g., NIH, FDA, CDC) Congressional briefings

13 HRSA s Implementation Approach, continued Parallel establishment of program components Contract components - competitive contracts Cord Blood Banks to collect NCBI Infrastructure components Advisory Council Accrediting Organization(s) During transition, HRSA will extend National Bone Marrow Donor Registry contract To ensure continuity of service

14 HRSA s Implementation Approach, continued Contract awards by 9/30/06 require interim approaches to: Technical requirements for CBB and HRSAfunded CBU Recognition of accrediting organization(s) Requirements regarding informed consent Interim provisions to be revisited in future years, with Advisory Council and public input

15 HRSA s Implementation Approach, continued The following schematic shows the contract structure for the program Does not show relationships for: Facilitating transplants Reporting Outcomes Data Program oversight

16 C.W. Bill Young Cell Transplantation Program: Contracting Structure Department of Health and Human Services $ HRSA/Division of Transplantation Advisory Council $ $ $ $ $ Accrediting Organizations Infrastructure Cord Blood Banks Cord Blood Coordinating Center Outcomes Database Bone Marrow Coordinating Center Public Interface Blood Stem Cell Single Point of Access Access Point for Stem Cell Sources Patient Advocacy Services HRSA Contract Organizations Other New Organizations or Relationships Transplant Centers Patients Referring Physicians

17 National Cord Blood Inventory: Highlights of HRSA Approach Banks funded through one-time, 10-year contracts, awarded competitively HRSA funds for 3 years Annual competitions for new cohorts of banks Add banks in new geographic areas fine-tune diversity of NCBI Permit funding new banks w/o long wait for funding opportunity Broaden the opportunity for Federal support Encourage improvement in quality across the industry Permit timely adjustments based on bank performance Available funds: ~ $14M for FY 2006; ~ $8M for FY 2007 HRSA reimbursement at fixed rate per unit, for X units HRSA expects to negotiate discounted prices necessary to make progress toward 150,000 units

18 National Cord Blood Inventory: Highlights of HRSA Approach, continued Interim technical requirements for NCBI (HRSA reimbursable) CBU include: Total nucleated cell count (TNC) 90 x 10 7 (without subtraction of nucleated red blood cells) CFU-GM assay must show growth TNC Viability 85% CD34+ content / viability measured no min. value At least 2 attached segments Negative for: infectious disease markers, bacteria and fungi, and clinically significant hemoglobinopathies Minimum human leukocyte antigen (HLA) typing: low/intermediate A,B; DR; all by DNA methods

19 National Cord Blood Inventory (NCBI): Highlights of HRSA Approach, continued Bank eligibility requirements include: Accreditation by an organization recognized by the Secretary Pending such recognition, accreditation by FACT or AABB Minimum public inventory of 500 CBU collected and banked by the CBB Request for Proposals (RFP) Issued April 28, 2006 Proposals received June 5, 2006 Awards to be made by ~September 30, 2006

20 Cord Blood Coordinating Center (CBCC): Highlights of HRSA Approach Functions include: Facilitate transplants with CBU from NCBI banks (NCBI CBU, Other CBU) Other participating banks Provide standardized search information on CBU to the Single Point of Access Facilitate further testing of CBU being evaluated for a patient Facilitate distribution of CBU Require submission of outcomes data Assist banks in public education and recruitment

21 Cord Blood Coordinating Center (CBCC): Highlights of HRSA Approach, continued Functions, continued: Coordinate contingency planning with banks Coordinate inter-bank proficiency testing program Compile data on CBU released for research RFP (published May 23, 2006) does not prescribe or prohibit a particular model or technology, consistent with Congressional direction RFP allows for subcontracting and consortia arrangements CBCC operating policy/procedures subject to HRSA approval CBCC will coordinate with other Program components through several working groups

22 Advisory Council Purpose Consider and make recommendations to the Secretary on matters related to the Program Steps in establishment under Federal Advisory Committee Act (FACA) include Draft & Final establishment packages to HHS Publish charter and call for nominations in the Federal Register Draft & Final Nomination Packages to HHS Secretary appoints members Announce first meeting in the Federal Register

23 Advisory Council, continued Membership Statute lists 15 required categories voting and non-voting ex officio members Other categories recommended to HRSA in RFI responses Number and categories of members will be specified in Federal Register Notice

24 Advisory Council, continued Statutory membership categories: Voting members: Reps of marrow donor centers and marrow transplant centers Reps of cord blood banks and participating birthing hospitals Recipients of bone marrow transplant Recipients of cord blood transplant Persons who require such transplants Family members of a recipient or a patient who has requested the assistance of the Program in searching for an unrelated donor of bone marrow or cord blood Persons with expertise in bone marrow and cord blood transplantation Persons with expertise in typing, matching, and transplant outcome data analysis Persons with expertise in the social sciences Basic scientists with expertise in the biology of adult stem cells Members of the general public Non-voting, ex-officio members: HRSA, FDA, NIH, DoD

25 Advisory Council, continued Other membership categories suggested to HRSA: Voting Members Representatives from transplant centers with extensive experience in unrelated donor transplants, including cord blood transplants Hematology and transfusion medicine researchers with expertise in adult stem cells from adult donors and cord blood Ethicists Experts in Obstetrics Experts in Cord Blood Processing Experts in Bio-informatics Representatives from Accreditation Programs Donor and Donor Representatives (family members of cord blood and adult donors) Non-voting, ex-officio members: CMS, CDC

26 Advisory Council, continued Possible Topics for Advisory Council: Targets for National Cord Blood Inventory & Adult Registry size & composition Research Priorities HPC transplantation Other therapy with cells from CB & BM Criteria for units for research CBU characteristics needed for good outcomes Criteria for choosing best stem cell source

27 Advisory Council, continued Possible Topics for Advisory Council: Program priorities based on reports of activity and performance Public & Professional Education - re donation Regulatory Policy including international harmonization of regulation Public and Private Insurers actions to increase donation and access to transplant State and Federal Government - actions (other than as insurers)

28 Some Work Beyond September Complete transition to new program structure Complete establishment of Advisory Council Revisit interim program requirements, with Advisory Council and public input, esp. re Accreditation Informed Consent Definition of a high-quality CBU Define targets for composition of NCBI, and size & composition of adult donor registry, in consultation with Advisory Council

29 Some Work Beyond September, continued Design & begin the Related Cord Blood Demonstration Project Future cycles of funding for CB banks Encourage research, in collaboration with other Federal agencies and Advisory Council, to, e.g.: Improve transplant outcomes CB and adult donor 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 Collect and report data re new uses of stem cells from CBU and adult donors

30 References Stem Cell Therapeutic and Research Act of 2005 (P.L ) Senate Report IOM Report National Cord Blood Inventory - Request for Proposals tml Cord Blood Coordinating Center Request for Proposals g.html

31 THANK YOU for your attention QUESTIONS?

32 My Contact Information James F. Burdick 5600 Fishers Lane, Room 12C-06 Rockville, Maryland