1. ` Division of Transplantation/OSP/HRSA Allocation of Pancreata for Whole Organ and Islet Transplantation James Burdick, M.D. Director Health Resources and Services Administration Division of Transplantation BRMAC Meeting October 10, 2003

2. Division of Transplantation/OSP/HRSA National Organ Transplant Act (NOTA) of 1984 Created Organ Procurement and Transplantation Network (OPTN) Prohibited purchase of transplantable organs (Section 301)

3. Division of Transplantation/OSP/HRSA Section 1138 of the Social Security Act A hospital that performs organ transplants must be a member and abide by the rules of the OPTN

4. Division of Transplantation/OSP/HRSA Transplant Programs 246 kidney 124 liver 139 pancreas 37 pancreas islet cell 44 intestine 139 heart 73 heart/lung 70 lung

5. Division of Transplantation/OSP/HRSA Islet Program Membership Currently, there are no OPTN membership criteria for islet programs The OPTN Kidney-Pancreas Transplantation Committee has developed criteria Public comment period closed October 4, 2003 for most recent proposal on islet program membership Proposed criteria will be submitted to the OPTN Board of Directors for final approval in November

6. Division of Transplantation/OSP/HRSA Proposed Islet Program Membership Criteria Must be located in a hospital at a center approved to perform whole pancreas transplants Must provide data on patients transplanted Must report the disposition of the islets (transplanted, discarded, other use) Must have a qualified surgeon, physician, and radiologist Must have access to ancillary personnel

7. Division of Transplantation/OSP/HRSA OPTN Policy 3.2 Waiting List All patients who are potential recipients of deceased organ transplants must be listed on the computer Waiting List OPTN Members shall not provide organs to non-member transplant centers

8. Division of Transplantation/OSP/HRSA Waiting List Size Candidates waiting as of September 19, 2003: 1445 pancreas 2418 kidney/pancreas 164 islet

9. Division of Transplantation/OSP/HRSA Allocation of Pancreata for Islet Transplantation Current OPTN allocation algorithm for pancreata gives priority for whole organ transplantation OPTN has approved requests for variances giving higher priority for allocation of pancreata for use in islet transplantation locally Approved variances generally limit the number of pancreata diverted for use in islet transplantation

10. Division of Transplantation/OSP/HRSA Variances Under Final Rule Experimental policies that test methods of improving allocation Accompanied by a research design Include plans for data collection and analysis Time limited Subject to same approval process as standard policies

11. Division of Transplantation/OSP/HRSA Current Pancreas Allocation Algorithm Candidates ranked by length of time on the waiting list Current algorithm gives highest priority to: –0-mismatch and highly sensitized candidates –Isolated and combined whole pancreas transplantation Efforts are made to place the pancreas locally, regionally, then nationally for whole pancreas transplantation before offering it for clinical islet transplantation If a pancreas still is not accepted, it is offered for research use (not clinical research)

12. Division of Transplantation/OSP/HRSA Proposed Pancreas Allocation Algorithm Donor age  50 years AND BMI  30 kg/m² Local isolated or combined whole pancreas, or combined solid organ-islet; then Regional then national isolated or combined whole pancreas; then Local, regional, then national islet; then Research

13. Division of Transplantation/OSP/HRSA Proposed Pancreas Allocation Algorithm (Cont.) Donor age > 50 years OR BMI > 30 kg/m² Local isolated or combined whole pancreas, or combined solid organ-islet; then Local, regional, then national islet; then Regional then national isolated or combined whole pancreas; then Research

14. Division of Transplantation/OSP/HRSA Issues/Concerns Non-OPTN member institutions doing islet transplants –FDA IND does not take into account that non- OPTN member institutions can not receive pancreata for clinical transplantation Pancreata allocated for research being used for clinical islet transplantation

15. Division of Transplantation/OSP/HRSA OPTN Response Proposal by the Kidney-Pancreas Committee All pancreata for clinical islet transplantation must be allocated through the OPTN/UNOS allocation system Pancreata initially allocated for research cannot be used for clinical transplantation unless re-allocated through the system

16. Division of Transplantation/OSP/HRSA Issues/Concerns (Cont.) Allocation of pancreas for islets –Pancreas is allocated to a specific patient on waiting list, not an institution –Allocation based on need, equitable access Any difference in cost/reimbursement for licensed product vs IND product?

17. Division of Transplantation/OSP/HRSA Issues/Concerns (Cont.) Islets as a licensed/commercial product –May shift procurement focus towards ideal islet donors –May shift a center’s listing focus –Dual regulation (HRSA & FDA) –NOTA section 301 implications?

18. Division of Transplantation/OSP/HRSA Issues/Concerns (Cont.) Whole pancreas transplantation is a proven therapy—how does islet transplantation compare Patients adequately informed about both options (whole pancreas vs. islet transplant) Transplant program expertise –Surgical management of complications of islet transplant procedure –Management of immunosuppression

19. Division of Transplantation/OSP/HRSA Contact Information James Burdick, M.D. (301) 443-7577 jburdick@HRSA.gov DoT Website [www.hrsa.gov/osp/dot] –OPTN Final Rule OPTN Website [www.optn.org] SRTR Website [www.ustransplant.org]