1. STATE HEALTH PLAN REQUEST FOR REVIEW OF DONOR COVERAGE BENEFITS Presented by: S. Elizabeth Sharf August 2015

2. Background  Hematopoietic stem cell transplantation or HCT (historically called a “bone marrow transplant” or BMT) remains the only curative treatment option for a wide range of diseases that evolve out of the hematopoietic (blood) system such as:  Leukemias Acute Lymphoblastic Leukemia (ALL) Acute Myelogenous Leukemia (AML)  Myelodysplastic syndromes (MDS)  Aplastic Anemia  These patients must undergo a BMT in order to destroy their own blood forming system and replace it with a donor’s.  This type of transplant is called an allogeneic BMT.  An allogeneic transplant is when cells from a family member, unrelated donor or umbilical cord blood unit are used for transplant.  This is also called a related donor transplant if the donor is a family member or an unrelated donor transplant if the donor is not a family member.

3. Solid Organ Donors vs. Bone Marrow Donors  Solid organ donors (heart, kidney, lung, etc.) must be a compatible match with the recipient’s blood type:  A, B, O or AB  Hematopoietic cell transplant (BMT) donors need to be as closely matched as possible to the recipient’s Human Leukocyte Antigens (HLAs) for the best transplant outcomes. 1  This is not the same as ABO blood typing.  HLAs are proteins on almost all cells in the human body.  Found on the surface of white blood cells  Help the immune system tell the difference between body tissue and substances that are not from your own body

4. HLA and Ethnicity  A patient’s likelihood of finding a match within an unrelated donor registry ranges from 66% to 97%, depending on race and ethnicity.  Many people have very complex HLA tissue types, due to human migration over time. This is especially true for individuals of diverse racial and ethnic heritage.  When it comes to matching human leukocyte antigen (HLA) types, a person is more likely to find a donor from the same racial or ethnic background. This is because HLA markers used in matching are inherited.

5. BMT Benefit Language For patients who need an allogeneic transplant, the State Health Plan has benefit coverage for:  the work-up of the selected donor,  the procurement of the donor cells  the coverage of the transplant event.

6. BMT Donor Search Restrictions The current donor search coverage provided through the NC State Health Plan has a maximum benefit of $10,000

7. Finding a Donor NC TRANSPLANT CENTERS Duke University UNC Hospitals Wake Forest Baptist Health Carolinas Medical Center About 70%, or 7 out of 10 patients, who need a transplant do not have a suitable HLA donor match within their family. Buccal Swab Blood Sample

8. FatherMother A 01:01 A 68:02A 02:01 A 11:01 B 08:01 B 14:02B 18:01 B 40:01 DRB1 03:01 DRB1 13:03 DRB1 03:01 DRB1 13:02 Patient A 01:01A11:01 B 08:01B40:01 DRB103:01DRB113:02 EXAMPLE of FAMILY HLA TYPING

9. Child 1Child 2Child 3 A 01:01 A 02:01 A 68:02 A 11:01 A 68:02 A 02:01 B 08:01 B 18:01 B 14:02 B 40:01 B 14:02 B 18:01 DR 03:01 DR 03:01DR 03:01 DR13:02 DR 13:03 DR 03:01 Child 4 PATIENT – Child 5 A 23:01 A 11:01 A 01:01 A 11:01 B 44:03 B 40:01 B 08:01 B 40:01 DR 15:03 DR13:02DR 03:01 DR 13:02 FatherMother A 01:01 A 68:02A 02:01 A 11:01 B 08:01 B 14:02B 18:01 B 40:01 DRB1 03:01 DRB1 13:03 DRB1 03:01 DRB1 13:02

10. Finding an Unrelated Donor When no family members are a match, an unrelated donor is required and searches are performed through donor registries such as Be The Match Registry or DKMS.

11. Costs  Approximate cost to have HLA typing performed at a large university hospital can be $3,000/donor.  If HLA typing must be performed on an unrelated donor, additional registry charges* will be incurred:  One time formal activation fee = $1095  Sample obtained from domestic donor = $ 530  Infectious Disease Results = $ 425  Sample obtained from international donor = $ 400-$2300 * = approximate

12. Potential Cost of Unrelated Domestic Donor HLA Typing  $955 to obtain the blood sample, send to the transplant center for typing and infectious disease markers performed  $3000 to perform HLA typing at a large university hospital __________________________________________  $4,000 total cost/unrelated domestic donor typing

13. Cost and Timing  $10,000 Donor Search Benefit  This amount can be quickly exceeded and create a barrier to a successful outcome for the transplant recipient.

14. Strategies to stay within the $10K benefit While these strategies may protect the patient financially, it may also cause an unnecessary delay in securing an allogeneic donor.  Counsel patients regarding donor search limitations and obtain their permission to potentially exceed $10K in order to find a donor in a timely fashion  Seek financial assistance for the patient through the donor registry grants prior to activating a formal search – may not qualify  Utilize search strategy analysis to select best donors

15. BMT Statistics - State of NC  The current population of NC is 9.9M  In 2013, 256 allogeneic transplants were performed in NC (Source: HRSA website)  SHP members comprise 7% of NC population 700,000 members ( Source: SHP website)  Using 7% SHP/NC population, 18 of those 256 transplants would be for SHP members

16. Approximate additional Costs to the SHP If donor search coverage benefits were raised from $10K to $50K:  18 transplants x $40K (additional donor coverage) = $720K FY14 SHP expenses = 2.83B (SHP website)  $720K additional benefit coverage represents an increase of 0.025% This hypothetical projection does not include those patients who have donor searches performed but will never get to transplant.

17. The need to find a donor can happen to anyone… Orange County Judge Carl Fox

18. …when we least expect it.

19. An increase in the donor search coverage benefit maximum would allow the employees of the state of North Carolina to be able to search for the best possible donor in the most timely fashion which should be the goal for SHP members. In Conclusion

20. QUESTIONS? sam.sharf@unchealth.unc.edu Thank you for the opportunity to present this information.