1. OPTN Differences in the Opportunity for Transplantation within the US Co-chairs: S. McDiarmid MD and K. Olthoff MD Working Group 4: “Improve the Efficiency, Effectiveness, and Equity of the OPTN’s Policies and Processes”

2. OPTN Working Group 4 Members M Barr MD M Barr MD A Benedi A Benedi J Burdick MD J Burdick MD B Cosimi MD B Cosimi MD R Freeman MD R Freeman MD D La-Pointe-Rudow RN D La-Pointe-Rudow RN J Lake MD J Lake MD M Levy MD M Levy MD B Nuesse RN, BSN B Nuesse RN, BSN UNOS STAFF C Sommers JD C Sommers JD M McBride M McBride P Oldham BSS P Oldham BSS P Schaeffer RN, BSN P Schaeffer RN, BSN J Shaftel RN J Shaftel RN D Surlas RN D Surlas RN R Wiesner MD R Wiesner MD H H Wong MD JD H H Wong MD JD M Zamora MD M Zamora MDGUESTS R Durbin MBA R Durbin MBA H Krakauer MD PhD H Krakauer MD PhD A Leichtman MD A Leichtman MD G McBride RN MPH G McBride RN MPH F Port MD PhD F Port MD PhD M Stegall MD M Stegall MD Thankyou!!

3. OPTN Differences in Transplantation in the US Ethical Imperative Candidates for solid organ transplantation have life threatening disease Candidates for solid organ transplantation have life threatening disease Given there are not enough available organs, the allocation and distribution of a life threatening resource should not advantage or disadvantage any one individual or group of individuals, with similar medical characteristics, over another Given there are not enough available organs, the allocation and distribution of a life threatening resource should not advantage or disadvantage any one individual or group of individuals, with similar medical characteristics, over another

4. OPTN Differences in Transplantation in the US Federal Imperative – the Final Rule “inter-transplant program variance to be as small as can be reasonably achieved in any performance indicator” “inter-transplant program variance to be as small as can be reasonably achieved in any performance indicator”

5. OPTN Differences in Transplantation in the US What determines variation in who gets transplanted? Access to the list Access to the list Access to the donor organ Access to the donor organ What data demonstrates the major cause of differences in transplantation? What is the measure we should be trying to make more equitable?

6. OPTN Differences in Transplantation in the US Performance Indicators Common to all Organs Access to the waiting list Access to the waiting list Death (or removal ‘too sick’ for transplant) on waiting list Death (or removal ‘too sick’ for transplant) on waiting list Rate of transplantation Rate of transplantation Missed opportunities when active on the list Missed opportunities when active on the list Rate of organ refusal Rate of organ refusal DSA performance DSA performance allocation efficiency allocation efficiency

7. OPTN Differences in Transplantation in the US Performance Indicators can be evaluated for various patient groups: examples Age Age Ethnicity Ethnicity Severity of illness Severity of illness Diagnosis Diagnosis Insurance status Insurance status Center effects Center effects Patient location - geography Patient location - geography

8. OPTN Differences in Transplantation in the US Current system of geographical boundaries which define patient location UNOS defined regions. UNOS defined regions. Donor Service Areas (DSAs) Donor Service Areas (DSAs)

9. OPTN PR & US VI HI DE MD MA RI Differences in Transplantation in the US UNOS Regions 1 6 5 11 4 3 2 10 7 9 8

10. OPTN Differences in Transplantation in the US How were Regions established?  With establishment of the OPTN: for administrative and representative purposes  Intended to recognize existing relationships, local interests, and diversity across country  No intent necessarily to equalize populations or number of centers across the country

11. OPTN Differences in Transplantation in the US Donor Service Areas

12. OPTN Differences in Transplantation in the US How were DSAs established?  Generally intended as the first unit of organ distribution  DSA territories designated by CMS  Variable in terms of: - Number of transplant centers - Square mileage; inclusion of a part of a state, entire state, or multiple states - Population, candidate/donor ratios and characteristics, and procurement rates

13. OPTN Differences in Transplantation in the US Given different sources of possible variation why focus on geographic differences based on DSAs and UNOS regions? Together they form the basis of the current allocation and distribution system for deceased donor organs. Together they form the basis of the current allocation and distribution system for deceased donor organs. Boundaries of DSAs and UNOS regions not developed for purposes of organ distribution - a system that is feasible to be changed Boundaries of DSAs and UNOS regions not developed for purposes of organ distribution - a system that is feasible to be changed Most other variables – age, ethnicity, disease etc can not be changed with possible exceptions of insurance/payor status and center effects Most other variables – age, ethnicity, disease etc can not be changed with possible exceptions of insurance/payor status and center effects

14. OPTN Differences in Transplantation in the US As we consider geography: Policies of organ distribution have been built on a basic principle of patient and donor location: Policies of organ distribution have been built on a basic principle of patient and donor location: local first, region second, national last local first, region second, national last Is this valid for all organs? Is this valid for all organs? Is kidney different? Is kidney different?

15. OPTN Differences in Transplantation in the US Patient Location – the effect of ‘geography’: Issues- Who owns the organ – the locale in which it was recovered? Who owns the organ – the locale in which it was recovered? “the unifying force of biology requires at least a national perspective” Henry Krakauer Henry Krakauer Are patients harmed by geographic variations in access to transplantation? Are patients harmed by geographic variations in access to transplantation? Some regional variation is to be expected – how much is acceptable? Some regional variation is to be expected – how much is acceptable?

16. OPTN Differences in Transplantation in the US Who gets transplanted? Who gets transplanted? Performance Indicator: Access to the Waiting List

17. OPTN Differences in Transplantation in the US Access to the Waiting Listing Is this the purview of the OPTN/UNOS? Is this the purview of the OPTN/UNOS? Is the scope of our concern only after a candidate is listed? Is the scope of our concern only after a candidate is listed?

18. OPTN Differences in Transplantation in the US Access to the waiting list dependent on: Incidence and prevalence of end-organ failure Incidence and prevalence of end-organ failure Medical practice patterns Medical practice patterns Referral patterns Referral patterns Listing criteria Listing criteria Patient education Patient education Insurance/payor regional differences Insurance/payor regional differences

19. OPTN Differences in Transplantation in the US Should the OPTN try to influence medical care and practice for patients eligible for transplantation? YES: Otherwise the benefit of transplantation is denied to this group of eligible, yet unlisted, eligible patients Within the scope of the Final Rule’s requirement to develop standardized listing criteria as well as policies to address socioeconomic impediments to transplantation

20. OPTN Differences in Transplantation in the US Available Data Evaluating Access to Waiting List Quantitative for kidney – good data available from USRDS Quantitative for kidney – good data available from USRDS Qualitative for heart and liver Qualitative for heart and liver - based on an estimate of the incidence of end stage disease in a given population unit compared to the actual percent of the predicted eligible population actually listed - based on an estimate of the incidence of end stage disease in a given population unit compared to the actual percent of the predicted eligible population actually listed

21. OPTN Alan Leichtman MD

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30. OPTN Who gets transplanted? Who gets transplanted? Performance Indicators after listing Performance Indicators after listing Death (removal) on the list Death (removal) on the list Transplant Rate Transplant Rate - both can controlled for several variables such as age, diagnosis, medical urgency, geography - both can controlled for several variables such as age, diagnosis, medical urgency, geography Which variable(s) is most relevant?

31. OPTN Differences in Transplantation in the US Existing Differences in Performance Indicators after listing Given Final Rule emphasis to prioritize allocation of DD organs by medical urgency one approach to evaluating geographic differences should compare death on list and transplant rates by severity of illness

32. OPTN Differences in Transplantation in the US How is Medical Urgency Currently Defined? Organ Specific: Liver: MELD/PELD system Liver: MELD/PELD system Heart:: status categories – 1a,1b, 2 Heart:: status categories – 1a,1b, 2 Lung: risk of death without transplant Lung: risk of death without transplant Kidney: net lifetime survival benefit?? Kidney: net lifetime survival benefit??

33. OPTN Differences in Transplantation in the US Liver MELD/PELD severity of illness has been well validated as an accurate measure of probability of death within 3 months of listing MELD/PELD severity of illness has been well validated as an accurate measure of probability of death within 3 months of listing In place since Feb 2002 In place since Feb 2002 Extensive data already available examining regional and DSA differences in death waiting and rate of transplant Extensive data already available examining regional and DSA differences in death waiting and rate of transplant

34. OPTN Probability of Transplant and Death/Too Sick within One Year for Adult Liver Registrations Added 3/1/2002-2/29/2004 By Region Initial MELD Score 15+

35. OPTN Probability of Transplant and Death/Too Sick within One Year for Adult Liver Registrations Added 3/1/2002-2/29/2004 By DSA Initial MELD Score 15+

36. OPTN Differences in Transplantation in the US Heart: Severity of illness measured by status 1a, 1b, 2

37. OPTN Probability of Transplant and Death/Too Sick within One Year for Adult Heart Registrations Added 1/1/2002-12/31/2004 By Region Status 1A at Listing

38. OPTN Probability of Transplant and Death/Too Sick within One Year for Adult Heart Registrations Added 1/1/2002-12/31/2004 By DSA Status 1A at Listing

39. OPTN Percent of Adult Heart Transplants Status 1A for Transplants Performed During 1/1/04- 6/30/05 by DSA

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41. OPTN Differences in Transplantation in the US Kidney What is relevant measure of severity of illness? How can comparable patients be identified?

42. OPTN Probability of Transplant and Death/Too Sick within One Year for Adult Kidney Registrations Added 1/1/2002-12/31/2004 By Region PRA < 20% at Listing

43. OPTN Probability of Transplant and Death/Too Sick within One Year for Adult Kidney Registrations Added 1/1/2002-12/31/2004 By DSA PRA < 20% at Listing

44. OPTN

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46. OPTN Differences in Transplantation in the US Pancreas Unique in solid organ transplantation Unique in solid organ transplantation The supply of solitary pancreata generally is greater than the demand The supply of solitary pancreata generally is greater than the demand Simultaneous Pancreas and Kidney is limited primarily by the availability of the kidney Simultaneous Pancreas and Kidney is limited primarily by the availability of the kidney Major issues facing pancreas and islet allocation involve procurement and placement Major issues facing pancreas and islet allocation involve procurement and placement

47. OPTN Differences in Transplantation in the US Other Sources of Differences related to Geography DSA Performance DSA Performance Differences in donor quality Differences in donor quality

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50. OPTN Differences in Transplantation in the US Conclusions from the Data Patient location, as currently defined by Regions and DSAs, is the most important cause of differences in access to listing and transplantation Patient location, as currently defined by Regions and DSAs, is the most important cause of differences in access to listing and transplantation Is it right that where I live can dictate my chances of getting on a list, getting a transplant and of dying on the list Is it right that where I live can dictate my chances of getting on a list, getting a transplant and of dying on the list

51. OPTN Differences in Transplantation in the US What is the right metric to assess differences once listed? What is the right metric to assess differences once listed? For comparison of differences in access to transplantation based on patient location, what should we be seeking to ‘equalize’ across the US?: For comparison of differences in access to transplantation based on patient location, what should we be seeking to ‘equalize’ across the US?: the person who is sickest? the person who is sickest? the person to gain the most benefit from receiving an organ? the person to gain the most benefit from receiving an organ?

52. OPTN Differences in Transplantation in the US Working Group Recommendation: Working Group Recommendation: “The access to an organ should be similar across the US for patients with a similar transplant benefit”

53. OPTN Differences in Transplantation in the US Benefit of Transplantation: How to define? Organ specific. For liver and heart (lung)– urgency of tx to save life paramount For liver and heart (lung)– urgency of tx to save life paramount General consensus General consensus - net life years gained most relevant - net life years gained most relevant Quality of life? how best to measure Quality of life? how best to measure - how relevant is QOL in the face of critical organ shortage for immediately life saving transplantation for liver and heart (lung) recipients - how relevant is QOL in the face of critical organ shortage for immediately life saving transplantation for liver and heart (lung) recipients

54. OPTN Differences in Transplantation in the US Liver – Benefit of Transplant Using MELD score concept of who would most benefit from receiving a donor liver at the time of offer was developed. Using MELD score concept of who would most benefit from receiving a donor liver at the time of offer was developed.

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61. OPTN Differences in Transplantation in the US Lung – Benefit of Transplant Combination of : risk of death without transplant risk of death without transplant probability of post transplant survival probability of post transplant survival

62. OPTN Differences in Transplantation in the US Heart: Benefit Net life years gained with transplant Net life years gained with transplant Status 1a and 1b benefit most from transplant Status 1a and 1b benefit most from transplant Proposed broader sharing for 1a and 1b Proposed broader sharing for 1a and 1b

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65. OPTN Differences in Transplantation in the US Kidney – Definition of benefit Net lifetime survival benefit = survival with transplant – survival without transplant Then compare time to transplant for “comparable” patients

66. OPTN Differences in Transplantation in the US Pancreas Can use the same Net Lifetime Survival Benefit May not be necessary in ranking for allocation given the lack of scarcity

67. OPTN Differences in Transplantation in the US Conclusions: Geography is the most important cause of differences in access to transplantation both before and after listing Geography is the most important cause of differences in access to transplantation both before and after listing Patients, and the lives lost, are the victim of these differences Patients, and the lives lost, are the victim of these differences

68. OPTN Differences in Transplantation in the US Conclusions: Given the arbitrary geographic definitions of regions and DSAs, the differences proven by current data mandate that an urgent re-evaluation of the geographic units of organ distribution is undertaken Given the arbitrary geographic definitions of regions and DSAs, the differences proven by current data mandate that an urgent re-evaluation of the geographic units of organ distribution is undertaken

69. OPTN Differences in Transplantation in the US Conclusions: The data are compelling: the magnitude of the differences in opportunity for transplantation for patients within the US is in direct contravention of both ethical principles of providing life saving medical care without prejudice, and the mandate of the Final Rule The data are compelling: the magnitude of the differences in opportunity for transplantation for patients within the US is in direct contravention of both ethical principles of providing life saving medical care without prejudice, and the mandate of the Final Rule

70. OPTN Differences in Transplantation in the US Conclusions: We know better now how to rank the order in which patients should be transplanted based on their degree of medical urgency, (allocation) but we have made little progress in getting the organ to the appropriately identified patients (distribution). We know better now how to rank the order in which patients should be transplanted based on their degree of medical urgency, (allocation) but we have made little progress in getting the organ to the appropriately identified patients (distribution).

71. OPTN Differences in Transplantation in the US Conclusions: Transplant benefit, defined for each organ, is the metric by which comparable access to transplantation should be measured. Transplant benefit, defined for each organ, is the metric by which comparable access to transplantation should be measured. Transplant benefit is inherently just – not a utility tool Transplant benefit is inherently just – not a utility tool

72. OPTN Differences in Transplantation in the US Timeline for Progress Timeline for Progress Definition and evaluation of transplant benefit are currently at different stages for each organ Definition and evaluation of transplant benefit are currently at different stages for each organ Will need experience with new systems Will need experience with new systems Evaluations of accuracy and completeness Evaluations of accuracy and completeness Then able to begin measuring differences in access for candidates with similar transplant benefit Then able to begin measuring differences in access for candidates with similar transplant benefit

73. OPTN Differences in Transplantation in the US Question: System will need to accept some degree of variation System will need to accept some degree of variation How much is acceptable? How much is acceptable?

74. OPTN Differences in Transplantation in the US Challenges in Changing Distribution Limiting cold ischemia time – what is reasonable? Limiting cold ischemia time – what is reasonable? Organ specific Organ specific Does it vary with donor quality Does it vary with donor quality Is there any justification for the current concept of ‘local first’ for liver, heart, lung donors? Is there any justification for the current concept of ‘local first’ for liver, heart, lung donors?

75. OPTN Differences in Transplantation in the US Challenges in Changing Distribution Currently the local preference concept is driven by the OPO being the initiating factor in donor distribution Currently the local preference concept is driven by the OPO being the initiating factor in donor distribution Broader sharing of donor livers and hearts already in place and modeling predicts that death on the waiting list will be reduced Broader sharing of donor livers and hearts already in place and modeling predicts that death on the waiting list will be reduced How ‘broad’ can ‘broad’ be ? How ‘broad’ can ‘broad’ be ?

76. OPTN Differences in Transplantation in the US Challenges in Changing Distribution Should units of distribution be based on equalizing population density? Should units of distribution be based on equalizing population density? Distance from donor to recipient based on population density in place for kidney distribution in New England Distance from donor to recipient based on population density in place for kidney distribution in New England Should distance from donor to potential recipient be a factor? Should distance from donor to potential recipient be a factor? - concentric circles based on distance from donor: in place for heart distribution - concentric circles based on distance from donor: in place for heart distribution

77. OPTN Differences in Transplantation in the US Previous attempts to change distribution units. In 2000-2001 liver models were examined changing organ distribution boundaries – pre MELD In 2000-2001 liver models were examined changing organ distribution boundaries – pre MELD Combined contiguous DSAs in 7 different configurations to cover populations >9 million Combined contiguous DSAs in 7 different configurations to cover populations >9 million 17 different allocation sequences modeled 17 different allocation sequences modeled Inter-region variation reduced but fewer transplants and more deaths Inter-region variation reduced but fewer transplants and more deaths Freeman et al. Liver Trans 2002; 8:659 Freeman et al. Liver Trans 2002; 8:659 WHY?? WHY??

78. OPTN Differences in Transplantation in the US Challenges in Changing Distribution Focus was only on patient location - no account taken of medical urgency Focus was only on patient location - no account taken of medical urgency Need to “focus more on the patients who need it most, more than where they live” Freeman Need to “focus more on the patients who need it most, more than where they live” Freeman

79. OPTN Differences in Transplantation in the US Recommendations to the Board: Establish a central resource of expertise – both clinical and statistical, to develop organ specific proposals to reduce differences in transplantation, as a result of patient location, for patients with a similar transplant benefit Establish a central resource of expertise – both clinical and statistical, to develop organ specific proposals to reduce differences in transplantation, as a result of patient location, for patients with a similar transplant benefit

80. OPTN Differences in Transplantation in the US Recommendations to the Board: Require each organ specific committee to examine and discuss the proposals and concepts, requesting additional data and modeling as needed, and propose policies that would reduce geographic differences Require each organ specific committee to examine and discuss the proposals and concepts, requesting additional data and modeling as needed, and propose policies that would reduce geographic differences

81. OPTN Differences in Transplantation in the US Recommendations to the Board: Consider the effect of allowing local/regional variances to continue – may confound effects of new policies Consider the effect of allowing local/regional variances to continue – may confound effects of new policies Consider the likely impact, and response from small centers, if ‘local first’ concept is largely made obsolete Consider the likely impact, and response from small centers, if ‘local first’ concept is largely made obsolete

82. OPTN Differences in Transplantation in the US Recommendations to the Board: Require a progress report from each committee in one year after central resource provides initial concepts Require a progress report from each committee in one year after central resource provides initial concepts After initial proposals are made involve the transplant community as a whole to develop broad based consensus After initial proposals are made involve the transplant community as a whole to develop broad based consensus Aim to convene a Consensus Conference within 2 years Aim to convene a Consensus Conference within 2 years

83. OPTN Ask not what your country can do for you, but what you can do for your country. John F. Kennedy

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85. OPTN High RR of Waitlisting Does Not by Itself Explain a Low RR of Deceased Donor Transplant (R 2 = 0.17; p < 0.0001) (R 2 = 0.17; p < 0.0001) Rate of Deceased Donor Transplant Rate of Waitlisting N=20 N=10 N=5 N=16

86. OPTN WLRR>1, TxRR >1 WLRR>1, TxRR<1 WLRR 1 WLRR<1, TxRR <1 HI Waitlisting Rate* (WLRR) and Deceased Donor Transplantation Rate (TxRR)* by State, 1996-2004 WAWA OR CO NM AZ UT MT CA AK NV ID WY NJ PA NY CT RI MA VT MI MN IA MO NE IN OH WI IL ND SD KS TX OK AR LA TN MSAL WV VA NC SC GA FL DC DE MD *WLRR is adjusted for age, gender, ESRD cause, incidence year, comorbid conditions, dialysis unit type, employment at incidence, insurance, and BMI and censored at death, living donor transplant, or end of study; TxRR is adjusted for age, gender, ESRD cause, incidence year, comorbid conditions, dialysis unit type, employment at incidence, insurance, and BMI and censored at removal from the waitlist or end of study. Compared to National Average of 1.0. KY ME NH Differences in Transplantation in the US

87. OPTN A tale of two cities, two patients and one liver….. Mr A and Mr B both have hepatitis C and are listed on the same day for a liver transplant. Mr A lives in Loonyville, California. His doctors tell him he is very sick and that over the next 3 months he only has a 50:50 living chance of getting a liver before he dies. Mr B lives 25 miles away from Mr B in Normaltown Oregon. His doctors tell him that even without a new liver he has a better than 80% chance of being alive in 3 months A donor liver becomes available in Mr B’s hometown 2 months after he is listed and he is transplanted. 2 weeks later Mr A, 25 miles away, dies. Can this happen in the land of equal opportunity??