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The ethics of risk and innovation
http://www.jhltonline.org
EDITORIAL
The ethics of risk and innovation Heather J. Ross, MD From the Toronto General Hospital, Cardiology, 200 Elizabeth Street, NCSB 11C-1203, Toronto, ON, Canada M5G 2C4
In the current issue of JHLT we revise the 2006 indications for heart transplant and add an in-depth review of a growing number of other possible indications for heart transplant. Historically the field of transplantation has been characterized by innovation, setbacks and challenges, scientific investigation and discovery, understanding, refinement, improvement and ultimately wide scale acceptance and uptake. This cycle was seen in the pioneering days of heart, lung, and heart-lung transplantation and mechanical circulatory support. Victims of our own successes, we find ourselves faced with new or expanding indications for potential high risk transplant candidates, patients in need. What has changed is that this new challenge occurs in the context of an absolute scarcity of donor hearts and established expectations of transplant outcomes. Despite worldwide campaigns in donor awareness and promotion, there remains a fixed or dwindling1 resource of donor hearts as compared to the growing recipient pool, resulting in a significant mismatch between donor availability and recipient need. Transplanting one individual necessarily disadvantages another who remains wait-listed, facing disease progression and high wait list mortality. Some of the waitlist risk has been abrogated through the use of mechanical circulatory support (MCS). However not all listed patients are candidates for MCS. Transplant programs must ensure equity and transparency in the assessment of transplant candidacy, listing status priority and donor heart allocation process. They have a fiduciary duty to their patients, a duty to the listed patients and a duty to the donor to be good stewards of the scarce donor resource. As Dr. Norheim stated, the clinician’s concern for expected outcomes is grounded in the goal of maximising health through efficient allocation of resources2. In an era of balanced score cards and transparency, the majority of transplant programs are routinely monitored by governing bodies to ensure they maintain benchmark results3. In the US, the OPTN Membership and Standards Committee reviews program specific reports (PSR)4,5, looking at observed versus expected outcomes and 1 year patient and graft survival6. Although the PSR is adjusted for donor and recipient risk, not all risk factors are taken into account, for example desensitization for those patients with a
high PRA6,7. Furthermore, in the United States, CMS oversees those programs that receive Medicare funding6. Historically programs with substandard results have been put on probation or closed if their results fall below a certain threshold, potentially closing sites that offer high level care. Private insurers use the data published by SRTR to determine which centres will perform transplants on their insured patients6,7. This watchdog climate may make programs risk averse. A recent survey of US heart transplant programs found that 8 of 137 heart transplant programs did not meet CMS 1-year patient survival criteria7. It is unclear whether this was due to true deficiencies in their transplant programs or inadequate risk case adjustment7. In a recent informal survey, 70-80% felt their program tolerates less risk as a result of the program specific reports6. From the transplant program viewpoint, a major concern is that transplanting high risk patients with potentially inferior outcomes may result in bad press, censure, probation or closure of the program. A recent renal transplant study found that centres with a higher risk cohort of patients were more likely to be identified as poor performers8. This may lead to penalizing those centers willing to take on risk, ultimately condemning innovation. Historically, as a community we have been able to extend criteria for transplantation through the willingness of centres to push boundaries, through innovation and experience, while maintaining excellent outcomes (for example the evolution of the age cut-off, diabetes, etc). However, currently certain subgroups of patients (e.g. amyloid cardiomyopathy, Fontan), have outcomes that are inferior to the historic low-risk transplant candidate, a patient with dilated cardiomyopathy9,10. These competing forces result in tension between equity and access to heart transplantation, required outcomes for program specific thresholds versus achievable outcomes, and finally the risks for the patient (accepted or denied transplant) and the risk threshold that programs potentially in fear of censure will accept. How then do we balance the pioneering nature of transplantation, the need to advance the field and understand who is best served with transplant, the increasing needs and numbers of potential ‘high-risk’ recipients, our duty to distribute the scarce resource to those most likely to achieve
1053-2498/$ - see front matter r 2015 International Society for Heart and Lung Transplantation. All rights reserved. http://dx.doi.org/10.1016/j.healun.2015.05.009
2
The Journal of Heart and Lung Transplantation, Vol ], No ], Month ]]]]
excellent short and long term benefit, all the while maintaining ‘industry’ standard outcomes? There is no easy answer. Hence, an important justification for the decisions that are made is that they have been arrived at as a result of due process11. Accountability for reasonableness (A4R) operationalizes the abstract concept of fairness12,13. It is a normative framework that is often used in priority setting decision making12,14. A4R relies on 4 guiding principles (relevance, publicity, appeals, and enforcement)12,14. 1. Relevance ensures that decisions are based on the most up to date evidence and principles. Further, given the same information that fair minded people in similar circumstances would find the principles to be relevant and come to the same decision. Nationally and internationally this corresponds to individuals with specific content expertise developing guidelines for eligibility criteria based on all available relevant data, including published outcomes. At a local program level multidisciplinary review of potential candidates further ensures that each individual transplant candidate is given their due. 2. Publicity ensures that the process used to determine the decision is both transparent and publicly available. We achieve this through presentation and publication of specific guidelines, including sharing the mechanism/ process by which the recommendations came about. Furthermore, programs must clearly state their specific criteria for candidacy and make them freely available to any interested party. 3. The appeals process allows an opportunity for appeal or revision of the decisions. This is often more difficult to achieve in transplantation. Dispute resolution is complex. Although patients and advocacy groups can support and push to have an individual listed for transplant, the local program is not required to comply if they feel the risk is prohibitive. Appeals are therefore often achieved in transplantation through freely offering a second opinion at an alternate centre. At the macro level revisiting guidelines on a timely basis provides the opportunity to ensure criteria are revisited and revised in light of new information. 4. The final A4R principal is a mechanism to ensure that these fair processes are implemented (enforcement). This resides in the purview of the local organ procurement organization, the governing body (e.g. UNOS) and detailed program specific reports. These mechanisms ensure that each program is using appropriate national and international criteria for patient listing, donor allocation, whilst maintaining expected outcomes12. Each transplant program needs to ensure that governing and censoring bodies are using appropriate case risk weighting7 that accurately represents the patient undergoing transplant. As a community we are obligated to promote further research to better understand who will and will not benefit from transplant. Once known, these results need to be widely disseminated. As information comes available we need to share with the broader community and re-assess, revise and renew criteria for transplant candidacy as indicated. For those disease specific conditions deemed at significantly higher risk,
we should consider regional centres of excellence, ensuring adequate disease specific case volumes and concentrating the expertise needed throughout the continuum of care for that condition15. As the ASTS recommended, in order to protect ‘innovation’, patients being transplanted under a research protocol should not be considered within that institutions outcome cohort6,7. A constantly iterative process of review of listing criteria, donor allocation, and recipient outcomes; and revision as necessary in the face of new information and publicly sharing this information should enhance the transparency, fairness and equity of heart transplantation16.
Disclosure Statement The author of this manuscript has no conflicts of interest or relevant disclosures.
References 1. The registry of the international society for heart and lung transplantation: thirtieth annual report. Addison, Texas: International Society for Heart and Lung Transplantation; 2014. http//www.ishlt.org/registries/ slides.asp?slides=heartLungRegistry). Accessed April 25, 2015. 2. Norheim OF. Clinical priority setting. BMJ 2008;337:a1846. 3. Burch M. Is heart transplantation for adult congenital heart disease an appropriate use of a scarce resource? Heart 2010;96(15):1172-3. 4. Dickinson DM, Arrington CJ, Fant G, Levine GN, Schaubel DE, Pruett TL, et al. SRTR program-specific reports on outcomes: a guide for the new reader. Am J Transplant. 2008;8(4 Pt 2):1012-26. 5. VanWagner LB, Skaro AI. Program-specific reports: implications and impact on program behavior. Curr Op Organ Transplant 2013;18(2): 210-5. 6. Kasiske BL, McBride MA, Cornell DL, Gaston RS, Henry ML, Irwin FD, et al. Report of a consensus conference on transplant program quality and surveillance. Am J Transplant. 2012;12 (8):1988-96. 7. Abecassis MM, Burke R, Klintmalm GB, Matas AJ, Merion RM, Millman D, et al. American Society of Transplant Surgeons transplant center outcomes requirements–a threat to innovation. Am J Transplant. 2009;9(6):1279-86. 8. Schold JD, Srinivas TR, Howard RJ, Jamieson IR, Meier-Kriesche HU. The association of candidate mortality rates with kidney transplant outcomes and center performance evaluations. Transplantation 2008;85(1):1-6. 9. DePasquale EC, Nasir K, Jacoby DL. Outcomes of adults with restrictive cardiomyopathy after heart transplantation. J Heart Lung Transplant. 2012;31(12):1269-75. 10. Lamour JM, Kanter KR, Naftel DC, Chrisant MR, Morrow WR, Clemson BS, et al. The effect of age, diagnosis, and previous surgery in children and adults undergoing heart transplantation for congenital heart disease. J Am Coll Cardiol. 2009;54(2):160-5. 11. Ham C. Priority setting in the NHS: reports from six districts. BMJ 1993;307(6901):435-8. 12. Daniels N, Sabin JE. Setting Limits Fairly: Can We Learn to Share Medical Resources? New York: Oxford University Press; 2002. 13. Daniels N, Sabin JE. Accountability for reasonableness: An update BMJ 2008;337:a1850. 14. Danjoux NM, Martin DK, Lehoux PN, Harnish JL, Shaul RZ, Bernstein M, et al. Adoption of an innovation to repair aortic aneurysms at a Canadian hospital: A qualitative case study and evaluation. BMC. Health Services Research. 2007;7:182. 15. Arnaoutakis GJ, George TJ, Allen JG, Russell SD, Shah AS, Conte JV, et al. Institutional volume and the effect of recipient risk on short-term mortality after orthotopic heart transplant. J Thor Cardio Surg 2012; 143(1):157-67: 67 e1. 16. Ladin K, Hanto DW. Rationing lung transplants–procedural fairness in allocation and appeals. New Engl J Med 2013;369(7):599-601.
EDITORIAL
The ethics of risk and innovation Heather J. Ross, MD From the Toronto General Hospital, Cardiology, 200 Elizabeth Street, NCSB 11C-1203, Toronto, ON, Canada M5G 2C4
In the current issue of JHLT we revise the 2006 indications for heart transplant and add an in-depth review of a growing number of other possible indications for heart transplant. Historically the field of transplantation has been characterized by innovation, setbacks and challenges, scientific investigation and discovery, understanding, refinement, improvement and ultimately wide scale acceptance and uptake. This cycle was seen in the pioneering days of heart, lung, and heart-lung transplantation and mechanical circulatory support. Victims of our own successes, we find ourselves faced with new or expanding indications for potential high risk transplant candidates, patients in need. What has changed is that this new challenge occurs in the context of an absolute scarcity of donor hearts and established expectations of transplant outcomes. Despite worldwide campaigns in donor awareness and promotion, there remains a fixed or dwindling1 resource of donor hearts as compared to the growing recipient pool, resulting in a significant mismatch between donor availability and recipient need. Transplanting one individual necessarily disadvantages another who remains wait-listed, facing disease progression and high wait list mortality. Some of the waitlist risk has been abrogated through the use of mechanical circulatory support (MCS). However not all listed patients are candidates for MCS. Transplant programs must ensure equity and transparency in the assessment of transplant candidacy, listing status priority and donor heart allocation process. They have a fiduciary duty to their patients, a duty to the listed patients and a duty to the donor to be good stewards of the scarce donor resource. As Dr. Norheim stated, the clinician’s concern for expected outcomes is grounded in the goal of maximising health through efficient allocation of resources2. In an era of balanced score cards and transparency, the majority of transplant programs are routinely monitored by governing bodies to ensure they maintain benchmark results3. In the US, the OPTN Membership and Standards Committee reviews program specific reports (PSR)4,5, looking at observed versus expected outcomes and 1 year patient and graft survival6. Although the PSR is adjusted for donor and recipient risk, not all risk factors are taken into account, for example desensitization for those patients with a
high PRA6,7. Furthermore, in the United States, CMS oversees those programs that receive Medicare funding6. Historically programs with substandard results have been put on probation or closed if their results fall below a certain threshold, potentially closing sites that offer high level care. Private insurers use the data published by SRTR to determine which centres will perform transplants on their insured patients6,7. This watchdog climate may make programs risk averse. A recent survey of US heart transplant programs found that 8 of 137 heart transplant programs did not meet CMS 1-year patient survival criteria7. It is unclear whether this was due to true deficiencies in their transplant programs or inadequate risk case adjustment7. In a recent informal survey, 70-80% felt their program tolerates less risk as a result of the program specific reports6. From the transplant program viewpoint, a major concern is that transplanting high risk patients with potentially inferior outcomes may result in bad press, censure, probation or closure of the program. A recent renal transplant study found that centres with a higher risk cohort of patients were more likely to be identified as poor performers8. This may lead to penalizing those centers willing to take on risk, ultimately condemning innovation. Historically, as a community we have been able to extend criteria for transplantation through the willingness of centres to push boundaries, through innovation and experience, while maintaining excellent outcomes (for example the evolution of the age cut-off, diabetes, etc). However, currently certain subgroups of patients (e.g. amyloid cardiomyopathy, Fontan), have outcomes that are inferior to the historic low-risk transplant candidate, a patient with dilated cardiomyopathy9,10. These competing forces result in tension between equity and access to heart transplantation, required outcomes for program specific thresholds versus achievable outcomes, and finally the risks for the patient (accepted or denied transplant) and the risk threshold that programs potentially in fear of censure will accept. How then do we balance the pioneering nature of transplantation, the need to advance the field and understand who is best served with transplant, the increasing needs and numbers of potential ‘high-risk’ recipients, our duty to distribute the scarce resource to those most likely to achieve
1053-2498/$ - see front matter r 2015 International Society for Heart and Lung Transplantation. All rights reserved. http://dx.doi.org/10.1016/j.healun.2015.05.009
2
The Journal of Heart and Lung Transplantation, Vol ], No ], Month ]]]]
excellent short and long term benefit, all the while maintaining ‘industry’ standard outcomes? There is no easy answer. Hence, an important justification for the decisions that are made is that they have been arrived at as a result of due process11. Accountability for reasonableness (A4R) operationalizes the abstract concept of fairness12,13. It is a normative framework that is often used in priority setting decision making12,14. A4R relies on 4 guiding principles (relevance, publicity, appeals, and enforcement)12,14. 1. Relevance ensures that decisions are based on the most up to date evidence and principles. Further, given the same information that fair minded people in similar circumstances would find the principles to be relevant and come to the same decision. Nationally and internationally this corresponds to individuals with specific content expertise developing guidelines for eligibility criteria based on all available relevant data, including published outcomes. At a local program level multidisciplinary review of potential candidates further ensures that each individual transplant candidate is given their due. 2. Publicity ensures that the process used to determine the decision is both transparent and publicly available. We achieve this through presentation and publication of specific guidelines, including sharing the mechanism/ process by which the recommendations came about. Furthermore, programs must clearly state their specific criteria for candidacy and make them freely available to any interested party. 3. The appeals process allows an opportunity for appeal or revision of the decisions. This is often more difficult to achieve in transplantation. Dispute resolution is complex. Although patients and advocacy groups can support and push to have an individual listed for transplant, the local program is not required to comply if they feel the risk is prohibitive. Appeals are therefore often achieved in transplantation through freely offering a second opinion at an alternate centre. At the macro level revisiting guidelines on a timely basis provides the opportunity to ensure criteria are revisited and revised in light of new information. 4. The final A4R principal is a mechanism to ensure that these fair processes are implemented (enforcement). This resides in the purview of the local organ procurement organization, the governing body (e.g. UNOS) and detailed program specific reports. These mechanisms ensure that each program is using appropriate national and international criteria for patient listing, donor allocation, whilst maintaining expected outcomes12. Each transplant program needs to ensure that governing and censoring bodies are using appropriate case risk weighting7 that accurately represents the patient undergoing transplant. As a community we are obligated to promote further research to better understand who will and will not benefit from transplant. Once known, these results need to be widely disseminated. As information comes available we need to share with the broader community and re-assess, revise and renew criteria for transplant candidacy as indicated. For those disease specific conditions deemed at significantly higher risk,
we should consider regional centres of excellence, ensuring adequate disease specific case volumes and concentrating the expertise needed throughout the continuum of care for that condition15. As the ASTS recommended, in order to protect ‘innovation’, patients being transplanted under a research protocol should not be considered within that institutions outcome cohort6,7. A constantly iterative process of review of listing criteria, donor allocation, and recipient outcomes; and revision as necessary in the face of new information and publicly sharing this information should enhance the transparency, fairness and equity of heart transplantation16.
Disclosure Statement The author of this manuscript has no conflicts of interest or relevant disclosures.
References 1. The registry of the international society for heart and lung transplantation: thirtieth annual report. Addison, Texas: International Society for Heart and Lung Transplantation; 2014. http//www.ishlt.org/registries/ slides.asp?slides=heartLungRegistry). Accessed April 25, 2015. 2. Norheim OF. Clinical priority setting. BMJ 2008;337:a1846. 3. Burch M. Is heart transplantation for adult congenital heart disease an appropriate use of a scarce resource? Heart 2010;96(15):1172-3. 4. Dickinson DM, Arrington CJ, Fant G, Levine GN, Schaubel DE, Pruett TL, et al. SRTR program-specific reports on outcomes: a guide for the new reader. Am J Transplant. 2008;8(4 Pt 2):1012-26. 5. VanWagner LB, Skaro AI. Program-specific reports: implications and impact on program behavior. Curr Op Organ Transplant 2013;18(2): 210-5. 6. Kasiske BL, McBride MA, Cornell DL, Gaston RS, Henry ML, Irwin FD, et al. Report of a consensus conference on transplant program quality and surveillance. Am J Transplant. 2012;12 (8):1988-96. 7. Abecassis MM, Burke R, Klintmalm GB, Matas AJ, Merion RM, Millman D, et al. American Society of Transplant Surgeons transplant center outcomes requirements–a threat to innovation. Am J Transplant. 2009;9(6):1279-86. 8. Schold JD, Srinivas TR, Howard RJ, Jamieson IR, Meier-Kriesche HU. The association of candidate mortality rates with kidney transplant outcomes and center performance evaluations. Transplantation 2008;85(1):1-6. 9. DePasquale EC, Nasir K, Jacoby DL. Outcomes of adults with restrictive cardiomyopathy after heart transplantation. J Heart Lung Transplant. 2012;31(12):1269-75. 10. Lamour JM, Kanter KR, Naftel DC, Chrisant MR, Morrow WR, Clemson BS, et al. The effect of age, diagnosis, and previous surgery in children and adults undergoing heart transplantation for congenital heart disease. J Am Coll Cardiol. 2009;54(2):160-5. 11. Ham C. Priority setting in the NHS: reports from six districts. BMJ 1993;307(6901):435-8. 12. Daniels N, Sabin JE. Setting Limits Fairly: Can We Learn to Share Medical Resources? New York: Oxford University Press; 2002. 13. Daniels N, Sabin JE. Accountability for reasonableness: An update BMJ 2008;337:a1850. 14. Danjoux NM, Martin DK, Lehoux PN, Harnish JL, Shaul RZ, Bernstein M, et al. Adoption of an innovation to repair aortic aneurysms at a Canadian hospital: A qualitative case study and evaluation. BMC. Health Services Research. 2007;7:182. 15. Arnaoutakis GJ, George TJ, Allen JG, Russell SD, Shah AS, Conte JV, et al. Institutional volume and the effect of recipient risk on short-term mortality after orthotopic heart transplant. J Thor Cardio Surg 2012; 143(1):157-67: 67 e1. 16. Ladin K, Hanto DW. Rationing lung transplants–procedural fairness in allocation and appeals. New Engl J Med 2013;369(7):599-601.