Legislative Milestones

In the late 1960s, improvements in mechanical ventilation and other medical measures that sustained cardiopulmonary function enabled some patients who had experienced respiratory arrest or severe brain injuries to recover. Other patients, whose injuries were more extensive or who experienced a longer period of anoxia before restoration of circulation, could remain unconscious on a ventilator indefinitely. Postmortem examinations of patients in the latter group revealed brain damage inconsistent with their ever regaining consciousness or spontaneous circulatory-respiratory functions. Physicians developed methods of diagnosing when this loss of brain function was permanent and proposed that such patients could be declared dead even while a ventilator and associated medical interventions provided circulation of oxygenated blood (Report of the Ad Hoc Committee, 1968). In the period that followed, experts further elaborated the criteria and methods for determining death based on irreversible loss of brain functions, and the traditional methods of diagnosing death based on loss of circulation were clarified (DeVita et al., 1993; Guidelines for the determination of death, 1981; Halevy and Brody, 1993). Beginning in 1981, most states adopted the Uniform Determination of Death Act, which set forth two standards under which physicians were permitted to apply accepted medical criteria to determine that death had occurred, based either on the loss of circulatory and respiratory functions or on loss of all brain functions, including the brainstem (President's Commission, 1981). For both sets of criteria, the diagnosis of death requires both the cessation of function and irreversibility (Guidelines for the determination of death, 1981). The use of neurological criteria for the determination of death has gained wide medical, legal, ethical, and public acceptance in the United States, although debates continue (Bernat, 2005; Greer et al., 2020; Laureys, 2005). During the 1970s and 1980s, organ donation in the United States involved almost exclusively brain-based determinations of death. Beginning in the early 1990s, however, protocols were developed for donation after circulatory determination of death (DCDD), and, despite some controversy, this approach has become an increasingly important source of organs from deceased donors (Domínguez-Gil et al., 2021). In some areas of the country, the practice of DCDD never went away; however, the increases in DCDD started with the advent of a 2003 national Organ Donation Breakthrough Collaborative (see Chapters 6 and 7 for greater discussion of procurement of DCDD organs and national quality improvement efforts).

National Organ Transplant Act (NOTA)

In 1983, the growing demand for organ transplantation, controversies regarding the allocation of organs, and concerns about payment for organs prompted members of Congress to propose the creation of a formal, privately administered network to more effectively procure and equitably allocate deceased donor organs. This proposal became the National Organ Transplant Act (NOTA), which was adopted on October 19, 1984. NOTA authorized the creation of the OPTN to regularize and make more efficient and equitable the system for obtaining and distributing organs, based on an improved matching process (see Box 2-1).¹ NOTA specified that the OPTN would be operated by a private, nonprofit organization under federal contract. The Act also banned the purchase or sale of human organs for transplantation but allowed transplant professionals, hospitals, transporters, and organ procurement organizations (OPOs) to receive compensation for the services they provide (HHS, 2022). NOTA also permits living donors to receive reimbursement for the costs they bear in donating (such as travel, lost income, etc.), and it encourages the honoring of an individual's documented wishes with respect to organ donation.

BOX 2-1

FUNCTIONS OF THE ORGAN PROCUREMENT AND TRANSPLANTATION NETWORK (OPTN).

The Act also created the U.S. Task Force on Organ Transplantation to examine and report back to Congress on a broad range of issues including the technical, practical, and ethical limitations on sharing organs. In its April 1986 report, the Task Force concluded that “donated cadaveric organs are a national resource,” whose distribution ought not to be based on “accidents of geography.” This means that, to the extent technically feasible, every person in the nation in need of a transplant—not simply those who live in the area where an organ is donated—should be equally considered a potential recipient.² After the Task Force's report was submitted, the Secretary issued the OPTN contract to a nonprofit organization, the United Network for Organ Sharing (UNOS), based in Richmond, Virginia.

NOTA has been amended many times since 1984. In 1988 and again in 1990, the original scope of the OPTN's responsibilities—namely, to assist OPOs in distributing organs that “cannot be placed within [their] service areas”—was broadened, in line with the Task Force's conclusion, to assist OPOs “in the nationwide distribution of organs equitably among transplant patients.”³

The Omnibus Reconciliation Act of 1986 required all hospitals performing organ transplants to be members of the OPTN and to abide by its rules to receive Medicare and Medicaid payments.⁴ The Omnibus Health Amendments of 1988 required the OPTN to “establish membership criteria and medical criteria for allocating organs and provide to members of the public an opportunity to comment with respect to such criteria.”⁵

On September 8, 1994, the Health Resources and Services Administration (HRSA), a unit within the Department of Health and Human Services (HHS), issued a Notice of Proposed Rulemaking for a regulation governing the operation of the OPTN. Although the public comment period was supposed to end in December 1994, the department continued to accept comments on the OPTN's operations and its policies for allocating organs.⁶ Two years later, in November 1996, the department officially extended the period for public comment on the proposed rule, due to controversy over revisions in the liver allocation policies being proposed by the OPTN's board.⁷ Even after HRSA published the OPTN Final Rule on April 2, 1998,⁸ Congress twice delayed its going into effect.⁹ HRSA finally implemented the Final Rule on March 6, 2000.¹⁰ The OPTN board is composed of transplant physicians, recipients, candidates, family members, deceased donor families, recipient families, living donors, transplant hospitals, OPO representatives, and members of the public who are organized into various committees that are delegated with policy-making authority. In establishing the OPTN's regulatory framework, the rule instructs its board of directors to draft policies “based on sound medical judgment…to achieve the best use of donated organs”¹¹ and gives the board discretion to develop and implement these policies. But, in accordance with congressional enactments, the Final Rule also states that the Secretary of HHS must approve “significant” policies promulgated by the OPTN, such as the allocation rules for each type of organ, before they become federally enforceable.¹² OPOs, hospitals, and other entities that are members of the OPTN are expected to adhere voluntarily to the policies and bylaws adopted by the board to govern the OPTN's internal operations. These internal policies do not go through the process of Secretarial approval and are therefore not deemed to be federal rules.

Approach to Establishing Ambitious Goals

In all three of the cases outlined above, the goals established by HHS were extremely ambitious, but they were also grounded in the proven practices and outcome levels already achieved by the highest performers in the system. The quality improvement goal on donation was established using the levels of performance achieved by the top 5 to 10 percent of organizations (e.g., 5 percent of the 500 largest trauma centers). After 4 years of intensive quality improvement, nearly 80 percent (392) of the 500 largest trauma hospitals in the nation were recognized by HHS for achieving the 75 percent donation rate target.

The 2003–2004 goal that 10 percent of deceased donors originate with DCDD was achieved in a similar 4-year time frame, representing a more than double increase from the original 4 percent national rate in 2003. At that time, few OPOs were pursuing DCDD donors, and only 6 of the 59 had achieved a rate where 10 percent or more of their deceased donors came from DCDD.¹⁴ In both of these organ donation and transplant cases, and in the case of the national patient safety goal of a 40 percent reduction in preventable hospital harm within 4 years, HHS successfully used ambitious time-limited goals to substantially increase the overall performance of the system, based on the proven practices and the levels of achievement of the highest performers in the system.

When establishing ambitious goals of this nature, it is important to be mindful of potential unintended consequences. For example, in establishing a goal to increase the percentage of deceased donors coming from DCDD, it is often useful to incorporate balancing measures to ensure that the total numbers of donors coming from donation after neurological determination of death (DNDD) do not drop as a result of the ambitious DCDD goal. In recommending ambitious goals like those outlined in this report, it is the intention of the committee that the goals be pursued and obtained in ways that represent meaningful progress and true effects that benefit the patients we all serve, not as a result of gaming or manipulation of data (HRSA, 2018).

The goals recommended in this report are as ambitious as each of the prior examples detailed above. Achieving the recommended targets outlined in this report will require commitment, persistence, and focused attention to learning, testing, spread, and improvement of known best practices that are already being used by the highest performers in the system.

Conclusion 2-1: The current organ transplantation system is unduly fragmented and inefficient. The system's component parts—physicians caring for patients with organ failure, donor hospitals, organ procurement organizations, the OPTN, transplant centers, the SRTR, CMS and other payers, among others—do not operate as a fully integrated system. Likewise, the entities with oversight responsibilities each oversee particular components, but none monitors the performance of the system as a whole in producing predictable, consistent, and equitable results.

Conclusion 2-2: The organ transplantation system could save additional lives and be more equitable if its component parts functioned in a more cohesive fashion and were overseen by a single entity, or by several entities operating in a coordinated fashion with common goals and unified policies and processes. Such alignment of all components and oversight responsibilities would allow the public and Congress to ascertain whether the system is fairly and efficiently maximizing the benefits provided by organ donation and transplantation.

Conclusion 2-3: Since deceased donor organs are a national resource, the fairest way to allocate them to patients on the waiting list is on a national, continuous basis, in accordance with the OPTN Final Rule 2000 as most recently revised by HHS. The committee recognizes that some members of the transplant community feel strongly that deceased donor organs procured in a particular geographic area should be retained for allocation to wait-listed patients in that area.

Conclusion 2-4: By setting ambitious goals in its prior quality improvement initiatives in organ transplantation, HHS has achieved significant progress in increasing the number of successful transplants performed nationally.

Recommendation 1: Develop national performance goals for the U.S. organ transplantation system.

HHS should identify and substantially reduce or eliminate the existing variations among donor hospitals, OPOs, and transplant centers in the rates of organ donation, DCDD procurement and transplantation, acceptance of offered organs, and nonuse of donated organs, to improve the quality of, and foster greater equity in, organ donation and transplantation. HHS should also use the proven capabilities of the highest-performing donor hospitals, OPOs, and transplant centers to establish bold goals to drive national progress toward greater equity, higher rates of organ donation, procurement and transplantation of organs from donors after circulatory determination of death, and acceptance of offered organs, along with lower rates of nonuse of donated organs, to increase the total number of organs procured and transplants performed.

These goals can inform the development and use of various levers of influence including organized programs of quality improvement, payment policies, regulations, technical assistance, and public education campaigns. The goals should be continuously reviewed (at least annually) and updated as results are obtained, and as new, higher levels of organizational performance are achieved. HHS should

• Build on the initial CMS goals established in the kidney transplant collaborative, and establish a national goal for all transplant centers to reduce donated kidney nonuse rates to 5 percent or less.
• Establish new national goals to do the following:
  • Improve donation among minority populations and disadvantaged populations, and increase transplantation rates among minority and disadvantaged populations, based on the proven practices of donor hospitals, OPOs, and transplant centers which have the highest rates in these areas.
  • Increase the number of organs procured from medically complex donors. In particular, increase DCDD donors to at least 45 percent of all deceased donors, with no reductions in the numbers of organs procured from donors from neurological determination of death.
  • Improve offer acceptance levels for each organ type to those achieved by the 5 to 10 percent highest-performing transplant centers for that organ type nationally.
  • Increase the number of transplants to at least 50,000 by 2026.

Current OPTN Policy Development Process

The OPTN's current policy development process is lengthy and complex (see Figure 2-3).

FIGURE 2-3

The OPTN 10-step policy development process. SOURCE: OPTN policy development process explanatory document. https://optn.transplant.hrsa.gov/media/3115/optn-policy-development-process-explanatory-document.pdf (accessed November 18, 2021).

Advantages and Disadvantages of the OPTN Policy Development Approach

The OPTN's current policy development process has advantages and disadvantages. Its advantages include transparency, collaboration, and centralization. For instance, to ensure ample stakeholder involvement throughout the policy development process, multiple stakeholder individuals and organizations are encouraged to collaborate with the responsible OPTN committee(s) in the development of national transplant policies and medical criteria. During the public comment period feedback is solicited from those closely aligned within the organ transplantation system, and most importantly, the overall public who are outside the system. Following the close of the public comment period, the relevant OPTN committee addresses the issues raised by publishing written responses. Stakeholder and public engagement with the policy-making process is an advantage of the OPTN approach, providing important feedback and direction for policy-making and strengthening public trust in the process. A recent change was made to enhance the role of the Policy Oversight Committee, which is responsible for harmonizing and prioritizing policy-making work and timelines across OPTN committees. Additionally, centralized rulemaking enables OPTN policy makers to focus on a set of predetermined policy objectives.²

The disadvantages of the process relate to its length, complexity, and challenges in implementation. Because the OPTN's rulemaking process seeks engagement and input from a broad range of passionate stakeholders, the policy process requires multiple committee reviews to avoid possible unintended consequences across the OPTN. Although rare, additional public comment periods may also be necessary if substantial policy revisions are made as a result of public comment. As an operating committee of the OPTN, the Policy Oversight Committee advises the OPTN Board of Directors and Executive Committee in the development of strategic policy priorities, prioritization and coordination of policy and committee projects with broad implications for the OPTN, evaluation of policy and committee proposals prior to public comment, assessment of the impact of proposed policies, and confirmation that the OPTN committees justify proposals in compliance with policy development requirements. The Policy Oversight Committee has helped to avoid situations in which committee projects are placed on hold and started again, sometimes multiple times. The consensus-driven nature of the OPTN policy development process can create slowness and policy implementation challenges can further delay the process, such as when stakeholders are in disagreement about how best to allocate organs and categorize patient prioritization on the waiting list. Recently, this already lengthy process has been further exacerbated by litigation.¹⁷

The steps in the OPTN policy-making process are also complex and variable in the time allotted for public comment periods, as well as the overall time taken from committee project approval to OPTN board approval. For example, a federal study of the similarities and differences in the processes the OPTN used to change the liver and lung allocation policies revealed variations in public comment periods for informing the policy development process. The current liver allocation policy included a 25-day public comment period; the 2017 liver allocation policy included two separate 62-day and 64-day public comment periods; and the current lung allocation policy had a retroactive 61-day public comment period (GAO, 2020). Significant variability exists in terms of the average time it takes from committee project approval to final board approval. For example, as presented during the virtual public session of the OPTN's December 6, 2021, Board meeting,¹⁸ between 2009 and 2021 the longest period of policy development was 9.6 years for the revised kidney allocation system in 2013 (see Figure 2-4). A project on liver distribution redesign modeling in 2017 took 6.4 years from committee project approval to board approval. Since 2019, project timelines have shown a downward trend with the 2021 continuous distribution policy for lungs taking 2.6 years. Implementation time for each policy was not included in the presented analysis.

FIGURE 2-4

Average time from committee project approval to board approval. SOURCE: “OPTN Contract Requirement 3.3.2.: Policy Development Process Metrics – Metric 9: Average Time from Committee Project Approval to Board Approval.” OPTN Policy (more...)

There is limited information and analyses of the overall performance of the policy-making process in meeting the stated goals of the OPTN. Based on the evidence of variability in overall OPTN policy development timelines and challenges in maintaining stakeholder agreement on contentious issues in patient prioritization and organ allocation, the committee believes there is an opportunity for the OPTN to draw on previously untapped external organizations with expertise in managing complex stakeholder involvement through a transparent and collaborative process.

Conclusion 2-5: The OPTN policy-making process includes extensive committee reviews that aim to involve all stakeholders, but the nature of the reviews contributes to variability and a general slowness in policy development and implementation. Identifying and prioritizing strategic objectives through process changes in committee responsibilities and implementing continuous distribution as the uniform policy model have been effective in the last 2 years in decreasing the time for policy approval by the OPTN Board of Directors; however, opportunities for improvement in policy development and implementation timelines exist.

Conclusion 2-6: Organizations such as the National Quality Forum (NQF) are skilled in the development and use of timely, multistakeholder, consensus-based developmental processes, like the Measure Applications Partnership (MAP). The work of the NQF MAP is tied to, and guided by, specific regulatory and payment timelines of participating HHS agencies and their associated rulemaking processes. The experience and expertise of the NQF and/or other similar organizations can provide valuable insights to inform improvements in speed and agility, as well as stakeholder engagement and consensus of OPTN policy development work.

Recommendation 2: Improve the OPTN policy-making process.

HHS should hold the OPTN and HRSA accountable for developing a more expedient and responsive policy-making process including increasing racial, ethnic, professional, and gender diversity on the boards and committees responsible for developing OPTN policies. HHS should use the agreed on policy priorities established by the OPTN Policy Oversight Committee to establish contractual deadlines for completion of these policy-making priorities. HHS should consider requiring the OPTN to work with and receive support from an external organization, such as the NQF or the National Academy of Public Administration, with expertise in guiding federal programs through unique challenges in leadership and stakeholder collaboration. HHS should require the OPTN to consider the following elements of the policy-making process:

• Proven approaches by others, such as the NQF Measure Applications Partnership, for meeting aggressive timelines with intensive, consensus-based, multistakeholder policy development processes;
• Optimal board size and stakeholder balance;
• Continuous and concurrent versus sequential policy-making processes;
• Managing strategic priorities and ensuring priority items have sufficient momentum, institutional memory, and timelines;
• Alternative governance models; and
• Appropriate tools and processes for evaluating the effectiveness of the policy-making process.

Vascularized Composite Allotransplantation

Vascularized composite allotransplantation (VCA) is a form of solid organ transplantation involving the transplantation of multiple structures that may include skin, bone, muscles, blood vessels, nerves, and connective tissue as a functional unit in patients with major tissue loss or injury. A VCA may provide an alternative treatment option when reconstructive surgery is not feasible or effective. VCA transplants are performed to improve functional outcomes and quality of life and thus intend to be life enhancing, and are not considered lifesaving. For example, some recipients received a VCA organ after traumatic injury or limb loss through trauma (e.g., war-related exposure to explosive devices or traffic accidents).

In the United States, the first unilateral upper extremity transplant was performed in 1999. In the past decade, more than 90 individuals have received a VCA (American Society of Transplantation, 2021). HHS redefined VCAs as organs, as opposed to tissue, in 2014, which enabled the OPTN to assume oversight of policies pertaining to authorization for deceased donation, allocation, and distribution of VCA organs. CMS redefining VCA as an organ would allow increased alignment across performance metrics in the transplantation system. VCA transplants include upper extremity, face, uterus, penis, larynx, abdominal wall, and others. Most VCA organs are donated by deceased donors; however, uterus donors may also be living donors.

The field of VCA is growing. The surgical procedures themselves are generally no longer construed as experimental procedures, considering the mastery of microsurgical techniques and immunosuppressive approaches have placed reconstructive surgery at the highest rung in the surgical ladder (Cetrulo et al., 2017; Kaufman et al., 2019). However, the determination of whether VCA is a standard of care is a complex matter. Some countries consider certain forms of VCA as being a standard of care (Kaufman et al., 2019).

In the United States, all transplant programs have traditionally been required to obtain institutional review board (IRB) oversight to perform VCA. However, some VCA programs are no longer functioning under IRB oversight. Currently, VCA costs are covered by transplant programs. Insurance companies do not currently pay for VCA because they view VCA as not being the standard of care. A major challenge to becoming a standard of care is the small number of patients undergoing each type of VCA procedure. Without enough information on long-term functional outcomes among enough recipients to control for demographic and clinical variables, it is unclear whether the procedure is effective and worth the risks (ethically) and resources (financially). Insurance companies are awaiting long-term outcomes data before covering these procedures.

For reasons delineated, the broad scope of the study task and the limited time frame for this study, the committee did not conduct an in-depth analysis of VCA transplantation. The committee considered the implications that increased use of VCA could have on organ donation including some of the challenges that may arise as the public increasingly confronts difficult questions of whether they, as individuals, would approve donation of their organs (e.g., face, uterus, or penis) upon their death. Public opinion is generally supportive of VCA donations from deceased donors (Rodrigue et al., 2017); however, given the limitations of educational materials on VCA (Rasmussen et al., 2020), the transplant community will need to develop efforts to educate the public about how to make informed decisions about donating VCA organs and explain how VCA organs will be procured and used.

Key Use Statistics and Trends by Organ²⁰

The landscape of kidney transplantation in the United States is characterized by promising trends and ongoing challenges. The overall number of kidney transplants has continued to increase over the past 6 years, reaching 24,273 in 2019—more than 70 percent of which were deceased donor organ transplants. However, the number of available donor kidneys is far surpassed by the number of patients awaiting kidney transplant, and the extent of the shortage is far greater than other organs. From 2014 to 2019, only approximately 25 percent of patients on the waiting list received a deceased donor kidney transplant within 5 years, and this varies significantly based on donation service area—from 15.5 to 67.8 percent. In 2019, the nonuse rate for deceased donor kidneys was 20.1 percent overall (Israni et al., 2021). As will be described in Chapter 6, the probability of receiving a deceased donor kidney transplant within 3 years of waiting list placement varies across transplant centers in the United States, with 16-fold variance between centers. Waiting patients at transplant centers with low offer acceptance rates have only a 4 percent chance of getting a transplant within 3 years. Conversely, patients waiting at transplant centers with high offer acceptance rates have a 65 percent chance of getting a transplant within 3 years (King et al., 2020).

Of note, the rate of nonuse of hepatitis C virus (HCV)-positive kidneys has been declining since 2014 and by 2019, the rate was nearly equivalent to that of HCV-antibody-negative kidneys. In terms of resource use, the average length of hospital stay after transplant for kidney recipients was 7.4 days in 2019, a slight decline since 2008. The rate of reported hospital readmission during the first year posttransplant was 48.1 percent in 2018.

More than 1,000 pancreas-alone and kidney-pancreas transplants were performed in 2019—a number that remained relatively stable compared to 2018, as did the number of wait-listed patients awaiting transplant. The number of deceased organ pancreas transplants has increased marginally since the 2014 revision of the Pancreas/Kidney-Pancreas Allocation System. However, a concerning issue is the high rate of nonuse for deceased donor pancreata—25.4 percent in 2019—compared to other organs; this may be related to relatively short pancreas transplant wait times and the current state of the art for pancreas transplantation. For pancreas recipients, the average length of transplant hospitalization in 2019 was 11.5 days and the rate of hospital readmission during the first year posttransplant was almost 62 percent; both of these have declined in recent years.

Almost 8,900 liver transplants were performed in 2019, 94 percent of which were deceased donor transplants. HCV-positive donor organs have continued to increase since 2008, representing 9.7 percent of deceased donor livers in 2019. Both the number of new waiting list registrations and the number of transplants performed have been on the rise, while both the median waiting time for candidates with a Model for End-Stage Liver Disease (MELD) score of 15–34 and the number of transplants performed for patients with exception points decreased.²¹ The nonuse rate for deceased donor livers was 9.6 percent in 2019 compared to 8.6 percent in 2018, likely driven by an increase in nonused organs from donors aged 55 years or more. For liver recipients, the average length of transplant hospitalization in 2019 was 20.8 days and the rate of hospital readmission during the first year posttransplant was almost 60 percent among those who received a transplant in 2018; both of these have remained relatively stable since 2008.

Intestine transplants represent the smallest proportion of solid organ transplants performed in the United States.²² Intestine transplantation is a maturing field, and advances in intestine failure therapies have resulted in fewer patients being added to the waiting list for intestine transplant alone or for intestine transplant in combination with other organs. Just 81 deceased donor intestine transplants were performed in 2019. The nonuse rate for deceased donor intestines was relatively low, at about 6 percent. For intestine recipients, average length of transplant hospitalization in 2019 was about 61 days, which has varied substantially over the past decade. The rate of hospital readmission during the first year posttransplant was almost 96 percent among those who received a transplant in 2018—far higher than any other organ.

Around 3,600 heart transplants were performed in 2019—a slight increase over the previous year—but the number of candidates on the waiting list continued to increase.²³ The number of donor hearts increased by almost 64 percent between 2008 and 2019, when the number reached an all-time peak. The nonuse rate for deceased donor hearts was less than 1 percent, the lowest of all organs, although this rate has fluctuated over the past decade. For heart recipients, the average length of transplant hospitalization in 2019 was about 49 days, trending upward in recent years. The use of some preoperative devices or days of requiring extracorporeal membrane oxygenation as a bridge to transplantation could account for the lengthier hospital stay for some heart transplant recipients. Heart transplant patients without these interventions have much shorter hospital stays (e.g., less than 10 days). The rate of hospital readmission during the first year posttransplant was just under 40 percent, which was also the lowest rate of all organs.

The number of lung transplants performed each year in the United States is continuing to rise, increasing by 52.3 percent over the past decade and reaching an all-time peak of almost 2,800 transplants in 2019. This trend is likely attributable to increasing numbers of wait-listed candidates as well as the number of donors, which has increased by 62 percent in the last 10 years. The mortality rate for individuals on the lung waiting list decreased 14.6 percent in 2019, which is an important positive trend given the increasingly older and sicker lung candidate population. The nonuse rate for deceased donor lungs was more than 6 percent in 2019. For lung recipients, average length of transplant hospitalization in 2019 was around 34 days, which has generally increased in recent years. The rate of hospital readmission during the first year posttransplant was close to 52 percent.

From 2013 to 2017, 8,246 multiorgan transplants (MOTs) were performed in the United States, with 1,853 occurring in 2017 (OPTN, 2019). The most frequent type of MOT across this 4-year period were kidney–pancreas, kidney–liver, and kidney–heart, respectively. Additionally, the rate of MOT has been increasing over the last 2 decades (OPTN, 2019). MOTs, however, create issues of inequities, given that each combination of organs has its own allocation strategies and prioritization of those awaiting a MOT has not been standardized across the different organs. For example, some organ combinations require a candidate to join a single, combined, or multiple organ waiting lists. Confusion has arisen because of the varying levels of OPO discretion regarding their ability to “choose which MOT combinations get allocated if there are multiple MOT combinations possible from the same donor” (OPTN, 2019). Additionally, across the United States the current prioritization according to Policy 5.10: Allocation of Multi-Organ Combinations is that a MOT is typically prioritized above single organ transplant at the local level, which can also affect equity differently across the country depending on the volume of MOTs in the differing geographic areas.

Cost-Effectiveness of Transplantation by Organ

This section provides an overview of recent studies that have estimated the cost-effectiveness of kidney, liver, heart, and lung transplantation. It should be noted that even though the OPTN/SRTR's Annual Data Report releases data suitable for use in cost-effectiveness modeling of solid organ transplantation, up-to-date cost-effectiveness studies are not always available for all organs. Although cost-effectiveness analyses provide important information, they are only one element among others guiding health policy. There is debate about methodological and ethical shortcomings of the “quality-adjusted life year” (QALY), the most typically used measure of health gains in cost-effectiveness assessments of medical interventions (Rand and Kesselheim, 2021).

Cost-Effectiveness of Kidney Transplantation

Kidney transplantation is associated with a significant improvement in patient survival and quality of life across the spectrum of organ quality compared with maintenance dialysis (Axelrod et al., 2018; Massie et al., 2014; Whiting et al., 2000; Wolfe et al., 1999, 2009). Recent estimates (Axelrod et al., 2018) indicate that end-stage kidney disease (ESKD) patients who receive a living donor kidney transplantation (LDKT) or deceased donor kidney transplantation (DDKT) experience significantly longer expected survival than ESKD patients who receive dialysis therapy in terms of derived QALYs: 10 years with dialysis therapy, 4.03 QALYs; LDKT (human leukocyte antigen [HLA]-compatible, well-matched living donor [LD]), 6.34; LDKT (ABO-incompatible LD), 6.12; LDKT (HLA-incompatible LD), 5.47; DDKT (KDPI²⁴ < 85), 6.07; DDKT (Public Health Service [PHS] increased risk), 5.91; DDKT (KDPI > 85), 5.20. Kidney transplants are also the most cost-effective treatment for ESKD,²⁵ although the extent of the cost savings varies by quality of the donor organ.

Axelrod et al. (2018) estimate that from the payer's (i.e., Medicare's) perspective, low-to-moderate KDPI (≤ 85) DDKTs are cost saving over 10 years compared to dialysis ($49,017 vs. $72,476 per QALY), as are high-KDPI (> 85%) DDKT compared to dialysis ($63,531 vs. $72,476 per QALY). Despite the additional therapeutic procedures needed to facilitate complex transplants such as ABO-incompatible LDKT, they also represent cost savings ($59,564 per QALY) compared to remaining on dialysis. Compared to DDKT, HLA-compatible LDKT was more cost effective for compatible donors with up to three HLA mismatches ($39,939 per QALY) and for compatible donors with four to six HLA mismatches ($41,016 per QALY). Consistent patterns were found upon repeat analysis using a 20-year time horizon; all transplantation options resulted in cost-effectiveness ratios lower than the commonly cited willingness-to-pay threshold of $100,000 per QALY (Neumann et al., 2016).

Cost-Effectiveness of Liver Transplantation

Recent studies found liver transplantation to be cost effective (less than $100,000 per QALY). For instance, Dageforde et al. (2013)²⁶ studied the cost-effectiveness of liver transplantation with DNDD organs and DCDD organs by considering two waiting list strategies: (1) only DNDD organs and (2) both DNDD and DCDD organs (DNDD + DCDD). Over a 10-year horizon, the DNDD + DCDD waiting list strategy was more cost effective (5.6 QALYs; cost of $69,000/QALY) than the DNDD-alone strategy (6.0 QALYs; cost of $61,000/QALY) because of the decreased waiting list mortality and pretransplant morbidity associated with the former strategy.

In the United States, the current practice is for HCV-negative patients only to accept HCV-negative livers. Bethea et al. (2019) compared the cost-effectiveness of this current strategy versus a strategy of accepting any HCV-negative or HCV-positive livers, with recipients of HCV-positive livers receiving preemptive direct-acting antiviral therapy. For patients with a MELD score of 28,²⁷ it was cost effective for them to accept any liver. The incremental cost-effectiveness ratio of accepting any liver versus accepting only HCV-negative livers was $62,600/QALY. Incremental cost-effectiveness ratios of receiving any liver were less than $100,000 for patients with a MELD score of 22 or greater; the strategy was also cost effective for patients with low MELD scores that may not accurately reflect disease severity.

Sarasin et al. (2001) study found that in the United States, LDLT can be cost effective for patients with early hepatocellular carcinoma when waiting times for transplantation exceed 7 months.²⁸ A study using more recent data²⁹ found that over a 10-year period, both LDLT and deceased donor liver transplantation are cost effective compared to no transplant (i.e., medical management of cirrhosis) (Northup et al., 2009). Being on a waiting list with possible deceased donor liver transplant (DDLT) versus being on a waiting list with possible DDLT or LDLT were associated with 4.4 and 4.9 QALYs, respectively. The DDLT or LDLT strategy had an incremental cost-effectiveness ratio of $106,788 over DDLT only.

Cost-Effectiveness of Heart Transplantation

The 2013 OPTN/SRTR Economics Annual Data Report presented a cost-effectiveness analysis of heart transplantation—the most expensive of the major transplants—that compared transplant recipients who had received a ventricular assist device (VAD) procedure with those who had not (Schnitzler et al., 2015). The total expected discounted cost of care from 1 year prior to the transplant through 20 years posttransplant was lower for a patient with a VAD ($505,000) than patients without a VAD ($525,000). However, patients with a VAD gained fewer discounted QALYs (5.53) than patients without a VAD (6.28). Thus, the cost per QALY was greater for patients with a VAD ($91,000) than those without a VAD ($84,000), although both are within the conventional willingness-to-pay threshold (less than $100,000).

Cost-Effectiveness of Lung Transplantation

Schnitzler et al. (2014) used Medicare data linked to SRTR data to examine the effect of allocation based on the lung allocation score (LAS) on the cost-effectiveness of a lung transplant compared with medical management of end-stage pulmonary disease, finding that the relative cost-effectiveness of a lung transplant is correlated with LAS. Specifically, incremental cost-effectiveness ratios—measuring additional costs per QALY gained from a transplant—increased with LAS. Even at the highest LAS scores, the cost-effectiveness of lung transplants is within the conventionally cited limits.³⁰

Conclusion 2-7: Transplantation, independent of organ type, is a cost-effective intervention overall. Although SRTR has been releasing current data on pre- and posttransplant costs suitable for cost-effectiveness modeling, there is a need for up-to-date cost-effectiveness analyses for all organs to ensure that current data on effectiveness and costs are taken into account. There is debate about methodological and ethical shortcomings of cost-effectiveness analyses. In particular, cost-effectiveness may fail to capture features relevant to equity. Cost-effectiveness analyses are only one factor among others guiding health care policy choices.