Legislative and Regulatory Changes Affecting the US Transplant System

Organ transplantation improves survival and quality of life for individuals with end-organ damage, driven in large part by significant strides in our understanding of the underlying immunology, a slowly expanding armamentarium of therapeutic choices, along with improvements in racial equity for waitlisted patients after implementation of Kidney Allocation System (KAS) 2014, not to mention the excitement around emerging innovations such as immune tolerance and xenotransplantation. These strides have occurred in parallel with a recent, rapid uptick in the number of solid organ transplants being performed driven, in part, by a new regulatory framework for organ procurement organizations (OPOs) that has been credited with creating significantly more accountability in the system.

Underneath this surface of innovation and growth, however, significant headwinds facing the system have limited the patient-level benefits of these advances. The system has focused attention on the fortunate few patients who ultimately received a transplant; maximizing outcomes for these individuals was the primary—and perhaps the only—definition of success for a long time. As a result, it should come as little surprise that the growth in transplantation has occurred in parallel with increasing allocation inefficiency,¹ failure to improve access to the waitlist,² considerable variation in access to transplantation even after reaching the waitlist,³ and even a steady decrease in the proportion of individuals with kidney failure being waitlisted for a kidney transplant over the past decade. Organ utilization rates have steadily declined, driven, in part, by organ quality labels that are suboptimally designed and inefficient allocation systems coupled with a general lack of transparency in the organ offer process.^4,5 Among organs that are transplanted, only a small minority are allocated to the recipient at the top of the allocation sequence for that organ—and this is likely getting worse with more organs being allocated through work-arounds, such as list diving and out-of-sequence placement, which undermines the objective and equitable nature of the allocation system.^6–8 Equally concerning is that efforts to mitigate racial and socioeconomic disparities in access to the kidney transplant waitlist do not appear to have moved the needle in a meaningful manner in the past two decades.²

For a system as complex as the national transplantation system that oversees the allocation of deceased donor organs, the desire to tackle these challenges with large-scale process overhauls must understandably be tempered with caution. Large system changes can result in significant unintentional consequences, such as decreasing allocation efficiency and rising organ discard rates (e.g., KAS250). However, caution can also create the perception of a preference for the status quo, and this is precisely the challenge faced by a system that is seen to advance allocation strategy or policy slowly, failing to adequately recognize, measure, or address several disparities or, for that matter, assess whether it was meeting the needs of patients with end-organ failure who would benefit from a transplant rather just those fortunate enough to reach the waitlist.⁹

Challenges to the status quo have resulted in a broad recognition that the system needs more innovation, growth, equity, and engagement to serve patients better. One potential approach to introduce greater innovation and more rapid iterative changes in the system is to inject a sense of urgency and competition in the system. The current contract for the Organ Procurement and Transplantation Network (OPTN) has been held by a single contractor, the United Network for Organ Sharing, since its inception, and the National Organ Transplantation Act required the organization to hold the contract to manage the OPTN to have prior experience in the role, essentially eliminating any other organization from competing for this role. While this has provided invaluable stability, continuity, and institutional memory, it has also been posited to have adversely affected innovation and improvement.

These concerns drove the bipartisan passage of the Securing the US OPTN Act in September 2023, which aimed to promote system improvements by “authoriz[ing] Health Resources and Services Administration (HRSA) to award multiple grants, contracts, or cooperative agreements to support the operation of the network and eliminat[ing] a cap on the amount of funding available for supporting the network.”¹⁰ Passage of this legislation was necessary to facilitate the changes envisioned by HRSA that constitute the modernization of the OPTN, including the introduction of multiple contractors for different aspects of the transplant system, such as technology infrastructure, governance, operations, research (innovation), and quality improvement. After the recent appropriation of $59 million by Congress, HRSA completed the first step in the changes envisioned in the modernization effort with the creation of an OPTN board that is independent of the contractor board at the end of March and is poised to release opportunities for multiple contractors to bid for various parts of the transplantation infrastructure.

These legislative changes have occurred in parallel to a federal collaborative effort by the Centers for Medicare & Medicaid Services (CMS) and HRSA called the Organ Transplantation Affinity Group (OTAG), which has established five goals that address the twin aims of improving the performance of and equity in the US organ transplantation system (Table 1).¹¹ Several strategies identified by OTAG are under consideration to supplement the OPTN modernization initiative that is being spearheaded by HRSA. For example, a new value-based payment model from CMS titled Increasing Organ Transplant Access that is “focused on accountability for quality and cost, access, and health equity” has been proposed by the CMS Innovation Center (https://www.reginfo.gov/public/do/eAgendaViewRule?pubId=202310&RIN=0938-AU51). A prior care model—the ESRD Treatment Choices—is also attempting to increase home dialysis modality and transplantation rates in the United States in the first-ever attempt in a randomized controlled trial for a policy intervention at that scale with limited success (https://www.cms.gov/priorities/innovation/data-and-reports/2024/etc-2nd-eval-rpt). This model is accompanied by the ESRD Treatment Choices Learning Collaborative, which is focused on facilitating a dramatic increase in the number of kidney transplants being performed, improved utilization of less-than-ideal kidneys, and a reduction in the number of kidneys discarded.

Table 1.

Organ Transplantation Affinity Group's proposed plan of action¹¹

Aims	Goals	Strategies
Improve transplant system performance	• Reduce variation of pretransplant and referral practices• Increase availability and use of donated organs• Increase accountability for organ procurement and matching	• Improve transparency of pretransplant and referral practices• Establish criteria for standardization and transparency of weightless practices• Remove barriers to the use of donated organs, including living and medically complex organs• Ensure organ acquisition payment policies promote equity and support organ procurement• Identify performance measures and incentives to drive accountability for systems change• Support US organ transplant system operations, including embedding CQI efforts across the system
Improve transplant system equity	• Promote equitable access to transplants• Empower patients, families, and caregivers to actively engage in the transplant journey	• Ensure OPTN policy-making processes and policies create equitable outcomes across populations• Improve transparency of referral and weightless processes for patients, families, and caregivers• Enhance patient, family and caregiver education and engagement

CQI, continuous quality improvement; OPTN, Organ Procurement and Transplantation Network.

More recently, the OPTN Data Advisory Committee has provided, on HRSA's request, a proposal for the collection of new prewaitlisting data elements that would allow the OPTN to identify individuals being referred for transplantation and initiate evaluation for a transplant and the outcomes of the selection committee review at the end of the evaluation process. These data elements would help provide a better understanding of the denominator of patients potentially in need of an organ transplant in addition to providing, for the first time, national data on the hurdles in access to transplant and the associated disparities that exist in the steps leading up to the waitlist. More granular data from OPOs on the referral of potential deceased donors are being sought in an attempt to better understand OPO processes, performance, and the sources of existing variation. These new data collection efforts at the national level are consistent with the strategies outlined by the OTAG in their effort to improve the transplant system. These data have the potential to create unprecedented transparency in the transplant system by creating the opportunity for a better understanding of the current challenges and limitations in the steps to transplant and to identify practice variations and opportunities for improvement in an effort to achieve the eventual goal of improved, equitable access to transplantation for those who would benefit from an organ transplant.

In summary, the US organ transplant system, although the largest in the world, faces several challenges today but has been given an unprecedented opportunity for improvement and modernization by legislators and regulators. Increased competition and multiple contractors for different elements of the OPTN will mean greater coordination and oversight needed to realize the potential of the HRSA modernization effort (https://www.hrsa.gov/optn-modernization). Improved and broader data collection will provide a better understanding that would ideally inform both clinical practices and public policy, including allocation algorithms, as we move forward. Similarly, alternative payment models that recognize that the quality of life and better survival that accrue to patients with a solid organ transplant can only happen with the efforts of a large multidisciplinary team responsible for coordinating care along a complex continuum and are equally critical at this juncture. It is imperative for all stakeholders in the transplant community to engage with the modernization effort for the benefit of all patients who could benefit from a transplant and not merely the fortunate few who are lucky enough to reach the waitlist or receive a transplant. This means encouraging greater transparency of the system with improved equity and utility using an objective, efficient organ allocation system that minimizes work-arounds like list diving and out of sequence placements while continuing to advocate for the resources necessary to help overcome the current challenges to improved access to care.

Supplementary Material

jasn-35-1278-s001.pdf ^{(1.3MB, pdf)}

Disclosures

Disclosure forms, as provided by each author, are available with the online version of the article at http://links.lww.com/JSN/E721.

Funding

S. Mohan: Kidney Transplant Collaborative. S.A. Husain: Kidney Transplant Collaborative and NIDDK (K23 DK133729).

Author Contributions

Conceptualization: Sumit Mohan.

Writing – original draft: Sumit Mohan.

Writing – review & editing: Syed Ali Husain, Sumit Mohan.