Thoracic Organ Transplantation Committee Regional Review Board Guidance for Adult Congenital Heart Disease Exception Requests Thank you <Regional Councillor>, and thank you for the opportunity to present the Thoracic’s Committee’s proposal, “Regional Review Board Guidance for Adult Congenital Heart Disease Exception Requests,” currently out for public comment. Thoracic Organ Transplantation Committee

What problem will the proposal solve? Concern that adult congenital heart disease (ACHD) candidates will be disadvantaged under the new policy: Higher urgency statuses are device-driven Variability in review board decision-making for ACHD exception requests Challenging to objectively quantify severity of illness In December 2016, the OPTN Board of Directors approved the Thoracic Committee’s “Modification to the Adult Heart Allocation” proposal. During the development of that proposal, the Committee received feedback from the heart transplant community that revealed concerns that adult congenital heart disease candidates would be disadvantaged under the proposed policy, as there are often fewer inotropic and mechanical support options for these patients. The Committee considered the following concerns: Higher urgency statuses are device-driven For both anatomic and physiologic reasons, these candidates are less frequently helped by mechanical support and are higher risk when mechanical support is used than non-congenital heart disease candidates., Variability in review board decision-making for adult congenital heart disease exception requests The evaluation and award of exception requests for adult congenital heart disease candidates may vary from region to region because there is variable, limited, and inconsistent congenital heart disease expertise on regional review boards. Challenging to quantify severity of illness Because of limited data and challenges in reproducibly quantifying the severity of disease in a highly heterogeneous population, a variety of congenital heart disease candidates (likely with different mortality risks) have been grouped together within the new policy. The Committee acknowledged that some adult congenital heart disease candidates may have higher mortality and may not be candidates for the mechanical support options, but there was insufficient data to support changes in the proposed policy. While planned data collection should improve stratification of adult congenital heart disease candidates to higher tiers due to inconsistency of congenital heart disease expertise across review boards, reliance on the exception pathway may lead to greater geographic disparities.

What are the proposed solutions? Suggests objective criteria to define a pathway to the higher urgency statuses for ACHD Provides: Guidelines about statuses appropriate for specific conditions Rationale and context to justify the recommendations, potentially helping review boards without a CHD expert Specific, objective criteria the RRBs can use to evaluate exception requests, potentially increasing standardization of decision-making Should help RRBs recognize more medically urgent ACHD candidates requesting exceptions and can grant access to the higher urgency statuses. To help mitigate these challenges, the Committee agreed to draft guidance for the regional review boards with the goal of outlining objective criteria to standardize the evaluation and decision-making of adult congenital heart disease exception requests. This guidance suggests objective criteria to define a pathway to the higher urgency statuses for adult congenital heart disease candidates. Crafted largely in response to concerns from the transplant community, this guidance provides: Guidelines regarding which statuses would be appropriate for specific conditions Rationale and context to justify the recommendations, potentially helping review boards without a congenital heart disease expert Specific, objective criteria the RRBs can use in evaluating exception requests, potentially increasing standardization of decision-making If utilized, the RRBs should be able to recognize more medically urgent adult congenital heart disease candidates and can grant access to the higher urgency statuses.

What are the proposed solutions? Two broad categories: Single ventricle ACHD candidates Criteria suggests exception pathways for status 1, 2, 3 Dual ventricle ACHD candidates Criteria suggests exception pathway for status 3 Candidate must be admitted to the transplant hospital that registered the candidate to the waiting list. The Committee proposes recommendations that will establish exception pathways for statues 1, 2 and 3 for single ventricle adult congenital heart disease candidates. For dual ventricle adult congenital heart disease candidates, the Committee suggests criteria for a status 3 exception. Please review the proposal to evaluate the proposed criteria for each pathway, but for all exception requests, the candidate must be admitted to the transplant hospital that registered the candidate to the waiting list.

How will members implement this proposal? Heart programs should consider this guidance when submitting exception requests for their adult congenital heart disease candidates Guidelines are voluntary and not prescriptive of clinical practice No additional data collection required This proposal impacts heart transplant transplant programs. Programs should consider this guidance when submitting exception requests for their adult congenital heart disease candidates. However, these guidelines are for voluntary use by members and are not prescriptive of clinical practice or of regional review board decisions. This proposal does not require additional data collection.

How will the OPTN implement this proposal? Anticipated Board review date: Dec. 2017 Guidance posted on OPTN website once adult heart allocation system policy changes are implemented Members will not be evaluated for compliance based on guidance If public comment is favorable, the Committee plans to bring this guidance to the Board of Directors in December 2017. Upon Board approval, the OPTN Contractor will publish this guidance to the “Resources” section of both the OPTN and other websites concurrently with full implementation of the new adult heart allocation system. The OPTN Contractor will work with the Committee to develop a training specifically for regional review board representatives and alternates. The content of this guidance will be included as part of that training. This proposal will not require programming in UNet. Guidance from the OPTN does not carry the weight of policies or bylaws. Therefore, members will not be evaluated for compliance with the guidance in this document.

Questions? Kevin Chan, MD Committee Chair kevichan@med.umich.edu Kimberly Uccellini, MS, MPH Committee Liaison Kimberly.Uccellini@unos.org Thank you again for this opportunity to present this proposal. I am happy to answer any questions, or you can reach out to our Committee Chair, Kevin Chan, or Committee liaison, Kim Uccellini.

Backup slides

Supporting Evidence This proposal is informed primarily by clinical consensus, due to the lack of data supporting elevating this diverse patient population to higher urgency statuses as well as the lack of data regarding specific clinical, hemodynamic, or laboratory data that might assist with identifying a higher risk population. The RRBs operate based on medical judgment and clinical consensus, so guidance developed via clinical consensus for a body whose decisions are made by clinical consensus is reasonable. When relevant, OPTN descriptive analyses and TSAM results referenced in the modifications to the adult heart allocation system proposal were considered, as well as current peer-reviewed literature. In addition, the Subcommittee reviewed relevant feedback pertaining to this patient population from both public comment cycles. This figure shows waitlist mortality rates by simulation and by status. Rates in status 1 differed substantially from rates in statuses 2-7, so the figure was separated into two panels with different y-axis limits. Status 4 is not limited to CHD candidates; it includes candidates with: Dischargeable Left Ventricular Assist Device (LVAD) without Discretionary 30 Days Inotropes without hemodynamic monitoring Ischemic Heart Disease with Intractable Angina Amyloidosis, or Hypertrophic or Restrictive Cardiomyopathy Re-transplant   The TSAM results showed for candidates in statuses 3-7, waitlist mortality rates were similar under current rules and under allocation by statuses. Specific CHD diagnoses were not stratified in the TSAM cohort analyzed Scientific Registry of Transplant Recipients. HR2015_01, 2015.