ADVISORY COMMITTEE ON ORGAN TRANSPLANTATION (ACOT)
U.S. Department of Health and Human Services (HHS)
Conference Call * March 7, 2013
Welcome — Patricia Stroup, Executive Secretary, ACOT
Ms. Stroup opened the meeting by commenting that the ACOT had a full agenda and was scheduled to meet until 4:00 pm. She noted that there were several categories of participants on the call – so, in order to facilitate conversation among group members, the ACOT and Working Group members have been given “presenter” status so they can make comments and answer questions. She commented that she was looking forward to full discussion, and introduced the chair, Dr. Barr.
Welcome and Opening Remarks — Mark Barr, M.D., Chairperson, ACOT
Dr. Barr noted that this was one of two ACOT meetings scheduled for 2013; the next meeting will occur, in person, in August or September and will be announced later in the year. As Ms. Stroup noted, the meeting agenda included reports from the ACOT Working Groups, as well as reports from other stakeholders working on issues about which the ACOT is interested. Dr. Barr introduced the first presentation.
Program Report — Chris McLaughlin, Chief of the Organ Transplantation Branch, Division of Transplantation, Health Resources and Services Administration (HRSA)
Mr. McLaughlin described that his Branch manages the Organ Procurement and Transplantation Network (OPTN) and Scientific Registry of Transplant Recipients (SRTR) contracts. He said that his presentation would cover Branch activities and Education Branch activities occurring since the ACOT’s last meeting. Some of these specific areas were also on the meeting agenda as separate items, so he planned to give more of an overview for those topics.
Mr. McLaughlin gave an update on the OPTN Kidney Allocation Policy. The OPTN Kidney Committee released its proposal for public comment in the Fall of 2012. The proposal was intended to enhance post-transplant survival benefit; increase utilization of donated kidneys; and increase transplant access for biologically disadvantaged candidates. The proposal included several features, including:
For the SRTR Liver Region Redesign Project, HRSA provided additional funding to SRTR to develop an evidence-based approach to optimize the geographic distribution of livers. The goal was to design allocation policies using a principles-based framework and mathematical optimization. The OPTN Liver Committee must achieve consensus on a principle to optimize in regional allocation (e.g., a primary metric for evaluating any proposed allocation system). Mathematical optimization will be used to identify aggregate geographic areas that maximize and equalize the chosen principle across all areas. Much of the modeling is complete; the OPTN Board charged the Liver Committee with the task of identifying a principle to guide the optimization process by June 2013.
On the OPTN Living Kidney Donation Policy, OPTN approved three Living Kidney Donor policies in the Fall of 2012: Medical & Psychosocial Evaluation, Informed Consent, and Follow-up. The Living Donor Committee developed checklists to aid programs in meeting the policies, which have been uploaded onto the OPTN website.
The Living Liver Donor policies were being developed and the Committee was also working on a policy proposal on Independent Living Donors.
On the Status of Vascularized Composite Allografts (VCA) and Notice of Proposed RuleMaking, the proposed rule was published on December 17, 2011 and the comment period closed on February 14, 2012. Twenty-eight comments were received, all favorable. The Final Rule is undergoing review and there will be more news on this soon.
On the OPTN Contract Extension/Re-competition, the OPTN contract, in place since 2005, has been extended through June 30, 2013. HRSA published a Request For Information in 2012 seeking input on several issues, including: the use of subcontracting, the OPTN National Kidney Paired Donation program, enhanced public participation, enhanced OPTN education efforts, modifying OPTN’s research capacity, and improving IT capacity and responsiveness. A Request for Proposals was posted on February 22 with proposals due by April 5, 2013.
On the OPTN Deceased Donor Potential Study, HRSA provided $1.6 million in direct funding to United Network for Organ Sharing (Center for Transplant System Excellence) to conduct a study evaluating donor potential in the U.S. The findings were expected to serve as basis for evidence-based goal-setting including: demographic and epidemiological approaches, systems dynamics modeling, measuring variables that affect donor potential, and addressing the full range of donor potential. Findings will be completed by end of the current OPTN contract.
On the OPTN/SRTR Program Specific Report Conference Follow-up, the Consensus Conference on Transplant Program Quality and Surveillance occurred last year. Short- and long-term action items were developed and are available on the SRTR web site. The PSR Manual of Operations was developed, which details the SRTR process for risk-model development and oversight. There is a three-year cycle of model development that includes the formal input and involvement of OPTN Committees; the kidney model will be the first to be rebuilt. OPTN input was sought on the variables; based on the SRTR Technical Advisory Committee recommendation, the methodology used to revise the PSRs will be based on a Bayesian methodology. This is in development. The SRTR met with the OPTN Membership and Professional Standards Committee to discuss this and develop new flagging criteria.
On the SRTR PSR and Organ Procurement Organization(OPO) Report Improvements, the SRTR has improved the output and the format of the OPO program-specific reports. These now use color and an enhanced visual presentation of the data. The data are the same, but were presented in new ways. This OPO reports contain measures of the expected donor yield using the models approved by the OPTN Board in 2011. The SRTR also released a Donor Yield Calculator tool, which is available for download on the secure websites. OPOs can use these to track observed and expected donor yield using the new models. A new OPO report format is being developed and reviewed.
Mr. McLaughlin added something that did not appear on the slides, but that has taken a lot of time (and consumed much of SRTR’s time): the effort to regain access to the Social Security Death Master File, to obtain the data needed for the PSR calculations. The data have been unavailable for more than a year now, but an agreement was signed between the Social Security Administration and Centers for Medicare and Medicaid Services (CMS) last week to provide access to these data. The contractors will verify the data to use for the PSR and other contract activities. The hurdle has been overcome and hopefully the next round of PSRs will be released on time.
On the HRSA/CMS Alignment Activities: consistent with the ACOT’s Recommendation #55, HRSA, CMS, and the OPTN contractor have continued to work to identify areas where they can harmonize oversight and regulation of transplant centers and OPOs. The first effort was a crosswalk of transplant center requirements from the CMS Transplant Center Conditions of Participation and the OPTN Bylaws & Policies. The crosswalk will enhance efforts underway to modify OPTN and CMS requirements, and survey processes to address areas that are similar but not identical. The findings may lead to modifications in OPTN and/or CMS oversight activities. This topic will also be discussed later in the ACOT meeting.
The Public & Professional Educational Branch has conducted several efforts to increase donations, including the “50+” campaign, a Workplace Hospital Campaign, and a range of promotional materials. The goal of the 50+ Campaign is to increase registry enrollment among people ages 50 and over, in partnership with the National Institute on Aging and the Administration on Aging. Messages promote the concepts that any age is the right age to sign up to be an organ, eye, and/or tissue donor, and that people should not rule themselves out, as age and medical condition do not automatically disqualify one from donating. The campaign was launched by HHS Assistant Secretary for Health, Dr. Howard Koh, with a Radio Media Tour that aired on 2,736 stations and reached 18 million listeners. The 50+ Campaign was driven by the large percentage of people over 50 who are on the waiting list (64%), the percentage of organ recipients in this age group (60%), and the fact that some donors over 50 believe they are too old to donate (32%). The campaign seeks to change these facts.
The goal of the Workplace Hospital Campaign is to involve hospitals and state hospital associations in organ registry enrollment. Partners in this campaign include 700 hospitals, 88% of OPOs, and 48% of state hospital associations. The Branch is actively beginning social media campaigns.
The Branch launched organdonor.gov on Facebook and posts several times a week, including both stories of organ recipients; content from organdonor.gov; and information about current events, news, and shareable graphics. The Branch has succeeded in getting a range of full-page Public Specific Announcment into popular magazines; it is also making a video to help demystify the donation and transplantation process.
On the HHS Innovation Entrepreneurs Program, DoT was chosen to participate in this HHS-wide program designed to increase innovative thinking and problem-solving in the Department. An external Entrepreneur/Fellow (a former United Parcel Service executive and an expert in supply chains) joined DoT for a year; the Fellow will work with the OPTN to identify a potential new system for electronic identification and tracking of organs. OPTN has an ad hoc committee guiding efforts to develop a “process map” to generate electronic organ labels and the scanning capability to track organs from procurement to transplantation. It is also looking at processes to scan the labels at various points during the procurement and transport process to ensure additional safety and tracking as the organ goes through the system; this will ensure additional confidence that the received organ was what they thought it was. A simulation is being conducted at Northwestern next week and at Virginia Commonwealth University over the next few months.
Dr. Barr asked, in terms of the problems sharing information between HHS, Social Security Administration (SSA), CMS and the release of the PSR, if the agreement would be long-term? He asked if a change in legislation was needed to address these problems, noting that the delay concerned many in the transplant field. Mr. McLaughlin responded that the effort was to create a permanent solution; it is more complicated than it used to be, but DoT thinks it will work and be long-term. It takes more effort from contractors, but it is not extreme and DoT is hopeful that it will work. Statutory changes could be implemented to make things simpler, but the Branch wanted to make sure it could go ahead without further interruption.
Dr. Barr asked if the ACOT could do anything to help. The response was that the DoT thinks that this solution will work (this has not always been the case) and it will keep pushing. It’s a reasonable resolution. Mr. McLaughlin stated that he was not sure what the ACOT could do other than make a recommendation if the process does not work, to provide the needed data. He committed to keeping the ACOT updated on how this was going and if the ACOT meets again in August, the hope is that the next round of PSRs will be released by then and he can report on that. If there are obstacles that prevent the PSRs from going out, it will be clear by the next ACOT meeting.
Dr. Eason noted that the ACOT had recommended eliminating the Donor Service Area (DSA) as the first unit of allocation. He said that Mr. McLaughlin had given a great update in terms of the Liver Committee, but commented that the most recent revision of the Kidney Allocation Proposal still had the DSA as the first unit of allocation. Mr. McLaughlin responded that the OPTN Board challenged each of the organ-specific committees to identify a principle of equitable allocation across regions. The model being developed for livers could be used for other organs as well. This was the intent: if the process is successful with livers, then the SRTR will be asked to work on kidneys next. The committee is aware that the solution does not fully address geographic distribution issues yet. Some organs are being shared more broadly, but this is just a first step.
OPTN Update — Brian Shepard, Acting CEO, UNOS
Mr. Shepard commented that some of what he was going to present about had already been mentioned by Mr. McLaughlin. He was going to talk about policy development, what UNOS has been doing with CMS, special research projects, and a strategic approach that is starting to use in an across-the board way (e.g., not a committee-specific way).
In policy development, Mr. McLaughlin mentioned that the Living Donor Kidney Policies were approved at the last Board meeting; the policies cover medical evaluation, consent, and follow-up. The previous policies required transplant centers to establish their own criteria and policies to evaluate consent. UNOS then assessed the centers to see if they follow their own policies. Now, as of February 1, there are consistent standards for all centers. The Living Donor Committee is also looking at liver policies that would follow a similar framework with some liver-specific tweaks. This should be released for public comment in the Fall of 2013.
For kidney allocation, the policy Mr. McLaughlin discussed addresses high discard rates. The kidney proposal does not change the fundamentals of the regional distribution process, but it is not incompatible with alterations in that process either. This will work if the sharing level is DSA, regional, or something new that comes out of modeling. It is not an answer to the regional equity questions, but will not prevent us from addressing those questions in the future.
In terms of the coordination with CMS, the crosswalk would be discussed later in the meeting, but one notable aspect in its development was that the crosswalk was originally an internal identification tool. It was intended to be a way for HRSA stakeholders to assess its policies against CMS policies in order to identify overlaps and prioritize what areas to work on first. But, it turned out that the process created a tool that can also be used by the field to help quickly identify requirements from HRSA and CMS that might apply to the same subject. It has become a tool and a useful reference document for others as well. And, it will works for the original purpose, too.
One of the things that came out of the crosswalk was the creation of ABO sample forms. These are voluntary forms; centers are not required to use them. UNOS staff worked with CMS to develop the forms, which contain all of the elements of both sets of requirements. A center can use the form to be guided through all of the items needed for compliance with OPTN and CMS requirements.
The next step will be to achieve better alignment of the policies themselves. These are being rewritten currently, and CMS is participating in this process. The sample forms also help communicate to the community that the crosswalk was the starting line, not the finishing line.
The two research projects have already been mentioned; the deceased donor potential study is nearly done. The demographic and statistical experts are working to see if there are ways to look at this. It is going to be interesting and will spur conversations about how we identify these donors, etc. Those could be national and/or local conversations.
The second research project is the electronic tracking and transportation project. This project has also engaged process engineering folks at Northwestern. The approach has been to look at the whole process in a process engineering format, and to identify weak points and potential failures. It represents a substantial opportunity over simply trying to react to the most recent failure. There will be opportunities to do more of this in the future as well.
Mr. Shepard closed by mentioning some small, but interesting, items; combined, they indicate the OPTN’s strategic approach. The organ-specific committees are very independent and the benefits of that kind of independence must be maintained. But, there are priorities that need to be set at a higher level. To this end, the Board adopted a strategic plan in June 2012, and created its own mechanisms for providing oversight into the IT process and the relationship between the required fees and workload. In the Fall public comment cycle, there was a recommendation to create a Board Finance Committee, to connect finance and workload decisions. OPTN is about to release for public comment a bylaw proposal to redefine the Vice President’s role on the OPTN Board so that person could spend more time on broad financial and strategic national issues. In sum, the direction is towards a more national, consistent, and strategic approach to transplantation.
Dr. Barr asked if there was anything the ACOT can do. He noted that, behind the scenes, there have been a lot of Work Groups with membership from both the ACOT and outside experts. Dr. Barr did not want to put Mr. Shepard on the spot but asked that he let the ACOT know if it can help. Mr. Shepard thanked Dr. Barr for that offer.
ACBTSA Update — James Berger, MS MT (ASCP) SBB, Executive Secretary, ACBTSA
Dr. Barr noted that Mr. Berger is the Executive Secretary of the Advisory Committee on Blood & Tissue Safety Availability (ACBTSA); the ACOT and the ACBTSA meetings will now always include reports from the other committee. This is the second time the ACOT has had this cross-committee report.
Mr. Berger serves as the Senior Director of Blood Safety in the Office of HIV/AIDS and Infectious Disease. This meeting presents a great opportunity to share information between committees and to prevent duplication of efforts.
The ACBSTA met on December 6-7, 2012, to discuss policy implications related to emerging research involving blood and tissue products available during public health emergencies and provide recommendations to the Secretary on this topic.
The ACBSTA considered if it should provide recommendations to advance innovations in blood and tissue products supporting emergency preparedness; whether there was an opportunity to develop the supply-chain infrastructure for emergency preparedness of blood and tissue products; and if HHS should provide more resources for research and development of blood and tissue products and supply-chain infrastructure to assist with emergency preparedness.
The principal recommendations were to address the lack of a system to manage tissues during a disaster; and to enhance the blood system to respond to, and recover from, disaster preparedness. Three subcommittees were formed to further explore the proposed recommendations, led by:
Recommendations about areas of potential enhancement for the blood system were as follows:
a. Reserves of end products and manufacturing consumables
b. Logistic support for transportation, information technology systems & communication
c. Connectivity with other disaster response system
d. Designation of committed blood and plasma donors and essential blood center staff as healthcare responders
3. Advancements of new products that consist of extended storage of blood products and products not requiring cold storage:
a. Extended storage blood products and products not requiring cold storage (e.g. modified, plasma, platelets, and red blood cells and oxygen carrying solutions)
b. Rapid low technology screening tests for infectious diseases (i.e., HIV)
c. Pathogen reduced blood components
d. Stem cell based products and growth factors
4. Regulatory flexibility
Recommendations about the preparedness of the blood system were as follows:
1. Identify unmet needs including funding
2. Characterize and address barriers
3. Establish evidence to project the likely risks and benefits of possible interventions
Recommendations about the preparedness of the tissue system (these are somewhat redundant to the other areas) was as follows:
1. Support a gap analysis to address issues of maintaining safety and availability of tissue products.
The next ACBTSA meetings will be held on June 5–6, 2013, and December 4-5, 2013, in the NIH Conference Room (5635 Fishers Lane, Terrace Level, Rockville).
There were no questions.
Workgroup Reports: CMS/OPTN Alignment — Danielle Cornell, R.N., B.S.N., CPTC, CMS-OPTN Workgroup Chair
Ms. Cornell provided background about the Work Group so new ACOT members understand how activities lead up to Recommendation #55. The Work Group discussed misalignments that it believed may have led to issues — such as the decline in donors and increase in the number of discards — that have been observed since the CMS regulations went into effect.
The Work Group discussed misalignments that included the fact that, under the Community of Practice, transplant centers are penalized when they do not stay above an outcomes threshold. There is a lack of good definition on Expanded Criteria Donor (ECD); the current SRTR model does not account for transplant centers that have large wait lists or want to take a risk on a more marginal organ but feel that they cannot take that chance. OPOs have high yield measures that include the number of organs transplanted – with bad definitions – so there was no incentive to go for a small number of organ donors.
Goals and data from the OPTN and the SRTR analysis are important — but these goals and the performance measures must be aligned in order to maximize every donation and transplantation opportunity and provide risk adjustment so organs are not wasted. Of course, the expectation is for good overall outcomes for every transplant center, while recognizing that there are transplant centers that do not have good overall outcomes and need assistance. It is not an easy task for CMS.
As a result, the Work Group developed Recommendation #55, which calls for CMS to work with a wide variety of groups to create the best possible solution. Ms. Cornell read the Recommendation (see below):
Ms. Cornell turned the presentation over to Mr. Walsh to discuss the regulations crosswalk project and then come back to the Recommendation.
Workgroup Reports: CMS/HRSA/OPTN Analysis of Regulations Crosswalk — Bob Walsh, DOT, HRSA
Mr. Walsh introduced himself as the HRSA OPTN Project Officer. The crosswalk and the November 2012 webinar were the result of long-term discussions between CMS and HRSA about the misalignment and overlap between the OPTN bylaws and CMS’ Conditions of Participation.
The effort was not intended to be a regulatory analysis of the Conditions of Participation and Conditions of Coverage; it was intended to look at policies and bylaws for transplant centers and identify where misalignment exists and opportunities to coordinate efforts between CMS and OPTN. The effort actually pre-dated the ACOT’s Recommendation and grew out of concerns expressed by the community. CMS wanted to better coordinate this area.
As noted, the crosswalk was intended to be an internal tool for HRSA, OPTN, and CMS to use to better understand the issues. The three goals were to identify the misalignments; identify the next steps for modifications to create better alignment; and create tools to help the transplant centers.
Progress has been made in each area. The misalignments have been identified; it was interesting to note that, while there were issues, they were less dramatic than feared. OPTN and CMS are working to modify policies and guidelines to address the issues. Discussions are on-going about better coordination for transplant surveys. Clearly, there was an alignment between the recent passage of new OPTN policies on living donors and for the living donor requirements for CMS; this was likely to foster coordination in the future.
As the webinar was created, it became clear that the crosswalk tool was useful for the community as well, to help the centers understand both compliance and the surveys. He encouraged interested stakeholders to look at the crosswalk and the webinar, and provided his contact information if anyone had questions or comments to share.
Workgroup Reports: Recommendation 55 Follow Up — Alexandra Glazier, J.D., M.P.H.; Danielle Cornell, R.N., B.S.N., CPTC; Daniel Schwartz, M.D., CMS
Dr. Schwartz described that he is the Chief Medical Officer at the CMS Survey and Certification Group. He started by noting that there had been a change in leadership on the transplant team at CMS – Karen is now the Division Director of Nursing Homes. The group can continue to call on her expertise, however, and the team at CMS is excellent. CMS team members have experience and come from transplant programs themselves. They are recruiting for Karen’s replacement, and Dr. Schwartz will be acting until that person is hired.
Dr. Schwartz provided an update on CMS activities. First, he noted that CMS surveys need to be unannounced surveys, and that CMS does not have the authority to let others do the survey process or deem transplant programs. CMS has been in discussions with HRSA about alignment and this is an important piece of its work. The greatest overlap was in the area of the donor surveys – staff will keep talking about how the agencies can work together to accomplish the same goals and decrease programs’ burden.
On the survey process, CMS has had a few rounds of rule-making to reduce the burdens. The most recent efforts made changes to the transplant survey process; this has been released for comment. The proposal is to lengthen the time span between surveys in order to reduce provider burden and target the agency’s efforts more strategically. CMS was exploring whether other data could be used to get same information. CMS was being more flexible in how it applies the survey rules. In the past, for instance, if programs had not reached their 95% reporting level (to the OPTN), it might have triggered an on-site survey. But, many times, CMS found that the programs were very close to achieving the 95% target and felt it could back off on that. The same applies for the volume requirements. These changes should be welcomed in the community.
CMS looked at its internal processes and survey processes and was about to launch a pilot to attempt to improve efficiency and quality. It was not yet clear what this would look like, but staff were working on it to reduce burden, recognizing that it is a burden to have the survey team on-site. He offered to report on the progress at the next ACOT meeting.
In terms of OPO outcome regulations, CMS has participated in a lot of meetings with the Association of Organ Procurement Organizations (AOPO), HRSA staff, UNOS, etc. about what to do about these regulations. The staff are hoping to have some discretion in the survey and certification group, and look at the data around non-compliance. They are working with the clinical standards group that writes the regulations and want the draft language to be finalized by the end of March. The consensus was that they are on the right track. The goal was to finalize the regulation text, then attach it as a rider to another CMS rule (a payment rule that occurs annually), so it can be changed quickly before end of the fiscal year. The proposed rule will come out in the next few months and should address some of the big issues around compliance and certification of OPOs.
Dr. Schwartz closed by stating that CMS is looking for opportunities to reduce overlap and burden.
Ms. Glazier said that it was very helpful to get an update about what is happening around Recommendation #55. These actions are a good start. She noted that the Recommendation addressed the need to look at higher reconciliations embedded in yield measures for OPOs, and how they mesh with outcomes measure for transplant centers. This has created system-wide inefficiencies and organ wastage. The misalignment was at the 30,000-foot level; hence, the Recommendation’s goal was to create unification between transplant centers and OPO regulations — not just between these groups. She asked what efforts were happening on this bigger picture.
Dr. Schwartz responded that CMS was aware of concerns and some of the problems inherent in the three measures. When they were put into regulation in 2006, there was agreement but issues have arisen since then, due to changes in transplant medicine and the use of metrics. He said that CMS was aware of the issues, as well as of the new OPTN yield measures and the benefits these measures have over the current measures.
He noted that it is hard to change regulations, however. CMS has focused on doing what it can to make changes during the re-certification cycle for OPOs. CMS noted the new OPTN measure and there may be something about this in the preamble; this is open for discussion. CMS was aware of the issues; he asked the ACOT not to think that CMS was not looking at this closely, because it is. Dr. Schwartz said that the next step may be to align OPO measures with transplant center outcomes. CMS was open to suggestions about how to do this; it has been focusing on OPO outcome regulations in the short-term.
Ms. Glazier asked if the ACOT could help to CMS address the larger misalignment by identifying more specific strategies. The response was that CMS works closely with HRSA on this. It would definitely help to have specific problems identified and prioritized, and a set of solutions presented for each. That would help CMS to think strategically.
Ms. Glazier suggested that the ACOT create a Work Group on specific strategies and solutions; bring them to the next ACOT meeting for endorsement; and then hand them over to the Secretary as recommendations. Ms. Levine added that CMS could also have representatives on such a Work Group.
Ms. Cornell suggested that the ACOT dissolve this Work Group and create a new one. Dr. Barr said that, based on guidance Ms. Levine, this would be possible and it was an appropriate time to create a new Work Group to help implement the suggestions. He asked the ACOT members to email Ms. Stroup if they want to be on the new Work Group, and then calls will be scheduled for the members.
Dr. Schwartz suggested that the Work Group include both CMS and HRSA in any recommendation language, so the agencies could move forward together. Dr. Barr said that was a good idea; he noted that these were not solely CMS issues by any means. Ms. Stroup will send an e-mail to the ACOT members; once the key components are in place, ACOT can tap other individuals who may have expertise and interest in participating in this group.
Dr. Barr asked if there were other issues that CMS wanted the ACOT to be aware of, and the response was no. Dr. Schwartz noted that Recommendation #55 was very helpful for focusing CMS’ efforts and engaging with HRSA. Dr. Barr said he was happy to hear that positive feedback; the ACOT members care passionately about this issue, so it was great to hear that the recommendation was welcome. He noted that the ACOT wanted to help CMS follow through, as well.
Ms. Cornell commended CMS and Dr. Schwartz for taking the lead on this area. The Recommendation has not yet been endorsed by the Secretary (due to a lack of time, not a lack of interest) but CMS was really focusing on it anyway, which is great.
Report on the Technical Expert Panel on Determination of Death — James Bernat, M.D.
Dr. Bernat provided an update on the long-standing project with the Technical Expert Panel on Death Determination. This panel has been studying this issue and preparing a manuscript about applying the theory and principle on controlled organ donation to the uncontrolled donation environment. He has presented on this issue before at other ACOT meetings.
The manuscript has been completed; the paper went through eight revisions on the consensus points, and three revisions on the actual manuscript. The Panel decided that an emergency medicine journal was the best place to publish it because Emergency Room physicians will do death determinations in an uncontrolled donation situation. The paper was submitted to the Annals of Emergency Medicine on February 25, 2013.
HRSA’s attorneys added a disclaimer, similar to one that appeared in the first paper published, noting that the Panel was not an official government body. Three panelists declined to sign: Professor James Childress (agreed with findings but was too busy to participate), and Dr. Douglas White and Dr. Scott Halpern, who disagreed with some elements of the argument.
Dr. Bernat is waiting to submit this to the ACOT until a final version has been approved; it is also available from several ACOT members, who have copies of it. He read the abstract:
One barrier for implementing programs of uncontrolled organ donation after the circulatory determination of death is consensus on the precise moment of death. Our panel was convened to study this question after we performed a similar analysis on the moment of death in controlled organ donation after the circulatory determination of death.
We concluded that death could be determined by showing the permanent or irreversible cessation of circulation and respiration. Circulatory irreversibility may be presumed when optimal CPR efforts have failed to restore circulation and the period has elapsed thereafter during which auto-resuscitation to restored circulation could occur.
We advise against the use of post-mortem organ support technologies that re-establish circulation of warm oxygenated blood because of their risk of retroactively invalidating the required conditions upon which death was
Even though the occlusion balloon that has been used in some protocols technically solves this particular problem, the panel consensus was that this was undesirable because it was contrived specifically for the purpose of declaring death; hence, the intervention might raise questions regarding complicity in the patient’s death.
D. Barr asked about the timeline. Dr. Bernat responded that the manuscript was submitted 10 days ago; the editor has already asked for some references so he expected that the reviews would arrive within a month, and then the Panel members will do the revision. It is hard to get responses and revisions done with 13 authors. Dr. Bernat expressed the hope that the final document would be completed within three months, although this depends on the responsiveness of the journal.
Dr. Barr asked if there was anything the ACOT needed to do around this, given the ACOT’s representation on the group. Dr. Bernat said he did not know if the ACOT has influence on various protocols within medical centers on death determination. Most of these have not rolled out for uncontrolled circumstances. There may be a way to address death determination protocols in the medical groups and have the ACOT influence how this is carried out.
Dr. Barr clarified that he was actually asking about something much more mundane: if the ACOT needed to sign off on the paper or something like that. He suggested the death determinations are more appropriate for JAHCO to consider. Ms. Levine commented that there were no additional legal requirements for the ACOT; she added that the ACOT could endorse the Panel’s findings, or recommend further research in this area, but it is not required to do so.
Dr. Malinowsky commented by chat what the group thought about how uncontrolled Donation after Cardiac Death (DCD) should or could occur? Dr. Bernat responded that the Panel specifically did not address that question (although some panelists wanted to). The Panel thought that its charge was not to do so. In the early work, the Panel created a flow chart of a process of an uncontrolled DCD and there was a lot of input on this. But, it quickly got out of hand – and it was not really what the Panel was intended to do. The charge specifically included the question of death determination in an uncontrolled DCD protocol. The Panel’s purview included where within the protocol the determination occurred, but not the process for determination per se. The Panel did not take an advocacy or protocol position, other than in making the overall recommendation.
Mr. Hodges asked by chat about the points of dissent for those who did not sign, and if their objections reflect widely held views. Dr. Bernat responded that one had to do with the “permanent and irreversible” concept from the first paper. Dr. Halperin was at an ACOT meeting and said this was not accepted; that was an element in his refusal to sign on. Dr. White did specify his reason. He did sign the first paper, but may have changed his mind. Dr. Bernat said he did not know how widely held the objections were; he had not seen any survey data on these issues. From his own travels and debates in scholarly literature, however, he agreed that there are influential people who disagree with the Panel’s position. But, his impression was that more people agree — although he does not have these statistics.
Workgroup Reports: Kidney Paired Donation — Andrew Schaefer, Ph.D., Workgroup Co-Chair; Dorry Segev, M.D., Workgroup Co-Chair
Dynamic Challenges Inhibiting Optimal Adoption of Kidney Paired Donation: Findings of a Consensus Conference — Dorry Segev, M.D.
Dr. Schaefer said he would give a quick introduction, and then turn over the presentation to Dr. Segev to provide background for those who were not at the face-to-face meeting. He noted that it was good to refresh some of the issues, and then discuss a draft recommendation from the Work Group. The Work Group wants to get a lot of input before moving forward.
Dr. Segev went over excerpts from his Fall 2012 presentation to the ACOT on Kidney Paired Donation (KPD) and reviewed the KPD Consensus Conference held in March 2012. Dr. Segev’s overview of KPD illustrated the various matches and types of chains.
KPD is growing dramatically in the U.S. and now reaches about 500 transplants a year, thanks to the establishment of computer matching to optimize the process. Non-directed donations have also grown due to press coverage and education on donations. Now, KPD and non-directed donations comprise about 12% of live donor transplants in the U.S. This means that someone other than the donor determines the recipient, relying on registries, centers, etc. to pick the recipient.
There are lots of versions of KPD administration in the U.S. UNOS has one, the national kidney registry has one, as does the Alliance for Paired Donation – many groups are working on this. Some are making unilateral decisions (i.e., only engaging their own patients on their own list). It’s a potpourri right now.
Questions remain about chains, including:
• Are longer chains really better, or do they just attract more media?
• When do you stop the chain?
• To whom does the last kidney go (e.g. which list does it go to, and to what patient on the list)?
Another question is what are the priorities for matching (is it “allocation”?). What are the priorities? Are they the same as with deceased donors, like peds have a priority? There is no transparency right now. Another question is optimization; do you use dynamic versus batch optimization?
Ever since Johns Hopkins shipped the first kidney from San Francisco for one of these kidney exchanges and showed it was safe, this practice has expanded significantly. It was basically the standard of care in exchanges between centers, but questions exist about the safety and logistics given the multiple airline segments and potential for loss and misplacement. Those are looked at to some extent, but not fully.
Financial questions are the biggest area and a big barrier to expanding KPD. Dr. Segev suggested that it was possible to triple the annual transplants to more than 1,000, but that finances have a large impact. Usually, the donor bills the recipient’s insurance; this is especially complicated when they are at different centers, in different states and have different state Medicaid. Who covers donor complications? Who pays for multiple donor/Non-Directed Donor (NDD) evaluations? If you have a highly sensitized donor, and want to maximize success, what happens when 10 people are worked-up and 9 are not used? Who pays for it? We want the donors to be safe and to know that their care will be covered.
There are eight areas of extra costs with KPD:
1. Evaluation of incompatible donors
2. Evaluation of NDDs
3. Histocompatibility testing
4. Center-level administration
5. KPD program administration
6. Kidney shipping costs
7. Donor surgeon professional fees
8. Donor complications/follow-up
In financing KPD, the goal is to transfer calls from the donor hospital to the recipient hospital, eliminate the volume disparity between centers, reimburse for donor services by out-of-network providers, present consistent and predictable costs for payers, and be compliant with CMS. Dr. Segev recently published a potential strategy to achieve these goals through a Standard Acquisition Charge (SAC). It is not a trivial effort to define the fee and agree on it; for example, should it be a national SAC or a center-level SAC? To address these issues, a Consensus Conference was held in March 2013.
The recommendations are on the website and are as follows:
• All potential living donors should be informed about KPD early in the educational process, prior to compatibility testing
• A centralized information resource for NDDs should be developed by the transplant community. Because of their potential to trigger multiple transplants, all NDDs should be informed about KPD.
• The greatest benefit for candidates can be achieved in a single well-functioning registry that encompasses the successful aspects of currently operating registries
• A National SAC would best serve KPD in the U.S. financial model.
Dr. Segev highlighted the phrases “well-functioning” and “can be achieved”. There was some feeling in the community that certain centers have creative ideas and should be free to do them on their own (e.g. San Antonio transplant center has highest volume due to its use of single center kidney donation program); there is a need to not block creativity. A National SAC provides opportunities to foster change and innovation; avoid overly heavy regulation; protect donor safety; ensure reimbursements; and create transparency in the process.
The payor recommendations mirrored this:
• The designation of a national organization to administer and provide oversight to KPD would best meet the needs of expanding access to Kidney Transplant (KT) in a fair and equitable manner.
• We are impressed by a number of ingenious and resourceful regional and local approaches that have been used.
• However, considering the scope of the national KT needs, we believe that a national system that maintains the foresight and flexibility to foster innovative approaches to KPD will allow management of one seamless national effort.
• To be successful, a national KPD program would be managed under the auspices of HRSA. (Irwin et al, AJT, 2012)
Dr. Schaefer introduced the recommendation from the Work Group, which was designed to be more of a carrot than a stick: it allows flexibility and lets single centers do what they need to do, but offers a huge advantage to participating in a more national effort.
Kidney paired donation (KPD) plays an emerging role in the United States, now comprising more than 10% of live donor kidney transplants. The current decentralized organization of KPD programs is not optimal in terms of equity of access, broad participation by centers and patients, donor safety, and transparency. Providing a nationally accessible KPD registry with incentives to participate in this registry rather than in smaller, decentralized programs would improve equity of access and facilitate participation by centers and patients. Implementation of a standardized reimbursement model (such as a standard acquisition charge) would improve donor safety by ensuring medical care for donors. Evaluation of all KPD programs by a centralized group would improve transparency.
To address these issues, we recommend that the Secretary identify a national KPD contractor responsible for implementing a nationally accessible KPD registry, identifying optimal matching strategies, and encouraging participation by all transplant centers. The contractor would also be responsible for:
• Administering a standardized reimbursement model for KPD costs, donor workups, and post-donation medical care;
• Evaluation of registries and transplant centers that choose to perform KPD outside of the national registry;
• Balancing the needs of current and future patients; and
• Striving towards equity in patient access to kidneys.
As incentive for participation in the national registry, the standardized reimbursement payments should only be available to those centers fully participating in the registry.
The recommendation attempts to maintain the spirit of the Consensus Conference, but also reflects the concern that this is allocation and a national registry is needed to address that fact. It is a growing and exciting possibility but there are benefits to a national registry to increase fairness and access, and donor safety. Dr. Segev added that the goal was to be consistent with the Consensus recommendation, continue to allow the needed flexibility, but take KPD out of the Wild West and put some money behind it, to make it work more efficiently. Donor safety and finances are two critical issues that require something to be done with money at a national level.
Dr. Barr commented that this was a huge area of concern and impacts a lot of people. Presenting the draft language provides an opportunity to have a conversation with the ACOT members and other stakeholders participating in the call. He asked Ms. Levine if she had any comments. Ms. Levine responded that the recommendation was clear and that the ACOT members were experts making a recommendation about what the Secretary should do. The recommendation should not describe how she should do it (e.g., such as through a contract), but the recommendation could describe reasons for specific ways to do it (e.g., why a contract works well).
Dr. Barr added that there was precedence for this because KPD is allocation and allocation is handled through contracts. His opinion was that the ACOT could make this language more specific.
Dr. Segev said that, ideally, contracts ensure that the money was put behind this issue. There is no desire for an unfunded mandate for KPD – there was a real need for financing to make donors safe, and to improve the financial model for administering KPD. Dr. Barr agreed that if there were no financial platform, it would hurt the field. Dr. Schaefer added that the potential increase of 1,000 more donations was very beneficial and the SAC would create a win-win-win.
Dr. Gerber said that he was struck by the two goals; one was to ensure there is protected safety in the KPD realm, and get out of the Wild, Wild West. The second was to create a model for a SAC. He asked why this situation was different from a living donor who does not come through the KPD process. Another regulatory body was already overseeing this, so shouldn’t this already be happening through the current mechanisms? Was the goal about not stifling innovation?
Dr. Segev responded that the recommendation was not to address donor safety in the context of all living donors; it was more about addressing the additional issues that stem from KPD. If one cannot figure out who pays for follow-up (e.g., the donor is under a different state’s Medicaid), then the donors would be at greater risk and may not get follow-up care. It’s different from a regular donation.
In terms of allowing the continued innovation, the Work Group tried to make it an incentive, not a punishment. To get the SAC, a center would have to fully participate in the registry, which might have some allowable variations so creativity continues while the financial and safety issues are addressed. Centers could do what they want to, but would not get the SAC. It is like the way that regions can do things their own way – there are variations that can be allowed but also can be evaluated and are transparent and fair.
Dr. Gerber said he accepted what Dr. Segev said, up until the variance part. He said he had an uncomfortable feeling when there are multiple different entities because it was not clear who was in charge of the contract, and that can influence others in terms of what they are doing. Variations among regions do not have the same a financial impact on the patient or centers; they are more of an allocation variance.
Dr. Segev suggested a few scenarios based on current structures. Say there was a national contractor, and a center that has a successful single-center program — they could welcome to continue what they are doing. In a single center, there are fewer financial issues, because it looks like a conventional live transplant. The single center would have the right to keep doing what they are doing, and does not need the SAC because they have it figured out and get paid through the organ acquisition charge.
If there is a national contractor that does things one way and a multi-center group that has been successful with KPD but did not manage to land the national contract, and wants to continue doing their multi-center stuff… if the contractor is harsh and does not allow it, then the group can continue to do what it is doing today. One example would be for the multi-center group to charge the individual transplant vendors for every transplant they facilitate and that money goes into whatever it is going to cover and the transplant centers are happy. That would be allowed to continue; we are not saying nobody else can do anything, but we are seeing the financial infrastructure established the standardized reimbursement made available to those who participate.
The second competing group that has been disallowed by a variance by the main group, either they have the potential of applying for that contract and being what they wish the contractor would be, or it involves 150 centers, and they would have every right in the world to complain about the contractor and push for their variance to go through. Then they can get the SAC. Whatever this does, it would not make it impossible to do anything that is currently being done today, because today it is done without the SAC. That is the reason we tried to go with incentives for participation rather than disincentives for being innovative.
Dr. Eason said that added some clarification; but he was still confused about how this improved donor safety over what’s already being done by OPTN / UNOS. It seemed that it was redundant to the current oversight. The response was that maybe the recommendation needed to be more specific; there is great work happening on safety already. The recommendation was about KPD-specific safety: making sure donors get follow up care and it is clear and paid for – that’s all. Lots of centers have had issues figuring out who is paying for donor complications and follow up. It’s been fine so far but would be more guaranteed with a standardized reimbursement mechanism.
Dr. Segev noted that several people had raised this issue, so the Work Group needed to make sure the wording did not imply that the new contractor would also put together donor safety issues; the new contractor would only worry about the issues of donor reimbursement.
John Hodges commented by chat that all living donor candidates face the potential of complications, and equal care in making clear what their options will be for covered care. The speakers agreed that, when the recipient is at a different center, this puts the donor at risk and that’s all we are talking about.
A participant asked whether the OPTN infrastructure could be used to create a policy or oversight to cover KPD donors, rather than creating another entity to provide oversight? The response was that the donor safety issue in KPD is a financial one. The only way to ensure that donors get coverage was for a financial entity to manage it. And then, this was creating something new that is beyond the scope of the current OPTN contractor.
A participant said that, in terms of safety, when the donor operation occurred at a center other than the recipient center, it required significant costs such as work ups, surgery, etc. It was debatable how much oversight that entity has on allocation, transparency, and such. But, KPD donor safety was different when they were in different states (in terms of contracts, Medicaid vs. Medicare, etc.) and this can only be addressed by an over-riding entity that provides financial support. A SAC fee would require investment that gets paid back at the end.
Dr. Gerber said that this was even more of an argument for this staying in the OPTN. There are living donors who are directed living donors. The thought of a contractor that has some component of oversight but not all oversight… it creates the potential for conflict because there were two groups with a role in some mechanism of this sort, driving the process moving forward in a positive way, and potentially coming into conflict with one another.
A participant commented that one of the reasons the KPD community has been reluctant to have all of this happen through the OPTN is that OPTN had been trying to do this for several years and there have been other mechanisms that have been more successful. There is a feeling OPTN wants a contractor to be more streamlined and aimed only at KPD, which is a rapidly changing and dynamic field. It needs to be more nimble than the OPTN contractor, which has a million things to do. Any time an OPTN committee approves an allocation change, it takes three and a half years to program it into their computers — that is not the pace of KPD. In 2005, there were three centers doing five transplants, now there are 500-plus with rapidly changing computer models and new and innovative ideas. It would be harmful to the field to have a contractor that was not entirely dedicated to KPD and designed around the needs and the unique aspects of KPD.
C. Wright asked by chat if this should not be something that covers all living donors. Some living donors travel to donate, and may experience financial and health care problems if they have complications when they return home post-donation. These donors should have the same financial assurances that were being suggested for KPD. The response was that KPD was unique in that the living donor usually donates to a selected recipient and it was very clear whose insurance pays (the recipient’s). In a KPD, the donor did not know who the recipient was, where they live, what state their insurance was in, etc. The KPD contractor needs to be focused on KPD and its unique issues.
Ms. Luebke commented by chat: All the more reasons for Medicare to cover all donors. Dr. Barr said that many people agreed with that statement.
Harvey Mysel commented by chat that the system developed for deceased donor transplants is so different than what was needed for living donation in KPD that the community should start from the basics and develop a new program. Even the UNOS living donor program was not using the OPOs. Dr. Segev said he read this as agreeing about the need for a separate KPD contract rather than tacking it onto the current contract. It should be separate due to unique aspects of KPD that have to be addressed specifically.
Dr. Barr said that this was very useful feedback. In his opinion, the recommendation seemed to reflect the spirit of the Consensus Conference. There were some wording
issues and other concerns that the Work Group needs to clarify.
John Hodges commented by chat: So, you're moving forward aware of the double standard inherent in this proposal? Dr. Segev said that it was important to move ahead on the KPD end of things. The ACOT could consider a recommendation for the general issue of donors who travel and ensuring they get appropriate follow up. He hoped the field could learn from KPD and apply it to other donors.
Ms. Glazier said that she thought this was a good recommendation but it did not include the issue that KPD was about allocation; this means there was a significant difference with it in terms of transparent and public trust. She expressed the view that it was totally appropriate for the ACOT to make a recommendation on this.
Donna Luebke commented by chat: What is the benefit to taxpayers to fund a national KPD contractor when nothing has to change? Would the KPD contractor get the KPD registration fee or would there be another registration fee (beside the OPTN and UNOS fees) for the insurers to pay? Dr. Segev responded that he did not agree nothing needed to change. It is not sustainable to keep it going the way it was now. The financial benefit model clearly supports anything that will sustain an innovative live donor practice. Every time someone is transplanted with a live donor who would otherwise not get a donation, Medicare saves something like half a million dollars. This has to be addressed, since it has turned into allocation.
Dr. Barr agreed about the need to do something on this and said there was a sense of urgency felt by the Work Group. This recommendation was not ready for a vote but it should move ahead; he asked for a final recommendation to be presented for vote at the August meeting. He agreed with the comments about the urgency of the situation and the expanding clinical domain.
John Hodges commented by chat: Equal protection under the law? Harvey Mysel commented by chat: Yes, equal protection… also to save unproductive lives and to save money that is currently being spent on kidney dialysis. Dr. Barr commented that every time we do one of these successfully and safely there is a huge financial advantage for the taxpayer.
John Hodges commented by chat: Any substantive difference in treatment could be interpreted as incentive, or even coercion. The response was that it is a huge financial advantage to do these donations. The goal for a SAC to be available to those only to those who fully participate in the registry is intended as an incentive. We do not want to make it a requirement and hamper innovation. With single centers, it was less complicated so they do not need the SAC if they want to do innovative things.
Fully participating in the registry is another part of the incentive – there was a concern that if only partial participation was allowed, centers might distinguish what patients go into the registry and keep the easy ones. Full participation was designed as an important incentive. It was not the case that, if a center did not send all of their patients to the national registry, it would be cut off for life. The suggestion was that, if there was a reasonable paradigm for dealing with some patients in the center and some in the national registry, the contractor could assess this. This would leave the flexibility in the contractor’s hands, which is why the contractor had to fully focus on KPD and be nimble and innovative – it cannot be an entity that was not KPD-focused.
Ms. Levine raised potential legal issues. HHS needs to explore if statutory authority existed for any entity other than OPTN to do KPD allocation. There was also an issue if CMS reimbursed providers for a new fee and whether that could be made under a national registry at one level, but not to other providers that perform the same service on their own. The procedure was the same in both instances. But, she said, this could be figured out later; the ACOT should recommend the best way to go forward and staff can work on how to modify the authority later on.
Dr. Barr described the next step; the Work Group will work on wording in order to address the comments from this meeting. He asked that the final proposed recommendation be ready for vote at the ACOT’s August meeting. This would put the issue into the spotlight; the ACOT will do the field a service by accelerating the conversation. The Work Group membership can be expanded; if anyone wants to join it, please email Ms. Stroup.
Dr. Gerber asked when Ms. Levine would have guidance on the legal issues about payment and whether another entity could do this work. He said that information would be helpful as the group proceeded in making the recommendation. Ms. Levine said that DoT might want to see the ACOT recommendation beforehand, however, and recommended that the ACOT proceed with the work. Dr. Gerber said he agreed that this was allocation; he would like to see maximum incentives to participate and minimize any redundant regulatory activity going forward.
Dr. Schaefer apologized for not listing the Work Group members in the slide and asked that to be updated in the PDF posted on the website.
Ms. Corning from CMS commented that Dr. Schwartz had mentioned that there was a proposed rule out about changes in transplant centers and a few things about OPOs. She noted that she had sent a link (http://www.gpo.gov/fdsys/pkg/FR-2013-02-07/pdf/2013-02421.pdf) where people can see the rule; the comment period ends on April 8. She invited participants to email her with questions / comments. Ms. Stroup said she would distribute the link to the ACOT members. Dr. Barr noted that all participants in the call can also submit comments, and the ACOT members can submit as representatives of their organizations and/or as individuals.
Research Barriers Management and Innovation — David Gerber, M.D.
Dr. Gerber noted that he is the ACOT co-chair for the Research Barriers Work Group; Sandy Feng is the non-ACOT co-chair. This is a product from the August meeting at which Doctors Feng and Abt presented on an American Society of Transplant Surgery presentation, and Dr. Halperin shared an AST presentation on research barriers. The Work Group was designed to bring together the background and see how to move forward.
The Work Group primarily focused on the donors and research involving deceased organ donors, but there were some components that translate to the recipients, as well. Organ quality limits transplant outcomes; there has been a decrease in ideal donors with a corresponding increase in marginal or expanded criteria donors within all organ systems.
Donor quality defines post-transplant outcomes according to risk of graft loss, patient death or need for re-transplantation. Inferior organ quality engenders recipient morbidity
and mortality, which is germane to the patient and to the specific programs. There was a need for innovation and research in donor intervention and treatment in order to:
• Increase the number of organs available for transplantation
• Mitigate waitlist morbidity and mortality
• Improve the quality of organs used for transplantation
• Mitigate recipient morbidity and mortality
Dr. Gerber showed a slide to illustrate the complex process of getting from donor to recipient for donor-based research. For clinical research, the areas that come up include IRB approval, how protocols get disseminated, getting consent, etc. How can move field forward given the complexities? The obstacles to research and innovation in brain-dead deceased donors, include scientific, ethnical, logistical, and regulatory obstacles.
The problem was that the current ethical, logistical, and regulatory infrastructure was inadequate to support effective donor intervention and treatment studies. The magnitude and complexity of the challenges require guidelines to facilitate the optimal design and safe execution of clinical trials in deceased donors. The goal was to develop something broad enough to be used in all donor-related studies and help move the process forward
The Work Group has had two conference calls in last several months; its membership includes ACOT, HRSA, and other experts. Defining the challenges was the major first hurdle; questions include:
• What is required for performing research on brain-dead deceased donors? Institutional Review Board (IRB) consent; other legal issues?
• How do we engage widespread donor research activities? What is the role of donor hospitals, and what is the potential impact on organ distribution?
• What ethical issues and societal perspectives impact this issue?
• Develop/codify a process-based strategy that can be applied to multiple research endeavors versus “one-off” solutions that are not translatable, sustainable or focused on low-risk interventions.
There have been several publications on donor research activities, which Dr. Gerber described. There has been a lot of variability in practice in this area. In the U.S., less deceased donor research has been published than in Europe. One study from 2009 got verbal rather than written consent, and no consent was sought from the recipient. In a 2010 study, no consent was sought from the donor or recipient; the study was approved by IRBs in each of the 12 centers. In a second 2010 study, no consent was sought either. Dr. Gerber explained the rationales given for why consent was not sought.
With respect to the legal basis for research authorization in deceased donors, organ donation and associated research is based on gift law (donation is viewed as gifting and not an informed decision by health care). This was background to consider going forward.
There are challenges to donor consent for research, which include:
• Lack of consensus about whether autonomy and risk are meaningful concepts for the deceased
• Proximity of research to death/grieving
• Patients rarely have indicated research preferences
• Appropriateness of “general research consent” to research.
In terms of donor focus around research, IRB was not an issue for donor because the donors are not living subjects. But, “autonomy” and “risk” are meaningful concepts for the deceased. State statutory legal requirements under the Uniform Anatomical Gift Act (UAGA) could apply depending on the research. Health Insurance Portability and Accountability Act (HIPAA) applies to deceased Protected Health Information (PHI) and has research standards that should be considered. There was a precedent for genetic studies, as it potentially affects family members. One question was whether research would have a negative impact on gifting intent of decedent or decedent’s family.
The Work Group also discussed the role of the Donor Hospital with respect to the IRB, as the research is coordinated through the HCP and potentially the OPO rather than the donor facility. Guidance was needed from Office of Human Research Protections (OHRP) and legal opinions, and has been sought.
In terms of the recipient focus, there were several challenges, including:
• IRB issue for recipients (logistical challenges)
• What happens with a potential recipient who doesn’t want to participate
• Can recipients reasonably opt out?
• Would data submitted to the OPTN be accessible without consent?
• Can the transplant community guide patients regarding probabilities and magnitudes of risks involving the organ?
In terms of IRB logistics, there were also issues:
• Is there a need for multiple IRBs versus a national IRB model? There is a precedent for the latter from AIDS trials and pediatric oncology trials.
• Meaningful disclosure issues with transplant recipients. Examples include: Centers for Disease Control (CDC) high risk donors, ECD.
• Need for IRB to help minimize risks to recipients and minimize coercion to recipients. Examples include a novel agent that impacts kidney function; what does it do to extra-renal organs
Dr. Gerber presented possible solutions, noting that these were general, not from the Work Group specifically.
• Donor (surrogate) consent: Would crafting specific consent documents for individual studies be superior to generalized research consent?
o The role of consent for organ donation and local/state implications
o Without recipient consent the functionality of any research study is severely limited
• Recipient consent: waiver should be sought (as consent couldn’t achieve goals):
o Could build into generalized prospective consent.
o What would this “waiver” cover? Do we need a waiver?
o UNOS / OPTN data: there may be precedent that this should be available to researchers without additional consent
o One certainly cannot do anything to a recipient (collect specimens or additional data or anything else) without consent as recipient would clearly be a human subject
• Novel, cross-cutting regulatory structures
o Based within professional societies? Regional Review Boards (RRBs) or OPTN?
o Legal risks to transplant surgeon and center
o Recipient autonomy and decision-making issues
HRSA was planning a conference on donor-based research. Potential action items coming out of the Work Group would include recommendations, an ACOT recommendation endorsing the Conference. The latter could include a focus to include operational strategies, to create a model that can be applied to donor-focused research trials by establishing precedent or templates. The ACOT could recommend that the organizers of the HRSA conference engage experts with prior involvement in trials with design challenges (e.g., DOD, Vaccine trials, etc.).
Dr. Barr noted that said Dr. Feng was on a flight and could not join the conversation, but there were other American Society of Transplant Surgeons folks in the chat room who might be able to respond.
Dr. Gerber said that he and Dr. Feng have talked; both of them were very focused and committed that the HRSA Conference be valuable. It should be very processed-focused. As background, he said that there had been more than two years of discussion on how to do this research (focused on IRB and donor hospitals). Dr. Gerber’s personal perspective was that the Conference should be process-focused because it will help the donor hospitals and the OPOs. Donor families get approached about this and it would help the OPOs address it and ensure that families are well-informed.
On the recipient side, the situation is fuzzier; there were issues about recipients making an informed consent about accepting an organ that has been involved in research.
Dr. Barr asked Dr. Beigay to talk about the OHRP’s perspective on this, since she was on the conference call with OHRP. She reported that this seems very do-able. There is a need to address these issues but if the varying efforts are aligned there is the chance to move forward on this. Dr. Beigay expressed confidence that they can move ahead, given the discussion with OHRPs.
Pete Abt suggested that, as the Consensus Conference was being planned, organizers should reach out to White House Office on Science & Technology. This office has worked with industry on consent around difficult trials. There were some useful position papers on-line, as well.
Dr. Gerber commented that they have wanted to connect with some of the people who have been involved in these trials. There is operational precedent in capturing the intellectual history. In terms of a recommendation, he wondered if it was better to wait until after the HRSA conference and then develop a recommendation from the ACOT.
Dr. Barr said that, given how complex this issue is and how many entities are engaged, his suggestion was to fist see the output from the Conference, which will include participation from the ACOT members. This has been a long-standing issue and (like with KPD), the fact that the ACOT’s discussion is bringing it to the forefront is a service in and of itself. The recommendation itself is less important as seeing that the consensus meeting is happening. There is no urgency; the ACOT must make sure its recommendations are consistent with legal statute, as well.
Ms. Glazier said that there is a lot of confusion in this area, but that there are also answers to some of these questions. We need to sort through the answers that exist and see where there are remaining issues and gaps that ACOT could weigh in on. Then, the ACOT can focus on the remaining barriers and highlight them.
Dr. Barr asked if she could share her experience in this area, since her background was very interesting. Ms. Glazier noted that, in her previous work, she advised academic centers on human subject, compliance, and legal issues – she currently teaches a class on this issue in the law school. She said she was happy to help the ACOT work through these issues.
Dr. Barr asked what the timeline was for the consensus conference. The response was that it would occur sometime in the fall. Dr. Barr said that the timing of the next Work Group meeting can be influenced by that; the group should shoot for having a few conversations before the ACOT’s August meeting so people can be brought up to speed. They may not be able to do anything until after the consensus conference, but could look at what the VA has done and learn from them.
Peter Abt said that previous trails often have involved medications that were standard, approved, and low-risk. In the future this might not be true; the risks might be greater in the future. Dr. Barr agreed and said it was inevitable that this would involve UNOS and OPTN. There have been some attempts / proposals in some DSAs that highlight the need for recipient safety. Low-risk trials are easier, of course. High-risk ones, with the complexities of local and regional sharing, are very different issues. When there are systemic issues that involve altering the donor itself, there are all these complex IRB issues and a need for a mechanism to address them. There have been proposals, and even attempts, made to do some research studies without knowledge throughout the transplant network. UNOS has to be at the table.
Dr. Gerber said that this is one way we can help remedy these dilemmas that have occurred in the past. Patients should be able to express interest in (or turn down) organs that have been in a clinical trials, etc. If an organ crosses out of a DSA, patients need to have had the chance to say they are interested in that type of organ. Dr. Barr agreed and said this crosses over into allocation issues.
Darren Malinoski commented by chat: The Research Outcome Consortium studies pre-hospital /emergency room/trauma bay interventional Randomized Controlled Trials (RCTs); they obtain “community consent” and have an elaborate process for that. Dr. Gerber said he has experience in that type of consent.
Raymond Benza commented by chat: Will this also include research on donor related tissue that is not manipulated prior to donation? Dr. Gerber said the ex vivo lung study is a precedent for that. Darren Malinoski commented by chat: In terms of ex-vivo interventions, the issue can become more complicated if a large amount of blood needs to be drawn from the donor prior to organ recovery in order to conduct the ex-vivo intervention (i.e., heart in the box).
Dr. Barr said that was a good point; there have been complaints from the liver community in the areas where the study was being done. The donor is being exsanguinated to prime the system and this is unacceptable; the procedure is being rushed. The trial was not run through UNOS or other programs (e.g., liver, lung) and this sort of thing should be captured by the mechanism. Ex vivo in itself does not preclude it from having an impact on other organs. Having Ms. Levine, and Dr. Halperin, and Ms. Glazier present to think about legal, ethical issues will be helpful. If the ACOT members want to be on the Work Group, please tell Ms. Stroup.
Update on Vascularized Composite Allografts (VCA) — Ms. McDiarmid
Ms. McDiarmid provided an update on what is happening in the field that might benefit from the ACOT’s input.
VCA numbers continue to increase, although they remain small when compared to solid organ transplant. The issues to the field about VCA jurisdiction are very important. Right now, a hand or face is not considered to be an organ. So they fall under the Food and Drug Administration (FDA)’s jurisdiction (because they are considered to be tissue).
It is important for VCA to be defined as an “organ” because of the needed involvement of OPOs in terms of getting consent from donors, handling graphs, etc. HRSA made a determination in 2011 that this should go forward and that VCA should be redefined as an organ. But, there is no structure for moving this goal into reality.
Programs (there are now at least 12 programs doing hand transplantation and 3 doing face transplantation) have no standardization for procurement, allocation, etc. There is no list. The centers have had to create their own relationships with OPOS to make sure these transplants happen in the right way, and happen safety, to address disease transmission, ethics around family permission etc.
The numbers of programs are increasing and the numbers of transplants as well (especially for hands). To date, centers have done 35-40 hands, and maybe 3-4 faces. The complexity of the approach (medically, surgically, ethically, socially) has a broad impact. Outcomes have been very good in terms of survival. It is important to do collaborative studies to advance the science.
Dr. Barr commented that the ACOT made a recommendation on VCA and suggested putting it under OPTN rather than under the FDA (it was either Recommendation #53 or #54). Is there enough of a problem and lack of progress that the ACOT can do anything, or should we leave it to UNOS? What happened to the recommendation?
Ms. Stroup said that there is a notice for proposed rule-making out on this and the comments have been received. HRSA has to propose a final rule and then implement it.
Ms. McDiarmid said that the Secretary signed off on this more than a year ago, yet the final rule has not yet occurred. The ACOT’s help in pushing it forward would be very helpful. The potential for difficulties and public feeling disenfranchised is very real. There is a need for more transparency in this difficult situation.
Dr. Barr said that the ACOT made the recommendation quickly and he was dismayed that there has been so much delay. The DOD is interested in this because of the wounded soldiers coming back home. There were a lot of interested parties in this issue, and
everybody felt that it was a white hat issue. This was the right thing to do.
Ms. Levine agreed that it can be frustrating but noted that there has been substantial engagement about this issue in the department. When the ACOT writes its report on the meeting, it may wish to highlight the ACOT’s continued interest in this area, and its interest in getting this issue resolved as quickly as possible.
Dr. Barr said that he and Ms. Stroup would work with Ms. Levine on that. The ACOT does not usually write a letter or highlight anything, but could do so in this case, and refer to the recommendation and the fact that the ACOT is interested in expediting the activity around VCA. When the ACOT made the recommendation, VCA was tiny but it has really started growing now. This cannot be sat on somewhere.
Ms. McDiarmid suggested that the ACOT make a new recommendation specifically on expediting the rulemaking around VCA. Ms. Levine reported that she had looked up the status and the OMB has the final rule under review there. It is not at HRSA or HHS anymore. OMB has 60 days or so to review it, so, absent major changes, this should happen soon.
Update of Donor Management Activities — Teresa Beigay, Ph.D., DoT, HRSA
Dr. Beigay presented an overview of the donation and transplantation Community of Practice Committees. The process is moving the work of the Collaborative into the community. The meeting and the task forces are being run by the Alliance, in partnership with HRSA.
The first subject is the strategic planning process. The Donation and Transplantation Community of Practice (DTCP) is working to create an action plan for 3-5 years and create goals to accomplish. Dr. Beigay showed a slide that illustrated the strategic process and the inter-relationships at work. There are meetings and task forces with a lot of interplay in terms of improving practice. They also interact with OPTN, SRTR, ACOT, etc. There are several task forces that create a lot of products and tools for the community. Each has different work groups, as well.
The Donor Management Task Force’s best practices workgroup has put out a toolbox that includes resources for OPO and donor hospitals. The declaration of death workgroup has focused on the American Academy of Neurology (AAN) guidelines and training for community hospitals on how to conduct a brain-death evaluation, and ensure consistency in how brain death is declared. The scientists’ knowledge workgroup is looking at standard outcome measures, organ-specific measures, consent issues etc.
One of the transplant center task force’s first projects was to identify ways to recognize transplant centers, specifically at the National Learning Congress. Previously, transplant centers were recognized mostly due to volume, but the task force felt that quality was important to. It developed awards criteria for recognizing transplant programs that are performing well in terms of quality. They are also working on a quality resource similar to a toolbox, but taking the change package developed in the Collaborative and transforming that into a quality resource.
The quality improvement task force is looking at how to use data to improve performance. They have developed a quarterly scorecard and are looking at ways to look at the DSA performance as a whole — not just at the OPO or transplant center — to assess how they function together as a team. The task force has an annual Performance Improvement Summit that offers an educational opportunity for the community
A few other things that have come out of the work that the ACOT has done, in terms of helping identify needs. The brain death testing webinar came out of the donor management task force’s declaration of death workgroup. The webinar is targeted at the community, specifically at physicians and critical care staff at hospitals and OPOs. They have had two live webinars to date with 1750 participants. They have had a lot of impact.
The webinars are stored on the health care community website and the Alliance website as well, and are accessible to anybody.
The donor management research consensus meeting is a community of practice. It is not just a HRSA meeting; HRSA and the Alliance worked in partnership on it. The long pathway that Dr. Gerber described on research; we involve representatives of our national partners in planning. We are not defining the content specifically; we want to get the planning committee to develop the specific content and the approach for that meeting.
In terms of the progress on the three national goals, the goal for the conversion rate has been surpassed, but has been at a plateau for the last few of years. More improvement can probably be made. A lot of activities are geared toward what can be done to continue to increase conversion rates involving donor designation authorization, how to help increase honoring those authorizations and others, and other issues related to conversion.
The goal for organs transplanted per donor, there has been a decline in this over the last few years. There has been an increase in DCD donation, so there is a corollary there. We are exploring what can be done to increase the number of organs recovered per donor. Donation after circulatory determination of death has surpassed the goal and continues to increase. At this point, we want to look at some of the national variation; the range is incredible and goes from something like 1% at the bottom to almost 40% across DSAs.
Discussions at the ACOT help us decide what, as a group, we should do moving forward.
Dr. Gerber asked about the DCD goal of 10 percent, and whether this had an impact on potential decreased donors as the number creeps up. OPOs are a bit stuck between getting more decreased donors and not losing some of the brain dead donors. Dr. Beigay responded that this was a driver of the declaration of death workgroup. The annual donor management summit always stresses that DCD should be encouraged — but not at the expense of brain death donations. Teamwork has to occur between OPO and hospitals to address this. They are also looking, as part of the strategic planning process, for ways to look at national goals that may be better; this may not be the best goal anymore.
Jan Finn commented by chart: DCD donation goals do not involve recovering donors as DCD to meet the DCD percent goal. Donation by brain death is always the first priority for recovery. I agree with Teresa, just validating practices in the community.
Dr. Barr noted that Ms. Glazier just sent an e-mail of a public notice that the Secretary’s ruling will come out at the end of the month. She added that, in New England, there are three VCA programs, so they have had to create their own allocation system. Hopefully, if the ruling comes out at the end of the month, there will be more dialogue and that would get right to the OPTN.
PHS Guidelines — Matt Kuehnert, MD, ACOT ex-officio member, CDC
Dr. Kuehnert said that his report would be brief. The PHS guidelines have been cleared through all of the PHS agencies, and is waiting for Secretary Sebelius’ signature. It is on her desk. As soon as that happens, it will go to Public Health Reports, which is the Surgeon General’s report, and will be published in August or October issue. It may be on-line before that, though.
There was no discussion.
Dr. Barr asked that if members or the public have suggestions for presentations at the August meeting, or new Work Groups to be formed, to please email himself or Ms. Stroup with suggestions.
Dr. Barr asked if there were any prepared comments. Ms. Stroup said that one person had had a question and Ms. Stroup suggested he speak during this period. But, she did not see his name on the list of participants. The operator opened the phone lines to the public. There were no comments.
Ms. Stroup said that new appointments to the ACOT would be made in the next month, etc. The ACOT is looking for new nominees as well.
A motion was made, and seconded, to adjourn. The meeting was adjourned at 3:30 pm.