Advisory Committee on Organ Transplantation

Advisory Committee on Organ Transplantation (ACOT)
Fall Meeting
November 4 & 5, 2004

Doubletree Hotel and Executive Meeting Centers
Rockville, Maryland

Thursday, November 4, 2004

Welcome

Nancy Ascher

Nancy Ascher welcomed the meeting participants and noted that the three areas of concentration—valuable consideration, fair treatment, and waitlist—are reflected in the subcommittees' titles. Dr. Ascher then introduced Dr. James Burdick who spoke about the progress achieved in regard to the Advisory Committee on Organ Transplantation's (ACOT) first 28 recommendations.

Update on ACOT's Recommendations

James Burdick

James Burdick filled in for Thom Balbier, Executive Director of ACOT, who was unable to attend the day's meeting. Dr. Burdick asked that anyone who wished to make a public comment please speak to Sherry Whipple to arrange a time to address ACOT. He thanked Sherry Whipple for her part in planning ACOT meetings.

Dr. Burdick acknowledged Mr. Balbier's efforts to bring in Ex-officio members from four of HRSA's sister agencies. Ruth Solomon of the U.S. Food and Drug Administration (FDA), Marcia Newton of the Centers for Medicare & Medicaid Services (CMS), Nancy Bridges of the National Institutes of Health (NIH), and Matt Kuehnert of the Centers for Disease Control and Prevention (CDC) are all here in that capacity. This cross-disciplinary approach will help ACOT discern what needs to be done and make recommendations that are likely to bear fruit.

Insofar as the ACOT membership, Dr. Burdick noted that nomination packages are in the works for the new members. The incumbents will continue until the new people come in.

Dr. Burdick briefed the meeting participants on the status of ACOT's first 28 recommendations. He said that we are close to but have not yet garnered the approval of the U.S. Department of Health and Human Services (HHS) for recommendations 29 through 35. He noted that approval is likely a matter of the bureaucratic process, which requires some time. He noted that a conference call at a later date may be the best way to discuss the status of these recommendations. Below are some notes regarding the progress of certain of ACOT's recommendations 1-28:

• Recommendation 1 (ethical principles/informed consent standards): About a year ago, the Organ Procurement and Transplantation Network (OPTN) Ad Hoc Living Donor Committee agreed that ACOT's informed consent document be adopted as a template, as recommended by the Secretary. The document is being circulated for comment. In principle, this recommendation has been accepted.
• Recommendation 2 (independent advocate for living donors): The Secretary has announced his support for this recommendation, and the OPTN Ad Hoc Living Donor Committee agrees that this recommendation is appropriate. However, the type of professional to serve in this capacity should not be stipulated by the committee. This recommendation, then, has been endorsed in principle.
• Recommendation 3 (database for health outcomes of all living donors): This issue is a complex one, according to Dr. Burdick. It has been OPTN's responsibility to collect information early after living donation. The data collection process has been strengthened because the amount of data returned has been unsatisfactory. Progress is being made in regard to having early medical issues identified through the OPTN data collection process for 1-2 years of follow-up for all living donors at all centers. Long-term morbidities or penalties are being addressed by an NIH program getting underway. Its goal is to look in a focused way at the physiology of living donors using a well-studied subgroup.¹ Nevertheless, it is impractical to follow every living donor forever. By using OPTN data for short-term effects and the NIH data for long-term effects, it will be possible to get a good picture of health outcomes for living donors. We need to decide what is reasonable to do.
• Recommendation 4 (resource center for living donors and their families): The Secretary has announced his support for this recommendation.
• Recommendation 5 (extend present OPTN living donor preference for kidney transplants to all organs): The OPTN has considered this and has expressed concerns about whether the policy should be extended beyond kidney allocation preference.
• Recommendation 6 (delete requirements for HLA typing for liver transplants): On January 24, 2003, CMS and CDC published revised CLIA regulations to implement a variant of the ACOT recommendation.
• Recommendation 7 (establish a process to verify qualifications of centers to perform living liver or lung donor transplants): Dr. Burdick stated that some details of this recommendation are still under discussion but that there has been some progress in this direction.
• Recommendation 8 (increasing education and awareness of dialysis patients as to transplant options): CMS has been very involved in this area and is interested in supporting this effort. In addition to overseeing dialysis programs, CMS is having the End-Stage Renal Disease (ESRD) Network look into the availability of transplantation to patients on dialysis.
• Recommendation 9 (eliminating disparities in organ transplant rates): The Secretary has expressed strong support of this recommendation. Many papers have been published on this topic. Within OPTN, there have been studies of impact of minority access and what policies might be desirable. This area remains a work in progress. Dr. Burdick noted that recommendation 34 is related to this recommendation.
• Recommendation 10 (legislative strategies for coroners and medical examiners): Dr. Burdick said that the HHS Organ Donation Breakthrough Collaborative has been a major success. Nested within the Collaborative are important initiatives to eliminate organ refusals by medical examiners and coroners.
• Recommendation 11 (addition of organ and tissue transplantation in curriculum standards for public education): The Secretary, in concert with the U.S. Department of Education, has recommended development of curricula aimed at middle- and high-school students, as well as college students. An organ donation program for high-school students was recently announced by the Secretary.
• Recommendation 12 (best practices for managing potential donors): Dr. Burdick spoke about the successes of the Breakthrough Collaborative, which is in its second year. Teams were drawn from 850 hospitals to identify best practices and then spread the findings. The Collaborative has resulted in major improvements in conversion rates (goal of 75% conversion. Overall donation in the country is up 10%, no doubt attributable to the Collaborative, at least in part.
• Recommendation 13 (as a CMS condition of participation, require that hospitals with more than 100 beds identify an advocate for organ and tissue donation): The Secretary supports the principle that underlies this recommendation. Several leadership organizations—including the American Hospital Association (AHA) and the Joint Commission on Accreditation of Healthcare Organizations (JCAHO)—have been working with HHS to identify such an advocacy process.
• Recommendation 14 (as a CMS condition of participation, require that hospitals establish policies and procedures for donation after cardiac death [DCD]): Hospitals should maximize organ retrievals from DCD. Again, the Collaborative is having an effect. Subsequent recommendations address this issue, too. CMS is reviewing and revising conditions of participation. Dr. Burdick suggested that the ACOT members keep apprised of developments in this regard.
• Recommendation 15 (As a CMS condition of participation, require that hospitals notify the OPO prior to withdrawal of life support, and if patient is a potential donor, OPO shall reimburse hospital for appropriate costs related to extending life support): The Association of Organ Procurement Organizations (AOPO) and OPTN have addressed this issue leading to development of further ACOT recommendations (29 & 30) in November 2003.
• Recommendation 16 (regulatory framework for transplant centers and organ procurement organizations should be based on continuous quality improvement principles): CMS has been interested area in this for some time as has been the Health Resources and Services Administration (HRSA). Developments are coming down the road.
• Recommendation 17 (failure to identify potential donors should be considered a serious medical error): The Breakthrough Collaborative has focused on rewards, not punishment. The process has not involved review and censure. JCAHO has taken this approach to heart. There may be some independent moves within the community, but little action has occurred on this front thus far. Recommendation 29 will address this issue further.
• Recommendation 18 (strengthen JCAHO accreditation provisions regarding organ donation): JCAHO has published a white paper on this topic and participated in the best practices collaborative.
• Recommendation 19 (honoring the donor's wishes): The Secretary supports this recommendation, but this area remains somewhat controversial.
• Recommendation 20 (full implementation of UAGA with regard to donor rights): HHS and HRSA fully support this recommendation. AOPO participated in the National Conference of State Legislators.
• Recommendation 21 (rename and broaden the scope of the present cadaver donor registration form): The OPTN expanded and renamed the what is now called the Deceased Donor Registration Form on September 3, 2003, which completes the implementation of this recommendation.
• Recommendation 22 (encourage use of split livers): The Secretary has supported this recommendation, at least in concept. OPTN has also worked on this issue. These actions indicate that the topic is valuable and worthy of attention. Logical issues have prohibited full implementation of this recommendation, however. We are getting more information on split liver transplantation that may inform further deliberations on this recommendation.
• Recommendation 23 (identify a single reporting mechanism for clinical outcomes data): Although UNOS has a standardized request for information (RFI), many insurance companies continue to request additional information about transplantation outcomes. Work is continuing on this recommendation. An OPTN meeting is bringing together the transplant community and payers in a two-way process.
• Recommendation 24 (issue a request for proposals to elucidate relative risk of graft loss after transplantation): Dr. Burdick said that getting at real clinical issues was the intent behind this recommendation. NIH has taken a lead on this recommendation by planning and implementing programs to investigate basic science issues.
• Recommendation 25 (minimum Model for End-Stage Liver Disease [MELD] score as a lower limit for transplantation): There is concern that sicker patients in some regions are not receiving livers while healthier patients in other areas are getting livers. In addition, patients with low MELD scores have a better chance of remaining alive if they are not transplanted. This recommendation was addressed at a joint conference in 2003 with the OPTN Liver and Intestinal Organ Transplantation Committee. The proposal is still out for comment. Many questions remain and will stimulate more discussion. Nevertheless, this topic has received much positive activity.
• Recommendation 26 (endorsement of Section 105 of Senator Frist's bill [S. 573]): Defining valuable consideration has been the focus of much activity within ACOT. This promising recommendation is a work in progress. Some appropriate action is likely, said Dr. Burdick.
• Recommendation 27 (elimination of 3-year time limit for Medicare coverage for immunosuppressive drugs for kidney transplant patients): The Secretary could take action along these lines as could the U.S. Congress. This recommendation has been heard, and relevant activities are underway.
• Recommendation 28 (elimination of current requirement that patients must have been Medicare-eligible when they underwent organ transplantation in order to receive immunosuppressive drug benefits when they subsequently become Medicare-eligible through age or disability). Dr. Burdick said that there is no specific progress to report at this time, but this recommendation can be considered a work in progress.

Flora Solarz made two comments. First, the intent behind Recommendation 4 was to provide consistent information to all donors across transplant centers and to have a live staff person available who would be independent of the hospital and who could engage potential donors and their families in dialogue and deal with their questions. UNOS has made great strides in putting together information, but there has been no progress in putting a live person in place. Second, Ms. Solarz said that in regard to the database of outcomes for living donors (Recommendation 3) we need to look at the toll that living donation can take on someone who is trying to save a life. There are other important implications besides health outcomes (e.g., psychosocial, financial, implications for medical, disability, and life insurance). If we look only at survival, we are not giving appropriate attention to the spectrum of consequences faced by living donors.

Dr. Ascher responded that the NIH study will address a larger universe of data, including things brought up by Ms. Solarz. The question is: How can these issues be addressed in an affordable way? Dr. Burdick said that some aspects (e.g., insurance implications) might be easier to address than others (e.g., psychosocial consequences). Perhaps we can look at the easier aspects first. He also said that it might be difficult for the government to hire someone to serve as a donor liaison because of the costs involved and other logistical problems.

Robert Gibbons noted that Recommendation 25 netted two proposals by OPTN in regard to (1) regional sharing and (2) minimum listing criteria. These have been passed and implemented.

Kathy Turrisi observed that one problem has been the public perception that ACOT has not been moving fast enough on some agenda items. Some recommendations were made with the intent of moving recommendations and agenda items along more quickly. It seems, though, that we are still bogged down and not getting things in place although ACOT and UNOS have done a great deal of work. We should put some time points in place to show that we are moving along. Dr. Burdick responded that approximately one-third of ACOT's recommendations have had some aspects put in place. Some recommendations have taken some time but will be implemented. In its three and a half years, ACOT members can feel good about the progress achieved.

Another participant noted that Recommendation 28 contains two conflicts. CMS will work to resolve these.

Report from the OPTN Transplant Administrator's Work Group on the Request for Information (RFI) Form

Kathy Turrisi

Ms. Turrisi discussed clinical outcomes reporting. She noted that a standard RFI form is available, but payers often request many other items of information. How can we solve that problem? She outlined some progress that has been made in this area. For example, there will be an annually updated, Web-based version of the RFI form available. Information may then be faxed or mailed to insurance companies. Data from the Scientific Registry of Transplant Recipients (SRTR) can be downloaded.

On July 22, Ms. Turrisi represented ACOT at a meeting in Chicago with insurance administrators and UNOS. At that meeting, the participants asked insurance representatives about the types of information they need. Many payers have addendums to request information other than that on the RFI. Many companies have been asked to forward their addendums, which will be added to the 2006 RFI.

She noted that some insurers commented that SRTR data seem to be outdated. The insurers rely on transplant centers to provide the most recent information. Ms. Turrisi commented that the insurers asked about how they can get more timely data. The insurers want to know what centers are doing to fix their problems. Ms. Turrisi reported that the insurers said they want to see some kind of practice plan within transplant centers to ensure that timely data can be made available by sources other than the centers themselves.

Fritz Port commented that the criticism about outdated data appears to be invalid. Follow-up data must be based on an entire year of data collection. If the insurers just want counts, SRTR can provide up-to-the-minute data. SRTR has uniform data and captures additional follow-up from Social Security master files. Dr. Port said that SRTR's data continue to improve.

Ms. Turrisi said there is room for improvement. Questions in the RFI address other items. Some centers are not even approached by insurance companies. Such centers have taken corrective actions but they are still being punished years later. Some centers provide data that purports to be more recent than SRTR data. Again, this is a work in progress.

Alan Leichtman of SRTR said that SRTR's data are accurate and better than what had been provided by the centers. In terms of timeliness, if we are reporting 1-year follow-up, it requires closure of one year. SRTR is limited by the laws of the calendar. It important that insurers understand the framework that underpins data collection and reporting.

Roger Evans described a scenario in which a center gets in trouble, the center then recruits new staff and tells insurers that the center has improved. The data provided by centers must be considered questionable. He also made the point that insurers compete with each other through networks. There is a great deal of overlap among the networks. With all of the addendum materials, insurers are competing to get the best networks.

Ms. Turrisi reiterated that her focus has been to present the feedback about what insurers say they want. We have made progress because the insurers have indicated that they will look at the RFI addendums. The RFI will be standardized for 2006 and it will be Web-based.

Dr. Ascher asked if insurers think they will be held responsible if suboptimal outcomes occur at centers of excellence. Ms. Turrisi said that, indeed, insurers have expressed that concern.

Proposal to Amend Section 301 of the National Organ Transplant Act (NOTA)

Gail Agrawal

The Valuable Consideration Subcommittee (Subcommittee A) presented recommendations related to two issues: valuable consideration and presumed consent.

Valuable Consideration

Ms. Agrawal recalled discussions at the May ACOT meeting about why it would be desirable to get clarification and greater specificity in regard to the broad and somewhat confusing prohibition of valuable consideration in the context of organ donation. This subcommittee proposed that the Secretary seek some authority to define the prohibition on valuable consideration through amendment of existing regulations to provide for beneficial and ethical means to promote organ donation.

Ms. Agrawal drafted some language for a recommendation, which has been reviewed by HRSA staff:

• The Advisory Committee on Organ Transplantation (ACOT) recommends that the Secretary of Health and Human Services seek authority to identify and exclude certain practices from the definition of "valuable consideration" in section 301(a) of the National Organ Transplant Act, as amended. The Secretary's authority should be limited to legitimate and beneficial practices that are intended to increase the supply of human organs, without creating a commercial market for the purchase or sale of human organs or posing a risk of coercion of a potential donor or donor family. In addition, the Secretary should be required to obtain an appropriate independent ethical evaluation before excluding any practice from the prohibition on valuable consideration.
• ACOT has concluded that a process to limit the scope of "valuable consideration" would encourage the development of ethical practices to increase the supply of human organs and provide certainty to the transplant community about the scope of permissible activities. Regulatory authority is both more flexible and more responsive to innovation than an expanded statutory list of practices that are not included in the term "valuable consideration." The notice and comment period will provide an opportunity for public and professional input into any proposed regulation.
• ACOT, therefore, recommends that Section 301 of the National Organ Transplant Act be amended in its entirety to read as follows:
• (a) Prohibition
  It shall be unlawful for any person to knowingly acquire, receive, or otherwise transfer any human organ for valuable consideration for use in human transplantation if the transfer affects interstate commerce.
• (b) Penalties
  Any person who violates subsection (a) of this section shall be fined not more than $50,000 or imprisoned not more than five years, or both.
• (c) Definitions
  For purposes of subsection (a) of this section:
• (1) The term "human organ" means the human (including fetal) kidney, liver, heart, lung, pancreas, bone marrow, cornea, eye, bone, and skin or any subpart thereof and any other human organ (or any subpart thereof, including that derived from a fetus) specified by the Secretary of Health and Human Services by regulation.
• (2) The term "valuable consideration" does not include the reasonable payments associated with the removal, transportation, implantation, processing, preservation, quality control, and storage of a human organ, the expenses of travel, housing, and lost wages incurred by the donor of a human organ in connection with the donation of the organ, or such other practices that the Secretary of Health and Human Services shall designate by regulation pursuant to subsection (d) of this section.
• (3) The term "interstate commerce" has the meaning prescribed for it by section 321(b) of Title 21.
• (d) The Secretary of Health and Human Services shall propose and promulgate regulations to ensure that interpretations of subsection (c)(2) do not impede legitimate and beneficial practices that are intended to increase the supply of human organs available for transplantation, provided, however, that any practice that poses a risk of coercion in connection with the donation of a human organ or of the creation of a commercial market for the purchase or sale of human organs is not a legitimate or beneficial practice within the meaning of this subsection (d). In considering legitimate and beneficial practices that will be excluded from the prohibition on valuable consideration set forth in subsection (c)(2), the Secretary shall seek an ethical evaluation from an appropriate entity, including without limitation the Institute of Medicine and the President's Council on Bioethics, or such similar or successor entity."

Ms. Agrawal observed that this approach would preclude the creation of a commercial market and it cannot enrich donors. We do not want the rewards to be so great as to generate a coercive effect. We also provided a mechanism for an ethical review of the proposal prior to being put out for the notice and comment period. The effect of this recommendation is to bring the idea of clarifying valuable consideration to the fore. Ultimately, congressional action would be required.

Frank Delmonico applauded this effort because ACOT would be opposing commercial markets in organs for transplantation. Hans Sollinger commented that the recent Denver case plus the fact that some 500,000 people are on dialysis indicate that this measure may not be enough. He commented that a presentation by Janet Radcliffe-Richards in Vienna intrigued him about the idea of having a controlled system operating above the table to monitor what happens. He said that the growing demand for organs in the coming years will likely drive a black market in organs: "We must offer oversight; we cannot just say, don't do it." Dr. Ascher advocated oversight without commercialization. Ms. Agrawal said that the recommendation reflects the opinion of the subcommittee—purchase and sale of organs are not permissible. If we want to consider purchase and sale, then the subcommittee does not want to offer this recommendation.

Bill Harmon asked if this proposed amendment of NOTA would preclude the Wisconsin tax benefit. Ms. Agrawal responded that the Secretary and HHS staff would have to make that determination. In her opinion, that would not be purchase or sale of an organ. Whether it constitutes coercion or purchase/sale would be a question to address. Emily Marcus Levine observed that the Wisconsin law is permissible because it reimburses for donor expenses already allowable under NOTA.

There was some discussion about whether OPOs can help people find matching donors outside the construct of for-profit organizations. The concept of someone being monetarily enriched by donating organs is the problematic area. We need to find an ethical way to bring people together.

Dr. Sollinger said that presently some altruistic individuals are becoming living donors, but the situation is likely to move away from an altruistic approach and to move toward commercial markets in organs, such as those that exist in Pakistan and India. ACOT has heard only one side of this issue and the world does not necessarily agree with the view we are trying to support. Dr. Sollinger suggested that ACOT should invite speakers to present the other side. Several participants suggested moving forward on this recommendation because ACOT has been opposed to pure buying and selling of organs. Nevertheless, ACOT should consider hearing from presenters on the more controversial topic of commercial markets in organs. Dr. Harmon said that this recommendation would allow us to go forward. If we open the question about buying and selling organs today, we will not be able to reach an agreement soon. This step does not prohibit the next step. He noted that some nations (e.g., Kuwait) have reconsidered and eliminated their buy/sell market.

Carlton Young pointed out that we have a limited resource that we are trying to parcel out. We are trying to protect the disenfranchised while trying to implement everything we can do improve organ donation. We must improve the supply side.

Dr. Ascher then moved to insert the word "unregulated" before the word "commercial." Dr. Sollinger seconded the motion. Ms. Agrawal called for further discussion. Ms. Agrawal cautioned that such a modification of the proposal might imply the obverse of the statement's intent. Dr. Delmonico said that if we open the door for a regulated market, then we will be going back to ground zero. Dr. Ascher responded that we do not know where this field is going. Adding the word "unregulated" gives us some modicum of control. Dr. Harmon said he agreed with Dr. Delmonico, that this recommendation will be a lightning rod if accepted. Ms. Turrisi suggested taking out the word "commercial." The word "unregulated" is important. Ms. Solarz said that substituting this language is likely intended to give us flexibility down the road because it takes so much time to get a regulation through the process. Nevertheless, this is a slippery slope. Those who are disenfranchised by our health care system would be exploited. It would be anathema to allow people to sell pieces of themselves, even in a regulated situation. She said she would not back this proposed modification. It pains her to think that our system could encourage selling of organs even if regulated. We must be above that. We have an organ shortage, but we cannot have people risk themselves because they do not have what they need.

Dr. Sollinger said that it is not necessarily true that the poor and disadvantaged will bear the brunt of this (buying/selling of organs). But, that does happen every day. Think of how many of the rich and famous are serving in Iraq. He expressed concern that we are facing an eventual black market in organs. It has happened already in so-called civilized countries. We must be realistic. We have been very unilateral, but we must expand this discussion.

Amadeo Marcos stated that it is the transplant center that has the ultimate control in preventing black markets. Dr. Delmonico said that the American Society for Transplantation has discussed a commercial market in organs, but has expressed its opposition. The Congress considered it via Senator Frist's bill and rejected it. It was rejected by Senator Kennedy's staff as well. Just because a black market might be coming is no justification for creating a regulated market in organs, according to Dr. Delmonico.

Ms. Agrawal called a vote to amend the language in the proposed recommendation by inserting the word "unregulated" before the word "commercial." The vote was 4 for, 9 opposed, 2 abstentions. The motion failed.

Ms. Agrawal called a vote to adopt the recommendation as originally written (and reproduced above). The vote was 15 for, 0 opposed, 1 abstention. The motion passed, and the recommendation was adopted as written.

Presumed Consent

Ms. Agrawal recalled that Phil Berry has previously presented to ACOT on the topic of presumed consent. Moving toward a presumed consent model would have to be done at the State level because the Uniform Anatomical Gift Act (UAGA), which make provisions for consent is a State law, not a Federal law. The subcommittee proposes language for a recommendation to encourage the Secretary to support State initiatives for demonstration or pilot projects for making anatomical gifts under a presumed consent model:

• The Advisory Committee on Organ Transplantation (ACOT) recommends that the Secretary of Health and Human Services encourage States to undertake demonstration projects to test the feasibility of adopting a model of presumed consent to organ donation. The current system for the donation of human organs from deceased donors is based on a default assumption that individuals prefer not to donate their organs after their death. A policy of presumed consent would include as a default assumption that individuals do prefer to donate their organs for transplantation at death. Because of the life-saving potential of transplantation, a presumed consent model would be a moral improvement over the current system, provided individual autonomy is appropriately protected through a system of declining to donate.
• The Advisory Committee on Organ Transplantation, therefore, recommends that the Secretary authorize, encourage, and support State demonstration projects to design and implement "presumed consent" models for making anatomical gifts. The precise design of such models should be left to the states, provided that any State demonstration project authorized by the Secretary shall include provisions to ensure adequate notification and education of the citizens of the State and a method designed to permit any person not wishing to consent to an anatomical gift at death to register a decision to refuse to make an anatomical gift.

Ms. Agrawal envisions that a State could come to HHS with a proposal and bring the design to the Secretary with parameters and safeguards. The Secretary and staff would review the proposal. The object would be to see if these programs might increase the supply of organs.

Dr. Berry moved to approve the recommendation. Dr. Delmonico seconded the motion. Ms. Agrawal opened the floor for further discussion.

Susan Gunderson moved to change the words "consent to an anatomical gift" in the last sentence to "authorize an anatomical gift." Dr. Berry seconded the motion. Ms. Agrawal asked for discussion. She said that under a presumed consent model it is different from an anatomical gift. The default position is non-authorization. Mr. Seely warned that the phrase "presumed consent" has been used for years now. We do not want to muddy the waters or distract from the important issue at hand. Ms. Gunderson suggested changing only the last sentence and not the title of the recommendation.

Mike Seely said that States that have registries in place might be able to see if a presumed consent model would affect donation rates. Dr. Berry mentioned that he celebrated his 18th birthday last week (18 years since his liver transplant). More than 87,000 people are on the waitlist for livers now. We must look at alternatives. It is speculated that presumed consent or presumed authorization might be better, but it is mere speculation until we do it. Any State that has the wherewithal to make it happen should be allowed to do so for several years to be an adequate test to see once and for all whether it makes a difference. If a handful of States undertake such demonstrations and show a positive change in donation rates, then we can say that presumed consent is a viable alternative to what we are doing now. Until we do the studies, we will keep talking about it. It is time to see if it will work.

Ms. Agrawal called for a vote on proposing the recommendation with the following change: In the last sentence, change the words "consent to an anatomical gift" in to "authorize an anatomical gift." The vote was 14 for, 0 opposed, 0 abstentions. The motion passed, and the recommendation was adopted as amended.

Report from HRSA's Independent Expert Panels on Statistical Methods for the Analysis of Organ Transplant Data: Analytic Methods and Simulation Modeling

Gregory Fant

Gregory Fant said that ACOT's role in increasing the public's confidence in the integrity and effectiveness of the national transplantation system requires current and reliable analytic data on which to base decisions. HRSA is committed to ensuring that the analytic results relied upon in the decision-making process are current, correct, and reliable.

To that end, HRSA is charged to enter competitive contracts to provide analytic and simulation-modeling support. The proposals are evaluated according to their technical merit. The contract is presently with SRTR. SRTR works with OPTN committees to better understand the statistical nature of the research questions of particular committees and then employs the most appropriate statistical methods to address research questions. HRSA monitors the SRTR contract, not only to verify the completion of contract deliverables, but also to assess the content of the analysis submitted by SRTR.

As part of the ongoing commitment to ensure that analytic results are current, correct, and reliable, HRSA convened two panels of independent statistical experts to review and assess the analytic methodologies and simulation modeling developed and utilized to serve OPTN and ACOT. The two independent expert panels (IEPs) were held in Chicago from September 19 through 21, 2004. Dr. Fant provided biosketches of the experts and agendas for both sessions.

Both HRSA and SRTR made presentations to the panel members. OPTN also made presentations regarding simulation modeling. The panelists listened to the presentations and asked relevant questions. As part of the process, some preliminary findings were presented to the Scientific Advisory Committee on October 12, 2004. Many of the issues and related questions were highly technical. The panels are still working on their consensus documents, but Dr. Fant presented the highlights of their recommendations. (Dr. Fant will provide the final consensus documents to Mr. Balbier when they become available.)

Insofar as analytic methods are concerned, the IEP indicated that:

• Semiparametric and fully parametric survival analysis techniques may be appropriate for a given research question when used by statistical experts.
• The Cox proportional hazards model (and its extensions), as a semiparametric survival analysis technique, is a standard statistical method that has the flexibility and the advantage of being able to address more than 95% of survival analysis needs that include the consideration of risk factors.
• There is a need to better define such concepts as benefit of transplantation, candidate ranking, and so forth before selecting a statistical method. It is possible that more than one statistical technique may be appropriate.

The highlights from the IEP report on simulation modeling pertain to several different topics:

• Should changes in a patient's state (e.g., died, transplanted, removed from list) be drawn from the histories of existing patients or should they be generated by probabilistic models? The IEP recommended that the disease progression component of a simulation model should try to model the underlying biology of the process—this is the path followed by SRTR.
• How should events post-transplant be generated? The IEP indicated that these events are important. Simultaneously predicting patient and graft survival is crucial. However, the panelists would argue that it is incorrect to specifically model actual post-transplantation/re-transplantation rates in simulation modeling.
• Insofar as providing for and assessing the impacts of changes in OPTN rules, how should national rules be represented? The IEP recommends that any model of the allocation system should accurately represent the actual rules under which the current (or any proposed) system operates. The current SRTR model has a flexible structure and appears to represent faithfully many of the local variances in transplantation allocation.

The IEP offered some overall impressions in regard to analytic methods. First, the panelists said that the statistical approaches used by the SRTR to analyze data are standard and state-of-the-art methods widely used by statisticians. Second, they note that other innovative, cutting-edge methods are also being developed.

The simulation modeling IEP stated that the Simulation Allocation Models (SAMs) developed by the SRTR for liver (LSAM), thoracic-heart/lung (TSAM) and kidney-pancreas (KPSAM) represent appropriate modeling approaches. They appear to be reliable and useful. All three SAMs have undergone various levels of validation and are still evolving, as is to be expected.

Dr. Fant reminded the group that the responsibility for the assurance of the quality of the analysis belongs collectively to ACOT, OPTN, HRSA, and SRTR.

Dr. Gibbons commented that convening a group of statisticians who have not done work in this area has overlooked the problem of highly competing risks. If one tried to add up the probabilities from many SRTR analyses, they total more than 1. The other thing that is so unique about this area that would be missed by the IEPs is that this is a nested problem. Centers are nested within OPOs. Centers and OPOs do things in their own ways. They exhibit different degrees of aggressiveness in transplantation and procurement. The effect of geographic variability may be small or large. Which factors predict OPO variability and center variability? It may be possible to find associations in the data that do not really exist because the variability is understated. SRTR has raised the bar in the analyses from where they started. Dr. Gibbons said that it is critical to educate the independent expert panels about the field and the process to learn what issues are important.

Dr. Fant reminded the group that he was presenting only the highlights of the IEPs' initial findings. He said that the IEPs are indeed addressing the key issues of competing risk and informative censoring. In fact, there were two presentations on these issues at the Chicago meetings. Fritz Port further noted that SRTR has a new method to deal with informative censoring.

Some Additional Topics

James Burdick

Dr. Burdick briefly addressed the dilemma of how to measure the impact of activities related to organ donation awareness and acceptance. This dilemma highlights the successes of the HHS Breakthrough Collaborative. Many activities are underway nationwide to improve donor education and information. Conversion rates are improving even in hospitals that are not participating in the Collaborative, perhaps because of the Spread Initiative. This is an issue that researchers must deal with: isolation of the effects of a given initiative.

On another topic, Dr. Burdick noted that one requirement in the Frist bill was for a study of the ethical impact of efforts to improve organ donation. The Institute of Medicine (IOM) is to carry out a wide-ranging and intensive study. The IOM will be asked to assess interventions so that we could begin to think to the extent about how a given process is making a difference. An example is the presumed consent model that was just voted on. We could find out if it works and then replicate it. A challenge to the IOM is to tell us the impact of what we are doing.

Roger Evans said that many of these same issues arise with quasi-experimental designs. The other problem is that continuous improvement has its own problems (e.g., whether one can sustain the gains). Attributing causality is very difficult. Dr. Evans further noted that the wide-ranging and far-reaching activities associated with the Collaborative complicate the task of making new grants because of the challenges of evaluation.

Friday, November 5, 2004

Update on the HHS Breakthrough Collaborative and the Latest Secretarial Organ Initiative

Dennis Wagner and Virginia McBride

Mr. Balbier welcomed the presenters and introduced Dennis Wagner and Virginia McBride who head up the HHS Breakthrough Collaborative, which has been a tremendous success. The Secretary has recognized their work and recently bestowed the Distinguished Service Award on them, along with Lorah Tidwell and Jade Perdue from the Division of Transplantation. The AOPO also presented Mr. Wagner and Ms. McBride with the Executive Director's Award.

Mr. Wagner explained that a collaborative is an intensive, full-court press to facilitate breakthrough transformations in the performance of organizations based on what already works. The HHS Organ Donation Breakthrough Collaborative is committed to saving or enhancing thousands of lives a year by spreading known best practices to the Nation's largest hospitals to achieve organ donation rates of 75% or higher.

The first collaborative involved 95 hospitals, and 43 OPOs. The second collaborative comprises 131 hospitals, and 50 OPOs, plus 50 or so teams participating via satellite. The participating large hospitals account for approximately 50% of the donor potential in the Nation.

Mr. Wagner then presented some typical results achieved by hospitals participating in the Collaborative. The Washington Regional Transplant Center had 14 donors in October compared to its monthly average of 8. Their record is 18. Their conversion rate is usually in the range of 30% or 40%. Since the September learning session, five of six eligible donors were converted.

In the Midwest Transplant Network, four of the five hospitals that participated in the first Collaborative achieved average conversion rates of 75% or greater. The hospitals in the network increased the number of DCD donors from three in 2003 to 24 in 2004. Hospital team members from the second Collaborative resolved to pursue DCD at the September learning session and had already accomplished two DCDs by October 10.

One of the hospitals affiliated with the University of Wisconsin OPO and T.E.A.M. improved conversion rate from 50% to 77%. The Theda Clark Medical Center improved its conversion rate from 71% to 94%. This hospital achieved the Nation's longest streak of 20 donors before nondonor and is still going. (The number of donors before a nondonor is a measure collected by the Collaborative of consecutive successful donor requests.) The number of DCD donors increased from one or two per year to a total of nine in 2003 and six in 2004. The hospital is adding staff and making some operational changes to accommodate the increased number of donors.

The University of Wisconsin-OPO is on pace to break the record number of donors per year, again. Notable with this team is the involvement of the surgeon community. This appears to contribute to their results. Dr. Sollinger said that Tony D'Alessandro, a transplant surgeon, was appointed to the OPO. He is unusually committed. Much credit goes to him, and the OPO is situated next to the clinic. The members of the OPO see the successful patients walk by every day. They are reminded daily of the good they do. He suggested that other hospitals invite the OPO staff to make rounds, visit the clinic, and so forth in order to see the fruits of their work.

Virginia McBride reviewed some Action Period 3 results and said that the Collaborative's new process measures reflect a new practice and a greater urgency to refer to OPOs timely.

The new practice is making appropriate requests; i.e., the right person, the right time, in the right way. It may be that the request involves one person or several. The requestor may be the OPO, surgeon, social workers, or others. The appropriate request rate in the Collaborative hospitals has gone from about 75% to 90%. Right now it is around 82%.

For the year prior to the Collaborative, nationwide conversion rates hovered around 50%. Since the initiation of the Collaborative, conversion rates have trended upward. In October 2004, conversion rate was 62%. There remains a need, however, to continue to widen the gap between the number of "yes" and "no" responses to the request.

Another goal of the Collaborative is to decrease medical examiner denials. During the last quarter, for the 95 teams in the Collaborative, only three such refusals have occurred. Coroners have been invited to join the Collaborative.

Ms. McBride said that another measure is for hospitals to get to a 75% conversion rate — one month at a time. For the July-September 2003 quarter, only 17% made the 75% goal, but in August 2004, 70% made the goal. For the first time, the Nation is going over 600 donors per month nationally.

She made the point that increasing organ donation is all about the "ones":

• One donor at a time;
• One donor family at a time;
• One transplant candidate at a time; and
• One month at a time.

Mr. Seely commented that in his local area, one of the most remarkable things is that they are measuring things in a tangible way. That information guides the area hospitals and helps them discern where to push and where to put resources.

Dr. Higgins said that one of their administrators came back from the learning session very excited, saying that it had been an almost evangelical experience. He noted that the hospitals in the Collaborative are already committed and high performing. He said that the best practices need to be catalogued and disseminated to every hospital in the country because conversion rates in non-Collaborative hospitals need to be targeted more. That would make a real impact on nationwide conversion rates. Ms. McBride said that 60 hospitals are participating in the Spread Initiative. An ever-widening circle of hospitals is being impacted by the Collaborative. The process and outcome measures are becoming standards of practice.

Dr. Higgins asked if there is a legitimate reason not to donate. In many respects, 100% donation would be ideal, but are there any legitimate reasons why a person should not be a donor? Ms. McBride responded that the Collaborative's goal is to achieve 75%, a reflection that there are legitimate reasons not to donate. The success of the Collaborative is based not on barriers, but on successes. It is a very positive approach. Carlton Young said that these results are great, but what about the hospitals that are not be achieving the set goals? Do they get feedback? Ms. McBride responded that the Collaborative is in partnership with JCAHO and UNOS. Every hospital CEO gets an e-mail stating what the hospital's monthly conversion rate is. The CEOs can compare their performance to others'.

Mr. Seely said that they use the Collaborative's results to quickly realign by tapping into veins of information. The Collaborative provides a context and framework for change that often is not easy. Nevertheless, such changes allow us to transplant more patients and serve the public by getting us organized. Ms. McBride listed six high-leverage changes that can make big changes in conversion rates:

1. Advocate organ donation as a mission.
2. Involve senior leadership to get results.
3. Deploy a self-organizing OPO/hospital team.
4. Practice early referral/rapid response.
5. Learn and master effective requesting.
6. Implement DCD. A full-service OPO offers families a chance to donate after brain death or after cardiac death.

These changes are all necessary for a successful donation program. In successful donation programs, physician and clinical champions are known. A process is in place for conducting real-time death record reviews. The OPO presence is manifest in the hospital, often through an in-house coordinator. Focused change agenda is based on analysis of current hospital data. Team huddles are the norm, during which all the important players meet and talk about how the donation situation will be handled. Clinical triggers are in use. After-action reviews are the norm. Effective requesting is in place and in action. These pieces were all in place after the first Collaborative.

Mr. Seely said that the Collaborative is yielding results. It would be a shame if this effort lost its momentum at the end of the funding cycle. He advocated appropriate funding to keep this going. He suggested the possibility of tracking data from the first Collaborative for 5 years, not just one.

Dr. Marcos applauded the Collaborative's DCD effort. Every hospital should offer DCD as an option, although the practice is not accepted everywhere. He noted, though, that DCD must be accepted by transplant programs. Ms. McBride said that the OPOs must recognize that even if local programs do not use DCD organs, other programs do and the local programs still have an obligation.

Ms. McBride showed a video on mastering effective requesting in culturally diverse communities. It demonstrated the importance of diversity training to learn about differences in grieving processes. Trust is the key. Minority requesting works. The requestor does not necessarily have to look the same as the family does, but the requestor must be aware and sensitive. The teams receive copies of all the videos.

Dennis Wagner said that the teams have learned what works and have implemented those practices. He said that 50% of donor potential resides in 226 hospitals. There are about 6,000 hospitals in the United States. The Collaborative is seeking to increase the donor pool in the country through the Spread Initiative, which is aimed toward non-Collaborative hospitals. The OPOs and hospitals have identified 90 people to be Spread leaders for the country. They are leaders in their donor service areas (DSAs). They meet periodically. A knowledge management system (Web-based) will be used to track implementation.

Dennis Wagner said that the teams have learned what works and have implemented those practices. He said that 50% of donor potential resides in 226 hospitals. There are about 6,000 hospitals in the United States. The Collaborative is seeking to increase the donor pool in the country through the Spread Initiative, which is aimed toward non-Collaborative hospitals. The OPOs and hospitals have identified 90 people to be Spread leaders for the country. They are leaders in their donor service areas (DSAs). They meet periodically. A knowledge management system (Web-based) will be used to track implementation.

He concluded with two final sets of data. During the time period of the Collaborative, participating hospitals have achieved 19% increase in organ donation; nonparticipating hospitals achieved a 7% increase. Not all of these increases are due to the Collaborative, but it is likely that a substantial portion is.

Report on Transplantation Extramural Grant Program

Mary Ganikos

Mary Ganikos (Chief of the Education Branch) invited two grantees to speak about their work. Sheldon Zink of the University of Pennsylvania and Susan Morgan of Rutgers presented. One program is geared toward increasing intent to donate, and the other is aimed at increasing consent rates in hospital. One is community-based, and the other is hospital-based.

Ms. Ganikos said that the Division of Transplantation has three grant programs:

• Social and behavioral interventions to increase organ and issue donation;
• Media-based grass-roots efforts to increase minority solid organ donation; and
• Clinical interventions to increase organ procurement.

The Division requires that grantees utilize a consortium approach. Some beautiful teamwork has developed as a result. The programs must have a scientific design and include a rigorous evaluation component. The Division identifies performance measures that each program must approach. The programs must measure impact on consent (hospital-based programs) or the impact on intent to donate with verifiable family notification (community-based programs).

Ms. Ganikos described two new 2004 projects:

• Evaluation of an early education program to increase live kidney donation (University of Maryland Surgical Associates); and
• The impact of lay health advisors on African Americans' intent to donate (National Kidney Foundation of Michigan).

Each application is reviewed by a technical review panel. The Division offers technical assistance workshops and pre-application workshops. The programs address different settings and populations. Target populations have been general and minority. Diverse topic areas have been proposed and studied. She distributed some project findings via handouts.

Ms. Ganikos reviewed some general lessons learned:

• African Americans are less likely to sign organ donation cards, obtain less knowledge regarding organ and tissue donation, are more distrustful of the medical system, have less social support for organ donation, and are less willing to discuss organ and tissue donation issues with their families.
• Matching requesters (cultural background) with that of the family is associated with improved consent rates (from 33.2% for non-similar to 48.7% for culturally similar requestors).
• African Americans and Latinos generally are more comfortable discussing donation in familiar venues such as churches. They welcome the opportunity to discuss medical issues with medical professionals, which can increase willingness to donate.
• For Latinos, media campaigns and community education are strongly related to improving donation knowledge, attitudes, intent to donate, and family discussions of organ and tissue donation.

In terms of lessons learned from consent (hospital-based) programs, Ms. Ganikos pointed out that:

• Discussions with mothers who have donated a child's organs may enhance the likelihood of consent by newly bereaved parents (72.6% for intervention group versus 58.3% for comparison group).
• Using a presumptive approach works. It is strongly associated with increased consent rates.
• A multi-pronged, hospital-wide intervention can substantially improve organ and tissue referral and donation rates, resulting in a near doubling of organ donors.
• A hospital-wide intervention for DCD is positively associated with staff knowledge about DCD, numbers of hospitals with DCD protocols, and the number of DCD performed.
• Professional actors can be a useful resource in a consent simulation for training hospital and OPO staff.
• In-house coordinators in level 1 trauma centers can be effectively replicated in various areas of the country. This project started under a previous initiative that was very successful. They replicated that study in four different cities, which achieved equally good results. In-house coordinators have been a major contributor to increased donation rates.

Public education studies, according to Ms. Ganikos, have shown that:

• Donation education at the worksite is associated with a significantly greater declaration of intent to donate.
• A donor registry and related public education on deceased and living donation are associated with increased consent and living kidney donation.

Lessons learned from school programs include:

• An educational donor Website is strongly related to high school students' increases in donation knowledge, attitudes, and registry contact.
• After a high school intervention consisting of a classroom and a parent component, an intervention group demonstrated significantly greater increases in family discussions and donation decisions as well as the parents' signing donor cards.

The Division funds three types of programs: pilot studies, replication studies, and roll-out studies. Program results are being publicized through journal articles, grantee publications, the Breakthrough Collaborative, the North American Transplant Coordinators Organization (NATCO) annual meeting, Progress in Transplantation journal, and OPTN communications.

Dr. Ascher asked why no data were available for projects begun in 2001. Ms. Ganikos replied that the three-year projects were funded in late September 2001. They then had to go through the institutional review board process. Data will likely be available in next 4 or 5 months. Most grantees requested no-cost extensions. She did note, however, that one project begun in 2000 is behind schedule.

A Study of the Presumptive Approach to Consent for Organ Donation

Sheldon Zink

Sheldon Zink spoke about the presumptive approach to consent. The premise of the research is that given the opportunity to save a life, most people would do it. Organ donation is the right thing to do. She said that their primary research objective was to examine the effectiveness of the presumptive approach to increase the probability of consent for solid organ donation.

The study was assisted by an external advisory committee, consisting of 11 experts in the field of organ procurement and transplantation, communication, psychology and donor family representatives. In collaboration with a linguistic anthropologist, the committee worked to develop appropriate language for presumptive requests. (Dr. Zink mentioned that she will make available copies of their training manual upon request.)

They worked out a flowchart depicting the consent process, highlighting the points where there are opportunities for presumptive approaches. Dr. Zink outlined the major components of the presumptive approach:

First is the intention to be presumptive. The requester should not focus on the family's possible responses. The requester should serve as the voice of the recipient and guide the family toward the goal of donation. It is important to use open-ended questions to keep the conversation going and obtain a series of "small" yeses throughout the family discussion.

Second, the presumptive introduction represents a subtle but important shift for the family. The idea is to present organ donation as a normal step for families.

Third, the presumptive transition elicits empathy for potential recipients and helps the families focus on the good that could come from their tragedy. It pairs loss with gain, death with life, tragedy with heroism. The transition helps the family move from death to a positive look forward to a hopeful future.

Fourth, the transplant coordinator's role shifts to one of an advocate for both the recipients and the donor families. This helps the family to see the decision as the right thing to do by allowing them to understand the great benefits of donation. Dr. Zink mentioned that mentioning potential recipients to donor families had been considered taboo because of possible guilt feelings that may arise if donation is denied.

Fifth, presumptive phrases serve as strong affirmations of donation and help the family be confident in their decision to donate. The requester should use phrases such as "when you donate" (instead of "if you donate").

Sixth, the presumptive ask is an affirmative movement toward donation, open-ended, and focuses on moving forward in the consent process. It also reduces the stress on the family in their time of grief. The requester uses language like "Are there any final questions you would like me to answer before we continue?" This step is a trigger to start the psychosocial paperwork.

Dr. Zink described their research approach used to gauge the effectiveness of the presumptive approach. She encouraged ACOT to develop standard definitions for such terms as "donor potential," "consent rate," and "eligible donor." She noted that data collection will end in December 2004 and then showed some of their preliminary data. Among their findings:

• Mentioning potential recipients (in a general way) increased consent rates.
• Approaching the family before brain death declaration increased consent rates.
• Mentioning organ donation prior to the arrival of the transplant coordinator increased consent rates.
• Knowing the donor's wishes to donate increased consent rates.

Dr. Zink concluded by saying that the best practices initiative is working, but the idea of solid research is critical. We need to identify best practices for the OPOs. We need to sort out the Hawthorne effect, and we need transparency of data. She noted that the Collaborative's data and AOPO data are not made available.

Public Education through Worksite Interventions

Susan Morgan

Susan Morgan said that some people feel qualified altruism; they want the recipient to be deserving of a donated organ. She is working on the public education end. Their studies are funded through the Division of Transplantation. Each project builds on the findings of the project before. Dr. Morgan mentioned studies of three worksite campaigns (quasi-experimental design):

• United Parcel Service;
• University Worksite Organ Donation Project; and
• New Jersey Workplace Partnership for Life.

For worksite campaigns, Dr. Morgan said that they are developing principles and procedures for constructing effective worksite campaigns, which include the following:

1. Specifically target barriers to donation and family communication.
2. Feature employees who have been touched by organ donation and can be resources for worksite programs.
3. Use materials in prepackaged kits (instructions, sample ads, etc.).
4. Use an organizational checklist to help OPOs make informed decisions about how best to use scarce resources.

Dr. Morgan highlighted some findings of their studies. Theory-driven campaigns are effective in improving knowledge, attitudes, and behaviors relevant to organ donation. Nevertheless, there are previously unexamined factors that are barriers to the willingness to donate. One of the most important barriers, identified during studies of family communication about donation, is the mass media. (Several publications describing these studies and their findings have appeared in national journals and presented at conferences, garnering two Top Paper awards.) She suggested that these findings may be of interest to ACOT.

There is much published literature on donation attitudes. Dr. Morgan said that they have learned from qualitative investigations of how families communicate about organ donation that medical mistr