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Statement on H.R. 2418- Organ Procurement and Transplantation Network Amendments of 1999 by William F. Raub, PH.D.
Deputy Assistant Secretary for Science Policy
Office of the Assistant Secretary for Planning and Evaluation
Office of the Secretary
U.S. Department of Health and Human Services

Before the House Committee on Commerce, Subcommittee on Health and Environemnt
September 22, 1999

Good afternoon, Chairman Bilirakis, Congressman Brown, and other members of the Subcommittee. I am William Raub, Deputy Assistant Secretary for Science Policy at the U.S. Department of Health and Human Services. I appreciate your invitation to be here today to discuss H.R. 2418, a bill to reauthorize the National Organ Transplant Act (NOTA). I also will testify in general about national organ transplantation policies and the constructive discussions we have had with our colleagues in the transplant community about those policies.

Thanks in large part to the passage of NOTA in 1984, the transplant network is very different from the system that existed then. Organ transplantation is no longer experimental; it is routine. The figurative handful of centers comprising the network in 1984 has grown into an enterprise of 891 organ-specific transplant programs. Technological advances allow excised organs to last much longer and to be transported much farther than they could fifteen years ago.

In many respects, NOTA and the transplant network it spawned have served patients well. We have an organized national system requiring a structured approach to organ transplantation that did not previously exist. Allocation of scarce organs is done more cooperatively than in the past. Advances in drugs for immunosuppression and techniques for lengthening cold ischemic time have resulted in high survival rates for transplant patients.

Many organizations and individuals deserve high praise for the achievements of the Organ Procurement and Transplantation Network (OPTN): the United Network for Organ Sharing (UNOS); surgeons, physicians, nurses, and other health professionals who have committed themselves to the practice of transplantation medicine; laboratory and clinical scientists who continue to generate new knowledge and technology that drives transplantation medicine to ever-greater success; staff of the Organ Procurement Organizations (OPOs), who work so diligently to acquire and transport organs to transplant programs; patients, who provide invaluable insights as to how OPTN processes might be improved; and, most of all, organ donors and their loved ones, whose decisions to share the gift of life enable transplantation medicine to flourish.

Their efforts have made organ transplantation available to more and more chronically ill patients. Last year more than 21,000 Americans received organ transplants. But more than 4,000 people on transplant waiting lists died because of the scarcity of donated organs. As these lists grow, many more will die as the system continues to strain under the demand for organs.

We in the Administration believe that our first priority must be to increase organ donation. To that end, in December 1997, the Department launched its National Organ and Tissue Donation Initiative to foster partnerships between public- and private-sector organizations to enhance public education about the need for donation and to recruit potential donors. The centerpieces of this initiative are a rule, modeled upon legislation in Pennsylvania, requiring hospitals participating in Medicare to expand their reporting of imminent deaths and deaths to OPOs and a new $5 million grant program to learn more about what works in organ donation. We are pleased to report that organ donation increased by more than 5 percent last year as a result of our collective efforts, and we are hopeful that the upward trend will continue. Nevertheless, we recognize that the need for transplantation is growing faster than the supply of organs and that continued emphasis on both organ donation and equitable organ allocation is necessary.

A second priority for the Administration is to ensure that the OPTN established by NOTA works equitably and provides the best possible outcomes for patients. The role of the federal government in this area was recently affirmed by the Institute of Medicine (IOM), which was directed by Congress last year to study the national organ transplantation network. Two months ago, IOM reported that:

The federal government, as well as the transplantation community, has a legitimate and appropriate role to play in ensuring that the organ procurement and transplantation system serves the public interest, especially the needs and concerns of patients, donors, and families affected by it. The [IOM] learned of numerous instances in which weak governance tends to undermine the effectiveness of the system...Weak oversight has compromised accountability at all levels, permitted poor procedures for data collection and analysis to persist, and allowed the system to operate without adequate assessment of performance.

As I said, the OPTN should be commended for its wonderful accomplishments. Nevertheless, we cannot ignore the persistent flaws and unfairness in the system. The most medically urgent patients do not always receive priority. Patients with similar levels of disease may have different outcomes, depending on where they live or list. Distrust among transplant surgeons and hospital Administrators sometimes impedes broader sharing of organs. True measures of equity to judge the OPTN do not exist.

In recognition of the need for public-sector oversight of the OPTN and in response to provisions of the Consolidated Omnibus and Emergency Supplemental Appropriations Act of 1999, the Department has increased its efforts to assess the performance of transplant programs. With the assistance of staff from UNOS, the contractor for both the OPTN and the Scientific Registry, Department staff analyzed OPTN patient outcome data for liver and heart transplants with respect to three critical issues:

the likelihood that, having been listed as a transplant candidate, a patient will receive a organ within one year;

the likelihood that a patient will die within one year of listing while awaiting transplantation; and

the likelihood that a patient will still be alive one year after listing, irrespective of whether he or she underwent a transplant procedure.

After risk adjustment (i.e., adjustment for differences in the mix of patients= health status from program to program), the analyses revealed substantial differences in outcomes from one transplant program to another. The principal findings for liver transplants illustrate this:

ten percent of the programs have a risk-adjusted rate of transplantation within one year of listing of 71 percent or more; whereas, for another ten percent of the programs, the rate is 25 percent or less;

the likelihood of dying within one year of listing while awaiting a transplant ranges from less than 8 percent to more than 22 percent; and

the likelihood of surviving one year after listing as a transplant candidate or a recipient ranges from approximately 65 percent to almost 86 percent.

The analogous values for heart transplants are 36-72 percent (transplantation within one year of listing), 9-23 percent (death within one year of listing while awaiting a transplant), and 67-84 percent (survival for one year after listing irrespective of whether transplanted or not).

In the course of performing these analyses, Department staff identified gaps in the data currently collected by the Scientific Registry - e.g., additional clinical details about patients= conditions at the time of listing (which could improve risk adjustment) and additional data on clinical complications (which could help in assessing quality of life following transplantation). The Department intends to encourage UNOS, in its management of the OPTN and its operation of the Scientific Registry, to broaden the scope of data collection and make increased use of program-specific performance analyses.

Although these findings warrant further study to determine the precise reasons for such variances in patient outcomes, one thing is clear. Where a patient lives or lists often does more to determine whether he or she gets a transplant than do medical considerations. Patients in like circumstances are being treated differently, in clear contradiction of the premise of NOTA that we have an equitable national system of organ transplantation in the United States .

Many of the flaws that I have discussed are addressed in the final rule for the OPTN that was issued by the Department in April, 1998, after extensive public comment and three days of public hearings. The rule requires the OPTN to develop policies that will result in standard listing practices and medically based definitions of patient status categories. The rule also requires policies that will encourage broader geographic sharing of organs so that the most medically urgent patients receive transplants, based on the sound medical judgment of physicians under this general rubric. The rule leaves the policy decisions to the OPTN with oversight by the Department.

In reference to the final rule, I again quote from the IOM report:

Vigilant and conscientious oversight and review of programs and policies are critically important to ensuring accountability on the part of the OPTN and other participants in the organ procurement and transplantation system. The Final Rule appropriately places this responsibility with the federal government. The [IOM] believes that this is an important aspect of the Final Rule and charge that should be pursued by the federal government in close cooperation with the full range of participants in the transplant community.

The Department clearly concurs with the IOM. There should be vigilant government oversight and close cooperation with the transplant community. This is the balance we sought in the HHS rule.

Mr. Chairman, we have taken very seriously the charge we were given by the Congress last year to work collaboratively with the transplant community to clarify the intent and effect of the rule. We very much appreciate the many hours that those in the community have spent meeting with us and their constructive approach in identifying apparent problems and potential solutions through oral and written comments. In addition, we have carefully reviewed the IOM report and recommendations and have met with representatives of the IOM Committee on two separate occasions. As I have indicated, we are also fortunate to have additional data pursuant to the study provisions in the omnibus bill.

In response to these helpful comments and discussions, we have committed to revise the rule in important areas. For example, we intend to clarify the rule so there is no doubt that the OPTN will develop allocation policies. We intend to ease any lingering concerns of the transplant community about the Secretary=s regulatory authority by instituting an independent advisory Committee B as recommended by IOM B to review major differences between the Secretary and OPTN on policy matters. Other areas of potential clarification or revision have been discussed in detail with transplant community representatives. I believe our revisions will address the major concerns we have heard from the transplant community yet maintain the essential framework of the rule requiring standardization of certain practices and encouraging broader geographic sharing of organs.

Consistent with the law, it is our intent that the rule, once revised, go forward to address inequities in the system identified by the Department and IOM. However, congressional review of the OPTN is essential, and the Department looks forward to working together with you, Mr. Chairman, and other members of Congress to develop legislation to reauthorize NOTA. Reauthorization should primarily address the needs of patients and also maintain the requirement of NOTA that there be a national, equitable system of organ transplantation in the United States . We recommend that reauthorization reinforce the role of the federal government in overseeing the OPTN, in accordance with the IOM recommendations, and that the statute continue to leave management and policy development of the OPTN in the private sector.

The Department is concerned that H.R. 2418 does not do enough to preserve and strengthen many of the attributes of the OPTN that have placed transplantation firmly within the medical mainstream. Indeed, in some instances, the bill takes a step backward. The Department, therefore, opposes H.R. 2418 in its current form. The following paragraphs describe our principal concerns.

  • While calling for a national system to match organs and individuals who need organ transplants, the bill does nothing to decrease the reliance on arbitrary geographic boundaries and the inequities that result. Commenting specifically on allocation of livers, IOM concluded that Athe fairness of the organ procurement and transplantation system, and its effectiveness in meeting its stated goals, would be significantly enhanced if the allocation of scarce donated livers were done over larger populations than is now the case.@ Medical outcomes, waiting time and the possibility of fatality should not depend on the geographic location of a transplant program at which a patient is wait-listed.
  • H.R. 2418 does not ensure that patients and referring physicians can obtain the kinds of program-specific information they need to make decisions about whether, when, and where to seek transplantation. Although H.R. 2418 provides for program-specific data on such items as the probability of receiving an organ transplant, the waiting time for similarly situated patients, and the medical outcomes for similarly situated patients, the bill does not specify that this information also be timely and easy to use. Out-of-date information can lead referring physicians and patients to make less than optimal decisions.
  • H.R. 2418 would erode the role of the federal government in providing oversight of the OPTN. The OPTN, by its structure, is not able to incorporate into its activities the public policy considerations that underpin the NOTA. The Department believes that it must continue to be an active partner with the private-sector in striving to fulfill the goals of the OPTN. Indeed, the overarching purpose of the OPTN rule is to clarify the nature and extent of oversight by the Department while ensuring a continued prominent role for transplantation professionals, patients / patient-advocates, and other organizations and individuals in the private sector.
  • As you know, under the U.S. Constitution, a private entity cannot perform functions inherent to the federal government. Yet the bill does not stipulate that binding policies for the OPTN be approved by the federal government.
  • Although H.R. 2418 requires that the Board of the OPTN have physician representation of no less than 50 percent, it makes no such percentage requirements for patients and donor families, who we believe should have a significant representation on the Board.

In summation, Mr. Chairman and members of the Subcommittee, the Department is committed to working with you and the members of the transplant community to create policies that improve the quality of care and the equity of our organ allocation system. While we have had our differences, our recent discussions with the transplant community have been quite promising. We hope that this spirit of cooperation can extend to our discussions on reauthorization of NOTA as well. Thank you for the opportunity to testify today.

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