Transplant Statistics: Annual Report : Appendix C

LEGISLATIVE ORIGINS - A NATIONAL NETWORK AND REGISTRY

By the early 1980s, organ transplantation had brought renewed hope to thousands of people suffering from diseases of the heart, liver, kidneys, lungs, and other organs. Nevertheless, many patients were having difficulty obtaining transplants, leading some to seek assistance through media coverage. As issues drew congressional attention in 1983 and 1984, it became clear that a more comprehensive approach to transplantation was necessary. In October 1984, Congress passed PL 98-507, known as the National Organ Transplant Act (NOTA). Among other things, NOTA provided for the establishment of a Task Force on Organ Procurement and Transplantation, an Organ Procurement and Transplantation Network, and a scientific registry of the recipients of organ transplants.

As required by law, the Secretary of Health And Human Services assembled a 25-member Task Force to conduct comprehensive studies of the medical, legal, ethical, economic, and social issues relevant to human organ transplantation. Experts in medicine, immunology, law, theology, ethics, allied health, the health insurance industry, and public advocacy were joined by representatives of the Office of the Surgeon General of the Public Health Service, the National Institutes of Health, the Food and Drug Administration, the former Health Care Financing Administration (now the Centers for Medicare and Medicaid Services), and staff from the Health Resources and Services Administration (HRSA).

Upon completion of the Task Force report, in 1986 HRSA issued a request for proposals to establish a National Organ Procurement and Transplantation Network (OPTN) under NOTA. On September 30, 1986, the initial contract to establish and operate the OPTN was awarded to the United Network for Organ Sharing (UNOS). Legislation in the same year was enacted mandating that, as a condition of participation in the federal Medicare/Medicaid programs, organ procurement organizations (OPOs) and transplant hospitals must be members of the newly established OPTN (Omnibus Budget Reconciliation Act, PL 99-509). In 1987HRSA issued an additional request for proposals - to establish and maintain a Scientific Registry of Transplant Recipients (SRTR) to support the ongoing evaluation of the scientific and clinical status of solid organ transplantation in the United States. The SRTR contract was awarded to UNOS on September 30, 1987.

Provisions of NOTA were amended in 1988 and 1990, including expansion of OPTN responsibilities to increase the donor organ supply and assist OPOs in nationwide placement of organs not placed locally. Both the OPTN and SRTR contracts were competitively awarded with UNOS without interruption through December 1996 with a period of performance ending September 30, 2000. In September 2000, UNOS was again awarded the OPTN contract, including all data collection activities; the SRTR contract was awarded to University Renal Research and Education Association (URREA), in collaboration with the University of Michigan. The OPTN and SRTR, now operated by two different organizations, collaborate and share data to support important policy and clinical issues in transplantation.

FEDERAL OVERSIGHT

The Division of Transplantation (DoT), a division of HRSA within the United States Department of Health and Human Services (HHS), administers the OPTN and SRTR contracts and provides federal oversight to both programs. Under the terms of each contract, UNOS and URREA conduct numerous tasks and projects to meet the federally established goals of the OPTN and the SRTR, respectively . Written reports on the progress and completion of these tasks are provided regularly to the HRSA Project Officer and the Director of the DoT. Additionally, representatives of the DoT serve as ex officio, nonvoting members of the OPTN/UNOS Board of Directors and committees, and the SRTR Scientific Advisory Committee (SAC) and its subcommittees.

On April 2, 1998, HHS published in the Federal Register a final rule for operation of the OPTN, with opportunity for public comment. This rule, as amended on July 1, 1998; October 20, 1999; and December 21, 1999, became effective on March 16, 2000. It establishes requirements and procedures for operation of the OPTN, including membership in the OPTN, listing transplant candidates on a nationwide computer network, allocating organs, and maintaining records and reporting by member OPOs and transplant hospitals. Under the terms of the final rule, policies and bylaws developed through the OPTN/UNOS committees and Board of Directors are subject to additional review and oversight by the Secretary, allowing requirements of OPTN members that will be enforceable under the Social Security Act.

PURPOSE AND REQUIREMENTS

The OPTN and the OPTN Database

Since its inception, the OPTN's purpose has been to improve the effectiveness of the nation's organ procurement, distribution, and transplantation system by addressing issues regarding the availability of, and access to, donor organs for patients with end-stage organ failure.

The OPTN database is a fundamental element of the OPTN and its purposes. Specific goals articulated in the Task Force on Organ Transplantation recommendations, the NOTA, and the HRSA contract include:

• To collect, in a computer system, data on all transplant recipients, transplant programs,organ donors, and organ procurement organizations in the United States
• To provide a database that enables regular periodic analysis and reporting on the efficacy of transplantation nationwide
• To provide a national database for basic and clinical research on organ transplantation

The OPTN database consists of data about donors, waiting list patients, and information pertinent to organ allocation, as well as posttransplant recipient data, from the time of transplant until graft failure or recipient death, whichever comes first. Using OPTN organ-specific data collection forms, transplant candidate and recipient data are collected, processed, and validated. Validated data are entered into the OPTN database. Data collected include comprehensive medical and histocompatibility information about donors, registrants, and recipients, as well as graft and patient survival information. (See Appendix I for detailed information on the OPTN data collection system.)

Subject to disclosure restrictions under various statutory and regulatory policies, data collected and generated by the OPTN are available to members of the OPTN, the Board of Directors and committees serving the OPTN, UNOS and URREA staff, the medical community, government officials, private organizations, and the general public. The data have been used in a variety of ways, perhaps the most important of which is to serve as the basis for the development of transplantation policies. With the aid of accurate and timely information about previous transplants and their outcomes, the transplant community can identify specific factors that balance the number of transplants, graft survival, time to transplant, and other measures of both medical utility and justice in organ allocation. (See Appendix H for specific issues being addressed with the aid of OPTN data.)

Specific tasks required by the OPTN contract include:

• Elect a Board of Directors composed of representatives from the professional transplant community; transplant candidates, recipients, organ donors, and family members; voluntary health organizations; and public members
• Establish and maintain a committee system with diverse representation to develop OPTN policy
• Operate and maintain a national computer list of patients waiting for kidney, pancreas, kidney-pancreas, liver, intestine, heart, lung, and heart-lung organ transplants
• Develop and maintain a computer-based organ allocation system and an Organ Center that allow 24-hour transplant program access to the donor/recipient matching system
• Collect data as required and necessary to operate the computer-based matching system
• Maintain a data system that serves as a resource for the Government and other interested parties, with information about patients waiting for transplants, donors and recipients of donated organs, donor/recipient matching and organ allocation, and donor/recipient histocompatibility. The system contains medical and demographic data about every transplant recipient transplanted since October 1, 1987, from time of transplant until time of graft failure or patient death, whichever comes first
• Work with the SRTR contractor to prepare an annual report
• Work with the SRTR contractor to develop and manage the Data Working Group, which makes recommendations on data collection processes and new data to be collected by the OPTN

The SRTR

The SRTR exists to support ongoing evaluation of the scientific and clinical status of solid organ transplantation in the United States. The SRTR is responsible for conducting inferential statistical analyses of transplantation data in support of the OPTN, HHS, the Secretary's Advisory Committee on Organ Transplantation (ACOT), and other interested parties. (See Appendix H for current research activities of the SRTR.) The results of this research serve to:

• Support the OPTN in developing policies guiding organ allocation and other issues
• Support the performance analysis of transplant programs, OPOs, and other OPTN members
• Provide useful information about transplantation for a range of decision-makers, including medical professionals, transplant candidates, transplant recipients, organ donors, donor families, and the general public
• Support investigative efforts by payers, researchers, and other interested parties
• Support the development of computer simulation (modeling) of alternative organ allocation systems
• Specific tasks required by the SRTR contract include:
• Produce an Internet-based report of institution-specific outcomes data for every transplant center and organ procurement organization semiannually
• Prepare a biennial report to Congress on the scientific and clinical status of organ transplantation in the United States
• Establish and maintain the Scientific Advisory Committee (SAC) and the Post-Transplant Tumor Registry Technical Advisory Committee (PTTRTAC)
• Respond to requests for data from committees serving the OPTN, HRSA, and the general public
• Perform special studies as requested by HRSA
• Work with the OPTN contractor to prepare an annual report
• Work with the OPTN contractor to develop and manage the Data Working Group, which makes recommendations on data collection processes and new data to be collected by the OPTN

STRUCTURE

The OPTN and SRTR serve complimentary roles in a national system of organ procurement, transplantation, and evaluation.

UNOS and the OPTN Organization

In fulfilling its purposes as previously described, the OPTN includes an intricate network of members, committees, Board of Directors, regional administration, and internal administration that works together to develop policies and membership criteria through consensus-based processes.

Board of Directors. To conform to OPTN contract requirements, the OPTN elects annually new members to the Board of Directors, which includes representatives from groups of OPTN members and the public at large. The Board is composed of members representing each of the OPTN/UNOS regions (one regional councillor per region), and representatives from the following OPTN categories: Patient and Donor Families, Thoracic Organ Transplantation, Histocompatibility Laboratories, Organ Procurement Organizations, Transplant Coordinators, Voluntary Health Organizations, Medical/Scientific Organizations, Minority Transplantation, and General Public Members. Also included on the Board are members of the transplant community serving as President, Immediate Past President, Vice President/President Elect, Vice President of Patient and Donor Affairs, Treasurer, and Secretary. Representatives of HRSA also serve as ex officio, nonvoting members of the Board as well. The summary composition of the 2002-2003 Board is shown in Appendix Table C-1 and a complete listing is provided as well, along with the 2001-2002 Board.

Membership. In carrying out its functions, the OPTN has established membership criteria and medical criteria for allocating organs and a process for providing an opportunity for members of the public and other interested persons to comment with respect to these criteria. The OPTN admits and retains as members all organ procurement organizations, hospitals, and histocompatibility laboratories that apply for membership, agree to comply with OPTN rules and requirements, and meet certain other criteria.Pursuant to its contract with HRSA, the OPTN Board of Directors has adopted bylaws and policies developed with the aid of organ transplant professionals and patient representatives. These bylaws and policies have been submitted to the Secretary of HHS for review and are considered voluntary guidance to OPTN members until approved as OPTN rules and requirements by the Secretary of HHS. As the OPTN contractor, UNOS is responsible for keeping these bylaws and policies current and for monitoring compliance by OPTN members.

OPTN members fall into one of two categories, institutional members and public members. Institutional members include transplant centers, OPOs, histocompatibility laboratories, and consortia. Public members include the following: 1) private, nonprofit voluntary health organizations that promote organ donation nationwide or serve the interests of transplant patients and their families; 2) private, nonprofit medical or scientific membership organizations with involvement in transplantation; and 3) individuals representing the general public. General public members commonly represent such fields as theology, ethics, health care financing, social and behavioral sciences, patients, patient advocates, and donor families.

The composition of the OPTN membership as of August 2002 is displayed in Appendix Table C-2. OPTN member transplant programs are enumerated according to type in Appendix Table C-3. (See Appendix D for a complete list of all members by institutional type.)

For administrative purposes, the country is divided into 11 geographic regions. Each region is assigned a UNOS staff administrator to assist in coordinating regional activities. Additionally, each region is represented on the Board of Directors and on each of the OPTN's permanent standing committees. The current regional devisions are depicted in Figure III-2.

Policy Development Process. It is through committees, constituted to ensure geographic, individual, and organizational representation, that the OPTN Board of Directors formulates policies for approval. OPTN committees may develop policy recommendations on their own initiative or as directed by the Board or other constituencies or individuals. The Board considers policy proposals on all issues affecting transplantation. Each proposal is accompanied by a description of the underlying rationale, including a summary of medical, ethical, and scientific evidence upon which the proposed policy is based. The Board may approve, disapprove, or amend the proposed policy statement or return it to committee for further consideration.

OPTN members and the public at large are given an opportunity to contribute to the policy development process. Policy statements developed by committees are distributed to OPTN members, the Federal Government, and other interested individuals who have requested to receive the documents for comment before they are submitted to the Board for approval. They also may be made available to the general public via media coverage, public hearings, and the OPTN website. Following a public comment period (generally 45 days), the committee reviews and amends the policy, as it deems necessary, and then submits the final policyproposal to the Board of Directors for deliberation and a decision.

OPTN Committees

As of August 1, 2002, the OPTN had 16 standing committees (per Bylaw Article VI), one joint working group with the SRTR contractor, and two ad hoc committees. Committee members are recommended by the regional councillors and/or selected to provide broad expertise for committee activities. Committees receive input from regional subcommittees, the transplant community, and the public. The 2002-2003 OPTN standing, joint, and ad hoc committees are listed below. For a description of each committee's charge and scope of activities, go to the OPTN web site at www.optn.org, click on members, then committees. (See Appendix H for descriptions of recent research interests and activity.)

1. Communications

2. Data Advisory

3. Ethics

4. Executive

5. Finance

6. Histocompatibility

7. Kidney/Pancreas Organ Transplantation

8. Liver/Intestinal Organ Transplantation

9. Membership and Professional Standards

10. Minority Affairs

11. Organ Availability

12. Organ Procurement Organization

13. Patient Affairs

14. Pediatric Transplantation

15. Thoracic Organ Transplantation

16. Transplant Administrators

17. Joint OPTN/SRTR Data Working Group

18. Ad Hoc International Relations

19. Ad Hoc Living Donor

URREA AND THE SRTR

The SRTR is operated by the University Renal Research and Education Association (URREA), a not-for-profit research foundation specializing in medical and health outcomes research, in collaboration with the University of Michigan. The principal investigators from the SRTR include prominent experts in the field of transplantation, biostatistics, epidemiology, and health-outcomes research. The research conducted by the SRTR is guided by the SRTR Scientific Advisory Committee (SAC), which has been established to advise the SRTR, the Data Working Group (DWG), the OPTN/UNOS Board of Directors, the various OPTN/UNOS committees, and the HHS including ACOT.

SAC Mission Statement. The SAC shall maintain an analytic framework and the data resources necessary to support the activities of various communities and organizations actively involved in the field of transplantation. The SAC shall be actively involved in the development of research agendas that contribute to the science and practice of treating patients with end-stage organ failure. The SAC shall ensure that the SRTR provides the informational and analytical resources necessary to assist the United States solid-organ transplant community to develop both clinical and health care policies and monitor outcomes of such policies. Additionally, in its advisory role, the Committee shall be responsible for upholding ideals that promote the fair and efficient utilization of the scarce resources devoted to transplantation and recommend database interfaces with other appropriate organizations. Finally, the SAC shall evaluate the quality, accuracy, and completeness of procurement and transplant data and ensure that the SRTR makes these data available to individuals and organizations that demonstrate legitimate goals for studying the effectiveness and efficiency of solid organ transplantation in the United States.

Annual Goals of the SAC.

The overall responsibilities of the SAC include the following

• Guide and review research activities of the SRTR, including responses to OPTN committee requests
• Review publications prepared individually by the SRTR or jointly by the SRTR and the OPTN
• Oversee coordination of the OPTN/SRTR Data Working Group (DWG)
• Provide scientific guidance and support to both the OPTN and the HHS, including the ACOT
• For the second year of the SRTR contract, the SAC has set the following goals:

• Provide counsel to the SRTR contractor on the objectives, approach, methods and format for various research projects, including the following:

  1. Program-specific reports for individual OPOs and transplant programs
  2. The 2002 OPTN/UNOS Annual Report, published in conjunction with the OPTN contractor
  3. The 2003 Biennial Report to Congress on the Scientific and Clinical Status of Organ Transplantation
  4. Research reports and articles submitted to peer-reviewed journals
  5. Work conducted in support of the OPTN and the ACOT
  6. Organ allocation modeling activities
• Advise the SRTR contractor on objectives, approach, methods, and format for both ongoing research and new areas of inquiry, thus helping determine the SRTR research agenda
• Advise the SRTR contractor regarding the release of data to outside organizations; grant approval for such release as appropriate
• Advise the DWG on the clinical and scientific data needed to support the functions of the SRTR and the OPTN, including data about morbidity and functional impairment in transplant candidates and recipients and the cost and/or resource utilization associated with transplantation
• Advise the OPTN, including its board of directors, committees, subcommittees, and working groups; report as a committee to the OPTN Board of Directors
• Meet in full four times a year, with at least one meeting in the Washington, D.C., metropolitan area

In addition, the PTTRTAC, as a subcommittee of the SAC, will advise the SRTR and OPTN contractors on scientific and medical issues concerning posttransplant tumors. Particular emphasis will be given to collection and reporting of posttransplant tumor incidence data collected by the OPTN contractor.

SAC Composition. As mandated by HRSA, the SAC is composed of "representatives of the kidney, liver, heart, heart-lung, intestine, and pancreas transplant communities with qualifications in the areas of clinical, statistical, and/or epidemiologic research related to transplantation, multi-center data collection, quality control and utilization, and biostatistics." The SAC must also include representatives from relevant OPTN/UNOS committees, especially those that are organ-specific. A representative of the OPTN contractor serves as an ex officio nonvoting member of the SAC; other ex officio nonvoting members include the SRTR contract project officer, the director of the Division of Transplantation, and representatives from other government agencies. Other members of the SAC include patients, economists, and OPO professionals. The SAC is organized with a chairperson and vice chairperson; the vice chairperson succeeds the chairperson.

The SAC membership is listed below.

John P. Roberts, MD - Chairman

University of California San Francisco

San Francisco, CA

Dale Distant, MD - Vice Chairman

State University of New York Health Science Center

Brooklyn, NY

William M. Bennett, MD

Northwest Renal Clinic

Portland, OR

William R. Coleman

Brooklyn, NY

Suzanne L. Conrad, RN, MS, CPTC

Iowa Donor Network

Iowa City, IA

Harold I. Feldman, MD, MSCE

University of Pennsylvania School of Medicine

Philadelphia, PA

Daniel S. Gaylin, MPA

National Opinion Research Center

Washington, D.C.

Steven M. Goldman, PhD

University of California Berkeley

Berkeley, CA

Tom Greene, PhD

The Cleveland Clinic Foundation

Cleveland, OH

Sundaram Hariharan, MD

Medical College of Wisconsin

Milwaukee, WI

Camille A. Jones, MD, MPH

Boston, MA

Nancy G. Kutner, PhD

Emory University School of Medicine

Atlanta, GA

Suzanne V. McDiarmid, MB, ChB

University of California Los Angeles

Los Angeles, CA

Robin N. Pierson III, MD

University of Maryland Medical Center

Baltimore, MD

Richard E. Pietroski, MS

Transplantation Society of Michigan-Gift of Life

Ann Arbor, MI

Bruce R. Rosengard, MD

University of Pennsylvania School of Medicine

Philadelphia, PA

Steven K. Takemoto, PhD

UCLA Immunogenetics Center

Los Angeles, CA

Ex Officio Members

Michael W. Dreis, PharmD, MPH

SRTR Project Officer

HRSA Division of Transplantation

Bethesda, MD

Erick B. Edwards, Jr., PhD

OPTN/UNOS

Richmond, VA

Joel Greer, PhD

Centers for Medicare and Medicaid Services

(Formerly Health Care Financing Administration)

Baltimore, MD

Shiv Prasad, PhD

National Institute of Allergy and Infectious Diseases - NIH

Bethesda, MD

Joyce Somsak

Acting Director, HRSA Division of Transplantation