Medicare Program; Hospital Conditions of Participation: Requirements for Approval and Re-Approval of Transplant Centers To Perform Organ Transplants

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Centers for Medicare and Medicaid Services

42 CFR Parts 405, 482, and 488

[CMS-3835-P]

RIN 0938-AH17

Medicare Program; Hospital Conditions of Participation: Requirements for Approval and Re-Approval of Transplant Centers To Perform Organ Transplants

AGENCY:

Centers for Medicare and Medicaid Services (CMS), HHS.

ACTION:

Proposed rule.

SUMMARY:

This proposed rule would set forth the requirements that heart, heart-lung, intestine, kidney, lung, and pancreas transplant centers must meet to participate as Medicare-approved transplant centers. These proposed revised requirements focus on an organ transplant center's ability to perform successful transplants and deliver quality patient care as evidenced by good outcomes and sound policies and procedures. We are proposing that approval, as determined by a center's compliance with the proposed data submission, outcome, and process requirements would be granted for 3 years. Every 3 years, approvals would be renewed for transplant centers that continue to meet these requirements. We are proposing these revised requirements to ensure that transplant centers continually provide high-quality transplantation services in a safe and efficient manner.

DATES:

We will consider comments if we receive them at the appropriate address, as provided below, no later than 5 p.m. on April 5, 2005.

ADDRESSES:

In commenting, please refer to file code CMS-3835-P. Because of staff and resource limitations, we cannot accept comments by facsimile (FAX) transmission. You may submit comments in one of three ways (no duplicates, please):

1.

Electronically.

You may submit electronic comments on specific issues in this regulation to

http://www.cms.hhs.gov/regulations/ecomments.

(Attachments should be in Microsoft Word, WordPerfect, or Excel; however, we prefer Microsoft Word.)

2.

By mail.

You may mail written comments (one original and two copies) to the following address ONLY: Centers for Medicare & Medicaid Services, Department of Health and Human Services, Attention: CMS-3835-P, PO Box 8013, Baltimore, MD 21244-8013.

Please allow sufficient time for mailed comments to be received before the close of the comment period.

3.

By hand or courier.

If you prefer, you may deliver (by hand or courier) your written comments (one original and two copies) before the close of the comment period to one of the following addresses. If you intend to deliver your comments to the Baltimore address, please call telephone number (410) 786-9994 in advance to schedule your arrival with one of our staff members.

Room 445-G, Hubert H. Humphrey Building, 200 Independence Avenue, SW., Washington, DC 20201; or

7500 Security Boulevard, Baltimore, MD 21244-1850.

(Because access to the interior of the HHH Building is not readily available to persons without Federal Government identification, commenters are encouraged to leave their comments in the CMS drop slots located in the main lobby of the building. A stamp-in clock is available for persons wishing to retain a proof of filing by stamping in and retaining an extra copy of the comments being filed.)

Comments mailed to the addresses indicated as appropriate for hand or courier delivery may be delayed and received after the comment period.

Submission of comments on paperwork requirements.

You may submit comments on this document's paperwork requirements by mailing your comments to the addresses provided at the end of the “Collection of Information Requirements” section in this document.

For information on viewing public comments, see the beginning of the

SUPPLEMENTARY INFORMATION

section.

FOR FURTHER INFORMATION CONTACT:

Eva Fung (410) 786-7539. Marcia Newton (410) 786-5265. Jeannie Miller (410) 786-3164. Rachael Weinstein (410) 786-6775.

SUPPLEMENTARY INFORMATION:

Submitting Comments:

We welcome comments from the public on all issues set forth in this rule to assist us in fully considering issues and developing policies. You can assist us by referencing the file code CMS-3835-P and the specific “issue identifier” that precedes the section on which you choose to comment.

Inspection of Public Comments:

All comments received before the close of the comment period are available for viewing by the public, including any personally identifiable or confidential business information that is included in a comment. CMS posts all electronic comments received before the close of the comment period on its public website as soon as possible after they have been received. Hard copy comments received timely will be available for public inspection as they are received, generally beginning approximately 3 weeks after publication of a document, at the headquarters of the Centers for Medicare & Medicaid Services, 7500 Security Boulevard, Baltimore, Maryland 21244, Monday through Friday of each week from 8:30 a.m. to 4 p.m. To schedule an appointment to view public comments, phone 1-800-743-3951.

I. Background

A. Key Statutory Provisions

The Medicare statute contains specific authority for prescribing the health and safety requirements for facilities furnishing end stage renal disease (ESRD) care to beneficiaries, including renal transplant centers, pursuant to section 1881(b)(1) of the Social Security Act (the Act). Section 1102 of the Act (42 U.S.C. 1302) authorizes the Secretary to publish rules and regulations “necessary for the efficient administration of the functions” with which the Secretary is charged under the Act. Section 1871(a) of the Act authorizes the Secretary to “prescribe such regulations as may be necessary to carry out the administration of the insurance programs under this title.” In 2003, 13,278 donors (deceased and living) provided organs in the U.S., and 25,468 transplants (deceased and living donor) were performed, yet 83,731 patients waited for a transplant at the end of 2003. Given the relative scarcity of donated organs compared to the number of people on transplant waitlists and the critical need to use these limited resources efficiently, we believe the proposed conditions of participation (CoPs) for transplant centers are necessary to: (1) Protect other potential Medicare beneficiaries who are waiting for organs for transplantation; (2) establish sufficient quality and procedural standards to ensure that transplants are performed in a safe and efficient manner; and (3) reduce Medicare expenses by decreasing the likelihood that a transplant will fail.

Section 1864 of the Act authorizes the use of State agencies to determine providers' compliance with the CoPs. Responsibilities of States in ensuring compliance with the CoPs are set forth in regulations at 42 CFR part 488, Survey, Certification, and Enforcement Procedures. Under section 1865 of the Act and § 488.5 of the regulations,

hospitals that are accredited by the Joint Commission on Accreditation of Healthcare Organizations (JCAHO) or the American Osteopathic Association (AOA) are not routinely surveyed by State agency surveyors for compliance with the conditions but are deemed to meet most of the requirements in the hospital CoPs based on their accreditation. In order to receive deemed status, hospitals accredited by the JCAHO, the AOA, or other national accreditation programs with deeming authority under § 488.6 of the regulations must meet requirements that are at least as stringent as the Medicare CoPs. (

See

Part 488, Survey and Certification Procedures.) Therefore, an accreditation organization could apply for and receive approval of deeming authority for the proposed hospital CoPs for transplant centers if the accreditation organization demonstrates that it has requirements for transplant centers that are at least as stringent as the proposed CoPs.

B. Department Activities Related to Organ Donation and Transplantation

1. Department Commitment To Increasing Organ Donation and Transplantation

At the end of 2003, there were 83,731 Americans waiting for organ transplants. About 25,468 patients on the waitlist received organ transplants (deceased and living donor), and approximately 6,879 persons died waiting for an organ to become available. Promotion of organ donation, which would increase the number of transplant recipients by increasing organ availability, is of paramount importance to the Department of Health and Human Services (the Department). On April 17, 2001, Secretary Tommy Thompson launched his “Gift of Life Donation Initiative,” a multi-level approach to increasing organ, tissue, and marrow donation. The Secretary has directed agencies within the Department to make organ, tissue, and marrow donation a top priority. The Secretary's initiative focuses on 5 elements: (1) A model donor card program, (2) a national forum on donor registries, (3) a national “ Gift of Life” medal to honor donor families, (4) a model curriculum on organ donation for drivers' education classes, and (5) the “Workplace Partnership for Life” program, which involves collaboration with companies and employer groups to make information on organ donation available to all employees.

We are revising the current Medicare requirements for heart, intestine, kidney, liver, and lung centers and adding new Medicare requirements for heart-lung and pancreas centers by proposing transplant center hospital conditions of participation. The proposed CoPs would ensure that all Medicare-approved transplant centers provide quality transplantation services so that organs, once recovered, are not wasted. This proposed rule would not apply to the Medicaid program.

2. Transplantation Criteria Town Hall Meeting

We held a Town Hall Meeting on December 1, 1999 (

See

64 FR 58419) to discuss current medical and scientific evidence regarding potential criteria for approval of transplant centers for Medicare coverage. Approximately 150 people attended the meeting. Attendees included representatives from the Organ Procurement and Transplantation Network (OPTN), staff from transplant centers, health policy and clinical researchers, transplant recipients and their families, physicians and other clinicians, and government officials.

The format for the meeting included four subject-related panel presentations followed by an opportunity for comments from the attendees. The panel topics included: (1) Aspects of facilities linked to coverage, (2) methodologies for measuring outcomes, (3) data used for approving centers, and (4) thresholds for approving centers. In addition to the planned panel topics, the meeting provided for an open forum during which ideas not covered in the topic panels could be shared. To accommodate the views of those who could not attend the meeting, we provided an opportunity for members of the community to share their views in writing.

Comments from the Town Hall Meeting expressed widely divergent views. However, the ideas shared during this meeting and the written public comments were considered seriously and significantly influenced the development of this proposed rule. Our staff has also attended meetings, conferences and training to stay abreast of the latest advancement and issues associated with transplantation.

C. Current Medicare Policy Regarding Transplantation

1. Kidney Transplant Centers

Section 1881 of the Act authorizes benefits for individuals who have been determined to have ESRD, including dialysis and transplantation services. Section 1881(b)(1)(A) of the Act provides an explicit direction to the Secretary of Health and Human Services to develop requirements for kidney (renal) transplantation services under the Medicare program. We fulfilled this responsibility through regulations published on June 3, 1976 (41 FR 22511). These requirements are codified at 42 CFR part 405, Subpart U. Under the Conditions for ESRD coverage, renal transplant centers must meet all appropriate conditions of coverage, which address issues such as compliance with applicable Federal, State, and local laws and regulations; Governing body; Patient long-term program and patient care plan; Patients' rights; Medical records; and Physical environment. In addition, the conditions of coverage include the following criteria specifically for kidney or renal transplant centers:

•

Minimum utilization rates.

The regulations classify renal transplant centers that meet all the other conditions for coverage of ESRD services at 42 CFR 405, Subpart U into the following 4 categories according to the center's minimum utilization rates (annual volume): (1) Unconditional status, (2) conditional status, (3) exception status, and (4) not eligible for reimbursement for that ESRD service. (

See

42 CFR 405.2122.) Unconditional status is assigned to a center that performs 15 or more transplants per year. Conditional status is assigned to a center that performs 7 to 14 transplants per year. (

See

42 CFR 405.2130.) If a center does not meet the minimum utilization rate for unconditional or conditional status, it may, under certain circumstances, be approved for a time-limited exception status. A center that does not meet the requirements for conditional or unconditional status and is not granted an exception status under § 405.2122(b) is not eligible for reimbursement for that ESRD service. (

See

42 CFR 405.2122.)

•

Director of Renal Transplantation.

Renal transplant centers must be under the direction of a qualified transplant surgeon or a physician who is responsible for: (1) Participating in the selection of suitable treatment modalities for each ESRD patient; (2) ensuring adequate training of nurses in the care of transplant patients; (3) ensuring tissue typing and organ procurement services are available either directly or under arrangement; and (4) ensuring transplantation surgery is performed under the direct supervision of a qualified transplant surgeon (

See

42 CFR 405.2170).

•

Minimal Service Requirements

. Renal transplant centers must meet the following minimal service requirements: (1) Be part of a Medicare-approved and participating hospital; (2) be under the supervision of the hospital administrator and medical staff; (3)

participate in a patient registry program with an OPO for patients who are awaiting deceased donor transplantation; (4) utilize a qualified social worker to evaluate transplant patients' psychosocial needs, participate in care planning of the patients and identify community resources to assist the patient and family; (5) utilize a qualified dietitian who will, in consultation with the attending physician, assess the nutritional and dietetic needs of each patient, recommend therapeutic diets, provide diet counseling to patients and their families, and monitor adherence and response to a prescribed diet; (6) utilize a laboratory that is approved under 42 CFR Part 493 and that can perform cross-matching of recipient serum and donor lymphocytes for pre-formed antibodies by an acceptable technique on a 24-hour emergency basis, and (7) utilize the services of an organ procurement organization (OPO) to obtain deceased donor organs, and have a written agreement covering the services (See 42 CFR 2171).

Even though the ESRD conditions of coverage contained at 42 CFR part 405, subpart U include some kidney transplant center provisions, the proliferation of patient and living donor issues and our desire to standardize requirements for transplant centers necessitate a broader regulatory framework for the oversight of kidney transplant centers. Therefore, we have concluded that it is logical for us to replace the requirements contained in Part 405, Subpart U that pertain solely to renal transplant centers with approval and re-approval requirements for kidney transplant centers in these proposed hospital CoPs for organ transplant centers. Specifically, we propose to delete § 405.2120 through § 405.2134, § 405.2170 through § 405.2171, and the definitions for “histocompatibility testing,” “ESRD Network,” “Network organization,” “organ procurement,” “renal transplantation center,” “transplantation service,” and “transplantation surgeon” contained in § 405.2102. The proposed transplant center CoPs are both outcome and process-based and would collectively ensure that transplantation services furnished in all types of transplant centers are safe and efficient.

Generally, the provisions contained in the proposed transplant center CoPs are applicable to all types of transplant centers. However, kidney transplantation differs from other types of organ transplants in some ways. For example, section 1881(b)(1)(A) of the Act explicitly provides for Medicare kidney transplants while coverage of most transplant services are provided under the general “reasonable and necessary” authority of section 1862. Also, whereas organ transplantation is the only treatment option for patients with end-stage heart, liver, lung or intestinal failure, dialysis is an alternative treatment for ESRD patients when transplantation is not feasible. To underscore the distinct nature of kidney transplants and kidney transplant centers, we have included some provisions that are specific only to kidney transplant centers in the proposed hospital CoPs for transplant centers. The following proposed CoPs for approval and re-approval of transplant centers contain provisions that are specific only to kidney transplant centers (see Section II. Provisions of the Proposed Regulation for further discussion of the requirements):

• Condition of participation: Patient and living donor selection (proposed § 482.90(a)(1));

• Condition of participation: Patient and living donor management (proposed § 482.94(c)(3)); and

• Condition of participation: Additional requirements for kidney transplant centers (proposed § 482.104).

2. Extra-renal Organ Transplant Centers

Beginning in 1987, we published several notices in the

Federal Register

delineating our coverage policies regarding various organ transplants. On April 6, 1987, the Health Care Financing Administration (HCFA), now known as CMS, published a ruling (52 FR 10935) (HCFAR 87-1) announcing Medicare's national coverage policy on heart transplants. On April 12, 1991, we published a final notice (56 FR 15006) announcing Medicare's national coverage decision on liver transplants in adults. On February 2, 1995, we published a notice with comment (60 FR 6537) announcing Medicare's national coverage decision on lung transplants.

In these notices, we stated that the transplants in adults were medically reasonable and necessary and covered by Medicare under section 1862 (a)(1), 42 U.S.C. 1395y(a)(1), when performed on carefully selected patients in centers that meet certain criteria. As discussed in these notices, we based these policies on research carried out by the Battelle Human Affairs Research Center (heart) and the Public Health Service's Center for Health Care Technology (liver and lung). The specified center criteria for heart, liver, and lung transplant centers included the following:

•

Patient selection

. A center must have specific written patient selection criteria for each organ type and an implementation plan.

•

Patient management

. A center must have adequate patient management plans and protocols that include therapeutic and evaluative procedures for the waiting period, in-hospital period, and post-transplant phases of treatment.

•

Commitment

. The center must make a sufficient commitment of resources and planning of the transplant center to demonstrate the importance of the center at all levels. Indications of this commitment must be broadly evident throughout the center. The center must use a multidisciplinary team that includes representatives with expertise in the appropriate organ specialty (

e.g.

, hepatology, cardiology, or pulmonology) and the following general areas: Vascular surgery, anesthesiology, immunology, infectious diseases, pathology, radiology, nursing, blood banking, and social services.

•

Facility plans

. The center must have facility plans, commitments, and resources for a program that ensures a reasonable concentration of experience.

•

Maintenance of data

. The center must agree to maintain and, when requested, submit data to CMS.

•

Organ procurement

. The center must be located in a hospital that is a member of the OPTN as a transplant hospital, and abide by its approved rules. The center must also have an agreement with an OPO.

See Section II Provisions of the Proposed Regulations (Proposed Section 482.72) for further discussion of the OPTN rules.

•

Laboratory services

. The center must make available, either directly or under arrangements, laboratory services to meet the needs of patients.

•

Billing

. The center must agree to submit claims to Medicare only for transplants performed on individuals who have Medicare-covered conditions.

•

Experience and survival rates

. The center must demonstrate experience and success with organ transplants. The center staff must have performed a specified volume of transplants for each organ type (12 or more adult heart or liver transplants or 10 or more lung transplants) for covered conditions in each of the two preceding 12-month periods. Additionally, the center must demonstrate a minimum actuarial 1-year and 2-year survival rate. Heart transplant centers must demonstrate actuarial survival rates of 73 percent for 1 year and 65 percent for 2 years. Liver centers must demonstrate a 1-year actuarial survival rate of 77 percent and

a 2-year actuarial survival rate of 60 percent for adult patients. Lung transplant centers must demonstrate a 1-year actuarial survival rate of 69 percent and a 2-year actuarial survival rate of 62 percent.

On July 26, 2000, we issued a national coverage decision (

http://www.cms.hhs.gov/mcd/viewdecisionmemo.asp?id=75

), which was implemented in a program memorandum (

See

Program Memorandum AB-00-95,

http://www.cms.hhs.gov/manuals/pm_trans/2000/memos/comm_date_dsc.asp

) with an effective date of October 11, 2000. This decision announced a revision to the volume criterion for transplant centers to require 12 transplants over a 12-month period for heart and liver transplant centers, and 10 transplants over a 12-month period for lung transplant centers and to eliminate the 2-year minimum experience requirement. The memorandum was issued in response to concerns raised by hospitals that open a new transplant center staffed by an experienced team that has transferred from another Medicare-approved center. The hospitals stated that a new center, staffed with an experienced team, should receive immediate Medicare approval rather than wait at least 2 years until the center was able to demonstrate that it had performed the required volume of transplants. In response to these concerns, we solicited scientific evidence from the transplant community on the relationship between low-volume centers, transplantation team experience, and outcomes. Our analysis of the scientific literature and the information we received indicated that center volume could serve as a proxy for the 2-year minimum experience requirement. In other words, the evidence we reviewed pointed to the fact that volume is a more accurate indicator of outcome than time (see CAG-00061,

http://cms.hhs.gov/ncdr/memo.asp?id=75

, for summary of relevant clinical literature). Thus, new centers staffed with an experienced team that perform a high volume of transplants could be expected to produce satisfactory outcomes.

As of July 1, 1999, Medicare covers whole organ pancreas transplantation for diabetic patients, when it is performed simultaneously with or after a kidney transplant. (

See

sections 35-82 of Coverage Issues Manual.) Effective for services provided on or after April 1, 2001, Medicare covers isolated intestinal transplant, combined liver-intestinal transplant, and multivisceral transplant. Coverage for all three types of intestinal transplants is limited to patients who have irreversible intestinal failure and who have failed total parenteral nutrition (TPN). To be Medicare-approved, an intestinal transplant center must have an annual volume of 10 transplants with a 1-year actuarial patient survival rate of 65 percent (See Program Memorandum AB-01-58).

D. Living Donors

Since 1990, living donation has become the fastest growing source of kidneys for kidney transplants and, more recently, of livers for liver transplants. In 2001, the number of living donors exceeded the number of deceased donors for the first time. There were 12,591 organ donors in the U.S. in 2001; 6,510 were living donors and 6,081 were deceased donors. In 2003, the number of living donors continued to exceed the number of deceased donors. In 2003, there were 13,278 organ donors in the U.S.; 6,821 were living donors and 6,457 were deceased donors. Living donor transplantation provides an alternative to deceased donor transplantation for a growing number of waitlist patients. Of the 25,468 transplants performed in the U.S. in 2003, 6,811 were living donor transplants, which is a 3.0 percent increase from the 6,616 living donor transplants performed in 2002. Meanwhile, the number of deceased donor transplants rose by 2.0 percent from 18,292 in 2002 to 18,657 in 2003.

As living donor transplantation increases, there is growing concern over the safety of living donors. Most of the living donor transplant data reported are for kidney and liver transplants. Other types of living donor transplants are rare and data are scarce. For example, among the 6,811 living donor transplants performed in 2003, 6,468 were kidney transplants, 321 liver transplants, 15 lung transplants, 0 pancreas transplant, and 4 intestinal transplant. 3 kidney-pancreas transplants were performed. The risk of donor death for living kidney donors has been very low. In the 46-year history of living donor kidney transplantation, the risk of donor death is estimated to be approximately 0.03 percent.

For example, if we look at the 6,468 living donor kidney transplants performed in 2003 (out of a total of 15,138 living and deceased kidney transplants performed in the U.S. in 2003), we estimate that fewer than 2 of those transplants would result in donor death. Although there is a relatively low risk of donor death for living kidney donors, recent research seems to indicate that living kidney donation may increase the donor's morbidity. For example, a United Network for Organ Sharing (UNOS) study indicated that a total of 56 previous living donors were identified as having been listed for transplantation. It is unknown if more living kidney donors had suffered from renal failure as well (Ellison MD, McBride MA, Taranto SE, Delmonico FL, Kauffman HM. “Living Kidney Donors in Need of Kidney Transplants: A Report From the Organ Procurement and Transplantation Network.

Transplantation,

2002 November 15; 74(9): 1349-51). Living renal donation has long-term risks that may not be apparent in the short term, which leads us to believe that potential donors should be informed of these long-term risks.

The risk of donor death for living liver donors is higher than the risk of donor death for living kidney donors. In the 13-year history of living donor liver transplants (LDLTs), the risk of donor death has been estimated to be approximately 1 percent. Living liver donors face a higher risk of morbidity and mortality than living kidney donors due in part to complications from blood clotting, bile duct leakage, and infections. Furthermore, the rapid growth of adult LDLT as an alternative to deceased transplantation has resulted in great variation in surgical techniques, center volumes and recipient and donor selection criteria.

In addition to concerns over donor morbidity and mortality, there is also growing concern about the lack of standard guidelines governing living donor selection and post-operative care. For example, in 2002, a living liver donor death was reported in a transplant hospital in New York. The New York Department of Health launched an investigation into the donor's death and found that the donor's post-operative care was inadequate and fragmented. The New York Department of Health's investigation report concluded that inadequate staffing was a contributing factor in the donor's death (“NY Department of Health charges inadequate staffing a factor in live donor's death at Mt. Sinai Hospital,”

Transplant News

, March 15, 2002, at 5.).

Accurate physical and psychosocial assessments of the suitability of prospective donors are imperative to reduce the likelihood of harm to healthy donors. In the absence of national guidelines for donor selection, it is difficult to ensure that living donations are performed safely. Currently, there are few worldwide registries to track living donor outcomes. The OPTN, however, gathers 1-year post-donation

follow-up data on living donors in the US.

Section 1881(d) of the Act entitles any individual who donates a kidney for transplant surgery to Medicare benefits under parts A and B with respect to such donation. Medicare does not have a national coverage determination regarding extra-renal living donor transplants. In the absence of a national coverage determination, however, Medicare contractors may make local coverage determinations either on a claim-by-claim basis or through local medical review policies. We have some concerns about the lack of standardized recipient and donor selection criteria, best practices in living donation procedures, a national outcomes database of donors' long-term follow-up and the variability in surgical expertise, volumes and center resources given the growth in living donor transplants. More systematic data collection and reporting of donor and recipient mortality and morbidity are needed to further assess the risk of death for living donors and the benefit for recipients. Generally, we believe living donation is a very promising medical practice. Therefore, in order to protect the safety of living donors and guarantee the more efficient use of human organs, we have proposed some minimal requirements for transplant centers performing living donor transplants that would apply to all Medicare-approved centers that perform living donor transplants. In accordance with our authority to establish standards necessary for the health and safety of individuals furnished services in hospitals, we believe we possess sufficient authority to prescribe rules for this practice. We invite public comments on these proposed requirements for living donor selection and living donor rights (

see

Section II. Provisions of the Proposed Regulations for a detailed discussion of these proposed requirements). We also request comments on whether we need to establish additional criteria for transplant centers performing living donor transplants.

[If you choose to comment on this issue, please include the caption “CRITERIA FOR CENTERS PERFORMING LIVING DONOR TRANPLANT” at the beginning of your comments.]

E. Why We Are Proposing New CoPs for Transplant Centers

Our current Medicare coverage policies for extra-renal organs are based on the “reasonable and necessary” provision, Section 1862(a)(1)(A) of the Act. (“[N]o payment may be made under part A or part B for any expenses incurred for items or services—(1)(A) which * * * are not reasonable and necessary for the diagnosis or treatment of illness or injury or to improve the functioning of a malformed body member.”) Generally a medical procedure will be covered if its safety and efficacy have been adequately demonstrated by scientific evidence and the medical community has generally accepted the procedure. In the

Federal Register

notices announcing the Medicare coverage policies for heart, liver, and lung transplants, we stated that organ transplants in adults were reasonable and necessary when performed on carefully selected patients in facilities that meet certain criteria.

In the past decade, however, the medical community has made remarkable strides in organ transplantation, and data on successful transplant outcomes are compelling. Organ transplantation is generally very effective and successful. Patients who have received transplants benefit substantially from these life-saving procedures in terms of improved quality of life and longer lifetime. Aided by ongoing evolution in pharmacology and transplant technology, organ transplantation is no longer regarded as an experimental procedure by the medical community and most health insurance companies. Instead, transplantation has become the mainstream operation for many patients who are in the end stage of organ failure.

Furthermore, cutting-edge medical technology and pharmacology have raised graft and patient survivals significantly, such that we recognize that the survival standards that we had established previously for heart, liver, and lung centers may be too low. The national mean 1-year patient survival rates for heart, liver, and lung transplants performed in all transplant centers are much higher than the 1-year patient survival thresholds we established in our earlier national coverage decisions for Medicare approval of heart, liver, and lung transplant centers.

Furthermore, the current requirements for heart, liver, and lung centers established threshold requirements for Medicare reimbursement but do not include criteria for re-evaluating the ongoing performance of approved heart, liver and lung centers. Since organ transplantation is a medical procedure that depends completely on organs donated from an appropriate donor, any potential outcome failure should be minimized to minimize organ wastage. Ongoing evaluation of a transplant center's outcomes would serve as a valuable oversight tool for guaranteeing that donated organs are used efficiently. By establishing criteria for data submission, outcome measures, and process requirements, we can assume that Medicare-approved transplant centers would continue to provide a sufficient quality of transplantation so that organ wastage due to transplant failure would be decreased.

We believe it is important to promulgate regulations that will allow CMS to take advantage of advances in medical technology and establish standards for facilities that will ensure that Medicare beneficiaries receiving care at Medicare-approved transplant centers receive quality transplantation services. We are proposing rules that will encourage centers to seek approval to perform transplants on patients and that will include reasonable requirements necessary to produce a high probability of success. We believe these rules will lead to more efficient usage of donated organs and enhance effective administration of the Medicare program. We are proposing to codify the requirements for the approval and re-approval of transplant centers as an option for hospitals under part 482, Subpart E. These regulations would apply to heart, heart-lung, intestine, kidney, liver, lung, and pancreas centers. For purposes of this regulation, intestine centers are those Medicare-approved liver transplant centers that perform intestinal transplants, combined liver-intestinal transplants, and multivisceral transplants. Pancreas centers are those Medicare-approved kidney transplant centers that perform pancreas transplants, alone or subsequent to a kidney transplant, and that perform kidney-pancreas transplants.

The requirements for Medicare-approved transplant centers have been published over the years in the

Federal Register

, the Coverage Issues Manual, and 42 CFR part 405, subpart U. Locating the Medicare requirements for different organ types has proven difficult for hospitals desiring to become Medicare-approved transplant centers. Therefore, we are proposing to include the criteria for all of the organ transplant types (

i.e.

, heart, heart-lung, intestine, kidney, liver, lung, and pancreas) in the same CFR part: 42 CFR part 482. Although we received some comments during the Town Hall Meeting in December 1999 expressing the view that kidney transplant center criteria should remain with the ESRD facility conditions, we believe it will facilitate ease of reference and understanding if all the transplant center criteria are

consolidated into a specific set of hospital policies.

Entities that request approval as a Medicare transplant center must first meet all of the hospital CoPs in 42 CFR part 482; however, inclusion of the organ transplant center criteria in the hospital CoPs does not imply that every hospital must meet the criteria in order to participate in Medicare. Rather, the transplant criteria represent an optional status based on conditions that are applicable only to hospitals that choose to apply for Medicare approval as a transplant center. Each type of organ transplant center would be approved separately, so only the approval of the individual organ-specific transplant center would be threatened if it were found non-compliant with the CoPs for transplant centers. That is, the hospital would not face the automatic loss of its Medicare approval as a hospital (or the loss of Medicare approval for other transplant centers) if one transplant center in the hospital were found to be noncompliant with the CoPs for that type of transplant center.

II. Provisions of the Proposed Regulations

For the reasons discussed previously, we propose to set forth new hospital CoPs for the approval and re-approval of transplant centers at part 482, subpart E of this chapter. Following is a discussion of the specific requirements contained in the proposed conditions.

Special Requirements for Transplant Centers (Proposed Section 482.68)

The requirements for approval and re-approval of transplant centers contained in this proposed rule represent special requirements that a transplant center must meet in order to receive Medicare approval as an organ-specific transplant center. Therefore, we propose a hospital that has a Medicare provider agreement must meet the CoPs specified in § 482.70 through § 482.104 in order to be granted approval from CMS and to receive reimbursement for providing transplant services. We propose that unless we specify otherwise, the CoPs specified in § 482.70 through § 482.104 apply to all transplant centers addressed in this proposed rule (

i.e.

, heart, heart-lung, intestine, kidney, liver, lung, and pancreas transplant centers).

We also propose that transplant centers seeking Medicare approval meet the hospital conditions of participation specified in § 482.1 through § 482.57. In other words, if the hospital in which a transplant center operates is terminated from Medicare, the transplant center would also lose its Medicare approval. However, loss of a transplant center's approval status would not automatically lead to termination of the hospital's provider agreement.

Definitions (Proposed § 482.70)

For clarity, we propose standardizing the usage of certain terms by proposing definitions for “transplant hospital,” “transplant program,” and “transplant center.” Sometimes CMS has used the term “transplant center” interchangeably with the term “transplant hospital” and sometimes it has used it interchangeably with the term “transplant program.” We propose defining “transplant hospital” as a hospital that furnishes organ transplants and other medical and surgical specialty services required for the care of transplant patients. A transplant hospital may have one or more types of organ transplant programs operating within the same hospital. Based on the definition of “transplant program” set forth at 42 CFR 121.2, we propose defining a “transplant program” as a component within a transplant hospital that provides transplantation of a particular organ type. Under the proposed definitions for “transplant hospital” and “transplant program”, we propose to use “transplant center” interchangeably with “transplant program” in this proposed rule.

We propose to delete the definitions for “histocompatibility testing,” “ESRD Network,” “network organization,” organ procurement,” “renal transplantation center,” “transplantation service,” and “transplantation surgeon” contained in § 405.2102. To emphasize the distinct statutory requirements that kidney transplant centers have to meet and to clarify usage of three terms in the proposed CoPs for transplant centers, we propose to retain in § 482.70 the definitions for “ESRD,” “ESRD network,” and “network organization” from § 405.2102.

We propose adding a definition for “adverse event” because we propose requiring a center to establish a written policy to address adverse events that occur during any phase of an organ transplantation case. The proposed definition for “adverse event” is derived from the JCAHO definition of an “adverse event” and provides examples of adverse events that may occur in a transplant center.

To reduce confusion, we also propose definitions for the particular types of organ transplant centers addressed in this proposed rule that perform multi-organ transplants. We propose including definitions for “heart-lung transplant center,” “pancreas transplant center,” and “intestinal transplant center” as they are used in this proposed rule.

These definitions, as we propose to include them, are contained in the regulatory text at proposed § 482.70.

Proposed General Requirements for Transplant Centers

Condition of Participation: OPTN Membership (Proposed section 482.72)

The OPTN was established under section 372 of the Public Health Service (PHS) Act, as enacted by the National Organ Transplant Act of 1984 (Pub. L. 98-507), and amended by Public Law 100-607 and Public Law 101-616. Section 372 of the PHS Act requires the Secretary to provide, by contract, for the establishment and operation of the OPTN to manage the national organ allocation system, to increase the supply of donated organs, and to perform related activities. Since 1986, the Health Resources and Services Administration's (HRSA) Division of Transplantation (DoT) has administered a contract with UNOS to operate the OPTN. On October 20, 1999, HRSA published regulations governing the operation of the OPTN at 42 CFR Part 121 (64 FR 56650).

The primary functions of the OPTN are (1) to ensure that critically-ill and medically-qualified patients have equitable access to organs; (2) to ensure the safe and efficient recovery and use of scarce vital organs; and (3) to collect, maintain, and track information on all transplants and transplant patients from the time of surgery until graft failure or patient death. Although the OPTN regulations referred to above include some provisions that apply to OPTN members, including transplant centers, the OPTN regulations at § 121.4 also require the OPTN to establish policies for its members in order to achieve the goals of the OPTN. As required by the OPTN regulations at § 121.4, policies are established concerning organ procurement and transplantation for OPTN members. These policies established by the OPTN are legally enforceable against OPTN members if the Secretary approves them and they are published in the

Federal Register

in accordance with § 121.4. The Secretary enforces the OPTN policies, or rules, pursuant to the procedure laid out at § 121.10. To date, no OPTN policies have been approved by the Secretary.

Until enactment of the Omnibus Budget Reconciliation Act (OBRA) of 1986 (Pub. L. 99-509), membership in the OPTN was voluntary. However, section 9318 of the OBRA of 1986 added section 1138(a)(1)(B) to the Act to require hospitals that perform organ

transplants to be members of and abide by the rules and requirements of the OPTN as a condition for participation in the Medicare and Medicaid programs. In accordance with section 1138(a)(1)(B) of the Act, the hospital condition of participation for organ, tissue, and eye procurement at § 482.45(b)(1) requires that a hospital in which organ transplants are performed must be a member of the OPTN and abide by the OPTN rules that have been approved by the Secretary. We propose that transplant centers must be located in a transplant hospital that is a member of and abides by the rules and requirements of the OPTN as set forth in § 482.45(b)(1), which are enforceable under § 121.10. We propose that no transplant hospital would be considered to be out of compliance with section 1138(a)(1)(B) of the Act, or with the proposed rule, unless the Secretary had given the OPTN formal notice that he or she approved the decision to exclude the transplant hospital from the OPTN and had notified the center in writing.

Condition of Participation: Notification to CMS (Proposed section 482.74)

The current requirements for coverage of heart, liver and lung transplants require a Medicare-approved transplant center to report immediately to CMS any events or changes that would affect its approved status. Specifically, a center is required to report to us, within a reasonable period of time, any significant decrease in its experience level (for example, volume) or survival rates, the departure of key members of the transplant team or any other major changes that could affect the performance of heart, liver or lung transplants at the facility. There are no requirements for kidney transplant centers to report significant changes to CMS. We are proposing to require each transplant center to report immediately to CMS information on any significant changes that would affect its approval, such as an unusually large number of patient deaths during or shortly after transplant that could impact the center's 1-year patient survival rates or a change in key staff members, such as the individual the transplant center designates to the OPTN as the center's “primary transplant surgeon” or “primary transplant physician.” This would be a new requirement for kidney, pancreas, heart-lung, and intestine transplant centers. We believe this requirement is necessary for all transplant centers to ensure that each transplant center maintains the resources and commitment needed to safely and efficiently perform transplants throughout its approval period.

Condition of Participation: Pediatric Transplants (Proposed Section 482.76)

Section 4009(b) of the Omnibus Budget Reconciliation Act of 1987 (OBRA 1987) (Pub. L. 100-203) indicates that pediatric heart transplant centers are Medicare-approved heart transplant centers if they meet certain criteria. Public Law 100-203 specified the following criteria: (1) The hospital's pediatric heart transplant center is operated jointly by the hospital and another facility that is Medicare-approved; (2) the unified program shares the same transplant surgeons and quality assurance program (including oversight committee, patient protocol, and patient selection criteria); and (3) the hospital demonstrates to the satisfaction of the Secretary that it is able to provide the specialized facilities, services, and personnel that are required by pediatric heart transplant patients (See Section 35-87 of the Coverage Issues Manual). We currently use criteria for pediatric liver and lung transplant centers similar to the criteria that were specified by Congress for pediatric heart transplant centers. (See Section 35-53.1 of the Coverage Issues Manual for liver transplants and 60 FR 6537 for lung transplants.)

Since many centers that perform pediatric transplants are not jointly operated by another facility that is Medicare-approved, we propose to require all transplant centers, adult and pediatric, that wish to be reimbursed for pediatric transplants performed on Medicare beneficiaries to specifically request Medicare approval to perform pediatric transplants. We would approve and re-approve the center to perform pediatric transplants using the procedures described in proposed § 488.61. A center that wishes to be approved to perform pediatric transplants would have to meet the conditions of participation contained in § 482.68 through § 482.74 and § 482.80 through § 482.104 with respect to its pediatric patients. However, given Congress's intent that pediatric heart centers could participate in Medicare if they meet the requirements described in section 4009(b) of OBRA 1987, we are proposing to retain the statutory criteria as an option for heart transplant centers that wish to become Medicare-approved to perform pediatric heart transplants. In other words, a center that wishes to be approved to perform pediatric heart transplants may be approved by meeting the data submission, outcome, and process requirements proposed in this regulation, or the center may be approved by meeting the criteria in section 4009(b) of OBRA 1987.

Although all transplant centers that wish to be reimbursed for transplants performed on pediatric Medicare beneficiaries would have to request Medicare approval to perform pediatric transplants, we believe it is necessary to distinguish between two different types of centers that may provide pediatric transplantation services. In some centers, patients are predominantly adults (

i.e.

, 18 years or older) and only a few pediatric transplants are performed. In other centers, pediatric transplant programs are separate from the adult programs and may be operated by departments of pediatrics or children's hospitals where a majority of transplants are performed on pediatric patients (

i.e.

, patients younger than 18).

We propose that in centers where patients are predominantly (≤50 percent) adult patients, the center would need to have Medicare approval to perform both adult and pediatric transplants in order to be reimbursed for transplants performed on pediatric Medicare beneficiaries. Since few transplants are performed on children in such centers, we propose that loss of Medicare approval to perform adult transplants, whether voluntary or involuntary, would result in loss of Medicare approval to perform pediatric transplants. However, loss of Medicare approval to perform pediatric transplants would not affect the center's Medicare approval to perform adult transplants.

Likewise, we propose that a center that predominantly (≥50 percent) provides transplantation services to pediatric patients (

i.e.

, a pediatric center) would need to have Medicare approval to perform both pediatric and adult transplants in order to be reimbursed for transplants performed on adult Medicare beneficiaries. In this case, however, loss of Medicare approval to perform adult transplants would not impact the center's Medicare approval to perform pediatric transplants while loss of Medicare approval to perform pediatric transplants, whether voluntary or involuntary, would result in loss of Medicare approval to perform adult transplants. Usually, centers that predominantly serve pediatric patients will transplant only a few young adults (18 or 19 years old) who wish to maintain continuity of care but have aged beyond the pediatric patient classification. Because of the occasional adult patients being transplanted at the pediatric centers and the relatively few pediatric transplants in general, we are not requiring a minimum number of

transplants (adult or pediatric) for pediatric centers. We are requesting comments on our proposed methodology for approving and re-approving centers that perform pediatric transplants.

[If you choose to comment on this issue, please include the caption “CENTERS PERFORMING PEDIATRIC TRANSPLANTS” at the beginning of your comments.]

Proposed Transplant Center Data Submission and Outcome Requirements

Condition of Participation: Data Submission and Outcome Measure Requirements for Initial Approval of Transplant Centers (Proposed section 482.80)

[If you choose to comment on this section, please include the caption “OUTCOME MEASURE REQUIREMENTS” at the beginning of your comments.]

A. Overview

Our intent in promulgating this rule is to establish quality standards for approval and re-approval of transplant centers participating in Medicare. We intend to focus regulations on the actual care being furnished and the outcomes of that care, rather than solely on the underlying policies and procedures.

The Institute of Medicine (IOM) highlighted the importance of focusing on outcomes in its report (“Organ Procurement and Transplantation: Assessing Current Policies and the Potential Impact of the DHHS Final Rule”), published on July 22, 1999. In its recommendation on Federal oversight, the IOM articulated its view that the Department should include greater use of patient-centered, outcome-oriented performance measures for OPOs, transplant centers, and the OPTN.

Some representatives from the transplant community that attended the CMS Town Hall Meeting held in December 1999 also voiced a similar opinion that transplant center performance should be assessed using patient-centered outcome measures. However, there was no consensus on how to design an outcome-oriented system for evaluating center performance.

We recognize the fact that transplant outcomes and practices can be assessed from multiple perspectives, and there is no one single criterion that can adequately evaluate the performance of a transplant center. Therefore, we are proposing to evaluate a center's performance by measuring a center's outcomes and experience, in combination with some specific process requirements we believe will ensure the quality of the transplant center.

In developing a proposed framework for the initial approval of transplant centers, we have included criteria of significance to an outcome-based evaluation system. We are proposing criteria for timely and complete data submission, patient survival, and graft survival.

B. Data Submission Requirements for Initial Approval of Transplant Centers

1. Current Medicare Data Submission Requirements

Under current transplant policies for heart, liver, and lung centers and the current regulations for renal transplant centers, centers applying for Medicare approval are required to supply data to CMS. As appropriate, these applicants must report every heart and liver transplant performed since 1982, every lung transplant performed since January 1, 1990, or every kidney transplant performed during the most recent year of operation and during each of the preceding 2 calendar years. The current criteria for approval of heart, liver, and lung transplant centers require centers to agree to maintain and routinely submit to CMS, in a prescribed standard format, summary data about patients selected, protocols used, and short- and long-term outcomes on Medicare and non-Medicare patients undergoing transplantation.

2. Data Collection and the OPTN

In addition to supplying transplant data to CMS, transplant centers also collect and submit transplant data to the OPTN. Under the Department's Health Information Privacy Rules at 45 CFR 164.512, which implement the privacy provisions of the Health Insurance Portability and Accountability Act (HIPAA), covered entities are permitted to use and disclose protected health information to OPOs or other organizations engaged in the procurement, banking, or transplantation of organs, eyes, or tissues from deceased donors. Therefore, data submission to the OPTN is an exception under HIPAA with respect to organ transplants. The OPTN database utilizes electronic submission, review, and modification features through a secure, encrypted web-based system. Under contract with HRSA, the OPTN develops policies concerning data submission as well as policies concerning organ procurement and transplantation.

The OPTN requires its members to submit organ-specific data electronically to the OPTN through the use of standardized forms. There are a total of 26 different organ-specific forms containing more than 3,500 data fields. Transplant centers are responsible for submitting the appropriate organ-specific forms for each center using six form types. The OPTN also specifies time frames in which each form must be submitted to the OPTN. Below is a description of the six forms for which transplant centers are responsible and the due dates established by the OPTN for each form:

•

Transplant Candidate Registration Form

includes waitlist data as well as other clinical and organ-specific information collected prior to transplant. There is a form for each organ type: Kidney-pancreas, kidney, pancreas, liver, intestine, heart, lung, and heart-lung. The OPTN requires transplant centers to submit the organ-specific Transplant Candidate Registration Form to the OPTN within 30 days of the form generation date.

•

Transplant Recipient Registration Form

includes the patient status at discharge, pre- and post-transplant clinical information, as well as treatment data. The form is generated when the patient receives a transplant and is removed from the waitlist. There is a form for each organ type: kidney-pancreas, kidney, pancreas, liver, intestine, and thoracic (

i.e.

, heart, lung, and heart-lung). The OPTN requires transplant centers to complete the organ-specific Transplant Recipient Registration Form when the transplant recipient is discharged from the hospital or six weeks following the transplant date, whichever is first. The OPTN also requires transplant centers to submit the organ-specific Transplant Recipient Registration Form to the OPTN within 60 days of the form generation date.

•

Transplant Recipient Follow-up Form

is generated six months post-transplant (excluding thoracic) and on the transplant anniversary for every living organ recipient with a functioning graft. It includes patient status, clinical, and treatment information. There is a form for each organ type: Kidney-pancreas, kidney, pancreas, liver, intestine, and thoracic. The OPTN requires transplant centers to submit the organ-specific Transplant Recipient Follow-up Form to the OPTN within 30 days of the form generation date unless the transplant recipient dies or experiences a graft failure. In such circumstances, the OPTN specifies that transplant centers are required to submit the organ-specific Transplant Recipient Follow-up Form to the OPTN within 14 days of the recipient's death or graft failure.

•

Post Transplant Malignancy Form

is generated after a malignancy has been reported on the Transplant Recipient Follow-up Form. The OPTN requires transplant centers to submit the Post Transplant Malignancy Form to the OPTN within 30 days of the form generation date.

•

Living Donor Registration Form

collects data for all living organ donors. The OPTN requires transplant centers to submit the Living Donor Registration Form to the OPTN within 30 days of the form generation date.

•

Living Donor Follow-up Form

includes patient status and clinical information collected on the living donor at intervals of six months and one year post-transplant. The OPTN requires transplant centers to submit the Living Donor Follow-up Form to the OPTN within 30 days of the form generation date.

The OPTN also includes a data submission standard that requires, among other things, 95 percent of the required forms to be completed within 90 days of their due date.

3. The Scientific Registry of Transplant Recipients (SRTR) and the Center-Specific Reports

Once the OPTN collects the required data, the SRTR, which is run by the University Renal Research Education Association (URREA) under contract with HRSA, analyzes the OPTN data and creates national and center-specific reports. Regulations at 42 CFR 121.11 require the SRTR to make center-specific information on the performance of transplant centers available over the Internet and requires the SRTR to update these data at least every 6 months. URREA updates the center-specific reports every January and July, and makes the center-specific reports available over the Internet at

http://www.ustransplant.org.

The SRTR center-specific reports contain a variety of statistical tables based on the transplants performed at each center in the US. The center-specific reports contain information on each center's performance; including statistics on each center's waitlist activity, deceased and living donor transplant recipient characteristics and outcomes (including patient and graft survival), and donor characteristics. The SRTR also prepares national summary reports of these topics by center. Below, we provide a more detailed description of some of the statistics available in the center-specific reports.

The most important outcome for a lifesaving technology such as transplantation is whether the patient survives the procedure. Currently, the SRTR center-specific reports provide observed and expected patient survival rates for adult and pediatric patients at the 1-month, 1-year, and 3-year reporting time point for each center. For calculation of the 1-month, 1-year, and 3-year patient survival statistics, the SRTR center-specific reports use transplants that occurred during a 2.5-year interval before a report is published. In order to maximize follow-up of patients that were transplanted towards the end of the 2.5-year interval, there may be a significant lag between the time that the last transplant in the 2.5-year period occurred and the time that patient survival statistics are reported. For example, the July 2003 center-specific reports contain 1-month and 1-year patient survival statistics for abdominal transplants (for example, kidney, kidney-pancreas, intestine, liver, and pancreas transplants) that were performed at a center between January 1, 2000 and June 30, 2002 and for thoracic transplants (for example, heart, heart-lung, and lung transplants) that were performed between January 1, 2000 and June 30, 2002. In the future, the SRTR plans to calculate 1-month and 1-year survival statistics using 2.5-year cohorts for all organs. The 3-year patient survival statistics include transplants performed between January 1, 1998 and December 31, 1999. Additionally, the SRTR center-specific reports include adult patient survival rates and pediatric patient survival rates for deceased and living donor transplants.

A center's observed patient survival rate is an estimate of the fraction of patients in each cohort that would still be alive at the reporting time point had follow-up data been received up to that time. The SRTR uses the Kaplan-Meier method to calculate a center's observed patient survival rate from the OPTN follow-up data and the Social Security Death Master File (SSDMF) data. The Kaplan-Meier method is a standard statistical technique for estimating survival at the reporting time point by assuming that the failure rate would have been the same for those patients lost to follow-up as was observed for patients with complete follow-up data.

Recognizing that some patients are lost to follow-up for reasons beyond a transplant center's control, such as a patient's change of residence, change of providers, or unreported death, the SRTR began augmenting the OPTN data by tracking all transplant patients “lost to follow-up” through the SSDMF. Although there are some flaws in the SSDMF data, it has enhanced the SRTR's ability to determine if patients “lost to follow-up” had died or were still thought to be alive on a certain date. In addition to enhancing the accuracy of the SRTR's center-specific reports, URREA has determined that the additional data obtained from the SSDMF seems to increase the reported survival rates of some centers.

A center's expected patient survival rate is a risk-adjusted statistic that provides an estimate of the fraction of patients who would be expected to be alive at each reported time point based on the national experience for similar patients. The SRTR uses the Cox proportional hazards regression model to calculate each center's expected patient survival rate.

The Cox model is a statistical modeling technique that is widely used in the analysis of survival data. The Cox model is flexible in the types of data, event rate patterns, and covariates it can handle. It can model dependence of event rates on patient and donor characteristics in a variety of ways including time dependent proportional hazards (covariates), which are extremely useful for modeling the effect of current patient status on mortality and for modeling both short term and long term covariates effects on event rates. Information about the Cox model can be found on the Internet. For example, background on the Cox model can be found at

http://members.aol.com/johnp71/prophaz.html.

The Cox model is designed to evaluate the outcomes among the recipients at one center, compared to what would be expected, had those same patients received a transplant at an “average” center. One of the most important features of the Cox model is the identification of the adjustment factors that could affect transplant outcomes. These factors are chosen using clinical input supported by statistical analyses. The clinical input comes from the constant review of SRTR models by experts on the OPTN committees and the Secretary's Advisory Committee on Organ Transplantation (ACOT). The Secretary established the ACOT to enhance organ donation, ensure the system of organ transplantation is grounded in the best available medical science, ensure the public that the system is as effective and equitable as possible, and thereby increase public confidence in the integrity and effectiveness of the transplantation system. Some non-statistically significant factors are also included in the Cox models used to calculate expected patient survival in order to improve validity and public acceptance of the models.

Historically, there have been more than 100 models fit for each center-specific report release (

e.g.

, models by organ, by age group, by living/deceased donor, by follow-up time period, by graft/patient survival). Currently, the models used to calculate 1-month and 1-year patient survival are based on the same cohort of patients. The SRTR plans to begin to use a single model to calculate survival, as this would allow for more stable estimation of factors for the 1-month results, which currently are based on relatively few events. This will assure consistency in the expected values for the overall transplant population and the subpopulations of living and deceased donor recipients.

The specific risk adjustment factors that affect transplant outcomes identified in the Cox model and their weights are subject to change with each updated analysis. Semi-annually (every January and July), the SRTR assesses the goodness of fit and stability of a survival model using the index of concordance. The index of concordance is a measure of a model's ability to fit the mortality outcomes for each patient. In order to assess the stability of the models, for each center-specific report release, the models will be fit using the same list of covariates to a series of successive cohorts of transplant recipients. In addition, the values of the coefficients will be reported for each of the models while outcomes are evaluated relative to the norm, or the “average.” Significant changes in the index of concordance and the coefficients over a period of time will help to identify the factors that require closer evaluation in order to be sure that the models are as up to date as possible.

In the future, the SRTR plans to complete a table for each of the center-specific report post-transplant models. The table will include the index of concordance, the coefficients, and p-values for the coefficients when the model is fit for transplants during the 2.5-year cohort used for the current center-specific report release as well as that for the two previous releases. This table will be posted publicly on the SRTR Web site (

http://www.ustransplant.org

) at the time of the preview site, which is approximately 1 month before the center-specific report public release date. It is intended to allow users to assess the stability of the models. If the fit of the models or the coefficients of the factors change markedly, one would be careful to evaluate the models to be sure that they are as up to date as possible. If the fit and coefficients do not change markedly, one could be assured that the models are stable.

For purposes of example, the Cox models used in the July 2004 center-specific reports to calculate expected 1-year patient survival rates for deceased donor adult transplants contained the following factors. (Analytic Conventions—Guide to the Center-Specific Reports,

http://www.ustransplant.org/programs-report.html

). Factors for kidney transplants included: diagnosis, donor age, donor history of hypertension, donor meets expanded donor criteria for deceased kidney, donor race, donor serum creatinine, donor cause of death, human lymphocyte antigen (HLA) mismatch, peak panel reactive antibody (PRA), recipient age, recipient ethnicity, recipient medical condition, recipient race, and year of ESRD treatment. Factors for liver transplants included: diagnosis, ABO (

i.e.

, blood types A, B, AB, and O) compatibility, donor Hispanic/Latino, donor age, donor and recipient in the same region but not the same OPO, donor and recipient not in same region or OPO, donor race, donor cause of death, non heart beating donor, recipient portal vein thrombosis, recipient age, recipient any previous transfusions, recipient ascites, recipient creatinine, recipient ethnicity, recipient height, recipient incidental tumor found at time of transplant, recipient insulin dependent diabetes, recipient medical condition, recipient on life support, recipient previous abdominal surgery, recipient race, and split or partial liver. Factors for heart transplants included: diagnosis, donor age, donor cause of death, ischemia time, recipient creatinine, recipient height, recipient medical condition, recipient on extracorporeal membrane oxygenation (ECMO), and recipient on ventilator. Factors for lung transplants included: cardiac index, diagnosis group B, diagnosis group C, diagnosis group D, diagnosis, donor age, donor body surface area, donor history of diabetes, donor race, donor cause of death, percent predicted forced vital capacity (FVC), ischemia time, New York Heart Association (NYHA) class, oxygen required at rest, recipient age, recipient creatinine, recipient female, recipient on ventilator, recipient race, and pulmonary artery (PA) hemodynamics mean by diagnosis interaction.

As in patient survival, the SRTR also calculates observed and expected 1-month, 1-year and 3-year graft survival statistics. Using the Kaplan-Meier method, the SRTR calculates observed graft survival rates for each of the reporting time points (

i.e.

, 1-month, 1-year, and 3-year) from OPTN and SSDMF data. Cox models are used to calculate expected graft survival statistics for each of the reporting time points. The factors predictive of graft survival models are generally similar to those predictive of patient survival models and generally include an indicator for whether or not this was the first transplant of this type. Again, 1-month, 1-year, and 3-year graft survival statistics in the center-specific reports are stratified by age (

i.e.

adult or pediatric) and by donor type (

i.e.

deceased or living) and are calculated using only transplants that occurred during a 2.5-year interval before a report is published.

4. Proposed Data Submission Requirements

Since the SRTR center-specific reports contain a wealth of information about transplant center outcomes and the SRTR prepares its analytical reports from the data that transplant centers are already self-reporting to the OPTN, we propose that the SRTR's center-specific reports could form the foundation for our outcome evaluation system. However, we need to be certain of the completeness of the data used to evaluate each center's outcomes.

In July 2001, an article that appeared in the

Milwaukee Journal Sentinel

(“Transplant Rate Reports Don't Tell Whole Story,”

http://www.jsonline.com/alive/column/jul01/marccol30072701.asp

, July 27, 2001) questioned the data used by the SRTR to generate and publish the center-specific reports. The article charged that some centers were getting away with reporting less than half of follow-up data required by the OPTN. Incomplete data can be attributed to several factors, including lost to follow-up. However, the article also alleged that some centers were purposely submitting incomplete data to skew their survival results. In order to ensure that the data used by the SRTR for analysis and compilation of the national and center-specific reports are comprehensive and accurate, we believe that it is important that we establish requirements for timely and complete reporting of data to the OPTN.

As discussed earlier, the OPTN includes a data submission standard that requires, among other things, 95 percent of the required forms to be completed within 90 days of their due date. We propose a similar data submission requirement. We propose, at § 482.80(a) that no later than 90 days after the due date established by the OPTN, heart, heart-lung, intestine, kidney, liver, lung, and pancreas transplant centers must submit to the OPTN at least 95 percent of required data submissions on all transplants

(deceased and living donor) performed at the center. We believe it is important to maintain this 90-day grace period to ensure that transplant data collection and compilation are as complete and accurate as possible.

We propose that required data submissions include, but not be limited to, the submission of the appropriate organ-specific OPTN forms for transplant candidate registration, transplant recipient registration, and recipient follow-up. Requiring timely and complete submission of data will ensure up-to-date and meaningful data.

C. Outcome Measure Requirements for Initial Approval of Transplant Centers

1. Current Medicare Outcome Measure Requirements

Under the current transplant policies, transplant centers applying for Medicare approval of a heart, liver or lung transplant center are required to report their 1-year and 2-year actuarial (unadjusted) patient survival rates using the modified Kaplan-Meier method. The modified Kaplan-Meier method estimates survival at the reporting time point by treating those patients lost to follow-up as dead on the day following the last ascertained survival.

The current actuarial survival standards for heart transplants were developed in 1986. According to those standards, a center is required to demonstrate an actuarial survival rate of 73 percent for 1 year and 65 percent for 2 years for patients who have had heart transplants since January 1, 1982 at that center. Current criteria for approval as a liver transplant center were developed in 1991 and require an actuarial survival rate of 77 percent for 1 year and 60 percent for 2 years for the time period the center is using to calculate survival. The criteria for lung transplants were published in our February 1995 notice of Medicare policy for lung transplants. The criteria require centers to maintain a 1-year actuarial survival rate of 69 percent and a 2-year actuarial survival rate of 62 percent for all transplant cases occurring on or after January 1, 1990.

The Medicare National Coverage Decision that we issued in October 2000 requires intestinal centers to have a 1-year actuarial survival rate of 65 percent for intestinal and multivisceral transplants. The required intestinal threshold is based on a weighted average of the national 1-year patient survival rates for small bowel transplantation, small bowel/liver transplantation, and multivisceral transplantation data from the literature reports on the international intestinal transplant registry. There are no survival standards in place for kidney, pancreas, and heart-lung transplant centers for Medicare approval.

2. Appropriateness of Current Survival Criteria

At the time the survival criteria for heart, liver and lung transplants were developed, organ transplants were largely viewed as experimental procedures and the survival criteria were designed to be high enough to ensure that Medicare-approved transplant centers were high-quality institutions but low enough to ensure that centers did not exclude high-risk patients. Aided by remarkable advances in medicine and cutting-edge technology, survival rates for heart, liver, and lung transplant patients have steadily increased since our criteria were established. For example, according to the 2003 OPTN/SRTR Annual Report, the unadjusted 1-year patient survival figures for transplants performed between 2000-2001 for deceased donor heart, liver, and lung transplantation were 86 percent, 86 percent, and 78 percent, respectively. The recent national 1-year patient survival rates are considerably higher than the corresponding Medicare 1-year patient survival standards of 73 percent for heart, 77 percent for deceased donor liver, and 69 percent for lung transplantation. It seems clear that the Medicare survival criteria currently used for Medicare approval of heart, liver, and lung centers would not be appropriate under an outcome-oriented set of standards.

We believe it is necessary for us to establish outcome measure requirements for transplant centers to protect patient safety and, given the scarcity of donor organs, to ensure that donor organs, once recovered, are transplanted effectively and are not wasted. In an effort to assure that transplant centers furnish transplantation services efficiently, we believe we need to establish a system for approval and re-approval of transplant centers that focuses on a center's outcomes. A center's outcomes serve as indicators of the center's ability to furnish transplantation services successfully. Since we are proposing a system that focuses heavily on outcomes, it is critical that the outcome standards reflect current conditions. Consequently, we are proposing significant changes in the standards that would be applicable to Medicare approval.

Moreover, we believe our responsibility to ensure that transplantation services are furnished safely and efficiently is no less important to those beneficiaries in need of kidney transplants than those in need of heart, liver, or lung transplants. Therefore, we are proposing to develop survival criteria for kidney transplant centers.

3. Proposed Outcome Measure Requirements for Heart, Kidney, Liver, and Lung Centers

It has been widely acknowledged by the transplant community that a transplant center's performance should be measured on the basis of its outcomes. However, there is no consensus on how to develop an outcome-oriented evaluation system. In developing an outcome-oriented system for evaluating center performance, some issues we considered are what types of measures should be used, how many measures to include, and whether to include both short and long-term outcomes.

The transplant community considers post-transplant outcomes, such as patient and graft survival, to be the “gold standard” for evaluating a transplant center's performance. While post-transplant outcomes, which measure the outcomes of transplant recipients, are widely accepted as meaningful measures of transplant center performance, organ transplantation is both a short and long-term experience.

We currently evaluate a center's performance on the basis of a single outcome measure, patient survival. For the purposes of this proposed rule we considered continuing to evaluate a center's performance on the basis of a single outcome measure. However, this approach could encourage centers to neglect other outcomes. For example, a kidney center might focus its efforts on ensuring that a kidney recipient survives to the detriment of the survival of the graft, since dialysis provides an alternative to death for kidney recipients with a failed graft.

Additionally, we are concerned that use of patient survival rates alone would not paint a complete picture of the quality of transplants performed at a center. While patient survival rates measure patient mortality, patient survival rates do not measure patient morbidity or the success of the actual transplantation procedure. Therefore, we are not proposing to limit outcome criteria for initial approval to patient survival; we are proposing a graft survival criterion as well.

We do not propose to use graft survival exclusively because patient survival is also an important measure for assessing a transplant center's quality. For example, if a transplant

center lost grafts only due to patient deaths, its outcomes may not be poor with respect to graft survival. However, since patient deaths are supposed to occur less frequently than graft loss due to re-transplants and dialysis, this transplant center may have a significantly lower than expected patient survival.

Therefore, we are proposing to use both graft and patient survival as outcome measures that would portray a center's actual performance more accurately. The proposed outcome measure requirements, like the other proposed requirements for initial approval, serve as one of several requirements that transplant centers seeking initial approval would have to meet in order to begin furnishing transplantation services that are covered by Medicare.

We also considered looking at both short-term and long-term outcomes, such as the 2-year statistics we currently require. However, we realize that long-term outcomes are more susceptible to exogenous factors not directly related to the transplantation procedure. After careful analysis of these issues, we propose using 1-year patient survival and 1-year graft survival (and in certain circumstances, 1-month patient survival and 1-month graft survival in lieu of 1-year patient survival and 1-year graft survival) as outcome measures for initial approval. We propose to require centers to meet both the 1-year patient survival and 1-year graft survival requirements separately. We propose to assess a transplant center's 1-year patient and graft survival by comparing a transplant center's expected 1-year patient and graft survival rate to its observed 1-year patient and graft survival rate for all transplants performed in the center, including living donor transplants if applicable. We propose to review a center's observed patient and graft survival against its expected patient and graft survival using a methodology that was developed by the SRTR and used by the OPTN. (This methodology, including its development, is discussed in detail below.) We propose to review a center's outcomes using the patient and graft survival data contained in the most recent SRTR center-specific report.

We also propose to review adult and pediatric outcomes separately if a center other than a lung transplant center requests Medicare approval to perform pediatric transplants. For most organ types, the SRTR has developed separate Cox models for calculating expected patient and graft survival statistics for adult (18 and older) and pediatric (younger than 18) patients. For lung transplants, however, the SRTR stratifies recipient outcomes using other categories—(1) patients that are 12 and older or (2) patients that are less than 12. Since most lung transplants performed on pediatric patients, which is traditionally defined as patients that are younger than 18 years old, are performed on older children, we propose to use the 1-year patient survival data on patients who are at least 12 years old to assess both adult and pediatric outcomes.

a. Proposed Outcome Evaluation Methodology

Some of the attendees in the CMS Town Hall Meeting expressed the view that transplant centers should be evaluated on the basis of risk-adjusted outcomes because risk adjustment can reduce the impact of patients' diverse risk factors on survival rates. We agree that risk adjustment addresses the potential to inadvertently penalize centers for transplanting high-risk patients or using organs from extended criteria donors. We believe risk adjustment can level the playing field for all transplant centers. As such, we propose an evaluation system that relies on the SRTR's risk-adjusted data.

The SRTR methodology, which was adopted by the OPTN's Board of Directors in June 2003, was designed to update deficiencies in prior OPTN methods. A discussion of prior methods used by the OPTN is available in the OPTN Proposal Archive, March 14, 2003-32 Proposals (Proposed Modifications to OPTN/UNOS Bylaw Appendix B (Criteria for Institutional Membership), Section III (Transplant Programs) at

http://www.optn.org/policiesAndBylaws/publicComment/proposalsArchive.asp

. The current SRTR method, which is being proposed for use by CMS, uses a three-pronged approach that takes into consideration (1) statistical certainty; (2) the value of the finding for allocating resources to perform on-site surveys; and (3) the need for taking action. This three-pronged approach provided the OPTN's Membership and Professional Standards Committee (MPSC) with a balanced tool for assessing transplant center performance without creating excessive demand on the resources of the MPSC.

Specifically, the SRTR methodology compares observed outcomes to expected outcomes using three tests: (1) The p-value to test for statistical significance, (2) the number of observed events (

i.e.

, patient deaths or graft failures) minus the number of expected events (O−E), and (3) the number of observed events divided by the number of expected events (O/E). When a transplant center crosses over the thresholds for all three tests, it is identified for further review by the OPTN.

The first prong of the three-pronged approach of the SRTR methodology is statistical certainty, which is based on assessing whether the difference between the observed number of deaths or graft failures is statistically significantly more than the expected number. Statistical tests often use p-values to distinguish whether chance can or cannot be ruled out or chance is a likely or unlikely explanation for the differences documented between two observations. The p-value measures the statistical significance (or evidence) for testing a hypothesis. Usually, this hypothesis is either that two numbers are equal to each other or that a number is different from zero. A p-value of less than 0.5 (indicating that there is less than a 5 percent chance that any observed difference offered by random chance alone) is often considered “statistically significant”. Consequently, the p-value helps to identify centers where chance is an unlikely explanation for the differences between the center's observed events and its expected events.

A low p-value generally indicates that chance is an unlikely explanation for the differences between the actual and expected outcomes. The MPSC determined that a p-value less than 0.05 would be adequate to assure the statistical certainty of the difference between the observed and expected number of deaths or graft failures.

The second prong of the three-pronged approach of the SRTR methodology is the value of the finding for allocating resources to perform on-site surveys. The number of observed events minus the number of expected events (that is, the number of patient deaths or graft failures a transplant center would expect to have based on its patient population) helps to identify centers with relatively large numbers of unexpected events. The OPTN uses the results of this test to determine how to allocate its limited resources available for the review of centers. This avoids allocation of resources to centers with only a small fraction of unexpected deaths. The SRTR proposed a threshold value for each test. The MPSC determined that the number “3” (that is, 3 more patient deaths or graft failures than expected) would be adequate to assure that there was meaningful clinical information to assess for deficiencies in a transplant center (O−E>3). Few smaller centers are expected to show statistical significance (

i.e.

, show a p-value <0.05) because, from a statistical perspective, it hard to rule out chance when working with

small numbers. Therefore, one could expect that fewer small centers than large centers potentially would be identified using the SRTR methodology.

The OPTN MPSC recognized that it would need to be able to appropriately flag smaller cohorts, especially since the center-specific reports separate adult and pediatric transplants. As such, in 2001, the SRTR presented some analyses that would help the OPTN MPSC decide upon the minimum number of transplants needed in order for the SRTR methodology to flag smaller cohorts. Transplant centers that performed fewer transplants than this minimum number would not be reviewed using the SRTR methodology.

Although a single death has a much greater impact on a center's patient survival rate in a smaller center than in a larger center, the OPTN MPSC felt that the percentage difference when working with smaller cohorts was less useful from a clinical perspective because of the smaller numbers. For example, a transplant center that performs 10 transplants and loses 1 graft has a 90 percent survival rate whereas a center that performs 11 transplants and loses 2 grafts has an 82 percent survival rate. Although the difference between 90 percent and 82 percent may appear to be significant, when only 10 transplants have been performed, the absolute difference between the loss of 1 graft and 2 grafts is small. The MPSC felt that this type of difference was not sufficient to distinguish small cohorts. Therefore, the MPSC asked the SRTR to help them determine the minimum number of transplants required for the SRTR methodology to flag a transplant center and to have that “flag” be clinically appropriate.

In deciding upon the minimum number of transplants required for use of the SRTR methodology, the OPTN recognized that small transplant centers had to have a minimum excess of graft failures/deaths before there was adequate clinical information to evaluate for deficiencies in the transplant center. Since the minimum number of excess graft failures/deaths was determined to be 3, a transplant center would have to perform at least 4 transplants in order to have an excess of 3 deaths. However, performing 4 transplants and having a 100% graft failure/death rate was not clinically acceptable. Therefore, the SRTR developed a scenario in which a transplant center's expected graft failure/mortality rate was 10 percent, but its actual graft failure/mortality rate was 50 percent. Using this scenario, the SRTR methodology could flag cohorts as small as 8 transplants. Based on this finding, the OPTN MPSC decided to use the SRTR methodology on cohorts (adult or pediatric) of at least 9 transplants. As the number of transplants increase, the clinical concordance of observed and expected mortality rates should also increase.

The third prong of the approach of the SRTR methodology is the need for taking action. The MPSC determined that it would need to take action when it determined that the observed number of deaths or graft failures was 50 percent more than expected (O/E>1.5).

We applaud the SRTR's effort to strive for better ways to identify under-performing transplant centers. We have carefully reviewed and evaluated the SRTR's methodology for flagging under-performing transplant centers. We believe the SRTR approach to handling small centers is reasonable. To address concerns that the methodology could be perceived as being more lenient towards smaller centers, we analyzed transplant center data from the most recent SRTR center-specific report and found that it flagged centers of all size ranges. Of the 72 small centers (9-25 transplants), 15% were flagged.

Adult Programs Flagged Based on Center Size

Center size

Number of programs

(1)

Number of programs flagged (patient/graft/both)

(2)

Flagged/program

(2)/(1)

<9

71

0 (0.0%)

0%

9-25

72

11 (20.4%)

15.3%

26-50

98

11 (20.4%)

11.2%

51-100

121

13 (24.1%)

10.7%

101-200

111

15 (27.8%)

13.5%

201-500

60

3 (5.6%)

5.0%

>500

8

1 (1.9%)

12.5%

Total

541

54 (100.0%)

We believe that the analyses we conducted shows that the p-value test performs very well for centers with at least 9 transplants. Given the fact that an adult center has to have performed 9 transplants in order to enable the SRTR methodology to capture differences during the 2.5 year cohort period, we believe the SRTR methodology can maintain a delicate balance between able to identify the outliers in both large and small centers. We are requesting comments on the appropriateness of proposing this approach.

We propose adapting the general framework of the SRTR methodology to assess a heart, liver, lung, or kidney transplant center's outcomes for our use. That is, we propose that if a transplant center's observed 1-year patient survival rate and 1-year graft survival rate is lower than the expected 1-year patient survival rate and 1-year graft survival rate, respectively, we would use the three SRTR tests (p-value, O−E, and O/E) to determine whether a center's observed survival rates were unacceptably low and whether thus the center would require CMS follow up.

For each of the outcome measures we proposed for initial approval of heart, liver, lung, and kidney centers, we propose establishing minimum thresholds for the p-value, O−E, and O/E tests. One of the primary concerns expressed by beneficiaries at our Town Hall Meeting was access to their choice of transplant centers. Therefore, we want to establish a mechanism whereby all transplant centers that perform at or near their expected outcomes are able to obtain initial Medicare approval for transplantation. We recognize that the threshold we establish for each test would affect the quality of care, number and location of centers, and access to centers. It is our goal to establish thresholds to ensure access while ensuring that Medicare beneficiaries receive high quality organ transplantation services. After careful evaluation of SRTR's analysis and OPTN's reasoning, we propose to adopt

thresholds that mirror those adopted by the OPTN.

Specifically, for each outcome measure, we propose considering the center's patient and graft survival rate to be acceptable as long as the center's observed patient and graft survival rate is higher than the center's expected patient and graft survival rate. If a center's observed patient and graft survival is lower than its expected patient or graft survival, we would still consider the center's patient and graft survival rate to be acceptable, unless all three of the following thresholds are crossed over:

• The one-sided p-value is less than 0.05;

• The number of observed events minus the number of expected events (O−E) is greater than 3; and

• The number of observed events divided by the number of expected events (O/E) is greater than 1.5.

Our justification for these thresholds is the same as that of the OPTN when it adopted the thresholds in June 2003. A one-sided p-value less than 0.05 can loosely be interpreted to mean that there is a 95 percent probability that the difference between a center's observed patient or graft survival rate cannot be explained by random fluctuations. Therefore, we believe that establishing the threshold for the p-value at 0.05 provides us with reasonable assurance that a transplant center's observed patient or graft survival rate truly cannot be attributed to external factors that may also influence patient or graft survival, as opposed to being the result of a random fluctuation (

i.e.

the difference between the observed and expected is statistically significant). A difference between the observed number of events (

i.e.

, patient deaths or graft failures) and the number of expected events that is greater than 3 indicates that 3 or more of the observed events were unexpected. In establishing the threshold for the O−E test at 3, our goal was to strike a balance between establishing a threshold that is high enough to avoid identifying centers where the absolute number of unexpected events is very small and establishing a threshold that is low enough to reflect that a non-trivial number of patients were affected. When the quotient of the number of observed events divided by the number of expected events is greater than 1.5, this indicates that a substantial fraction (more than 50 percent) of the observed events were unexpected. Therefore, the proposed thresholds for the O−E and O/E tests help to identify centers in which a relatively large portion of the center's transplants resulted in an unexpected adverse outcome (

i.e.

, patient death or graft failure).

For each outcome measure, we propose that only when a heart, liver, lung, or kidney center crosses over the thresholds established for all three tests, would we consider the center not to be in compliance with the requirements for that particular outcome measure. For example, we would consider a center that demonstrates a p-value of 1.00, O−E of 5.0, and O/E of 2.0 based on the 1-year patient survival data contained in the most recent SRTR center-specific report to meet the patient survival requirement because one of the three thresholds (that for the p-value test) was not crossed over. On the other hand, a center that demonstrates a p-value of 0.01, O−E of 5.0, and O/E of 1.9 for its patient survival data would cross over the thresholds for all three tests; therefore, we would not consider the patient survival requirement to be met.

Transplant centers would have to meet the requirements for each of the outcome measures (

i.e.,

patient survival and graft survival) separately. In other words, a center that meets the requirements for patient survival but not for 1-year graft survival would not meet the proposed outcome measure requirements. By considering centers whose observed outcomes are lower than their expected outcomes to be acceptable unless they cross over the thresholds for all three tests, we believe that we can be reasonably assured that any center identified using this methodology will have both a statistically significant and non-trivial number of unexpected deaths or graft failures. Centers in which the number of unexpected events is relatively large but not statistically significant or in which the number of unexpected events is statistically significant but relatively small would not be inadvertently penalized under this proposed methodology.

We are proposing that an adult transplant center requesting Medicare approval would have to have 1-year patient and 1-year graft survival follow-up data on at least 9 transplants of the appropriate organ type during the 2.5-year period reported in the most recent center-specific report. In other words, centers that perform fewer than 9 transplants generally would not be eligible for Medicare approval under our proposal. We are asking for comments as whether requiring the minimum number of 9 transplants during the 2.5-year period is acceptable for this application of the SRTR methodology.

CMS is cognizant that requiring a minimum number of transplants may appear to limit access to transplantation for Medicare beneficiaries. However, given that the proposed minimum number of transplants of 9 is lower than the current Medicare requirements (12 transplants over a 12-month period for heart and liver transplant centers, and 10 transplants over a 12-month period for lung and intestinal transplant centers), we do not believe this requirement would lessen current access to transplant centers. As stated earlier, our analysis of the most recent SRTR center-specific reports indicates that approximately 71 adult transplant centers performed fewer than 9 transplants in the most recent 2.5-year period. It appears that the majority of the smaller cohorts involved pediatric cases, transplant centers at children's hospitals, or centers in transition. After careful analyses, we found that 45 of those centers were the adult component of a pediatric center, which does not have to meet the proposed volume requirement. Of the remaining 26 centers, only 11 are currently active according to the records of the OPTN. Of those 11 centers, there are 5 heart centers, 1 kidney center, 2 liver centers and 3 lung centers. Also, four centers have 7-8 transplants (and could easily reach 9 transplants); 2 centers are affiliated with a large transplant center; one center recently opened; and 2 centers are located in cities with a nearby transplant center.

OPTN requirements are similar to those we propose. The OPTN currently requires that heart, kidney and liver transplant centers perform a minimum of one transplant every 3 months, which equals approximately 9-10 transplants over the course of 2.5 years. Although lung transplant programs are required to perform a transplant only once every 6 months, there were only 3 lung centers that did not perform at least 9 transplants.

Given the very specialized care that needs to be provided to children, as well as the relatively few children who are Medicare beneficiaries, we did not want to restrict access to this group by setting a volume threshold that was inappropriately high. Although we have stated we would review pediatric outcomes separately if a transplant center requests Medicare approval to perform pediatric transplants, we propose not to require such centers to perform a minimum number of pediatric transplants prior to their request for approval. Most centers that would request Medicare approval to perform pediatric outcomes are likely to perform only 2 or 3 transplants per year. Analyses conducted by HRSA's DoT staff indicate that a minimum volume requirement that would still allow the

SRTR's methodology to flag poor-performing centers would preclude most children's hospitals from being able to request Medicare approval. The OPTN, also recognizing the infrequency of pediatric transplantation, requires that only one transplant per year be performed to demonstrate that the pediatric center is functionally active. We request comments on this proposal.

We recognize that there may be some concerns related to our proposed minimum number criterion because the current Medicare volume standards for heart, liver, lung, and intestinal centers are higher. Medicare currently requires heart and liver transplant centers to perform 12 transplants over a 12-month period, and lung and intestinal transplant centers to perform 10 transplants over a 12-month period. Historically, we have used volume as a proxy for outcome. Since we now have risk-adjusted outcome measures, we believe it would be insufficient to propose a volume standard that would be viewed as arbitrary or unscientific. Instead, our volume requirement should only reflect the minimum number of transplants needed for the SRTR to be able to flag a poor-performing center, that is, 9 transplants performed during the reporting period.

If a heart center is requesting Medicare approval in December 2004, we would rely on the 1-year patient and graft survival data contained in the July 2004 SRTR center-specific report. Since the July 2004 report contains 1-year patient and graft survival data on transplants performed between January 1, 2001 and December 31, 2002, we would expect that the July 2004 center-specific report include 1-year patient and graft survival information on at least 9 heart transplants that were performed between January 1, 2001 and December 31, 2002. Meanwhile, a kidney transplant center that requests Medicare approval in December 2004 would be expected to have 1-year patient and graft survival follow-up information on at least 9 kidney transplants that were performed between January 1, 2001 and June 30, 2003, since the SRTR used a 2.5-year cohort in the July 2004 center-specific report to report patient and graft survival statistics for abdominal organs.

This lower volume criterion may also raise the concern that a center could perform 9 transplants quickly and then not perform a transplant for 12 months and yet become or remain Medicare approved. However, we believe this scenario is unlikely to occur because of additional oversight provided through the OPTN. In 1996, the MPSC of the OPTN proposed changes to the bylaws that would define a functionally inactive transplant center's responsibility to patients on the waiting list. In order to identify such centers, the MPSC set forth criteria that would trigger further investigation of transplant center functional inactivity. Initially, the MPSC considered a transplant center to be functionally inactive if it did not perform a transplant within a 3-month period. As the MPSC has gained greater understanding of the impact of the organ procurement and allocation process on a center's ability to perform transplants, it has revised the initial criteria for determining whether a center is functionally active: for heart, liver and kidney centers—a transplant every 3 months; for lung centers—a transplant every 6 months; for children's hospitals—a transplant once a year. In addition to these frequency standards, the MPSC also reviews organ offers and turndowns at centers that have not performed a transplant recently to determine whether the reason for inactivity is due to lack of suitable organ offers or inadequate resources at the transplant center. If the OPTN determines that a transplant center is functionally inactive, the transplant center is no longer eligible to receive organs for transplantation, and therefore, can no longer perform transplants. These OPTN reviews offer additional oversight to assure the public and Medicare that the organ transplant centers are truly functionally active at the time of Medicare approval and re-approval. We request comments on our proposal to focus more heavily on a center's outcomes by eliminating volume as a separate standard and integrating volume into our outcomes assessment.

b. Evaluation of Alternatives to the SRTR Methodology

Based on our analysis of the July 2004 SRTR center-specific reports, we believe that a majority of the heart, kidney, liver, and lung centers would be able to meet the proposed 1-year patient and 1-year graft survival requirements. Using data from the July 2004 SRTR center-specific reports, approximately 10.0 percent of all heart, kidney, liver, and lung centers that perform adult transplants have observed outcomes that are lower than their expected outcomes and cross over the proposed thresholds for the three tests in terms of both 1-year patient survival and 1-year graft survival. In other words, if all heart, kidney, liver, and lung centers that perform adult transplants were to seek initial Medicare approval simultaneously, approximately 10.0 percent of the 541 heart, kidney, liver, and lung centers that perform adult transplants would not be able to meet the proposed outcome measure requirements. Also, approximately 1.9 percent of the 309 heart, liver, lung, and kidney centers that perform pediatric transplants have observed outcomes that are lower than their expected outcomes and meet the proposed thresholds for all three tests. We invite comments on the proposed outcome measures and their thresholds. We specifically solicit data and evidence that may support alternative thresholds, especially thresholds that may be specific to a particular organ transplant type.

We also welcome comments on the methodology itself. We understand that the OPTN continuously reviews this methodology and may make modifications to the methodology or the thresholds for the three tests in the future. In the event that the OPTN decides to modify the methodology or any of the thresholds currently used, we would consider adopting the modified methodology or thresholds through notice and comment rulemaking.

In addition, we explored two options for applying the SRTR methodology. We would like to take this opportunity to welcome comments on these other options as well. In one option, a heart, kidney, liver, or lung center whose observed outcomes are lower than its expected outcomes would be considered to have unacceptable outcomes if it met the proposed thresholds for just two of the three tests (hereafter referred to as option 1. When we analyzed the data in the July 2004 SRTR center-specific reports, we discovered that option 1 would identify approximately 15.7 percent of the heart, kidney, liver, and lung centers that perform adult transplants and 4.2 percent of the heart, kidney, liver, and lung centers that perform pediatric transplants.

A second option consists of considering a center's outcomes to be unacceptable if its observed outcomes are lower than its expected outcomes and the center met the proposed threshold for just one of the three tests (hereafter referred to as option 2. If option 2 were selected, approximately 41.6 percent of the heart, kidney, liver, and lung centers that perform adult transplants would fail to meet the proposed 1-year patient survival and 1-year graft survival requirements and approximately 67.0 percent of the heart, kidney, liver, and lung centers that perform pediatric transplants would fail to meet the proposed 1-year patient survival and 1-year graft survival requirements.

Considering a transplant center's outcomes to be unacceptable when the center's observed outcomes are lower than its expected outcomes and the center crosses over the proposed threshold for just one or two of the three tests is more stringent than our proposal. However, we are concerned that under this option, we would be conducting inspections on centers where the differences between the observed and expected events are relatively large but not statistically significant, thus diverting resources that should be expended surveying centers where the differences between the observed and expected events are both large and statistically significant. Therefore, we are proposing to consider a center's outcomes to be unacceptable only when a center's observed outcomes are lower than its expected outcomes and the center crosses over the proposed thresholds for all three tests. We are inviting comments on the merits of our proposed approach.

For comparison, we have summarized the results of our analysis of the effects of our proposal as well as options 1 and 2 in the table below. We used data from the July 2004 center-specific reports to perform this analysis. We did not, however, screen out centers that performed fewer than 9 adult transplants when we conducted this analysis. Therefore, some of the centers that perform adult transplant that were identified using the proposed methodology or using option 1 or option 2 may not be eligible to request Medicare approval because they did not perform 9 adult transplants during the 2.5-year period reported in the July 2004 center-specific reports.

Number and Percent of Centers Identified as Failing To Meet Proposed Outcome Measure Requirements Under Proposal and Options 1 and 2, By Organ and Transplant Type (Adult or Pediatric)

Organ type

Number (n) and percent (%) of centers identified using:

Adult transplants

Proposal

n

%

Option 1

n

%

Option 2

n

%

Pediatric transplants

Proposal

n

%

Option 1

n

%

Option 2

n

%

Heart

11

8.7

20

15.9

45

35.7

0

0.0

3

4.4

18

26.5

Liver

11

10.3

15

14.0

43

40.2

3

4.2

4

5.6

19

26.8

Lung

7

10.0

8

11.4

25

35.7

0

0.0

0

0.0

10

100.0

Kidney

25

10.5

42

17.6

112

47.1

3

1.9

6

3.8

160

100.0

All Organs

54

10.0

85

15.7

225

41.6

6

1.9

13

4.2

207

67.0

c. Special Circumstances in Which 1-Month Patient and 1-Month Graft Survival May Be Used in Lieu of 1-Year Patient and 1-Year Graft Survival

We are also proposing that, under certain circumstances, we would review a center's outcomes using 1-month post-transplant data in lieu of 1-year post-transplant data. We recognize that transplant teams sometimes move from one hospital to another to open a new transplant center. It is not uncommon for new centers staffed with an experienced team to have good outcomes. These new centers that request Medicare approval may not have 1-year patient and graft survival data (including follow-up data from at least 9 adult transplants performed during the 2.5-year period reported in the SRTR center-specific reports). At a minimum, 1-month post-transplant data can demonstrate the success of the transplantation procedure as well as the skill of the transplantation team. We believe that in the absence of 1-year post-transplant outcomes, 1-month post-transplant outcomes can capture early graft and patient deaths due to poor transplantation skills and poor donor and/or recipient selection. These data are important in the assessment of a new transplant center.

Therefore, we are proposing that a new transplant center may request initial approval using 1-month patient and 1-month graft survival data if the key members of the center's transplant team performed transplants at a Medicare-approved transplant center for a minimum of 1 year prior to the opening of the new center and if the transplant center's team meets the human resources requirements at § 482.98. If these specific conditions are not met, the new center must be reviewed using 1-year post-transplant patient and graft survival follow-up data. A new center with an experienced team requesting initial Medicare-approval that does not have 1-year patient and graft survival follow-up data (including 1-year follow-up data on at least 9 adult transplants for centers requesting Medicare approval to perform adult transplants) in the most recent SRTR center-specific report would have to ask the SRTR to generate a customized report of the center's 1-month patient and 1-month graft survival statistics for all transplants performed in the previous 1-year period. The SRTR would generate these customized reports using the same models as those used to generate the center-specific reports.

When 1-month post-transplant outcomes are used, we would review the center's 1-month patient and graft survival rates for all transplants performed at the center during the previous 1-year period using customized reports. We would evaluate the center's 1-month outcomes using the same SRTR methodology that we propose for evaluating transplant centers' 1-year outcomes. The transplant center would need to have follow-up data on at least 9 transplants of the appropriate organ type. Instead of 1-year follow-up data on at least 9 transplants performed at the center during the 2.5-year period reported in the SRTR center-specific reports, however, the center would need a customized report with 1-month follow-up data on at least 9 transplants performed during the previous 1-year period.

Centers which gain Medicare approval based on 1 month data would be reevaluated based on 1 year data when it became available. We are requesting comments on the frequency with which we should assess these centers after they are approved.

If a center other than a lung transplant center requests Medicare approval to perform pediatric transplants on the basis of its 1-month patient and graft survival data, we would continue to review the adult and pediatric outcomes separately. We do not propose a volume criterion for approving centers to perform pediatric transplants when a center's 1-year patient and graft survival data are used. Therefore, we do not propose a volume criterion for Medicare approval of a center to perform pediatric

transplants when 1-month patient and graft survival data are used.

4. Proposed Outcome Measure Requirements for Heart-Lung, Intestine, and Pancreas Centers

Due to the limited volume of heart-lung, intestinal, and pancreas transplants performed nationwide, the OPTN has not been able to gather enough transplant data on these organ types for the SRTR to develop Cox models for calculating expected survival statistics for these types of transplants. We prefer not to gauge a transplant center's performance on the basis of unadjusted data. Unadjusted data, or a center's observed outcomes, do not take into account variation among transplant centers, such as differences in patient case-mix. We believe evaluating a transplant center on the basis of unadjusted data could potentially discourage centers from performing transplants on severely ill or high-risk patients. Therefore, for heart-lung, intestinal, and pancreas transplant centers, we propose no outcome measure requirements at this time. In the event that the SRTR develops risk-adjustment models for heart-lung, intestinal, or pancreas transplant survival rates in the future, we will consider establishing outcome measure requirements for heart-lung, intestinal, or pancreas transplant centers through rulemaking.

When the Medicare coverage criteria for heart transplants were published in 1987, heart-lung transplants were considered to be experimental and were not covered by Medicare. When the Medicare coverage criteria for lung transplants were published in 1995, we stated that Medicare would cover heart-lung transplants for beneficiaries with progressive end-stage cardiopulmonary disease when they were provided in a facility that was approved by Medicare for both heart and lung transplantation. Although Medicare began covering heart-lung transplants as well as single and double lung transplants, we did not establish separate survival criteria for heart-lung transplants. Instead, lung centers were required to have an aggregate 1-year survival rate of 69 percent and an aggregate 2-year survival rate of 62 percent. In calculating its survival rates, centers were asked to include single and double lung transplants, as well as heart-lung transplants.

When the SRTR calculates statistics for lung transplants, however, the SRTR does not include heart-lung transplants because there is a separate category of data for heart-lung transplants. Even though the SRTR has a separate category for heart-lung transplant data, the data are not risk-adjusted. We propose that a heart-lung center, as defined in the proposed definition for a “heart-lung transplant center,” would need to meet just the proposed data submission requirements to be compliant with the proposed Data Submission and Outcome Requirements for Initial Approval of Transplant Centers CoP. In light of the proposed definition for “heart-lung transplant center,” which requires heart-lung centers to be located in a hospital that has Medicare-approval to perform both heart and lung transplants, and the fact that only 33 heart-lung transplants were performed in the U.S. in 2002, we believe that we would have reasonable assurance that the heart-lung center has sufficient expertise to perform heart-lung transplants successfully. We believe skill and expertise in both heart and lung transplantation are sufficient for ensuring that a center is able to perform high quality heart-lung transplants and that separate patient and graft survival rate criteria for heart-lung centers would not be necessary. Again, we request comments on the appropriateness of this approach for evaluating heart-lung transplant centers, as well as alternatives to this approach.

The Medicare coverage decision for multivisceral and intestinal transplants was issued on October 4, 2000 and only covers services provided on or after April 1, 2001. Since only 299 intestinal transplants were performed from 2000 through 2002, it is probable that the current Medicare 1-year patient survival threshold of 65 percent for intestinal transplants continues to be relevant. We are reluctant to establish outcome measure requirements on the basis of unadjusted data. Unlike heart-lung centers, intestinal centers do not have to be affiliated with any other type of center under current Medicare requirements. Historically, however, intestinal centers have evolved as an extension from the liver transplant centers. In 2002, there were 107 intestinal transplants, of which only 44 were intestine alone transplants. Given the historical affiliation of intestinal transplant centers with liver transplant centers and the very small number of intestinal transplants being performed, we are proposing that there not be any outcomes or volume criteria for intestinal transplantation. We believe that the proposed definition for “intestinal transplant center,” which requires transplant centers to be located in a hospital that has Medicare approval to perform liver transplants, would be sufficient. Intestinal transplant centers would need to meet the proposed data submission requirements. We are requesting comment on the appropriateness of the proposal to approving intestinal transplant centers in light of the absence of risk-adjusted outcomes data for intestinal transplantation, the very low frequency of this type of procedure, and potential concerns that setting volume standards would further limit access to a rare procedure.

Of the 1,369 deceased donor pancreas transplants performed in the United States in 2003, 502 were performed alone or subsequent to a kidney transplant and 867 were performed simultaneously with a kidney transplant (

i.e.,

kidney-pancreas transplants). According to the July 2003 SRTR national summary report, the national mean 1-year patient survival rate for adult pancreas transplants performed alone or subsequent to a kidney transplant is 96.01 percent and the national mean 1-year graft survival rate is 78.34 percent. Since the number of pancreas transplants performed alone or subsequent to a kidney transplant is very small, the outcomes are generally very good, and the SRTR has not established a risk-adjustment model for pancreas transplants performed alone or subsequent to a kidney transplant, we do not propose any outcome measure requirements for pancreas transplant centers. We believe that the proposed definition for “pancreas transplant center,” which requires transplant centers to be located in a hospital that has Medicare approval to perform kidney transplants, would be sufficient. As with heart-lung and intestinal transplant centers, a pancreas transplant center would still need to meet the data submission requirements to be in compliance with the proposed Data Submission or Outcome Requirements for Initial Approval of Transplant Centers CoP at § 482.80. We request comments on the appropriateness of this approach to evaluating pancreas transplant centers in light of the lack of risk-adjusted data for pancreas transplants that are performed alone or subsequent to a kidney transplant.

We note that these standards would not apply to infusions of pancreatic islet cells, a procedure sometimes termed “islet cell transplantation”. Under section 733 of the Medicare Prescription, Drug Improvement, and Modernization Act (MMA) (Pub. L. 108-173), Medicare pays for some investigational islet transplantation procedures. Our pancreas standards would be inappropriate for these islet procedures which do not involve a whole organ or require the same skills

and expertise as surgical transplantation of whole organs.

D. Summary of Proposed Data Submission and Outcome Measure Requirements for Initial Approval, by Organ Type

Since the requirements proposed in § 482.80 vary by organ type, the following table summarizes the data submission and outcome measure requirements that each type of organ transplant center would have to meet under this proposed CoP.

Type of center

Proposed data submission and outcome measure requirements for initial approval

Heart, Kidney, Liver, or Lung

• Timely submission of at least 95 percent of required data on all transplants

1

performed to OPTN; and

• As long as a center has 1-year post-transplant follow-up on at least 9 transplants that were performed during the 2.5-year period reported in the most recent SRTR center-specific report and the center's observed 1-year patient and graft survival rate is higher than its expected 1-year patient and graft survival rate, the center's outcomes would be acceptable.

• If the center's observed 1-year patient and graft survival rate is lower than its expected 1-year patient and graft survival rate, the center's patient and graft survival could still be acceptable, unless all 3 of the following thresholds are crossed:

(1) p-value < 0.05,

(2) O−E > 3, and

(3) O/E > 1.5.

Heart-lung

• Timely submission of at least 95 percent of required data on all heart-lung transplants performed to OPTN.

Intestine

• Timely submission of at least 95 percent of required data on all intestinal, combined liver-intestinal, and multivisceral transplants performed to OPTN.

Pancreas

• Timely submission to the OPTN of at least 95 percent of required data on all pancreas and kidney-pancreas transplants performed.

Condition of Participation: Data Submission, and Outcome Measure Requirements for Re-approval of Transplant Centers (Proposed § 482.82)

A. Overview

The current Medicare policies on organ transplants do not have criteria for re-approval of transplant centers. In 2000, 37 percent of Medicare-approved heart transplant centers fell below the Medicare-required volume or survival rate criteria and yet still retained their Medicare-approved status. We believe there is a need to establish criteria for evaluating the ongoing performance of Medicare-approved transplant centers, including post-approval criteria for data submission and outcomes. Without these criteria, we are unable to be assured that once a transplant center becomes Medicare-approved it continues to provide transplantation services in a safe and efficient manner. Given that outcome measures are important indicators of transplantation quality, periodic re-assessment of these indicators, along with the requirement for complete and timely submission of data, would serve as a valuable oversight tool for ensuring that once a transplant center becomes Medicare-approved, it can continually demonstrate a minimum level of commitment to and expertise in transplantation. Therefore, we are proposing specific data submission and outcome requirements for re-approval.

1

Each transplant center must submit data on all transplants performed at the center, including living donor transplants if applicable, because CMS will review outcomes for all transplants of the appropriate organ type performed at the center.

B. Proposed Data Submission Requirements for Re-approval of Transplant Centers

As we proposed for initial approval, we also propose that no later than 90 days after the due date established by the OPTN, heart, heart-lung, intestine, kidney, liver, lung, and pancreas transplant centers must submit to the OPTN at least 95 percent of required data submissions on all transplants (deceased and living donor) performed at the center over the 3-year approval period. As in initial approval, we propose required data submissions include, but not be limited to, submission of the appropriate OPTN forms for transplant candidate registration, transplant recipient registration, and transplant recipient follow-up for the type of organ(s) transplanted.

C. Proposed Outcome Measure Requirements for Re-approval of Transplant Centers

We propose using the same outcome measures for the re-approval of heart, kidney, liver, and lung centers that we propose for initial approval of these centers. However, while we proposed to give transplant centers the option of using 1-month post-transplant outcomes under certain conditions for initial approval, we are not proposing a similar option for re-approval. Each heart, kidney, liver, and lung center would have to use 1-year patient and graft survival data contained in the most recent SRTR center-specific report for re-approval. We would also review outcomes for all transplants performed at the center, including living donor transplants, if applicable.

Furthermore, each heart, kidney, liver, and lung center that has Medicare approval to perform adult transplants would need to have 1-year post-transplant follow-up on at least 9 adult transplants of the appropriate organ type performed during the 2.5-year period reported in the most recent SRTR center-specific report. Except for lung transplant centers, we would review outcomes for pediatric and adult patients separately if a center has Medicare approval to perform pediatric transplants. As with initial approval, transplant centers that have Medicare approval to perform pediatric transplants would not need to perform a minimum number of pediatric transplants. As we stated earlier, requiring centers to perform a minimum number of pediatric transplants would preclude many centers from obtaining Medicare approval to perform pediatric transplants. Again we request comments on our proposed approach to evaluating pediatric transplant centers' outcomes.

For the same reasons discussed for the proposed outcome measure requirements for initial approval, we also propose adopting the same methodology for evaluating a heart, kidney, liver, or lung transplant center's outcomes that we propose for initial approval. As long as the center's

observed outcomes are higher than the center's expected outcomes, the center's outcomes would be acceptable. If a center's observed outcomes are lower than its expected outcomes, the center's patient and graft survival could still be acceptable, unless all of the following three thresholds are crossed:

• The one-sided p-value is less than 0.05;

• The number of observed events minus the number of expected events (O−E) is greater than 3; and

• The number of observed events divided by the number of expected events (O/E) is greater than 1.5.

Again, we propose that when a center's observed patient and graft survival is lower than the expected patient and graft survival and the center crosses over all three thresholds for a particular outcome measure, we would not consider the center to be in compliance with the requirements for that particular measure. Centers still would have to meet the outcome requirements for each outcome measure separately. In other words, a heart, kidney, liver, or lung center in which both the observed 1-year patient survival rate and the observed 1-year graft survival rates are lower than the expected survival rates would have acceptable outcomes unless the center crosses the thresholds for all three tests (

i.e.

, p-value, O−E, and O/E) with respect to its observed and expected 1-year patient survival rates and with respect to its observed and expected 1-year graft survival rates.

We welcome comments on the proposed thresholds for re-approval of heart, kidney, liver, and lung centers and on the methodology itself. Given that failure to meet the outcome measure requirements would not necessarily result in denial of re-approval, as it would for initial approval, we specifically request comments on whether we should consider a heart, kidney, liver, or lung center's outcomes to be unacceptable if the center crosses the thresholds for all three tests as proposed or whether we should consider a heart, kidney, liver, or lung center's outcomes to be unacceptable if the center crosses the thresholds for just one or two of the three tests, as discussed earlier.

For re-approval of heart-lung, intestinal, and pancreas centers, we propose the same requirements as we do for initial approval of heart-lung, intestinal, and pancreas centers. For heart-lung, intestinal and pancreas transplant centers, we do not propose any outcome measure requirements since we feel that at this time skill and expertise in heart and lung transplantation, in liver transplantation, and in kidney transplantation, respectively, are sufficient. We request comments on our proposed approach to evaluating heart-lung, intestine, and pancreas transplant centers' outcomes.

D. Summary of Proposed Data Submission and Outcome Requirements for Re-Approval, by Organ Type

Since the proposed data submission and outcome requirements for re-approval vary by organ type, the following table summarizes the data submission and outcome measure requirements that each type of organ transplant center would have to meet under this CoP.

2

Each transplant center must submit data on all transplants performed at the center, including living donor transplants if applicable, because CMS will review outcomes for all transplants of the appropriate organ type performed at the center.

Type of center

Proposed data submission and outcome measure requirements for re-approval

Heart, Kidney, Liver, or Lung

• Timely submission of at least 95 percent of required data on all transplants

2

performed to OPTN; and

• As long as a center has 1-year post-transplant follow-up on at least 9 transplants that were performed during the 2.5-year period reported in the most recent SRTR center-specific report and the center's observed 1-year patient and graft survival rate is higher than its expected 1-year patient and graft survival rate, the center's outcomes would be acceptable.

• If the center's observed 1-year patient and graft survival rate is lower than its expected 1-year patient and graft survival rate, the center's patient and graft survival would still be acceptable, unless all 3 of the following thresholds were crossed:

(1) p-value < 0.05,

(2) O−E > 3, and

(3) O/E > 1.5.

Heart-lung

• Timely submission of at least 95 percent of required data on all heart-lung transplants performed to OPTN.

Intestine

• Timely submission of at least 95 percent of required data on all intestinal, combined liver-intestinal, and multivisceral transplants performed to OPTN.

Pancreas

• Timely submission to the OPTN of at least 95 percent of required data on all pancreas and kidney-pancreas transplants performed.

Proposed Transplant Center Process Requirements

A. Overview

We believe sound policies and processes are keys to ensuring quality care for patients. State agency surveys of hospitals with transplant centers indicate that deficiencies are usually associated with inadequate or poor implementation of patient management policies and procedures, inadequate staffing, and poor or inadequate monitoring of QAPI programs. We believe it is critical to include process-oriented requirements in the regulation in addition to data submission and outcome requirements. The combination of outcome-oriented and process-oriented requirements will enhance efficient usage of donated organs and thereby decrease organ wastage. The process requirements that we are proposing promote efficiency in the Medicare program and are based heavily on accepted standards of practice in the transplantation field and on continuous quality improvement efforts that have been proven to improve outcomes. To reduce burden on providers, we are revising or eliminating specific requirements that currently apply to heart, kidney, liver, and lung centers and proposing only requirements that will ensure the overall quality of transplant centers for all transplant types. Proposing that transplant centers meet process requirements is intended to promote the quality of transplant services.

The well-being of living donors is as important as the well-being of transplant recipients. Consequently, based on the Secretary's authority under section 1861(e)(9) of the Act to require hospitals to meet requirements “necessary in the interest of the health and safety of individuals who are

furnished services in the institution,” we have proposed several process requirements we believe are necessary to protect the health and safety of prospective living donors.

B. Current Requirements

Currently, kidney transplant centers are covered under applicable regulations in § 405.2135 through § 405.2160 and specific kidney transplant regulations in § 405.2170 through § 405.2171. The current regulations for kidney transplant centers require, among other things, a kidney transplant center to be under the general supervision of a qualified transplant surgeon or a qualified physician-director, serving as the director of renal transplantation and responsible for the following: (1) Participating in the selection of suitable treatment modalities for each patient; (2) ensuring adequate training of nurses in the care of transplant patients; (3) ensuring tissue typing and organ procurement are available either directly or under arrangement; and (4) ensuring transplantation surgery is performed under the direct supervision of a qualified transplantation surgeon(§ 405.2170).

The regulations also require a kidney transplant center to meet specific minimal service requirements: (1) Be part of a Medicare certified and participating hospital; (2) participate in a patient registry program with an OPO certified or recertified under part 486, subpart G ; (3) be under the supervision of the hospital administrator and medical staff; (4) utilize a qualified social worker to evaluate transplant patients' psychosocial needs, participate in care planning of the patients and identify community resources to assist the patient and family; (5) utilize a qualified dietitian who will, in consultation with the attending physician, assess the nutritional and dietetic needs of each patient, prescribe therapeutic diets, provide diet counseling to patients and their families, and monitor adherence and response to a prescribed diet; (6) utilize a laboratory that is approved under 42 CFR Part 493 and that can perform histocompatibility testing on a 24-hour emergency basis, and (7) utilize the services of a designated organ procurement organization(§ 405.2171).

The current Medicare transplant policies for heart, liver, and lung centers have specific process requirements for patient selection, patient management, commitment, facility plans, maintenance of data, organ procurement, laboratory services, and billing.

C. Proposed Process Requirements

Our goals in developing the CoPs are to ensure the quality of care provided in transplant centers and to increase the number of successful transplants. We believe that the OPTN also shares these goals. We believe it will be beneficial for us to adopt certain aspects of the OPTN policies, as they are specific to current practice, in our proposed process requirements. We specifically invite comments on this proposal.

To keep process-oriented requirements to a minimum and to reduce burden on providers, we are proposing only requirements that are directly related to patient outcomes or that are necessary for data collection purposes to ensure the efficient operation of the Medicare program. We propose that our process requirements address the following subjects: (1) Patient and living donor selection, (2) organ recovery and receipt, (3) patient and living donor management, (4) QAPI, (5) human resources, (6) organ procurement, and (7) patients' and living donors' rights, and (8)additional requirements for kidney transplant centers. We want to emphasize that our overall focus is on the continuous, integrated care process that a patient experiences across all aspects of transplantation.

1. Condition of Participation: Patient and Living Donor Selection (Proposed Section 482.90)

[If you choose to comment on this section, please include the caption “PATIENT AND LIVING DONOR SELECTION” at the beginning of your comments.]

We believe transplant centers should have an active role in the management of patients prior to transplantation. We propose to require centers to utilize written patient selection criteria in making determinations regarding a patient's suitability for placement on the waitlist and a patient's suitability for transplantation. When a patient is placed on the center's waitlist or is selected to receive a transplant, we propose that the center must document in the patient's medical record the patient selection criteria that were utilized. We are also asking for comments on whether transplant centers should be required to make the patient selection criteria available to patients, either routinely or upon request.

We have not specifically defined patient selection criteria in the proposed rule because transplant technology is continually changing. We want to preserve centers' flexibility in identifying organ transplants that are medically reasonable and necessary in light of the most recent transplantation research and the needs of transplant recipients. However, we propose that the patient selection criteria must ensure fair and non-discriminatory distribution of organs.

In general, organ transplants, should be performed only on carefully selected patients whose medical needs cannot be met by other therapies (except for kidney transplants where the dialysis option may continue to exist). We propose that before a transplant center selects a patient for extra-renal transplant, the center would have to consider or employ all other appropriate medical and surgical therapies that might be expected to yield both short and long-term survival comparable to transplantation.

We are proposing an exception to this patient selection requirement for kidney transplant candidates because while kidney transplantation is the preferred treatment for patients with kidney failure, ESRD patients, unlike patients with other types of end-stage organ failure, have an alternative dialysis treatment option available to them, when kidney transplant is not feasible or when the graft has failed. Renal replacement therapy, which is required when kidney functions fall below 10-15 percent, includes either dialysis or kidney transplants.

Studies have shown that dialysis does not seem to yield survival comparable to transplantation. Kidney transplantation has many advantages, such as a lifestyle free from dialysis, a better quality of life and a longer life expectancy. However, kidney transplants have risks, such as surgical complications, rejection, and life-long maintenance medications and associated side effects. Therefore, dialysis continues to be a viable treatment option for an ESRD patient whose kidney transplant was unsuccessful.

We propose that a prospective transplant candidate must receive a psychosocial evaluation prior to placement on the waitlist. Although a person may be medically suitable for transplantation, he or she may have inadequate social support or coping abilities, or may be unable to demonstrate adequate adherence to a therapeutic regimen, which could then put the graft, and ultimately the transplant recipient at risk.

We also propose that before a transplant center places a patient on its waitlist, the candidate's medical record would have to contain documentation that the candidate's blood type has been determined. Requiring documentation

of the candidate's blood type would ensure that transplant centers are verifying the accuracy of vital data necessary to match the transplant candidate to a potential donor. We are specifically requesting comments on this proposal.

Like organ transplant candidates, we believe potential living donors should be carefully selected. Unlike deceased donor transplantation, living donor transplantation presents an ethical quandary in that living donation represents the only area of medicine in which an otherwise healthy individual is subject to surgical risk for somebody else's benefit. Any benefits to the donor are primarily psychological. We propose that transplant centers performing living donor transplants would have to use written living donor selection criteria to determine the suitability of candidates for living donation. We propose that the center must document in the transplant candidate's and living donor's medical records the living donor's suitability for donation. We have not proposed specific living donor selection criteria for transplant centers because there are no established guidelines concerning the selection of living donors at this time. Until living donor standards are established, we propose that the centers' living donor selection criteria must be consistent with the general principles of medical ethics. We propose that prior to donation, a prospective living donor must receive a medical and psychosocial evaluation. We also propose that the transplant center must document that the living donor has given informed consent, as required under § 482.102.

2. Condition of Participation: Organ Recovery and Receipt (Proposed Section 482.92)

As reported in The

Charlotte Observer,

a recent death of a transplant recipient was caused by transplantation of organs from a donor of an incompatible blood type. The incident was attributed to a combination of system errors that occurred during the organ procurement, organ receipt, and transplant processes. Another death was attributed to a miscommunication of blood types between the center's laboratory and the transplant team (Grady, Denise and Lawrence K. Altman, “Suit Says Transplant Error Was Cause in Baby's Death in August,”

The New York Times,

12 March 2003, Section A, Page 23, Column 5). These two events might have been avoided if certain steps were actively taken to validate the ABO (

i.e.

blood type) compatibility and other key data elements.

Under the current policies for heart, liver and lung transplants and the current regulations for renal transplant centers, there are no provisions addressing procedures for transplant centers to ensure that donor organ and transplant recipient data are compared, or to prevent the transplantation of mismatched organs. The OPTN rules specify that an OPO with an organ available for transplantation must obtain a “match run” for that organ type from UNOS. The match run lists potential recipients on the waitlist who are the correct size and blood type to receive the organ that is available. The OPTN also requires the OPO to provide the transplant center with written documentation of the potential donor's age, sex, and race, appropriate laboratory values, blood type, ABO or HLA typing, vital signs, cause of brain death and diagnosis, and current medication and transfusion history. However, these OPTN policies are voluntary. To prevent transplant mishaps caused by blood type mismatch, we propose that transplant centers would need to have written protocols for organ recovery and organ receipt. We propose that the protocols would have to ensure that the transplant center validates the donor's and the recipient's blood type and other vital data. Examples of vital data about the donor and the recipient that a transplant center should validate include, but are not limited to, appropriate laboratory values, vital signs, current medication and transfusion history. We also propose assigning responsibility for ensuring the medical suitability of donor organs for transplantation into the intended recipient to the transplanting surgeon, or the surgeon in the transplant center receiving the organ offer for his or her patient.

We propose that a center's protocols for organ recovery specify that a transplant center's organ recovery team would have to review and compare the recipient and donor data before recovery takes place. We also propose that when an organ arrives at the center, the transplanting surgeon and at least one other individual at the transplant center would have to verify that the donor's blood type and other vital data are compatible with transplantation of the intended recipient prior to transplantation. These verifications would ensure that transplant centers are actively taking steps to avoid transplantation of mismatched organs throughout the organ distribution process and would also prevent wastage of organs in the event a mismatch was not discovered until the organ(s) arrived at the transplant hospital.

We also propose that a center's protocols for organ recovery and receipt would have to ensure that the transplanting surgeon and at least one other individual at the transplant center verifies that the living donor's vital data (including blood type) are compatible for transplantation of the intended recipient, immediately before the removal of the living donor organ(s) and, if applicable, prior to the removal of the recipient's organ(s).

3. Condition of Participation: Patient and Living Donor Management (Proposed Section 482.94)

Under the current policies for heart, liver and lung transplants, a center is required to have adequate patient management plans and protocols that include therapeutic and evaluative procedures during the waiting, in-hospital, and discharge phases of transplantation. The current conditions for coverage for ESRD services require each ESRD facility, which includes renal transplant centers, to maintain for each patient a written long-term program and a written patient care plan to ensure that each patient receives the appropriate modality of care and the appropriate care within that modality. We believe that a patient's care should be managed during every stage of transplantation, starting with the patient's evaluation for placement on a center's waitlist and through the patient's discharge from the hospital following transplant, to ensure that the services provided meet the patient's care needs and that the patient is involved in his or her care. We propose that centers must have written patient management policies and patient care planning for pre-transplant, and through the patient's discharge from the hospital following transplant. It is equally important to ensure that living donors receive services that meet their care needs throughout the various stages of donation, starting with donor evaluation and continuing through the donor's immediate discharge from the hospital post-donation. Therefore, we propose that centers performing living donor transplants must have written donor management policies for the donor evaluation, donation, and through the donor's discharge from the hospital following donation. We propose that a transplant center must ensure that each patient or living donor is under the care of a multidisciplinary patient care team coordinated by a physician during all phases of transplantation or living donation.

A center's initial responsibility for a transplant patient begins when he or she is evaluated for placement on that center's waitlist, regardless of whether or not the patient is on another center's waitlist. Effective waitlist management, in our view, means installing and maintaining a reliable administrative system that tracks patient status and provides accurate updated patient data on demand. Inaccurate information on waitlist patients may create a situation where a center may initially agree to accept organs that are offered to them but later decline them at the last minute when they discover that the organs are not suitable for the intended recipients. In order to prevent organs from being wasted once they are recovered, we are proposing a standard specifically for waitlist management.

In 2002, the Clinical Practice Committee of the American Society of Transplantation issued guidelines regarding waitlist maintenance based on a questionnaire sent out to 287 transplant centers, of which 192 responded. The guidelines specifically recommend annual follow-up or assessment of potential transplant recipients as deemed appropriate to ascertain transplant status. Although we do not specifically propose annual follow-up or assessment of transplant candidates, we believe transplant centers need to reassess patients placed on their waitlist to ensure that (1) the center's information on the patient is accurate and (2) the transplant is still medically indicated. We are proposing that transplant centers keep their waitlists up to date, including updating waitlist patients' clinical information on an ongoing basis. We also propose that the transplant center must remove a patient from its waitlist when the patient receives a transplant or dies, or if there is any other reason why the patient should no longer be placed on a center's waitlist (for example, the patient's health could deteriorate or improve to the point that a transplant would no longer be medically suitable or a patient could voluntarily ask to be removed from a center's waitlist). We propose requiring transplant centers to notify the OPTN of the patient's removal from the center's waitlist no later than 24 hours after such removal. This timely notification to the OPTN of a patient's removal from a center's waitlist is crucial. Not only would this notification provide patients with confirmation of their removal from a center's waitlist, but the OPTN would also rely on this information to keep the national waitlist current. Prompt notification of a patient's removal from the waitlist provides more accurate data to facilitate accurate patient placement on the waitlist. Prompt notification of patient's removal from a center's waitlist would also enhance the accuracy of the SRTR data analyses. Furthermore, OPOs have a very narrow window of opportunity for allocating recovered organs to the appropriate recipient. Some OPOs have complained that transplant centers sometimes agree to accept an organ for a particular individual only to discover later that the individual has already received a transplant or has died prior to receiving a transplant.

We are proposing a requirement at § 482.94(c) that transplant centers maintain up-to-date and accurate patient management records for each patient who receives an evaluation for placement on a center's waitlist and who is admitted for organ transplant. We believe that accurate patient records are especially crucial in determining a patient's readiness for transplants. Accurate information about a patient's transplant status needs to be readily available to individuals involved in the care of the patient, and to the patients themselves. For example, we have found that in some cases, after a kidney dialysis patient is evaluated for placement on a center's waitlist, the patient's status is not communicated to the dialysis facility or to the patient. The patient, and the dialysis facility, may believe he or she has been placed on a waitlist, only to find months later that the transplant center is waiting for the patient to undergo further clinical testing.

Given that time on the waitlist is often one of the factors that determine which patients ultimately are transplanted, we propose that for each patient who has received an evaluation for placement on a center's waitlist, the transplant center must document in the patient's record that it has notified each patient of his or her placement status. Specifically we propose that the center must notify the patient of: (1) The patient's placement on the center's waitlist; (2) the center's decision not to place the patient on its waitlist; or (3) the center's inability to make a determination regarding the patient's placement on its waitlist because further clinical testing or documentation is needed.

After a patient is placed on a center's waitlist, we believe it is the transplant center's responsibility to provide waitlisted patients with an annual update of their waitlist status. We propose that once a patient is placed on a center's waitlist, the center must document in the patient's record that the patient has been notified of his or her waitlist status at least once a year, even if there is no change in the patient's placement status. In addition, we propose that no later than 10 days after a patient's removal from a center's waitlist for reasons other than death or transplantation (such as the patient's voluntary withdrawal from the waitlist or a change in the patient's medical status such that a transplant is no longer indicated), the center must document in the patient's record that the patient has been notified of his or her removal from the waitlist. For dialysis patients, we propose that the transplant center also must document in each patient's record that both the patient and the patient's usual dialysis facility are informed of the patient's transplant status or of changes in the patient's transplant status. In the event there are changes in a dialyzed patient's transplant status, we believe it is imperative for dialysis facilities to have up-to-date and accurate information about kidney transplant candidates to ensure adequate care and coordination between the dialysis facility and the transplant center prior to transplantation. In the case of patients admitted for organ transplants, we propose that the patient records contain written documentation of multidisciplinary care planning during the pre-transplant period and multidisciplinary discharge planning for the patient's post-transplant care.

In addition, we propose requiring transplant centers to make available social and nutritional services, furnished by qualified social workers and dietitians, to patients and living donors. The current kidney transplant center regulations at § 405.2171 require centers to provide a qualified social worker to evaluate transplant patients' psychosocial needs, participate in care planning of patients, and identify community resources to assist the patient and family. Similarly, we believe social services, such as assisting and supporting patients and their families in maximizing the social functioning and adjustment of the patient, are important to all transplant patients and living donors. Therefore, we are proposing that social services, furnished by a qualified social worker, be made available to all transplant patients, living donors and their families. Based on the definition of “qualified social worker” contained in § 405.2102, we propose to define a qualified social worker as an individual who meets licensing requirements in the State in which practicing, and (1) has completed a course of study with specialization in clinical practice, and holds a masters degree from a graduate school of social work accredited by the

Council on Social Work Education; or (2) has served for at least 2 years as a social worker, one year of which was in a transplantation program, and has established a consultative relationship with a social worker who has obtained the education described above.

The current kidney transplant center regulations at § 405.2171 also require a qualified dietitian, in consultation with the attending physician, to assess the nutritional and dietetic needs of each patient, recommend therapeutic diets, counsel patients and their families on prescribed diets and monitor adherence and response to diets. All transplant patients and living donors may need dietary modifications, permanently or temporarily, to maintain balances in fluids, electrolytes, and macro or micro-nutrients. We are proposing that nutritional assessments and diet counseling, furnished by qualified dietitians be made available to all transplant patients and living donors. Based on the definition of “qualified dietitian” contained in § 405.2102, we propose to define a qualified dietitian as an individual who (1) is eligible for registration by t

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