Organ Procurement and Transplantation Network

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Health Resources and Services Administration

42 CFR Part 121

Organ Procurement and Transplantation Network

AGENCY: Health Resources and Services Administration, HHS.

ACTION: Final rule.

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SUMMARY: This document sets forth improvements to the final rule

governing the operation of the Organ Procurement and Transplantation

Network (OPTN), published in 1998. It reflects the advice of a panel

convened by the National Academy of Science's Institute of Medicine, as

called for in the Department's appropriation act for 1999. It also

reflects comments on the 1998 rule and consultation with

representatives of the organ transplantation community, as recommended

in the same legislation; and it summarizes new transplant data

developed in the period since enactment of the appropriations act.

DATES: The final rule published on April 2, 1998, 63 FR 16296, adding

42 CFR part 121 with an effective date of October 1, 1998, as amended

on July 1, 1998, 63 FR 35847, did not take effect under section 213(a)

within Public Law 105-277, 112 Stat. 2681, 2681-359 through 2681-360,

approved October 21, 1998. The April 2, 1998 rule as amended by this

document, is effective on November 19, 1999.

FOR FURTHER INFORMATION CONTACT: D.W. Chen, M.D., M.P.H., Director,

Division of Transplantation, Office of Special Programs, Health

Resources and Services Administration, 5600 Fishers Lane, Room 7C-22,

Rockville, MD 20857, telephone 301-443-7577.

SUPPLEMENTARY INFORMATION: On April 2, 1998, the Department of Health

and Human Services (HHS) published in the Federal Register a final rule

pertaining to the operation of the Organ Procurement and

Transplantation Network (63 FR 16296). In accordance with the National

Organ Transplant Act (NOTA) of 1984, as amended, the purpose of the

final rule is to help achieve the most equitable and medically

effective use of human organs that are donated in trust for

transplantation. Toward this end, the final rule establishes

performance goals intended to bring about:

(1) Standardized criteria for placing patients on transplant

waiting lists, (2) standardized criteria for defining a patient's

medical status, and (3) allocation policies that make most effective

use of organs, especially by making them available whenever feasible to

the most medically urgent patients who are appropriate candidates for

transplantation. The final rule also sets standards for availability of

organ transplantation data, and it addresses the governing structure of

the OPTN. No provision of the final rule is intended to interfere with

the discretion of individual health professionals and patients in

medical decision-making, and the rule looks to the OPTN to design organ

allocation policies. At the same time, the rule defines the policy

oversight responsibilities of the Secretary of HHS. In concert with

efforts to encourage organ donation, the final rule is intended to help

make best use of the limited number of organs available for

transplantation.

The final rule invited further comments, which have been received

and reviewed. In addition, the Omnibus Consolidated and Emergency

Supplemental Appropriations Act for 1999 delayed implementation of the

final rule until October 21, 1999. (This Omnibus Act, Public Law 105-

277, at section 101(f) of Division A, enacted the Department of Labor,

HHS, and Education, and Related Agencies Appropriations Act, 1999.

Within the latter act, section 213 included provisions related to the

final OPTN rule, 112 Stat. 2681, 2681-359 through 2681-360. Hereafter,

for ease of reference, we will refer to section 213 of the

Appropriation Act, or simply section 213.) Section 213 called for

independent review through the National Academy of Science's Institute

of Medicine. It also suggested development of improved information on

the effectiveness of the transplantation system, including center-

specific information if possible. Finally, it suggested further

discussions between HHS and representatives of the transplant

community. Each of these areas has been addressed.

I. Background

A. Legislative and Regulatory History

Legislative and regulatory history are outlined in the preamble to

the April 2, 1998, final rule. In addition to the underlying statute

(sections 371-376 of the Public Health Service Act, as enacted by the

National Organ Transplant Act of 1984, and as subsequently amended), of

particular importance is section 1138 of the Social Security Act,

enacted in 1986. This legislation requires 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. This provision subjects a transplant hospital's

entire Medicare and Medicaid participation, and thus in reality its

economic survival, to OPTN policy and enforcement. A similar provision

in section 1138 affects funding under Medicare and Medicaid for organ

procurement organizations (OPOs). But authority for establishing

conditions of participation in Medicare and Medicaid resides with the

Secretary and cannot be exercised by another party without either

oversight authority or delegation. Thus, review and oversight authority

of OPTN policies by the Secretary of HHS is made even more necessary by

section 1138. A Federal Register notice published on December 18, 1989

(54 FR 51802) addressed this need by stating that no OPTN policies are

legally binding ``rules or requirements'' of the OPTN for purposes of

section 1138, unless they have been approved by the Secretary. The

final rule published April 2, 1998, defines the structure for such

review and approval, thus setting the stage for OPTN ``rules or

requirements'' that would be enforceable on transplant hospitals and

OPOs under section 1138.

In October 1998, section 213 of the Appropriation Act delayed

implementation of the final rule to October 21, 1999. Section 213

directed that the Institute of Medicine conduct a review of the current

policies of the OPTN and the final rule. Section 213 also suggested

that the Secretary ``may conduct a series of discussions with the OPTN

in order to resolve issues raised by the final rule.'' In addition,

section 213 indicated a need for improved availability of data on

transplantation and transplant center performance.

B. Institute of Medicine Report

The Institute of Medicine (IOM) issued its report, Organ

Procurement and Transplantation, on July 22, 1999. The report included

five major recommendations. The Department has relied heavily on the

guidance in the IOM report in reviewing the provisions of its final

rule. In general, the IOM report validates the concerns that gave rise

to the final rule and the approaches taken in the rule:

Recommendation 1: Establish Organ Allocation Areas for Livers.

The committee recommends that the DHHS Final Rule be implemented by

the establishment of Organ Allocation Areas (OAAs) for livers--each

serving a population base of at least 9 million people (unless such

an area would exceed the limits of acceptable cold ischemic time).

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OAAs should generally be established through sharing arrangements

among organ procurement organizations to avoid disrupting effective

current procurement activities.

Recommendation 2: Discontinue Use of Waiting Time as an

Allocation Criterion for [Liver Transplant] Patients in Statuses 2B

and 3. The heterogeneity and wide range of severity of illness in

statuses 2B and 3 make waiting time relatively misleading within

these categories. For this reason, waiting time should be

discontinued as an allocation criterion for status 2B and 3

patients. An appropriate medical triage system should be developed

to ensure equitable allocation of organs to patients in these

categories. Such a system may, for example, be based on a point

system arising out of medical characteristics and disease prognoses

rather than waiting times.

Recommendation 3: Exercise Federal Oversight. The Department of

Health and Human Services should exercise the legitimate oversight

responsibilities assigned to it by the National Organ Transplant

Act, and articulated in the final rule, to manage the system of

organ procurement and transplantation in the public interest. This

oversight should include greater use of patient-centered, outcome-

oriented performance measures for OPOs, transplant centers, and the

OPTN.

Recommendation 4: Establish Independent Scientific Review. The

Department of Health and Human Services should establish an

external, independent, multidisciplinary scientific review board

responsible for assisting the Secretary in ensuring that the system

of organ procurement and transplantation is grounded on the best

available medical science and is as effective and as equitable as

possible.

Recommendation 5: Improve Data Collection and Dissemination.

Within the bounds of donor and recipient confidentiality and sound

medical judgment, the OPTN contractor should improve its collection

of standardized and useful data regarding the system of organ

procurement and transplantation and make it widely available to

independent investigators and scientific reviewers in a timely

manner. The Department of Health and Human Services should provide

an independent, objective assessment of the quality and

effectiveness of the data that are collected and how they are

analyzed and disseminated by the OPTN.

In addition, the General Accounting Office (GAO) made findings in

two other areas required by section 213: the possibility of legal

liability of OPTN members arising from their peer review activities and

the confidentiality of information. Regarding liability, the General

Counsel of the GAO found no apparent conflict between the final rule

and State laws governing peer review. Regarding confidentiality, the

General Counsel found that the Secretary of HHS has authority under the

final rule to decide that the public interest in disclosure of

information about organ transplants outweighs the interest in

confidentiality.

C. Discussions With the Transplant Community

Representatives of HHS met with members of the transplant community

on numerous occasions in the period immediately following publication

of the final rule. Since enactment of section 213, representatives of

HHS have met on 11 separate occasions with representatives of 11

transplant organizations: United Network for Organ Sharing (UNOS, the

current OPTN contractor), Transplant Recipients International

Organization, American Liver Foundation, National Transplant Action

Committee, National Minority Organ and Tissue Transplant Education

Program, National Kidney Foundation, Patient Access to Transplantation

Coalition, American Society of Transplantation, American Society of

Transplant Surgeons, North American Transplant Coordinators

Organization, and the American Nephrology Nurses Association. On

September 15, 1999, an additional meeting with representation invited

from all of these organizations took place to discuss together issues

that had been surfaced.

Clarifications

HHS is further clarifying these issues with this publication:

``National'' lists: The final rule does not require single

national lists for allocation of organs, beyond the national registry

lists already utilized by the OPTN. As underscored by the IOM

recommendations, it is the Department's goal to achieve sharing of

organs broad enough to achieve medically effective results for

patients, especially by providing organs for patients with greatest

medical urgency who are appropriate candidates for transplantation.

When using the terms ``greatest medical urgency,'' or ``most medically

urgent,'' the Department is referring to transplanting those patients

whose medical condition, in the judgment of their physicians, makes

them suitable candidates for transplantation. The final rule directs

the OPTN to overcome as much as possible arbitrary geographic barriers

to allocation that restrict the allocation of organs to patients with

greatest medical urgency who are appropriate candidates for

transplantation and that are not based on medical criteria. Broader

sharing was an essential element of the IOM's findings.

Most Medically Urgent Patients: The final rule follows,

and intends to expand, existing policy in serving most medically urgent

patients first, again, referring to patients who are suitable

candidates for transplantation. It is not the Department's intention to

require transplantation of patients too ill to benefit; the final rule

specifically prohibits policies that might result in such futile

transplantations and organ wastage. Providing available organs to

patients with greatest medical urgency who are appropriate candidates

for transplantation is already the policy of the OPTN within allocation

areas. Transplant priority for patients with greatest medical urgency,

whenever they are medically suitable, follows the tenets of medical

practice generally and is already accepted throughout the transplant

community and general public.

Medical Factors Affecting Organ Movement: The final rule

fully recognizes limitations on movement of organs resulting from

medical factors, especially limits of ischemic time. As recommended by

the IOM report, and as intended by the 1998 final rule, sharing of

organs should be broad enough to enable medically effective use of

organs, especially to enable organs to reach the most medically urgent

patients, but ischemic time limits and any other medical factors

affecting the viability of the organ must be considered in designing

allocation policies.

Small and Medium Sized Transplant Centers: The Department

does not expect the final rule to cause the closure of small or medium

sized transplant centers or otherwise diminish access to

transplantation for certain populations, including those living in

rural areas. The IOM report did not find evidence that the rule would

have such effects; and a report by the HHS Office of Inspector General

(``Fostering Equity in Patient Access to Transplantation: Local Access

to Liver Transplantation,'' dated August 1999) concluded that

geographic distribution of liver transplant centers is unlikely to

change as a result of national policies on organ allocation. The

Department is concerned that patient access to transplant services not

be adversely affected by closure of centers that are providing quality

care, including small and medium sized centers. Thus, the amendments

below include provision for monitoring any effects of policy changes on

small and medium sized centers. However, HHS and the OPTN should work

together to ensure that all transplant programs, regardless of volume,

are providing quality care to candidates and recipients.

Designated Transplant Program Requirements: The final rule

carries forward the policies in the proposed

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rule that provided separate staffing and organizational ``designated

transplant program'' requirements for non-Medicare participating

transplant programs and those that are certified as Medicare approved

transplant programs. The Department has received comments similar to

those submitted in response to the proposed rule, suggesting that

uniform standards be applied for designation status. The Department

continues to have no objection to this suggestion in principle, but

believes that the OPTN should submit such standards for the Secretary's

consideration as possible changes to the Medicare conditions for

coverage of organ transplants, which currently contain similar

requirements.

Secretarial Oversight and Enforceability of OPTN Policies

Virtually all commenters agreed that HHS should exercise an

oversight role over OPTN policies, although there were different views

among the participants as to how such oversight should be carried out.

Exercise of HHS oversight was also one of the five primary

recommendations of the IOM report. Further, as explained in

``Legislative and Regulatory History'' above, section 1138 of the

Social Security Act elevates OPTN membership and policies to the status

of requirements for participation in Medicare and Medicaid for

transplant hospitals and OPOs, thus necessitating Secretarial review

and oversight authority over those policies. The final rule provides

the framework for such oversight as well as the framework for creating

a body of enforceable OPTN policies.

An additional recommendation by the IOM was establishment of an

independent scientific review board ``for assisting the Secretary in

ensuring that the system of organ procurement and transplantation is

grounded on the best available medical science and is as effective and

as equitable as possible.'' In response to this recommendation in the

IOM report as well as comments received, the Department intends to

create such an advisory board, the Advisory Committee on Organ

Transplantation. The Department intends to implement the IOM's

recommendations that this Committee have several key responsibilities.

As recommended by the IOM, the Committee will provide ``timely,

nonpartisan review'' to ``assist the Secretary in managing the system

in a manner that best serves the public interest.'' It will also, as

recommended by the IOM, ``help provide objective information and advice

for future directions for the [organ transplantation] system.'' It

would also, as recommended by the IOM, ``help insure that policies and

procedures are evidence-based and guided by the best available

scientific and medical precepts.'' In order that the Committee fulfill

this latter responsibility, Sec. 121.4 (b)(2) and (d) have been revised

to reflect this role.

When the OPTN proposes enforceable policies, the Secretary will ask

the Committee for its views on the proposals when the proposals are

published in the Federal Register for public comment. The Committee's

views, public comments, and the Department's views will then serve as

the basis for discussions with the OPTN. If, after these discussions,

the Secretary wishes to direct that the OPTN revise its proposals, the

OPTN will have the opportunity to suggest revisions. If the Secretary

does not agree with the OPTN's revised approach (or if it does not

respond in a timely manner), the Secretary may require the OPTN to take

other appropriate actions. However, the Secretary will ask the

Committee for its views on the specific proposed actions before

transmitting them to the OPTN. A similar approach may also be used

should the Secretary review other OPTN policies, or elect to evaluate

critical comments received by the Secretary relating to the manner in

which the OPTN is carrying out its duties.

It is not the desire, nor is it the intention, of the Department to

interfere in the practice of medicine. Decisions about who should

receive a particular organ in a particular situation involve levels of

detail, subtlety, and urgency that must be judged by transplant

professionals. The Advisory Committee will greatly assist the Secretary

with respect to the medical and scientific components of OPTN policies.

The medical community has substantial contributions to make within the

deliberative process for developing OPTN policies, as well as in

individual decisions involved in clinical transplantation practice.

The rule also has been revised to emphasize that the Secretary's

review is intended to ensure consistency between OPTN policies and the

National Organ Transplant Act and this regulation. This revision is

intended to emphasize, as the IOM did in its report, that the

Secretary's oversight will further the public interest, a role assigned

to the Department by the National Organ Transplant Act and articulated

in this regulation.

OPTN Board Composition

Participants expressed a variety of views on requirements

concerning the composition of the OPTN Board of Directors. Some

participants believed that the rule should require, not merely

authorize, the Board to include at least 50 percent representation of

transplant physicians and transplant surgeons, to ensure a

preponderance of medical expertise. Others suggested more even division

of representation among transplant physicians and transplant surgeons,

other non-physician transplant professionals, and candidates,

recipients, donors, their families, and the general public. Concern was

also raised that a combination of percentage representation

requirements with specific categorical representation requirements

would make the Board so large as to be unwieldy, if the Board chose to

allow 50 percent representation of transplant physicians and surgeons.

The Department has reorganized and revised the Board and Executive

Committee composition provisions to strengthen the role of transplant

physicians and surgeons on the Board, consistent with the rule's thrust

that allocation policy (one of the OPTN's most important

responsibilities) be based on objective and measurable medical criteria

and sound medical judgment, to strengthen the role of transplant

candidates, recipients, donors, and their families on the Board and its

Executive Committee, and to provide the OPTN greater flexibility in

determining the appropriate size for the Board. This document includes

amendments that identify categories of membership, but do not require a

specific number of members from each category. This amendment requires

approximately 50 percent transplant physician or transplant surgeon

membership, instead of no more than 50 percent, and specifies at least

25 percent transplant candidates, transplant recipients, organ donors,

and family members.

We have retained the provision designed to avoid even an appearance

of a conflict of interest by requiring that transplant candidates,

recipients, donors and family members on the Board not have an

``employment or similar relationship'' with certain entities and

individuals involved in transplantation. However, we received comments

suggesting that such individuals may have exceptional commitment or

knowledge and should not be automatically disqualified from Board

membership, and that, in any event, the Board should have additional

flexibility in this area. We have revised this provision to authorize

the Board to waive this requirement for up to half of

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these members. We expect the Board to use this flexibility consistent

with the rule's goal of broad involvement of patients, recipients,

donors, families and the public in the formulation of transplant

policy.

Broader Geographic Sharing of Organs

The final rule's emphasis on broader sharing of organs is being

clarified through this document. Establishment of liver allocation

areas broad enough to provide for medically effective allocation of

organs was the leading recommendation of the IOM report. Some

commenters expressed concern about the need for the transplant system

to use standard criteria for listing patients and assigning their

urgency status, and likewise the need for enforcement mechanisms to

ensure that medically urgent patients who are appropriate candidates

for transplantation are not disadvantaged through misuse of listing

criteria or priority rankings. The final rule calls on the OPTN to

develop such standard criteria, and to monitor compliance with them,

prospectively if appropriate. Further, by establishing a framework for

Secretarial review and approval of OPTN policies, as well as review and

evaluation procedures for the OPTN, the rule provides a foundation for

enforcement of these standard criteria.

Frequency and Timeliness of Data

Most participants expressed support for enhanced frequency and

timeliness of data. Likewise, the IOM report strongly urged

improvements in data collection and dissemination, both for physician

and patient information and to provide outcome data that may improve

understanding of best medical practices. As OPTN contractor, UNOS

expressed concern about its ability to meet the frequency requirements

in the April 2 final rule. The Department has decided to retain the 6

month data presentation requirement. The Department recognizes that

UNOS' concerns stem in part from its belief that certain types of data

may not need to be updated as frequently as others. Therefore, the

Department has added a provision that would permit longer intervals for

certain data.

The Department recognizes the progress that UNOS has made in

increasing the availability of program-specific information for use by

patients, families, physicians, and payors. To respond to the

contractor's concerns regarding its ability to meet the frequency of

the reporting requirement in the final rule, HHS will not require the

submission of the first program-specific report under

Sec. 121.11(b)(1)(iv) until June 30, 2000. This will allow OPTN member

organizations adequate time to become fully Y2K compliant and ensure

that all data submitted to the OPTN is done so electronically, and will

enable the contractor to meet the Department's and the IOM's

expectations that information be more timely and accessible.

Use of Waiting Time

In general, the IOM found the emphasis on cumulative waiting times

to be inappropriate as a measure of equity in the transplant system and

as a criterion for allocation for less medically urgent patients,

pointing instead toward ``more meaningful indicators of equitable

access'' such as ``status-specific rates of pretransplantation

mortality and transplantation.'' The IOM report indicated, however,

that the use of ``waiting times in status'' for the most medically

urgent liver transplant patients (those in status 1 and 2A) was ``an

appropriate criterion, along with necessary medical criteria.'' For

less medically urgent patients (statuses 2B and 3), the IOM recommended

that the OPTN discontinue use of waiting time as an allocation

criterion and instead develop ``an appropriate medical triage system .

. . to ensure equitable allocation of organs to patients in these

categories.'' HHS generally agrees with these findings, although the

Department believes that waiting time in status (unlike cumulative

waiting time) can be one among several useful criteria in assessing

variability in results for patients at different transplant centers. To

date, waiting times have been used in examining the performance of the

transplant system in part because waiting times are used by the OPTN as

an allocation criterion, and in part due to lack of better measures. It

is for these reasons that reducing any variations in ``waiting time in

status,'' especially for the most medically urgent patients, was

included as a performance measure in the final rule published April 2.

In addition, the IOM recommendation points again to the need for better

data to provide alternatives to waiting time as a performance measure.

Based on the IOM's recommendations and comments from the transplant

community, the Department has made additional refinements to the rule's

discussion of waiting times.

The Department's approach in this section follows the

recommendations of the IOM and responds to issues raised by commenters.

First, the Department agrees with the IOM recommendations that

``overall'' waiting times are an inappropriate measure. The concept of

using ``waiting time in status'' is, however, permitted as a factor in

allocation policy.

Second, Sec. 121.8(b)(4) requires the OPTN to use performance

indicators to assess transplant program performance and to seek to

reduce the variations among transplant programs with respect to

selected performance indicators. This ``performance indicator''

approach is consistent with the IOM's recommendation that data be used

to assess transplant program performance. Among the alternatives

available to the OPTN is the performance indicator ``waiting time in

status.'' Consistent with the IOM's approach, if the OPTN retains

waiting time in status for allocation purposes for medically urgent

categories similar to current Status 1 and 2A in its revised liver

allocation policies, the Department would expect the OPTN to use

waiting time in status as a performance indicator for liver patients,

along with necessary medical criteria.

Regarding the general approach of reducing variations among

transplant programs with respect to selected performance indicators, we

also expect the OPTN to work towards improving, where possible, the

outcomes under these indicators. For example, if the OPTN used the

performance indicator pretransplantation mortality rates for liver

patients by medical status, as recommended by the IOM, then the

Department would expect the OPTN to seek to reduce the variations in

this performance indicator by improving pre-transplant survival at

programs where it fell significantly below the national rates.

We also note that, although Sec. 121.8(b)(2) requires that the

medical characteristics of patients within each category be as similar

as possible, the IOM observed that the current liver status categories

2B and 3 were heterogeneous. As a result, some patients in these

categories need life-saving transplants sooner than others. The other

patients, often with longer waiting times, can, nevertheless, wait

longer periods of time without increased risk of death. Therefore, the

IOM concluded that the OPTN should not use waiting times as a criterion

for patients in these categories. Some commenters, however, suggested

that the OPTN would have difficulty further refining its existing

status categories. Commenters also requested that the OPTN be allowed

to continue to use waiting times in some fashion for these patients.

This rule provides the OPTN flexibility to continue to use waiting

times for patients in these categories but would require that such use

not

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override medical urgency considerations.

However, the Department expects, as the IOM concluded, that broader

sharing of organs should occur for all patients and that organs will go

to more medically urgent patients who are appropriate candidates for

transplants before being offered to patients whose condition permits

them to wait longer for a transplant.

OPTN Review of Member Compliance With Final Rule Requirements and

Mandatory OPTN Policies

Many members of the transplant community expressed concern about

how best to promote compliance with OPTN policies. Section 121.8(a)(7)

has been added to emphasize that the OPTN should especially promote

compliance with approved allocation policies through prospective and

retrospective reviews of programs' compliance with allocation policies.

In addition, the OPTN is required by Sec. 121.10 to conduct reviews and

evaluations of each OPTN member's compliance with these rules and

approved OPTN policies. Thus, the OPTN is required to implement a

review process to ensure that individuals receiving transplants are

accurately listed and in proper classification categories to receive

organs. Currently, UNOS liver and thoracic Regional Review Boards

(RRBs) provide retrospective review of designation of status 1 and 2A

patients for livers and 1A patients for hearts. The Department will

explore with the OPTN contractor issues related to the conduct of

prospective and/or retrospective reviews of all listings and changes in

status categories to assure that programs are making appropriate

classification determinations. Reviews, prospective and retrospective,

might be performed by existing OPTN RRBs. In addition, the Secretary

may ask independent third parties, such as the Joint Commission on the

Accreditation of Health Organizations (JCAHO), or Utilization and

Quality Control Peer Review Organizations (PROs) established under Part

B of title XI of the Social Security Act, to monitor the OPTN

enforcement system by independently conducting audits of the work of

the RRBs.

Incentives for High Performing OPOs

Concern has been expressed that, by emphasizing broader sharing of

organs, the final rule might bring about reduced organ donation. The

Department disagrees, and the IOM report found some evidence that,

where broader sharing is currently occurring, donations have increased.

In response to these concerns, however, HHS has considered the

possibility that positive rewards might be offered for high performing

OPOs, to add to incentives for organ donation. The Department believes

that high performance by OPOs should be rewarded in a way that does not

disadvantage patients by compromising one of the fundamental objectives

that the final rule is trying to achieve--namely broader sharing of

organs. Therefore, the Department encourages the OPTN to develop and

recommend to the Secretary policy incentives to reward high-performing

OPOs. In addition, in response to longer-standing concerns, HHS' Health

Care Financing Administration (HCFA) is reviewing the way it currently

measures OPO performance.

Policies to Address Socioeconomic Barriers

Some in the transplant community have expressed concern that the

final rule would require transplant hospitals to make their own

financial resources available to pay for transplant and follow-up care

for patients unable to pay. However, this was not the intention of the

April 2 final rule. The rule calls on the OPTN Board of Directors to

recommend policies that would reduce inequities in access resulting

from socioeconomic status and ensure that the registration fee itself

does not represent a barrier to transplantation.

Registration Fees

One commenter objected to Secretarial review of that portion of

registration fees paid by OPTN members (and indirectly by patients and

their insurers) that represents expenditures by the contractor that are

not directly related to the tasks performed under the contracts with

HHS. The final rule specifies that the Secretary has oversight of that

portion of the registration fee directly related to operation of the

OPTN.

Health Resources and Services Administration (HRSA)-HCFA Cooperation

A commenter noted the need for increased coordination between HRSA

and HCFA on transplantation issues within their respective areas of

responsibility. HRSA and HCFA have pursued several cooperative efforts

to achieve increased organ donation, a goal of the Administration's

National Organ and Tissue Donation Initiative, which was launched in

December 1997. On June 22, 1998, HCFA published a final rule (42 CFR

part 482) regarding Medicare Hospital Conditions of Participation,

which requires hospitals to refer all deaths and imminent deaths to

local OPOs and conduct donation request training programs for

appropriate staff representatives. In 1999, HRSA and HCFA jointly

sponsored projects to encourage collaboration between hospitals and

OPOs in effectively implementing this regulation. HCFA's responsibility

for OPO performance standard establishment, certification and re-

certification of OPOs, and OPO waiver request review involves close

cooperation with HRSA to identify practices most likely to benefit

donor families and transplant patients, and that impact current organ

allocation policy. In addition, HCFA and HRSA are working together to

enhance and better coordinate collection, reporting, and analysis of

organ procurement and transplant data in an effort to assure optimum

performance of the OPTN.

D. Data

Section 213 called for ``timely and accurate program-specific

information on the performance of transplant programs.'' The IOM

report, in reviewing 68,000 medical records, made a significant

contribution in the data area, although the report also cited the

paucity of data available and recommended improved data collection and

dissemination. In addition, UNOS recently has added Internet-based

capability, both for providing information to physicians and the public

and for collecting data from its members.

Finally, HHS has completed new transplant program-specific analyses

that show varying outcomes for patients among different transplant

hospitals. Department staff analyzed OPTN patient outcome data for

liver and heart transplants with respect to three critical issues: (1)

The likelihood that, having been listed as a transplant candidate, a

patient will receive an organ within one year; (2) the likelihood that

a patient will die within one year of listing while awaiting

transplantation; and, (3) the likelihood that a patient will still be

alive one year after listing, irrespective of whether he or she

underwent a transplant procedure. After risk adjustment (i.e.,

adjustment for differences in the mix of patients' health status from

program to program), the analyses revealed substantial differences in

outcomes from one transplant program to another. The principal findings

for liver transplants illustrate that:

Ten percent of the programs have a standardized risk-

adjusted rate of transplantation within one year of listing of 71

percent or more; whereas,

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for another ten percent of the programs, the rate is 25 percent or

less;

The likelihood of dying within one year of listing while

awaiting a transplant ranges from less than 8 percent to more than 22

percent; and

The likelihood of surviving one year after listing as a

transplant candidate or a recipient ranges from approximately 65

percent to almost 86 percent.

The analogous values for heart transplants are 72 and 36 percent

(transplantation within one year of listing), 9 and 23 percent (death

within one year of listing while awaiting a transplant), 67 and 84

percent (survival for one year after listing irrespective of whether

transplanted or not).

In the course of performing these analyses, Department staff

identified gaps in the data currently collected by the Scientific

Registry--e.g., additional clinical details about patients' conditions

at the time of listing (which could improve risk adjustment) and

additional data on clinical complications (which could help in

assessing quality of life following transplantation). The Department

has provided these analyses to UNOS and has encouraged it, in its

management of the OPTN and its operation of the Scientific Registry, to

broaden the scope of data collection and make increased use of program-

specific performance analyses. The analyses are included in the U.S.

Department of Health and Human Services 1999 Report to Congress on the

Scientific and Clinical Status of Organ Transplantation.

II. Public Comments

Between April 2 and September 16, 1998, we received a total of

approximately 2,500 comments on the final rule. (Letters with petitions

or with form letters attached were counted as one comment. HHS received

a total of approximately 20,000 form letters.) The majority of the

comments reflected issues addressed in ``Clarifications'' above. This

document includes changes intended to make these issues clear. Other

issues raised by commenters were discussed in the meetings conducted

this year pursuant to section 213 of the Appropriation Act, and they

are also outlined above.

III. Changes in the Regulatory Text

As a result of the comments received, the Department has made

several modifications to the final rule published on April 2, 1998.

Some changes have been made to clarify the regulatory language. Other

revisions to the regulatory text add provisions or modify requirements

from the previously published final rule.

1. Definition of Organ

The Department has deleted bone marrow from the definition of organ

in Sec. 121.2 because it falls within the scope of a different

statutory authority. Although the NOTA refers to bone marrow for

purposes of the Scientific Registry, subsequent legislation established

a separate program to address ``unrelated'' bone marrow transplants. A

commenter recommended that the definition be expanded to include

intestine, stomach, or a collection of human cells that perform a vital

function of an organ, including any organ containing vasculature that

carries blood after transplantation. In the Preamble to the 1998 rule,

the Department stated: ``The inclusion of other organs, such as the

stomach and intestines, not only would have an impact on other

requirements in these regulations such as the development of allocation

policies, certification of designated transplant programs, and

establishment of training requirements but also would affect OPO

requirements to procure these organs in accordance with HCFA rules.

Thus, the Department believes it would be premature for this rule to

specify other organs in addition to those already named. Instead, the

Department will direct the OPTN contractor to consider which organs or

parts of organs, if any, should be subject to OPTN policies, and to

submit recommendations to the Secretary.'' The Department's position on

this issue remains unchanged.

2. National List

The term ``national list'' has been replaced with ``waiting list''

in Sec. 121.2, and throughout the final rule. The term ``national

list'' was incorporated into the regulation to reflect statutory

language in section 372 of the Public Health Service (PHS) Act, 42

U.S.C. 274, which requires the OPTN to ``establish a national list of

individuals who need organs.'' Current OPTN allocation convention

derives subordinate lists from a single database and current OPTN

policy allocates zero-antigen mismatched kidneys nationally, due to

scientifically demonstrated improvements in patient and graft survival

resulting from this policy. Furthermore, ischemic times and patient

outcomes make such an approach appropriate in the case of zero-antigen

mismatched kidneys. If supported by scientific evidence, the Department

has no objection to this approach.

3. Composition of OPTN Board of Directors

The Department wishes to ensure adequate patient, donor and family

representation on the OPTN Board of Directors, while giving the OPTN

sufficient flexibility to constitute a balanced and effective Board.

Thus the Department has included a requirement under Sec. 121.3(a) that

the Board of Directors shall include at least 25 percent transplant

candidates, transplant recipients, organ donors, and family members. In

response to comments, the Department also has revised Sec. 121.3(a)(1)

to enable the OPTN to govern itself with greater flexibility than was

provided by the 1998 rule. The revised language maintains the

requirement that the Board of Directors include representatives of

OPOs, transplant centers, voluntary health associations, transplant

coordinators, histocompatibility experts, other non-physician

transplant professionals, and the general public, but does not mandate

a specific number of members from each category. The Secretary believes

that the less prescriptive language in this revision will better allow

the OPTN itself to determine the appropriate size of, and

representation on, its Board of Directors, while achieving a balance

among physician, patient, donor, family and other representatives.

Section 121.3(a)(2) has been revised. That paragraph prohibited

those Board members who were identified as transplant recipients,

transplant candidates, organ donors, family members, or members of the

general public to be employees of, or have similar relationships with,

specified categories of institutional members required to be on the

Board. The revised paragraph is more flexible, as described more fully

above.

As discussed above, Sec. 121.3(a) has been revised to require that

approximately 50 percent of the Board members be transplant surgeons or

transplant physicians, rather than the language of the April 2, 1998,

rule requiring no more than 50 percent, and that at least 25 percent of

its members be transplant candidates, transplant recipients, organ

donors, and family members. The comparable requirements for the

Executive Committee of the Board have been similarly revised.

Transplant physicians or transplant surgeons elected to the Board or

Executive Committee under other categories must be counted toward the

requirements of these paragraphs of the final rule.

[[Page 56656]]

Furthermore, the requirement for a two year term for Board members

in former Sec. 121.3(a)(4) has been deleted. Board members have diverse

backgrounds and will require different periods of time to become

familiar with the complex issues coming before the Board. Thus, we

believe that it is appropriate for the OPTN to determine for itself the

length of the term for Board members, subject to Departmental review.

4. Socioeconomic Issues

As articulated in the April 2, 1998, rule, the Department is

concerned that all patients in the country have access to

transplantation and encourages the OPTN to work toward this goal.

Several members of the transplant community, however, commented that

the provisions of Sec. 121.4 addressing socioeconomic issues would

require transplant hospitals to make their own financial resources

available to pay for transplantation and follow-up care for patients

unable to pay. In response to these comments, the Department has

revised this section to specify that paragraph (a)(3)(i) refers only to

the registration fee and has revised paragraph (a)(3)(ii) to clarify

that resources for patients unable to pay should be sought from all

available sources.

5. Secretarial Review of OPTN Policies

In response to comments asking which OPTN policies are to be

submitted to the Secretary, the Department has modified the language of

Sec. 121.4(b)(2) to provide that the Board of Directors is required to

provide the Secretary with proposed policies that the OPTN recommends

be enforceable under Sec. 121.10 (including allocation policies) and

others as specified by the Secretary. As discussed above, the rule has

been revised to adopt the IOM's recommendation that the Advisory

Committee assist the Secretary in reviewing OPTN policies and practices

as well as to indicate the purposes of the Secretary's review.

The timing requirement has also been changed from 30 days to 60

days before implementation of the proposed policy to provide a more

realistic estimate of the time required for review by the Advisory

Committee and the public, should such review be necessary.

6. Registration Fee

One commenter objected to Secretarial review of the patient

registration fee, maintaining that this fee is paid voluntarily by OPTN

members for the services provided to them by the contractor. The

Department agrees that a portion of the current fee represents a

voluntary payment by OPTN members to the contractor for services

outside the direct operation of the OPTN on behalf of patients, while

another portion represents the payment provided by patients and their

insurers for the operation of the OPTN system itself. Consequently, the

Department has modified the language of Sec. 121.5(c) to indicate that

the portion of the registration fee subject to Secretarial oversight is

that portion directly related to operation of the OPTN; any other fee

may only be charged on a voluntary basis to OPTN members. In this

regard, the Department would interpret the ``reasonable costs'' for

operating the OPTN to include additional costs of compliance under

Sec. 121.8(a)(7) and reviews and enforcement under Sec. 121.10.

7. Human Immunodeficiency Virus (HIV)

Commenters suggested revising the language of Sec. 121.6(b) to

authorize transplantation of organs from HIV positive donors to HIV

positive recipients. The Department has revised Sec. 121.6(b) to

reflect the language of the statute. We note, however, that HCFA

regulations governing OPOs, at 42 CFR 486.306(q), require OPOs to

screen donors to ``[e]nsure that appropriate donor screening and

infection tests, consistent with the OPTN standards and the CDC

[Centers for Disease Control and Prevention] guidelines * * * are

performed * * * to prevent the acquisition of organs that are infected

with the etiologic agent for acquired immune deficiency syndrome.'' The

OPO regulations require that OPO donor screening meet the two

thresholds of the OPTN standards as well as the CDC guidelines. OPOs

must comply with the CDC ``Guidelines for Preventing Transmission of

Human Immunodeficiency Virus Through Transplantation of Human Tissue

and Organs'' as appended to the regulations for OPOs (see 42 CFR part

486, Subpart G, Appendix A). As a result, the OPO regulations will

still preclude acquisition of an organ from an HIV-positive donor for

transplantation. The OPTN may propose standards permitting such

transplantation to the Secretary for consideration and potential change

in existing CDC guidelines.

8. Criteria for Listing Patients

The 1998 rule set as a performance goal that the OPTN standardize

objective and measurable medical criteria for including patients on the

waiting list. In drafting the language of that section, the Department

expected that the criteria developed for adding patients to the waiting

list would inherently contain criteria for removing patients from the

list. Commenters pointed out that the rule should be specific in this

respect. The Department adopted this suggested clarification in

Sec. 121.8(b)(1).

9. Organ Allocation

The Department received many comments on this section, especially

former Sec. 121.8(a). We have reorganized this entire section for

clarity and addressed points raised by the IOM as well as several

issues raised by commenters. Some commenters asked that we clarify the

OPTN's ability to have different allocation policies for different

types of organs (or combinations of organs) to be transplanted.

Language to this effect is now found in Sec. 121.8(a)(4). The

Department wishes to emphasize that this means that the OPTN may take a

different approach in defining priority ranking under Sec. 121.8(b)(2)

for organs like kidneys where the technology of renal dialysis permits

some flexibility in determining the timing of a transplant. Similarly,

a different approach may also be taken where such ``rescue'' techniques

are available for other organs. Such alternatives may be used,

consistent with sound medical judgment.

Other commenters suggested that the concepts of using sound medical

judgment, avoidance of futile transplants or wastage of organs, and

promotion of the efficient use of organs should be applicable to all

the performance goals. Language adopting this suggestion is now found

in Sec. 121.8(a)(5).

We have added to Sec. 121.8(a)(5) a provision that allocation

policy seek to promote patient access to transplants, an issue Congress

asked the IOM to address. As discussed above, we have also added at

Sec. 121.8(a)(7) language to promote compliance with and enforcement of

approved allocation policies.

We have revised the discussion of medical urgency now found in

Sec. 121.8(b)(2). We have made clear that the need to rank patients or

categories of patients in order of decreasing medical urgency only

applies to otherwise medically appropriate candidates for transplants.

This is consistent with the provisions found in Sec. 121.8(a) that

require allocation policies be developed in accordance with sound

medical judgment and avoidance of futile transplants and organ wastage.

[[Page 56657]]

Some commenters suggested that the rule was unclear as to how

``medical urgency'' applies to kidney allocation policy. We revised

this section in response to comments that the term ``status

categories,'' as currently used for liver and heart patients, is not

used for kidney patients. (Instead, a point system is used to rank

patients when an organ becomes available.) The use of the term

``patients or categories of patients'' in this section makes clear that

ranking patients rather than categories of patients is permitted under

this rule. As discussed above, we intend for ranking to be applied in

the context of the factors listed in Sec. 121.8(a), especially in

accordance with sound medical judgment. Therefore, we believe that

there may well be different approaches to kidney allocation policy than

those for other types of organs, perhaps along the lines of the current

policies, which take into account such factors as immunologic

compatibility between the donor and patient, whether the patient's

immune system is highly sensitized, and other medical factors.

Commenters suggested that the Department closely monitor the

changes to allocation policies made after the initial reviews required

under this section to ensure that the new policies are achieving the

desired improvements in the allocation system. The Department intends

to monitor the effects of these changes closely and in consultation

with the OPTN. In addition to this monitoring and consultation, the

Department will formally determine whether further changes are

necessary six months and 12 months after the changes to allocation

policies made after the initial reviews go into effect.

Finally, as discussed above, we have given the OPTN additional

flexibility with respect to performance indicators, including waiting

times, in response both to comments received and the IOM report.

The Department wishes to emphasize, however, that these changes are

not intended to limit the ability of the OPTN to address special

situations such as the unique needs of young children.

10. Department of Veterans Affairs Hospitals

The term ``Dean's Committee'' has been deleted from

Sec. 121.9(a)(3), as this is not a term currently used by the

Department of Veterans Affairs. Currently, the Department of Veterans

Affairs, Veterans Health Administration, designates specific VA medical

centers to carry out organ transplantation. To cover the possibility

that transplants may also be carried out in other Federal hospitals, as

well as those owned and operated by the Department of Defense (DoD),

transplant programs in DoD or other Federal hospitals have been added

to those eligible to receive organs for transplantation under

Sec. 121.9(a).

11. Enforcement

Section 121.10(c)(1) has been edited to clarify that appropriate

enforcement action may include termination of a transplant program's

reimbursement under Medicare and Medicaid. In addition, the Department

wishes to clarify that the regulation permits the OPTN to develop

policies that will contain lesser or intermediate level sanctions that

may be taken by the OPTN, but these policies must first be approved by

the Secretary in order for them to be enforceable.

12. Reporting Requirements

Section 121.11(b)(2) has been amended to include transplant program

costs among the items to be reported by transplant hospitals to the

OPTN and the Secretary. Although the language in the previously

published final rule was sufficiently broad to permit the Secretary to

specify that cost information be submitted, it was felt that its

specific inclusion in the rule would ensure that such information would

be made available on a timely basis when requested, consistent with

section 213. Because of the difficulty in defining costs for these

purposes, the Department will accept measures of resource utilization.

13. Effect of the Regulation on State Laws (former Sec. 121.12)

The inclusion of Sec. 121.12 in the 1998 regulation was intended to

be consonant with longstanding Constitutional principles regarding the

relationship between the Federal and State governments. It reflected

the HHS belief that Congress intended the statutory scheme it

established under NOTA to result ``in the nationwide distribution of

organs equitably among transplant patients.'' Section 372(b)(2)(D) of

the Public Heath Service Act. Nevertheless, because the Department

views this result as flowing from the statutory scheme, the section of

the regulation articulating the Department's views on the matter is

unnecessary as a legal matter. Accordingly, Sec. 121.12 has been

removed.

14. Advisory Committee on Organ Transplantation

The Department intends to implement the recommendation of the IOM,

as discussed above, to create an independent, multidisciplinary

scientific advisory board which will assist the Secretary in,

``ensuring that the system of organ procurement and transplantation is

grounded on the best available medical science and is as effective and

as equitable as possible.'' Constitution of such an advisory committee

and its consultation by the Secretary, as appropriate, in the words of

the IOM, ``would also enhance public confidence in the integrity and

effectiveness of the system.'' The Department has added a new

Sec. 121.12 to provide for the establishment of an Advisory Committee

on Organ Transplantation. The Committee, to be established in

accordance with the Federal Advisory Committee Act [5 U.S.C. App.],

will be available to the Secretary to provide comments on proposed OPTN

policies and other matters related to transplantation. The Committee

will be composed of individuals drawn from diverse backgrounds such as

health care public policy, transplantation medicine and surgery, non-

physician transplant professions, biostatistics, immunology, health

economics, epidemiology, bioethics, and law. As part of this process of

establishing the Committee, the Secretary intends to solicit

nominations for Committee members from the transplant community and the

general public.

IV. Impact Analyses

We have examined the impact of this amendatory language as required

by Executive Order 12866, section 202 of the Unfunded Mandates Reform

Act of 1995 (Pub. L. 104-4) and the Regulatory Flexibility Act (RFA)

(Pub. L. 96-354). Executive Order 12866 directs agencies to assess

costs and benefits of available regulatory alternatives and, when

regulation is necessary, to select regulatory approaches that maximize

benefits. The Unfunded Mandates Reform Act of 1995 also requires that

agencies prepare an assessment of anticipated costs and benefits before

proposing any rule that may mandate an annual expenditure by State,

local, or tribal governments of $100 million or more.

Under the Regulatory Flexibility Act (5 U.S.C. 601-612), if an

action has a significant economic effect on a substantial number of

small businesses, the Secretary must specifically consider the effects

on small business entities and analyze regulatory options that could

lessen the impact of the rule.

Section 1102(b) of the Social Security Act requires us to prepare a

regulatory impact analysis for any regulation that may have a

significant impact on the operations of a substantial number of small

rural hospitals.

[[Page 56658]]

The amendatory language set forth in this document makes no changes

that have a significant economic effect on State, local or tribal

governments, hospitals or patients; therefore, we certify that no

additional regulatory analysis is required. We have also concluded,

based on the findings of the Institute of Medicine and the General

Accounting Office under section 213(b), discussed earlier in this

Preamble, and the Secretary certifies, that this amendatory language

would not have a significant economic impact on a substantial number of

small entities; therefore, a regulatory flexibility analysis is not

required.

We are also not preparing a rural impact statement since we have

determined, and the Secretary certifies, that this amendatory language

would not have a significant impact on the operations of a substantial

number of small rural hospitals.

The earlier analyses from the April 2, 1998, final rule remain

applicable to that rule and are not altered by these amendments.

List of Subjects in 42 CFR Part 121

Health care, Hospitals, Organ transplantation, Reporting and

recordkeeping requirements.

Dated: October 13, 1999.

Claude Earl Fox,

Administrator, Health Resources and Services Administration.

Approved: October 15, 1999.

Donna E. Shalala,

Secretary.

Accordingly, 42 CFR part 121 is amended as follows:

PART 121--ORGAN PROCUREMENT AND TRANSPLANTATION NETWORK

1. The authority citation for part 121 is revised to read as

follows:

Authority: Sections 215, 371-376 of the Public Health Service

Act (42 U.S.C. 216, 273-274d); sections 1102, 1106, 1138 and 1871 of

the Social Security Act (42 U.S.C. 1302, 1306, 1320b-8 and 1395hh).

2. Paragraph (b) of Sec. 121.1 is revised to read as follows:

Sec. 121.1 Applicability.

* * * * *

(b) In accordance with section 1138 of the Social Security Act,

hospitals in which organ transplants are performed and which

participate in the programs under titles XVIII or XIX of the Social

Security Act, and organ procurement organizations designated under

section 1138(b) of the Social Security Act, are subject to the

requirements of this part.

3. Amend Sec. 121.2 as follows:

a. Remove the definition for the ``National list''.

b. Amend the definition of ``OPTN computer match program'' by

revising the words ``national list'' to read ``waiting list''.

c. Amend the definition of ``Organ'' by removing the words ``and

for the purpose of the Scientific Registry, the term also includes bone

marrow''.

d. Amend the definition of ``Organ procurement organization'' by

revising the words ``Section 1138(b)'' to read ``section 1138(b)''.

e. Amend the definition of ``Organ procurement and transplantation

network or OPTN'' by revising the words ``Section 372'' to read

``section 372''.

f. Amend the definition of ``Scientific Registry'' by revising the

words ``Section 373'' to read ``section 373''.

g. Amend the definition of ``Transplant candidate'' by revising the

words national list'' to read ``waiting list''.

h. Add a definition for ``Waiting list'' in alphabetical order.

The addition reads as follows:

Sec. 121.2 Definitions.

* * * * *

Waiting list means the OPTN computer-based list of transplant

candidates.

4. Amend Sec. 121.3 as follows:

a. Revise the heading of paragraph (a).

b. Revise paragraph (a)(1).

c. Remove paragraph (a)(2).

d. Remove paragraph (a)(3).

e. Remove paragraph (a)(4).

f. Remove the heading of paragraph (b).

g. Redesignate paragraph (b)(1) as paragraph (a)(2) and revise it.

h. Redesignate paragraph (b)(2) as paragraph (a)(3) and amend the

newly designated paragraph (a)(3) by removing the paragraph heading.

i. Redesignate paragraph (b)(3) as paragraph (a)(4) and amend newly

designated paragraph (a)(4) by removing the paragraph heading.

j. In newly designated paragraph (a)(4)(ii), revise the term

``potential transplant candidates'' to read ``transplant candidates,

transplant recipients, organ donors and family members''.

k. Remove paragraph (b)(4).

l. Redesignate paragraph (c) as paragraph (b).

m. Redesignate paragraph (d) as paragraph (c) and revise the word

``Status'' in the heading to read ``status''.

n. Redesignate paragraph (e) as paragraph (d) and revise it.

The revisions read as follows:

Sec. 121.3 The OPTN.

(a) Organization of the OPTN. (1) The OPTN shall establish a Board

of Directors of whatever size the OPTN determines appropriate. The

Board of Directors shall include:

(i) Approximately 50 percent transplant surgeons or transplant

physicians;

(ii) At least 25 percent transplant candidates, transplant

recipients, organ donors and family members. These members should

represent the diversity of the population of transplant candidates,

transplant recipients, organ donors and family members served by the

OPTN including, to the extent practicable, the minority and gender

diversity of this population. These members shall not be employees of,

or have a similar relationship with OPOs, transplant centers, voluntary

health organizations, transplant coordinators, histocompatibility

experts, or other non-physician transplant professionals; however, the

Board may waive this requirement for not more than 50 percent of these

members; and

(iii) Representatives of OPOs, transplant hospitals, voluntary

health associations, transplant coordinators, histocompatibility

experts, non-physician transplant professionals, and the general

public.

(2) The Board of Directors shall elect an Executive Committee from

the membership of the Board. The Executive Committee shall include at

least one general public member, one OPO representative, approximately

50 percent transplant surgeons and transplant physicians, and at least

25 percent transplant candidates, transplant recipients, organ donors,

and family members.

* * * * *

(d) Effective date. The organization designated by the Secretary as

the OPTN shall have until June 30, 2000, or six months from its initial

designation as the OPTN, whichever is later, to meet the requirements

of this section, except that the Secretary may extend such period for

good cause.

5. Amend Sec. 121.4 as follows:

a. Revise paragraph (a)(3)(i).

b. Revise paragraph (a)(3)(ii).

c. Revise paragraph (b)(2).

d. Revise paragraph (c).

e. Revise paragraph (d).

f. Amend paragraph (e) introductory text by adding the word

``shall'' after the words ``implement policies and'', and by revising

the word ``them.'' in paragraph (e)(1) to read ``them; and''.

The revisions read as follows:

[[Page 56659]]

Sec. 121.4. OPTN policies: Secretarial review and appeals.

(a) * * *

(3) * * *

(i) Ensuring that payment of the registration fee is not a barrier

to listing for patients who are unable to pay the fee;

(ii) Procedures for transplant hospitals to make reasonable efforts

to obtain from all available sources, financial resources for patients

unable to pay such that these patients have an opportunity to obtain a

transplant and necessary follow-up care;

* * * * *

(b) * * *

(2) Provide to the Secretary, at least 60 days prior to their

proposed implementation, proposed policies it recommends to be

enforceable under Sec. 121.10 (including allocation policies). These

policies will not be enforceable until approved by the Secretary. The

Board of Directors shall also provide to the Secretary, at least 60

days prior to their proposed implementation, proposed policies on such

other matters as the Secretary directs. The Secretary will refer

significant proposed policies to the Advisory Committee on Organ

Transplantation established under Sec. 121.12, and publish them in the

Federal Register for public comment. The Secretary also may seek the

advice of the Advisory Committee on Organ Transplantation established

under Sec. 121.12 on other proposed policies, and publish them in the

Federal Register for public comment. The Secretary will determine

whether the proposed policies are consistent with the National Organ

Transplant Act and this part, taking into account the views of the

Advisory Committee and public comments. Based on this review, the

Secretary may provide comments to the OPTN. If the Secretary concludes

that a proposed policy is inconsistent with the National Organ

Transplant Act or this part, the Secretary may direct the OPTN to

revise the proposed policy consistent with the Secretary's direction.

If the OPTN does not revise the proposed policy in a timely manner, or

if the Secretary concludes that the proposed revision is inconsistent

with the National Organ Transplant Act or this part, the Secretary may

take such other action as the Secretary determines appropriate, but

only after additional consultation with the Advisory Committee on the

proposed action.

(c) The OPTN Board of Directors shall provide the membership and

the Secretary with copies of its policies as they are adopted, and make

them available to the public upon request. The Secretary will publish

lists of OPTN policies in the Federal Register, indicating which ones

are enforceable under Sec. 121.10 or subject to potential sanctions of

section 1138 of the Social Security Act. The OPTN shall also

continuously maintain OPTN policies for public access on the Internet,

including current and proposed policies.

(d) Any interested individual or entity may submit to the Secretary

in writing critical comments related to the manner in which the OPTN is

carrying out its duties or Secretarial policies regarding the OPTN. Any

such comments shall include a statement of the basis for the comments.

The Secretary will seek, as appropriate, the comments of the OPTN on

the issues raised in the comments related to OPTN policies or

practices. Policies or practices that are the subject of critical

comments remain in effect during the Secretary's review, unless the

Secretary directs otherwise based on possible risk to the health of

patients or to public safety. The Secretary will consider the comments

in light of the National Organ Transplant Act and the regulations under

this part and may consult with the Advisory Committee on Organ

Transplantation established under Sec. 121.12. After this review, the

Secretary may:

(1) Reject the comments;

(2) Direct the OPTN to revise the policies or practices consistent

with the Secretary's response to the comments; or

(3) Take such other action as the Secretary determines appropriate.

* * * * *

Sec. 121.5 [Amended]

6. Amend Sec. 121.5 as follows:

a. In paragraph (a), add the words``, consistent with the OPTN's

criteria under Sec. 121.8(b)(1),'' after the word ``individuals''.

b. In paragraph (b), revise the words ``national list'' to read

``waiting list''.

c. In paragraph (c), revise the words ``national list'' to read

``waiting list'' and add the phrase ``calculated to cover (together

with contract funds awarded by the Secretary) the reasonable costs of

operating the OPTN and shall be'' after the words ``amount of such fee

shall be'.

7. Paragraph (b) of Sec. 121.6 is revised to read as follows:

Sec. 121.6 Organ procurement.

* * * * *

(b) HIV. The OPTN shall adopt and use standards for preventing the

acquisition of organs from individuals known to be infected with human

immunodeficiency virus.

* * * * *

Sec. 121.7 [Amended]

8. Paragraph (d) of Sec. 121.7 is amended by revising the words

``paragraph (b) of this section'' to read ``paragraph (b)(2) of this

section'.

9. Revise Sec. 121.8 to read as follows:

Sec. 121.8 Allocation of organs.

(a) Policy development. The Board of Directors established under

Sec. 121.3 shall develop, in accordance with the policy development

process described in Sec. 121.4, policies for the equitable allocation

of cadaveric organs among potential recipients. Such allocation

policies:

(1) Shall be based on sound medical judgment;

(2) Shall seek to achieve the best use of donated organs;

(3) Shall preserve the ability of a transplant program to decline

an offer of an organ or not to use the organ for the potential

recipient in accordance with Sec. 121.7(b)(4)(d) and (e);

(4) Shall be specific for each organ type or combination of organ

types to be transplanted into a transplant candidate;

(5) Shall be designed to avoid wasting organs, to avoid futile

transplants, to promote patient access to transplantation, and to

promote the efficient management of organ placement;

(6) Shall be reviewed periodically and revised as appropriate;

(7) Shall include appropriate procedures to promote and review

compliance including, to the extent appropriate, prospective and

retrospective reviews of each transplant program's application of the

policies to patients listed or proposed to be listed at the program;

and

(8) Shall not be based on the candidate's place of residence or

place of listing, except to the extent required by paragraphs (a)(1)-

(5) of this section.

(b) Allocation performance goals. Allocation policies shall be

designed to achieve equitable allocation of organs among patients

consistent with paragraph (a) of this section through the following

performance goals:

(1) Standardizing the criteria for determining suitable transplant

candidates through the use of minimum criteria (expressed, to the

extent possible, through objective and measurable medical criteria) for

adding individuals to, and removing candidates from, organ transplant

waiting lists;

(2) Setting priority rankings expressed, to the extent possible,

through objective and measurable medical criteria, for patients or

categories of patients who are medically suitable candidates for

transplantation to receive transplants. These rankings

[[Page 56660]]

shall be ordered from most to least medically urgent (taking into

account, in accordance with paragraph (a) of this section, and in

particular in accordance with sound medical judgment, that life

sustaining technology allows alternative approaches to setting priority

ranking for patients). There shall be a sufficient number of categories

(if categories are used) to avoid grouping together patients with

substantially different medical urgency;

(3) Distributing organs over as broad a geographic area as feasible

under paragraphs (a)(1)-(5) of this section, and in order of decreasing

medical urgency; and

(4) Applying appropriate performance indicators to assess

transplant program performance under paragraphs (c)(2)(i) and

(c)(2)(ii) of this section and reducing the inter-transplant program

variance to as small as can reasonably be achieved in any performance

indicator under paragraph (c)(2)(iii) of this section as the Board

determines appropriate, and under paragraph (c)(2)(iv) of this section.

If the performance indicator ``waiting time in status'' is used for

allocation purposes, the OPTN shall seek to reduce the inter-transplant

program variance in this indicator, as well as in other selected

performance indicators, to as small as can reasonably be achieved,

unless to do so would result in transplanting less medically urgent

patients or less medically urgent patients within a category of

patients.

(c) Allocation performance indicators. (1) Each organ-specific

allocation policy shall include performance indicators. These

indicators must measure how well each policy is:

(i) Achieving the performance goals set out in paragraph (b) of

this section; and

(ii) Giving patients, their families, their physicians, and others

timely and accurate information to assess the performance of transplant

programs.

(2) Performance indicators shall include:

(i) Baseline data on how closely the results of current allocation

policies approach the performance goals established under paragraph (b)

of this section;

(ii) With respect to any proposed change, the amount of projected

improvement in approaching the performance goals established under

paragraph (b) of this section;

(iii) Such other indicators as the Board may propose and the

Secretary approves; and

(iv) Such other indicators as the Secretary may require.

(3) For each organ-specific allocation policy, the OPTN shall

provide to the Secretary data to assist the Secretary in assessing

organ procurement and allocation, access to transplantation, the effect

of allocation policies on programs performing different volumes of

transplants, and the performance of OPOs and the OPTN contractor. Such

data shall be required on performance by organ and status category,

including program-specific data, OPO-specific data, data by program

size, and data aggregated by organ procurement area, OPTN region, the

Nation as a whole, and such other geographic areas as the Secretary may

designate. Such data shall include the following measures of inter-

transplant program variation: risk-adjusted total life-years pre-and

post-transplant, risk-adjusted patient and graft survival rates

following transplantation, risk-adjusted waiting time and risk-adjusted

transplantation rates, as well as data regarding patients whose status

or medical urgency was misclassified and patients who were

inappropriately kept off a waiting list or retained on a waiting list.

Such data shall cover such intervals of time, and be presented using

confidence intervals or other measures of variance, as may be required

to avoid spurious results or erroneous interpretation due to small

numbers of patients covered.

(d) Transition patient protections.--(1) General. When the OPTN

revises organ allocation policies under this section, it shall consider

whether to adopt transition procedures that would treat people on the

waiting list and awaiting transplantation prior to the adoption or

effective date of the revised policies no less favorably than they

would have been treated under the previous policies. The transition

procedures shall be transmitted to the Secretary for review together

with the revised allocation policies.

(2) Special rule for initial revision of liver allocation policies.

When the OPTN transmits to the Secretary its initial revision of the

liver allocation policies, as directed by paragraph (e)(1) of this

section, it shall include transition procedures that, to the extent

feasible, treat each individual on the waiting list and awaiting

transplantation on October 20, 1999 no less favorably than he or she

would have been treated had the revised liver allocation policies not

become effective. These transition procedures may be limited in

duration or applied only to individuals with greater than average

medical urgency if this would significantly improve administration of

the list or if such limitations would be applied only after

accommodating a substantial preponderance of those disadvantaged by the

change in the policies.

(e) Deadlines for initial reviews. (1) The OPTN shall conduct an

initial review of existing allocation policies and, except as provided

in paragraph (e)(2) of this section, no later than November 16, 2000

shall transmit initial revised policies to meet the requirements of

paragraphs (a) and (b) of this section, together with supporting

documentation to the Secretary for review in accordance with

Sec. 121.4.

(2) No later than February 15, 2000 the OPTN shall transmit revised

policies and supporting documentation for liver allocation to meet the

requirements of paragraphs (a) and (b) of this section to the Secretary

for review in accordance with Sec. 121.4. The OPTN may transmit these

materials without seeking further public comment under Sec. 121.4(b).

(f) Secretarial review of policies, performance indicators, and

transition patient protections. The OPTN's transmittal to the Secretary

of proposed allocation policies and performance indicators shall

include such supporting material, including the results of model-based

computer simulations, as the Secretary may require to assess the likely

effects of policy changes and as are necessary to demonstrate that the

proposed policies comply with the performance indicators and transition

procedures of paragraphs (c) and (d) of this section.

(g) Variances. The OPTN may develop, in accordance with Sec. 121.4,

experimental policies that test methods of improving allocation. All

such experimental policies shall be accompanied by a research design

and include data collection and analysis plans. Such variances shall be

time limited. Entities or individuals objecting to variances may appeal

to the Secretary under the procedures of Sec. 121.4.

(h) Directed donation. Nothing in this section shall prohibit the

allocation of an organ to a recipient named by those authorized to make

the donation.

10. Amend Sec. 121.9 as follows:

a. Amend paragraph (a)(1) by removing the words ``and Medicaid''

after the word ``Medicare''.

b. Amend paragraph (a)(2)(vi) by adding a comma after the word

``radiology''.

c. Amend paragraph (a)(2)(vii) by adding a comma after the word

``recipients''.

d. Revise paragraph (a)(3).

The revision reads as follows:

Sec. 121.9 Designated transplant program requirements.

(a) * * *

(3) Be a transplant program in a Department of Veterans Affairs,

[[Page 56661]]

Department of Defense, or other Federal hospital.

* * * * *

Sec. 121.10 [Amended]

11. Amend paragraph (c)(1) of Sec. 121.10 by removing the word

``or'' before the words ``termination of an OPO's reimbursement'', and

by adding the words ``, or such other compliance or enforcement

measures contained in policies developed under Sec. 121.4'' after the

words ``Social Security Act''.

12. Amend Sec. 121.11 as follows:

a. Revise paragraph (a)(1)(i) by removing the word ``national''

after the word ``computerized''.

b. Revise paragraph (b)(1)(iv).

c. Amend paragraph (b)(2) by adding the words ``costs and'' before

the word ``performance''.

The revision reads as follows:

Sec. 121.11 Record maintenance and reporting requirements.

* * * * *

(b) * * *

(1) * * *

(iv) Make available to the public timely and accurate program-

specific information on the performance of transplant programs. This

shall include free dissemination over the Internet, and shall be

presented, explained, and organized as necessary to understand,

interpret, and use the information accurately and efficiently. These

data shall be updated no less frequently than every six months (or such

longer period as the Secretary determines would provide more useful

information to patients, their families, and their physicians), and

shall include risk-adjusted probabilities of receiving a transplant or

dying while awaiting a transplant, risk-adjusted graft and patient

survival following the transplant, and risk-adjusted overall survival

following listing for such intervals as the Secretary shall prescribe.

These data shall include confidence intervals or other measures that

provide information on the extent to which chance may influence

transplant program-specific results. Such data shall also include such

other cost or performance information as the Secretary may specify,

including but not limited to transplant program-specific information on

waiting time within medical status, organ wastage, and refusal of organ

offers. These data shall also be presented no more than six months

later than the period to which they apply;

* * * * *

13. Sec. 121.12 is revised to read as follows:

Sec. 121.12 Advisory Committee on Organ Transplantation.

The Secretary will establish, consistent with the Federal Advisory

Committee Act, the Advisory Committee on Organ Transplantation. The

Secretary may seek the comments of the Advisory Committee on proposed

OPTN policies and such other matters as the Secretary determines.

[FR Doc. 99-27456 Filed 10-18-99; 9:46 am]

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