[Federal Register Volume 64, Number 202 (Wednesday, October 20, 1999)]
[Rules and Regulations]
[Pages 56650-56661]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 99-27456]
[[Page 56649]]
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Part VI
Department of Health and Human Services
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Health Resources and Services Administration
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42 CFR Part 121
Organ Procurement and Transplantation Network; Final Rule
Federal Register / Vol. 64, No. 202 / Wednesday, October 20, 1999 /
Rules and Regulations
[[Page 56650]]
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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).
[[Page 56651]]
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,
[[Page 56655]]
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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