- Provision of the best care
to the largest number of patients possible, with priority
given to patients, and not individual physicians or
transplant centers.
- Insuring equal access to
transplantation regardless of race gender or ability
to pay.
- The improvement of transplant
outcomes based on the sound scientific principles.
In preparation for federal
legislation relating to transplantation (including the
reauthorization of the Transplant Act of 1984 as well
as funding for the National Institutes
of Health (NIH) and proposed administrative initiatives
by the Executive Branch), the ASTP reviewed two important
federal government supported studies of organ transplantation.
We chose as a frame of reference the Congressionally
mandated (P.L. 98-507) Department of Health and Human
Services Report of the Task Force on Organ Transplantation,
dated April 1986. We prepared a "score card" to
determine which of the Task Force recommendations should
have been and were not implemented during the past ten
years. In addition, we reviewed the landmark 1991 Institute
of Medicine (IOM) report "Kidney Failure and the
Federal Government" funded in the Ominbus Reconciliation
Act (OBRA) 1987.
It
is clear that considerable progress has been achieved
since the release of the Task Force Report in 1986 which
focused on enhancing organ donation, the quality of patient
care, access, coverage, basic and clinical research.
For example, the sophisticated, volunteer-run Organ Procurement
Transplant Network (OPTN) has developed a single national
system for organ sharing, the United Network for Organ
Sharing (UNOS). Congress extended anti-rejection drug
benefits for kidney transplant patients from 12 to 36
months under the Medicare entitlement. Also, there have
been steady improvements in transplantation morbidity
and mortality. Surveys confirm that an overwhelming percentage
of Americans are aware and are in favor of the "gift
of life" related to organ donation. The organ procurement
organizations (OPOs) were consolidated from 120 to 64.
Required request laws were enacted in all 50 states.
The amount of support for biomedical research related
to transplantation at NIH has increased in the past decade.
Not withstanding these advances, our "scorecard" suggests
there is much more to be done in the next decade to build
on the advances of the previous decade. The agenda of
the Task Force and the recommendations of the IOM report
suggest to us specific areas that need to be addressed
in the next ten years which we hope will be designated
as the Decade of Transplantation:
- Organ Donation
- Organ
Allocation
- Access
to Transplantation
- Biomedical
Research
1.
Organ Donation: The harsh reality is
that there is a growing disparity between the number
of available organs and the number of patients on
the transplant waiting list. In spite of very considerable
improvements in the system of organ retrieval and
allocation it simply has not kept pace with demand.
The following charts place the seriousness of the
problem in perspective. The number of patients on
the UNOS waiting list in 1990 was 21,914 and has
risen by 133% to over 51,000 currently. The number
of donors has only increased by 43% over the same
time period. The increased number on the lists leads
to longer waiting times and tragically the death
of nearly 10 patients on the list every day due to
progressive organ failure.
U.S.
Organ Transplants by Organ and Donor Type - 1988
through 1995
|
Organ/Donor
Type
|
Year
|
|
1988
|
1989
|
1990
|
1991
|
1992
|
1993
|
1994
|
1995
|
| Kidney |
Cadaveric |
7230 |
7086 |
7785 |
7732 |
7697 |
8170 |
8383 |
8598 |
| Living |
1811 |
1902 |
2094 |
2390 |
2533 |
2850 |
3009 |
3212 |
| Total |
9041 |
8988 |
9879 |
10122 |
10230 |
11020 |
11392 |
11810 |
| Liver |
Cadaveric |
1713 |
2199 |
2676 |
2931 |
3031 |
3404 |
3592 |
3882 |
| Living |
0 |
2 |
14 |
22 |
33 |
36 |
60 |
44 |
| Total |
1713 |
2201 |
2690 |
2953 |
3064 |
3440 |
3652 |
3926 |
| Pancreas |
Cadaveric |
244 |
413 |
526 |
530 |
554 |
772 |
840 |
1022 |
| Living |
5 |
4 |
2 |
1 |
3 |
2 |
2 |
6 |
| Total |
249 |
417 |
528 |
531 |
557 |
774 |
842 |
1028 |
| Intestine |
Cadaveric |
0 |
0 |
5 |
12 |
22 |
34 |
23 |
44 |
| Living |
0 |
0 |
0 |
0 |
0 |
0 |
0 |
1 |
| Total |
0 |
0 |
5 |
12 |
22 |
34 |
23 |
45 |
| Heart |
Cadaveric |
1669 |
1696 |
2096 |
2121 |
2170 |
2295 |
2338 |
2360 |
| Living |
7 |
9 |
12 |
4 |
1 |
2 |
3 |
0 |
| Total |
1676 |
1705 |
2108 |
2125 |
2171 |
2297 |
2341 |
2360 |
| Lung |
Cadaveric |
33 |
93 |
202 |
401 |
535 |
659 |
708 |
848 |
| Living |
0 |
0 |
1 |
4 |
0 |
7 |
15 |
23 |
| Total |
33 |
93 |
203 |
405 |
535 |
666 |
723 |
871 |
| Heart-Lung |
Cadaveric |
74 |
67 |
52 |
51 |
48 |
60 |
70 |
69 |
| Living |
0 |
0 |
0 |
0 |
0 |
0 |
0 |
0 |
| Total |
74 |
67 |
52 |
51 |
48 |
60 |
70 |
69 |
| Total
Organs |
Cadaveric |
10963 |
11554 |
13342 |
13778 |
14057 |
15394 |
15954 |
16823 |
| Living |
1823 |
1917 |
2123 |
2421 |
2570 |
2897 |
3089 |
3286 |
| Total |
12786 |
13471 |
15465 |
16199 |
16627 |
18291 |
19043 |
20109 |
Source:
UNOS Scientific Registry data as of September 7, 1996
OPTN
Waiting List at Year's End - 1988 to 1995
|
All
Organs
|
|
Year
|
Kidney
|
Liver
|
Pancreas
|
Kidney
- Pancreas
|
Heart
|
Lung
|
Heart
- Lung
|
Total
|
|
1988
|
13943 |
616 |
163 |
0 |
1030 |
69 |
205 |
16026 |
|
1989
|
16294 |
827 |
320 |
0 |
1320 |
94 |
240 |
19095 |
|
1990
|
17883 |
1237 |
473 |
0 |
1788 |
308 |
225 |
21914 |
|
1991
|
19352 |
1676 |
600 |
0 |
2267 |
670 |
154 |
24719 |
|
1992
|
22376 |
2323 |
126 |
778 |
2690 |
942 |
180 |
29415 |
|
1993
|
24973 |
2997 |
183 |
923 |
2834 |
1240 |
202 |
33352 |
|
1994
|
27498 |
4059 |
222 |
1067 |
2933 |
1625 |
205 |
37609 |
|
1995
|
31149 |
5701 |
286 |
1239 |
3468 |
1932 |
208 |
43983 |
Source:
UNOS OPTN Waiting List on the last day of each year
Furthermore, the median waiting times have increased dramatically for all organs
from 1988 to 1995. For example, the wait for a kidney went from 400 days
to 842 days, a liver 33 days to 254 days and a heart 122 days to 313 days.
The
reasons for the lack of transplantable organs continue
to bedevil the entire community, but family refusal remains
the leading cause of loss of potential donors, averaging
over 40%. The Task Force suggested "that substantial
improvements in organ donation would ensue through new
innovations and expanded programs in public and professional
education and coordination of efforts of the many organizations
and agencies that engage in these activities." Five
years later the Institute of Medicine Committee recommended
that "increasing the donation of kidneys receive
the highest priority in the coming decades." The
Task Force expressed concern regarding the fragmentation
of this effort, which unfortunately is a problem. It
is hard to measure the impact of any single effort in
organ donation given the delay between signing a donor
card and actual donation.
We
urge federal and state governments, providers, and the
patient communities to establish a working compact directed
at translating the extremely high public awareness of
the "gift of life" into a pro-active national
effort to increase organ donation. To address this fundamental
concern we propose that the Congress reauthorize the
Transplant Act of 1984 and broaden the authority of federal
agencies to increase support for organ donation initiatives
(e.g. the President's proposed budget increase for the
Division of Organ Transplantation). It is noteworthy
that the Transplant Act of 1984 has not been reauthorized
since 1990. We hope to see congressional action in the
105th Congress that will incorporate our recommendations
into action.
- The
Congress should establish a federal government council
to coordinate the transplant programs of the Health
Care Financing Administration (HCFA), the Assistant
Secretary of Health, the NIH, FDA and the Division
of Transplantation (DOT). It is very important to bring
these participating agencies together to share and
coordinate their resources. Transplant professionals
should be included to provide insight into the problems
and possible solutions.
- The
Congress should establish a certification process for
the OPO's and Organ Procurement Coordinators. This
should set high standards for organ retrieved rates
among OPO's and find solutions to help the low retrieving
OPO's. Barring lack of improvement, mechanisms to allow
better managed OPO's to take over retrieval areas should
be put in place.
- The
Congress should insist that the annual report on the "State
of Transplantation" be executed as provided by
statute, to date this has not been done by the Department
of Health and Human Services (HHS). The report should
include the following information: who is paying for
the transplants, how much they cost, and what percentage
of patients are in the managed care setting. It should
also contain information on access to care and coverage.
- The
Congress should provide for a study of the proliferation
of transplant centers and in particular determine the
minimum volume necessary to optimize outcomes. In addition
a study should compare center volume and experience.
Proliferation of centers must also be studied in the
context of cost effectiveness and patient access to
determine the optimal number of transplant centers.
- The
Congress should authorize increased directed funds
to identify and implement new and innovative programs
to increase organ donation.
- The
Congress should direct the Department of HHS to issue
the multiple transplantation regulations promulgated
by UNOS.
- The
Congress should dramatically increase direct support
for biomedical research. (See section
on Biomedical Research).
In
addition to public initiatives, we propose that the private
sector embark on a new paradigm that embraces past successes
and the wisdom gained from failed ventures. We will encourage
the community to reach a consensus among the providers,
patients, business, and labor to identify and support
the best possible public donation programs. This should
include continued support and, where indicated, an expansion
of existing programs, such as the national efforts of
the Coalition on Donation, the National Kidney Foundation
and UNOS.
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2.
Organ Allocation: Many of the Task Force
recommendations on organ sharing have been carried
out through the highly successful, single, national
system for organ sharing. The volunteers at UNOS
continue to work towards a fair and equitable allocation
of organs for the entire country. In addition, the
Scientific Registry has done a good job of providing
scientific and clinical data relating to transplantation.
We continue to share the Task Force concerns about
the high discard rates of organs and the lack of
progress in regionalization of histocompatibility
typing. We propose:
- The
Scientific Registry develop policies to make the system
more user friendly concerning access to data and its
use.
- A
mechanism to minimize the persistent problem of organ
discard rates. In 1995, 1,200 kidneys, 500 livers and
250 hearts were procured but discarded.
- The
implementation of the Task Force recommendation to
regionalize histocompatibility typing. To accomplish
this, the federal government needs only to authorize
the OPTN to proceed with the process.
- The
Congress needs to embrace the OPTN guidelines so that
the contractor would have the authority to resolve
disputes in allocation.
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3.
Access to Transplantation: The issues
related to access to transplantation are complex
and controversial for the entire transplant community.
Certain principles, however, are widely accepted
by all parties involved in transplantation. First,
each donated organ is a national resource and subject
to stewardship by all segments of our society. Second,
to paraphrase the observation of Speaker O'Neill
that "all politics are local," most transplantation
issues are local. However, there are key issues which
need to be addressed in the next decade to assure
the national system is fair relating to the equitable
distribution of organs.
The
ASTP believes that the current system of patient selection
and allocation is a process in progress. To build upon
and enhance the existing system we propose:
- The
development of uniform medical criteria for listing
each solid organ category (heart, lung, liver, pancreas
and kidney).
- To
assure that patients make informed choices regarding
transplantation, the HCFA and private insurance carriers
should annually advise patients of their treatment
options.
- All
patients that meet uniform, accepted criteria for a
transplant should have access to the procedure regardless
of their ability to pay.
- The
development of a national education program aimed at
the education of minorities about the "gift of
life," as well as the medical consequences of
a transplant. Critical to this effort is a thoughtful
review of previous minority education programs.
- The
government should extend Medicare coverage for transplant
patients for the life of the graft rather than the
current thirty-six (36) month coverage period.
- The
payment for anti-rejection drugs for transplants should
be extended from the current thirty-six (36) months
to the life of the graft. The ESRD program for example,
would experience dramatic savings by extending graft
life through appropriate drug regimens.
- With
Medicaid reform the central government should assure
that all states have uniform eligibility and coverage
criteria for transplantation.
- The
growth of managed care has raised very serious questions
about access to transplantation. The ASTP strongly
recommends that the federal government assert its leadership
to assure each managed care plan provide equal access
to transplantation.
- There
is disturbing evidence that transplant recipients experience
employment discrimination. The Congress should schedule
hearings to determine the extent of discrimination
in employment, insurance coverage, etc. and move to
amend the Society Security Act, the job training program,
and the Vocational Rehabilitation Act to eliminate
discrimination and design programs to share the cost
of coverage with transplant recipients based on income
and remove disincentives.
Pediatric
patients comprise a small but extremely important sub-group
of transplant recipients. It is notable that the task
force on organ transplantation did not contain any pediatric
specialists and its report did not address the unique
characteristics of pediatric transplantation. UNOS, however,
has established a Pediatric Committee to address these
issues. As a result, pediatric patients are provided
preference in organ transplant allocation and some of
the unique indications for transplantation present in
pediatric recipients have been adopted as specific listing
criteria. Unfortunately, however, the unique but extremely
important aspects of pediatric transplantation may be
overlooked in the broader discussions.
We therefore propose: Organ
allocation to children to be given a high priority, listing
criteria for all organ transplant categories reflect
the unique indications for transplantation in pediatric
recipients, all funding sources, including Medicaid and
Medicare, private insurances and HMOs recognize the additional
cost necessary for appropriate provision of transplantation
procedures to children, particularly infants and young
children, children be provided access to specialized
pediatric transplant facilities and resources which address
their unique needs.
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4.
Biomedical Research: Research
is central to all of the other transplantation
proposals set forth in this paper. We submit that
increased funding for transplantation research
will lead to solutions that save lives. In 1986,
the Task Force recognized the importance of research
by suggesting it receive the highest priority.
The IOM report recommended "that basic research
continue to receive high priority." To set
science priorities, the Task Force queried surgeons,
internists, pathologists, immunologists, biologists,
and basic scientists concerning their views on
future transplantation research.
A majority of the respondents
replied that the chief obstacle to progress in organ
transplantation is both acute and chronic organ rejection
and the lack of better methods to suppress and monitor
the immune system. One specific area of focus is methods
to induce tolerance. Respondents also agreed on the need
to improve methods of preserving organs for longer periods
and understanding the organ injury that occurs with brain
death, and the need for more basic and clinical research.
The need for more research involving xenografts was also
cited. Research initiatives since 1986 have indeed made
progress in all of the areas cited by the Task Force.
In the area of liver transplantation, patient survival
has increased from 60% at one year in 1986 to over 80%
in 1995 through the use of newer immunosuppressive drugs
and better technique. Furthermore, research in preservation
has extended preservation from 4 to 24 hours.
The ASTP believes that we
are on the threshold of many important breakthroughs
in the areas of research cited by the Task Force such
as, rejection-immunosuppression tolerance and xenografts.
Next to issues related to the supply of organs for patients
on the waiting lists, those of basic and clinical research
are of the highest priority. Clinical and basic transplantation
funding at the NIH must be increased. We propose that
Congress, through the authorization and appropriations
process, expand the general transplantation research
authority of NIH. In particular, we recommend that Congress
designate the following high priority initiatives at
the NIDDK, the NIAID and the NHLBI.
- NHLBI
- Establish the creation
of databases for end stage heart disease to understand
the disease and plan for future needs. Heart
failure is the number one admitting volume diagnosis
in the Medicare system, the longest DRG, and
has the highest readmission rate. There is an
estimated one million people with advanced heart
failure and they impact the health care budget
as much or more than any other disease group.
Specifically, the creation of a database for
end-stage heart failure patients, similar to
the ESRD now funded by the NIDDK and HCFA.
- Clinical trials in
immunosuppression in heart transplantation.
- Research on chronic
rejection applying promising new technology such
as intravascular ultrasound.
- NIAID
- Basic and clinical
immunology stressing an understanding of immunologic
mechanisms of tolerance and autoimmunity, evaluation
of chronic transplant rejection, and immunosuppression
in transplant patients.
- Basic immunology
stressing the response to xenotransplants and
methods to overcome the response.
- Further structures
on identification and treatment of infectious
disease risks associated with xenotransplantation.
- NIDDK
- Studies to improve
the survival of renal transplants, including
improved mechanisms of donor and recipient matching.
- Trials to compare
the outcome of combined kidney-pancreas transplant
to kidney alone for diabetic patients.
- Priority increased
in funding for pancreatic islet cell transplantation.
In addition, we are encouraged by the cooperative
study undertaken by the NIDDK and HCFA concerning
pancreatic/kidney transplant patients.
- Continuation of the
liver transplant database.
Further, we recommend that
the NIAID be designated as the lead coordinating institute
for the NIH transplantation research effort in the next
decade, The Decade of Transplantation.
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Conclusion: The
message that the ASTP intends to send is that transplantation
in America has come a very long way since the original
Transplant Act of 1984 and the findings and recommendations
of the Task Force it established. The next decade should
build upon these accomplishments and address those
proposals that have been only partially addressed,
as well as new problems and challenges. At a time of
zero-based budgeting the task is to balance the need
with the cost. We submit the benefit to cost ratio
of transplantation is very favorable. In 1991, the
IOM, in reference to kidney transplants reported:
From Medicare's perspective,
a successful transplant is cost-effective compared
to dialysis. HCFA data show that the annualized Medicare
expenditure for a dialysis patient was $32,000. Patients
with functioning kidney grafts require a higher first-year
per-capita expenditure of $56,000, but they cost Medicare
only $6,400 on average in succeeding years. Given certain
assumptions about patient and graft survival rates,
it has been estimated that the cumulative dialysis
and transplantation cost reach a break-even point in
about three years. From then on transplantation provides
a net financial gain to Medicare (PW Eggers, HCFA,
personal communication, 1990).
It eventually comes down to
doing a better job. This means making sure required-request
laws are fully implemented in each state, seeking more
effective ways of educating the public and health care
professionals, initiating improvements of the OPO's and
their performance, enhancing transplantation biomedical
research in the public and private sectors and assuring
that access is fair and equitable so that no one in need
of a transplant is denied access for reasons of cost,
race, creed or gender. To meet these unfulfilled needs
certain changes must occur.
The
federal government must reassert its leadership in supporting
and fostering innovative
programs in cooperation with the private sector. The
private sector must move beyond its traditional response
to the needs of patients and question its assumptions
regarding donation, supply, access, and research. Both
the public and private sectors must explore every possible
way to have our citizens embrace Dr. Elbert Tuttle's
observations, "Sharing of essentials for survival
is one of the cherished attributes, whether it be food,
drink, shelter, blood, air, or organs. Becoming one's
brother's keeper enhances a donor's self esteem and generates
a sense of being valued in the recipient so that both
gain meaning in their lives."1
1. Controversies in
Organ Donation, A Summary Report. The National
Kidney Foundation, Inc., June 1993.
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In testimony during a Congressional
hearing, Organ Transplants: Choices and Criteria
Who Lives, Who Dies and Who Pays, Joshua Wiener,
Ph.D. of the Brooking Institute presented the following
table:
COST
PER QUALITY ADJUSTED YEARS OF PROLONGED LIFE BETWEEN
TREATMENT OPTIONS
|
WITHOUT TRANSPLANT
|
ADDITIONAL YRS.
|
TOTAL COST
|
COST PER YR.
|
| End Stage
Heart Disease |
.54
|
$40.000
|
$74.074
|
| End Stage Liver Disease |
.54
|
$50.000
|
$92.592
|
| Leukemia |
1.07
|
$60.000
|
$66.074
|
|
|
|
|
|
WITH TRANSPLANT
|
ADDITIONAL YRS.
|
TOTAL COST
|
COST PER YR
|
| End Stage
Heart Disease |
9.03
|
$210.000
|
$23.256
|
| End Stage Liver Disease |
9.03
|
$220.000
|
$24.363
|
| Leukemia |
9.03
|
$225.000
|
$30.991
|
