Organ Transplants: Allocation Policies Include Special		 
Protections for Children (28-SEP-01, GAO-01-498).		 
								 
Pediatric patients in need of an organ transplant face a shortage
of donated organs. The number of pediatric organ donors has	 
remained relatively constant from 1991 to 2000, despite a drop in
potential donors. The number of adult donors rose 45 percent	 
during the same period, in large part because donor eligibility  
criteria have been expanded to include older donors and donors	 
with diseases that have been prohibited in the past. Waiting	 
lists for organs for pediatric patients have more than doubled.  
Compared to adults, however, children account for a small number 
of transplant candidates. The degree to which pediatric organs	 
are transplanted into adults varies by organ. Pediatric patients 
appear to be faring as well as or better than adult patients,	 
both while on the waiting list and after transplantation.	 
Allocation policies for kidneys, livers, and hearts provide	 
several protections for children awaiting transplants. The	 
priority a child receives takes into account differences between 
children and adults in the progression and treatment of end stage
organ disease, with the policies differing for each organ.	 
-------------------------Indexing Terms------------------------- 
REPORTNUM:   GAO-01-498 					        
    ACCNO:   A02049						        
  TITLE:     Organ Transplants: Allocation Policies Include Special   
Protections for Children					 
     DATE:   09/28/2001 
  SUBJECT:   Adults						 
	     Children						 
	     Diseases						 
	     Organ Procurement and Transplantation		 
	     Network						 
								 

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GAO-01-498
     
Report to Congressional Requesters

United States General Accounting Office

GAO

September 2001 ORGAN TRANSPLANTS

Allocation Policies Include Special Protections for Children

GAO- 01- 498

Page i GAO- 01- 498 Pediatric Organ Transplants Letter 1

Results in Brief 2 Background 3 Demand for Organs for Pediatric Patients
Continues to Outpace

Supply 6 Most Pediatric Organs Are Transplanted Into Adults 12 Pediatric
Patients Generally Fare As Well As or Better Than Adult

Patients on Critical Measures 19 Allocation Policies Aim to Provide
Protection for Pediatric Patients 25 Concluding Observations 27 Agency
Comments 28

Appendix I Insurance Coverage for Immunosuppressive Drugs for Children 30

Sources of Payment for Transplants 30 Medicare Coverage for
Immunosuppressive Drugs 31 Medicaid Coverage for Immunosuppressive Drugs 32
Private Insurance Coverage for Immunosuppressive Drugs 33

Appendix II Causes of Death Most Likely to Result in Organ Donation 35

Appendix III Distribution of Kidneys, Livers, and Hearts, 1994 Through 1999
37

Appendix IV GAO Contact and Staff Acknowledgments 39

Tables

Table 1: Mortality Rates in Children by Age for Deaths Most Likely to Result
in Organ Donation, 1989 and 1997 7 Table 2: Organ Recovery from Potential
Pediatric Donors at 31

Organ Procurement Organizations, and Reasons for Nonrecovery, 1997 and 1998
12 Table 3: Causes of Death Most Likely to Result in Organ Donation 36 Table
4: Distribution of Kidneys, 1994 through 1999 37 Contents

Page ii GAO- 01- 498 Pediatric Organ Transplants

Table 5: Distribution of Livers, 1994 through 1999 37 Table 6: Distribution
of Hearts, 1994 through 1999 38

Figures

Figure 1: Pediatric and Adult Cadaveric Donors, 1991 Through 2000 8 Figure
2: Numbers of Pediatric and Adult Patients on Waiting Lists,

1991 Through 2000 10 Figure 3: Distribution of Pediatric Kidneys, Livers,
and Hearts by

Age of Recipient, 1994 Through 1999 13 Figure 4: Distribution of Pediatric
Kidneys by Age of Donor and

Recipient, 1994 Through 1999 14 Figure 5: Distribution of Pediatric Livers
by Age of Donor and

Recipient, 1994 Through 1999 16 Figure 6: Distribution of Pediatric Hearts
by Age of Donor and

Recipient, 1994 Through 1999 18 Figure 7: Median Waiting Times by Organ and
Age 20 Figure 8: Deaths on the Waiting List, 2000 21 Figure 9: One- Year
Survival Rates for Transplants Performed in

1999 23 Figure 10: Five- Year Survival Rates for Transplants Performed in

1994 and 1995 24 Figure 11: Expected Sources of Payment for Pediatric
Kidney,

Liver, and Heart Transplants, 1997 Through 1999 31

Abbreviations

AOPO Association of Organ Procurement Organizations EPSDT Early and Periodic
Screening, Diagnostic and Treatment

Program ESRD end- stage renal disease FPL federal poverty level HHS
Department of Health and Human Services HRSA Health Resources and Services
Administration OPTN Organ Procurement and Transplantation Network UNOS
United Network for Organ Sharing

Page 1 GAO- 01- 498 Pediatric Organ Transplants

September 28, 2001 Congressional Requesters Organ transplantation can offer
individuals with end stage organ disease the opportunity for a healthy life.
Organ transplants can be particularly beneficial for children with organ
failure. However, pediatric organ transplants involve some special
considerations. 1 Children with end stage organ disease face different
medical consequences than adults. Disease progression can be faster in
children, and their physical and mental development may be affected if they
do not receive an organ transplant early in their illness.

With the scarcity of donor organs, children, as well as adults, may wait a
long time for a transplant or die while waiting. You raised concerns about
the supply of organs and the number of pediatric organs that are allocated
to adults. You also raised concerns about how children in need of an organ
transplant are faring in comparison with adults waiting for a transplant and
whether organ allocation policies sufficiently recognize the unique needs of
children. Specifically, you asked us to determine (1) the trends in organ
donation and demand among adults and children and the factors that affect
pediatric donation, (2) the extent to which pediatric organs are
transplanted into adults, (3) whether pediatric patients are disadvantaged
in terms of waiting times and survival, and (4) how the national organ
allocation policies compare for adult and pediatric patients.

In conducting this study, we interviewed officials and obtained documents
from the Department of Health and Human Services? (HHS) Health Resources and
Services Administration (HRSA), which regulates and provides oversight of
the Organ Procurement and Transplantation Network (OPTN). We also
interviewed officials and obtained documents on organ allocation policies
and data on donation, transplant, survival, and waiting times from the
United Network for Organ Sharing (UNOS), the organization that coordinates
the OPTN for HHS. The OPTN develops policies for organ allocation, maintains
the waiting list, and tracks the OPTN data on each patient. We also
interviewed officials from the Health Care Financing Administration (now the
Centers for Medicare and Medicaid Services) about coverage criteria for
organ transplants and

1 The pediatric population includes children aged 17 and younger.

United States General Accounting Office Washington, DC 20548

Page 2 GAO- 01- 498 Pediatric Organ Transplants

medication therapy because of Medicare coverage for disabled children and
children with end stage renal disease. Additionally, we visited six
transplant centers that represented different geographic locations and
perform a high volume of pediatric organ transplants. At these centers, we
interviewed medical and other transplant services personnel and obtained
relevant documents about donation and allocation issues, survival measures,
and coverage for medication therapy. Representatives from the local organ
procurement organization attended meetings we held at four centers, and we
separately interviewed officials from two other organ procurement
organizations. We also obtained and analyzed data on the causes of death
most likely to result in organ donation from the Centers for Disease Control
and Prevention?s mortality and population database. Because the overwhelming
majority of the organs used in transplant operations come from cadaveric
donors, the discussion on donor organs in this report is limited to
cadaveric organs. We conducted our work from March 2000 through September
2001 in accordance with generally accepted government auditing standards.

Pediatric patients in need of an organ transplant continue to face a
shortage of donated organs. From 1991 through 2000, the number of pediatric
organ donors each year has remained relatively constant, despite a drop in
the number of potential donors. The number of adult donors has increased 45
percent during the same period, in large part because donor eligibility
criteria have been expanded to include older donors and donors with certain
diseases that were not accepted in the past. Simultaneously, the demand for
organs for pediatric patients has grown substantially, with the number of
children on waiting lists for organ transplants more than doubling. However,
compared to adults, children account for a small number of transplant
candidates. Several factors can prevent the recovery of organs from a
potential donor, such as refusal by the family to give consent for donation,
failure by health professionals to identify potential donors or approach
families, and refusal by medical examiners and coroners to release the body.
Nonetheless, organs are recovered from a higher proportion of potential
pediatric donors than potential adult donors.

Most pediatric organs are transplanted into adults because adults make up
the vast majority of patients waiting for an organ transplant and therefore
are more likely to be at a higher status on local organ waiting lists than
children. The degree to which pediatric organs are transplanted into adults
varies by organ. In particular, adult patients receive more pediatric
kidneys than pediatric patients do, partly because of the importance of
Results in Brief

Page 3 GAO- 01- 498 Pediatric Organ Transplants

tissue- type matching criteria in the allocation of kidneys. The picture
differs for livers and hearts, where organs from children aged 11 to 17 are
often transplanted into adults, because of size considerations, but organs
from children under 10 are usually transplanted into pediatric patients.
Adult organs are also transplanted into children, but in much smaller
numbers because of size and matching criteria.

Pediatric patients appear to be faring as well as or better than adult
patients, both while on the waiting list and after transplantation. On key
measures such as time on the waiting list, deaths while waiting for a
transplant, and post- transplant survival, children do as well as or better
than adults, with some exceptions for very young patients and heart
transplant patients. Pediatric patients generally wait fewer days on average
than adults for transplants, have lower death rates on the waiting list, and
have equivalent or better rates for 1- and 5- year post- transplant
survival.

Allocation policies for kidneys, livers, and hearts provide a number of
protections for children awaiting transplants. The organ transplant
community has recognized the distinctive needs of children waiting for a
transplant, and the OPTN has revised organ allocation policies over the past
decade to consider the pediatric patient. The priority a child receives
takes into account differences between children and adults in the
progression and treatment of end stage organ disease, with the policies
differing for each organ. For example, the kidney policy strives to reduce
waiting time for pediatric patients, in part because of the difficulties
they experience with dialysis, and pediatric liver patients now receive
priority for livers from pediatric donors because research has shown that
pediatric livers will improve pediatric patients? chances of survival after
transplant.

HHS and UNOS provided technical comments on a draft of the report which we
incorporated, where appropriate.

Transplants are performed for organs such as kidney, liver, heart,
intestine, pancreas, heart- lung, and kidney- pancreas. However, the kidney,
liver, and heart are the most commonly transplanted organs. In 2000, doctors
performed 13,333 kidney, 4,950 liver, and 2,197 heart transplants. 2

2 Approximately 39 percent of the kidney transplants and 7 percent of the
liver transplants were from living donors. Background

Page 4 GAO- 01- 498 Pediatric Organ Transplants

Of these, children made up 617 of the kidney recipients, 569 of the liver
recipients, and 274 of the heart recipients. Organ transplants were
performed at 261 centers, which had one or more specific organ transplant
programs, in 1998. Some of these centers accept both adults and children,
and others are for children only. 3 In 1998, pediatric kidney transplants
were performed at 129 of the 241 centers that performed kidney transplants;
pediatric liver transplants were performed at 77 of the 116 centers that
transplanted livers; and pediatric heart transplants were performed at 54 of
the 134 centers that transplanted hearts.

In 1984, Congress enacted the National Organ Transplant Act (P. L. 98- 507),
which requires HHS to establish the OPTN. In 1986, HHS awarded the OPTN
contract to UNOS, which operates the network under HRSA?s oversight. The
OPTN develops national transplantation policy, maintains the list of
patients waiting for transplants, and fosters efforts to increase the
nation?s organ supply. 4 OPTN members include all transplant centers, organ
procurement organizations, and tissue- typing laboratories.

Only a small fraction of those who die are considered for organ donation.
Most cadaveric organs derive from donors who have been pronounced brain-
dead as a result of a motor vehicle collision, stroke, violence, suicide, or
severe head injury.

When an organ becomes available, staff from the local organ procurement
organization typically identify potential recipients from the OPTN
computerized waiting list. Patients are ranked on the OPTN waiting list
according to points assigned on the basis of time waiting, medical urgency,
5 organ size, and the quality of the tissue- type match between the donor
and the potential recipient, as determined by antigen matching. 6 The
criteria that determine the order of candidates on the list are applied or

3 No count is maintained of the number of programs that are specifically for
pediatric patients. 4 HHS issued final regulations governing the operation
of the OPTN, which became effective on March 16, 2000. Because of concern
over geographic disparities in organ allocation, the Final Rule authorizes
continuous evaluation and revision of organ allocation policies to achieve
an equitable national allocation system.

5 Medical urgency is measured differently for different organs and may
include such factors as life expectancy and intensity of current treatment.
6 Tissue type is determined by identification of six human- leukocyte-
associated antigens. Organ Donation and

Allocation

Page 5 GAO- 01- 498 Pediatric Organ Transplants

defined differently for each type of organ and for pediatric versus adult
patients. With certain limitations, organs from pediatric donors can be
transplanted into adults, and vice versa. The UNOS computer matches each
patient in the OPTN database against a donor?s characteristics and then
generates a different ranked list of potential recipients for each
transplantable organ from the donor. Organs are generally allocated first to
patients waiting in the local organ procurement organization?s service area,
with priority based on a patient?s severity of illness. If a matching
recipient is not found locally, the organ is offered regionally and then
nationally. 7 Organ allocation policies are revised from time to time to
reflect advancements in medical science and technology.

Title XXI of the Children?s Health Act of 2000 (P. L. 106- 310, October 17,
2000) requires the OPTN to recognize the differences in organ
transplantation needs between children and adults and adopt criteria,
policies, and procedures that address the unique health care needs of
children. In addition, the OPTN is to carry out studies and demonstration
projects for improving procedures for organ procurement and allocation,
including projects to examine and to increase transplantation among
populations with special needs, such as children and racial or ethnic
minority groups. Finally, the act requires the Secretary of HHS to conduct a
study and make recommendations regarding the (1) special growth and
developmental issues that children have before and after transplant; (2)
extent of denials by medical examiners and coroners to allow donation of
organs; (3) other special health and transplantation needs of children; and
(4) costs of the immunosuppressive drugs that children must take after
receiving a transplant and the extent of their coverage by health plans and
insurers. (For a discussion of children?s access to these necessary
medications, see app. I.) The Secretary must report to the Congress by
December 31, 2001.

7 The OPTN has divided the country into 11 regions for allocating organs.
Under certain circumstances, regional or national sharing may occur from the
outset when medically appropriate or when interregional sharing arrangements
exist. For example, regional sharing is allowed for the highest- priority
liver patients. Children?s Health Act of

2000

Page 6 GAO- 01- 498 Pediatric Organ Transplants

Pediatric patients in need of an organ transplant continue to face a
shortage of donated organs. From 1991 through 2000, the number of pediatric
organ donors each year has remained relatively constant, even though the
number of potential pediatric donors decreased. The number of adult donors
has increased significantly during the same period, in large part because
donor eligibility criteria have been expanded to include older donors and
donors with certain diseases that were not accepted in the past.
Simultaneously, the demand for organs for pediatric patients has grown
substantially, with the number of children on waiting lists for organ
transplants more than doubling. However, compared to adults, children
account for a small number of transplant candidates. Several factors can
prevent the recovery of organs from a potential donor. Refusal by the family
to give consent for donation is the primary reason for nonrecovery of an
organ, but failure by health professionals to identify potential donors or
approach families and refusal by medical examiners and coroners to release
the body also account for significant losses of transplantable organs.
Nonetheless, organs are recovered from a higher proportion of potential
pediatric donors than potential adult donors.

The number of pediatric donors has held relatively steady despite a drop in
the number of potential donors. Our analysis of 1989 through 1997 mortality
data for children showed a 20- percent decline in deaths of the kinds that
are most likely to result in organ donation, such as those resulting from
head trauma, motor vehicle collisions, and violence. (See app. II for a
complete list of these causes of death.) Mortality for potential donors up
to age 19 years declined from 24,069 deaths in 1989 to 19, 327 in 1997, the
latest data available at the time of our analysis (see table 1). Demand for
Organs

for Pediatric Patients Continues to Outpace Supply

Number of Pediatric Organ Donors Has Remained Relatively Constant

Page 7 GAO- 01- 498 Pediatric Organ Transplants

Table 1: Mortality Rates in Children by Age for Deaths Most Likely to Result
in Organ Donation, 1989 and 1997

1989 1997 Age, years Number of

deaths Mortality rate a Number of deaths Mortality rate a

Under 1 6, 604 163 3,958 102 1- 4 2, 032 14 1,566 10 5- 9 1, 712 10 1,328 7
10- 14 2,242 13 2,143 11 15- 19 11,479 63 10,332 54

Total deaths 24,069 19,327

a The Centers for Disease Control and Prevention calculates death rates for
infants (aged less than 1 year) as the number of deaths per 100,000 live
births. For other ages, the mortality rate is based on the number of deaths
per 100,000 population.

Source: Centers for Disease Control and Prevention, ?Compressed Mortality
File, CDC Wonder on the Web? (http:// wonder. cdc. gov), Apr. 13, 2000.

OPTN data show that from 1991 through 2000, while the number of pediatric
donors remained relatively constant, the number of adult donors increased 45
percent (see fig. 1). 8 The large increase in the number of adult donors is
primarily due to changes in the criteria for accepting organs from a donor.
At one time, organs were accepted only from someone who had been declared
brain- dead and was relatively young and free from diseases that could
affect organ quality. However, because of the continuing shortage of
transplantable organs, transplant professionals have gradually expanded the
criteria for acceptable organs. Older individuals and persons with certain
medical conditions who previously would have been excluded from donating
organs can now be donors. 9 From 1991 through 2000, the number of cadaveric
donors aged 50 to 64 increased 108 percent, and the number of cadaveric
donors aged 65 or older increased 272 percent.

8 Throughout this report, we use OPTN data that were current as of the date
cited. These data are subject to change based on future data submissions or
corrections. 9 Individuals not routinely considered for organ donation
include diabetics and those with systemic infections or abnormal organ
functions.

Page 8 GAO- 01- 498 Pediatric Organ Transplants

Figure 1: Pediatric and Adult Cadaveric Donors, 1991 Through 2000

Source: OPTN, June 4, 2001.

The number of children waiting for a transplant has increased over time, but
not as much as for adults (see fig. 2). OPTN data show that the number of
pediatric patients awaiting transplants increased from 1,010 in 1991 to
2,299 in 2000, a 128- percent increase. The number of adults on the waiting
list has increased even faster, from 23,709 in 1991 to 77,047 in 2000, a
225- percent increase. These increases have been spurred by advances in
Demand for Pediatric

Transplants Has More Than Doubled Over the Past Decade

Adults aged 65 years or older Adults aged 18- 64 years Children aged 17
years or younger

1991 1992 1993 1994 1995 1996 1997 1998 1999 2000 Number of donors

0 1000

2000 3000

4000 5000

6000

4526 4520 5984

4861 5100

5361 5418 5478 5798 5822

Page 9 GAO- 01- 498 Pediatric Organ Transplants

medical science and technology, which have made transplantation a more
acceptable medical procedure; improvements in immunosuppressive medications,
which have increased survival rates; and an increase in the incidence of
certain diseases that lead to end stage organ failure. Despite these
increases, the proportion of patients awaiting transplant who are children
has remained fairly constant from 1991 through 2000, at between 3 and 4
percent overall.

Page 10 GAO- 01- 498 Pediatric Organ Transplants

Figure 2: Numbers of Pediatric and Adult Patients on Waiting Lists, 1991
Through 2000

Source: OPTN, June 4, 2001.

Number on waiting list

77,047 1,010

23,709 1,206

28,209 1,338

32,056 1,409

36,275 1,589

42,348 1,744

48,386 1,889

54,817 2,122

62,301 2,159

69,951 2,299

1991 1992 1993 1994 1995 1996 1997 1998 1999 2000

Adults Children aged 17 years or younger

0 10,000

20,000 30,000

40,000 50,000

60,000 70,000

80,000

Page 11 GAO- 01- 498 Pediatric Organ Transplants

Several factors can prevent the recovery of organs from potential pediatric
and adult donors and thus contribute to the continuing shortage of
transplantable organs for both children and adults. For example, for many
potential donors, families refuse to give consent for organ donation. For
others, health care professionals may fail to offer the families the
opportunity to donate. 10 Further, some medical examiners and coroners
believe that the need to preserve forensic evidence in certain types of
cases, such as suspected child abuse and sudden infant death syndrome, makes
it impossible for them to allow organ donation to proceed.

The Association of Organ Procurement Organizations (AOPO) recently conducted
a study at 31 organ procurement organizations on the reasons why potential
adult and pediatric donors do not become organ donors. The study found that
consent was not given for 39 percent of potential donors and only 41 percent
of suitable individuals actually become organ donors. 11

AOPO provided us with the survey data from the referral, request, and organ
recovery processes for the pediatric patients. As our analysis shows in
table 2, of the 2,420 potential pediatric donors, organs were recovered in
1,230 cases, or about 51 percent of pediatric cases, a rate higher than the
overall donation rate. Family refusal (25 percent) was the most common
obstacle to organ recovery, but this occurred less frequently for potential
pediatric donors than for the entire group of potential donors.

10 Centers for Medicare and Medicaid Services regulations require hospitals
that participate in Medicare and Medicaid to refer all deaths and imminent
deaths to organ procurement organizations as potential donors.

11 S. Conrad, L. Brigham, E. Sheehy, Association of Organ Procurement
Organizations (AOPO) Death Record Review Study. Paper presented at AOPO
Briefing of Federal Officials; Nov. 15, 2000, Rockville, Md. Several Factors
Can Inhibit

Both Pediatric and Adult Organ Donation

Page 12 GAO- 01- 498 Pediatric Organ Transplants

Table 2: Organ Recovery from Potential Pediatric Donors at 31 Organ
Procurement Organizations, and Reasons for Nonrecovery, 1997 and 1998

Number Percentage of total

Total potential pediatric donors 2, 420 Recovery of at least one organ 1,
230 51 No organ recovery 1,190 49 Reason for nonrecovery a

No referral to organ procurement organization 291 b 12 Referred to organ
procurement organization but family not approached 153 c 6 Referred to organ
procurement organization but family refused 597 25 Consent obtained but
organs not recovered 136 d 6

a Numbers do not total to 1,190 because of missing information for 13
records. b Hospital staff ruled out 123 cases; families denied consent in 97
cases; medical examiners or coroners refused referral in 14 cases; and 57
cases were not referred for other reasons. c The potential donor was
determined to be medically unsuitable in 60 cases; medical examiner and

coroner refusals occurred in 38 cases; cardiac arrest occurred before the
request could be made in 18 cases; no placement could be made for the organ
prior to the request in 5 cases; and the request was not made for other
reasons in 32 cases. d The organs were determined to be medically unsuitable
in 47 cases; cardiac arrest occurred prior to

recovery in 36 cases; unsuccessful placement occurred in 21 cases; medical
examiner or coroner denials occurred in 18 cases; and the organs were not
recovered for other reasons in 14 cases.

Source: GAO analysis of data from the AOPO Death Record Review Project.

Most pediatric organs are transplanted into adults because adults make up
the vast majority of patients waiting for an organ transplant and therefore
are more likely to be at a higher status on local organ waiting lists than
children. However, the degree to which pediatric organs are transplanted
into adults varies by organ. In particular, adult patients receive more
pediatric kidneys than pediatric patients do, partly because of the
importance of tissue- type matching criteria in the allocation of kidneys.
12 While most pediatric kidneys are transplanted into adults, adult kidneys
are sometimes transplanted into children. The situation is different for
livers and hearts, where organ size is an important determinant of
suitability. Livers and hearts from children under 10 are usually
transplanted into pediatric patients, whereas those from children aged 11

12 Although first priority is given to patients for whom a donor kidney is a
perfect match, antigen- matching criteria are becoming less important
because of improvements in immunosuppressive therapies. Most Pediatric
Organs

Are Transplanted Into Adults

Page 13 GAO- 01- 498 Pediatric Organ Transplants

to 17 are usually transplanted into adults. Figure 3 shows the distribution
of pediatric kidneys, livers, and hearts to pediatric and adult recipients.
(See app. III for a detailed listing of the distribution of kidneys, livers,
and hearts by age of donor and recipient.) Pediatric livers and hearts that
are given to adults have sometimes been refused beforehand for a pediatric
patient by the patient?s physician for various medical or logistical
reasons. 13 Adult organs are also transplanted into children, but in much
smaller numbers.

Figure 3: Distribution of Pediatric Kidneys, Livers, and Hearts by Age of
Recipient, 1994 Through 1999

Source: OPTN, June 17, 2000.

Although the majority of pediatric kidneys are transplanted into adults,
some adult kidneys are transplanted into children. From 1994 through 1999,
adult donors provided 81 percent of the kidneys procured and pediatric
donors provided 19 percent (see table 4 in app. III). Of the adult kidneys,
4 percent were transplanted into children. Of the pediatric kidneys, 93
percent were transplanted into adults. Figure 4 shows the

13 Comparable refusal data are not available for kidney transplants. Most
Pediatric Kidneys Are

Transplanted Into Adults, While Most Pediatric Patients Receive Kidneys From
Adults

93 7

63 37

Percentage into adults Percentage into children

Kidneys Livers Hearts Percentage

61 39

Page 14 GAO- 01- 498 Pediatric Organ Transplants

distribution of pediatric kidneys by age of donor and recipient. During that
period, 32 percent of the kidneys given to pediatric recipients came from
children, and 68 percent came from adults.

Figure 4: Distribution of Pediatric Kidneys by Age of Donor and Recipient,
1994 Through 1999

Note: Percentages may exceed 100 due to rounding. Source: OPTN, June 17,
2000.

Kidneys from pediatric donors are most often transplanted into adults
because children make up only a small portion of the kidney waiting list 14
and because of the importance of antigen matching as a ranking factor for
this organ. Also, the matching criteria for kidneys generally do not include
the size (weight and height) of the donor and recipient. When kidneys

14 At the end of 1998, 648 children were on the waiting list, compared to
41, 744 adults.

2 3 2 94

Age of donor 0- 5

Age of donor 6- 10

Age of donor 11- 17

1 2 3 94 2 2 6

90

Age of recipient (years)

0- 5 6- 10 11- 17 18+

Percentage

Page 15 GAO- 01- 498 Pediatric Organ Transplants

from small children are given to adults, they are typically transplanted en
bloc, meaning that both kidneys are transplanted into the recipient.
Transplant center representatives told us that adult kidneys are often
preferred for children because of the larger kidney mass. If complications
occur, the larger kidney is more apt to continue functioning than a small,
pediatric kidney.

For liver transplants, the sizes of the donor and the recipient are factors
that are considered to obtain an organ of compatible size. From 1994 through
1999, adult donors provided 78 percent of the livers procured and pediatric
donors provided 22 percent (see table 5 in app. III). Of the adult livers, 4
percent were transplanted into children. Of the pediatric livers, 63 percent
were transplanted into adults, but this varied greatly by age of the donor.
Most livers (81 percent) from donors aged 5 years or younger went to
recipients in the same age group, and 4 percent went to adults. For the 6-
to 10- year- old donors, 47 percent of the livers went to adult recipients,
and for the 11- to 17- year- old donors, 89 percent of the livers went to
adult recipients. Figure 5 shows the distribution of pediatric livers by age
of donor and recipient from 1994 through 1999. During that period, 72
percent of the livers given to pediatric recipients came from children, and
28 percent came from adults. Liver Transplants Present a

More Varied Picture, but Most Children Receive Pediatric Livers

Page 16 GAO- 01- 498 Pediatric Organ Transplants

Figure 5: Distribution of Pediatric Livers by Age of Donor and Recipient,
1994 Through 1999

Note: Percentages may exceed 100 due to rounding. Source: OPTN, June 17,
2000.

Unlike kidneys and hearts, livers can be reduced in size or split to
accommodate the size of the recipient. A reduced- size liver from an adult
donor can be transplanted into a pediatric patient. A split liver can yield
a portion for an adult and a portion for a child. 15 However, the number of
livers that are either reduced or split is small. From 1994 through 1999,
fewer than 2 percent of donor livers were reduced for transplantation, and

15 The liver consists of a right and left lobe. A reduced- size liver graft
consists of the left lobe of an adult liver, with the right lobe discarded.
In split- liver transplantation, the left lobe of an adult liver is
transplanted into a child, and the right lobe is transplanted into an adult.

4 11

4 81

Age of donor 0- 5

Age of donor 6- 10

Age of donor 11- 17

5 2

4 89 25

15 13 47

Age of recipient (years)

0- 5 6- 10 11- 17 18+

Percentage

Page 17 GAO- 01- 498 Pediatric Organ Transplants

about 1 percent were split for transplantation. Although using reduced or
split livers can provide a needed transplant for children, initial studies
found that survival rates were lower for pediatric recipients of these types
of liver transplants. 16 However, a recent OPTN analysis of 1997- 99
transplants has shown similar 1- year survival rates for whole and split-
liver transplants.

Sometimes an organ from a pediatric donor is transplanted into an adult even
though there is a higher- ranking pediatric patient waiting. This only
occurs if the transplant center refuses the organ for the higher- ranked
patient. According to OPTN data, 1,122 liver transplants occurred during
1997 and 1998 in which an adult recipient received a pediatric organ. Of
these, 222 livers were each refused for at least one potential pediatric
recipient who was ranked higher on the waiting list than the adult
recipient. The most common reasons for refusing the pediatric liver for a
pediatric patient involved administrative reasons (e. g., medical judgment,
transportation, logistics, and distance concerns) (33 percent), donor size
and/ or weight (26 percent), and poor donor quality (18 percent). 17

From 1994 through 1999, adult donors provided 75 percent of the hearts
procured and pediatric donors provided 25 percent (see table 6 in app. III).
Of the adult hearts, 3 percent were transplanted into children. Of the
pediatric hearts, 39 percent were transplanted into adults, but this varied
greatly by age of donor. For heart transplants, organ size is critically
important both to proper functioning and to proper fit into the chest
cavity. Hearts from small children, aged 5 years or younger, are therefore
likely to be transplanted into children of the same age group. Of the hearts
recovered from donors aged 5 years and younger, 93 percent were transplanted
into recipients in the same age group, and 1 percent went to adults. During
the same period, adults received about 24 percent of the hearts from donors
aged 6 through 10 years, and 89 percent from donors aged 11 through 17
years. Figure 6 shows the distribution of pediatric hearts by age of donor
and recipient. During that period, 83 percent of the

16 For the period 1990 through 1996, the rate of graft survival for children
at 1 year for whole- liver grafts was 70. 9 percent, whereas the rates for
reduced- liver grafts and split- liver grafts were 61. 1 and 60. 3 percent,
respectively (N. Scott Adzick and Michael L. Nance,

?Pediatric Surgery, Second of Two Parts,? New England Journal of Medicine,
Vol. 342, No. 23 (2000), pp. 1726- 32).

17 Poor donor quality can result from problems with a donor?s medical
condition, such as hypertension, cardiac arrest, evidence of infection, or
diabetes. Heart Transplants Are

Highly Dependent on Organ Size

Page 18 GAO- 01- 498 Pediatric Organ Transplants

hearts given to pediatric recipients came from children, and 17 percent came
from adults.

Figure 6: Distribution of Pediatric Hearts by Age of Donor and Recipient,
1994 Through 1999

Note: Percentages may exceed 100 due to rounding. Source: OPTN, June 17,
2000.

OPTN data indicate that 664 heart transplants occurred during 1997 and 1998
in which a pediatric organ was transplanted into an adult. Of these, 75
hearts were each refused for at least one pediatric patient who was ranked
higher on the waiting list than the adult recipient. In these instances, the
most common refusal reasons were donor quality (17 percent), donor size and/
or weight (17 percent), administrative reasons (14 percent), and abnormal
echocardiogram (14 percent).

1 1 5

93

Age of donor 0- 5

Age of donor 6- 10

Age of donor 11- 17

1 2 8

89

Age of recipient (years)

0- 5 6- 10 11- 17 18+

Percentage

19 35 22 24

Page 19 GAO- 01- 498 Pediatric Organ Transplants

Although the patterns vary by organ and present a complex picture, pediatric
patients appear to be faring as well as or better than adult patients, both
while on the waiting list and after transplantation. Data from the OPTN and
HHS on four key measures- time on the waiting list, deaths while waiting for
a transplant, and 1- and 5- year post- transplant survival- show that
children appear to fare as well as or better than adults, with some
exceptions for very young patients and heart transplant patients. Other
measures of importance for pediatric patients, such as growth and
development, are not routinely part of the current OPTN data collection.
Pediatric patients wait fewer days on average than adults for transplants.
With the exception of infants under 1 year of age and heart transplant
patients, death rates for pediatric patients on the waiting list are lower
than those for adults. Again with the exception of infants under 1 year old,
post- transplant survival rates for children generally appear to be
equivalent to or better than those for adult patients at the 1- and 5- year
post- transplant points. However, because the number of pediatric patients
is small, variation across time by even a few pediatric patients on any of
these measures could result in relatively large changes in the percentages.
We report on the most current data available.

In general, pediatric patients wait fewer days than adults for transplants
(see fig. 7). Adults are likely to wait about twice as long as children for
a kidney transplant. For patients added to the waiting list for a transplant
in 1997, the median waiting time for pediatric kidney recipients ranged from
389 days for 6- to 10- year- olds to 548 days for 11- to 17- year- olds,
while for adults the range was from 1,044 days for 18- to 34- year- olds to
1,150 days for 50- to 64- year- olds. For livers and hearts, the median
waiting time for adult candidates was two to three times as long as it was
for children. For livers, median waiting times for patients added to the
waiting list in 1999 ranged across age subgroups from 182 to 318 days for
children through age 10. For children aged 11 to 17 years, however, the
waiting time was similar to waiting times for adults. Candidates aged 11
through 17 years waited 746 days, whereas adult waiting times ranged across
age subgroups from 636 to 795 days. Across all age groups, waiting times for
hearts were much shorter than they were for kidneys and livers because
survival is lower without a transplant. Among heart transplant candidates
added to the waiting list in 2000, median waiting times for children ranged
from 52 to 86 days and for adults from 137 to 242 days across the different
age subgroups. Pediatric Patients

Generally Fare As Well As or Better Than Adult Patients on Critical Measures

Pediatric Patients Generally Spend Less Time on the Waiting List Than Adults

Page 20 GAO- 01- 498 Pediatric Organ Transplants

Figure 7: Median Waiting Times by Organ and Age

a Median waiting time for kidney patients under 1 year of age was not
calculated owing to the small number of patients. Note: Data are for
patients added to the waiting list for a transplant for a kidney in 1997, a
liver in 1999, or a heart in 2000.

Source: OPTN, June 8, 2001.

The death rates for pediatric patients on the waiting list vary considerably
by organ, with pediatric patients having slightly lower rates than adults
for kidneys and livers, but higher rates than adults for hearts (see fig.
8). In Pediatric Deaths While

Waiting Vary by Organ

0 100

200 300

400 500

600 700

800 900

1,000 1,100

1,200 Median waiting time (days) by age group

Kidney (1997) Liver (1999) Heart (2000) a <1 1- 5 6- 10 11- 17

18- 34 35- 40 50- 64 65+ 496

389 548

1044 1103

1150 1136 182 205

318 746

636 758 744

795 52 86

60 67 137

222 242 184

Age group (years)

Page 21 GAO- 01- 498 Pediatric Organ Transplants

2000, death rates for children waiting for a kidney transplant ranged from 0
to 92 per 1,000 patient risk years (i. e., years on the waiting list),
whereas for adults they ranged from 36 to 104. Infants under 1 year old who
were awaiting liver or heart transplants had considerably higher death rates
than other pediatric or adult age groups; however, pediatric patients aged 1
year or older waiting for a liver transplant had lower death rates than
adults. For patients waiting for a heart transplant, pediatric patients of
all age groups had higher death rates than did adults.

Figure 8: Deaths on the Waiting List, 2000

a Patient- years at risk is calculated as the number of deaths for every
1,000 patient years on the waiting list, based on the actual time waiting
for each patient listed. b Of 16 kidney patients under age 1, none died
while on the waiting list.

Source: OPTN, June 8, 2001.

0 50

100 150

200 250

300 350

400 450 Annual death rate (per 1,000 patient risk years a )

Age group (years) Kidney Liver Heart

<1 1- 5 6- 10 11- 17

18- 34 35- 40 50- 64 65+ 0 b

92.2 52.8

22 35.5 60.2

171.2 71.4 67.5

54 102.8 94.1

441.1 292.2

201.2 201.6 166.6

107.8 141.3

180 103.7 78.9

119.6 147

Page 22 GAO- 01- 498 Pediatric Organ Transplants

With the exception of infants under 1 year old, post- transplant survival
rates (i. e., the percentage of patients alive at 1 and 5 years after
transplant) for children generally appear to be as good as or better than
those for adults (see figs. 9 and 10). In general, 1- year survival rates
vary more by type of organ than they do by age group, with kidney transplant
recipients having the highest survival rates and heart transplant patients
having the lowest survival rates. Overall, survival rates for children at 5
years after transplant are better than adult survival for kidneys and
livers. Children 5 years old and younger have lower 5- year survival rates
for heart transplants. Pediatric Survival After

Transplant Is Generally As Good As or Better Than That for Adults

Page 23 GAO- 01- 498 Pediatric Organ Transplants

Figure 9: One- Year Survival Rates for Transplants Performed in 1999

a Survival rates for infants under 1 year of age for kidney transplants
could not be calculated because of an insufficient number of patients.
Source: OPTN, June 8, 2001.

Kidney a Liver Heart 0 20

40 60

80 100

<1 1- 5 6- 10 11- 17

18- 34 35- 40 50- 64 65+

Age group (years) Percentage of patients alive 1 year after transplant

82.7 94.9

92 97.9 97.6 96.7

93.1 88.6

85 86.1 87.5 88.4 92.5

88.8 84.9

77.6 81.5 81.5

90.5 84.2 82.8

89 82.9

Page 24 GAO- 01- 498 Pediatric Organ Transplants

Figure 10: Five- Year Survival Rates for Transplants Performed in 1994 and
1995

a Survival rates for infants under 1 year of age for kidney transplants
could not be calculated because of an insufficient number of patients.
Source: OPTN, June 16, 2001.

0 20

40 60

80 100

<1 1- 5 6- 10 11- 17

18- 34 35- 40 50- 64 65+

Age group (years) Kidney a Liver (1995) Heart

91.7 97

93.3 92.2 86.1

76.1 61.5

84.7 79.3

93 87.3

82.1 78.5

73.6 67.7

53.7 57.7

67.7 70.6 72.1 72.3 71.9 70.8

Percentage of patients alive 5 years after transplant

Page 25 GAO- 01- 498 Pediatric Organ Transplants

Organ allocation policies provide a number of protections for children
awaiting transplants. The organ transplant community has recognized the
distinctive needs of children waiting for a transplant, and the OPTN has
revised organ allocation policies over time to consider the pediatric
patient. The priority a child receives takes into account differences
between children and adults in the progression and treatment of end stage
organ disease. Prolonged waiting times can be more harmful for children than
for adults because disease progression in children can be faster and their
growth and development can be compromised without timely transplantation.
The policies differ for each organ. For example, waiting time requirements
for kidney transplants are less stringent for pediatric patients than for
adult patients because of the unique problems children experience with end
stage renal disease, including difficulties with dialysis. For livers,
research showing better survival for pediatric patients who received a
pediatric liver led to a policy change giving priority for pediatric livers
to pediatric patients. For hearts, medical urgency status is determined
differently for pediatric patients because pretransplant treatments
appropriate for adults, such as heart assist devices, cannot always be used
for children who are waiting for transplants.

Current kidney allocation policy provides several protections for pediatric
kidney patients because of the unique problems they experience in
association with end stage renal disease. These problems include dialysis
difficulties and disruption of growth and development due to renal failure.
Early transplantation can avoid or ameliorate many of the effects of end
stage renal disease experienced by pediatric patients.

One advantage the allocation policy gives to pediatric patients concerns
waiting time, one factor in determining priority for obtaining a transplant.
Waiting time for children is measured from when they are placed on the
waiting list, whereas, since changes to the adult kidney allocation policy
in January 1998, waiting time for adults begins when they reach a certain
stage of disease. Therefore pediatric patients can begin moving up in
priority on the waiting list at an earlier point in their disease
progression than can adult patients.

In addition, pediatric patients receive higher priority for kidney
allocation at the time of listing and until they reach 18 years of age,
based on their age at listing. The criteria for granting this priority were
first implemented by the OPTN in 1990 and have been altered several times,
most recently in November 1998. Kidney transplant candidates less than 11
years of age at Allocation Policies

Aim to Provide Protection for Pediatric Patients

Kidney Allocation Policy Geared to Reduce Waiting Time for Pediatric
Patients

Page 26 GAO- 01- 498 Pediatric Organ Transplants

listing are assigned four additional points, and candidates aged 11 through
17 years are assigned three additional points. 18

Another advantage was introduced by the OPTN in November 1998. It provides
that patients who are less than 18 years old at listing, and have not
received a transplant within a specified amount of time, must be the first
in line to receive available kidneys, except for those that must be
allocated to a patient with a perfect antigen match, to a patient needing a
kidney plus a nonrenal organ, or to a patient whose immune system makes it
difficult to receive organs. These specified times are within 6 months of
listing for candidates up to and including 5 years of age, 12 months for
those from 6 to 10 years, and 18 months for those from 11 to 17 years.

The liver allocation policy for pediatric patients has been revised several
times since 1994 to address conditions and challenges unique to pediatric
patients. Children with chronic liver disease may deteriorate rapidly and
unpredictably. Their growth and development may also be affected. The policy
revisions redefine medical urgency criteria, focus on disease progression in
children, and recognize factors distinctive to pediatric liver candidates.

In June 2000, the OPTN approved a policy to give pediatric liver transplant
patients preference over adult patients for livers from pediatric donors.
Prior to the implementation of this change, the age of the donor was not a
factor. Now, a pediatric liver is offered to a pediatric patient before an
adult patient with the same medical urgency within the same organ
distribution area. If no local matches occur in a given medical urgency
category, the pediatric liver will be offered to a pediatric patient before
an adult patient with the same medical urgency at the regional level. This
change was made in response to the finding that pediatric liver transplant
recipients have higher survival rates and better graft survival 19 if they
are transplanted with a pediatric liver rather than an adult liver. A study
showed that pediatric patients receiving livers from pediatric donors during
1992 through 1997 had a 3- year graft survival rate of 81 percent,

18 According to current allocation policies, patients are ranked on the OPTN
waiting list according to points assigned on the basis of time waiting and
medical urgency. 19 Graft survival refers to the length of time the
transplanted organ continues to function. Liver Allocation Policy

Seeks to Provide Pediatric Patients With Pediatric Organs

Page 27 GAO- 01- 498 Pediatric Organ Transplants

compared to 63 percent for children receiving an adult liver. Adults,
however, had similar 3- year graft survival rates regardless of donor age.
20

The OPTN policy also provides an advantage for pediatric patients with
chronic liver failure. The policy places these patients at the highest
medical urgency level when their condition worsens, a provision that is not
in place for adult patients with chronic liver failure. Moving pediatric
patients to the highest category provides the advantage of access to donated
organs locally and regionally before all patients in lower categories.

The heart allocation criteria have also been revised recently to reflect
differences in treatment and progression of heart disease between children
and adults. Before these revisions, the use of certain mechanical assist
devices or other monitoring and treatment therapies was required for any
patient to be included in the highest medical urgency categories. However,
because some of these devices and therapies are generally not used with
pediatric patients, the OPTN removed this requirement for pediatric patients
in January 1999. The OPTN implemented two further revisions in May 2000. One
change allows pediatric patients on the waiting list for a heart to retain
their medical urgency status when they turn 18 rather than being subject to
adult criteria. Another revision gives priority to pediatric heart
transplant candidates, within each medical urgency category, for hearts
recovered from 11- to 17- year- old donors.

Children constitute a small proportion of patients in need of an organ
transplant, but organ allocation policies have been designed to provide this
vulnerable population with some special protections. Our examination of
transplantation patterns across age groups and recent data on waiting times
and death and survival rates indicates that pediatric patients do not appear
to be at a disadvantage in the competition for scarce organs. These data
show comparable or better outcomes for pediatric patients even before the
most recent policy changes, such as the change to prioritize pediatric
livers for pediatric recipients.

20 Sue V. McDiarmid, Darcy B. Davis, Eric B. Edwards, ?Improved Graft
Survival of Pediatric Liver Recipients Transplanted With Pediatric- Aged
Liver Donors,? Transplantation, Vol. 70, No. 9 (Nov. 15, 2000), pp. 1283-
91. Data from this study were presented to OPTN for consideration prior to
publication. Heart Allocation Policy

Gives Some Priority to Pediatric Patients

Concluding Observations

Page 28 GAO- 01- 498 Pediatric Organ Transplants

We provided HHS with the opportunity to comment on a draft of this report.
HHS provided technical comments, which we have incorporated where
appropriate. We also provided a draft of the report to UNOS, and it provided
technical comments, which we have incorporated where appropriate.

As agreed with your offices, unless you publicly announce the contents of
this report earlier, we plan no further distribution of it until 30 days
from the date of this letter. We will then send copies to others who are
interested and make copies available to others who request them. If you or
your staffs have any questions about this report, please call me at (202)
512- 7119. Another contact and key contributors to this report are listed in
appendix IV.

Janet Heinrich Director, Health Care- Public Health Issues Agency Comments

Page 29 GAO- 01- 498 Pediatric Organ Transplants

List of Requesters

The Honorable John D. Dingell Ranking Minority Member Committee on Energy
and Commerce

The Honorable Sherrod Brown The Honorable William J. Coyne The Honorable
Diana L. DeGette The Honorable Frank R. Mascara The Honorable John E.
Peterson The Honorable Henry A. Waxman House of Representatives

Appendix I: Insurance Coverage for Immunosuppressive Drugs for Children

Page 30 GAO- 01- 498 Pediatric Organ Transplants

Coverage for immunosuppressive medications may be extended to children under
Medicare, Medicaid, and private insurance. Pediatric patients may also gain
access to prescription drug coverage through special state insurance
programs for children. However, both adults and children may have difficulty
in obtaining and retaining insurance coverage for the expensive
immunosuppressive medications 1 necessary for survival following
transplantation. Further, gaps in coverage may occur during a transition
from one type of insurance to another. For example, if a parent loses
Medicaid eligibility, a child?s eligibility status could also be affected.
In addition, coverage problems can arise for both Medicaid- and
privateinsurance- covered pediatric patients when they reach adulthood.
Transplant recipients covered by Medicaid as children may become ineligible
for continued coverage if they are able to obtain employment as they reach
adulthood. Children covered by private insurance under a parent?s policy may
be unable to afford coverage, given their expensive preexisting medical
condition, when they grow too old to be covered by a parent?s policy. Data
on the costs of immunosuppressive medications, actual payments, and patient
cost- sharing by the various insurers are not readily available, so the
level of the coverage cannot be specified with certainty.

The proportion of transplant patients covered by different insurance
programs can be used to derive an indication of coverage for
immunosuppressive medications. Data from the Organ Procurement and
Transplantation Network (OPTN) on the expected sources of payment for the
pediatric transplants performed from 1997 through 1999 may serve as a
general estimate of the share of immunosuppressive medications for children
paid for by Medicare, Medicaid, and private insurance. OPTN data show that
4,835 transplants were performed on children up to age 17 from 1997 to 1999.
Of these, 2,775 transplants were for livers and hearts, and 2,060 were for
kidneys. As figure 11 shows, private insurance paid for almost half of the
pediatric transplants for these three organs performed from 1997 through
1999, while Medicaid paid for 25 percent and Medicare paid for 14 percent of
these transplants. For the same period, Medicare paid for an estimated 30
percent of pediatric kidney transplants because of

1 In 1999, the average annual charges for immunosuppressive medications for
kidney, liver, and heart transplant recipients ranged from $11, 400 to $13,
600 (Richard H. Hauboldt, Cost Implications of Human Organ and Tissue
Transplantations, An Update: 1999

(Brookfield, Wisc.: Milliman & Robertson, Inc., 1999)). Appendix I:
Insurance Coverage for

Immunosuppressive Drugs for Children Sources of Payment for Transplants

Appendix I: Insurance Coverage for Immunosuppressive Drugs for Children

Page 31 GAO- 01- 498 Pediatric Organ Transplants

its special coverage for kidney patients under the End- Stage Renal Disease
(ESRD) program.

Figure 11: Expected Sources of Payment for Pediatric Kidney, Liver, and
Heart Transplants, 1997 Through 1999

Source: OPTN, April 23, 2000.

Medicare coverage for transplants and the associated medications is provided
to children either under a special entitlement to the Medicare program
created by the Congress for those diagnosed with ESRD or by virtue of a
parent?s enrollment as an eligible Medicare beneficiary.

The Medicare program has special entitlement rules for patients with ESRD,
the stage of kidney impairment that is considered irreversible and requires
either regular dialysis or a kidney transplant to maintain life. To be
eligible for Medicare entitlement as an ESRD patient, the patient generally
must have been on dialysis for 3 months and must be (1) entitled to a
monthly insurance benefit under title II of the Social Security Act (or an
annuity under the Railroad Retirement Act), (2) fully or currently insured
under Social Security, or (3) the spouse or dependent child of a Medicare
Coverage

for Immunosuppressive Drugs

47.1% 6.1%

8.4% 14%

24.5% Other

Not reported Medicare

Medicaid Private insurance

Appendix I: Insurance Coverage for Immunosuppressive Drugs for Children

Page 32 GAO- 01- 498 Pediatric Organ Transplants

person who meets at least the first 2 requirements. 2 Currently, ESRD
patients? entitlement to Medicare- and thus coverage for immunosuppressive
medications- ends 36 months after a transplant is performed. 3

In contrast, individuals who are eligible for Medicare under other
entitlement rules- that is, age 65 or disabled, and eligible for Social
Security or Railroad Retirement benefits- currently receive unlimited
coverage for immunosuppressive drug medications for the life of the
transplant under Part B. 4 Originally, Medicare limited immunosuppressive
drug coverage to 1 year. However, the Omnibus Budget Reconciliation Act of
1993 (P. L. 103- 66) expanded this coverage with a series of annual 6- month
increases beginning in 1995. As a result, by 1998, Medicare patients
received immunosuppressive medication coverage for 36 months after a
transplant operation. In 1999, the Medicare, Medicaid, and SCHIP Balanced
Budget Refinement Act of 1999 (P. L. 106- 113) extended this
immunosuppressive drug coverage benefit for an additional 8 months. Most
recently, the Medicare, Medicaid and SCHIP Benefits Improvement and
Protection Act of 2000 (P. L. 106- 554) eliminated all time limits for
immunosuppressive drug coverage under Part B of Medicare.

Medicaid is a joint federal/ state entitlement that annually finances health
care coverage for more than 40 million low- income individuals, over half of
whom are children. Medicaid coverage for children is comprehensive, offering
a wide range of medical services and mandating coverage based upon family
income in relation to the federal poverty level (FPL). Federal law requires
states to cover children up to age 6 from families with incomes up to 133
percent of the FPL, and children ages 6 to 15 for incomes up to 100 percent
of the FPL. Medicaid benefits are particularly important for children
because of Medicaid?s Early and Periodic Screening, Diagnostic, and
Treatment (EPSDT) Program. EPSDT, which is

2 For ESRD participants, Medicare pays secondary to employer- sponsored
group health plans for the first 30 months. 3 An ESRD patient may be
eligible for immunosuppressive drug coverage if, after 36 months, the
patient is otherwise entitled to Medicare based on age and/ or disability. 4
Medicare insurance consists of two parts, Part A, which covers in- patient
hospital expenses, and Part B, which covers other medical expenses including
physician, outpatient hospital, laboratory, and other services such as
immunosuppressive drug therapy. Beneficiaries must pay a premium for Part B
coverage, currently $50 per month, and are also responsible for Part B
deductibles, coinsurance, and copayments. Medicaid Coverage

for Immunosuppressive Drugs

Appendix I: Insurance Coverage for Immunosuppressive Drugs for Children

Page 33 GAO- 01- 498 Pediatric Organ Transplants

mandatory for categorically needy children, provides comprehensive, periodic
evaluations of health and developmental history, as well as vision, hearing,
and dental screening services to most Medicaid- eligible children. 5 Under
EPSDT, states are required to cover any service or item that is medically
necessary to correct or ameliorate a condition detected through an EPSDT
screening, regardless of whether the service is otherwise covered under a
state Medicaid program. This would include immunosuppressive drugs. 6

Private insurance, such as employer- sponsored health plans, generally
covers all aspects of organ transplants, including follow- up care and
necessary medications. Information is not readily available on private
insurance coverage specifically for immunosuppressive medications. However,
according to a 1998 national survey of employer- sponsored health plans,
nearly all employers that offer health benefits include benefits for
outpatient prescription drugs. 7 In addition, a Kaiser Family Foundation
survey of employer health benefits found that 96 percent of all firms with
conventional fee- for- service plans and 99 percent of those with managed
care plans cover prescription drugs. 8

5 The categorically needy includes low- income children; pregnant women;
aged, blind, or disabled people meeting Supplemental Security Income program
requirements; and individuals who are eligible to receive federally assisted
income maintenance payments. The EPSDT benefit is optional for the medically
needy population, who generally have too much income to quality for
Medicaid, but have ?spent down? their income by incurring medical and/ or
remedial care expenses. See 42 USC 1396( a)( 10)( C). If a state chooses to
provide one EPSDT service, it must provide all EPSDT services to medically
needy individuals under age 21. The medically needy comprise individuals
whose income, resources, or both exceed the levels for the categorically
needy, but who cannot afford to pay their medical bills.

6 The Institute of Medicine reported that kidney, heart, and liver
transplants are covered services under Medicaid in nearly all states. See
Organ Procurement and Transplantation, ?Assessing Current Policies and the
Potential Impact of the DHHS Final Rule,?

(Washington, D. C.: National Academy Press, 1999). Generally, once a
transplant is approved, immunosuppressive drugs and medications to maintain
the organ are also approved.

7 Mercer/ Foster Higgins, National Survey of Employer- Sponsored Health
Plans 1998, a survey of all U. S. employers with 10 or more employees. 8 The
Kaiser Family Foundation and Health Research and Educational Trust, Employer
Health Benefits, Annual Survey, 1999 (Menlo Park, Calif., and Chicago, Ill.,
1999). Private Insurance

Coverage for Immunosuppressive Drugs

Appendix I: Insurance Coverage for Immunosuppressive Drugs for Children

Page 34 GAO- 01- 498 Pediatric Organ Transplants

Privately insured organ transplant patients most likely will incur
additional expenses for medications, however, such as out- of- pocket
expenses for deductibles and copayments, because of limits on coverage. A
recent survey of employers with 1,000 or more employees on strategies to
control prescription drug expenditures found that 6 percent of employers cap
annual benefits and 10 percent are considering doing so. 9 The study also
found that 41 percent of employers limit the quantities of prescription
drugs and 7 percent are considering it. Moreover, 40 percent of employers
now require higher copayments than previously, and 39 percent are
considering it.

9 Hewitt Associates, Health Care Expectations: Future Strategy and
Direction, 2001

(Lincolnshire, Ill.: 2001).

Appendix II: Causes of Death Most Likely to Result in Organ Donation

Page 35 GAO- 01- 498 Pediatric Organ Transplants

The causes and circumstances of death that could reasonably result in a
declaration of brain death and from which organ donation might be possible
are listed in table 3. 1 We used the International Classification of
Diseases, 9th Revision, Clinical Modification (ICD- 9- CM) codes to classify
deaths by causes and circumstances. 2

1 See Organ Procurement Organizations: Alternatives Being Developed to More
Accurately Assess Performance (GAO/ HEHS- 98- 26, Nov. 26, 1997), where we
developed this list.

2 Medical staff use ICD- 9- CM codes at the time of death to indicate cause
of death on the death certificate. Appendix II: Causes of Death Most Likely
to

Result in Organ Donation

Appendix II: Causes of Death Most Likely to Result in Organ Donation

Page 36 GAO- 01- 498 Pediatric Organ Transplants

Table 3: Causes of Death Most Likely to Result in Organ Donation ICD- 9 CM
Code Description

430- 438 Cerebrovascular disease 798.0 Sudden infant death syndrome E810-
E825 a Motor vehicle accident E830 Accident of watercraft causing submersion
E832 Other accidental submersion or drowning in water transport accident
E850- E858 Accidental poisoning by drugs, medicinal substances, and
biologicals E910- E913 Accidental submersion, suffocation, and other foreign
bodies E920 Accidents caused by cutting and piercing instruments or objects
E922 Accidents caused by firearm missile E930- E950. 5 Drugs, medicinal and
biological substances causing adverse effects in therapeutic use,

suicidal and self- inflicted poisoning by solid or liquid substances E953-
E955. 4, E956, E958. 5 Suicide E962.0 Assault by poisoning E963 Assault by
hanging and strangulation E964 Assault by drowning E965- E965. 4 Assault by
firearms and explosives E966 Assault by cutting and piercing instrument E970
Injury due to legal intervention by firearms E974 Injury due to legal
intervention by cutting and piercing instrument E980.0- E980.5 Poisoning,
undetermined whether accidentally or purposely inflicted E983 Hanging,
strangulation, or suffocation undetermined whether accidentally or purposely

inflicted E984 Drowning, undetermined whether accidentally or purposely
inflicted E985- E985. 4 Injury by firearms, undetermined whether
accidentally or purposely inflicted E986 Injury by cutting, piercing
instruments, undetermined whether accidentally or purposely

inflicted a E codes permit the classification of environmental events,
circumstances, and conditions as the cause of injury, poisoning, and other
adverse effects.

Appendix III: Distribution of Kidneys, Livers, and Hearts, 1994 Through 1999

Page 37 GAO- 01- 498 Pediatric Organ Transplants

The following tables show the distribution of kidneys, livers, and hearts
procured from all donors from 1994 to 1999, by age of donor and recipient.

Table 4: Distribution of Kidneys, 1994 through 1999 Recipients

Donors 0- 5 years 6- 10

years 11- 17 years Total

children Total

adults, 18 and over Total

recipients

0- 5 years 27 23 47 97 1,412 1,509 6- 10 years 29 38 99 166 1,479 1,645 11-
17 years 55 88 227 370 5,477 5,847 Total children 111 149 373 633 8,368
9,001 Total adults, 18 and over 168 272 902 1,342 36,449 37,791

Total 279 421 1,275 1,975 44,817 46,792

Source: OPTN, June 17, 2000.

Table 5: Distribution of Livers, 1994 through 1999 Recipients

Donors 0- 5 years 6- 10

years 11- 17 years Total

children Total

adults, 18 and over Total

recipients

0- 5 years 1, 052 148 51 1,251 55 1,306 6- 10 years 215 124 107 446 399 845
11- 17 years 151 53 149 353 2,982 3,335 Total children 1, 418 325 307 2,050
3,436 5,486 Total, adults, 18 and over 326 128 341 795 18,128 18,923

Total 1,744 453 648 2,845 21,564 24,409

Source: OPTN, June 17, 2000.

Appendix III: Distribution of Kidneys, Livers, and Hearts, 1994 Through 1999

Appendix III: Distribution of Kidneys, Livers, and Hearts, 1994 Through 1999

Page 38 GAO- 01- 498 Pediatric Organ Transplants

Table 6: Distribution of Hearts, 1994 through 1999 Recipients

Donors 0- 5 years 6- 10

years 11- 17 years Total

children Total

adults, 18 and over Total

recipients

0- 5 years 752 44 3 799 9 808 6- 10 years 66 118 76 260 81 341 11- 17 years
11 44 180 235 1,959 2,194 Total children 829 206 259 1,294 2,049 3,343 Total
adults, 18 and over 8 20 236 264 9,978 10,242

Total 837 226 495 1,558 12,027 13,585

Source: OPTN, June 17, 2000.

Appendix IV: GAO Contact and Staff Acknowledgments

Page 39 GAO- 01- 498 Pediatric Organ Transplants

Marcia Crosse, (202) 512- 3407 In addition to the above, Donna Bulvin,
Charles Davenport, Roy Hogberg, Behn Miller, and Roseanne Price made key
contributions to this report. Appendix IV: GAO Contact and Staff

Acknowledgments GAO Contact Acknowledgments

(201043)

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