Finkelstein BS, Silvers JB, Marrero U, Neuhauser D, Cuttler L. Insurance Coverage, Physician Recommendations, and Access to Emerging TreatmentsGrowth Hormone Therapy for Childhood Short Stature. JAMA. 1998;279(9):663-668. doi:10.1001/jama.279.9.663
From the Departments of Pediatrics (Drs Cuttler and Finkelstein and Ms Marrero), Pharmacology (Dr Cuttler), and Epidemiology and Biostatistics (Drs Finkelstein and Neuhauser), School of Medicine, and the Weatherhead School of Management (Dr Silvers), Case Western Reserve University, Cleveland, Ohio.
Context.— There is concern in both the medical community and the general public
about mechanisms of medical decision making and the interplay of physician
and insurer decisions in determining access to care.
Objective.— To examine the medical process influencing access to growth hormone
(GH) therapy for childhood short stature by comparing coverage policies of
US insurers with the treatment recommendations of US physicians.
Design and Participants.— Independent national representative surveys were mailed to insurers
(private, Blue Cross/Blue Shield, health maintenance organizations, programs
for Children with Special Health Care Needs, and Medicaid programs, n=113),
primary care physicians (n=1504), and pediatric endocrinologists (n=534) with
response rates of 75%, 60%, and 81%, respectively. Each survey included identical
case scenarios. Primary care physicians were asked decisions about referrals
to pediatric endocrinologists. Endocrinologists were asked GH treatment recommendations.
Insurers were asked coverage decisions for GH therapy.
Main Outcome Measures.— Insurer coverage decisions for GH in specific case scenarios were compared
with the recommendations of primary care physicians and pediatric endocrinologists.
Results.— Physician recommendations and insurance coverage decisions differed
strikingly. For example, while 96% of pediatric endocrinologists recommended
GH therapy for children with Turner syndrome, insurer policies covered GH
therapy for only 52% of these children. Overall, referral and treatment decisions
by physicians resulted in recommendations for GH therapy in 78% of children
with GH deficiency, Turner syndrome, or renal failure; of those recommended
for treatment, 28% were denied coverage by insurers. Similarly, GH therapy
would be recommended by physicians for only 9% of children with idiopathic
short stature, but insurers would not cover GH for the vast majority of these
children. Furthermore, the data indicated considerable variation among insurers
regarding coverage policies for GH (P<.01).
Conclusions.— Access to GH therapy differs depending on the type of insurance coverage.
The deep discord between physician recommendations and insurance coverage
decisions, exemplified by these findings, represents a major challenge to
mechanisms of health care decision making, access, and costs.
THE INFLUENCE of insurance on access to health care, especially access
to expensive specialist care and treatment, is a major concern.1- 3
However, little is known about the spectrum of coverage across insurers or
about the agreement between physician recommendations and insurer policies
for specific therapies. These concerns are particularly important for emerging
and semielective treatments related to quality of life (such as treatments
for short stature, infertility, obesity, and aging4),
in which consensus about optimal utilization may be lacking.
In this article, we focus on insurer and physician decisions regarding
growth hormone (GH) therapy for childhood short stature for several reasons.
First, GH therapy is representative of many treatments whose use depends on
a process involving primary care physicians, specialists, and insurers. Pediatric
endocrinologists are the specialist group almost exclusively responsible for
prescribing GHs for short children and are considered experts in the area.5 However, the overall impact of their decisions on
GH use is influenced by referrals from primary care physicians and coverage
decisions of insurers. Second, GH therapy is at times semielective, is rarely
needed for life-threatening situations, and is very costly (approximately
$14000 per year for a child weighing 20 kg).6
Finally, optimal GH use has been the subject of debate.7- 12
Traditionally, GH therapy has been used for children who have classical GH
deficiency (GHD)11,13 due to a
lack of natural GHs. However, the medical literature and available guidelines
suggest diverse criteria for defining GHD and identifying appropriate candidates
for treatment.7- 13
Physicians may and do prescribe GHs for conditions other than GHD.5,12,14,15 The
literature indicates that GHs may benefit children with short stature due
to conditions such as Turner syndrome and chronic renal insufficiency (CRI).
Moreover, GH use has been suggested for certain short children who do not
have a defined medical disorder (ie, familial, constitutional, or idiopathic
These children constitute the largest number of candidates for GH therapy
and represent the first major threshold in nontraditional GH use that may
be followed by other applications, including GHs and derivatives for aging,
the acquired immunodeficiency syndrome, and obesity.4
In this study, we asked 3 major questions: (1) What are insurer policies
for the coverage of GH therapy in the treatment of childhood short stature?
(2) Are insurer policies comparable to the recommendations of expert physicians?
(3) How do insurer policies for GH therapy interact with physician decisions
in influencing GH utilization and costs?
A written survey instrument was developed by a team of experts in endocrinology,
survey methodology, and health care financing. The preliminary survey was
pretested with administrative and medical personnel employed by insurers.
The final questionnaire used an experimental design based on survey methodology
in which each insurer was asked to report the following: (1) general policies
for GH therapy; (2) whether their organization would cover all or part of
the costs of GH therapy for childhood short stature due to specific medical
conditions such as GHD, CRI, or Turner syndrome. These conditions were selected
to include disorders for which GH therapy was approved (GHD, CRI) or not approved
(Turner syndrome) by the Food and Drug Administration (FDA) and for which
physician consensus to recommend or use GHs ranges from moderate (CRI) to
very high (GHD, Turner syndrome)5; and (3)
whether their organization would cover GH therapy for case scenarios of children
who are short but do not have classical GHD or other defined medical disorders
(ie, idiopathic short stature). (Note: GH therapy was approved by the FDA
for Turner syndrome on December 31, 1996, after this survey took place.)
The format for the case presentations of children with idiopathic short
stature involved systematic variation of experimental variables to create
a controlled environment for the study of GH coverage decisions. Insurers
were presented with 4 case scenarios (cases A-D; Figure 1), differing only in the physiological growth variables
concerning degree of short stature (height, 2 or 3 SDs below the mean for
the child's age) and the rate of growth (the 3rd to 10th percentile or below
the third percentile for age, designated as 4.5 and 3.2 cm per year [1.8 and
1.3 in per year],16 respectively). The case
descriptions were designed to be representative of the population of relatively
short, slow-growing children (height, >1.5 SDs below the mean, growth rate
below the 10th percentile for age16) with the
common and often difficult-to-differentiate conditions of idiopathic short
stature, familial short stature, and constitutional delay in growth and development.4,13,17 The case descriptions
were also designed to match the cases presented in separate surveys (described
below) to primary care physicians for referral decisions, and to pediatric
endocrinologists for treatment recommendations. Case descriptions included
information typically used by each group (eg, insurer surveys indicated that,
for each case scenario, GH therapy had been "prescribed by an authorized physician").
To control for other variables, the clinical context of the scenarios was
described in detail. The patient presented was a 10-year-old boy or girl (the
average age of children presenting to endocrinologists for short stature and
the approximate midrange of potential candidates18)
with no other abnormalities on physical examination, normal peak GH levels
(15 µg/L) in response to a stimulation test,5,7,13
and all other laboratory test results normal (including free thyroxine, thyrotropin,
complete blood cell count, erythrocyte, sedimentation rate, urinalysis, chemistry
profile, and, in females, karyotype), excluding the diagnosis of classical
GHD or other medical causes of short stature.
All state Medicaid agencies were surveyed since preliminary investigation
indicated likely state-to-state variation.19
Because some state public programs for Children with Special Health Care Needs
(CSHCN)20 act as the payer of last resort for
the underinsured and uninsured, we surveyed these programs from 16 states
(Alabama, Arizona, California, Florida, Georgia, Indiana, Maine, Maryland,
Michigan, Minnesota, Nebraska, New Hampshire, New Mexico, North Carolina,
Ohio, and South Carolina) chosen by stratified sampling according to variation
in physician-to-population ratio and geographic diversity,21
and to match the sampling frame used for the survey of primary care physicians
as described below. The distribution of health insurer coverage in the 16
states is representative of the US population. To capture private insurer
decisions, Blue Cross and Blue Shield (BC/BS) agencies from these states were
surveyed. In addition, we targeted the largest 5 private US indemnity insurers22 (60% of all lives covered by such plans), and the
largest 25 individual health maintenance organizations (HMOs) in the United
States in 1993 (covering 35% of all individuals enrolled in HMOs).23
The survey was mailed to the medical director of each organization in
late 1994 and early 1995, with follow-up telephone calls. A second mailing
was sent to nonresponders. Several measures were taken to ensure accuracy
of the data. We asked each respondent to indicate how well the survey responses
captured their organization's policies (choices included fully, partially,
and not at all). We also asked each respondent to indicate how much involvement
he or she personally has in decisions made regarding coverage of GH therapy
(choices included never involved, involved in an advisory capacity, direct
involvement with setting policies, oversee implementation and compliance with
policies, and approve all cases seeking coverage for GH therapy). In addition,
after receipt of the completed survey, each insurer was sent a personalized
summary of the information for verification.
We conducted separate national surveys of primary care physicians and
pediatric endocrinologists to address referral practices and treatment decisions,
respectively, for childhood short stature.
The primary care physician survey was mailed to 1504 practitioners (equal
numbers of family practitioners and general pediatricians), selected at random
by the Division of Survey and Data Resources of the American Medical Association
from the same 16 states described under the insurer survey.21
The survey included the same case descriptions as the insurer questionnaire.
Physicians were asked whether they would refer each case to a pediatric endocrinologist
for further evaluation, using a 5-point scale (categories included definitely
would not, not likely to, not sure, likely to, definitely would refer) with
the last 2 categories taken as decisions to refer.
A separate survey was sent to all members of the Lawson Wilkins Pediatric
Endocrine Society, the largest professional group of pediatric endocrinologists
in the United States and the only one devoted exclusively to endocrine disorders
in children (n=534, excluding physicians involved in survey development and/or
employed by the government or industry). Data from that survey were recently
reported5 and are presented here for comparison
with results from insurers and primary care physicians. The endocrinologists
were asked to indicate whether they would recommend GH therapy for children
with CRI or Turner syndrome, and for cases of idiopathic short stature matching
those presented to primary care physicians and insurers, with additional medical
information provided on the bone age x-ray film.
Census data on the US population of children,24
together with assessments of disease prevalence from the medical literature,14,25,26 were used to determine
the potential number of US children eligible for GH treatment because of the
3 medical disorders (GHD, Turner syndrome, and CRI). In evaluating the number
of children represented by the 4 cases of idiopathic short stature, we used
census data and the conditional presence of low or very low growth rates within
designated height categories, estimated using the Delphi method27
with a panel of 8 pediatric endocrinologists. Since the primary care physician
may not diagnose the cause of short stature in children with medical conditions
such as Turner syndrome, his or her decision to refer would typically be based
on stature and growth patterns alone. Therefore, referral patterns for the
4 cases of idiopathic short stature, based on height and growth rate, were
used to estimate referrals by primary care physicians for children with medical
causes of short stature. The cost of GH therapy, ascertained from the literature6 and corroborated by pharmacists and GH manufacturers,
was combined with standard dosages6 to yield
an annual cost of $700 per kilogram of body weight (applied to each case presented
based on average weights).16
The response rate for insurers was 75% (private insurers [80%], BC/BS
[50%], HMOs [64%], CSHCN programs [82%], and Medicaid programs [84%]). Over
80% of respondents indicated that they had direct involvement with setting
policies for GH coverage, oversaw implementation and compliance with policies,
or approved all cases seeking GH therapy within their organization. Faxed
verification of responses indicated that the data were accurate.
All insurers reported having a policy for the coverage of GH therapy,
with the exception of 9 state Medicaid programs. As shown in Table 1 (top), 28% of Medicaid agencies reported that they always cover GH therapy if prescribed by an authorized
physician and almost one quarter (22%) of CSHCN programs reported that they never cover any part of GH costs. Other payers fell between
these 2 extremes, covering GH therapy only under certain circumstances.
Heterogeneity also existed among third-party payers as to the type of
physician authorized to prescribe GHs. As shown in Table 1 (bottom), CSHCN programs and HMOs tend to restrict GH coverage
to prescriptions from pediatric endocrinologists, whereas most Medicaid agencies
and private insurers allow other physician groups (including pediatric endocrinologists,
adult endocrinologists [internal medicine], pediatricians, primary care physicians,
nephrologists, and/or "any physician") to prescribe GHs.
Insurer policies for GH treatment varied strikingly for conditions other
than classical GHD. For example, coverage policies for GH treatment of CRI
varied significantly among insurer groups, with approvals ranging from 36%
of CSHCN programs to 79% of Medicaid programs (P<.02, Table 2). For Turner syndrome, GH coverage
ranged from 25% of BC/BS insurers to 81% of Medicaid programs (P<.02, Table 2). For
each of the 4 children with idiopathic short stature, variation in coverage
was marked (P<.001). None of the private and BC/BS
insurers approved GHs for any of the cases presented, whereas approximately
half (48%-50%) of Medicaid agencies approved GHs for each case. Eight percent
of the CSHCN programs approved GHs for cases C and D, and 6% of HMOs approved
coverage for case D.
Based on the literature,28- 30
the distribution of insurance coverage for US children was assessed as follows:
private insurance (24%), BC/BS (25%), Medicaid (21%), HMO (17%), and CSHCN/uninsured
(13%). There are no data indicating different patterns of insurance for short
stature children; this distribution, together with insurer responses about
policies for GHs, was therefore used to construct weighted average approval
rates for GH coverage. The weighted average indicates that for each of the
4 cases with idiopathic short stature, 10% to 13% of children would have insurance
that covers GH treatment. For children with defined medical conditions, 94%
of children with classical GHD and slightly more than half of children with
Turner syndrome (52%) or CRI (58%) are likely to have insurance coverage for
We compared the proportion of primary care physicians who would refer
short children to endocrinologists, the proportion of endocrinologists who
would recommend GH therapy, and the weighted mean insurance coverage of GH
treatment for several causes of short stature. There was considerable discord
between physician recommendations and insurance policies for all conditions
other than GHD. For example, whereas 96% and 68% of pediatric endocrinologists
recommended GH therapy for Turner syndrome and CRI, insurers would cover GH
therapy for only 52% and 58% of these children, respectively.
For the 4 cases of idiopathic short stature, the differences between
physician recommendations and insurance coverage were particularly marked,
as illustrated in Figure 2. Among
primary care physicians, decisions to refer children to specialists ranged
from 6% (for a moderately short and slow-growing child, case A) to 95% (for
a very short and very slow-growing child, case D). While less than 5% of endocrinologists
recommended GHs for the first child (case A), recommendations rose progressively
depending on the height and growth rate of the child, with 58% of endocrinologists
recommending GHs for the most severely affected child (case D). By contrast,
insurer decisions were more categorical, with approvals for coverage of 10%
to 13% of children in each of the 4 cases. Thus, there was a 2- to 4-fold
mismatch between the recommendations of endocrinologists and the coverage
policies of insurers, with at least 1 reversal (ie, proportion of insurers
approving GH was greater than physician recommendations).
Combining the initial cohort of children with GHD, Turner syndrome,
CRI, and idiopathic short stature with the survey results for (1) primary
care physicians' referral decisions, (2) endocrinologists' GH treatment recommendations,
and (3) insurer approvals of GH coverage allows assessment of the proportion
of children excluded at each step of the decision process (Figure 3). While we recognize that families play a key role in GH
use by the emphasis placed on stature, physician-seeking behaviors, and willingness
to undertake treatment,18 data are lacking
on the factors that determine parental decisions and therefore the models
assume that all potential candidates would present to primary care physicians
to initiate the process. Approximately 66% of children with idiopathic short
stature are likely not to be referred to an endocrinologist by a primary care
physician (Figure 3, left); of those
referred, 74% are likely not to be recommended for GH treatment by endocrinologists.
Thus, 91% of the cohort are not recommended for GH treatment by physicians,
leaving only 9% to be considered by insurer groups. Yet, insurer groups, on
average, do not cover GH treatment for 89% of these remaining children. Therefore,
only 1% of the initial cohort of children with idiopathic short stature would
ultimately access GH treatment.
For the 3 medical causes of short stature (GHD, Turner syndrome, and
CRI) (Figure 3, right), 22% of children
are excluded by the 2 physician decisions, leaving 78% for insurer considerations.
Insurer groups, on average, do not cover the costs of therapy for 28% of these
children, leaving 56% of the initial cohort of children with both physician
recommendations and treatment coverage.
Using the results illustrated in Figure
3 together with estimated costs of therapy,6
we estimated the potential cost of GH treatment for US children. At the high
end of potential costs, if all short, slow-growing US children ages 4 to 15
years (approximately 1 million) obtained GH treatment, potential annual costs
could reach over $18 billion. Physician recommendations and insurer coverage
policies, ascertained from the current data, would reduce this amount to approximately
$357 million. If only children with 1 of the 3 medical conditions (GHD, Turner
syndrome, and CRI), a physician recommendation for GH, and insurance coverage
for GH were treated, there would be approximately 13400 candidates, at an
annual cost of $196 million. Thus, the potential costs to the United States
for GH treatment of children could range from a low of $196 million to a high
of $18 billion.
The overall importance of insurance coverage is clear. Patients without
either public or private insurance often have reduced access to medical care
and poor medical outcomes, compared with insured patients.1,3
However, the influence of type of insurance on access to and use of specific
treatments—particularly in the context of physician recommendations—is
not well understood. The current analyses provide insight into the role of
insurance in treatment utilization processes for specialized medical therapies.
The data indicate significant variation among US insurers regarding
coverage policies for GH therapy, supporting the concept that there are major
discrepancies in access to treatment as a function of third-party payers.
These findings are consistent with the few earlier retrospective assessments
for other conditions, undertaken in selected locations and populations,31,32 and extend them by conducting analyses
across insurance groups and geographic sites.
The contrast between physician recommendations for GH therapy and the
coverage decisions of insurers is striking. Primary care physicians appear
to use discretion in referring short children to specialists who, in turn,
are fairly selective in their treatment recommendations (ie, the overwhelming
majority would not recommend GH treatment for a moderately short child such
as case A). Despite the sequential medical process that favors relatively
limited GH use, insurance adds a further significant level of restriction.
For Turner syndrome, almost half of the children recommended for GH treatment
by endocrinologists would not, on average, have insurance that covered treatment,
and 42% of children with CRI who are recommended for treatment would not have
coverage. For idiopathic short stature, pediatric endocrinologists were selective
in their recommendations based on physiological patient characteristics, whereas
insurer policies were less responsive to these characteristics. Thus, for
3 of 4 cases presented, far fewer children would have insurance coverage for
GH therapy than would be recommended for treatment by physician experts. The
insurer therefore plays a critical role in influencing access to this treatment
and, in conjunction with other determinants, is instrumental in limiting GH
therapy to 1% of the initial cohort of children with idiopathic short stature
and 56% of children with 1 of 3 defined medical conditions.
While these findings cannot establish whether current practices are
right or wrong, questions remain as to the appropriate role of the insurer
and about how final decisions should be made regarding access to treatments.2,33,34 The discord between
physicians and insurers may result, in part, from current debates about what
constitutes deficiency and disease, and what testing criteria ought to be
used in the determination of a GH disorder.4,7,10,11
Nevertheless, judging from the data, many payers do not agree with the expert
opinion of pediatric endocrinologists. The apparent discounting by insurers
of physician recommendations may not be limited to GHs. Reports within the
lay press 35,36 suggest similar
conflicts in other settings, such as bone marrow transplants in cancer patients.
Federal agencies have begun to consider such issues.37
The current data, to our knowledge, are the first to systematically analyze
and quantify the implications of disagreements between physicians and insurers.
Although disagreements may be difficult to resolve, our findings underscore
their importance in determining differential access to treatment and, as such,
indicate the necessity of serious efforts toward resolution.
In interpreting the findings, several limitations are noted. It is possible
that coverage policies reported from an insurer's national headquarters (eg,
private insurers) may not reflect local variation or "tailor-made" individual
policies, although respondents to our survey did indicate that policies are
consistent across product lines. Categorical policy decisions may also be
mutable by pressures such as lawsuits32 and
individual lobbying. The absence of global claims data precludes comparison
with these survey results, although the figures derived from our survey data
are consistent with those reported elsewhere. In addition, although our response
rates were generally high, plans not responding might have different policies
from those of the responders. The increasing trend toward state Medicaid managed
care plans may impose different prescribing and approval patterns within a
more restrictive environment. It is also recognized that some children and
their families may be self-referred to endocrinologists, while some children
with distinct medical disorders may be missed or misdiagnosed at an early
point in the sequential decision process. Furthermore, we assumed independent
decision making when modeling exclusion processes. It is possible that physician
decisions and insurer decisions are influenced by each other, although we
controlled for insurance in the survey to prescribing physicians by indicating
the out-of-pocket costs of GH treatment (after insurance coverage) for the
cases presented. Physician and insurer decisions may also be altered by future
changes in treatment costs, although the price of GH has remained relatively
stable despite new manufacturers and the loss of Orphan Drug status in 1995.
In summary, this study provides insight into the sequential process
of medical decision making found in most nonemergency or subspecialty care.
It illustrates how medical referral and expert opinion serve as powerful forces
in limiting treatment utilization to a subset of potential patients even in
cases as ambiguous as short stature of unknown cause. The fact that insurance
coverage further limits access may simply reflect a reasonable cost-benefit
calculus at work. Alternatively, it may represent inappropriate denial of
care. Nonetheless, the discrepancy between physician treatment recommendations
and insurance coverage, exemplified by the current findings, constitutes a
critical challenge to health care delivery with serious ramifications for
access, costs, and outcomes.