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Coverage for pediatric GH therapy varies widely in idiopathic short stature
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Among children with idiopathic short stature who do not test as growth hormone-deficient, the decision as to who receives coverage for GH therapy is unrelated to the degree of shortness or predicted adult height, according to a new analysis published in Endocrine Practice.
In the 15 years since the FDA approved the use of GH for children with idiopathic short stature, use of the therapy has expanded, with at least seven companies marketing GH therapy for pediatric indications, Paul Kaplowitz, MD, PhD, former chief of endocrinology at Children’s National Medical Center in in Washington, D.C., and colleagues wrote in the study background. However, across the U.S., insurers vary in their willingness to approve GH therapy for short children without GH deficiency.
“The decisions insurances companies make regarding whether to cover growth hormone or not are somewhat random and not really based on the degree of shortness and the anticipated final height, which are things that endocrinologists feel are very important,” Kaplowitz told Endocrine Today. “The take-home message is we need to figure out ways to make growth hormone for those who do not fit into the category of growth hormone deficiency more available. The question is really how best to do that.”
In a retrospective chart review, Chandar and colleagues analyzed data from 87 patients who underwent GH stimulation testing (glucagon followed between July 2009 and April 2017 to identify idiopathic short statue, defined as a height < –2.25 standard deviation [SD], subnormal predicted adult height and a peak GH of > 10 ng/mL.) Within the cohort, 47 of those patients (29 boys) had a GH therapy request submitted to insurance (mean age, 9 years; mean height velocity, –2.83 SD; mean growth velocity, 4.4 cm per year). The mean predicted adult height based on bone age was –2.5 SD, equaling 62 inches for men and 58 inches for women. Most patients (74%) had private insurance.
Of the 47 patients with a request for GH therapy, only seven patients (15%) received approval after the initial submission, according to researchers. According to the review, the letters from insurance companies in most cases included reasons for rejection such as “does not meet criteria, as [idiopathic short stature] is not approved for GH therapy” or “GH therapy is not done for cosmetic indications.”
Of the 40 rejections, 30 were appealed. Researchers observed that the low predicted adult height, which was not included in the initial statement of medical necessity form, was emphasized on appeal. “In this process, an additional 10 patients were approved,” the researchers wrote. “Of the 30 patients for whom GH therapy was not approved, 12 families paid for GH out of pocket, 10 received GH through drug company patient access programs and eight were not treated.”
Of 39 patients who eventually received GH therapy, researchers had follow-up measurements for 21, observing a mean height increase of 0.6 SD at 1 year, but noting a “wide range” of increases from 0.2 SD to 1.1 SD.
The researchers noted that the decision to recommend a trial of GH therapy for idiopathic short stature should be “weighed carefully” and that families should not receive overly optimistic projections as to the long-term benefit.
“While some companies have modified their treatment guidelines to make GH accessible for very short children with normal GH testing, in the [Washington, D.C.] area at least, a peak GH of < 10 ng/dL is still the primary criteria used for approval,” the researchers wrote.
Kaplowitz said cost of therapy remains a concern.
“Growth hormone is expensive, partly because its usage extends, potentially, over many years,” Kaplowitz said. “It can easily run $15,000 to $25,000 a year. You can see why insurance companies are not wild about covering all cases of idiopathic short stature. Getting the key stakeholders together is one way we might be able to work through this issue. However, it would take the recognition of people in my field that we can’t ignore the concerns of the insurance companies about how expensive growth hormone is.”
– by Regina Schaffer
For more information:
Paul Kaplowitz, MD, PhD, can be reached at Children’s National Health System, 111 Michigan Ave NW, Washington, DC 20010; email pkaplowi@childrensnational.org.
Disclosures: The authors report no relevant financial disclosures.
Perspective
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The FDA approved GH therapy in 2003 for idiopathic short stature, defined as children with height below 1.2 percentile (<–2.25 SD) without evidence of GH deficiency and a “growth rate unlikely to attain an adult height within the normal range.” In the 15 years since the FDA approval, pediatric endocrinologists in practice have increasingly faced denials from insurance companies for patients who meet the criteria. Chandar and colleague have analyzed this trend in this retrospective chart review that confirms what most of us in practice have been experiencing. Their analysis is from a single large urban children’s hospital in the Washington, D.C. area, but their results are generalizable to most states and insurance plans.
The authors raise six important points to be considered by the pediatric endocrine community to help decrease the overall cost of GH therapy while allowing approvals for treatment of very short children. These include a height cut-off of less than or equal to –2.5 SD (0.6 percentile) instead of –2.25 SD (1.25 percentile); a growth rate of less than 4.5 cm per year or less than the 25th percentile for age, except in very short children; a predicted adult height of no more than 64 in for boys and 60 in for girls; no GH testing in short children with normal IGF-I levels meeting the aforementioned criteria; a starting dose of 0.24 mg/kg/week (20% less than the “standard dose” of 0.3 mg/kg/week); and stopping treatment once the child reaches normal range in height for those children who are growing well after onset of puberty.
The treatment of idiopathic short stature with GH therapy has been controversial since the FDA approval. Increasing demand for GH therapy in non-GH-deficient children has led to increased rate of insurance denials for treatment. It is time that the pediatric endocrine community revisit this issue so GH is still available for use in very short children who need it the most to help them get even closer to normal, functional adult height.
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