US Growth Hormone Use in the Idiopathic Short Stature Era: Trends in Insurer Payments and Patient Financial Burden

Abstract

Objective To investigate trends in prevalence and expenditures of growth hormone (GH) use by US youth in the last 15 years, a period during which the US Food and Drug Administration (FDA) approved GH treatment of idiopathic short stature (ISS), and insurers imposed greater barriers to GH treatment reimbursements. Design With the use of 2001 to 2016 OptumInsight commercial claims data, we analyzed trends in claims of GH drugs among beneficiaries aged 0 to 18 years (n = 38,857 beneficiaries receiving GH). Outcome measures included annual prevalence of GH claims and annual total insurer and total patient payments for GH claims. t Tests were used for linear time trends in outcomes. The percentage of beneficiaries switching GH brands also was calculated. Results The number of members with GH claims per 10,000 beneficiaries under age 18 rose steadily from 5.1 in 2001 to 14.6 in 2016, without a dramatic change around 2003, the ISS approval date. Mean total GH expenditures decreased (−26% in constant dollars), as did the estimated insurance paid amount (−28%). However, mean total patient spending increased by 163%. Beneficiaries switching GH brands in the year ranged from 1.4% to 3.6% in 2001 to 2007 and from 5.1% to 8.8% after, with 25.6% switching in 2009 and 13.9% switching in 2015. Conclusions The FDA ISS approval was not a watershed event in the steady increase in GH use by US youth. Progressive restrictions on coverage and formulary preference coverage strategies appear to have succeeded in lowering total expenditures and insurer burden of GH treatment per beneficiary. However, those savings were not passed on to patients who bore greater burdens financially and from brand switches.

Real-World Treatment Patterns and Outcomes of Growth Hormone Treatment Among Children in Israel Over the Past Decade (2004–2015)

“…However, GH treatment rates for ISS vary between countries. Such variation may be attributed to differences in insurance coverage rules, access to health care, and attitudes toward treating children with ISS by patient families and/or their treating pediatricians and endocrinologists ( 24 , 27 ).…”

Growth Hormone Stimulation Testing Patterns Contribute to Sex Differences in Pediatric Growth Hormone Treatment

“…The rate of false-positive results may be as high as 50% [26-29]. Second, because many insurance providers deny coverage for GH treatment of ISS [7], pediatric endocrinologists may order the test to be able to assign a GHD diagnosis to justify GH treatment for children with ISS.…”

“…Further, despite absence of sex differences in distribution of height z (HtZ)-scores in a large pediatric primary care population in the USA[3], boys outnumbered girls 2:1 for all growth hormone (GH) indications and 3:1 for idiopathic short stature (ISS) in 4 US pediatric GH registries [3, 4]. The burden of GH treatment includes expense – USD 35,000–52,000 per inch of height gained [5, 6] with a mean of USD 1,099 per year paid by the commercially insured patient in 2016 [7] – potential side effects [8], and daily injections [9].…”

Comparative effectiveness of East Asian traditional medicine for treatment of idiopathic short stature in children

“…As effective standard therapies for pediatric ISS are lacking and GH treatment is controversial, [ 4 , 5 ] various EATM treatments such as acupuncture, herbal medicine, and chuna, which have been used for thousands of years, are currently employed for the treatment of pediatric ISS patients in clinical settings. However, clinical studies that directly compare the effectiveness of EATMs are rare.…”

“…In July 2003, the US Food and Drug Administration approved the use of growth hormones (GHs) for ISS treatment [ 3 ] ; however, the treatment is still controversial because of variable efficacy and high costs. [ 4 , 5 ] Consequently, a guideline was released recommending against the use of GHs for the treatment of ISS. [ 6 ]…”