SUN-249 Growth Hormone Utilization By US Youth: Trends as Insurance Pushes Back after FDA-Approved Expansion

Abstract

In 2003 US FDA approved growth hormone (GH) treatment for idiopathic short stature (ISS), expanding potential eligibility for GH treatment from 1:3500 children with GH deficiency to the shortest 1.2% of the US population, at a potential estimated national cost of $40 billion. In response, over the last decade or so, the insurance industry has been instituting progressive restrictions on coverage for GH treatment, adopting formulary preference coverage strategies as a cost-containing measure, adopting more stringent plan-specific criteria for coverage, and denying any coverage for GH treatment of ISS. Using Optum administrative claims data (Clinformatics DataMart™ 7.0, 2017), which covers an annual 18 million individuals enrolled in commercial health plans, we sought to investigate how utilization of GH drugs by youth has changed in response to FDA approval of GH treatment for ISS, and secular changes in utilization as insurers have imposed greater barriers to GH treatment reimbursements. For our analysis, we included individuals aged 0-18 years who had insurance coverage between 2001 and 2016 for 6 or more months in any given year. Linear time trends in prevalence and financial burden were tested using t-tests with Bonferroni correction. Because of the long time frame covered, we analyzed payments in both nominal dollars and real dollars (1982-84 CPI-deflated). The number of members with GH prescriptions per 10,000 beneficiaries under age 18 rose steadily from 5.1 in 2001 to14.6 in 2016 (total on GH=38,857), without a dramatic change around 2003, the ISS approval date. The proportion of females receiving GH treatment dropped (34% to 28%) as did the proportion of white youth (80% to 77%), while proportions of black (3% to 4%) and Asian (1% to 4%) youth increased. Mean and median ages were 12-13 throughout. Total GH expenditures decreased (by 6% in nominal and 27% in real dollars), as did the estimated insurance-paid amount (by 8% in nominal and 28% in real dollars). However, mean copayments, deductibles and total patient financial burden increased (total up by 234% in nominal and 161% in real dollars). Between 2001 and 2007, beneficiaries switching GH brands at least once in the year ranged from 1.4 to 3.6%. After 2007, the range rose to 5.1-8.8%, with 25.6% switching in 2009 and 13.9% switching in 2015. In conclusion, the FDA ISS approval was not a watershed event in the steady increase in GH utilization by US youth. Progressive restrictions on coverage and formulary preference coverage strategies appear to have succeeded in lowering the total cost and insurer burden of GH treatment. However, those savings were not passed on to patients, who bear higher burdens financially and from brand switches. These patterns are consistent with broader US trends of increased patient cost-sharing and aggressive use of formularies and coverage restrictions to limit insurer exposure to high specialty drug prices.