Patients Pay More for Growth Hormone, Suffer Restrictions on Use

Tara Haelle

April 15, 2019

NEW ORLEANS — Costs associated with human growth hormone (GH) prescriptions in the US have dropped overall, particularly for insurance companies, but patient's families have seen costs more than double in the past 15 years, according to a poster presented last month at ENDO 2019: The Endocrine Society Annual Meeting.

This follows the approval of GH for idiopathic short stature (ISS) by the US Food and Drug Administration (FDA) in 2003, which led to expectations that prescriptions for this medication would increase substantially; however, background information in the poster reveals this change didn't pan out.

But the findings also reveal that insurance companies, fearing an explosion in prescriptions, proceeded to regularly change the "preferred" GH product in their formularies, causing aggravation for physicians and their staff, in terms of time spent on paperwork and other bureaucracy, and for patients and their families.

For the latter, this can lead to breaks in therapy as patients struggle to navigate different GH brands and their delivery devices.

"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," concluded lead author Adda Grimberg, MD, a pediatric endocrinologist and scientific director of the Growth Center at the Children's Hospital of Philadelphia, Pennsylvania.

"Because GH is a very expensive medication that requires years of daily injections, cost-containment efforts by insurance providers have increasingly encroached upon and eroded patient–physician decision making, autonomy, and clinical care," she stressed in a prepared statement.

"It was important for us to understand the extent of how these restrictions by insurance companies were impacting families and GH use overall."

Asked to comment, Alan Rogol, MD, a pediatric endocrinologist and professor emeritus of the University of Virginia, Charlottesville, told Medscape Medical News that, although the findings are not surprising at all, the research and analysis are well-reasoned and well-done.

"They did their homework and have a proper dataset," he noted, praising the work of Grimberg and her coauthor, Genevieve P. Kanter, PhD, of the University of Pennsylvania Leonard Davis Institute of Health Economics, Philadelphia.

He added that this pattern is occurring with more than just GH. The situation is even worse, he said, with pediatric insulin prescriptions, which are life-saving. "It's a familiar story in US healthcare," he said.

GH Approved for Idiopathic Short Stature 15 Years Ago

The US FDA's approval of GH for idiopathic short stature (ISS) in 2003 led to expectations that prescriptions for this medication would increase substantially, from 1 in 3500 children for GH deficiency to hundreds of thousands of children diagnosed with ISS. Those with a height more than 2.25 standard deviations below average (based on age, sex, and population) meet the criteria for ISS, equivalent to the shortest 1.2% of the population, according to background information in the poster.

But that expectation did not pan out: data from the poster show no substantial relative increase in prescriptions since 2001.

Grimberg analyzed Optum administrative claims data (an estimated 18 million patients are annually enrolled in commercial health plans) to understand prescribing patterns for GH among patients aged 0-18 years who had insurance coverage for at least 6 months in any year in 2001-2016.

Prescriptions did steadily increase from 5.1 patients/10,000 beneficiaries in 2001 to 14.6 patients/10,000 beneficiaries in 2016. But no bumps occurred around 2003, suggesting the expected boom from the FDA approval of GH for ISS failed to materialize.

Regardless, insurance companies enacted restrictions to offset the $40 billion anticipated increase in costs from the expansion of GH indication (which never occurred).

Over the past decade, those restrictions have included "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," according to Grimberg's poster.

Insurance companies subsequently saw a drop in payout costs as GH expenditures per patient declined — and patient' cost burdens rose.

Total expenditures related to GH dropped by 27% in real dollars (6% in nominal dollars), and insurance payments dropped 28% in real dollars (8% in nominal). At the same time, however, patients' costs more than doubled: average copayments, deductibles, and overall financial costs increased 161% in real dollars (234% in nominal).

Insurance Companies Have Regularly Altered Preferred GH, Causing Hassle

One of the key strategies insurance companies have used to control their costs — regularly changing which GH product is preferred in their formulary — has added administrative burdens to pediatric endocrinologist practices, as well as stress and anxiety to patient families, Grimberg, also a professor of pediatrics at the University of Pennsylvania Perelman School of Medicine, told Medscape Medical News.

Although the drug's active ingredient does not differ across brands, each company has a different patented device for administering it.

"The pens are not equal, so the parents like one pen versus another pen, but they have no say. We have no say, and every single time they get switched we have to retrain them."

Different pens also use different cartridges with different dosage amounts, and a cartridge cannot mismatch a pen. "I have a family who did that for 2 months before they came in to see me and realized that the colors didn't match, so there's potential safety issues as well," she explained.

The proportion of beneficiaries who switched GH drug brands at least once a year first increased from 1.4% in 2001 to 3.6% in 2007 before beginning to climb exponentially. An estimated 5.1%-8.8% switched brands at least once just after 2007; that number had reached 25.6% in 2009 before dropping again to 13.9% in 2015.

Switching also means more paperwork for the pediatric endocrinologist and staff, and more time for a nurse practitioner to retrain the family on how to administer the medication, Rogol commented to Medscape Medical News.

"If you're changing year by year by year, it's a drain on secretarial and nurse time," he said.

And all that paperwork — and the phone calls and paperwork associated with writing appeals for denied claims, Grimberg added — can contribute to treatment lapses.

Rogol agrees: "The most important implication is that this can and does lead to breaks in therapy," he l stressed. "It might be a week or it might be a month as you wind your way through the system."

Demographic Shifts in Prescribing Too

Although gender disparities in GH prescribing patterns have been well documented for years as favoring boys over girls, Grimberg's research also reveals that these disparities have worsened. Girls comprised only 28% of patients receiving GH treatment in 2016, compared with 34% in 2001. However, a significantly greater prevalence of ISS among boys than girls has not been found.

Over those years, the proportion of patients receiving GH treatment also saw racial shifts: the percentage of white youth comprising the patient population fell from 80% to 77% while it increased from 3% to 4% among black youth and 1% to 4% among Asian youth.

"I really wanted to get these data out there to educate the clinicians," Grimberg said, because such lack of transparency exists regarding cost agreements between pharmaceutical companies and insurance companies.

No external funding was declared. Grimberg has reported receiving consulting fees from Pfizer. Rogol has reported no disclosures related to this topic, but consults for Acerus Pharmaceuticals, Clarus Therapeutics, Ferring Pharmaceuticals, Lipocine, and Viramal, and recently testified on behalf of these companies before the FDA. He has also consulted for Ultragenyx.

ENDO 2019. Presented March 24, 2019. Abstract SUN-249.

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