July 29, 2015
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Hemophilia therapies drive outpatient pharmacy costs for publicly insured children

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Medications for the treatment of hemophilia are a considerable driver of outpatient pharmacy expenditures for publicly insured children with serious chronic conditions, according to study results.

An analysis of outpatient pharmacy products in California between 2010 and 2012 showed antihemophilic agents accounted for more than 40% of the costs, even though they were only used to treat 0.4% of the population studied.

Sonja M. Swenson, BA, the research coordinator at the Center for Health Policy and Center for Primary Care and Outcomes Research at Stanford University, and colleagues retrospectively analyzed paid insurance claims for 34,330 children aged 0 to 21 years in California.

The investigators determined outpatient pharmacy expenditures totaled $475.7 million, and this accounted 20% of total health care expenditures.

Per-child pharmacy expenditures ranged from 16 cents to $56.84 million, for an average of $13,857 per child (median, $791; interquartile range, [IQR] = 127-5,873).

“Our study underscores the potential effect of new, expensive but efficacious pharmaceuticals on the public insurance programs for children with chronic illness,” the researchers wrote. “These findings may inform efforts to enhance value in these programs, particularly as new insurance frameworks, such as accountable care organizations, are considered.”

Blood formation, coagulants and thrombosis agents accounted for 41.9% of the expenditures. Antihemophilic factor, a protein that necessitates blood clotting and is deficient in patients with hemophilia, made up 98% of that group, or 40.9% of all pharmacy expenditures.

Children with an antihemophilic factor paid claim accounted for 0.4% of the entire cohort.

The average per-child expenditure for antihemophilic factor was $1.34 million. For those children with antihemophilic factor claims who were enrolled for the entire 3 years of the analysis, the average annual expenditure was $634,054 (median, $152,280; IQR = 19,434-393,000).

As a comparison, the next largest percentage of total pharmacy expenditures was 9.2% for central nervous system drugs, with a per-child average of $1,869.

“Antihemophilic factor is highly efficacious and essential in caring for children with children with hemophilia, putting pressure on public programs to seek improved pricing mechanisms for antihemophilic factor and other highly efficacious, high-cost medications,” Swenson and colleagues wrote.

Limitations for this analysis included a lack of clinical data, lack of in-patient pharmacy data and the study’s cross-sectional design. Also, researchers excluded children enrolled in managed care, and data were limited to 3 years.

The investigators indicated that a state-to-state examination may provide further insights. The average annual expenditure for children with anthemophilic factor claims over 3 years significantly surpassed North Carolina’s Medicaid program, which was $233,968 in fiscal year 2012, as well as Medicaid programs in 10 other states.

“Public programs for children with serious chronic illness vary between states, and care should be taken in making direct program comparisons,” Swenson and colleagues wrote. “Greater transparency of use and costs, and cross-state collaboration, may increase health care value as states revise programs.” – by Anthony SanFilippo

Disclosure: The researchers report no relevant financial disclosures.