Pediatric Hemophilia Dominates Pharmacy Expenditures

Outpatient pharmacy spending totaled 20% of total health care expenditures.

Hemophilia therapies account for 41% of outpatient pharmacy expenditures among publicly insured children with serious chronic illness.

This finding, which was published as a research letter in JAMA, highlights the need to contain costs for pediatric hemophilia treatments. These pricey medications are increasing health care costs for children with chronic illness, who are likely to survive infancy now more than ever, a JAMA press release noted.

In analyzing this trend, Stanford University researchers examined expenditures for outpatient pharmacy products used by nearly 35,000 publicly insured children with serious chronic illness during 3 years.

They studied paid claims for children ages 0 to 21 years using the California Children’s Services (CCS) paid claims data set from 2010 to 2012. This included children enrolled through fee-for-service care for a minimum of 6 continuous months.

The records showed that outpatient pharmacy expenditures totaled more than $475 million, or 20% of total health care expenditures. The costs varied widely at the individual level, ranging from 16 cents to nearly $57 million per child, with average and median per-child costs of $13,857 and $791, respectively.

Blood formation, coagulation, and thrombosis agents accounted for 42% of outpatient pharmacy expenditures. Within this class, antihemophilic factor, a protein that is lacking or deficient in those with hemophilia A, represented 41% of all pharmacy expenditures.

Notably, children with a paid claim for antihemophilic factor represented only 0.4% of the cohort. The average per-child expenditure for this treatment was more than $1.3 million. Among children who had antihemophilic factor claims and were enrolled in CCS for all 3 years of the study, the average and median per child annualized expenditures were $634,054 and $152,280, respectively.

“Antihemophilic factor is highly efficacious and essential in caring for children with hemophilia, putting pressure on public programs to seek improved pricing mechanisms for antihemophilic factor and other highly efficacious, high cost medications,” the study authors concluded. “Our study underscores the potential effect of new, expensive but efficacious pharmaceuticals on public insurance programs for children with chronic illness. These findings may inform efforts to enhance value in these programs, particularly as new insurance frameworks, such as accountable care organizations, are considered.”

After antihemophilic factor, the next largest percentage of outpatient pharmacy total expenditures was for central nervous system agents (9.2%) at an average per-child cost of $1869.