Hemophilia therapies account for the largest portion of pharmacy expenditures among publicly insured children with serious chronic illnesses in California, according to a study published in JAMA.
Hemophilia therapies accounted for 41% of expenditures for these children, even though hemophilia patients made up only 0.4% of the group studied.
Researchers said this finding suggests a need to improve pricing for hemophilia therapies and other high-cost medications. However, they noted that pricing varies from state to state.
Sonja M. Swenson, of Stanford University in California, and her colleagues conducted this research, analyzing paid claims for children (ages 0-21 years) using the California Children’s Services (CCS) paid claims data set (2010-2012).
CCS provides insurance coverage, care coordination, and a regionalized system of pediatric specialty care facilities for approximately 180,000 publicly insured children with serious chronic illnesses.
The data set includes age, sex, race/ethnicity, county of residence, enrollment dates, primary and secondary eligible diagnoses, claim diagnoses, and procedures for every enrollee. This study included children enrolled through fee-for-service care for at least 6 continuous months.
The researchers examined the records of 34,330 children. Outpatient pharmacy expenditures totaled $475,718,130 (20% of total healthcare expenditures).
Per-child pharmacy expenditures ranged from $0.16 to $56,849,034. The average and median per-child expenditures were $13,857 and $791, respectively.
Expenditures for all products analyzed were as follows:
| Product Class | Expenditures | % of Total
Expenditures |
No. of Children | Expenditures/
Child |
|---|---|---|---|---|
| Blood formation, coagulation, and thrombosis agents | $199,498,843 | 41.9% | 3499 | $57,016 |
| Central nervous system agents | $43,633,418 | 9.2% | 23,351 | $1869 |
| Electrolytic, caloric, and water balance | $39,617,776 | 8.3% | 10,959 | $3615 |
| Anti-infective agents | $35,827,958 | 7.5% | 26,165 | $1369 |
| Respiratory agents | $29,614,645 | 6.2% | 16,706 | $1173 |
| Hormones and synthetic substitutes | $24,722,256 | 5.2% | 8542 | $2894 |
| Enzymes | $13,294,509 | 2.8% | 27 | $492,389 |
| Gastrointestinal drugs | $12,500,330 | 2.6% | 11,817 | $1058 |
| Heavy metal antagonists | $6,983,828 | 1.5% | 108 | $64,665 |
| Cardiovascular drugs | $6,173,792 | 1.3% | 4031 | $1532 |
Hemophilia expenditures
As seen in the above table, the product class of blood formation, coagulation, and thrombosis agents accounted for the greatest share of outpatient pharmacy expenditures (42%).
Antihemophilic factors represented 98% of this class’s expenditures, or 41% of total pharmacy expenditures. Children with an antihemophilic factor paid claim were 0.4% of the entire cohort (n=145). And the average per-child expenditure for antihemophilic factor was $1,343,262.
Among children with antihemophilic factor claims who were enrolled for all 3 years studied, the average and median per-child annualized expenditures were $634,054 and $152,280, respectively.
The researchers said these results suggest a need for better pricing for hemophilia therapies, but it’s important to note that expenditures vary from state to state.
For instance, CCS’s mean per-child antihemophilic factor annual expenditure ($634,054) significantly surpassed that of North Carolina’s Medicaid program ($233,968 in fiscal year 2012) and Medicaid programs in 10 other states ($148,215 in 2008).
