Healthcare resource utilization among haemophilia A patients in the United States

Summary. Advances in therapy have improved life expectancy and quality of life of patients with haemophilia A. Due to the chronic and complex management of this disease, particularly, the development of inhibitors, little is known about their health resource utilization in the real‐life setting over time. The aim was to assess the distribution and trend of healthcare resource utilization among US haemophilia A patients with and without inhibitors. The MarketScan® Database, was queried to identify individuals with ≥1 year continuous enrolment, two medical diagnoses of haemophilia A and claims for factor VIII or bypassing agent (to infer inhibitor status) during 2001–2007. Haemophilia‐related cost was estimated from inpatient, outpatient and pharmacy claims. Annual cost differences were assessed by age and over a 4‐year period for those with continuous enrolment. Among 51 million covered lives, 1044 haemophilia patients were identified, of whom 981 (94%; mean age = 21.2 years) did not have an inhibitor. The median haemophilia‐related cost for these patients was $63,935 per patient per year. When normalized by weight, annual cost was stable (no statistically significant differences) among 312 non‐inhibitor patients (mean age = 21.8 years) with 4‐year continuous data. While there was a wide distribution of haemophilia‐related cost among the 63 individuals with an inhibitor (mean age = 15.4 years), only 0.6% of the total haemophilia patients had costs exceeding $1 million per patient per year. This study indicated that most haemophilia A patients were inhibitor‐free with relatively stable annual costs over time. There was a wide distribution of haemophilia‐related cost for inhibitor patients, while the proportion of patients who incurred extreme high cost was low.