In Reply We agree with Drs Ruble and Brixner that not all patients with hemophilia are the same. These patients represent a wide range of severity, and the cost skewing within the group reflects that. Hemophilia is not unique in this—most serious pediatric conditions demonstrate a range in severity.
Ruble and Brixner accurately describe the reasons for this variation, including presence of inhibitors, and note that patients with inhibitors have higher costs of therapy.1 We do not dispute these facts. Our Research Letter was not meant to be a profile of patients with hemophilia in California, but an analysis of overall outpatient pharmacy expenditures by children with serious chronic illness.
Chamberlain LJ, Swenson SM, Wise PH. Pharmacy Expenditures for Children With Serious Chronic Illness—Reply. JAMA. 2016;315(7):706-707. doi:10.1001/jama.2015.16981