Substantial Annual Health Care Costs Associated With Hemophilia A

Hemophilia A management incurs significant health care costs, which vary widely depending on population characteristics and prophylaxis intensity, according to findings published in the Journal of Managed Care & Specialty Pharmacy.

Hemophilia A is a rare genetic bleeding disorder characterized by a deficiency of blood clotting factor VIII (FVIII), which results in prolonged bleeding and difficulty in clotting. To gain insights into the health care resource utilization and costs associated with managing hemophilia A in the United States, a targeted literature review was conducted.

Researchers conducted a comprehensive search of databases such as Embase, MEDLINE, and Cochrane Database of Systematic Reviews, covering the period from 2010 to 2022. They also included gray literature, such as abstracts, posters, conference presentations, and reports from the Institute for Clinical and Economic Review. In total, 40 publications met the eligibility criteria. 

The findings revealed a wide range of annual health care costs per patient, varying from $213,874 to $869,940. The primary driver of these costs was the expense and intensity of prophylactic treatment with FVIII replacement concentrates, bypassing agents, and the recently introduced therapy emicizumab. The estimated treatment costs exhibited significant heterogeneity, influenced by factors such as hemophilia A severity, treatment type and intensity, patient age, weight, and inhibitor status, according to the findings. 

Patients with inhibitors, which are antibodies that neutralize FVIII, incurred substantially higher costs. Furthermore, among a subset of adult patients without inhibitors, annual FVIII treatment costs were found to increase over time. 

Only 2 studies reported indirect costs, with annual expenses of $13,220 and $27,978 for patients without and with inhibitors, respectively. Additionally, parents of children with hemophilia A incurred costs of $8252 for nonmental health services and $258 for mental health services on an annual basis.

The researchers emphasized the need for more robust studies in different settings and subpopulations to fully understand the societal and economic impact of hemophilia A. Additionally, the impact of emerging therapies on costs and the assessment of indirect costs and productivity loss should be further investigated, they said.

Reference:

Chen Y, Cheng SJ, Thornhill T, Solari P, Sullivan SD. Health care costs and resource use of managing hemophilia A: A targeted literature review. J Manag Care Spec Pharm. 2023;29(6):647-658. doi:10.18553/jmcp.2023.29.6.647