Health care resources and costs associated with delivering gene therapy for hemophilia in clinical practice

Abstract

Background: The debate around the cost-effectiveness of gene therapy for hemophilia has largely centered on its price (€1-€3.5 million per individual). While previous studies have explored care organization for gene therapy delivery, none have evaluated the potential resource utilization and associated costs in a real-world setting. Objectives: This study aimed to estimate the health care resources and costs of delivering gene therapy for hemophilia in clinical practice. Methods: We conducted a bottom-up microcosting study and constructed a process map outlining each step of care. Data on resource use were obtained from clinical trial protocols and translated to reflect real-world clinical practice through semistructured interviews. Dutch unit costs were assigned to each resource, and mean total costs per individual were calculated for hemophilia A and B. Sensitivity analyses were performed to assess the potential range of consumed resources and costs. Results: In clinical practice, delivering gene therapy for hemophilia is expected to require resources such as personnel time, hospital visits, laboratory tests, liver function assessments, drugs, hospital facilities, medical consumables, and office equipment. The estimated total cost for an eligible individual without liver function abnormalities, covering screening, pretreatment preparation, administration, and first-year follow-up, is €28,696 (€20,873-€48,973) for hemophilia A and €20,511 (€18,175-€36,310) for hemophilia B. Conclusion: Delivery of hemophilia gene therapy requires significant resources, incurs substantial costs, and demands additional organizational infrastructure within treatment facilities. These findings may aid stakeholders to better plan implementation of these innovative therapies into clinical practice, as well as inform economic evaluations and reimbursement discussions.