Background: The clinical benefits of prophylaxis in patients with hemophilia are well-established and include the following: reduced bleeding episodes, prevention of joint damage, decreased inhibitor development, and improved health-related quality of life. However, the cost-effectiveness of prophylaxis is still not clear. Procedures: We reviewed the published hemophilia prophylaxis economic models focusing on utility assumptions. Results: We found six cost-utility studies that compared prophylaxis and on-demand regimens. These studies reported remarkably different results, using utility values based on different assumptions and data sources. Conclusions: We suggest that cooperation among key stakeholders (clinicians, patient organizations and health-care decision makers) as a means of collecting evidence-based and experiential data to represent both the utility and the quality of life changes for patients with Hemophilia A who are treated with prophylaxis or receive on-demand treatments may represent a winning strategy with which to resolve the outstanding issues related to health technology assessments in the care of patients with hemophilia. Pediatr Blood Cancer 2015;62:1826-1831.
- Cost Utility Analysis (CUA)
- Health Technology Assessment (HTA)
- Health-related quality of life (HRQoL)
- Quality-adjusted life years (QALY)
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health