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.

Valente, M., Cortesi, P., Lassandro, G., Mathew, P., Pocoski, J., Molinari, A., et al. (2015). Health economic models in hemophilia A and utility assumptions from a clinician's perspective. PEDIATRIC BLOOD & CANCER, 62(10), 1826-1831 [10.1002/pbc.25543].

Health economic models in hemophilia A and utility assumptions from a clinician's perspective

CORTESI, PAOLO ANGELO
Secondo
;
MANTOVANI, LORENZO GIOVANNI
Penultimo
;
2015

Abstract

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.
Articolo in rivista - Review Essay
Cost Utility Analysis (CUA); Health Technology Assessment (HTA); Health-related quality of life (HRQoL); Hemophilia; Prophylaxis; Quality-adjusted life years (QALY); Hemophilia A; Hemorrhage; Humans; Cost-Benefit Analysis; Models, Economic; Quality Assurance, Health Care; Oncology; Pediatrics, Perinatology and Child Health; Hematology; Medicine (all)
English
1826
1831
6
Valente, M., Cortesi, P., Lassandro, G., Mathew, P., Pocoski, J., Molinari, A., et al. (2015). Health economic models in hemophilia A and utility assumptions from a clinician's perspective. PEDIATRIC BLOOD & CANCER, 62(10), 1826-1831 [10.1002/pbc.25543].
Valente, M; Cortesi, P; Lassandro, G; Mathew, P; Pocoski, J; Molinari, A; Mantovani, L; Giordano, P
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10281/100105
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