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Health economic models in hemophilia A and utility assumptions from a clinician's perspective
Author(s) -
Valente Michele,
Cortesi Paolo A.,
Lassandro Giuseppe,
Mathew Prasad,
Pocoski Jennifer,
Molinari Angelo C.,
Mantovani Lorenzo G.,
Giordano Paola
Publication year - 2015
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.25543
Subject(s) - medicine , perspective (graphical) , intensive care medicine , quality of life (healthcare) , health care , medline , nursing , artificial intelligence , computer science , political science , law , economics , economic growth
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. © 2015 Wiley Periodicals, Inc.

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