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The Scientific World Journal
Volume 2015, Article ID 596164, 9 pages
Review Article

Haemophilia A: Pharmacoeconomic Review of Prophylaxis Treatment versus On-Demand

1Department of Public Health and Infectious Diseases, Sapienza University of Rome, Piazzale Aldo Moro 5, 00185 Rome, Italy
2Institute of Hygiene, Catholic University of the Sacred Heart, Largo Agostino Gemelli 8, 00168 Rome, Italy

Received 8 July 2014; Accepted 6 November 2014

Academic Editor: Angelos E. Hatzakis

Copyright © 2015 Brigid Unim et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.


Objectives. Haemophilia A is a congenital disorder of coagulation that mainly affects males and causes a considerable use of resources, especially when hemophilic patients are treated with prophylaxis. The aim of the present review was to discuss and appraise the methodological aspects and results of published economic evaluations of haemophilia A treatments in the last decade. Methods. The literature search, performed by consulting four engines, covered studies published between 2002 and 2014. Full economic evaluations published in English language were identified and included in the review. A quality assessment of the studies was also carried out based on Drummond’s checklist. Results. After careful evaluations of the identified records, 5 studies were reviewed. Primary and secondary prophylaxis resulted cost-effective compared to on-demand therapy: the ICER of primary prophylaxis ranged from €40.236 to €59.315/QALY gained, while the ICER of secondary prophylaxis was €40.229/QALY gained. Furthermore, 60% were high quality and 40% were medium quality studies. Conclusions. The review underlines the cost-effectiveness of prophylaxis versus on-demand treatment and the different methodological approaches applied. Further economic evaluations are required with models that reflect the clinical reality and consumption of resources in each country.