title: Haemophilia A: pharmacoeconomic review of prophylaxis treatment versus on-demand.
abstract: OBJECTIVE
  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.
authors: Department of Public Health and Infectious Diseases, Sapienza University of Rome, Piazzale Aldo Moro 5, 00185 Rome, Italy.
authors: Brigid
authors: 0000-0002-6522-9098
authors: B
authors: Unim
authors: Institute of Hygiene, Catholic University of the Sacred Heart, Largo Agostino Gemelli 8, 00168 Rome, Italy.
authors: Maria Assunta
authors: MA
authors: Veneziano
authors: Department of Public Health and Infectious Diseases, Sapienza University of Rome, Piazzale Aldo Moro 5, 00185 Rome, Italy.
authors: Antonio
authors: A
authors: Boccia
authors: Institute of Hygiene, Catholic University of the Sacred Heart, Largo Agostino Gemelli 8, 00168 Rome, Italy.
authors: Walter
authors: W
authors: Ricciardi
authors: Department of Public Health and Infectious Diseases, Sapienza University of Rome, Piazzale Aldo Moro 5, 00185 Rome, Italy.
authors: Giuseppe
authors: G
authors: La Torre
mesh_terms: D017281:Cost of Illness; D003362:Cost-Benefit Analysis; D016527:Drug Costs; D005169:Factor VIII / Q000191:economics / Q000627:therapeutic use; D017048:Health Care Costs; D006467:Hemophilia A / Q000188:drug therapy / Q000191:economics* / Q000517:prevention & control; D006801:Humans; D008297:Male
chemical_list: D005169:Factor VIII