title: Health economic models in hemophilia A and utility assumptions from a clinician's perspective.
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.
  
  
  METHODS
  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.
authors.affiliation: Bocconi University, Milano, Italy.|Bayer HEOR Intern, Whippany, New Jersey.
authors.forename: Michele
authors.initials: M
authors.lastname: Valente
authors.affiliation: Research Centre on Public Health (CESP), University of Milan-Bicocca, Monza, Italy.
authors.forename: Paolo A
authors.initials: PA
authors.lastname: Cortesi
authors.affiliation: Department of Biomedical Science and Human Oncology-Pediatric Unit, University of Bari "Aldo Moro,", Bari, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Lassandro
authors.affiliation: Bayer HealthCare Pharmaceuticals, Whippany, New Jersey.|University of New Mexico, Albuquerque, New Mexico.
authors.forename: Prasad
authors.initials: P
authors.lastname: Mathew
authors.affiliation: Bayer HealthCare Pharmaceuticals, Whippany, New Jersey.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Pocoski
authors.affiliation: Department of Hematology and Oncology, Thrombosis and Hemostasis Unit, Giannina Gaslini Children's Hospital, Genoa, Italy.
authors.forename: Angelo C
authors.initials: AC
authors.lastname: Molinari
authors.affiliation: Research Centre on Public Health (CESP), University of Milan-Bicocca, Monza, Italy.
authors.forename: Lorenzo G
authors.initials: LG
authors.lastname: Mantovani
authors.affiliation: Department of Biomedical Science and Human Oncology-Pediatric Unit, University of Bari "Aldo Moro,", Bari, Italy.
authors.forename: Paola
authors.initials: P
authors.lastname: Giordano
mesh_terms: D003362:Cost-Benefit Analysis; D006467:Hemophilia A / Q000188:drug therapy* / Q000191:economics*; D006470:Hemorrhage / Q000209:etiology / Q000517:prevention & control; D006801:Humans; D018803:Models, Economic; D011785:Quality Assurance, Health Care / Q000592:standards
keywords: Cost Utility Analysis (CUA); Health Technology Assessment (HTA); Health-related quality of life (HRQoL); Hemophilia; Prophylaxis; Quality-adjusted life years (QALY)