title: Social/economic costs and quality of life in patients with haemophilia in Europe.
abstract: OBJECTIVE
  The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with haemophilia in Europe.
  
  
  METHODS
  We conducted a cross-sectional study of patients with haemophilia from Bulgaria, France, Germany, Hungary, Italy, Spain Sweden and the UK. Data on demographic characteristics, health resource utilisation, informal care, loss of labour productivity and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. The costs have been estimated from a societal perspective adopting a bottom-up approach.
  
  
  RESULTS
  A total of 401 questionnaires were included in the study, of which 339 were collected from patients with haemophilia and 62 from caregivers. The lowest average annual cost per person was reported in Bulgaria (€6,660) and the highest in Germany (€194,490). Our results demonstrate both a large difference from country to country in the average annual cost per patient in 2012 and the driving role of drugs in costs. Drugs represent nearly 90 % of direct healthcare costs in a majority of the countries analysed (Hungary, Italy, Spain and Germany). In Bulgaria, France and Sweden, however, healthcare services (visits, tests and hospitalisations) prevail. Costs are also shown to differ between children and adults. The mean EQ-5D index score for adult patients was 0.69 and mean EQ-5D VAS was 66.6. The mean EQ-5D index score for carers was 0.87 and mean EQ-5D VAS was 75.5. In the disability score, 60 % showed no disability and measuring caregiver burden with the Zarit Index produced an overall mean score of 25.3.
  
  
  CONCLUSIONS
  We have shown that haemophilia is associated with a substantial economic burden and impaired HRQOL. Studies on cost of illness and HRQOL are important for haemophilia as the future of this disease is likely to change with the development of new innovative treatments. The introduction of these treatments will most likely impact future costs related to haemophilia.
authors.affiliation: Centre for Research on Health and Social Care Management (CERGAS), Bocconi University, Via Roentgen 1, 20136, Milan, Italy. marianna.cavazza@unibocconi.it.
authors.forename: Marianna
authors.initials: M
authors.lastname: Cavazza
authors.affiliation: National Centre for Rare Diseases, Istituto Superiore di Sanità (ISS), Rome, Italy.
authors.forename: Yllka
authors.initials: Y
authors.lastname: Kodra
authors.affiliation: Centre for Research on Health and Social Care Management (CERGAS), Bocconi University, Via Roentgen 1, 20136, Milan, Italy.
authors.forename: Patrizio
authors.initials: P
authors.lastname: Armeni
authors.affiliation: National Centre for Rare Diseases, Istituto Superiore di Sanità (ISS), Rome, Italy.
authors.forename: Marta
authors.initials: M
authors.lastname: De Santis
authors.affiliation: University of Castilla-La Mancha, Talavera de la Reina, Toledo, Spain.|Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain.
authors.forename: Julio
authors.initials: J
authors.lastname: López-Bastida
authors.affiliation: Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain.|Fundación Canaria de Investigación Sanitaria (FUNCANIS), Las Palmas de Gran Canaria, Spain.
authors.forename: Renata
authors.initials: R
authors.lastname: Linertová
authors.affiliation: Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain.|University of Castilla-La Mancha, Toledo, Spain.
authors.forename: Juan
authors.initials: J
authors.lastname: Oliva-Moreno
authors.affiliation: Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain.|Evaluation and Planning Service at Canary Islands Health Service, Santa Cruz de Tenerife, Spain.
authors.forename: Pedro
authors.initials: P
authors.lastname: Serrano-Aguilar
authors.affiliation: Institute of Rare Diseases Research, ISCIII, SpainRDR & CIBERER, Madrid, Spain.
authors.forename: Manuel
authors.initials: M
authors.lastname: Posada-de-la-Paz
authors.affiliation: National Centre for Rare Diseases, Istituto Superiore di Sanità (ISS), Rome, Italy.
authors.forename: Domenica
authors.initials: D
authors.lastname: Taruscio
authors.affiliation: "Aldo and Cele Daccò" Clinical Research Center for Rare Diseases, Mario Negri Institute for Pharmacological Research, Ranica (Bergamo), Italy.
authors.forename: Arrigo
authors.initials: A
authors.lastname: Schieppati
authors.affiliation: Institute of Rare Diseases, Plovdiv, Bulgaria.|Department of Social Medicine and Public Health, Faculty of Public Health, Medical University of Plovdiv, Plovdiv, Bulgaria.
authors.forename: Georgi
authors.initials: G
authors.lastname: Iskrov
authors.affiliation: Department of Health Economics, Corvinus University of Budapest, Budapest, Hungary.
authors.forename: László
authors.initials: L
authors.lastname: Gulácsi
authors.affiliation: Centre for Health Economics Research Hannover (CHERH), Leibniz Universität Hannover, Hanover, Germany.
authors.forename: Johann Matthias Graf
authors.initials: JM
authors.lastname: von der Schulenburg
authors.affiliation: Department of Social Policy and LSE Health, London School of Economics and Political Science, London, UK.
authors.forename: Panos
authors.initials: P
authors.lastname: Kanavos
authors.affiliation: URC Eco Ile de France, AP-HP, Paris, France.|Université Paris Diderot, Sorbonne Paris Cité, ECEVE, UMRS 1123, Paris, France.|INSERM, ECEVE, U1123, Paris, France.
authors.forename: Karine
authors.initials: K
authors.lastname: Chevreul
authors.affiliation: The Swedish Institute for Health Economics, Lund, Sweden.
authors.forename: Ulf
authors.initials: U
authors.lastname: Persson
authors.affiliation: Centre for Research on Health and Social Care Management (CERGAS), Bocconi University, Via Roentgen 1, 20136, Milan, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Fattore
mesh_terms: D000293:Adolescent; D000328:Adult; D017028:Caregivers; D017281:Cost of Illness; D003430:Cross-Sectional Studies; D005060:Europe; D005260:Female; D017048:Health Care Costs / Q000706:statistics & numerical data; D006467:Hemophilia A / Q000191:economics* / Q000523:psychology; D006801:Humans; D008297:Male; D008875:Middle Aged; D005791:Patient Care / Q000191:economics; D011788:Quality of Life; D018434:Sickness Impact Profile; D012959:Socioeconomic Factors; D011795:Surveys and Questionnaires; D006113:United Kingdom; D055815:Young Adult
keywords: Haemophilia; Health related quality of life; Rare diseases; Social burden; Societal costs