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Social/economic costs and quality of life in patients with haemophilia in Europe

  • Marianna CavazzaEmail author
  • Yllka Kodra
  • Patrizio Armeni
  • Marta De Santis
  • Julio López-Bastida
  • Renata Linertová
  • Juan Oliva-Moreno
  • Pedro Serrano-Aguilar
  • Manuel Posada-de-la-Paz
  • Domenica Taruscio
  • Arrigo Schieppati
  • Georgi Iskrov
  • László Gulácsi
  • Johann Matthias Graf von der Schulenburg
  • Panos Kanavos
  • Karine Chevreul
  • Ulf Persson
  • Giovanni Fattore
  • BURQOL-RD Research Network
Original Paper

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.

Conclusion

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.

Keywords

Rare diseases Haemophilia Health related quality of life Societal costs Social burden 

Notes

Acknowledgments

The authors wish to thank: National Alliance of People with Rare Diseases (NAPRD), Bulgaria; Alliance Maladies Rares, France; ACHSE, Germany; Hungarian Federation of People with Rare and Congenital Diseases (RIROSZ), Hungary; Federazione Italiana Malattie Rare (UNIAMO), Italy; the Consulta Nazionale delle Malattie Rare, Italy; Rare Diseases Sweden; Federación Española de Efermedades Raras (FEDER), Spain; Rare Disease UK and Rare Diseases Europe (EURORDIS); Bulgarian Haemophilia Society; Association française des hémophiles, France; Interessengemeinschaft Hämophiler e.V., Germany; Magyar Hemofília Egyesület, Hungary; FEDEMO—Federazione delle Associazioni Emofilici ONLUS and A.E.L.—Associazione emofilici del Lazio, Italy; Swedish Hemophilia Society, Sweden; Asociación Andaluza de Hemofilia Canf Cocemfe and Asociaciónde Hemofilia de la Comunidad de Madrid, Spain; Haemophilia Society, UK.

Compliance with ethical standards

Funding

Supported by the Social/Economic Burden and Health-Related Quality of Life in Patients with Rare Diseases in Europe Project, which received funding from the European Union within the framework of the Health Programme (grant A101205). The Executive Agency of the European Union is not responsible for any use that may be made of the information contained herein.

Conflict of interest

The authors declare that they have no conflicts of interest.

Supplementary material

10198_2016_785_MOESM1_ESM.docx (16 kb)
Supplementary material 1 (DOCX 16 kb)
10198_2016_785_MOESM2_ESM.doc (41 kb)
Supplementary material 2 (DOC 41 kb)

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Copyright information

© Springer-Verlag Berlin Heidelberg 2016

Authors and Affiliations

  • Marianna Cavazza
    • 1
    Email author
  • Yllka Kodra
    • 2
  • Patrizio Armeni
    • 1
  • Marta De Santis
    • 2
  • Julio López-Bastida
    • 3
    • 4
  • Renata Linertová
    • 4
    • 5
  • Juan Oliva-Moreno
    • 4
    • 6
  • Pedro Serrano-Aguilar
    • 4
    • 7
  • Manuel Posada-de-la-Paz
    • 8
  • Domenica Taruscio
    • 2
  • Arrigo Schieppati
    • 9
  • Georgi Iskrov
    • 10
    • 16
  • László Gulácsi
    • 11
  • Johann Matthias Graf von der Schulenburg
    • 12
  • Panos Kanavos
    • 13
  • Karine Chevreul
    • 14
    • 17
    • 18
  • Ulf Persson
    • 15
  • Giovanni Fattore
    • 1
  • BURQOL-RD Research Network
  1. 1.Centre for Research on Health and Social Care Management (CERGAS)Bocconi UniversityMilanItaly
  2. 2.National Centre for Rare Diseases, Istituto Superiore di Sanità (ISS)RomeItaly
  3. 3.University of Castilla-La ManchaTalavera de la ReinaSpain
  4. 4.Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)MadridSpain
  5. 5.Fundación Canaria de Investigación Sanitaria (FUNCANIS)Las Palmas de Gran CanariaSpain
  6. 6.University of Castilla-La ManchaToledoSpain
  7. 7.Evaluation and Planning Service at Canary Islands Health ServiceSanta Cruz de TenerifeSpain
  8. 8.Institute of Rare Diseases Research, ISCIII, SpainRDR & CIBERERMadridSpain
  9. 9.“Aldo and Cele Daccò” Clinical Research Center for Rare Diseases, Mario Negri Institute for Pharmacological ResearchRanica (Bergamo)Italy
  10. 10.Institute of Rare DiseasesPlovdivBulgaria
  11. 11.Department of Health EconomicsCorvinus University of BudapestBudapestHungary
  12. 12.Centre for Health Economics Research Hannover (CHERH)Leibniz Universität HannoverHanoverGermany
  13. 13.Department of Social Policy and LSE HealthLondon School of Economics and Political ScienceLondonUK
  14. 14.URC Eco Ile de France, AP-HPParisFrance
  15. 15.The Swedish Institute for Health EconomicsLundSweden
  16. 16.Department of Social Medicine and Public Health, Faculty of Public HealthMedical University of Plovdiv PlovdivBulgaria
  17. 17.Université Paris Diderot ParisFrance
  18. 18.INSERM, ECEVE ParisFrance

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