Abstract
Objectives
Our goal was to provide data on the economic burden and health-related quality of life (HRQOL) of patients with cystic fibrosis (CF) and their caregivers in Europe.
Methods
A cross-sectional study was carried out on adults and children with CF in eight European countries. Patients completed an anonymous questionnaire regarding their socio-demographic characteristics, use of healthcare services and presence of a caregiver. Costs were calculated with a bottom-up approach using unit costs from each participating country, and HRQOL was assessed using EQ-5D. The principal caregiver also answered a questionnaire on their characteristics, HRQOL and burden.
Results
A total of 905 patients with CF was included (399 adults and 506 children). The total average annual cost per patient varied from €21,144 in Bulgaria to €53,256 in Germany. Adults had higher direct healthcare costs than children, but children had much higher informal care costs (P < 0.0001). Total costs increased with patients’ level of dependence. In adults, mean utility fell between 0.640 and 0.870, and the visual analogue scale ranged from 46.0 to 69.7. There was no difference in caregiver HRQOL regardless of whether they cared for an adult or a child. However, caregivers who looked after a child had a significantly higher burden (P = 0.0013).
Conclusions
Our study highlights the burden of CF in terms of costs and decreased HRQOL for both patients and their caregivers throughout Europe.
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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); Cystic Fibrosis Bulgaria; Centre de Référence Maladies Rares pour la Mucoviscidose de Nantes-Roscoff, France; Mukoviszidose e.V. Bundesverband Cystische Fibrose, Germany; Országos Cisztás Fibrózis Egyesület, Hungary; L.I.F.C. (Lega Italiana Fibrosi cística), Italy; Federación Española de Fibrosis Quística and Asociación Madrileña, Catalana, Extrmeña y Andaluza contra la Fibrosis Quística, Spain; Cystic Fibrosis Trust, UK.
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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.
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The authors declare that they have no conflicts of interest.
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Chevreul, K., Michel, M., Brigham, K.B. et al. Social/economic costs and health-related quality of life in patients with cystic fibrosis in Europe. Eur J Health Econ 17 (Suppl 1), 7–18 (2016). https://doi.org/10.1007/s10198-016-0781-6
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DOI: https://doi.org/10.1007/s10198-016-0781-6