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Social/economic costs and health-related quality of life in patients with cystic fibrosis in Europe

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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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References

  1. Orphanet: cystic fibrosis. http://www.orpha.net/

  2. Prévalence des maladies rares, données bibliographiques. Classement par ordre alphabétique des maladies ou groupes de maladies (2013) http://www.myobase.org/doc_num.php?explnum_id=9206

  3. Association française pour le dépistage et la prévention des handicaps de l’enfant: Bilan d’activité 2012 (2013) http://www.afdphe.org/sites/default/files/bilan_activite_2012.pdf

  4. Bradley, J.M., Blume, S.W., Balp, M.-M., Honeybourne, D., Elborn, J.S.: Quality of life and healthcare utilisation in cystic fibrosis: a multicentre study. Eur. Respir. J. 41, 571–577 (2013)

    Article  PubMed  Google Scholar 

  5. Abbott, J., Hurley, M.A., Morton, A.M., Conway, S.P.: Longitudinal association between lung function and health-related quality of life in cystic fibrosis. Thorax 68, 149–154 (2013)

    Article  PubMed  Google Scholar 

  6. Hegarty, M., Macdonald, J., Watter, P., Wilson, C.: Quality of life in young people with cystic fibrosis: effects of hospitalization, age and gender, and differences in parent/child perceptions. Child Care Health Dev. 35, 462–468 (2009)

    Article  CAS  PubMed  Google Scholar 

  7. European Commission—rare diseases. http://ec.europa.eu/health/rare_diseases/policy/index_en.htm

  8. Dewitt, E.M., Grussemeyer, C.A., Friedman, J.Y., Dinan, M.A., Lin, L., Schulman, K.A., Reed, S.D.: Resource use, costs, and utility estimates for patients with cystic fibrosis with mild impairment in lung function: analysis of data collected alongside a 48-week multicenter clinical trial. Value Health J. Int. Soc. Pharmacoecon. Outcomes Res. 15, 277–283 (2012)

    Article  Google Scholar 

  9. Heimeshoff, M., Hollmeyer, H., Schreyögg, J., Tiemann, O., Staab, D.: Cost of illness of cystic fibrosis in Germany: results from a large cystic fibrosis centre. PharmacoEconomics. 30, 763–777 (2012)

    Article  PubMed  Google Scholar 

  10. Van Gool, K., Norman, R., Delatycki, M.B., Hall, J., Massie, J.: Understanding the costs of care for cystic fibrosis: an analysis by age and health state. Value Health J. Int. Soc. Pharmacoecon. Outcomes Res. 16, 345–355 (2013)

    Article  Google Scholar 

  11. Mahoney, F., Barthel, D.: Functional evaluation: the Barthel Index. Md. State Med. J. 14, 61–65 (1965)

    CAS  PubMed  Google Scholar 

  12. Collin, C., Wade, D.T., Davies, S., Horne, V.: The Barthel ADL Index: a reliability study. Int. Disabil. Stud. 10, 61–63 (1988)

    Article  CAS  PubMed  Google Scholar 

  13. Hérbert, R., Bravo, G., Préville, M.: Reliability, validity, and reference values of the Zarit Burden Interview for assessing informal caregivers of community-dwelling older persons with dementia. Can. J. Aging 19, 494–507 (2000)

  14. Van den Berg, B., Brouwer, W.B.F., Koopmanschap, M.A.: Economic valuation of informal care. An overview of methods and applications. Eur. J. Health Econ. HEPAC Health Econ. Prev. Care. 5, 36–45 (2004)

    Article  Google Scholar 

  15. McDaid, D.: Estimating the costs of informal care for people with Alzheimer’s disease: methodological and practical challenges. Int. J. Geriatr. Psychiatry 16, 400–405 (2001)

    Article  CAS  PubMed  Google Scholar 

  16. Hodgson, T., Meiners, M.: Cost-of-illness methodology: a guide to assessment practices and procedures. Milbank Memorial Fund Q./Health Soc. 60, 429–462 (1982)

  17. Herdman, M., Gudex, C., Lloyd, A., Janssen, M., Kind, P., Parkin, D., Bonsel, G., Badia, X.: Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). Qual. Life Res. Int. J. Qual. Life Asp. Treat. Care Rehabil. 20, 1727–1736 (2011)

    Article  CAS  Google Scholar 

  18. Tarricone, R.: Cost-of-illness analysis. What room in health economics? Health Policy Amst. Neth. 77, 51–63 (2006)

    Article  Google Scholar 

  19. The Campbell and Cochrane Economics Methods Group, The Evidence for Policy and Practice Information and Coordinating Centre, CCEMG—EPPI-Centre Cost Converter v.1.2. http://eppi.ioe.ac.uk/costconversion/default.aspx

  20. Horvais, V., Touzet, S., François, S., Bourdy, S., Bellon, G., Colin, C., Durieu, I.: Cost of home and hospital care for patients with cystic fibrosis followed up in two reference medical centers in France. Int. J. Technol. Assess. Health Care 22, 525–531 (2006)

    Article  PubMed  Google Scholar 

  21. Huot, L., Durieu, I., Bourdy, S., Ganne, C., Bellon, G., Colin, C., Touzet, S., REMU study: Evolution of costs of care for cystic fibrosis patients after clinical guidelines implementation in a French network. J. Cyst. Fibros. Off. J. Eur. Cyst. Fibros. Soc. 7, 403–408 (2008)

    Article  Google Scholar 

  22. Briesacher, B.A., Quittner, A.L., Fouayzi, H., Zhang, J., Swensen, A.: Nationwide trends in the medical care costs of privately insured patients with cystic fibrosis (CF), 2001–2007. Pediatr. Pulmonol. 46, 770–776 (2011)

    Article  PubMed  Google Scholar 

  23. Assurance Maladie: Prévalence des ALD au 31/12/2009. http://www.ameli.fr/l-assurance-maladie/statistiques-et-publications/donnees-statistiques/affection-de-longue-duree-ald/prevalence/frequence-des-ald-au-31-12-2009.php

  24. Assurance Maladie: Coût des ALD en 2009 dans la population du régime général. http://www.ameli.fr/l-assurance-maladie/statistiques-et-publications/donnees-statistiques/affection-de-longue-duree-ald/cout/cout-des-ald-en-2009.php

  25. Eidt-Koch, D., Wagner, T.O.F., Mittendorf, T., Graf von der Schulenburg, J.-M.: Outpatient medication costs of patients with cystic fibrosis in Germany. Appl. Health Econ. Health Policy 8, 111–118 (2010)

    Article  PubMed  Google Scholar 

  26. Stolk, E.A., Busschbach, J.J., Vogels, T.: Performance of the EuroQol in children with imperforate anus. Qual. Life Res. Int. J. Qual. Life Asp. Treat. Care Rehabil. 9, 29–38 (2000)

    Article  CAS  Google Scholar 

  27. Polinder, S., Meerding, W.J., Toet, H., Mulder, S., Essink-Bot, M.-L., van Beeck, E.F.: Prevalence and prognostic factors of disability after childhood injury. Pediatrics 116, e810–e817 (2005)

    Article  PubMed  Google Scholar 

  28. Willems, D.C.M., Joore, M.A., Nieman, F.H.M., Severens, J.L., Wouters, E.F.M., Hendriks, J.J.E.: Using EQ-5D in children with asthma, rheumatic disorders, diabetes, and speech/language and/or hearing disorders. Int. J. Technol. Assess. Health Care 25, 391–399 (2009)

    Article  PubMed  Google Scholar 

  29. Willems, D.C.M., Joore, M.A., Hendriks, J.J.E., Wouters, E.F.M., Severens, J.L.: Cost-effectiveness of a nurse-led telemonitoring intervention based on peak expiratory flow measurements in asthmatics: results of a randomised controlled trial. Cost Eff. Resour. Alloc. CE. 5, 10 (2007)

    Article  Google Scholar 

  30. Secnik, K., Matza, L.S., Cottrell, S., Edgell, E., Tilden, D., Mannix, S.: Health state utilities for childhood attention-deficit/hyperactivity disorder based on parent preferences in the United Kingdom. Med. Decis. Mak. Int. J. Soc. Med. Decis. Mak. 25, 56–70 (2005)

    Article  Google Scholar 

  31. Matza, L.S., Secnik, K., Mannix, S., Sallee, F.R.: Parent-proxy EQ-5D ratings of children with attention-deficit hyperactivity disorder in the US and the UK. PharmacoEconomics. 23, 777–790 (2005)

    Article  PubMed  Google Scholar 

  32. The World Bank: GDP per capita (current US$). http://data.worldbank.org/indicator/NY.GDP.PCAP.CD

Download references

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.

Author information

Authors and Affiliations

  1. URC Eco Ile de France, AP-HP, Hôtel Dieu, Paris, France

    Karine Chevreul, Morgane Michel & Karen Berg Brigham

  2. Université Paris Diderot, Sorbonne Paris Cité, ECEVE, UMRS 1123, Paris, France

    Karine Chevreul & Morgane Michel

  3. INSERM, ECEVE, U1123, Paris, France

    Karine Chevreul & Morgane Michel

  4. Université Paris Est, Créteil, France

    Karen Berg Brigham

  5. University of Castilla-La Mancha, Talavera de la Reina, Toledo, Spain

    Julio López-Bastida

  6. Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain

    Julio López-Bastida, Renata Linertová, Juan Oliva-Moreno & Pedro Serrano-Aguilar

  7. Fundación Canaria de Investigación Sanitaria (FUNCANIS), Canary Islands, Spain

    Renata Linertová

  8. University of Castilla-La Mancha, Toledo, Spain

    Juan Oliva-Moreno

  9. Evaluation and Planning Service at Canary Islands Health Service, Santa Cruz de Tenerife, Spain

    Pedro Serrano-Aguilar

  10. Institute of Rare Diseases Research, ISCIII, SpainRDR and CIBERER, Madrid, Spain

    Manuel Posada-de-la-Paz

  11. National Center for Rare Diseases, Istituto superiore di sanita (ISS), Rome, Italy

    Domenica Taruscio

  12. Centro di Ricerche Cliniche per Malattie Rare Aldo e Cele Daccò, Istituto di Ricerche Farmacologiche Mario Negri, Ranica (Bergamo), Italy

    Arrigo Schieppati

  13. Institute of Rare Diseases, Plovdiv, Bulgaria

    Georgi Iskrov

  14. Department of Social Medicine and Public Health, Faculty of Public Health, Medical University of Plovdiv, Plovdiv, Bulgaria

    Georgi Iskrov

  15. Department of Health Economics, Corvinus University of Budapest, Budapest, Hungary

    Márta Péntek

  16. Centre for Health Economics Research Hannover (CHERH), Leibniz Universität Hannover, Hannover, Germany

    Johann Matthias Graf von der Schulenburg

  17. Department of Social Policy and LSE Health, London School of Economics and Political Science, London, UK

    Panos Kanavos

  18. The Swedish Institute for Health Economics, Lund, Sweden

    Ulf Persson

  19. Centro di Ricerche sulla Gestione dell’Assistenza Sanitaria e Sociale (CERGAS), Università Commerciale Luigi Bocconi, Milan, Italy

    Giovani Fattore

Authors
  1. Karine Chevreul
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  2. Morgane Michel
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  8. Manuel Posada-de-la-Paz
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  13. Johann Matthias Graf von der Schulenburg
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  15. Ulf Persson
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Consortia

BURQOL-RD Research Network

Corresponding author

Correspondence to Karine Chevreul.

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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.

Additional information

Members of the BURQOL-RD Research Network are listed in the Appendix.

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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

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