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Social/economic costs and health-related quality of life in patients with Duchenne muscular dystrophy in Europe

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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 Duchenne muscular dystrophy (DMD) in Europe.

Methods

We conducted a cross-sectional study of patients with DMD from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden, and the UK. Data on demographic characteristics, healthcare resource utilization, informal care, labor productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. Costs have been estimated from a societal perspective adopting a bottom-up approach.

Results

A total of 422 questionnaires were included in the study; 268 of which were collected from patients with DMD and 154 from caregivers. The average annual cost per person in 2012 ranged from €7657 in Hungary to €58,704 in France. Direct non-healthcare costs are the main component of whole costs and informal care is the main driver of non-healthcare costs. Costs are also shown to differ between children and adults. With regard to HRQOL of adult patients, the EQ-5D VAS score and EQ-5D index scores were 50.5 and 0.24, respectively. The corresponding EQ-5D VAS and EQ-5D index scores for caregivers were 74.7 and 0.71, respectively.

Conclusions

We have estimated the average annual cost per patient with DMD in eight European countries adopting a social perspective, and to our knowledge this is the first study with such a wide perspective. The results on costs show a considerable gap between Eastern and Western European countries. Non-healthcare costs range from 64 to 89 % of overall costs and informal care is to a great extent the main driver of this cost category. The HRQOL of people with DMD is much lower than that of the general population.

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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; Rare Diseases Europe (EURORDIS); Deutsche Gesellschaft für Muskelkranke e.V., Germany; Genitori con figli affetti da distrofia muscolare di Duchenne e Becker, Italy; Misko Alapítvány, Gyógyító Jószándék Alapítvány—Izombeteg Gyermekek Alapítványa (Duchenne betegekért DMD) Hungary, RBU, Sweden; Federación Española de Enfermedades Neromusculares, Asociación de Enfermedades Neuromusculares de la Comunidad Valenciana, Asociación Aragonesa de Enfermedades Neuromusculares, Asociación de Enfermos Neuromusculares de Castilla La Mancha and Asociación Ourensana de EM, ELA, Parkinson y otras enfermedades neurodegenerativas, Spain; Muscular Dystrophy Campaign, UK.

Author information

Authors and Affiliations

  1. Centre for Research on Health and Social Care Management (CERGAS), Bocconi University, Via Roentgen 1, 20136, Milan, Italy

    Marianna Cavazza, Patrizio Armeni & Giovanni Fattore

  2. National Centre for Rare Diseases, Istituto Superiore di Sanità (ISS), Rome, Italy

    Yllka Kodra, Marta De Santis & Domenica Taruscio

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

    Julio López-Bastida

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

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

  5. Fundación Canaria de Investigación Sanitaria (FUNCANIS), Las Palmas de Gran Canaria, Spain

    Renata Linertová

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

    Juan Oliva-Moreno

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

    Pedro Serrano-Aguilar

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

    Manuel Posada-de-la-Paz

  9. Centro di Ricerche Cliniche per le Malattie Rare “Aldo e Cele Daccò”, Mario Negri Institute for Pharmacological Research, Ranica, Bergamo, Italy

    Arrigo Schieppati

  10. Institute of Rare Diseases, Plovdiv, Bulgaria

    Georgi Iskrov

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

    Márta Péntek

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

    Johann Matthias Graf von der Schulenburg

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

    Panos Kanavos

  14. URC Eco Ile de France, AP-HP, Paris, France

    Karine Chevreul

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

    Ulf Persson

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

    Georgi Iskrov

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

    Karine Chevreul

  18. INSERM, ECEVE, U1123, Paris, France

    Karine Chevreul

Authors
  1. Marianna Cavazza
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  2. Yllka Kodra
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  3. Patrizio Armeni
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  4. Marta De Santis
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  8. Pedro Serrano-Aguilar
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  11. Arrigo Schieppati
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  12. Georgi Iskrov
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  13. Márta Péntek
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  14. Johann Matthias Graf von der Schulenburg
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  15. Panos Kanavos
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  16. Karine Chevreul
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  17. Ulf Persson
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  18. Giovanni Fattore
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Consortia

BURQOL-RD Research Network

Corresponding author

Correspondence to Marianna Cavazza.

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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 listed in the Annex I of the ESM.

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Cavazza, M., Kodra, Y., Armeni, P. et al. Social/economic costs and health-related quality of life in patients with Duchenne muscular dystrophy in Europe. Eur J Health Econ 17 (Suppl 1), 19–29 (2016). https://doi.org/10.1007/s10198-016-0782-5

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  • DOI: https://doi.org/10.1007/s10198-016-0782-5

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