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Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe

Author

Listed:
  • Márta Péntek

    (Corvinus University of Budapest)

  • László Gulácsi

    (Corvinus University of Budapest)

  • Valentin Brodszky

    (Corvinus University of Budapest)

  • Petra Baji

    (Corvinus University of Budapest)

  • Imre Boncz

    (Institute for Health Insurance, University of Pécs)

  • Gábor Pogány

    (Hungarian Federation of People with Rare and Congenital Diseases (RIROSZ))

  • Julio López-Bastida

    (University of Castilla-La Mancha
    Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC))

  • Renata Linertová

    (Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)
    Fundación Canaria de Investigación Sanitaria (FUNCANIS))

  • Juan Oliva-Moreno

    (Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)
    University of Castilla-La Mancha)

  • Pedro Serrano-Aguilar

    (Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)
    Evaluation and Planning Service at Canary Islands Health Service)

  • Manuel Posada-de-la-Paz

    (Institute of Rare Diseases Research, ISCIII, SpainRDR & CIBERER)

  • Domenica Taruscio

    (National Centre for Rare Diseases, Istituto Superiore di Sanità (ISS))

  • Georgi Iskrov

    (Institute of Rare Diseases
    Medical University of Plovdiv)

  • Arrigo Schieppati

    (Centro di Ricerche Cliniche per Malattie Rare Aldo e Cele Daccò, Istituto di Ricerche Farmacologiche Mario Negri)

  • Johann Matthias Graf Schulenburg

    (Leibniz Universität Hannover)

  • Panos Kanavos

    (London School of Economics and Political Science)

  • Karine Chevreul

    (URC Eco Ile de France, AP-HP, Hôtel Dieu
    Université Paris Diderot, Sorbonne Paris Cité, ECEVE, UMRS 1123
    INSERM, ECEVE, U1123)

  • Ulf Persson

    (The Swedish Institute for Health Economics)

  • Giovanni Fattore

    (Bocconi University)

Abstract

Objectives To assess the health-related quality of life (HRQOL) of patients with mucopolysaccharidosis (MPS) and their caregivers and to quantify the disease-related costs from a societal perspective. Methods In the context of a multi-country study of rare diseases (BURQOL-RD project), a cross-sectional survey was performed among MPS patients in seven European countries. Data on demographic characteristics, health resource utilization, informal care, and loss of labor productivity were collected. The EQ-5D, Barthel index (BI), and Zarit burden interview (ZBI) questionnaires were used to assess patients’ and their informal caregivers’ quality of life, patients’ functional ability, and caregivers’ burden, respectively. Results Altogether, 120 patients (children 62 %, females 40 %) and 66 caregivers completed the questionnaire. Patients’ mean age was 16.5 years and median age at diagnosis was 3 years. Adult patients’ average EQ-5D and EQ VAS scores varied across countries from 0.13 to 0.43 and 30.0 to 62.2, respectively, mean BI was 46.7, and ZBI was 32.7. Mean informal care time was 51.3 h/week. The mean total annual cost per patient (reference year 2012) was €24,520 in Hungary, €25,993 in France, €84,921 in Italy, €94,384 in Spain, and €209,420 in Germany. Costs are also shown to differ between children and adults. Direct costs accounted for most of the costs in all five countries (80, 100, 99, 98, and 93 %, respectively). Conclusions MPS patients experience substantial loss of HRQOL and their families take a remarkable part in their care. Although utilization of health and social care resources varies significantly across countries, MPS incurs considerable societal costs in all the countries studied.

Suggested Citation

  • Márta Péntek & László Gulácsi & Valentin Brodszky & Petra Baji & Imre Boncz & Gábor Pogány & Julio López-Bastida & Renata Linertová & Juan Oliva-Moreno & Pedro Serrano-Aguilar & Manuel Posada-de-la-Pa, 2016. "Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 17(1), pages 89-98, April.
  • Handle: RePEc:spr:eujhec:v:17:y:2016:i:1:d:10.1007_s10198-016-0787-0
    DOI: 10.1007/s10198-016-0787-0
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    References listed on IDEAS

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    2. Michela Meregaglia & Elena Nicod & Michael Drummond, 2023. "The estimation of health state utility values in rare diseases: do the approaches in submissions for NICE technology appraisals reflect the existing literature? A scoping review," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 24(7), pages 1151-1216, September.
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    4. Petra Baji & Dominik Golicki & Valentina Prevolnik-Rupel & Werner B. F. Brouwer & Zsombor Zrubka & László Gulácsi & Márta Péntek, 2019. "The burden of informal caregiving in Hungary, Poland and Slovenia: results from national representative surveys," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 20(1), pages 5-16, June.

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    More about this item

    Keywords

    Mucopolysaccharidosis; Health-related quality of life; Cost-of-illness; Caregiver; EQ-5D;
    All these keywords.

    JEL classification:

    • I19 - Health, Education, and Welfare - - Health - - - Other

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