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Social/economic costs and health-related quality of life in patients with Prader-Willi syndrome in Europe

Author

Listed:
  • 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)
    Canary Islands Foundation for Health Research (FUNCANIS))

  • Juan Oliva-Moreno

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

  • Manuel Posada-de-la-Paz

    (Spain RDR and CIBERER)

  • Pedro Serrano-Aguilar

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

  • Panos Kanavos

    (London School of Economics and Political Science-Health Research Centre)

  • Domenica Taruscio

    (Istituto superiore di sanità (ISS))

  • Arrigo Schieppati

    (Istituto di Ricerche Farmacologiche Mario Negri)

  • Georgi Iskrov

    (Institute of Rare Diseases
    Medical University of Plovdiv)

  • Petra Baji

    (Corvinus University of Budapest)

  • Claudia Delgado

    (Federación Española de Enfermedades Raras (FEDER))

  • Johann Matthias Graf Schulenburg

    (Leibniz University)

  • Ulf Persson

    (The Swedish Institute for Health Economics)

  • Karine Chevreul

    (AP-HP
    Université Paris Diderot, Sorbonne Paris Cité, ECEVE, UMRS 1123
    INSERM, ECEVE, U1123)

  • Giovanni Fattore

    (Bocconi University)

Abstract

Objective The aim of this study was to determine the economic burden from a societal perspective and health-related quality of life (HRQOL) of patients with Prader-Willi syndrome (PWS) in Europe. Methods We conducted a cross-sectional study of patients with PWS from Spain, Bulgaria, Hungary, Germany, Italy, the UK, Sweden and France. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. Results A total of 261 patients completed the questionnaire. The average annual costs ranged from € 3937 to € 67,484 between countries; the reference year for unit prices was 2012. Direct healthcare costs ranged from € 311 to € 18,760, direct non-healthcare costs ranged from € 1269 to € 44,035, and loss of labour productivity ranged from € 0 to € 2255. Costs were also shown to differ between children and adults. The mean EQ-5D index score for adult PWS patients ranged between 0.40 and 0.81 and the mean EQ-5D visual analogue scale score ranged between 51.25 and 90.00. Conclusion The main strengths of this study lie in our bottom-up approach to costing and in the evaluation of PWS patients from a broad societal perspective. This type of analysis is very scarce in the international literature on rare diseases in comparison with other illnesses. We conclude that PWS patients incur considerable societal costs and experience substantial deterioration in HRQOL.

Suggested Citation

  • Julio López-Bastida & Renata Linertová & Juan Oliva-Moreno & Manuel Posada-de-la-Paz & Pedro Serrano-Aguilar & Panos Kanavos & Domenica Taruscio & Arrigo Schieppati & Georgi Iskrov & Petra Baji & Clau, 2016. "Social/economic costs and health-related quality of life in patients with Prader-Willi syndrome in Europe," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 17(1), pages 99-108, April.
    • Handle: RePEc:spr:eujhec:v:17:y:2016:i:1:d:10.1007_s10198-016-0788-z
    DOI: 10.1007/s10198-016-0788-z
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    References listed on IDEAS

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    13. Patrizio Armeni & Marianna Cavazza & Entela Xoxi & Domenica Taruscio & Yllka Kodra, 2021. "Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal," IJERPH, MDPI, vol. 18(3), pages 1-18, January.
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