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

authored by

BURQOL-RD Research Network , 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, Claudia Delgado, Johann Matthias Graf von der Schulenburg, Ulf Persson, Karine Chevreul, Giovanni Fattore

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.

Organisation(s)

Institute of Insurance Business Administration

External Organisation(s)

Universidad de Castilla-La Mancha
Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)
Canary Islands Foundation for Health Research (FUNCANIS)
Institute of Rare Diseases Research (IIER)
Canary Islands Health Service
London School of Economics and Political Science
Istituto Superiore di Sanita
Institute of Pharmacological Research Mario Negri IRCCS
Institute of Rare Diseases
Medical University of Plovdiv
Corvinus University of Budapest
Federación Española de Enfermedades Raras (FEDER)
The Swedish Institute for Health Economics
AP-HP Assistance Publique - Hopitaux de Paris
Institut national de la santé et de la recherche médicale (INSERM)
Università Commerciale Luigi Bocconi
Universite Paris 7

Type

Article

Journal

European Journal of Health Economics

Volume

17

Pages

99-108

No. of pages

10

ISSN

1618-7598

Publication date

04.2016

Publication status

Published

Peer reviewed

Yes

ASJC Scopus subject areas

Economics, Econometrics and Finance (miscellaneous), Health Policy

Sustainable Development Goals

SDG 3 - Good Health and Well-being

Electronic version(s)

https://doi.org/10.1007/s10198-016-0788-z (Access: Closed)