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

authored by

BURQOL-RD Research Network , 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-Paz, Domenica Taruscio, Georgi Iskrov, Arrigo Schieppati, Johann Matthias Graf von der Schulenburg, Panos Kanavos, Karine Chevreul, Ulf Persson, Giovanni Fattore

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.

Organisation(s)

Center for Health Economics Research Hannover (CHERH)

External Organisation(s)

Corvinus University of Budapest
University of Pecs
Hungarian Federation of People with Rare and Congenital Diseases (RIROSZ)
Universidad de Castilla-La Mancha
Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)
Canary Islands Foundation for Health Research (FUNCANIS)
Canary Islands Health Service
Istituto Superiore di Sanita
Institute of Rare Diseases
Medical University of Plovdiv
Institute of Pharmacological Research Mario Negri IRCCS
London School of Economics and Political Science
Hopital Hotel-Dieu AP-HP
Institut national de la santé et de la recherche médicale (INSERM)
The Swedish Institute for Health Economics
Università Commerciale Luigi Bocconi
Universite Paris 7
Institute of Health Carlos III (ISCIII)

Type

Article

Journal

European Journal of Health Economics

Volume

17

Pages

89-98

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/s10198-016-0787-0 (Access: Closed)