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

authored by

BURQOL-RD Research Network , Marianna Cavazza, Yllka Kodra, Patrizio Armeni, Marta De Santis, Julio López-Bastida, Renata Linertová, Juan Oliva-Moreno, Pedro Serrano-Aguilar, Manuel Posada-de-la-Paz, Domenica Taruscio, Arrigo Schieppati, Georgi Iskrov, Márta Péntek, Johann Matthias Graf von der Schulenburg, Panos Kanavos, Karine Chevreul, Ulf Persson, Giovanni Fattore

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.

Organisation(s)

Center for Health Economics Research Hannover (CHERH)

External Organisation(s)

Università Commerciale Luigi Bocconi
Istituto Superiore di Sanita
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
Institute of Health Carlos III (ISCIII)
Institute of Pharmacological Research Mario Negri IRCCS
Institute of Rare Diseases
Medical University of Plovdiv
Corvinus University of Budapest
London School of Economics and Political Science
AP-HP Assistance Publique - Hopitaux de Paris
Institut national de la santé et de la recherche médicale (INSERM)
The Swedish Institute for Health Economics
Universite Paris 7

Type

Article

Journal

European Journal of Health Economics

Volume

17

Pages

19-29

No. of pages

11

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/s10198-016-0782-5 (Access: Closed)