TY - JOUR

T1 - Social/economic costs and health-related quality of life in patients with Duchenne muscular dystrophy in Europe

AU - BURQOL-RD Research Network

AU - Cavazza, Marianna

AU - Kodra, Yllka

AU - Armeni, Patrizio

AU - De Santis, Marta

AU - López-Bastida, Julio

AU - Linertová, Renata

AU - Oliva-Moreno, Juan

AU - Serrano-Aguilar, Pedro

AU - Posada-de-la-Paz, Manuel

AU - Taruscio, Domenica

AU - Schieppati, Arrigo

AU - Iskrov, Georgi

AU - Péntek, Márta

AU - von der Schulenburg, Johann Matthias Graf

AU - Kanavos, Panos

AU - Chevreul, Karine

AU - Persson, Ulf

AU - Fattore, Giovanni

N1 - Funding Information: Supported by the Social/Economic Burden and Health-Related Quality of Life in Patients with Rare Diseases in Europe Project, which received funding from the European Union within the framework of the Health Programme [Grant A101205]. The Executive Agency of the European Union is not responsible for any use that may be made of the information contained herein.

PY - 2016/4

Y1 - 2016/4

N2 - 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.

AB - 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.

KW - Duchenne muscular dystrophy

KW - Health-related quality of life

KW - Rare diseases

KW - Social burden

KW - Societal costs

UR - http://www.scopus.com/inward/record.url?scp=84962026588&partnerID=8YFLogxK

U2 - 10.1007/s10198-016-0782-5

DO - 10.1007/s10198-016-0782-5

M3 - Article

C2 - 27038625

AN - SCOPUS:84962026588

VL - 17

SP - 19

EP - 29

JO - European Journal of Health Economics

JF - European Journal of Health Economics

SN - 1618-7598

IS - 1

ER -