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

Marianna Cavazza (), Yllka Kodra, Patrizio Armeni, Marta Santis, Julio López-Bastida, Renata Linertová, Juan Oliva (), Pedro Serrano-Aguilar, Manuel Posada- de-la-Paz, Domenica Taruscio, Arrigo Schieppati, Georgi Iskrov, Márta Péntek, Johann Matthias Graf Schulenburg, Panos Kanavos, Karine Chevreul, Ulf Persson and Giovanni Fattore
Additional contact information
Marianna Cavazza: Bocconi University
Yllka Kodra: Istituto Superiore di Sanità (ISS)
Patrizio Armeni: Bocconi University
Marta Santis: Istituto Superiore di Sanità (ISS)
Julio López-Bastida: University of Castilla-La Mancha
Renata Linertová: Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)
Pedro Serrano-Aguilar: Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)
Manuel Posada- de-la-Paz: Institute of Rare Diseases Research, ISCIII, SpainRDR and CIBERER
Domenica Taruscio: Istituto Superiore di Sanità (ISS)
Arrigo Schieppati: Mario Negri Institute for Pharmacological Research
Georgi Iskrov: Institute of Rare Diseases
Johann Matthias Graf Schulenburg: Leibniz Universität Hannover
Panos Kanavos: London School of Economics and Political Science
Karine Chevreul: URC Eco Ile de France, AP-HP
Ulf Persson: The Swedish Institute for Health Economics
Giovanni Fattore: Bocconi University

The European Journal of Health Economics, 2016, vol. 17, issue 1, No 3, 19-29

Abstract: 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.

Keywords: Rare diseases; Duchenne muscular dystrophy; Health-related quality of life; Societal costs; Social burden; I140 (search for similar items in EconPapers)
Date: 2016
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Citations: View citations in EconPapers (17)

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DOI: 10.1007/s10198-016-0782-5

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