Science and Research

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

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 euro7657 in Hungary to euro58,704 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.

  • Cavazza, M.; Kodra, Y.; Armeni, P.; De Santis, M.; Lopez-Bastida, J.; Linertova, R.; Oliva-Moreno, J.; Serrano-Aguilar, P.; Posada-de-la-Paz, M.; Taruscio, D.; Schieppati, A.; Iskrov, G.; Pentek, M.; von der Schulenburg, J. M.; Kanavos, P.; Chevreul, K.; Persson, U.; Fattore, G.; Burqol-Rd Research Network

Keywords

  • Adolescent
  • Adult
  • Caregivers/statistics & numerical data
  • Child
  • Child, Preschool
  • *Cost of Illness
  • Cross-Sectional Studies
  • Europe
  • Female
  • *Health Care Costs/statistics & numerical data
  • Humans
  • Male
  • Muscular Dystrophy, Duchenne/*economics/psychology
  • Patient Care/economics
  • *Quality of Life
  • Sick Leave/economics
  • Sickness Impact Profile
  • Socioeconomic Factors
  • Surveys and Questionnaires
  • United Kingdom
  • Young Adult
  • *Duchenne muscular dystrophy
  • *Health-related quality of life
  • *Rare diseases
  • *Social burden
  • *Societal costs
Publication details
DOI: 10.1007/s10198-016-0782-5
Journal: The European journal of health economics : HEPAC : health economics in prevention and care
Pages: 19-29 
Work Type: Original
Location: BREATH
Disease Area: COPD
Partner / Member: LUH
Access-Number: 27038625
See publication on PubMed

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