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

Abstract

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.

Duchenne muscular dystrophy and caregiver burden: a systematic review

“…In total, seven articles measured overall caregiver burden, [9][10][11][12][13][14]22 five HRQoL, [10][11][12][13][14] four stress, 14,[16][17][18] six work life, informal care, and/or associated cost burden, 11,[19][20][21][22]28 five family problems and functioning, 9,14,15,23,24 six sleep, 13,15,21,[25][26][27] five pain and/or discomfort, 12,13,21,23,24 two strain, 13,28 nine anxiety and/or depression, [12][13][14][15]23,24,[26][27][28] two overall caregiver health, 23,24 one self-esteem and mastery (i.e. that life chances are under one's own control), <...>…”

“…Across studies and countries, the utility (i.e. HRQoL as valued by the general population) of caregivers to patients with DMD has been estimated at between 0.51 and 0.87 . Possible reasons for this variability include general cultural aspects (e.g.…”

“…Concerning measures of overall burden in DMD, the mean global ZBI score has been estimated at between 15 (in France) and 37 (in Sweden), which may be compared with estimates for neuromuscular diseases in general (23), irritable bowel syndrome (22), Alzheimer's disease (29), obsessive–compulsive disorder (29), and Parkinson disease (24) . Interestingly, despite the notable variability across specific countries within studies, mean ZBI scores from pooled samples of caregivers from Cavazza et al . and Landfeldt et al .…”

“…Of the 21 included articles, 20 recorded data using surveys, [9][10][11][12][13][14][15][16][17][18][19][20][21][23][24][25][26][27][28][29] seven through interviews, 9,14,15,17,22,27,29 six using a combination of interviews and surveys, 9,14,15,17,27,29 and one using clinical examinations. 27 Moreover, as evident from Table I, a wide range of instruments were used to investigate different aspects of caregiver burden.…”

Social/economic costs and health-related quality of life in patients with rare diseases in Europe

“…DMD is a rapidly progressive form of muscular dystrophy, causing increasing loss of muscle function and weakness [25][26][27]. Cavazza et al [28] estimated an average annual cost per patient with DMD that ranged from €7657 in Hungary to €58,704 in France. Direct non-healthcare costs are the main component of costs, and informal care is its main driver.…”