TY - JOUR

T1 - Social/economic costs and health-related quality of life in patients with epidermolysis bullosa in Europe

AU - BURQOL-RD Research Network

AU - Angelis, Aris

AU - Kanavos, Panos

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 - Brodszky, Valentin

AU - von der Schulenburg, Johann Matthias Graf

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 has 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 here.

PY - 2016/4

Y1 - 2016/4

N2 - Background: The aim of this study was to determine the social/economic costs and health-related quality of life (HRQOL) of patients with epidermolysis bullosa (EB) in eight EU member states. Methods: We conducted a cross-sectional study of patients with EB from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden and the United Kingdom. Data on demographic characteristics, health resource utilisation, informal care, labour 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. Results: A total of 204 patients completed the questionnaire. Average annual costs varied from country to country, and ranged from €9509 to €49,233 (reference year 2012). Estimated direct healthcare costs ranged from €419 to €10,688; direct non-healthcare costs ranged from €7449 to €37,451 and labour productivity losses ranged from €0 to €7259. The average annual cost per patient across all countries was estimated at €31,390, out of which €5646 accounted for direct health costs (18.0 %), €23,483 accounted for direct non-healthcare costs (74.8 %), and €2261 accounted for indirect costs (7.2 %). Costs were shown to vary across patients with different disability but also between children and adults. The mean EQ-5D score for adult EB patients was estimated at between 0.49 and 0.71 and the mean EQ-5D visual analogue scale score was estimated at between 62 and 77. Conclusion: In addition to its negative impact on patient HRQOL, our study indicates the substantial social/economic burden of EB in Europe, attributable mostly to high direct non-healthcare costs.

AB - Background: The aim of this study was to determine the social/economic costs and health-related quality of life (HRQOL) of patients with epidermolysis bullosa (EB) in eight EU member states. Methods: We conducted a cross-sectional study of patients with EB from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden and the United Kingdom. Data on demographic characteristics, health resource utilisation, informal care, labour 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. Results: A total of 204 patients completed the questionnaire. Average annual costs varied from country to country, and ranged from €9509 to €49,233 (reference year 2012). Estimated direct healthcare costs ranged from €419 to €10,688; direct non-healthcare costs ranged from €7449 to €37,451 and labour productivity losses ranged from €0 to €7259. The average annual cost per patient across all countries was estimated at €31,390, out of which €5646 accounted for direct health costs (18.0 %), €23,483 accounted for direct non-healthcare costs (74.8 %), and €2261 accounted for indirect costs (7.2 %). Costs were shown to vary across patients with different disability but also between children and adults. The mean EQ-5D score for adult EB patients was estimated at between 0.49 and 0.71 and the mean EQ-5D visual analogue scale score was estimated at between 62 and 77. Conclusion: In addition to its negative impact on patient HRQOL, our study indicates the substantial social/economic burden of EB in Europe, attributable mostly to high direct non-healthcare costs.

KW - Cost-of-illness

KW - Epidermolysis bullosa

KW - European Union

KW - Health-related quality of life

KW - Rare disease

KW - Social cost

UR - http://www.scopus.com/inward/record.url?scp=84964240158&partnerID=8YFLogxK

U2 - 10.1007/s10198-016-0783-4

DO - 10.1007/s10198-016-0783-4

M3 - Article

C2 - 27107597

AN - SCOPUS:84964240158

VL - 17

SP - 31

EP - 42

JO - European Journal of Health Economics

JF - European Journal of Health Economics

SN - 1618-7598

IS - 1

ER -