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


CONCLUSIONS
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.
Department of Social Policy and LSE Health, London School of Economics and Political Science, London, UK. a.n.angelis@lse.ac.uk.
Aris
A
Angelis
Department of Social Policy and LSE Health, London School of Economics and Political Science, London, UK.
Panos
P
Kanavos
University of Castilla-La Mancha, Talavera de la Reina, Toledo, Spain.|Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain.
Julio
J
López-Bastida
Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain.|Fundación Canaria de Investigación Sanitaria (FUNCANIS), Las Palmas de Gran Canaria, Spain.
Renata
R
Linertová
Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain.|University of Castilla-La Mancha, Toledo, Spain.
Juan
J
Oliva-Moreno
Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain.|Evaluation and Planning Service at Canary Islands Health Service, Santa Cruz de Tenerife, Spain.
Pedro
P
Serrano-Aguilar
Institute of Rare Diseases Research, ISCIII, SpainRDR and CIBERER, Madrid, Spain.
Manuel
M
Posada-de-la-Paz
National Center for Rare Diseases, Istituto superiore di sanità (ISS), Rome, Italy.
Domenica
D
Taruscio
Centro di Ricerche Cliniche per Malattie Rare Aldo e Cele Daccò, Istituto di Ricerche Farmacologiche Mario Negri, Ranica (Bergamo), Italy.
Arrigo
A
Schieppati
Institute of Rare Diseases, Plovdiv, Bulgaria.|Department of Social Medicine and Public Health, Faculty of Public Health, Medical University of Plovdiv, Plovdiv, Bulgaria.
Georgi
G
Iskrov
Department of Health Economics, Corvinus University of Budapest, Budapest, Hungary.
Valentin
V
Brodszky
Centre for Health Economics Research Hannover (CHERH), Leibniz Universität Hannover, Hannover, Germany.
Johann Matthias Graf
JM
von der Schulenburg
URC Eco Ile de France, AP-HP, Paris, France.|Université Paris Diderot, Sorbonne Paris Cité, ECEVE, UMRS 1123, Paris, France.|INSERM, ECEVE, U1123, Paris, France.
Karine
K
Chevreul
The Swedish Institute for Health Economics, Lund, Sweden.
Ulf
U
Persson
Centre for Research on Health and Social Care Management (CERGAS), Bocconi University, Milan, Italy.
Giovanni
G
Fattore
D000293:Adolescent; D000328:Adult; D017028:Caregivers; D002648:Child; D017281:Cost of Illness; D003430:Cross-Sectional Studies; D004820:Epidermolysis Bullosa / Q000191:economics* / Q000523:psychology; D005060:Europe; D005062:European Union; D005260:Female; D017048:Health Care Costs / Q000706:statistics & numerical data; D006801:Humans; D008297:Male; D008875:Middle Aged; D005791:Patient Care / Q000191:economics; D011788:Quality of Life; D018582:Sick Leave / Q000191:economics / Q000706:statistics & numerical data; D018434:Sickness Impact Profile; D012959:Socioeconomic Factors; D011795:Surveys and Questionnaires; D006113:United Kingdom; D055815:Young Adult
Cost-of-illness; Epidermolysis bullosa; European Union; Health-related quality of life; Rare disease; Social cost