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

Abstract

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.

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

“…Rare diseases commonly place a large burden on family and caregivers, the impact of which is usually not taken into consideration in standard cost-effectiveness analyses (94)(95)(96). In light of the lack of appropriate HTA tools that can incorporate benefits and costs specific to rare disease treatments beyond the standard cost per QALY, e.g., socio-economic aspects, Multi-Criteria Decision Analysis (MCDA) is an approach that could be considered.…”

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

“…The estimated prevalence of EB in European Union countries is 2.4 cases per 100,000 inhabitants [29,30]. Angelis et al [31] estimated average annual costs per patient with EB that ranged between countries from €9509 to €49,233. Direct healthcare costs ranged from €419 to €10,688, direct non-healthcare costs ranged from €7449 to €37,451, and loss of labour productivity ranged from €0 to €7259.…”

Oral Health-related Quality of Life in Patients with Dystrophic Epidermolysis Bullosa

“…Therefore, it is not surprising to hypothesize that EB patients may develop a poor general quality of life associated with psychological impairment since childhood. Several studies have investigated this aspect and concluded that general quality of life in EB patients can be poor (Horn et al, 2002;Tabolli et al, 2009;Jeon et al, 2016;Angelis et al, 2016), but data on psychological aspects of EB have been conflicting. Some have reported no manifestation of anxiety and/or depressive symptoms (Fortuna et al, 2016) or other psychiatric disorders (Andreoli et al, 2002), whereas others have found a high prevalence of psychosocial problems and psychiatric symptoms (Margari et al, 2010).…”