Health-Related Quality of Life of Patients with Epidermolysis bullosa and Carers Using a Time Trade-Off Approach in the UK General Population to Elicit Utilities for Health Technology Assessment

Background: Epidermolysis bullosa (EB) is a complex group of rare, inherited skin disorders that blister easily, imposing substantial burden on patients and carers. There is a lack of published evidence in the UK quantifying health-related quality of life in EB patients and carers as utility values suitable for use in Health Technology Assessment (HTA). Methods: A time trade-off (TTO) study was conducted with 120 UK general public members to estimate utility values for EB patient and primary carer vignettes depicting varying EB severity. An exploratory sub-study involving six carer and clinical experts was conducted to estimate utility values of second carers. Results: 115 general public participants were included in the final TTO analysis. Mean utility values declined as disease severity increased, from 0.82 in the least severe state, to 0.54 in the most severe. Carer utilities were higher than the equivalent patient health states, but with a similar pattern of decreasing utility with increasing EB severity, ranging from 0.85 to 0.64. The secondary carer sub-study, in six respondents estimated a second care burden factor relative to the primary carer of 77%. Conclusions: The utilities estimated for both EB patients and their carers have relevance for future economic evaluations and HTA of new EB therapies.