OBJECTIVES: The quantification of carer utility impacts in cost effectiveness analyses (CEA) has become prominent in recent years, especially in rare diseases. However, there is a lack of precedent for valuing utilities where more than one informal carer is involved. Epidermolysis bullosa (EB) is a rare, genetic skin disease affecting children and adults where more than one family member is typically involved in the care of the patient. A time trade-off study, previously conducted in the UK general public to inform health state utility values for both patients and primary carers, was used in CEA for a novel EB treatment, developed for NICE highly specialised technology (HST) appraisal. This estimated a utility value of 0.64 for the main carer in the highest severity health state, but did not explore second-carer impact and utility.

METHODS: A supplementary exercise was performed via an online questionnaire administered by the DEBRA EB patient support organisation, whereby EB carers and healthcare professionals were asked to evaluate the second-carer utility impact, relative to the primary carer. Vignette descriptions of an EB patient in the highest severity health state and typical carer were provided for context.

RESULTS: Six respondents estimated that the average impact of an additional carer, relative to the main carer utility, was 77% (range: 60%-100%), resulting in an average utility value of 0.70 for second carers of patients in the most severe EB health states.

CONCLUSIONS: This exploratory study has shown there are utility impacts associated with second-carer involvement in EB. As this was an initial study in a small sample used to inform HST appraisal, further data collection is necessary to confirm results and provide second-carer utility values that can be used in future economic evaluations for EB and other rare diseases.