Doxycycline compared with prednisolone therapy for patients with bullous pemphigoid: cost-effectiveness analysis of the BLISTER trial

Abstract

Background Bullous pemphigoid (BP) is an autoimmune blistering skin disorder associated with significant morbidity and mortality. Doxycycline and prednisolone to treat bullous pemphigoid were compared within a randomized controlled trial (RCT). Objectives To compare the cost‐effectiveness of doxycycline‐initiated and prednisolone‐initiated treatment for patients with BP. Methods Quality‐of‐life (EuroQoL‐5D‐3L) and resource data were collected as part of the BLISTER trial: a multicentre, parallel‐group, investigator‐blinded RCT. Within‐trial analysis was performed using bivariate regression of costs and quality‐adjusted life‐years (QALYs), with multiple imputation of missing data, informing a probabilistic assessment of incremental treatment cost‐effectiveness from a health service perspective. Results In the base case, there was no robust difference in costs or QALYs per patient at 1 year comparing doxycycline‐ with prednisolone‐initiated therapy [net cost £959, 95% confidence interval (CI) −£24 to £1941; net QALYs −0·024, 95% CI −0·088 to 0·041]. However, the findings varied by baseline blister severity. For patients with mild or moderate blistering (≤ 30 blisters) net costs and outcomes were similar. For patients with severe blistering (> 30 blisters) net costs were higher (£2558, 95% CI −£82 to £5198) and quality of life poorer (−0·090 QALYs, 95% CI −0·22 to 0·042) for patients starting on doxycycline. The probability that doxycycline would be cost‐effective for those with severe pemphigoid was 1·5% at a willingness to pay of £20 000 per QALY. Conclusions Consistently with the clinical findings of the BLISTER trial, patients with mild or moderate blistering should receive treatment guided by the safety and effectiveness of the drugs and patient preference – neither strategy is clearly a preferred use of National Health Service resources. However, prednisolone‐initiated treatment may be more cost‐effective for patients with severe blistering.