OBJECTIVES: To identify evidence gaps in the literature on the burden of illness and treatment of bullous pemphigoid (BP) to support the launch of efgartigimod to treat this rare disease.
METHODS: A structured literature review was conducted from 25 October 2012 through to 25 October 2022 in PubMed, Embase, and Cochrane regarding disease description, epidemiology, humanistic and economic burden, treatment guidelines, and treatment patterns. Desktop searches were conducted for health technology assessments (HTAs), competitor labels, and ongoing clinical trials.
RESULTS: Based on available epidemiology data, the incidence (and prevalence) of BP has been increasing since 2003. This could be due to the aging population, with a greater number of individuals at risk for BP. Although data suggest that the incidence of BP is increasing, the true burden of BP is likely underestimated due to the poor study design of index studies. BP follows a chronic-remitting course that can be self-limiting. Partial or complete remission can occur at a median time of 6.7 months, with relapses occurring at a median time of 15.9 months. BP is associated with a negative impact on health-related quality of life. No economic burden studies were found for direct costs specific to BP; only 1 study was found for inpatient burden. There are currently no licensed treatments for BP, but several therapies are used off-label, either as monotherapy or in combination, or are generic. Corticosteroids have been the standard treatment for decades, but they are associated with significant adverse events, especially in older adults. HTAs are currently limited, as only two published HTA decisions on treatments for BP were identified.
CONCLUSIONS: Although there have been recent advancements in the understanding of BP pathogenesis, new treatments are still needed to control disease activity. There are currently no licensed treatments for BP.
