OBJECTIVES: To identify evidence gaps in the literature of the burden of illness and treatment of pemphigus vulgaris (PV) and pemphigus foliaceus (PF) to support the launch of efgartigimod to treat these rare diseases.

METHODS: A literature review using a structured search strategy was conducted from 1 July 2011 to 26 October 2021 in PubMed, Embase, and Cochrane regarding disease description, epidemiology, humanistic and economic burden, treatment guidelines, and treatment patterns. Online desktop searches for health technology assessments, competitor labels, and ongoing clinical trials were also conducted.

RESULTS: Epidemiology data are limited, and the true incidence and prevalence of pemphigus variants are not completely known worldwide. Unlike ICD-9 codes, ICD-10 codes are specific to PV and PF subtypes, but only a few epidemiology studies have used ICD-10 codes to date. There is a lack of studies that show the effect of treatment to prevent serious complications (e.g., opportunistic infections, cardiovascular disease), hospitalization, and death. Although health-related quality of life is significantly affected in patients with PV/PF, few studies have estimated it, and most used generic instruments. Correspondingly, two studies have reported the impact of PV/PF on caregivers’ quality of life (emotional, physical, and with daily activities). Only a few studies that estimate the economic burden of PV/PF are available. No cost-effectiveness (CE) analyses were found, including CE models that supported health technology assessment decisions. Few treatment-pattern studies were found, but one study was found that included rituximab after it was approved.

CONCLUSIONS: There are many gaps in the literature to support the launch of a new product in pemphigus, including epidemiology for budget-impact models, humanistic and economic burden studies, CE models, and treatment-pattern studies.