OBJECTIVES: Cemiplimab is the first treatment approved in patients with aBCC who have progressed on or are intolerant to an HHI based on Study 1620 (NCT03132636), an open-label, multicentre, single arm, non-randomised trial. Prior to cemiplimab availability, there was no standard of care after HHI for advanced BCC patients. The objectives of the current study were to identify and characterise clinical trials and observational studies evaluating the response rates, progression-free survival, or overall survival (OS) of patients with aBCC following HHI receiving systemic therapy or best supportive care (BSC).

METHODS: A systematic literature review was performed to identify relevant studies through predefined searches of the EMBASE, MEDLINE and CENTRAL databases (no date restriction) and relevant conferences (Jan-2018 to Oct-2021). Data were extracted regarding study and patient characteristics and outcomes of interest.

RESULTS: Twelve studies were identified reporting outcomes in aBCC patients following HHI. Beyond Study 1620 (n=138), OS Kaplan-Meier data were only identified from one retrospective study evaluating BSC (n=15). Sample size was limited and differences were observed in patient and clinical characteristics (i.e., age, disease severity, performance status, reasons for HHI discontinuation, and prior therapy). For other studies identified, seven Phase 1/2 clinical trials and three observational studies evaluated seven unlicensed systemic interventions. Response rates varied substantially and should also be interpreted with caution given small sample size (n<10 in nine studies, n=31 in one study) and potential selection bias for observational studies.

CONCLUSIONS: Study 1620 represents the largest trial to date in aBCC following HHI. In view of the small sample size and heterogenous nature of studies of other possible treatment options, including BSC, Study 1620 represents a new benchmark for efficacy in this setting.