Effectiveness, safety and utilization of vismodegib in locally advanced basal cell carcinoma under real‐world conditions in Germany – The non‐interventional study NIELS

Background Basal cell carcinoma (BCC) can arise by the uncontrolled proliferation of cells from multiple epidermal compartments due to aberrant activation Hedgehog (Hh) signalling pathway. Vismodegib, a small-molecule inhibitor this pathway, is approved for treatment patients with locally advanced (la) BCC inappropriate surgery or radiotherapy symptomatic metastatic (m) BCC. Objectives The aim non-interventional study was assess effectiveness special focus on duration response (DOR), safety and utilization vismodegib laBCC in daily practice Germany. Methods This (NIS) observed according German label clinical practice. All available who had received at least one dose between commercial availability Germany (02 August 2013) 3 years before end (31 March 2016) could be included were documented retrospectively and/or prospectively up years. Primary variable DOR. Assessment tumour carried out treating physicians. Exploratory variables vismodegib, decision makers therapy method evaluation. statistical analyses descriptive. Results Between September 2015 2019, 66 26 centres. objective rate (ORR) 74.2% disease control (DCR) 90.9%. median DOR 15.9 months (95% CI: 9.2; 25.7; n = 49 response). progression-free survival (PFS) 19.1 time (TTR) 2.7 months. A total 340 adverse events reported 63 (95.5%) patients; no new signals identified. Conclusions NIS NIELS shows laBCC. It confirms transferability results pivotal trial into routine