Vismodegib Use in Clinical Practice: Analysis of a United States Medical Claims Database

February 2018 | Volume 17 | Issue 2 | Original Article | 143 | Copyright © February 2018

C. William Hanke MD,a Shivani K. Mhatre PhD,b David Oliveri BS,c Marko Zivkovic PhD,c Ivor Caro MD,b Daniel Bergström PhD,b* Keith Dawson MS,b and Camelia S. Sima MDb

aLaser and Skin Surgery Center of Indiana, Carmel, IN bGenentech, Inc., South San Francisco, CA cGenesis Research, Hoboken, NJ *Current affiliation: Nutricia, Solna, Sweden

BACKGROUND: Information is limited on the use of vismodegib for treatment of advanced basal cell carcinoma beyond the setting of clinical trials. OBJECTIVE: To investigate the treatment patterns and characteristics of patients treated with vismodegib in clinical practice. METHODS: A longitudinal, retrospective cohort study was undertaken using data from a US commercial insurance claims (Truven Health Analytics MarketScan) database. Eligible patients were ≥18 years of age, with ≥1 claim for vismodegib from January 2012 to December 2015. RESULTS: A total of 321 patients were included in the analysis. Approximately 20% of the patients took 1 or more treatment breaks of ≥ 30 days each before treatment discontinuation. Median duration of vismodegib treatment before the first treatment break and discontinuation was 4.0 and 5.5 months, respectively. Older age ( > 65 years) and absence of Gorlin syndrome were associated with increased risk for treatment interruption or discontinuation. Overall, 47% and 36% of patients underwent surgery or radiotherapy within the 6 months before and after vismodegib initiation, respectively. CONCLUSIONS: Real-world evidence indicates that vismodegib is being used in clinical practice as part of combination treatment strategies. J Drugs Dermatol. 2018;17(2):143-148.


Basal cell carcinoma (BCC), representing approximately 80% of nonmelanoma skin cancers, is the most commonly diagnosed human cancer worldwide,1 and some evidence suggests that the worldwide incidence of BCC is increasing.2BCC is characterized by local spreading and a low tendency to metastasize. Although more than 95% of the cases of BCC are successfully treated by surgery or, less frequently, radiation therapy, infrequently the tumor can progress to advanced BCC (aBCC; composed of locally advanced BCC and metastatic BCC).3,4 Standard treatment such as surgery and radiotherapy may be inappropriate for aBCC because these treatments are unlikely to effect a cure or, in the case of surgery, may result in substantial disfigurement.5,6 Studies have shown that the Hedgehog signaling pathway is a key driver in the pathogenesis and progression of BCC and is abnormally activated in nearly all cases.1,4,7 To date, 2 Hedgehog pathway inhibitors, namely vismodegib and sonidegib, have shown clinical efficacy in halting disease progression for patients with aBCC.4 Vismodegib is approved for the treatment of aBCC in which the disease has recurred after surgery or in which patients are not candidates for radiation therapy.8,9 Sonidegib is approved for use in locally advanced BCC but not metastatic BCC.10Although data from a study assessing the use of vismodegib in a large cohort of patients representative of routine clinical practice (STEVIE study, a multicenter clinical trial evaluating vismodegib in aBCC, identifier: NCT01367665),11 and the RegiSONIC Disease Registry Study ( identifier: NCT01604252)12 are available, additional information about real-world use of this drug is still needed. The objective of the current study was to evaluate the use of vismodegib in routine clinical practice in patients with aBCC in the United States to better inform decision makers and clinicians regarding patient experience. The clinical characteristics of patients, treatment patterns of vismodegib (duration of use, interruptions, discontinuation), and other treatments received around the time of vismodegib administration were evaluated. 


Data Source 

This analysis used data from the Truven Health Analytics MarketScan Commercial Claims and Encounters (CCAE) and