Related Experiment Video
Updated: Feb 28, 2026
A Rapid Screening Workflow to Identify Potential Combination Therapy for GBM using Patient-Derived Glioma Stem Cells
Published on: March 28, 2021
A Matching-Adjusted Indirect Comparison of Sonidegib and Vismodegib in Advanced Basal Cell Carcinoma
Dawn Odom1, Deirdre Mladsi1, Molly Purser1
1RTI Health Solutions, 200 Park Offices Dr., Research Triangle Park, NC, USA.
Objectives:
Based on single-arm trial data (BOLT), sonidegib was approved in the US and EU to treat locally advanced basal cell carcinomas (BCCs) ineligible for curative surgery or radiotherapy. Vismodegib, the other approved targeted therapy, also was assessed in a single-arm trial (ERIVANCE). We examined the comparative effectiveness of the two drugs using a matching-adjusted indirect comparison (MAIC) versus an unadjusted indirect comparison.
Methods:
After comparing trials and identifying potential prognostic factors, an MAIC was conducted to adjust for differences in key patient baseline characteristics. Due to BOLT's small sample size, the number of matching variables was restricted to two. Efficacy results for sonidegib were generated so that selected baseline characteristics matched those from ERIVANCE and were compared with published ERIVANCE results.
Results:
Matching variables were baseline percentages of patients receiving prior radiotherapy and surgery. After weighting, sonidegib objective response rate (ORR) and median progression-free survival (PFS) were effectively unchanged (prematched versus postmatched ORR and PFS, 56.1% versus 56.7% and 22.1 versus 22.1 months, resp.). Vismodegib's ORR and PFS were 47.6% and 9.5 months.
Conclusions:
Comparative effectiveness of sonidegib versus vismodegib remains unchanged after adjusting BOLT patient-level data to match published ERIVANCE baseline percentages of patients receiving prior surgery and radiotherapy.
Insights
A matching-adjusted indirect comparison found sonidegib and vismodegib have similar effectiveness for advanced basal cell carcinoma (BCC). Adjusting for patient characteristics did not change sonidegib's outcomes compared to vismodegib.
Area of Science:
- Oncology
- Clinical Pharmacology
Background:
- Sonidegib and vismodegib are targeted therapies for locally advanced basal cell carcinoma (BCC) ineligible for surgery or radiotherapy.
- Both drugs were approved based on single-arm trial data (BOLT for sonidegib, ERIVANCE for vismodegib).
- Direct comparative data between these two agents is lacking.
Purpose of the Study:
- To compare the effectiveness of sonidegib versus vismodegib for advanced BCC.
- To assess the impact of patient baseline characteristics on comparative effectiveness using a matching-adjusted indirect comparison (MAIC).
Main Methods:
- A matching-adjusted indirect comparison (MAIC) was performed using data from the BOLT and ERIVANCE trials.
- Key patient baseline characteristics (prior radiotherapy and surgery) were identified as matching variables.
- Sonidegib efficacy data was weighted to match the baseline characteristics of the ERIVANCE trial population.
Main Results:
- After matching, the objective response rate (ORR) for sonidegib remained 56.7% and median progression-free survival (PFS) was 22.1 months.
- Vismodegib demonstrated an ORR of 47.6% and a median PFS of 9.5 months.
- The MAIC indicated that sonidegib's effectiveness was not significantly altered by adjusting for baseline differences.
Conclusions:
- The comparative effectiveness of sonidegib versus vismodegib for advanced BCC remains consistent after adjusting for baseline patient characteristics.
- MAIC is a valuable method for comparing treatments approved via single-arm trials when direct head-to-head data is unavailable.
