A Matching-Adjusted Indirect Comparison of Sonidegib and Vismodegib in Advanced Basal Cell Carcinoma

Table 1

Overview of trial designs, including outcome definitions.

Trial characteristics	BOLT	ERIVANCE
Study description	(i) Multicenter, international, randomized, double-blind, phase 2 study to investigate the safety and efficacy of sonidegib; (ii) Patients were randomized to receive either 200 mg or 800 mg of sonidegib [5]	(i) Single-arm, multicenter, international, nonrandomized, phase 2 study to investigate the safety and efficacy of vismodegib [8]
Key inclusion criteria	(i) Histologically confirmed diagnosis, with measurable disease of ≥1 lesion, ≥10 mm in at least 1 dimension by MRI or color photograph; (ii) Patients were not amenable to radiation therapy, curative surgery, or other local therapies; (iii) Patients were not required to have received any prior therapy [5]	(i) Histologically confirmed diagnosis, with measurable disease of ≥1 lesion, ≥10 mm in the longest dimension; (ii) Patients were considered to be inoperable or medically contraindicated to surgery; (iii) Patients were required to have been given radiotherapy unless radiotherapy was contraindicated or inappropriate [8]
Key exclusion criteria	(i) Life expectancy was not mentioned; (ii) Presence of superficial multifocal BCC that may be considered unresectable was not mentioned [5]	(i) Patients with life expectancy <12 weeks [8]; (ii) Patients with superficial multifocal BCC that may be considered unresectable due to breadth of involvement [9]
Periods for reported results (minimum duration of follow-up)	(i) Primary analysis (6 months of follow-up) [14]; (ii) 12-Month update (12 months of follow-up) [5]; (iii) 18-Month update (18 months of follow-up) [7]	(i) Primary analysis (9 months of follow-up) [8]; (ii) 6-Month update (15 months of follow-up) [15]; (iii) 12-Month update (21 months of follow-up) [16]; (iv) 18-Month update (27 months of follow-up) [17]; (v) 24-Month update (33 months of follow-up) [18]; (vi) 30-Month update (39 months of follow-up) [19]
Primary efficacy endpoint	(i) ORR by central review	(i) ORR by central review
Other efficacy and safety outcomes available	(i) DOR; (ii) Complete response rate; (iii) PFS; (iv) Overall survival; (v) Time to response; (vi) Specific adverse events	(i) DOR; (ii) Complete response rate; (iii) PFS; (iv) Overall survival; (v) Specific adverse events
Assessment of tumor response	(i) mRECIST: composite assessment of MRI (per RECIST v1.1) [20], photograph (per WHO [21]), and histology; (ii) Prespecified sensitivity analysis using ERIVANCE-like criteria	(i) Composite assessment of MRI or photograph (per RECIST v1.0) [22], ulceration, and histology

BCC: basal cell carcinoma; DOR: duration of response; mRECIST: modified RECIST; MRI: magnetic resonance imaging; ORR: objective response rate; PFS: progression-free survival; RECIST: Response Evaluation Criteria in Solid Tumors; WHO: World Health Organization. Only patients with locally advanced BCC in the 200 mg arm are included in this analysis.