ABSTRACT
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.
ABSTRACT
OBJECTIVES: To assess patients' preferences and estimate willingness to pay (WTP) for gastroesophageal reflux disease (GERD) treatments. METHOD: Patients were randomly selected from a multicenter clinical study to participate in the discrete choice experiment (DCE) survey. Relevant treatment attributes were identified through literature review, clinical expert consultation, and focus groups. The DCE included 14 choice tasks composed of six attributes, three treatment profiles, and a "none"option considering orthogonality, D-efficiency, and level balance, while keeping patient response burden reasonable. Individual-level preferences and WTP were estimated by aggregate-level conditional logit and hierarchical Bayes analyses. RESULTS: Our sample of 361, drawn from a clinical trial, had a mean age of 57 years, were primarily women (53%), and rated their GERD symptoms as mild/moderate (31%) and moderately severe/severe (7%). Most important attributes of GERD treatment were (in order) as follows: avoiding side effects, sleeping discomfort, daytime discomfort, dietary changes, medication cost, and treatment frequency. Simulations found that patients are willing to pay an additional US $36 to reduce susceptibility to side effects from moderate to mild or to decrease the frequency of sleeping discomfort. Patients 65 years or older were willing to pay less for daytime discomfort relief, while women would pay more to avoid sleeping discomfort. CONCLUSIONS: Key factors concerning patients with GERD and their preference for treatment features to control GERD symptoms were confirmed. A DCE estimated WTP by GERD sufferers for relief from symptoms and avoidance of side effects using relevant treatment costs. These findings may help guide clinical treatment decisions for individual patients to improve GERD symptom control.
