OBJECTIVES: This study provides real-world data (RWD)on patients with advanced, recurrent/metastatic squamous cell oesophageal cancer and treated with nivolumab (Opdivo®) after at least one prior systemic therapy.

METHODS: Adult patients who initiated nivolumab as part of United Kingdom (UK) Early Access to Medicines Scheme Program (EAMS) and consented to observational research component of the EAMS were included. Data collection included patient characteristics, treatment history, nivolumab treatment patterns, disease progression and survival status. Patients completed EQ-5D-3L at baseline and biweekly for up to 24 weeks. Adverse events (AE) were collected. Descriptive analyses were performed. Kaplan-Meier method evaluated treatment persistence, progression-free survival (PFS), and overall survival (OS).

RESULTS: 87 oesophageal cancer patients enrolled in EAMS (June –November 2020) and 79 patients received at least one nivolumab treatment. Of these, 44 patients (55.7%) participated in the observational research and were followed for 12 months. Patients (median age = 66.5 years) were mostly male (65.9%) and white (88.6%). Median nivolumab treatment duration was 2.6 months (95% CI: 2.0-3.0). In available data, median PFS was 3.8 months (95% CI:2.6-4.9) and 19.3% (95% CI:7.0-36.1) of patients remained disease progression-free at 12 months. Median OS was 5.5 months (95% CI:3.9–not estimable); 18 patients had died by end of follow-up. Among those who completed baseline EQ-5D, the mean EQ-VAS score was 63.4 (SD:18.3) and mean health utility scores were 0.6 (SD:0.2) at baseline. Of 73 AEs, 68 were serious AEs and 2 SAEs were related to nivolumab.

CONCLUSIONS: RWD collected in EAMS suggested that treatment duration and 12-month PFS, HRQoL, and treatment safety were consistent with the clinical trial population. Shorter OS in EAMS aligned with the expectation of a diverse population in real-world setting. These data demonstrate UK EAMS are an effective setting to demonstrate value of new medicines ahead of market access.