NICE has approved nivolumab with ipilimumab for use within the Cancer Drugs Fund as an option for adults with untreated advanced renal cell carcinoma (RCC) that is intermediate- or poor-risk according to the International Metastatic Renal Cell Carcinoma Database Consortium criteria.
The decision is based on the findings of the CheckMate 214 trial, an open-label, randomised controlled trial of nivolumab with ipilimumab versus sunitinib.
In an interim analysis (August 2017) at a median follow-up of 25 months, nivolumab with ipilimumab was associated with improved progression-free survival (PFS) and overall survival (OS) compared to sunitinib in people with intermediate- or poor-risk RCC.
Median PFS was 11.0 months (95% CI 8.3-15.2) for nivolumab with ipilimumab and 8.3 months (95% CI 7.0-9.8) for sunitinib (hazard ratio [HR] 0.76; 99.1% CI 0.60-0.95). Median OS was not reached (95% CI 28.2 to not evaluable) for nivolumab with ipilimumab and was 26.0 months (95% CI 22.1 to not evaluable) for sunitinib (HR 0.63; 99.8% CI 0.44-0.89). Based on the strength of the survival benefit shown, CheckMate 214 was stopped early.
However, the NICE appraisal committee noted that trials stopped early for benefit can overestimate the benefit. It also noted that the data were too immature to establish the long-term effect of treatment.
NICE acknowledged that the combination has the potential to be cost-effective, but said more evidence is needed to address the clinical uncertainties. Hence, it has approved nivolumab with ipilimumab for use in the Cancer Drugs Fund only for untreated advanced RCC, while the manufacturer collects further data.
The decision will be reviewed after August 2021 when further evidence is expected.