Pembrolizumab becomes the first and only immunotherapy to be recommended by NICE for urothelial carcinoma patients

NICE has issued final guidance recommending the use of the immunotherapy KEYTRUDA® (pembrolizumab) within the cancer drugs fund (CDF) as an option for the treatment of locally advanced or metastatic urothelial carcinoma in adults who have had platinum-containing chemotherapy.

“Outcomes of patients with chemotherapy refractory metastatic bladder cancer remain stubbornly poor. Pembrolizumab is the first immune check point inhibitor in this setting to show extended survival in this group of patients. This opens a new chapter in the treatment of bladder cancer. NICE’s recommendation for pembrolizumab is a bright ray of light for patients with this difficult disease,” explained Thomas Powles from Barts Cancer Centre (London, UK).

The recommendation states that pembrolizumab should be stopped at 2 years of uninterrupted treatment or earlier in the event of disease progression and the conditions in the managed access agreement for pembrolizumab are to be followed.

Inclusion in the CDF demonstrates that NICE considers that pembrolizumab has potential to satisfy the criteria for routine use on the NHS for this group of bladder cancer patients but before making a final decision on routine commissioning it needs more investigation, through data collection in the NHS in clinical practice or from further research.

NICE based its decision on the randomized, pivotal, phase III KEYNOTE-045 study evaluating pembrolizumab monotherapy compared to investigator-choice chemotherapy (paclitaxel, docetaxel, vinflunine) in the treatment of patients with metastatic or locally advanced, urothelial cancer that had recurred or progressed following platinum-based chemotherapy.

KEYNOTE-045 demonstrated that pembrolizumab extended overall survival by almost 3 months more than chemotherapy (median overall survival was 10.3 months (95% confidence interval [CI], 8.0 to 11.8) in the pembrolizumab group, compared with 7.4 months (95% CI, 6.1 to 8.3) in the chemotherapy group. Treatment-related adverse events occurred in 60.9% of the patients treated with pembrolizumab, compared with 90.2% of those who received chemotherapy.