The aim of this project, which was commissioned by NICE through the NIHR HTA Programme as part of the NICE’s Single Technology Appraisal process, was to review the evidence for the clinical and cost-effectiveness of Pembrolizumab for the treatment for the treatment of adults with advanced or recurrent PD-L1 positive non-small cell lung cancer i) whose disease has progressed after platinum-containing doublet chemotherapy and ii) whose disease has progressed on both platinum-containing doublet chemotherapy and targeted therapy for epidermal growth factor receptor (EGFR) or anaplastic lymphoma kinase (ALK) positive tumours. The clinical effectiveness evidence submitted by the company (Merck Sharp & Dohme Ltd) responsible for manufacturing Pembrolizumab (Keytruda) consisted of three RCTs: KEYNOTE-010, a phase II/III head-to-head RCT that compared pembrolizumab with docetaxel; KEYNOTE-001 (Parts C and F) a phase I trial due to its initial dose escalation, which evolved into multiple phase II-like sub-studies through a series of expansion cohorts that assessed the effects and safety of pembrolizumab (no comparator); and LUME-LUNG-1, a phase III trial that compared docetaxel plus nintedanib with docetaxel plus placebo. The Aberdeen HTA Group acted as the Evidence Review Group (ERG) for the project and provided an independent critique of the clinical and cost-effectiveness evidence submitted by the company.

After taking into consideration the company submission and the findings of the ERG’s critique, NICE released clinical guidance (TA428, January 2017). Pembrolizumab is currently recommended, within its marketing authorisation, as an option as an option for treating locally advanced or metastatic PD L1 positive non small cell lung cancer in adults who have had at least one chemotherapy (and targeted treatment if they have an epidermal EGFR or ALK positive tumour), only if: i) Pembrolizumab is stopped at 2 years of uninterrupted treatment and no documented disease progression, and ii) the company provides Pembrolizumab in line with the commercial access agreement with NHS England.


Miriam Brazzelli;




Brazzelli, M, Hawkins, N, McKeekin, N, Goulao, B, Cooper, D, Sharma, P, Fraser, C, Nicolson, M, Wu, O. Pembrolizumab after platinum chemotherapy for treating advanced, recurrent, PD-L1 positive, non-small cell lung cancer. . Evidence Review Group report in support of the NICE STA University of Aberdeen: Health Services Research Unit; 2016 URL: