OBJECTIVES : Advances in the understanding of the molecular pathogenesis of non-small cell lung cancer (NSCLC) are paving the way for new treatments targeting specific patient populations harbouring molecular biomarkers. This study analyses the health technology assessment (HTA) decisions for treatments in non-endothelial growth factor receptor (EGFR)/ anaplastic lymphoma kinase (ALK) positive patients.

METHODS : HTAs for the treatment of NSCLC corresponding to EMA assessments from 2009 onwards were identified from an internal database. HTAs where EGFR/ ALK mutations were specified in the target indication were excluded. Assessments by NICE (England and Wales), SMC (Scotland), HAS (France), G-BA (Germany), PBAC (Australia), and CADTH (Canada) were reviewed.

RESULTS : 55 HTAs were identified for 8 treatments for NSCLC in non-EGFR/ ALK positive patients, where 72.7% were for programmed death-(ligand)-1 (PD-(L)1) inhibitors. In 78.1% of all HTAs, the treatment was recommended or demonstrated added benefit (90% for PD-(L)1 inhibitors). CADTH, SMC, and NICE had the highest number of positive outcomes (85.7%, 88.9% and 90.9%); however, all recommendations by CADTH were conditional on the cost-effectiveness being improved to an acceptable level. NICE’s Cancer Drugs Fund, which allows provisional access while additional data is collected to support clinical/ cost-effectiveness, was a frequent access route for new treatments (50%). HAS and the G-BA determined added benefit in 66.7% and 70% of HTAs, respectively. The lowest number of positive outcomes was by PBAC, where the treatment was recommended in 55.6% of HTAs, likely due to the nature of Australian HTAs, where the manufacturer often needs to adjust the appraisal with a resubmission. Overall survival was a key driver for decision making across all countries, although uncertainties in cost-effectiveness was often cited as a reason behind a negative decision.

CONCLUSIONS : The majority of HTAs had a positive outcome, with a higher approval rate for PD-(L)1 inhibitors. There were, however, different outcomes between countries.