Presentation (CTI)

OBJECTIVES: Overall survival (OS) is widely recognized by health technology assessment (HTA) bodies as the gold standard endpoint in oncology trials. To evaluate positive reimbursement decisions despite uncertainty in OS, we reviewed Canadian HTAs in lung cancer indications.
METHODS: HTA recommendations for lung cancer indications from 2019-2024 were retrieved from CDA. Corresponding submissions for these products were retrieved from the INESSS website. Information on product, trial design, final decisions, and critique were extracted from HTAs that received a positive recommendation.
RESULTS: Of 422 HTAs by CDA between 2019-2024, 51 were in lung oncology indications/solid tumour indications that included patients with lung cancer. CDA noted OS uncertainty in 24 submissions. Positive recommendations were issued for 18 submissions, whereas 6 received a negative recommendation. Non-comparative trial data were submitted for all HTAs that received a negative CDA recommendation and seven that received a positive recommendation. OS uncertainties were mainly due to immature OS data (n=7), for which most pivotal trials submitted by the sponsor were ongoing at time of submission (n=5). In corresponding INESSS submissions, 12 products received a positive recommendation. Nine products had differing recommendations between the agencies. Of the 30 positive HTAs, the HTA bodies noted oral convenience (n=20), manageable toxicity profile (n=17), CNS benefit (n=15), maintenance of quality of life (n=6) as part of their rationale for recommendation.
CONCLUSIONS: Although OS remains a gold standard endpoint, limitations such as time needed to collect OS data presents as a challenge in clinical trials. CDA and INESSS have issued positive recommendations in the absence of mature or non-statistically significant OS data. Notably, CDA recently implemented time limited recommendations as part of modernizing their review processes, to help balance timely access to promising new therapies with considerations under high uncertainty; future investigations will provide an understanding of the impact of these changes in decision-making processes.