OBJECTIVES: Perform a descriptive analysis to understand influential factors including RWE in health technology assessment (HTA) decision-making when assessing oncology submissions with phase II / single arm trial data.

METHODS: Oncology submissions to the Canadian Agency for Drugs and Technologies in Health (CADTH) that contained phase II / single arm trial data between 2018 and 2022 were obtained and analyzed from the CADTH website. Information from the final recommendation, clinical, and economic reviewers reports was used to determine factors that likely influenced HTA recommendations. Upon completing this preliminary analysis, certain files were further investigated to understand the methodology of RWE used, any learnings that could be used for future RWE submissions by manufacturers, and their assessment in jurisdictions outside of Canada.

RESULTS: A total of 34 submissions were identified between 2018-2022. Of these, 20 (59%) received a positive recommendation (i.e., list or list with conditions) while 14 (41%) received a negative recommendation (i.e., do not list). 85% of positive recommendations recognized a net clinical benefit despite uncertainty. Files with negative recommendations noted high uncertainty in net clinical benefit and a majority of these noted that conducting a phase 3 trial was feasible. Of the 10 submissions that used RWE, 9 used international data sources while 1 used Canadian data. 8 of these received a positive recommendation, while 2 received a negative recommendation. Mostly, RWE was used to inform comparative efficacy.

CONCLUSIONS: Positive net clinical benefit and infeasibility of conducting a Phase III clinical trial were associated with positive recommendations for Phase II/single arm trial data. Analyses using RWE were critiqued and often noted to be interpreted with caution. It is unclear whether the inclusion of RWE was sufficient to mitigate uncertainty in HTA decision making. Differences in HTA assessments of RWE submissions could lead to inequity in access globally.