OBJECTIVES: Early-phase evidence at CADTH often receives a Do Not Reimburse recommendation. CADTH’s 2022-2025 Strategic Plan aims to address uncertainty and identify new ways to bridge evidence gaps. In April 2023, CADTH released a proposal on time-limited recommendations. This observational research aims to compare Canada’s oncology drug access with other HTA markets and identify factors influencing clinical success at CADTH.

METHODS: CADTH’s website was searched for files submitted with phase I or II evidence, and issued recommendations from January 2021 through May 2023. Recommendations were analysed to identify clinical drivers of outcomes. Websites of INESSS (Quebec, Canada) and seven international HTA bodies with accessible English information were searched (September 19, 2023) to determine outcomes and funding mechanisms.

RESULTS: 9/27 (33%) early-phase oncology submissions at CADTH received negative recommendations. INESSS reviewed 26/27 CADTH files; 14 (54%) had negative outcomes. Lower rates of negative outcomes were observed for SMC (2/13, 15%) and NICE (3/18, 17%). Common limitations in the CADTH negative recommendations included lack of statistical methods (9/9, 100%), non-comparative trials (7/9, 78%) with a RCT considered feasible for 5/7 (71%), and lack of HRQoL assessment (4/9, 44%). 16/18 (89%) positive files were non-comparative with a RCT considered feasible for 5/16 (31%). 13/18 (72%) lacked statistical methods, and 3/18 (17%) lacked HRQoL assessment. Despite similar ITC limitations, 1/9 (11%) negative and 6/15 (40%) positive files with an ITC resulted in directional efficacy conclusions. 6/9 (67%) negative and 15/18 (83%) positive recommendations mentioned significant unmet need; however, only positive recommendations concluded that the drug addressed the need (17/18, 94%).

CONCLUSIONS: Identifying success factors at CADTH was challenging due to inconsistencies, though RCT feasibility and HRQoL assessment may be influential. Adopting a consistent Canadian deliberative framework and funding mechanism for early-phase evidence, like the United Kingdom and Scotland, could enhance transparency, reduce regional disparities, and improve patient access.