Abstract

Objectives

To characterize real-world evidence (RWE) in Canada’s Drug Agency (CDA-AMC) submissions, describe its designs and purposes, and summarize the agency/committee views on its use.

Methods

We conducted a comprehensive environmental scan of the CDA-AMC website to retrieve all reports and recommendations of reimbursement review submissions published between January 2020 and June 2024. RWE utilization was classified by therapeutic area, study design, geographic data source, and intended purpose. Reviewer and committee comments were thematically coded.

Results

Of the 294 submissions screened, 274 were included in this study. A total of 70 submissions (25.5%) contained RWE. These submissions cited 113 distinct real-world data/RWE sources, namely: retrospective cohorts (58%), prospective cohorts (20%), and other designs (22%). Geographic origin was predominantly the United States (39%) or Europe (38%); only 12 sources (11%) were Canadian. RWE often informed comparative effectiveness (54%) and evidence gaps (52%). Reviewer critiques focused on data quality (70%) and generalizability (61%); lack of Canadian data was identified in 50% of submissions. Within the Canadian subgroup, generalizability concerns decreased (40%), but data quality issues remained common (70%). A total of 83% of submissions received “reimburse with conditions” recommendations.

Conclusions

RWE has become routine in CDA-AMC submissions, yet remains largely non-Canadian and limited in its applicability. More formal guidance on transportability analyses, quantitative bias assessment, and improved standards across clinical and economic domains could enhance evidence generation, submission quality, and health technology assessment confidence in clinical and pharmacoeconomic information. These findings may inform ongoing refinement of RWE checklists and expectations, providing a benchmark against which future use of RWE can be measured and tracked.