A Comprehensive Review of Real-World Evidence (RWE) Use in Submissions to Canada's Drug Agency (CDA)
Author(s)
Cal Shephard, MSc1, Eon Ting, MBA, MSc1, Paul Arora, PhD2, Alind Gupta, PhD3, Ghayath Janoudi, MSc, PhD4, Winson Y. Cheung, MPH, MD5.
1AstraZeneca, Toronto, ON, Canada, 2Inka Health, Toronto, ON, Canada, 3University of Toronto, Mississauga, ON, Canada, 4Loon, Ottawa, ON, Canada, 5Alberta Health Services, Calgary, AB, Canada.
1AstraZeneca, Toronto, ON, Canada, 2Inka Health, Toronto, ON, Canada, 3University of Toronto, Mississauga, ON, Canada, 4Loon, Ottawa, ON, Canada, 5Alberta Health Services, Calgary, AB, Canada.
OBJECTIVES: The clinical relevance of RWE is increasing, but its role and impact in sponsor submissions to CDA are unclear, particularly when non-Canada data are used. This study aims to characterize the use of RWE in CDA over a five-year period.
METHODS: We conducted a comprehensive review of all sponsor submissions to CDA from January 2020 to June 2024. We summarized submissions that incorporated RWE and described CDA decisions and committee recommendations.
RESULTS: Of the 614 reports screened that represented 274 sponsor submissions, 70 considered RWE and met the study inclusion criteria for review. Among them, 44.2% were from pERC (oncology) and 55.7% were from CDEC (non-oncology). Most submissions focused on first-line (44.2%) and second-line (25.7%) therapies. RWE informed clinical evidence, indirect treatment comparisons, and economic models in 47.1%, 41.4%, and 67.1% of cases, respectively. Data sources for RWE were reported in 42.8% of submissions and frequently originated from the US (20.0%) and Europe (17.1%) rather than Canada (4.3%). The majority of RWE was based on retrospective cohort studies (52.9%). The number of submissions utilizing RWE increased over time from 7 in 2020 to 20 in 2023. CDA feedback on the submitted RWE emphasized specific areas for improvement, such as poor generalizability (90.0%), small sample sizes (52.9%) and lack of Canadian data (37.1%). CDA recommendations among the 70 submissions included reimbursement with conditions (82.8%), do not reimburse (15.7%), and time-limited reimbursement (1.4%).
CONCLUSIONS: Submissions using RWE have increased significantly from 2020, but there are concerns regarding generalizability, especially with the use of non-Canadian data. Transportability methods may offer a direct way to address concerns about applying non-Canadian RWE to Canadian contexts and should be considered in the future to improve health technology assessments in Canada.
METHODS: We conducted a comprehensive review of all sponsor submissions to CDA from January 2020 to June 2024. We summarized submissions that incorporated RWE and described CDA decisions and committee recommendations.
RESULTS: Of the 614 reports screened that represented 274 sponsor submissions, 70 considered RWE and met the study inclusion criteria for review. Among them, 44.2% were from pERC (oncology) and 55.7% were from CDEC (non-oncology). Most submissions focused on first-line (44.2%) and second-line (25.7%) therapies. RWE informed clinical evidence, indirect treatment comparisons, and economic models in 47.1%, 41.4%, and 67.1% of cases, respectively. Data sources for RWE were reported in 42.8% of submissions and frequently originated from the US (20.0%) and Europe (17.1%) rather than Canada (4.3%). The majority of RWE was based on retrospective cohort studies (52.9%). The number of submissions utilizing RWE increased over time from 7 in 2020 to 20 in 2023. CDA feedback on the submitted RWE emphasized specific areas for improvement, such as poor generalizability (90.0%), small sample sizes (52.9%) and lack of Canadian data (37.1%). CDA recommendations among the 70 submissions included reimbursement with conditions (82.8%), do not reimburse (15.7%), and time-limited reimbursement (1.4%).
CONCLUSIONS: Submissions using RWE have increased significantly from 2020, but there are concerns regarding generalizability, especially with the use of non-Canadian data. Transportability methods may offer a direct way to address concerns about applying non-Canadian RWE to Canadian contexts and should be considered in the future to improve health technology assessments in Canada.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
MSR133
Topic
Methodological & Statistical Research
Disease
No Additional Disease & Conditions/Specialized Treatment Areas