In lock step with its US counterparts, Health Canada this week announced that it’s seeking to better use real-world evidence (RWE), while addressing certain specifics in protocol development and data quality concerns.
As real-world data (RWD) sources increase in quantity and quality, Health Canada explains how “prospectively planned clinical trials have been and continue to be considered the most robust tool for providing evidence of drug safety and efficacy,” although RWE has the potential to provide significant value when controlled clinical trials are not feasible or are challenging to conduct.
“We encourage RWE submissions (1) that aim to expand evidence-based indications for populations often excluded from clinical trials (ex: children, seniors, and pregnant women); (2) for drugs/diseases where clinical trials are unfeasible such as may be the case with rare diseases; and/or (3) where clinical trials are unethical, as may be the case during emergencies where dosages from animal studies may need to be extrapolated to treat humans potentially exposed to chemical or biological threats,” Health Canada says.
But “the potential for bias (e.g. confounding), and issues that may impact the robustness of the findings” are a cause for concern, the regulator adds. Health Canada offers sponsors 15 key elements that should be considered for each research protocol, including data management and quality control, and ethical and data protection issues.
With two principal data collection techniques for building RWE, Health Canada explains how prospective data collection “requires similar rigour as would be anticipated for a controlled clinical trial,” while retrospective data collection “has additional elements that should be addressed.
“Due to the retrospective nature of these designs, the statistical assumptions may require testing to ensure their validity (e.g. comparability of study arms). All procedures should be clearly articulated and transparent. Validation of data sets should be clearly described and include information addressing data completeness (e.g. methodology to address missing data),” Health Canada adds.
Health Canada also explained how it is collaborating with its Health Technology Assessment partners to establish a joint document later this year that will optimize and formalize the use of RWE across the drug life cycle.
Similarly, the US Food and Drug Administration unveiled
its RWE framework last December to provide a high-level view of how it is looking at the use of RWD and RWE. And earlier this month, FDA approved a new indication for a cancer drug
thanks to the use of electronic health record data.
Optimizing the Use of Real World Evidence to Inform Regulatory Decision-Making
Elements of Real World Data/Evidence Quality throughout the Prescription Drug Product Life Cycle