On April 16, 2019, Health Canada announced a new position to leverage real-world evidence (RWE) in decision-making to improve access to treatments in Canada. Along with the Canadian Agency for Drugs and Technologies in Health (CADTH) and Quebec’s Institut National d’Excellence en Santé et en Services Sociaux (INESSS), Health Canada alerted industry partners that they are now open to high quality RWE submissions as a way to expand the evidence base for treatments.  

The increased focus on RWE is one tactic to strengthen the Canadian regulatory system, a major initiative of Health Canada for 2019. In March, the agency released a document, “The Elements of RWD/Evidence Quality throughout the Prescription Drug Product Life Cycle,” outlining 15 principles to guide protocol in the generation of RWE, from clearly defining the research question to planning the dissemination of results. These principles are in line with recommendations from ISPOR’s RWE Task Force and FDA’s 2018 framework on RWE generation.


Prioritizing use cases for RWE

In addition to providing recommendations for best practices, Health Canada, CADTH, and INESSS are developing a joint statement regarding the use of RWE throughout the drug life cycle, also to be released in 2019. To inform their guidance, a workshop was held in collaboration with the Institute for Health Economics in October 2018, bringing together stakeholders to examine use cases for RWE in the regulatory and reimbursement process in Canada.  

Two key themes emerged from these activities:

  1. Generating RWE is a contextual process: Real-world data must be fit for purpose, as well as minimally processed, transparent, and traceable. Analyses should be pre-specified, transparent, and reproducible.  
  2. The opportunity to maximize use of RWE throughout the drug life cycle is significant. High-priority use cases identified in the October workshop included turning to RWE for regulatory approval when there are no comparative trials. RWE can also be used to reduce levels of uncertainty and assumptions made during the HTA process around treatment options not captured in RCTs, and for monitoring these assumptions. RWE can also inform and enable conditional reimbursement.

A hierarchy of evidence or fit-for-purpose data?

While Health Canada and CADTH move forward to adopt RWE throughout the drug life cycle, information from the October workshop continues to label RWE as secondary to evidence produced by randomized controlled trials, the preferred evidence source for effectiveness estimates among regulatory and HTA agencies in Canada.

Assuming a hierarchical structure of evidence quality in which RWE sources are viewed as less than RCTs, however, reinforces hesitancy to employ RWE. With broadening recognition that RCTs cannot answer all research questions, and as the validity of RWE studies is established, a new perspective is emerging.

In an April 2019 commentary in Clinical Pharmacology & Therapeutics, Sebastian Schneeweiss, M.D., Sc.D., writes, “In the end, all discussions of data quality culminate in the same question: Are the data appropriate for this specific study question? Even in a narrow therapeutic area, no single data source or standardization method can answer all questions."

The question is not which data are superior, but which data are fit for purpose?

The question is not which data are superior, but which data are fit for purpose?


Open dialogue leads to trust

The open dialogue between Health Canada, CADTH, and INESSS, industry, and researchers is a model as all stakeholders develop a common language and strategies in working with RWE. Through these conversations, stakeholders are establishing trust in shared data and transparent methodologies. Together, we can improve access to treatments for all patients.