anada has a long history of developing the requisite infrastructure and research expertise to collect and assess administrative data and other real world insights. Processes for using this information have often been laborious and, thus, application has frequently been limited to decision making at the provincial and/or federal government level.

However, we’ve seen significant evolution of technology, systems, tools, standards, and methodologies related to real world evidence (RWE) generation over the past several years. In addition, regulators around the world are giving very serious consideration to the use of RWE to inform their decisions. It is important that all key players in the Canadian pharmaceutical system align to these developments, to ensure timely patient access to medications that is consistent with other jurisdictions.

Regulatory

Health Canada initiated the Regulatory Review of Drugs and Devices (R2D2) modernization process in 2017. This initiative specifically states the department’s intention to “make better use of real world evidence to support regulatory decisions across a product’s life cycle for both drugs and medical devices.” For example, a specific project has been identified—Strengthening the use of real world evidence for drugs—with the goal of identifying opportunities for enhanced use of RWE throughout the drug life cycle, mapping potential RWE sources, developing and implementing a RWE strategy and implementation plan for the use of RWE in regulatory decision making for drugs, and consulting with stakeholders on the RWE strategy. This project is expected to be completed by fall 2022.

Health Canada’s work must be put into the context of international developments. For instance, for the past two years, the FDA has more frequently commented on the role of RWE for evaluating drug safety and effectiveness. Janet Woodcock and colleagues recently published an overview of the program being developed, stemming from the 21^st Century Cures Act, to evaluate the use of RWE to support approval of new indications for approved drugs or to satisfy post-approval study requirements. This is expected to build significantly on drug safety work which leverages claims and pharmacy data in its Sentinel System. These evolving approaches to RWE that will eventually be promoted by the FDA through this program will set a new standard for all other regulators.

Health Technology Assessment (HTA)

Historically, Canadian HTA bodies have been somewhat reluctant to consider RWE in their appraisal processes. Recent HTA reviews and the new mandate for Canada’s largest HTA organization—the Canadian Agency for Drugs and Technologies in Health (CADTH)—imply an emerging role for RWE in evidence assessment and outcomes evaluation.

In the context of individual HTA reviews, two recent recommendations for cancer medications suggest a “conditional” reimbursement approach linked to future and/or ongoing evidence generation. This represents a very new type of recommendation, one that certainly opens the door to coverage with evidence development (including reassessment of products) and creates a potential link for RWE to inform reassessments and/or product listing agreements.

Earlier this year, CADTH released its 2018 – ‘21 strategic plan, Transforming How We Manage Health Technologies. This plan will move CADTH from an HTA organization toward a health technology management (HTM) paradigm. HTM is broader than HTA: It encompasses assessment at all phases of a technology’s life cycle, increased collaboration and engagement with a broad range of stakeholders, and comprehensive implementation support to ensure that best practices actually drive decisions.

It is expected that RWE would have a prominent role in delivering this new HTM mandate, both in terms of evidence assessment as well as evaluation of outcomes after implementation of HTA recommendations.

Pricing Negotiations

The pan-Canadian Pharmaceutical Alliance (pCPA) undertakes pricing negotiations for brand name and generic drugs on behalf of publicly funded drug programs across Canada. The pCPA has hinted that it sees the potential for performance- and/or outcomes-based product listing agreements (PLA) for certain products, which points to a possible role for RWE in monitoring and/or assessing the impact of these PLAs. The potential format of these PLAs (e.g., conditional reimbursement, managed access plans, coverage with evidence development, etc.) is not currently clear; however, the focus on outcomes implies that RWE may be an important component within them.

Other Considerations

It is important for Canadian decision makers to monitor international developments, and to learn from and leverage those developments to inform their own policy positions and ensure alignment. Taking an approach to RWE in Canada that is significantly different from global practices will ultimately undermine the efforts of decision-makers and companies involved in RWE activities.

Stakeholder engagement must be integrated into the assessment of options and models for RWE initiatives, from both a policy and an operational perspective. As consistently demonstrated by the US’ Patient-Centered Outcomes Research Institute (PCORI), patients and clinicians must have a central and very active role in research prioritization, project design (e.g., what outcomes are most important to measure) and interpretation of RWE efforts.

Capacity for RWE in Canada continues to expand. The recent launch of the Alberta Real World Evidence Consortium is an example of new resources being targeted to this field of research. Its mandate is to support the development of quality RWE generation and utilization, by leveraging Alberta’s large, mature and comprehensive health system data depositories. The Consortium intends to serve a broad client base, giving industry and other health system partners access to coordinated resources, expertise, processes, and data to ensure high-quality RWE studies.

Implications

• After many years of discussion, Canada now seems primed to make decisions on the role that RWE could and will play in the pharmaceutical ecosystem.
• Canadian decision-makers must ensure that policies and processes are set up to accept and leverage these data in making sound decisions that support patient access to medications. Transparency and consistency in the manner that RWE informs these assessments and decisions will be key.
• Learnings from global and local experiences must be brought to bear to create an environment for RWE that addresses the needs of Canadian patients, providers, and policy-makers.

References available upon request.