Real-World Evidence for Decision-Making

Real-World Evidence

Real-world evidence (RWE) is evidence about the use, safety, and effectiveness of a drug, medical product, or technology based on data from a real-world health care setting. It is playing an increasingly important role in health care policy and practice decision-making.

To learn more about RWE, read Real-World Evidence Primer.

Guidance for Reporting Real-World Evidence

We have partnered with Health Canada, the Institut national d’excellence en santé et en services sociaux (INESSS), and other health system stakeholders to advance the integration of real-world evidence (RWE) into decision-making. Guidance for Reporting Real-World Evidence lays the foundation for the use of RWE in regulatory approval and Health Technology Assessment (HTA) in Canada, starting with the principles for transparent reporting of RWE studies.

Guidance for Reporting Real-World Evidence

Partnerships and Stakeholders

We serve as the Chair of the collaborative Real-World Evidence Steering Committee. The committee is helping to develop a strategic framework for Canada’s use of RWE to support decision-making.

The Real-World Evidence Steering Committee consists of the following working groups:

• The Guidance for Reporting Real-World Evidence Working Group developed clear and comprehensive guidance on the conduct, reporting, and appraising of RWE studies concerning the safety and effectiveness of health technologies for regulatory approval and health technology assessment in Canada.
• The Real-World Evidence Oncology Working Group provides advice and support on the optimal use of RWE for health care decision-making related to oncology therapeutics in Canada and the planning and execution of learning projects.
• The Real-World Evidence Non-Oncology Working Group provides advice and support on the optimal use of RWE for health care decision-making concerning non-oncology therapeutics in Canada and the planning and execution of learning projects.

We also engage with several types of stakeholder groups to support and provide guidance on the optimal use of RWE. These groups include:

• commercial RWE service providers
• private drug plans
• rare disease research networks and businesses
• governments
• health system partners
• general public
• patients with rare disease
• rare disease patient organizations
• pharmaceutical industry
• health care professionals
• rare disease data holders
• researchers and academics.

Drugs for Rare Diseases

We share with Health Canada the challenge of evaluating drugs for the treatment of smaller populations or rare disease and has recognized the need to develop knowledge, capabilities, and competencies related to RWE to meet this challenge.

During a presentation at the 2021 CADTH symposium entitled Real-World Evidence and Health Technology Assessment: Past, Present, and Future, we launched a learning period to better understand how to optimize the use of RWE to inform decision-making for drugs for rare diseases.

This learning period launched the creation of the Best Brains Exchange (BBE) co-hosted by us, the Canadian Institutes of Health Research (CIHR), and the Canadian Organization for Rare Disorders (CORD). Learnings from the BBE and from subsequent consultations with a variety of health system stakeholders helped us identifying 4 pillars necessary for optimal integration of RWE into decision-making processes.

Development of these 4 pillars and related activities were presented at our Information Day in April 2022 and will support optimal integration of RWE into decision-making processes. They are:

1. multi-stakeholder engagement and dialogue
2. real-world data generation and access
3. collaborative partnerships
4. guidance for RWE.

Learning Projects

Our learning projects for RWE fall into the following categories:

• RWE to support optimization of care for rare diseases:
  • pediatric glioma
  • pediatric spinal muscular atrophy
  • amyotrophic lateral sclerosis
  • primary biliary cholangitis
  • pediatric cystic fibrosis
• RWE to support optimal use of drugs:
  • plaque psoriasis
  • multiple myeloma
  • ulcerative colitis
  • ozempic
  • dementia real-world evidence bundle for health system readiness:
    • Clinician Panel: Understanding the Portrait of Dementia Care in Canada: Our Panel of Experts
    • Patient Panel: Dementia Concerns and Considerations: Our Panel of People with Lived Experience
    • Canadian Medical Imaging Inventory:
      • Canadian Medical Imaging Inventory Service Report: The Role of Neuroimaging in Drugs Targeting Amyloid-Beta in Alzheimer Disease: Part 1
      • Canadian Medical Imaging Inventory Service Report: The Role of PET-CT in Drugs Targeting Amyloid-Beta in Alzheimer Disease: Part 2
    • Devices: An Overview of New and Emerging Technologies for Early Diagnosis of Alzheimer Disease
    • Pharmaceuticals: Utilization of Cholinesterase Inhibitors for Alzheimer Disease in Canada
    • Infusion Clinics: The Availability of Privately Funded IV Infusion Clinics in Canada

Case Studies

We are actively gathering case studies from organizations that generate or integrate RWE into their work, including:

• Reflections on the Canadian Bleeding Disorders Registry: Lessons Learned and Future Perspectives.
  • The Value of Patient Registries: Common Needs for Rare Diseases Editorial

International Initiatives

We are currently participating in several international RWE and real-world data initiatives, including:

• Council for International Organisations of Medical Sciences working groups, including the Working Group XIII on Real-World Data and Real-World Evidence in Regulatory Decision-Making
• International Network of Agencies for Health Technology Assessment (INAHTA) Real-World Evidence Learning Group
• International Society for Pharmacoepidemiology (ISPE) Real-World Evidence Task Force
• RWE4Decisions – Real-World Evidence for Decisions.

Contact

If you have questions about our work to support RWE for decision-making, contact us at [email protected].