Guidance for Reporting Real-World Evidence (RWE) [published May 2023] is a comprehensive document developed by the Canadian Agency for Drugs and Technologies in Health (CADTH) and Health Canada to provide clear guidance on the conduct, reporting, and appraisal of RWE studies for regulatory approval and health technology assessment (HTA) in Canada.

Guidance Objectives:

1. Ensure regulators and HTA agencies have ample information for evaluating a study’s appropriateness for decision-making.
2. Establish core RWE study reporting standards that align globally.
3. Prioritize transparent reporting while acknowledging RWD and RWE challenges.

Implementation: Given the diverse sources and uses of RWD, this guidance offers flexibility for various RWE applications. Some RWE studies might not need all components described here. The guidance anticipates updates as the RWE field evolves.

RWE Utility:

While randomized controlled trials (RCTs) are the gold standard for establishing the safety and efficacy of health technologies, they often lack wider applicability due to specific target populations. RWE, derived from RWD, has benefits like the inclusion of under-represented populations in trials, such as children, older adults, and diverse ethnic groups. Its expanded sample sizes and longer follow-ups offer a richer dataset than clinical trials, providing insights into healthcare provider and patient perspectives about accessibility and usability of health technologies. For instance, RWE is instrumental in pharmacovigilance and monitoring adverse events.

RWE Limitations: Concerns include biases, confounding factors, and the lack of rigorous outcome ascertainment as seen in clinical trials. Clear standards are essential for transparent reporting of RWE.

RWE Guidance Structure: Guidance is provided for the reporting of 12 study components as follows:

1. Research Questions and Study Design
2. Setting and Context
3. Data Specifications, Access, Cleaning Methods, and Linkage
4. Data Sources, Data Dictionary, and Variables
5. Participants
6. Exposure Definitions and Comparators
7. Outcomes
8. Bias, Confounding, and Effect Modifiers or Subgroup Effects
9. Statistical Methods
10. Study Findings
11. Interpretation and Generalizability
12. Limitations

In summary, this recent guidance aims to harmonize global and Canadian reporting standards for RWE through expert consensus. It includes a Recommendations Checklist to ensure compliance and is designed to be adaptable across different types of RWE studies, laying the groundwork for best practices and methodological rigor in using RWE for regulatory and HTA purposes.