Real-world evidence (RWE) is evidence about the use, safety, and effectiveness of a medical product, technology, or drug that is based on or derived from analysis of data generated in a real-world health care setting.
Real-world data (RWD) includes information about the health of individuals or the delivery and/or outcomes of health care that is collected outside of traditional clinical trials and thus reflects results within the context of the particular health care system.
Potential sources of RWD include:
RWD can be collected by patients and patient groups, physicians and other health care providers, regulatory agencies, governments, and pharmaceutical and other private companies. The data may vary in quality and are not always fit for the purpose of informing decision-making.
Real-world evidence comes from the analysis of RWD. However, RWD can only serve as evidence for decision-making if it is considered to be relevant and reliable information.
Randomized controlled trials are often considered the gold standard method of gathering evidence about the safety and efficacy of a drug or treatment. In these types of studies, a precisely defined group of patients are enrolled and randomly assigned to either a group receiving the intervention being studied or to a control group who do not receive that intervention.
Clinical trials may have limitations, including the following:
Real-world evidence can be used to:
Real-world evidence has the potential to fill gaps in clinical evidence that are generated in clinical trials and can facilitate a more comprehensive assessment of the safety, efficacy, and effectiveness of drugs or health technologies to a broader population, over a longer time period, and which considers the context of the Canadian health care system.
The role of real-world evidence can be especially important to meet the challenge of assessing drugs submitted for approval to treat rare disorders. In these cases, it can be challenging to develop robust clinical evidence due to the small number of patients affected by the condition, the impracticality of conducting a randomized controlled trial, or ethical concerns.
Real-world evidence can help decision-makers determine if the conditions or scope of use for a product should be revised. It can also help determine the optimal order of drugs within a drug sequence to optimize their benefits. Finally, real-world evidence can help health care providers and patients determine whether a drug is the right choice to meet their treatment goals.
It is also important to evaluate a drug or intervention throughout its life cycle is increasingly recognized. Real-world evidence can support the ongoing assessment of a product’s effectiveness and safety within the Canadian health care setting.
For more insight on the integration of real-world data and evidence to support decision-making in the pharmaceutical sector, please see a recent State of Knowledge report from the Institut national d’excellence en santé et en services sociaux (INESSS). The report from INESSS, translated by CADTH, provides a narrative review of the literature on the use of real-world evidence and real-world data in assessments to support the decision-making process in formulary listing drug reviews. The state of knowledge document is solely for information purposes and does not constitute a position paper or recommendations.
Real-world evidence is currently being used in Canada to:
Challenges associated with using real-world evidence to inform decision-making include: