Real-world evidence (RWE) can be of value to support comparative effectiveness of drugs and technologies by providing additional information about their use for a variety of patients in real contexts of care. However, the integration of RWE in appraisals can be challenging, and INESSS felt the need to reinforce and explicit the underlying methodological and theoretical foundations.

A comprehensive literature review was carried out, followed by collaborative development work by members of the methodological and assessment teams.

The literature review led to a common understanding of RWE underlying principles and fed the subsequent phases of the project. Three factors were identified as driving the integration of RWE in reimbursement appraisals at INESSS. Specifically, (i) the design and conduct of the real-world studies are done in accordance with best practices, (ii) the results are presented transparently and include all relevant information to assess the quality of the study and the data, and (iii) the RWE submitted is appropriate and relevant for decision-making. This third component is further ascertained by considering the decisional context (what are the circumstances motivating the submission of RWE and how does it correlate or not with existing evidence?), the data (is the dataset fit for decision needs?) and the study methods (are study design and analytical methods robust enough?). Globally, INESSS considers the integration of RWE in appraisals and its weighting, in relation with the (more traditional) available evidence, to be a case-by-case exercise.

The characterization of the main factors driving the integration of RWE in reimbursement appraisals at INESSS serves as a basis for communicating the requirements for evaluation submissions by sponsors. It further reinforces INESSS capabilities in assessing innovations, which can imply an appraisal of value at various moments along the lifecycle and with a diversity of evidence types. Considering the rapidly evolving literature and international experience, this work is expected to evolve too, and will be updated as needed.