This guideline recommends international standards for, and promotes harmonization of, the general principles on planning, designing, analysing, and reporting of noninterventional studies that utilize fit-for-use data for safety assessment of medicines. The guideline outlines recommendations and high-level best practices for the conduct of non-interventional pharmacoepidemiologic safety studies to streamline the development and regulatory assessment of study protocols and reports.
This is the first harmonised guidance for non-interventional studies aimed at generating real-world evidence on the safety of medicinal products (key principles may also apply to effectiveness studies).
The guideline recommends an iterative approach to study development, focusing on assessment of data fitness-for-use, feasibility assessments to guide study design, and further refinement of design based on feasibility results.
The recommendations are intended to minimise the conduct of multiple studies on the same safety concern for regulatory submissions, improve the regulatory acceptance of the protocol across regions, and ultimately to support regulatory decision making.
Keywords: real-world data (RWD), pharmacoepidemiology, non-interventional studies, safety, post-authorisation