ICH M14 guideline published

The final version of the ICH M14 Guideline “General Principles on Planning, Designing, Analysing, and Reporting of Non-interventional Studies That Utilise Real-World Data for Safety Assessment of Medicines” has been published on the ICH Website.

This is the first harmonised guidance for non-interventional studies aimed at generating real-world evidence on the safety of medicinal products, while key principles can also be applied to effectiveness studies when RWD are included. 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. It also emphasises the importance of prespecifying and documenting key decisions around exposure, outcome, and covariate definitions, analysis plans, data management, and other aspects, and encourages transparency in study conduct, reporting, and dissemination of results. Interaction with regulators for key decisions throughout the study process is recommended. 

The ENCePP Guide on Methodological Standards in Pharmacoepidemiology has been extensively leveraged to develop the guideline, together with other relevant regulatory and non-regulatory guidance. For an overview and key features, please refer to the corresponding presentation.