ICH M14 guideline on general principles on plan, design and analysis of pharmacoepidemiological studies that utilize real-world data for safety assessment of medicines - Scientific guideline

The use of pharmacoepidemiological studies as a source of evidence for regulatory decision-making has increased globally, and multiple guidelines and best practice documents have been developed by health authorities and professional societies. Generation of robust evidence to be used for regulatory purposes relies on the quality of the data and the application of sound pharmacoepidemiological methods. 

This guideline provides internationally harmonised guidance and outlines recommendations and high-level best practices for the conduct and analysis of non-interventional pharmacoepidemiological studies using fit-for-purpose data for the assessment of the safety of medicines (drugs, vaccines, and other biological products). It aims to streamline the development and regulatory assessment of study protocols and reports, and improve their ability to be accepted across health authorities.

To ensure that all concerned European stakeholders are given the opportunity to bring forward their views on technical and operational aspects to advance the guideline work, EMA is coordinating a European public consultation until 30 August 2024, in parallel to the global public consultation launched by ICH.

Keywords: real-world data (RWD), pharmacoepidemiology, non-interventional studies, safety, post-authorisation

Current version

ICH M14 guideline on general principles on plan, design and analysis of pharmacoepidemiological studies that utilize real-world data for safety assessment of medicines Step 2b

Consultation dates: 30/05/2024 to 30/08/2024Draft: consultation openReference Number: EMA/CHMP/ICH/155061/2024Summary:

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