In May 2024, the International Council for Harmonisation (ICH) released the M14 guideline “ICH M14 Guideline on general principles on plan, design and analysis of pharmacoepidemiological studies that utilize real-world data for safety assessment of medicines. This guideline focuses on the planning, design, and analysis of pharmacoepidemiological studies using real-world data (RWD) for the safety assessment of medicines, thus aligning global practices and supporting regulatory decisions.
As an ICH guideline, it aims to establish international standards and harmonise practices for conducting pharmacoepidemiological studies, enhancing their reliability and regulatory acceptance globally.
It includes recommendations for studies using RWD to evaluate post-marketing safety of medicinal products, such as drugs, vaccines, and biological products. The guideline also considers primary data collection when necessary for the study.
Notably, it provides a robust framework for generating reliable evidence from RWD, covering key aspects like study design, data sources, target populations, exposures, outcomes, and covariates. It addresses potential biases and confounding factors, ensuring the reliability of study results.
Detailed guidance is provided on developing study protocols, including considerations for data standardisation and data quality. It emphasises the importance of systematic feasibility assessments and clear research questions.
Additionally, the guideline outlines best practices for data management, including roles and responsibilities of data holders and researchers. It also covers statistical analysis methods, including handling of missing data and conducting sensitivity analyses.
The draft version of the guideline is currently open for public consultation until 30 August 2024. Stakeholders are encouraged to provide feedback to ensure comprehensive and effective guidance.
Here the complete document.