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ICH M14 Guideline: General Principles on Plan, Design and Analysis of Pharmacoepidemiological Studies That Utilize Real-World Data for Safety Assessment of Medicines

Recently the International Council for Harmonisation (ICH) announced on 24 May, 2024 the availability of draft guidance for the industry entitled “General Principles on Plan, Design and Analysis of Pharmacoepidemiological Studies That Utilize Real-World Data for Safety Assessment of Medicinesthat guides on how real-world data (RWD) can be used to support drug development.  


Epidemiological studies are a key component in detecting, characterizing, and evaluating safety concerns or signals and may be descriptive or inferential.


The use of pharmacoepidemiological studies for regulatory decision-making has increased globally, and health authorities and professional societies have developed multiple guidelines and best practice documents.


This guideline is crucial for ensuring the reliability and validity of studies that inform regulatory decisions and enhance patient safety. This guidance aims to recommend international standards and promote harmonization of general principles for planning, designing, and analyzing observational (non-interventional) pharmacoepidemiological studies that use fit-for-purpose data to assess the safety of medicines, including drugs, vaccines, and other biological products.


Real-world data refers to health-related information collected from various sources outside of traditional clinical trials. These sources can include electronic health records (EHRs), insurance claims, patient registries, and even data from wearable devices. Integrating RWD into pharmacoepidemiological studies allows for a more comprehensive understanding of how medicines perform in everyday settings, providing valuable insights into their safety profiles.

This guidance provides a framework for developing adequate evidence through RWD, identifies the research question that these studies will address, identifies fit-for-purpose data to address a particular research question through feasibility assessments, and develops a protocol for designing and conducting pharmacoepidemiological safety studies with the help of experts in the field.


As the use of RWD continues to grow, adherence to the ICH M14 principles will be crucial for maintaining the integrity and reliability of safety assessments in the pharmaceutical industry. For more detailed information, refer to the full guidelines available here.


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