M14 General Principles on Planning, Designing, Analyzing, and Reporting of Non-Interventional Studies That Utilize Real-World Data for Safety Assessment of Medicines; International Council for Harmonisation; Guidance for Industry; Availability

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled "General Principles on Planning, Designing, Analyzing, and Reporting of Non- interventional Studies That Utilize Real-World Data for Safety Assessment of Medicines." The guidance was prepared under the auspices of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH). The guidance outlines general principles on planning, designing, analyzing, and reporting non-interventional studies that utilize real-world data for safety assessment of medicines (i.e., drugs, vaccines, and other biological products). The recommendations in this guidance, while focused on safety, are applicable to non-interventional studies assessing effectiveness and are aligned and complementary with the FDA's other guidances on the generation real world evidence. The guidance includes recommendations and high-level best practices for the conduct of these studies, including articulating the research question, selecting appropriate data sources, defining key variables, addressing potential biases and confounding, conducting analyses, reporting, and submission. The guidance is intended to streamline the development and regulatory assessment of postmarketing non-interventional safety studies that include real-world data. The guidance replaces the draft guidance "M14 General Principles on Plan, Design, and Analysis of Pharmacoepidemiological Studies that Utilize Real-World Data for Safety Assessment of Medicines" issued in July 2024. FDA is also announcing the withdrawal of the guidance entitled "Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Healthcare Data Sets," published May 2013.

Abstract as published in the Federal Register.

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