FDA Guidance on Real-World Data for Medicine Safety Assessment

FDA Guidance Documents

Published March 3rd, 2026

Detected March 4th, 2026

Summary

The FDA has issued final guidance on using real-world data for medicine safety assessments, replacing a previous draft and withdrawing an older guidance. This document provides principles for planning, designing, analyzing, and reporting non-interventional studies utilizing real-world data for drugs and biological products.

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What changed

The FDA has released a final guidance document, "General Principles on Planning, Designing, Analyzing, and Reporting of Non-interventional Studies That Utilize Real-World Data for Safety Assessment of Medicines." This guidance, developed under the International Council for Harmonisation (ICH), outlines best practices for using real-world data in postmarketing safety studies for drugs and biological products. It replaces the July 2024 draft guidance and withdraws the May 2013 guidance on pharmacoepidemiologic studies. The document covers research question articulation, data source selection, bias mitigation, analysis, reporting, and submission.

This guidance provides a framework for pharmaceutical companies and drug manufacturers to conduct and report non-interventional safety studies using real-world data. While focused on safety, the principles are applicable to effectiveness assessments and complement existing FDA guidance on real-world evidence. Companies should review the guidance to ensure their study designs and reporting practices align with the recommended principles to streamline regulatory assessment of postmarketing safety studies.

What to do next

Review the final guidance on real-world data for medicine safety assessment.
Update internal protocols for planning, designing, analyzing, and reporting non-interventional safety studies using real-world data.
Ensure alignment with ICH principles and FDA recommendations for postmarketing safety studies.

Source document (simplified)

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Guidance Documents

Docket Number: FDA-2024-D-2754 Issued by: Guidance Issuing Office Center for Drug Evaluation and Research Center for Biologics Evaluation and Research 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.