A proliferation of new data sources and sophisticated analytic methods are helping researchers transform real-world data (RWD) into real-world evidence (RWE) to address important healthcare questions. Regulators and policymakers recognize the power of policy to realize the full potential of data to improve patient care and outcomes.
In December 2024, FDA issued draft guidance on the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Guideline titled “E6(R3) Good Clinical Practice: Annex 2.” The ICH guideline addresses GCP considerations that arise from the use of a wider range of design elements and data sources, focusing on examples of trials that incorporate decentralized elements, pragmatic elements, and/or real-world data.
In July 2024, FDA issued draft guidance on the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Guideline titled “M14 General Principles on Plan, Design, and Analysis of Pharmacoepidemiological Studies That Utilize Real-World Data for Safety Assessment of Medicines.” The ICH guideline describes recommendations for post-marketing safety studies that use RWD.
In June 2023, the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) published a reflection paper titled “International Harmonisation of Real-World Evidence Terminology and Convergence of General Principles Regarding Planning and Reporting of Studies Using Real-World Data, with a Focus on Effectiveness of Medicines.” The ICH reflection paper seeks to enhance global regulatory harmonization and convergence for RWD/E.