Draft ICH M14 Guideline on Real-World Data for Safety Assessment

Following the previously published ICH reflection paper on Real-World Evidence (RWE) and Real-World Data (RWD) to support regulatory decision-making, the ICH M14 Guideline has been published in May for public consultation. The guideline will focus on non-interventional pharmacoepidemiological studies using RWD and will include basic principles that may apply to these studies when RWD elements are included. In addition, the document provides internationally harmonised guidance and outlines recommendations and high-level best practices for the conduct and analysis of non-interventional pharmacoepidemiological studies using fit-for-purpose data for the assessment of the safety of medicines (i.e., drugs, vaccines, and other biological products).

Synergies are foreseen with other ICH guidelines, for example E6(R3) and its Annex II, as well as ICH M11 (CeSHarP), currently under development. The ICH guideline M14 focuses on convergence of guidance and best practices across jurisdictions on planning and designing safety studies that use RWD, whereas the potential for RWE can be broadened to include assessing the effectiveness of medicines and analysing utilisation of marketed medicines administered in routine medical practice. The ICH guideline M11 covers general protocol design principles and approach used to develop the separate associated documents, i.e. the ICH M11 Clinical Electronic Structured Harmonised Protocol Template and the Technical Specification, that are acceptable to all regulatory authorities of the ICH regions. The scope of the M11 guideline focuses on protocol of clinical trials only, whereas RWD have been used mostly in non-interventional studies.

Comments should be provided to EMA by 30 August 2024. For more information please see the draft ICH M14 Guideline on General Principles on Plan, Design and Analysis of Pharmacoepidemiological Studies That Utilize Real-World Data for Safety Assessment of Medicines.