Regulatory Intelligence - 2025 Round Up
An overview of the key regulatory intelligence updates from FDA & EMA released in 2025.
Food and Drug Administration (FDA)
The FDA issued guidance on considerations for the use of Artificial Intelligence to support regulatory decision-making for Drug and Biological Products
The FDA issued a draft guidance document entitled ‘Considerations for the Use of Artificial Intelligence to Support Regulatory Decision-Making for Drug and Biological Products’. This guidance provides recommendations to sponsors and other interested parties on the use of artificial intelligence (AI) to produce information or data intended to support regulatory decision-making regarding safety, effectiveness, or quality for drugs. Specifically, this guidance provides a risk-based credibility assessment framework that may be used for establishing and evaluating the credibility of an AI model for a particular context of use (COU).
The ICH adopted E6(R3) Guideline on Good Clinical Practices
The International Council for Harmonisation (ICH) adopted its E6(R3) guideline, which aims to harmonize the framework for conducting clinical studies. The guideline received endorsement from the ICH Assembly, reaching Step 4 of the ICH process and indicating it is ready for adoption by regulatory authorities. The document was initially released as Step 2 and made available for public consultation on 19th of May 2023. The guideline includes some minor revisions from the draft version, including the addition of new language on data governance in Section 4. These include a new section 4.2.7 on retention and access to trial data and a new Section 4.2.8 on destruction of trial data. New subsections have been added to the section on computer systems, including Sections 4.3.3 on security, 4.3.4 on validation, 4.3.5 on release of trial-specific systems, 4.3.6 addressing computer system failures, 4.3.7 on technical support, and 4.3.8 on user management.
The FDA announced a plan to phase out the requirement for animal testing for monoclonal antibodies and other drugs
The FDA took a groundbreaking step to advance public health by replacing animal testing in the development of monoclonal antibody therapies and other drugs with more effective, human-relevant methods. The FDA’s animal testing requirement will be reduced, refined, or potentially replaced using a range of approaches, including AI-based computational models of toxicity and cell lines and organoid toxicity testing in a laboratory setting (so-called New Approach Methodologies or NAMs data). Implementation of the regimen will begin immediately for investigational new drug (IND) applications, where inclusion of NAMs data is encouraged, and is outlined in a roadmap also being released today. To make determinations of efficacy, the agency will also begin use pre-existing, real-world safety data from other countries, with comparable regulatory standards, where the drug has already been studied in humans.
The FDA published guidance on technical specifications for submitting clinical trials
The FDA published final guidance on technical specifications for submitting clinical trials for response assessments for treatments of acute leukemias. The guidance provides recommendations for datasets containing response assessment elements and summary-level efficacy outcomes in New Drug Applications (NDAs) and Biologics License Applications (BLAs). These specifications complement existing disease-specific guidance on clinical development and efficacy endpoints and focus solely on data elements related to efficacy evaluations in acute leukemias.
The FDA published draft guidance on scientific considerations in demonstrating biosimilarity to a reference product
The FDA published a draft guidance titled, “Scientific Considerations in Demonstrating Biosimilarity to a Reference Product: Updated Recommendations for Assessing the Need for Comparative Efficacy Studies (CES)”. This draft guidance describes considerations regarding a comparative clinical study or studies with efficacy endpoints (a CES) to support a demonstration of biosimilarity in a biologics license application (BLA) submitted under section 351(k) of the Public Health Service (PHS) Act. Although the 351(k) pathway generally applies to all biological products, this guidance focuses on therapeutic protein products, providing an overview of important scientific considerations for determining when a CES may inform a demonstration of biosimilarity.
The FDA unveiled the new plausible mechanism pathway
The Food and Drug Administration (FDA) published a paper on plausible mechanism pathway; a regulatory framework to accelerate approval of bespoke, patient-specific therapies when traditional trials are not feasible. The paper highlights that the marketing authorization will be considered after treatment of “several consecutive” patients under single patient investigational new drug (IND)/expanded access programs. Manufacturers will be able to leverage platform data from similar personalized products. As a post-marketing commitment, marketing authorization holders will be required to collect real world evidence to confirm efficacy and show lack of off-target edits/detect other safety signals. While it was originally indicated that the pathway would be for ultra-rare diseases, the article indicates that it will also be available for common diseases particularly those with a high unmet need. The pathway is also not limited to gene editing therapies, and the possibility of extending to other therapeutic classes is raised.
European Medicines Agency (EMA)
The EMA updated procedural advice for Orphan Medicinal Product Designation: Guidance for Sponsors
The European Medicines Agency (EMA) updated the “Procedural Advice for Orphan Medicinal Product Designation: Guidance for Sponsors” document. This document overviews and provides guidance for sponsors seeking to obtain an orphan medicinal product designation and has been updated for 2025.
The EMA released a draft guideline to support the inclusion of pregnant and breastfeeding individuals in clinical trials
The EMA published a draft guideline for public consultation, developed jointly with global regulators through the International Council for Harmonisation (ICH), aiming to promote the inclusion and retention of pregnant and breastfeeding individuals in clinical trials. This initiative seeks to address the current underrepresentation of these populations in clinical research – data from the Clinical Trials Information System (CTIS) indicate that less than 0.4% of EU clinical trials include pregnant participants, and only 0.1% include breastfeeding individuals. The guideline outlines scientific, regulatory, and ethical principles to ensure participant safety and data robustness, encouraging early engagement between medicine developers and regulatory authorities. Public consultation is open until the 15th of September 2025.
The EMA expanded the SEND data submission in proof-of-concept study for non-clinical dossiers
The EMA updated its guidance on the Standard for Exchange of Non-clinical Data (SEND) format, expanding its use in the proof-of-concept study for initial centralised marketing authorisation applications. This initiative aims to standardise the submission of non-clinical safety data, including toxicity, safety pharmacology, and reproductive studies, to enhance the efficiency and consistency of regulatory assessments. The EMA encourages voluntary participation from applicants and provides detailed instructions on the submission process.
The EMA published the ICH M14 guideline on Real-World Data
The EMA adopted the final Step 5 version of the International Council for Harmonisation (ICH) M14 guideline, providing international standards for planning, designing, analysing, and reporting non-interventional studies that utilise real-world data (RWD) for the safety assessment of medicines. The guideline offers harmonised standards on study design, data quality, transparency, and regulatory interaction to ensure the reliable use of observational data in post-marketing safety evaluations, enhancing the acceptance of real-world evidence. It will come into effect on the 18th of March 2026.
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