EMA, FDA Set Global Principles for AI in Medicines

The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have jointly agreed on ten principles for good artificial intelligence (AI) practice across the medicines lifecycle, marking a significant step toward global regulatory alignment in the use of AI in drug development and oversight. The principles are intended to guide the responsible use of AI technologies from early-stage research and clinical trials through manufacturing, regulatory decision-making, and post-market safety monitoring.

The newly identified principles provide broad, high-level guidance on how AI should be applied in evidence generation and continuous monitoring of medicines. They are relevant not only for pharmaceutical and biotechnology companies developing new therapies, but also for marketing authorisation applicants and holders that are increasingly relying on AI-driven tools to support data analysis, trial design, quality control, and pharmacovigilance.

Regulators said the principles will serve as a foundation for future, more detailed AI-related guidance in both jurisdictions, while also supporting enhanced international collaboration among regulatory authorities, standards-setting organisations, and other stakeholders. In the European Union, work is already underway to translate these principles into formal guidance, building on the EMA’s AI reflection paper published in 2024.

European Commissioner for Health and Animal Welfare Olivér Várhelyi described the initiative as an important milestone in renewed EU–US cooperation on novel medical technologies. He said the principles demonstrate how transatlantic collaboration can help maintain global leadership in pharmaceutical innovation while ensuring the highest standards of patient safety.

The use of AI across the medicines lifecycle has grown rapidly in recent years, reflecting advances in data science and computing power. As highlighted in the European Commission’s proposed Biotech Act, AI has the potential to significantly shorten development timelines and improve the efficiency of bringing safe and effective medicines to patients. The EU’s new pharmaceutical legislation also creates opportunities to test innovative, AI-driven approaches in controlled regulatory environments.

However, regulators emphasized that realizing these benefits requires careful governance and risk mitigation. A principles-based approach is intended to help regulators and industry manage challenges such as data quality, transparency, bias, and accountability, while remaining flexible as AI technologies continue to evolve.

The EMA–FDA initiative builds on discussions from the FDA–EU bilateral meeting held in April 2024 and aligns with the EMA’s broader strategy to 2028, which prioritizes data, digitalisation, and AI. With ethics and patient safety at the forefront, the agencies said they will continue to pursue global convergence on AI-related topics in close collaboration with international public health partners.