US and European medicines regulators have jointly issued a set of guiding principles to support the safe, effective and responsible use of artificial intelligence (AI) in drug development, marking a significant step toward regulatory alignment on emerging technologies.

The principles, developed by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), are intended to apply across the entire medicines lifecycle — from early research and non-clinical studies to clinical development, manufacturing and post-market monitoring. The framework seeks to encourage innovation while ensuring patient safety and the reliability of scientific evidence generated using AI tools.

Regulators emphasized that AI systems used in drug development should be fit for purpose, transparent, and supported by robust data governance and risk management practices. The principles stress the importance of clearly defining the role of AI within development processes, maintaining human oversight, and ensuring that AI-driven outputs can be explained, validated and reproduced.

The guidance also highlights the need for lifecycle management of AI models, recognizing that systems may evolve over time as new data are introduced. Developers are encouraged to continuously monitor performance, address potential biases, and manage changes in a controlled and documented manner.

By aligning on shared expectations, the FDA and EMA aim to reduce regulatory uncertainty, support global drug development programs, and facilitate the responsible adoption of AI technologies that have the potential to shorten development timelines, improve decision-making, and enhance the efficiency of pharmaceutical research.

The principles are expected to inform future regulatory guidance and help companies integrate AI into drug development in a way that balances technological advancement with high standards of quality, safety and scientific integrity.