The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have jointly issued new guidance on the use of AI in the development and regulation of medications.

The guidance sets out 10 principles on AI use in evidence generation and monitoring across all phases of a medicine, from early research and clinical trials to manufacturing and safety monitoring.

The principles dictate that the development and use of AI technologies in drug development must align with ethical and human-centric values, and must follow a risk-based approach with proportionate validation, risk mitigation, and oversight based on the context of use and determined model risk.

The guidance also states there must be a well-defined context of use, and that multidisciplinary expertise covering both the AI technology and its context of use are integrated throughout the technology’s life cycle.

As expected, the principles advise that AI technologies adhere to relevant legal, ethical, technical, scientific, cybersecurity, and regulatory standards. Plain language information should be provided to users and patients regarding the AI technology’s context of use, performance, limitations, underlying data, updates, and Interpretability.

Monitoring and evaluation

Data source provenance, processing steps, and analytical decisions must be documented in a detailed, traceable, and verifiable manner, in line with good practice requirements. AI technologies must undergo scheduled monitoring and periodic re-evaluation to ensure adequate performance, including addressing data drift.

Commenting on the publication of the new recommendations, Olivér Várhelyi, European Commissioner for Health and Animal Welfare, said: “The guiding principles of good AI practice in drug development are a first step of a renewed EU-US cooperation in the field of novel medical technologies. The principles are a good showcase of how we can work together on the two sides of the Atlantic to preserve our leading role in the global innovation race, while ensuring the highest level of patient safety.”

The use of AI technologies in drug development and regulation has increased significantly in recent years.

As emphasised in the European Commission’s Biotech Act proposal, AI holds great promise as a tool to accelerate the path from innovation to safe and effective medicines. The new pharmaceutical legislation accommodates the broader use of AI in regulatory decision-making, and creates additional possibilities for testing innovative AI-driven methods for medicines in a controlled environment.

EMA comments

“To realise these benefits, AI needs to be expertly managed, including the mitigation of risks. As AI continues to evolve, a principles-based approach will help regulators, pharmaceutical companies and medicines developers harness the potential of these technologies while ensuring patient and animal safety and regulatory compliance. Over time, these principles will be supplemented by additional EU guidance taking into account the applicable legal requirements and relevant new EU legislation in the area of medicines,” according to a statement from the EMA.