Regulations For Artificial Intelligence in Drug Development and Clinical Trials in US and EU

Objective: To review and compare the current regulatory frameworks governing artificial intelligence (AI) applications in drug development and clinical trials in the United States and Europe, highlighting core principles, challenges, and future directions. Methods: Comprehensive review of official regulatory documents, draft guidances1, legislative acts, and guidance papers published between 2020 and 2025 from key authorities, including the US Food and Drug Administration (FDA), European Medicines Agency (EMA), and European Union’s Artificial Intelligence Act.2 Comparative analysis focused on risk assessment frameworks, lifecycle management, ethical considerations, and stakeholder engagement. Results: The FDA's 2025 draft guidance1 introduces a seven-step, risk-based credibility assessment framework emphasizing AI model validation, lifecycle oversight, transparency, and defined contexts of use (COU). The EU’s AI Act2 categorizes AI applications by risk levels and mandates rigorous requirements for “high-risk” AI in drug development. EMA’s 2023 Reflection Paper3 complements these with ethical and human oversight emphases. Both regions prioritize data integrity, bias mitigation, and proactive regulatory engagement. However, differences remain in procedural detail and scope, resulting in regulatory heterogeneity but ongoing convergence efforts. Conclusions: US and European regulatory frameworks provide robust foundations to ensure safe and effective AI integration in pharma, balancing innovation with patient safety. Continued harmonization, refinement, and collaborative dialogue among stakeholders will be essential to address evolving AI technologies and promote global adoption.