Artificial intelligence (AI)

Artificial intelligence (AI) refers to computer systems designed to perform tasks that typically require human intelligence, including learning, reasoning, language processing, pattern recognition, and decision-making. Artificial intelligence comprises computational approaches such as machine learning, deep learning, natural language processing, generative AI, and other methods that perform cognitive tasks.

In clinical research, artificial intelligence is applied to activities such as protocol development, patient recruitment, medical imaging analysis, pharmacovigilance, data management, statistical analysis, and the generation or review of clinical documentation.

Scope

AI describes a class of technologies rather than a single method or product. Depending on the intended use, AI systems may function as decision-support tools, automate routine tasks, or generate content for human review.

Applications of artificial intelligence span the medicinal product lifecycle, including drug discovery, non-clinical research, clinical development, regulatory affairs, pharmacovigilance, and post-marketing activities.

Regulatory responsibilities

The use of artificial intelligence in clinical research does not replace existing obligations established by good clinical practice (GCP), clinical trial legislation, pharmacovigilance requirements, data protection legislation, or other applicable regulatory requirements. Sponsors and investigators remain responsible for regulatory compliance, participant safety, data integrity, and the reliability of data submitted to regulatory authorities.

Regulatory environment

The regulatory oversight of artificial intelligence in drug development and clinical research is evolving. Current requirements derive from several distinct categories of legislation, regulatory frameworks, initiatives, and voluntary standards. These categories are frequently discussed together but serve different purposes.

AI legislation

Within the European Union, Regulation (EU) 2024/1689 (Artificial Intelligence Act) 1 establishes a legal framework for AI systems based on a risk classification approach. Depending on their intended purpose and use, AI systems employed in drug development, clinical research, or healthcare may be subject to obligations defined by the Regulation.

Additional jurisdictions are developing legislation and regulatory policies governing the development, deployment, and oversight of artificial intelligence.

Existing regulatory frameworks

Artificial intelligence used in clinical research remains subject to existing pharmaceutical legislation and GxP requirements where applicable. These frameworks were not developed specifically for AI but continue to apply according to the intended use of the system.

Examples include:

  • ICH Good Clinical Practice (E6) and other applicable ICH guidelines governing clinical trials. At present, ICH has not published an AI-specific guideline
  • Regulation (EU) No 536/2014 (Clinical Trials Regulation) governing clinical trials conducted in the European Union
  • General Data Protection Regulation (GDPR), where AI systems process personal data
  • EU GMP Annex 11 and 21 CFR Part 11, where AI forms part of computerized systems used to create, modify, maintain, archive, retrieve, or transmit regulated electronic records
  • National legislation applicable within individual jurisdictions

Regulatory initiatives

Regulatory authorities have published strategic documents and initiated programmes addressing the use of artificial intelligence in medicines regulation.

The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) have identified artificial intelligence as a strategic priority for medicines regulation. The HMA–EMA Multi-annual Artificial Intelligence Workplan 2023–2028 2 outlines planned activities related to regulatory guidance, scientific evaluation, data governance, and the use of AI throughout the medicinal product lifecycle.

The U.S. Food and Drug Administration (FDA) has incorporated artificial intelligence into selected internal regulatory activities. In 2025, the agency introduced Elsa 3, a generative AI platform intended to assist FDA personnel with activities including scientific review, clinical protocol evaluation, adverse event assessment, inspections, and administrative tasks. According to the FDA, the platform operates under human oversight and is not trained on confidential information submitted by regulated industry.

The FDA also issued draft guidance for industry in 2025 on using artificial intelligence to generate information or data for regulatory decisions about the safety, effectiveness, or quality of drugs. The Considerations for the Use of Artificial Intelligence 4 guidance proposes a risk-based approach for assessing the credibility of an AI model for a specific context of use.

International organizations have also begun developing recommendations for the use of artificial intelligence in regulated activities. The Council for International Organizations of Medical Sciences (CIOMS) established Working Group XIV 5 to develop guidance on the use of artificial intelligence in pharmacovigilance. The group’s draft report discusses governance, validation, transparency, human oversight, documentation, and risk management for AI systems used in pharmacovigilance activities.

Voluntary standards and industry guidance

Several organizations have published standards and guidance relevant to the governance, quality management, and lifecycle management of artificial intelligence. These documents do not constitute legally binding regulatory requirements but may be used to support organizational governance and risk management.

Examples include:

  • ISPE GAMP 5 guidance for GxP computerized systems 6
  • ISO/IEC 42001, Artificial intelligence – Management system 7
  • NIST Artificial Intelligence Risk Management Framework (AI RMF) 8
  • WHO guidance: Ethics and governance of artificial intelligence for health 9

Related terminology

  • Clinical decision support system
  • Computerized system
  • Software as a medical device (SaMD)

References

  1. European Parliament and Council of the European Union. 2024. Regulation (EU) 2024/1689 (Artificial Intelligence Act). Available at: https://eur-lex.europa.eu/eli/reg/2024/1689/oj/eng ↩︎
  2. HMA–EMA Big Data Steering Group. 2023. Multi-annual Artificial Intelligence Workplan 2023–2028. Available at: https://www.ema.europa.eu/en/news/artificial-intelligence-workplan-guide-use-ai-medicines-regulation ↩︎
  3. U.S. Food and Drug Administration. 2025. FDA Launches Agency-Wide AI Tool to Optimize Performance for the American People. Available at: https://www.fda.gov/news-events/press-announcements/fda-launches-agency-wide-ai-tool-optimize-performance-american-people ↩︎
  4. U.S. Food and Drug Administration. 2025. Draft Guidance for Industry and Other Interested Parties: Considerations for the Use of Artificial Intelligence To Support Regulatory Decision-Making for Drug and Biological Products. Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-use-artificial-intelligence-support-regulatory-decision-making-drug-and-biological ↩︎
  5. CIOMS Working Group XIV. 2025. Artificial Intelligence in Pharmacovigilance (Draft for public consultation). Available at: https://cioms.ch/working_groups/working-group-xiv-artificial-intelligence-in-pharmacovigilance/ ↩︎
  6. International Society for Pharmaceutical Engineering (ISPE). 2022. GAMP 5: A Risk-Based Approach to Compliant GxP Computerized Systems, 2nd Edition. Available at: https://ispe.org/publications/guidance-documents/gamp-5-guide-2nd-edition ↩︎
  7. International Organization for Standardization (ISO). 2023. ISO/IEC 42001:2023, Information technology — Artificial intelligence — Management system. Available at: https://www.iso.org/standard/42001 ↩︎
  8. National Institute of Standards and Technology (NIST). 2023. Artificial Intelligence Risk Management Framework (AI RMF 1.0). Available at: https://www.nist.gov/publications/artificial-intelligence-risk-management-framework-ai-rmf-10 ↩︎
  9. World Health Organization (WHO). 2021. WHO guidance: Ethics and governance of artificial intelligence for health. Available at: https://www.who.int/publications/i/item/9789240029200 ↩︎

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