Methodological and regulatory considerations for causal AI in drug development.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 41760772.
- Also identified by DOI 10.1038/s41746-026-02477-w and PMC identifier 13065995.
- Licence recorded as CC BY.
- The licence permits redistribution, so the abstract is shown in full and the full text is available from the publisher.
Abstract
Advances in AI offer significant opportunities to enhance drug development. While several regulatory agencies have begun issuing guidance on AI adoption, its application to causal inference-a critical piece to understand treatment effects and inform regulatory decisions-remains limited. This paper reviews regulatory activities and examines statistical methodologies for AI-driven causal inference. We discuss key regulatory challenges and illustrate how AI adds value across diverse data sources and studies.