NEJM AI’s Post

End point evaluation is central to the evidence generation process in a clinical trial, and #ArtificialIntelligence (AI) tools have the potential to improve the efficiency and accuracy of this process. In 2025, the European Medicines Agency and U.S. Food and Drug Administration independently endorsed AIM-NASH, an AI-based tool for histologic assessment in clinical trials for metabolic dysfunction–associated steatohepatitis (MASH). As the first ever regulatory certification of an AI-enabled end point, this represents a milestone for the emerging development and use of AI tools for drug development. A new article examines the evidence and policy decisions underlying the endorsement and explores the clinical and regulatory implications of AI-enabled end points for the transformation of clinical trials. Read the Perspective “Adoption of Artificial Intelligence–Based End Points — A New Era for Clinical Trials” by Kushal Kadakia, MD, MSc, and Harlan Krumholz, MD, SM: https://nejm.ai/4wkloTl #AIinMedicine

  • Table outlining potential applications of artificial intelligence for clinical trial endpoints. It includes focus areas such as histopathology, anatomical imaging, and physiological imaging. Descriptions and current examples are provided alongside AI-enabled future possibilities for each category.

This is a real milestone and a long-overdue one. If the EMA and FDA can independently validate an AI-based histology endpoint, the regulatory path exists for AI-based clinical decision support as well. I practice medicine at sea. We lack the infrastructure for expert pathology review, specialist consultation, and sometimes even basic lab interpretation. The same rigor that validated AIM-NASH could validate AI-based tools for resource-constrained environments, cruise ships, rural clinics, forward operating bases where the standard of care is whatever the physician in the room can manage alone. The regulatory framework is no longer theoretical.

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