FDA Issues Draft Guidance on the Role of Artificial Intelligence in Supporting Regulatory Decisions
This framework marks the agency’s first official recommendation on the use of AI in drug development.
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The FDA announced it has issued a new draft guidance on the use of artificial intelligence (AI) in supporting regulatory decision-making on a drug or biological product’s safety, effectiveness, or quality. This guidance marks the agency’s first official action relating to the use of AI in drug development.1
The guidance provides a risk-based framework for sponsors to assess and establish the credibility of AI models and determine the necessary actions they must take to demonstrate it.
In a press release, FDA Commissioner Robert M. Califf, MD, said, “The FDA is committed to supporting innovative approaches for the development of medical products by providing an agile, risk-based framework that promotes innovation and ensures the agency’s robust scientific and regulatory standards are met. With the appropriate safeguards in place, artificial intelligence has transformative potential to advance clinical research and accelerate medical product development to improve patient care.”
A number of FDA divisions worked together on the guidance, including its human and animal medical product centers, Office of Inspections and Investigations, Oncology Center of Excellence, and Office of Combination Products. The agency also incorporated feedback from key stakeholders such as sponsors, manufacturers, technology developers and suppliers, and academics.
In particular, feedback was included from an FDA-sponsored expert workshop by the Duke Margolis Institute for Health Policy in December 2022 and over 800 comments on two discussion papers released by the FDA in May 2023 on the use of AI in drug development. Additionally, the agency cited its own experience as a key component in the development of this new guidance, which includes more than 500 drug and biological product submissions with AI components since 2016.
According to the FDA, the use of AI in drug development and regulatory submission has exponentially increased since 2016. “AI can be used in various ways to produce data or information regarding the safety, effectiveness, or quality of a drug or biological product. For example, AI approaches can be used to predict patient outcomes, improve understanding of predictors of disease progression and process and analyze large datasets (e.g., real-world data sources or data from digital health technologies),” the agency said in the press release.
The FDA has also announced the publication of another draft guidance that includes recommendations to support the development and marketing of safe and effective AI-enabled medical devices. If finalized, this guidance would be the first to provide comprehensive recommendations for these devices.2
In a different press release, Troy Tazbaz, director of the Digital Health Center of Excellence within the FDA’s Center for Devices and Radiological Health, said, “The FDA has authorized more than 1,000 AI-enabled devices through established premarket pathways. As we continue to see exciting developments in this field, it’s important to recognize that there are specific considerations unique to AI-enabled devices. Today’s draft guidance brings together relevant information for developers, shares learnings from authorized AI-enabled devices and provides a first point-of-reference for specific recommendations that apply to these devices, from the earliest stages of development through the device’s entire life cycle.”
In recent years, the FDA has been open with encouraging sponsors to engage with the agency early and often when it comes to new technology, including the use of AI. Both of these new guidances reinforce the agency’s commitment to embracing the use of AI in advancing clinical research.
References
1. FDA Proposes Framework to Advance Credibility of AI Models Used for Drug and Biological Product Submissions. News release. FDA. January 6, 2025. Accessed January 6, 2025. https://www.fda.gov/news-events/press-announcements/fda-proposes-framework-advance-credibility-ai-models-used-drug-and-biological-product-submissions
2. FDA Issues Comprehensive Draft Guidance for Developers of Artificial Intelligence-Enabled Medical Devices. News release. FDA. January 6, 2025. Accessed January 6, 2025. https://www.fda.gov/news-events/press-announcements/fda-issues-comprehensive-draft-guidance-developers-artificial-intelligence-enabled-medical-devices
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