The U.S. Food and Drug Administration (FDA) has taken a significant step in regulating the use of artificial intelligence (AI) in drug and biological product development by issuing its first draft guidance on the topic. This guidance provides recommendations for using AI to support regulatory decisions regarding a product's safety, effectiveness, and quality.
"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,” stated FDA Commissioner Robert M. Califf, M.D. He emphasized AI's transformative potential in advancing clinical research and accelerating medical product development, provided appropriate safeguards are in place.
A Response to Rapid AI Integration:
The FDA has observed a substantial increase in AI's use in drug development and regulatory submissions since 2016.
AI can analyze large datasets, including real-world data and data from digital health technologies, to predict patient outcomes and improve understanding of disease progression. This guidance addresses the need for a standardized approach to evaluating these AI-driven insights.
Focus on Model Credibility:
A central theme of the guidance is "model credibility"—trust in an AI model's performance within its specific "context of use." Context of use refers to how an AI model is applied to answer a particular research question. Recognizing the diverse potential applications of AI, the guidance establishes a risk-based framework for sponsors to assess and demonstrate the credibility of their AI models. This framework helps determine the necessary activities to establish that an AI model’s output is trustworthy. This approach aligns with the FDA's existing review practices for drug and biological product applications incorporating AI. The FDA encourages early engagement with sponsors regarding AI credibility assessment and its use in human and animal drug development.
Collaborative Development and Extensive Input:
This draft guidance is the result of collaborative efforts across the FDA's human and animal medical product centers, the Office of Inspections and Investigations, the Oncology Center of Excellence, and the Office of Combination Products, ensuring agency-wide consistency.
The FDA incorporated feedback from various stakeholders, including sponsors, manufacturers, technology developers, academics, and suppliers. This feedback includes:
Input from an FDA-sponsored expert workshop hosted by the Duke Margolis Institute for Health Policy in December 2022.
Over 800 comments received on two discussion papers published in May 2023 regarding AI use in drug development and manufacturing.
The FDA’s experience reviewing over 500 drug and biological product submissions containing AI components since 2016.
Public Comment and Future Steps:
The FDA is now seeking public comment on the draft guidance, "Considerations for the Use of Artificial Intelligence to Support Regulatory Decision-Making for Drug and Biological Products," for a 90-day period. The agency is particularly interested in feedback on the guidance's alignment with industry experience and the adequacy of available engagement options with the FDA regarding AI use. The FDA will review and consider these comments before finalizing the guidance.
Broader AI Regulatory Landscape:
This announcement complements a separate draft guidance published concurrently, focusing specifically on AI-enabled medical devices. These publications reflect the FDA's broader commitment to transparency and ensuring product safety and effectiveness while fostering innovation in the rapidly evolving field of AI in healthcare. The FDA’s medical product centers are dedicated to promoting the responsible and ethical application of AI in medical products and will continue developing policies that support innovation while upholding rigorous standards for safety and effectiveness.
Key Resources:
Draft Guidance: Considerations for the Use of Artificial Intelligence to Support Regulatory Decision-Making for Drug and Biological Products
Artificial Intelligence and Medical Products
Artificial Intelligence and Machine Learning (AI/ML) for Drug Development
Artificial Intelligence & Medical Products: How CBER, CDER, CDRH, and OCP are Working Together
About the FDA:
The FDA, a component of the U.S. Department of Health and Human Services, is responsible for protecting public health by ensuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, biological products, and medical devices. The agency also oversees the safety and security of the nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and regulates tobacco products.
Sources and related content https://www.fda.gov/
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