FDA kicks off 2025 with the release of draft guidance on AI use in drug and biological product development

Setting the stage for a transformative year in innovation, the US Food and Drug Administration (FDA) has issued draft guidance on AI in drug development, emphasizing credibility, risk-based frameworks, and public input to strike a balance between innovation and safety.

The FDA has released its first-ever draft guidance on the use of AI to support regulatory decisions for drug and biological product safety, effectiveness, and quality. Titled ‘Considerations for the Use of Artificial Intelligence To Support Regulatory Decision-Making for Drug and Biological Products’, the guidance outlines recommendations for sponsors and stakeholders to ensure AI model credibility in regulatory submissions.

“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, MD in the press release. He stressed the transformative potential of AI in advancing clinical research and accelerating medical product development to improve patient care.

Since 2016, the FDA has observed exponential growth in AI use for drug development and regulatory submissions. AI applications include predicting patient outcomes, analyzing disease progression, and processing large datasets such as real-world data (RWD) and digital health technologies. While these advancements are promising, the FDA has emphasized the need to ensure AI model credibility to build trust in their performance for specific applications.

Risk-based framework for AI credibility

A central component of the guidance is the introduction of a risk-based framework for evaluating AI model credibility. The FDA defines credibility as the, “trust, established through the collection of credibility evidence, in the performance of an AI model for a particular context of use.” This process involves defining how the model addresses a specific question of interest, assessing its risk, and developing plans to validate its outputs.

“The activities that may be used to establish credibility of AI model outputs should be commensurate with the AI model risk and tailored to the specific context of use,” the guidance states. Sponsors are encouraged to engage with the FDA early to discuss AI credibility assessments and development plans.

The draft guidance incorporates extensive feedback from stakeholders, including technology developers, sponsors, manufacturers, and academics. It reflects insights from over 500 regulatory submissions involving AI components since 2016, an expert workshop hosted by the Duke Margolis Institute for Health Policy in 2022, and over 800 comments on two related discussion papers published in 2023.

The FDA’s medical product centers, along with its Office of Inspections and Investigations and Oncology Center of Excellence, collaborated to ensure the framework applies consistently across the agency. The FDA also seeks public comment on the draft guidance within 90 days.

Additional AI guidance for medical devices

On the same day, the FDA issued another draft guidance specific to AI-enabled medical devices, titled ‘Artificial Intelligence-Enabled Device Software Functions: Lifecycle Management and Marketing Submission Recommendations’. This document outlines recommendations for lifecycle management, marketing submissions, and addressing transparency and bias in AI-enabled devices.

As AI continues to evolve, the FDA remains dedicated to supporting innovation while ensuring safety and effectiveness. “AI management requires a risk-based regulatory framework built on robust principles, standards, and best practices,” the agency stated, reinforcing its commitment to promoting responsible AI use in the medical field.