FDA Outlines AI Principles for Drug Development

The Food and Drug Administration released information Wednesday outlining how the agency and its partners should use artificial intelligence in drug development and evaluation.

The guidance, published in conjunction with the European Medicines Agency, lays out 10 principles for using AI across the drug product lifecycle, from early research to clinical trials and manufacturing. The agency said the goal is to maximize AI’s potential while maintaining reliable data, patient safety and regulatory standards.

“AI has the potential to transform drug development by reducing time and cost, ultimately improving health care,” Anindita Saha, acting associate director for data and AI policy at FDA’s Center for Drug Evaluation and Research, said in a LinkedIn post Wednesday. “As AI continues to evolve, international collaboration is helpful for harmonization for drug developers to advance innovation that benefits patients. These principles also align and build upon the device GMLP principles showing cross-sector learnings.”  

The principles give high-level guidance on AI use and are intended to “lay the foundation for developing good practice” for AI technology in drug development. Among other recommendations, the principles direct drug researchers and developers to document AI processing steps and analytical decisions so that it can be traceable and verified, to implement risk-based quality management systems, and to regularly perform scheduled monitoring and periodic re-evaluation to ensure adequate performance of the AI technologies. 

While the principles are not enforceable guidelines, they are intended to inform future regulatory policies and provide the framework for responsible AI use in drug research and development. 

“Among other innovations, AI technologies are anticipated to support a multi-faceted approach that promotes innovation, reduces time-to-market, strengthens regulatory excellence and pharmacovigilance, and decreases reliance on animal testing by improving the prediction of toxicity and efficacy in humans,” according to the publication.  

Aligning with Broader Federal AI Efforts

The FDA’s guidance follows the Trump administration’s push to rapidly adopt AI. The White House has issued multiple directives including an AI Action Plan, the Genesis Mission and most recently a memorandum to create a national AI framework and challenge state laws seeking to regulate AI.

The president’s AI Action Plan outlines steps to accelerate private sector innovation, build critical infrastructure and advance AI policy. Similarly, the Genesis Mission is a coordinated national effort to use AI to accelerate innovation and discovery. It includes building an integrated AI platform to “harness federal scientific datasets.”  

In July, President Donald Trump emphasized speed and American dominance in AI while signing three executive orders that called for reducing regulatory burdens and accelerating AI innovation. 

“We’re suddenly engaged in a fast-paced competition to build and define this groundbreaking technology,” Trump said. “America must once again be a country where innovators are rewarded with a green light, not strangled with red tape.” 

The orders called on agencies to make it easier to stand up necessary AI infrastructure, advocate for U.S.-developed AI technologies internationally and eliminate biased AI systems.