Regulators back AI and organ-on-a-chip technologies to modernize safety evaluation and accelerate patient access

The US FDA has announced a landmark move to phase out its traditional animal testing requirements for monoclonal antibody (mAb) therapies and other drugs — in favor of advanced, human-relevant technologies. The initiative aims to modernize drug evaluation, reduce R&D costs, and speed up patient access without compromising safety.

Under a new roadmap released by the agency, drug developers will be encouraged to replace or reduce animal studies in investigational new drug (IND) applications using a range of non-animal approaches, including artificial intelligence (AI)-driven simulations, cell-line assays, and organoid or organ-on-a-chip data — collectively known as new approach methodologies (NAMs). The FDA will also allow the inclusion of human safety data collected in countries with comparable regulatory standards.

“For too long, drug manufacturers have performed additional animal testing of drugs that have data in broad human use internationally,” said FDA Commissioner Dr Martin Makary. “By leveraging AI-based modelling, human organ model-based lab testing, and real-world human data, we can get safer treatments to patients faster and more reliably, while also reducing R&D costs and drug prices.”

A step change in regulatory science

Animal models — long seen as the gold standard in preclinical testing — are increasingly under scrutiny for their limited ability to predict human responses. The FDA’s updated guidance reflects growing confidence in alternatives that more accurately mimic human biology.

Key elements of the FDA roadmap include:

• Advanced simulations that model drug behavior and predict toxicity based on molecular composition

• Lab-grown human organ models, such as liver and immune organoids, that can identify risks not always seen in animals

• Regulatory incentives that may fast-track reviews where non-animal safety data meets required standards

• Global data harmonization, with inclusion of safety data from international settings where the drug is already in human use

This change also aligns with a broader push for regulatory modernization, supported by recent calls from Congress and sustained advocacy from the scientific community.

Industry already adapting

The FDA’s new direction is expected to accelerate the adoption of commercial-ready organ-on-a-chip technologies — including those developed by companies such as Emulate, which recently demonstrated that human-relevant platforms could match or outperform traditional animal models for assessing immunotoxicity in biologics.

Such technologies offer more granular, tissue-specific insights and may eventually replace whole-animal studies altogether for many drug classes.

A pilot program is set to launch later this year, enabling select monoclonal antibody developers to apply a non-animal testing strategy under direct FDA consultation. Insights from that pilot will feed into further policy refinement.

Commissioner Makary described the policy shift as “a win-win for public health and ethics,” adding: “Thousands of animals, including dogs and primates, could eventually be spared each year as these new methods take root.”