FDA Roadmap to Reducing Animal Testing in Preclinical Safety Studies
Capabilities
Transforming Preclinical Safety Studies with NAMs
In April 2025, the FDA issued a scientific roadmap to eliminate the use of animals for preclinical drug development. In it, New Approach Methodologies (NAMs) are described, including various in vitro, in silico, and ex vivo tools that are being developed and validated to provide physiologically relevant data, thereby eliminating interspecies differences that can confound safety and efficacy results.
Initial Focus on Monoclonal Antibodies and Broader Applications
The roadmap outlines a 3-year plan that relies on collaboration between industry, researchers, and regulatory agencies to enhance the translatability of nonclinical data, thereby improving the prediction of human outcomes. It will initially be applied to monoclonal antibodies, or mAbs, before being rolled out to other biologics, followed by new chemical entities.
NAM Integration in IND-Enabling Programs for CGT Products
For cell and gene therapy (CGT) products, the use of animals in preclinical development has been a distinctive challenge due to the lack of relevant models to study the in vivo effects of human cells and genes. The 2013 FDA guidance on preclinical assessment of CGT products addresses this challenge by recommending the use of animal disease models, transgenic animals, and hybrid pharm/tox studies for IND-enabling programs.
The guidance also recommends using in vitro studies (e.g., functional assays, immunophenotyping, morphologic evaluation) to identify potential safety issues and the mechanism of action (MOA) of an investigational CGT product, as well as to establish the biological relevance of a specific animal species to the particular product. The recommendations introduced in the 2013 guidance lay the foundation for the more comprehensive roadmap recently issued.
The USDA has long promoted animal welfare through the 3Rs (replacement, reduction, and refinement). The roadmap takes an additional step by describing how to achieve physiological relevance in non-animal test systems. As the roadmap and use of NAMs become more widely adopted, the time and cost to develop complex therapeutics should be significantly reduced, bringing safe and effective treatments to patients who need them.
Pharmaron’s Innovations
Since our foundation, Pharmaron has been developing different approaches that support these regulatory trends. Our sustained dedication to in vitro–in vivo correlation (IVIVC) has advanced our development of alternatives to conventional animal testing—approaching it not just as a regulatory requirement, but as a core scientific approach to improve predictive accuracy.
IDAS, Pharmaron’s proprietary technology, combines traditional dissolution testing to determine and quantify interactions with a bio-relevant membrane. IDAS can evaluate absorption, permeation, accumulation, biomarker regulation, and metabolism and test a finished dosage form from a tablet or capsule to suspension.
With state-of-the-art tools, our Computer-Aided Drug Design team routinely generates insights into data and proactively designs new compounds to guide medicinal chemistry teams in creating or expanding IP space, while addressing specific issues, such as potency, selectivity, physical properties or metabolic stability. in silico ADMET and Drug–like Property Calculations are utilized for a faster prediction of physical properties.
As regulatory landscapes continue to evolve, Pharmaron will continue to evolve with it. With extensive experience and deep expertise in nonclinical testing, we offer our partners not only state-of-the-art technologies but also the strategic guidance required to adapt to shifting regulatory demands.
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