Precision Medicine and the FDA Modernization Act 2.0: Catalyzing Innovation in Cardiovascular Therapy

Animal models are an essential part of preclinical research, serving to protect patients through safety and efficacy studies before human trials begin. Prior to the passage of the Food and Drug Administration (FDA) Modernization Act 2.0 in 2022, animal studies were generally required by regulators in preclinical safety evaluations. This Act amended the Federal Food, Drug, and Cosmetic Act to clarify that while animal testing had been the default standard, it is no longer a mandatory requirement for regulatory filings involving drugs and biological products1. Additionally, the Act explicitly specifies in vitro, in silico, and in chemico testing as alternatives to assess pre-clinical safety and efficacy. The FDA and others refer to these alternative platforms as New Approach Methodologies (NAMs) with an emphasis on the 3Rs of replacing, reducing and refining the use of animal testing2. This shift opens new pathways for developing and evaluating precision therapies in cardiovascular disease particularly if human-derived, genetically accurate models can outperform animal studies in predictive power and translatability.