Organoids in drug discovery and development: emerging technologies to transition beyond animal models

High translational failure rates and ethical concerns surrounding animal models are driving the development of technologies to improve the drug development process. Organoids, three-dimensional self-organizing structures derived from stem cells, provide scalable, human-relevant platforms that recapitulate aspects of native tissue architecture and physiology with the potential to enhance predictive assessment of drug efficacy and toxicity.

This review evaluates the potential for organoids to complement and, where scientifically feasible, reduce or eliminate animal testing. It highlights advancements and enabling technologies, including bioprinting, organ-on-chip, bioreactors, and machine learning, that support increasing organoid complexity and physiological relevance. Furthermore, it explores evolving regulatory frameworks and industry adoption while discussing practical challenges and ethical complexities to inform future development. The literature was surveyed up to August 2026 through PubMed and ClinicalTrials.gov, with industry reports retrieved through Google search.

The future of drug discovery lies in a strategic transition toward human-relevant systems. Organoid technologies provide value across stages of drug development, from high-throughput screening and mechanistic studies to predicting patient-specific treatment response, while reducing reliance on animal testing and supporting increased patient safety and efficiency. However, challenges in standardization, morphological complexity, heterogeneity, and cellular maturation currently limit their broad application.