Advanced In Vitro Models in Drug Development

This technical report discusses the emerging role of advanced in vitro models in drug development, outlining the challenges and opportunities associated with their adoption. Pharmaceutical companies invest significantly in drug discovery, yet a small percentage of drug candidates gain approval, primarily due to reliance on outdated experimental systems. The report highlights how advanced in vitro models, such as organoids and organ-on-a-chip systems, aim to provide more predictive safety and efficacy data compared to traditional 2D cell cultures and animal models. It also details the regulatory momentum towards acceptance of these models, including initiatives like the FDA Modernization Act 2.0. Despite their potential, the report identifies key challenges that hinder widespread adoption, including entrenched legacy models, validation requirements, decision-making silos, and nascent regulatory guidance. Strategies to overcome these challenges are discussed, emphasizing the need for validation and practical utility to enhance the acceptance of advanced in vitro tools in pharmaceutical workflows.