Organoids and organs-on-chips: Recent advances, applications in drug development, and regulatory challenges

Abstract

Organoids and organs-on-chips (OoCs) are rapidly evolving technologies for creating miniature human tissue models. They can mimic complex physiological functions and pathological conditions, offering more realistic platforms for disease modeling, drug screening, precision medicine, and regenerative therapies. The passing of the FDA Modernization Act 2.0 has reduced animal testing requirements for drug trials, marking a significant milestone in using advanced in vitro models such as organoids and OoCs for therapeutic discovery. Apart from technical and ethical challenges, regulatory issues persist in ensuring the reliability, scientificity, and applicability of these models in drug development. This perspective explores the concept, advancements, pros and cons, and applications of organoids and OoCs, particularly in drug research and development. It also examines global regulatory agencies' policies and actions on using these models in drug evaluation, aiming to guide industry standard setting and advance regulatory science.

Keywords: compliance challenges; drug discovery and development; organoids; organs-on-chips; regulatory perspectives.