Organoid models: Regulations and prospects for the development of investigational new drugs

ABSTRACT: Over the past decade, research associated with organoids has entered a golden era, marking a significant shift in the biomedical landscape. Organoids have been widely applied in the industrial sector and have gained recognition from regulatory authorities. However, amid this rapid expansion, a comprehensive and precise overview of the field has been lacking. Our review aims to fill this gap by presenting a comprehensive overview of the regulations and prospects for the development of investigational new drugs (INDs) using organoids. Drawing on our extensive experience in academic research and industrial applications, we meticulously analyzed the organoid field from two distinct perspectives. We explored the synergies between organoids and cutting-edge technologies such as single-cell omics, which highlights their crucial role in customizing personalized medicines, improving high-throughput drug screening, and establishing physiologically relevant disease models. Organoids offer advantages over traditional animal models, including high throughput, rapid results, cost-effectiveness, and strong clinical relevance. Our analysis delves into supportive policies from regulatory bodies such as the European Medicines Agency (EMA), Food and Drug Administration (FDA), and Center for Drug Evaluation (CDE) for the use of organoids. Our comprehensive analysis and reflective discourse provide a deep dive into the current landscape of drugs derived from organoids that have obtained IND status and the subsequent progress in clinical trials. We cautiously anticipate challenges and future directions in organoid research, particularly in terms of its potential impact on personalized patient care, novel avenues for drug discovery, and clinical research. In summary, our review presents a comprehensive exploration of organoid technology and prospects for the development of INDs using organoids, thereby providing reflections, summaries, and projections that can be beneficial for current researchers.