Organoids in drug development: from predictive models to regulatory integration

Drug Discovery Today 2026 Open access

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Authors: Xuyang Song, Xinrong Chen, Qing Chen, Hao Wang, Tao Zhang, Michael Z. Liao, Chunhong Liu, Hongtao Yu, Yanning Hao, Guodong Gu, Zehua Cheng, Zhiyao Zhu
+2 more Yongbin Zhang, Suchitra K. Hourigan
DOI: 10.1016/j.drudis.2026.104608
Cited by: 11
References: 148

Abstract

Organoids offer superior physiological relevance over traditional 2D and animal models. By recapitulating human tissue architecture, they enable predictive assessments of drug efficacy, toxicity, and pharmacokinetics (DMPK). Integrating artificial intelligence (AI)-driven modeling further expands their preclinical utility, particularly in oncology. Concurrently, regulatory agencies are establishing standards for organoid validation and qualification. In this review, we summarize current applications and discuss the scientific and regulatory requirements needed to bridge the translational gap. We emphasize that harmonized regulatory frameworks are essential to ensure scientific rigor and facilitate the broader adoption of organoid technologies in drug development.

Fields of Study & Subjects

Cancer Cells and Metastasis 3D Printing in Biomedical Research Mathematical Biology Tumor Growth

Publication details

Journal / source
Drug Discovery Today
Publisher
Elsevier BV
Volume
31
Issue
2
Pages
104608–104608
Publication date
21 January 2026
Language
EN