Organoids in drug development: from predictive models to regulatory integration.

Area of Science:

• Biotechnology
• Drug Development
• Oncology

Background:

• Organoids offer enhanced physiological relevance compared to traditional 2D and animal models.
• They recapitulate human tissue architecture, enabling predictive assessments of drug efficacy, toxicity, and pharmacokinetics (DMPK).

Purpose of the Study:

• To review current applications of organoids in drug development.
• To discuss scientific and regulatory requirements for bridging the translational gap.
• To emphasize the need for harmonized regulatory frameworks for organoid technology adoption.

Main Methods:

• Literature review of organoid applications in preclinical drug development.
• Analysis of current scientific and regulatory landscapes.
• Discussion of challenges and opportunities for organoid technology integration.

Main Results:

• Organoids are valuable tools for predicting drug efficacy, toxicity, and DMPK.
• Artificial intelligence (AI)-driven modeling enhances organoid utility, especially in oncology.
• Regulatory agencies are developing standards for organoid validation and qualification.

Conclusions:

• Harmonized regulatory frameworks are crucial for ensuring scientific rigor.
• Broader adoption of organoid technologies in drug development requires standardized validation and qualification processes.
• Organoids, augmented by AI, represent a significant advancement in preclinical research.