Organoids and New Approach Methodologies in Drug Discovery: From Target to industry

Drug discovery is undergoing a generational shift. The FDA Modernization Act 2.0, the EPA’s NAM roadmap, and EMA’s evolving guidance are accelerating industry adoption of non-animal models. Organoids, organs-on-chip, engineered 3D tissues, and complementary in silico and in vitro tools — collectively, New Approach Methodologies (NAMs) — are moving from proof-of-concept into everyday use across screening, discovery, safety, potency, and IND-enabling studies.

This Research Topic convenes leaders from academia, the pharmaceutical industry, and regulatory agencies to benchmark where the field stands in 2026, where it is going, and what translational and regulatory gaps remain as we enter this new era. While the papers can draw on specific indications or model systems, the themes of what challenges remain for full adoption into the drug discovery and regulatory workflow should be approached agnostically.

Potential Themes to Be Covered include classes of NAMs, tissue models, pipeline position (i.e. different phases of drug discovery), and the regulatory landscape. Original research/technical papers, case studies, reviews, editorials/perspectives, and commentaries are welcome. Both academic and industry researchers are invited to participate, and commentary or discussion on the differences and unique challenges of these two fields is welcome. Further discussion and consideration of future trends and next steps (AI-enabled phenotypic readouts, patient-avatar organoids, GLP-grade MPS, multi-organ chips, etc.) are also of interest.

Article types and fees

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Data Report
• Editorial
• FAIR² Data
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• ... View all formats

Articles that are accepted for publication by our external editors following rigorous peer review incur a publishing fee charged to Authors, institutions, or funders.

Article types

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Data Report
• Editorial
• FAIR² Data
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• Original Research
• Perspective
• Review
• Systematic Review
• Technology and Code

Keywords: Organoids, microphysiological systems, organ-on-chip, New Approach Methodologies (NAMs), iPSC-derived models, IND-enabling studies, phenotypic screening, tissue-chip, engineered 3D tissues, preclinical drug discovery, qualification, context-of-use, human functional data

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.