New Frontiers of Drug Development Through the Use of New Approach Methodologies

Clinical Snapshot

Key Findings

• Effect Size: Not applicable - qualitative meeting report

• Primary Outcome: Summary of expert consensus on NAMs implementation in drug development

• Nnt Or Sensitivity: Not applicable - no quantitative outcomes assessed

Clinical Application

Implementation depends on regulatory acceptance, technical expertise, and validation requirements Relevant to TGA regulatory pathways; aligns with international harmonisation efforts through ICH guidelines Pharmaceutical researchers, regulatory scientists, drug development professionals

Abstract

On November 2025, AAPS PharmSci 360 convened a symposium that included experts in the application of various types of New Approach Methodologies (NAMs). They shared their experiences and insights on topics that included microphysiological systems (MPS), the use of 3D organoids, and in silico tools. MPS systems were explored from the perspective of their testing and qualification through an academia-industry-government tissue chip testing consortium whose mission is to perform context-of-use-based testing of MPS and conduct comparative evaluations to support the use of MPS systems in safety assessments. 3D organoids were described and insights shared on how and when they may provide an appropriate tool for evaluating drug safety and effectiveness, and their use in supporting personalized medicine. Regarding in silico tools, examples were provided to describe their growing utility in predicting drug toxicity, population variability, and its potential benefits over traditional evidence-based toxicology. These tools include in silico models (e.g., physiologically based pharmacokinetic models), Artificial Intelligence (AI), and Machine Learning (ML) which, unlike other aspects of model informed drug development, reduce reliance on predefined models and allows for the integration of diverse sources of data. Computationally, these tools can generate predictions in hours where it would otherwise have taken weeks or months (and extensive experimentation). Within this meeting report, we highlight the key issues discussed during that symposium and share additional aspects to consider when developing a NAMs-based roadmap for specific contexts of use.

References

1. 1.Martinez, M. N., Mochel, J. P., Allenspach, K., Zdyrski, C., Krishna, S., & Rusyn, I. (2026). New Frontiers of Drug Development Through the Use of New Approach Methodologies. The AAPS Journal. https://doi.org/10.3389/fcell.2023.1083175