The International Society for Stem Cell Research (ISSCR) Consortium on Advanced Stem Cell-Based Models in Drug Discovery and Development has submitted comments to the U.S. Food and Drug Administration (FDA) in response to the agency’s draft guidance , General Considerations for the Use of New Approach Methodologies in Drug Development Guidance for Industry.
The consortium, a global collaboration of academic scientists, industry leaders, and regulatory experts convened by the ISSCR, welcomed the FDA’s efforts to advance the integration of new approach methodologies (NAMs) into drug development while emphasizing the importance of maintaining a flexible, fit-for-purpose regulatory framework that can adapt to rapidly evolving technologies.
“NAMs, including stem cell-derived models such as organoids, engineered tissues, and computational systems, have tremendous potential to improve the predictive power, efficiency, and human relevance of preclinical drug development,” said Shuibing Chen, co-chair of the consortium. “The FDA’s draft guidance is an important step forward in supporting innovation while helping to establish needed framework for the use of NAMs in drug development.”
In its comments, the consortium commended the FDA for developing a clear, principle-based framework centered on context of use, human biological relevance, technical characterization, and fit-for-purpose evaluation. The consortium also offered recommendations intended to strengthen the guidance and ensure it remains adaptable across a wide range of NAM technologies and applications.
Among the key recommendations were:
Clarifying that the level of biological complexity in a NAM should align with its intended context of use, supporting both high-throughput reductionist systems and more complex models.
Encouraging evaluation of NAMs based not only on comparison to traditional animal models, but also on their ability to generate human-relevant insights in areas where conventional methods may be limited.
Expanding recognition of computational modeling, artificial intelligence, and hybrid in vitro–in silico approaches as increasingly important components of modern drug development.
Recognizing the distinct characterization needs of stem cell-derived NAMs, particularly induced pluripotent stem cell (iPSC)-based systems, and encouraging future FDA guidance or workshops specific to these technologies.
The consortium emphasized that stem cell-derived NAMs offer unique opportunities to model human biology and disease , helping improve translational relevance while supporting broader efforts to modernize preclinical testing strategies.
Supported by founding members Bayer and Burroughs Wellcome Fund, learn more about the consortium’s aims and scope .
About ISSCR
Across more than 80 countries, the International Society for Stem Cell Research is the preeminent global, cross-disciplinary, science-based organization dedicated to advancing stem cell research and its translation to medicine.