NAMs and the pragmatic path to safer first-in-human studies

An Expert View from Steve Bulera, CVP and CSO, Discovery and Safety Assessment, Charles River (NYSE: CRL).

Nearly a decade ago, the phrase "New Approach Methodologies” or NAMs was formally coined to describe a broad range of tools and technologies, from organoids and organ-on-a-chip to computational models and skin sensitization tests, that aim to reduce the use of animals, shorten the time required for new medicine development, and increase patient safety. The goal of these approaches was to provide more human-relevant, mechanistic, and high-throughput alternatives to traditional animal-based studies of efficacy and toxicity.

Over time, the US Food and Drug Administration (FDA), the European Medicines Agency, and other regulatory authorities have selectively allowed some fully validated NAMs to be included in IND submissions, with a number of companies attempting to use a NAMs-based approach. Recently, the first IND approval was granted where the efficacy data was generated solely from human vascularized organoid-based combination studies.