Impact of the FDA roadmap and perspectives for regulatory use of new approach methodologies in Japanese pharmaceutical companies.

Pharmaceutical scientists develop and apply increasingly complex technologies from an expanding toolbox to create more effective and safer drugs. Consequently, the increasing human specificity of new modalities has highlighted the limitations of conventional animal studies. Recently, new approach methodologies (NAMs) have attracted attention for their potential to promote the 3Rs and human-relevant evaluation in pharmaceutical research and development, and have been applied not only in early discovery research but also in regulatory review. In April 2025, the US Food and Drug Administration released a roadmap outlining a stepwise transition from animal studies to NAMs and highlighting drug development tool qualification, raising the urgency for a coordinated response in Japan. The Japan Pharmaceutical Manufacturers Association (JPMA) conducted a survey of JPMA member companies in July 2025 to provide an overview of the status of NAMs in the Japanese pharmaceutical industry and to generate evidence for policy recommendations. While most responding companies were aware of the FDA roadmap, concrete internal responses were limited, and approximately half had not conducted NAMs research. The results indicate strong interest in promoting the 3Rs and improving non-clinical development, but limited experience with NAMs, especially for regulatory applications. Both internationally harmonized guidelines and the establishment of a domestic qualification system, including early consultation channels and a transparent public database, are required to promote the regulatory use of NAMs in Japan. The JPMA could serve as a central hub by facilitating proactive communication among domestic and international stakeholders, thereby promoting the broader implementation of NAMs in regulatory submissions.