[Current challenges and future perspectives of pharmacological testing using new approach methodologies].

Area of Science:

• Regulatory Science
• Pharmacology
• Toxicology

Background:

• Current drug development relies on in vitro and in vivo methods, with existing approaches showing effectiveness but lacking optimal human risk prediction.
• The need for improved accuracy in predicting adverse events in humans is critical for advancing drug safety and efficacy.
• Recent global health events, like the COVID-19 pandemic, have accelerated the adoption of novel methodologies in drug development.

Purpose of the Study:

• To review the current landscape and future potential of New Approach Methodologies (NAMs) in pharmacological assays.
• To highlight how NAMs contribute to safer and more effective drug development.
• To discuss the integration of NAMs for enhanced patient safety and streamlined regulatory review processes.

Main Methods:

• Exploration of in vitro methods, specifically utilizing human induced pluripotent stem cells (hiPSCs).
• Integration of computational approaches and in silico modeling for risk assessment.
• Review of animal alternative methods and their role in modern drug evaluation.

Main Results:

• NAMs demonstrate significant potential in improving the accuracy of predicting human adverse events.
• In vitro and computational methods are accelerating drug development efficiency and patient safety.
• These novel approaches support faster regulatory review timelines.

Conclusions:

• New Approach Methodologies (NAMs) are pivotal for advancing regulatory science and ensuring human longevity.
• The continued development and implementation of NAMs are essential for enhancing drug safety, efficacy, and the overall drug development pipeline.
• Pharmacological assay systems utilizing NAMs represent the future of robust and predictive drug development.