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[Non-clinical safety assessments using human cells for pediatric drug development]

Yuta Sakamoto1, Mitsuhiko Yamada2, Yasunari Kanda1

  • 1National Institute of Health Sciences.

Nihon Yakurigaku Zasshi. Folia Pharmacologica Japonica
|May 6, 2026
PubMed

Insights

New approach methodologies (NAMs) using human cell models aid pediatric drug development by improving non-clinical safety and efficacy assessments, addressing challenges like limited patient numbers in clinical trials.

Area of Science:

  • Pharmacology
  • Toxicology
  • Biotechnology

Background:

  • Pediatric drug development is critical due to unique physiological differences in children compared to adults.
  • Clinical trials in pediatric populations face challenges, including limited patient numbers.
  • Ensuring drug safety and efficacy in children requires robust non-clinical assessment strategies.

Purpose of the Study:

  • To explore the application of New Approach Methodologies (NAMs) in pediatric drug development.
  • To demonstrate the utility of human cell-based models for assessing pediatric drug safety and efficacy.
  • To discuss advancements in non-clinical assessment strategies for pediatric therapeutics.

Main Methods:

  • Utilized human cell-based models and organoids for drug development.
  • Applied in vitro models for assessing pediatric heart failure therapeutics.
  • Employed cell models for evaluating drug-induced liver injury risk in pediatric contexts.

Main Results:

  • Human cell models can recapitulate pediatric-specific characteristics for improved non-clinical assessments.
  • Demonstrated successful application of cell models in developing pediatric heart failure therapeutics.
  • Showcased the utility of in vitro models for assessing drug-induced liver injury risk in pediatrics.

Conclusions:

  • NAMs, particularly human cell models, offer promising alternatives to animal testing in pediatric drug development.
  • These models enhance the predictability of human drug responses and improve the safety and efficiency of clinical trials.
  • Continued development and implementation of NAMs are crucial for advancing pediatric drug development globally.

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