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Pediatric Developmental Safety Assessment: Are We Ready for the Next Thalidomide?

Gilbert J Burckart1, Susan M Abdel-Rahman1, Gelareh Abulwerdi1

  • 1Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.

Clinical Pharmacology and Therapeutics
|November 19, 2025
PubMed

Insights

Pediatric drug development has advanced significantly, but the potential risks of new medications on child development remain under-evaluated. Future research must prioritize assessing these developmental impacts to prevent unforeseen harm.

Area of Science:

  • Pediatric Pharmacology
  • Developmental Toxicology
  • Drug Safety

Background:

  • Over 1,000 products have been studied in pediatric patients in the last 20 years.
  • Advancements in understanding pediatric disease processes have driven progress.
  • The potential adverse effects of new drugs on pediatric development have been largely overlooked.

Purpose of the Study:

  • To highlight the overlooked risks of new drugs on pediatric developmental processes.
  • To emphasize the need for evaluating developmental impacts in pediatric drug studies.
  • To prevent potential tragedies similar to thalidomide.

Main Methods:

  • This study is a review and critical analysis of existing pediatric drug development practices.
  • It synthesizes current knowledge on drug effects on development.
  • It examines historical precedents and potential future risks.

Main Results:

  • Despite significant progress in pediatric drug studies, a critical aspect—the impact on development—remains under-assessed.
  • There is a lack of predictive models for developmental toxicity in pediatric populations.
  • Historical events underscore the potential for severe, unforeseen consequences.

Conclusions:

  • Pediatric drug development requires a paradigm shift to include rigorous assessment of developmental effects.
  • Proactive evaluation of developmental risks is crucial for ensuring child safety.
  • Failure to address this oversight risks repeating past tragedies.

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