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Accelerating Pediatric Oncology Drug Development: Advances in Clinical Pharmacology, Trial Design, Non-Clinical

Ya-Feng Wen1, Xiaoning Wang1, Sarandeep Boyanapalli1

  • 1Clinical Pharmacology Sciences, Gilead Sciences, Inc., Foster City, CA, USA.

Journal of Clinical Pharmacology
|July 24, 2026
PubMed

Insights

Pediatric oncology drug development is accelerating due to targeted therapies and innovative trials. Key strategies include reduced nonclinical data and model-informed approaches to improve pediatric cancer treatment.

Area of Science:

  • Oncology
  • Pharmacology
  • Regulatory Science

Background:

  • Pediatric oncology drug development faces challenges like rare diseases, heterogeneity, and ethical trial constraints.
  • Despite barriers, pediatric cancer therapy approvals are increasing due to molecularly targeted treatments, regulatory evolution, and novel trial designs.

Purpose of the Study:

  • To review regulatory requirements for pediatric oncology drug development in the US, EU, and globally.
  • To analyze 33 US FDA-approved pediatric oncology drugs (2018-2025), focusing on data generation for dose justification.
  • To discuss challenges in pediatric clinical operations, formulation, and the integration of real-world data, external controls, and AI/ML.

Main Methods:

  • Review of recent regulatory requirements for pediatric oncology drug development.
  • Analysis of 33 US FDA-approved oncology drugs for pediatric populations between 2018 and 2025.
  • Discussion of common challenges, formulation development, and emerging data strategies.

Main Results:

  • Recent approvals demonstrate successful generation of nonclinical and clinical data for pediatric oncology drug development.
  • Strategies for dose-finding and justification are highlighted through reviewed drug approvals.
  • Emerging trends include reduced nonclinical data requirements and increased use of model-informed drug development.

Conclusions:

  • Advances in molecularly targeted therapies, regulatory adaptations, and innovative trial designs are accelerating pediatric oncology drug approvals.
  • Model-informed drug development and reduced nonclinical data requirements support dose selection and optimize pediatric trial design.
  • Addressing clinical operational challenges and leveraging real-world data, external controls, and AI/ML are crucial for future pediatric cancer drug development.
Keywords:
clinical pharmacologyclinical trial designmodel‐informed drug developmentoncologypediatricreal‐world evidence

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