FDA approvals for pediatric cancer indications using children’s oncology group associated trials data
Menée aux Etats-Unis à partir de l'analyse des données 2000-2024 du site de la "Food and Drug Administration" (FDA), de la base clinicaltrials.gov et de la base du "Children’s Oncology Group" (COG), cette étude examine l'utilité des données des essais cliniques du COG pour l'autorisation des traitements destinés aux patients pédiatriques atteints d'un cancer
Background : The Children’s Oncology Group (COG), formed in 2000, is a large international trial network that has conducted over 330 clinical trials for pediatric, adolescent, and young adult cancers. The role of COG trial data for Food and Drug Administration (FDA) approval of pediatric oncology indications was assessed since 2000.
Methods : All FDA approvals for pediatric oncology indications between January 1, 2000, and December 31, 2024, were reviewed. Data were collected from the FDA website, clinicaltrials.gov and COG database.
Results : Twenty-five (47%) of the 53 FDA pediatric indication approvals for 37 different drugs or biologics utilized COG clinical trial data for the approval of the pediatric indication. The 21 COG studies used for approvals were supported by three different partnership models, with 14 indications (26% of all pediatric approvals) supported by data from 15 trials utilizing the COG cooperative agreement award from the National Cancer Institute (NCI).
Conclusion : Almost half of FDA approved pediatric indications for oncology therapeutics were supported by data from COG trials, highlighting the effectiveness of this public–private partnership model in producing the level of clinical evidence required for regulatory success.
Journal of the National Cancer Institute , résumé, 2026