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June 10, 2021

Master Protocols and Adaptive Trial Designs to Develop Tumor-Agnostic Drugs for Children: Essential Tools in the Era of the Research to Accelerate Cures and Equity Act

Author Affiliations
  • 1Harvard-MIT Center for Regulatory Science, Harvard Medical School, Boston, Massachusetts
  • 2Dana-Farber Cancer Institute/Boston Children’s Hospital, Boston, Massachusetts
  • 3Pediatric Therapeutics and Regulatory Science Initiative, Computational Health Informatics Program, Boston Children’s Hospital, Boston, Massachusetts
  • 4Department of Pediatrics, Harvard Medical School, Boston, Massachusetts
JAMA Oncol. 2021;7(9):1281-1282. doi:10.1001/jamaoncol.2021.1508

Progress in drug development has steadily improved survival from childhood malignant tumors over the past decades, but there is still a tremendous need for safe and effective drugs with which to treat pediatric cancers. Between 10% and 30% of children with cancer die within 5 years of diagnosis.1 Secondary tumors and treatment-related toxic effects, such as cognitive and endocrine dysfunctions, are also common among young survivors.

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