Accelerating Pediatric Cancer Drug Development: Challenges and Opportunities for Pediatric Master Protocols

Tahira Khan, Mark Stewart, Samuel Blackman, Raphaël Rousseau, Martha Donoghue, Kenneth Cohen, Nita Seibel, Mark Fleury, Bouchra Benettaib, Raleigh Malik, Gilles Vassal, Gregory Reaman

Research output: Contribution to journalArticlepeer-review

10 Scopus citations


Although outcomes for children with cancer have significantly improved over the past 40 years, there has been little progress in the treatment of some pediatric cancers, particularly when advanced. Additionally, clinical trial options and availability are often insufficient. Improved genomic and immunologic understanding of pediatric cancers, combined with innovative clinical trial designs, may provide an enhanced opportunity to study childhood cancers. Master protocols, which incorporate the use of precision medicine approaches, coupled with the ability to quickly assess the safety and effectiveness of new therapies, have the potential to accelerate early-phase clinical testing of novel therapeutics and which may result in more rapid approval of new drugs for children with cancer. Designing and conducting master protocols for children requires addressing similar principles and requirements as traditional adult oncology trials, but there are also unique considerations for master protocols conducted in children with cancer. The purpose of this paper is to define the key challenges and opportunities associated with this approach in order to ensure that master protocols can be adapted to benefit children and adolescents and ensure that adequate data are captured to advance, in parallel, the clinical development of investigational agents for children with cancer.

Original languageEnglish (US)
Pages (from-to)270-278
Number of pages9
JournalTherapeutic Innovation and Regulatory Science
Issue number2
StatePublished - Mar 1 2019


  • clinical trial design
  • drug development
  • master protocol
  • pediatric cancer
  • regulatory policy

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Public Health, Environmental and Occupational Health
  • Pharmacology (medical)


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