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Considerations on Design and Analysis of External Control in Pediatric Oncology.

Jingjing YeHaitao PanGregory ReamanSatrajit RoychoudhuryChengxing LuLindsay A RenfroYuan JiRong LiuYing YuanWeidong Zhang
Published in: Medical research archives (2024)
Pediatric cancer consists of a diverse group of rare diseases. Due to limited patient populations, standard randomized and controlled trials are often infeasible. As a result, single-arm trials are common in pediatric oncology and the use of external controls is often desirable or necessary to help generate actionable evidence and contextualize trial results. In this paper, we illustrate unique features in pediatric oncology clinical trials and describe their impact on the use of external controls. Various types of relevant external control data sources are described in terms of their utility and drawbacks. Statistical methodologies and design implications with external control are discussed. Two recent case studies using external controls to support pediatric oncology drug development are described in detail.
Keyphrases
  • palliative care
  • clinical trial
  • phase iii
  • phase ii
  • open label
  • randomized controlled trial
  • childhood cancer
  • squamous cell carcinoma
  • drinking water
  • electronic health record
  • artificial intelligence
  • genetic diversity