Statistical Considerations in Pediatric Cancer Trials: Report of American Statistical Association Biopharmaceutical Section Open Forum Discussions

Sridhara, Rajeshwari; Marchenko, Olga; Jiang, Qi; Barksdale, Elizabeth; Alonzo, Todd A.; Amatya, Anup; Arons, David; Bliu, Alex; Choo, Qiuyi; Coory, Michael; Donoghue, Martha; Ehrlich, Lori A.; Filho, Leis; Fox, Elizabeth; Freidlin, Boris; Goodman, Nancy; Hawkins, Douglas S.; Häring, Dieter A.; Karres, Dominik; Kolb, E. Anders; Mao, Hejun; Kalyani, P; Naranjo, Arlene; Pappo, Alberto S.; Posch, Martin; Price, Karen; Raven, Andrew; Rantell, Khadija; Renfro, Lindsay A.; Rivera, Donna R.; Roy, Pourab; Shan, Minghua; Simon, Richard H.; Singh, Sonia; Smith, Malcolm A.; Theoret, Marc R.; Thomas, Marius; Thomas, Zachary M.; Thompson, Andrew; Tian, Hong; Tymofyeyev, Yevgen; Vallejo, Jonathon; Wathen, Kyle; Ye, Jingjing; Pazdur, Richard; Reaman, Gregory H.

doi:10.1080/19466315.2023.2238650

Cited by 2 publications

(1 citation statement)

References 11 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The rarity of pediatric cancer renders it impractical to execute gold standard randomized clinical trials in majority of conditions. Additionally, clinical equipoise may be difficult to assess when a drug is already approved for adults and is available on the market (50). Rather than "nice-to-have", external control data emerges to be necessary in many instances to demonstrate efficacy either by supplementing or replacing control data in a prospective clinical trial.…”

Section: Discussionmentioning

confidence: 99%

Global Statistics and Data Sciences (GSDS), BeiGene USA, Fulton, MD

Ye,

Pan,

Reaman

et al. 2024

MRAJ

View full text Add to dashboard Cite

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.

show abstract

Section: Discussionmentioning

confidence: 99%