A Bayesian approach in design and analysis of pediatric cancer clinical trials.

Pharmaceutical Statistics
Jingjing YeRajeshwari Sridhara

Abstract

It is well recognized that cancer drug development for children and adolescents has many challenges, from biological and societal to economic. Pediatric cancer consists of a diverse group of rare diseases, and the relatively small population of children with multiple, disparate tumor types across various age groups presents a significant challenge for drug development programs as compared to oncology drug development programs for adults. Due to the different types of cancers, limited opportunities exist for extrapolation of efficacy from adult cancer indications to children. Thus, innovative study designs including Bayesian statistical approaches should be considered. A Bayesian approach can be a flexible tool to formally leverage prior knowledge of adult or external controls in pediatric cancer trials. In this article, we provide in a case example of how Bayesian approaches can be used to design, monitor, and analyze pediatric trials. Particularly, Bayesian sequential monitoring can be useful to monitor pediatric trial results as data accumulate. In addition, designing a pediatric trial with both skeptical and enthusiastic priors with Bayesian sequential monitoring can be an efficient mechanism for early trial cessation for bo...Continue Reading

References

Jan 1, 1968·The Journal of Pediatrics·H Shirkey
Jun 30, 1995·Statistics in Medicine·J B Greenhouse, L Wasserman
Sep 4, 2007·Biometrics·Satoshi MoritaPeter Müller
Feb 17, 2010·Clinical Trials : Journal of the Society for Clinical Trials·Beat NeuenschwanderDavid J Spiegelhalter
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Apr 1, 2018·Pharmaceutical Statistics·Nigel DallowTimothy H Montague
May 16, 2018·Therapeutic Innovation & Regulatory Science·Tahira KhanGregory Reaman

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Citations

Nov 28, 2020·Pediatric Blood & Cancer·Alemayehu Y AkaluJingjing Ye
Apr 30, 2021·Clinical Cancer Research : an Official Journal of the American Association for Cancer Research·Julie A SchneiderRichard Pazdur

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