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

This article provides a summary of discussions from the American Statistical Association (ASA) Biopharmaceutical (BIOP) Section Open Forum organized by the ASA BIOP Statistical Methods in Oncology Scientific Working Group in coordination with the US FDA Oncology Center of Excellence and LUNGevity Foundation on June 24, 2021, and January 13, 2022. Diverse stakeholders engaged in a discussion on how best to use various innovative clinical trial designs in designing future pediatric oncology trials. While standard randomized controlled trials are preferred to evaluate treatment effect in an unbiased manner, given the rarity of pediatric cancers, innovative strategies are needed to promote and assure timely cancer drug development in pediatric populations. The discussions highlighted the need to consider innovative designs with less stringent Type I error specification and Bayesian designs borrowing from external control data, or borrowing treatment effect information from adult data, or both. Such designs are available in the literature and some examples are summarized under the FDA Complex Innovative Trials Design Pilot Program (https://www.fda.gov/drugs/development-resources/complex-innovative-trial-design-meeting-program). Early consultation with global regulatory agencies for pediatric clinical trials can provide a better understanding of different features of the clinical trial design options for successful pediatric cancer drug development. [ABSTRACT FROM AUTHOR]