
Faculty, Staff and Student Publications
Publication Date
1-1-2024
Journal
Medical Research Archives
Abstract
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.
Keywords
Bayesian analysis, Pediatric oncology, combination therapy, external controls, prior clinical trial, registry
DOI
10.18103/mra.v12i1.5088
PMID
39026931
PMCID
PMC11257159
PubMedCentral® Posted Date
7-18-2024
PubMedCentral® Full Text Version
Author MSS
Published Open-Access
yes
Included in
Bioinformatics Commons, Biomedical Informatics Commons, Genetic Phenomena Commons, Medical Genetics Commons, Oncology Commons