Considerations on Design and Analysis of External Control in Pediatric Oncology

Authors

Jingjing Ye
Haitao Pan
Gregory Reaman
Satrajit Roychoudhury
Chengxing Lu
Lindsay A Renfro
Yuan Ji
Rong Liu
Ying Yuan
Weidong Zhang

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