Considerations on Design and Analysis of External Control in Pediatric Oncology.
Med Res Arch
; 12(1)2024 Jan.
Article
in En
| MEDLINE
| ID: mdl-39026931
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.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Language:
En
Journal:
Med Res Arch
Year:
2024
Document type:
Article
Affiliation country:
Moldova
Country of publication:
United States