Perspectives on informative Bayesian methods in pediatrics

ABSTRACT

Bayesian methods have been proposed as a natural fit for pediatric extrapolation, as they allow the incorporation of relevant external data to reduce the required sample size and hence trial burden for the pediatric patient population. In this paper we will discuss our experience and perspectives with these methods in pediatric trials. We will present some of the background and thinking underlying pediatric extrapolation and discuss the use of Bayesian methods within this context. We will present two recent case examples illustrating the value of a Bayesian approach in this setting and present perspectives on some of the issues that we have encountered in these and other cases.

KEYWORDS:

• Bayesian
• clinical trials
• pediatric
• extrapolation
• external data

Acknowledgements

The authors would like to thank Gregory Levin, Stefanie Kraus, Roberto De Lisa and Juan Jose Abellan Andres for their helpful comments and suggestions.

Disclosure statement

The authors report there are no competing interests to declare.

Additional information

Funding

The author(s) reported there is no funding associated with the work featured in this article.