gms | German Medical Science

Bayesian methodology is often proposed in the context of rare diseases and pediatric development plans where resources are limited in terms of patients for clinical trials. In the special case of pediatric extrapolation substantial evidence for efficacy of a medical product is often already available in adults. This information can be used in the extrapolation exercise to borrow information for efficacy evaluation in the pediatric population. However, little is said about the true impact of this borrowing on the evaluation of outcome in the pediatric study and even less is said about the ability to set-up pre-specified strategies for decision making as required in the regulatory context.

Several Bayesian and frequentist methods are applied to an exemplary extrapolation exercise which consists of already conducted trials in the adult population and differing assumptions about the efficacy in the pediatric population. Based on the results differences and similarities between meta-analyses of pediatric and adult trials and Bayesian extrapolation with efficacy priors based on adult data are explored and discussed.

Die Autoren geben an, dass kein Interessenkonflikt besteht.

Die Autoren geben an, dass kein Ethikvotum erforderlich ist.