Generating the evidence needed to extrapolate efficacy of anti-seizure medications from adults to pediatric patients aged ≤ 4 years with focal onset seizures: a partial extrapolation exercise.

In Europe, addition of a new indication to the product information of anti-seizure medications (ASMs) usually requires support through specific clinical studies in the sought epilepsy (sub)population. An extrapolation exercise, wherein existing information derived from one patient (sub)population can be used to make inferences about another, could provide an alternative route to support these changes. Currently partial extrapolation of efficacy data from adults with focal onset seizures (FOS) to pediatric patients aged 4 years and older with FOS is considered established. This project aims to evaluate how partial extrapolation in patients younger than 4 years with FOS can be justified.