A review on the role of extrapolation as basis for paediatric marketing authorization applications of medicines in the EU.

ABSTRACT:

Aims

For new medicines, drug companies obtain regulatory approval on the strategy to generate evidence in the paediatric population, which can be supported by extrapolation of evidence obtained in a reference population. This study investigated whether paediatric marketing authorization applications (PMAAs) supported by extrapolation based on exposure-matching were more successful-i.e. approval of the targeted paediatric population-and efficient-i.e. duration of the drug development-compared to PMAAs not supported by extrapolation.

Methods

Data was extracted from completed paediatric investigation plans (PIPs), associated drug labels and public assessment reports published on the European Medicines Agency website. Assessment reports were evaluated to assess whether PMAAs wer

SUBMITTER: Simons CWM 

PROVIDER: S-EPMC12035585 | biostudies-literature | 2025 May

REPOSITORIES: biostudies-literature