Table 4 –

Overview of Regional Pediatric Development Regulations

Agency	Pharmacokinetic Testing for Dosing	Requirement for an Appropriate Pediatric Formulation	Safety Endpoints	Efficacy Endpoints	Extrapolation	Pediatric Plan
EMA	Yes	Yes	Yes	Usually required	Not routinely acceptable	End of phase 1, amended as needed
FDA	Yes	Yes	Yes	Not required, if extrapolation supports efficacy	Yes	End of phase 21

¹To the FDA within 60 days of the end-of-phase 2 meeting or no later than 210 days before planned new drug application (NDA) submission, but not mandatory for orphan drug development programs.

FDA=Food and Drug Administration, EMA=European Medical Agency