Table 1.

Comparison of regulatory requirements in the US vs the EU [28]

	US BPCA	US PREA (following the RACE for Children Act)	EMA EC 1901/2006
Voluntary/mandatory	Voluntary	Mandatory	Mandatory
Incentive	6-month patent extension	None	6-month patent extension*
Document	Written request from FDA	iPSP	PIP
Products with orphan designation	Included	Included if RACE for Children Act stipulation met	Included
Timing of submission of plan/waiver request	When data are available—must be submitted before loss of exclusivity to receive the incentive	End of phase 2 in adults and before phase 3 (required before submission of NDA/BLA)	Ideally at the end of phase 1 in adults
Decision-making body	FDA PeRC	FDA PeRC	EMA PDCO

BLA Biologics License Application, BPCA Best Pharmaceuticals for Children Act, EMA European Medicines Agency, EU European Union, FDA Food and Drug Administration, iPSP initial pediatric study plan, NDA New Drug Application, PDCO Paediatric Committee, PeRC Pediatric Review Committee, PIP pediatric investigation plan, PREA Pediatric Research Equity Act, RACE Research to Accelerate Cures and Equity, US United States

*Orphan-designated products may be granted a 2-year extension of exclusivity