Spring Intro 2023

30/03/2023

What & Why?

EU Orphan disease eligibility criteria • Affects less than 5 in 10000 • US: condition affects fewer than 200,000 patients • Insufficient return on company investment without incentives • Life-threatening or chronically debilitating • Not a requirement for US orphan designation • No existing satisfactory therapy or will provide significant benefit compared to existing therapies

The Organisation for Professionals in Regulatory Affairs

55

What & Why ?

Incentives • Access to Centralised Procedure and Protocol Assistance • 10 years market exclusivity • Additional 2 years on completion of agreed PIP • Reduced fees (Protocol Assistance, Inspection and MAA) • Research grants • Additional national measures • Tax incentives, pricing, access/reimbursement incentives

The Organisation for Professionals in Regulatory Affairs

56