- In Europe, there are about 7,000 rare diseases affecting 36 million people. Roughly 1 in 17 people will be affected by a rare disease at some point in their life
- Orphan designation criteria:
- Intend to treat/prevent/diagnosis of a life-threatening or chronically debilitating disease
- prevalence must not be more than 5 in 10,000
- there is no satisfactory treatment currently available, or the new treatment offers significant benefit to patients
- The EMA’s COMP reviews submissions for orphan drug designations and provides a positive or negative opinion. Based on this, the European Commission may then grant Orphan Designation
In this section we review EU Orphan Drug Designation, outlining the benefits for pharmaceutical companies and summarising the regulations for Orphan Drug market exclusivity. We detail the pricing and reimbursement journey as, after authorisation, a significant challenge for orphan drugs lies in convincing payors and regulators in each European market that the drug has sufficient value to justify pricing and reimbursement.