Table 3.
Article 58 versus Orphan Designation: comparison of requirements and incentives
| Regulatory aspects | Article 58 | Orphan Medicinal Product |
|---|---|---|
| Medicinal Product | ||
| Pre-submission phase | ||
| Eligibility | Needed (in collaboration with WHO) | Needed (assessed by COMP) |
| SME status | Can be granted | Can be granted |
| Scientific advice | Possible | Possible |
| PIP | Not legally required | Legally required (compliance check prior to MAA submission) |
| Accelerated review request | Possible | Possible |
| Evaluation phase | ||
| Environmental risk assessment | Not legally required | Legally required |
| Data applicable to EU population | Not required | Required |
| Application fee reduction | Eligible (case-by-case) | Yesa |
| Inspections fee reduction | Eligible (case-by-case) | Full fee reduction |
| CHMP opinion | ||
| Conditional or exceptional circumstances | Possible | Possible |
| Post-opinion phase | ||
| Marketing authorisation (EEA) | No (allows future MAA submission in the EU) | Yes |
| Market exclusivity | No (since no EU MAA) | Yes |
| PhV system / RMP | Needed (adapted to local use) | Needed |
| PSUR | Submission mandatory | Submission mandatory |
| Fee reductions | Eligible (case-by-case) | Yesa |
COMP: Committee for Orphan Medicinal Products; EEA: European Economic Area; EMA: European Medicines Agency; MAA: marketing authorisation application; PhV: pharmacovigilance; PIP: paediatric investigation plan; PSUR: periodic safety update report; RMP: risk management plan; SME: micro, small and medium-sized enterprises.
a See details in the explanatory fee note: http://www.ema.europa.eu/docs/en_GB/document_library/Other/2014/04/WC500164415.pdf