3. Special cases in marketing authorisations: Orphan Medicines

Orphan designation offers incentives to encourage development of orphan medicines and their availability. Incentives for sponsors include

• a 10-year period of market exclusivity once an orphan medicine is authorised, which is extended by two years for medicines that also have complied with an agreed paediatric investigation plan (see below).
• eligibility for Union and member state initiatives which support research and development of orphan medicines (e.g., research grants under EU Framework Programmes for Research and Innovation, E-Rare, a transnational project for research programmes on rare diseases)
• reduced fees for regulatory activities. This includes reduced fees for protocol assistance, marketing-authorisation applications, inspections before authorisation, applications for changes to marketing authorisations made after approval, and reduced annual fees.
• incentives in EU Member States for designated orphan medicines are available in the European Commission's Inventory of Union and Member State incentives to support research into, and the development and availability of, orphan medicinal products — state of play 2015

Of note: Some 60% of designated orphan medicines are intended for paediatric use since many childhood diseases are rare. Medicines authorised across the EU with the results of studies from a paediatric investigation plan included in the product information are eligible for an extension of their supplementary protection certificate. For designated orphan medicines, the incentive is an additional two years of market exclusivity.

Legal Basis:

Regulation (EC) No 141/2000
Commission notice on the application of Articles 3, 5 and 7 of Regulation (EC) No 141/2000 on orphan medicinal products setting out its interpretation on certain matters relating to the implementation of the designation and the market exclusivity provisions.
For a detailed inventory of the underlying legal basis see: EMA - Legal framework: orphan designation