This section provides information on the incentives available to sponsors of medicines that have obtained orphan designation and the activities that take place after a designation has been granted.
Sponsors who obtain orphan designation benefit from a number of orphan incentives, including protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity once the medicine is on the market. Fee reductions are also available depending on the status of the sponsor and the type of service required.
Sponsors must submit an annual report on development to the Agency summarising the status of development of the medicine.
Applications for marketing authorisation for designated orphan medicines are assessed by the Committee for Medicinal Products for Human Use (CHMP). Sponsors also need to submit an application for maintenance of the orphan designation in order to be eligible for the ten-year market exclusivity incentive. For more information, see marketing authorisation and market exclusivity.
Transfers of orphan designation from one sponsor to another are possible. Transfers are free of charge.
Sponsors can also request removal of an orphan designation.
Sponsors of medicines with orphan designation should also remember to apply for a paediatric investigation plan (PIP), deferral or waiver once phase-I clinical studies are complete. For more information, see Rewards and incentives for paediatric medicines.
A valid and completed paediatric investigation plan (PIP) could make the sponsor eligible for the two-year marketing exclusivity extension onto the ten-year marketing exclusivity which is granted at the time of review of the orphan medicinal designation. Each orphan designation for a product linked to a separate orphan condition is eligible for a two-year extension if this is accounted for in the PIP. The extension is granted by the European Commission based on the positive compliance check from the Paediatric Committee and opinion from the CHMP. For further information please refer to Questions and answers on the procedure of PIP compliance verification at EMA, and on paediatric rewards.
Guidance for sponsors:
- Orphan incentives
- Annual report on development
- Marketing authorisation and market exclusivity
- Transfers of orphan designation
- Changing the name or address of a sponsor
- Removal of an orphan designation
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