This page describes the a range of incentives offered in the European Union (EU) for medicines that have been granted an orphan designation by the European Commission.
The Agency provides a form of scientific advice specifically for orphan medicines called protocol assistance. This allows sponsors to get answers to their questions on the types of studies needed to demonstrate the medicine's quality, benefits and risks, and information on the significant benefit of the medicine.
Update: Protocol assistance is available at a reduced charge for designated orphan medicines, linked to a fee-reduction scale that depends on the status of the sponsor. There is no restriction on the number of times a sponsor can request protocol assistance.
For more information, see:
- Scientific advice and protocol assistance
- European Medicines Agency guidance for companies requesting scientific advice and protocol assistance
The Agency encourages sponsors to consider coordinating the timing of protocol assistance from the Agency with request for scientific advice from the United States Food and Drug Administration (FDA). Parallel scientific advice with the FDA is available:
- General principles: European Medicines Agency - FDA parallel scientific advice
- FDA Center for Biologics Evaluation and Research: procedures for Parallel Scientific Advice with European Medicines Agency
A scientific advice service is also available at the Japanese Pharmaceuticals and Medical Devices Agency (PMDA).
Access to the centralised authorisation procedure
All designated orphan medicines are assessed for marketing authorisation centrally in the European Union. This allows companies to make a single application to the European Medicines Agency, resulting in a single opinion and a single decision from the European Commission, valid in all EU Member States. Sponsor’s may also have access via orphan designation to conditional approval, which is conducted under the centralised procedure.
For more information, see central authorisation of medicines.
Ten years of market exclusivity
Authorised orphan medicines benefit from ten years of protection from market competition with similar medicines with similar indications once they are approved. This period of protection is extended by two years for medicines that also have complied with an agreed paediatric investigation plan which is granted at the time of review of the orphan medicinal designation.
- For more information, see marketing authorisation and market exclusivity and Questions and answers on the procedure of PIP compliance verification at EMA, and on paediatric rewards.
Additional incentives for micro, small and medium-sized enterprises (SMEs)
Companies classified as SMEs benefit from further incentives when developing medicines with orphan designation. These include administrative and procedural assistance from the Agency's SME office and fee reductions.
- For more information, see SME office.
Companies applying for designated orphan medicines pay reduced fees for regulatory activities. This includes reduced fees for protocol assistance, marketing-authorisation applications, inspections before authorisation and applications for changes to marketing authorisations made after approval, and reduced annual fees.
Fee reductions are revised each year in relation to the budget available.
The Agency does not offer research grants for sponsors of orphan medicines, but funding is available from the European Commission and other sources:
- Horizon 2020, the EU Framework Programme for Research and Innovation (see the theme Personalising Health and Care which covers New therapies for rare diseases);
- E-Rare, a transnational project for research programmes on rare diseases;
- International Rare Diseases Consortium (IRDIRC).
Grants are also available for sponsors considering research in the United States or Japan:
- United States: Food and Drug Administration: Orphan products grants program
- Japan: National Institute of Biomedical Innovation: Services to promote development of medicinal products for rare diseases
Incentives in Member States
The incentives available for designated orphan medicines in EU Member States are detailed in the 2013 Report on the state of the art of rare disease activities in Europe from the European Union Committee of Experts on Rare Diseases (EUCERD).
For more information, contact the medicines regulatory authority in your country:
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