Orphan incentives

The European Union (EU) offers a range of incentives for medicines intended for the treatment of small numbers of people.

Orphan designation

To benefit from these incentives, sponsors intending to develop orphan medicines must first submit an application to the European Medicines Agency requesting orphan designation for their medicine. This assessment is free of charge.

Information on this process is available under how to apply for orphan designation.

Incentives for designated orphan medicines

Sponsors with medicines designated as orphan medicines by the European Commission can benefit from a range of incentives:

Protocol assistance

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 benefits and risks, and information on the significant benefit of the medicine. Questions can concern studies looking at the quality, non-clinical and clinical aspects of a medicine. Protocol assistance is free of charge, both for initial requests and for follow-up requests.

• European Medicines Agency guidance for companies requesting scientific advice and protocol assistance
• Scientific advice and protocol assistance

Access to the centralised authorisation procedure

All designated orphan medicines can be 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.

• Central authorisation of medicines

Ten years of market exclusivity

Authorised orphan medicines benefit from ten years of protection from market competition once they are approved.

Fee reductions

Companies applying for designated orphan medicines pay reduced fees for regulatory activities. This includes reduced fees for marketing-authorisation applications, inspections before authorisation, applications for changes to marketing authorisations made after approval and annual fees:

• Executive decision on fee reductions

Grants

The Agency does not offer financial grants for sponsors of orphan medicines, but funding is available from the European Commission:

• Seventh Framework Programme for Research and Technology
• International Rare Diseases Consortium (IRDIRC)

Some EU Member States also offer grants for the development of orphan medicines:

• France: Fondation Maladies Rares
• Italy: Telethon
• Spain: Instituto de Salud Carlos III

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

Incentives are available for designated orphan medicines in European Union Member States:

• Inventory of Community and Member States' incentive measures to aid the research, marketing, development and availability of orphan medicinal products

For more information, contact the medicines regulatory authority in your country:

• National competent authorities (human)