This section provides guidance and procedural information on applying for orphan designation for medicines for rare diseases in the European Union (EU). It also includes information on what happens after a designation has been granted, including the incentives available for sponsors developing orphan medicines.
Orphan designation in the product lifecycle
To qualify for orphan designation, a medicine must meet a number of criteria:
- it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
- the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
- no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.
Applications for orphan designation are examined by the European Medicines Agency's Committee for Orphan Medicinal Products (COMP), using the network of experts that the Committee has built up. The evaluation process takes a maximum of 90 days from validation.
For information on how to apply, see how to apply for orphan designation.
After orphan designation
Sponsors who obtain orphan designation benefit from a number of 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.
When planning the development of their medicinal product, sponsors should consult the relevant scientific guidelines.
Sponsors must submit an annual report 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 10-year market exclusivity incentive. Sponsors may also need to submit an evaluation of orphan similarity.
For more information, see activities after orphan designation.
The Agency encourages companies developing orphan medicines to check whether they can be classified as a micro, small or medium-sized enterprise (SME). These companies benefit from further incentives, including administrative and procedural assistance from the Agency's SME office and fee reductions. For more information, see SME office.
Horizon 2020 call 'New therapies for rare diseases' (updated)
Horizon 2020 is the biggest ever EU research and innovation funding programme, running from 2014 to 2020.
Under its call New therapies for rare diseases, the European Commission will provide funding to clinical trials on substances that have received an orphan designation from the European Commission based on a recommendation from the COMP, where the clinical trial design takes into account recommendations from protocol assistance provided by EMA’s Scientific Advice Working Party (SAWP).
Prospective applicants should consider the timelines for both the orphan designation and protocol assistance procedures, to determine if they can meet the deadlines set by Horizon 2020:
For more information, see:
- Legal background
- How to apply for orphan designation
- Activities after orphan designation
- Guidance and forms
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- Guidance and forms
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