Targeted consultation on the guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one

Public health community

Targeted stakeholders

The stakeholders involved in the development of orphan medicinal products.

Objective of the consultation

The current revision of the Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another addresses the European Medicines Agency new online platform to submit applications for designations as orphan medicinal products.

This guideline is intended to provide supplementary advice on how to compile the information that should be provided by sponsors in an application for orphan medicinal product designation.

The comments received will be taken into account by the European Commission in the finalisation of the Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another.

The consultation document

• PDF version
• Word format with track changes

Protection of personal data

The European institutions are committed to protecting and respecting your privacy. As this service/application collects and further processes personal data, Regulation (EU) 2018/1725 of the European Parliament and of the Council of 23 October 2018 on the protection of natural persons with regard to the processing of personal data by the Union institutions, bodies, offices and agencies and on the free movement of such data, is applicable. More information is available here.

Specific privacy statement

Please consult the Privacy Statement.

Additional information:

• Orphan medicinal products
• Orphan medicinal products - Major developments
• EMA - orphan medicinal products

How to submit your contribution

Stakeholders are invited to comment on this consultation by 28 April 2019 at the latest. Responses should preferably be sent to sante-pharmaceuticals-B5ec [dot] europa [dot] eu (sante-pharmaceuticals-B5[at]ec[dot]europa[dot]eu)

They can also be sent by post to Directorate-General for Health and Food Safety, Unit SANTE B/5, BE-1049 Brussels. The subject line of the letter or email should contain the reference "TSC on Revision of the Guideline on the format and content of orphan applications".

When submitting your response, please include your name and e-mail address and specify if you are responding as an individual or as a representative of an organisation. If you represent an organisation, please indicate its name and category (company/business; public authority (local, regional, national, international); patient organisation; healthcare professional organisation; academic; NGO; other).

Contact details

Any queries about the targeted stakeholder consultation should be sent to: Unit B5 - Medicines: policy, authorisation and monitoring, Directorate-General for Health and Food Safety sante-pharmaceuticals-B5ec [dot] europa [dot] eu (sante-pharmaceuticals-B5[at]ec[dot]europa) by referring by containing the reference "TSC on Revision of the Guideline on the format and content of orphan applications”.