Orphan designation: research and development

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The European Medicines Agency provides support, including regulatory guidance and incentives, for the research and development of medicines for rare diseases in the European Union (EU)

United Kingdom’s (UK) withdrawal from the EU

EMA has published important information to help users of the centralised procedure prepare for the expected consequences of 'Brexit', including their obligations related to establishment within the European Economic Area (EEA). For more information, see UK’s withdrawal from the EU.

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Horizon 2020 call 'New therapies for rare diseases' 

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:

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