EU releases revised orphan drug designation guidelines
Rules clarify how sponsors should define the medical benefits
Revised guidelines have been released for the European Union (EU) pharmaceutical sector on how companies should apply for their medicines to be designated an orphan drug, and hence gain additional intellectual property protection and assistance with regulation, including potential administrative fee reductions.
The new rules have been released by the European Commission and clarify how sponsors should define the medical benefits and target treatment for their planned drug. They also allow electronic submission of applications to be made using a common US Food & Drug Administration/European Medicines Agency application form.
The new system also allows pharma companies to change their applications to take account of new scientific discoveries made while the medicines’ impact is explored.
'During the development of the product, the classification of a disease may change and the designated condition may need to be modified to better reflect the indication that the sponsor intends to request at the time of marketing, therefore, an existing designation can now be amended,' the Commission said.
The guidance text explains that the 'granted therapeutic indication at the time of marketing authorisation' should be backed by an 'assessment of the quality, safety and efficacy data submitted with the marketing application', noting that this 'may be different to the one proposed at the time of orphan designation application'.
It added that other changes, such as using a new salt or international non-proprietary name that does not affect treatment options should be addressed in an annual report 'or by submitting a new application for an orphan designation'.