European Commission launches orphan drug controls review
Following concerns that they may slow the development of rare diseases that are not found in the EU
The European Commission is undertaking a review of European Union (EU) orphan drug market approval controls, after concerns that they may slow the development of rare diseases that are not found in the EU.
In a consultation paper, the EC said the current maximum EU outbreak threshold for an illness targeted by a drug regarded as an orphan is five in 10,000 people – orphan drugs receive accelerated market approval because of potential low returns for pharmaceutical companies.
But the paper noted that a disease which is not found in the EU, but is found elsewhere, such as Ebola, 'can very rapidly become a serious threat to public health'. As a result, the Commission is proposing that this orphan drug threshold is made more flexible. 'It may therefore be appropriate to apply a risk-based approach,' said the Commission.
On the other hand, the Commission is proposing tightening other controls. As companies cannot obtain an orphan designation for a new pharmaceutical form if they already have such an authorisation for the same active substance, some have been re-submitting applications via third parties, with orphan rights being transferred back later.
'This practice can be considered as an attempt to circumvent the intention and the purpose of this provision,' said the Commission, which wants to block this move.
Brussels also wants to clarify that a drug must have 'a significant benefit over authorised products or other methods of treatment used in the EU' to secure orphan designation.
The proposals are subject to public consultation until 15 February 2016.