The Commission has said it wants to ‘streamline the regulatory framework’ underpinning the assistance given to manufacturers of orphan drugs and to adapt the policy to ‘technical progress,’ and has asked for comments from the pharma industry and other interested parties on how it should proceed.
In its preliminary text put out for comments, Brussels proposes clarification of how sponsors might demonstrate a significant benefit compared with authorised medicines and where and how a new pharmaceutical form could be justified. ‘Furthermore, in view of the development and further integration of the European pharmaceutical market, it seems appropriate to remove the possibility of claiming a significant benefit based on a potential increased supply,’ states the paper, although it acknowledges that some member states had suggested that the medicinal products prepared in a (hospital) pharmacy should be considered in the assessment of the significant benefit.
The Commission, noting that it had refused orphan designation for products intended for diseases that had been declared eradicated by the WHO, comments that the outbreak of Ebola had shown that an infectious disease with a very low prevalence in the EU can very rapidly become a serious threat to public health and ‘it may therefore be appropriate to apply a risk-based approach’ for deciding what is or is not an orphan drug.
As defined by the European Medicines Agency (EMA), orphan medicines are medicines developed to treat rare diseases deemed to be life-threatening or chronically debilitating to no more than five in every 10,000 people. Given that in normal market conditions, the pharmaceutical industry has little interest in researching and marketing medicines in such relatively small quantities, the EU offers incentives to encourage their manufacture, setting strict qualification criteria. At the heart of these is the requirement that any new Orphan Medicinal Product (OMP) has to demonstrate that it offers ‘significant benefit’ to patients over and above what is already available.
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