EMEA awaiting guidelines for orphan drug breaches

The European Medicines Agency (EMEA) is waiting for new guidelines that could be used to reduce the period of market exclusivity granted to orphan drugs.

Although the orphan drug legislation is generally considered to have been highly successful, some small biotech companies are privately furious that a minority of products with orphan drug status - notably Novartis's Glivec - have become blockbusters. But this is very much an exception, said the EMEA's executive director Thomas Loenngren.

"It is not for us to regulate blockbusters," he stressed. "Glivec happened to be a product with many indications. That is acceptable according to the legislation."

But Loenngren pointed out that the orphan drug legislation introduced in 2000 contains a mechanism for re-evaluating designated orphan drugs in cases where the "profitability is far beyond what was expected".

European law provides for a period of 10 years of market exclusivity for orphan medicinal products. However Article 8(2) provides that this period may be reduced to six years if it is established that the designation criteria are no longer met.

The review procedure can be triggered if a member state informs the EMEA that in a particular case there are "indications suggesting that the orphan drug designation criteria are no longer met". The Committee on Orphan Medicinal Products would then review the evidence and advise the Commission whether the market exclusivity should be maintained or reduced.

Asked about the affordability of new drugs in general, Loenngren stressed that the high price of some medicines, particularly in oncology, was not an issue for the EMEA. However drug pricing was part of the remit of the Pharmaceutical Forum, whose final report was expected this autumn.

The forum - a platform where the Commission, member states, MEPs and pharma industry players can exchange information and views - has three working groups examining drug pricing, added therapeutic value and patient information.

Martin Terberger, head of DG Enterprise's pharmaceuticals unit, has already made clear that the Commission does not intend to have a central role in preparing an EU-wide medicine pricing and reimbursement system. However, that does not necessarily exclude agreement on methodologies to assess the value of innovation.

The added therapeutic value working group aims to improve consensus at European level between member states on the data needed to assess the relative effectiveness of new products compared with established products.

Loenngren said: "In the group on added therapeutic value and the pricing group, there is discussion on harmonisation of principles for pricing and reimbursement in the EU and some discussion about the methodology for added therapeutic value.

"In the autumn, when the final result of the pharma forum should be presented, we will see how far member states want to go to discuss this on a European level. Otherwise it is an issue that will be discussed individually in each member state."