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EMA sees backing for more orphan drugs


The European Medicines Agency has said it has seen a rising trend in the number of orphan medicines recommended for marketing authorisation.

In a Monday statement, the EMA said a total of 11 out of 81 medicines recommended by its CHMP scientific committee in 2013 were for treatment of rare diseases.

This continued a year-on-year trend in the number of marketing authorisation recommendations in recent years. The CHMP recommended eight in 2012 and four in 2011.

The volume of applications for orphan designation submitted to the agency has increased 22% since 2009 and the number seeking development advice increased from 82 requests in 2012 to 108 requests in 2013.

The EMA said one of the explanations for the trend offered by its Committee for Orphan Medicinal Products (COMP) is that developers are making better use of the tools offered by the agency and the European Orphan Regulation to support development.

In 2013 the CHMP granted conditional marketing authorisations for four orphan medicines, compared with one in 2012 and none in 2011.

This follows an increasing number of companies requesting advice on the procedure, which allows a marketing authorisation from less complete data.

Around 40% of applications for orphan designation are submitted by small and medium sized companies.

The EMA added it expects to see more parallel applications for orphan regulations with other regulators and has been collaborating on this with the U.S. regulator since 2008 and the Japanese regulator since 2012.

Around half of all orphan designation applications was submitted in paralel with other regulators in 2013 and a similar trend is expected in 2014.

The EMA in March will host at its offices a joint workshop with the U.S. Food and Drug Administration and the Japanese regulatory authorities, focusing on the orphan programmes in the three territories.

The EMA said that two medicines with an orphan designation had been through its programme of parallel scientific advice for health technology assessment. This is a key area of collaboration between the EMA and EUnetHTA, which focuses on scientific cooperation in HTA in Europe.

The COMP will this year raise awareness of the need for designated orphan medicines to demonstrate a significant benefit in order to obtain 10-year market exclusivity.

Developers can ask for advice on demonstrating significant benefit - and the EMA said it received 28 such requests in 2013.

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