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EMA to expand orphan drug HTA collaboration

Will further develop the concept of parallel scientific advice in 2014

The European Medicines Agency (EMA) is set to continue to offer orphan drug manufacturers the opportunity to receive parallel advice on benefit-risk, significant benefit and relative effectiveness.

The last of these is supplied by health technology assessment (HTA) bodies and is part of the EMA's existing collaboration with the EUnetHTA European network.

So far two medicines with an orphan designation have been through the Agency's programme of parallel scientific advice, but the EMA wants more manufacturers to make use of the process.

To that end the regulator's Committee for Orphan Medicinal Products (COMP) will this year further develop the concept, which encompasses advice from the CHMP, the COMP and HTA bodies.

The COMP is also set to launch an awareness drive on the need for orphan drugs to demonstrate a significant benefit in order to obtain 10-year market exclusivity, a major incentive provided by the European Orphan Regulation.

Developers can request protocol assistance for advice on how to demonstrate a significant benefit and in 2013 the COMP received 28 requests for protocol assistance related to the demonstration of significant benefit.

Overall the number of orphan drugs approved in Europe has doubled over the last two years but numbers remain low, rising from four in 2011 to 11 last year.

Nevertheless their numbers are steadily increasingly and in 2013 accounted for nearly 14 per cent of the medicines recommended by the EMA, with products given the green light including Otsuka's Deltyba and Lucane Pharma's Lucane both for multi-drug resistant tuberculosis.

It's a trend the regulator's Committee for Orphan Medicinal Products (COMP) suggests could be explained by life sciences companies making better use of the tools and support they receive from the EMA and the Orphan Regulation.

Bruno Sepodes, chair of the COMP, said: “12 years after the European Orphan Regulation came into force, it is undoubtedly serving its purpose, with more and more orphan medicines reaching patients with rare diseases each year.

"The joint efforts of the EMA committees, including the COMP, the Paediatric Committee and the CHMP, in providing scientific advice to companies during the development of their medicines, have been a driving force behind this success.”

The development follows the EMA's announcement at the end of last year that it plans to introduce more fee reductions for orphan drugs.

Article by
Dominic Tyer

14th February 2014

From: Regulatory

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