EU orphan designations rise
pharmafile | February 19, 2013 | News story | Research and Development, Sales and Marketing | COMP, EMA, Orphan, hta
The number of orphan drug designations in the EU is set to rise to record levels this year, according to the European regulator.
The European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) says more than 150 are expected in 2013: this compares with 107 designations granted in 2011 and 148 in 2012.
In all, orphan medicines were the subject of 19 applications for marketing authorisation last year, compared with 14 in 2011.
The upward trend is, COMP says, evidence of the success of orphan incentives, in particular the EU’s Orphan-Medicinal-Product Regulation introduced 12 years ago.
And COMP now says it is bolstering its efforts this year to speed the development of orphans, in particular by taking more account of patient views and developing links with international health technology assessment (HTA) groups.
“The work of the COMP is evolving and constantly adapting to better serve and address patients’ needs, the growing scientific knowledge on rare diseases and the regulatory framework,” explains COMP chair Bruno Sepodes.
The committee is to proactively request contributions from expert patients on issues such as a drug’s contribution to patient care, improvement in quality of life and possible use in specific indications.
As part of this move it says it will ‘strengthen its interactions’ with disease-specific patient organisations and the European Organisation for Rare Diseases (EURORDIS).
It will also collaborate more closely with HTA bodies such as NICE to gain a better understanding of orphan designation.
COMP has already started working closely with EUnetHTA, an organisation which promotes HTA co-operation in Europe.
Since 2008 it has had an agreement with the FDA which allows applications for orphans to be submitted in the US and Europe at the same time: 62% were put forward in parallel last year.
COMP wants to move towards similar arrangements with authorities in Japan and Canada.
Drugs can be granted orphan status at any stage of their development if they are intended to treat very serious conditions affecting not more than five in 10,000 people in the EU, or diseases for which development would not be commercially viable without incentives.
Sponsors of orphan drugs in Europe get market exclusivity for ten years along with special help in getting the authorisation process to flow smoothly.
Paying for such treatments is a question which the Scottish government has recently addressed, launching a fund to meet orphan costs last month.
Its new £21 million pot comes into being next month, and is earmarked through to April 2014 and will be used to cover treatments not recommended by the Scottish Medicines Consortium (SMC).
Adam Hill
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