Orphan drug Kalydeco now available in Wales

In Regulation & Government by Tim Peplow


The much-celebrated orphan drug Kalydeco (ivacaftor) will be made available to the estimated 13 eligible patients with cystic fibrosis in Wales, confirmed Mark Drakeford, Welsh Assembly Minister for Health and Social Services, last Friday (May 10th). Not only will this be great news for the manufacturers, Vertex, but it’s also great news for patients and those who campaigned for the drug to be made equally accessible across the UK.

“We are delighted that the Minister has recognised that patients in Wales should have the same access as people in the UK to this medicine,” said Ed Owen, Chief Executive of the Cystic Fibrosis Trust. “I would like to thank Mr Drakeford for listening to the voice of people with cystic fibrosis in Wales.”

The news comes after the All Wales Medicines Strategy Group (AWMSG) had recommended against funding the orphan drug – which is estimated to be about £180,000 per patient per year.

According to the Cystic Fibrosis Trust, about 4% of patients across the UK have the G551D mutation for which Kalydeco is licensed. In a statement, Vertex said they were encouraged to learn the Chief Pharmaceutical Officer in Wales has been asked by the minister to set up a review of the Welsh appraisal process for orphan and ultra-orphan drugs.

Are you pleased to see Kalydeco being made increasingly available in Europe?

This news comes amid reports that the orphan drug market is set to reach $127 billion by 2018. This infographic shows some more important facts about orphan drugs and rare diseases.

If you want to know more about innovation and strategy in the orphan drug market, you might be interested in attending the World Orphan Drug Congress Asia, 18-19 June 2013, Singapore, or the 4th World Orphan Drug Congress Europe, in Geneva on the 14th November 2013.