Thursday 18 January 2018

Deal finally reached for 'vital' cystic fibrosis drug

Hazel Robinson, of Drumlish, Co Longford, who has been sleeping outside the Dáil in the hope of getting long-term access to Orkambi for daughter Gypsy Ann (14). Photo: Tom Burke
Hazel Robinson, of Drumlish, Co Longford, who has been sleeping outside the Dáil in the hope of getting long-term access to Orkambi for daughter Gypsy Ann (14). Photo: Tom Burke
Eilish O'Regan

Eilish O'Regan

Around 600 people with cystic fibrosis finally reached the end of a gruelling campaign last night after it was confirmed they will be offered two groundbreaking drugs from the beginning of next month.

But the price for one of the drugs Orkambi - the most expensive medicine ever purchased here - has to remain "confidential".

The confidentiality clause emerged after Health Minister Simon Harris announced the game-changer drug would be available to around 550 people with cystic fibrosis from May.

Another drug Kalydeco will also be extended to younger children between the ages of two and five years.

Agreement "in principle" has been reached between the HSE and Vertex, makers of both medicines, and final details have yet to be worked out.

The news was greeted with relief by Cystic Fibrosis Ireland, which has mounted a long-running campaign to make Orkambi available here.

The drug, which improves lung function and cuts hospitalisations, was originally rejected as too expensive after Vertex demanded €159,000 per person for the medicine.

Jillian McNulty, whose life has been transformed since she started on Orkambi as part of a trial, said last night she was overwhelmed that the fight of more than two years was finally over.

"Within a few months my body started changing after starting on the drug which treats the underlying causes of the disease," she said.

Philip Watt of Cystic Fibrosis Ireland said around 550 cystic fibrosis patients are waiting to try Orkambi. It is unclear how many will benefit, but experts say it could be as many as 150. Another 40 younger people are waiting for Kalydeco.

"There will be around 30pc of the CF population that still have no drug that treats the underlying cause of their condition in Ireland," he said.

That is why the "pipeline deal" - which is a feature of this agreement - is so important, he said. There also may be better drugs for those on existing Vertex drugs coming down the line.

Vertex recently issued very positive news on phase three clinical trials for drug Tezacaftor which is also showing dramatic improvements in lung function.

The agreement secures availability of new drugs from Vertex coming on stream in the future and an end to similar campaigns.

Irish Independent

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