HSE seems set to reject ‘ground-breaking’ cystic fibrosis drug

Orkambi had been ‘considered unjustifiably expensive and not sufficiently cost effective’

The HSE seems set to reject a “ground-breaking” but highly expensive drug for patients with cystic fibrosis in the public health system.

The HSE said on Sunday that to date the drug, Orkambi, had been “considered unjustifiably expensive and not sufficiently cost effective” by both the National Centre for Pharmacoeconomics (NCPE) and the HSE drugs committee.

The Sunday Business Post reported that a formal announcement by the HSE was imminent after its drugs committee recommended against funding Orkambi at a recent meeting.

The newspaper said the committee had concluded that the drug did not deliver sufficient benefits to patients to justify its annual price tag of €159,000.


Cystic Fibrosis Ireland, the organisation that represents people with the condition, said on Sunday that it would fight any move by the HSE to reject the new drug. It called on the Minister for Health Simon Harris to intervene to ensure Orkambi was made available to about 550 patients which it said could benefit from "from this life-lengthening drug" .

The HSE said it had undertaken considerable engagement and negotiation with the manufacturer Vertex over a protracted period, in an effort to secure a significant price reduction for Orkambi with a view to having it approved for reimbursement for people with cystic fibrosis in Ireland.

"The HSE is disappointed around the level of meaningful engagement by the manufacturer (Vertex) in lowering the exorbitant price of Orkambi for Irish patients (and indeed equally for cystic fibrosis patients in the UK, Canada, Scotland and Australia)."

“The HSE has recently completed its most recent round of negotiations with Vertex. On foot of this, the HSE’s drugs committee (together with a NCPE recommendation and other expert advice) is in the process of sending its latest recommendation on the cost-effectiveness of Orkambi to the HSE directorate.

“The HSE directorate will then make a decision taking into consideration the manufacturers latest price offer.”

Mr Harris, on Sunday wrote to Cystic Fibrosis Ireland and said he fully supported the HSE in its attempts “to lower the exorbitant price” the company was seeking to charge for Orkambi for Irish patients.

He said it required two sides to negotiate “and Vertex clearly has a critical role in this regard”.

Mr Harris said while the HSE had not concluded its assessment process, “I want to assure cystic fibrosis patients and their families that I do not see this as the end of the process”.

“I want everyone to work to try and find a way forward but let me be clear, this will require Vertex to further engage on a meaningful and innovative basis and to significantly reduce their price.”

Different process

Mr Harris said Orkambi had not yet been approved for reimbursement by any public health service in Europe. He said while the drug was available in Germany and France, it was through a very different process.

"Ireland, the UK, Canada and Australia all find themselves trying to negotiate without success so far. Clearly the time has come for a different approach from Vertex. I now intend to write to fellow health ministers in these countries engaged with Vertex on this matter to seek an understanding of their experience and perspective in addressing this."

“I can only imagine how stressful a time this must be for many families and that is why I want to assure them that we will not leave any stone unturned on this matter - but equally Vertex need to do likewise.

“ In the meantime it is important to remember and to remind cystic fibrosis patients that Vertex have confirmed (as stated on the NCPE website) that patients currently receiving Orkambi under the Managed Access Scheme will continue to receive the drug until reimbursement is finalised in Ireland.”

Highest rate globally

The chief executive of Cystic Fibrosis Ireland Philip Watt said: "With around 1,200 people with cystic fibrosis here in Ireland, we have the highest rate of the disease per head of population in the world. We also have some of the most severe forms of cystic fibrosis.

“It is rare that a therapy comes along that has been shown to reduce hospitalisation by up to 40 per cent and further it significantly retards the progression of cystic fibrosis for those who stand to benefit from this drug. This drug would have resulted in other major savings to the HSE from both reduced hospitalisation and less reliance on other expensive CF drugs and reduced dependency on transplants.”

Martin Wall

Martin Wall

Martin Wall is Washington Correspondent of The Irish Times. He was previously industry correspondent