Cystic Fibrosis Ireland Vow to Fight Decision of HSE to Turn Down Orkambi

Cystic Fibrosis Ireland Vows to Fight Decision of HSE to Turn Down Ground-breaking CF Drug Orkambi: Calls for Minister Harris to Intervene

27th November 2016

Cystic Fibrosis Ireland Vows to Fight Decision of HSE to Turn Down Ground-breaking CF Drug Orkambi: Calls for Minister Harris to Intervene

Philip Watt, CEO, Cystic Fibrosis Ireland, comments:

'Cystic Fibrosis Ireland, Ireland's national charity supporting people with cystic fibrosis (CF), has today expressed its dismay and its opposition to the news - leaked to a Sunday newspaper - that the Health Service Executive will not fund Orkambi, the ground-breaking drug for CF.'

'We will fight this decision and we call on the Minister for Health, Simon Harris TD, to use his Ministerial powers to intervene to ensure that Orkambi is made available in Ireland for the 550 patients who stand to benefit from this life-lengthening drug.'

'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 hospitalisations 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.'

'The long-term benefits of Orkambi have been confirmed by Professor Stuart Elborn, formerly of the City Hospital Belfast and now the lead CF clinician in the Brompton Hospital London. The data confirming the impact of Orkambi was presented at the North American CF Conference in October 2016.'

'Cystic Fibrosis Ireland is angry at the cynical way the HSE has conveyed this information to our patients, some of whom are very ill. The decision was leaked to a Sunday newspaper even before the Minister was informed, never mind patients. It’s a disgraceful and heartless form of communication.'


For further information: Philip Watt, CEO, Cystic Fibrosis Ireland, is available on 087 637 0557


Letter Received from Minister for Health Simon Harris on November 27th 2016

Dear Philip,

I wanted to write to you today on foot of the article in the Sunday Business Post regarding the drug, Orkambi.

I am extremely eager that your members, patients with CF and their families are kept fully informed of the process which the HSE has been engaged in with the drugs company, Vertex. I am both annoyed and upset that details seemed to leak regarding these discussions today and I understand the HSE has issued a statement to clarify the position.

Firstly, let me state that I have not yet received a decision from the HSE Directorate regarding Orkambi. The HSE is responsible for the assessment of new medicines, as you know, which is a statutory process.

Secondly, following the recommendation of the NCPE, the HSE has been engaged in negotiations with Vertex to try and find a way forward. I am frustrated that this seems to have been extraordinarily difficult. I understand that the HSE has engaged extensively with the company and has sought a significant price reduction.  I fully support the HSE in its attempts to lower the exorbitant price the company is seeking to charge for Orkambi for Irish patients.  It takes two sides to negotiate and Vertex clearly has a critical role in this regard.

Thirdly, as you are aware, the assessment process is a statutory process. The Oireachtas, in legislating for this process in 2013, gave full responsibility to the HSE.  My Department can only consider requests for funding requirement for a new drug if the HSE decides to reimburse the drug but does not have the resources to fund the medicine. 

Fourthly, whilst, again, the HSE has not concluded their assessment process, I want to assure CF 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.

Orkambi has not yet been approved for reimbursement by any public health service in Europe. Whilst the drug is available in Germany and France, it is 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 CF 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.

Philip I will keep you updated and please do keep in touch,

Best wishes.

Yours sincerely,


Simon Harris TD

Minister for Health

Letter written by CFI CEO, Philip Watt to Minister for Health Simon Harris on November 27th 2016

Thank you Minister 

On behalf of CFI I acknowledge your letter and phone call and we will be responding to the points you raise more comprehensively when I have had a chance to discuss them with our association. From the responses I have heard this morning I know that many families are devastated by the news to refuse funding of the ground breaking drug Orkambi. 

We take this opportunity to ask for a meeting with you as soon as possible to appeal this decision and to explore if there are grounds in your letter  that this is not the the end of the process. The HSE have been very disrespectful in the way that this has been conveyed to our patients through an apparently cynical leak to a Sunday newspaper at a very late hour and we have had scores of phone calls, texts and messages this morning from very upset parents and people with CF who share our sense of anger. I would request that immediate action is taken on this heartless method of communicating potentially life altering information to patients in this country, some of whom are extremely ill. We ask again that you use your powers as Minister to expedite the reimbursement of Orkambi as soon as possible as we know you have the powers to do so. 

Yours Sincerely,

Philip Watt


Cystic Fibrosis Ireland