Bobby Aylward (Carlow-Kilkenny, Fianna Fail)
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I am disappointed the Minister for Health, Deputy Simon Harris, is not here. The matter is the urgent need for the Minister for Health to request an update from the HSE drugs group on reimbursement of the drug Vimizim.

Martin Kenny (Sligo-Leitrim, Sinn Fein)
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Two of the children who use this drug are in the Public Gallery. One of them is from Leitrim and one from Kilkenny. Both of them are very anxious and worried that they have not got any decision. They really depend on getting this drug approved. There are only three children in the country using it. I appeal for something to happen in this regard.

Bobby Aylward (Carlow-Kilkenny, Fianna Fail)
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I thank the Leas-Cheann Comhairle for selecting this matter for discussion. The drug Vimizim is produced by BioMarin in its manufacturing operations in Shanbally, Co. Cork. It has been granted market authorisation in many countries worldwide for treatment of Morquio A syndrome. This is a rare, degenerative, potentially life threatening genetic condition that affects major organ systems in the body. It can substantially limit the quality of life of those affected and patients rarely live beyond the second or third decade of life.

This drug, Vimizim, is the first and only enzyme replacement therapy designed to target the underlying cause of this disease, which currently affects only ten known patients in Ireland. As my colleague has said, one of these patients is an 11 year-old girl from my constituency, Carlow-Kilkenny, who has battled bravely to lead a robust and fun filled life despite this challenging medical condition. This little girl’s family were delighted when their daughter was one of two Irish children included in the trials for Vimizim which began in September 2011. I want to read a previous statement from her mother, who is in the Gallery, into the record:

This is a progressive and damaging condition. I don't know how she will be without the medicine. It gives her stamina and energy and it is our only chance for her.

Unfortunately this brave young girl could lose her access to this drug very shortly. The voluntary drug supply that has been given to her is being withdrawn next Friday. From next Friday, this child will not have this drug that has given her the quality of life of the last couple of years.

The HSE drugs group has denied a number of applications for reimbursement. It has denied it twice so far. BioMarin submitted an application for reimbursement to the HSE in 2015. On 15 June 2017 it received a letter from the HSE confirming the decision not to reimburse the drug Vimizim in Ireland. The decision not to reimburse the drug on the grounds of insufficient clinical data and lack of value for money is surprising, given the nature of rare disease medicines and the acceptance of these in other countries. BioMarin met with the HSE again in July 2017 and resubmitted additional evidence and a new commercial offering for consideration at the HSE drugs group meeting on 18 September 2017. On 13 November 2017 while attending the HSE on another matter, BioMarin was informed that the HSE would not reimburse Vimizim for Irish patients. That was the second time.

The negative feedback from the HSE for a second time was particularly unfortunate, given that the drug is currently available to children and adults with Morquio A syndrome in many countries following an assessment of the very same data. The drug is currently commercially available to patients in the United Kingdom, our next door neighbours, Northern Ireland which is joined to us, Germany, Spain, France, Italy, the Czech Republic, Hungary, countries in the Middle East, the Russian Federation, Brazil, Canada, and the United States.

BioMarin requested a meeting with the HSE again in December 2017 and agreed to submit a new application, which was due to be discussed at the HSE drugs group meeting on 18 January 2018, just a couple of weeks ago. However, since this date they have had no communication from the drugs group, which leaves both patients and the company in the dark as to whether the drug will be reimbursed or not. We understand from parliamentary questions, of which I submitted many, however, that the drug was discussed at this meeting. I know very well the Minister of State will state that responsibility for decisions on reimbursement is solely that of the HSE drugs group and he is bound by legislation not to intervene. Surely the Minister can contact the HSE drugs group directly on behalf of the young people who are here today, their families and the other eight known Irish people with this condition and demand that they urgently report on their most recent meeting of 18 January.

Jim Daly (Cork South West, Fine Gael)
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I thank the Deputies for raising this issue. It was raised this morning as well during Leaders Questions. I acknowledge the people in the Gallery who have more than a passing interest in this serious issue and in ensuring that we get a satisfactory outcome.

Securing access to new and innovative medicines in a timely manner is a key objective of the Minister for Health and the Health Service Executive. However, these new products are often very expensive, with significant potential impact on the health service budget. The Oireachtas has put in place a robust legal framework in the Health (Pricing and Supply of Medical Goods) Act 2013, which gives full statutory powers to the HSE to assess and make decisions on the reimbursement of medicines, taking account of a range of objective factors and expert opinion as appropriate. The Act specifies the criteria to be applied in the making of reimbursement decisions which include the clinical and cost effectiveness of the product, the opportunity cost and the impact on resources that are available to the HSE. In reaching its decision, the HSE will examine all the relevant evidence and will take into account such expert opinions and recommendations as are appropriate including from the National Centre for Pharmacoeconomics, NCPE.

This ensures that reimbursement decisions are made on objective scientific and economic grounds. The HSE has advised the Department that, following an intensive process, it has decided not to reimburse Vimizim, which is for the treatment of Morquio A syndrome. I appreciate that this condition is a serious one and that the families of children diagnosed with this illness are extremely anxious to obtain any treatment which they believe will alleviate its symptoms. The HSE drugs group, which reviewed the effectiveness of the drug, did not consider the evidence for its clinical benefit to be sufficiently strong in the context of the proposed cost and budget impact.

The applicant company was notified on 7 November 2017 of the HSE’s decision not to add Vimizim to its reimbursement list. The HSE advised the company that it would be open to reviewing any additional clinical evidence if this becomes available. The company submitted a new application for reimbursement in December 2017. This was discussed at the January 2018 drugs group meeting and will be considered further by the HSE leadership in the coming weeks.

Bobby Aylward (Carlow-Kilkenny, Fianna Fail)
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The HSE drugs group, in its decision not to reimburse Vimizim, has on both occasions stated that there is a lack of clinical evidence on the effectiveness of the drug. They have acknowledged that the budget impact to cover the cost was relatively low. The drugs group itself agreed that cost was not an issue. When we talk about drugs we worry mostly about the cost. The drug is available in most of the European Union, including Northern Ireland, our neighbours, and is actually manufactured in Ireland. The manufacturing company applied again to the HSE drug group with new evidence late last year after the drug was rejected at the September meeting. This evidence was largely from the managed access agreement that the company are running in the UK, which the UK accepted. If the clinical evidence has proven acceptable in other countries and the HSE has acknowledged that the cost is not the primary barrier to reimbursement, then the drug should be made available to those who need it.

In respect of the cost, there are figures in the public domain which indicate a very high costing for the use of the drug per patient. These figures are factually incorrect as we are dealing with a weight-based drug, which means that the publicly quoted amount is substantially higher than what the price per patient would be because they are small and the weight per patient means less cost. It is also worth bearing in mind that in the decision not to reimburse the drug the cost was not cited as a significant factor in the decision.

In respect of the managed access agreement, BioMarin has an arrangement in the UK with the National Institute for Health and Care Excellence, the NHS and the patient group where patients who suffer from Morquio A syndrome sign a contract which states they must attend all medical sessions and that, if after a year the medical records note that there has not been an improvement in their circumstances, they agree to come off the drug. In England, if the drug does not work, there is an agreement to stop using it if it is not having any effect. This has the benefit of ensuring that patients who do benefit from the drug can use it and that those who do not discontinue treatment, ultimately ensuring that the costs associated with the drug are solely associated with those patients who benefit from the drug. Clinicians who currently work in this space in Ireland have drawn up a similar guideline in Ireland and the patients are also willing to engage on this basis going forward.

In respect of the HSE drugs group challenges, the decision-making process is non-transparent which leaves the patients and the pharmaceutical company in the dark. The current nature of the drugs reimbursement process as a whole is unsustainable. There are problems associated with the quality adjusted life year based analysis for rare and orphan drugs.

There are two little girls looking down on the Minister of State here today. Next Friday, they will not have this drug, which has helped them over the past four years. Will the Minister of State please, on humanitarian grounds, bring this issue back to the task force and explain the situation? It will involve only a small cost to the taxpayers of this country.

Jim Daly (Cork South West, Fine Gael)
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I assure the Deputy that I take very seriously my responsibilities in the Department of Health. Indeed, the Deputy takes his responsibilities very seriously, which includes being a representative for his constituents, as indeed does Deputy Martin Kenny. The Deputy makes a very good case, and I certainly will convey the very well presented and well-felt feelings for the two girls, his constituents, who are affected by this.

As the Deputy and the House will appreciate, neither I nor the Minister for Health, Deputy Harris, can tell the Department or the HSE what drugs to okay and what drugs to reject. There is a process in place as a result of the Health (Pricing and Supply of Drugs) Act 2013 which was passed in this House. The criteria is now set in law. It is a strict criteria because we cannot have a situation where drug companies can charge the State, funded by the working and contributing classes of this country, any price they want. There is no magic pot out there. We have to have a structure and there has to be a system in place. While that system is in place, I appreciate that there is a lot of anxiety on the part of the families now. My Department has written to the HSE and asked it to ensure that the care of these two people is paramount in all considerations, and that they will be contacted and kept informed of any decisions and assisted in any way possible. I will redouble those efforts, on behalf of my Department, and convey that to the HSE today, and I can assure the Deputy of that on foot of his request. I cannot go any further and give any confirmation at this stage as to what drugs will or will not be reimbursed by the State.