Diarmuid Wilson (Fianna Fail)
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I would like to share time with Senator Rose Conway-Walsh.

Paul Coghlan (Fine Gael)
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Is that agreed? Agreed.

Diarmuid Wilson (Fianna Fail)
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I welcome the Minister of State, Deputy Catherine Byrne, and thank her for attending this debate on the issue of the drug Translarna, the medication for Duchenne muscular dystrophy, a degenerative condition which affects five young boys in this country. While this issue has been raised and discussed numerous times in the Lower House and this House, including last Tuesday by Senator Rose Conway-Walsh and also by Deputy James Browne, who held a briefing in the AV room, the lack of action is very disappointing.

The disease's progressive nature requires urgent action and the HSE's refusal to fund the drug has only exacerbated the situation. The manufacturer of Translarna, PTC Therapeutics, will soon make a new application to the National Centre for Pharmacoeconomics. I implore the Minister to treat this application with priority, given the time-critical nature of the condition of these boys. This drug would improve both the quality of day to day life of the boys and their life expectancy and productivity. Having spoken to some of the families on Tuesday of this week, their determination to fight for a better life for their sons shone through in a profound way. Facing up to the injustice not only of their children's condition, but also to the denial of this treatment, requires serious strength. I can tell the Minister of State that while they may be small in number, they have the strength to fight and they will do so, with our assistance on this side of the House.

I remind the Minister of State that the responsibility for these boys' lives lies with the Government. It is not just her job or the job of the Minister, Deputy Simon Harris, but their duty to provide them with the best possible care, given their incredibly tough situation. Will the Minister ensure the boys and their families are not left waiting another two years for a definitive decision? On 19 June, the Minister, Deputy Harris, said there would be no delay in the processing of this application. I sincerely hope he ensures this is the case. These boys and their families do not have two years. Translarna is available in 22 European countries to 400 children just like these boys. I hope the Minister of State is willing to do what she can to ensure they are afforded the same chance of life as their peers in the UK and on the Continent.

Rose Conway Walsh (Sinn Fein)
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I thank Senator Wilson for sharing time. He knows how passionate I am about the subject of Translarna and the boys who are waiting for it. I thank the Minister of State for coming to the House.

The Minister, Deputy Harris, is acutely aware of this subject and also of what needs to be done. I welcome the fact PTC Therapeutics and the HSE have come to an agreement and that the legal case is now taken off the table and will be discontinued in the High Court. That presents us with a small window of opportunity in which a revised application can be submitted. What is key is that we do not start the process all over again, that the information and clinical evidence that has already been submitted is taken into account and that we do not have to go through the two to three year process that applied originally. What needs to be outlined at this stage is the exact process and the timeline for that. In fairness, that needs to be given to PTC Therapeutics and to be made transparent. I ask the Minister of State for that information.

In terms of the price negotiation, the Minister, Deputy Harris, will also know the HSE failed to negotiate the price on the original application. That is why it was bewildering to get one's head around how a cost-benefit analysis could be done without having the proper negotiated price. That needs to be done at an early stage.

I know this can be settled in a matter of weeks or a couple of months. By the time we come back in September, these boys should be able to get the Translarna drug and not have to wait any longer. As Senator Wilson outlined, the impact of them not having it, in terms of their walking being discontinued, is not acceptable. We have this window of opportunity but the Minister needs to intervene because the HSE will not do it in a timely manner unless the Minister gets involved. I ask him to ensure the meeting between PTC and the HSE is facilitated as quickly as possible.

Catherine Byrne (Dublin South Central, Fine Gael)
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On behalf of the Minister, Deputy Simon Harris, I thank the Senators for raising the matter. Ataluren, also known by the brand name Translarna, is indicated for the treatment of Duchenne muscular dystrophy, DMD, a genetic disorder characterised by progressive muscle degeneration starting in early childhood. I understand that access to potentially beneficial drug treatments is an important issue for people with DMD. The Minister, Deputy Harris, appreciates that DMD sufferers and their families face enormous day-to-day challenges in dealing with this condition.

The Health (Pricing and Supply of Medical Goods) Act 2013 gives full statutory powers to the HSE to assess and decide on the reimbursement of medicines, taking account of a range of objective factors and expert opinion, as appropriate. The Act specifies criteria for reimbursement decisions. These include the clinical and cost effectiveness of the product, the opportunity cost and the impact on resources that are available to the HSE.

Ataluren is a high cost medicine manufactured by PTC Therapeutics for the treatment of a subgroup of patients with DMD, a rare disease. The medicine has conditional market authorisation for Europe from the European Medicines Agency, the condition being that the company carries out further clinical trials to determine the clinical efficacy of the drug. Applications for reimbursement were considered by the HSE drugs committee and leadership team in 2016 and 2017. The applications were considered carefully. However, the final decision was that the HSE was not in a position to fund the medicine on the basis of the current clinical evidence of effectiveness and the prices being charged by the pharmaceutical company. The HSE informed the applicant company of this decision, in keeping with the requirements of the 2013 Act. The applicant appealed the HSE's decision to the High Court, which is part of the statutory process under section 27 of the 2013 Act.

On 6 July 2018, the Department was informed by the HSE that a settlement of the High Court action had been reached. The terms of settlement stipulate that in the event that the manufacturer submits a new application to add ataluren to the reimbursement list, it would be reviewed by the HSE in a timely fashion having regard to the relevant statutory framework.

The Minister for Health is very much aware that this protracted process is upsetting to the patients and their families who are affected by this condition. As of yesterday, 11 July, the manufacturer has not submitted an application for reimbursement of Translarna. I call on the company to come to back to the table with a fair offer so that patients may access this treatment as soon as possible.

Paul Coghlan (Fine Gael)
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I call Senator Wilson.

Diarmuid Wilson (Fianna Fail)
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I give way to my colleague, Senator Conway-Walsh.

Paul Coghlan (Fine Gael)
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I advise Senator Conway-Walsh that she has one minute.

Rose Conway Walsh (Sinn Fein)
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I thank the Leas-Chathaoirleach and Senator Wilson. I also thank the Minister of State for her response.

The application by PTC will be submitted by the end of the month. It would be helpful and important to outline the process and timeline. We have to remember these boys. Both Senator Wilson and I know these families so they have become human beings and not just numbers to us. One of the families affected is from County Cavan and they could get the drug if they travelled five miles up the road. I know the other family as they are from County Mayo. I do not want them put in a position where they must uproot their family and move to the North in order to get the drug.

As has been said, the European Medicines Agency has granted approval. The evidence is there. Even if more evidence needs to be gathered, a conditional approval would be fine so that the boys can have the drug. It is important the process is completed as quickly as possible. We do not want to call the Minister of State or the Minister for Health to the House again. We believe that this is a pretty straightforward case. I thank everyone involved but above all I thank the families for bearing with this matter and remaining in the State-----

Diarmuid Wilson (Fianna Fail)
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Hear, hear.

Rose Conway Walsh (Sinn Fein)
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-----in the hope that Translarna will be granted to them.

Catherine Byrne (Dublin South Central, Fine Gael)
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I assure Senators Wilson and Conway-Walsh that the Minister for Health will do everything possible to ensure that access is approved as soon as possible for this life-changing drug to be given to the families, and particularly the children. We all know how important it is to keep our children well. Nobody likes to see children suffer. I will urge the Minister when I see him that everything is done as soon as possible to sort out the complications connected with the pricing and everything else. I will make sure to do that when I return to the other House.

Paul Coghlan (Fine Gael)
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I thank the Minister of State and the Senators.

Rose Conway Walsh (Sinn Fein)
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I thank the Minister of State.