Today is world Rare Disease Day, which is a day to raise awareness among the public and decision makers about the impact of rare diseases on people's lives. On 20 September last, more than six months ago, I raised the issue of spinal muscular atrophy, SMA, with the Tánaiste and the need for the HSE to improve the reimbursement for the drug, Spinraza. This has been raised on a weekly basis by many Deputies across the House since. As we stated, SMA is one of the most horrific diseases imaginable.

We had a meeting this morning, hosted by Deputy Ó Caoláin, in the audiovisual, AV, room where it would have broken a stone heart to hear the stories from the families and from those with the condition. SMA debilitates lives and until the arrival of Spinraza, there was nothing to tackle it effectively. Those living with SMA spoke this morning about the impact of Sprinraza, this miracle drug, and the new opportunities it presented to them. They laid out those testimonies in a painful and direct manner, but along with the pain, there was bravery, courage and a raw ambition to live lives to the fullest. There was an ambition on the part of those older patients with SMA and an ambition on the part of those parents who are seeing lives that they thought they would not see because before the arrival of Sprinraza, the average lifespan of somebody with SMA was two years.

The HSE does not seem to share that ambition or to understand that pain because, on Friday last, it told families again that it would not approve Spinraza for reimbursement and thereby took that chance away. It has sentenced sufferers and their families to continued suffering and to emigration to one of the 25 countries that have approved Spinraza medically and financially. It was laid out clearly to us this morning and I regret that this decision has potentially sentenced some of those sufferers to death because without Spinraza they will not survive SMA.

Twenty-five countries have approved Spinraza and can stand over the clinical effectiveness of the drug. Twenty-five countries have come to a deal with Biogen on the pricing of Spinraza, and yet the Government cannot. A number of Deputies have met Biogen representatives who have engaged with the HSE since September in a detailed manner. They have put a deal on the table, which is considerably less than the prices the HSE is quoting publicly. It strikes me that the HSE is deliberately misleading people about the potential cost of this drug in order to change the course of the debate.

I thank the Deputy. The time is up.

At the same time, the HSE is ignoring a potential €90 million in savings that have been pointed out to it by Deputy Brassil through the use of what are known as biosimilars.

Please, the time is up.

Why is the HSE using misinformation to add to the distress of families with SMA? Why will its staff not engage properly with Biogen regarding the deal that is on the table as opposed to a deal that they are making up? Can the Tánaiste ensure that the Minister for Health will meet directly with the families and the patients in the manner the Deputies did this morning to understand the consequences of this decision?

Before we proceed, I implore Members to adhere to the time limits provided for questions.

I thank Deputy Calleary for raising this sensitive and important issue, which has been raised repeatedly. When I have taken Leaders' Question, it has come up almost every second week for many months now.

SMA, as has been described, is a terrible condition with awful consequences for sufferers, and I am sure extraordinary stress for families as well. I completely understand the stress and frustration that each family must be under and the anxiety caused as they wait for news about funding of this drug.

The Government wants an agreement to be reached so that sufferers have access to the best medication. However, we cannot ignore recommendations from the HSE in ensuring that we spend appropriate amounts on many drugs for rare conditions. Two have been approved so far this year through the system that we are discussing.

The price list sought by the company, according to information that I have, amounts to €600,000 per patient for the first year and €380,000 per patient per annum thereafter.

That has changed.

Let me give the House the information that I have. I certainly stand open to questions.

Following detailed consideration of an application from the manufacturer, Biogen, the HSE leadership team made the decision that it was unable to recommend reimbursement of Spinraza at the price quoted on 12 February. On Thursday last, 21 February, the HSE wrote to the company informing it that the executive proposed to refuse reimbursement at the current price offering but had left the door open for the company to come back with a more realistic offering. Under the terms of the Health (Pricing and Supply of Medical Goods) Act 2013, as I am sure Deputy Calleary will be aware, the company has 28 days to come back to the HSE with a proposal.

The Government is keen to see agreement reached here. We want the HSE to work proactively with the company so that we can find a middle ground position that the company can accept in terms of pricing and that the HSE can recommend to the Government, which can allow this drug to be made available. That is where we are. The HSE has not recommended that there should not be reimbursement for Spinraza. It has made a recommendation that it cannot do it at the price on offer.

The time is up.

I encourage the company to engage and ensure that we find a way, for patients and families who are stressed and suffering because of the anxiety and the wait on this issue, to bring that wait to an end if it is possible to do so.

Those prices the Tánaiste is quoting are out by up to 50%. He has been given information-----

I have been given a note by the HSE.

-----but we have spoken to the company. I accept that the Tánaiste has been given incorrect information. That is why he needs to take charge of this as deputy prime minister. The families have no confidence in the HSE or in the management of the Department of Health to see this through. Those prices are way out and they cannot be used to stop families accessing a better future.

The Taoiseach, of course, tweeted this morning, in the lack of anything of substance, that today is international Rare Disease Day and, "Up to 300k people in Ireland will be affected by a rare disease in their lifetime. We will do better in helping these patients & families in Ireland". Let the Government do better. Let it do better for the parents of children and adults with SMA and give them access to something that will transform their lives. We want the Tánaiste and the Minister for Health to meet these parents and patients to understand how important this is and the transformative nature of this drug because the HSE clearly does not. I ask the Tánaiste to get the right information.

I will verify the information that I have. I have a date next to that reimbursement price that was proposed of 12 February, which I suspect may be accurate. Perhaps there has been a change since then in the price that is on offer. If there is, there is a structured mechanism for the company to engage.

Do it again.

I ask the Deputy not to trivialise this important issue. We want to get it resolved. There is an absolute willingness on the part of the Government to do that. However, when we are talking about facilitating drugs for rare conditions, as we have been done this year, if we ignore price as part of the discussion, we could pay money that could go into other drugs.

There is €90 million in savings on the table.

Of course we want the best drugs to be available for rare conditions and other conditions. They are going through the assessment process. Cost must be a part of that discussion. The onus is on the company to come back with a proposal that I certainly hope the HSE will be able to consider favourably.

Today, in the audiovisual room, Deputies, Senators and support staff listened, many with tears in their eyes - I was facing them - and unashamed of the fact. They listened to the most heart-rending appeals of parents, children and adult survivors all contending with spinal muscular atrophy, SMA, in their lives. The stories of their lives and the lives of the many others who joined them afterwards at the gates of Leinster House need to be heard by every Deputy and Senator without exception. Along with colleagues from all parties and independent voices, I have been consistently raising the issue of approval of Spinraza for all SMA sufferers. I ask the Tánaiste to note that those who have turned 18 years of age and who have lived beyond childhood are equally entitled to this chance of not only improved life but life itself. This is world Rare Disease Day. It should have been a day of celebration for the 25 children and the 50 people aged over 18 who live with the cruel denial of the ability to live full and active lives. It should have been a day of celebration for their heartbroken parents, their extended families and all who care for them. It would have been if the HSE had approved Spinraza for reimbursement when its senior leadership team met two weeks ago on 14 February, St. Valentine's Day, but it did not. Despite the fact that Spinraza has been approved in 25 of the 28 member states of the European Union, and that its efficacy and effectiveness have been clinically established and demonstrated in a significant number of the cases of people who are accessing the drug, Ireland, with Estonia and the United Kingdom, remains unmoved. Scotland has acted unilaterally within the UK and has approved Spinraza for types 1, 2 and 3. With the UK exiting the European Union, Ireland and Estonia will stand alone as the only EU countries not to face up to their responsibilities to all their children and to those who have survived into adult life.

I am sick and tired of appealing to the Government and the Minister for Health, Deputy Harris, in respect of this issue and so many others. When the Minister and the Department fail to set the policy and fail to instruct the HSE to act in a responsible way on a given issue - and in this instance where the lives of very ill children are at stake - it galls and angers me. Against the almost daily statements of his and the Government's commitment to children's healthcare services and the new national children's hospital, the State is continuing to fail a small cohort of 25 children suffering from a devastating wasting disease. Will the Tánaiste undertake to act now as deputy leader of the Government, as an accountable public elected representative and as a parent?

I assure the Deputy that I, as deputy head of Government and as a parent, try to act every day in terms of the responsibility I have. I do not think we should be trying to paint the HSE as some kind of cold body that does not want to grant reimbursement for Spinraza. It does want to grant it. The HSE rare diseases technology review committee recommended that it should be approved. As it moves through the process cost must be part of the discussion and because the HSE does not believe the company involved is offering value for money there is a stalling in the process. This is why the HSE has written to Biogen to this effect. I certainly hope that what was suggested earlier by Deputy Calleary, that the cost proposal has changed, can be a factor in the response the company makes to the HSE's letter.

Make no mistake, the Government wants this issue to be resolved. We want our children and the families who love them to get the support of the State in respect of rare diseases and the drugs that are very expensive but that are absolutely justifiable in those cases. We also have to ensure the State gets value for money because if it does not, we will spend money that could otherwise fund other really valuable drugs that are also needed and other families and victims will not get the support they need. In the context of the dealings the State has with the pharmaceutical sector and drugs companies, we have to ensure that there is a fair pricing model and that we have a process that does not allow children and their trauma and stories to be used as a way of trying to get prices agreed.

I will speak to the Minister for Health and the Taoiseach on this directly but it is important that politicians are not the ones who make decisions on what is or is not value for money in terms of healthcare and that we have a system we can rely on to do this. Again, I encourage the company and the HSE to try to resolve the issue because I know how many people in the House understandably continue to take a very personal interest in it.

I hope that what the Tánaiste is saying is the case in terms of the Government's keenness in wanting to see this resolved. I charge that those who are representing the Government and us in terms of the engagement with Biogen are not demonstrating the same commitment. Make no mistake about it, the figures that Deputy Calleary has shared with the House are accurate. I have also met Biogen and I can state that an imaginative proposal that would leave the cost for those aged under 18 alone - just dealing with children - at approximately €9 million for two year's provision has been put forward. Figures have been bandied about and put out on the airwaves. The Government must take direct responsibility on this engagement because trusting and not exploring the full facts makes it complicit in the failure. If the Tánaiste was at the gates of the House but an hour ago to speak to those families and meet those children, he would not give the answers he is offering. He would instead make this matter his number one priority. I ask him to commit to doing exactly this from this moment forward.

This is a priority, just like it was a priority to get other rare disease drugs approved to date in 2019. There are families awaiting and needing decisions on reimbursement and the absence of those decisions has been going on for too long. In the case of Spinraza, it has been going on for too many months. We also have to ensure that we have in place a system which makes decisions on the basis of the facts in order to ensure that we get the drugs we need for the families and, in particular, the children who need them. We must also ensure we spend money in a way that guarantees we get value for all the money we spend throughout the healthcare expenditure profile, for rare diseases and other diseases. This is where we are at on this. We want this issue resolved for the families concerned. There needs to be a response from Biogen to the effect that persuades the HSE leadership that its proposal represents value for money and is comparable with the price of the drug in other countries. I will certainly speak to the Minister for Health about this issue. It is the responsibility of the Government to ensure that the figures are accurate but we must also ensure that experts make decisions in technical areas such as this. This is why we have in place the existing structure and systems.