Batten Disease: Access to Drugs

Motion made, and Question proposed, That this House do now adjourn.—(Mike Freer.)

Mr Speaker, may I begin by thanking you for allowing me this Adjournment debate and for your personal encouragement to me to bring it forward? My gratitude goes beyond that; I also thank you for the way you so encourage this House to hold the Government—the Executive: those who rule us—to account.

I am also grateful to the hon. Member for Newcastle upon Tyne North (Catherine McKinnell) and the terrific campaign she has been running in relation to Batten disease; she has done more than almost anybody else to highlight it. I note that two of my hon. Friends on the Front Bench, my hon. Friends the Members for Macclesfield (David Rutley) and for Pudsey (Stuart Andrew), have a great interest and a constituency concern in this issue, although they are not allowed to intervene on me for obvious reasons. I want to put their concern on the record. I know that others too might wish to intervene on this very important subject.

This evening’s debate is about my constituent, Max. Max is a little boy; he is eight years old. He is a lively boy, and those of us who have children know what eight-year-olds are like—what a joy they are and how wonderful their spirit is. But Max has this horrible disease. Batten disease is perhaps the cruellest disease that one can imagine as a parent. We all see our children grow; we see them learn to walk and then to talk, and to run and to do all the things that children do. Batten disease means that they then go backwards. It tends to hit at about two—on a child who has shown no signs until then. The talking stops and the walking becomes more difficult. The average life expectancy of a child with Batten disease is between six and 12.

The blow to parents, and to grandparents and families, that this is and must be, is so hard to bear and so difficult; it is so sad for them to see a child who they would hope would be going on into adulthood instead declining, and declining steadily. It is a neurodegenerative disease. To put it in layman’s terms, it is essentially dementia of the young: all that we see of Alzheimer’s in people in their 70s, 80s and 90s is instead happening to a child.

But there is a drug that delays this. It is not a cure and it does not reverse the disease, but it seems to stop its progression—nobody knows for how long. It is called Brineura and it has been shown to be effective on a number of children who have taken it. So far in this country there are only two children for whom it would be suitable who are not receiving it, one of whom is Max. The others are receiving it as part of a trial that has been successful and is still being funded by the drug company—but that might not continue for ever, so there is an argument for them as well. I mentioned earlier the enormous contribution that the hon. Member for Newcastle upon Tyne North has made, and I am very glad to see her in her place; without her, I do not think this really important matter would have achieved the publicity that it has received.

My hon. Friend will know about my constituent, Michal Luc, who is in exactly the same situation. We talk to parents who see their children degenerating and dying before their eyes. Does he agree that they cannot understand how we can argue over money when their children’s lives are disappearing before their very eyes?

I completely agree with my hon. Friend.

Generally, I recognise the need for public expenditure constraint. Money always has to come from somewhere; it has to be either taxed or borrowed. However, in a country that spends over £800 billion a year, and £120 billion or whatever it is a year on the national health service, can we not find just over £6 million a year for this small number of children who have a terrible disease that can be held at bay?

I very much commend the hon. Gentleman for securing this hugely important and timely debate. Does he share my concern that we seem to be witnessing a bit of a blame game between NHS England, NICE and BioMarin? Does he agree that they just need to get round the table and resolve this issue one way or another, even if it requires the Minister to bring them together and knock some heads together to get them to come to a resolution? The rapid-acting nature of Batten disease means that my constituents Nicole and Jessica Rich and the other children who are affected just do not have the time for this wrangling to carry on.

The hon. Lady is absolutely right. The terrible thing is that in the month that has passed since I first raised this matter in the House, Max’s condition will have slightly deteriorated, and in every month that goes on while we are debating this, not just Max but all the children with this condition will go downhill. That is what happens with this disease.

There are questions to be asked about the structure of policy on rare diseases, and about the Government’s response and what powers they have. As I said at the beginning, accountability through this House is of fundamental importance. By law, the Secretary of State still maintains overall responsibility for the provision of healthcare in this country. It is the Secretary of State who is accountable. We cannot make NICE accountable; it has not been structured to be accountable. It does not appear in the Chamber to tell us what it is doing—that is done by second degree, through Ministers. We really need to know what, if any, reserve powers Ministers may have to be able to do something about the situation.

Can something be done? Can a budget exception be provided, so that funds may be made available for these rare diseases? Can something be done, as has been done for cancer treatments, to provide money where exceptionality can be seen? Of course these drugs are expensive: they affect so few people, and the drugs companies will not develop them if they cannot at least make their money back. Can something be done as in other areas, particularly cancer, to ensure that the drugs can be provided? Can the rule changes in 2017 that made it harder to fund rare disease drugs be reviewed and possibly reversed? Since 2017, the financial aspect has become much more significant than it was before.

Although I accept, of course, that there is a need to look at costs, when we are talking about eight-year-old children, we are not talking about a cost for people who only have weeks or months to live, but about a child who could have years of a high quality of life ahead of him. That must be where most of us as taxpayers think it is right to spend money and where we think that the moral case for spending money is extraordinarily strong.

Does the hon. Gentleman share my concern that the impact of rare conditions such as Batten disease is not just felt in the child’s physical health, but in their mental health and the mental health of their wider family? The system for judging what is value for money and how our NHS should spend its money needs to take a much broader approach when calculating the value of these medicines in those circumstances. It needs to get it right.

The hon. Lady puts it so well—and it is not just the family, but the community. On Saturday, the village of East Harptree, a small village in North East Somerset, came together for its annual village fête. All the funds raised were to try and help Max. He is at the local primary school, East Harptree Primary School. The week before, they had the school races. All the children had gone back a few yards so that Max could win, for the first and only time in his life, the race at his school. That is such a wonderful example of community. If communities can do that, surely the Government can help too, because it is not just Max and not just his wonderful family who are trying so hard to do the right thing for him. A whole community would be pleased, and would feel it was being taken notice of, if Max were helped—all his schoolfriends and schoolteachers and the whole community in East Harptree.

The hon. Gentleman gives a really powerful example. The whole of the community in Newcastle knows about Nicole and Jessica Rich and is doing everything it can to support them in this journey. This not only affects those two beautiful children; it also affects their family in a huge way, and the whole community.

For that reason, I beg the Minister today to recognise that this is not only about reaching the right decision, but about doing it with urgency. Every day, there is an impact on their deteriorating health, and there is also the impact on the parents of supporting those children with a debilitating condition and living with the agony of not knowing what future lies ahead—whether the medicine that will save their children’s lives will be funded or not.

I so agree with the hon. Lady. I am of course primarily talking about Max, my constituent, but to take the drug away from children who are already getting it would be unconscionable. I simply do not believe that any reasonable person—any politician or any administrator—would think that the right thing to do. It is bad enough not to give the drug to a child who could benefit; to withdraw it would be so utterly wrong that I cannot believe that that could happen.

When something can be done, it is hard for it not to be done and for us to allow it not to be done. It is frustrating that it is so hard to change and that there seems to be nobody who can decide it. Everyone one talks to says it is not up to them. NICE is bound by its guidelines, NHS England is bound by NICE, and the Secretary of State is bound by the legal interpretation of what the Health and Social Care Act 2012 provides, but none of that is good enough. We need action. Ultimately, it is Ministers, through Parliament, who are able to act.

Let me finish with what Max’s father, Simon Sewart, who has been doing so much to look after his son, wrote:

“I have always understood that life is no fairy tale with a happy ending, but when you learn that your beautiful child has a disease, as horrific as Batten Disease, your world changes forever and your heart is broken.

NICE announced, just 24 hours after Max’s diagnosis, that the first ever treatment for CLN2 Batten Disease will not be funded.

At a time when you should be taking care of your child, your other children, and enjoying precious time together as a family, you instead find yourself spending all of your time writing emails and letters, speaking to journalists and TV news programmes, communicating with your MP and with doctors in other countries where the ERT is available.

Expending all of your energy in fighting the extraordinary decision by NICE and NHSE. And all the time, you see your child decline, day by day. And all the time, you just want to expend your energy on them, on holding them, on playing with them, on laughing and smiling with them, on running with them, on walking with them, on talking with them, on looking around at the world with them; on all these things. With them.

I congratulate my hon. Friend the Member for North East Somerset (Mr Rees-Mogg) on securing this important debate on what is a heartbreaking issue, as I am sure you will agree, Mr Speaker. He and other Members have spoken up movingly and with great passion about and on behalf of their constituents, and I am grateful to them for doing so. I understand how vital it is for patients and their families to be able to access new medicines as quickly as possible. This is one of the hardest types of debates that one has to respond to as a Minister, when we can put ourselves in the position of the families up and down the country who are facing such a desperate situation.

I will endeavour to respond as fully as I can to the issues that my hon. Friend and other Members raised, but I should begin by saying, sadly, that I am unfortunately unable to comment on matters relating to the availability of Brineura, a drug used to treat Batten disease, as this is currently subject to an active judicial review procedure.

As we have heard from hon. Members, Batten disease is a terrible condition that progresses rapidly, leading to loss of speech, mobility and vision, progressive dementia and early death. It is a rare genetic disease, and it is estimated that around three to six children in the UK are diagnosed each year, with around 30 to 50 children living with the condition. Current treatment options are limited to symptomatic relief and supportive care. I fully understand how vital any new treatment option could be to the families of children with this rare and devastating condition.

The Government want patients, including those with rare diseases such as Batten disease, to be able to benefit from effective new treatments. It is in the interests of all NHS patients that we have a system in place for making evidence-based—

On a point of order, Mr Speaker. This important debate is about whether this drug is going to be available for these young children, and you will share my concern that we are not going to get the answers we want because it is supposedly prevented by a judicial review. Can you rule on whether we should be able to have the information we seek in this debate for the parents who are watching?

I am extremely grateful to the hon. Gentleman for his point of order. He anticipates me, because his discernment is such that he will have observed me in conversation with the Clerk at the Table. Let me say, for the avoidance of doubt, that the content of the Minister’s reply is, of course, a matter for her, and it is not something with which I would seek to interfere. That is not for me.

However, for the purposes of clarification, I want to make this point. I am not aware that this matter is sub judice, as I have not received prior notification that it is. I am not aware that it is, I have not been informed that it is, and the Clerks have not been informed that it is. If it is not sub judice, nothing whatsoever precludes the Minister from commenting on this case. If it is sub judice, as colleagues will know, it is within the competence —I use that term in the technical sense—of the Chair to waive the sub judice rule, which it would most certainly be my instinct to do.

What the Minister says is a matter for the Minister, but it would not be right, as far as I can tell, to say that it is not possible, in a legal sense, for the Minister to comment on this matter. The Minister is the Minister, and the Minister’s answer on the specifics is sought. If the Minister wishes to proffer that answer, she can do so.

Thank you, Mr Speaker. You have made my position even more difficult. I am very grateful for your advice.

The advice I have been given by the legal team within the Department of Health and Social Care is that anything I say could potentially influence the outcome of judicial proceedings that may be ongoing.

The Minister is nothing but solicitous and courteous. I have found her unfailingly courteous in her dealings with me over many years, and I certainly do not seek to make life difficult for her, but my concern is with the rights of Members of this House and of the people they are here to represent.

Frankly, if lawyers within the Department of Health and Social Care are of that view, it would have been courteous to consult the officials of the House. As far as I am aware, the officials of the House have not been consulted, and I have not been consulted about this matter. I am simply saying that if the Minister wishes to respond to the specifics, which manifestly the hon. Member for North East Somerset (Mr Rees-Mogg) was going to raise—that is the rationale for the debate—it is perfectly open to her to do so.

If the hon. Member for North East Somerset is not able to secure satisfaction on this occasion—the Minister must judge what she wants to say in response—and if he wishes to pursue the matter further in another Adjournment debate, during the course of waiting for which further time will unfortunately have been lost, that debate will be available to him. I am determined that this matter will be effectively raised in the Chamber of the House of Commons by the hon. Gentleman and by other Members.

Thank you for clarifying that, Mr Speaker. I very much share the passion that I detect my hon. Friend the Member for North East Somerset feels about this issue, which I know is shared by Members on both sides of the House.

On the basis of what Mr Speaker has said, surely if those who are taking this issue to the courts withdrew their challenge, or if the Minister gave them a satisfactory answer to help the hon. Member for North East Somerset (Mr Rees-Mogg), it would not be sub judice. The Minister has the power to do that.