Batten Disease: Access to Drugs

Andrew Griffiths Excerpts

Tuesday 16th July 2019 Commons Chamber

[Mr Jacob Rees-Mogg (North East Somerset) (Con)]

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.

This drug Brineura has been given a quality-adjusted life years rating by the National Institute for Health and Care Excellence of 30, which is the highest rating that it gives. That means that the drug is thought to provide 30 extra years of life of good quality. That is a stunning achievement for any drug, and it has been given the highest rating and the highest amount of funding, but unfortunately that amount of funding is not enough. The pricing cannot be agreed between NHS England, NICE and BioMarin, the manufacturer.

BioMarin is a drug company that needs to make a return on the amount of money it has spent. To be fair to the company, it spent $696 million last year on research and development and made a pre-tax loss of $142 million, so it is not an enormously profitable, rapacious company that is being difficult. One might think, however, that having lost $142 million, it might quite welcome a little bit of income from the national health service. If I were one of its shareholders, I might suggest that it would be a good idea to do something with the national health service so that the company could get some income back on its $696 million of research and development expenditure in 2018. Without an agreement between the buyers and sellers, Max will not receive the drug and his standard of life will decline month by month.

[Andrew Griffiths (Burton) (Con)]

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?

[Mr Rees-Mogg]

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?

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[The Minister for Care (Caroline Dinenage)]

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—

[Andrew Griffiths]

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?

[Mr Speaker]

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.

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[Caroline Dinenage]

I am very tempted to do so, but I would like to try to make progress on some of the things I can say, and then we can maybe move on to a more satisfactory conclusion.

[Andrew Griffiths]

On a point of order, Mr Speaker.

[Mr Speaker]

Of course the Minister should have the opportunity to do so, but I think it is only right that I hear the point of order, if it is a genuine point of order.

[Andrew Griffiths]

Could you explain the rules to me, Mr Speaker? If it is proved that today’s Adjournment debate is not sub judice and that the information could have been proffered to the House in a way that would have satisfied both my hon. Friend the Member for North East Somerset (Mr Rees-Mogg) and you, would it be in order for the responsible Minister to come to this place to make a statement before the recess, so that those valuable days are not lost? Do you think it would be sensible for the Minister to make that commitment now?

[Mr Speaker]

It is certainly open to the Minister to do so, and I think that it would, without question, be to the advantage of the House if such an oral statement were proffered before the House goes into recess. What I should say to the hon. Gentleman and to other colleagues is that, to my knowledge—forgive me, but I am dealing with this matter literally on my feet—the case has not been set down; it is not listed for court consideration. There are no current proceedings dated. Therefore, as we understand it—my advisers and I—it cannot be said to be sub judice. Sometimes a judgment might be made about the possibility of prejudicing a case—that is a wider consideration–but the real issue is: is it sub judice? Our answer, on the best information available, is that it is not, and so I think a statement would be helpful. Alternatively, if Members wish to exercise their ingenuity in raising important and of course legitimate points of order, and the Minister wishes in the meantime to consult her officials so that a full and detailed response on the specifics can be provided, that is open to her. I recognise that that might be challenging at such short notice and she may not wish to do that, but it would certainly be helpful to have a substantive response, preferably this evening, and a full oral statement before we go into recess. I would say to the House that I am giving the ruling that I am giving both on the basis of a decade’s experience as the occupant of the Chair and on the strength of top-quality procedural advice.

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[Caroline Dinenage]

That is a really good question, and I think that is one reason why NICE is looking again at its systems. It must be possible, because around 80% of new drugs are recommended for NICE approval and approved by NICE. It is clearly possible for new drugs to make it through even when they have not been around for a long enough period to see the very long-term consequences of their impact. It is really important in cases such as this, in which the opportunity—

[Andrew Griffiths]

On a point of order, Mr Speaker. I seek your advice, because we have a precedent for a situation that can arise on numerous occasions. In the nine years I have been in the House, I have heard of many debates in which right hon. and hon. Members have brought forward demands for treatments for rare diseases. We call the Minister to the Dispatch Box to answer—to be accountable. If we have a situation, going forward, where the Minister can claim sub judice, on advice from officials—it is not the Minister; she is taking advice from her Department—and be allowed to hide behind that cloak of anonymity and prevent the House from doing its job, which is to establish the facts and find out the truth behind the matter, that clearly prevents the House from being able to do its job.

You would share my concern, Mr Speaker, that that would prevent Members in this House from serving their constituents in a way that they would be expected to do. Therefore, can you ensure that you will go away today and look at this situation and make sure that, if this procedure has gone wrong, Departments are told very clearly that they cannot hide behind sub judice and that they must be candid with this House, regardless of the advice they have been given by officials.

[Mr Speaker]

That is a most helpful point of order from the hon. Gentleman, to which, essentially, the answer is a simple yes. I will happily go away and reflect on how Departments might usefully be advised to proceed in these matters. Beyond that, there are two points. First, as I have been advised, the matter is not sub judice. It has been suggested to me by a very constructive member of the Treasury Bench that advice to the effect that the case might be sub judice was proffered, though not to the particular Clerk whom I have been consulting, but that advice was not judged to be correct. Put simply, as far as we can see, the matter is not sub judice.

Secondly, however, and this is an extremely important point in the context of the specific debate that the hon. Member for North East Somerset has secured, the issue is that of ministerial intervention, and therefore whether or not the case is sub judice—I repeat that I am advised that it is not—the matter of ministerial intervention is manifestly one for Ministers, and it is therefore a matter upon which a Minister from the Bench can comment and would widely be expected to do so. Frankly, this question of the legal status is not germane to the capacity of a Minister to respond to the specifics of the debate. That is the situation.

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[Caroline Dinenage]

NICE now operates two separate programmes for the assessment of new medicines: first, a technology appraisal programme through which NICE assesses the vast majority of new medicines; and secondly, a highly specialised technologies programme that is reserved for the evaluation of very high cost drugs for the treatment of very small numbers of patients suffering from very rare diseases in England who are treated in a handful of centres in the NHS.

Where NICE recommends a treatment for use on the NHS, NHS commissioners are legally required to make funding available so that it can be prescribed to patients. This is reflected in the NHS constitution as a right to NICE-approved treatments. The intention of NICE is to have a system that means that the public can have confidence that the price paid by the NHS is consistent with the improvement in health outcomes that the medicine brings, ensuring fairness and the best possible use of funding for patients and the NHS.

As I said to the hon. Member for Newcastle upon Tyne North (Catherine McKinnell), NICE has recommended around 80% of products it has assessed. Through its important work, many thousands of patients, including patients with rare diseases, have benefited from access to effective new treatments.

It is right that NICE’s processes continue to evolve with developments in science, healthcare and the life sciences sector. That is why it keeps its methods and processes updated through periodic review that includes extensive engagement with stakeholders, including patient representatives, drug manufacturers and clinicians. In this spirit of continuous development, through the 2019 voluntary scheme for branded medicines pricing and access, which was published in December, the Government announced that NICE would be undertaking a review of its methods and processes in 2019-20 for both its technology appraisal and the highly specialised technologies programme.

[Andrew Griffiths]

I thank the Minister for the honourable way in which she has played a straight bat in what is a very sticky wicket; I am grateful to her for that. She mentioned that she was prepared to come back and make a statement to the House, for which I think we are all grateful. Would she just confirm whether she intends to make that statement before the House rises for the summer recess?

[Caroline Dinenage]

Yes, I will come back and make that statement before the House rises for the summer recess, with the correct legal recommendations.

The NICE review of its methods and processes has now begun. That will be quite helpful for some hon. Members who are questioning whether those processes are as they should be. Although I do not think it would not be appropriate for me to pre-empt the review by commenting in detail on its scope, I can assure hon. Members that it will be wide-ranging and will be carried out with full consultation with stakeholders.

My hon. Friend the Member for North East Somerset spoke about access to the new drug Brineura for his constituent Max. NICE is currently developing recommendations for the NHS on the use of Brineura through its highly specialised technologies programme for the evaluation of drugs for the treatment of patients with very rare diseases. It has not yet published that final guidance. I understand that NHS England and NHS Improvement have been in discussions with the manufacturer of Brineura, BioMarin, for some time to seek a deal that would enable NICE to recommend the drug for use on the NHS, but an agreement has unfortunately not been possible so far. As I said at the beginning, NICE’s assessment of Brineura is potentially going to be subject to legal proceedings.

Let me move on to individual funding requests. Where a treatment is not routinely available on the NHS, including where NICE has not yet made a recommendation on its use, NHS clinicians are able to apply for funding through an individual funding request. This can be done when a clinician believes that their patient is clearly different from other patients with the same condition, or where their patient might benefit from the treatment in a different way from other patients.

In the absence of NICE’s final recommendations on Brineura, I am aware that NHS England and NHS Improvement have recently considered an individual funding request for my hon. Friend’s constituent Max that it has unfortunately been unable to support, because he would not be regarded as an exceptional case to existing policy. I understand how devastating that decision will be for Max, his parents and all those involved. However, if my hon. Friend’s constituents and their clinicians think that the process has not been followed correctly, they can ask for a review of the decision within 28 days. If a patient’s situation changes or more clinical evidence becomes available about the effectiveness of a treatment, they may also submit additional information to be considered and discussed.

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[Caroline Dinenage]

My understanding is that if the manufacturer of this vital drug gets around the table with NICE and suggests a price within a reasonable scale, NICE would be very open to listening. That is my understanding. Certainly, I understand calls from Members across the House for NHS England, NICE and the manufacturer to get around the table to try to find a solution. That seems to me to be the most sensible way forward.

[Andrew Griffiths]

I thank the Minister for giving way again. The language she is using is the same as that we hear in a lot of these debates. She has said that many of these families and young children are receiving the drug on compassionate grounds from the drug company. For a parent whose child is not getting that drug to be told that somebody else’s son or daughter is getting compassion but theirs is not—the Minister is a mother herself, so she will understand this—is like a dagger through the heart. Will she do all she can to ensure that that compassion is extended to all those mums and dads who are looking on tonight?

[Caroline Dinenage]

Of course I will commit to doing everything I can to making sure that all the very valid and easy-to-understand sentiments that have been expressed by Members across the House are sent to all the parties involved in this discussion. I completely share Members’ desire to find a resolution to this and ensure that families get the answers they need. In the meantime, I thank all Members for the part they have played in today’s debate, the questions they have asked and the great interest they have shown in this issue. As I say, I will come back to the House shortly with fuller answers to some of those questions.