Batten Disease: Access to Drugs

Caroline Dinenage Excerpts

Tuesday 16th July 2019 Commons Chamber

[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.

[Caroline Dinenage]

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.

[Mr Speaker]

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.

[Caroline Dinenage]

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.

[Mr Jim Cunningham (Coventry South) (Lab)]

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.

[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.

--- Later in debate ---

[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.

[Caroline Dinenage]

Thank you, Mr Speaker. I am very grateful—

[Mr Rees-Mogg]

On a point of order, Mr Speaker. My point of order follows on from what you said and I hope it may be helpful to the Minister in giving her a moment to reflect on what you have just said. I would express a broader concern on this sub judice question, because, as you rightly said, it is always the prerogative of this House to discuss what it wishes to discuss, regardless of what is going on in any court. The sub judice rule has only ever been a self-denying ordinance of this House, to feel that it should not trample on things that were immediately before another court, rather than something that can be enforced upon us. In that respect, we are different from any other place in the country—other than of course their lordships’ House—which would be subject to sub judice as a matter of law. We are subject to it only in so far as we feel that it is wise to be cautious and prudent, and not to interrupt another of the separated powers within our judicial system. Therefore, it seems to me that whenever Ministries decide to cite sub judice rules, it would be wise to have consulted Mr Speaker or one of the Clerks in advance, in order to have some clarity before the debate begins.

[Mr Speaker]

Yes, it is a matter of prudence on the part of the House of Commons; it is not a cloak behind which the Executive branch should seek to hide. I know that the Minister would not attempt to do so, but any advice that might be thought to be intended to allow that to happen would be ill viewed by right hon. and hon. Members, and most certainly by their constituents.

[Caroline Dinenage]

I take on board all the comments that you have made, Mr Speaker, and indeed that hon. Members have made. I need to go away and reflect on this. I would like to make some progress with what I feel confident to be able to talk about today, and then, if inspiration does not approach me from anywhere else in this Chamber in the next few moments, I will commit to coming back with a fuller response if that is possible before we break for recess, if that would be acceptable to you, Mr Speaker.

[Catherine McKinnell]

I thank you for your guidance on this issue, Mr Speaker. I wonder whether the Minister would accept that the legal action that is being discussed would be unnecessary if NHS England, the National Institute for Health and Care Excellence and BioMarin got around the table and resolved this issue. On the broader point, on 21 May I and a number of colleagues, including the hon. Member for North East Somerset, met the Secretary of State to discuss this issue. He has since written to me to say that he is

“doing all he can within the legal constraints to resolve this”

situation. I was therefore hoping that the Minister could clarify what exactly that means, and what progress is being made as a result of that meeting and the representations we have made today.

[Caroline Dinenage]

I know that the Secretary of State met the hon. Lady and other Members recently, and this is something that he cares about passionately. We are doing everything we can in the Department of Health and Social Care to try to move the position forward. The hon. Lady is right to say that had this issue been satisfactorily resolved, we would not be having this discussion at all, we would not be talking about legal proceedings and the situation would hopefully be a lot easier to resolve.

I have only a short amount of time left to be able to give some sort of response to my hon. Friend the Member for North East Somerset.

[Mr Rees-Mogg]

We have 40 minutes left, Mr Speaker, so we are not short of time.

[Mr Speaker]

No, we have plenty of time. We can continue until 7.30. [Interruption.] Oh yes, we have plenty of time. And of course, there will be an opportunity for either a ministerial statement or an urgent question between now and Thursday, so we have, if I may say so, bucket-loads of time to deliberate on this important matter. I know that that message will be extremely well received by the Minister.

[Caroline Dinenage]

Thank you, Mr Speaker.

The legal position, as I understand it, is that NHS England and NHS Improvement have been in discussions with the manufacturer BioMarin for some time to try to seek a deal that would enable NICE to recommend the drug for use on the NHS, but so far an agreement has unfortunately not been possible. As hon. Members know, and as I said at the beginning of my speech, NICE’s assessment is currently subject to an ongoing legal procedure. NICE has published its draft recommendation and was unfortunately unable to recommend the use of the drug, despite its meeting the criteria for consideration under the highest cost-effectiveness threshold. NICE has not yet re-published its final word on this issue, and that may provide more clarity. In the meantime, it is of course open to the company to enter into an agreement with NHS England that would make the drug cost-effective and would make the legal procedure unnecessary.

[Mr Jim Cunningham]

If NHS England got a satisfactory agreement with the drug manufacturer, would that have any bearing on the decision of the Secretary of State or on NICE?

[Caroline Dinenage]

I do not think it would contravene any legal recommendations if I were to say that if the manufacturer, NHS England and NICE were to get around a table and agree a satisfactory price, there would be no need for the legal challenge. In actual fact, the desperately poorly children we have heard about today would therefore be able to get the treatments that they desperately need.

[Catherine McKinnell]

Does the Minister share my concern that one reason why NICE turned down Brineura is that there is not enough long-term evidence about the drug’s effectiveness? That does prompt the question, which I am sure people at home would wonder about: how can we secure that long-term evidence when the treatment is so new—yet so effective, in my view—but is not even available on the NHS in this country? How do we ever find enough evidence to match the criteria that NICE is looking for?

[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.

[Caroline Dinenage]

Thank you, Mr Speaker. I do apologise for the further confusion that is here today. We are trying to seek further guidance on this—

[Mr Rees-Mogg]

Will my hon. Friend allow me?

[Caroline Dinenage]

In a moment.

The advice that I have been given is that I have to be very careful on the legal procedure because of the fact that it is not a legal procedure between individuals and the Department, but between individuals and NICE. I do not want anything that we say potentially to negatively impact on a family’s opportunity to get these very important drugs for their children.

[Mr Speaker]

Very important decisions are subcontracted to NICE, but policy responsibility, as the hon. Member for North East Somerset has pointed out, is that of the Government. We do not have Government by NICE; we have Government in the case of health policy through the Department of Health and Social Care.

[Caroline Dinenage]

I give way.

[Mr Rees-Mogg]

I am grateful to the Minister for giving way. I understand that discussions have been rapidly going on while we have had points of order and various other things happening in this Chamber. I am not sure whether this is orderly, Mr Speaker, but I happened to notice that there has been a return to the officials’ box of advisers. I wonder whether inspiration might now be forthcoming and heading in the direction of the Minister and whether I am now giving time for such advice to be passed through, or whether the telephone calls that I heard were taking place have not been as fruitful as I might have hoped.

[Mr Speaker]

I am not in a position to know that immediately for the simple reason that the Speaker does not possess eyes in the back of his head, and I have been focusing on the hon. Gentleman, the Minister and other colleagues, rather than on the occupants of the officials’ box. I have seen a note that has been circulating and a Minister is attempting to assist one of my advisers. I assume the Minister is attempting to do so on the basis of information provided. Whether there is something that is so valuable that the Minister in question is about to furnish the answering Minister with it, I do not know, but I am allowing a suitable opportunity for that missive to be passed to the Minister for Care, who is, of course, entitled to digest and reflect upon its contents and to decide in the light thereof how she wishes to continue, but I repeat that there is no shortage of time. I am very grateful to the Minister for her characteristic courtesy.

[Caroline Dinenage]

We are in a difficult position because the advice that has been forthcoming from the Department of Health and Social Care is not the same as the advice being given by the brilliant Clerks here. That puts me in a very difficult position, which is why I would like to commit to returning to the House with a statement once I have been able to pursue this matter a little bit further.

The Government want patients, including patients 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 decisions on whether new medicines should be made routinely available to patients. That is why we have NICE, which makes independent, evidence-based recommendations for the NHS.

--- Later in debate ---

Motion made, and Question proposed, That this House do now adjourn.—(Jackie Doyle-Price.)

[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.

[Catherine McKinnell]

Does the Minister understand how deeply hurtful the situation is to those families who are receiving this treatment on compassionate grounds from the drug company and how difficult it must be for them to listen to this debate? Not only are the Government seeking to hide behind sub judice rules when in fact they should be giving the answers that those families deserve; they are also sending families round and round, suggesting that they make applications based on individual circumstances and that they appeal decisions, when they just want to care for their very sick children. Is there nothing more that the Minister can offer those families who are listening to this debate?

[Caroline Dinenage]

I completely understand what the hon. Lady says, and I completely agree with her. I would love to be able to solve this problem straightaway and give these children the lifeline they need. Unfortunately, that is not the case today. I have committed to make a statement before the House finishes for recess at the end of next week, so I will come back shortly with all the information I am allowed to give. I really do not want her to think that this in any way suggests that I do not understand how the families must be feeling. I can only begin to imagine how desperate it must be to have such a poorly child.

[Mr Jim Cunningham]

I do not want to be difficult for the Minister, because she is doing a very difficult job, but could she clarify something? I got the impression from something she said a minute or two back that if NICE and NHS England concluded negotiations and agreed a price with the drug manufacturer, that would be acceptable to the Government.

[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.

[Mr Speaker]

Ordinarily, the proceedings would now conclude, and they will do so shortly. However, I think it important that our proceedings should be intelligible not only to right hon. and hon. Members but others who are interested in our proceedings but are not Members of this House. To try to achieve that objective, I want to add, by way of conclusion, the following.

I am advised by my officials that the Department of Health and Social Care claims that this matter—the subject of the debate—is sub judice because an application for a judicial review has been made. In the light of that, let me explain. Under the sub judice resolution of this House,

“Civil proceedings are active when arrangements for the hearing, such as setting down a case for trial, have been made”.

The Department has not supplied evidence that this test is met. Therefore, I stand by what I said earlier on advice.

By way of conclusion, I say to the Minister—echoing, I think, the sentiments of colleagues—that her commitment made in the circumstances, at very short notice, to return to the House before the summer recess to make an oral statement is appreciated, and we look forward to that statement taking place either tomorrow or on Thursday. Policy is a matter for the Government and not the Chair, but the hon. Member for North East Somerset and other colleagues who are similarly interested may rest assured that, for so long as this matter is not resolved to their satisfaction and they wish to bring it to the House for questioning, interrogation and debate again, as far as the occupant of the Chair is concerned, there will be multiple opportunities for them to do so. The matter will not go away.

I thank the Minister once again for her courtesy and consideration in difficult circumstances, and I thank all Members who have contributed to the debate not on their own accounts but in the interests of their constituents, very anxious parents and families, and sympathetic people right across the country.

Question put and agreed to.