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Batten Disease

Volume 663: debated on Monday 22 July 2019

(Urgent Question): To ask the Secretary of State to intervene to ensure that funding is provided to treat those suffering from Batten disease.

I thank my hon. Friend for his question; he is a great champion for his constituents. We must ensure that all children with Batten disease receive world-class care and support. The Secretary of State has met families of children who suffer from the condition and has seen at first hand how cruel the disease can be. I pay tribute to my constituent Melanie Moffatt, whose amazing care for her daughter Matilda has been truly inspiring.

The whole House will recognise that a key element of providing world-class care is getting access to the most effective new medicines. The National Institute for Health and Care Excellence is the expert body, independent of the Government, that provides authoritative, evidence-based guidance for the NHS on whether new drugs and treatments represent an effective use of resources. If they do, the NHS is obliged to provide funding. In 2013, NICE introduced its highly specialised technologies programme, which supports access to drugs for very rare diseases such as Batten disease, through additional funding—up to £300,000 per quality-adjusted life year. However, companies still need to price their products appropriately and fairly. In this instance, Brineura has not been made available at a price that could be recommended by the NHS. NHS England stands ready to do a deal at a reasonable price, but this has not been possible so far. I urge BioMarin to sit down again with NICE and NHS England, as NICE has not yet published its final guidance, so that a fair and reasonable price can be agreed.

I assure the House that my Department and the NHS are working as hard as possible to improve the broader care and support for patients with rare diseases, including Batten disease. I reassure all Members that the Department is committed to ensuring that all patients with rare diseases have access to world-class medicines, care and support.

I thank the Minister for her response. I am grateful that she has not hidden behind sub judice on this occasion. Could she confirm to me that, while NHS England is obliged to follow a positive recommendation from NICE, it has the legal discretion not to follow a negative recommendation and can decide to pay for a drug? In the event that NHS England will not do this, what powers—if any—does the Secretary of State have to ensure that a drug is made available? If that cannot be done, in what way is NHS England accountable to Parliament for the decisions it makes, or is it entirely above accountability?

The point at issue today is that Brineura has now been available for two years and it is available in many other countries at a price that has been agreed between their authorities and BioMarin. We now know that three more children—leaving five altogether in this country, including my constituent Max—who ought to be receiving this drug are not. They suffer from a condition that means that they degenerate relatively quickly, and this drug can stop the decline in their condition. It is therefore urgent that this matter is addressed quickly, rather than continuing to allow time to pass with sick children getting worse. It really is a most important and pressing issue. In instances where the drug companies and NHS England cannot agree, but where other countries have agreed, I wonder whether there could be any system of arbitration to determine what is a fair price, because the development of these drugs is exceptionally expensive.

I thank my hon. Friend for his questions. I will attempt to answer all of them.

In terms of governance, no, NICE is not above accountability. Ministers set the framework for NICE, which is a non-departmental body. The reason it was established was to have fairness—so that there was no postcode lottery on access to various drugs. It is important that medical experts and scientists make these decisions rather than politicians. Regular governance meetings are held between the Department and NICE. There is a framework agreement. Where the Secretary of State considers that NICE is failing, or has failed, to discharge its functions or to do so properly, he can direct NICE to discharge functions. If NICE were to fail to comply with the Secretary of State’s direction in those circumstances, he could discharge such functions himself. There is therefore a strong and robust governance system with regard to NICE.

It is not always very helpful to use other jurisdictions as a comparison because we do not know the exact price that has been agreed. In addition, different systems have different healthcare populations and do not necessarily have the equivalent of our national health service.

Turning to access to Brineura, I pay tribute to my hon. Friend and to Max’s family. I know from the very moving testimony by him and by other hon. Members such as the hon. Member for Newcastle upon Tyne North (Catherine McKinnell) and from speaking to my constituent Melanie on numerous occasions that this is an absolutely dreadful disease. That is why we want the NICE process to be able to bring drugs to market as quickly as possible. Drug companies find this drug difficult to develop—that it is very expensive. It is not necessarily a drug that will be paid for by having millions of sufferers globally, and therefore a different system needs to be in place. That is why the bar for QALY is so much higher.

My hon. Friend’s suggestion on arbitration is very interesting, and I will take it away. On NHS England and the negative procedure, yes, in theory we could do that, but it is unlikely if NICE does not recommend a process. Overall, where a drugs company and NICE are unable to come to an agreement—we see this with other medication as well—Ministers urge the company to carry on negotiating to have a fair price, because every pound spent on one drug is a pound that we cannot spend on a drug for another sick person.

Thank you, Mr Speaker, for granting this urgent question. I thank and congratulate the hon. Member for North East Somerset (Mr Rees-Mogg) for securing it following his Adjournment debate last week. I do not doubt that he would have preferred the Minister to have come before the House voluntarily, rather than being forced to come here today for his urgent question.

Time and again, we come to this place to talk about a drug and its benefits to patients, only to be told that no matter how good it is, people cannot access it on the NHS. Among all the politics, there are people, including children like Max, who are suffering. No parent wants to hear a critical diagnosis for their child who has not yet really experienced childhood, let alone reached adulthood.

As we have heard, Brineura, a drug made available by BioMarin, could stop the progression of Batten disease. An assessment by NICE has found that Brineura could provide 30 extra years of good-quality life to patients. But, as has become expected when we discuss drugs for rare diseases in this place, Brineura is not available for patients on the NHS. NICE confirmed earlier this year that it was unable to recommend the use of Brineura on the NHS because of cost-effectiveness. The drug costs over £500,000 per person for each year’s treatment. BioMarin has another drug for rare diseases—Kuvan, for patients with phenylketonuria, or PKU. PKU patients do not have access to Kuvan, because it is also deemed not to be cost-effective. Does the Minister agree that the NICE appraisal process is just not fit for purpose when it comes to assessing the suitability of drugs and treatments for rare diseases?

Access to Brineura would help to give patients and families their child back, and it would allow them to enjoy time with their child and treasure special moments with them. As time ticks on without access to the drug, parents will witness their child’s condition deteriorate. No parent wants to see that, so we really need an appraisal process that captures rare diseases effectively.

Will the Minister step in and personally urge BioMarin, NHS England and NICE to meet and come to an agreement? Families do not want just warm words from the Minister; they want and need access to medicines now. I hope that this urgent question will result in real change in how we address rare diseases.

In answer to my hon. Friend the Member for North East Somerset (Mr Rees-Mogg), I urged BioMarin to get back around the table with NHSE and NICE and come to a fair and reasonable price. NICE has already approved drugs for 75% of rare diseases through its technology appraisal programme, including drugs for idiopathic pulmonary fibrosis and neuroblastoma. NICE’s process and review methods are constantly reviewed, and they are internationally respected. NICE knows that it has to keep up to date with developments in science, medicine and healthcare. There is a periodic review going on at the moment, and that includes extensive engagement with stakeholders.

I thank the Minister for coming today and providing more information. On Friday, I met the parents of Michal, one of my constituents. Michal is four years old, and he was diagnosed with Batten in February. He has already lost almost all his ability to walk and speak, and his parents are desperate to get him access to this drug.

I understand what the Minister says about it not always being helpful to compare access to drugs in different countries, but this drug is available in 20 countries, including Wales. If Michal lived in Bangor rather than Burton, he would be getting the drug that could stop the progression of this disease now. It is simply not acceptable to say, “Let’s not compare what happens here with what happens in other parts of this country, the United Kingdom,” and we need to know more about that.

The Minister talks about her desire to get BioMarin around the table. Time is of the essence for these children —every single day matters when it comes to stopping this disease in its tracks—so will she agree to pick up the phone to BioMarin and personally ask it to come around the table to negotiate with NICE? If Wales can afford to give children this drug, the Minister must have an idea about the scale of the difference between what we can afford to pay in England, and what Wales is paying. We have to find a solution to make this drug available to parents and children here in England.

I pay tribute to my hon. Friend and to the parents of Michal. This dreadful disease is so upsetting, not only for the children affected and their families, but for their wider communities. Health care in Wales is devolved. I again urge BioMarin to get back round the table, but I reassure my hon. Friend that I will make contact with the chief executive of NHS England to make sure that he is taking forward negotiations with BioMarin—he is the negotiating party—and I will let my hon. Friend know when I have done so.

Thank you, Mr Speaker, for granting this urgent question. I thank the hon. Member for North East Somerset (Mr Rees-Mogg) for securing it. The families of children with Batten disease have been left dangling for far too long, and the delay in a positive decision being arrived at—for what is a really obvious use of NHS funding, if we were to ask any taxpayer out there —is just too painful for many of them to bear. The stress and anxiety they are being caused is completely unacceptable.

The Minister acknowledges that this is a dreadful disease, but it is a dreadful disease that has a treatment—a highly effective treatment. It does not just score 30 QALYs; it has been acknowledged that it scores way beyond that. NHS England is adhering to an arbitrary cap set by NICE. Will the Minister please confirm whether NHS England can use a budget exemption in these circumstances to deal with the very tiny number of children who are affected, and what will she do practically—and what has she done since last week—not just to urge but to get BioMarin round the table with NHS England and NICE to get a positive outcome for these families and these children?

I thank the hon. Lady for her question. She has spoken to me and spoken in this House about Nicole and Jessica Rich. I agree that it is a highly effective treatment, but NICE sets the guidelines because it is made up of the independent experts and they are the ones responsible for the number of QALYs. However, as I have already said, it is constantly reviewing its guidelines in the light of the best available evidence. I have already reassured the House that I will make sure that I make contact with NHS England so that it is driving forward the process with BioMarin.

I have looked after a number of children with Batten disease in my career, and no one should underestimate the horrific nature of this condition with which a child develops apparently normally and then gets the horrific diagnosis that they will suffer neurodegeneration. I completely respect the importance of NICE being independent, and in general I do not get involved in these debates, but I believe I should do so in this one, because I actually think that NICE has this wrong. This drug does not make a little bit of difference—it does not have the effect of making someone die a couple of weeks later; it makes a phenomenal difference to the quality of life for these children. Yes, the trials have been short so far, but over a reasonable period it makes a massive difference, and I think we should do everything we can. I have heard the Minister say that she will ask the chief executive of NHS England to get BioMarin back round the table. How long will she give him to achieve that, and if he does not succeed, what will she herself do to ensure that these children get these drugs as soon as possible?

I pay tribute to my hon. Friend for all the work she has done as a clinician. I can only say again—I know this is very disappointing for the House—that we have to rely on the NICE process to be independent. I hear what the House is saying about some people having doubts about the process, but, again, it is under review. NICE is internationally respected, and it has been going for 20 years. Yes, these are exceptionally difficult cases, but this is why, as custodians of NHS funds, we have to be very careful, because every pound we spend on one drug is a pound we cannot spend on another. I hear what my hon. Friend says about this being a life-changing drug, and I hope that BioMarin, NHSE and NICE will, and we would urge them to, carry on with their negotiations.

There can be very few things as painful for a parent as knowing, once their child has been diagnosed, that there is potential treatment out there that may make a radical difference to their life, and it feels as though some bureaucrats—whether or not they are medical bureaucrats—are saying no. These little things in my hand, which would not have been prescribed for me if I had gone to the doctor a year ago, now cost £7,000 a month to the NHS, and I am delighted that I am able to receive them. However, I do want to make sure we have a proper system to ensure, for the most rare conditions, that there really is a possibility of making things available.

There may be only three dozen cases in the UK at the moment, which means there are probably about 900 in Europe, and if we include the Commonwealth, probably several thousand more. Why do we not have Governments in the world sitting round the table together with people from the pharmaceutical companies, who are not the baddies in this—these are the people, I think including the hon. Member for North East Somerset (Mr Rees-Mogg), whose investment made these pills available for me, in part; investment in these pharmaceutical companies is a good thing—to make sure that more of these rare—disease conditions can be treated?

We are determined to improve treatments for people living with rare diseases. As the hon. Gentleman pointed out, they have to be treated differently because fewer people are affected by them. We have the rare diseases strategy, and we are trying to use genomics better to diagnose and treat diseases. We are trying to be the first health service to put genomics into day-to-day health delivery, which will enable us to diagnose and treat diseases such as Batten more quickly. We have care co-ordinators for patients with rare diseases and we are trying to ensure that those who live to adulthood are cared for better, but what the hon. Gentleman said about having an international approach is valid.

The Minister rightly speaks about NICE’s important role in eliminating postcode lotteries. Does she agree that NICE’s independence is vital to ensuring availability to patients once an agreement is reached with BioMarin, wherever those patients are from, whether Penwortham in her constituency or Pensnett in mine?

My hon. Friend the Member for Rhondda (Chris Bryant) made the point about international co-operation, which already exists. A European agency examining the treatment of rare diseases was established in January. It is funded by €101 million and the UK is currently a participant. My question for the Minister is, will we still be, in the event of withdrawal after 31 October?

There will be co-operation with other medicines agencies, and I have no doubt that future co-operation will also come under any agreement that we reach with our European partners following our withdrawal from the European Union.

Sadly, this is not the first time that we have been here discussing how to make a highly specialised drug available for people generally and the talks with companies. May I add my request to my hon. Friend to act urgently to ensure that the review of NICE is undertaken with speed and that the full range of appropriate stakeholders is included in the discussions to take NICE forward?

I completely agree with my hon. Friend. I have answered debates here and in Westminster Hall about the medical treatments for rare diseases. To reassure both patients and their families and Members of this place, we need to ensure that the review of NICE processes is robust and transparent.

Less than half of all available rare disease treatments licensed by the European Medicines Agency are reimbursed in the UK for patients to access freely through the NHS, compared with 93% in Germany and 81% in France. With respect, Minister, the parents of those young children with Batten disease have seen those figures as well. They are desperate for the medication for their loved ones, so will she agree to an urgent review of the funding for such treatments for UK citizens?

We are putting record amounts of funding into the NHS, but I would rest again on the independence of the NICE process and the fact that it is experts and clinicians who are making these decisions. I agree that these are dreadful decisions and it is very hard for us to make them, which is why we rely on that expert advice. I would say to the hon. Gentleman that other jurisdictions are not always a good comparison.

I thank the hon. Member for North East Somerset (Mr Rees-Mogg) for securing this urgent question.

Will the Minister, while working to secure the funding of the drug Brineura to help sufferers of Batten disease such as my young constituent Kaycee Bradshaw, look at how we can help to prevent companies such as BioMarin from charging extortionate fees for life-changing drugs? Sadly, this company also charges beyond the NICE framework for Kuvan, a vital drug needed by my young constituent Liam, who suffers from PKU. BioMarin made a net-product revenue increase for 2018 of $31.3 million from Brineura and $26.1 million from Kuvan, and $1.5 billion from across its range of drugs. This, by my standards, is a clear example of playing profits with people’s lives. It hurts even more that it is children who are suffering. It is not on. It is time that Governments got together and took heed. We do not know what other countries are paying. It might be less than our £300,000 or it might not, but something must happen. Get together and put the pressure on, but please, please secure these drugs for our children.

I pay tribute to Kaycee and Liam. The hon. Lady makes a very important point. We want pharmaceutical companies to develop their medicines here, so that they are brought to the market here first and our constituents have access to them. However, we also have an obligation to spend taxpayers’ money in a very fair way, so that every penny we spend is spent correctly and appropriately. When it comes to PKU, Orkambi or Brineura, what we are all—NHS England and all of us here—saying to the drug companies is that we will pay a price, but we want it to be a fair price.

Earlier in this Parliament, I supported a young constituent of mine in securing access to Brineura. Health is a devolved matter in Wales, but the NICE recommendations are still very important. The problem I have seen over the past four years, unfortunately, is that those guidelines do not work particularly well when a disease is extremely rare. Does the Minister plan to look again and review the guidelines, so that people are not penalised simply because the condition they have is rare?

Right hon. and hon. Members have made clear to me their concerns about the NICE process for rare diseases. A review is ongoing, and I will keep a very close eye on it.

I am very grateful to the hon. Member for North East Somerset (Mr Rees-Mogg) and to the Minister. I have a sense that we will very likely be returning to this matter in September, if not before.