Transcript for Parliamentary Debate on drugs for rare diseases

Transcript of Parliamentary Debate on Drug Funding for Rare Diseases – 20th Jan 2015 – Westminster Hall

Taken from

Drugs (Ultra-rare Diseases)

10.59 am
Greg Mulholland (Leeds North West) (LD):

I am delighted to have the opportunity to raise this hugely important issue, which affects a number of children across the country represented here today by a good number of right hon. and hon. Members. Although the debate is scheduled for only half an hour, I will take interventions from Members who wish to raise constituency cases. I am happy to do so because this is an important issue that affects families across the country. At the moment, 88 children in the United Kingdom have Morquio. There are 2,500 people with Duchenne muscular dystrophy, but 50 have a nonsense mutation, which falls into the category of an ultra-rare disease. We are talking about 138 people, 111 of whom are currently on treatment trials with the two drugs that are relevant to this debate.

I am delighted that there was a powerful lobby last week on behalf of the group with the nonsense mutation of Duchenne muscular dystrophy. Families from across the country gathered in Portcullis House and then presented a 24,000-strong petition calling for Translarna. I was with the hon. Member for Blaydon (Mr Anderson), who chairs the all-party group on muscular dystrophy, and the right hon. Member for Chesham and Amersham (Mrs Gillan), who was there with her constituent Archie Hill. We were joined by Liam and Saul, two other boys with the condition. We were all delighted that the hon. Member for Blaydon managed to ask a question at Prime Minister’s Question Time, and the answer gave hope to all those children because the Prime Minister gave a very personal answer comparing his own son to Archie—he had a picture of Archie playing football. The Prime Minister came to speak to us, and he said that he would personally do what he could, which echoes the Minister’s work. I thank the Minister for his personal assistance, which has been extremely helpful. I have met him twice, and I know he is very engaged on this matter, which I appreciate.

Mrs Cheryl Gillan (Chesham and Amersham) (Con):

I am grateful to the hon. Gentleman for giving way. I pay tribute to him for his work on this subject, which is second to none. I also thank him for mentioning my constituent Archie Hill and his parents, who have campaigned tirelessly for Translarna for their son. Does the hon. Gentleman agree that it is important that NHS England takes on board what the Prime Minister said to us, and to the families whom we represented, at Downing street last week by introducing a plan that enables Translarna to be available to those children who could benefit now, rather than waiting for the bureaucracy that is tying the drug up in knots? It could be available for those children now.

Greg Mulholland:

Absolutely. It is a pleasure to be working on this issue with the right hon. Lady and other right hon. and hon. Members from both sides of the House. This is a personal issue for me, too. My attention was drawn to the issue when Simon and Katy Brown came to see me with their son, Sam, in 2012. Sam was then four, and he is now six. Sam is receiving Vimizim, which is the only drug that clinically works for Morquio. Both drugs have been shown to have a very significant impact on the health of these individuals, changing what they are able to do with their lives, which is crucial.

David Simpson (Upper Bann) (DUP):

I thank the hon. Gentleman for giving way. I congratulate him on securing this debate. Is the supply of drugs purely down to finances, or is it totally bureaucracy? What is it? Why is there a hold-up on such an important issue?

Greg Mulholland:

That is a good question, and it is the nub of the issue. It is not finances. Finances are clearly an issue, but it is important to get the message out that they are not the cause of the hold-up. There is an element of bureaucracy in the process, which I will address. I know that the Minister is seeking to ensure that we have a proper process, but ultimately we have to make decisions based on the effectiveness of the drugs. In this case, both drugs have been shown to work and are licensed and used by health systems in other countries.

Caroline Nokes (Romsey and Southampton North) (Con):

I thank my hon. Friend for giving way, and I congratulate him on securing this debate. On effectiveness, would he agree—I hope the Minister also concurs—that it is about timing, particularly with Translarna? The drug will extend the ability of young boys to maintain their mobility and to keep out of a wheelchair, which is why it is so critical that the bureaucracy is speeded up for individuals such as my constituent Jagger Curtis and his dad, James. They need the drug now, not in six, 12 or 18 months’ time. It comes down to ensuring that the drug is available when it will be effective.

Greg Mulholland:

Absolutely. That is very much the case with Morquio, too. Simon and Katy Brown have told me that the drug is having a huge impact now. I met Sam and saw him running around when he visited my surgery. When there is deterioration in such conditions, the clock can never be turned back, which is why we are urging the Minister to address the situation. I am delighted that we had meetings with him. All the organisations involved, the MPS Society, the Muscular Dystrophy Campaign, Action Duchenne and Joining Jack, are urging the Minister and NHS England to find a way to ensure that all these children, not only the 111 who are currently on the trials—some of whom are, of course, receiving placebos—but all 138 children with these conditions, are able to access the drugs now. We have asked for a decision on that by the end of January.

We are in this situation because a decision was supposed to have been taken by NHS England on 15 December 2014, but a letter was sent by the MPS Society and a young man with Morquio syndrome, Kamal—I am delighted that his family are visiting Parliament today—and on receipt of that letter NHS England, realising that its process was potentially discriminating against people with ultra-rare conditions, pulled the entire process, leaving all these families in limbo. NHS England has a responsibility to put another proper, robust process in place.

Alec Shelbrooke (Elmet and Rothwell) (Con):

I am grateful to my hon. Friend for giving way. I will be meeting my constituent Angela Paton on Friday afternoon. She was part of the trial, which helped her immensely, but she is worried that the drug may now be withdrawn. Would my hon. Friend like me to report to him after that meeting on Friday?

Greg Mulholland:

Absolutely. It is critical that we all work together on this issue. Indeed, I would like to hear from the MPs for all these families across the country so that we can have one voice to say to NHS England and the Minister, who has been very helpful, that we need a solution and that we need to hear some news by the end of January.

NHS England is now consulting on a new process, and it has said that it will take 90 days. That may seem a reasonable time to come up with a process, but considering that the old process was flawed, there needs to be something to fill the gap that enables all these children to access the drugs now. At the moment, the drugs in this case are being supplied through the good will of the drug companies: BioMarin in the case of Vimizim and PTC in the case of Translarna. Both companies are engaged in the process, both have a part to play and both are involved in dialogue with the Minister and NHS England.

I will briefly explain the two conditions so that people understand. Morquio is caused by the lack of an enzyme needed to break down certain chains of sugar molecules used in building bones, cartilage, tendons and other bodily tissues. Those unbroken molecules are stored in parts of cells called lysosomes, which become swollen, disrupt cell functions and cause progressive damage. Babies with the syndrome grow normally, but growth slows significantly after 18 months. Those severely affected stop growing at about age eight, and their final height may be three or four feet, which has many effects on their quality of life. There is no cure for Morquio, but the enzyme replacement therapy Vimizim, for which clinical trials are ongoing at the moment, has been shown to be effective. As we have said, any delay with the drug will cause damage that cannot then be reversed.

Kerry McCarthy (Bristol East) (Lab):

Before I came in, I was speaking to my hon. Friend the Member for Alyn and Deeside (Mark Tami), who would have liked to be here but who has a meeting and sends his apologies. When reading the testimonies from the MPS Society UK, I was struck by how significant a difference the drug makes to children’s energy levels. Obviously, clinical trials and other formal assessments are important, but the personal testimonies from the families about the changes that they have seen in the children and how much energy the drug gives them are far more compelling than any scientific assessment could be. Does the hon. Gentleman agree that listening to the families is important?

Greg Mulholland:

It is crucial, but that also tallies with the medical evidence. It has been shown that that particular treatment stabilises symptoms, slows deterioration and has a hugely positive impact on quality of life. Children can do more and lead more normal lives; they have more energy and stamina. People with Morquio can live full lives and go on to education and employment, but childhood is their only opportunity to take a drug to slow the effects of the disease.

Duchenne muscular dystrophy, also caused by a mutation, affects young boys specifically. It also has no cure and gets worse over time. It begins by affecting a particular group of muscles and then muscles more widely, leading to difficulty walking, running, jumping, standing up and climbing stairs. Children with Duchenne muscular dystrophy may end up in a wheelchair fairly young, and are certainly predicted to become wheelchair-bound between the ages of eight and 14 as their muscles weaken and they lose their ability to walk.

Mrs Gillan:

The thing that came home to me was that those children need to access such drugs quickly, while they are still walking. Is that not why the time scale is so urgent? As soon as the child is no longer ambulatory, the drug will not have an effect. That is why we must have a speeded-up timetable and access to personal budgets for such drugs.

Greg Mulholland:

The right hon. Lady is absolutely right. Without such drugs, boys with Duchenne and children with Morquio are deteriorating now while waiting. They were expecting a decision on 15 December about whether they would be able to access those two drugs.

Translarna changes the natural course of the mutation in Duchenne muscular dystrophy, slows the decline in physical functioning and can therefore also play an important role in reducing the burden that the condition places not only on the boys who have it but on their families. They can do more, lead normal lives and see their boys do normal things with their siblings.

The number of people affected by the nonsense mutation in Duchenne is very small: there are only 50, 34 of whom are currently in the Translarna trial. The number expected to be eligible for Translarna is about 80 to 90 people, so we are not talking about huge numbers. Some of those people, incidentally, are not yet diagnosed; it is believed that that is the largest potential figure. Vimizim is already licensed in various countries: more than 20 European countries have access to it outside clinical trials, including France, Germany, Italy, Denmark and the Czech Republic. Translarna has been given conditional approval by the European Medicines Agency to treat boys with the nonsense mutation. Data gathered from clinical trials of Translarna indicates that the drug, as well as being effective, is safe. Results of the phase 2B trial were encouraging. Boys who received a low dose of Translarna could walk an average of 31 metres farther than boys receiving the placebo. Translarna is already available in Italy, Germany, Spain and France.

The clear message from all the families and organisations representing people with both those conditions is that we cannot wait for the drugs. NHS England has a responsibility, but so does the Department of Health, because the abolition of the previous highly specialised commissioning service led to an unfit-for-purpose process that had to be scrapped in the face of the pre-action. There is a moral and potentially a legal responsibility to find a way to make that decision. We are now already more than a month past the day when those families were expecting a decision that could literally be life-changing for them.

We understand, of course, that NHS England must put in place a proper process, but I urge the Minister to carry on doing what he is doing and the Prime Minister, who has taken a personal interest in the issue, to find a way to allow all those children to access these two drugs, which have been shown to be effective and to have a hugely transformative effect on their lives and those of their families. I will carry on working with the Minister and the two drug companies, but I urge him to listen to this message. We cannot wait 90 days. We need an interim solution, and I hope that we can have that by the end of January, soon as that is. I will carry on working with him and colleagues throughout the House until we get that news.

11.16 am
The Parliamentary Under-Secretary of State for Business, Innovation and Skills (George Freeman):

I congratulate the hon. Member for Leeds North West (Greg Mulholland) on his tireless work on this issue, and colleagues across the House, including the hon. Member for Blaydon (Mr Anderson), my right hon. Friend the Member for Chesham and Amersham (Mrs Gillan) and others here today.

I thank the hon. Member for Leeds North West for his kind words about the work that I have been trying to do for him, and about the Prime Minister’s signal of support. The issues are incredibly complex and do not lend themselves to an easy waving of a ministerial wand, but we are committed to finding a solution.

The hon. Gentleman has been tireless in his support of one of his constituents, six-year old Sam Brown from Otley, who has the very rare Morquio syndrome. A new treatment is now available called Vimizim, from which Sam has already benefited as part of a clinical trial. I wish to state my support for Sam and his family, and for all those who suffer from the disease, including those in the trial who have access to the drug when others currently do not. I also pay tribute to the hon. Gentleman’s support for the family of another young boy, Archie, who has Duchenne muscular dystrophy, a very rare form of muscular dystrophy that affects only boys. Archie’s family want him to be treated with a new medicine, Translarna.

I will say a little about the background to the diseases and what we are trying to do about them. Both conditions are very rare—there are about 80 children living with Morquio syndrome in England, and about 140 boys with Duchenne muscular dystrophy—so we are talking about a very small number of children with those life-limiting conditions. However, rare diseases are not rare: there are between 5,000 and 10,000 known types of rare disease, and an estimated one in 17 people will be affected by a rare disease in their lifetime, amounting to some 3 million suffers in the UK alone.

The truth is that the more we know about the human genome and the behaviour of genes in disease development, the more we understand its complexity. In cancer particularly, we know that the tumour itself mutates at different stages of the disease. The more we know about genetics, the more we discover that diseases that we thought yesterday were one disease in fact break down into different bundles of rare disease. New knowledge, technology and advances in biomedicine are a wonderful thing, but that does not detract from the fact that the NHS operates with finite resources and that difficult funding decisions must be made daily.

I was delighted to meet Sam’s mother and Archie’s family early in December, along with the hon. Gentleman and representatives of the Society for Mucopolysaccharide Diseases, to whose work I pay tribute, and of the Muscular Dystrophy Campaign. As the hon. Gentleman mentioned, we had a number of meetings over the Christmas period. I was delighted to meet patient groups and the manufacturers of Vimizim and ataluren just before Christmas. In that meeting, I asked the patient groups and companies to set out their proposals, which they have now done. I am grateful to them, and I have passed on that information to NHS England.

This morning, I met NHS England’s clinical director of specialised services, James Palmer, and its director of specialised commissioning, Richard Jeavons, and I will convene a further meeting shortly to pursue the issues that the hon. Gentleman has raised this morning. Since he first made me aware of this issue, I have been absolutely determined to bring as much ministerial focus to it as I can. I am also grateful for his acknowledgement of the Prime Minister’s support. The Prime Minister and I are both determined to ensure, without compromising due process, that the case for these children and their families is properly heard, and that the system works as it is supposed to.

I am acutely aware of the urgency behind the hon. Gentleman’s comments today and that is why I have taken the unusual step of trying to broker an agreement on what we might do to help children affected by these diseases, but I must stress that it is for NHS England, which in the end is the responsible commissioner, to make any decisions about making funding available so that the treatments are available on the NHS. It will act on the best clinical advice from the UK’s specialist body, the National Institute for Health and Care Excellence.

I will say a little more about the options for accelerating that process in a moment, but first I will talk about our approach to improving access to treatments for rare diseases generally, because I know that this debate is being watched closely by others who have an interest in a number of other drugs and conditions, in the commissioning process, and in NHS England’s prioritisation and decision-making framework. In setting the scene, I remind right hon. and hon. Members of the pressures that the NHS faces, particularly on budgets for rare diseases. The emergence of new treatments, the increasing personalisation of medicines, the end of the one-size-fits-all model and the possibilities offered by the rapid advances that we are making in genomic medicine and diagnosis are all putting immense pressure on NHS England’s resources for the commissioning of services for rare conditions.

Ideally, of course, we would want to fund all the treatments that are shown to benefit patients in any way, but we have to make difficult decisions about how we spend the money that we have available. That is why we have put clinicians in charge of the process, so that they can make decisions based on patient benefit and on the best health economic assessments that we can make. The painful truth is that with finite resources, when we make a decision in one case to accept a drug, we will make a decision elsewhere to reject, and we have a duty to all to ensure that we make those decisions fairly.

For people with rare conditions, their families, carers and clinicians, having access to the latest and most effective treatments is obviously critical, and I am absolutely committed to ensuring that patients with rare diseases have access to the latest and most effective treatments that represent value to the NHS and the taxpayer, as well as delivering benefits to patients. That is why we recently introduced the early access to medicines scheme, which aims to give patients with life-threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation or licence where there is clear unmet medical need. I am delighted that initial products have been brought forward in the last six months under that scheme.

More generally, our strategy for life sciences sets out an ambitious longer-term plan to improve the wider environment for health and life sciences companies in this country. Recently, I launched a major review of the landscape in the UK for bringing innovative medicines and medical technologies to patients much more quickly, and I will soon announce the chair, the terms of reference, the scope and the timetable of that review.

We are not in any way complacent. The truth is that the challenges in this sector, which are being driven by the pace of technological change, demand that in our policy-making framework, in the Department of Health and in NHS England, we adapt the way in which we handle these processes. Because of their rarity and the low patient populations, services for rare conditions are directly commissioned nationally by NHS England as specialised services. They account for approximately 14% of the total NHS budget and represent spending of about £14 billion a year. Both Morquio syndrome and Duchenne muscular dystrophy fall within these national specialist commissioning arrangements.

As right hon. and hon. Members are aware, NHS England is considering draft clinical commissioning policies for both Vimizim and ataluren. I understand that they are being considered as part of NHS England’s wider prioritisation process for funding in 2015-16. NHS England’s clinical priorities advisory group formulates recommendations on the commissioning of new treatments for rare diseases in England. It is made up of clinicians, patient representatives and commissioners of health services.

In summer 2014, a decision-making aid for the prioritisation of new interventions and treatments was developed by a partnership of stakeholders, including more than 250 patient representatives. It was due to be used for the first time in early December 2014, but on 28 November 2014 NHS England decided to postpone its introduction, in response to concerns that some patients affected by rare diseases might be disadvantaged by its application. The legal process about that must now run its course. I understand that NHS England is, rightly, reviewing the appropriate approach to prioritising new treatments and interventions within specialised commissioning in response to those concerns. A 90-day consultation on the prioritisation framework and decision-making process for commissioning decisions on new treatments will be launched by NHS England shortly. This morning, I again raised the importance and urgency of that consultation process.

I know that patients and their families are understandably concerned that it may take a long time for a decision to be made by NHS England on whether it will fund the drugs, and that in the interim the children affected will not receive them. However, I am delighted to say that NHS England has assured me that the consultation will have no impact on the decision-making timetable for commissioning NHS services from April 2015 onwards. In addition, it has assured me that existing treatments will continue to be commissioned, ensuring that support for patients is maintained. NHS England understands that the manufacturer, Bio Marin, is providing Vimizim under an expanded access arrangement to those patients who are on the clinical trial until an NHS England policy decision has been made.

Since April 2013, NICE, which is responsible for the evaluation of selected high-cost low-volume drugs under its highly specialised technologies programme, has been playing an important role in ensuring that commissioning decisions are based on a robust and thorough assessment of the available evidence. NICE has recently been asked to evaluate Vimazim under this programme, and it is also considering whether to develop guidance on Translarna. That is a very positive step, and I look forward to receiving NICE’s proposals on future topics that will be considered. I know that NICE will also be keen to learn lessons from its recent experiences with the new highly specialised technologies process, to make that process as efficient and effective as possible.

For my part, I am absolutely determined to continue playing the active role that I have taken on in the last few months, to drive this process and give it the focus that it requires. I am delighted to have confirmed with NHS England that it will continue to meet the treatment costs. I have signalled, and will continue to signal, to NICE, without compromising its processes, the strength of the case that has been made by Members and patient groups to put Translarna on the list, and to consider whether it can expedite its process in any way, but I do not want to compromise that process in any way. I will also ask NICE to ensure that it uses its review of the experience of the HST programme to explore how we can speed up both this process and others in due course.

Finally, I am committed to continuing to work with the companies to see whether I might be able to help broker some kind of planning arrangement that might encourage NICE to make the decision that I know everyone in Westminster Hall today would like to hear.

Mrs Gillan:

I am grateful to the Minister and I congratulate him on taking up the cudgels on this issue and trying to move it forward. The Muscular Dystrophy Campaign has asked whether the individual funding requests from patients would be a route to secure access to Translarna while the Minister is waiting for due process to take its course, because I am afraid that muscular dystrophy waits for no man and no process.

George Freeman:

I understand; my right hon. Friend makes an important point. In fact, I raised it this morning in my meeting with NHS England. My understanding is that NHS England will continue to consider individual applications for Translarna through its individual funding request process from patients who may be exceptional. However, my understanding is that such cases really do have to be exceptional. In reality, the members of the whole group that we are considering are more or less suffering from the same condition and therefore they may not qualify under those criteria. I merely share that with my right hon. Friend because I myself raised that point this morning with NHS England.

Greg Mulholland:

I stress to my hon. Friend the Minister that we are discussing two conditions and two drugs, Translarna and Vimazim. I also have to say to him again that we understand that NHS England has to put a process in place; of course it does, because the process it had put in place was not fit for purpose. Does he accept that NHS England has a legal, as well as a moral, responsibility in this regard? It certainly has a moral responsibility. Having said that the decision will be made on 15 December, NHS England cannot now hide behind saying, “There needs to be a new process,” when this situation is their fault in the first place. We are now a month on from that initial deadline, and there needs to be an interim solution to somehow allow these 138 children to access the two drugs in the meantime, and before that process is complete.

George Freeman:

I certainly accept the moral case; I think that everyone would accept that there is a moral duty to get this matter right and to try to make these decisions on the right basis and on the basis of the right evidence. The legal position, given the legal challenge, is more complicated, and it has triggered a formal process of reappraisal. As I have said, I will meet NHS England officials to urge them to try to expedite that process as best they can. However, I must stress that I do not want to get into a situation where we compromise due process and inadvertently undermine a case. What I want to see is a NICE decision being made as quickly as possible, and I will urge NICE to expedite that process in every way it can, so that we get the right decision that we all want.

11.30 am

Sitting suspended.