Drugs (Ultra-rare Diseases)

Greg Mulholland Excerpts

Tuesday 20th January 2015 Westminster Hall

[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.

--- Later in debate ---

[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 its 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.