Drugs: Ultra-rare Diseases

Greg Mulholland Excerpts

Tuesday 16th June 2015 Westminster Hall

[Mr David Crausby (in the Chair)]

The Speaker’s Commission on Digital Democracy recently recommended the use of regular digital public discussion forums to inform debates held in Westminster Hall. A digital debate has taken place on Twitter ahead of today’s debate on access to drugs for ultra-rare diseases. For this reason, Mr Speaker has agreed that for this debate members of the public can use hand-held electronic devices in the Public Gallery. Photos, however, must not be taken. I encourage Members who wish to refer to the Twitter debate to call it the rare diseases Twitter debate, rather than using people’s individual Twitter names.

[Greg Mulholland (Leeds North West) (LD)]

I beg to move,

That this House has considered access to drugs for ultra-rare diseases.

I am delighted to have the chance to speak on this important topic today. I was also delighted to lead and take part in the historic Twitter debate yesterday, which was a great success. On top of the very strong show of support from Members of all parties, the fact that nearly 1 million people took part in the debate yesterday shows how important the issue is.

I got involved in the issue because Katy and Simon, the parents of Sam Brown, a six-year-old boy in my constituency, came to see me. In 2009, when Sam was 16 months old, he was diagnosed with Morquio syndrome, an ultra-rare disease that 88 people in the United Kingdom have. It is a degenerative life-limiting condition with a typical life expectancy of around 25 years. It limits considerably what those suffering from it can do. All of us here can only imagine what it must feel like as a parent to receive the devastating news that your child will deteriorate before your eyes, not live to an old age, and may not even see much, if any, of their adulthood. Imagine how it feels when a nurse rings up and says, “There might be a treatment, but it is only a trial.” Of course, on hearing such news, what parent would not want to sign up for a trial for the drug Vimizim, supplied by the drug company BioMarin? That is exactly what Katy and Simon did: they signed up Sam to the trial without hesitating.

For the past three years, Sam has been doing a 100-mile trip from Otley to Manchester every Thursday to get Vimizim, his enzyme replacement therapy. Without it, Sam would see his growth stunted more than it already is, with further skeletal deformities and possible heart and vision problems. With Vimizim, Sam’s parents, and, even more importantly, Sam’s medical team, say that he is clearly physically more capable and stronger, with more stamina than ever before. To quote Katy, his mother:

“The drug has given him the freedom to be a child again.”

I ask right hon. and hon. Members to take the opportunity to share the single, “There is a Boy”, produced by the Keep Sam Smiling campaign and produced at his primary school, the Whartons in Otley, where they have shown huge support to an ordinary little lad who wants to be an ordinary boy and an ordinary man. The video for the single shows Sam being a fireman, a doctor and an astronaut, the kinds of things that he has the right to hope one day to be, but he can have that hope only if he gets treatment and is able to continue to take Vimizim.

We are here today because, after three and a half years, in just nine days’ time, Sam’s access to Vimizim looks set to be cut off.

[Jon Trickett (Hemsworth) (Lab)]

The hon. Gentleman knows that I represent the grandmother of Sam Brown. This debate is important. As the hon. Gentleman has said, the mother has already testified to how Sam is stronger and fitter as a result of taking the medication. NICE has said that it is

“likely to provide valuable clinical benefits for certain aspects of the condition”.

Even if it does not provide a full cure, how can the treatment for that wonderful young boy be axed?

[Greg Mulholland]

The recommendation from NICE is strange—I will come on to that—given that, clearly, the drug is effective.

Sam and other children and adults with Morquio disease are not the only people being let down. There are other conditions. I have been working with Members and organisations on the mutation of Duchenne muscular dystrophy and tuberous sclerosis. We have come together to campaign as one to say that we need a better way of approving drugs for ultra-rare conditions. At the moment we have a system in this country where people with ultra-rare diseases are discriminated against, and that must stop.

[Rehman Chishti (Gillingham and Rainham) (Con)]

I pay tribute to the hon. Gentleman for the work that he has done and for securing this debate. On other rare conditions, I have a very sick two-year-old in my constituency who suffers from neuroblastoma, a rare form of cancer that only 100 children suffer from each year. It is difficult to accept that my constituent has to raise money and travel to the United States to get treatment. We should ensure that children or anyone suffering from rare conditions, such as Ruby Young and those in the hon. Gentleman’s constituency, get the treatment they need at the first port of call in their own country.

[Greg Mulholland]

The hon. Gentleman is right to say there are other such conditions. I will not be able to mention them all today, but other Members may wish to do so. I will concentrate on the three conditions that I have been working on: Morquio, Duchenne and tuberous sclerosis. Some 180 people suffer from those conditions. I am sorry to say that all those people and their families have been hugely let down by the repeated failure of process by NHS England and by the thick wall of bureaucracy and utter lack of accountability.

[Mark Tami (Alyn and Deeside) (Lab)]

Like the hon. Gentleman, I have been involved in the issue of Morquio. The correspondence that we have had seems to want to blame the company; the company says it has not had the information; and patients suffer. This matter has been drawn out, and we now have the news from NICE.

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[Greg Mulholland]

I thank the hon. Gentleman for his intervention. It has been a pleasure working with him and others. We must continue to do so. That leads me on to the fiasco of the decision-making process. The leadership of NHS England should hang their heads in shame over the way they have handled this. There is also a responsibility on the shoulders of the Minister, who I know cares about this, but he needs to get a grip of NHS England and the way that it has failed families. Part of the problem goes back to the passing of the Health and Social Care Act 2012, which led to the disbanding of the advisory group for national specialised services in April 2013. That advisory group was the expert body that advised on specialist treatments and services, and it was respected by many rare disease charities.

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

I congratulate the hon. Gentleman on securing this debate. There are muscular dystrophy treatments in Europe that have suddenly been halted in this country. I hope the Minister can give us a good answer on that because people are suffering while there are delays. In some instances, it could shorten their lives.

[Greg Mulholland]

I hope that we get answers today and a real promise of intervention from the Minister.

[Mrs Cheryl Gillan (Chesham and Amersham) (Con)]

I pay tribute to the hon. Gentleman’s work on this issue. I joined this campaign because of Archie Hill, a constituent of mine aged 10 who has Duchenne muscular dystrophy. No matter what the Minister says about drugs such as Translarna and the process that the hon. Gentleman is about to outline, which has been disgraceful, that drug is available in other European countries and we have still not cleared it for patients in the UK.

[Greg Mulholland]

Indeed. It was a pleasure to meet the right hon. Lady’s constituent, Archie, and his parents. These young people are inspiring us to campaign. She is absolutely right. We are debating the European Union Referendum Bill today in the Chamber. Other EU countries, and some non-EU countries, regard these treatments as effective and affordable, yet we do not.

I will fast-forward from the scrapping of the previous body to October 2014, when NHS England came out with the scorecard system. That is despite one of the clinicians involved, Dr Chris Hendriksz, saying on 22 October in an email:

“I would suggest the scoring is not used at all for decision making this round and I would rather have people acknowledging that they are making random decisions than to try and give some credibility to a process that was deeply flawed.”

That is from one of the senior clinicians.

NHS England none the less went ahead with the scorecard system to decide which funding should be prioritised. Suzanne Mallah and her 10-year-old boy Kamal, who has Morquio and is another inspiring young person whom I have been delighted to meet, saw that that was not only haphazard but discriminatory. With the help of the MPS Society, they threatened legal action on 28 November against NHS England on the basis that the scorecard was clearly discriminatory, that there was no policy explaining it and that there had been no public consultation on its use. Just one week after that, on 2 December, NHS England announced that it was suspending use of the scorecard because the MPS Society and Kamal were right and it was wrong.

[Mr David Anderson (Blaydon) (Lab)]

The hon. Gentleman makes a good case. Is it not also the case that clinicians have not been listened to all the way through this, in the same way that they were not listened to when the Health and Social Care Act went through? That is what has led us to where we are. I have been the chairman of the all-party group on muscular dystrophy for 10 years. We had a very good working relationship with the specialised commissioning groups, which were effective in getting medication of this type to people, but the bureaucracy created by the Act was against clinicians’ wishes, which is why we are here today. NHS England has a lot to answer for. The Government’s decision to ignore the voice of professionals has put us in this position.

[Greg Mulholland]

It has been a pleasure to work with the hon. Gentleman and the APPG on muscular dystrophy on the Translarna part of the campaign. He is absolutely right. We want not only an acknowledgment from the Minister that the current processes are not fit for purpose and not fair on those with ultra-rare diseases, but a drive to overhaul them.

[Mary Glindon (North Tyneside) (Lab)]

I congratulate the hon. Gentleman on securing the debate. Does he agree that one of the best ways to help people suffering from ultra-rare diseases is Muscular Dystrophy UK’s suggestion of a fund to ring-fence money for these rare diseases?

[Greg Mulholland]

That is a powerful suggestion, as is using the surplus from the tariffs that drug companies are expected to pay to form part of a fund. There certainly needs to be an overhaul.

[Sir Gerald Howarth (Aldershot) (Con)]

Will the hon. Gentleman give way?

[Greg Mulholland]

I will give way, but I am conscious that I have not gone through the process yet.

[Sir Gerald Howarth]

We are all extremely grateful that the hon. Gentleman has been so generous in giving way. Like him, I was at Downing Street last week, supporting my constituent Harry Barnley, who suffers from Duchenne muscular dystrophy. The headquarters of the Batten Disease Family Association are in Farnborough in my constituency. The hon. Member for North Tyneside (Mary Glindon) referred to ring fencing. Part of the problem is that there is a very small number of these cases and they are very expensive to treat. I wonder whether we should either ring-fence some funding or introduce a surcharge on prescription charges generally paid by the public, so that the funding issue is taken out of it. There are two issues: the clinical issue and the funding. If we remove the funding issue, we can concentrate on the clinical issue.

[Greg Mulholland]

I thank the hon. Gentleman for his contribution. I am sure the Minister will want to consider that in his drive for an appropriate system.

After NHS England suspended the use of the scorecard on 2 December, a meeting of the NHS England clinical priorities advisory group on 15 December was called off. That is when we started campaigning for an interim process while NHS England went back to the drawing board. NHS England refused to do that, which I am sorry to say left all these families in the dark, with no idea what would happen next or in what timescale. NHS England then launched a consultation on 27 January, with a new process for deciding which drugs to fund that closed on 27 April. We still have not heard the decision. We have been told that there may be a decision on 25 June, although that has not been confirmed in writing. I hope that the Minister will give confirmation today.

Linked to that are the recent NICE recommendations, and particularly those on Vimizim. Even though we were clearly told by NHS England that its decision on 25 June would not be dependent on NICE, it now says that it will not approve Vimizim because NICE will not do so in the short term. The whole thing is a fiasco and an embarrassment. I understand the Minister’s argument that we cannot have political interference. However, the Secretary of State for Health made clear when he appeared before the Public Administration Committee in the previous Parliament that he accepts that the buck stops with him. When things are wrong and when bureaucrats are failing, it comes to his desk and to the Life Sciences Minister’s desk. I urge the Minister to take that up.

I pay tribute to the MPS Society for its amazing campaigning, and particularly to the chief executive Christine Lavery, whose son Simon had Morquio and died in 1982 aged just seven. Her passion and her colleagues’ passion have inspired me and others, and we will continue to work with them. The enzyme replacement therapy produced by BioMarin, Vimizim, is currently supplied on a free trial by BioMarin to 34 patients around the country out of a total of 88 patients, so more people with Morquio are not getting Vimizim than are.

The list price for Vimizim is £395,000 per person per year. In October, BioMarin proposed a fixed-term arrangement with NHS England to supply the drug at a lower price for a number of years. After BioMarin’s offer in October, NHS England did not even reply, despite repeated follow-ups, forcing BioMarin to announce in February that it would cease to supply the drug after 11 May; that date was then extended to 25 June. Having heard nothing, BioMarin said that it would have to withdraw the drug.

[Mark Tami]

Gracie Mellalieu in my constituency, fortunately being in Wales, will get the drug until October, but there is still that cut-off point. Is the hon. Gentleman amazed, as I was, at the total lack of engagement from NHS England, even when that offer was made?

[Greg Mulholland]

It is absolutely disgraceful and I urge the Minister to properly take that up. We have not had answers or justifications, although there can be no justification for NHS England behaving in that way. NICE’s decision not to recommend approving Vimizim in the short term has already been deemed to be flawed by those involved, including the MPS Society and clinicians, because it fails to consider BioMarin’s offer and has assumed that the cost of the drug will be the original £395,000. How has that happened? NICE also took months to put together the interim guidance, but has given only until next Tuesday to receive the extra evidence that it has asked for. Surely that is an unfair timeline for response.

As of 28 April 2015—which, incidentally, is a year after Vimizim was approved by the European Medicines Agency, meaning that it is approved in 20 European countries, including France, Germany and the Czech Republic—the drug was still not available in the UK, because NHS England has failed to put in place arrangements for funding it. Does the Minister not share the sense of frustration, anger and disbelief that the NHS refuses to fund the drug when so many of our neighbours do? More fundamentally, Earl Howe gave patients an assurance that their access to the drugs that they need would not depend on the cost per quality-adjusted life year measure. Can the Minister tell us why his Department has gone back on that assurance? That is exactly what it appears to have done.

I appreciate that the Minister has taken the time to meet us, but I remind him of the 11-page letter that he asked the organisations to send him some 11 weeks ago. We expected him to respond to that, as it was a complaint about NHS England’s handling of the matter, yet he simply passed it on for NHS England to respond to. That is not what we asked him to do, and the response does not address the points that we made to him, at his request, about how NHS England has failed people. I ask him again to reply directly and properly, and to investigate the mishandling of the situation by NHS England.

Duchenne muscular dystrophy has been mentioned. Again, I highlight the campaigning of organisations such as Muscular Dystrophy UK, Joining Jack, Action Duchenne, the Duchenne Family Support Group, the Duchenne Children’s Trust, Alex’s Wish and the Harrisons Fund. Those groups share the MPS Society’s frustration at the process. As many hon. Members know, Duchenne muscular dystrophy is a condition affecting only boys, and numerous potential treatments are in late clinical trial. Translarna, in particular, received conditional approval funding in the EU in August 2014. This clearly effective drug is being funded in a number of countries, including Greece, even given its economic situation, yet we are still no closer to hearing whether it will be funded here. I hope to hear positive news on that drug today.

I pay tribute to the Tuberous Sclerosis Association and the work of Jayne Spink and her colleagues. For those who do not know, tuberous sclerosis is a condition that causes the growth of tumours in organs, including the brain, eyes, heart, kidneys, skin and lungs, and a range of associated health problems, including epilepsy, learning difficulties and behavioural problems. The drug everolimus has been found to be effective in shrinking the tumours, extending life and improving quality of life, but although it was licensed for use in patients with tuberous sclerosis in February 2013, NHS England has failed to draw up a prescribing policy. At least two people have already died since the drug was licensed; Chris Kingswood, a consultant nephrologist, said that Julie Brooker’s death in January 2013 was “absolutely preventable” if she had been given access to everolimus.

[Jo Churchill (Bury St Edmunds) (Con)]

My constituent William needs that drug. The issue for his family is the timeline, which the hon. Gentleman mentioned. They have waited two years and been told that it may be another year, but they have said to me that William might not have that much time and that, like the woman the hon. Gentleman just mentioned, he might no longer be with them by then. Those parents are fighting for their son.

[Greg Mulholland]

The hon. Lady is right: none of these children or families has time. All those conditions deteriorate irreversibly. She is right that it has been 28 months since the drug was approved, yet patients are no closer to accessing it. What will the Minister do to speed up a commissioning policy for everolimus?

I turn to Batten disease, another condition already mentioned. I pay tribute to the Batten Disease Family Association. Batten disease is another condition that I had not heard of until I was approached by my constituents Duncan and Lynsey Brownnutt. I have been pleased to join Duncan to support some of his amazing fundraising efforts. This summer, he is off on a wonderful cycling trip to the Arctic Circle with his friend Rod to raise money, but the day after the general election, his six-year-old daughter Ellie Mae passed away from Batten disease.

Batten disease is another condition currently without any cure. It includes increasing visual impairment, complex epilepsy with severe seizures, decline of speech, language and swallowing skills, deterioration of motor skills resulting in loss of mobility and ultimately death. Potential treatment for Batten disease is not even being considered for 25 June. If the situation of the other conditions is still unclear and their drugs have been turned down, when will action be taken on treatment for Batten disease?

[Mrs Flick Drummond (Portsmouth South) (Con)]

May I just point out that BioMarin is also developing a treatment to alleviate some of the symptoms of Batten disease?

[Greg Mulholland]

The Batten Disease Family Association explained that to me when I met with representatives, but unfortunately that is not even in the consideration for 25 June. That is why we need an overhaul.

We have a five-year Parliament. I hope that the Minister will serve as the Life Sciences Minister for a considerable time, if not for the whole Parliament. His challenge as the Life Sciences Minister, as well as dealing with the accountability deficit that clearly exists in NHS England’s decision making, must now be to initiate a proper process for the approval of drugs for rare conditions. Of course there are cost implications, and of course drugs must be effective, but the situation is that there are effective drugs that this country is not funding, while other countries with less strong economies are finding the money in their health services to fund them.

[Rehman Chishti]

The hon. Gentleman talks about funding, but one aspect that precedes funding is awareness of such diseases. For example, the Government’s “Be Clear on Cancer” campaign does not take into account rare conditions and cancers such as neuroblastoma, from which my constituent, who is near death, is suffering. The Government must ensure that rare conditions are part of the bigger campaign, so that the people suffering from them get the help that they need as well.

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[Greg Mulholland]

The hon. Gentleman is right. I am afraid to say that politically, particularly at election time, there is not enough focus on rare conditions and too much focus on more common conditions in order to appeal to a broader group of people. We cannot allow that to lead to discrimination against people with ultra-rare conditions.

I will finish with two quotes. The first is from the framework agreement with NHS England, which clearly lays out that the Minister and the Secretary of State can and should intervene. Paragraph 4.11.3 says:

“If the Secretary of State considers that NHS England is significantly failing in its duties and functions, he is able to intervene and issue directions to NHS England. This also applies where he or she considers NHS England has failed to act in the interests of the health service.”

Clearly, that is what NHS England has done, and he must now act and get a grip of this process.

I will leave the final word today to Katy Brown, the amazingly courageous mother of six-year-old Sam, because we can imagine the devastation that she felt after the flawed NICE decision not to recommend approving the drug for the time being, knowing full well that NHS England will just use that decision as its cue to say no on or before 25 June. Katy has said that, if that is the case,

“Sam is being handed a death sentence…He is being denied his freedom, his independence and his future.”

That is not something that any of us should allow when we have a drug that is affordable if we have a system such as that in other European nations and that is clearly clinically effective. We need major change and, Minister, we need it quickly.