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Drugs: Ultra-rare Diseases

Volume 597: debated on Tuesday 16 June 2015

[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.

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.

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?

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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?

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.

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.

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.

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?

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.

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.

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?

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

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.

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.

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.

It is a pleasure to serve under your chairmanship, Mr Crausby.

I am pleased to be the first to congratulate the hon. Member for Leeds North West (Greg Mulholland) on securing this important debate. I also pay tribute to him for the enormous amount of work he has done on Morquio syndrome, which he has raised many times in the House and in Westminster Hall. He has also held numerous meetings and led delegations to Downing Street. He has worked assiduously on behalf of his constituent, Sam Brown, and, as we have heard this afternoon, he has worked on not only Morquio syndrome, but a range of ultra-rare diseases. He has done an excellent job today of highlighting the problems, the delays in funding and the amount of time it has taken simply to get these drugs through the approval process.

Rather than focusing on those aspects, I will talk about the human cost of these diseases, highlighting the case of my constituent, Jagger Curtis, who is just seven years old—he will be eight in August—and a pupil at Romsey Abbey primary school in my constituency.

Last Wednesday, Jagger was one of the brave boys who walked up Downing Street to hand-deliver his letter to the Prime Minister, which was an incredible experience for him and his parents. It was a really important part of their campaign to highlight the need for funding and approval of Translarna, because Jagger suffers from Duchenne muscular dystrophy.

Of course, Translarna is a relatively new drug. I say “relatively”, because it has been used in European countries since last year; it received conditional approval from the European Commission in August 2014. Yet here in the UK, as the hon. Gentleman has said, we are still waiting.

Duchenne muscular dystrophy is a very serious condition that affects about 2,500 people in the UK, almost all of them boys. It causes muscle weakness, leading to a dramatic loss of muscle function. Typically, patients will lose the ability to walk in their early teens; they will require respiratory support by their mid teens, and they are likely to die either of heart failure or respiratory failure before they reach 30. I cannot emphasise enough what a devastating condition it is and how brave families are when they have to face up to and deal with the reality of a Duchenne diagnosis.

Currently, the only treatments available address the symptoms, rather than the cause of Duchenne. They include the prescription of steroids, which of course have some very severe side effects, including sudden and dramatic weight gain, mood swings, which can be particularly difficult to contend with in teenage boys, and thinning bones.

As has been said this afternoon, Duchenne is a rare condition, with very few sufferers in the UK, and only about 10% to 15% of them have what is referred to as “the nonsense mutation”, which makes them eligible for treatment with Translarna. In some respects, Jagger is very lucky, because he is one of the boys with the nonsense mutation and is therefore eligible for Translarna. Currently, he is still mobile, which is absolutely critical when the use of Translarna is being considered, because it cannot be prescribed after a patient has lost their mobility. Translarna has the best chance of having a beneficial effect while the boys can still move around. Once they have lost their ambulation, it is too late and the opportunity has been missed.

Jagger’s parents, Julie and James, were told late last year that he was a suitable candidate for Translarna, and they genuinely believed that they were within a few weeks of going to the hospital and picking up a prescription for the one drug that they had been told could make a difference to their son. In November 2014, they had no idea that they would still be waiting for the drug now and that it still would not have finished going through the administrative process by the end of June. We are now seven months on from the day that they had expected to go and collect a prescription, but there has still not been a decision and they simply do not know what the outcome of this process will be.

During that time, of course, Julie and James have watched their son lose some of his mobility; his muscles have wasted away further. More than anything else, they desperately want an extension of the time in which Jagger is able to move around by himself, without the need for a wheelchair.

In his letter to the Prime Minister last week, Jagger wrote that he wanted to keep on playing football forever, just like his friends. He is an enormous Saints fan, and one of his proudest moments was going on to the pitch at St Mary’s to lead the team out. There is a fantastic photograph that he included in his letter to the Prime Minister, showing him shaking hands with the Saints manager, Ronald Koeman. In every other way, Jagger is a lively, lovely, normal little boy, who has a massive love for football, but, and it is a huge but, unlike most seven-year-olds Jagger has already been fitted for a wheelchair. His parents have had to make the necessary preparations—it was difficult, even heartbreaking, but they had to do it—to ensure that when Jagger’s mobility is more restricted a wheelchair will be ready and waiting for him so that he can still get around.

For Jagger and every other boy with Duchenne muscular dystrophy who has the nonsense mutation, the clock is ticking. In fact, it has been ticking since last August, when Jagger’s parents and others had their hopes raised that there was a treatment that was about to become available on the market. That treatment could give boys such as Jagger the chance to see out their time at primary school without needing a wheelchair, so that, as Jagger himself puts it, he can run around with his friends and be like any other normal little boy.

My hon. Friend the Member for Leeds North West—I should refer to him as the hon. Gentleman now, but old habits die hard, and on this subject he has been a great friend and a great campaigner; I pay tribute to him for that—along with Muscular Dystropy UK and Action Duchenne, has done great work to highlight the problems that people have faced in getting approval for Translarna in the UK. We expect a decision on Translarna at the end of June, and the company that manufactures it, PTC Therapeutics, indicated last week that it was ready to go, had stocks available and could supply it as and when it was needed.

If that drug is given the green light at the end of June, it will be distributed here, but the boys I have mentioned today have already waited for far too long, and this drug is the only one that is giving them any hope. I know the Minister has been most diligent for some months; he has listened to all we have had to say in this Chamber, in the House, on Twitter and indeed in the media. However, as we have heard, there are real concerns about how long the approval process has taken and about how complicated it has been, as well as about some of the inconsistencies and contradictions about when the drug might be made available. I hope that the Minister will make some comment on that.

I am conscious that there are many Members here in Westminster Hall this afternoon who want to contribute, so I have deliberately kept my remarks short. I will conclude with the words of Jules Geary, because I do not think anyone else could better summarise how her family feels:

“It is hard enough watching your child have to go through losing their muscles. For the drug to work, Jagger still needs to be mobile, so we simply don’t have time to wait. We have been given hope through this drug. We just can’t let it be taken away again.”

First, I congratulate the hon. Member for Leeds North West (Greg Mulholland) on bringing this matter to Westminster Hall for consideration. Westminster Hall is well filled today because we all have constituents who are suffering and do not have access to the drugs needed to combat these rare diseases. I also congratulate the hon. Gentleman on his hard work on this issue, for which he is well renowned; we have all said that, but it is the truth, and we all want him to know that we know it.

I am glad this debate has occurred, because it is on a subject that affects many people in my constituency. We have heard some stories and we will hear more before this debate is over.

The diseases we are considering may be rare, but collectively they affect the lives of 3 million people across the United Kingdom. That emphasises that everything must be done to create a comprehensive initiative for providing care to those affected by these difficult and challenging diseases.

Rare diseases tend to be life-threatening or chronically debilitating. There are between 6,000 and 8,000 rare diseases. Each one affects less than 0.1% of the UK’s population, but Rare Disease UK calculates that 75% of these illnesses affect children.

We are here today on behalf of our constituents, but we are also focusing very much on young people across the United Kingdom of Great Britain and Northern Ireland who have these problems.

The ultra-rare diseases that have been mentioned include Morquio disease, Duchenne muscular dystrophy and tuberous sclerosis. I would also add Prader-Willi syndrome, which some of my constituents suffer from.

The chance of improving people’s quality of life depends very much on a narrow timescale. It requires quick diagnosis, treatment and drug provision, so that drugs can be accessed when they are proven to be most effective. In other words, as every Member who has spoken has said, time is of the essence—the people who are suffering need help now, not in six or 12 months. It is our duty to make that timeline as transparent and effective as possible within the finite resources we have, and I understand the problems the Minister has. There must be adequate assistance for practitioners, to allow for timely diagnosis and the timely provision of drugs and treatment.

The hon. Gentleman has been very consistent on this issue, and he is right: as those of us in the all-party group on muscular dystrophy have found, one of the main reasons for delays is that clinicians—particularly GPs—do not see these diseases very often, and when they do, they are sometimes lost as to where to go. Once a disease is diagnosed, the people suffering from it should have no worse access to treatment than people with much more common diseases—surely that is the issue that has to be addressed. Once a disease is identified, we have to get to grips with it, and people have to get the medication and the support they need, so that they can get on and live the best life they can.

I agree wholeheartedly. I am sure the Minister has heard us all say that time is of the essence and that we should strike right away. That is what we are about.

The health and social care professionals involved in the diagnosis, treatment and care of these patients face difficult tasks. As I was saying, there must be adequate assistance for them, to allow for timely diagnosis and the timely provision of drugs and treatment. There also needs to be sufficient funding UK-wide.

In Adjournment debates and other debates about these issues, I have always referred to Queen’s University in Belfast and to the importance of research and development. Queen’s University is one of the universities that do research, and it works in conjunction with the Health Department. Perhaps the Minister could therefore give us some idea what the Government are doing on research and development to ensure that new drugs are found.

My hon. Friend mentions research. As he will know, I am involved in a campaign in Northern Ireland and across the United Kingdom on complex regional pain syndrome. The condition affects children, but it mostly affects adults from the age of 50 onwards, and people can lose limbs to it. One in every 3,000 people is affected, and many lives have been destroyed. We need more research to find a drug to cure this condition, and research funding needs to be put in place so that that research can be done.

I thank my hon. Friend for his intervention. That is a message that I, too, believe in, and I am sure the Minister will respond positively.

Leaving aside all the statistics we have heard today, we need to imagine the emotional strain these things put on people and their families, and we have had examples of that. Only 35% of patients are aware of a licensed treatment for their condition. There is something wrong when that is the case. How come only 35% of people know there is something there for them? How are the Government addressing that? I am not attacking the Minister—that is not how I work—but how do we move things forward in a positive fashion? Of that 35%, 89% are able to access the treatment, but 11% are not. Therefore, 65% of people are not aware of the drugs, and of the 35% who are, a proportion are not able to get them.

Like others, I want now to touch on Duchenne muscular dystrophy. If Translarna is given at the correct time, we can prolong the sufferer’s mobility. My constituents deserve to have access to that drug as soon as possible, and that is what I would like to see happen. The effects of long waiting times and uncertainty are widespread, and although ultra-rare diseases affect the few, their effects for those who suffer from them are an inescapable reality and should be treated with the utmost seriousness.

Families deserve a solution to the continual failure to establish a lawful, robust and transparent commissioning service that enables the rare disease community to access new drugs in an equitable and timely manner and to avoid situations such as those we have spoken about, where crucial windows of opportunity pass by. This is a crisis—it cannot be described as anything else. People are in trouble, and they need our help now.

Let me quickly pay tribute to the lady who looks after the Northern Ireland Rare Disease Partnership, Christine Collins. Last year, we met the Under-Secretary of State for Health, the hon. Member for Battersea (Jane Ellison), to discuss these matters. We were clearly moving forward, and the Minister was very responsive. The background information for the debate says that, in November 2013, the UK Department of Health and the devolved Governments published the UK strategy for rare diseases. In June 2014, the Northern Ireland Assembly endorsed it and gave a commitment to publish an implementation plan, and last year’s meeting provided an opportunity to underline the need for that to happen. Perhaps the Minister can give us some idea today of what discussions he has had with his fellow Minister to move things forward so that we can deliver on that commitment.

The debate has dealt with access to drugs. It has also given us an opportunity to bring out the gaps in the patient experience. Let us remember the patients, the families, the children and all those who suffer. They require a co-ordinated response from not only the health service and the social services, but research bodies and the relevant charities. I hope that the common experiences we have described signal the urgent need for access to these vital treatments. I remind all those in a position to have a tangible impact on drug access that while we are debating these issues, somebody else is falling into the trap and will, unfortunately, be unable to access the necessary drugs. I urge the Minister to respond positively, and I thank the hon. Member for Leeds North West again for giving us all a chance to speak about this issue.

Order. I intend to call the three Front Benchers at 3.30 pm. Three Members are standing, so if they can keep their contributions to less than four minutes, they will all get in.

I pay tribute to the hon. Member for Leeds North West (Greg Mulholland) for raising this issue and for his untiring work. I agree with my hon. Friend the Member for Romsey and Southampton North (Caroline Nokes) and the hon. Member for Strangford (Jim Shannon), who made many of the points I hoped to make.

I am grateful to have a short time to raise the case of my constituent Archie Hill and his parents’ tireless campaigning to get access to Translarna for him. It is inspirational to see how this family, and many others, have campaigned for their children. I can imagine nothing worse than watching one’s child slowly lose their mobility, knowing that their life expectancy will not be as great as ours might be.

Translarna is available in other European countries. As we have heard, it is available in Greece, which is not in the best economic health. Only recently, in Germany, the Federal Joint Committee determined that it provides a benefit for ambulatory patients aged five years and above with the nonsense mutation. The rise in the PTC Therapeutics share price on the back of that announcement shows that the company is well placed, and its drug is being recognised right around the world.

There is an irony here. If the decision coming down the track goes against making Translarna available to the patients who deserve it so much, the question arises as to whether this is about cost. The decision will almost definitely be made on a cost basis. Day by day, I see millions being spent in my constituency on High Speed 2 when we cannot spend £150,000 to keep a 10-year-old boy ambulatory and enjoying his life. We must question where a Government’s priorities are, when there are such people in front of us and we see the pie-in-the-sky projects that Governments of all complexions sometimes choose to pursue.

The point I really want to make is that if the decision is against providing the drug—bear in mind the failed processes that it has gone through—the Government have a golden opportunity to rescue the dish from the fire. I do not think it will necessarily fall out of the frying pan. On 8 July the Chancellor of the Exchequer will deliver his emergency Budget. We have previously created a cancer drugs fund, so that expensive drugs could be available to save lives. Will the Minister have conversations with the Treasury to see whether the Chancellor will on 8 July announce an access fund for drugs for rare conditions? If Translarna was one of the drugs on the list, it would be available in time for Archie Hill and the other children we have heard about today. To me, the awful thing is that time is running out. I do not think that letting time run out for those children would be the mark of a civilised Government, when the cost involved is small compared with some other expenditures that Governments make.

I add my congratulations to those that have been offered to the hon. Member for Leeds North West (Greg Mulholland) on tabling the debate. His passion and commitment on the subject are second to few.

Last week in Bath I met an incredibly impressive constituent of mine named Sarah Long. Many hon. Members will already know of her from the Twitter debate yesterday. She met me to discuss the benefits that she has received from Vimizim, the enzyme replacement therapy to address the cause of Morquio A. She is estimated to be one of just 88 people in the UK who need the drug. While she has been on Vimizim she has experienced dramatic changes, which have become gradually more apparent. She told me that since being on the drug her ability to use her lungs has been transformed. Before she started treatment she needed almost constant access to oxygen, and today she needs just four hours of nebulising. Pre-Vimizim, Sarah found it difficult to talk, but Vimizim has given her voice back to her. The idea of a return to the former days obviously fills her with dread.

Following recent conversations with the MPS Society, the National Institute for Health and Care Excellence announced on 4 June that it is leaning towards not recommending Vimizim for treating people with MPS IVA, or Morquio. That is only a preliminary recommendation and is not its final guidance; the decision may change after consultation. I hope in the interests of my constituent that it will change. I request my hon. Friend the Minister to lean heavily on NHS England before 25 June as it decides about reimbursement with respect to Vimizim on an interim basis, while NICE completes its decision making.

The date of 25 June is critical, as hon. Members have said. If NHS England announces a positive decision, all those who want treatment and who meet the criteria will be allowed access to treatment, regardless of whether they were on the clinical trial. If NHS England follows NICE’s current position and the decision is negative, BioMarin will immediately withdraw compassionate use from those in England who are receiving treatment.

As hon. Members will know, MPS can lead to reduced life expectancy. However, Sarah is in her forties. We have heard an awful lot in the debate about treatments that support young people, but Vimizim also supports adults, if they manage to get to such an age. If NICE gathered more evidence from people such as Sarah, the Minister would see that Vimizim has worked for her and for and others. The real injustice is that her quality of life has dramatically improved, but it appears that NICE is unable to conduct a peer review because of the lack of cases. I hope that the Minister will be able to look into that Catch-22 case.

Being on the drug has dramatically reduced the cost of my constituent’s care, because the amount of time on oxygen has fallen. That is also an obvious cost reduction to the taxpayer. NICE clearly needs to acknowledge the significance of clinical expertise in its processes, and to address its current expertise shortfalls to prevent other constituents with a rare disease from having to suffer the same problems. However, if a further extension to Vimizim is granted, it must be available to all ages and not just children.

I am delighted that the Government have published a strategy for rare diseases, but a strategy is only as good as its implementation. The strategy highlights a commitment to protecting patients with rare diseases and emphasises the need to improve and deliver effective interventions quickly, equitably and sustainably. I hope the strategy will put my constituent in a good place. I am pleased that the Government are leading the way on scientific and pharmaceutical research, but what good are those things if they do not reach those who most need them? The rare diseases strategy is excellent, but will the Minister provide the House with an update on an ultra-rare disease strategy? I would be most grateful if he were to have the time to meet my constituent in the coming days, given the urgency of the matter.

I thank and commend the hon. Member for Leeds North West (Greg Mulholland) for his work.

I find it heartbreaking that we have today heard about many constituents who have had access to drugs that have given them hope and improved their lives but which have then been taken away. Like the hon. Member for Leeds North West, I have a constituent who suffers from Morquio, Angela Paton. She is 35 years old; it has taken 35 years to find a drug that works, and it is now being taken away from her. Matthew Firth is another constituent, a young man with special needs who can no longer get a basic cream that he needs.

The case of Abi Longfellow is much in the news at the moment. She is a 12-year-old girl with a rare form of dense deposit disease. She needs a kidney transplant to live. Her father has been prepared in the past 12 months to give her his kidney. He should have had the operation on Friday, but for that to go ahead she needs the drug eculizumab. NHS England and NICE say that the drug will not work in Abi’s case—she has a very rare form of DDD—but there is research from the US, Canada and Italy indicating that the drug does work.

I thank the Prime Minister for intervening and asking NHS England to examine the case, and I ask my hon. Friend the Minister to ensure that that happens. There is research available; I find it difficult to understand whether, when the likes of NHS England and NICE say the drug will not work, that is just a tick-box exercise, or whether they look at research from outside this country. It is important to consider that. I would like a joined-up approach between NICE, NHS England and the Department of Health. I ask the Minister to consider the matter comprehensively, and to ensure that NHS England and NICE look into it.

I will now call the Front-Bench Members; there may be a vote, in which case I shall suspend the sitting for 15 minutes. The new rules allow Mr Mulholland to wind up the debate briefly, if there is time, but for that to happen, the Minister must be allowed enough time.

I was a breast cancer surgeon for more than 30 years, and I often experienced the situation that has been described in the debate with my patients and new cancer drugs. We were turned down for Kadcyla earlier this year. With cancer, it is often end-of-life research that later translates to early treatment research. People read things in the paper and say, “Oh, £90,000 for six months of life—that doesn’t make sense.” Inevitably, however, those drugs move forward. We have a different system in Scotland, and while listening to the debate I have been struck by how what is required is a system that is open and can be approached, and which looks from all angles.

In Scotland, the Scottish Medicines Consortium considers drugs as NICE does, and it considers worldwide evidence. It will work up a drug in detail. The balance for us seems to be slightly more on effectiveness than cost, although obviously cost is part of it. Our impression is that, for NICE, cost would sometimes be a bigger component. They are both looking at cost-effectiveness, and we all know there is not an infinite pot of money.

What has changed in our system over the last year is that we have combined our cancer drugs fund with our rare diseases drug fund and simply called it the new drugs fund. The amount in the fund has been quadrupled from £20 million to £80 million, which means that in any year it is a little more flexible in responding to demand, whether that is for drugs for rare diseases or for a new cancer drug. NICE only assesses three drugs a year, so rare drugs are never going to get that work-up. They need a separate system. In Scotland, we have pathways to follow for rare diseases and ultra-diseases.

The biggest change in Scotland in the last year is patient and clinician evaluation. If the evidence for a drug is so strong that it will go through on the nod and there is not an issue, that is fine and PACE is not engendered; but if things look more finely balanced or the drug will not go through, patient groups or drug companies can request a PACE assessment. That will involve expert clinicians, patients and patient groups, and allows people to get slightly outside the numbers and talk about life change, quality of life and money saved in respect of other aspects of the NHS—things that perhaps do not appear in a research paper. That is what is required: a system.

At the end of the day, the system will not produce a favourable result for every single person and every single new drug in the world, but it has to be fair. We cannot have things not being looked at properly, or individual requests being used as the main way of accessing a drug. The system I am talking about is meant to be a transition—supporting young people, for example, who have been on a trial, by giving them access on a compassionate basis, while we get through the paperwork prior to a drug being accepted. It cannot be left as the main method.

That system sounds excellent. I commend the Scottish Government on their work. That could be a great help to my constituent, Mr Trystan James, who suffers from tuberous sclerosis complex and is reliant on a clinical trial drug to deal with a life-threatening tumour. Of course, his drug prescription is therefore completely at the discretion of the drugs company and his family are going from one prescription to the next. That relates to what the hon. Member for Strangford (Jim Shannon) said about emotional pressure on families. I commend the Scottish Government on their work.

The important thing to remember is that if this is all done by individual requests, the NHS does not go to the companies. We need to realise that companies have often made investment over decades and that nine out of 10 drugs they research will go nowhere, but it is important to have a wider debate with companies to get the best price. Hon. Members mentioned that some companies are willing and able to reduce the price to get a drug in.

Drugs are licensed. We must not mix up licensing with funding. Licensing is about asking, “Is this drug safe and proven at a basic level?”, not anyone coming in and saying, “Rare plant juice will cure everything.” These are licensed drugs that we could prescribe—a doctor has the right to prescribe them—but the NHS has to make the decision about whether to fund them; those are funding decisions, not licensing decisions.

It is important that families know what the pathway is and how they move on when their clinician takes a case forward. It is important that they know they can respect decisions and how to lobby at the next step, and that they feel their voice is being listened to. We feel that PACE has, over more than a year, allowed us to do that. Clinicians in Scotland got frustrated about decisions going through without us informing that decision.

There could be a system that sits on the side of NICE, or a sub-group. One of NICE’s three assessments will never be given over to a drug intended for 88 patients when it is also assessing drugs that might be taken by 500,000 people. Rare diseases would always fall behind, and that is why those must have their own system and why the patient voice must be heard in these ways. Obviously, things have changed with the Health and Social Care Act 2012, but I commend such a structure to the Minister.

The hon. Lady is making a thoughtful contribution. Does she agree that it would be worth all Administrations in these islands, who together form the British-Irish Council, collaborating on these issues, particularly borrowing from the good example being developed in Scotland, and seeing whether there can be common achievement and common advances, and perhaps even creating some common funding stream, as well as the discrete funds that she has talked about?

Obviously, devolution gave us the power to do things differently, but I do not think that we should re-invent the wheel. Often, we will accept work done by NICE or re-evaluate it quickly, to see whether things should be applied differently, but we do not just go back to the beginning. However, I am sure that ideas can be shared in both directions.

It seems that certain drugs were left as orphans when the system changed. We know that patients with the brain tumour form of tuberous sclerosis, which the hon. Member for Carmarthen East and Dinefwr (Jonathan Edwards) mentioned, can access the drug through the cancer drugs fund, but if they have a kidney tumour and are treated by urologists they are not part of that system and simply will not be aware of it. Such random unfairness exists.

There is a forum and association, driven by the Health Minister, that discusses matters together with the three regions. A UK-wide strategy is already in place. The process is allowing that to happen already. However, it is delayed and has not happened yet; that is why we are concerned.

I think it is a matter of what ideas go on the table and what is being discussed in the meetings. Good ideas are going ahead. I commend the idea of including patients and clinicians in evaluations, because the numerical data from trials will often be small due to the nature of the diseases in question, and we will have to look wider. The problem for children is that if these drugs are to prevent deformity, they have to be got in early. People with Morquio already have the changes. We do not know yet how much change could be prevented, or how much saving there could be on a person’s disability in the long term if metastatic breast cancer treatments, which eventually become adjuvant treatments, are given earlier.

I commend the system I have talked about. I know it is difficult and challenging, but it is clearly fair, with an interim period for compassionate reasons, and people know where their voice should be heard.

It is, as ever, a pleasure to speak under your chairmanship, Mr Crausby. I, too, commend the hon. Member for Leeds North West (Greg Mulholland) on securing this important debate on access to medicines for ultra-rare diseases and on his contribution to this debate. He has pursued the issue doggedly through debates and questions, and it is right that he has been allotted time to bring these matters to the attention of the Government today.

I commend the UK parliamentary outreach team for hosting the online debate on this issue yesterday on Twitter, using #RDdebate. The public have had an opportunity to contribute to and inform this debate, and that is valuable. I am aware that many are watching us this afternoon. I also welcome the Minister.

When viewed collectively, it is more than apparent that rare diseases are simply not that rare. One in 17 people will be affected by a rare disease at some point in their life, which means that some 3.5 million people in the UK have a rare disease. About 75% of rare diseases affect children and almost one in three rare disease patients will die before their fifth birthday. These are sobering statistics and it is clear that more must be done.

In June 2009, the previous Labour Government adopted the Council of the European Union’s recommendation on action in the field of rare diseases, which recommended that member states should establish and implement plans or strategies for rare diseases. Following the work set out under the Labour Government, the coalition published the UK strategy in November 2013, and NHS England published its statement of intent with regard to the UK strategy in February last year. Since then, we have had the five-year forward view, which reaffirms NHS England’s commitment to achieving better outcomes for people with rare diseases. While each of the publications is a step in the right direction, so much more needs to be done, as many have said this afternoon. The health reforms of the Health and Social Care Act 2012, which was introduced by the coalition, have seen patients and professionals left to navigate a labyrinth to access particular medicines that in many cases have already been approved and received licences.

We have heard already about tuberous sclerosis complex. It is a rare genetic condition that is estimated to affect 1 million people worldwide. Those with the condition develop non-cancerous tumours, often in the brain, eyes, heart, kidney, skin and lungs. Often, TSC patients are at risk of complications, and surgical removal of the tumours is not always an option. It can have a massive and often severe impact on a person’s quality of life. We have heard about a drug called Everolimus that has been developed to treat some tumours associated with TSC; it has been granted market authorisation by the European Medicines Agency. However, despite being licensed in the UK 28 months ago, it has not been appraised by NICE. It is only available through the NHS on an individual basis or through the cancer drugs fund, resulting in significant inequalities in patient access.

Another example we have heard about throughout this debate, and on other occasions in recent weeks in the House, is Duchenne muscular dystrophy, which leaves many patients wheelchair-dependent by the age of 12. The drug Translarna received conditional approval in the EU in August 2014 for the treatment of DMD. However, almost a year on, too many boys who could benefit are still awaiting a decision on funding from NHS England. I commend my hon. Friend the Member for North Tyneside (Mary Glindon), who has done so much to raise awareness of the issue on behalf of her constituents. As we have heard, many Members from all parts of the House also have constituents who are affected, and the hon. Member for Romsey and Southampton North (Caroline Nokes) made a passionate contribution. Each day of delay sees the boys come closer to losing the ability to walk, by which point they would no longer be eligible for the drug.

Countries across Europe have already approved the drug. The UK has taken a leading role in clinical trials for Translarna, but we are lagging behind other European countries in the delivery of the drug to patients. Will the Minister tell us why we have fallen so far behind? I understand that NHS England is set to take a decision on funding shortly. We often hear the word “shortly”, so will he provide a further update and clarify and qualify what “shortly” actually means?

The system to approve prescription is confusing and frankly chaotic. There are seven pathways through which drugs for rare diseases can be evaluated and made available to patients. I will not go through every one of them, but it is clear that there is no clarity in the process to decide on which pathway a particular drug will be put. In particular, owing to a lack of clarity and transparency in the process, information on how or why one medicine evaluation approach or access route is selected over another is simply not available. Will the Minister outline the steps the Government will take to clarify the process, to speed up decisions and to make those decisions more open, so that patients can better understand the process?

I have specific questions for the Minister on two of the pathways: the highly specialised technology evaluation programme, which is administered by NICE, and Evaluation through Commissioning, which is administered by NHS England. There is significant concern that they could limit access to medicines for people with rare diseases. There are widely held concerns that the process in the highly specialised technology evaluation programme, introduced following the 2012 Act to appraise medicines for rare diseases, is too opaque and that the topic selection process is out of date. Does he have any plans to work with NICE to update the selection criteria for the pathway, as they do not take into account conditions defined by genetics, biomarkers or differences in clinical presentations?

Do the Government have any plans to increase the resources available to NICE to evaluate drugs through the highly specialised technology evaluation programme route, given that it is only resourced for three drugs appraisals a year, despite the European Medicines Agency licensing more than four times that amount? Finally, it is essential that patient groups have input on the process by which the drugs upon which patients rely are appraised, but a consultation on the programme has not yet been announced. When does the Minister expect that to take place?

Evaluation through Commissioning is a specialised commissioning pathway to conduct pilots to collect data to inform the decision-making process on funding for specialised commissioning proposals. It is more than a year since Commissioning through Evaluation was expanded, and a few months since it was rebranded as Evaluation through Commissioning. Will the Minister update members on the progress the process has made in expanding patient access to drugs? As I understand it, no medicine has been selected for the programme. When does he expect that to change? Will he update the House on the effectiveness of the early access to medicines scheme to date?

There are more than 6,000 rare conditions. A disease can be described as rare, but having a rare disease is clearly anything but. Improving access to medicines and treatments for the 3.5 million people affected by rare diseases is crucial in improving their quality of life. We have heard moving personal stories from Members from all parts of the House in this debate. I was looking on Twitter at some of the contributions made by members of the public in the past 24 hours. One tweeter said:

“If I could go on the #vimizim I could start to work and pay taxes for others to get hold of the drugs they need”.

That is just one contribution among many, and I ask the Minister to reflect on them after the debate. Many issues need addressing to improve the system of medicine appraisals, and we have touched on just a few today. I hope the Minister will take on board what Members have said, and I look forward to his reply.

I start by thanking Mr Speaker and the Speaker’s Office for granting this debate and for allowing the Twitter debate, which has been a big step forward for public engagement. I thank the hon. Member for Leeds North West (Greg Mulholland) for bringing the debate to this forum this afternoon. I suspect that in the 14 minutes I have available, I will not be able to answer every question, but I have made a detailed log and, with permission, perhaps I can write all those present with answers if I run out of time.

I pay tribute to the parents and the patients, some of whom are here today, whom I have got to know over the past few months, particularly Sam Brown and his mother, Katy, Jagger Curtis and Archie Hill. Others have mentioned the MPS Society and Christine, and the many people in Action Duchenne and the Muscular Dystrophy UK group. This campaign raises some of the hardest issues at the heart of public health and the NHS, and is being driven hard by the parents and patients with active representation from all parties in the House. It is my job to respond as best as I can and to try to put in place a policy landscape, but I pay tribute to them for their work in raising difficult issues that need to be dealt with. I do not think anyone can fail to be moved by the situation that the parents and children find themselves in. I assure them, as I do everyone else in the Chamber, that I wish there were an easy solution.

It is absolutely right that every child and patient in this country should ask for and expect the very best from our NHS, but it is equally true that, as a taxpayer-funded, universal, free-at-the-point-of-use, comprehensive health service, we simply cannot afford to provide every single treatment. I will say some more about the pressures on the system, particularly in the field of rare diseases.

As several Members have been kind enough to point out, this is one of the issues on which I have worked most tirelessly since taking office as the first Minister for Life Sciences last year. I have had several meetings with the hon. Member for Leeds North West and campaigners. Indeed, the Prime Minister and I spent more time talking about this subject than any other in my first nine months in office. I continue to work with NHS England to help it develop a more appropriate mechanism for the transparency and timeliness of its processes in all the specialist services. I have met MPs from all parties, patient groups, drug companies, campaigners and children, and I will continue to be happy to do so.

These are some of the most complex, difficult and life-changing decisions that any Department has to deal with. It is in everyone’s interest that such decisions are taken not by politicians but by clinicians and healthcare professionals, whose job it is to make those decisions—indeed, they do it for us every day of every week of every year. I thank them for that.

I want to discuss the context in which the challenge of rare diseases is developing, and what the Government are doing about it. I also want to discuss the timetable for the specific drugs that have been mentioned. I will then deal with some of the questions that were asked. We are at the forefront of an extraordinary revolution in biomedicine that is increasing pressure on all healthcare systems throughout the developed world, and will continue to do so. There are currently more than 6,000 rare diseases, and it is estimated that one in 17 people will suffer from a rare disease at some point in their lives. Therefore, more than 3 million people have a rare disease in the UK. The NHS is attempting to put in place a fair mechanism for dealing with their needs as best as it can.

The term “ultra-orphan” has no formal or legal definition, but it is taken to mean a disease

“affecting fewer than 500 people in England”,

which means a prevalence of around one in 100,000 patients. Having come to the House after a career in biomedical research, I know well that rare-disease pressure is going to grow exponentially as the extraordinary advances in genomics and biomedical research mean that we discover that more and more diseases that we used to think of as a one-size-fits-all blockbuster are rarer diseases that require stratified, targeted and, ultimately, personalised therapies. I can assure the House that the Prime Minister, who has experienced first hand the huge pain of rare disease in a family, feels that personally. We have devoted time to trying to tackle it, and will continue to do so. Indeed, that is part of the reason why the Prime Minister created my role: part of my remit in government is to tackle some of these issues.

We are doing a number of important things on rare diseases. We have put £20 million into funding the National Institute for Health Research’s rare diseases translational research collaboration and £900,000 into funding to support the work led by Public Health England to establish the first UK rare diseases register. We are leading the work with other EU countries and key colleagues to develop a European reference network to support research. We have also launched the precision medicine initiative and the rare disease consortium, but we are going further.

Central to the mission of the new ministry of life sciences is dramatically accelerating UK leadership in the field, which is why we have established Genomics England. We are the first nation on earth to seek to sequence the full genome of 100,000 NHS patients and combine that with clinical data to form the world’s reference database for targeted and stratified diagnostics and treatments. That is why we have launched the stratified medicines initiative, and why I have launched the early access to medicines scheme.

A number of colleagues challenged me about whether we were getting a grip on this: I have launched a review of the accelerated access to innovative medicines and technologies for that reason. I can assure Members that the scope of the review has struck a chord around the world. We are looking at NICE and at the regulator, and the vision of the review—I have asked for first recommendations this autumn—is to look at how we can dramatically accelerate the timeline for innovative medicines to come into the NHS, dramatically shorten the timeline for patients, and unlock what is essentially the great win-win at the heart of the NHS. We can use its research potential—its genomic and clinical informatics potential—as the world’s only integrated healthcare system to drive research into new drugs and bring down the time and cost of developing them. That way we can get drugs tested and developed here, to the benefit of our patients, while putting this country at the forefront of the revolution again.

We need to remember that it typically costs a billion to a billion and a half pounds and takes 10 to 15 years to develop a new medicine. That is unsustainable for the industry and for us. We cannot afford to pay the premium price at the end of patent life that the industry requires. We are leading the global race to put in place a new landscape. I fear that the solution will not come in time to solve the particular funding issue that has been mentioned, either this month or this quarter, or even this year. Nevertheless, we are making rapid progress. We will look back with pride on the UK’s leadership in this field.

NHS England has in place very carefully worked out decision-making processes for making drugs for rare diseases available to patients, and I want briefly to outline how they work. Because of their rarity and the low patient populations, services for rare conditions are commissioned nationally by NHS England, as opposed to locally by clinical commissioning groups. These specialised services include 146 prescribed medical services set out in legislation and account for approximately 14% of the total NHS budget—£14 billion a year. It is worth remembering the price of the system. For just this one class—the Translarna drugs alone—we are talking about hundreds of millions of pounds over a lifetime. We have to reduce the cost. We simply will not be able to afford the price required by the companies for every single new class that comes on stream.

The NHS England specialised commissioning process has been set out very carefully. It starts with one of the 68 clinical reference groups in NHS England creating a commissioning policy, which is produced by clinicians and other medical professionals. The commissioning policy is referred to one of the care boards and then to a clinical panel, which assesses the draft policy against the known evidence, with particular regard to clinical effectiveness and cost-effectiveness. The supported policies are passed on to the clinical priorities advisory group, which ensures that due process has been followed and makes a recommendation to the specialised commissioning oversight group. It considers the appropriateness and relative priority of new and existing treatments. The final sign-off is by the specialised commissioning committee, an NHS board sub-committee. NHS England’s clinical priorities advisory group formulates recommendations on the basis of clinical advice. I stress to colleagues across the House that it is not in anyone’s interest for Ministers ever to attempt to intervene in clinical decisions.

I want to touch on the timetables for the drugs mentioned by a number of colleagues: Translarna and Vimizim. On Translarna, the clinical priorities advisory group developed the clinical commissioning policy for the treatment of the mutation, and the policy was out for consultation between 24 March and 23 April. The group is considering the draft commissioning policy today and tomorrow and will make a recommendation to the specialised commissioning oversight group very shortly. The oversight group will consider the recommendations on 24 June and make recommendations to the specialised commissioning committee. The committee will make recommendations on 30 June and then make a decision on whether to commission Translarna nationally until NICE releases final guidance.

Before purdah, I was delighted to refer Translarna as a topic for evaluation by NICE’s highly specialised technologies programme. It is unfortunate that the general election fell right in the middle of the consultation process; that explains some of the difficulty we had dealing with the correspondence, as the hon. Member for Leeds North West mentioned. Final guidance on Translarna is expected in February 2016; draft guidance will be complete by the end of this year.

Similarly, Vimizim is being considered by the clinical priorities advisory group today and tomorrow and a recommendation will go to the oversight group. That recommendation will be considered on 24 June, and the final recommendations will be made on 30 June for subsequent consideration. NICE’s highly specialised technologies programme will release final guidance on Vimizim in October 2015. It is important to point out that NICE has not yet issued its final guidance on Vimizim to the NHS. I encourage patients, the public, professionals and the manufacturer, BioMarin, to engage with the ongoing consultation.

I have several questions to answer with just under 120 seconds remaining. It would not be appropriate for me to try to spin through every one, so with colleagues’ permission, I will write with detailed answers to them all. Several Members from across the House asked whether we could do something to raise money more quickly to purchase these drugs. I am discussing with the Chancellor the whole issue of how we purchase specialist drugs and put in place a landscape so that we are not only bringing drugs more quickly into the NHS and unlocking its power as a research engine, but updating our commissioning structures.

The accelerated access review that I am leading does not just address how we light the runway in terms of regulations and NICE and the Medicines and Healthcare Products Regulatory Agency’s processes to bring drugs to proof of concept in the system more quickly; it specifically looks at how we can commission better. It also deals with the cancer drugs fund. I hear the comments from north of the border—I used to advise Scottish Enterprise on this whole field. We will look at whether we might put in place some kind of innovative medicines fund for rare diseases and specialist drugs to support testing medicines within the system in a research medicine setting, particularly for rare diseases.

In the next few years, Genomics England and our leadership of genomic insights into diagnostics and new drugs will bring on a range of potential new therapies. We need to ensure that England has a landscape for testing those drugs that is compatible with Scotland. That may well mean that we will not pay premium retail prices to manufacturers at the end of a traditional phase III or phase IV development process, but build a new model of commissioning based genuinely on evaluation, thus unlocking the power of the NHS as the world’s greatest research engine.

Motion lapsed (Standing Order No. 10(6)).