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Cystic Fibrosis

Volume 603: debated on Tuesday 8 December 2015

I beg to move,

That this House has considered access to medicines for people with cystic fibrosis and other rare diseases.

As always, it is a pleasure to see you in the Chair, Sir Edward. I thank Carly Jeavons from Dudley for contacting me to suggest that we hold this debate and for what she has taught me about cystic fibrosis; Ed Owen, the chief executive of the Cystic Fibrosis Trust, and all his staff for their help and advice in organising this debate; and all the right hon. and hon. Members who are here to take part and to speak up for their constituents with cystic fibrosis and other long-term conditions.

Three years ago, Carly Jeavons was at a crossroads. She did not know whether to leave work and face financial turmoil or to continue working while risking her physical wellbeing and mental health. She struggled to breathe and had a lung function of around 44%. Every day she was taking around 90 tablets and undertaking around two hours of physiotherapy, and she spent two weeks in hospital every three months. In September 2014, Carly was offered the opportunity to participate in a clinical trial for a new type of treatment. Initially on a blind clinical trial, she was unsure what treatment she was taking, but later found out that it was a new treatment called Orkambi. The treatment has enabled her to spend more time with her family, and she has been able to go on holiday. She now attends a cystic fibrosis clinic every eight to 12 weeks, rather than every four.

Personalised medicines can have a transformational impact, not only for people with cystic fibrosis, but for a range of other illnesses. Without a more effective process for appraising such medicines, however, patients are unable to access new and innovative treatments. That is why I called for this debate. Cystic fibrosis is a life-shortening inherited disease that affects more than 10,000 people in the UK. It causes the lungs and digestive system to become clogged with mucus, making it hard to breathe and digest food. The damage to the lungs caused by cystic fibrosis means that many people come to rely on a lung transplant to stay alive. There is no cure, but many treatments are available to manage it, including physiotherapy, exercise, medicine and nutrition. Tragically, the median survival age is just 28.

Cystic fibrosis care has long been limited to managing symptoms and decline, but now, after 25 years of research, the Cystic Fibrosis Trust says that there is a pipeline of precision medicines that target particular cystic fibrosis mutations and seek to correct the basic underlying genetic defect. This new type of personalised medicine, which targets the defective gene, is a testament to modern science, and provides an opportunity to tackle this life-shortening inherited disease. As a contributor to the human genome project, British science has played a leading role in creating this new era of genomic medicines, and the UK is a global centre for clinical trials such as the one that my constituent Carly participated in. That work continues through the NHS’s 100,000 Genomes Project.

The first precision medicine for cystic fibrosis, Kalydeco, targets a mutation that only a little more than 4% of people with cystic fibrosis in the UK have. On that medicine, patients have shown increased lung function and slower progression of lung disease, and the number of hospital admissions has fallen by more than half. There are predictions that some people on the drug could expect a near-normal life expectancy. Orkambi is the next precision medicine for cystic fibrosis. It is being developed by Vertex, based here in London. It targets a mutation that around 50% of people with cystic fibrosis have, and, like Kalydeco, it has the potential to offer significant health benefits. Orkambi is now licensed for use in the EU and will soon begin its separate appraisals for clinical and cost-effectiveness across the NHS, covering England, Scotland, Wales and Northern Ireland. Work in this area is also important for people affected by muscular dystrophy and related conditions, with a number of drugs in late-stage clinical trials and one, Translarna, which is used to treat Duchenne muscular dystrophy, undergoing appraisal by the National Institute for Health and Care Excellence.

Muscular dystrophy is a progressive condition, often rapidly so, meaning that delays at the regulatory, approvals and funding stages can make all the difference to whether someone can access a treatment. Genomics England is currently delivering the 100,000 Genomes Project, the aim of which is to create a new genomic medicine service for the NHS. The project is focused on rare diseases and cancer. The developments in cystic fibrosis treatment and the impact of the new medicines have already demonstrated the human benefit from work in this area, but the current single technology appraisal system may not enable access to personalised medicines.

The existing NICE appraisal system makes decisions on the efficacy of a drug based on 24 weeks of clinical trials data. It fails to take into account the long-term benefits to sufferers’ quality and length of life. The focus on measuring the benefits of a treatment in terms of quality-adjusted life years does not work for genetic diseases such as cystic fibrosis, because it massively underestimates the impact that the drugs have on quality of life over the long term. It also fails to take account of the wider societal benefits of these medicines, such as the way they can help sufferers or their carers to get into work. In short, the existing system cannot provide an accurate assessment of new treatments, such as Orkambi, which offer long-term, preventive stabilisation of cystic fibrosis. It may say no too soon to treatments that require time for their value to be realised.

This debate is not about spending more money on drugs. In fact, it is the opposite: it is about making sure that we are helping people with conditions such as cystic fibrosis in the most cost-effective way, which could actually reduce hospital admissions and enable them to work more easily. According to the Cystic Fibrosis Trust, new genomic treatments could be available to 90% of people with cystic fibrosis within five years, but under the way in which NICE currently appraises medicines, none of those drugs is likely to be approved for use in the NHS. The system simply is not set up to assess personalised medicines where the patient target audience is, by definition, increasingly small. The situation affects many other rare diseases beyond cystic fibrosis, but without reform, research into precision medicines of this kind could dry up and a once-in-a-lifetime opportunity to beat cystic fibrosis and other rare diseases could be missed.

The Government recognise that change is needed. The creation of the Office for Life Sciences and the accelerated access review are among various initiatives to investigate reform. We need a system that gives new treatments the chance to prove their full effectiveness with long-term, real-world data. We have started to see that in other disease areas, with the development of the first managed-access agreement between a manufacturer and NHS England, which will allow unapproved treatments to undergo long-term testing before requiring full approval. That new model has the potential to be applied across the entire system.

I welcome the establishment of the accelerated access review to find ways of speeding up access to innovative new drugs and treatments. The interim report on the review emphasised the importance of flexibility and anticipating potentially transformative technologies, both those on the horizon and those already available. Such innovative transformative medicines should be seen as part of the solution. We need the NHS to give clinicians and patients time to assess how new precision medicines might slow the decline of diseases, and we need a system that gives medicines the chance to prove their true effectiveness with long-term, real-world data.

Cystic fibrosis is a test bed for reform because the Cystic Fibrosis Trust hosts the UK cystic fibrosis registry, an anonymised database that lists the 10,583 people in the UK with cystic fibrosis. The registry already provides real-world data to health commissioners and pharmaceutical companies so that they can monitor the efficacy of treatments. That makes cystic fibrosis a unique testing ground to pilot a new appraisal system for innovative medicines that could be applied to treatments for a wide range of conditions beyond cystic fibrosis. Orkambi could be the first treatment piloted. This is a once-in-a-generation opportunity to beat cystic fibrosis. Like Carly Jeavons, the 4,000 people in the UK eligible for Orkambi do not have time to wait for the system to catch up.

Last night, hundreds of people with cystic fibrosis, along with their families and carers, took part in an online digital discussion on social media that enabled them to share their experience and opinions directly with Members of Parliament ahead of this debate. Simon, who took part in that debate, said that it is

“hard to state the significance on quality of life”

that new drugs had given him. He said that he

“now had a stable job”

and is

“in the middle of getting a mortgage”.

Lorraine, who cares for two children with cystic fibrosis, told us that these new treatments mean that she can go back to work and worry less about outliving her children. Michael said they will enable him to focus on his career without fear that he will have to give it up as he gets more unwell. Kelly pointed out that having healthier people who need less hospitalisation could save the NHS in the long run. Last night’s discussion and this debate are supported by Parliamentary Outreach, which aims to enable people with cystic fibrosis to come together and express their views.

Cystic fibrosis is a uniquely cruel condition. The people who suffer from it are unable to come together because they are vulnerable to the different bacteria that grow in their lungs. Although those bugs are usually harmless to people who do not have cystic fibrosis, they can settle in the lungs of those who do and harm them. Our discussion last night and this debate are important because they enable people with cystic fibrosis, who do not normally get the chance to speak up, to be heard. They show that if we embrace new technology and think of new ways of opening up democracy beyond the walls of Westminster, people such as Carly, Lorraine, Michael and Kelly can be heard.

The system needs to change. We need NICE reform and an appraisal system fit for a future that includes personalised medicines, which cannot be approved too soon. In the current system, decisions about a drug’s efficacy are based on 24 weeks of clinical trials data, but for new medicines such data are not available. The system needs to account for the development of data over time, and for cystic fibrosis it needs to account for the fact that the value of the new medicines will be realised over time.

Cystic fibrosis is a test bed for reform. The Government must agree to explore ways of collaborating with the trust. I know that the Minister is meeting the Cystic Fibrosis Trust later today to discuss some of these issues. There has been major investment in the life sciences in the UK, but we cannot continue to invest in developing innovative new medicines if patients cannot access such treatments.

I have several questions for the Minister. Can he update the House on the timings for developing proposals for a new system for appraising new medicines? Will he consider meeting the CFT to discuss working with it to develop a system for managed access to medicines that includes a CF registry? Can he comment on the safeguards that will be in place to ensure equality of access to medicines under any new scheme? Will he consider amending the appraisal process for the new drugs to give more weight to the societal benefits for sufferers and their carers? What is the Government’s latest thinking on following Scotland and Northern Ireland by introducing a ring-fenced fund for rare disease drugs in England? Will he write to the chief executive of the National Institute for Health Research to ask how his organisation plans to work with specialist muscle centres to address concerns about the lack of clinical trial capacity for Duchenne muscular dystrophy? Finally, how can NICE and NHS England be given greater powers to negotiate the best price with pharmaceutical companies to ensure that new treatments are not held up or rejected on the grounds of cost?

It is a pleasure to serve under your chairmanship, Sir Edward. I warmly welcome the Minister, who, I am afraid, is very familiar with what I am speaking about today; I hope he gives me an A for effort and persistence. Given that we have spent so much time discussing access to Translarna, perhaps in his winding-up speech he will have some good news for me and my constituent.

I congratulate the hon. Member for Dudley North (Ian Austin). I am absolutely delighted that he secured this debate on access to medicines for people with cystic fibrosis and other rare diseases. Like me, he knows how important this issue is for families up and down England. I have been looking at the issues surrounding Duchenne muscular dystrophy for what seems like many years—in truth, it has been for just over a year. Only 90 boys affected by the disease in England are eligible for this drug, and the number is slightly larger across the whole of the United Kingdom.

Duchenne muscular dystrophy is a devastating condition that leads to full-time wheelchair use between the ages of eight and 11. It is a progressive, muscle-wasting disease that eventually affects the muscles involved in the respiratory and cardiac functions. Sadly, few with the condition live to see their 30th birthday. I have been working with Muscular Dystrophy UK, which fights causes to do with muscle-wasting conditions. I pay tribute to that organisation for all the support and help it gives. It not only informs Members of Parliament, but helps people affected by those diseases. My constituent, young Archie Hill, is an inspiration to everybody in this area. He has been campaigning for many years, and he and his family are indefatigable in their efforts to get the right medicine at the right time to these boys.

I congratulate my hon. Friend the Member for Dudley North (Ian Austin) on securing this timely debate. As the right hon. Member for Chesham and Amersham (Mrs Gillan) will recall, some months ago we all went to Downing Street to petition to get something done about muscular dystrophy. I am sure she would agree that one of the big problems is that even if the new treatments are okay, there is always a long run-in, in which negotiations take place between the Government and the pharmaceutical companies.

The hon. Gentleman is absolutely right. I pay tribute to the other colleagues in the House who took part in that petition. That truly cross-party effort aimed to draw attention to the drugs that are not readily and fully available to our constituents. I was grateful that it was a cross-party delegation, because such things are much stronger when they take place in an atmosphere of good co-operation across the board rather than a political atmosphere. We saw parliamentarians at their best, so I thank the hon. Gentleman for attending that lobby at No. 10 Downing Street, which was inspired partly by Muscular Dystrophy UK and partly by the families it supports.

The issue for me is the drug that the hon. Member for Dudley North referred to. Translarna is its trademark name; it is called ataluren. It is produced by a company called PTC Therapeutics, which calls it its “lead product candidate” for these disorders. I know that the Minister is familiar with PTC Therapeutics, and I hope that in his winding-up speech he will refer to any contact he has had with the company. One of the issues surrounding the efficacy and licensing of the drug is the cost, so I hope the Minister will update us on that situation.

PTC Therapeutics states that the drug is a

“novel, orally administered small-molecule compound for the treatment of patients with genetic disorders due to a nonsense mutation. Ataluren is in clinical development for the treatment of Duchenne muscular dystrophy caused by a nonsense mutation…and cystic fibrosis caused by a nonsense mutation…Ataluren was granted conditional marketing authorization in the European Union under the trade name Translarna”.

I believe that it is already available in France, Germany, Italy and Spain. It is the first treatment approved for the underlying cause of Duchenne muscular dystrophy, which is a complicated condition.

Nonsense mutations are implicated in a variety of genetic disorders. They create a premature stop signal in the translation of the genetic code contained in the mRNA. That prevents the production of full-length, functional proteins. The company says that

“ataluren interacts with the ribosome, which is the component of the cell that decodes the mRNA molecule and manufactures proteins, to enable the ribosome to read through premature nonsense stop signals on mRNA and allow the cell to produce a full-length, functional protein. As a result…ataluren has the potential to be an important therapy for muscular dystrophy, cystic fibrosis and other genetic disorders for which a nonsense mutation is the cause of the disease.”

The importance of access to Translarna cannot be overstated. Boys such as my constituent Archie Hill have been waiting since August 2014 for a decision on whether Translarna will be approved in England. As I said, it is the first licensed drug to tackle an underlying genetic cause of Duchenne’s. It would help to keep Archie and these other boys walking for longer and potentially delay the onset of the devastating symptoms affecting the heart and lungs that I referred to earlier.

NICE’s appraisal of the drug is ongoing, but the families have not yet been made aware of when guidance will be issued, leaving them facing an anxious wait over the Christmas period. Over the time I have known Archie and his family, I have seen his mobility decrease; it is depressing to see such an active, energetic, lively, intelligent young man, who has his life before him, being denied a drug that could well keep him active for longer and improve his quality of life.

My right hon. Friend is making a powerful argument. She is right to say that we must improve access to new medicines, which can transform the lives of people such as her constituent Archie. Does she agree that new medicines may also reduce hospital admissions, which would have a huge impact on the NHS?

I thank my hon. Friend for that intervention. He is absolutely right. There is no doubt that increasing the length of time that these young people can be kept active and mobile will inevitably reduce the amount of time that they spend requiring treatment in other health settings.

I also want to describe the emotional journey. Seeing anybody suffering with a muscle-wasting condition is terribly draining, because they fade before one’s eyes. That is why the drug is so important, particularly for young people suffering from Duchenne’s. I turn now to my constituent’s mother, Louisa Hill, for a quotation. She said:

“Decision makers need to understand the impact on children of even a small change. It gives them more time to run and play football with their friends. It’s really buying precious time. Archie will have to deal with very difficult mental and physical challenges as his condition progresses. Translarna is buying time for Archie just to be a kid.”

If you are not touched by that statement from a mother, I do not know what you would be touched by.

Translarna is not the only potential therapy that could benefit Archie. For example, others, such as utrophin upregulation, which involves injecting a protein called utrophin into the muscles to compensate for the loss of dystrophin in boys and young men with Duchenne’s, are in a later stage of clinical trial. It is vital that the process of moving such drugs from the laboratory to the clinic is expedited, including ensuring that appraisal processes are as swift as possible; that secure funding is available to help meet the costs of new drugs; and that NHS England and NICE have effective mechanisms to negotiate an appropriate price with drug companies.

On 14 October, I had the temerity to question the Prime Minister on Translarna at PMQs. He referred to the cancer drugs fund and its role in reducing the costs of drugs for rare types of cancer. A similar model would help for rare disease drugs for conditions such as Duchenne muscular dystrophy. The Prime Minister said:

“The cancer drugs fund has helped to reduce the costs that the companies charge. We need to see that in other areas, too.”—[Official Report, 14 October 2015; Vol. 600, c. 313.]

The Government’s accelerated access review provides an important route through which such issues could be addressed. I hope that the Minister will have his feet held to the fire by the Prime Minister’s answer.

Research into treatments for Duchenne’s is at a promising stage, with a range of potential therapies in late stage clinical trials. As I said, Translarna is already licensed in Europe, but the UK muscle centres where trials are conducted are reporting that given the growth in clinical trials they lack the resources, such as staffing levels and equipment, to keep pace. As a result, centres report that they are turning away new trials—not because of bad science, but because of a lack of capacity. [Interruption.] I see the Minister shaking his head. He knows that the situation is serious and I hope he will comment on it.

That lack of capacity risks causing a bottleneck in drug development and gives boys such as Archie Hill less chance to enrol on a trial that could allow them access to a new therapy. A clinical trial capacity audit, conducted by Muscular Dystrophy UK as part of the “Newcastle Plan” of joint working with UK Duchenne charities to address clinical trial capacity, corroborated the reports and also found that:

“Work on clinical trials is not counting towards specialist training at many centres for medical doctors, physiotherapists and nurses”

which is affecting trainee participation. In addition, it was found that a

“lack of acknowledgment of research in clinical job planning means that already overstretched clinical staff are having to carry out research activities in their own time. This is consequently severely limiting centres’ abilities to take part in research.”

It also found that the process of setting up a clinical trial can be excessively bureaucratic. Perhaps the Minister, with his experience in this area, will be able to comment on that.

I am disappointed that Archie Hill and the other boys suffering from Duchenne’s do not have access to Translarna. The process has seemed to take an incredible length of time, and I hope that the Minister will be able to do something about it. Like the hon. Member for Dudley North, I have a series of questions that I want to put to the Minister, which may help him when he sums up.

First, will the Minister commit to meet representatives of Muscular Dystrophy UK? I would be grateful for that, and it would be helpful for him to discuss the accelerated access review, particularly in the context of the emerging treatments for Duchenne’s. Secondly, I do not suppose that he can say this, but when can families such as Archie’s expect to be notified of NICE’s guidance on access to Translarna on the NHS? It is the obvious question and one that I hope he can answer.

Thirdly, will the Minister ask the chief executive of the National Institute for Health Research’s clinical research network how his organisation plans to work with specialist muscle centres to address concerns over the lack of clinical trial capacity, particularly for Duchenne’s? The hon. Member for Dudley North referred to the latest thinking in Scotland and Northern Ireland, such as introducing a ring-fenced fund for rare diseases. I hope that that might be a recommendation of the accelerated access review.

I do hope that the Minister will be able to give us some optimism. Boys such as Archie Hill are an inspiration to us all. For one so young, he is very mature in his attitude towards not only his Duchenne muscular dystrophy, but other children suffering from rare diseases. He has great capacity for humanity and for tireless campaigning. This will be the second Christmas since I met him that he will be waiting for an outcome on Translarna. Will the Minister talk to PTC Therapeutics, to NICE and to anyone else to whom he can reach out, to ensure that this year the Christmas present for Archie Hill and other boys in England is to have access to ataluren or Translarna?

It is a pleasure to serve under your chairmanship, Sir Edward.

I thank my hon. Friend the Member for Dudley North (Ian Austin) for proposing today’s debate on cystic fibrosis and on the future of the drug therapy. I thank the cystic fibrosis team at York hospital. I have met with them and discussed at length their innovative service, which is at the cutting edge of provision for those with cystic fibrosis and takes on board the need for clinical excellence and the sterile conditions that we have heard about—they work the service around the patient, not the patients around the service. I also thank the people at the Cystic Fibrosis Trust for their time.

I emphasise the points made by the right hon. Member for Chesham and Amersham (Mrs Gillan). Her tireless campaigning was triggered by the inspiration of Archie Hill from her constituency and presses for the need to make progress on the right therapeutic responses for those with Duchenne muscular dystrophy. We would all like to see progress with Translarna.

I want to take a wider view of the therapeutic measures for those who experience cystic fibrosis. I am a physiotherapist by training and have worked for 20 years in the NHS with respiratory and neurological conditions, so I have a real understanding of the people who experience cystic fibrosis. There has been massive change in the management of that condition in my time in practice, in particular in physical therapy. Treatment is now more dynamic in support of individuals—physical treatment, rather than a more static treatment, especially when dealing with mucus clearance and building up lung capacity. That is all about the treatment and management of symptoms, however, similar to the drug regime that accompanies the physical therapy.

We have seen progress, therefore, but today we are debating a step change in our approach to cystic fibrosis. We are trying to provide hope to the 10,000 people who happen to have cystic fibrosis. Looking at a new generation of drugs might provide that hope. Orkambi is a drug that targets abnormal proteins, which will deal with the symptoms. When we look at drug therapy for cystic fibrosis, we should be looking not only at the immediate impact, which so many drugs do, but at the long-term effect. Every instance of a chest infection brings about damage to the lungs, as people have to expectorate continually, and that has long-term implications that can be fatal for some.

It is vital that we look at early intervention, which is what Orkambi is all about—about bringing a step change in the treatment process for those with cystic fibrosis. By targeting the proteins we have the opportunity to ensure that the cells in the lungs are healthy, which will produce longevity among patients. It is hoped that the new drug will bring improvement to about 50% of people with cystic fibrosis, which in itself will be a seismic change in the outcomes for them. It will have a profound impact.

I encourage the Government not to be nervous about cost, because costs for someone with cystic fibrosis are already high and cannot be underestimated. I will focus on existing costs, such as the cost of frequent visits to hospital, including the frequent use of intravenous drugs. A large proportion of people are on IV drugs for approximately one month a year, which is costly. People also have to be in sterile conditions, because the risk of further infection is incredibly high. Ongoing therapeutic intervention with drugs or physiotherapy has significant bearing on costs. There are also costs to do with managing a high-calorie but healthy diet.

Another expense is the drugs. Cystic fibrosis is not on the list of diseases for which people get free medication. Will the Minister look at that? When the list was drawn up, people with cystic fibrosis were not living into adulthood, so we should re-examine it. There are the costs of having lung transplants, if people require one, and any drugs that prevent future lung transplants have to be a positive, despite the risks, because people will be brought long-term hope.

There is the cost to an individual of education, which for many will have a disturbed pattern—in and out of school—and the impact on long-term employment opportunities. Even if in work, many people find it difficult to hold down a job, because the nature of the disease often takes them out of the workplace and they have to organise and balance their day with fitting in physio and the demands of drug therapy and diet.

Finally, there is the cost of care. Rarely is only one person involved in care for any of the diseases that we are talking about—a network of care is put around an individual with such a disease. Moving to a precision, early-intervention drug, therefore, is a way to bring in resource management, which can be positive not only for the individual, but for the NHS as a whole.

The result of what is being called for today would be positive economically and for people’s lives. In my short contribution, I want to ask the Minister to address the timeline for progress. There is obviously discussion in Europe, such as on the European regulations for Orkambi, and we want to see the timeline tightened up, so that people can have real hope in the new year that they will get access to the drug, because each time someone has a chest infection it has an impact on their long-term future. Time is not something that so many have, so my plea is for progress on securing access to the drug for those with cystic fibrosis.

It is a pleasure to serve under your chairmanship, Sir Edward. It is also nice to see the Minister in his place again—whatever the debate might be, there are few for which the Minister and I are not in the same room at the same time.

I thank the hon. Member for Dudley North (Ian Austin) for bringing this important issue to Westminster Hall. It affects my constituents and I am here to speak on their behalf—this is the place for us to do that as elected representatives. As he mentioned in his introduction, in Northern Ireland we have had some good news, with money set aside for rare diseases. Any approach to such diseases needs to be innovative and to take into account all those who contribute, be they academics, researchers or hard-working charities who provide support for those suffering from cystic fibrosis and their families.

I also commend the right hon. Member for Chesham and Amersham (Mrs Gillan) and the hon. Member for York Central (Rachael Maskell) who have spoken. They are doughty campaigners on behalf of those who have Duchenne and on many other issues. It is good to see them in their places and making valuable contributions.

We are surely duty-bound to support and fund those who fight for the sufferers and those developing new treatments. The debate is very much about how we develop new treatments and move forward.

I also congratulate the hon. Member for Dudley North (Ian Austin) on bringing the debate. Does my hon. Friend agree that pharmaceutical companies need to be sent a message that their work in research is not about large profits; it is about curing rare diseases? We saw that difficulty whenever we approached pharmaceutical companies on meningitis B: some companies held out for large profits at the expense of people who were suffering.

I thank my hon. Friend for focusing on the pharmaceutical companies. They can do a great deal and there is also a role for Government and the NICE guidelines, which direct the direction in which pharmaceutical companies will proceed. The companies are driven not always by profit or margins; criteria also indicate to them what to do.

We should be ever mindful that people are suffering through no fault of their own, so we need to help them move forward. It is good to see facts and figures that show that, on average, a child born in the 21st century with cystic fibrosis will live for more than 50 years. There have been tremendous advances. The innovation and hard work done by charities and researchers is too often forgotten, but it has brought about real results, with new precision medicines treating not just the symptoms, but the underlying cause of the condition. We must go further in that direction. To be fair, cystic fibrosis is one condition that we are probably treating rather than solving at the moment, but we need to see a future where everyone with cystic fibrosis can live a life unlimited, which the facts show is more achievable today than ever before.

Unfortunately, precision medicines are expensive and, as my hon. Friend the Member for Upper Bann (David Simpson) said, it is difficult to predict the cost-effectiveness of new treatments. However, we need to get those treatments and try them out to move forwards. I understand that the Government are considering how we can speed up access to innovative treatments, which I think comes under the NICE guidelines. Will the Minister respond to that in his speech? There are proposals to approve new drugs provisionally while using real-world data to assess their benefits. I welcome that and look forward to seeing more of it.

May I put on record my thanks to the Northern Ireland Rare Disease Partnership under the chairmanship of Christine Collins, who happens to be one of my constituents? We have worked together over the years on this matter. Indeed, in the previous Parliament we spoke to the Under-Secretary of State for Health, the hon. Member for Battersea (Jane Ellison), in a private meeting. She was supportive and allowed us to make positive progress. As everyone knows, health in Northern Ireland is a devolved matter. The Minister there, my colleague, Simon Hamilton, has set aside about £3 million for the partnership, which shows there are positive approaches in Northern Ireland and a positive way forward. Perhaps that could be emulated across the whole of the United Kingdom of Great Britain and Northern Ireland.

It is estimated that one in 2,500 babies in the UK will be born with cystic fibrosis and there are more than 9,000 living with the condition. The facts are stark. It most commonly affects white people of northern European descent—it is much less common in other ethnic groups. Those are the facts, which in my constituency means that we are looking at virtually the whole populace. Other constituencies will have similar demographics, so it is concerning to hear that, but it is encouraging that research has advanced so much that we can pinpoint such factors so that we know where problems could arise.

Babies are screened for cystic fibrosis at birth using a heel-prick test as part of the NHS’s newborn screening programme. The NHS and Ministers responsible are taking correct steps to diagnose such conditions at an early stage. Treatment for cystic fibrosis is not curative, but it seeks to manage symptoms. Medications including steroids, antibiotics, insulin and bronchodilator inhalers are often used. Nutritional advice and physiotherapy for airway clearance are commonly part of management.

Cystic fibrosis patients may also be suitable for lung transplants. NICE provides a number of guidelines on specific treatments for cystic fibrosis, which it is currently updating. They are due to be published in 2017. On organ transplants, I believe that we should all be considered to be donors unless we say otherwise. The Welsh Assembly has taken steps to bring in that in Wales and such legislation is pending in other regions of the United Kingdom as well, but whenever we see stories about those who are managing but no more and for whom a lung transplant would be the beginning of a new life, perhaps we should emphasise the organ transplant system and find a method to make progress on that.

The hon. Member for York Central rightly referred to families. We focus on those who have cystic fibrosis, but let us also focus on those who support their loved ones at times of hardship and difficult health symptoms. I will also plead the case for Prader-Willi syndrome. I have a number of constituents who have it, but that is not unique by any means to my consistency; it is seen across Northern Ireland. We do not hear much about this, which is another muscular wasting disease and also an eating disease—it is an obsessive disease.

The right hon. Member for Chesham and Amersham talked about Duchenne muscular dystrophy. I have constituents who suffer from that and I have attended events just across the way with people from across the UK with it. It comes in different levels and types, but, as she said, there have been advances in medication. The Minister may refer to those in his reply, but we also need to focus on how we can help those families.

Recent developments show that innovation is working in advancing treatment of cystic fibrosis. I commend the Department for its work. I will also mention the hard work done by universities in partnership with private business and enterprise to come up with innovative ideas for new drugs. We can never underestimate the importance of what they do. Just as others speak highly of their universities, I do so of Queen’s University Belfast and Ulster University which are bringing forward innovative ideas for advances in medicine and other things. We could work well together with them on this.

I spoke earlier of the hidden or forgotten sector: the voluntary charities, of which there are many. Where would we be without them and their dedicated researchers? Such people often dedicate their lives to helping humanity overcome disease. The Cystic Fibrosis Trust is just one example. It is the largest charity funder of cystic fibrosis research in the UK. Last year it invested more than £3 million in groundbreaking research and it plans to invest a further £3.5 million by the end of this financial year. By adding our support and funding where possible, we can add to the great work being done and make a real life-changing difference for those with cystic fibrosis and their families.

It is a pleasure to serve under your chairmanship, Sir Edward. I applaud the hon. Member for Dudley North (Ian Austin) for bringing this timely debate. If any fact highlights the importance of this, it is that the median survival age is just 28. That really highlights the issue. If that does not focus minds on the need to do something, nothing will. He also touched on quality of life. We must remember that it is not just about statistics and medical reports. It is about the life of not just the sufferer, but the families involved. I am grateful to be able to take part in the debate.

The hon. Gentleman also mentioned issues relating to NICE, its assessments and medicines. I am obviously a Scottish Member, and things are slightly different in Scotland, so I was grateful that Members mentioned the differences. One thing we have is the Scottish Medicines Consortium, which assesses medicines a bit quicker, putting them through the peer-approved clinical system. That is a good practice, which the Minister should perhaps look at. Having said that, we are also still waiting for the assessment of Orkambi, and we hope to have it around April, so there is still a delay in getting things through for everyone.

The right hon. Member for Chesham and Amersham (Mrs Gillan) made some good points. I was interested to hear about muscular dystrophy, which is not an issue I know much about, although the situations people face are obviously very similar. She highlighted the impact on families and the importance for children and young people. When we hear people’s life expectancy, that really highlights just how devastating this issue is.

The hon. Member for York Central (Rachael Maskell) made interesting points about therapeutic measures. Her key message was about providing hope, and I share her view on that. I hope that this Government and all the Governments in the devolved Assemblies take on board the message that we should not be nervous about costs. That message needs to go out from here very strongly.

The hon. Member for Strangford (Jim Shannon) highlighted the different and positive practices in Northern Ireland, which, again, I find interesting. I am sure there are things we can learn from each other’s areas. One positive in Scotland is that the Scottish Government have the UK-leading new medicines fund, which, in May, more than doubled the support it provides, from £40 million to £90 million. That will affect all rare diseases, including cystic fibrosis. There are therefore things we can do, and there is good practice we can demonstrate and lead the way on.

Another thing we did in Scotland was to abolish prescription charges. Before we did that, two thirds of all paid-for prescriptions were for long-term conditions. That was another financial impact on the families we are talking about, who already have enough difficulties.

With those comments, I look forward to hearing the Minister’s view. I hope we have sent a strong message to not only the Government here, but the Governments in our devolved Assemblies.

It is a pleasure to be back, having spent four and a half weeks in cold, wet Oldham, running the Labour party by-election campaign. I pay tribute to my fellow shadow Ministers for standing in for me in numerous Westminster Hall debates. It is good to be back, and it is good to see you in the Chair, Sir Edward.

I pay tribute to my hon. Friend the Member for Dudley North (Ian Austin) for making sure that this important debate could take place. He is right that we need to make sure that access to pharmaceuticals is one of the most important policy areas. With the results of the accelerated access review coming out in the new year, the effectiveness of NICE is very much on the agenda.

I also pay tribute to the right hon. Member for Chesham and Amersham (Mrs Gillan), who spoke powerfully about not only Archie, but Duchenne muscular dystrophy, which is a terrible disease, and she is right that we need to do much more to make sure drugs are available to treat it. I hope the case she made for Translarna will not fall on deaf ears with Ministers, because such drugs can make a big difference to the quality of life of children such as Archie. The right hon. Lady has put that case very powerfully in her question to the Prime Minister, and in her contribution today.

In June 2009, the previous Labour Government adopted the European Council recommendation on action in the field of rare diseases, which recommended that member states should establish and implement plans and strategies for rare diseases. Following on from work set out before the 2010 election, the coalition Government published the UK strategy in November 2013. 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, and when the Minister concludes the debate, I am sure he will give us more detail about how he sees the points made by my hon. Friends the Members for Dudley North and for York Central (Rachael Maskell), the hon. Member for Strangford (Jim Shannon) and the hon. Member for Linlithgow and East Falkirk (Martyn Day), who leads for the SNP on these matters, and the right hon. Member for Chesham and Amersham. Rare diseases are a crucial part of the five-year forward view, and given that the UK leads in life sciences, there is no reason why we cannot start to push the boundaries on what is achievable in respect of the drugs available for rare diseases.

The problem is that, although each of the publications I mentioned set out some laudable intentions, the actions arising from those publications have been baby steps in comparison with what we actually need. Changes resulting from the Health and Social Care Act 2012 have left patients and professionals to navigate a labyrinth when accessing medicines that, in many cases, have already been approved or have received licences. That really should not be happening.

Before the last election, the Opposition said unambiguously that we would reform NICE from top to bottom to remove the requirement to enforce competition rules and to ensure that access to medicine was decided on the basis of a medical justification, balanced with consideration of how much money we had available. I think we all now agree that NICE needs some reform. The current appraisal system makes decisions based on 24 weeks of clinical trials data, but that understates the efficacy of drugs that provide long-term stabilisation of a condition.

Other Members have spoken of the frustration cystic fibrosis patients have felt at not being able to access new treatments because those will not be approved given the way NICE appraises them. As my hon. Friend the Member for York Central said, the NICE system is not set up to assess precision medicines, and the issue extends well beyond cystic fibrosis to other rare diseases. Members have spoken powerfully about cystic fibrosis, and what we have heard about could be an excellent platform for testing new ways of doing things, and that could, indeed, also be the case with muscular dystrophy. The appraisal system for innovative medicines needs to be overhauled and to be adapted to include personalised medicines. Until the system is prepared to look at the value of new medicines over time, instead of looking for more rapid effects, it will not be suitable for its purpose.

I have some specific questions for the Minister. I am concerned that the highly specialised technologies evaluation programme could limit access to medicines for people with rare diseases. There are widely held concerns that the process, which was introduced after the 2012 Act to appraise medicines for rare diseases, is opaque and that the topic selection process is out of date. Does the Minister have plans to work with NICE to update the selection criteria for the pathway? Am I right that the process does not take into account conditions defined by genetics, biomarkers or differences in clinical presentation? Will he look again at that? Will he, as other hon. Members have asked, meet representatives of Muscular Dystrophy UK and the Cystic Fibrosis Trust to discuss those matters?

I accept that there has been huge investment in life sciences in the UK, but the current system, which encourages investment in technology and does not facilitate patient access to it, is unsustainable and wrong. Without such reform as we have discussed, funding for research on the relevant medicines could dry up and we could lose crucial shots at tackling a lot of rare diseases once and for all. The accelerated access review is a timely opportunity to take a careful look at how people get access to the kinds of medicines that might change their lives. It would be a tragedy if we threw that opportunity away.

I do not doubt for a moment that the Minister is fully behind every Member taking part in the debate in wanting to expedite the availability of the drugs in question, and I am keen to hear more about how the Government plan to do that. I commend Members for speaking so powerfully in a consensual cross-party manner in today’s debate and at other times when we work in Parliament to promote such causes. I hope that the Minister will answer all the questions that have been asked, and offer a glimmer of hope for those people who seek access to such drugs.

It is a pleasure to serve under your chairmanship, Sir Edward. I congratulate the hon. Member for Dudley North (Ian Austin) and other hon. Members from across the House who have spoken. This timely debate has been incredibly powerful—not that there has been much disagreement in it. It has been an opportunity to raise important issues that I am dealing with, and I am grateful to colleagues for acknowledging that.

The debate is particularly timely because I am convenor of a major summit today on accelerated access for faster cures. There is a precision medicine summit in London and the Association of Medical Research Charities has just held its annual conference, at which I exhorted members to come to my table with ideas about how to accelerate novel treatments and give the charities more of a voice. A powerful and helpful debate is going on.

I pay tribute to the work of the Cystic Fibrosis Trust, which is among a number of charities that lead the debate on innovative treatments and medicines. Its leader Ed Owen in particular plays an important role in that; but so do Carly, Lorraine, Michael, Kelly and the other people who have been mentioned. Many of the charities do extraordinary work to articulate the experience of patients who suffer from disease and bring it to the policy table in a powerful way. It is a change in policy making that I am keen to accelerate.

The debate goes to the heart of the challenge and opportunity that precision medicines represent for our system and the landscape of assessment, testing, approvals and reimbursement, as well as the growing role of charities and the patient voice. Those things are passions of mine and I want to discuss why, in the next few months and the years ahead, there will be dramatic progress.

The Government and I wholeheartedly support the cystic fibrosis campaign’s central aim of ensuring that as many people with CF as possible will have access to personalised medicines by 2020. That sets an inspiring and clear goal and I relish the attempt to deliver it. I want to make some remarks about the condition, about what NHS England and the NHS in Scotland and Northern Ireland are doing about treatment today, about the rare diseases and precision medicine landscape, and about the reforms that I am pushing to try to deal with the issues that have been raised.

I have had a career in biomedical research, so it is an extraordinary privilege to have been given my role by the Prime Minister, who has personal experience of the tragic consequences of genetic disorders affecting children. I am delighted to share with the House the fact that my passion to lead in this field, and unleash the power of the NHS and our research expertise in a new landscape for accelerated access, is exceeded only by the Prime Minister’s.

As hon. Members know, cystic fibrosis is the most common life-limiting inherited condition in the UK. It affects about 10,500 people in England—and more, of course, in Scotland, Northern Ireland and Wales—more than half of whom are adults. Cystic fibrosis is one of the UK’s commonest life-threatening inherited diseases. It is caused by a single defective gene. As a result, the internal organs and especially the lungs and digestive system become clogged. That results in chronic infections, inflammation in the lungs and difficulty digesting food.

The number of adults living with CF is gradually increasing over time, because of improvements in diagnosis from newborn screening and new treatments. The condition affects everyone differently—that is an important point—but for many it involves a rigorous daily treatment regime including physiotherapy, oral, nebulised and occasionally intravenous antibiotics, and taking enzyme tablets with food. For those who are very ill with cystic fibrosis and who have very poor lung function, daily life can be a struggle as basic tasks can leave them breathless. Some patients use a wheelchair to get around, and use oxygen to help them breathe.

For patients and their families, managing the condition is extremely challenging. That is made worse by the absence of an effective treatment or cure—or, as several colleagues have explained today, by the tantalising presence of a possible treatment or cure that cannot yet be administered to them or their suffering loved ones. I pay tribute to patients who grapple with the disease day in, day out, and who have done so for years, for their patience as we try to bring new solutions to the table. Current treatments generally target the complications rather than the cause of the condition. Treatments can be broadly classified as nutritional support, relief of airway obstruction, treatment of airway infection and, ultimately, lung transplantation.

What are the Government doing? I want first to talk about what the NHS is doing in England and in Scotland and the other devolved areas, and then to say something about what we are doing more strategically to tackle the new landscape.

Since April 2013 NHS England has been responsible for securing high-quality outcomes for patients with cystic fibrosis as part of its remit to deliver specialised services. Its service specifications for cystic fibrosis—one for adults and one for children—set out what providers must have in place to offer high-quality care and support equity of access to services for patients with cystic fibrosis, wherever they live. The NHS England cystic fibrosis clinical reference group has developed a number of clinical policies for the treatment of patients with cystic fibrosis and it reviews outcomes with the Cystic Fibrosis Trust and with patients and charities.

As we have heard, Scotland, leading within the United Kingdom—and it is not the first time—has launched a dedicated fund worth £40 million this year to give patients greater access to new medicines, as the Scottish Health Secretary, Alex Neil, has announced today. The £40 million new medicines fund expands and replaces the rare conditions medicines fund established in March 2013, giving health boards access to greater resources. In 2013-14 the rare conditions medicines fund supported the cost of 45 different medicines, benefiting more than 200 patients, including ivacaftor for cystic fibrosis as well as other treatments for related rare diseases.

NHS England is investing significant resources into the provision of new medications that work directly on the genes causing cystic fibrosis. Since 2013, it has routinely commissioned ivacaftor or Kalydeco for the treatment of cystic fibrosis in those with a certain gene mutation affecting only 5% of the CF population. Earlier in 2015, that indication was extended to an additional eight mutations for patients aged six years and above. NHS England is considering a policy proposition for extending the use of ivacaftor for the same gene mutations to children aged two to five years. It will consider the evidence base and be included with other therapies requiring investment as part of NHS England’s prioritisation process for specialised services for 2016-17.

Several colleagues raised the matter of Orkambi. Some drugs for cystic fibrosis will be considered by NICE through its technology appraisal process, including Orkambi, which, as many will know, is lumacaftor in combination with ivacaftor. NICE is currently developing technology appraisal guidance on the use of Orkambi for the treatment of patients with cystic fibrosis. It currently expects to issue final guidance in July 2016. NHS England will commission drugs where there is a positive NICE technology appraisal, and I will say something about the changes that we envisage in the landscape in that respect.

NHS England operates a horizon-scanning process to identify new treatments and the cystic fibrosis clinical reference group advises on the development of services for patients and keeps relevant published literature under review. Where NICE is not considering a therapy, NHS England can consider the evidence base and may propose commissioning treatments through its policy development process. I shall say something shortly about changes that we are considering in the way NHS specialist commissioning might embrace the new freedoms in the accelerated access review to accelerate the commissioning of rare disease treatments.

In fact, ivacaftor is something of a mild success story. NHS England commissioned it earlier than might otherwise have been expected, having agreed, in discussion with the company that makes it, a flexible pricing model. We want to see more of that sort of innovation.

I am grateful to the hon. Member for Denton and Reddish (Andrew Gwynne) for giving me some time to answer the various questions asked, which I will try to do in some detail. First, I want to set the scene in terms of why this debate is happening and why this landscape is under such pressure. The truth is that breakthroughs in genomics and informatics—our ability to understand patients’ genetic predisposition to different diseases and to respond to different drugs, as well as the availability of large-scale data sets, including individualised patient treatment histories and anonymised cohort studies—are transforming the traditional pathway for drug R and D, which normally takes years. It now takes roughly 15 years and $2 billion to bring the average drug to patients.

Genomics and informatics, particularly for some of the rare genetic diseases, allow us to take time, cost and risk out of the development pathway in a profound way. That is driving opportunity and challenge in our system; the Prime Minister created this post and put me in it to ensure we respond to that challenge with ambition.

My hon. Friend the Minister is absolutely right to say that the medical landscape is changing hugely at the moment, but does he feel that the wider implications of new medicines are being fully explored by NHS England and NICE? We have heard about the huge consequences of cystic fibrosis for not only the sufferer but their wider family and the NHS. Does he feel that those wider consequences are being fully explored?

My hon. Friend raises an important point. Over the past few decades, the NHS across the UK has played an inspiring role in leading a lot of the breakthroughs in new treatments, but we have become latterly a slower adopter of the very treatments we often helped to discover. That is partly because the pressure of an ageing society and the rising cost for the health system today of just treating existing conditions are extremely challenging. In some areas, that has made innovations appear a cost to the system, when in fact good innovations may come with a cost spike on day one but generally lead to downstream savings in years 2, 3 and 4.

My hon. Friend puts his finger on a profound challenge at the heart of this landscape: in order really to assess the impact of innovative treatments, we need a much better handle on the existing costs, many of which are hidden, that come with a diagnosis. For that reason, I am spearheading work in the Department of Health to drive through a system of per-patient costing, so that we can begin to get a much clearer handle on what a CF diagnosis means on day one for both the patient and the health economy. That will allow NICE and NHS England to develop much more intelligent systems for assessing whether an innovation really represents good value.

Genomics and informatics are changing the landscape; for that reason the Prime Minister has created my post and we have launched a series of initiatives. On genomics, we have launched a groundbreaking £300 million initiative to sequence the genomes from 100,000 NHS patients of cancer and rare diseases. We have also launched 11 genomic medicine centres across the NHS, so that genomics is fundamentally embedded in our health system. On informatics, we have released huge amounts of cohort data to drive research, and we just announced in the comprehensive spending review a major £3.5 billion programme to invest in NHS digital infrastructure to support that.

We have launched precision medicine and cell therapy catapult centres with the Medical Research Council and industry partners to lead in both understanding causal mechanisms of rare diseases and developing and accelerating new treatments. We continue to fund the excellent National Institute for Health Research, for which it is my privilege to be responsible, to the tune of £1 billion a year, and we committed this year in the CSR to fund it throughout this Parliament, at a cost of £5 billion. We have funded the £700 million Francis Crick Institute, and roughly £2 billion of the drugs budget is allocated to new medicines and new treatments in this Parliament.

There is a major commitment, in terms of science and funding, to trying to tackle this issue, but crucially we need policy reforms to ensure that breakthroughs in science can be harnessed for much quicker benefits for patients. That is what the accelerated access review and a number of other initiatives, such as the test bed programme and the vanguards I am running with NHS England, are about—trying to ensure we can change the pathways for getting innovation into our health system for much quicker patient benefit.

I want to say something about the accelerated access review and the specialist commissioning reforms that NHS England is putting in place. I know all Members here take an interest in this subject, so I hope they will be aware that I have launched the independent AAR to ask and answer one big question: what can we better do to harness the extraordinary infrastructure here in the UK in terms of our deep science research base, our NHS-NIHR research base and our NHS daily treatment platform?

The NHS is the fifth biggest organisation in the world, making millions of diagnoses and carrying out millions of treatments every day. Its original founding mission was to be a research organisation, but unless we better capture the data on those interventions, we are still practising, in many cases, blind medicine; we are not harnessing that intelligence enough to inform treatment.

I have asked that the AAR tackles three big questions. First, what can we do to allow the innovators—the developers of new drugs and innovations—quicker access to patients, to reach the all-important moment of proving an innovation works in patients? Secondly, what can we do to harness our leadership in genomics and informatics in order to create a more intelligent system for NICE and NHS England, with more flexibilities, so that they can assess, adopt, approve and reimburse innovations using real-time data about real patients? That will allow us to develop a more flexible set of pathways and adaptive tools with which to embrace this revolution.

When a drug comes to us with a genomic biomarker and we know that it will work for a certain sub-cohort of patients, that profoundly changes the risk dynamic of a traditional pharmaceutical clinical trials programme and should allow us to accelerate adoption for particular patient groups.

Within those considerations, will the Minister also look at international evidence, so that we are looking at not only our own clinical trials but those on a global scale? Clearly, developments are global rather than just national.

The hon. Lady makes an important point. I have been to Washington three times and to Berlin, Paris and Brussels to highlight that while the UK is leading in this field, we need a transatlantic—European and American—agreement on how we move things forward. That is why I am convening and chairing a summit this afternoon with the Washington-based FasterCures campaign, which is a cross-party group on the Hill pushing for innovations in this space. I have been talking to the Commission about the European framework. I want the UK to be the best entry point into the European market, but I also want the European regulatory framework to be consistent and coherent; that is an important point.

The second question I have asked the AAR to look at is: what freedoms, flexibilities and new pathways can we envisage giving NICE and NHS England, particularly in the field of specialist commissioning? For CF, the decision to purchase ivacaftor is a national one, made by an NHS England specialist commissioning unit. I would like that unit to work much more closely with the Department of Health pricing team, so that where we can offer a company faster access to a key patient cohort, data and genomic information, we are able to do a much better deal with the company.

At the moment, we are operating the Translarna and Vimizim programme in the existing landscape. I share colleagues’ frustration, but it is important we go through due process. I do not think anyone wants a world in which Ministers decide what drugs come through on the basis of political pressure, tempting though it may be. I have done everything I can this year to expedite the existing process.

Following the positive news on Vimizim, I am hopeful about Translarna—a similar drug. NICE has been consulting on the process, and I believe the company has been engaging with NICE on pricing. I am hopeful that there will be a decision in the next few months to parallel the one on Vimizim, but that decision is not in my gift: it is up to NICE, which is rightly working on the basis of the very best clinical evidence.

I had better crack on; I will come to the questions that my right hon. Friend the Member for Chesham and Amersham (Mrs Gillan) asked later, if I may.

Hon. Members have raised a number of questions and I want to deal with them all. The hon. Member for Dudley North asked about timings for the accelerated access review. We have had an interim report. I have asked for final recommendations in the spring—in March or April—and also that the review considers whether the process should go on. I want recommendations that we can implement quickly, but equally these discussions are complex and we may well need to go on to look at other bits of the landscape. I would be delighted to meet the Cystic Fibrosis Trust—in fact, that is already arranged; we are meeting this afternoon at the summit that I have organised.

The hon. Gentleman also asked about safeguards, which is a very important point. Although all of us share a recognition of the need to accelerate access, nothing in what is happening must in any way undermine patient trust and confidence in safety and protections. That is an important balance to strike. Nothing in what we are doing in any way looks at changing the legal basis in terms of negligence, consent or the clinical trials framework. The issue is about ensuring that our systems have the flexibilities to embrace the very latest science, and particularly, in this case, genomic biomarkers.

The hon. Gentleman asked about amending the NICE appraisal process to weight wider societal costs. At this point, the review is not specifically looking at the internal mechanics of NICE’s current high-technology appraisal process, but we are looking at giving it, with its new flexibilities and freedoms, a suite of different types of innovation that might come through. We are particularly looking at where that can be ring-fenced and targeted at particular patient groups.

The hon. Gentleman asked about a ring-fenced fund. As I said, we are looking at the allocation that we have had from the Treasury, which is about £4 billion extra on drugs in this Parliament, £2 billion of which is more or less the existing demand driven by demographic change. There is about a £2 billion allocation in there for new medicines. The difficulty is that the drugs that we might consider now to be most worthy of ring-fencing and accelerating may not be the ones that in five years’ time, on the basis of the clinical evidence, we look back on and say, “Why did we not accelerate that?”

We want to make sure, through the AAR, that we are putting in place a system that gives us the flexibilities to pull through those drugs that have the most transformational effect. But let me be clear: we are looking at wanting to build in, over the next few years, a wider understanding of the real costs to our health economies—local and national—of different forms of disease. That is why the Secretary of State and I are leading on per-patient costing so that, in due course, we can develop a more intelligent system to reflect that.

The hon. Gentleman asked about the NIHR and specialist muscle centres. This debate covers a number of different disease areas, and it is a tribute to the NIHR’s research network that more and more charities are now wanting to build centres of excellence. In the forthcoming NIHR five-year funding cycle, looking at the biomedical research centres and the biomedical research units, I am keen to make sure we consider where we can bring funding in from charities to complement that core research network.

Finally, the hon. Gentleman asked about the accelerated access review and the powers that we are looking at for NICE and NHS England. I do not want to pre-empt the findings of that independent review, but I have asked that the review looks precisely at how we can make it easier for NICE and NHS England to work more closely together. Specialist commissioning would be an obvious place to start to share those data and look at how we can get a better deal for everybody—for patients, the system and the economy.

My right hon. Friend the Member for Chesham and Amersham asked about Translarna and Vimizim and how quickly we may be able to get good news for Archie. I pay tribute to my right hon. Friend; she has been a very doughty campaigner on this matter during the last year. I share her frustration that in the existing system, due process has to be gone through and that, although we have expedited this as much as we can, it has taken a long time. I pay tribute, as she has, to Archie. He, like so many of these patients, is an inspiring example of the very best of this sector and of this country. They are people who have the most reason to complain, but tend to be the least likely to and the most inspiring, given their generosity about the system and their demand that we take their suffering and use it to make sure that others do not have to suffer.

I have touched on the timetable. I am very hopeful that we should get a decision from NICE on the basis of the secondary consultation early in the new year.

I thank the Minister for the way in which he is still pursuing this matter on behalf of my constituent and the other boys. However, does he share my frustration? I know we have to go through due process, but why does due process have to take so long? Every day matters to these children and to their quality of life. I cannot impress enough on the Minister, NICE and anybody else watching this debate, that due process must be executed in a more timely fashion. This is nothing short of torture for these boys and these families. I know that the Minister has tried very hard, but I just hope that the people at NICE will be listening to this. I appeal to them directly through him to make a positive decision on this before Christmas; it would be the best Christmas present that these boys and their families could have.

My right hon. Friend makes her point as powerfully as ever. I shall not add to it; it has been put on the record very clearly.

My right hon. Friend asked about contact with the company. It is not for Ministers to get actively involved—much as, at times, I would like to—in negotiating these deals, but I have made contact with the company, both on Vimizim and Translarna, to urge it to be as flexible as it can in discussions. I can only say that I am hopeful that it will have been able to reach a point where NICE feels able to make a recommendation.

Part of the reason why due process is important is that when NICE makes a recommendation, NHS England is bound in law to provide the drug in perpetuity, so it is a major cost undertaking. In some cases, these drugs cost £200,000 or £300,000 a year, so it is a commitment of several hundred million pounds from NHS England. Other patients would say, “We must make sure that when you make a decision like that, it is done properly.” However, I share my right hon. Friend’s frustration that a lot of these breakthroughs scientifically mean that we ought to be able to speed things up.

My right hon. Friend asked whether the Prime Minister is holding my feet to the fire. She need not worry; I am as passionate about this as ever and very impatient to make sure that the AAR is landed with some good recommendations.

My right hon. Friend made an excellent point about NIHR staffing. I am working with the chief medical officer and the NIHR on that at the moment. A number of our clinical research facilities could, with a few more staff, turn over more and do more trials work. There is an opportunity for us to get more people internationally to enrol in NIHR training—in clinical trials and translational research training—which would give us more capacity and allow us to move things along faster.

The hon. Member for York Central (Rachael Maskell) raised an important point about cost. I have touched on the work that we are doing on per-patient costing to try and make sure that we develop a system that more intelligently captures the real cost of disease.

I am grateful to the hon. Member for Denton and Reddish, the Opposition spokesman, for his comments. I congratulate him on the by-election victory. He asked about NICE reform, which I have touched on, through the AAR. We do not want to interfere with or undermine NICE’s independence and their “gold standard” reputation, but we want to create a place in which the accelerated access review gives them the freedoms that they are, indeed, helping to shape.

In conclusion, this debate has highlighted not only the challenges from the rising costs of new drug discovery—£200,000 to £400,000 a year for patients in the rare disease space—and the pressure on the one-size-fits-all model of assessment, but the opportunities for us to unleash our leadership in genomics and informatics to create a new landscape. That is why this week, the Association of Medical Research Charities conference and my summit this afternoon, and the accelerated access review work is creating momentum for a new landscape for accelerated pathways for patient-led innovation.

I think we will look back in two or three years at this as a crucial turning point at which the system that was set up to assess a very one-size-fits-all, 20th-century model was rapidly adapted, creating new opportunities for patient-led innovations and charities such as the CF Trust to bring through innovations that benefit their patients more quickly.

If I may, Sir Edward. I thank you for chairing this debate. I thank the Minister and the Opposition spokesman for what they have said. It was really interesting to listen to the Minister and my hon. Friend the Member for York Central (Rachael Maskell) bringing to bear the deep expertise that they have gained from their careers before coming into Parliament. The right hon. Member for Chesham and Amersham (Mrs Gillan) spoke really movingly, and incredibly passionately and powerfully, about Archie Hill.

Most of all, I want to thank the people at the CF Trust and my constituent, Carly Jeavons, for raising this issue with me. I think this debate shows exactly how Parliament and politics should be working—with our constituents raising issues with us, us coming here to speak up on their behalf, and the Minister responding to their concerns—so I am very grateful indeed for that.

Question put and agreed to.


That this House has considered access to medicines for people with cystic fibrosis and other rare diseases.