I beg to move,
That this House has considered the implications of the Accelerated Access Review for cystic fibrosis and other conditions.
It is a pleasure to serve under your chairmanship, Sir Alan. I start by thanking three Dudley residents—Carly Jeavons, Samantha Carrier, and Samantha’s fiancé Rob Evans—for contacting me about accessing new treatments, and for what they have taught me about cystic fibrosis. I also thank Ed Owen, the brilliant former chief executive of the Cystic Fibrosis Trust, Darren O’Keefe and all of the staff at the trust for all their help and advice in organising the debate. Finally, I thank all of the right hon. and hon. Members who have taken the trouble to come here today to speak up on behalf of their constituents who have cystic fibrosis and other long-term conditions.
Just over a year ago, I was contacted by Carly about her work with the Cystic Fibrosis Trust to push for new treatments, such as Orkambi, to be offered on the NHS. She has continued to campaign, and I had the pleasure of joining her, her father Robert and her son Corey to deliver a 15,000-name petition to Downing Street earlier this year. Carly said to me:
“Before, I was always exhausted, I couldn’t work the hours I was contracted to and I had a little boy, Corey, to look after. I couldn’t do everything I needed to do and keep on top of my health, but this drug has given me some control back. I can now do everyday things and walk to the park with my five-year-old, which I could never do before. I personally feel like I have got better and better the longer I have been on it. I have a new way of living.”
Thanks to a clinical trial of Orkambi, Carly now needs to visit a cystic fibrosis clinic less than half as frequently as she used to. That allows her to carry on working, to go on holiday with her family and to do the things that the rest of us take for granted. She continues to benefit from Orkambi, but only thanks to a compassionate use scheme offered by the drugs manufacturer, Vertex. She and other users of Orkambi need the certainty that they will be able to benefit from the drug well into the future with NHS support, which is why we are here today.
I also thank Samantha Carrier, another Dudley resident, who is campaigning to raise awareness and raise funding after her young daughter, Daisy, was diagnosed with cystic fibrosis. Samantha has seen the difference that drugs such as Orkambi can make, and she wants her own daughter to have access to them as soon as possible, so that she can live as full a life as possible. She has told me about the hours of care and support that her daughter needs every day—which makes work so much more difficult for many parents of children with cystic fibrosis.
Samantha said to me:
“I am not ashamed to say I didn’t know how to cope with it all. But one day you wake up and you realise ‘This is it now’. All we can do as a family is try to do our best by her and give her the best life we can.”
I have been very moved by Carly, Samantha and Rob’s determination for something positive to come out of these diagnoses. I think their fundraising and campaigning for the Cystic Fibrosis Trust is nothing short of inspirational.
Personalised medicines can transform life for people with cystic fibrosis and a range of diseases, including muscular dystrophy and Alzheimer’s, but without a process for appraising these medicines that is fit for purpose, patients are unable to access these innovative medicines. That is why we called for today’s 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 that cystic fibrosis causes to the lungs means that many people eventually need a lung transplant. There is no cure for cystic fibrosis but many treatments are available to manage it, including physiotherapy, exercise and nutrition. The median survival age is just 28. What people like Carly, Sam and countless other families across the country need to hear today is the hope that a way forward can be found that will bring an end to an agonising and unnecessary wait that has gone on for well over a year now.
Orkambi was licensed in November 2015. It is a first-of-a-kind personalised medicine that treats the cause, not just the symptoms, of those with a particular mutation of the genetic defect that causes cystic fibrosis. Around half of the people with cystic fibrosis in England stand to benefit. Personalised medicines offer a revolution in cystic fibrosis care. People in countries such as France, Germany and America who have been on the drug for some time are beginning to report total transformations in their health, with some improving enough to come off the lung transplant waiting list—on which one in three people with cystic fibrosis die. Clinicians in England are desperate to prescribe Orkambi. Those who are prescribing it, on compassionate grounds, report that the drug, which halves hospital admissions—that lasts for months—for people with cystic fibrosis, could help ease the severe and worsening shortage of beds on cystic fibrosis wards.
I stood in this Chamber a year ago to raise concerns that the appraisal process for Orkambi was not suited to an innovation of this kind. The existing National Institute for Health and Care Excellence appraisal system makes decisions on the efficacy of a drug based on 24 weeks of clinical trials data, but fails to take into account the long-term benefit 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 benefits for society of these medicines, such as the way that they can help sufferers or their carers get into work. In short, the existing system cannot provide an accurate assessment of new treatments that offer long-term, preventive stabilisation of cystic fibrosis.
I highlighted that, due to those concerns, the Cystic Fibrosis Trust was proposing an innovative solution under which real-world, long-term data could be gathered using the UK cystic fibrosis registry. The registry already provides real-world data to health commissioners and pharmaceutical companies, so that they can monitor the efficacy of treatments.
My hon. Friend is making an incredibly important point. I congratulate him on securing this debate, which will give hope to the many people out there who suffer with cystic fibrosis. Is he aware of new 96-week data that have recently been published that show that Orkambi slows decline in lung function by around 42%? Those data were not available to NICE when it made its appraisal. Do those data alone not make the case for a further accelerated review process on this absolutely compelling?
My right hon. Friend is completely right; he raises a point I will make shortly. It is good that he is here to support people with cystic fibrosis in his constituency, and to bring his knowledge and experience of the national health service to bear in the debate.
The Cystic Fibrosis Trust’s proposal would provide foundations for a managed access scheme for the drug. That was in line with the interim findings of the accelerated access review, which recommended the merits of such an approach and referred to the UK cystic fibrosis registry as an exemplar. I will say more about the accelerated access review in a few moments.
As expected, seven months later NICE referred to a lack of long-term data in rejecting Orkambi for use in the NHS. That was despite Orkambi’s being proven to halve hospitalisations and NICE’s recognising it as a
“valuable new therapy for managing cystic fibrosis”
with significant clinical benefits, as well as
“wider benefits to society for people with cystic fibrosis and carers of people with cystic fibrosis.”
I congratulate the hon. Gentleman on securing this important debate. He correctly points out that this is not just about the way in which Orkambi improves quality of life, which I know is extremely important, but about cutting hospital admissions. That has to be taken into account when we look at the wider cost implications of the drug. What we need is time for the drug to be given the chance to prove its worth.
The hon. Gentleman is completely right to say that Orkambi could reduce hospital admissions, and could shorten the amount of time people spend in hospital when they have been admitted.
In its statement, NICE referred directly to the trust’s proposal as a potential solution to the shortage of long-term data. With the NICE process exhausted and seven months wasted, we hoped that the way would be clear for direct negotiations between the drug manufacturer Vertex and NHS England, which would allow for a speedy resolution to the situation. However, Department of Health officials then demanded that the drug be put through a rapid review process, which, at 16 weeks, is anything but.That process is based on exactly the same criteria that had just seen Orkambi denied to those who need it. Vertex has declined to enter the process, because of the certainty that it will come to nothing.
New data published in October at the North American cystic fibrosis conference, which my right hon. Friend the Member for Leigh (Andy Burnham) mentioned, are based on 96 weeks of trials and show that Orkambi slows the decline in lung health by up to 42%. That is comparable with the 47% slow in decline caused by the transformational treatment Kalydeco, which is widely available in the UK for a less common mutation of cystic fibrosis. Those data were unavailable to NICE but clearly illustrate that drugs such as Orkambi need the chance to prove their worth in the long term. That also underlines the fact that we now have a situation where people with cystic fibrosis face discrimination by genotype, because they are being denied the same level of treatment that people with a different genetic mutation of cystic fibrosis receive.
Twelve months after licensing, negotiations are at a standstill. I understand that Vertex is keen to offer a substantial discount, but for commercial reasons would need to do so confidentially. It would like to take up the trust’s offer of monitoring the effectiveness of Orkambi for a trial period. That could build on the American data and allow NHS England to conduct final negotiations based on an accurate reflection of the drug’s effectiveness.
I would like to thank the hon. Gentleman for securing such an important debate. One of the beauties of cystic fibrosis data is that they capture 99% of all people with the disease, so could truly be used as an exemplar. The accelerated access review calls for accurate monitoring via data, and this offers an ideal chance to do that.
The hon. Lady is completely correct. It is good that she is here in the Chamber, making these important points.
Vertex is also keen to explore flexible reimbursement schemes, which would allow the NHS to manage the overall budget impact of the treatment. However, the inflexible current system insists that any offer has to be made public, rejects the trust’s solutions and offers no scope for flexible reimbursement schemes. That brings me to the accelerated access review, which was commissioned to speed up access to innovative new drugs and treatments such as Orkambi. The review was finally published in October, after a long delay, and recommends that NICE reviews its processes. It calls directly for the current system to change, to include more emphasis on the confidential commercial arrangements, flexible reimbursement arrangements and collection of real-world data that I and other Members have referred to. Those recommendations could be the key to reaching a deal that delivers Orkambi to those desperate to receive it.
When the review was commissioned last year by the Minister’s predecessor, the hon. Member for Mid Norfolk (George Freeman), he spoke of how accelerating the uptake of transformational technologies in the 21st century would attract investment in research and innovation to help us earn the prosperity we need as an advanced economy. When the review was published in October, NHS England’s chief executive, Simon Stevens, said that creating headroom for faster and wider uptake of important new patient treatments would create opportunities for the UK’s globally successful life sciences sector. The failure to deliver Orkambi undermines that vision.
We have a rigid and inflexible system, and warnings that it is not fit for purpose have been ignored throughout the process. Instead of embracing the opportunity for an innovative solution, we have been offered further negotiations based on criteria that have already failed once. That is a waste of time and taxpayers’ money and sends completely the wrong signal to a global life sciences industry currently questioning future investments here in the UK. Hugh Taylor, the review’s chair, set out the need for commitment and collaboration across Government, the NHS and the life sciences industry to make the review’s proposals a reality.
The review sets out criteria for transformational treatments that should be fast-tracked for access. Orkambi meets those criteria. It presents the perfect opportunity to put many of the review’s proposals to the test, to illustrate the commitment and collaboration needed and to demonstrate how we can come together and adapt in the light of new information. It is predicted that 95% of people with cystic fibrosis could benefit from a personalised medicine within five years. Coming up with a solution for Orkambi—one that makes sense to the NHS as well as reflecting the investment that goes into these treatments—will give us a genuine opportunity to beat this condition.
I am sure people will benefit from the review’s proposals in the years to come, but that must not be at the cost of Orkambi, which is available now. Many people with cystic fibrosis, as well as their families and carers, such as my constituents Carly Jeavons and Samantha Carrier, are watching this debate. Many of them are forced to spend weeks and months of each year in hospital, and most of all they want a chance to be able to do the everyday things we all take for granted, such as raising a family, planning a holiday or breathing without struggling. They have already endured needless delays, and as time goes on those delays present an obstacle to investment in future treatments to beat cystic fibrosis. That is not the vision set out by the accelerated access review.
Muscular Dystrophy UK is calling, among other things, for ring-fenced, protected funding for rare diseases. That was not included in the review to which my hon. Friend refers. Does he feel that that possibility should at least be considered as a way forward at some point?
That is a really good point, and I am pleased that my hon. Friend raised it. I am sure the Minister will want to respond to that.
Tragically, we have to face the fact that many people are dying now. They do not have time to wait for the Government to respond to the review or for NICE to enter a lengthy consultation on its processes. They want to see the Government get on with exploring how Orkambi can reach those who need it without delay. If the Government create the conditions for constructive negotiations, the manufacturers will play their part, just as the Government themselves need to be flexible in order to deliver transformational treatments such as Orkambi.
I would like to ask the Minister the following questions. Does he think it is right that people in this country are considering moving to France or Germany in order to save their children’s lives by giving them Orkambi, which is now proven to halt the progression of their children’s decline? What does that say about a Britain trying to project its place as being at the cutting edge of the life sciences sector? Will the Minister provide assurances to people watching today that the Government are listening, and that everything possible will be done to explore progressing the negotiations on Orkambi in 2017? Will he reassure them that we are capable of finding a solution next year that will bring an end to this cruel and unnecessary wait?
Will the Minister seek guidance from Government, NICE and NHS England on how the recommendations in the accelerated access review can be used to break the deadlock in negotiations? Will he meet Vertex and the Cystic Fibrosis Trust to discuss that? Samantha Carrier points out that in the 1970s, the life expectancy of cystic fibrosis sufferers was only five years old. Thankfully, that has increased greatly, but the rules for free prescriptions have not moved. When people become 18, they have to pay for their medication, despite the fact that they need these drugs to stay alive. Will the Minister look at that issue?
This is exactly how Parliament and politics in our country should work. It is our job to listen to our constituents and come here to stand up and speak out on their behalf. People like Carly Jeavons struggle to work or spend time with their family and do other things that the rest of us take for granted because they have to undergo hours and hours of treatment. New treatments have helped Carly, but others are missing out on these new drugs at the moment. People like Sam and Rob are having to come to terms with what this condition means for their newborn child, at the same time as having to care for her. All three of them—Sam, Rob and Carly—are devoting hours to raising funds or campaigning for better treatments for people with cystic fibrosis. They are an inspiration to us all; will the Minister meet Carly Jeavons, Samantha Carrier and Rob Evans and listen to them directly?
Six Members have made written requests to speak. Our plan, under the guidelines, is to bring in the Front-Bench spokespeople at 10.30 am, so we have little time left. If speakers and anyone making interventions are very succinct, we will get in as many Members as possible in this important debate.
The accelerated access review is important because it is designed
“to speed up and simplify the process for getting the most promising new treatments and diagnostics safely…to patients.”
That is good news for all Members of Parliament who have in their constituencies people who need access to innovative treatments. Sir John Bell states that the review
“addresses one of the most important issues the National Health Service is confronting; how best to access innovation for the benefit of patients and to improve health care efficiency.”
It should therefore be welcomed and receive support from both the Government and the NHS.
The report has the dual intentions of improving NHS productivity through better use of technology and of promoting the UK as a destination for life sciences. It is clear about the areas that need to be addressed: horizon scanning, data collection, regulatory decision making, clinical and cost-effectiveness assessment and commercial decision and uptake support.
I shall focus briefly on the data collection element of the report because that is what will enable treatments to come forward and help patients. The accelerated access review sets out a mechanism for collecting data on “strategically important medical technologies”. There is a clear need to collect data on technologies and their impact on the healthcare system. The review suggests that one approach should be a “commissioning through evaluation” system, whereby
“complex medical technologies or diagnostic products that significantly change clinical pathways are rolled out in a number of specialist providers who are well-placed to collect impact data and build expertise around pathway change. Following this period, should the technology prove its value after assessment by NICE, it should enter routine commissioning and benefit from supported uptake”.
In a recent debate that I secured on diabetes, I referred to commissioning through evaluation because I fully support the intent of that objective and believe that collecting data in that manner is an effective means of developing real-world data to support the uptake and use of modern treatments across the NHS. That type of evidence development is currently under way in the NHS, and I would like to look at one current commissioning through evaluation programme, which has been in operation since 2013. The programme launched for several technologies, covering a range of conditions. It included procedures to prevent strokes, improve the mobility of children with cerebral palsy, help patients with heart failure and improve radiotherapy for lung and liver cancer. I recognise that this debate is about cystic fibrosis in particular, but I am trying to make the point that as we collect data and bring forward the treatments, we need to ensure that they get to the people who most need them, including those whom we are talking about today.
The programme to which I have referred was structured in two phases. First was the evidence development phase, in which patients would receive the treatments and data would be collected. Second was the evidence assessment phase, in which data would be analysed and a routine commissioning policy developed. We have now reached the point in the process at which the number of procedures originally commissioned has been reached and patients will no longer be given the procedures until a formal commissioning decision has been made.
However, in answer to a parliamentary question in July, the Department of Health said it would take between one and two years to carry out the analysis. Recently, NHS England has stated that formal commissioning policies will not be in place until 2019. Those patients who would benefit from the procedures face the prospect of a two-year wait.
If we focus on just one procedure, we can see the impact that that will have on patients. Selective dorsal rhizotomy is a procedure that supports children with cerebral palsy to have increased mobility in later life. There is a narrow window in a child’s development in which they can receive the treatment. A two-year gap in commissioning will mean that some children never benefit from the procedure.
This debate was initiated by the hon. Member for Dudley North (Ian Austin), whom I thank for giving me the opportunity to speak. Its title on the Order Paper is “Implications of the Accelerated Access Review for cystic fibrosis and other conditions”. I have referred to other conditions, but I want to finish my speech by reading out a letter from a constituent, Christine Edwards, relating to cystic fibrosis. I need add nothing to what she writes:
“Dear Mr Thomas…My niece’s boyfriend, Taylor, has cystic fibrosis. He is a lovely young man and I think it is tragic that his life expectancy is so short. At the moment he is doing pretty well and his health is strong enough to support him going off to University…he did so this year.
The reality is though he can expect his health to decline and with it, his quality of life. Drugs like Orkambi offer such tremendous hope as slowing lung health decline not only offers him the potential to increase his life span directly but also allow him more opportunity to benefit from future treatment development.”
Christine Edwards also writes:
“The 2,700 people with cystic fibrosis in England desperate to access Orkambi do not have the time to wait for the development of a Strategic Commercial Unit to consider a wide range of commercial arrangements. Nor do they have the time to wait for NICE and NHS England to consult on their processes.”
That letter sums up why the Government must intervene and accelerate access to these transforming medical treatments. As Mrs Edwards states, patients cannot wait.
I therefore call on my hon. Friend the Minister to intervene and ask NHS England to give patients access to these innovations by ensuring a rapid and transparent decision-making process for all the innovative treatments currently undergoing commissioning through evaluation. That process should be supported by examining all available evidence and delaying while a small sample of data is analysed. I also call on the Minister to ensure patient access throughout the assessment phase by continuing to fund the procedures until a routine commissioning policy is in place, and to look at the operation of the system in the future and ensure that the design of any programme delivers continued patient access from the start of the programme through to a routine commissioning policy being in place. Finally, I call on the Minister—this is extremely important and will help large numbers of patients with cystic fibrosis and other conditions—to support wider stakeholder input into the system from those who have experience. That would include working with patient groups and industry representatives and would ensure that the NHS had the most accurate information.
It is a pleasure to serve under your chairmanship, Sir Alan. I thank my hon. Friend the Member for Dudley North (Ian Austin) for securing this very important debate.
The recommendations in the accelerated access review have been a cause of hope for many families throughout the UK. I welcome those recommendations and hope that the Minister today will reiterate the Government’s commitment to their implementation. I hope that he will also give consideration to a number of possible areas of clarification that I and, I am sure, other hon. Members will raise. However, the priority is to secure the most advanced and best medicine and technology for patients much more quickly than at present. Early access to new drugs can enhance and extend lives. It is vital not only that the UK keeps pace with other countries in approving new treatments, but that there is a consistent standard within the UK. I recognise that today’s debate focuses on NHS England, but I hope that a strong precedent is set for patients in Northern Ireland.
A few days ago, I received a letter from a constituent who feels that there is now hope. She has a four-year-old child with cystic fibrosis, which affects many of the organs in his body. She says that his life expectancy is 37, but it would be much better if there were access to Orkambi, that necessary drug. There was sadness on her part that NICE did not see fit to license the drug because of cost and a lack of necessary data. I hope that a change can take place to enable the drug to be made available.
Time limits mean that I cannot touch on every condition relevant to the accelerated access review, so I echo the points raised by my hon. Friend the Member for Dudley North and will focus on another condition to which the review relates and which has already been mentioned by my hon. Friend the Member for Bootle (Peter Dowd)—muscular dystrophy. The effects of that condition are also progressive and can range from mild to severe disability. There is a serious impact on the lives of those with the condition and their families. Sadly, it can also result in premature death, typically in childhood or early adulthood. Accelerating access to new and effective medicines and treatments is clearly vital for those affected by muscle-wasting conditions.
The recommendations around faster assessments by NICE, the flexible approval arrangement and enabling NHS England to negotiate price and flexible commercial deals at the early stages have been welcomed by Muscular Dystrophy UK. The charity has also pressed for funding to be attached to the early access to medicines scheme, so I was pleased to see its inclusion in the recommendations. However, I would draw the Minister’s attention to one recommendation in particular that I ask him to consider carefully. The accelerated access review has recommended a transformation designation and an accelerated pathway for some drugs. For some conditions that is wholly the right approach; however, in the case of muscular dystrophy the aim is to slow down muscle wastage as opposed to transforming or stopping the progression of the disease, and the medicines for muscular dystrophy are therefore incremental. I am sure that the disadvantage of incremental drugs is by no means the aim here, so I ask the Minister to take account of that and to ensure that treatments and medicines for muscular dystrophy are not overlooked.
Finally, Sir Alan, we are talking about treatments that can prolong a child’s ability to walk and to live, having profound effects on their quality of life and that of their family and carers. I look forward to the Minister’s clarification on this issue, and to an acceleration by NICE of the licensing of the drug Orkambi for cystic fibrosis sufferers.
I am pleased to speak in today’s debate and join others in congratulating the hon. Member for Dudley North (Ian Austin) on securing it. I join him in congratulating the Cystic Fibrosis Trust on its work, as well as other cystic fibrosis organisations and charities across the rest of the UK on theirs. As chairman of the all-party parliamentary group on rare, genetic and undiagnosed conditions, I take a particular interest in this subject and in particular how the approach taken by NICE when deciding on funding for drugs is unsuitable for drugs aimed at rare or genetic conditions.
As other Members have said, Orkambi is a particularly interesting development and is part of a new wave of gene-specific precision medicine. It tackles the underlying causes of cystic fibrosis—in this case, the defective CFTR gene—rather than simply treating the symptoms. We should be embracing this new technology and creating appropriate ways for these drugs to be approved and funded, so as not to discriminate against those with a rare or genetic disease. There may be fewer people who suffer from a rare or genetic condition, but I urge the Minister to do all he can to improve their chances. I know that he is a passionate advocate for that particular case.
The NICE process of recommendation understandably relies on data to commission the use of drugs on the NHS. However, the process is currently very rigid, which works against new, often life-changing drugs that only have trial data as evidence. That is exactly what happened with Orkambi, which only had data from a 24-week clinical trial when making its application—similar to a range of other drugs available on the market at the moment—yet evidence shows that it brings significant clinical benefits, as well as wider benefits to society, for people with cystic fibrosis and their carers.
It is not just the lack of available data that discriminates against drugs aimed at genetic and rare conditions. The NICE single technology appraisal process does not adequately reflect the potential benefits of the medicine in protecting future health deterioration or the wider holistic and societal benefits. Its thresholds for cost-effectiveness also work against those with a rare disease. Fundamentally, the diseases are rare by nature and therefore there are only a small number of eligible patients. That should not be a barrier, and we all agree that we need a system that can help those patients.
In short, the accelerated access review, which was brought about after the hard work of my hon. Friend the Member for Mid Norfolk (George Freeman), potentially holds the answers to the problems that currently beset the NICE system. It recognises that the innovative nature of new medicines means that they are unlikely to be approved through the current methods and proposes new guidelines. The new approach will help to ensure that the UK sets itself up as the best possible place to develop new drugs and, I hope, for Orkambi. The Minister might not be able to give me the answer now, but I would like him to write to me on the predicted result of the reduction of the drug spend through NHS England—as we recently heard in a series of evidence sessions held by the APPG for rare diseases—from 7% to about 3.5%, and on how the accelerated access review budget will be increased to compensate.
The Government are set to respond to the recommendations shortly. I hope that their proposal will benefit drugs such as Orkambi that are at the forefront of life science innovation because they treat the underlying causes of the disease, not just the symptoms, thus resulting in a lifetime of health and wellbeing benefits and savings.
The hon. Gentleman is right to focus on the development of these drugs. My concern is not just that patients are denied access to life-changing drugs, but that our pharmaceutical industry finds this a frustrating country in which to develop new drugs and to ensure that they are available to people such as the constituents of my hon. Friend the Member for Dudley North (Ian Austin).
The right hon. Lady is quite right that pharmaceutical companies, in the rare diseases space in particular, find this country a very frustrating place to come to. The message that we are going to support the industry to bring drugs to market here is not loud and clear, and there have been a range of delays and process errors. I know that the Minister and previous Ministers have tried to address this issue, but it has been a very slow, difficult and arduous process, because the message has not been heard loudly and clearly enough.
The difficulty that Orkambi is currently facing in getting funded perfectly displays the problems faced by many other innovative drugs that aim to treat rare or genetic diseases. As chair of the APPG, I get contacted by many people across the country who are desperate to see potentially life-changing drugs approved by NICE. There is a clear deficiency in the process for this type of drug, so I hope that the Minister can today announce a pilot process to show that the UK is committed to leading in this field and providing hope for all those sufferers of rare and genetic diseases.
Before we proceed, I say to Members that we are running very close to the line now. A number of Members have taken the trouble to write in and I need to try to call them all, so I ask each Member to restrict whatever they bring up to a maximum of five minutes, or hopefully less.
It is a pleasure to speak in this debate. I congratulate the hon. Member for Dudley North (Ian Austin) on bringing it forward. One of the major issues that I seek to raise with Government, as the Democratic Unionist party’s health spokesperson, is the treatment of rare diseases and cancers.
Cystic fibrosis is a most debilitating life-limiting disease. It is believed that one in every 2,500 babies in the UK is born with cystic fibrosis. It is a disease that affects too many households in our nation and as such one that we must address to the fullest degree and in the best way possible. As a member of the all-party parliamentary group on cystic fibrosis, I have a great interest in this work and noted, with a small amount of hope, what was being labelled as a wonder drug—Orkambi, which was touted as having significantly reduced hospital admissions and slowing the decline in lung function in people with the genetic mutations that it targets. However, we all know that this year NICE was unable to recommend Orkambi, despite acknowledging the drug as an effective treatment for the management of cystic fibrosis. Since then, negotiations between the manufacturer Vertex, the Government and NHS England have reached a deadlock. Orkambi is a precision medicine that treats the underlying genetic cause of cystic fibrosis rather than just the symptoms, and is therefore very important.
Like the hon. Member for Dudley North and others, I would like to quote people from my constituency and from Northern Ireland—one is from my constituency and one is not. I was emailed by a man from Castlederg, the hometown of my mother’s family, regarding the failure of the NHS and NICE to recommend this drug for the prescription list. Although I had read much about the drug, the human aspect was made so clear in his letter:
“With the power to lift so many of the limits cystic fibrosis can place on people with the condition, it’s vital that access is granted without delay.”
I believe that many of us in this Chamber are here to highlight and draw attention to the plight of our constituents who are crying out for the hope that this drug could bring—the difference of quality years of life for someone suffering from cystic fibrosis. My friend from Castlederg also wrote about this example of a young lady who quite clearly needs help:
“I have first hand knowledge of this drug Orkambi because my daughter Rachel who suffers from cystic fibrosis has been on it for over three years now. Rachel took part in clinical trials for two and a half years and it has transformed her life. Her lung functions have risen by 19%”.
These are more than stats—this is about her life and how Orkambi has changed it.
A constituent of mine, Charlene Barr, passed away at the age of 20, just days before she was due to visit this House to campaign for cystic fibrosis drugs. I ask the House to pay tribute to her and her family for the fantastic work they do in Northern Ireland to raise awareness of this issue and of cystic fibrosis.
My hon. Friend has put his constituent’s name in Hansard as part of this debate, and I believe that is a fitting tribute to her.
My friend from Castlederg also wrote of Rachel:
“Her lung functions have risen by 19%, she has gained a stone in weight and has had very little coughs or colds in this period of time. In CF terms this is massive.”
He said that Rachel was 25 years of age last January and that she
“is currently doing a PhD at University and Orkambi has really given her so much energy and strength to be able to carry out such a big undertaking. Rachel has been very fortunate as Vertex have kept her on Orkambi after the trials because I suppose it would be bad looking on their part if they took her off it...I think that its only right that people that are eligible for this drug should be given the chance to receive it and to prolong their lives for many, many years and maybe even save their lives. The problem is that NICE have told the NHS that it’s too expensive at around £104,000 per year. What price do you put on a person’s life?”
I understand the way things work and I understand well the arguments regarding the likes of pancreatic cancer drugs that could add an extra year to someone’s life versus more money for the research to find a cure, but this drug could make a life such as Rachel’s much better and could help her. The new 96-week data published recently show that Orkambi slows decline in lung function, which is the main cause of death among people with cystic fibrosis, by 42%. The data were unavailable to the NHS, as others have said, but they are available now. We look to the Minister to ensure that the opportunity is available for people to have Orkambi. People who are on Orkambi through the compassionate use programme are beginning to report total transformations in their health, including enough improvement to come off the lung transplant list.
I understand the time restrictions, but I will give one more example. So many people have contacted me, including Martin Keefe, whose beautiful granddaughter, Evie-May, was diagnosed with CF at three weeks old. She is now seven years old. Surely this is the time to begin this treatment, so that she has less irreversible lung damage and can look forward to a longer, healthier life. To be clear, I am not a scientist, a doctor or a researcher, but as an elected representative, I can listen to the difference that these drugs have made and could make to people’s lives—to Rachel’s life, Evie-May’s life and the lives of many others. The research that was not available at the time of the NICE guidelines is now available and it is compelling. With great respect, we are all conveying compelling evidence and information directly to the Minister.
The review is an opportunity to do the right thing by those suffering from this disease, particularly those such as Evie-May and Rachel, who has noticed such a change. It is for those people that I ask the Department of Health to end the stalemate and make a new decision. We look forward to the Minister’s giving them the Christmas present that they want and that we in this House all wish for. I understand the budgetary constraints, but the benefit of the drug appears to outweigh the financial cost. Rachel, Evie-May and others like them, UK-wide, deserve the chance to have the drug.
It is a pleasure, as ever, to see you in the Chair, Sir Alan. I thank the Backbench Business Committee for granting us this debate. I also thank the Cystic Fibrosis Trust, which has not just been instrumental in getting MPs here today, but does great work, as my hon. Friend the Member for Dudley North (Ian Austin) said, in supporting people with cystic fibrosis and their families. I think that Ed Owen is now moving on to other things and I particularly pay tribute to him for his work.
As I have mentioned before in this place, I have a 12-year-old niece, Maisie, who was diagnosed at Christmas in 2004 as one of the 10,500 people in the UK with cystic fibrosis. Thankfully, she is doing very well. In recent weeks, the number of constituents who have contacted me whose children, grandchildren or friends’ children have cystic fibrosis has been revealing to me. Sadly, I also have constituents whose children have died from the condition. The youngest cystic fibrosis patient I have been contacted about was born in April this year. I have also been contacted by a couple of people who have 19-year-olds who are both at university, and the issue of prescription charges mentioned by my hon. Friend the Member for Dudley North has particularly hit home. One constituent said that, although she was very proud that her son had now gone to university hundreds of miles away, he was now responsible for managing his condition, which created some anxieties for her. She said that he was very angry that, despite being classed as a disabled student, he had to pay for his prescription. I hope that the Minister takes that on board because we have been promised a review of exemptions for many years.
As we have heard, Kalydeco, the drug that treats one particular mutation of cystic fibrosis, helps about 4% of patients in the UK. It was approved back in 2013 and the results have been dramatic, increasing lung function and reducing the time people have to spend in hospital on intravenous antibiotics. It has made a huge difference to patients’ quality of life and all the signs indicate that it could significantly improve life expectancy. Now we have Orkambi, which is a combination of the drug that is marketed as Kalydeco and another drug. Orkambi targets a different mutation that affects more than 4,000 people, so it could help almost half the CF population in the country.
The clinical studies that became available last year indicated a significant reduction of 30% to 39% in lung infections and inflammations that lead to irreversible lung scarring and the need for a lung transplant. One in three patients who need a double lung transplant because of cystic fibrosis die while they are still on the waiting list. I hope that organ donation is also on the Minister’s radar. Orkambi could be absolutely life-transforming. Despite that, NICE rejected it back in June because of doubts about its cost-effectiveness. It recognised that the drug is potentially a “valuable new therapy” with “significant clinical benefits”, but it concluded that the cost per “quality-adjusted life year” is too high.
I acknowledge that NICE has a difficult job in assessing all the potential treatments for a range of conditions, and that it operates on a tight budget, but, as the Cystic Fibrosis Trust has argued and as we have heard this morning, the processes that NICE uses can be flawed because they rely on short-term data for a lifelong condition. We heard from my right hon. Friend the Member for Leigh (Andy Burnham) that new data show that after 96 weeks of treatment—NICE only looked at the 24-week clinical trials—the decline in lung function, which is the main cause of death for people with cystic fibrosis, slows by 42%. If NICE had had that data, it would have rated Orkambi’s cost-effectiveness much higher.
I am partly speaking from personal experience, but I also know from my constituents’ very sad stories that people are desperate for something to address these problems. The accelerated access review was commissioned because the Government recognised the weaknesses in the NICE process. For patients whose life depends on the outcome of the report and its implementation, it has been a long wait. I urge the Minister not to delay any longer and to consider the Cystic Fibrosis Trust’s request to apply the AAR recommendations to the deadlocked Orkambi negotiations. Confidential commercial agreements would free Vertex to make the NHS its best offer, and commercial agreements under the Cancer Drugs Fund are confidential. Flexible pricing arrangements would allow the consideration of alternative models to manage costs, such as a price cap that is not exceeded even if patient numbers rise, and the collection of real-world data would allow a more accurate assessment of the drug’s effectiveness. The hon. Member for Bury St Edmunds (Jo Churchill), who has now left the Chamber, mentioned the fact that 99% of cystic fibrosis patients are on the database and that there is a lot of available information about them, which makes them ideal to pilot the scheme. I hope that the Minister considers that.
It is a pleasure to serve under your chairmanship, Sir Alan. I congratulate my hon. Friend the Member for Dudley North (Ian Austin) on leading this important debate. We have long needed to address the fact that the UK is trailing behind on patient access to new medicine. A.A. Gill who, just three days ago, sadly died of cancer, wrote:
“The NHS represents everything we think is best about us.”
But in his final column, he revealed that he was denied immunotherapy that might have helped him to live longer. He said that NICE,
“the quango that acts as the quartermaster for the health service, won’t pay.”
His experiences are striking, but they are a symptom that is all too common of a system that is struggling to cope. When Labour established NICE it soon became a world leader in approaching the profound and challenging question of how to allocate scarce resources fairly. Although the question remains the same, the times have changed and the pace of innovation has increased, as has the cost.
We were all pleased when the Government’s long-awaited and much-delayed accelerated access review was finally published. Simply put, we need innovative new drugs to reach patients quickly at a price that the NHS can afford, but it is not clear that the accelerated access review has solved the conundrum. There are already signs, since publication, that yet more problems are emerging. NHS England’s sudden and unexpected consultation on the QALY—quality-adjusted life years—threshold for highly specialised technologies risks running counter to the spirit of the AAR and introducing yet another gatekeeper.
The AAR recommends a fully funded early access to medicines scheme, but we need a positive response from both the Government and NHS England because the danger is that, with other countries having funded early access companies, we risk seeing clinical development work moving away from the UK.
As we have heard, the Cystic Fibrosis Trust has suggested that the AAR made several recommendations that could enable access to drugs such as Orkambi through flexible pricing arrangements and the gathering of real-world data to prove the drug’s effectiveness. Does the Minister agree that those recommendations will make a difference to people affected by cystic fibrosis? If so, will he commit to implementing the recommendations?
Other hon. Members have told us of real-world examples, and I will quote my constituent Julian Wheel:
“My youngest daughter, a local Cambridge primary school teacher for over 15 years, recently had a new daughter, diagnosed with cystic fibrosis. It imposes major changes on her and her partner’s lives in caring for her—time, difficult nutritional choices and the fear of recurrent infection, not to mention the additional and regular workloads imposed on the NHS staff at Addenbrooke’s, the local GP practice in Harston and healthcare visitors.”
He says that their family receive terrific support from the cystic fibrosis clinic and local surgery practice, but this new drug could relieve the suffering and improve their quality of life. He says it could offer “real hope.”
New drugs are expensive, but incentivising innovation should be a priority. The Government must ensure there are effective mechanisms that can help to address the affordability challenges that new treatments are likely to present. A balance will need to be struck between setting a price that rewards and incentivises innovative research and setting a price that is also affordable to the NHS. Will the Minister establish a strategic commercial unit in NHS England to consider flexible pricing models?
Recommendations such as a fully funded early access to medicines scheme could make real inroads, but of course that depends on the Government supporting their implementation. The BioIndustry Association points out that innovation is impaired because the current early access to medicines scheme is not funded, and the lack of funding poses a barrier to many small biotech companies engaging with the scheme.
Across the board, organisations have called for a strong response from the Government to the accelerated access review. I am not sure we yet have that. Will we have one today?
I congratulate my hon. Friend the Member for Dudley North (Ian Austin) on the spirit in which he introduced the debate and on his consensual tone. I also commend him for the quality of the case he outlined in his excellent speech. He could not have done a better job of representing his constituents and all those represented by the Cystic Fibrosis Trust. My hon. Friend the Member for Bristol East (Kerry McCarthy) is right to pay tribute to Ed Owen, the departing chief executive of the CF Trust, who made an enormous difference to so many people during his tenure.
I sympathise with the Minister, because I have been there when such difficult issues have arisen. I assure him that there is no party politics in this room today. We have heard excellent speeches from both sides of Westminster Hall on issues of great importance to our constituents, and Members have made their points in that spirit.
I was involved in the establishment of NICE, which my hon. Friend the Member for Cambridge (Daniel Zeichner) mentioned, and it did become a world leader. I am the first to say that it can never be right for politicians to sit in judgment on treatments—judging that those who shout the loudest should therefore get the treatment. NICE was established for an important reason and, as a Minister, I always sought as best I could to stick to NICE’s judgments and not to undermine them. On occasion, however, treatments would come along that were, quite simply, exceptional and that could not be considered within the narrow confines of the NICE appraisal process. Those treatments were often innovative and related to chronic conditions where the drug, if used, might have a long-term beneficial social impact, rather than an impact that would necessarily return money to the NHS budget.
To be open about the shortcomings of the NICE process that we established, NICE was not able to consider the wider public budget, the Department for Work and Pensions budget and other budgets. My hon. Friend the Member for Dudley North spoke about people being able to work and care for their kids, and often the failure to fund a drug has a much wider social cost, yet the narrow process applied by NICE often did not take that into account. Orkambi is one such treatment where we need exceptional consideration of its potential wider impact. The accelerated access review has given the keys to the Minister. There are things that can be done, and we all urge him to use those flexibilities today.
I could say a lot more, but the best way to use the time remaining to me is to refer to some of my constituents, many of whom have been in touch to encourage me to speak today. My office manager, Karen Aspinall, has a son in his 20s who has CF. Through her, I know how it is to live day to day with the challenges presented by CF.
I close with the direct words of my constituent, Leigh resident Philip Grimshaw. He is 28 years old and his words say far more than I could. He and his sister Melissa were diagnosed with cystic fibrosis when they were very young, and this is what he wrote to me:
“Melissa was diagnosed with CF as a baby after being very unwell since birth, and I was diagnosed as a result of this, at 7 years old. All our lives we have had to take a cocktail of medications and have had frequent stays in hospital.
In my opinion Orkambi would, amongst other things, reduce the number of hospital stays and also reduce the need for occasional extra antibiotics due to CF related illness (because we would be in better health as a result of Orkambi). Both of these mentioned would save the NHS money. I understand that it’s not a cheap medication but neither is a two week hospital stay, on a specialised ward, on extra antibiotics, six times or more a year.
I do think that the stress of losing our mum had an impact on Melissa’s health.”
Sadly, Melissa died in 2013. The letter continues:
“The problem with CF is that once your health starts to slide, it’s very difficult to bring back to where it should be. If Melissa was on Orkambi then it could have kept her in a better state of health and prevented her becoming as ill as she was and would have prevented the worst.
As for me; I’m looking to settle down and have children in the near future. Orkambi would help me to watch my children grow up rather than live to the predicted age of being in my 50s. I have had the dilemma of whether or not to pay into a pension because I won’t live to see retirement age and maybe even not long enough to be able to take a lump sum out at 55!
Orkambi can change that. It would be nice to have the confidence to know that I could see my children graduate university, start jobs and even have children of their own.”
I am sure that Philip’s words would be shared by many other people in their 20s, or younger, with cystic fibrosis if they had the opportunity to vent them in this place. They are the appropriate words on which to finish.
I recognise the difficulties, but we were here before with Kalydeco and we managed to find a way through. As my hon. Friend the Member for Bristol East said, nobody now doubts that that drug has made a huge difference to so many lives. We are in exactly the same position again, so let us learn from that experience. Let us not test people’s patience. Let us get the appraisal process moving towards a positive resolution. I realise that that is a challenge, but Ministers sometimes need to cut through the bureaucracy. The Minister is a good man, and I urge him to do that today.
Access to new drugs seems to be almost the commonest theme of debates in Westminster Hall. Having spent years as a breast cancer specialist involved in trials, I can say that it is really frustrating to have access to drugs within a trial and then lose that access when the drug is passed. The United Kingdom can be up to five years behind Europe or America in accessing such drugs. We talk all the time about having more research on brain tumours and other diseases, but that does not help us if, at the end of the research, our patients cannot get the drugs.
Cystic fibrosis is one of the commonest of what we call the rare diseases. It involves a problem with transmission of salts through membranes, which results in incredibly thick mucus that clogs various organs, most commonly the lungs. As the right hon. Member for Leigh (Andy Burnham) mentioned, if it is diagnosed late, damage will already have been done. The earlier patients with cystic fibrosis are treated, the less damage is done and the healthier they are. The life expectancy has changed from childhood to middle age, due to a combination of approaches.
I am shocked to hear that people in England with cystic fibrosis have to pay for their prescriptions, because that would amount to quite a lot of money; they are on a lot of medication. We do not have prescription charges at all in Scotland, because you get an increased rate of people not collecting their prescription, or going to the chemist and saying, “Excuse me, dear, which are the two most important drugs for me to take?” and that ends up not being cost-effective. I would have thought that people with a chronic condition should at least have their names on a list as being exempt. I would have thought that that was the least the Government could do.
The right hon. Member for Leigh said that we have been through this with Kalydeco and many other drugs. Orkambi is a synergistic combination of Kalydeco, or ivacaftor, with lumacaftor, which makes it work much better. They are the first drugs that are not just antibiotics or mucolytics; they are trying to attack the disease itself. In that sense, they are transformative. The problem in the access review is that the definition of “transformative” going forward will not necessarily help those drugs. We do not suddenly find a drug that is a cure for any of these conditions; we move step by step, often adding drugs together or making new discoveries.
There is a real concern among those who develop drugs that in the consultation between NICE and NHS England, the levels considered acceptable for such highly specialised treatments are being changed. The problem is that if we send out the message to people with rare conditions, “I’m sorry; you’re just outside the pale,” we will be letting them down. We need a different approach. I think we need a different conversation for all drugs. The NHS in the United Kingdom brings a cohesive system that allows for follow-up data and allows a lot more information to be sent back to companies over time, which is not easily available in other countries. That should be on the table as part of the negotiations.
I have a real concern, going forward after Brexit, that in this country we will be further down the list for people to even apply for licences here. It may well be that the application to the Medicines and Healthcare Products Regulatory Agency for 60-odd million people in the United Kingdom may well cost an amount very similar to an application to the European Medicines Agency for a market of 450 million. That means we could end up in the same position as Canada, where it often takes about a year before a company decides to apply for a licence. The problem is that if, going forward, companies see that they must pay to apply to NICE, which will turn them down so they will have to pay to apply again, they may just decide that it is not worth the candle. That must be taken into consideration.
Obviously, England has the Cancer Drugs Fund, the idea of which is to allow a little bit of flexibility on access to new drugs, which are often expensive, but it does not help you if you do not have cancer. In Scotland, ours is a new medicines and rare diseases fund, which as a proportion of the population is three times the size of CDF, so it is more flexible. It cannot be a long-term solution for such drugs, or the funds would get sucked up, but it is important that when we are going through a phase of considering the real-life use of expensive drugs we have some flexibility for patients, and not just cancer patients.
We had a debate in the main Chamber last week about the Health Service Medical Supplies (Costs) Bill, and one discussion involved funding. In Scotland, the pharmaceutical rebate goes to fund the new medicines and rare diseases fund. In England, it goes back into core funding, which means that along the line, the beneficiary is the Treasury. If the NHS is managing attribution and access to new drugs in such a way that it gets a rebate, it should be able to use that to access more innovative medicines. That is why the pharmaceutical industry agreed to it. It also creates a better relationship with the pharmaceutical industry. We cannot have a situation where the industry just pulls a price out of the air and we must rise to it—of course we must get value for money—but it is really important that we do not leave people with certain conditions knowing that there is utterly no chance that they or their children will access treatment.
It is an honour to serve under your chairmanship, Sir Alan. I thank my hon. Friend the Member for Dudley North (Ian Austin) for securing this important debate. I note that as he said, it is just over a year since he first brought to the House a debate on cystic fibrosis.
I appreciate all hon. Members who have attended and spoken in this debate to show their support for the cause; it is one that we must urgently get right. Members have shared many moving cases involving their constituents whose lives Orkambi could save and would certainly transform. My hon. Friend the Member for Dudley North mentioned Carly Jeavons and Sam and Rob, the parents of Daisy. The hon. Member for Strangford (Jim Shannon) spoke about Evie-May, and my hon. Friend the Member for Bristol East (Kerry McCarthy) mentioned her niece Maisie. My right hon. Friend the Member for Leigh (Andy Burnham) spoke about his office manager Karen Aspinall and her son, as well as Philip and his sister Melissa, who sadly died. Philip believes that Orkambi would have helped his sister and would certainly help him, as he also suffers from cystic fibrosis. Those people believe that their lives would be transformed by Orkambi. I believe that too, and the evidence supports it, as we have heard in detail.
I thank all hon. Members who have spoken in this debate, including the hon. Member for St Ives (Derek Thomas), the hon. Member for Bath (Ben Howlett) and my hon. Friend the Member for Cambridge (Daniel Zeichner), for their excellent contributions, as well as the many others who have made valuable interventions. I also thank the Cystic Fibrosis Trust for its dedicated campaigning on the issue, and the 20,000 people who have been involved in its survey, in the digital debate here in Parliament, and in petitions and e-action. The concerns and the need for action are clear, and it is up to the Minister to give all those people beyond this place the answers that they need.
In my contribution, I will set out why the Opposition want to see the Government do more on innovative drugs, through case studies involving Orkambi. I will touch on issues of access to Orkambi and other drugs for those living with cystic fibrosis and expand into the recommendations of the accelerated access review, which can do much to address many of the issues involving access to new drugs.
Although it is welcome that the prescription drug Kalydeco was given the go-ahead by NHS England last week for two to five-year-olds as part of re-prioritisation, Orkambi remains an issue. There is currently a deadlock in negotiations between the pharmaceutical company Vertex, the Government and NHS England for the drug to be accessible to the 2,700 people who stand to benefit from it. As we have heard in detail today, that is all down to rejection of the drug under NICE’s appraisal system because there is a lack of long-term data. Although it is welcome that NICE recognises the treatment as effective in managing cystic fibrosis, it is clear that we desperately need a new system under which drugs can be better accessed, especially those that show that they can benefit patients. We have also heard about new data that NICE did not take into account and that would have showed 42% effectiveness.
Orkambi has been shown to halve the amount of hospitalisation of cystic fibrosis sufferers, and 96-week data published recently showed that it can help to slow lung function decline by 42%. The data are also backed up by anecdotal evidence from people who have accessed Orkambi through the compassionate use programme and are beginning to report transformations in their health—some are reporting enough improvement to come off the lung transplant list. That information is all positive. It should be made better available for consideration as part of the appraisal process; it should also form part of the negotiations between Vertex, the Government and NHS England. However, when we see a deadlock, all of that information is for naught. Thousands of people are suffering irreversible lung damage that could be stopped if the current impasse between those around the negotiating table was broken. Those who will suffer the most are stuck in the middle.
It is up to the Government to facilitate the end of the deadlock so that people can access Orkambi and see their lives transformed. One way to do that is to begin the job of implementing the recommendations set out in the accelerated access review, which the Opposition welcome. The goal of speeding up access to drugs by cutting four years off the time needed to bring new medicines to patients is something that we should all welcome; we need to see whether it can be achieved. The review has the potential to change the philosophy of the NHS in line with the five-year forward view, but also to help to maintain our global lead in life sciences. The recommendations set out in its final report have the potential to transform how we provide drugs and treatments, ensuring that we see innovation in drugs, diagnostic tools and healthcare developments. However, there still remain issues around thresholds for new drugs, which NICE and NHS England are currently consulting on. I understand that some associations and charities have raised concerns about that, and I hope that the Minister will update us on some of those discussions.
My hon. Friend is right to be so positive about many aspects of the accelerated access review. However, as she has mentioned, there are concerns that new definitional ruts could be created by some of the terms of the review, which could lead to some patients and some promising drugs being trapped in exactly the sort of deadlock that she has described.
My hon. Friend is right to raise those concerns. We do not want to move into a new system that will create new unintended consequences. Perhaps the Minister will touch on that in his speech.
Although some are calling for interim solutions to help people who are stuck waiting for the accelerated access review’s recommendations to be implemented, it is also important that the Government get on with implementing those changes. The review was announced more than a year ago and was published two months ago now. It is important to remember that the transformation that we all want to see will not happen straight away, but it is still right that we keep up the pressure for the recommendations to be implemented. There are many such recommendations, and I hope that the Minister will be able to update us today on the progress on each of them. There are two in particular that illustrate what can be done to resolve the deadlock around Orkambi—the immediate establishment of an accelerated access partnership and the setting up of a new flexible strategic commercial unit.
The accelerated access partnership is one way in which, through co-ordination and collaboration across the system, we could see drugs brought on to the market more quickly to benefit patients who need access to them. I would be interested to hear from the Minister what progress has been made on its creation, especially in conjunction with the issues surrounding the deadlock on Orkambi.
It is clear that the strategic commercial unit could help to benefit those who wish to see Orkambi offered on the NHS. The unit could work with those involved in this dispute to end the current deadlock through facilitation of the flexibility and transformational change promised by the accelerated access review. That would go some way towards helping to access data on drugs such as Orkambi and getting them out to patients. There is a willingness out there for that flexibility to be brought into the system; for example, the Cystic Fibrosis Trust has offered to use the UK cystic fibrosis registry to help to provide essential data that can help to prove how effective drugs can be and what more needs to be done. We have already heard how substantial that registry is; it includes 99% of sufferers. I understand that the trust’s offer has been welcomed by all sides in the negotiations but is blocked due to the lack of progress in implementing the changes set out in the review. I hope that the Minister will give us some clarity on when the unit will be created and when we can see a culture shift within the system that will allow for flexibility to accept data and information that show how much effect these drugs have on people’s lives.
Does the hon. Lady share my concern about drugs for other conditions, such as sofosbuvir for hepatitis C? Even after they get NICE approval, those more expensive drugs are now being rationed at the NHS England stage. At the moment we are fighting to get through NICE, but it needs to be a smooth path all the way through.
The cost of drugs sometimes leads the NHS into the terrible and unfortunate situation in which rationing seems to become the norm. There can also be a postcode lottery, which is another element that we need to look at. The price of drugs really is the crux of the issue.
In conclusion, I hope that the Minister will offer some insight into the progress being made on the recommendations of the accelerated access review. The case of Orkambi can help to drive through these changes and to end this deadlock, which, as we have heard, is causing unnecessary suffering for those living with cystic fibrosis. The review has established a space for change and for patients to access new and innovative drugs and treatments. It is important that there is no stalling or delay in transforming the system, because people’s lives depend on the changes called for by the review. I am sure that the Minister will keep that in mind when he goes back to his officials.
It is good to serve under your chairmanship today, Sir Alan. I congratulate the hon. Member for Dudley North (Ian Austin) on leading the charge in this debate. The right hon. Member for Leigh (Andy Burnham) rightly said in his very good speech that this is not really a political issue. Every Member in this Chamber has constituents who would benefit from these drugs. There are 10,500 people in the country with cystic fibrosis and it is massively important that we do everything we can to make progress on the issue. I also congratulate the Cystic Fibrosis Trust on its work and on its “Stopping the Clock” campaign. Debates such as this give prominence to these issues and to the need to make progress.
The debate is really about two drugs, a drugs company and an evaluation process. I shall speak about all of those and about where we are going with the accelerated access review. The two drugs are Kalydeco, which applies to something like 4% of cystic fibrosis sufferers, and Orkambi, which would apply to a further 40% of sufferers. Both are relatively small populations: for Kalydeco it is something like 400 people in England, and for Orkambi it is something like 2,700 or 3,000. Kalydeco has been routinely available on the NHS since 2013. As mentioned today, it was extended on 4 December to children aged two to five. It makes a big difference and we are pleased to have made that progress. Both Kalydeco and Orkambi are produced and owned by a Boston-based drugs company called Vertex, which I shall talk about later.
Orkambi could be used by around 3,000 patients. It has a price of something like £100,000 per annum—the implication being that the cost of its approval in England would be in the order of £300 million or £400 million a year. As several Members have said, it is obviously right that there is a process that weighs that cost of £300 million to £400 million a year against other NHS priorities and other drugs. That process is the NICE process. A number of comments have been made about the efficacy of that process, and it has been suggested that it may have deficiencies in respect of providing precision drugs to small numbers of users. I will try to address those concerns. I think everybody agrees that we need a consistent method of evaluating these matters, and there needs to be a way forward based on that.
When NICE evaluated Orkambi in July, it found that it had clinically significant and important benefits, which several Members have spoken about. There is no dispute about that, but the evaluation process—which is based on quality-adjusted life years, as has been said—also found that it was not cost-effective. I spent some time last night reading the NICE evaluation, and make the point to colleagues and other Members that it was not a near miss. It looks like there is a factor of 10 in NICE’s evaluation of its cost-effectiveness. I guess that is largely driven by the price of £100,000 per annum and what that would mean.
It is obviously reassuring to everybody that the Minister has taken such a close interest in the issue before coming to the debate. He says it was not a near miss. That may have been the case on the data that NICE had, but does he accept the point made by my hon. Friend the Member for Dudley North (Ian Austin) and other Opposition Members that those data were very limited indeed? The 96-week trial data that are now available would probably have produced a very different overall calculation.
To be honest, I am not qualified to have an opinion on that. The right hon. Gentleman rightly said that decisions of this sort should not be made by politicians and that there has to be a process around them. It is clear that if NICE is presented by Vertex with new clinical data, or indeed new price data—this is perhaps equally relevant, but we have not really discussed it—a review could be carried out quickly without any need for us to go through the whole process again. There is a precedent for that, and if those data exist and Vertex presents them, they would be looked at. I give my commitment, and certainly that of the Minister responsible for this policy area, that that would be the case and there is no impediment to that. I do not want to raise false hopes by saying that, and I do not think I have done so. The fact that it is not a near miss—it is possibly out by a factor of eight or 10—implies that there is quite a lot of work to do on pricing.
It is worth recapping what other countries have done. Orkambi is available in Germany, although it appears from the data available that its use there is quite mixed, with perhaps no more than one in five eligible people having access to it. In France, the other country in Europe that has authorised it, Vertex has booked no sales yet this year. The picture seems quite mixed in those countries. The countries that have not authorised Orkambi include Scotland.
In my speech I mentioned a young girl from my constituency, Rachel, who has been on the Orkambi trials and shown exemplary improvements in her health. That is an example we can all point to of where goodness has come out of the drug for those who have had the opportunity to have it, and that is true not only in my constituency but throughout the whole of the United Kingdom of Great Britain and Northern Ireland.
There is no dispute that the drug works, and there is no dispute at all that it is life-changing. The issue before us is the extent to which it justifies a price tag of £300 million to £400 million versus other NHS priorities. All I can say on that is that it is right that the decision is not made by politicians, for the reasons given earlier by the right hon. Member for Leigh.
I was discussing the countries that have so far not authorised Orkambi. Neither Scotland nor the Republic of Ireland accepted that it was cost-effective, and it is not used in Scandinavia or Canada either.
The Minister mentioned Scotland and the Republic of Ireland, where there are clearly challenges—we only have to look at the pictures in The Irish Times yesterday to see the graffiti about Orkambi in Dublin. Will he commit to working with colleagues from across these islands to use the underdeveloped and underused machinery of the British-Irish Council to literally get our act together when it comes to rare diseases? We should combine our purchasing power when negotiating with the drugs companies and ensure that there are much better networks for referral and treatment. We should improve that collaboration and literally get our act together on these islands.
I, too, saw the press. I think the Republic of Ireland drugs Minister has talked about writing around to that effect, and it would be a great idea were we to use our combined procurement muscle in that regard. He is certainly pressing at an open door.
I wish to spend a little time talking about Vertex. The company owns the drugs we are discussing and is worth $18 billion. As well as looking at the NICE review last night, I spent quite a lot of time looking at Vertex’s financial position. The company needs to sell these products; indeed, its continued functioning as a major pharmaceuticals company depends on that. Its share price has fallen by a third during the course of this year—I estimate that is a loss of value of something like $7 billion—because its sales are not adequate. There needs to be a meeting of minds here. I am sure that people from Vertex are listening to this debate, as will people from other places, too. We all want a solution whereby the drug becomes available at a cost-effective price, but the negotiation is not a one-way street; Vertex is part of it as well. Were the company to come forward with different pricing data, those would be looked at very quickly. At some point in the future—I know it will be a long time—the drug will be available generically, although I accept that that will not give hope to some of the people we have heard about today.
In the couple of minutes I have remaining, I wish to discuss the accelerated access review, which was a manifesto commitment we made at the election. We set up the review panel. The basic intent was to enable transformative drugs to come forward more quickly and for there to be, as Members have mentioned, a commercial unit in the NHS that is empowered to do deals and bring treatments forward more rapidly. In October the review team and panel published the final report, to which something like 600 stakeholders contributed. It is a valuable piece of work and we know its direction of travel: bringing drugs into the system more quickly, allowing the NHS to set priorities for the drugs its wants, and giving drugs companies some notice and knowledge that if they develop drugs, they will be used. That will mean that a lot of the commercial discussions can happen earlier and progress can be made more quickly.
The Government are reviewing the results of the accelerated access review. There is much in it, if not all of it, that will be accepted, although I am not in the position to accept it today—that is not my role here. We do, though, want to make progress, which should give some hope for the potential of another review of the matters we have been discussing. Nevertheless, I must say again to Government and Opposition Members that the NICE process and the people carrying it out—they are rigorous scientists and serious doctors—need to be treated and understood with respect. We can all agree that the current situation is heartbreaking for many people. The world has a drug that would change people’s lives, but the world has not rolled that drug out to them because of real and reasonable financial issues. I accept that that is a very difficult thing to explain to people and it is very difficult to accept.
Motion lapsed (Standing Order No. 10(6)).