Accelerated Access Review

Lord Austin of Dudley Excerpts

Tuesday 13th December 2016 Westminster Hall

[Ian Austin (Dudley North) (Lab)]

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.

[Andy Burnham (Leigh) (Lab)]

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?

[Ian Austin]

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.”

[Julian Sturdy (York Outer) (Con)]

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.

[Ian Austin]

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.

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

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.

[Ian Austin]

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.

[Peter Dowd (Bootle) (Lab)]

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?

[Ian Austin]

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?

[Several hon. Members]

rose—