Accelerated Access Review

Andy Burnham 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.”

--- Later in debate ---

[Andy Burnham (Leigh) (Lab)]

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.

--- Later in debate ---

[The Parliamentary Under-Secretary of State for Health (David Mowat)]

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.

[Andy Burnham]

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.

[David Mowat]

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.