Cystic Fibrosis

Rachael Maskell Excerpts

Tuesday 8th December 2015 Westminster Hall

[Rachael Maskell (York Central) (Lab/Co-op)]

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

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

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

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

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

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

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

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

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

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

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

--- Later in debate ---

[George Freeman]

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

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

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

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

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

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

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

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

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

[Rachael Maskell]

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

[George Freeman]

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

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

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

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