Cystic Fibrosis

Lord Austin of Dudley Excerpts

Tuesday 8th December 2015 Westminster Hall

[Ian Austin (Dudley North) (Lab)]

I beg to move,

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

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

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

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

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

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

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

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

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

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

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

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

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

“hard to state the significance on quality of life”

that new drugs had given him. He said that he

“now had a stable job”

and is

“in the middle of getting a mortgage”.

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

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

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

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

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

--- Later in debate ---

[Ian Austin]

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

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

Question put and agreed to.

Resolved,

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