Cystic Fibrosis

Julian Sturdy Excerpts

Tuesday 8th December 2015 Westminster Hall

[Mrs Gillan]

The hon. Gentleman is absolutely right. I pay tribute to the other colleagues in the House who took part in that petition. That truly cross-party effort aimed to draw attention to the drugs that are not readily and fully available to our constituents. I was grateful that it was a cross-party delegation, because such things are much stronger when they take place in an atmosphere of good co-operation across the board rather than a political atmosphere. We saw parliamentarians at their best, so I thank the hon. Gentleman for attending that lobby at No. 10 Downing Street, which was inspired partly by Muscular Dystrophy UK and partly by the families it supports.

The issue for me is the drug that the hon. Member for Dudley North referred to. Translarna is its trademark name; it is called ataluren. It is produced by a company called PTC Therapeutics, which calls it its “lead product candidate” for these disorders. I know that the Minister is familiar with PTC Therapeutics, and I hope that in his winding-up speech he will refer to any contact he has had with the company. One of the issues surrounding the efficacy and licensing of the drug is the cost, so I hope the Minister will update us on that situation.

PTC Therapeutics states that the drug is a

“novel, orally administered small-molecule compound for the treatment of patients with genetic disorders due to a nonsense mutation. Ataluren is in clinical development for the treatment of Duchenne muscular dystrophy caused by a nonsense mutation…and cystic fibrosis caused by a nonsense mutation…Ataluren was granted conditional marketing authorization in the European Union under the trade name Translarna”.

I believe that it is already available in France, Germany, Italy and Spain. It is the first treatment approved for the underlying cause of Duchenne muscular dystrophy, which is a complicated condition.

Nonsense mutations are implicated in a variety of genetic disorders. They create a premature stop signal in the translation of the genetic code contained in the mRNA. That prevents the production of full-length, functional proteins. The company says that

“ataluren interacts with the ribosome, which is the component of the cell that decodes the mRNA molecule and manufactures proteins, to enable the ribosome to read through premature nonsense stop signals on mRNA and allow the cell to produce a full-length, functional protein. As a result…ataluren has the potential to be an important therapy for muscular dystrophy, cystic fibrosis and other genetic disorders for which a nonsense mutation is the cause of the disease.”

The importance of access to Translarna cannot be overstated. Boys such as my constituent Archie Hill have been waiting since August 2014 for a decision on whether Translarna will be approved in England. As I said, it is the first licensed drug to tackle an underlying genetic cause of Duchenne’s. It would help to keep Archie and these other boys walking for longer and potentially delay the onset of the devastating symptoms affecting the heart and lungs that I referred to earlier.

NICE’s appraisal of the drug is ongoing, but the families have not yet been made aware of when guidance will be issued, leaving them facing an anxious wait over the Christmas period. Over the time I have known Archie and his family, I have seen his mobility decrease; it is depressing to see such an active, energetic, lively, intelligent young man, who has his life before him, being denied a drug that could well keep him active for longer and improve his quality of life.

[Julian Sturdy (York Outer) (Con)]

My right hon. Friend is making a powerful argument. She is right to say that we must improve access to new medicines, which can transform the lives of people such as her constituent Archie. Does she agree that new medicines may also reduce hospital admissions, which would have a huge impact on the NHS?

[Mrs Gillan]

I thank my hon. Friend for that intervention. He is absolutely right. There is no doubt that increasing the length of time that these young people can be kept active and mobile will inevitably reduce the amount of time that they spend requiring treatment in other health settings.

I also want to describe the emotional journey. Seeing anybody suffering with a muscle-wasting condition is terribly draining, because they fade before one’s eyes. That is why the drug is so important, particularly for young people suffering from Duchenne’s. I turn now to my constituent’s mother, Louisa Hill, for a quotation. She said:

“Decision makers need to understand the impact on children of even a small change. It gives them more time to run and play football with their friends. It’s really buying precious time. Archie will have to deal with very difficult mental and physical challenges as his condition progresses. Translarna is buying time for Archie just to be a kid.”

If you are not touched by that statement from a mother, I do not know what you would be touched by.

Translarna is not the only potential therapy that could benefit Archie. For example, others, such as utrophin upregulation, which involves injecting a protein called utrophin into the muscles to compensate for the loss of dystrophin in boys and young men with Duchenne’s, are in a later stage of clinical trial. It is vital that the process of moving such drugs from the laboratory to the clinic is expedited, including ensuring that appraisal processes are as swift as possible; that secure funding is available to help meet the costs of new drugs; and that NHS England and NICE have effective mechanisms to negotiate an appropriate price with drug companies.

On 14 October, I had the temerity to question the Prime Minister on Translarna at PMQs. He referred to the cancer drugs fund and its role in reducing the costs of drugs for rare types of cancer. A similar model would help for rare disease drugs for conditions such as Duchenne muscular dystrophy. The Prime Minister said:

“The cancer drugs fund has helped to reduce the costs that the companies charge. We need to see that in other areas, too.”—[Official Report, 14 October 2015; Vol. 600, c. 313.]

The Government’s accelerated access review provides an important route through which such issues could be addressed. I hope that the Minister will have his feet held to the fire by the Prime Minister’s answer.

Research into treatments for Duchenne’s is at a promising stage, with a range of potential therapies in late stage clinical trials. As I said, Translarna is already licensed in Europe, but the UK muscle centres where trials are conducted are reporting that given the growth in clinical trials they lack the resources, such as staffing levels and equipment, to keep pace. As a result, centres report that they are turning away new trials—not because of bad science, but because of a lack of capacity. [Interruption.] I see the Minister shaking his head. He knows that the situation is serious and I hope he will comment on it.

That lack of capacity risks causing a bottleneck in drug development and gives boys such as Archie Hill less chance to enrol on a trial that could allow them access to a new therapy. A clinical trial capacity audit, conducted by Muscular Dystrophy UK as part of the “Newcastle Plan” of joint working with UK Duchenne charities to address clinical trial capacity, corroborated the reports and also found that:

“Work on clinical trials is not counting towards specialist training at many centres for medical doctors, physiotherapists and nurses”

which is affecting trainee participation. In addition, it was found that a

“lack of acknowledgment of research in clinical job planning means that already overstretched clinical staff are having to carry out research activities in their own time. This is consequently severely limiting centres’ abilities to take part in research.”

It also found that the process of setting up a clinical trial can be excessively bureaucratic. Perhaps the Minister, with his experience in this area, will be able to comment on that.

I am disappointed that Archie Hill and the other boys suffering from Duchenne’s do not have access to Translarna. The process has seemed to take an incredible length of time, and I hope that the Minister will be able to do something about it. Like the hon. Member for Dudley North, I have a series of questions that I want to put to the Minister, which may help him when he sums up.

First, will the Minister commit to meet representatives of Muscular Dystrophy UK? I would be grateful for that, and it would be helpful for him to discuss the accelerated access review, particularly in the context of the emerging treatments for Duchenne’s. Secondly, I do not suppose that he can say this, but when can families such as Archie’s expect to be notified of NICE’s guidance on access to Translarna on the NHS? It is the obvious question and one that I hope he can answer.

Thirdly, will the Minister ask the chief executive of the National Institute for Health Research’s clinical research network how his organisation plans to work with specialist muscle centres to address concerns over the lack of clinical trial capacity, particularly for Duchenne’s? The hon. Member for Dudley North referred to the latest thinking in Scotland and Northern Ireland, such as introducing a ring-fenced fund for rare diseases. I hope that that might be a recommendation of the accelerated access review.

I do hope that the Minister will be able to give us some optimism. Boys such as Archie Hill are an inspiration to us all. For one so young, he is very mature in his attitude towards not only his Duchenne muscular dystrophy, but other children suffering from rare diseases. He has great capacity for humanity and for tireless campaigning. This will be the second Christmas since I met him that he will be waiting for an outcome on Translarna. Will the Minister talk to PTC Therapeutics, to NICE and to anyone else to whom he can reach out, to ensure that this year the Christmas present for Archie Hill and other boys in England is to have access to ataluren or Translarna?

--- Later in debate ---

[The Parliamentary Under-Secretary of State for Life Sciences (George Freeman)]

It is a pleasure to serve under your chairmanship, Sir Edward. I congratulate the hon. Member for Dudley North (Ian Austin) and other hon. Members from across the House who have spoken. This timely debate has been incredibly powerful—not that there has been much disagreement in it. It has been an opportunity to raise important issues that I am dealing with, and I am grateful to colleagues for acknowledging that.

The debate is particularly timely because I am convenor of a major summit today on accelerated access for faster cures. There is a precision medicine summit in London and the Association of Medical Research Charities has just held its annual conference, at which I exhorted members to come to my table with ideas about how to accelerate novel treatments and give the charities more of a voice. A powerful and helpful debate is going on.

I pay tribute to the work of the Cystic Fibrosis Trust, which is among a number of charities that lead the debate on innovative treatments and medicines. Its leader Ed Owen in particular plays an important role in that; but so do Carly, Lorraine, Michael, Kelly and the other people who have been mentioned. Many of the charities do extraordinary work to articulate the experience of patients who suffer from disease and bring it to the policy table in a powerful way. It is a change in policy making that I am keen to accelerate.

The debate goes to the heart of the challenge and opportunity that precision medicines represent for our system and the landscape of assessment, testing, approvals and reimbursement, as well as the growing role of charities and the patient voice. Those things are passions of mine and I want to discuss why, in the next few months and the years ahead, there will be dramatic progress.

The Government and I wholeheartedly support the cystic fibrosis campaign’s central aim of ensuring that as many people with CF as possible will have access to personalised medicines by 2020. That sets an inspiring and clear goal and I relish the attempt to deliver it. I want to make some remarks about the condition, about what NHS England and the NHS in Scotland and Northern Ireland are doing about treatment today, about the rare diseases and precision medicine landscape, and about the reforms that I am pushing to try to deal with the issues that have been raised.

I have had a career in biomedical research, so it is an extraordinary privilege to have been given my role by the Prime Minister, who has personal experience of the tragic consequences of genetic disorders affecting children. I am delighted to share with the House the fact that my passion to lead in this field, and unleash the power of the NHS and our research expertise in a new landscape for accelerated access, is exceeded only by the Prime Minister’s.

As hon. Members know, cystic fibrosis is the most common life-limiting inherited condition in the UK. It affects about 10,500 people in England—and more, of course, in Scotland, Northern Ireland and Wales—more than half of whom are adults. Cystic fibrosis is one of the UK’s commonest life-threatening inherited diseases. It is caused by a single defective gene. As a result, the internal organs and especially the lungs and digestive system become clogged. That results in chronic infections, inflammation in the lungs and difficulty digesting food.

The number of adults living with CF is gradually increasing over time, because of improvements in diagnosis from newborn screening and new treatments. The condition affects everyone differently—that is an important point—but for many it involves a rigorous daily treatment regime including physiotherapy, oral, nebulised and occasionally intravenous antibiotics, and taking enzyme tablets with food. For those who are very ill with cystic fibrosis and who have very poor lung function, daily life can be a struggle as basic tasks can leave them breathless. Some patients use a wheelchair to get around, and use oxygen to help them breathe.

For patients and their families, managing the condition is extremely challenging. That is made worse by the absence of an effective treatment or cure—or, as several colleagues have explained today, by the tantalising presence of a possible treatment or cure that cannot yet be administered to them or their suffering loved ones. I pay tribute to patients who grapple with the disease day in, day out, and who have done so for years, for their patience as we try to bring new solutions to the table. Current treatments generally target the complications rather than the cause of the condition. Treatments can be broadly classified as nutritional support, relief of airway obstruction, treatment of airway infection and, ultimately, lung transplantation.

What are the Government doing? I want first to talk about what the NHS is doing in England and in Scotland and the other devolved areas, and then to say something about what we are doing more strategically to tackle the new landscape.

Since April 2013 NHS England has been responsible for securing high-quality outcomes for patients with cystic fibrosis as part of its remit to deliver specialised services. Its service specifications for cystic fibrosis—one for adults and one for children—set out what providers must have in place to offer high-quality care and support equity of access to services for patients with cystic fibrosis, wherever they live. The NHS England cystic fibrosis clinical reference group has developed a number of clinical policies for the treatment of patients with cystic fibrosis and it reviews outcomes with the Cystic Fibrosis Trust and with patients and charities.

As we have heard, Scotland, leading within the United Kingdom—and it is not the first time—has launched a dedicated fund worth £40 million this year to give patients greater access to new medicines, as the Scottish Health Secretary, Alex Neil, has announced today. The £40 million new medicines fund expands and replaces the rare conditions medicines fund established in March 2013, giving health boards access to greater resources. In 2013-14 the rare conditions medicines fund supported the cost of 45 different medicines, benefiting more than 200 patients, including ivacaftor for cystic fibrosis as well as other treatments for related rare diseases.

NHS England is investing significant resources into the provision of new medications that work directly on the genes causing cystic fibrosis. Since 2013, it has routinely commissioned ivacaftor or Kalydeco for the treatment of cystic fibrosis in those with a certain gene mutation affecting only 5% of the CF population. Earlier in 2015, that indication was extended to an additional eight mutations for patients aged six years and above. NHS England is considering a policy proposition for extending the use of ivacaftor for the same gene mutations to children aged two to five years. It will consider the evidence base and be included with other therapies requiring investment as part of NHS England’s prioritisation process for specialised services for 2016-17.

Several colleagues raised the matter of Orkambi. Some drugs for cystic fibrosis will be considered by NICE through its technology appraisal process, including Orkambi, which, as many will know, is lumacaftor in combination with ivacaftor. NICE is currently developing technology appraisal guidance on the use of Orkambi for the treatment of patients with cystic fibrosis. It currently expects to issue final guidance in July 2016. NHS England will commission drugs where there is a positive NICE technology appraisal, and I will say something about the changes that we envisage in the landscape in that respect.

NHS England operates a horizon-scanning process to identify new treatments and the cystic fibrosis clinical reference group advises on the development of services for patients and keeps relevant published literature under review. Where NICE is not considering a therapy, NHS England can consider the evidence base and may propose commissioning treatments through its policy development process. I shall say something shortly about changes that we are considering in the way NHS specialist commissioning might embrace the new freedoms in the accelerated access review to accelerate the commissioning of rare disease treatments.

In fact, ivacaftor is something of a mild success story. NHS England commissioned it earlier than might otherwise have been expected, having agreed, in discussion with the company that makes it, a flexible pricing model. We want to see more of that sort of innovation.

I am grateful to the hon. Member for Denton and Reddish (Andrew Gwynne) for giving me some time to answer the various questions asked, which I will try to do in some detail. First, I want to set the scene in terms of why this debate is happening and why this landscape is under such pressure. The truth is that breakthroughs in genomics and informatics—our ability to understand patients’ genetic predisposition to different diseases and to respond to different drugs, as well as the availability of large-scale data sets, including individualised patient treatment histories and anonymised cohort studies—are transforming the traditional pathway for drug R and D, which normally takes years. It now takes roughly 15 years and $2 billion to bring the average drug to patients.

Genomics and informatics, particularly for some of the rare genetic diseases, allow us to take time, cost and risk out of the development pathway in a profound way. That is driving opportunity and challenge in our system; the Prime Minister created this post and put me in it to ensure we respond to that challenge with ambition.

[Julian Sturdy]

My hon. Friend the Minister is absolutely right to say that the medical landscape is changing hugely at the moment, but does he feel that the wider implications of new medicines are being fully explored by NHS England and NICE? We have heard about the huge consequences of cystic fibrosis for not only the sufferer but their wider family and the NHS. Does he feel that those wider consequences are being fully explored?

[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.