Cystic Fibrosis

Cheryl Gillan Excerpts

Tuesday 8th December 2015 Westminster Hall

[Mrs Cheryl Gillan (Chesham and Amersham) (Con)]

It is a pleasure to serve under your chairmanship, Sir Edward. I warmly welcome the Minister, who, I am afraid, is very familiar with what I am speaking about today; I hope he gives me an A for effort and persistence. Given that we have spent so much time discussing access to Translarna, perhaps in his winding-up speech he will have some good news for me and my constituent.

I congratulate the hon. Member for Dudley North (Ian Austin). I am absolutely delighted that he secured this debate on access to medicines for people with cystic fibrosis and other rare diseases. Like me, he knows how important this issue is for families up and down England. I have been looking at the issues surrounding Duchenne muscular dystrophy for what seems like many years—in truth, it has been for just over a year. Only 90 boys affected by the disease in England are eligible for this drug, and the number is slightly larger across the whole of the United Kingdom.

Duchenne muscular dystrophy is a devastating condition that leads to full-time wheelchair use between the ages of eight and 11. It is a progressive, muscle-wasting disease that eventually affects the muscles involved in the respiratory and cardiac functions. Sadly, few with the condition live to see their 30th birthday. I have been working with Muscular Dystrophy UK, which fights causes to do with muscle-wasting conditions. I pay tribute to that organisation for all the support and help it gives. It not only informs Members of Parliament, but helps people affected by those diseases. My constituent, young Archie Hill, is an inspiration to everybody in this area. He has been campaigning for many years, and he and his family are indefatigable in their efforts to get the right medicine at the right time to these boys.

[Mr Jim Cunningham (Coventry South) (Lab)]

I congratulate my hon. Friend the Member for Dudley North (Ian Austin) on securing this timely debate. As the right hon. Member for Chesham and Amersham (Mrs Gillan) will recall, some months ago we all went to Downing Street to petition to get something done about muscular dystrophy. I am sure she would agree that one of the big problems is that even if the new treatments are okay, there is always a long run-in, in which negotiations take place between the Government and the pharmaceutical companies.

[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 ---

[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.

[Mrs Gillan]

rose—

[George Freeman]

I had better crack on; I will come to the questions that my right hon. Friend the Member for Chesham and Amersham (Mrs Gillan) asked later, if I may.

Hon. Members have raised a number of questions and I want to deal with them all. The hon. Member for Dudley North asked about timings for the accelerated access review. We have had an interim report. I have asked for final recommendations in the spring—in March or April—and also that the review considers whether the process should go on. I want recommendations that we can implement quickly, but equally these discussions are complex and we may well need to go on to look at other bits of the landscape. I would be delighted to meet the Cystic Fibrosis Trust—in fact, that is already arranged; we are meeting this afternoon at the summit that I have organised.

The hon. Gentleman also asked about safeguards, which is a very important point. Although all of us share a recognition of the need to accelerate access, nothing in what is happening must in any way undermine patient trust and confidence in safety and protections. That is an important balance to strike. Nothing in what we are doing in any way looks at changing the legal basis in terms of negligence, consent or the clinical trials framework. The issue is about ensuring that our systems have the flexibilities to embrace the very latest science, and particularly, in this case, genomic biomarkers.

The hon. Gentleman asked about amending the NICE appraisal process to weight wider societal costs. At this point, the review is not specifically looking at the internal mechanics of NICE’s current high-technology appraisal process, but we are looking at giving it, with its new flexibilities and freedoms, a suite of different types of innovation that might come through. We are particularly looking at where that can be ring-fenced and targeted at particular patient groups.

The hon. Gentleman asked about a ring-fenced fund. As I said, we are looking at the allocation that we have had from the Treasury, which is about £4 billion extra on drugs in this Parliament, £2 billion of which is more or less the existing demand driven by demographic change. There is about a £2 billion allocation in there for new medicines. The difficulty is that the drugs that we might consider now to be most worthy of ring-fencing and accelerating may not be the ones that in five years’ time, on the basis of the clinical evidence, we look back on and say, “Why did we not accelerate that?”

We want to make sure, through the AAR, that we are putting in place a system that gives us the flexibilities to pull through those drugs that have the most transformational effect. But let me be clear: we are looking at wanting to build in, over the next few years, a wider understanding of the real costs to our health economies—local and national—of different forms of disease. That is why the Secretary of State and I are leading on per-patient costing so that, in due course, we can develop a more intelligent system to reflect that.

The hon. Gentleman asked about the NIHR and specialist muscle centres. This debate covers a number of different disease areas, and it is a tribute to the NIHR’s research network that more and more charities are now wanting to build centres of excellence. In the forthcoming NIHR five-year funding cycle, looking at the biomedical research centres and the biomedical research units, I am keen to make sure we consider where we can bring funding in from charities to complement that core research network.

Finally, the hon. Gentleman asked about the accelerated access review and the powers that we are looking at for NICE and NHS England. I do not want to pre-empt the findings of that independent review, but I have asked that the review looks precisely at how we can make it easier for NICE and NHS England to work more closely together. Specialist commissioning would be an obvious place to start to share those data and look at how we can get a better deal for everybody—for patients, the system and the economy.

My right hon. Friend the Member for Chesham and Amersham asked about Translarna and Vimizim and how quickly we may be able to get good news for Archie. I pay tribute to my right hon. Friend; she has been a very doughty campaigner on this matter during the last year. I share her frustration that in the existing system, due process has to be gone through and that, although we have expedited this as much as we can, it has taken a long time. I pay tribute, as she has, to Archie. He, like so many of these patients, is an inspiring example of the very best of this sector and of this country. They are people who have the most reason to complain, but tend to be the least likely to and the most inspiring, given their generosity about the system and their demand that we take their suffering and use it to make sure that others do not have to suffer.

I have touched on the timetable. I am very hopeful that we should get a decision from NICE on the basis of the secondary consultation early in the new year.

[Mrs Gillan]

I thank the Minister for the way in which he is still pursuing this matter on behalf of my constituent and the other boys. However, does he share my frustration? I know we have to go through due process, but why does due process have to take so long? Every day matters to these children and to their quality of life. I cannot impress enough on the Minister, NICE and anybody else watching this debate, that due process must be executed in a more timely fashion. This is nothing short of torture for these boys and these families. I know that the Minister has tried very hard, but I just hope that the people at NICE will be listening to this. I appeal to them directly through him to make a positive decision on this before Christmas; it would be the best Christmas present that these boys and their families could have.

[George Freeman]

My right hon. Friend makes her point as powerfully as ever. I shall not add to it; it has been put on the record very clearly.

My right hon. Friend asked about contact with the company. It is not for Ministers to get actively involved—much as, at times, I would like to—in negotiating these deals, but I have made contact with the company, both on Vimizim and Translarna, to urge it to be as flexible as it can in discussions. I can only say that I am hopeful that it will have been able to reach a point where NICE feels able to make a recommendation.

Part of the reason why due process is important is that when NICE makes a recommendation, NHS England is bound in law to provide the drug in perpetuity, so it is a major cost undertaking. In some cases, these drugs cost £200,000 or £300,000 a year, so it is a commitment of several hundred million pounds from NHS England. Other patients would say, “We must make sure that when you make a decision like that, it is done properly.” However, I share my right hon. Friend’s frustration that a lot of these breakthroughs scientifically mean that we ought to be able to speed things up.

My right hon. Friend asked whether the Prime Minister is holding my feet to the fire. She need not worry; I am as passionate about this as ever and very impatient to make sure that the AAR is landed with some good recommendations.

My right hon. Friend made an excellent point about NIHR staffing. I am working with the chief medical officer and the NIHR on that at the moment. A number of our clinical research facilities could, with a few more staff, turn over more and do more trials work. There is an opportunity for us to get more people internationally to enrol in NIHR training—in clinical trials and translational research training—which would give us more capacity and allow us to move things along faster.

The hon. Member for York Central (Rachael Maskell) raised an important point about cost. I have touched on the work that we are doing on per-patient costing to try and make sure that we develop a system that more intelligently captures the real cost of disease.

I am grateful to the hon. Member for Denton and Reddish, the Opposition spokesman, for his comments. I congratulate him on the by-election victory. He asked about NICE reform, which I have touched on, through the AAR. We do not want to interfere with or undermine NICE’s independence and their “gold standard” reputation, but we want to create a place in which the accelerated access review gives them the freedoms that they are, indeed, helping to shape.

In conclusion, this debate has highlighted not only the challenges from the rising costs of new drug discovery—£200,000 to £400,000 a year for patients in the rare disease space—and the pressure on the one-size-fits-all model of assessment, but the opportunities for us to unleash our leadership in genomics and informatics to create a new landscape. That is why this week, the Association of Medical Research Charities conference and my summit this afternoon, and the accelerated access review work is creating momentum for a new landscape for accelerated pathways for patient-led innovation.

I think we will look back in two or three years at this as a crucial turning point at which the system that was set up to assess a very one-size-fits-all, 20th-century model was rapidly adapted, creating new opportunities for patient-led innovations and charities such as the CF Trust to bring through innovations that benefit their patients more quickly.