Stevens-Johnson Syndrome and Toxic Epidermal Necrolysis

Debate between George Freeman and Cheryl Gillan

Wednesday 13th July 2016 Westminster Hall

[George Freeman]

Many of these conditions have been known about for years, but it is only now that we are really beginning, through genomics and infomatics, to get a handle on how we might track, spot earlier and use big data to analyse cause and effect and develop new medicines that could intervene. Some of these conditions that have been thought of as never treatable are now becoming treatable because of the pace of biomedical progress. We need to inform our trainee clinicians not to think, “Well, I’m sorry. You’ve got a diagnosis; there is nothing we can do about it. People have suffered for 80 years.” There is a genomics programme, an accelerated access review for new medicines and an early access to medicines scheme, and we are beginning to accelerate getting new cures through into treatment. I will raise the issue of greater awareness of rare disease and what is available for them with the agencies responsible for training medical students.

The right hon. Lady raised the idea of an awareness week, which I think is an excellent idea. The truth is there are many rare diseases and I foresee a clamour for every rare disease to have a week, for which there would not be enough weeks in the year. It may be that one has a rare dermatological conditions awareness week, which would heighten awareness. There may be different ways to do that but her idea is first class. She also talked about money for research; she would not be doing her job if she did not. The Government spend a considerable amount of money on research. The NIHR has a policy of not identifying particular diseases and earmarking money to them but, following the debate, I will raise with the NIHR how much is being spent that would be relevant for sufferers of SJS. I know it is taking steps to amend its research criteria in the years ahead so that it is responding to the progress made in the genomics programme and others.

The right hon. Lady made an excellent point about gene testing. The reason I am so inspired by that quiet revolution is that we are now at a point at which we can start to gene test patients, profile them and get targeted medicines to them. That is already happening with cancer and some other diseases. For the new drugs we have launched in the NHS this year for Hep C, it turns out we can profile which patients will respond in six weeks, in eight weeks or in 12 weeks. That is driving a new model of reimbursement that sits at the heart of my accelerated access review.

Lastly, the right hon. Lady raised the important issue of side effects and the wider science of drug side effects, which the Government are investing in through a whole series of programmes in the Department of Health and NHS England. Understanding side effects can be a cue to the science of new cures. I hope she is reassured that we are taking that seriously and I will follow up—or will ensure my successor follows up, if I am no longer in post after today—the points she has sensibly raised.

[Mrs Cheryl Gillan (in the Chair)]

Order.

Motion lapsed (Standing Order No. 10(6)).

Oral Answers to Questions

Debate between George Freeman and Cheryl Gillan

Tuesday 10th May 2016 Commons Chamber

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

T8. My constituent Archie Hill and his parents, Louisa and Gary, were really excited when, on 15 April, the National Institute for Health and Care Excellence recommended that the drug Translarna—a breakthrough drug for children with Duchenne muscular dystrophy —should be funded by NHS England. Hon. Members can imagine what happened when, on 4 May, NICE unexpectedly announced that it required extra time to come to an agreement with NHS England. What is going on? We thought this drug had been cleared. Time is of the essence, because the boys affected are eligible for this drug only if they are still walking. Can we please look into this issue, and can we please go back to the original timetable? After all, these boys have had to wait several years to get to this stage.

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

I commend my right hon. Friend, and we have had numerous discussions over the last year on this subject. She can rest assured that I am actively doing everything I can to make sure we expedite this. She will understand that there are important negotiations with NHS England, NICE and the company at the moment, which are key to making sure we can get this drug accelerated quickly.

Specialist Neuromuscular Care and Treatments

Debate between George Freeman and Cheryl Gillan

Tuesday 15th December 2015 Westminster Hall

[Mrs Gillan]

Thank you very much, Mr Brady. I was not sure whether you were going to say something more from the Chair before I spoke again. May I welcome you warmly to the Chair? Perhaps we will get through this debate together without knowing too many details about the procedure.

I also welcome the Minister to his position. When one is a Minister, one sometimes finds oneself in debates where it is déjà vu all over again, as they say. I am afraid that this is probably going to be one of those debates, but it is no less serious than the debate that we had last week on this subject, and I am grateful for the opportunity that the House has given me to reinforce that debate, by allowing this debate today. I see colleagues in the Chamber who were also here last week, and they will know how passionately I feel about this subject; indeed, many of my colleagues, from all parties in the House, feel passionately about it.

I really am delighted to have an opportunity to speak about this subject, because as I think we all know, there are more than 60 different types of muscular dystrophy and related neuromuscular conditions. It is now widely accepted that approximately 1,000 children and adults for every 1 million of the population in the UK are affected by these muscle-wasting conditions, and it is estimated that some 70,000 people right across the UK are affected.

I appreciate that there are other areas that we could discuss under this topic, and I am sure that we will hear from colleagues about them. However, I will use this opportunity to concentrate on muscular dystrophies such as Duchenne muscular dystrophy, on which I have been working hard with my constituents, the Hill family, in order to gain access to a drug called Translarna.

Around 2,500 children and adults in the UK have Duchenne and almost all of them are male. The condition is caused by the lack of a vital muscle protein called dystrophin. It leads to muscles weakening and wasting over time, and to increasingly severe disability. The vital heart and breathing muscles are affected, which often causes devastating cardiac and respiratory difficulties. In older patients, assisted ventilation can be required, which necessitates 24-hour care. Some patients have to undergo a tracheostomy procedure and, sadly, few people live with this condition past their 30th birthday.

Duchenne has a huge impact on families and on the individuals who suffer from it. Only about 100 boys are diagnosed with it every year in the UK, but it is hard to overstate the devastation to the individual and the surrounding family that it causes. The diagnosis is really hard to come to terms with, and the family must deal with huge challenges as the condition progresses and the patient grows older. It usually leads to full-time wheelchair use, surgery for scoliosis, which often involves inserting iron rods into the patient’s back, and the use of full-time assisted ventilation.

As the Minister knows, there is a very brave little boy who is my constituent. He is called Archie Hill and his parents, Gary and Louisa Hill, together with his brother, Leyton, have campaigned tirelessly for access to Translarna. To put things in context for my colleagues who are here for this debate, I can do no better than to use the words, once again, of Gary and Louisa Hill, which I hope will help people to understand the devastation that this condition causes:

“Being told your child will probably die before you, has to be the most devastating thing you can tell anyone. Archie was diagnosed in 2008. Over the next couple of years we became very reclusive, barely getting out the car at school drop off, sometimes not even answering the phone...we wanted to grieve on our own (grieving is not too strong a word). We’re angry, we look at other families and wonder why us?”

They wonder why it has happened to their beautiful child. They blame themselves, even though they know it is not their fault.

The emotional effect on siblings is really apparent, although I have to say that, having met Leyton, I know he is a fantastic support to his brother and to his mother and father. He is an integral part of this team and should be equally praised for his courage and perseverance. I know that he struggles with his concentration, and that he is deeply affected by his brother’s condition, but he is also a very brave little boy coping with this in his family.

Archie faces huge day-to-day challenges. His parents say:

“He is taken out of lessons for physio on a daily basis. He suffers from…mood swings”.

I find that hardly surprising. They go on:

“Every so often he will ask us questions about his condition; does it only affect my legs? Do I always have to take this medicine? Why do I have to wear the night splints?”

He asks all the sorts of questions that a child of his age would ask their parents when they knew that they were suffering from this condition.

Despite that, Archie has great stamina, and he has spent whole days here campaigning, marching up to Downing Street and telling the Prime Minister what he wants and what the Prime Minister should do about it. Quite frankly, he is one of the pluckiest little spirits that I have ever met in my life.

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

indicated assent.

[Mrs Gillan]

I see the Minister nodding his head; I know that he agrees; anybody who has met Archie will know that what I am saying is true.

It is not just Archie who is affected by this condition. Sue Barnley, whose son Harry would benefit from Translarna, says:

“If Harry could get Translarna now whilst he is the best he is ever going to get, ie not gaining any more skills, only deteriorating then this will enable us to have more fun on a day to day basis. We gain a lot of support from our family and friends already, this will only increase as time goes on.”

She goes on to say:

“It seems cruel that the drug is there to make a massive difference in our lives, yet it is totally out of reach. Living with Duchenne is like living under a very dark cloud, we as parents know what Harry’s…future holds, to have some extra time living for the ‘here and now’ would make a BIG BIG difference, time to make and treasure some extra memories before our lives become totally engulfed by this awful disease.”

As the Minister knows, I have worked quite closely with Muscular Dystrophy UK and I have nothing but praise for that organisation, because it goes the extra mile for the people it represents. In my experience, the way that it deals with parliamentarians, offering them briefs and helping them, is second to none. It is an organisation that I trust, and I believe that it gives us the right information at the right time. It says that for older boys and men who have this condition, the respiratory function is compromised and the challenges get even greater for them and their families, because they have to engage with and face what many find to be truly frightening aspects of the condition.

One mother with whom Muscular Dystrophy UK works closely was called out to her son’s residential home at 2 am one weekend in September due to an emergency incident. Although her son was not hospitalised long term, he was experiencing increasing difficulties, and his mother told us that

“he is very conscious of his own mortality.”

Other young men are hospitalised frequently and often for long periods of time due to chest infections, which are very difficult to shift and are life-threatening. The current time of year is a frightening time for young men with Duchenne, because as we all know, respiratory infections abound, but in their case, hospitalisation is much more likely than it is for other people.

The Minister knows that Translarna is available from a company called PTC Therapeutics. We should put the benefits of Translarna in the context of a very short life. The early loss of the ability to walk is associated with a faster progression of the disease, and the later stages, as I have just described, are frightening and absolutely devastating. In a short life, the main goal is to spend as much of that life as possible in the best state of health and with the best quality of life. The National Institute for Health and Care Excellence—we are waiting for its decision on Translarna—must apply significant weight to any benefits that can be obtained through the use of Translarna in the context of that short and limited life. A delay in any of the devastating consequences of the disease, no matter how short, contributes to quality of life.

While Translarna is not yet licensed for use as an end-of-life medicine—it is still to be tested in clinical trials with older patients—evidence from existing trials shows that it delays the progression of the disease during a significant stage of a boy’s life. The trials also indicate that it is likely to delay the end of life, as a proxy measure. NICE has to give special consideration to the limited life expectancy of these boys when it is looking at this issue.

Translarna was the first licensed drug to tackle the underlying genetic cause of Duchenne and to keep boys walking for longer. Boys with the specific nonsense mutation of Duchenne, such as my constituent Archie Hill, have been waiting a year and a half since the European Medicines Agency approval in May 2014 for a decision on whether Translarna will be approved in England. It was a conditional approval, but the rubber stamp with it meant that the drug was then available in such countries as France, Spain, Italy, Germany and Denmark. That prompts the obvious question: if a European citizen can travel to any of those countries and get Translarna, why can they not get Translarna here in England?

I appreciate that there is a process that has to be gone through, and that due process needs to be followed, but it seems a cruel and unusual punishment that we have been waiting for more than a year and a half to see whether the drug can be made available to children in our country. As things stand, families face the prospect of a further agonising delay to NICE’s decision over Christmas. If I have stressed it once, I have stressed it over and over again: every day counts as those boys lose ambulation and become ineligible for Translarna.

Boosting clinical trial capacity for Duchenne muscular dystrophy is important. As Translarna becomes available for treatment, as I hope it will, it will apply only to 10% to 15% of boys with Duchenne. Other treatments are beginning to emerge. With the growth in clinical trials for Duchenne, specialist muscle centres—that is where the studies are conducted—are reporting that they are having to turn studies away due to a lack of resources and capacity. As part of a new initiative by Duchenne charities to address those concerns, Muscular Dystrophy UK has conducted an audit of clinical trial capacity and submitted that to the accelerated access review as evidence confirming that worrying picture. If the issue is not addressed, as the Minister knows—he is nodding his head in agreement—there is a risk that the promising drugs for Duchenne that are in the pipeline and in clinical trial will not continue to improve and meet their potential, hampering the search for effective treatments.

Muscular Dystrophy UK’s audit also found an excessively high clinical workload being placed on small and overstretched teams, which means that they are unable to participate in clinical trials through, for example, recruiting patients. That also means that children affected by Duchenne are unable to enrol in trials where they could access a new therapy. To aid the development of clinical trials, it is important that standards of NHS care for Duchenne patients are high across the country to ensure that patients on clinical trials are generally in the same state of health and physical shape. While there are some centres of excellence, such as Newcastle and Great Ormond Street, other parts of the country have much less developed services, and essential therapies, such as specialist physiotherapy, are not regularly provided.

Centres have also expressed concern that excess treatment costs—the additional costs of treating patients enrolled in research—are not being reimbursed to centres by clinical commissioning groups. That is a clear point of friction, and it limits the centres’ ability to take part in research. NHS England recently issued guidance on the issue, but it is not yet clear whether that will be enforced in practice.

Turning to the NICE guidelines on uncommon neurological conditions, a huge problem faces families and health professionals because there is no NICE guideline for any muscular dystrophies or neuromuscular conditions, which is why NHS England has asked NICE to develop clinical guidance on the assessment, diagnosis and referral of uncommon neurological problems. Muscular Dystrophy UK attended the initial scoping workshop on 11 November and will be participating in the consultation, which I understand has already started. While muscular dystrophies and neuromuscular conditions were listed as among the many conditions covered by the guideline and despite past assurances from NICE, there is concern that the focus on muscle-wasting conditions might be minimal unless the complexities of the conditions are highlighted. Given the internationally recognised standards of care for Duchenne and spinal muscular atrophy, it is disappointing that the NICE guidelines that are being developed are far more generic than the original guideline proposed by NICE to cover uncommon neuromuscular conditions in a letter to Muscular Dystrophy UK in November 2013.

Muscular Dystrophy UK has proposed that the guidelines should address the following: paediatric neuromuscular services specifically for Duchenne muscular dystrophy, in conjunction with current guidelines; the use of steroids as effective therapy in terms of the age when the optimal effect can be achieved, whether there should be a continuous or intermittent dosing regime, and how to manage the side effects; spinal surgery to correct or prevent scoliosis, with evidence regarding the optimum age and management pre and post surgery; and respiratory support, with a comparison of the evidence regarding invasive and non-invasive interventions, including comparisons with experiences in Denmark, where evidence suggests that men with Duchenne are living into their 40s because of the relatively high standard of respiratory support. So far as adult neuromuscular services are concerned, the guidelines need to address: diagnosis and the importance of GPs recognising the conditions, making early referrals and ensuring effective links from primary into tertiary care; respiratory support, as I have talked about before; and cardiac support, including regular monitoring to detect and address the deterioration of the heart through the progression of muscle-wasting conditions.

High costs can be involved in unplanned emergency admissions due to Duchenne and other muscle-wasting conditions, and in living with such conditions. There is a cost attached to not taking action to implement preventative care. Access to specialist multidisciplinary care, including access to respiratory, cardiac and physiotherapy support, can contribute to reducing avoidable, unplanned emergency admissions to hospital. A clinical audit of emergency hospital admissions that was led by Professor Mike Hanna revealed in June 2012 that 40% of these costly admissions could have been prevented if patients had been able to access expert tertiary care, specialist physiotherapy and—this is the worst finding of all—vital medical equipment. It has been estimated that addressing those issues could save the NHS more than £32 million a year as the appropriate proportion of NHS spending on neuromuscular services.

The cost of living with Duchenne is enormous. In the first study of its kind, academics found that the overall care for each patient with Duchenne cost the UK economy about £71,000 a year, giving a national total of £120 million. That survey was led by Newcastle University and a team in Sweden. Some 770 patients and their primary caregivers in the UK, Germany, Italy and the US were asked to complete a questionnaire on their experience of living with Duchenne and its impact on their need to access medical care, employment, leisure time and quality of life. The direct cost of the illness across all countries was at least eight times higher than the average health expenditure per person, and the figure for the UK was 16 times higher. The overall figures included medical treatment as well as the cost associated with the loss of employment among caregivers. In the UK, nearly half of caregivers reduced their working hours or stopped working completely owing to their relatives’ Duchenne muscular dystrophy. I therefore have several questions that I hope the Minister will answer during his winding-up speech.

When we discussed access to medicines last week in Westminster Hall, the Minister mentioned that he had made contact with NICE about both Translarna and Vimizin. I hope he feels that he may have reached a point with NICE such that he can talk about those drugs. I understand that they are used in similar situations, so if there is good news about Vimizin, we hope there will be good news about Translarna, and vice-versa.

Will the Minister provide more details on ensuring standards of care for muscular dystrophy and neuromuscular conditions? I really hope that clinical trials will be developed, so will he say a little more about that? I also want him to ensure that NICE gives more prominence to muscular dystrophy and neuromuscular conditions in the development of the uncommon neurological conditions guidelines, as was outlined in the original proposal.

Lastly, I hope the Minister will join me in congratulating Muscular Dystrophy UK on its work to develop information and resources for people with muscle-wasting conditions and to support health professionals through its “Bridging the Gap” project. More than 400 GPs and 150 physiotherapists completed the online learning modules about muscular dystrophy. The charity has sent out 4,500 alert cards for specific muscle-wasting conditions and 300 care plans, which is a positive step forward to improve how we treat and look after our patients with Duchenne.

I finish with a plea to the Minister. When I asked for this debate, as he knows, it was entirely based on trying to get Translarna cleared for Archie Hill. The Hill family went on holiday today, I think shortly before the debate began. I do not know what the Minister can do to speed the process along but, for the Hill family and Archie, and for all the other children and their parents at this time of year, if the Minister could ask NICE to bring forward a positive decision on Translarna, it would be the best Christmas present that any parent or child could get.

--- Later in debate ---

[George Freeman]

In the few minutes I have available, let me say a few things about the main issues raised. I pay tribute to Muscular Dystrophy UK, Robert Meadowcroft, Emily Crossley, the Duchenne Children’s Trust, Action Duchenne and all the other organisations that work so hard in this area, and specifically on the two or three key drugs.

I remind the House that the decision from NICE on Vimizim is due before the end of the year. Without breaching due process, I have asked that, if that decision is in the pipeline, it can be made as quickly as possible, ideally before we all break up for the Christmas holidays. That is not in my gift, but I made that request. Similarly, I have requested that the Translarna decision, which I believe is due in February, is similarly expedited. However, again, that is not in my gift, and while during the year the Prime Minister and I have urged NICE and NHS England to do everything they can to expedite their decision making on those drugs, we do not have the power—rightly, in my view—to step in and breach process. It is fair to all patients in the NHS that decisions are taken properly.

[Mrs Gillan]

The Minister has given us a sympathetic hearing and I know that he has done everything in his power to try to bring forward that decision. Quite rightly, it is not his decision to make, but, through him, may I appeal to the men and women who are making that decision? If they have any humanity about them, they ought to make a positive announcement before Christmas.

Cystic Fibrosis

Debate between George Freeman and Cheryl Gillan

Tuesday 8th December 2015 Westminster Hall

[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.

Oral Answers to Questions

Debate between George Freeman and Cheryl Gillan

Thursday 12th February 2015 Commons Chamber

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

T5. The Minister responsible for life sciences will be familiar with Daiichi Sankyo, an innovative drug company based in Chesham and Amersham. It is launching a novel oral anticoagulant to help prevent strokes more effectively in people who suffer with atrial fibrillation. The uptake of such drugs has been repeatedly blocked by the NHS despite guidance from the National Institute for Health and Care Excellence that calls for their use. What can the Minister do to clear that blockage so that the drugs can be used to benefit our patients?

[The Parliamentary Under-Secretary of State for Business, Innovation and Skills (George Freeman)]

I am indeed familiar with the great work of Daiichi Sankyo, and that of my right hon. Friend in supporting its investment in her constituency. I recently had the pleasure of going up to open a new facility. She raises an important point, and the appointment of a Minister responsible for life sciences at the Departments for Business, Innovation and Skills and of Health, where I have responsibility for NICE and the Medicines and Healthcare products Regulatory Agency, allows us to begin to ensure that our health system better supports our life sciences cluster. The review I recently launched of speedier access for innovative medicines will tackle the issue of uptake that my right hon. Friend has rightly raised.

Drugs (Ultra-rare Diseases)

Debate between George Freeman and Cheryl Gillan

Tuesday 20th January 2015 Westminster Hall

[The Parliamentary Under-Secretary of State for Business, Innovation and Skills (George Freeman)]

I congratulate the hon. Member for Leeds North West (Greg Mulholland) on his tireless work on this issue, and colleagues across the House, including the hon. Member for Blaydon (Mr Anderson), my right hon. Friend the Member for Chesham and Amersham (Mrs Gillan) and others here today.

I thank the hon. Member for Leeds North West for his kind words about the work that I have been trying to do for him, and about the Prime Minister’s signal of support. The issues are incredibly complex and do not lend themselves to an easy waving of a ministerial wand, but we are committed to finding a solution.

The hon. Gentleman has been tireless in his support of one of his constituents, six-year-old Sam Brown from Otley, who has the very rare Morquio syndrome. A new treatment is now available called Vimizim, from which Sam has already benefited as part of a clinical trial. I wish to state my support for Sam and his family, and for all those who suffer from the disease, including those in the trial who have access to the drug when others currently do not. I also pay tribute to the hon. Gentleman’s support for the family of another young boy, Archie, who has Duchenne muscular dystrophy, a very rare form of muscular dystrophy that affects only boys. Archie’s family want him to be treated with a new medicine, Translarna.

I will say a little about the background to the diseases and what we are trying to do about them. Both conditions are very rare—there are about 80 children living with Morquio syndrome in England, and about 140 boys with Duchenne muscular dystrophy—so we are talking about a very small number of children with those life-limiting conditions. However, rare diseases are not rare: there are between 5,000 and 10,000 known types of rare disease, and an estimated one in 17 people will be affected by a rare disease in their lifetime, amounting to some 3 million suffers in the UK alone.

The truth is that the more we know about the human genome and the behaviour of genes in disease development, the more we understand its complexity. In cancer particularly, we know that the tumour itself mutates at different stages of the disease. The more we know about genetics, the more we discover that diseases that we thought yesterday were one disease in fact break down into different bundles of rare disease. New knowledge, technology and advances in biomedicine are a wonderful thing, but that does not detract from the fact that the NHS operates with finite resources and that difficult funding decisions must be made daily.

I was delighted to meet Sam’s mother and Archie’s family early in December, along with the hon. Gentleman and representatives of the Society for Mucopolysaccharide Diseases, to whose work I pay tribute, and of the Muscular Dystrophy Campaign. As the hon. Gentleman mentioned, we had a number of meetings over the Christmas period. I was delighted to meet patient groups and the manufacturers of Vimizim and ataluren just before Christmas. In that meeting, I asked the patient groups and companies to set out their proposals, which they have now done. I am grateful to them, and I have passed on that information to NHS England.

This morning, I met NHS England’s clinical director of specialised services, James Palmer, and its director of specialised commissioning, Richard Jeavons, and I will convene a further meeting shortly to pursue the issues that the hon. Gentleman has raised this morning. Since he first made me aware of this issue, I have been absolutely determined to bring as much ministerial focus to it as I can. I am also grateful for his acknowledgement of the Prime Minister’s support. The Prime Minister and I are both determined to ensure, without compromising due process, that the case for these children and their families is properly heard, and that the system works as it is supposed to.

I am acutely aware of the urgency behind the hon. Gentleman’s comments today and that is why I have taken the unusual step of trying to broker an agreement on what we might do to help children affected by these diseases, but I must stress that it is for NHS England, which in the end is the responsible commissioner, to make any decisions about making funding available so that the treatments are available on the NHS. It will act on the best clinical advice from the UK’s specialist body, the National Institute for Health and Care Excellence.

I will say a little more about the options for accelerating that process in a moment, but first I will talk about our approach to improving access to treatments for rare diseases generally, because I know that this debate is being watched closely by others who have an interest in a number of other drugs and conditions, in the commissioning process, and in NHS England’s prioritisation and decision-making framework. In setting the scene, I remind right hon. and hon. Members of the pressures that the NHS faces, particularly on budgets for rare diseases. The emergence of new treatments, the increasing personalisation of medicines, the end of the one-size-fits-all model and the possibilities offered by the rapid advances that we are making in genomic medicine and diagnosis are all putting immense pressure on NHS England’s resources for the commissioning of services for rare conditions.

Ideally, of course, we would want to fund all the treatments that are shown to benefit patients in any way, but we have to make difficult decisions about how we spend the money that we have available. That is why we have put clinicians in charge of the process, so that they can make decisions based on patient benefit and on the best health economic assessments that we can make. The painful truth is that with finite resources, when we make a decision in one case to accept a drug, we will make a decision elsewhere to reject, and we have a duty to all to ensure that we make those decisions fairly.

For people with rare conditions, their families, carers and clinicians, having access to the latest and most effective treatments is obviously critical, and I am absolutely committed to ensuring that patients with rare diseases have access to the latest and most effective treatments that represent value to the NHS and the taxpayer, as well as delivering benefits to patients. That is why we recently introduced the early access to medicines scheme, which aims to give patients with life-threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation or licence where there is clear unmet medical need. I am delighted that initial products have been brought forward in the last six months under that scheme.

More generally, our strategy for life sciences sets out an ambitious longer-term plan to improve the wider environment for health and life sciences companies in this country. Recently, I launched a major review of the landscape in the UK for bringing innovative medicines and medical technologies to patients much more quickly, and I will soon announce the chair, the terms of reference, the scope and the timetable of that review.

We are not in any way complacent. The truth is that the challenges in this sector, which are being driven by the pace of technological change, demand that in our policy-making framework, in the Department of Health and in NHS England, we adapt the way in which we handle these processes. Because of their rarity and the low patient populations, services for rare conditions are directly commissioned nationally by NHS England as specialised services. They account for approximately 14% of the total NHS budget and represent spending of about £14 billion a year. Both Morquio syndrome and Duchenne muscular dystrophy fall within these national specialist commissioning arrangements.

As right hon. and hon. Members are aware, NHS England is considering draft clinical commissioning policies for both Vimizim and ataluren. I understand that they are being considered as part of NHS England’s wider prioritisation process for funding in 2015-16. NHS England’s clinical priorities advisory group formulates recommendations on the commissioning of new treatments for rare diseases in England. It is made up of clinicians, patient representatives and commissioners of health services.

In summer 2014, a decision-making aid for the prioritisation of new interventions and treatments was developed by a partnership of stakeholders, including more than 250 patient representatives. It was due to be used for the first time in early December 2014, but on 28 November 2014 NHS England decided to postpone its introduction, in response to concerns that some patients affected by rare diseases might be disadvantaged by its application. The legal process about that must now run its course. I understand that NHS England is, rightly, reviewing the appropriate approach to prioritising new treatments and interventions within specialised commissioning in response to those concerns. A 90-day consultation on the prioritisation framework and decision-making process for commissioning decisions on new treatments will be launched by NHS England shortly. This morning, I again raised the importance and urgency of that consultation process.

I know that patients and their families are understandably concerned that it may take a long time for a decision to be made by NHS England on whether it will fund the drugs, and that in the interim the children affected will not receive them. However, I am delighted to say that NHS England has assured me that the consultation will have no impact on the decision-making timetable for commissioning NHS services from April 2015 onwards. In addition, it has assured me that existing treatments will continue to be commissioned, ensuring that support for patients is maintained. NHS England understands that the manufacturer, Bio Marin, is providing Vimizim under an expanded access arrangement to those patients who are on the clinical trial until an NHS England policy decision has been made.

Since April 2013, NICE, which is responsible for the evaluation of selected high-cost low-volume drugs under its highly specialised technologies programme, has been playing an important role in ensuring that commissioning decisions are based on a robust and thorough assessment of the available evidence. NICE has recently been asked to evaluate Vimazim under this programme, and it is also considering whether to develop guidance on Translarna. That is a very positive step, and I look forward to receiving NICE’s proposals on future topics that will be considered. I know that NICE will also be keen to learn lessons from its recent experiences with the new highly specialised technologies process, to make that process as efficient and effective as possible.

For my part, I am absolutely determined to continue playing the active role that I have taken on in the last few months, to drive this process and give it the focus that it requires. I am delighted to have confirmed with NHS England that it will continue to meet the treatment costs. I have signalled, and will continue to signal, to NICE, without compromising its processes, the strength of the case that has been made by Members and patient groups to put Translarna on the list, and to consider whether it can expedite its process in any way, but I do not want to compromise that process in any way. I will also ask NICE to ensure that it uses its review of the experience of the HST programme to explore how we can speed up both this process and others in due course.

Finally, I am committed to continuing to work with the companies to see whether I might be able to help broker some kind of planning arrangement that might encourage NICE to make the decision that I know everyone in Westminster Hall today would like to hear.

[Mrs Gillan]

I am grateful to the Minister and I congratulate him on taking up the cudgels on this issue and trying to move it forward. The Muscular Dystrophy Campaign has asked whether the individual funding requests from patients would be a route to secure access to Translarna while the Minister is waiting for due process to take its course, because I am afraid that muscular dystrophy waits for no man and no process.

[George Freeman]

I understand; my right hon. Friend makes an important point. In fact, I raised it this morning in my meeting with NHS England. My understanding is that NHS England will continue to consider individual applications for Translarna through its individual funding request process from patients who may be exceptional. However, my understanding is that such cases really do have to be exceptional. In reality, the members of the whole group that we are considering are more or less suffering from the same condition and therefore they may not qualify under those criteria. I merely share that with my right hon. Friend because I myself raised that point this morning with NHS England.