Specialist Neuromuscular Care and Treatments — [Mr Graham Brady in the Chair]

– in Westminster Hall at 2:30 pm on 15th December 2015.

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Photo of Dame Cheryl Gillan Dame Cheryl Gillan Conservative, Chesham and Amersham 2:30 pm, 15th December 2015

I beg to move,

That this House
has considered access to specialist neuromuscular care and treatments.

Photo of Graham Brady Graham Brady Chair, Conservative Party 1922 Committee

Mrs Gillan, you have the opportunity to speak to the motion.

Photo of Dame Cheryl Gillan Dame Cheryl Gillan Conservative, Chesham and Amersham

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.

Photo of Dame Cheryl Gillan Dame Cheryl Gillan Conservative, Chesham and Amersham

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 life and with the best quality of health. 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.

Several hon. Members rose

Photo of Graham Brady Graham Brady Chair, Conservative Party 1922 Committee

Order. Five or six Members want to participate and I want to get on to the winding-up speeches by no later than 3.35 pm. Although there is no formal time limit on speeches, if Members can keep their remarks to around six or seven minutes, we will be able to accommodate everybody.

Photo of Rachael Maskell Rachael Maskell Shadow Minister (Defence) 2:53 pm, 15th December 2015

It is a pleasure to serve under your chairmanship, Mr Brady. I congratulate Mrs Gillan on speaking with such passion once again about her constituent and neuromuscular disorders. Such disorders often do not receive the profile of other medical conditions, yet for each of the 70,000 people affected, they are all-encompassing. I want to talk about a range of services and the challenges within them. I recognise that the conditions affect adults as well as children. I declare my interest as a physiotherapist who has worked in this field in the NHS for 20 years and is registered with the Health and Care Professions Council. I want to examine three themes: specialist services and how they are delivered; transition; and a timely response at the time of need.

I will deal first with specialist services. A multidisciplinary team is essential for delivering services to people with neuromuscular disorders, but access can often be challenging. District hospitals have teams of staff who specialise in paediatrics, neurology or other fields, but those people might not have the specific skills that are needed when dealing with neuromuscular disorders. Practitioners are practised in the principles of such conditions, but might not be as familiar with particular syndromes, given that there are so many—60 or more, as we have heard. This situation is likely to be exacerbated in the community when general practitioners and community physios do not have the specific skills, so it is important to ensure that people with these conditions can access those with the right skills who understand the pathology of the disorder and the specialist treatment that is required.

For instance, there are two specialist centres in London and one in Oxford for the whole of the south-east, so people have to travel vast distances to consult a specialist team. Owing to the nature of their disability, that can be very challenging, and the centres can even prove to be inaccessible, especially as their disease advances. How do we bring specialist services to those with neuromuscular disorders? How do we train staff to provide optimum care in the community, and how do we provide a rapid self-referral service when that is needed?

If I take muscular dystrophy as an example, paediatric patients in York benefit from Leeds general infirmary’s outreach service. That provides an opportunity for families to meet specialist practitioners but, obviously, some have to travel to those services. Will the Minister ensure that funding for that outreach hub-and-spoke model of service provision will continue and that clinicians will be able to travel to deliver their services, either individually or as a team?

It is important that services are placed in appropriate locations. For example, if a patient will benefit from hydrotherapy, we need hydropools to be available. Anisa Kothia, a member of the York muscle group, has a son, Yusef, who has Duchenne muscular dystrophy, and hydrotherapy is a vital component of his treatment. It relaxes his muscles so that his limbs can be taken through their range of movements, and the buoyancy of the water helps his movement. Will the Minister support a national review between the Department of Health and the Department for Communities and Local Government into hydrotherapy provision and ensure that any deficits are addressed?

Ongoing services rely on clinicians with less of a specialism, however, so that requires professionals to be trained and the provision of regular updates, which is why Gita Ramdharry, associate professor at St George’s,

University of London, and Kingston University, has been working with Muscular Dystrophy UK to develop new online physiotherapy training. Will the Minister set aside resources to ensure that we can have specialist online training to equip professionals to provide the optimum treatment? Obviously, more specialist care training is also needed which, for physio, can be very hands-on.

I have had discussions with senior clinicians in other fields about global medical education. I think that that is relevant for neuromuscular disorders, because such disorders often require a global view that enables recruitment from around the world to participate in webinars, to examine case studies and academic papers, and to demonstrate learning by making a submission. We need to take medical training to another level, so will the Minister look at that more deeply, particularly with regard to neuromuscular conditions?

Before I move on, I want to highlight that much of the care for long-term progressive conditions is provided by the family. Good self-management is key, as Labour’s expert patient initiative has proven. If the professional knows the patient and their condition, the treatment will be optimal and will provide the best support. For example, a chest infection often accompanies a neuromuscular disorder, and a chest physio who knows the patient will know how best to support sputum clearance with a combination of the best postural drainage, manual support, and expectoration or suction techniques. Knowing exactly what the patient needs is critical, and can be life-saving.

All physios have the competencies required to treat a patient, but knowing the individual can make the difference. Rapid access to services can be transformative, and self-referral is very important, so will the Minister ensure that all services provide rapid-access routes to the appropriate clinicians and that all patients can self-refer, rather than having to go through the normal access channels? In north and west Yorkshire, we have only one neuromuscular care adviser to cover more than 3,500 adults and children. Will the Minister recognise the need to provide additional professionals in that role, including in north Yorkshire?

I have previously talked about the need to review the transition between child and adolescent mental health services, and adult mental health services. We should do the same for neuromuscular disorders, because using someone’s age as a measure is arbitrary. The pathology of Duchenne muscular dystrophy is more likely to be understood in paediatric services than adult services, owing to the number of children who, sadly, still do not make it to adulthood. A person’s medical team and physios know that individual and know how to progress their treatment. It is entirely arbitrary and nonsensical that someone’s birthday should determine that they have to transfer to another team.

Continuity of service provision is important. The condition of those who do reach adulthood is often at an advanced stage, so they need continuity. The findings in the field are that young adults are often lost in the service and then re-emerge later with problems that were preventable. Will the Minister take a serious look at the interface between paediatric and adult services right across the Department of Health, and particularly with regard to neuromuscular disorders, because surely specialism should override age?

We should be making a timely response to need. A worsening situation has been observed across the spectrum of neuromuscular disorders. It has been seen by clinicians in practice, and now constituents are writing to me about it. Orthotics, wheelchairs and equipment must be in place when they are needed. Infants and children grow, and disease processes may degenerate, so the combination of the two means that expediency is important. Patients are waiting far too long for appropriate equipment, and that is essentially an issue of under-resourcing and poor prioritisation.

If someone is measured for a chair, they need that chair, but people are waiting month upon month before their chair arrives. While they are waiting, they will be positioned inappropriately and might not even have enough support for their frame. That can exacerbate pain, as well as compromise a patient’s musculoskeletal situation and, dangerously, their respiratory function. There is absolutely no excuse for that. When a chair arrives, a patient needs it, so we need to ensure that we get the right equipment in the right place at the right time.

Planning for what equipment will be required is part of the management process, because people must always be prepared for the next stage, and the outsourcing of services has made the situation far worse. With life-limiting conditions, there is no time to wait. Will the Minister agree to carrying out an urgent review of the situation? Will he ensure that, starting on 1 April 2016, there will be a waiting-time marker for the renewal and provision of equipment so that the time between the initial request for an assessment and the patient receiving the equipment is measured?

For someone with a rare condition, their future depends on the whole NHS and care service working around them to provide optimum support. I have not touched on research and pharmaceuticals, nor on advances in science, but there are things that can be done immediately that can really change someone’s outcomes. We need the best provision and to give individuals hope to ensure that we can extend their life and improve their quality of life. I look forward to hearing the Minister’s response.

Photo of Caroline Nokes Caroline Nokes Chair, Speaker's Advisory Committee on Works of Art 3:04 pm, 15th December 2015

As ever, it is a pleasure to serve under your chairmanship, Mr Brady. I pay tribute to my right hon. Friend Mrs Gillan, who secured this important debate today. It is on a recurring theme which she brings us back to time and again. Not only does she work tirelessly to highlight the case of her constituent, Archie Hill, but she is extremely knowledgeable, so I will endeavour not to repeat any of her comments because I want to give other Members the chance to contribute.

Like my right hon. Friend, I have a constituent who suffers from Duchenne muscular dystrophy. Jagger Curtis is a pupil at Romsey Abbey primary school and is just eight years old. I have highlighted Jagger’s case in this Chamber before and I have raised it twice at Prime Minister’s questions, but, like Archie, Jagger is still waiting for an answer on whether he will be allowed to have Translarna. I will focus briefly on the issue of treatment, but mainly I want to echo my right hon.

Friend’s calls, asking when families such as Jagger’s can expect to be notified of guidance from the National Institute for Health and Care Excellence on access to Translarna.

I know the Minister is aware of the time sensitivities of access to this drug—it has to be prescribed while the patient is still mobile—so I ask him to consider meeting me and my constituents, Jules Geary and James Curtis, who are Jagger’s parents, to discuss how the process might be accelerated. It might not necessarily be accelerated now, for their son, but it should be for the other hundred boys who will be diagnosed with Duchenne muscular dystrophy every single year. We have been waiting many months for a decision on Translarna, and every deadline appears to result in a decision to prolong matters further. There are concerns about the clinical trial capacity for drugs developed to assist neuromuscular conditions; will the Minister please assure me that he is actively promoting the network of specialist muscle centres as a means to overcome that problem?

Other issues associated with Jagger’s care have also raised huge concerns. He has had to wait far too long for his specialist wheelchair, and there seems to be little understanding that the chair is a lifeline for Jagger and his parents. Last week he went on holiday to Tenerife, and his wheelchair arrived literally just in time—the very day before he went on holiday—despite the fact that it was ordered back in May. Rachael Maskell referred to the fact that children grow and change, and their needs change. It seems to be an absolute tragedy that a child can wait all those months, and then by the time the chair arrives the danger is that it will no longer be correct for their condition.

Jagger still has his mobility, but his parents have done an arguably very difficult thing in making sure that he has that chair. Inevitably, as a child with a condition in which his muscles deteriorate, he tires really easily, so that chair is his lifeline and his access to continued mobility now and in future. He is still suitable for treatment with Translarna—he is one of the boys who has the nonsense mutation—but his family feels as though the clock is ticking very quickly.

Muscular Dystrophy UK has highlighted the problems with wheelchair provision throughout the country. Clinical commissioning groups are now responsible for commissioning wheelchair services. I am really conscious that in west Hampshire there have been delays for many people, not just Jagger. For those with neuromuscular conditions, as the hon. Member for York Central said, it is all about getting the right chair at the right time. It can be particularly devastating for children to have to wait for a chair that enhances their freedom.

I recently saw that Jagger’s mother had posted on Facebook a picture of him proudly showing off his new chair. However, it also said that the family had launched a GoFundMe page to buy a powered sitting and standing chair to enable Jagger to live his life as fully and actively as possible. It is heartbreaking that they are trying to fund that through GoFundMe because they do not have the confidence that the CCG is going to provide that sort of facility for them.

In the south-east we lag behind the rest of the country in the provision of neuromuscular care and adviser support. As we have heard, there are two specialist treatment centres in London and one in Oxford. That is a two-hour journey for a child in a wheelchair, coming via Waterloo, and his parents tell me that it is incredibly difficult for them to do that whenever Jagger needs to come to London for treatment. There is also a problem with specialist guidance. These families are looking for support—they need support, advice and information.

We are lucky that in Romsey we have brilliant GPs, but Jules Geary told me the tale of trying to get Jagger diagnosed. As a first-time mum, she was often dismissed as a worried mother, when in fact she was the one who knew her son best and knew that there was a problem. I do not blame the GPs at all, because if 100 boys are diagnosed every year, one would not expect a GP in Romsey to see it very often. It was not until James read an article in the Daily Mail about Duchenne muscular dystrophy that he pointed his finger and said, “Those are the same symptoms that Jagger has.” They took that article to the GP, and it was only then that the specialist tests were done on Jagger.

I know that we keep returning to this subject, but that is because it is important. It is right that we keep returning to it. Quality of life, especially for children, is crucial. I hope the Minister will look round this Chamber this afternoon and hear the cross-party and apolitical calls for help for those with such conditions, and I hope he will address some of the points that we raise.

Photo of Margaret Ritchie Margaret Ritchie Social Democratic and Labour Party, South Down 3:10 pm, 15th December 2015

It is a pleasure to serve under your chairmanship, Mr Brady. I congratulate Mrs Gillan on securing this debate on a vital issue that affects many young people throughout the UK.

As the right hon. Lady said, neuromuscular diseases come in many different forms. In fact, there are about 60 different types of muscular dystrophy and related neuromuscular conditions, which makes it difficult for the NHS to provide clear-cut statistics on the number of people affected by such diseases. However, research undertaken by Muscular Dystrophy UK suggests that out of every 1 million of the UK’s population, approximately 1,000 children and adults are affected by such muscle-wasting conditions. On that basis, we can estimate that some 70,000 of our constituents, of whom approximately 2,000 live in Northern Ireland, are affected by those conditions.

Another way of totalling the scale of the issue is to look at the admission rates of those with neuromuscular diseases to accident and emergency departments. Muscular Dystrophy UK undertook work on that issue and found that in Northern Ireland in 2011, 787 people with a neuromuscular condition were admitted to A&E departments requiring emergency treatment, at an estimated cost of £2.2 million. Those figures are broadly in line with the GB average. There were 28,000 emergency admissions in the UK, at a cost of £81 million. Relying on the emergency services to fill the gaps in treatment for people with such conditions robs people of their independence and costs the NHS much more than a well-designed system that helps people to manage their conditions and avoids emergencies.

I am sure everyone in this Chamber is in agreement on this issue and wants the best possible treatment and care to be provided to people living with the effects of this cruel disease. Unfortunately, we are not there yet. There is still much work to do—in particular, on an issue that the right hon. Lady already referred to: Duchenne muscular dystrophy and the need for Translarna to be commissioned by NICE and approved by its guidelines. It is important that that happens, because Translarna is already in use in France, Germany, Italy and Spain. Families in those countries can use it, but families here are waiting for it.

Photo of Will Quince Will Quince Conservative, Colchester

One of my constituents has two sons with Duchenne. Does the hon. Lady agree that it is deeply regrettable that they are considering moving to France and commuting back to work so that their sons have the vital access to those drugs?

Photo of Margaret Ritchie Margaret Ritchie Social Democratic and Labour Party, South Down

I thank the hon. Gentleman for his intervention. I agree. His point illustrates that we urgently need a decision from the Minister. I hope the Minister provides us with some welcome information on that issue. It is deeply regrettable that families will go through Christmas not knowing for sure whether the drug will be approved. In the new year, NICE’s decision must not be delayed further. We must end the difficult wait of those families and children.

In Northern Ireland, there has been a commitment for more adult neuromuscular nurse specialists and adult neuromuscular consultants. I share the hope that, when combined with increased care adviser support, the new specialists will begin to improve our currently overstretched services, although there are still valid concerns about how that can be carried out effectively in the context of broader reorganisation and funding scarcity. If we are to achieve the standard of care we all want, much more must be done to co-ordinate better and join up services to ensure patients with muscle-wasting conditions get the help they need efficiently and effectively.

Before I conclude, I want to mention one of my constituents, a lady called Michaela Hollywood, who is wheelchair-bound and was born with spinal muscular atrophy. She was born without ears and is permanently in a wheelchair. She is now 25 years old. She received a Points of Light award, and on Thursday last week she was with the Prime Minister when the Christmas tree lights were turned on in Downing Street. She is on the BBC’s list of the 100 most inspirational women. She received her undergraduate and master’s degrees from Ulster University, and she hopes to go back to do her PhD. She is a lady of immense capacity. She is a campaigner for young people like her with muscle-wasting conditions and, although she spends every day of her life in a wheelchair, she very much enjoys every one of those days because she is a constant campaigner with enormous zeal for life.

Michaela gave evidence to the all-party group on muscular dystrophy in the Northern Ireland Assembly for its report on specialist neuromuscular care. What she said is most important, because it highlights the need for joined-up Government thinking, whether here at Westminster and in the Department for Health or in the devolved Administrations. She said:

“There’s physiotherapy and hydrotherapy, trying just to cover everything. I do receive physiotherapy but it’s a tricky issue because when you’re under 18, with a neuromuscular condition, you have respiratory physio in the community; when you’re over 18 and in the community, with a neuromuscular condition, you’re with disability physios, even though you’re deemed as having a respiratory problem. So that I think is something that is a prime example of the disjointed care that we’re receiving. If we have one specialist multidisciplinary team…that would make things so much easier. If we had a physio that concentrated on neuromuscular diseases but also had experience within respiratory areas, that would make things easier. Also, if we had a cardiologist who pretty much had a good knowledge all round, that would help too.”

Michaela’s words make the case for a joined-up service better than any of us could, so I will end by simply reiterating her appeal for specialist multidisciplinary teams for the treatment of muscular dystrophy to be established. I call on NICE to make its decision on Translarna with the utmost urgency. I hope the Minister will give us some favourable answers to alleviate the distress that is felt by many people throughout the UK.

Photo of Jim Shannon Jim Shannon Shadow DUP Spokesperson (Health), Shadow DUP Spokesperson (Transport), Shadow DUP Spokesperson (Equality) 3:18 pm, 15th December 2015

It is a pleasure to speak in this debate. It is always a pleasure to follow Ms Ritchie, who made an impassioned plea on behalf of her constituent. Every one of us has constituents who suffer from Duchenne muscular dystrophy and other diseases and problems, so it is always good to make a plea on behalf of them in this Chamber. As always, it is good to see the Minister and the shadow Minister here.

I congratulate Mrs Gillan on setting the scene for us all. She clearly outlined the issues, and asked questions of the Minister that we all endorse and support. I thank all other Members who have spoken. I am my party’s spokesperson on health, and I hope to find out about promising developments on the mainland that can be taken back to Northern Ireland, and that thoughts from Northern Ireland can be explained to the Minister. Perhaps the combination of the two can help us to look forward together, in ways that are to our advantage.

As other hon. Members have said, there are some 60 different types of neuromuscular condition, and it is estimated that around 60,000 to 70,000 people in the UK live with neurological conditions that affect their muscle function. Duchenne muscular dystrophy has been mentioned in particular. A couple of my constituents have that condition, and I am aware of the issues. At an event at the Methodist central hall across the way, people with the condition from across the United Kingdom of Great Britain and Northern Ireland clearly explained their needs, the issues—such as the massive advances in medical technology—and the best approaches. There are different levels of Duchenne, and what is medicine for one may not be medicine for another; that is the point I am trying to make.

In Northern Ireland, an estimated 1,600 people in the Southern Health and Social Care Trust area alone are diagnosed with neurological illnesses each year. That is equivalent to the number diagnosed with a major cancer; the conditions we are debating are of as great a relevance as some better-known ones. An estimated 34,000 people in Northern Ireland suffer from a disabling long-term neurological or neuromuscular condition such as epilepsy, multiple sclerosis, Parkinson’s disease, or one of the rare disorders such as motor neurone disease or muscular dystrophy, including Duchenne muscular dystrophy.

Neurological symptoms such as headaches are one of the most common reasons for seeing a general practitioner, and they account for up to 8% of emergency department visits.

Last year, a new dedicated out-patient centre for neurology, with some responsibility for neuromuscular issues, was opened in Northern Ireland. That has been a positive development in the Province, helping with the nationwide momentum for access to treatment, and better treatment, for those living with neurological conditions. The new facility helps us to move towards meeting some of the latest standards in caring for people with long-term neurological and neuromuscular conditions. Since it opened, feedback has been very positive. Improved access to a modernised facility makes life much easier for patients and greatly improves their overall experience of their care.

I understand that the proposed multidisciplinary team will include neuromuscular consultants, neuromuscular physiotherapists and speech and language therapists, along with other care professionals. One of the key service outcomes, which I welcome, is that all patients with long-term neurological conditions have an individual care plan. Caroline Nokes commented on access to wheelchairs; it is important for each individual to have a wheelchair that suits them. That might seem a small thing to some people, but to the patients, it is massive. With 60,000 to 70,000 people in the UK living with neurological conditions, we must take those conditions seriously and prioritise our responsibility to those living with them.

A landmark decision could end the agonising wait for the delivery of the treatment that we are talking about to all those eligible to benefit in England, Wales and Northern Ireland. For those in Northern Ireland who benefited from the dedicated out-patient service I mentioned, I am sure this would be an additional boost. Nationwide, the momentum is now in favour of those living with neurological conditions. That may not always be how it feels, but there is a step forward, and we have to look at that, and at how to deliver that better. Although the move is very well intentioned and welcome overall, concerns have been raised and reservations expressed.

I know that the Minister will respond to what has been said about the NICE conditions. Lately he seems regularly to make particular reference to NICE in responding to debates in Westminster Hall and the main Chamber, and I think that is what Members are interested in. There is a need for a specific NICE response. It has asked for further clarification from PTC Therapeutics of the degree of benefit that its drug Translarna provides in the treatment of a type of Duchenne muscular dystrophy. DMD is one of a group of muscular dystrophies, which are inherited genetic conditions that cause the body to produce too little dystrophin, a substance crucial for muscle functioning. Seeing some of the young patients who have the condition focuses one’s attention quickly on how critical the availability of the medicine is. The condition leads to changes in the muscle fibres, which gradually weaken the muscles, resulting in an increasing level of disability. The decline in physical functioning in DMD leads to respiratory and cardiac failure and eventual death, usually before the age of 30.

Ataluren is the first licensed treatment for DMD that addresses the loss of dystrophin, the underlying cause of the condition. It has a conditional marketing authorisation in the UK for the treatment of DMD. The families of children affected, and Muscular Dystrophy UK, have campaigned for NHS England to make the drug available, since it became the first EU-approved drug for tackling Duchenne muscular dystrophy last August. The condition affects 2,500 people in the UK, causing increasingly severe disability and cutting lives short. It would be useful if the Minister and his Department could address NICE’s concerns, as it has asked questions. The right hon. Member for Chesham and Amersham referred to NICE and outlined four or five questions. I think that the focus that will emerge from the debate will be on how to improve the NICE guidelines and improve access to drugs throughout the United Kingdom of Great Britain and Northern Ireland.

Whenever I attend health debates in Westminster Hall, I ask whether we can have not just a regional strategy but a UK-wide one, bringing Northern Ireland, Scotland and Wales together. Will the Minister consider that? The four regions can benefit from each other’s knowledge. Collectively, we can do better.

Photo of Madeleine Moon Madeleine Moon Chair, Defence Sub-Committee 3:26 pm, 15th December 2015

I want to speak not so much about Duchenne muscular dystrophy as about Parkinson’s disease and motor neurone disease—particularly the latter, which is also a neuromuscular condition. I will consider the problem of getting appropriate diagnosis and in-care support from neurologists. That is critical. We have already heard about the difficulty that those living in the wrong part of the country have in obtaining a quick diagnosis and the right support as they progress through the condition.

Neurological disorders account for up to 20% of acute medical admissions, and there are more than 60 different neuromuscular conditions. I was helped to understand the patchwork problem that people experience in getting access to neurological advice, support and guidance by a report commissioned in 2014 by the Association of British Neurologists. With 60,000 to 70,000 people in the UK living with a neuromuscular condition, there is considerable pressure on neurologists, and on specialist diagnosis and support. In 2011, a joint report of the ABN and the Royal College of Physicians, “Local adult neurology services for the next decade”, recommended that all relevant patients be admitted to hospitals that had an acute neurological service led by consultant neurologists. We are nowhere near that position now.

In 2014 a survey by the ABN found that the likelihood of a patient with a neurological problem being seen by a neurologist varies dramatically depending on where they live. The availability of a neurological review varied according to the type of hospital the patient was admitted to—whether it was a neuroscience centre, a neurology centre or a district general hospital, with or without neurological support. It was significantly better to be seen at a neuroscience centre, where there was support on 91% of days. Those who went to a neurological centre got support from a neurologist on only 80% of days. District general hospitals with a neurologist based at them had access to a neurologist on only 58% of working days. In contrast, access for patients at district general hospitals with no resident neurologist was available on only 32% of days. That is totally unacceptable, when we know that access to such neurological support dramatically changes the impact on a patient; there is an effect on their capacity to continue their life, and on their prospects of being discharged from hospital with a good quality of life.

I am concerned about the fact that access to CT and MRI imaging varies between hospitals. MRI was available 24/7 at only 30% of sites in the UK. The 2014 study identified a wide variation in access to specialist services for patients presenting with acute neurological disorders. As liaison neurology services change the diagnosis and management of a high proportion of patients, improving outcomes and reducing length of stay, there is an opportunity to improve both patient care and cost-effectiveness.

The all-party parliamentary group on motor neurone disease recently heard emotional and dramatic evidence from Mark and Katy Styles. Mark worked in local government and Katy was a secondary school teacher before she gave up work to become Mark’s full-time carer. Mark has a form of motor neurone disease called Kennedy’s disease, which is slowly progressive and genetic, passed from mothers to sons. He lives in Canterbury, and they have to travel to University College hospital in London to see a neurologist. That is nonsensical. Support should exist locally, because his condition may soon deteriorate to the extent that he cannot access the neurological support that he and his wife need. When Katy was working, she earned £150 a day. She now gets £67 a week to provide the on-duty, 24/7 care that her husband needs. We must recognise the invaluable work of carers and ensure that they get the necessary support.

Some 30% of people with motor neurone disease will die within one year of diagnosis. It is absolutely critical that they get rapid access to help and support for their condition. I will not repeat all the words that have been said about access to wheelchairs, but I stress that if someone with MND has the wrong wheelchair, they may not be able to communicate that. An ill-fitting wheelchair causes acute pain, but MND sufferers rapidly lose their speech and the ability to move their hands and upper limbs. They cannot communicate that they are in pain or distress, so the provision of wheelchairs is critical.

The APPG commissioned a report last year called “Condemned to Silence” about access to communication support. The issue is not money, because the Government have put money in place for communication support; it is poor roll-out, and something must be done to improve it. I look forward to hearing from the Minister what steps are being taken to ensure that people are not condemned to die without access to communication support, which they need to talk to their loved ones and carers about their needs, and to avoid suffering the indignity of being unable to communicate if they are admitted to hospital.

I have two final points. First, access to care support is critical as conditions deteriorate. People cannot wait for further assessments, or be put on waiting lists for care support, when they often need double-handling care support. If people are not to be condemned to lie in beds, support must be available immediately. Access to hoists and high-low hospital beds is often delayed.

We must get faster at providing them. Secondly, access to finance to research such conditions is vital if we are to give families hope, and a sense that time and research will give them—and perhaps family members, who may inherit a condition—a better life in the future.

Photo of Richard Arkless Richard Arkless Scottish National Party, Dumfries and Galloway 3:33 pm, 15th December 2015

I thank Mrs Gillan for securing a debate on a subject that clearly touches many people for various reasons. It is also of course a pleasure to serve under your chairmanship, Mr Brady. Those Members who know my background as a lawyer and a businessman may be forgiven for asking, “Why are you here to speak about neuromuscular diseases?” I am not a member of the SNP health team, which is lucky to have the profound experience of my hon. Friends the Members for Central Ayrshire (Dr Whitford) and for East Kilbride, Strathaven and Lesmahagow (Dr Cameron), but I am developing something perhaps even more important and certainly more profound: I am living with motor neurone disease within my family.

If I can declare an interest of sorts, Mr Brady, my mother has motor neurone disease and, if Members will allow me, I will restrict my comments to that experience. I do not mean to sideline the 59 other important neuromuscular diseases; I want to ensure that what I say comes from a real place, not just a bunch of briefing notes. That notwithstanding, I want to add my voice to the calls to the Minister to persuade him to get Translarna approved as quickly as is humanly possible. I know that he looks sympathetically on that cause, and we have heard many vivid stories today about how it would affect people quickly.

Like everybody, I would lay claim to my mother—and indeed my wife—being the best that there is. She is the most selfless, dedicated mother in the world. She has literally lived her life for me, my brother, Nicholas, and my baby sister, Poppy—a baby who is now in her twenties. We have been her life. She has lived her life exclusively for us. My brother and sister and I will always be grateful for that. My mother was diagnosed 20 months ago. She kept it a secret with my dad for 16 of those months—thank goodness that she has my dad, with whom she has an unbreakable bond—so I have only been living with it for a few months. It is difficult and, as may become obvious during this speech, extremely raw.

During those 16 months, we witnessed the deterioration of my mother’s speech and joints. She explained the speech by way of fictional dentures that she had apparently had implanted. She explained the hands with reference to a historical arthritis problem in our family. Both were plausible and not really questioned up until the point that she decided to tell us. She did not tell me, my brother or sister or our extended family until after I was lucky enough to be in this place and make my maiden speech. She was determined that I should pursue my dream, and of course her dream, of making Scotland a better nation. I suspect that what she really wants now is a second referendum before it is too late, but I suppose that you get the point.

I will return to my mother later, but I want to pick up on some points made by other Members in this enlightening and touching debate. The right hon. Member for Chesham and Amersham is right to say that there are 60 different types of neurological disease, and I hope that she will forgive me for indulging myself with only one of them today. She talked about the Hill family, particularly Archie. I have this vivid image in my mind of Archie running up to 10 Downing Street and giving the Prime Minister what for—if only all of us could get that opportunity.

The speech of Rachael Maskell was touching indeed. She brings a wealth of practical experience, to which the Minister ought to listen carefully. I was particularly captured by her assertion that equipment needs to be made available when needed. It needs to be the right equipment in the right place at the right time, because time moves too quickly with such diseases. I hope that the Minister will pay careful heed to the hon. Lady’s practical experience, which has substantially benefited the debate.

I was particularly touched by the story of Jagger from Caroline Nokes. I hope that he enjoys his break in Tenerife and that the Minister will listen to calls for Translarna to be approved as soon as possible.

The debate has been consensual so far, and I want to take the politics out of what I am going to say. I want to touch on some things that Scotland is doing well on motor neurone disease, but I do not mean that to be a criticism of the UK Government. I hope that everybody’s ears are open. If we are doing things right, I sincerely hope that the UK can learn. If the UK is doing things right, my ears are open and Scotland can certainly learn. There is no politics in this whatsoever. As an aside, as new Member I have been frankly dismayed at how health services are politicised by both sides of the House. I have become increasingly amenable to the suggestion that perhaps the health service should not be run by politicians at all, but by people who have at heart the interests of the people whom we are here to serve.

Motor neurone disease is a neurological degenerative disease. In simple terms, the mind is fine and continues to operate with full function, but the body gradually gives up. The signals do not go from the mind to the body to make it work: that is how I think of it. Patients are affected differently. My brother-in-law died from it a couple of years ago, and his limbs were affected first. His legs started to give way for no apparent reason. However, my mother’s speech was the first thing to go—this was a lady who liked to talk, who seriously liked to talk! To have that stripped away from her must be incredibly difficult—and I know it is.

Scotland is doing many things well on motor neurone disease. Over the past year we have announced that we will double the number of motor neurone nurses. We are very much in the early stages, but the Scottish Government have provided funding to local authorities to ensure that things happen. At the moment local authorities are recruiting and assessing the need, and I hope that process will be speeded up as we go on.

That funding is a recognition of the difference that specialist nursing can make to motor neurone disease. My mother has to rely on a motor neurone nurse who comes from another region and who can only come on a part-time basis. A very proud woman, she was initially most reluctant to consider any form of help, but she has since come to realise what a benefit the nurse is. She has asked me to take up the cause of getting more motor neurone nurses throughout not only Scotland, but the rest of the UK—we are all human beings, despite our political views.

The change in Scotland has been praised in all quarters. Huge thanks are due to people such as Archie and Jagger. In Scotland a gentleman called Gordon Aikman, Christina McKelvie MSP, and all the staff at MND Scotland have done an incredible job of persuading the Government of the immediate need for those services. We have committed to giving a free voice box on the NHS and to paying families directly for support, not as a patronising gesture to pay people to look after their relatives, but to ensure that support is available if needed. We have also increased investment in palliative care.

I live in a town called Stranraer. The UK average is two people with motor neurone disease per 100,000; the Stranraer average is 13 per 10,000—an astronomical figure. I have asked the chief executive of our local health board to figure out exactly why—

Photo of Graham Brady Graham Brady Chair, Conservative Party 1922 Committee

Order. I am sorry, the hon. Gentleman must resume his seat. I am loth to interrupt a very personal story, but we need to leave time for both the official Opposition spokesperson and the Minister to wind up. I have allowed more than eight minutes, and I am keen to allow the same to the official Opposition.

Photo of Barbara Keeley Barbara Keeley Shadow Minister (Health) 3:42 pm, 15th December 2015

It is a pleasure to speak in the debate with you in the Chair, Mr Brady.

As others have done, I congratulate Mrs Gillan on securing the debate. She gave a moving account of the Hill family in the light of Archie’s diagnosis and of the impact on Archie and his family of Duchenne. We have also heard excellent speeches from my hon. Friends the Members for York Central (Rachael Maskell) and for Bridgend (Mrs Moon) and the hon. Members for Romsey and Southampton North (Caroline Nokes), for South Down (Ms Ritchie), for Strangford (Jim Shannon) and for Dumfries and Galloway (Richard Arkless).

The all-party group for muscular dystrophy has carried out essential work to raise awareness and understanding of the needs of people living with muscular dystrophy and other neuromuscular conditions. I congratulate the APG on the quality of its inquiries and reports. The right hon. Member for Chesham and Amersham also paid tribute to the work of Muscular Dystrophy UK, and I join her in that tribute.

Providing access to treatment for people with muscular dystrophy is complex, because it is a rare condition. There are challenges in delivering localised, specialised care to people who have multiple, complex needs, but that cannot be an excuse for poor-quality care. As we have heard, some 70,000 people are affected by a neuromuscular condition in the UK. We must ensure that the NHS delivers equal treatment for equal need and that those with complex needs may have access to the treatment and support necessary to help them achieve the best quality of life possible.

In 2009 the APG’s Walton report showed clear deficiencies in the provision and planning of, and access to, care for people living with neuromuscular conditions. It found cases where care was “inadequate and not acceptable”. Although the report offered many sensible recommendations to improve the quality of care, the potential for progress was limited by the coalition Government’s reforms of the NHS under the Health and Social Care Act 2012.

Given those reforms, the APG undertook a six-month inquiry that considered their impact, releasing another report in March this year. Sadly, the reorganisation of the NHS and other reforms had done little to improve access to and availability of treatment for patients with neuromuscular conditions. Sadly, in fact, the reforms had made it even harder for patients to access support as a result of significant regional variations in the commissioning and funding of services. That is the nub of what I want to say.

There is a failure to join up services, and confusion about responsibilities and processes is a common theme. The hon. Member for South Down gave us an excellent quote on that lack of joined-up services. At the national strategic level there has been no specific mention of neuromuscular conditions in the five-year forward view, nor anything in the consultation document on the draft NHS mandate, which suggests a failure to recognise the specific needs of such patients at the strategic level. In fact, the five-year forward view groups together rare diseases and cancers, but there is a great deal of difference between all the conditions that we have discussed today and rare cancers.

There is a lack of clear guidance on which bodies in the NHS fund certain services and, as we have heard, sometimes people are not even receiving crucial respiratory support. Locally, there were examples of clinical commissioning groups failing to fund sessions of specialist neuromuscular physiotherapy or to provide sufficient funding for people to receive the right wheelchair at the right time. That is clearly so important and was mentioned by my hon. Friend the Member for York Central and the hon. Member for Romsey and Southampton North. It must be right for a child to have a comfortable chair while growing up.

The coalition Government’s reforms have also contributed to a delay in decisions on the availability of drug treatment for Duchenne muscular dystrophy, a life-limiting illness that affects about 2,500 boys and adults. The right hon. Member for Chesham and Amersham talked about that so well. To have to wait with a condition such as Duchenne for a decision on the drug Translarna is clearly agonising. It is a shame that the issue has been caused by NHS England halting its assessments to review its processes. I was not present at a Westminster Hall debate last week in which I understand that the Minister present said that he was “hopeful” that access to the drug would become available:

“I am hopeful about Translarna…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”.—[Hansard, 8 December 2015; Vol. 603, c. 274WH.]

As he went on to say, however, the decision is not in his gift.

I hope that the Minister’s optimism is well founded, because as we have heard today it must be recognised that time is an important factor. The decision is different from some others, because the timing can affect the benefit that the boys will receive. I wish to ask, as everyone else has done, about that decision, and what he is doing to ensure that delays do not happen again. We must ensure that system of wider support is available for patients and their families and carers. In some cases people need 24-hour support and care; homes must be adapted; physiotherapy, speech and language therapy, and occupational therapy need to be available; and carers need access to the right advice and support, as has been said.

I am concerned, like others, that the Government’s failure to protect social care funding and other non-NHS health funding, such as training budgets, will mean that that wider network of support is not available when needed. The Walton report highlighted issues with social care back in 2009, but since then the number of people with access to publicly funded social care has fallen by 25%. The availability of the right support for people with specialist care needs is unlikely unless we have a sustainable funding settlement for both the NHS and social care. The difficulty with recent funding announcements, if the Minister intends to refer to them, is that the 2% social care precept and the better care fund are back-loaded funding mechanisms, with nothing this year and little next year.

We have heard about the regional differences in access to care. The Walton report highlighted that there were only 13 neuromuscular care co-ordinators when 60 were felt to be needed. My hon. Friend the Member for York Central said that there was only a single neuromuscular care adviser in North Yorkshire; in fact, no neuromuscular care adviser support is available for adults living with such conditions in Greater Manchester or the surrounding areas of the north-west. That is in spite of the fact that an estimated 8,100 people with a muscle-wasting condition live in the north-west. There is a need to ensure that clinical commissioning groups and other regional health organisations are aware of their responsibilities. Sadly, the findings of the 2015 APG report suggest little progress in the issue of unequal access.

Given the devolution deals on health and social care in certain parts of the country, will the Minister assure us that the inequalities in specialised services that we have heard about will be addressed and that the relevant bodies will be made aware of their responsibilities, which they do not seem to be at the moment? The debate has highlighted the fact that we have ingrained problems in our health and social care system. The lack of a sustainable funding settlement for social care and other recent reforms have led to fragmentation and instability in services. That means that inequalities in care sadly will continue. I urge the Minister to consider in full the most recent report and recommendations from the APG. We must ensure that people with neuromuscular conditions receive the care that they need, and that those inequalities in care are addressed.

Photo of George Freeman George Freeman The Parliamentary Under-Secretary of State for Business, Innovation and Skills, The Parliamentary Under-Secretary of State for Health 3:49 pm, 15th December 2015

It is a pleasure to serve under your chairmanship, Mr Brady. I am left with 10 minutes in which to try to deliver my speech and the answers that I have carefully prepared while listening to colleagues’ comments. If I run short of time, I will undertake to write to everyone in the Chamber with answers to the points raised.

I start by paying tribute to my right hon. Friend Mrs Gillan for securing the debate. She is a tenacious advocate on this issue, as on others. I join her in paying tribute to Archie and his family. I have met patients who suffer from these diseases and their families, and one’s heart goes out to them. One wants to pay tribute to the bravery with which they deal with their conditions. As is so often the case in the history of medical progress, the families, patients and carers are those who advocate and, in the end, win through to make their point heard, with the help of colleagues from across the House. My right hon. Friend eloquently paid tribute to the families of children with these disorders and diseases who, in many ways, suffer every bit as much as the patients who show such incredible fortitude. She asked me last week whether I would give her an A grade for effort and persistence. I will happily give her an A-plus in this end-of-term summary, but the people to whom we really want to give an A-plus are NICE and NHS England.

I want to touch on some of the excellent points that were raised. My right hon. Friend raised Vimizim and Translarna, so I will say something in detail about the timing of those decisions in a minute. She also made an important point about standards of care across the NHS in clinical trials, which was mentioned by numerous colleagues, and the importance of NICE giving more prominence to the time aspect of these conditions, which are unusual because they can deteriorate with every week’s delay in getting treatment.

Rachael Maskell gave us the benefit of her front-line clinical expertise. In case I run short of time, I shall say now that I will happily convene a meeting at the Department of Health with officials from my Department and NHS England, to which I invite colleagues from all parties who want to discuss the issues she and others raised about front-line care, because a range of practical issues about such care has been raised, in addition to access to drugs, and giving colleagues the chance to raise such points on behalf of their constituents would represent a powerful opportunity. The hon. Lady talked in particular about training and the interface of paediatric and specialist services, which I come across in connection with numerous different specialist conditions.

My hon. Friend Caroline Nokes spoke passionately about James, Jules and Jagger Curtis, and the importance of expediting those particular decisions and quicker assessment, as well as adoption in general. That is a passion of mine, which was why I launched the accelerated access review to look systemically at what we can do to expedite getting new medicines into the service. She also touched on the importance of wheelchair access.

Ms Ritchie talked eloquently about Michaela and the importance of specialist, multidisciplinary teams. Jim Shannon, who gets the prize for appearing in more debates with me than any other Member of the House, which is a tribute to his activism as the Democratic Unionist party’s spokesman on these issues, highlighted the importance of Belfast as a hub of research and regional strategies in Northern Ireland and spoke about his constituents. This is a devolved matter, and while I pay tribute to the work of researchers and medics in Northern Ireland, it is important that the devolved Administration in Northern Ireland put in place a similarly enlightened commissioning process.

Mrs Moon raised the broader issues of Parkinson’s and neurological disorders, while Richard Arkless spoke passionately about his mother’s suffering. Before I came to Parliament, I worked in Scotland and, as he highlighted, in this area, as in several others, Scotland pioneers some of the clinical commissioning work. The supreme irony of the debate was brought to light by his request that we depoliticise the NHS. For me, one of the greatest steps following the Health and Social Care Act 2012 was the separation of the NHS from the Department of Health. NHS England now operates under its own arm’s length management, subject to a mandate from Ministers

We do not control the NHS—believe me that if, for one afternoon, I could do that, I wish it was now. I would love nothing more than to pull the lever and give all these children the drugs that we all want them to get before Christmas, but that is not in my gift, and I suggest that it is in all our interests that it is not. It is right that such decisions are taken by NHS England and clinical professionals, advised by the very best people at NICE.

Photo of Barbara Keeley Barbara Keeley Shadow Minister (Health)

It is important that the NHS mandate covers these conditions because at the moment it does not. Something must be done to make sure that they are covered.

Photo of George Freeman George Freeman The Parliamentary Under-Secretary of State for Business, Innovation and Skills, The Parliamentary Under-Secretary of State for Health

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.

Photo of Dame Cheryl Gillan Dame Cheryl Gillan Conservative, Chesham and Amersham

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.

Photo of George Freeman George Freeman The Parliamentary Under-Secretary of State for Business, Innovation and Skills, The Parliamentary Under-Secretary of State for Health

My right hon. Friend made the point more powerfully that I could. On Vimizim, I am delighted that BioMarin, the company concerned, has, following exhortation from me and others, sat down with NHS England and gone through pricing flexibilities. I have been able to grant an access agreement. The drugs are incredibly expensive, which is what makes NICE’s work difficult, so if anyone from PTC Therapeutics, the company responsible for making Translarna, is watching, I urge them to sit down with NHS England and adopt a similar approach.

On specialist commissioning, we lived through and focused on difficult decisions, and this summer NHS England agreed to 23 new treatments, including Duodopa, which controls the symptoms of patients with Parkinson’s disease, wider access to proton beam therapy, and extending treatment with ivacaftor for cystic fibrosis involving G551D mutations.

We live in an age of extraordinary biomedical progress, but budgets struggle to keep up with the rate of progress that our scientists and researchers make. I know that this of little comfort to my right hon. Friend and to Archie and his family, but I hope that hon. Members agree that, in deciding which treatments should be procured on behalf of us all, due process must apply. However, I share their frustration, which was why I launched the accelerated access review, which will report independently to me in the spring. That substantial piece of work is examining whether we can do more to embrace breakthroughs in genomics and informatics to give NICE and NHS England new flexibilities to speed up how innovations for patients are assessed, as well as to explore new pathways and flexibilities for different models of reimbursement to get innovation through quicker.

I want to pick up the point that was made about specialist neuromuscular care. The truth is that there are few curative treatments for most of these diseases, so we are talking about the importance of routine care for people that is provided by local primary and secondary care services via local CCGs. However, a number of specialist services have been designated among the 146 that NHS England is responsible for commissioning nationally, which are set out in legislation and commissioned directly by NHS England through 10 area teams. Twenty-five specialised neurological treatment centres across England ensure that patients can access high-quality neurological care where they live.

As I said, I will be happy to convene a meeting in the Department with officials and NHS England to talk about how we can address some of the practical issues raised when providing front-line, integrated services. I fear that the clock is against my being able to go through the 22 questions for which I had answers prepared, so I close by saying that while we all would want to pull a lever to make this happen quickly, the truth is that we need a system that is rigorous, robust and evidence-based, but quicker, in recognition of the effects on these patients.

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

Sitting suspended for a Division in the House.