William Mead: 111 Helpline

Cheryl Gillan Excerpts

Tuesday 26th January 2016 Commons Chamber

[Mr Hunt]

I agree with the broad thrust of the hon. Lady’s remarks. Of course she speaks with the authority of an experienced clinician herself. In this case, the tragedy was that there was actually a doctor who spoke to the Mead family on the night before William died, and he did not spot the symptoms. It is not simply a question of access to a doctor, but ensuring that doctors have the training necessary. However, as she says, dealing with cases such as this can be very difficult. The doctor’s view on that occasion was that, because the child was sleeping peacefully, it was fine to leave him until morning when, tragically, it was too late. Other doctors would say that that is a mistake that could easily have been made by anyone, which is why the report is right to say that it is about not individual blame, but a better understanding of the risks of sepsis. She is right in what she says. As we are trying to join up the services that we offer to the public, it is a good principle to have one number that we dial when we need advice on a condition that is not life-threatening or a matter for a routine appointment with a GP, and 111 is an easy number to remember. However, we need to ensure that there is faster access to clinicians when that would count, and that those clinicians can see people’s medical records so that they can properly assess the situation.

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

As chair of the all-party group on sepsis, may I also pay tribute to the Mead family, who are now campaigning to ensure that no other child suffers in the same way as William? The Secretary of State has taken a great deal of interest in the UK Sepsis Trust and the work that it has been doing with the APPG. He will know that we are pressing for a campaign similar to the F.A.S.T campaign for strokes, as early diagnosis can save lives. Will he now consider very seriously funding such a campaign for sepsis, because there are thousands of deaths that could be prevented by a campaign that makes everyone aware of the signs of sepsis?

[Mr Hunt]

I am happy to undertake that the Under-Secretary of State for Health, my hon. Friend the Member for Battersea (Jane Ellison), will look urgently into whether such a campaign would be right. I can reassure my right hon. Friend that the package that we put together and announced last January did contain what most people felt was necessary, but we can always look at whether more needs to be done. I commend her for her campaigning on the issue of sepsis. On a more positive note, when the NHS has decided to tackle conditions such as MRSA and clostridium difficile, it has been very successful. In the past three years, the number of avoidable deaths from hospital-acquired harms—the four major ones—has nearly halved, so we can do this. We should be inspired by the successes that we have had to make sure that we are much, much better at tackling sepsis.

Oral Answers to Questions

Cheryl Gillan Excerpts

Tuesday 5th January 2016 Commons Chamber

[Jane Ellison]

I am glad the right hon. Gentleman mentions that point; the House may not be aware that we recently added four new rare diseases to the newborn heel-prick test, which has helped to detect more than 1,400 children with a rare disease. I am disappointed to hear that he feels that some parents had issues with follow-up, and of course we will look into that, but I think he will find that the UK rare diseases strategy, which was published in 2013 and contains 51 commitments from government, covers that. The first report back on that strategy will take place this spring and it is being done by the UK Rare Disease Forum. I am happy to speak to him afterwards about whether the excellent organisation he names is part of that.

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

One such rare disease is Duchenne muscular dystrophy. I am sure the Minister is aware that we are awaiting what we hope will be a positive decision from NHS England on a drug called Translarna, which could help boys with the disease. We were due to have that announcement yesterday. Does she have any further and better particulars on that? Will she update us on when we can expect an announcement, which we hope will be a positive one?

[Jane Ellison]

I know that my colleague, the Under-Secretary of State for Life Sciences, is working very hard on that matter, and is hoping to make an announcement soon. I am sure that, at that point, he will be able to update my right hon. Friend.

Specialist Neuromuscular Care and Treatments

Cheryl Gillan Excerpts

Tuesday 15th December 2015 Westminster Hall

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

I beg to move,

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

[Mr Graham Brady (in the Chair)]

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

[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.

[Several hon. Members rose—]

--- 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.

Southern Health NHS Foundation Trust

Cheryl Gillan Excerpts

Thursday 10th December 2015 Commons Chamber

[Mr Hunt]

I thank the right hon. Gentleman for his comments. I hope I did address that by saying that the first time was when we realised there were issues around the tragic death of Connor Sparrowhawk. That is what started the process and led to the independent investigation. Because NHS England wanted it to be very thorough, that investigation went right back to 2011 and up to 2015. It looked at all unexpected deaths in that period, and at the reporting culture and lessons that had or had not been learned as a result. A lot of action has been taken. I can also reassure the right hon. Gentleman that during that period we have been implementing the recommendations of the Francis report, which has meant that throughout the NHS there is a much greater focus on, and transparency in, patient safety.

It is important to give the NHS credit. During the past three years, we have actually seen a 25% increase in the number of reported incidents. I think people are treating this much more seriously than in the past, but there is much more to do.

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

I, too, welcome my right hon. Friend’s statement and the news that he plans to accept the recommendations of this very sobering report. Will he reassure the House that anyone found to have been deliberately contributing to patient neglect or failing to investigate avoidable deaths will be held to account both by the professional regulators and the full weight of the law?

[Mr Hunt]

I can of course give my right hon. Friend that assurance, but there is a note of hesitation in my response. That is partly because professional standards, as my right hon. Friend knows, are not a matter for politicians—they have to be set independently by the General Medical Council and the Nursing and Midwifery Council—and partly because if we are going to improve the reporting culture, which in the end is what the report is about, we have to change the fear that many doctors and nurses have that if they are open and transparent about mistakes they have made or seen, they will get dumped on. That is a real worry for many people. Part of this is about creating a supportive culture, so that when people take the brave decision to be open about something that has gone wrong they get the support that they deserve.

Cystic Fibrosis

Cheryl Gillan Excerpts

Tuesday 8th December 2015 Westminster Hall

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

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

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

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

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

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

[Mrs Gillan]

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

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

PTC Therapeutics states that the drug is a

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

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

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

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

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

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

[Julian Sturdy (York Outer) (Con)]

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

[Mrs Gillan]

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

--- Later in debate ---

[George Freeman]

The hon. Lady makes an important point. I have been to Washington three times and to Berlin, Paris and Brussels to highlight that while the UK is leading in this field, we need a transatlantic—European and American—agreement on how we move things forward. That is why I am convening and chairing a summit this afternoon with the Washington-based FasterCures campaign, which is a cross-party group on the Hill pushing for innovations in this space. I have been talking to the Commission about the European framework. I want the UK to be the best entry point into the European market, but I also want the European regulatory framework to be consistent and coherent; that is an important point.

The second question I have asked the AAR to look at is: what freedoms, flexibilities and new pathways can we envisage giving NICE and NHS England, particularly in the field of specialist commissioning? For CF, the decision to purchase ivacaftor is a national one, made by an NHS England specialist commissioning unit. I would like that unit to work much more closely with the Department of Health pricing team, so that where we can offer a company faster access to a key patient cohort, data and genomic information, we are able to do a much better deal with the company.

At the moment, we are operating the Translarna and Vimizim programme in the existing landscape. I share colleagues’ frustration, but it is important we go through due process. I do not think anyone wants a world in which Ministers decide what drugs come through on the basis of political pressure, tempting though it may be. I have done everything I can this year to expedite the existing process.

Following the positive news on Vimizim, I am hopeful about Translarna—a similar drug. NICE has been consulting on the process, and I believe the company has been engaging with NICE on pricing. I am hopeful that there will be a decision in the next few months to parallel the one on Vimizim, but that decision is not in my gift: it is up to NICE, which is rightly working on the basis of the very best clinical evidence.

[Mrs Gillan]

rose—

[George Freeman]

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

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

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

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

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

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

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

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

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

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

[Mrs Gillan]

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

[George Freeman]

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

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

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

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

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

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

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

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

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

Junior Doctors Contract

Cheryl Gillan Excerpts

Monday 30th November 2015 Commons Chamber

[Mr Hunt]

The hon. Lady is right that this is about the excess mortality rates of people admitted at the weekend—not of people who are already in hospital at the weekend. I am afraid that she is mistaken in her characterisation of the rest of the Government position. Clinical standards are clear: people admitted at the weekend, or at any time, should be seen by a consultant within 14 hours, but that is true in only one in eight hospitals across seven days of the week, which is why sorting out the consultant contract for urgent and emergency care matters. Although the opt-out in the consultant contract applies only to elective work, half as many consultants are available in A&E on Sunday as are available during the week, although Sunday is one of the busiest days of the week, so it is not just about junior doctors. However, if we are going to make life better for junior doctors, we need to make sure that they have more senior cover and do not feel clinically exposed, which is what independent studies have said they feel.

Governments of any party must have the right to set the terms and conditions of an employment contract. That is a right that no part of the public sector has moved away from, and it is a vital right for all employers. I have simply said that I will not move towards any new contract while negotiations are happening during this time-limited period. That was what my statement clearly said, and the BMA for its part has said that if this agreement is honoured, it will remove the threat to strike during that period.

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

I congratulate the Secretary of State on coming here today on this very important matter. All parts of the House support him in trying to find a negotiated solution to this knotty problem. However, if the strike goes ahead—although we very much hope that the BMA will see sense and agree to the terms so far put on the table—I understand that the BMA has not been willing to provide assurances that it will ask its members to provide urgent and emergency cover in areas where patients may be at risk. What more can the Secretary of State do to encourage the BMA to make that statement? That is what will be worrying patients out there.

[Mr Hunt]

On the overall picture, we must be clear that this is not about asking junior doctors to work a lot of extra hours for free. We expect that as we have increased take-up of seven-day services and more people working antisocial hours, particularly on Sundays, that might lead to a higher pay bill, but we need to make sure that the proposals for the workforce that we have at present protect average pay and mean that as we move to seven-day services, they are affordable by hospitals. To answer my right hon. Friend’s question, we respect the right of doctors to strike, even though it is very disappointing when they choose to do so, but they have said on this occasion, in a way that is quite unprecedented, that they will withdraw urgent and emergency care on 8 and 16 December. All we have said to them is that if there are areas where we are not able to make alternative arrangements for urgent and emergency care by, for example, using other front-line clinicians, we would like their support in those specific areas, not across the whole country, in asking junior doctors to step in on those cases in the interests of patient safety. We have not yet had those assurances, but we very much hope we will get them.

NHS (Contracts and Conditions)

Cheryl Gillan Excerpts

Monday 14th September 2015 Westminster Hall

[Dr Whitford]

I made the point that Bray’s paper talked about registered nurses—so, degree nurses—and that reflects the skill mix.

We need to know what the actual problem is rather than just running in and throwing ideas and policies around. Attacking staff who work very hard and for really long hours is not very fruitful. We need NHS staff to believe in the political decisions, the guidance and the direction being taken in the future, so I simply suggest that everyone in this House look at the way forward.

[Mrs Cheryl Gillan (in the Chair)]

When time permits, it is our practice in Westminster Hall that when a Member has been here for the opening speeches and then had to step out temporarily, we give them the opportunity to speak. So I call Andrea Jenkyns to speak.

Drugs: Ultra-rare Diseases

Cheryl Gillan Excerpts

Tuesday 16th June 2015 Westminster Hall

[Greg Mulholland]

I hope that we get answers today and a real promise of intervention from the Minister.

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

I pay tribute to the hon. Gentleman’s work on this issue. I joined this campaign because of Archie Hill, a constituent of mine aged 10 who has Duchenne muscular dystrophy. No matter what the Minister says about drugs such as Translarna and the process that the hon. Gentleman is about to outline, which has been disgraceful, that drug is available in other European countries and we have still not cleared it for patients in the UK.

[Greg Mulholland]

Indeed. It was a pleasure to meet the right hon. Lady’s constituent, Archie, and his parents. These young people are inspiring us to campaign. She is absolutely right. We are debating the European Union Referendum Bill today in the Chamber. Other EU countries, and some non-EU countries, regard these treatments as effective and affordable, yet we do not.

I will fast-forward from the scrapping of the previous body to October 2014, when NHS England came out with the scorecard system. That is despite one of the clinicians involved, Dr Chris Hendriksz, saying on 22 October in an email:

“I would suggest the scoring is not used at all for decision making this round and I would rather have people acknowledging that they are making random decisions than to try and give some credibility to a process that was deeply flawed.”

That is from one of the senior clinicians.

NHS England none the less went ahead with the scorecard system to decide which funding should be prioritised. Suzanne Mallah and her 10-year-old boy Kamal, who has Morquio and is another inspiring young person whom I have been delighted to meet, saw that that was not only haphazard but discriminatory. With the help of the MPS Society, they threatened legal action on 28 November against NHS England on the basis that the scorecard was clearly discriminatory, that there was no policy explaining it and that there had been no public consultation on its use. Just one week after that, on 2 December, NHS England announced that it was suspending use of the scorecard because the MPS Society and Kamal were right and it was wrong.

--- Later in debate ---

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

I pay tribute to the hon. Member for Leeds North West (Greg Mulholland) for raising this issue and for his untiring work. I agree with my hon. Friend the Member for Romsey and Southampton North (Caroline Nokes) and the hon. Member for Strangford (Jim Shannon), who made many of the points I hoped to make.

I am grateful to have a short time to raise the case of my constituent Archie Hill and his parents’ tireless campaigning to get access to Translarna for him. It is inspirational to see how this family, and many others, have campaigned for their children. I can imagine nothing worse than watching one’s child slowly lose their mobility, knowing that their life expectancy will not be as great as ours might be.

Translarna is available in other European countries. As we have heard, it is available in Greece, which is not in the best economic health. Only recently, in Germany, the Federal Joint Committee determined that it provides a benefit for ambulatory patients aged five years and above with the nonsense mutation. The rise in the PTC Therapeutics share price on the back of that announcement shows that the company is well placed, and its drug is being recognised right around the world.

There is an irony here. If the decision coming down the track goes against making Translarna available to the patients who deserve it so much, the question arises as to whether this is about cost. The decision will almost definitely be made on a cost basis. Day by day, I see millions being spent in my constituency on High Speed 2 when we cannot spend £150,000 to keep a 10-year-old boy ambulatory and enjoying his life. We must question where a Government’s priorities are, when there are such people in front of us and we see the pie-in-the-sky projects that Governments of all complexions sometimes choose to pursue.

The point I really want to make is that if the decision is against providing the drug—bear in mind the failed processes that it has gone through—the Government have a golden opportunity to rescue the dish from the fire. I do not think it will necessarily fall out of the frying pan. On 8 July the Chancellor of the Exchequer will deliver his emergency Budget. We have previously created a cancer drugs fund, so that expensive drugs could be available to save lives. Will the Minister have conversations with the Treasury to see whether the Chancellor will on 8 July announce an access fund for drugs for rare conditions? If Translarna was one of the drugs on the list, it would be available in time for Archie Hill and the other children we have heard about today. To me, the awful thing is that time is running out. I do not think that letting time run out for those children would be the mark of a civilised Government, when the cost involved is small compared with some other expenditures that Governments make.