Spinal Muscular Atrophy

Debate between John Hayes and Seema Kennedy

Thursday 25th July 2019 Commons Chamber

[The Parliamentary Under-Secretary of State for Health and Social Care (Seema Kennedy)]

What a pleasure it is to speak in the final parliamentary debate of what has been an historic and tumultuous term for all of us.

I congratulate my right hon. Friend the Member for South Holland and The Deepings (Sir John Hayes) on securing the debate on such a timely day, because we have had news on Spinraza in the last 24 hours. He outlined very movingly the symptoms of spinal muscular atrophy, which has been raised before in this House. The hon. Members for Bristol East (Kerry McCarthy), for York Central (Rachael Maskell) and for North Tyneside (Mary Glindon) are particular champions for sufferers.

SMA is a terrible disease that can have a terrible impact on the children who suffer from it, their families and the wider community. We in the Department know how important it is for them to be able to access effective treatment. It is a rare disease. It affects about 100 babies born each year. It is estimated that it affects between 1,200 and 2,500 children and adults in the UK. Until recently, there were no licensed treatments for it, so the condition has been managed using nutritional support, physiotherapy and social care.

Spinraza, as my right hon. Friend outlined, has been licensed across Europe for the treatment of SMA since 2017 and is very effective. However, it is extremely expensive and has been undergoing assessment by NICE to determine whether it would be an effective use of NHS resources. I say to him that QALYs do take account of wider costs—not just physical costs but social care costs. As far as I am aware, there is currently no difference in the quantitative evidence. He gave the examples of a man in his mid years—at 60—and a child.

I will go on to talk more broadly about the NICE process, but I am really pleased that only yesterday, NICE published its final guidance, meaning that this debate is very timely. The guidance recommends the use of Spinraza for many patients with SMA through a managed access agreement, which has been negotiated between NICE, NHS England and the manufacturer, Biogen. This makes Spinraza available to the NHS at a discounted price. Importantly, it also sets out arrangements for collecting evidence on the long-term impact of Spinraza, because at the moment the evidence is quite immature. The managed access agreement is one of the most comprehensive deals in the entire world and it brings Spinraza to one of the widest cohorts of eligible patients in any country. It was signed and supported by Spinal Muscular Atrophy UK, Muscular Dystrophy UK and TreatSMA. I put on the record my thanks and my tribute to all those organisations for the support they have given patients and families with the illness. That very welcome development has involved hard work and flexibility from all parties to reach this point; if only all parties in all the debates I have to speak in on this matter were as flexible.

[Sir John Hayes]

The Minister makes a good point about the managed access agreement and the amendments to it that she described. But the real issue here is whether Spinraza would benefit every single sufferer. If it would, it should be universally available.

[Seema Kennedy]

I know that there is an issue with some people suffering from SMA type 3, but I am not the person who would make the decision on whether Spinraza was effective or not; that is why NICE is there.

Children with the most severe form of SMA type 1 are already benefiting from treatment with Spinraza. Following yesterday’s announcement, eligible patients with types 2 and 3 will begin treatment as soon as possible within the next three months.

NICE has concluded that there is not sufficient evidence at this stage for the managed access agreement to cover some patients with SMA type 3 or any patients with type 4; that is why we are still carrying on collecting evidence. I realise how dreadfully painful that is for those not able to access Spinraza. The majority of patients will be eligible under the managed access agreement.

The parties to the agreement have agreed to keep the eligibility criteria for treatment under review during the five-year term of the agreement, so those criteria may be further extended in future if more evidence of benefit emerges over time. NHS England and I have acknowledged that the inclusion criteria associated with the managed access agreement could have been more clearly communicated when it was announced.

I turn to the NICE process altogether. This is an important system. It makes independent, authoritative, evidence-based decisions, which is essential so that taxpayers can be assured that the price we pay for new medicines reflects the value that they bring. It also helps ensure rapid access to effective new treatments for NHS patients. It has been going for 20 years and is internationally respected. There are both established and new pharmaceutical companies developing medicines for rare diseases, which takes an awfully long time, based here in the UK, and medicines can be brought to the market through the NHS very quickly. The Department is keen to press on with that.

We have a UK rare diseases strategy, which was set out in the NHS long-term plan. Genomics is a particularly important area, in which we want to innovate so that we have more comprehensive and precise diagnoses and allow patients to access the right drugs. We are committed to that. The NICE process has recommended more than 80% of the medicines appraised and 75% of medicines for rare diseases for some or all of the eligible patient population, but of course the processes must evolve. They have to keep going, taking into consideration developments in science, healthcare and the life sciences sector. That is why NICE keeps its methods and processes updated through periodic review, which includes extensive engagement with stakeholders, including patient representatives, drug manufacturers and clinicians.

Acquired Brain Injury

Debate between John Hayes and Seema Kennedy

Thursday 9th May 2019 Commons Chamber

[The Parliamentary Under-Secretary of State for Health and Social Care (Seema Kennedy)]

It is a real pleasure to respond to this excellent debate on behalf of the Government. I would like to begin by thanking the hon. Member for Rhondda (Chris Bryant) for securing the debate. I commend him for the all the work he has undertaken as chair of the all-party group on acquired brain injury. He began by giving us a very vivid description of the physical impact of brain injuries, and went on to describe a whole gamut of mental health and emotional effects they have on people—and, of course, their families. He set me a challenge and I shall endeavour to meet it.

My right hon. Friend the Member for South Holland and The Deepings (Sir John Hayes) spoke with his usual eloquence. He reminded us about the scale of ABI, and emphasised the complexity of the issue and how it touches on many areas. The hon. Member for Swansea East (Carolyn Harris) told us all about George, reminding us that ABI can have extremely unexpected and devastating effects on the people who live with it and their families.

My hon. Friend the Member for Ayr, Carrick and Cumnock (Bill Grant), with his long career in the fire service, saw many people who had an ABI. He set us a very interesting challenge about having compulsory helmets for cyclists. I will take that point away and speak to my colleagues in the Department for Transport about it. The hon. Member for Blaydon (Liz Twist) outlined how people “just don’t get it”. That is a very important point, and it shows how important it is that we are debating ABI here today. She made a point about carbon monoxide poisoning, and I am due to have a meeting with the hon. Member for Rhondda on that very issue.

The hon. Member for Mitcham and Morden (Siobhain McDonagh) told us that the excellent charity Headway, which was mentioned very many times during the debate, is based in her constituency. She reminded us that a brain injury can strike any of us at any time. We also heard from the hon. Member for Caithness, Sutherland and Easter Ross (Jamie Stone). He spoke very movingly, as he did in the debate last year, about his wife’s experience and his. All I can say is keep going on the crossword.

Brain Injury Awareness Week is from 20 to 26 May. I welcome the time Parliament that has set aside to discuss this issue. Prevalence estimates for ABI are problematic to make, but it is likely that the number living with ABI is definitely over half a million and could be as high as 1 million. The total cost of brain injury in the UK is estimated to be at least £1 billion per year. Charitable organisations such as Headway and the UK Brain Injury Forum, as well as other local and national groups that hon. Members have referred to, are highly valued by those affected. They raise awareness and provide help to those with the condition, as well as to their families and carers, and I want to put on the record our appreciation for everything they do.

As we have heard, in 2018 the APPG held a wide-ranging inquiry into the causes, impact and treatment of ABI. My predecessor, my hon. Friend the Member for Winchester (Steve Brine), to whom I pay tribute for the energy he brought to this matter, agreed to respond to that report, and my Department co-ordinated with officials across Whitehall to deliver that response on 19 February. In responding today, I will draw on key areas of the response to set out the relevant activity that is under way.

[Sir John Hayes]

I thank my hon. Friend for giving way, and I know she will address these matters with her usual acumen and assiduity. I spoke about dynamic disability and the pace of recovery, but will she take into account the fact that aligned with that is the subtlety of the effects of brain injury? Sometimes a person may be deemed to have recovered completely and to have returned to normal—whatever normal is—but their manner, meter and mood might have changed and their sense of appropriateness might have altered, and that has effects in education and employment, in particular, as well as in personal relationships.

[Seema Kennedy]

My right hon. Friend makes an extremely important point, showing that this is an issue not only for the health system and my Department but for others across Government.

While the majority of rehabilitation care is locally provided, NHS England commissions specialised services for patients with the most complex levels of need. For people who have ABI, neuro-rehabilitation that is timely and appropriate is an important part of their care. Access to high-quality rehabilitation improves outcomes for patients and can save money. The shadow Minister mentioned rehabilitation prescriptions. RPs reflect the assessment of the physical, functional, vocational, educational, cognitive, psychological and social rehabilitation needs of a patient and are an important element of rehabilitation care. The APPG report was clear that all patients with ABI should benefit from an RP.

NHS England’s major trauma service, where acute phase rehabilitation begins, sets out that patients should be reviewed by a rehabilitation consultant. The shadow Minister asked about this. The development of major trauma centres, which the hon. Member for Rhondda supported, has improved recruitment to this specialty, while the national clinical audit of specialist rehabilitation recommended that all trauma networks review access to rehabilitation consultants and make improvements. Patients should have either a rehabilitation consultant or an alternative clinician with skills and competencies in rehabilitation to provide an initial formulation and plan to complete and perform the initial RP. At discharge, all patients should have a patient-held record of their clinical information and treatment plan from admission as they move to specialised or local rehab.

The “National Clinical Audit of Specialist Rehabilitation for Adults Patients with Complex Needs Following Major Injury”, published in 2016, found that on average 81% of patients had a record of a rehabilitation prescription. The audit recommended that MTCs take action to improve compliance. The audit report appears to have had a significant impact. The latest data, from the last quarter of 2018, from the trauma and audit research network shows that the national average rose to a 95% completion rate for RPs. This is good news. NHS England has worked with patients, clinicians and charities to improve the RP design and set new standards for communication and involvement of patients, families and carers. It is hoped that the new RP will support the development of a rehabilitation dashboard to monitor the performance of the system. Audits play an important role in helping services to improve. The report also recommended that all organisations within a trauma network work together to review capacity.

The majority of rehabilitation care is commissioned and managed locally. To support commissioners to plan services for local populations, NHS England has produced a document, “Principles and Expectations for Good Adult Rehabilitation”, that describes what good rehabilitation looks like. Additional guidance covering adults and children sets out a commissioning model and the evidence base for delivering high-quality rehabilitation services.

The hon. Member for Rhondda mentioned that ABI spans many Departments, and I shall take away all the comments that concern my ministerial colleagues and will ask them to respond. On support for children with ABI in school, the special educational needs and disabilities system is designed to support all children and young people with additional needs. The arrangements for SEND are intended to support joint working between health, social care and education; multi-professional assessment of a child or young person’s needs involving relevant experts; and the development of an individual education, health and care plan to meet those needs. This should provide a basis for the sharing of information and expertise to ensure the needs of children and young people with ABI are supported in school.

The hon. Member for Blaydon mentioned the ABI card. The Department for Education has said that promotion of the card is a matter for individual schools, but as far as my Department is concerned, Professor Chris Moran, a national trauma director, said that he would be happy to promote the card in trauma networks, working with the Brain Injury Trust. The statutory guidance on supporting children with medical conditions at school covers a range of areas, including the preparation and implementation of school policies for supporting pupils, the use of individual healthcare plans, consulting with parents, collaborative working with healthcare professionals and staff training. The Department for Education continues to work with organisations such as the Health Conditions in Schools Alliance to help to raise further awareness of the duty on schools.

On prisons, there is an increasing body of evidence linking ABI to offending behaviour. NHS England’s liaison and diversion service has collaborated with Headway to develop workshops to improve awareness and identification of ABI in vulnerable offenders and the support available. The “train the trainer” workshops were designed so that attendees could return to their services and cascade workshop learning to their colleagues. Representatives of all NHS England-commissioned liaison and diversion services attended. Over the past two years, the Ministry of Justice has also piloted approaches to improve screening and support for prisoners through new link worker roles at six sites on the male secure estate. I take the point about female offenders and will speak to the relevant Minister. There was a pilot at a female prison between 2016 and 2018, but I will take away the point about the female estate.

I want briefly to touch on the point raised by my right hon. Friend the Member for New Forest East (Dr Lewis), the Chair of the Defence Select Committee. The veterans trauma network delivers comprehensive medical care to veterans, including those suffering from brain tumours, and, as he knows, the Prime Minister opened the successor Defence centre to Headley Court last year. We do not recognise his statistic that there are only two machines, but I will take that away and report back to him.