Potentially life-saving gene therapy now available on the NHS
Described as the ‘world's most expensive drug’, Zolgensma is now available on the NHS to treat children with spinal muscular atrophy, the leading genetic cause of death for children
Spinal muscular atrophy (SMA) is a genetic condition that causes weakening of muscles and problems with movement (NHS, 2020). Until 2 years ago, there were no treatments available for children diagnosed with this disease, but a landmark deal between the NHS and Novartis Gene Therapies has made this cutting edge gene therapy available on the NHS (NHS England, 2021).
Zolgensma, which has a list price of £1.795 million per dose, is a gene therapy that aims to target the cause of SMA. SMA is caused by a defect in a gene called SMN1 that produces a vital protein for nerves that control muscle movements (European Medicines Agency, 2021). The treatment uses a virus to deliver a corrected version of the SMN1 gene into the child's body (European Medicines Agency, 2021).
The use of gene therapy is part of the NHS Long Term Plan, which was released in 2019 and outlines the NHS plans to improve care for patients in the next 10 years (NHS, 2019). Part of the plan focuses on cutting edge treatments and therapies to save and improve lives. The NHS Chief Executive, Sir Simon Stevens, said it was ‘fantastic news’ that the treatment was now available on the NHS, and that ‘Zolgensma is the latest example of the life-changing therapies that the NHS is now routinely using to transform the lives of patients and their families’.
The treatment has been administered to its first patient, a 5-month-old baby who was born 6 weeks premature in December and was diagnosed with SMA in May. The treatment was given at Evelina London Children's Hospital, and three other specialist NHS centres in England are now confirmed as having been commissioned to administer this treatment (NHS England, 2021). These sites are the Manchester University NHS Foundation Trust, the Sheffield Children's NHS Foundation Trust, and the University Hospitals Bristol and Weston NHS Foundation Trust.
Zolgensma is only the latest in a chain of deals that the NHS has established to provide access to innovative medicines for patients in the last year. Other recent agreements included access to chimeric antigen receptors cell therapy, a treatment for children and young people with B cell lymphoblastic leukaemia and lymphoma (Cancer Research UK, 2021; NHS England, no date), and siponimod, a daily tablet used to treat secondary progressive multiple sclerosis (Multiple Sclerosis Society, 2020).
Matt Hancock, Health and Social Care Secretary, commented that ‘through the NHS Long Term Plan, we are doing everything we can to provide people with access to the latest generation of innovative medicines through the NHS’.
Planned children's recovery budget ‘falls far short’ of the mark
A coalition of campaigners has urged the government to reconsider its plans to invest only £1.4 billion in children's recovery after the COVID-19 pandemic
It has been more than a year since the UK first went into lockdown to combat the spread of coronavirus disease 2019 (COVID-19) (Gov.uk, 2020). After many months of school closures, alongside advice to stay at home and minimise social contact, there is already evidence that children and young people have been negatively affected by the pandemic (Mental Health Foundation, 2020).
The government announced on 2 June 2021 that it would be investing £1.4 billion to help children recover from the impact of the pandemic, especially on the lost learning resulting from school closures (Gov.uk, 2021). This includes £1 billion to support up to 6 million tutoring courses and £400 million to train and support early years practitioners and school teachers.
However, many have said that this funding is not enough to support recovery for children and young people across the UK. In May, the Education Policy Institute (2021a) released a report that found that a total of £13.5 billion would be required to ‘reverse the damage to pupils' learning as a result of the pandemic’. The report incorporated research conducted for the Department of Education on lost learning during the crisis (Education Policy Institute, 2021b), as well as economic modelling for the long-term impact on young people's employment chances and a review of the most effective policies that support pupils' wellbeing.
A coalition of campaigners from across sectors including education, charity, business and unions have signed two letters to the Prime Minister, urging further investment to be made to help children and young people recover from the pandemic (National Children's Bureau, 2021). The letters were coordinated by the National Children's Bureau and the Fair Education Alliance and in total have more than 240 signatures from leaders in education, charity and business.
The letters outline the issues facing children and young people after a year of ‘disruption, isolation and anxiety’, and call for the government to increase investment in staff development and interventions for pupils. They also state that wider investment is required, to address issues such as rising child poverty, increased waiting times for mental health services for children and young people, and tackling the overwhelming need for services to protect against abuse and neglect.
The letter coordinated by the Fair Education Alliance (2021) focused especially on the needs of disadvantaged children, stating that for these children in particular, ‘the crisis in education is not limited to the damage the pandemic caused’ and that it ‘merely exacerbated existing critical issues’. The letter outlined recommendations for the government, including investing the full £13.5 billion recommended by the Education Policy Institution, and focusing on a number of issues, including support and development for the education workforce, investment in enrichment activities for children from disadvantaged backgrounds and careers education for disadvantaged young people.
Dr Vanessa Ogden, Chair of the Fair Education Alliance, commented that ‘this moment presents an opportunity for our government to demonstrate in hard cash terms a genuine commitment to children in post-Covid recovery’ and that ‘we all stand to benefit from a future with well-educated, highly skilled and socially and mentally healthy workforce, undamaged by poor investment in pandemic recovery’.
‘The government announced on 2 June 2021 that it would be investing £1.4 billion to help children recover from the impact of the pandemic…In May, the Education Policy Institute…found that a total of £13.5 billion would be required’
School absences for COVID-19 have increased
The latest figures from the Education Settings Survey show that absences for COVID-19 in state-funded schools has almost doubled since the start of the summer term
The most recent data concerning school attendance in England was released on 8 June 2021, summarising attendance rates up to 27 May 2021 (Gov.uk, 2021). It shows that absence because of COVID-19 has almost doubled to 1.8% in state-funded schools, following a ‘period of stability’ at approximately 1% since the start of the summer term.
The data are from the Department for Education's daily education settings survey and is reported by schools. An absence for reasons relating to COVID-19 include those with a confirmed or suspected case of coronavirus, those who are self-isolating and those enrolled in schools that are closed because of COVID-19. These data do not include pupils who are shielding.
The largest proportion (1.1%) of the absent pupils are those who are self-isolating because of contact inside school. Geoff Barton, of the Association for School and College Leaders (2021), described the trend as ‘very worrying’, and said that ‘we are clearly now seeing the impact of the Delta variant feeding through into these statistics’.
As well as the increase in pupil absences resulting from COVID-19, there has been an increase in workforce absences because of COVID-19. The figures for 27 May show that 0.7% of teachers and school leaders and 0.6% of teaching assistants and other staff in state-funded schools were absent, up from 0.5% for both cohorts on 20 May.
Book of stories on school nursing released by Queen's Nursing Institute
‘Stories of School Nursing’ is a book featuring 19 case studies written by school nurses about their experiences in their profession. It has been released by the Queen's Nursing Institute, supported by Public Health England. It was written after a workshop was run in 2019 by Suzanne Gordon, an award-winning journalist and author.
Dr Crystal Oldman CBE, Chief Executive of the Queen's Nursing Institute said she was ‘delighted’ about the book's publishing, and that the aim was ‘to bring the incredible diverse, complex and challenging work of school nurses to a much wider audience’.
The stories showcase the vast range of experiences and responsibilities that school nurses have in their role. They range from discussing supporting children and young people who are carers for family members with a physical or mental health problem, to difficult issues such as handling self-harm and suicide. Throughout the book, the clear message that the work is that the work that school nurses do is ‘critically important’ for children and young people throughout the UK.
Suzanne Gordon wrote the foreword to the book, and describes how she felt ‘privileged’ to be part of a project aimed at recognising the ‘value of nursing’, especially as their work was made so much harder by the pandemic. Furthermore, the Institute does not wish this to be a ‘one-off event’, and there is currently a plan to collect stories from community nurses regarding their experiences during the COVID-19 pandemic.
Suzanne says that is was ‘an honour and a pleasure to help nurses write about their work. I hope that more members of the public, policy makers, hospital and healthcare administrators and even other nurses will listen to their voices and value their work’. She was ‘thrilled’ to have been made a fellow of the Queen's Nursing Institute in June 2019.
Further information and resources
- Queen's Nursing Institute book of stories: https://www.qni.org.uk/wp-content/uploads/2021/05/School-Nurse-Stories-2.pdf
