Historically, universal access to the multidisciplinary services that are essential to the SMA community has not been seamless. We have seen huge regional ‘postcode lottery’ differences in service standards and speed of access to a range of local services including wheelchair services, occupational therapy and physiotherapy. If the ICS can assess how many people within their footprint share needs for these specialised services and allocate budgets accordingly, this will indeed be a step towards regional equality.  

In principle, it does make sense that all funding for local care and health services are included in a pooled ICS budget. However, we do have concerns that if there is no reserve budget for specialised services locally, there may not be enough money in the ‘pot’. The ICS is focused on ensuring ‘public resources are used where they can have the greatest impact’1  At a local level, the rare disease community is incredibly small, we do worry that spending money to improve their quality of life may not be considered a ‘great impact’. Will it make it easier to fund expensive specialist equipment and care that many disabled children and adults rely on for day to day living?  

Another problem faced by our community, that could be resolved by this model, is addressing the lack of training and communication from leading hospitals to local clinicians. In addition to the current population of people of all ages living with SMA with varying levels of severity of impact and need for services and support across the UK, since 2017 and the advent of new drug treatments, we also now have a wonderful new generation of children living with SMA. These are children with complex disabilities and needs with families needing regular and convenient access to services and support. The SMA community need their specialists to be supporting their local clinicians. Local health visitors, GPs, physiotherapists, and nurses are currently not routinely trained in the day-to-day symptom management of SMA and the medical equipment that facilitates this. If experts from leading hospitals who know the SMA population and their needs, could educate their satellite local hospitals using the new technologies adopted through lockdown, the quality and equity of care would be much improved, and diagnosis would be more efficient.  

Specialised care and quality of life for the patient could really benefit from using technology ‘to reimagine care pathways’2. This provider collaboration is crucial in what is a potential new era for SMA.  

In the UK, there is one baby born with SMA every 5 days. 60% of those babies will be diagnosed with SMA Type 1 – the most severe form. Before 2007, without intervention for breathing difficulties, most children with SMA Type 1 were expected to live for less than two years. New drug treatments are potentially life changing but early treatment is vital to achieve the greatest benefits. Any delay in diagnosis, even a matter of weeks, allows the disease to rapidly progress. The further on in disease progression, the more severe a child’s disability will be for the rest of their lives, which has a huge impact not only on the child and their families but on the finances of the NHS.  

Without newborn screening for SMA in the UK. which would ensure the earliest possible treatment of the condition, there could not be more urgency to use the new ICS framework to share the expertise of specialists, through remote learning platforms established during the pandemic. Local clinicians need to be effectively trained, so that diagnosis can be as quick as possible. As well as focusing on the needs of the population as a whole, ICS groups need to understand their potential to improve care pathways within the rare disease community, improving communication between specialist hospitals and local services could not only transform lives but also save money in the long run.  

It is also encouraging to hear that mental health services will be part of the systematic approach. The impact of a diagnosis of a rare disease like SMA on a patient and their family is huge. We would like to see a greater consideration given to the mental health of our community, coordinated at a local level and based on the choices and individual needs of the families.  

We see many health inequalities within the SMA population across the UK, one of the most significant differences is between paediatric and adult care. We asked adults living with SMA about their experiences, and comments included, “I think they completely forgot I existed when I turned 18” and “Honestly I haven’t seen anyone unless I asked to be re-referred”. Within the new landscape of treatment for SMA it is more important than ever that adult patients have equal access to clinics. The systematic approach presents an excellent opportunity for clinicians to work closely together to create a smooth transition pathway and to ensure young adults do not feel abandoned by their health providers,  ‘ICS leadership have the opportunity to shape the strategic health and care priorities for the populations they serve’.3 

SMA is a condition which comes with symptoms that are treated by a multidisciplinary team. Within some hospital trusts, people are considered holistically, rather than as a series of illnesses, whereas in others they seem to be always ‘bouncing’ around between specialities. The ICS must be used to implement good practice seen in some leading hospitals such as GOSH where SMA clinics are already established. In one trip to the hospital families can see a number of specialists, respiratory consultants, neurology and orthotics. Other families, who may live a considerable distance away are making numerous trips for different clinics, often using NHS resources on multiple patient transport journeys. To reduce these health inequalities there must be greater integration not only across primary, secondary and tertiary care but also between the specialisms within them. 

Sharing skills across the ICS is another key to reducing health inequalities, working in partnership provides an opportunity to see high standards across the system, not just in the specialised hospital. After what is often a long admission in a tertiary hospital, families we work with get very anxious about local step down care. We would like to see ICS’ work to support local services to provide a smoother transition into community care. Investing in community provision could reduce the reliance on inpatient services.  

The framework for the ICS admits that ‘Many crucial features of strong system working- such as trust between partners, good leadership and effective ways of working – cannot be legislated for’ 4  Without legislation, it is essential that the patient voice is listened to throughout this period of change and indeed into the future. The function of the ‘critical friend’ that the patient voice provides is an invaluable resource for improvement of services and patient experience. In our experience NHS England has had a consistent approach to seeking our feedback as a patient representative body, so we fully support them taking a lead on involvement activities for nationally commissioned services. It is important too that the ICS are included, so that all parties can learn from the patient experience, especially at the highly specialised level.  

It appears that the proposed new way of working with population-based budgets, larger footprints, a wide range of different services working together on a huge array of individualised care pathways has massive potential for teething problems.  Its success will be dependent on the good practice of innovative leaders. Patient voices are the key to identifying where this good practice is happening and where it is not. We hope that the ICS will embed patient voice into their self-improvement practice, seeking involvement from patient communities at regular intervals and finding creative and collaborative solutions to problems.

It makes sense that commissioning of highly specialised services should remain on a national level. Populations of rare disease communities like ours are so small that it is not reasonable to expect many providers to have the specific skills, knowledge and expertise to meet needs such as administration of drugs, or collection of outcome measures data for managed access agreements. Quality of care would surely suffer if multiple centres were suddenly expected to deliver highly specialised treatments. 

The day to day services that maintain the condition, those that our community share with others such as physiotherapy, speech and language services or personal care assistance should be part of the ICS in order to provide a more holistic service closer to home. With population-based budgeting, we would hope that there would be more money allocated for 1:1 personal care for disabled people living in their own homes. The low wages in the sector have led to a crisis where adults have had to sacrifice their independence because they can not find reliable personal assistants. We would be very interested to know how the new ICS will affect people managing their own personal independence budgets. Does the national funding allocation for each ICS consider individuals who rely on personal independence payments?