Commission: Public services that work from the start
Topic: Prevention, early intervention and better public services for all
Answer to Question 1: How can Labour ensure our public health services prevent worsening population health, ensure pandemic preparedness, address widening health inequalities, and oﬀer early intervention programmes that reduce pressure on our communities and other services (in conjunction with wider social policy)?
The Rare Autoimmune Rheumatic Disease Alliance (RAIRDA) is asking Labour to consider the needs of people with rare diseases, specifically rare autoimmune rheumatic diseases (RAIRDs), in their public health policy.
As part of Labour’s prevention agenda, it is important to focus not only on preventing disease from occurring, but on reducing the impact of diseases when they do occur. Labour’s ongoing policy development needs to ensure that people with rare conditions have access to diagnosis within reasonable timeframes, care which is coordinated smoothly across specialties, protection from ongoing threats such as COVID-19 and future pandemics, and better access to treatments.
RAIRDs are conditions which are characterised by the body’s own immune system becoming overactive and attacking healthy tissues, often in multiple organs throughout the body simultaneously. This can lead to tissue or organ damage, which can be fatal. RAIRDs can affect many parts of the body, including joints, skin, lungs, kidneys and the heart. They can be split into two groups: connective tissue disorders (lupus, scleroderma, myositis, primary Sjögren’s syndrome) and systemic vasculitis (ANCA-associated vasculitis, giant cell arteritis, Takaysu’s Arteritis and Behçet’s disease).
RAIRDA is the first alliance linking clinical and patient organisations to campaign for improved care for people living with RAIRDs in the UK. RAIRDA is comprised of LUPUSUK, Vasculitis UK, Scleroderma and Raynaud’s UK, and the British Sjӧgren’s Syndrome Association.
It may not be possible to prevent RAIRDs from occurring as they (like 20% of rare diseases) are predominantly non-genetic. However, people with RAIRDs are facing poor outcomes which are preventable, including, in some cases, premature mortality. These poor outcomes are due to issues such as delays in diagnosis and treatment, poor care coordination and inadequate access to vaccines and priority treatment.
Whilst the UK Rare Diseases Framework (2021) has been a significant step towards improving care for rare diseases, there are gaps in the framework which mean that people with RAIRDs are still at real risk of preventably poor health, and are experiencing widening health inequalities compared to those with common conditions. The England Rare Diseases Action plan for 2023 does address some of these gaps, particularly regarding non-genetic diseases. However, there is further to go. RAIRDA’s policy paper, Resetting the Balance (2022), outlines a roadmap for better care and treatment for people with RAIRDs.
Another important consideration from a policy perspective is ensuring that there are incentives and metrics in the system that drive change in rare as well as more common conditions. RAIRDA’s co-chair is also the chair of the Independent Advisory Group (IAG) on quality standards to the Forum on the UK Rare Diseases Framework. The group is calling for the development of a National Institute for Health and Care Excellence (NICE) quality standard for rare disease. Quality standards set out priority areas for quality improvement in health, public health and social care, and can be used to help improve quality and measure progress via audits. This group is calling for this quality standard as they believe it will drive improvements in care for rare diseases, including RAIRDs.
If Labour’s policy could pay specific attention to policy focus on rare diseases, including RAIRDs, this would help combat the health inequalities faced by people living with these conditions.
Worsening Population Health
In the UK, there are over 170,000 people living with RAIRDs, and an estimated 3.5 million people living with rare diseases. Individually, these conditions are rare, but collectively, rare diseases are common and affect a significant proportion of the population. Yet, in part due to the backlog caused by COVID-19, the NHS is too focused on conditions which are common and easily treatable, to the detriment of the many people whose individual conditions are rarer.
For example, RAIRDA’s 2018 survey showed nearly half of respondents waited over three years from symptom onset to receive a RAIRD diagnosis. It is unfair that the significant proportion of the population who live with rare diseases have to wait for so much longer than those with low-complexity, high-volume conditions, often with significant impacts on their health through adverse outcomes. For example, of people who are diagnosed with the subtype ANCA associated vasculitis, approximately 15% will die within the first year.
To ensure that the health of these 3.5 million people is protected into the future, it is important that health policy and services take a greater focus on preventing people’s conditions from worsening. For example, if a quality standard for rare disease was in place with diagnosis targets, it would ensure that people are seen before their condition reaches a critical point, preventing future adverse outcomes which also place strain on NHS resources. Thus, prevention policy should include provisions for creating parity in the care and treatment received by those with both common and rare conditions.
This is a particularly pertinent point for people who are immuno-compromised, which includes many people with RAIRDs. Research from the RECORDER project in March – July 2020 showed that people with RAIRDs had a 54% increased risk of COVID-19 infection and more than twice the risk of COVID-19-related death compared to the general population.
Despite this, many had difficulties accessing vaccines and treatments during the most recent pandemic. For example, RAIRDA’s 2021 survey found significant issues with the rollout of the third primary dose to people with rheumatic conditions.
The Joint Committee on Vaccinations and Immunisation (JCVI) issued guidance stating that people with compromised immunity were eligible for a third primary dose and a subsequent booster. Yet two months after this guidance had been issued, around one-quarter (22%) of those who responded to RAIRDA’s survey had not been able to access a third primary dose, and for those who had, two in five respondents (41%) said that it was much more difficult to access than their previous doses.
To reduce mortality and morbidity in future pandemics, as well as in seasons where high levels of other respiratory viruses such as flu are prominent, we need to improve the identification of the most vulnerable, including people with RAIRDs, to ensure they have priority access to drugs and treatments. To address this there need to be better ways of identifying these people. This includes having their eligibility effectively and consistently recorded across primary and secondary care records, something which is not happening at the moment. Accurate and up-to-date digital coding of NHS records would mean that professionals can rapidly and reliably identify RAIRD patients who need priority access. Further, communication between people with RAIRDs and professionals needs to improve: people with RAIRDs should be aware of their right to access vaccines and treatments, and to have enough information to know how to do so.
If Labour’s policy is able to address the above areas, then it could potentially mitigate some of the impact of future pandemics or seasonal occurrences of flu and other respiratory conditions.
RAIRDs can be life threatening and significantly life-altering, yet prevention of these adverse outcomes through early diagnosis has not received sufficient focus. As mentioned above, RAIRDA’s survey showed that nearly half of respondents waited over three years from symptom onset to receive a RAIRD diagnosis. This means that people’s conditions are being allowed to progress far further than they would if an adequate diagnosis and treatment were received sooner.
Health policy which sets out clear targets for diagnosis and the start of treatment for people with rare diseases would drive improvements and prevent a greater burden on the NHS later down the line: similar targets have led to significant benefits for other more common conditions such as rheumatoid arthritis. For example, the The National Early Inflammatory Arthritis Audit, led by the British Society for Rheumatology, has shown steady improvement in services in England and Wales against the 2013 NICE quality standard for rheumatoid arthritis.
Widening Health Inequalities
After a diagnosis is reached, for people with RAIRDs, consistent monitoring and access to consultants for treatment reviews are a critical part of preventing unnecessary disease progression and adverse outcomes. As NHS services are too focused on common conditions, people with RAIRDs aren’t receiving the consistent, high quality treatment they need.
For example, recent reports from RAIRDA’s member charities suggest that some patients are being left months without drug monitoring or regular blood tests for disease activity. This means the current system is preventing some people with rare disease from experiencing an achievable level of good health. The resulting morbidity and mortality is preventable, if health policy and services take a greater focus on preventing people’s conditions from worsening.
The development of a National Institute for Health and Care Excellence (NICE) quality standard could be a useful tool to drive improved outcomes for RAIRDs. They set out priority areas for quality improvement in health, public health and social care, and can be used to help improve quality and measure progress via audits.
For example, if a quality statement around monitoring was put in place, it would mean that healthcare services would be audited against the statement, thus encouraging them to meet the minimum requirement for monitoring.
RAIRDA is grateful for the opportunity to feed into this important consultation, and hopes that improving care and treatment for rare diseases, particularly RAIRDs, will form part of Labour’s policy going forwards. RAIRDA is happy to provide any additional evidence required.
 Rutter, M. et al., “COVID-19 infection, admission and death among people with rare autoimmune rheumatic disease in England: results from the RECORDER project,” Rheumatology (2021).