28 Feb A Renewed Momentum? The England Rare Diseases Action Plan
Today – on Rare Disease Day – the Government unveiled the England Rare Diseases Action Plan 2025. It marks a key step in maintaining momentum in the care and support of those affected by rare conditions before the UK Rare Diseases Framework expires in 2026.
The Rare Diseases Action Plan outlines how the health system in England will tackle the overarching priorities of the 2019 Rare Disease Framework. The priorities are to:
- help patients get a diagnosis faster
- increase awareness of rare diseases amongst healthcare professionals
- better coordinate care
- improve access to specialist care, treatment, and drugs
The Plan reports on progress made against the previous 36 actions and introduces an additional three immediate, year-long actions. The new actions augment the Framework priorities to better coordinate care and improve access to innovations.
Action 37: Clinics for multi-system disorder
To improve care coordination, the Government has committed to introducing a structural methodology that incentivises providers to run clinics for multi-system disorders. Patients with complex conditions are usually required to attend multiple appointments, often with different healthcare professionals and at different locations. This places a significant burden on people already living with challenging sets of symptoms, and can have financial implications in terms of taking time off work and travelling to appointments. Multi-system clinics bypass this by enabling patients to see a team of specialists in one visit. This approach not only enables cohesive care, but also improves understanding of the condition for both patient and clinician. How these clinics will be incentivised is yet to be determined, but will likely be informed by broader reforms being considered as part the Government’s 10-Year Health Plan for the NHS.
Action 38: Reform of clinical trial regulation
This commitment refers to existing and ongoing plans to reform clinical trials regulations. The plans, which follow extensive consultation by the MHRA in 2023, go beyond rare diseases, with hope the changes will bolster international investment in the UK rare disease landscape by default. Additionally, the MHRA will develop guidance on increasing public involvement in clinical trials, emphasising best practices rather than strict legislative requirements.
The decision to reform clinical trial regulations undeniably speaks to the Government’s economic growth aims. For patients, this may mean increased access to treatment – but workforce support is vital to create an NHS fit for change.
Action 39: An operational framework for individualised therapies
It is now possible to develop therapies specific to a patient’s DNA. Such individualised therapies aim to address the root cause of their condition. Given such treatments would only be appropriate and provided to one patient, this presents significant challenges to traditional processes for medicine regulation, reimbursement and service delivery. Over the coming year, NHS England will develop a highly specialised service operational framework to address these challenges. This is due to be finalised February 2026 and subsequently reported.
The 39 Steps in the right direction
The new actions are positive steps in the right direction, especially when combined with the successes highlighted by the plan. For instance, the Generation Study to sequence the whole genomes of up to 100,000 babies has now started recruitment at 24 hospitals. Meanwhile, results of an NIHR evidence mapping project revealed more about the inequalities in accessing health services which exist for people living with rare conditions.
NHS England has also finalised a service description and provider selection plan for pilot Syndrome With A Name (SWAN) clinics. However, in England these will be put on pause pending new funding becoming available. Despite the success, the challenges this Government faces in funding ambitious policy changes cannot be overstated.
Real people, real impact
While it’s easy to get caught up in the language of policy change, we cannot forget the very real people who stand to be impacted by the Action Plan. Yesterday, representatives from the OVID team were delighted to attend our client Genetic Alliance UK’s Westminster Rare Disease Day Reception, where we were inspired by the exhibition of pieces from the “More Than You Can Imagine” anthology of rare patient experiences.
We were moved by Jillian Hastings-Ward’s performance of her poem outlining the challenges of caring for a child with a rare condition, aligned to the theme of “More than You Can Imagine”. We also heard a speech by Minister for Public Health and Prevention, Ashley Dalton MP, zebra-print socks and all! Alongside reaffirming her Government’s commitment to addressing the priorities in the Framework, the Minister announced the launch of the England Action Plan.
It’s undeniable that this Action Plan comes at a crucial moment, providing the new Government with an opportunity to reduce disparities in care and ensure rare disease care is not left behind as part of its broader and ongoing reforms. But with no word yet on whether the Rare Diseases Framework will be renewed when it expires in 2026, the questions remain about how the Labour Government will continue to prioritise rare diseases as it builds a “health service fit for the future”.