2025 England Rare Disease Action Plan: what does it mean for people affected by rare neurological conditions?

12/03/2025

Rare Disease Day 2025 (28 February) saw the publication of the 2025 England Rare Disease Action Plan, available in full here, but what does it mean for people affected by rare neurological conditions?  Our Policy and External Affairs Manager, Sam Mountney, takes a closer look.  

Fourth and final plan 

This is the fourth England action plan published by the Department of Health and Social Care. It builds on previous nation specific action plans and sets out actions and progress to deliver on four key national priorities of the UK Rare Diseases Framework. 

  • helping patients get a final diagnosis faster 
  • increasing awareness among healthcare professionals 
  • better co-ordination of care 
  • improving access to specialist care, treatment and drugs 

Around half of rare conditions are neurological so we were pleased to once again join several of our rare neurological conditions members in supporting the development of the plan as part of the England Rare Disease Patient Advisory Group coordinated by Genetic Alliance UK. Our involvement included providing feedback on earlier drafts, informed by the rare neurological community. 

New actions for 2025 

The 2025 plan includes 3 new actions alongside updates on progress against 36 actions from previous plans. New actions include: 

  • Clincs for multi-systems disorders – in repose to some of the challenges facing  people with multi-system disorders, this action will incentivize and implement clinics for multi-system disorders to reduce the number of individual appointments and the burden of care co-ordination often faced by people affected, their families and carers. 
  • Reform of the clinical trials regulations – forthcoming changes to rules around clinical trials will look to address some of the challenges facing rare disease clinical trials including supporting UK participation in multi-regional clinical trials and developing guidance around patient and public involvement in clinical trials. 
  • Developing an operational framework for individualised therapies – some medicines can now be tailored specifically for individuals with certain rare conditions but there are challenges with delivering them through the health system. This action will develop a framework to support equitable NHS delivery of individualised therapies where appropriate. 

We’ve summarised some of the updates on progress against priority areas and actions from previous actions plans below.  

Mental health remains an area of focus 

We know that rare neurological conditions can have a significant impact on mental health and too often, accessing appropriate mental health support a challenge for many. Ensuring that the plan recognised and acted on these concerns was once again a key focus. The 2025 plan once again reminds Integrated Care Boards (ICBs) and providers that mental health services should be offered based on need and not exclude anyone as a result of their neurological diagnosis. The annex to the 2025 plan also highlights inequities of access to services, including mental health services, for people affected by rare conditions. 

Going forward we will continue to work with our rare conditions members to highlight and address challenges around accessing appropriate mental health support. 

Care co-ordinators highlighted 

People with rare neurological conditions often require input and support from a wide variety of services across the health and care system, making effective co-ordination a critical aspect of good care. We were pleased to see the vital role of care co-ordinators highlighted in the 2025 action plan but were reminded of the challenges many people face with findings from the CONCORD care co-ordination study showing that just 12% of patients and 14% of parents or carers reporting having access to a formal care co-ordinator. 

The plan also includes a new action for 2025 (Action 37) to incentivize and implement clinics for multi-system disorders with a view to reducing the number of individual appointments and the burden of care co-ordination often faced by people affected, their families and carers. 

Transition from children’s to adult services 

There is limited reference to actions to improve the transition from childrens to adult services in the 2025 plan but we’re aware this remains a challenge for many. The plan does mention the updated NICE quality standard on transition from 2024, referenced in our 2024 action plan explainer, plan along with a note on a forthcoming NHS England healthcare transition framework, expected to be published in early 2025. 

We will continue to work with key stakeholders, including professional bodies, to build a consensus around improving transition for people affected by neurological conditions, including rare neurological conditions. Findings from My Neuro Survey 2025, expected to be published this summer, will also include an update on experiences of transition for children and young people and those who support them. 

What next for the UK Rare Disease Framework? 

The 2025 plan sets out more details on a range of actions and priority areas. We have focused on areas that we know are of critical importance to the rare neuro community and that we raised in our feedback to the Department on earlier drafts. 

In line with the timetable set out in the original UK Rare Disease Framework published in January 2021, this is the final plan of the 5-year framework. Together with colleagues from across the sector, we are calling for the vital work to improve services and support for people affected by rare conditions to continue past 2025/26. 

We welcome the commitment from the Government to work with the sector and governments across the UK to develop next steps for this important policy area. The UK Rare Disease Framework has been an important catalyst for action on some of the key challenges facing the rare neuro community, but there is still lots more to do.  

Importantly, the Framework did not have funding attached to some actions, which has hampered progress. The 2025 plan highlights work to develop Syndrome Without a Name (SWAN) clinics in England and self-reporting mechanisms for the NCARDS rare disease registry have been paused due to funding constraints. This must not be the case for future efforts to improve care for people affected by rare conditions. 

As an Alliance we will continue to champion this vital cause going forward alongside ongoing work through our Rare Conditions Subgroup and steering group role in the development of a Rare Disease Quality Standard. 

Ensuing the UK Neuro Forum delivers for the rare neuro community 

There are also new opportunities to help address some of these challenges through the UK Neuro Forum. The potential to discuss and explore challenges and solutions around accessing care across borders in the UK will be particularly relevant to people with rare neurological conditions. UK-wide discussions on research and the neuro workforce also have the potential to benefit the rare neurological community. 

As members of the UK Neuro Forum, we will ensure that the needs of people affected by rare neurological conditions are heard in these important discussions.