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Creating unity and equitable consistency across the home nations: Implementing the Rare Diseases Framework

With the four nations responsible for implementing their own action plans of the Rare Diseases Framework, how are we going to ensure that people have equitable access and support? How can the UK avoid a postcode lottery and end the current disparities between nations? 
Ben Allcock, a rare diseases policy officer in the Genomics Science and Emerging Technologies team in the Department for Health and Social Care, explains what the next steps are in implementing the Framework, and how they are balancing meeting the individual needs of each of the home nations with a commitment to health equity.
Ben Allcock
Q. Could you explain to us the next steps involved for the four nations in implementing the Rare Diseases Framework in the UK? 

All four nations have committed to publishing their action plans by the end of 2022, although some are expecting to publish earlier.

Following publication of the UK Rare Diseases Framework, each of the four UK nations has set up its own delivery/implementation board. These boards are responsible for drawing up and monitoring nation-specific implementation plans, detailing how each of the priorities of the Framework will be met. 

Our team has oversight of the England Rare Diseases Framework Delivery Group, which is currently meeting every six weeks to develop actions together with delivery partners (including NHS England, NICE, the MHRA, Health Education England, Public Health England, Genomics England and others), and representatives from the rare diseases patient and clinician community. These individual actions will be specific, measurable and timebound, and will demonstrate each organisation’s commitment to meeting the goals of the Framework.

All four nations have committed to publishing their action plans by the end of 2022, although some are expecting to publish earlier. Throughout development of the action plans, each nation will be engaging with members of the rare diseases community, to make sure the action plans are relevant and fit for purpose. Once published, the action plans will be updated and reviewed annually to measure progress and take new opportunities into account.

With each nation responsible for its own implementation, what safeguards will be in place to ensure the framework is going to be equitable throughout the UK? 

​To ensure coordination of rare diseases policy and action plan development across the UK, DHSC has also set up two UK-wide boards which will work together with the nation-specific implementation boards. The first is the UK Rare Diseases Framework Board, which will focus on high-level strategy and making sure all four action plans are aligned across nations.

  1. ​This board is co-chaired by Professor Chris Whitty (Chief Medical Officer for England), and senior officials from each of the four nations, who will serve as co-chairs on a rotational basis.
  2. ​Members include the chairs of each of the implementation boards, as well as policy and NHS representatives from each of the four UK nations, and representatives from the rare diseases community.
  3. This board will provide a UK-wide perspective and will review and offer advice on the action plans as they are developed.

The second is the UK Rare Diseases Forum, which we have set up to engage with the broader rare diseases community across the UK.

The Forum consists of two parts: a core group of about 30 members, representing patients and carers, industry, research, healthcare professionals and others, who meet regularly to provide specific input on the development of all four nations’ action plans; and 

An online platform, which is open to the broader rare diseases community. This is where we post updates, news, minutes of meetings and other important documents, to promote transparency and offer insight into the progress made across the four nations. Members of this platform are encouraged to engage through participating in chats, answering questionnaires and posting their own views on the progress being made towards implementing the Rare Diseases Framework.

Aside from these formal structures, the teams responsible for rare diseases policy in England, Scotland, Wales and Northern Ireland meet regularly to share updates and ensure our plans are coordinated.

Q. What are the challenges you foresee in achieving this and what solutions/activities will be needed to overcome these?

Developing nation-specific action plans involves balancing the specific health needs of the individual UK nations with the Government’s commitment to health equity and avoiding disparities between nations. With several of our delivery partners operating across the UK, we are currently working together with them to develop projects with a cross-nation reach.

Through a combination of nation-specific and UK-wide approaches (as outlined above) and by maximising our engagement with the diverse rare diseases community, we hope to identify and address any potential areas of inequality, and ensure that we improve the lives of people living with rare diseases, regardless of where they live.

​Contact our team to find out more about getting involved and signing up to the online platform for updates: gset@dhsc.gov.uk

These links are to third party, non-Alexion websites:

 Charity Perspective – Rhoda Walker, Chair – Northern Ireland Rare Disease Partnership

“The Northern Ireland Rare Disease Partnership are excited for the year ahead.   We are delighted to be part of the Northern Ireland (NI) Rare Disease Implementation Planning Group and to represent our nation on the UK Rare Disease Stakeholders Forum.

Momentum has been gathering, as never before in the world of rare disease and, for all the trials the community has faced because of the pandemic, there have been real moments of light and inspiration gained from the challenge to do things differently. 

We know we can only truly take advantage of this window of opportunity by working in partnership with all the other rare disease stakeholders, learning from each other, working to minimise duplication of effort, streamline existing services to work smarter, not harder and maximise on the potential synergies that partners bring to the table.

Rhoda Walker

Co-production, design and delivery is the way forward and we look forward to working hand-in-hand with our partners across the UK and Ireland. We encourage all involved to reflect on the asset base that already exists and how it might be supported and used more effectively, to consider simple before complex solutions, and to ensure the patients voice continues to be so well represented across all areas of programme management and delivery.”

NIRDP are committed to advocating, connecting, educating and innovating on behalf of the rare disease community in Northern Ireland.  We work to ensure that no-one is disadvantaged due to the rarity of their condition.  For more information or support contact us at info@nirdp.org.uk or call 07704476930.


Charity Perspective- Rick Thompson, CEO – Findacure

“When it comes to rare diseases, collaboration will always be king. Only by working together, uniting patients, charities, diseases, and countries can we deliver effective and meaningful change to the care and support available to those affected.

​The Rare Disease Framework provides a unique opportunity to drive change in rare diseases, and in particular to deliver equitable care to patients. However, this can only be reached through the careful coordination and collaboration of the health services of England, Scotland, Wales, and Northern Ireland.

It cannot be acceptable that two patients with the same condition living in Liverpool and Wrexham have access to wildly different treatment options and care, despite living no more than 30 miles apart. 

Rick Thompson

This type of situation remains far too common within each nation today, let alone between them. Rare diseases are tackled by pooling resources, connecting experience, and listening to those affected. Let us hope that the Rare Framework moves to implement this type of approach to deliver meaningful change for our community over the next five years.”

Findacure is a UK charity that is building the rare disease community to drive research and develop treatments. We are here to transform the world’s understanding of rare diseases and the devastating impact they have on people’s lives.


Charity Perspective –  Ceridwen Hughes, Creative Director – Same but Different

​”We welcome a UK-wide plan that listens first to the different challenges of implementation across nations and regions. We are based in Wales and provide rare disease support through our Rare Navigator service. We also work with individuals and families affected by rare disease throughout the UK to give them a stronger voice through the arts.

This breadth of experience has highlighted the commonalities in the rare disease experience across conditions and the need for a joined up approach between nations, regions and local health boards. 

Ceridwen Hughes

​If we’re going to see a real difference a cohesive and genuinely actionable plan is required, with clear accountability from the start.  We welcome a UK wide plan that offers transparency and coordination of care that truly represents the patient voice.”

By intently listening to these collective experiences, we collaborate and design support services that help meet each community’s need. We regularly partner with all types of organisations to create evocative and thought-provoking campaigns to raise awareness, bring change and ensure communities can get the support they need.

Date of preparation: August 2021
​Document No: UK/NP/0014

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