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"Working together we can secure the best use of scarce expertise and resources, maximising the health gain for all those with rare conditions" Alastair Kent OBE, Chair of RDUK

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You are here: Home / UK Rare Disease Strategy / Background to a Strategy

Background to a Strategy

This page provides some information on why we need a UK Strategy for Rare Diseases, what has been done so far, what RDUK has done and how you can help ensure that an effective strategy for rare diseases is implemented in the UK.

Please click the questions below to skip to each section:

Why is a UK Strategy for Rare Diseases needed?

In the UK it is estimated that 3.5 million people will be affected by a rare disease at some point in their lives. Despite the scale of the issue, a significant proportion of these patients do not receive an adequate response to their needs from the NHS.

It is not that those working within the NHS are uncaring or lack the will to support patients or families with rare diseases. Rather that the systems and structures are not in place to adequately respond to the needs of many of those affected, or that the limited resources available within the NHS are not used in the most efficient manner.

There are many examples of excellent practice for those with rare diseases throughout the UK, but unfortunately these are more often the exception rather than the rule.

A coherent strategy can bring together all the existing knowledge and expertise in a logical way to ensure that all patients with rare diseases in the UK are treated according to their specific needs and receive equitable, high quality services and support regardless of their location in the UK.

An effective strategy for rare diseases can secure the best use of available resources, maximise the health gain for all those with rare conditions and create a framework for the research necessary to support the development of innovative therapies for unmet medical needs.

For too long, patients with rare diseases have had to face inequitable access to services, treatment and support. Now is the time for the health departments of the UK to demonstrate their commitment to bringing an end to this inequity by addressing the situation with a well-planned, strategic approach.

Briefing Papers

Please follow the below links to read why a strategy for rare diseases would be beneficial in each of the home nations of the UK. These papers also include examples of good practice in each nation which we believe should be used as models on which to base new services.

For too long, patients with rare diseases have had to face inequitable access to services, treatment and support. Now is the time for the health departments of the UK to demonstrate their commitment to bringing an end to this inequity by addressing the situation with a well-planned, strategic approach.

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What has RDUK done for the development of a UK Strategy for Rare Diseases?

Since the UK adopted the European Union’s Recommendation on an action in the field of rare diseases, RDUK has been campaigning for a strategy for integrated service delivery for rare diseases. This would coordinate:

  • Research
  • Prevention, diagnosis and screening
  • Patient care, information and support
  • Delivery of coordinated care
  • Commissioning and planning of services and treatment

We worked with our members and the broad stakeholder community to gather evidence to inform our report ‘Improving Lives, Optimising Resources: A Vision for the UK Rare Disease Strategy’, which was launched on Rare Disease Day 2011.

This report is believed to be the most comprehensive and wide-ranging review of services for patients with rare diseases in the UK and was contributed to by over 1,000 people or organisations. It demonstrates how patients’ and families’ lives can be improved, and NHS resources optimised, by taking some creative approaches and by building on the innovative ways of service planning and delivery that are already proving successful in the UK.

You can download the full report here.

If you would like a hard copy, please email

Overview of the report

This report covers five broad topics that a strategy should address and outlines the current situation of each and recommendations to improve this situation.

Click here to view the summary of the report.

Click here to download the report summary as a pdf.

Media coverage

A considerable amount of media coverage was generated from the launch of this report and Rare Disease Day. A summary of this can be seen here.

Next Steps

Following the launch of the UK Strategy for Rare Diseases in November 2013, with Earl Howe announcing its key themes at an event held by RDUK to mark its publication, RDUK have continued to work on the implementation of the Strategy in practice. You can find out more about the launch and the UK Strategy here. We are now working with the various bodies and organisations tasked with the implementation of specific recommendations to help them effectively fulfil their obligations. We will also be carrying out work to assess the success of implementation of the strategy and the effect it has had on patients.

A re-constituted UK Rare Diseases Stakeholder Forum, of which RDUK is a member, which was originally set up to help develop the UK Strategy will also continue to monitor its implementation across the UK.

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Are the Health Departments going to implement a UK Strategy for Rare Diseases?

Yes. In November 2013, in response to the launch of the UK Strategy for Rare Diseases each UK health department committed to publish their response to the UK Strategy for Rare Diseases in time for Rare Disease Day 2014.

RDUK was pleased to see responses from England and Wales and you can find out what is happening in your nation here.

We are now waiting for further information from Scotland, and we are still waiting for Northern Ireland to publish their initial response (we expect this to be in the very near future and details will be published on the gov.uk website).

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How can you be involved in the implementation of the UK Strategy for Rare Diseases?

You can join RDUK for free to show your support for a UK strategy for rare diseases. The more people there are backing our campaign, the greater the pressure is on the health departments of the UK to consider rare diseases as a health priority and develop an effective strategy to target the issues currently experienced by the rare disease community. You’ll then also be signed up to receive our e-updates, where we’ll keep you informed on other ways in which you can contribute your experience or evidence, provide your opinions and participate in evidence-gathering activities to help influence the implementation of a UK Strategy.

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