Background to a Strategy

This page provides some information on the need to develop a 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 developed.

Please click the questions below to skip to each section:

• Why is a UK strategy for rare diseases needed?
• What is RDUK doing for the development of a UK rare disease strategy?
• Are the Health Departments going to develop a UK strategy for rare diseases?
• How can you be involved in the development of a UK rare disease strategy?

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 is needed to 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 would work to 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.

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What is RDUK doing for the development of a UK rare disease strategy?

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

For 18 months 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’. 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.

Following the launch of this report, RDUK will continue to work with health departments across the UK and decision makers across the UK to ensure that the recommendations set out in the report are taken on board in the development of the government’s strategy.

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Are the Health Departments going to develop a UK strategy for rare diseases?

Yes. The first confirmation came in July 2010 when Anne Milton MP, the Parliamentary Undersecretary for State, in response to a written question replied that –

“The Department is working with the Devolved Administrations and key interest groups to develop a plan for rare disease by 2013 in line with the Council’s recommendation.”

In November 2010, at the Europlan conference arranged by RDUK, a more detailed timeline of activity was confirmed by Dr Ed Jessop from the National Specialised Commissioning Team who represented the Department of Health. He informed us that the strategy will be developed over the summer of 2011 by the Department of Health in collaboration with the devolved nations, resulting in a 12 week public consultation in the autumn of 2011.

Dr Jessop, and representatives from the health departments of the devolved nations, have also expressed the desire not to duplicate the extensive work done by RDUK in producing our recommendations for the strategy and have shown a willingness to consider the recommendations set out in our report ‘Improving Lives, Optimising Resources’ in the development of their strategy. This willingness to collaborate was further elaborated at the RDUK Rare Disease Day 2011 receptions, where representatives of the Scottish, English and Northern Irish health departments gratefully received copies of RDUK’s report.

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How can you be involved in the development of a UK rare disease strategy?

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 development of a strategy.

When the health departments launch their own consultation, RDUK will be working with our members to inform and assist them in responding to the consultation to ensure that the strategy accurately and thoroughly reflects the needs of the rare disease community.

You can also contact your local politician to raise their awareness of rare diseases, and to encourage them to pledge their support to a rare disease strategy. If you would like help on how to do this, please contact .

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