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Date:
02-01-14

Author:
Empower

Getting ahead for those with rare diseases – The UK Strategy for Rare Diseases

Getting ahead for those with rare diseases – The UK Strategy for Rare Diseases

After a great deal of hard work by many the UK has now released it's Strategy for Rare Diseases.  The Strategy was needed to fulfill European Commission requirements but it's by no means a box ticking exercise: it provides a walk through some of the issues that have been affecting patients and their carers for years and it sets out a starting point for a great deal more work to deliver on it’s 51 commitments from now until 2020.

The Strategy covers the whole piece: from education and training for health professionals so that they can spot rare diseases, working collaboratively in research, empowering patients all the way through to how decisions are made about access to treatments. Speed is highlighted too: from speeding up diagnosis to faster health and social care support and faster sign up to trials. 

The research side is of particular interest as this is a where there is one of the biggest challenges for rare diseases: getting enough knowledge in one place to focus efforts and be confident in the results of trials. The Strategy highlights the importance of research and champions the involvement of patients and families in all types of research. It also recognises that there is a need to streamline processes such as research approval. It sets out the potential for much greater use of online tools: to find out about research, to take part and to share with others.  Some key commitments include:

• support patients to register on databases, where these exist;
• help patients to contribute to research and other activity related to rare diseases;
• examine how they can encourage service providers to involve patients in research and to ensure appropriate funding for excess treatment costs for research in rare diseases
• use portals to connect patients and relatives to enhance research participation and, where appropriate, promote self-enrolment to approved research studies with online consenting, self-reporting and use of social media
• explore the feasibility of the UK Clinical Trials Gateway including experimental medicine trials for rare diseases to provide information for patients and their families about research trials

Now each nation in the UK needs to draw up their implementation plans to deliver on the 51 commitments.  And not all of these will be easy to meet; some will probably only be in the eye of the beholder (for example, ‘appropriate’ procedures for evaluating the costs and benefits of treatments for patients as what is appropriate could be different depending on whether you’re the producer of the treatment, the NHS or the patient).

The opportunity now is instead of focusing on not leaving those with a rare disease behind, to get ahead for those with rare diseases.

Leela Barham is an independent health economist

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