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First strategy to focus on improving treatment for rare diseases

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A UK-wide strategy has been launched to help build understanding of rare diseases and boost research to find effective treatments.

The first UK Rare Diseases Strategy is intended to prevent people affected by rare diseases being “left behind” those with more common conditions, according to the government.

Key elements of the strategy include that every patient should have a clear personal care plan, which brings together health and care services.

In addition, there should be support for specialised clinical centres to “offer the best care”, and better education and training for healthcare professionals to help “ensure earlier diagnosis and access to treatment”.

The strategy also backs promoting the UK as a world leader in research and development to improve the understanding and treatment of rare diseases.

Launching the document at Great Ormond Street Hospital, health minister Lord Howe said: “Millions of people in the UK are affected by the thousands of different kinds of chronically debilitating illnesses that are defined as rare diseases.

“So when looked at as a whole, rare diseases are not rare and our focus should be on making sure that no one who suffers from one is left behind,” he said.

“For the first time, we are strengthening the links between research and the treatment and care of patients with rare diseases,” he added. “This is about putting those patients first, with better diagnosis, treatment and support for them and their families.”

A rare disease is defined as a life-threatening or chronically debilitating disease that affects five people or fewer in 10,000, and requires special, combined efforts to enable patients to be treated effectively.

There are already more than 5,000 rare diseases identified and it is estimated that one in 17 people will suffer from a rare disease in the course of their lifetime.

Along with the launch of the strategy, the government announced that the National Institute for Health Research was establishing a Rare Diseases Translational Research Collaboration with £20m of funding over four years.

Alastair Kent, chair of Rare Disease UK, was part of the stakeholder forum that helped shaped the strategy.

He said: “While the NHS has demonstrated that it is capable of delivering a world class service, the experience of patients has been that this is by no means a universal experience.

“Now, at least, patients can have a clear expectation of what the NHS aspires to provide for them, wherever they live in the UK. This is a huge step forward, and the publication of the Strategy will give hope and comfort to the 3.5 million people affected by rare diseases across the UK.”

The Specialised Healthcare Alliance said it welcomed the strategy as a means of “delivering a joined-up approach to services for people with rare conditions across the UK”.

Alliance director John Murray said the strategy “sends out a strong message” about the need to work together to improve care for people with rare conditions, whose interests can often be overlooked in the context of a locally-focused NHS.”

“The main challenge will be to ensure that all four nations develop tailored implementation plans to a clear timetable.”

“The commitment to ensure that all patients with rarer conditions receive an appropriate care plan is particularly welcome,” Mr Murray added.

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