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First U.K. rare diseases strategy launched

Friday, November 22, 2013

The U.K.’s first strategy to help build understanding of rare diseases and boost research to find effective treatments and therapies has been launched.

The U.K. Rare Diseases Strategy aims to ensure none of the three million people in the U.K. who are affected by rare diseases are left behind.

Key elements of the strategy include a clear personal care plan for every patient that brings together health and care services, with more support for them and their families; support for specialized clinical centers to offer the best care and support; better education and training for health and social care professionals to help ensure earlier diagnosis and access to treatment; and promoting the U.K. as a world leader in R&D to improve the understanding and treatment of rare diseases.

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

“There are already more than 5,000 rare diseases identified, and the total is steadily rising as new genetic discoveries explain previously unexplained disease patterns,” he said. “It is estimated that one in 17 people will suffer from a rare disease in the course of their lifetime. In the U.K., this means more than three million people will have a rare disease.

“Therefore, when looked at as a whole, rare diseases are not rare. They represent a significant cause of illness, making considerable demands on the resources and capacity of the NHS and other care services,” said Howe.

In parallel with the launch of the U.K. Rare Diseases Strategy, the government announced the National Institute for Health Research (NIHR) is establishing a Rare Diseases Translational Research Collaboration (TRC). With investment of $32.4 million over four years, the NIHR Rare Diseases TRC will help to increase research collaboration that will lead to improved diagnosis, treatment and care for people with rare diseases.

Professor David Goldblatt, director of Clinical Research and Development at Great Ormond Street Hospital and director of the NIHR Biomedical Research Center, said, “Many of the children attending Great Ormond Street Hospital suffer from rare diseases. Their families’ experience of delays in the recognition of their syndrome or the absence of a clear diagnosis can be profoundly affecting.”

“Rare diseases are under-researched at present and in response, the NIHR-funded Biomedical Research Center at GOSH/UCL is now focusing almost exclusively on rare disease experimental research,” he said. “While Great Ormond Street Hospital has made some great strides, for example in pioneering gene therapy, much more work needs to be done.”

Goldblatt said, “Our ambition is to invest in rare disease research and advance understanding and treatments for the benefit of patients across the world. The hospital’s charity is building a new Center for Children’s Rare Disease Research for the hospital and UCL Institute of Child Health, which will play a key role in developing therapies for many disorders for which no treatment or cure currently exists.”

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