Coalition launches rare disease strategy
pharmafile | November 22, 2013 | News story | Research and Development, Sales and Marketing | DoH, NHS, R&D, rare disease
The Department of Health has launched the UK’s first strategy on rare diseases as part of a bid to boost R&D and to provide better patient care.
Around three million people in the UK are thought to suffer from such conditions and the UK Strategy for Rare Diseases is being painted by government as a means for redressing the balance in a neglected area of health.
Launching it at Great Ormond Street Hospital, 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.”
It is an impressive statement of intent – but the establishment of the strategy appears to fly in the face of recent events: it comes just a month after NHS England pulled the plug on a £50 million fund to advance innovation in the investigation and treatment of rare conditions.
The Specialised Services Commissioning Innovation Fund (SSCIF) was developed to accelerate the adoption of innovative new approaches to specialised services.
Billed as an initiative that would “transform the way that new innovations are identified, tested and adopted”, it has been put on hold indefinitely after more than 600 applicants made submissions for funding.
However, Lord Howe insisted: “The UK already leads the way with ground-breaking research to better understand and treat these illnesses and this strategy will help cement our reputation as the driving force in this field.”
Others offered qualified support for the scheme. “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,” said Alastair Kent, chair of Rare Disease UK.
“With the development of the UK Strategy for Rare Diseases patients and families can have a clear expectation of what to expect from the NHS, wherever they live in the UK,” he added.
But while admitting it is a ‘huge step forward’, he warned: “Turning the UK Strategy’s aims into practical benefits for patients will require hard work and detailed planning.”
Professor David Goldblatt, director of clinical R&D at Great Ormond Street and director of the NIHR Biomedical Research Centre, acknowledged that rare diseases are under-researched at present.
“The NIHR-funded Biomedical Research Centre at GOSH/UCL is now focussing almost exclusively on rare disease experimental research,” he said. “[But] much more work needs to be done.”
The government suggests genomic research will be a major part of the picture and observers will recognise many of the strategy’s elements – for example, the pledge to provide a clear personal care plan for every patient that brings together health and care services reflects wider current practice when it comes to health.
Other measures include specialised clinical centres to offer 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 UK as a world leader in R&D.
MPs from the Parliamentary Group for Muscular Dystrophy in the UK have already called for a ring-fenced fund to pay for drugs for rare and ultra-rare conditions.
Adam Hill
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