National consortium of charities call for step changes in commissioning and planning processes for rare diseases to ensure equitable access to health services and treatments regardless of a patient’s location
Action Duchenne, the only national charity to campaign to find a cure and viable treatments for Duchenne Muscular Dystrophy, welcomes the publication of Rare Disease UK’s Strategy document. Entitled ‘Improving Lives, Optimising Resources: Vision for the UK Rare Disease Strategy’, the document is calling for a national approach to treating rare diseases to ensure better care, avoiding the current piecemeal approach which results in a postcode lottery.
Action Duchenne and the Duchenne Muscular Dystrophy community have long been campaigning for national commissioning and a national approach to providing services for those living with Duchenne. International consensus guidelines have been produced detailing the care for Duchenne by a group of 84 neuromuscular disease experts, published in Lancet Neurology, which was highlighted by the report as an example of good practice. The subsequent family guidelines explain the likely progression of Duchenne, and the care that they should expect. While these standards of care have been published, they are yet to be adopted by the NHS and under current arrangements are only capable of being provided at the two UK Centres of Excellence for Duchenne in London and Newcastle.
Action Duchenne together with Rare Disease UK are calling for:
• National Commissioning of Care services to move away for the current postcode lottery for care.
• Consistent implementation of the internationally agreed Standards of Care for Duchenne at Centres of Excellence, reasonably accessible to all
• Access to research and clinical trials, and a plan to deal with regulatory hurdles associated with treatments for rare diseases (low numbers of patients means it is impossible to get the high volumes of data required for licensing of tradition medicines)
• National planning by the Government/NHS for the provision of new genetic treatments as they become available.
Nick Catlin, CEO of Action Duchenne said, “Action Duchenne is committed to supporting Rare Disease UK in the launch of its national strategy. For too long now rare diseases have not received the attention they are due, simply because the NHS doesn’t really know how to cope with them. Many families in the UK affected by rare conditions, Duchenne muscular dystrophy included, are experiencing inadequate care because there is no expertise in the disease local to them.”
“The UK must find a way to provide better, more appropriate care to patients that is not reliant on local funding, which may not be available. This is particularly important as we start to see new genetic medicines being developed. However, the proposed GP Commissioning arrangements raise real concerns that general practitioners will not have the detailed understanding of complex but rare conditions like Duchenne to deliver the required care. Most local GPs have no experience of Duchenne and to prevent the continued postcode lottery, national commissioning for Duchenne and other rare conditions must remain a priority.
Action Duchenne is the only UK charity focused on finding treatments and a cure for Duchenne Muscular Dystrophy, the most common lethal genetic disorder that affects 1 in 3,500 male births. There are between 1500 and 2500 boys and young adults living with the condition in the UK, which is a severe and degenerative muscle wasting disease. It is caused by a defect in the gene which produces dystrophin, essential for muscle function. The disease progresses with weakening of muscles, ultimately leading to complete paralysis. The lack of dystrophin in the respiratory and cardiac muscles leads to a life expectancy in the late teens or early twenties. There is currently no cure or treatment to halt or reverse the onset of Duchenne which is progressive and life-limiting.
Action Duchenne is an active member of Rare Disease UK.
Notes to Editors:
About Action Duchenne
Established in 2001 Action Duchenne aims to support and promote innovative research into a cure and effective medicines for Duchenne/Becker Muscular Dystrophy. The charity, which is led by Duchenne families, aims to promote awareness of the condition, to improve care services, and provide access to a range of educational and support/development programmes for people living with Duchenne at every stage of the condition. This is achieved by working in partnership with government agencies, NHS and care organisations, other charities, academic, scientific and research groups, and biotech companies worldwide.
Action Duchenne is actively campaigning for best practice in care for Duchenne based on the publication of an international consensus document in Lancet Neurology in Jan/Feb 2010. The published article¹ was the product of an extensive review by 84 international experts in Duchenne Muscular Dystrophy diagnosis and care, and was supported by the Centers for Disease Control (CDC) in the US. It provides an expert guide to recommendations on the multidisciplinary care that should be available to all individuals with Duchenne. Several Duchenne patient organisations including Action Duchenne and the TREAT-NMD network have helped to produce a ‘Guide for Families’, based on the original academic article, which is written in language accessible to all without a medical background.
Since 2003 Action Duchenne has provided £4m for research projects and partnerships. The charity has worked with the MDEX consortium, Department of Health, and the Medical Research Council to deliver new clinical trials for Duchenne drugs. In addition, it has been instrumental in developing projects with biotech companies both in the UK and US including key projects with AVI Biopharma and Summit.
In 2005 Action Duchenne launched the DMD Patient Registry, a national database that holds gene information of people living with Duchenne that is used to speed up the recruitment of patients for clinical trials. For more information please visit: www.dmdregistry.org
In 2008 Action Duchenne launched the Include Duchenne project in partnership with Decipha. This programme addresses the learning and behaviour needs of those living with Duchenne. For more information please visit: www.decipha.org
Action Duchenne promotes social inclusion within the community for young men living with Duchenne through its Genius programme. For more information please visit: www.actionduchenne.org/geniusproject
In 2010 the National Advocacy Council was launched to spearhead the lobby for government funding for research into Duchenne and to improve access to Standards of Care. www.actionduchenne.org/NAC
For more information please visit: www.actionduchenne.org
CEO, Action Duchenne
Tel: 0208 556 9955
PR Artistry Limited
Tel: 01491 639500
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