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Rare disease treatments risk being 'entirely out of patients' reach', says MPs report

9 September 2013
Appeared in BioNews 721

A parliamentary report has recommended a ring-fenced fund to ensure access to 'low volume, high cost' treatments for rare diseases.

Several experimental treatments for the illnesses, estimated to affect over 3.5 million people in the UK, have entered clinical trials over the last decade, with one gene therapy approved for use by the European regulator late last year (see BioNews 680). But the All-Party Parliamentary Group (APPG) for Muscular Dystrophy says that for patients to really reap the benefits of research, the Government, NHS and health regulators need to change.

'Successfully developing an effective treatment is far from the end' of a drug's development, highlights Dave Anderson MP, chair of the APPG. 'We must focus on ensuring that if treatments are proven to be safe and effective, the UK is in a position to license and deliver them swiftly - something which we most certainly are not now'.

Anderson warns that without swift action 'effective treatments may remain entirely out of patients' reach'.

The APPG is particularly concerned about the effect of recent changes in how rare disease treatments are assessed and funded. Previously a special committee distributed a dedicated £100 million fund but England's share of this was absorbed into one for all specialised services in April. Scotland's fund will remain in place until 2014.

The new arrangement in England will mean that high cost, but potentially high benefit, new treatments for rare diseases would compete against lower cost treatments for common conditions in a battle for funding.

Meanwhile, an expanded remit for the National Institute for Health and Care Excellence (NICE) means that it will now decide whether or not to recommend rare disease treatments. In a key recommendation, the APPG urges NICE 'to assess treatments for rare conditions differently from less rare conditions' to ensure that patients do not miss out on treatments because of the high cost.

Elsewhere, the APPG calls on regulators to streamline processes so that drugs can be assessed more rapidly. The report drew on the evidence of relatives of children with degenerative rare diseases like Duchenne muscular dystrophy to highlight the race against the clock many affected young people face.

The APPG report drew a response from the Department of Health. A spokeswoman said: 'NHS patients with rare diseases already receive some of the best levels of care in the world, but we are committed to improving this further'.

'That is why we are working closely with other health departments and stakeholders to develop a UK strategy for rare diseases. This will be published by the end of the year'.

Access to rare disease drugs - new campaign report launched
Muscular Dystrophy Campaign (press release) |  3 September 2013
Call for rare conditions drugs fund
BBC News |  3 September 2013
Create drugs fund for rare diseases, say MPs
WebMD |  3 September 2013
Rare disease patients 'at risk'
ITV |  3 September 2013
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