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Drugs for Ultra-rare Diseases

I have been leading the national campaign #FundOurDrugsNOW to secure funding for drugs for those with ultra-rare diseases.

Keep Sam Smiling campaign

In December 2014, NHS England was forced to suspend its 'scorecard' system for deciding which drugs to prioritise for funding. The process was guilty of discrimination against those with ultra-rare diseases. NHS England failed to put an interim commissioning process in place while drawing up new long-term rules. This has left 218 people who are suffering from Morquio Syndrome, Tuberous Sclerosis Complex, and Duchenne Muscular Dystrophy without secure access to treatment, including Otley's own six-year old, Sam Brown.

Throughout 2015 Sam Brown has had to rely on the goodwill of drug manufacturers BioMarin to receive the drug Vimizim, which he needs to prevent the irreversible deterioration of his condition. It is grossly unfair to leave people without access to treatment simply because of the rarity of their disease, yet NHS England have repeatedly delayed decision-making on this issue and have twice failed to meet with me despite agreeing to attend meetings.

I have secured two debates on the issue of ultra-rare disease drugs in Parliament, first in January 2015 and later in June, where I received cross-party support for my demands. I have also gone further, and directly demanded that Health Ministers intervene to force NHS England to clean up its mess. In a letter to Mr Freeman at the Department of Health, I outlined the inefficient and ineffective action taken by NHS England. Mr Freeman passed the letter onto the NHS, but has received no response. It is not acceptable to hide behind layers of bureaucracy where the lives of disease sufferers such as Sam Brown are concerned.

Greg with Sam Brown and his family, outside 10 Downing StreetSix months after the NHS suspended its commissioning process, representatives finally revealed that a Clinical Priorities Advisory Group would meet on June 25th to tackle the arrangement for interim funding for ultra-rare disease treatment drugs. However, NHS England has again refused to take responsibility for the health of those with ultra-rare conditions by granting funding. Instead, they have passed the buck and will wait for the National Institute for Health and Care Excellence to publish a report on the issue.

After a long-fought campaign, the Vimizim drug to treat those with the ultra-rare disease Morquio Syndrome received final sign-off from health authorities on 16 December 2015.

Announcing the decision, the National Institute of Health and Care Excellence (NICE) published final guidance saying that Vimizim is now officially approved for use in the UK and can be funded by NHS England. This is a fantastic victory for the campaign and well received news for all those suffering from this terrible rare disease. I am happy that my friend Sam Brown can now readily receive the necessary drugs for his Morquio Syndrome.

However the fight does not stop here, other drugs such as Everolimus and Translarna must also now be signed off. That's why I will continue to campaign on behalf of all those who are affected by these incredibly rare diseases and #FundOurDrugsNOW campaign will continue to fight on to secure the much needed funding for these conditions.

UPDATE - 18 APRIL 2016

Translarna is to be made available for the treatment of Duchenne Muscular Dystrophy after a National Institute for Health and Care Excellence (NICE) recommendation

UPDATE - 28 JUNE 2016

I am delighted that Everolimus has now been approved and is available through the NHS to treat those suffering with the ultra-rare disease tuberous sclerosis complex (TSC) and as a result have kidney tumours, known as angiomyolipomas (AML).