Little Sam pens a heartending note to Prime Minister David Cameron

19 November 2014.......         Sam Brown, 6,  with  parents Katy and Simon from Otley. Sam has Morquio syndrome. He is being treated with drugs thanks to a trial in Manchester which have significantly improved his condition but cash for this will run out next month and the NHS is unlikely to pick up the tab.TJ100584h Picture by Tony Johnson
19 November 2014....... Sam Brown, 6, with parents Katy and Simon from Otley. Sam has Morquio syndrome. He is being treated with drugs thanks to a trial in Manchester which have significantly improved his condition but cash for this will run out next month and the NHS is unlikely to pick up the tab.TJ100584h Picture by Tony Johnson

The family of a six-year-old boy suffering from a rare genetic illness has asked Prime Minister David Cameron to step in to prevent his life-changing drug treatment from being scrapped.

South Hiendley born Katy Brown and husband Simon, said they have been fed “half truths and half promises” after the NHS scrapped its own system for approving drugs for very rare conditions, including Morquio syndrome which affects only 88 people in the UK.

Their son Sam has been given the drug Vimizim - the first and only drug for the condition - on compassionate grounds by its manufacturer BioMarin since he took part in a successful trial for the treatment.

But because of delays by NHS England in deciding whether to fund the drug, the company said it will have to end the free treatment - which costs between £14,000 and £20,000 a month - in May.

And last week the family delivered hundreds of letters from friends and family to Downing Street putting pressure on Mr Cameron to ensure a process to consider drugs for rare conditions is set up immediately.

Mrs Brown, now of Otley, said: “We still haven’t heard anything from David Cameron - it’s incredibly frustrating especially when Sam originally sent him a letter three weeks ago.

“It’s vital we get this funding. Sam’s been receiving this drug on a clinical trial now for almost three years and it’s having an amazing impact.

“His condition is degenerative but whilst he has been on the treatment he has grown, his physical abilities have improved. It really is changing his life and his future looks brighter.

“We’ve had a substantial amount of time now where we’ve just been part of a process mess-up. NHS England should have had a decision on the funding of the drug in December but that was delayed because the process was proved to be discriminatory.

“Since then it’s a been a series of half-truths and half-promises and still now we have no clarity about how and if this drug is going to be funded.

“That was made even more serious when we found out the drug company, because of the lack of communication from NHS England, has no choice but to stop the compassionate provision of the drug.

“We’ve seen the great results of this drug and to have it taken away will be devastating. It is completely wrong that a six-year-old should suffer the consequences of the NHS’s inability to put in place a robust process for funding drugs.”

A spokesperson for NHS England said:“It is the role of NICE to provide guidance on whether a new drug or treatment should be made available on the NHS following a thorough review of the evidence. Patient groups have asked also NHS England to consult with patients and the wider public about how we make these decisions in future. This will ensure the principles and process we follow are well informed, evidence-led and in line with the expectations of patients and the public. The consultation runs until the end of April. Until then, any decision that is urgent on clinical grounds will be dealt with quickly though our existing procedures. Clinicians, on behalf of their patients, will continue to be able to make a request (an Individual Funding Request) to NHS England for treatment that is not routinely available.”