The family of a six-year-old boy suffering from a rare genetic illness has urged Prime Minister David Cameron to step in to prevent his life-changing drug treatment being scrapped.
Katy and Simon Brown, of Otley, 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 £14,000 and £20,000 a month - in May.
The family yesterday 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 said: “It’s been an incredibly stressful and frustrating time. Sam’s been receiving this drug on a clinical trial now for almost three years and it’s having an amazing impact, it’s really changing his life.
“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.”
The family’s Leeds North West MP Greg Mulholland urged Ministers to intervene.
“NHS England really have behaved in a wholly unacceptable manner,” he said. “They haven’t responded to a letter by BioMarin with a proposal for going forward and that’s just simply not acceptable, considering people like Sam are relying on this drug and waiting on the NHS.”