SAM Brown, from Otley in my constituency, is six-years-old and one of just 105 people across the UK with the ultra-rare disease Morquio Syndrome.
This is a condition that can severely inhibit one’s growth, cause skeletal deformities, and also lead to a larger head, knock knees, widely spaced teeth and many other symptoms.
Damage caused by the condition is irreversible and if left untreated life expectancy is reduced to just 25. In June, Sam’s drug supply was temporarily cut off, thanks to a failure of process from NHS England, the Department of Health and the National Institute of Health and Care Excellence (NICE).
For over nine months now, I have fought hard to try and get Sam long-term, secure access to the drugs he needs. This whole issue goes back to the passing of the Health and Social Care Act 2012, which saw the disbanding of the Advisory Group on National Specialised Services.
This was the committee advising ministers which treatments for ultra-rare diseases the NHS should fund. Importantly, it was a system that had been supported by charities and campaigners. The situation since then has been one of confusion with NHS England and NICE duplicating work and it not always being clear how their parallel processes work together.
Late in 2014, NHS England set out a scorecard system for deciding which drugs to fund, despite concern from clinicians. They were forced to suspend it after the threat of a legal challenge that it was discriminatory. At the same time, the producers of the drug, a company called BioMarin, had in October proposed to NHS England that it supply the drug for five years.
Had NHS England agreed to this offer, it would have represented a 42 per cent saving on the price of the drug. However, NHS England did not even bother to reply. And Vimizim was not the only drug affected by this hold up in bureaucracy.
Translarna, a drug needed by 50 people in England who suffer from a rare form of Duchenne muscular dystrophy, and Everolimus, needed by 80 people who suffer from tuberous sclerosis complex, were two further drugs stuck in the enigmatic “system”.
The fight for these three drugs turned into the wider, cross-party, multi-organisational #FundOurDrugsNow campaign.
Over the weeks and months that followed, I raised the matter repeatedly in the House of Commons through questioning the Prime Minister, the Secretary of State for Health and the Life Sciences minister.
I tabled numerous parliamentary motions, led two debates, and we also had a number of rallies outside Parliament, the Department of Health and Downing Street.
Sam Brown and his parents came down from Otley to hand in letters at 10 Downing Street. Again we were fobbed off by ministers repeating the same line again and again: “These are matters for NHS England and ministers cannot intervene”.
This is simply not true. The Department of Health’s Framework Agreement with NHS England says: “If the Secretary of State considers that NHS England is significantly failing in its duties and functions he is able to intervene and issue directions to NHS England.”
Even when these are “matters for NHS England”, why is it that NHS England officials failed to turn up to two meetings I had with the Life Sciences minister? After the minister specifically requested a letter from us setting out our concerns, why did NHS England then take 11 weeks to reply? And, as mentioned earlier, why did NHS England not bother replying to the offer from BioMarin to supply the drug at a considerable saving to the taxpayer?
We were repeatedly told that NHS England was consulting on a draft policy for funding Vimizim, but the decision on July 2 not to fund it was based on interim guidance from NICE, so why were we repeatedly being pointed towards this NHS consultation?
Even the July announcement should have been made on June 25. It was unfair on the families to be told to expect a decision, be given this false hope, but then be told about repeated delays instead. And given that the UK led the way in the clinical trial for Vimizim, why is the drug available in more than 20 other European countries but not here? Why is it that countries including Brazil and Turkey, also with publicly-funded health services, have enabled access to Vimizim while the UK hasn’t?
The announcement today by the drugs company that it will resume the free supply of Vimizim is welcome news. But NICE must now make a positive decision to approve the drug when their final guidance on Vimizim is published in October. Only this will end the turmoil that many families have been through.
In the longer term, we need a better, faster process for approval of drugs for ultra-rare diseases when they are shown to be effective. Ministers must start taking responsibility and put one in place.
Greg Mulholland is the Liberal Democrat MP for Leeds North West.