Health bosses have the chance to give hope to a group of people whose lives are being marred by a cruel illness – and they must take it.
Morquio Syndrome, first recognised in 1929, has caused the early deaths of thousands of people over the years who have been stricken by the health issues like skeletal deformities, heart disease and loss of sight or hearing that come with it.
A clinical trial of the drug Vimizim has offered some hope for those affected, the results of which have given the likes of 41-year-old Angela Paton, from Kippax, “a new lease of life”.
Unfortunately, as we have seen through the ongoing battle for Vimizim to be funded by the NHS, it is not that simple.
NHS England told the 34 people on the trial that it would wait for guidance from the National Institute of Health and Care Excellence (NICE) before deciding whether to fund it.
The campaign to give people with rare conditions like Morquio much-needed drugs on the NHS, and the call to action from the family of adorable Morquio tot Sam Brown, from Otley, has shown the drug is desperately needed. But it appears finance is at the heart of the health service’s reluctance to fund Vimizim.
Draft guidance from NICE in June suggested they may not approve it for use in the UK due to concerns over cost – at £395,000 per person per year – and fears its clinical benefits have been “overestimated”.
The fact remains, before Vimizim there was no treatment for the 88 people in the UK with Morquio. It has proven a lightbulb moment for those who have survived a life of pain and disability through the condition.
Money should be no object, these people should be given the treatment they deserve.
THE BATTLE FOR DRUG FUNDING
A successful trial of the drug Vimizim for 34 people with Morquio Syndrome gave families fresh hope.
The trial began in 2012 and has since ended but a battle to get the NHS to fund it has ensued. BioMarin, Vimizim’s manufacturer, will continue to offer the drug to families until NICE decides whether to approve it on October 28.
Morquio is a genetic condition that means those with the syndrome are missing a vital enzyme. The Vimizim drug aims to replace that missing ingredient.
NHS England is awaiting NICE’s decision on whether to approve the drug. The body’s committee will meet once more before issuing its final guidance later this year.