Leeds rare condition patients await drug decision

Sam Brown, six, who has Morquio syndrome. Pictured with parents Katy and Simon from Otley.
Sam Brown, six, who has Morquio syndrome. Pictured with parents Katy and Simon from Otley.
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Patients of the ultra-rare Morquio Syndrome are expecting to receive a long-awaited decision today on whether the NHS will approve a life-changing drug for funding.

Bosses at NHS England are expected to deliver their verdict ‘imminently’ on whether to fund Vimizim until health regulator NICE publishes its final decision on the drug in October.

Angela Paton from Kippax, who has Morquio Syndrome.

Angela Paton from Kippax, who has Morquio Syndrome.

Many patients have now had their last infusion of Vimizim, which had been provided temporarily free of charge until last week, by manufacturer BioMarin to those who took part in the clinical trial.

These include Sam Brown, six, of Otley, whose desperate parents have now resorted to monitoring him for signs of deterioration. Mum Katy said: “It’s just dreadful. To sit there and measure things in the hope he will go backwards to prove the drug works is a dreadful thing for parents.”

Morquio Syndrome, which currently affects 88 people in England, is a degenerative condition which limits growth and mobility and shortens life expectancy to an average of 25.

Clincal trials have found Vimizim - which is used in over 30 countries, including France, Germany and Hungary - can slow down disease progression and symptoms, with patients reporting a better quality of life.

Morquio patients and their families fear the worst for NHS England’s decision after NICE published its interim guidance on Vimizim in June which indicated it may not be recommending it for approval in October.

The regulator questioned the drug’s value for money and clinical benefits. While acknowledging it provided “valuable” benefits “for certain aspects of the condition, including quality of life” , it felt BioMarin had “overestimated” these benefits and the drug “would not entirely stop disease progression”.

Angela Paton, of Kippax, who at the age of 41 is one of the UK’s oldest patients, said NHS England and NICE are ‘playing God’ with her and other patients’ lives.

She said she believes NICE is placing undue emphasis on cost.

In her feedback to NICE during its consultation she wrote: “If you do not deem this treatment to be value for money then I would like to ask what would be considered value for money. What is it worth to you to have enough energy and stamina each day to go out to work, earn a living and enjoy of the things that come with financial independence. What is it worth to you to be able to walk, to be able to breathe, to have enough breath in your lungs that you are able to string a sentence together. What is it worth to you to see your children grow up and live beyond the age of 25. How terrifying would it be if you knew that in all likelihood your fate would be an early death, most likely gasping for breath as your respiratory system failed you, and what would it be worth to you to avoid this fate. I am sure anyone would answer that these things are priceless and yet for us you do not think they are ‘value for money’.”

She added: “I’ve never felt so completely and utterly helpless. We are just a small group of 88 vulnerable children and adults who in the grand scheme of things have no voice and no power. I know that this treatment works, I know that it has vastly improved my quality of life and have been given a glimpse of the brighter and longer future that it will afford me but now I have no idea if that future will be taken away from me.”

An NHS England spokesman last night said the timing of their decision had yet to be confirmed but said it was ‘imminent’.

He added: “The independent medicines assessors NICE have raised real doubts about Vimizim, however there is an annual process where the NHS carefully identifies the best new treatments and that’s now happening.”