Leeds rare condition sufferer’s setback in fight for drug

Angela Paton from Kippax, near Leeds, who has Morquio Syndrome and faces losing the treatment which is allowing her to have a reasonable quality of life.
Angela Paton from Kippax, near Leeds, who has Morquio Syndrome and faces losing the treatment which is allowing her to have a reasonable quality of life.
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One of the UK’s oldest sufferers of the rare Morquio Syndrome has spoken of her devastation after health regulator NICE indicated it may not recommend a drug which she says has been life-changing.

Angela Paton, 41, of Kippax, said the news had been a massive blow in the long-running battle for long-term access to Vimizim, which she has been receiving on a temporary basis through the goodwill of drug company BioMarin, after taking part in the clinical trials.

NICE’s final decisionn will be given in October but this week it published preliminary findings, which questioned the drug’s value for money and clinical benefits.

While acknowledging it provides “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”.

Morquio Syndrome is an inherited condition which limits growth and mobility and shortens life expectancy to an average age of 25. There are currently 88 people living in England with the condition, including Angela who credits Vimizim with transforming her life and allowing her to keep working.

She spoke at the NICE committee meeting as a ‘patient expert’ and said yesterday she is stunned at their resulting announcement.

“I find it hard to believe that anyone with an ounce of humanity, who listened to the same evidence as I did and heard our accounts of the difference this treatment had made to our lives could come to the conclusion that Vimizim is not ‘value for money’.

“This is a drug that is allowing me to continue to work, pay tax and play an active role in society. It is also a drug without which some adults with Morquio would no longer be alive and yet to NICE it is not worth the cost. To me this feels like they are basically saying we are not worth the investment, that our lives are worthless.”

Angela fears this latest news may influence the decision of NHS England who are due to announce on June 25 whether they will provide interim funding for Vimizim until October.

She added: “In all the months we’ve been fighting this battle, I don’t think the situation has ever been as critical as it is now, I have certainly never been as worried and desperate as I am now. I now have a real fear that we may lose access to Vimizim, not just temporarily due to bureaucratic failings but that it may be taken away from us permanently because the people who make these decisions have considered it and decided that our lives are not worth the cost. This is devastating.”

A spokeswoman for BioMarin said they will be responding to NICE and remain confident they can address all concerns raised.

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