With NICE leading towards not regarding Vimizim on the NHS in England for Morquio, the disability I have which falls under Mucopolysaccharide, I have joined the UK-wide campaign to show why this treatments needs to be approved.
I have wrote an open letter to all MPs in this part of the country and well as all the party leaders in both the north and south of Ireland to apply whatever influence they have to get the treatment for everyone.
Dear
I am writing to you as someone who has Mucopolysaccharide Disease, Morquio Syndrome. In 2014 Vimizim was approved by the FDA and EMA as a enzyme replacement therapy for Morquio. On the 5th of June, NICE announced that they were leaning towards a negative recommendation, denying individuals with Morquio access to the life changing Vimzim. Their final decision is due in October.
Individuals with Morquio have been waiting over a year for a decision following the drug’s licensing in April 2014 by the European Medicines Agency, yet we are still months away from the final decision.
The licensed drug Vimzim, now reimbursed in over 20 countries, including France, Germany and Czech Republic, is the only treatment for the condition Morquio which addresses the underlying cause of the disease and there are no alternative treatments available.
Children and young adults who were on the clinical trial have continued to access treatment compassionately but this is due to end on the 25th June unless NHS England decide to approve the treatment on an interim basis until NICE make their final decision in October. If NHS England agree to fund the treatment all those in England who want treatment will have access. Currently children and young adults who were not on the clinical trial are deteriorating whilst they wait for an interim funding decision.
Children who started on Vimizim at a young age are now only using wheelchairs occasionally to enable them to cover more distance or preserve their energy when outdoors, or at school or don’t require them at all. Children’s stamina and energy have increased enabling them to keep up with their peers and participate in clubs and activities such as swimming, football, drama, and playing independently which had the disease followed its normal progression would not have been possible. Children on treatment have grown taller than the average person with Morquio. Their physical condition has much improved and they rarely complain about pain. The younger children who started treatment have seen their bones growing more “normally” and many have so far avoided a number of the surgical procedures associated with the disease.
I have not been on the clinical trial and over the last 5 years, I have noticed that my health has graduately deteriorated. Before my 30s, I was healthy young man, who was able to keep up with an active social life. However, over the years, this has slowly not been possible. On a flight from Lourdes, Frances to Belfast back in 2009, my respiratory system took a big hit, which I have not fully been able to recover from. From that point on, I have had to wear an oxygen machine at nights.
In 2012, my respiratory system took another hit and I was in hospital for a prolonged period of time and was in Intense Care for the day. Since that time, I now have to use a Nippy machine at night in bed.
My energy levels have not been the same since and have had to say “No” to lots of things that I have once been able to do, but cannot do anymore. I fear that over the years, this is only going to worsen and is why I am asking you to use your influence at Stormont and Westminster to apply pressure on NICE and the NHS England to give me and others with Morquio another chance at life and approve Vimizim. Vimizim has shown during extensive, robust clinical trials has shown reduction in disease progression, and associated symptoms resulting in a much enhanced life expectancy, improved health and quality of life.
Yours sincerely
Dermot Devlin
CC:
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