Leeds Sufferers Of Rare Condition Get Positive News
Morquio sufferers have finally got light at the end of tunnel on whether NICE will fund Vimizin.
Yorkshire Morquio sufferers have campaigned tirelessly to get Vimizin through the NHS. Up to now, only a small group of people had access to it through a clinical trial. One of those is Angela Patton from Leeds. She told Radio Aire today, she's exhausted from campaigning and it's a relief to have light at the end of the tunnel.
‘Although I am getting the treatment I am still exhausted from the battling and now Ill be able to get on with my life and enjoy it feeling well.’
‘It’s extending the process a bit longer, which means there’s a few more months before the guidance comes out. That’s not good for those who are not receiving treatment who were not part of the clinical trial. But overall it’s really good news.’
‘If we hadn’t fought the way we have I don’t think we would be in this position now. I think they would have failed it on cost, without giving us a fair hearing.’
NICE HST the drug decision making body have announced today, that they are leaning towards a positive recommendation, of the long awaited treatment elosulfase alfa, licensed Vimizim for individuals with MPS IVA, Morquio disease.
Today’s announcement is a huge relief to the English Morquio community that NICE HST are leaning to a positive decision. However, the decision comes with a request for a Managed Access Agreement that requires stringent start and stop criteria and a high level of compliance by patients and their clinical team.
Individuals with Morquio have been waiting since April 2014 when the drug was licensed by the European Medicines Agency, yet we appear to be months away from a final decision.
The licensed drug Vimzim, now reimbursed in over 30 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.
Untreated individuals with Morquio deteriorate steadily resulting in diminished independence, decreased endurance, impaired quality of life and early death and can only be managed palliatively. Vimizim however, 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.
NHS England were due to make a decision on interim funding of treatment in June 2015. However, seven days later on 2nd July 2015 NHS England decided not to make a decision and wait until NICE’s final decision at that time promised for the 28th October. Today’s announcement requiring further consultation will extend the delay for a final decision.
Currently children and young adults who were not on the clinical trial are deteriorating whilst they wait for NICE’s final decision as NHS England failed to make an interim decision on funding.
Christine Lavery, Chief Executive of the Society for Mucopolysaccharide Diseases said: “We are relieved by the announcement from NICE HST today and are working hard to complete the requested Managed Access Agreement. However, we call on David Cameron to now show compassion to the children diagnosed too late to participate in the clinical trial who are deteriorating whilst they wait for the final positive decision.
“Individuals with Morquio and their families have been emotionally drained by the catalogue of errors that has led to the dragging out of the decision making process by NHS England and the changing of goals. The Morquio community have had 16 months of waiting to receive Vimizim and the fact that we are still months from the final decision is an appalling way to treat those in our society whose health is deteriorating before their eyes.”