Wednesday 1st July 2015
CHILDREN AND YOUNG ADULTS FACE THE DEVASTATING CONSEQUENCES OF NHS ENGLAND FAILURES – DECISION EXPECTED TOMORROW
Those with the ultra-rare condition Morquio are left with no choice but to watch their health deteriorate as a result of NHS England bureaucracy and delay to a decision – again.
The Morquio community and the UK’s clinical expert centres played an instrumental part in the clinical trials for the development of the drug Vimizim, licensed by the EMA in April 2014. NHS Trusts benefited financially from carrying out the clinical trials and the treatment is now reimbursed in over 30 countries including Greece, Spain and Turkey.
Thirty-four British patients subjected themselves to years of hospital appointments and have spent hours each week hooked up to a drip to receive their Enzyme Replacement often in hospitals hundreds of miles from home. Family life and working life has fitted around the weekly routine of treatment in order to meet the high demands of the clinical trial only for the licensed treatment to be available outside our borders.
The condition Morquio has no cure and up until Vimizim was licensed no treatment. It is an ultra-rare condition affecting just 105 people in the UK, average life expectancy is just 25 years. The development of a treatment and subsequent licensing had given the Morquio community hope but now they feel like society has given up on them.
Christine Lavery Chief Executive of the Society for Mucopolysaccharide Diseases said: “Today’s announcement of yet another delay comes after months of false promises by the Prime Minster, Health Ministers and NHS England whose incompetence is playing with people’s lives. The emotional turmoil over the six months since a decision was originally expected in December and the lack of transparency and clarity around timescales of a decision is cruel. The health of individuals will start to worsen as a direct result of NHS bureaucracy and ministers failing to act.
“It is unforgiveable that NHS England have delayed the decision again on whether to reimburse Vimizim and the Morquio community should not be made to pay for the mistakes of NHS England.
“The impact has been life changing for young adults and children with Morquio and it is difficult to contemplate a future without Vimizim and a certain unnecessary premature death. There is no time to wait. We are hopeful that tomorrow will bring good news for the Morquio community”
The story so far
• UK lead on clinical trial – first patient in the world treated in Birmingham
• 30% of the clinical trial population treated in UK
• The drug Vimizim was licensed by European Medicines Agency (EMA) April 2014
• NHS England’s interim reimbursement decision expected December 2014
• NHS England’s decision making process was found to be discriminatory and the wait for a decision has continued
• Vimizim is now reimbursed in Czech Republic, Hungary, Austria, Denmark, France, Germany, Greece, Israel, Italy, Netherlands, Norway, Portugal, Spain, Sweden, Bahrain, Kuwait, Oman, Qatar, Saudi Arabia, United Arab Emirates, Russian Federation, Turkey, Indonesia, Japan, Brazil, Argentina, Chile, Colombia, Canada, United States
• Clinical trial patients treated in England who had been receiving treatment on compassionate grounds have now had free drug withdrawn following 14 months of extended access and NHS England’s lack of transparency surrounding a decision and timescales
For media enquiries or to arrange an interview, please contact Charlotte Roberts at firstname.lastname@example.org or 0345 389 9901
About The Society for Mucopolysaccharide Diseases
The Society for Mucopolysaccharide Diseases (The MPS Society) is the leading patient organisation in the world supporting over 1200 families in the UK and many more further afield whose children have an MPS or related disease. The MPS Society provides a pioneering advocacy service to individual families as well as fund innovative and life changing clinical and academic research. As a registered charity the Society is entirely supported by voluntary donations and fundraising.
About Mucopolysaccharidosis IVA (Morquio)
Morquio is a rare inherited degenerative disease which affects 105 children and young adults in the UK. Patients with the condition appear healthy at birth, but within 24-36 months start to show symptoms of severe problems.
The condition is a form of mucopolysaccharidosis, a metabolic disorder caused by the absence or malfunctioning of lysomal enzymes. The signs and symptoms of Morquio can affect many different parts of the body and frequently include heart, lung, visual, hearing and dental complications in addition to the skeletal abnormalities and difficulty breathing.
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 is a life changing enzyme replacement therapy (ERT) drug that targets the enzyme deficiency in people with Morquio. Following an extensive clinical trial, it was licensed by the European Medicines Agency (EMA) in April 2014. Vimizim has improved individual’s stamina and energy levels and enabled children to continue growing taller than the average untreated individual with the same condition. Children have required less surgical procedures and are less reliant on wheelchairs and mobility aids allowing for greater independence.
Individuals with Morquio rarely grow to more than three feet tall. The licensed treatment Vimizim has been found through extensive clinical trials to slow down disease progression and associated symptoms resulting in a much enhanced life expectancy, improved health and quality of life.
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.
Young adults have also seen huge improvements in their health and stamina and are describing that pain is more manageable and that their body feels lighter and no longer like they are moving through treacle. Prior to treatment many were concerned about their deterioration and having to give up work or PhD study Vimizim has removed that fear.
Vimizim is the only treatment available for those with Morquio with no alternative apart from managing symptoms to make the individual as comfortable as possible and providing palliative care.
Find out more at: www.mpssociety.org.uk or call us on 0345 389 9901