Devastation at HSE's refusal to fund life changing drug

A young Meath woman living with a very rare life limiting disease has been devastated by the HSE's refusal to fund a life-changing drug that could greatly improve her quality of life and her life expectancy.

June Elliott of Rathkenny suffers from the rare condition Morquio, which means she has repeated bouts of chest infections, chronic pain, lack of energy, has limited mobility and uses a wheelchair.She is also very short in stature.
When she was diagnosed at the age of four, her parents were told that she would probably not reach the age of ten, but she is now 32. Doctors still says that most patients up to now rarely make it out of their twenties.
Vimizim is a new drug that gained licensing approval from the European Medicines Agency (EMA) in 2014 and is showing great results in people using the drug.
Morquio is a rare inherited degenerative disease. Patients with the syndrome appear healthy at birth, but within 24-36 months start to show symptoms of severe health problems including heart disease, skeletal abnormalities, difficulty breathing and vision and hearing loss. There is no cure for Morquio, and, untreated, patients rarely live beyond their twenties.
“Patients in Northern Ireland are using it and there are great benefits. They have less chest infections and more energy and it is going to extend their life expectancy with so much less sickness,” June points out.
She said that Vimizim has been provided compassionately by manufacturers, BioMarin for the past three years to those who participated in the clinical trials. “BioMarin has agreed to continue providing Vimizim compassionately until the 5th December and after this date, people with the condition will no longer have access to this life changing drug.”
June was not involved in any trials herself but knows a few people who have and the huge difference it has made to their lives. “I would love to have the choice to go on this drug,” she says.
June is a daughter of Tom and Mary Elliott of Rathkenny and her diagnosis at four years old was a huge shock to her parents.
Depite the many problems she endures because of the condition, she attended Stackallen National School, the Mercy Convent in Navan and she currently works part time in ONM Buckets in Grangegeeth where she says her employers are great.
However, throughout her schooldays she missed a lot of time because of sickness and hospital appointments and her employers are very understanding of her condition.
Vimizim is manufacturered by BioMarin, an Irish company that provides a lot of jobs in Ireland.
According to the Irish Society for Mucopolysaccharide Diseases the HSE had “decided to refuse to reimburse Vimizim in Ireland due to lack of clinical data” in spite of the fact that it is currently funded by over 10 European countries including Northern Ireland.
According to the Society “Vimizim (Elosulfase alfa) brings multiple benefits: avoiding/delaying the requirement for invasive ventilation, improving cardiac function and muscle strength, improving respiratory function giving improved sleep which reduces sleep apnoea, lessens chest infections and breathing difficulties and increases energy and stamina. Vimizim also shows improved growth developing stronger posture which benefits mobility and organ function. Vision is preserved by preventing corneal clouding and pain levels become more manageable.”
June is angry that she cannot make the decision herself to use the drug, which by preventing repeated infection could greatly prolong her life.
She has seen a lot of difference in friends in Northern Ireland who are using the drug.
“They are now able to do things that people take for granted because they have more energy and stamina and pain levels are manageable.”
She is disappointed with the HSE's decision on behalf of all patients with Morquio in Ireland who have waited so long for a treatment to be developed and are now being denied access to it.
“The changes seen with using the drug, enable those with Morquio to complete normal day-to-day activities independently and offer the hope of very significant long-term benefits through extended use. One of these very significant benefits is to prolong life expectancy.”
“This drug is now being reimbursed in more than 10 European countries including Northern Ireland. Thus it is difficult to accept that some patients living on the island of Ireland have access to this vital drug while others will not,” she said.
“We are hopeful that the HSE will see sense, consider the clear positive benefits and make the right decision to ensure continuity of care for everyone.”
Vimizim is the first effective treatment for Morquio. It was developed by the pharmaceutical company BioMarin and was licenced on 28th April 2014 after extensive trials.It is an Enzyme Replacement Therapy (ERT) which overcomes the enzyme deficiency causing the disease. The HSE issued a Notice of Proposal not to support the reimbursement of Elosulfase alfa (Vimizim) to BioMarin in June 2017.
The Health (Pricing and Supply of Medical Goods) Act 2013 allows suppliers time to make representations on any proposed decision.
Those representations have been received and are being considered carefully. Until the time when the process has concluded and a formal decision has been communicated to BioMarin (the supplier) the application for reimbursement remains under consideration.