A tale of two treatments - Girls an hour apart are subject to a major difference in healthcare
Morquio is a lifelong condition which has left Grace Colgan and Aoife McGee as tall as a 2-year-old
Two years after she began fighting for her daughter (9) to get the only drug in the World that can help her, Grainne Cogan has vowed to keep going.
"I am not going away or giving up, this is our daughters' life," the mum who is based with her family in County Monaghan says.
An hour away in County Tyrone Aoife McGee, who is also nine, is receiving the drug and there has been a marked difference in her health.
Grace needs Vimizim, an expensive enzyme replacement drug, to treat Morquio, a lifelong condition which has left her as tall as a 2-3 year old and living with pain and fatigue that are getting worse.
Vimizim costs approximately €400,000 a year for an adult and the manufacturers, BioMarin made an application for it to be funded by the HSE.
As part of that process Vimizim was assessed by the National Centre for Pharmacoeconomics (NCPE) who decided in December last year not to recommend it for reimbursement.
Following the recommendation from the NCPE, the HSE examines all the evidence which may be relevant for the decision; the final decision on reimbursement is made by the HSE.
Grainne is continuing her campaign and is utterly frustrated that if she lived across the border, a short drive from their home in county Monaghan, her daughter’s life would be so different.
"We do not have any choices when it comes to treatments. This is the only drug in the World to treat Morquio. We do not have any alternative for Grace.
"It has been tested and cleared for use and it is used in lots of other countries and yet my daughter is suffering because it has not been approved for funding in Ireland."
The family, from Carrickmacross, thought their prayers were answered last year when the HSE approved their application for Grace to receive the drug under the Treatment Abroad Scheme.
However it was an error because the drug is not approved for use in the Republic and as such the approval cannot be put into action.
Aoife McGee lives one hours drive from Grace in Cookstown, county Tyrone. She is also 9 but unlike Grace she began on Vimizim last June and her mum Seanín said, "there has been a marked difference in Aoife's stamina and overall health."
"Most significantly she used to be very prone to chest infections which resulted in hospital stays and very strong antibiotics. This hasn't happened at all since commencing the therapy."
Aoife has more energy and instead of being "completely exhausted halfway through the school day," she can keep going until teatime without feeling too tired.
Seanín said the primary benefit will be Vimizim contributing to Aoife continuing to growing which will help her curved spine to straighten.
"I find it utterly reprehensible that two children, separated by such a short distance, can be subjected to such a major difference in vital health care. There just aren't adequate words to describe how it feels."
"What possible justification can there be for one little girl to be denied the same level of access to a drug that has been proven so effective across the EU, in the UK and right here in Northern Ireland, a handful of miles over a border? There is no justification. None whatsoever."
Dermot Devlin,who also lives in Northern Ireland, began receiving Vimizim less than three months ago and said, "it works."
"My physical well-being has improved greatly over the last 11 weeks and will continue to do so. I've spoken and made others who were on the Vimizim trial for 5 years and you would not recognise the differences in them as their health has so greatly improved."
"It makes a great differences to quality of life too. I am now able to take part in things that I would have found difficult a few short months ago and I can do it for longer. As a result, people can see the difference in my personality.
"I have always been a happy, positive sort of man, but I'm absolutely buzzing these days. People don't deny Grace the same opportunities to live as I have recently been given. That would be a sin."
Grainne said, "I am not giving up. In the last few weeks Grace has more pain in her legs and she limps more than she used to.
"She is very tired in school and finds it really tough to concentrate at times. When I hear about the difference Vimizim has made for Aoife and Dermot, it is unbelievable. I want that for my daughter! She deserves it!"
There are two other children receiving Vimizim in Ireland because they were part of the original clinical trial and it is normal procedure for the pharmaceutical company to continue to provide it for them.
"I am so happy for everyone else who gets Vimizim but it is not fair that Grace is not getting it. It is just not good enough to say ‘you are not getting the only drug that can help you.
"Two years after I started Campaign for Grace we are still waiting. There is no explanation for having a treatment and not giving it to someone who needs it."
In a statement the HSE said,"The HSE has met the company and no formal conclusion has been arrived at to date."