Amazing Grace
Nine year old with ultra-rare condition desperately wants State to approve costly drug
THE mother of a nine-year-old girl waiting to hear if the State will pay for the only drug in the world that will treat her ultra-rare condition has told the Irish Mail on Sunday: ‘In my heart, I do not expect it to be approved because it costs so much.’
Vimizim would slow the progress of Morquio, an enzyme disorder, and possibly help to repair some of the already devastating effects on Grace Cogan, who is in constant pain. But the medication costs an estimated €400,000 for one year’s supply for an adult.
Gráinne Cogan said: ‘I feel like my daughter is left to suffer because there is a price put on how much she is worth.’
Grace is one of only five children in Ireland with Morquio.
She celebrated her ninth birthday last week with her family in Carrickmacross, Co. Monaghan, but she is much shorter than her peers; she is only as tall as a two or threeyear-old.
‘This disease, which Grace was born with, has left her in constant pain. She wakes up every night with the pain in her joints.
‘We are so frustrated because Grace is living in pain and does not have to. She suffers needlessly.’
The manufacturers, BioMarin, have made an application for the drug to be funded by the HSE. As part of that process, Vimizim is being assessed by the National Centre for Pharmacoeconomics (NCPE).
Gráinne said: ‘We have no idea if the drug will be approved by the NCPE. It is out of our hands. We just have to wait and hope it’s the right decision for Grace. It is very frustrating for us knowing that there is a treatment available and in use in other EU countries like Scotland, England and Wales and Grace has not got access here.
‘If the NCPE approve the drug, we hope the HSE will agree to fund it just like the NHS are funding it in the UK. We know it is an expensive drug but money should not be the important factor here.’
Gráinne said that Grace had started to experience respiratory problems, a classic effect of Morquio, because her organs are continuing to grow but her skeleton is not. This has also put pressure on her spinal cord, resulting in two highrisk surgeries. Grainne said that Grace’s breathing had changed in recent weeks. ‘It is noisy, she has a cough and unless she gets Vimizim we will see more problems.’ The NCPE website says that the drug is still being assessed. The HSE said that, when the Health Technology Assessment (HTA) report is received, ‘it will be reviewed by the HSE and if required discussions will be had with the pharmaceutical company in relation to any issues identified through the HTA process. ‘The HSE Drugs group will consider the application and make a recommendation to HSE leadership,’ it said.
‘Money should not be an important factor here’