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'The bad days and stress for Grace are now over'


Grace Cogan at home Photo: Ciara Wilkinson

Grace Cogan at home Photo: Ciara Wilkinson

Grace Cogan at home Photo: Ciara Wilkinson

After two years of campaigning, a girl with an ultra-rare disease has been told she can have the only drug in the world that can treat it.

Morquio has left Grace (9) the size of a toddler and living with pain and fatigue that will not get better without Vimizim.

"The bad days and stress of trying to get access to it for Grace are now over. She will get a chance at a better quality of life," her mother Grainne Cogan said.

The HSE told the family that Grace will be able to receive Vimizim - an enzyme replacement therapy - at Temple Street Children's Hospital.

Ms Cogan said she was shaking when she heard the news that she, her family and friends have worked so hard for.

"To us this means everything. Nothing comes close to this feeling. I feel like a weight has been lifted off me," she said.

The family live near Carrickmacross, a short drive from Northern Ireland where Vimizim is provided by the NHS to other adults, and children, with the condition.

Vimizim costs around €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.

That blow came just weeks after the HSE, in error, approved their application for Grace to receive the drug under the Treatment Abroad Scheme (TAS).

Now the family's stress and heartache is over as the HSE, which has the final decision on the matter, told them they would provide it for Grace.

"Grace is a very special girl who deserves every chance at life and hopefully from now on her symptoms will improve," Ms Cogan said.

She is not giving up her fight for anyone else with Morquio to also get Vimizim.

"It does not mean that the drug is approved in the Republic but because of the HSE's error in approving it through the TAS they have organised for Grace to have access to the drug," she said.

"I was working when I got the call and I was shaking with total shock and excitement all day.

"It is still very important to me, however, that the drug gets approved within the Republic so that any person, child or adult, diagnosed with this progressive condition has access to treatment that would help them."

A HSE statement said that access to a drug therapy is only available where the specific drug has been approved in this jurisdiction.


But it added: "An individual patient was approved access to drug therapy under the Treatment Abroad Scheme (TAS) in error.

"Notwithstanding this error the HSE gave a commitment to honour the TAS approval as our decision had already been communicated to the family and a reasonable expectation of access to the non-approved drug had been raised.

"The HSE is finalising a solution for the patient on an individual exceptional basis in Ireland and we hope to have a service in place shortly to enable treatment to be delivered."

Irish Independent