HSE agrees to pay for medicine to treat rare condition

The HSE has agreed to pay for two children to receive the only medicine that can treat their rare disease.

They have been without the drug following a dispute over cost, but they and their families are overjoyed after the HSE said ‘yes’ to Vimizim.

“It is the most amazing news,” said Barbara Frow, whose daughter Grace, 10, along with Cezy Fosca, 12, stopped receiving the drug last month.

Grace, from Leitrim and Cezy, from Kilkenny, have Morquio, a form of Mucopolysaccharide disease (MPS) which causes lifelong problems including heart disease, skeletal abnormalities, vision and hearing loss, and respiratory issues.

Vimizim is the only medicine in the world to treat the condition and it replaces the enzyme that their body is missing.

A number of requests from pharmaceutical company BioMarin to be reimbursed were turned down by the HSE and last year the company said it could no longer provide it free to the girls. The girls initially received it more than five years ago as part of clinical trials, and BioMarin then kept providing it to them on compassionate grounds.

On Thursday, the families were told by their medical team in Temple St that it had been approved.

Cezy’s mother, Elena, said: “We were so delighted to get the good news and we are very happy for Cezy and for Grace to be back on track with Vimizim. It was a very stressful situation for us as a family. Cezy was without medicine for almost two months.”

“We can’t believe we’ve received the most amazing news for us. It’s even better than the Easter Bunny! The HSE phoned Temple St to finally, in response to the fourth application by BioMarin, give us a ‘yes’ for Vimizim funding by the HSE under a managed access scheme. We’re extremely thankful to the HSE for approving Vimizim for Grace and Cezy.

“The relief hasn’t even sunk in yet, it’s like a dream. It’s all we’ve been able to think of since August last.”

Cezy Fosca, 12, and Grace, have Morquio, a form of Mucopolysaccharide disease.

Ms Frow added: “We can finally get back to some kind of normal life now Grace has the chance of a healthy life again. Grace is over the moon with joy now she’s got her meds back and even the needles again haven’t phased her. She’s planning a bit of a party when school’s back.”

She said throughout their fight for continued access, “Grace has always been our main driving force.”

It was originally noted that Vimizim costs around €400,000 per adult and it is not known what the final price agreed by the HSE and BioMarin is.

In a statement, the HSE said that late last year, non-approval was “on the basis of cost”. A spokeswoman confirmed that it has now been approved: “Following negotiations over the past six months, the manufacturer has provided a revised offer that involves a significant benefit to the State and has reached a threshold that allowed approval by the HSE Drugs Committee.”

The HSE said: “Finalisation of contractual terms is under way today,” and it is expected that access will start shortly after Easter.

On its Facebook page, the Irish MPS society said, “After a long 12 months of campaigning & lobbying our TDs and Senators we are delighted to announce that all our hard work has paid off. On Thursday 29th March 2018 the HSE said Yes to Vimizim. We wish to thank everyone who has supported us in any way.”