Patients with rare diseases 'cannot wait' for another review into drug approval processes

Rare Diseases Ireland CEO Vicky McGrath: 'we need to find and design a system that takes some of the burden off the patient community and keeps them off the streets protesting.' Picture: Sam Boal/Collins

People living with rare diseases cannot wait for another review of drug approval systems, a representative group has warned, as it called for “heads to be banged together” to resolve the issue.

It follows an announcement last week by health minister Jennifer Carroll MacNeill of a review into how new medicines are approved here.

However, Rare Diseases Ireland CEO Vicky McGrath, raised concerns as it is only three years since a similar review was carried out by professional services firm Mazars for the Government.

“We need to move on today. It is underwhelming [to hear of a new review]. Is this Mazars 2.0?” she said.

“What changes did that report bring about for us? There’s still no real impact on the ground for people living with rare diseases and getting access to medicines in a timely fashion.

“From our perspective, we need to find and design a system that takes some of the burden off the patient community and keeps them off the streets protesting.”

In recent days, Craig Coady, whose son Paudie lives with the devastating disease Friedreich’s ataxia, has campaigned publicly for access to a drug called Skyclarys. He has already lost one son Rory to the disease, aged just 13.

Families of children with Duchenne muscular dystrophy also protested for access to a medicine called Givinostat. This was only recommended for reimbursement last week.

Ms McGrath said: “It’s just heartbreaking to see all of these families and individuals on the streets. I know it drives media coverage, but this is not the right approach for anybody.

“We need to knock the relevant heads together and say ‘right, in the next six months you need to design something so we don’t have anybody else going onto the streets to get access to a medicine’.”

She called on the pharmaceutical industry and the State to work out a plan, saying this must address backlogs and prevent future delays.

In the meantime, families need “some kind of compassionate programme” for emergency access, she added.

“The disease is constantly ticking away, and slowly but surely their abilities are being limited.”