Minister defends review process for breakthrough drug

The Government has yet to decide whether it will fund the breakthrough Friedreich’s Ataxia drug Skyclarys, with Minister for Health Jennifer Carroll MacNeill confirming the treatment remains under scientific and economic review.

Speaking to The Anglo-Celt during a visit to Monaghan Hospital, the Minister acknowledged the anxiety facing patients and their families, including Castleshane woman Aoife Gavan, who has become one of the leading voices in the campaign to secure State funding for the drug.

The Celt has previously reported how Ms Gavan believes she is "running out of time" to benefit from Skyclarys. The drug, the first treatment approved for Friedreich’s Ataxia, has been shown to slow progression of the rare degenerative neurological condition but it can’t reverse damage already done.

Campaigners have repeatedly stressed that every month without access risks patients losing abilities that cannot be regained.

Last month, Ms Gavan joined fellow campaigners outside Leinster House as the HSE considered the latest stage of the reimbursement process. While no decision emerged from that meeting, families continue to press the Government to approve funding for the treatment, which costs around €300,000 per patient annually.

Minister Carroll MacNeill said every medicine seeking reimbursement must undergo a rigorous two-stage assessment. "With all of the rare disease drugs, with all of the drugs generally, it goes through a two-stage process, testing the effectiveness of it and then seeing how the effectiveness relates to the price," she said.

The Minister said Skyclarys was approved by the European Medicines Agency and that the Government received a commercial proposal from its manufacturer in May before the HSE Drugs Group carried out an initial review earlier this month.

However, she said the evidence surrounding the treatment is less clear-cut than for some other medicines. "Across Europe it’s a pretty mixed outcome. I think there’s only 12 countries that have approved it and a lot of countries our sort of size have not approved it based on that mix of effectiveness and cost," she said.

"Belgium, the Netherlands, Denmark... there are a sizable number of countries that haven’t and it is one of those drugs where the science is more mixed, which is unfortunate."

Despite that, the Minister said she understood the hope the treatment represents for families.

"From a human point of view, from a patient perspective, I totally understand it," she said. "I know how significant any sense of a breakthrough can be for somebody’s life and for keeping a condition at bay."

She confirmed the application has now moved to the Rare Disease Technical Review Group, made up of clinicians who specialise in treating the condition, before any final recommendation is made.

"Our goal is always to try to get effective drugs to our patients. That’s what we want to do. That’s why we spend nearly €4 billion on drugs every year," she said.

Addressing reports that some Irish patients may relocate abroad to access Skyclarys, the Minister said availability across Europe is more limited than many believe.

"I know that there’s an idea that it’s widely available everywhere else. It isn’t necessarily true," she said, adding that several countries comparable in size to Ireland have either declined to reimburse the drug or have introduced strict eligibility criteria.

She also pointed to the difficulties smaller countries face when negotiating with pharmaceutical companies over high-cost medicines.

"We are trying to see how we can partner better with some of those other countries to increase our opportunity to negotiate," she said.

No timeline has been given for a final decision, leaving patients such as Aoife Gavan waiting to learn whether the treatment they believe could slow the progression of their condition will be made available through the HSE.