The HSE has approved reimbursement for a treatment for a rare genetic disorder, Friedreich’s ataxia, making it available to Irish patients.
The disease causes progressive damage to the nervous system, and individuals with the condition have a shorter than average life expectancy. About 200 people in Ireland are estimated to have the condition.
In February 2023, the drug omaveloxolone, branded as Skyclarys, was approved in the US for the treatment of the disease. Europe followed suit 12 months later.
Skyclarys is not a cure but patients and advocates say it has the ability to slow the progression of Friedreich’s ataxia by up to 55 per cent.
READ MORE
Last December, the National Centre for Pharmacoeconomics issued an assessment on the “cost-effectiveness” of Skyclarys. It recommended that the drug not be considered for reimbursement by the HSE, stating it would cost about €280,000 annually per patient.
On July 14th, the HSE Drugs Group did not recommend the drug for reimbursement and referred the application to the Rare Diseases Technology Review Committee “to seek further input”.
On August 11th, following the committee meeting, the group recommended that the drug should not be reimbursed. It stated the estimated cost was too high, as well as questioning the effectiveness of the treatment.
The HSE’s senior management team met on Tuesday to make a final decision on the drug, and approved it for reimbursement.
According to a statement from the HSE, Biogen, the manufacturer, made a “substantially improved financial offer” following “intensive discussions” over the cost of the medicine.
“Biogen has moved substantially from its previous position, and the HSE wants to express its appreciation of the flexible and constructive approach shown by the company,” the statement said.
The HSE said it decided to approve reimbursement of the drug based on the new commercial offering.
“It concluded that had it been asked to consider the same terms the HSE Drugs Group considered, it would have come to the same position as the Drugs Group and would not have proposed reimbursement,” it said in a statement.
In coming to its decision, the HSE acknowledged the “limited efficacy” and the high cost that remained despite the improved financial offer.
“However, it also agreed with the observations of the Drugs Group in relation to the substantial unmet need of patients suffering from what is a devastating disease and the absence of any other medicines to treat it,” it said.
“Engagements are ongoing with Biogen to finalise the administrative arrangements which will underpin reimbursement. The HSE will progress these engagements quickly, and understands that Biogen are also committed to finalising details at an early stage.”
The decision comes following months of campaigning and the Government facing significant political pressure from Opposition TDs calling for reimbursement of the drug.
A demonstration of Friedreich’s ataxia campaigners took place in Dublin on Sunday, led by Niamh Ní Hoireabhaird from Co Kildare, Emily Felix from Co Kilkenny and Aoife Gavan from Co Monaghan.
Rare Disease Ireland, an alliance of rare disease patient organisations, on Monday urged the HSE to continue negotiations on the price of Skyclarys, in the first such intervention the group has ever made.
Representative groups had called for political intervention, but Government officials maintained the process was non-political and independent of Leinster House.
Minister for Health Jennifer Carroll MacNeill described Tuesday as an “important day” for patients.
“Though I know the statutory process through which these decisions are made is complex, it is important that politics and politicians allow the space for science to be the decision maker,” she said.
“At a time when there is too much uncertainty in the world, Ireland will continue to stick to science and to the rule of law. Simply put, these processes must be independent and led by experts.”
Taoiseach Micheál Martin also welcomed the decision, adding it was important “the statutory process for approving the drug was brought to its conclusion.
“It has been a very challenging period for the families, and I wish to acknowledge that,” he said.
