Call over access to drug to treat Friedreich's Ataxia

Sinn Féin leader Mary Lou McDonald has urged the government to ensure families affected by the rare inherited neurological condition, Friedreich's Ataxia, can access the drug Skyclarys, which is set to be reviewed at an upcoming meeting. The disease, which progressively damages the nervous system, impairs mobility, balance, and coordination.

Skyclarys, the first and sole EU-authorised pharmaceutical treatment for Friedreich's Ataxia, has the potential to slow the progression of symptoms. Despite clinical trials demonstrating its efficacy, there are concerns that the drug may be referred to another committee for further consultation and decision-making. Last June, Taoiseach Micheál Martin had expressed his desire for patients to receive access to the drug as swiftly as possible.

McDonald emphasized the urgency of the matter, stating that the progressive and debilitating nature of the disease leaves no room for delay. A recent campaign by a patient living with Friedreich's Ataxia resulted in a High Court permission for Emily Felix to seek a judicial review of the Health Service Executive (HSE) decisions concerning Skyclarys access.

The HSE drugs group is scheduled to reconvene to deliberate on the reimbursement application for Skyclarys, with the Rare Diseases Technology Review Committee (RDTRC) having recently considered the matter. The HSE has acknowledged that the RDTRC must weigh factors such as cost-effectiveness, the overall impact on the drugs budget, and opportunity costs in its deliberations.