A decision is expected from the HSE on whether it will fund a treatment for a rare genetic disorder that has already been approved in both the United States and Europe.
The drug in question is omaveloxolone, sold under the brand name Skyclarys, which was first given the green light in the US for treating Friedreich’s ataxia, a progressive genetic condition that damages the nervous system and affects movement. European approval followed, raising hopes among patients and families in Ireland who have been waiting to see if the treatment will be made available here through the public health system.
Friedreich’s ataxia is a condition that typically begins in childhood or early adulthood. It causes increasing difficulty with coordination and walking, and over time can affect the heart and other organs. There is no cure, and for a long time treatment options have been largely focused on managing symptoms rather than tackling the underlying disease. Skyclarys represents a significant development because it is the first approved therapy specifically targeting the condition.
For Irish patients living with Friedreich’s ataxia, the wait for an HSE funding decision has been a stressful one. Rare disease treatments often come with high price tags, and the process of getting them approved for reimbursement through the Irish public health system can be lengthy. Families have been watching the progress of Skyclarys closely since its US approval, hoping Ireland would follow suit in making it accessible.
The HSE carries out its own health technology assessment process before agreeing to reimburse any new drug. This involves looking at the clinical evidence behind a treatment, weighing up its benefits and risks, and considering the cost to the health service. For rare diseases with small patient populations, these assessments can be particularly complex, as the pool of clinical trial data is often smaller than for more common conditions.
Patient advocacy groups in Ireland have been pushing for faster access to treatments for rare diseases more broadly, arguing that the current system can leave people waiting too long for therapies that could genuinely change their lives. The case of Skyclarys has become one of the more prominent examples of this ongoing tension between the pace of drug approvals internationally and the timeline for reimbursement decisions at home.
The number of people in Ireland living with Friedreich’s ataxia is relatively small, as is the case with most rare genetic disorders. But for those affected and their families, access to an approved treatment is not an abstract policy question. It is a matter of day-to-day quality of life and long-term health outcomes.
Once the HSE issues its decision, it will determine whether patients here can access Skyclarys through the public system or whether they would have to seek it through other means, which for many would simply not be a realistic option given the costs involved.
The expected decision has been anticipated for some time, and those in the rare disease community in Ireland will be watching closely when it finally comes.