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СпутникиРНSpaceXРоскосмосNASADisease causes progressive damage to the nervous system with those affected having shorter life expectancy
A treatment for a rare genetic disorder, Friedreich’s ataxia, should not be reimbursed by the Health Service Executive (HSE), its drug group has recommended.
The disease causes progressive damage to the nervous system, with individuals who have the condition having a shorter than average life expectancy.
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.
Despite its EU approval, the drug is not available through the HSE in Ireland.
Last December, the National Centre for Pharmacoeconomics (NCPE) issued an assessment on the “cost-effectiveness” of Skyclarys. It recommended the drug not be considered for reimbursement by the HSE, adding that it would cost around €280,000 per patient annually.
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”.
In a statement, the HSE said its drugs group met on Tuesday afternoon and considered reimbursing Skyclarys, among other drugs.
“The drugs group examined submissions and data from Friedreich’s Ataxia patients, their doctors and advocacy groups. The group discussed the profound and devastating impact that Friedreich’s Ataxia has on quality of life and life expectancy,” the statement said.
“The group acknowledged that current treatment of Friedreich’s Ataxia focuses on symptom management and best supportive care and that omaveloxolone (skyclarys), an orphan medicine, is the first licensed therapy for Friedreich’s Ataxia.”
However, the HSE said the assessment from the NCPE found there are “limitations and uncertainties associated with the available clinical efficacy data”.
“They also concluded that the current price was substantially above the level typically regarded as cost-effective in Ireland having regard to the limited efficacy of the drug,” the statement said.
The HSE said it was based on all of this evidence that the drugs group “has decided not to recommend this drug for reimbursement at this time”.
A final decision on reimbursement will be made by the HSE’s senior management team in two weeks’ time.
Patients and their families have been leading a campaign in recent weeks to make the drug available in Ireland.
Earlier this month, the High Court gave Emily Felix, one woman with Friedreich’s ataxia, permission to challenge what she said is the HSE delay on granting her access to the drug.