The Minister for Health, Stephen Donnelly TD is pleased to announce that the drug Patisiran (Onpattro) has been approved by the HSE for reimbursement in the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis.
hATTR amyloidosis is an inherited, rare, life-limiting, multi-systemic disease where there are limited treatment options currently available.
In order for the HSE to recommend in favour of reimbursement of Patisiran (Onpattro), the HSE has undertaken considerable engagement and negotiation with the manufacturer Alnylam over a protracted period.
Speaking today, Minister Donnelly said, “The reimbursement of Patisiran by the HSE is a welcome decision for hereditary transthyretin-mediated amyloidosis patients, their families and friends.
"Our country continues to make new and innovative treatment available to patients and the allocation of €50m in Budget 2021 for new medicines ensures that this continues. However, securing access to treatments for patients at an affordable price remains the key priority. I wish to take the opportunity to thank the HSE for the dedication it has put into the Patisiran negotiations over a long period, which required significant work and deserves recognition.”
The HSE now advise that they will develop and implement the required managed access programme, as required for reimbursement, in order for all eligible patients with hATTR amyloidosis with stage 1 or stage 2 polyneuropathy to access treatment with Patisiran in the near future.
ENDS//
Notes for Editors :
What is hereditary transthyretin-mediated (hATTR) amyloidosis?
hATTR amyloidosis is a very rare life-threatening genetic disease caused by mutations in the transthyretin (TTR) gene. This leads to the production of abnormal TTR protein which causes build-ups called amyloid deposits in the body’s tissues and organs (referred to as amyloidosis). If the amyloid deposits build up around the nerves this causes nerve damage (polyneuropathy).
What is Patisiran (Onpattro)?
Patisiran is an intravenous drug licensed for use in patients with hATTR amyloidosis, who have stage 1 or 2 polyneuropathy.
How many patients in Ireland are eligible for the medicine?
The National Centre for Pharmacoeconomics (NCPE) has estimated that on average nine patients will receive treatment with Patisiran in year one, rising to 45 patients in year five.
What Are the Next Steps?
The HSE will now develop and implement the required managed access programme, as required for reimbursement, in order for all eligible patients to access treatment with Patisiran. However, the HSE stress that they cannot determine an exact date for when reimbursement will commence due to certain requirements that are needed to support this reimbursement.
Useful links: http://www.ncpe.ie/wp-content/uploads/2019/01/Patisiran-plain-english-summary-24.02.2020.pdf