Drug for LHON not Recommended for Reimbursement at Current Price

The National Centre for Pharmacoeconomics (NCPE) today published its recommendation on reimbursement of the drug Idebenone (commercial name Raxone®) for the treatment of visual impairment in adolescent and adult patients with Leber Hereditary Optic Neuropathy (LHON). The NCPE report recommends that idebenone “not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatments.”

Following the recommendation from the NCPE, the HSE examines all the evidence which may be relevant for the decision; the final decision on reimbursement is made by the HSE. As part of the assessment carried out by the NCPE, Fighting Blindness, after consultation with a number of our members who are living with LHON, made a submission to the process. This submission was included in full in the final report to the HSE.

While we are disappointed with this recommendation, it is important to note that this is not a categorical no to reimbursement. It simply means that the assessment found that the drug does not present value for money to the State at the current price offered by the pharmaceutical company. This recommendation opens the door for negotiations between the company and the HSE to come to an arrangement on a price that is acceptable to both parties. Fighting Blindness enourages both the company and the HSE to begin talks as soon as possible and work to ensure that people with LHON in Ireland can get access to this treatment in a timely and equitable manner.

Leber hereditary optic neuropathy (LHON) is a genetic disease that leads to sudden central vision loss during young adult life, leaving only peripheral vision. Men are more likely to be affected than women. There is currently no treatment available for this condition in Ireland. LHON is a disorder caused by mutations in the genetic code of the mitochondria, which are small subunits that reside within the cell. Idebenone aims to protect cell membranes and mitochondria from oxidative damage. It has been shown in studies to improve vision by up to three letters on a standard eye chart after treatment over a 24-week period. More recent analysis of data from an Expanded Access Programme has shown that the number of people responding well to the treatment increased with a longer treatment duration of 12 and 24 months.

Background to this Decision

The European Commission granted a marketing authorisation valid throughout the European Union for Raxone in September 2015. A full pharmacoeconomic assessment of Raxone was commissioned by the HSE in June 2017 and a submission was made by the company Santhera Pharmaceuticals in November 2017. Fighting Blindness made a submission to the process in January 2018.

Fighting Blindness will keep you up to date with any developments in this area. If you have any questions about this process please contact Caitríona on 01 6789 004 or caitriona.dunne@fightingblindness.ie.

The full summary report is available on the NCPE website here.

If you would like information about research into LHON and other potential therapies that are currently being investigated, please contact the Fighting Blindness Research Department on 01 6789 004 or research@fightingblindness.ie.

Please note Fighting Blindness does not endorse any of the products, medications, treatments or information reported here, or on any of our communication platforms. Articles on Fighting Blindness websites and social media are intended for information purposes only. We strongly advise that you discuss all medications, treatments, and/or products with your eye doctor.