Fighting Blindness, the only patient led charity funding research into retinal diseases, is calling on the Health Service Executive (HSE) to reimburse a life-changing gene therapy, voretigene neparvovec (Luxturna®), and to provide access to people in Ireland living with severe vision loss due to an inherited retinal dystrophy (IRD) caused by biallelic RPE65 mutation.
This follows today’s publication by the National Centre for Pharmacoeconomics (NCPE) and its recommendation that ‘the HSE consider not funding voretigene neparvovec unless its cost effectiveness (value for money) can be improved.’
Luxturna® treats (RPE65-mediated) inherited retinal dystrophies, including forms of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). They involve progressive loss of vision, which ultimately leads to total blindness.
The NCPE will send their recommendation to the HSE who will look at all the relevant data about voretigene neparvovec (Luxturna®). The HSE makes the final decision on reimbursement.
When asked what difference this medicine would make, a parent of a two year old with an inherited retinal dystrophy shares “Our child is very young and potentially if she were to receive this treatment early in her life she might never have to know what is was like to have a visual impairment and just go on and have a normal life which is really what any parent would wish for their child.”
Dr Laura Brady, Head of Research adds, “For almost four decades, Irish patients have supported medical research in the hope of discovering treatments and cures and this community believes that one is long overdue. Inherited retinal degenerations are ‘actionable diseases’ with the potential to dramatically improve the overall well-being and quality of life for both the patient and wider family. Luxturna® is now available in the UK and other European countries and we wish to have the same opportunities afforded for our community in Ireland.
Kevin Whelan, CEO of Fighting Blindness urges the HSE to consider all the evidence available. “The degenerative nature of these conditions can limit every aspect of a patient’s life both on a day to day basis but also across their lifespan. With no other treatment available, Luxturna® fulfils a significant unmet need and health gap in Irish eye-care. It is the first and only medicine to address the underlying cause of these devastating conditions. Fighting Blindness firmly believes that with investment into this patient cohort, the costs associated with care and support across a patient’s lifetime will be reduced. It also fits the recent commitment to rare diseases under the current Programme for Government.”
The NCPE summary report in plain English is available on their website.
In July, Fighting Blindness submitted a supporting document to the health technology assessment for voretigene neparvovec (Luxturna®) in Ireland.
We will continue to campaign on your behalf for the reimbursement of voretigene neparvovec (Luxturna®) in Ireland.
Speaking to the media and raising national awareness will be an important element of our campaigning in the coming weeks. If you are the parent of a child living with retinitis pigmentosa (RP) or Leber congenital amaurois (LCA) and you’re willing to share your story – please do get in touch with us.
If you have any questions or would like to speak to us about sharing your story, please email firstname.lastname@example.org.