Tell policy makers what it’s like to live with retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA)

Friday 7 August, update: 

We can now confirm that this supporting document was submitted to the National Centre for Pharmacoeconomics (NCPE) on July 28, 2020. The NCPE is responsible for assessing new medicines in Ireland and their potential impact on Government budget.

On review of all the available evidence, the NCPE will then issue a final report with conclusions and recommendations to the HSE Corporate Pharmaceutical Unit (HSE-CPU).

We anticipate a decision by the HSE on whether this gene therapy will be reimbursed in Ireland later this year.

The role of patient involvement in decision-making processes like this is vital. This process has been a first for Fighting Blindness and the inherited retinal degeneration community in Ireland. Our submission would not have been possible without your feedback and contribution. Thank you for your support and we look forward to keeping you updated.

It is possible to track the status of this submission on the NCPE website.


Our primary mission is to cure blindness and advocate for access to therapies as they become available.

Luxturna™ (voretigene neparvovec) is the first gene therapy approved to treat vision loss caused by mutations in the RPE65 gene. Conditions caused by this type of mutation include very rare forms of Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP).

Given the rarity of the genetic mutation, there are just a small number of people in Ireland who may be eligible for Luxturna. However, this serves as proof that gene therapies can be effective for treating inherited retinal degenerations (IRDs). And it paves the way for future therapies that have the potential to treat a wider group of people.

The pharmaceutical company responsible for Luxturna, Novartis, has applied to the HSE to have it made available and reimbursed in Ireland. Because this drug is considered to have a high cost and a significant budget impact it must undergo a full assessment, called a Health Technology Assessment (HTA). It will evaluate all evidence surrounding the drug including effectiveness and impact on the Government budget.

The role of patient involvement and perspectives in decision-making processes like this is vital. As part of the HTA, Fighting Blindness will submit a supporting document to highlight, directly to the decision makers, the true lived experiences of these conditions.

To inform our submission, we’ve launched a survey to gather information about the impact of living with these conditions and the impact a new medicine might have on quality of life.

The survey is open to people living with retinitis pigmentosa (RP), Leber congenital amaurosis (LCA), including their families and carers. It contains nineteen questions divided into three sections and will take between fifteen and twenty minutes to complete.

The closing date for completion is Monday, June 29, 2020.