Why carry out this study?

Voretigene neparvovec (VN) is a gene therapy treatment for biallelic RPE65-mediated inherited retinal dystrophies, an extremely rare and previously untreatable condition that leads to complete blindness in almost all patients. The condition has a profound impact on patients’ quality of life, and visual impairment is associated with economic burden at the individual and societal levels.

The aim of the study was to determine if VN can be considered a cost-effective use of National Health Service (NHS) resources in the UK, by modelling the costs incurred and benefits gained from treatment, compared with the current standard of care.

What was learned from the study?

The cost-effectiveness model determined the incremental cost-effectiveness ratio (a measure of cost per unit benefit gained) to be £95,072 per quality-adjusted life-year gained.

This is lower than typical thresholds below which technologies for rare diseases are generally considered to be cost-effective in the UK, and so VN is expected to be a cost-effective use of NHS resources.