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New HTA Decisions in England
May 2020
Drug name
LUXTURNA™ (voretigene neparvovec)
Novartis Pharmaceuticals UK
Decision date
Therapeutic area
Eye conditions
Therapeutic sub area
Eye conditions: general and other
Voretigene neparvovec is recommended, within its marketing authorisation, as an option for treating RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherited retinal dystrophy from confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
Decision Detail
It is recommended only if the company provides voretigene neparvovec according to the commercial arrangement.
The clinical evidence came from 2 studies. Study 101/102 was a phase I, open-label, dose-escalating, safety profile study in which individual patients (n=12) had 1 injection of voretigene neparvovec in 1 eye. Study 301 was a phase III, open-label, randomised controlled trial comparing the long-term efficacy and safety of voretigene neparvovec (n=21) given in both eyes with best supportive care. Results from study 301/302 showed that, at year 1, patients in the voretigene neparvovec arm had improved multiluminance mobility test (MLMT) scores compared with no improvement in the BSC arm. The difference was both statistically significant (mean difference 1.60; 95% confidence interval [CI] 0.72 to 2.40; p=0.0013) and clinically meaningful according to the criteria defined by the company (change score of 1 or more light level). Improvements in the MLMT score seemed to remain steady until 3‑year follow up. At year 3, the proportion who passed the MLMT at 1 lux (lowest light level) was 60% (12/20) in the original voretigene neparvovec arm (study 301) and 89% (8/9) in the delayed voretigene neparvovec arm (study 302). This showed a sustained improvement in functional vision for patients who had voretigene neparvovec. There is no long-term clinical evidence, but it is biologically plausible that the treatment effect is likely to continue for decades. Based on the committee's preferred assumptions, the ICER was between £114,956 per QALY gained using the company's EQ‑5D utility values and £155,750 per QALY gained using utility values based on the total population from Rentz et al. (2014) and the committee's preferred discount rate of 3.5%. The results of the cost–utility analysis incorporating a 1.5% discount rate were between £60,908 and £86,118 per QALY gained. These results are associated with the company's list price and do not include the company's confidential commercial arrangement. The committee concluded that voretigene neparvovec met the criteria for a QALY weight of 1.2. The committee was satisfied that voretigene neparvovec would offer significant QALY gains, and therefore applied this weighting in its consideration of its value for money. Some assumptions in the economic modelling are uncertain, particularly around the utility values and how long the treatment effect lasts. Despite the uncertainties, voretigene neparvovec is likely to provide important clinical benefits for people with RPE65-mediated inherited retinal dystrophies, and is considered an appropriate use of NHS resources within the context of a highly specialised service. It is therefore recommended for use in the NHS.