An Economic Evaluation of Voretigene Neparvovec for the Treatment of Biallelic RPE65-Mediated Inherited Retinal Dystrophies in the UK

Abstract

Introduction: Voretigene neparvovec (VN) is a gene therapy and the first approved pharmacological treatment for biallelic RPE65-mediated inherited retinal dystrophies (IRD), a rare condition that starts in early life and causes vision to progressively deteriorate towards complete blindness. In a phase III trial, treatment with VN significantly improved functional vision and visual function, and in October 2019 the National Institute for Health and Care Excellence (NICE) Highly Specialised Technologies Enhanced Digital Features To view enhanced digital features for this article go to https://doi.org/10.6084/ m9.figshare.11637108.

Cost Effectiveness of Voretigene Neparvovec for RPE65-Mediated Inherited Retinal Degeneration in Germany

“…A decreasing treatment effect is mainly associated with a loss of QALYs and higher overall costs, primarily driven by the increase of indirect costs. This is consistent with the results of Johnson et al 10 and Viriato et al, 11 where the treatment effect was also identified as the largest individual driver. We expect that estimation of treatment effect duration may be one of the main factors for cost-utility analyses not only in VN, but also in many future gene therapies.…”

“…Because indirect cost data vary greatly between different countries, a direct comparison between studies is not possible. However, additional QALYs gained and ICURs of our results point in the same direction as the 2020 study by Viriato et al, 11 who estimated VN to be cost effective for the UK and assumed a gain of 6.4 QALYs. This study confirms our finding that substantial QALY gains can be attributed to the therapy when its effects are modeled with a life time perspective.…”

“…In the UK, a confidential discount on the price list has been agreed upon. 11 For Germany, price negotiations of VN may have a direct impact on pricing of future gene therapies and on their cost effectiveness. Our cost-utility analysis shows that VN is likely to be cost effective and may be beneficial not only to patients but also from a societal perspective in Germany.…”

“…We built a probabilistic lifetime model that simulates pathways of treatment-naïve patients with confirmed biallelic RPE65-mediated IRD. Consistent with previously published economic models for RPE65, we simulate 70 patients 10 in 1000 Monte Carlo simulations, 10,11 where parameters are varied by random draws according to their distribution. Patients are equally divided into two groups and chosen by random draws to be treated with VN or with standard of care (SoC).…”

“…Implementation of the phase III clinical trials' population to an RPE65 simulation model was already successfully performed in similar economic models. 10,11,14 In RPE65-mediated IRD, BCVA, which is often already significantly decreased, is typically relatively stable during the first life decade with a gradual decline starting around life year 15 to 20. 4 To reflect this natural disease progression, our model assumes a constant BCVA until life year 15 to 20.…”