Voretigene Neparvovec for Treating Inherited Retinal Dystrophies Caused by RPE65 Gene Mutations: An Evidence Review Group Perspective of a NICE Highly Specialised Technology Appraisal

Caroline Farmer (), Ash Bullement, David Packman, Linda Long, Sophie Robinson, Elham Nikram, Anthony J. Hatswell, G. J. Melendez-Torres and Louise Crathorne
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Caroline Farmer: University of Exeter
Ash Bullement: University of Exeter
David Packman: University of Exeter
Linda Long: University of Exeter
Sophie Robinson: University of Exeter
Elham Nikram: University of Exeter
Anthony J. Hatswell: University of Exeter
G. J. Melendez-Torres: University of Exeter
Louise Crathorne: University of Exeter

PharmacoEconomics, 2020, vol. 38, issue 12, No 5, 1309-1318

Abstract: Abstract The UK National Institute for Health and Care Excellence (NICE) considered evidence for voretigene neparvovec (VN; Luxturna®) for the treatment of RPE65-mediated inherited retinal dystrophies (IRD) within its highly specialised technology programme. This paper summarises the evidence provided by the company; the appraisal of the evidence by the Peninsula Technology Appraisal Group, who were commissioned to act as the independent evidence review group (ERG); and the development of the NICE guidance by the appraisal committee. The evidence presented by the company highlighted the significant lifelong burden of IRD for patients and carers. Evidence to support the effectiveness of VN was lacking, but the available evidence showed a modest, sustained improvement across a variety of vision-related outcomes. While patients would remain visually impaired, the committee considered that VN would prevent further deterioration in vision. The modelling approach used by the company had a number of limitations and relied heavily upon a large volume of clinical expert input to produce cost-effectiveness estimates with large uncertainty around long-term effectiveness. The ERG’s main concerns revolved around these long-term outcomes and the plausibility of utility values. The NICE committee were convinced that the clinical benefits of VN were important and an appropriate use of national health service resources within a specialised service. The committee concluded that a high unmet need existed in patients with RPE65-mediated IRD and that VN represents a step change in the management of this condition.

Date: 2020
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DOI: 10.1007/s40273-020-00953-z

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