Voretigene Neparvovec for Treating Inherited Retinal Dystrophies Caused by RPE65 Gene Mutations: An Evidence Review Grou
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REVIEW ARTICLE
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 Farmer1 · Ash Bullement1,2 · David Packman1 · Linda Long1 · Sophie Robinson1 · Elham Nikram1 · Anthony J. Hatswell1,2 · G. J. Melendez‑Torres1 · Louise Crathorne1
© Springer Nature Switzerland AG 2020
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.
Key Points for Decision Makers This appraisal demonstrates how clinical judgement, patient experience, and ‘biological plausibility’ may play an important role in addressing gaps in an evidence base. Attempts to develop complex economic models that provide a valid representation of patient experience need to also balance reliability when sample sizes are small. Analyses, such as threshold analyses, to explore the potential impact of uncertainties in a limited evidence base may be informative for decision making. * Caroline Farmer [email protected] 1
Peninsula Technology Assessment Group (PenTAG), University of Exeter, South Cloisters, St Luke’s Campus, Heavitree Road, Exeter EX1 2LU, UK
Delta Hat, Ltd., Nottingham, UK
2
1 Introduction Inherited retinal dystrophies (IRD) are a group of eye disorders caused by gene mutations that result in the gradual degeneration of photoreceptor cells on the retina [1]. In RPE65-mediated IRD, patients exhibit a mutation in the RPE65 gene, which (when functioning correct
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