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

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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.

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Acknowledgements

The views expressed in this paper are those of the authors and not necessarily those of the NHS, the NIHR SER programme, or the Department of Health and Social Care. Any errors are the responsibility of the authors. S. Bello, S. Dodman, M. Rezaei Hemami, A. Churchill, and A. Lotery all contributed to the ERG appraisal of VN. A. Churchill and A. Lotery acted as clinical advisors to the evaluation and reviewed the epidemiology and clinical-effectiveness sections of the report.

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Authors and Affiliations

  1. Peninsula Technology Assessment Group (PenTAG), University of Exeter, South Cloisters, St Luke’s Campus, Heavitree Road, Exeter, EX1 2LU, UK

    Caroline Farmer, Ash Bullement, David Packman, Linda Long, Sophie Robinson, Elham Nikram, Anthony J. Hatswell, G. J. Melendez-Torres & Louise Crathorne

  2. Delta Hat, Ltd., Nottingham, UK

    Ash Bullement & Anthony J. Hatswell

Authors
  1. Caroline Farmer
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  2. Ash Bullement
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  3. David Packman
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  4. Linda Long
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  5. Sophie Robinson
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  7. Anthony J. Hatswell
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  8. G. J. Melendez-Torres
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  9. Louise Crathorne
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Contributions

All authors commented on the submitted manuscript and have given their approval for the final version to be published. CF, LL, AJH, GJM-T, and LC critiqued the clinical burden and clinical effectiveness methods and evidence; SR critiqued the literature searches conducted by the company; AB, DP, EN, and AJH critiqued the health economic method and evidence and the company’s economic evaluation. LC supervised the appraisal and acted as guarantor. This article was not externally peer reviewed by PharmacoEconomics.

Corresponding author

Correspondence to Caroline Farmer.

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Funding

The appraisal of VN within the NICE HST programme was commissioned by the National Institute for Health Research Systematic Reviews Programme as project number 17/56/05.

Conflict of interest

CF, AB, DP, LL, SR, EN, AJH, GJM-T, and LC have no conflicts of interest that are directly relevant to the content of this article.

Data availability

Documents relevant to this appraisal (including the appraisal scope, CS, ERG report, stakeholder submissions, and NICE guidance issued) can be found on the NICE website at https://www.nice.org.uk/guidance/hst11.

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Farmer, C., Bullement, A., Packman, D. et al. Voretigene Neparvovec for Treating Inherited Retinal Dystrophies Caused by RPE65 Gene Mutations: An Evidence Review Group Perspective of a NICE Highly Specialised Technology Appraisal. PharmacoEconomics 38, 1309–1318 (2020). https://doi.org/10.1007/s40273-020-00953-z

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