ArticleAdvances in therapy2020
An Economic Evaluation of Voretigene Neparvovec for the Treatment of Biallelic RPE65-Mediated Inherited Retinal Dystrophies in the UK.
Article in Advances in therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 5 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 5 syntheses or guidelines pooled it, 34 citations in OpenAlex.
- A systematic review on the appropriate discounting rates for the economic evaluation of gene therapies: whether a specific approach is justified to tackle the challenges?International journal of technology assessment in health care · 2024Pooled it
- Evaluating the Quantity and Quality of Health Economic Literature in Blinding Childhood Disorders: A Systematic Literature Review.PharmacoEconomics · 2024Pooled it
- A systematic review of economic evaluations forInternational journal of technology assessment in health care · 2023Pooled it
- Capturing the Impact of Constraints on the Cost-Effectiveness of Cell and Gene Therapies: A Systematic Review.PharmacoEconomics · 2023Pooled it
- Economic Evidence on Potentially Curative Gene Therapy Products: A Systematic Literature Review.PharmacoEconomics · 2021Pooled it
- Patterns of X-Linked Retinitis Pigmentosa Genetic Testing in England and Implications for Service Provision.Ophthalmology science · 2026Article
- Article
- One down but many more to go: the state of gene therapy for inherited retinal disease.Regenerative medicine · 2025Review
- Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review.Healthcare (Basel, Switzerland) · 2025Review
- Estimating Australian Population Utilities for Inherited Retinal Disease Using Time Trade-Off.PharmacoEconomics - open · 2024Article
- The health care and societal costs of inherited retinal diseases in Australia: a microsimulation modelling study.The Medical journal of Australia · 2023Article
- Longitudinal analysis of health care costs in patients with childhood onset inherited retinal dystrophies compared to healthy controls.BMC ophthalmology · 2022Article
- Newer therapeutic options for inherited retinal diseases: Gene and cell replacement therapy.Indian journal of ophthalmology · 2022Review
- Cost-effectiveness of voretigene neparvovec in the treatment of patients with inherited retinal disease with RPE65 mutation in Switzerland.BMC health services research · 2022Article
- Challenges of Cost-Effectiveness Analyses of Novel Therapeutics for Inherited Retinal Diseases.American journal of ophthalmology · 2022Review
- Patient-Reported Health-Related Quality of Life in Individuals with Inherited Retinal Diseases.Ophthalmology science · 2022Article
- Critical Reflections on Reimbursement and Access of Advanced Therapies.Frontiers in pharmacology · 2022Article
- Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision.PharmacoEconomics · 2021Review
- Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy.Molecular diagnosis & therapy · 2020Review
- Cost Effectiveness of Voretigene Neparvovec for RPE65-Mediated Inherited Retinal Degeneration in Germany.Translational vision science & technology · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionVoretigene 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 (HST) process recommended VN for patients in England and Wales. We assessed the cost-effectiveness of VN compared with best supportive care (BSC) in individuals with biallelic RPE65-mediated IRD in the UK.
methodsA Markov model was developed to estimate the incremental cost per quality-adjusted life-year (QALY) gained for VN compared with BSC, from the perspective of the UK National Health Service and Personal Social Services. Phase III trial data were used to inform transition probabilities up to year 1, after which the treatment effect was assumed to be maintained for 40 years, followed by a decline in vision. A bespoke elicitation exercise involving clinical experts, patients and carers was conducted to estimate utility values for each model health state.
resultsAt list price, VN is associated with incremental costs of £612,404 and incremental QALYs of 6.4, resulting in an incremental cost-effectiveness ratio (ICER) of £95,072 per QALY gained. Voretigene neparvovec is associated with a significant undiscounted QALY gain (20.5) and is therefore eligible for additional QALY weighting under the NICE HST process; an ICER of up to £205,000 per QALY gained could be considered cost-effective under this framework.
conclusionThe results of the model show VN to be a cost-effective use of healthcare resources in the UK at list price. The availability of a commercial discount in the UK (as considered in the NICE appraisal) means that in reality the ICER will be even lower. Plain language summary available for this article.
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