ReviewPharmacoEconomics2020
Voretigene Neparvovec for Treating Inherited Retinal Dystrophies Caused by RPE65 Gene Mutations: An Evidence Review Group Perspective of a NICE Highly Specialised Technology Appraisal.
Review in PharmacoEconomics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
13 citing papers in PubMed, 2 syntheses or guidelines pooled it, 23 citations in OpenAlex.
- A systematic review of economic evaluations forInternational journal of technology assessment in health care · 2023Pooled it
- Economic Evidence on Potentially Curative Gene Therapy Products: A Systematic Literature Review.PharmacoEconomics · 2021Pooled it
- Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review.Healthcare (Basel, Switzerland) · 2025Review
- Real-world outcomes of Voretigene Neparvovec: a single-centre consecutive case series.Eye (London, England) · 2025Article
- Estimating Australian Population Utilities for Inherited Retinal Disease Using Time Trade-Off.PharmacoEconomics - open · 2024Article
- The extent and quality of qualitative evidence included in health technology assessments: a review of submissions to NICE and CADTH.International journal of technology assessment in health care · 2023Review
- [Inherited retinal diseases in Germany-Challenges in health care supply structure and diagnostics].Die Ophthalmologie · 2023Article
- Retinitis Pigmentosa: Burden of Disease and Current Unmet Needs.Clinical ophthalmology (Auckland, N.Z.) · 2022Article
- RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy.Orphanet journal of rare diseases · 2021Article
- Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision.PharmacoEconomics · 2021Review
- An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models.Journal of market access & health policy · 2021Review
- Cost Effectiveness of Voretigene Neparvovec for RPE65-Mediated Inherited Retinal Degeneration in Germany.Translational vision science & technology · 2020Article
- An Economic Evaluation of Voretigene Neparvovec for the Treatment of Biallelic RPE65-Mediated Inherited Retinal Dystrophies in the UK.Advances in therapy · 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
9 authors at 2 institutions in 1 country.
Funding
Abstract
The UK National Institute for Health and Care Excellence (NICE) considered evidence for voretigene neparvovec (VN; Luxturna
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.