Evidence map›Paper›PMID 40864185›Full record

ReviewDie Ophthalmologie2025

[Gene therapy methods for rare ophthalmological diseases in the light of distributional justice-An overview].

Carsten Fluck

Abstract readEnglish AbstractReview
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In one paragraph

Review in Die Ophthalmologie, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

1 author.

Carsten FluckJuristische und Wirtschaftswissenschaftliche Fakultät, Juristischer Bereich - Lehrstuhl für Öffentliches Recht, Martin-Luther-Universität Halle-Wittenberg, Universitätsplatz 3-5, 06099, Halle (Saale), Deutschland. carsten-trevor.fluck@jura.uni-halle.de.ORCID http://orcid.org/0009-0008-6895-7075

Funding

Bundesministerium für Bildung und Forschung 01GP2212
6 · The paper itself

Abstract

backgroundGene therapies offer promising approaches for the treatment of genetic diseases. With the approval of Neparvovec (Luxturna®, Novartis) in 2018, a gene therapy for the treatment of retinitis pigmentosa and Leber's congenital amaurosis in RPE65 mutations, this innovation has also reached ophthalmology practice. Despite their potential, gene therapies raise significant ethical and economic issues, particularly with regard to distributive justice, which could limit access and increase inequalities. AIM OF THE PAPER: This article examines distributive justice from an ethical perspective, focusing on the just rationing of health resources and the affordability of gene therapies for rare diseases. The aim is to highlight areas of tension between ethical demands, economic interests and the financial sustainability of the healthcare system.

methodsSelected literature will be reviewed to shed light on the distribution of scarce healthcare resources, potential financing models and the evaluation of appropriate pricing. In addition, challenges specific to ophthalmology are addressed.

resultsPay-for-performance models link costs to effectiveness, but do not address all challenges. Centralized data collection and negotiation structures increase efficiency and strengthen the position vis-à-vis manufacturers, while fund models such as the HIF promote the predictability of healthcare expenditure. In ophthalmology, questions arise regarding the prioritization of patient groups, intervention thresholds and the distribution of scarce resources, for example when treating the second eye after successful treatment of the first.

Indexed as

Eye DiseasesGenetic TherapyHealth Care RationingRare DiseasesSocial JusticeGermanyHumansDistributive justiceGene therapyOrphan drugsPay-for-performanceRare diseases

Identifiers

What OpenQuestion holds

Textmetadata
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Registered trials

None linked

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.