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

Abstract

Background: Gene 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.

Methods: Selected 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.

Results: Pay-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.