Résumé
The advent of gene therapies on the market has sparked great hopes in the management of genetic diseases, offering previously unimaginable prospects for cure. However, these treatments face significant challenges, particularly in terms of funding, pricing, and long-term effectiveness uncertainty. Therefore, to what extent is France and its healthcare system economically prepared for the arrival of gene therapy? What financing, pricing, and regulatory solutions should be considered for these innovative products? To address these questions, a mixed-method study was conducted, consisting of an in-depth literature review and semi-structured interviews with experts. These interviews allowed for the collection of diverse perspectives and enriched the understanding of the pricing and funding issues related to gene therapies in France. Stakeholders differ on solutions, but converge on the crucial importance of health economic analyses for establishing fair prices. Article 54 of the LFSS for 2023, intended to provide solutions, raises concerns due to its rigidity and the mandatory performance-based payment contracts. Proposed improvements, such as increased conventionnal flexibility and enhanced data regulation, are being considered. Adressing these challenges requires ongoing cooperation among all stakeholders to ensure equitable access to treatments while preserving the financial sustainability of the healthcare system.