Presentation (CTI)

OBJECTIVES: Gene therapies offer groundbreaking advancements in medicine, providing potential cures for previously untreatable genetic disorders. However, ensuring equitable access to these therapies presents a critical ethical challenge within the European Union (EU). This review aimed to assess the adequacy of the EU’s centralized market authorization framework and alternative pathways, such as the Hospital Exemption (HE) and Compassionate Use Program (CUP), in addressing access disparities.
METHODS: We conducted a critical review of the literature, including peer-reviewed articles, policy documents, regulatory guidelines, and grey literature. Sources were selected based on thematic relevance to key ethical challenges: access, affordability, and geographical disparities.
RESULTS: Although the EU’s centralized framework guarantees high safety, quality, and efficacy standards, its implementation reveals substantial barriers. High costs create significant financial challenges for healthcare systems and individuals, disproportionately affecting low-income countries and regions. Geographic disparities, amplified by fragmented national regulations and uneven healthcare infrastructure, limit patient access, particularly in rural areas. Alternative pathways like HE and CUP aim to improve access but are hindered by inconsistent implementation at the national level
CONCLUSIONS: This review highlights the need for a more cohesive and inclusive EU regulatory framework to ensure equitable access to gene therapies. Addressing economic and geographical barriers is important to realize the full potential of these innovations for all EU citizens.