Hospital Exemption in Italy: technical-scientific and regulatory aspects in comparison with Europe

Hospital Exemption (HE) represents a regulatory tool that allows the use of advanced therapy medicinal products (ATMPs) prepared on an individual basis for the treatment of patients with an unmet medical need and without viable therapeutic alternatives. However, the regulatory framework governing this tool varies significantly among European countries while providing accelerated access to such treatments. This article aims to analyze the applications for treatment with ATMPs prepared in a non-repetitive modality over the 10-year period of 2015 to 2025, focusing on the proposed therapies and the clinical outcomes achieved. The data reviewed concern ATMPs authorized for individual patients by the Italian Medicines Agency on the basis of the opinion regarding suitability for use issued by the Phase I Committee established at the Istituto Superiore di Sanità, the Italian scientific technical body that supports the national health care system. Particular attention is given to the critical challenges encountered in the administration of the HE pathway in the 10 years since the regulation came into effect. In a European context of limited regulatory harmonization, this article explores issues currently being discussed in European regulatory boards and anticipates the implementation of some items, including a monitoring system. It highlights the main operational challenges in HE practices and proposes management solutions to optimize their implementation. Such solutions include the creation of a centralized monitoring database for tracking patients, medical conditions, and products used.