Hospital exemption: a challenging milestone for the Andalusian Public Healthcare System

Abstract

Background & Aim Advanced therapy medicinal products (ATMPs) are a particularly novel class of medicines, which include gene and cell therapies, and tissue engineered products. Patients can access to ATMPs through clinical trials (research phase), authorized market drugs, hospital exemption or exceptionally under compassionate use. To reach the marketing authorization for ATMPs it is needed to follow the centralised procedure stablished by the Regulation (EC) No 1394/2007. In addition, the article 28 of this Regulation concerning to “hospital exemption” was included to recognize the small scale and developmental nature of the activities carried out in some hospitals, which calls for a degree of flexibility in the nature of regulatory requirements. This article establishes the exemption as follow “ATMPs prepared on a non-routine basis according to specific quality standards, and used within the same Member State in a hospital under the exclusive professional responsibility of a medical practitioner, in order to comply with an individual medical prescription for a custom-made product for an individual patient”. In agreement with this article, the hospital exemption ensure the accessibility and sustainability of the Public Health Care System without the use of non-commercialize products. Methods, Results & Conclusion In Spain, according to Royal Degree 477/2014, Hospital exemption request must be submitted using the Common Technical Document (CTD) structure, consists of five modules where set of specifications for an application dossier for the registration of Medicines. In this sense, The Andalusian Network for the Design and Translation of Advanced Therapies (AND&TAT) which belongs to the Andalusian Health Care System and coordinate a network of 10 GMP facilities, has leading the Hospital exemptions submissions in several Andalusian Hospitals a challenging activity for an Academic Institution with limited resources. To date, three requests from three different Hospitals have been presented to Spanish medicine agency and are pending on some clarifications to be approved. In parallel, efforts are ongoing to submit another three dossiers which pretend to be presented in near future.