December 16, 2024
ESGCT 2024: Exploring Hospital Exemption in ATMPs for Rare Diseases
The 31st European Society for Gene and Cell Therapies (ESGCT) Congress was held on 24-25 October 2024, in Rome, Italy. Johan Prévot, IPOPI’s Executive Director, took part in a panel session entitled “Towards the Accessibility of ATMPs for Rare and Ultra-rare Diseases with No Commercial Interest: Is there a role for Hospital Exemption?” moderated by Prof Claire Booth, UCL, London. Professor Alessandro Aiuti, SR-Tiget, Milan, provided an overview of challenges in ATMPs’ development and access to patients for rare and ultra-rare diseases, whilst Ms Paschalia Koufokotsiou, from the European Commission, went over the current meaning and legislation of Hospital Exemption (HE) in the EU. Sol Ruiz, Spanish Medicines Agency, discussed key standards for the harmonisation of HE across the EU.
Key points covered during the discussion included the fact that the use of the HE clause may constitute an alternative therapeutic approach for the treatment of rare and ultra-rare diseases which are not covered by the pharmaceutical industry. However, it was also highlighted that, currently, the application of HE in the different EU Member States is highly variable and used in different ways. The importance of facilitating the development of non-commercially viable ATMPs by academic institutions was also highlighted. It was recognised, however, that patients seeking HE treatment outside their home country face regulatory barriers. Harmonised frameworks and cross-border data sharing could help alleviate these issues. The importance of public-private partnerships was also highlighted by Mr Prévot as well as the importance of a well-regulated approach to ensure the safety and efficacy of ATMPs for patients. Still, on the issue of HE, the European Commission is currently finalising a report on the implementation of the concept of hospital exemptions in European Member States which will be published in Q1 2025.
IPOPI will continue to monitor developments and liaise with key stakeholders such as the AGORA foundation and the TRANSFORM alliance to ensure the EU provides a more optimal and harmonised framework for the regulatory pathway and the assessment of ATMPs, such as gene therapies, for the treatment of primary immunodeficiencies, including in the context of the revision of the EU pharmaceuticals’ legislation in the coming months.
