EU ATMP Hospital Exemption
• The Hospital Exemption has a legitimate role to play in meeting unmet patient needs. Equally, action should be taken to ensure that the use of the exemption does not undermine the rights of patients to safe and effective treatment.
• The Hospital Exemption should be interpreted and implemented in a strict and fully harmonised manner by all EU Member States, recognising that the exemption should only be applied in the absence of centrally authorised medicinal products. Commission guidance should support National Competent Authorities in achieving harmonisation.
• Measures should be adopted to improve transparency of the use of the Hospital Exemption, including though the establishment of a register of exempted products and the adoption of a harmonised Informed Consent Form.
• To safeguard incentives for innovation, steps should be taken to ensure the Hospital Exemption cannot be used an alternative route to market for developers of ATMPs.
EuropaBio calls on the European Commission to:
1) Clarify that the use of the HE should be restricted to the scenarios stipulated in the ATMP Regulation – for individual patients, within one Member State and on a non-routine basis. As regulatory exemptions should be interpreted conservatively by default, the Commission should make clear that any medicinal products approved pursuant to the ATMP Regulation should take precedence. There should be a harmonised strict interpretation and implementation by the EU Member States to recognise that the HE should only be applied in the absence of centrally authorised products.
2) Collaborate with the EMA, Heads of Medicines Agencies and National Competent Authorities, to provide clear harmonised guidance which ensures consistent interpretation and use of the HE across all Member States. This guidance should: • Provide an unambiguous and standardised definition of "non-routine" use and “within the same Member State”.
• Clarify how quality and PCV standards should be met.
• Make clear that Member States should ensure that GMP standards for HE products are equivalent to those for authorised ATMPs.
3) Work with National Competent Authorities and involve EU-funded programmes to establish a register of exempted medicinal products which monitors how these products are used and provides appropriate information relating to their safety and efficacy. Alternatively, National Competent Authorities should maintain a registry of sites, and require individual sites to contribute to a registry providing appropriate information relating to safety and efficacy. We recommend that there is a provision included within Commission guidance that ensures transparency relating to clinical data.
4) Work with National Competent Authorities to introduce a harmonised Informed Consent Form that must be completed prior to treatment for all exempted medicinal products.
5) Work with National Competent Authorities to ensure that the HE is not being used by developers of ATMPs as an alternative to the centralised approval pathway as set out in the ATMP Regulation.
Conclusion By putting forward these recommendations, EuropaBio and its members would like to engage the European Commission and all relevant stakeholders in a discussion on the future use of the HE in the context of the new EU Pharmaceutical Strategy and its accompanying actions.
Download the file below to read the full Position Paper.