The EBMT is a community of hospitals and healthcare professionals specialised in stem cell transplantation and cellular therapies. We are focused on innovation, research and the advancement of cellular and stem cell-based therapies to save and improve the lives of patients with blood-related disorders.
EBMT welcomes the Commission proposal for an EU regulation on standards of quality and safety for substances of human origin (SoHO) intended for human application, published on 14th July. Further information here: https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:52022PC0338&from=EN
We particularly welcome the introduction of the joint regulation approach. The implementation of a single Regulation that will directly apply in all Member States (MS) will promote harmonisation across MS and remove barriers to exchange of life-saving SoHOs. Leveraging European expert bodies to develop and maintain technical guidelines will give the framework the agility required to respond to changes in science and practice to ensure safety, quality and efficacy of SoHOs while promoting innovation in the sector. The extension of scope to include other SoHOs, like faecal microbiota, is another important part of future-proofing the SoHO framework.
We appreciate that the principle of voluntary unpaid donations has been maintained and that donor protection measures have been strengthened. To prevent that donors are exposed to avoidable risks, EBMT supports that donors’ health is monitored before, during and after donation and that donor health evaluations including data on Serious Adverse Occurrences (SAO) are registered. However, we request that clarification be included that SAOs are reportable to authorities only when they may be attributed to the SoHO activity and when they are unexpected. This would avoid reporting of SOA when they are known to be a result of the treatment of the SoHO recipient, such as Graft versus Host Disease, in order not to unduly burden SoHO entities.
We also welcome the establishment of a SoHO Coordination Board that will contribute to consistent implementation of the Regulation across MS and will improve interplay where SoHO become starting materials for ATMPs and other therapies manufactured under different legal frameworks, thereby contributing to a regulatory environment that enables the development of curative approaches to serious and frequently fatal diseases. As one of the leading medical and scientific organisations for cellular and stem cell-based therapies, EBMT supports a harmonised risk-assessment based authorisation process and clinical outcome monitoring proportionate to the identified risks to contribute to improving the lives and quality of care of patients with blood-related disorders. We also welcome Article 22(5) which allows for the use of verified existing clinical registries, thus avoiding duplication and reducing administrative burden for healthcare professionals. To ensure accurate reporting, adequate follow up and vigilance, EBMT encourages the Commission and MS authorities to exploit existing international consensus standards on terminology, definitions and robust traceability of SoHOs.
We also support Article 29(10) that allows authorities to take into account results from accreditation by international bodies. We believe that leveraging existing accreditation schemes such as JACIE will avoid duplication of inspection workload for facilities while not compromising on safety and quality.
Finally, EBMT looks forward to contributing to the ongoing legislative process as well as the implementation of the final Regulation and the development and update of standards to ensure the quality, safety and efficacy of SoHOs.
EBMT shares the views expressed by the CoRe SoHO consortium, the World Marrow Donor Association, National Marrow Donor Program/Be The Match and ICCBBA.