European Society for Blood and Marrow Transplantation
EBMT
We are a community of healthcare professionals 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.
ID: 652992023103-09
Lobbying Activity
Response to Revision of the Union legislation on blood, tissues and cells
8 Sept 2022
EBMT welcomes the Commission proposal for an EU regulation on standards of quality and safety for substances of human origin intended for human application
Leiden, 8 September 2022
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.
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.
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EBMT shares the views expressed by the CoRe SoHO consortium, the World Marrow Donor Association, National Marrow Donor Program/Be The Match and ICCBBA.
Read full responseResponse to A European Health Data Space
3 Feb 2021
The European Society for Blood & Marrow Transplantation (EBMT) warmly welcomes the EU's EHDS strategy. As the largest registry holder of its kind in Europe (>700k transplants reported), EBMT is rightly recognised as a key resource for not just scientific research in the field of cell therapy but for regulatory and other purposes. In 2019, EMA issued a positive qualification opinion on using the EBMT registry to support long-term follow-up of patients who received innovative cellular therapies for blood cancers. This in turn has led to EBMT entering into agreements with pharma to undertake long-term follow-up of authorised products in Post-Authorisation Safety Studies (PASS) based on secondary use of registry data. This experience has given us an insight into some of the challenges identified in this inception impact assessment.
Data are the foundation of policy decision making and scientific progress. A successful EHDS would address the principle question at stake which is access to data. It would substantially facilitate collaborative research ("Open Science") and regulatory oversight. It would minimise the data reporting burden currently borne by healthcare professionals and would lead to harmonisation of local interpretations of GDPR. It would do all of the above within a data protection framework that safeguarded the interests of the patient.
We agree with the problems already identified and would add the following for consideration:
- EHDS will also have to address data sharing with entities outside of the EU - science is international;
- There is a need for a clear, single interpretation of what is understood by primary and secondary use of data, interpretations in clinical study setting may vary from interpretations in other settings, moreover this definition has implications on study conduct and reporting obligations under GVP;
- Achieving successful data sharing will not bring any benefits unless it is combined with agreement on study requirements across Member States particularly for non-interventional studies. Without this, the data will not be usable for research;
- EU-level data protection bodies like EDPS and EDPB can only issue recommendations which Member States are not obliged to incorporate;
- Data governance - data subject rights and consent on who, how, when, for how long etc needs currently vary greatly and an agreed definition is needed for the lawful basis for collection and use. Other challenges to sharing data include proprietary, financial and fairness concerns about sharing data, particularly when involving companies. Questions like what safeguards will be implemented, what are the access criteria, how can we ensure that the costs and benefits of collecting, storing and using data are fairly distributed will need to be considered.
- Technology - interoperability remains a substantial obstacle which will need decisions to be taken on common data model standards so that data sources like registries can prepare themselves for the future;
- Sustainability of key clinical data sources are often dependent on non-for-profit organisations
- Fine-grained health data, particularly for rare diseases and/or rare treatments is often personally identifiable and can be difficult to anonymise and this could be more challenging where there is a need for linking different data sources and avoiding duplication of data from different sources.
Concerns around commercialisation of health data remain and even the terminology around a “European data market" are somewhat disquieting at a time when the EU itself is concerned about the influence of ‘Big Tech’. Such proposals should be carefully framed to reflect citizens’ and healthcare professionals’ concerns in this regard.
Read full responseResponse to Revision of the Union legislation on blood, tissues and cells
13 Dec 2020
EBMT agrees with the problems to be addressed as set out in Part A and highlights the following:
1. EBMT supports all efforts to minimise variability in how the framework is implemented at national level. Haematopoietic stem cells (HSC) cross national borders and these unique life-saving substances are vulnerable to regulatory delays or obstacles.
2. EBMT strongly supports the use of outcome data as an indicator of satefy and quality of SoHO and encourages the Commission to complement existing data frameworks rather than ‘reinventing the wheel’. The work of the GAPP Joint Action should be adopted and built upon and technology considered within the EU Health Data Space. Appropriate data protection and sharing measures also need to be considered as the current GDPR framework, while acknowledging the need for protection, also presents significant challenges to the reutilisation of such data for scientific or regulatory uses.
3. EBMT also agrees that the SoHO field is constantly progressing which requires a more agile framework to be able to respond to changes in science and practice. SoHO are likely to see growing applications as medicinal products as technology and research evolve e.g. stem cells for CAR-T cell therapy cross from the Tissues and Cells framework to the Medicine framework.
4. EBMT supports the principle of voluntary unpaid donation (VUD). The HSC sector has a long-established and succesful unrelated donor model which now has almost 40 million donors worldwide. Candidates to be donors come forward out of altruistic motives and any move towards commercialisation of this key process risks coercion and exploitation of donors, increased risk to patients, harm to local transplantation programs and international stem cell exchange, and the possibility of benefiting some patients while disadvantaging others.
5. EBMT agrees with the assertion that SoHO are not commodities. They should be legislated for at EU level based on public health principles and not on internal market ones irrespective of its end use. Commoditisation of substances could lead to EU citizens having differing access to healthcare depending on where they live and/or their income levels.
Policy options
Policy option 1 appears to be a step backwards to a potentially heterogeneous landscape and away from a common EU framework.
Policy option 3 is similar to the existing legislative framework which has been recognised as valuable but too rigid to be responsive to changes in science and practice.
Combining policy options would require very robust definitions. A single BTC framework will need to carefully assess the feasibility of using a common approach or whether different substances will require different rules.
EBMT therefore supports Policy option 2 with the following areas to be clarified:
1. The responsibilities of ECDC and EDQM must be very well defined if their outputs are expected to carry more weight.
2. Strengthening oversight and audit by the EC are welcomed as per option 1 but must be realistic and performed by experts in the field. Existing EU initiatives in this regard e.g. VISTART should also be built on and the professional associations could also be approached in this regard.
3. EBMT supports the inclusion of novel substances of human origin in the BTC framework with clear criteria whilst recognising the need for medicinal product oversight where applicable. Classifications should be make through a transparent multi-stakeholder process.
4. EBMT welcomes the proposal to strengthen donor vigilance, especially reporting adverse reactions and donor data capture including follow-up should be supported. The EC should capitalise on EU initiatives like ARTHIQS and TRANSPOSE as well as the expert input of the professional associations.
On the preliminary assessment of expected impacts
EBMT supports the analysis on likely economic and social impacts. Regarding fundamental rights, legislators must take into account Article 35 of the Charter
Read full response