The proposal for a Regulation on the safety and quality of substances of human origin (SoHO) is another building block of the European Health Union. It will promote pooling resources and achieving economies of scale. Its ambition is to create more opportunities for patients across the EU to access the treatment they need independently of where they live. It will also facilitate cross-border circulation of these critical health therapies as well as cross-border cooperation between public health authorities and it reinforces solidarity, while ensuring the same harmonised high level standards of quality and safety for all SoHO.
Figures speak for themselves: SoHO-based treatments are of high value, they provide large numbers of life-saving and life-enhancing therapies every year (25 million units of blood transfused, e.g. for surgery or trauma care; 36,000 stem cell transplants for blood cancers), life-creating therapies (medically assisted reproduction treatments, e.g. 940,000 cycles of IVF), and life-improving therapies (e.g 14,500 cornea transplants for restoring sight, 2,000 skin transplants for burn wounds and other injuries).
What is being adopted by the Commission?
The Commission tables a comprehensive revision of the existing legislation to ensure safety and quality of blood, tissues, and cells (BTC).
The proposal updates and reinforces the rules for safety and quality and extends them to other substances of human origin (SoHO), such as human breast milk, which were previously left unregulated at EU level.
The result is a proposal for a future-proof and robust framework which better protects donors, recipients treated by transfusion, transplantation or medically assisted reproduction, and offspring born from donated sperm or eggs and that fosters innovation in this crucial biotech sector.
This proposal builds upon the existing legal framework, which came into force in 2002 for blood, and 2004 for tissues and cells, containing parallel provisions for donor selection, quality and safety management and oversight.
To improve harmonisation, ensuring a uniform level of protection across the EU and simplifying cross-border exchange and access of SoHO therapies, the Commission proposes to repeal the Directives and replace them by a single Regulation that will be equally applicable in all Member States.
While this Regulation will bring a high level of safety and quality standards, it still leaves Member States the possibility to add more stringent requirements, in particular to ensure alignment to the set-up of national healthcare systems.
Why did the legislation on BTC need to be updated?
Having been in place for almost 20 years, the current legislation on blood and on tissues and cells is now outdated and does not reflect the health and societal evolutions. New infectious disease risks have emerged in the meanwhile and the biotechnologies available for preparing blood, tissues, and cells for clinical use have developed significantly.
The Commission published an evaluation in 2019 which showed that the framework had increased safety and quality across the EU, but the evaluation also identified five key areas where the legal framework no longer reflected the current state of the art.
Following a wide consultation with stakeholders, both on the objectives of the revision and its content, and incorporating lessons learnt from the COVID-19 pandemic, the proposed Regulation includes measures tackling each of these problems.
It ensures that:
(1) technical rules for safety and quality are responsive to changing risks and technologies
(2) donors and offspring are protected
(3) harmonisation of oversight requirements is strengthened
(4) innovation is supported, and
(5) measures supporting supply resilience are introduced.
What are the major changes compared to the current legislation?
The proposal combines measures that apply to regulatory authorities, SoHO entities and establishments preparing therapies, as well as healthcare providers in the sector.
The proposal includes a range of new measures which fill some gaps and were developed to support the functioning of the whole sector:
- The proposed Regulation covers all substances of human origin (except for solid organs for transplantation, which remain regulated separately under a dedicated Directive). These include blood, tissues, and cells, as well as other substances such as human breast milk or microbiota which come with similar safety and quality concerns. Other substances of human origin that may, in the future, be applied to patients will automatically fall within the scope of this legislation;
- SoHO have to be prepared and treated according to specific standards to ensure they are safe for patients. In addition to protecting patients, such standards will now be extended to protect offspring born from medically assisted reproduction as well as donors, including 15 million blood donors, over 34,000 stem cell donors and over 39,000 egg donors every year;
- To ensure that these rules continually reflect scientific progress, they will mostly be developed by scientific expert bodies active in the sector. This means that new evidence can be included more rapidly than before, and safety requirements can be kept up to date (e.g. testing for infectious or non-infectious diseases);
- Any entity conducting activities that affect the safety and quality of SoHO will have to register with their competent authorities. When these activities include processing and storage of SoHO, they will have to fulfil additional requirements to be authorised and inspected as a SoHO establishment, similar to current requirements;
- Entities working with SoHO will be required to report their annual activity data. These data will allow Member States to implement measures to improve donation collection rates when needed. Entities working with critical SoHO will need to alert their authority in the case of sudden fall in supply and will be required to have emergency plans in place;
- Oversight requirements will be proportionate and risk-based. This way, protection of patients, donors, and offspring can be increased without creating undue burdens for competent authorities. In addition, the Commission will support joint oversight activities (for example in authorising new SoHO preparations) as well as provide training, ICT support tools and opportunities for exchanging best practices.
How does the proposal enhance the EU-wide dimension of policies in this area?
The EU aims at helping facilitate innovation and research as well as the cross-sector collaboration. To simplify the work of innovators in the SoHO sector, the framework includes an EU-wide procedure for the authorisation of SoHO preparations. This procedure extends the currently applicable rules for preparation process authorisations in the Tissue and Cell Directive to the blood field and introduces an up-front risk-assessment followed by clinical outcome data collection requirements proportionate to the identified risks. The approach was developed and tested in a Joint Action carried out by a large number Member States.
The proposal also includes the establishment of the SoHO Coordination Board (SCB) as an advisory body that will support Member States in the implementation of the Regulation. The SCB will develop common good practices for inspection and vigilance, provide advice on the applicability of the Regulation, and generally support the competent authorities in the oversight of the sector.
The SCB will also increase cross-sector coherence and legal clarity by liaising with expert bodies and corresponding bodies in other legal frameworks regarding any regulatory borderline issues that might arise.
Does the proposal keep some provisions of the old legislative framework?
The proposal includes some provisions that mirror the old framework. For instance, Member States retain the full competence on any organisational and ethical decisions concerning the provision of SoHO-based treatments in their healthcare systems.
In addition, the principle of voluntary and unpaid donation has been upheld and improved in the proposal.
How will this legislation affect the lives of the citizens?
This new legislation will improve access to safe and proven SoHO therapies, which is a critical part of the healthcare system in each of the EU Member States.
The availability of these therapies is dependent on the willingness of citizens to make donations and on the efforts in, mainly public, blood and transplant services to prepare and offer therapies at affordable costs. Furthermore, academic centres continuously develop and share innovations, which allow citizens to access the latest SoHO-therapies across the EU.
The proposal provides for a tailored regulatory framework, adapted to these particularities of the sector, allowing safe access to high quality therapies based on continuous technological developments.
The proposal improves the protection of all citizens directly affected by transfusion, transplantation, and assisted reproduction as well as those citizens that donate to allow for these treatments to be carried out. For example, follow-up after donating or receiving certain types of SoHO will be required, in some cases for longer periods, so that any adverse reactions can be monitored and reported.
Furthermore, the proposed Regulation will optimise access to innovative therapies by making the authorisation subject to an upfront risk-assessment, thus ensuring a high standard of care for patients.
The increased harmonisation will improve access as well, as it will facilitate the exchange of SoHO between Member States and hence optimise treatment options for all EU patients. New measures requiring activity data monitoring, supply alerts to authorities and the development of emergency plans will help Member States to take action when the supply of critical SoHO is threatened.
What will be the roles of the European Centre for Disease Prevention and Control (ECDC) and the European Directorate for the Quality and Medicines and HealthCare?
Leveraging the common European expertise is to the benefit of all Member States, further strengthening the European Health Union.
Technical rules need to reflect high-quality and up-to-date evidence in order to ensure the safety and quality of SoHO and the protection of donors, recipients, and offspring.
The proposal therefore incorporates the expertise of the European Centre for Disease Prevention and Control (ECDC) and the European Directorate for the Quality of Medicines and HealthCare (EDQM[1]) of the Council of Europe.
Both the ECDC and the EDQM have proven expertise in the sector, providing guidance on how to protect donors and patients treated with SoHO from communicable diseases (ECDC) or any other risks to quality and safety (EDQM).
This guidance is already widely applied in the sector but will now become the primary means to meet the EU standards of quality and safety for SoHO.
Both expert bodies will use their capacity and experience to adapt their guidance to scientific progress and frequently changing threats. Entities that choose to apply different technical approaches can do so when they can demonstrate that they achieve equivalent standards of safety and quality to those achieved by following the ECDC and EDQM technical guidelines.
Will there be a role for digitalisation in this new framework?
Yes. The proposed Regulation puts forward the creation of an EU SoHO Platform, a central digital tool for authorities and stakeholders to facilitate data exchange and administration in the sector.
It will be developed and hosted by the Commission to serve the user needs: competent authorities, SoHO entities and establishments (including healthcare providers), patients, donors, and offspring, expert bodies and the Commission.
It will provide a central hub for access to information on registrations, authorisations and technical guidelines.
The platform will also allow collection and publication of data on donations, clinical use and possible adverse reactions, under clear governance and in line with data protection. The platform will improve communication and transparency and increase efficiency in the sector and will pre-empt the need for Member States to establish multiple data platforms and information dissemination tools, while safeguarding the privacy of health data.
What are the next steps?
The proposal will now be discussed by the Council and the European Parliament. Once the final text is agreed and adopted, it will come into force with a two-year transition period before most provisions apply and a three-year period for some particular provisions.
The Commission will adopt a series of Acts implementing certain provisions, such as import and traceability, in more details.
For More Information
Press release: European Health Union: Stronger rules for greater safety and quality of blood, tissues, and cells
Proposal for a Regulation on the safety and quality of substances of human origin
[1] The European Directorate for the Quality of Medicines & HealthCare (EDQM) is a directorate of the Council of Europe (CoE), which has 46 Member States, including all EU Member States,
The origin of EDQM date back to 1964, when the Convention on the Elaboration of a European Pharmacopoeia was adopted by the Committee of Ministers of the CoE. To date, the Convention has been signed and ratified by 39 Member States of the Council of Europe, including the 27 EU Member States, and by the EU.
The EDQM is responsible for the development of technical rules for safety and quality of SoHO through dedicated expert groups, then adopted by its European Committees on Blood transfusion and Organ Transplantation (CD-P-TS and CD-P-TO). These committees are intergovernmental structures answerable to the Committee of Ministers of the CoE. Members eligible to these Committees are the Members that have signed the Convention on the Elaboration of a European Pharmacopoeia.
