Explanatory Memorandum to COM(2022)338 - Standards of quality and safety for substances of human origin intended for human application - Main contents
Please note
This page contains a limited version of this dossier in the EU Monitor.
| dossier | COM(2022)338 - Standards of quality and safety for substances of human origin intended for human application. |
|---|---|
| source | COM(2022)338  |
| date | 14-07-2022 |
1. CONTEXT OF THE PROPOSAL
• Reasons for and objectives of the proposal
The Blood Directive - 2002/98/EC 1 and the Tissues and Cells Directive - 2004/23/EC 2 (the BTC legislation) have helped ensure the safety of millions of patients undergoing blood transfusion, transplantation and medically assisted reproduction. The legislation sets out quality and safety requirements for all activities from donation to human application (unless the donations are used to manufacture medicinal products or medical devices, in which case the legislation only applies to donation, collection and testing).
Every year, EU patients are treated with 25 million blood transfusions (during surgery emergency, cancer or other care), a million cycles of medically assisted reproduction, over 35,000 transplants of stem cells (mainly for blood cancers) and hundred thousands of replacement tissues (e.g., for orthopaedic, skin, cardiac or eye problems). These therapies are only available thanks to the willingness of fellow citizens to make altruistic donations.
In the European Union, the collection, processing and supply of each individual unit is typically organised on a local small-scale by public services, (academic) hospitals and non-profit actors.
After almost 20 years in place, the legislation no longer addresses the scientific and technical state of the art and needs to be updated to take into account developments that have taken place in the sector. While an evaluation of the BTC legislation 3 confirmed that it has brought very good levels of overall safety and quality in these sectors (less than one serious patient reaction for every 12,000 applications), shortcomings of the legislation were identified as follows:
• Patients are not fully protected from avoidable risks due to out-of-date technical rules;
• Blood, tissues and cells (BTC) donors and children born from donated eggs, sperm or embryos (offspring) are exposed to avoidable risks;
• Member States have divergent approaches to oversight that hampers cross-border exchanges of BTC;
• Full potential of BTC processed or used in new ways is not reached for patients;
• Patients are vulnerable to interruptions in EU supply of BTC.
The COVID-19 pandemic highlighted some of these shortcomings, in particular, those impacting on rules for preventing the risk of disease transmission by BTC and the lack of measures to ensure sufficiency of supply. The proposal aims to address these shortcomings, by revising the current legislation. The overall objective is to ensure a high level of health protection for EU citizens and ensure they have access to safe and effective BTC. As new technologies or risks will continue to emerge, it is desirable that the future framework is more effectively implemented, future proof, crisis resistant and agile enough to accommodate new risks and trends while continuing to provide appropriate safety and quality requirements. Being a REFIT initiative, areas for improving the efficiency of the legislation and simplifying its implementation by all stakeholders were also explored.
• Consistency with existing provisions in the policy area
The EU framework for safety and quality of substances of human origin (SoHOs) has currently three main Directives, respectively for Blood, Tissues and Cells, and Organs, together with implementing legislation. Each Directive sets safety and quality standards for all the steps from donation and collection from a donor body, over testing, processing, storage and distribution, to eventual application in the patients’ body. The current proposal covers blood, tissues and cells, and has links with the Organs Directive 4 , in particular regarding closer collaboration between Member States’ competent authorities for blood, tissues and cells, and those for organs, and regarding vigilance requirements.
Where BTC can be used in the manufacture of health products that are regulated by other Union legislation, or as the starting and raw material thereof, the SoHO framework applies on the first activities in the chain (donation, collection, testing) while these later activities (manufacturing, storage, distribution etc.) are regulated under these other appropriate legislative frameworks (e.g. medicinal products, including advanced therapy medicinal products, or medical devices) 5 . There are in place some mechanisms to ensure coherence between the BTC legislation and those adjacent frameworks. This proposal will reinforce the cooperation among those adjacent frameworks.
As part of the Pharmaceutical Strategy for Europe , there is an ongoing evaluation and revision of the pharmaceutical legal framework 6 . This proposal will feed into that work, in particular regarding the regulatory delineation between the BTC sector and the pharmaceutical sector. The delineating criteria are set by definitions in the pharmaceutical framework and are not altered by this proposal.
• Consistency with other Union policies
This initiative is part of the EU’s ambition to build a stronger European Health Union, so as to: (1) better protect the health of our citizens (including patients, donors and offspring); (2) equip the EU and its Member States to better prevent and address future pandemics (surveillance, data analysis, risk assessment, early warning and response) and (3) improve the resilience of EU health systems (sufficient supply of SoHOs).
The proposal further establishes links with the European Centre for Disease Prevention and Control (ECDC), for which the mandate has been proposed to be strengthened 7 , also in this field of SoHOs.
2. LEGAL BASIS, SUBSIDIARITY AND PROPORTIONALITY
• Legal basis
The SoHO legislation is based on Article 168(4)(a) of the Treaty on the Functioning of the European Union (TFEU). As a shared competence with the Member States, and in line with the principle of subsidiarity, this Treaty Article gives the EU a mandate to set out measures establishing high standards of quality and safety for SoHOs while allowing Member States to maintain or introduce more stringent protective measures. Member States remain responsible for decisions of an ethical and organisational nature, such as allowing the donation of certain SoHOs or deciding allocation of certain SoHOs or who may access certain SoHO therapies (e.g., access to in vitro fertilisation therapies). While the EU Charter on Fundamental Rights requires non-commercialisation of the human body which translates into a principle of voluntary unpaid donation in the EU legislation, it is for the Member States to define the detailed implementation of this principle within the context of each country. When a Member State chooses to allow a particular new practice that can bring ethical questions (such as testing or storage of embryos), the safety and quality of this practice are then regulated by the EU SoHO legislation.
• Subsidiarity (for non-exclusive competence)
Ever-evolving disease threats, such as Zika, Human Immunodeficiency Virus (HIV) or viral hepatitis B, C and D, which can be transmitted through SoHOs, constitute cross-border threats to public health. In addition, the exchange of SoHOs between Member States and with third countries is necessary for ensuring optimal patient access and sufficiency of supply. This is in particular the case for SoHOs that are used as personalised therapies where it is essential that a recipient is specifically matched with a donor. Increasing cross-border exchanges of SoHOs necessitate ever-closer cooperation between a number of health professional groups and authorities to ensure that SoHOs remain traceable from the donor to the recipient and vice versa.
Certain types of sector-specific expertise may also not be easily available in every Member State.
By providing a framework for cross-border cooperation, based on a common set of rules, and connected to sector-specific expertise, EU-level measures are best placed to address such issues effectively. Establishing high standards of quality and safety for SoHOs at EU level facilitates equal access to safe therapies for all EU citizens, and encourages circulation of SoHO materials and products between Member States. Providing for a common framework that supports joint practices will promote simplification and efficiency.
• Proportionality
The overall initiative is limited to aspects that Member States cannot achieve satisfactorily on their own, and where there is clear EU added value. Many of the pursued objectives can only be met through highly technical rules and guidance that require specific expertise for their regular updating. From the three policy options considered (see the impact assessment staff working document, section 5.2), the preferred option requires that blood and tissue establishments meet safety and quality standards by following guidelines developed and updated by nominated expert bodies such as the ECDC and the European Directorate for the Quality of Medicines & HealthCare (EDQM, a Directorate of the Council of Europe). This option provides the highest effectiveness and efficiency, avoids the need for re-developing guidelines, and can ensure a high level of harmonisation as well as rapid updating of standards.
The key added value of the EU approach in this proposal is to ensure, where appropriate, that common standards and guidelines make full use of the high level of latest scientific and technical expertise, as already available in expert bodies such as the ECDC and the EDQM, and therewith facilitate cross-border exchange of and access to safe SoHOs. In addition, the sharing of data through a common platform, and following common guidance, will enable policy making based on significantly more robust data.
As stated in the impact assessment staff working document (section 7.5), the proposal does not interfere with the right for Member States to maintain and introduce more stringent measures when they consider them necessary (Article 168 i of the TFEU) but does increase the level of safety and quality to be achieved in all Member States, thus reducing the need in most cases for more stringent measures that can create barriers to cross-border exchange and to patient access. In addition, the proposal will ensure the adoption of more stringent measures is made more visible so that exchanges can be more easily organised in full respect of those measures. Given that rules relating to ethical aspects of this field, or to healthcare organisation, are not included in the proposal, no special circumstances in individual Member States were identified that required a particular territorial variation in the measures to be applied.
• Choice of the instrument
The proposal takes the form of a new Regulation repealing two existing basic Acts, both Directives. A key element of the proposal is to establish more harmonised measures for Member States and organisations involved in collection, testing, processing, distribution, application of SoHOs, from donors to patients. Insufficient minimum harmonisation was identified as a key reason for reduced trust between Member States, resulting in reduced cross-border exchange and sub-optimal access for patients to SoHOs. A Regulation is considered the most suitable instrument since it does not require transposition and is directly applicable.
3. RESULTS OF EX-POST EVALUATIONS, STAKEHOLDER CONSULTATIONS AND IMPACT ASSESSMENTS
• Ex-post evaluations/fitness checks of existing legislation
The evaluation of the BTC legislation, published in 2019, identified the following gaps and shortcomings:
1. Patients are not fully protected from avoidable risks: the EU safety and quality requirements have not kept up to date with frequently changing scientific and epidemiological developments thus potentially exposing patients treated with BTC to avoidable risks. The ECDC provides up-to-date but non-binding guidance on safety measures, e.g. to address COVID-19 risks. The EDQM provides guidance on quality of BTC and many Member States put more stringent requirements in place. This situation can create legal confusion and unequal levels of safety and quality for patients. In addition, while new therapies have emerged since the BTC legislation was adopted, it is not always clear whether, and if so which, of the BTC Directives apply, leaving these substances unregulated or regulated in divergent ways (e.g., breast milk and faecal microbiota transplants). Some of these SoHOs do not meet the definitions of blood, tissues and cells included in the current legislation.
2. Divergent approaches to oversight cause unequal levels of safety and quality and barriers to the exchange of BTC across the EU: divergent national interpretations and implementations of the legislation lead to unequal protection and a lack of mutual trust between national authorities. This in turn creates barriers to cross-border exchange and to availability of BTC. These differences reflect the lack of common provisions for verification of effective implementation of inspection, authorisation and vigilance, and inconsistency in the levels of capacities, skills and independence required of inspectors supervising BTC establishments.
3. BTC donors and offspring (including children born from donated eggs, sperm or embryos) are exposed to avoidable risks: the current BTC legislation contains only very limited measures to protect and monitor BTC donors and offspring from donated sperm, eggs or embryos. In particular, the requirements to report donor adverse reactions are too limited and provisions for testing egg and sperm donors for genetic conditions are out of date with the technology available. Growing demand by commercial companies (e.g. egg banks for in vitro fertilisation, plasma collectors for medicinal product manufacture) increases the pressure to donate and consequently the need for robust donor protection measures.
4. The BTC legislation lags behind innovation: new ways of processing donations in BTC establishments may bring significant benefits. However, these new therapies can also put patients at risk, as current authorisation procedures for new BTC processes do not require evidence that risk is justified by benefits. Moreover, this lack of adequate procedures does not inspire trust and prevent healthcare actors from developing and adopting innovative processes. Besides risks and benefits, safety and quality measures also need to take account of the typical economic (public/non-profit) settings where BTC are developed and prepared, and of the often incremental and open-access nature of these innovations. In addition, there are sometimes difficulties in defining the borderlines for novel BTC with other regulatory frameworks, in particular where medicinal products and medical devices are concerned. This creates administrative burdens and implicit disincentives for BTC establishments, healthcare professionals and academia to innovate. This legal uncertainty issue requires further evidence gathering to allow its extent and consequences to be fully assessed.
5. The EU is vulnerable to interruptions in supply of some BTC: for some essential BTC, the EU is highly dependent on imports to ensure sufficiency. In particular, the EU relies on the United States for an adequate supply of plasma used to manufacture plasma-derived medicines. In the current legislation, sufficiency of supply through voluntary unpaid donation is encouraged, though without concrete measures to protect or increase supply. This approach has not proven adequate to protect EU patients from the risk of shortages or sudden supply disruption. The lack of EU and national monitoring provisions for the supply of BTC makes it difficult to predict EU supply interruptions and to take action to mitigate the risks to patients.
The proposal therefore provides measures to:
1. Ensure safety and quality for patients treated with SoHO therapies and fully protect them from avoidable risks linked to SoHOs.
2. Ensure safety and quality for SoHO donors and for children born from donated eggs, sperm or embryos.
3. Strengthen and allow for harmonisation of oversight practices among Member States.
4. Facilitate the development of safe and effective innovative SoHO therapies.
5. Improve the resilience of the sector, mitigating risk of shortages.
Objectives 1 and 2 are closely linked, as they both involve setting technical requirements for safety and quality to better protect EU citizens. While the EU has no mandate to intervene directly in supply management, reliable monitoring and notification of shortages would help Member States detect sudden drops in supply of SoHOs, trends towards shortages or dependencies on other Member States or on third countries, and would help them to take appropriate mitigation actions.
• Stakeholder consultations
Stakeholder consultations were a key step in the impact assessment phase for the revision of the legislative framework on blood, tissues, and cells. Consultation activities aimed to assess stakeholders’ views and opinions on (i) the validity of the findings of the evaluation (2019) 8 , (ii) the three proposed policy options described in an Inception Impact Assessment (IIA) 9 and (iii) the extent to which they would address the shortcomings identified in the evaluation, and their likely impacts.
Stakeholders were consulted via (i) the IIA publication for feedback, (ii) online surveys and questionnaires, (iii) hearings and participatory workshops with national competent authorities and stakeholders, (iv) bilateral meetings with stakeholder organisations and (v) interviews with specific stakeholders.
Overall, there is support among all stakeholders for the common measures proposed (revised legislation that allows for up-to-date technical guidance and fills existing legal gaps; strengthened oversight practices,; mechanism for legal advice whether and what SoHO requirements apply to a substance, if needed in coordination with other EU legal frameworks; tailored authorisation of SoHOs used or processed in new ways; and crisis preparedness and management). Stakeholders also expressed broad support for option 2 (technical rules set by expert bodies), which is seen as the most effective approach. Analysis of quantitative data from the public consultations confirmed that the degree of conflict among stakeholders was low, as this preference was widely agreed between all stakeholder categories. However, sector experts and national authorities also highlighted conditions that must be met for the option 2 approach to be a success, including the need for transparent drafting procedures that ensure that professionals as well as Member States can provide inputs, the need to allow for more stringent national requirements and the need to accommodate for geographic differences between the EU and the Council of Europe.
In addition, the analysis of respondents to the consultations highlighted once again the strong links between the blood sector on the one hand and the tissue and cell sector on the other, thus supporting the decision to combine both Directives into a single legal act on SoHOs (Organs being excluded).
Donors and patients as well as ethics bodies raised important points to be taken into consideration during the implementation phase of the new legal framework, regarding for example donor protection, voluntary unpaid donation principles or the use of new training opportunities for inspectors about fundamental rights (to further enable them to ensure that those are respected by establishments, and in particular non-discrimination of donors).
Many stakeholders also underlined the lack of legal clarity on the delineation with other EU legal frameworks (in particular medicinal products, including advanced therapy medicinal products, and medical devices) and indicated many cases where they believe SoHO-based therapies are not appropriately regulated, including some cases with a negative impact on feasibility to supply and eventual patient access. Stakeholders considered that the eventual choice for the most appropriate legal framework should aim in first place to ensure safety and quality, but also take account of the setting of, the costs for and the feasibility of providing access to safe and effective therapies. There was broad support for a dedicated SoHO legal advisory mechanism and for efficient coordination with advisory mechanisms in other sectors. It was largely considered that this will allow to strengthen legal clarity as well as improve interplay where SoHOs become starting materials for therapies manufactured under these other legal frameworks.
Also, while supportive, stakeholders pointed out that EU-level supply monitoring and crisis preparedness measures will demand significant efforts, without having any direct impact on the risk of shortages of critical SoHO. Finally, National Competent Authorities (NCAs) and blood and tissue establishments expressed concerns regarding some specific measures that would increase their costs or administrative burden. Consideration of EU level measures to provide support was taken into account when preparing the legal proposal.
An overview of the activities carried out and the results is available in Annexes 2 and 18 of the impact assessment staff working document.
• Collection and use of expertise
The Commission used the findings from the evaluation of the BTC legislation (2019). The impact assessment has been based on research and analyses done by the Commission. The Commission also contracted two external, independent consultant teams, to perform:
–A study, carried out by ICF S.A. supporting the impact assessment of the policy options, which gathered information on impacts and costs for stakeholders of the proposed measures and options, and further documented borderline case studies. The study also organised participatory workshops bringing stakeholders together to discuss various topics. The study was guided by a steering group composed of three senior experts in the field of blood, tissues and cells, who supervised the process and validated the study findings. The external support study will be published alongside this proposal.
–A feasibility study, carried out by Deloitte, focusing specifically on the costs, benefits and optimal approaches to the digitalisation of the sector. The preliminary report from that study is published as Annex 19 of the impact assessment staff working document.
Many of the 448 references in the BTC legislation evaluation were articles published in scientific journals and included data and evidence that was still relevant for the impact assessment of the policy options. In addition, a number of further scientific articles were published more recently and were also used as evidence sources for the impact assessment. These represent high quality evidence, due to the peer review process used by the publishers.
Evidence on costs is particularly difficult to gather in the SoHO sector, due to the predominant role of public sector organisations (public administrations, hospitals), where real costs related to SoHO activities are sometimes absorbed in overall hospital or institutional budgets. This explains the high variety of costs reported through the survey performed by the external study supporting the impact assessment, with NCAs and professionals. Sector experts, both from NCAs and from public establishments, were therefore brought together to identify and agree on reasonable average values and validate key assumptions that have been used to calculate costs.
The identified impacts of the proposed policy measures were subjected to a multi-criteria decision analysis to compare the effectiveness and efficiency of the options. To that end, the impact assessment piloted the tool developed by the Joint Research Centre of the European Commission, SOCRATES (SOcial multi-CRiteria AssessmenT of European policieS), using it to compare the different options based on previously established criteria.
• Impact assessments
The impact assessment analysed three policy options for the setting of safety and quality standards:
–Option 1 - Decentralised regulation: it provides blood and tissue establishments with the freedom to make reference to a variety of national and international guidance when conducting risk assessments of their own activities, with a view to setting their internal technical methods.
–Option 2 - Joint regulation: it requires blood and tissue establishments to follow the technical guideline developed and updated by nominated expert bodies.
–Option 3 - Central Regulation: it requires blood and tissue establishments to follow the safety and quality standards, all provided for in EU legislation.
The preferred option is Option 2. Joint regulation brings the highest effectiveness and efficiency as it builds on established expertise in the field of SoHOs to ensure that up-to-date standards are applied across the EU. Option 1 would allow changes to standards to be implemented more quickly, but with a high level of variation across the EU and a high workload on small blood and tissue establishments. Option 3 would allow for the highest level of harmonisation, but would require more time to adapt standards and bring additional costs for EU institutions.
This proposal therefore introduces high level standards in the legislative text for patient, donor and offspring protection and empowers the Commission to adopt implementing acts on the implementation of those standards if needed. Where there are no such implementing acts, professionals should, to meet these standards, apply safety and quality guidelines developed by the EDQM and the ECDC, in line with option 2. However, in line with option 1, it can also be acceptable to apply other, equivalent, guidelines accepted by national authorities and demonstrated as achieving equivalent standards of safety and quality. In the absence of a technical guideline from expert bodies, establishments can set their own technical method taking into account internationally recognised standards, scientific evidence and a documented risk assessment. This approach will facilitate an efficient and responsive implementation of safety and quality standards whenever risks and technologies change. It is proportionate in that it ensures EU legislation would be adopted for the implementation of a particular standard only when necessary and when it adds EU value (option 3).
In addition, a series of common measures was assessed, in particular to fill some legal gaps in the BTC framework, strengthen oversight, facilitate innovation, through advice on when the SoHO legislation is applicable and a (risk-) proportionate authorisation pathway for new processes, and manage SoHOs supply (crisis). The implementation of some of these common measures will be supported by guidelines from expert bodies (option 2).
Regarding the measure on establishing a SoHO advisory mechanism, this proposal does not propose any changes in the delineation of the borderline with the legal frameworks for pharmaceuticals or medical devices. The delineating criteria are defined in those other frameworks, in particular in Article 1 of Regulation (EU) 2017/745 (‘devices manufactured utilising derivatives of tissues or cells of human origin which are non-viable’), in Article 2 of Directive 2001/83/EC on medicinal products (‘intended to be placed on the market’ and ‘prepared industrially’), and consequently in Article 2 of Regulation (EC) No 1394/2007 on Advanced Therapy Medicinal Products (‘substantial manipulation’ and ‘not intended to be used for the same essential function’). This measure will rather facilitate coordination with established (or future) advisory mechanisms in those other frameworks.
The preferred option would ensure that citizens are better protected when donating, or being treated with a substance of human origin, with more harmonised and up-to-date safety and quality rules across the EU. In addition, offspring from medically assisted reproduction will be better protected, as will patients treated with currently unregulated SoHOs (e.g. therapeutic use of donated breast milk or bedside preparations of SoHOs).
The preferred option would bring a positive impact for professionals, in particular in blood and tissue establishments, when working with SoHO. Outdated, and sometimes costly, technical rules for safety and quality will be removed and replaced with standards based on the best scientific evidence and expertise available that will be updated in a timely manner (bringing efficiencies for establishments). The preferred option also allows for guidance to make common measures more efficient: a clear and (risk-) proportionate pathway will facilitate access to SoHOs prepared or used in new ways and supply-crisis management will be better aligned and coordinated.
The common measures will also strengthen oversight by competent authorities, by bringing in principles and new or more-efficient practices (e.g., joint inspections). Authorities will also benefit from more proportionate measures (e.g., risk based inspections) and EU level support (e.g. a digital platform, EU auditing of oversight systems, EU training courses for authority personnel). These measures will increase mutual trust and facilitate the collaboration between Member States, which should ultimately facilitate the cross-border exchanges of SoHOs, hence patient access.
Digitalisation will allow for further efficiencies in administrative processes, and the possibility for sharing of information will limit duplication of work across Member States.
The main costs relate to monitoring measures (donor, offspring, supply), to registration of bedside preparations of SoHOs and to the risk-proportionate pathway to authorise SoHOs processed or used in new ways. Those costs mostly fall on professionals in blood and tissue establishments, hospitals and clinics and, to a lesser degree, on competent authorities. EU measures can be considered to offset these costs for professionals and authorities, in particular in the adjustment phase and in particular through supporting digitalisation.
For EU institutions, the set-up of a common IT platform (the EU SoHO Platform) brings a significant cost, but will allow for a lightening of the (administrative) burden for national authorities and professionals. Further EU costs relate to coordination and co-funding of expert bodies.
• Regulatory fitness and simplification
This initiative is part of the Commission Work Programme 2021 under Annex II (REFIT initiatives).
The revision of the BTC legislation, with an approach proportionate to risks in different areas (authorisation or registration of establishments/entities, authorisation of new preparation processes, health monitoring of certain SoHO donors and offspring), brings opportunities for savings in the sector, and for carrying out some activities more efficiently with the same resources (e.g. risk-based inspections), though these opportunities have not always been fully quantified. The table below gives an overview of the main opportunities under the preferred option.
| REFIT Cost Savings – Preferred Option | ||
| Description | Amount | Comments |
| Graded oversight approach allows to oversee some establishments with lighter approach and less resources than today | EUR 4 m | 750 establishments eligible 10 , mainly saving on inspection costs for authorities and for themselves |
| Common IT-platform to share assessments of new SoHOs technologies reduces duplications | >EUR 2 m | Conservative estimate; Requests to authorise same new technologies are introduced and assessed in parallel across EU; Sensitive to unit cost of assessments and authorisations |
| Risk-based schedule allows to inspect same activities/establishments more efficiently (targeting high-risk activities) | Not quantified | Model has rather assumed this to be a cost-neutral measure as the same number of resources (inspectors) allow for more oversight on most complex activities |
| Recognition of authorisations of importing tissue establishments in other Member States, reduces need for ad-hoc import authorisations in different Member States | EUR 0.5 m | Applicable for almost 1 000 imports per year of blood stem cells (from bone marrow or peripheral blood) through a central registry (World Marrow Donor Association registry, subject to one joint authorisation) |
| Removing obsolete tests and systematic screening measures from the legislation | EUR 2 m (example – West Nile Virus NAT tests 11 ) | Very high potential, given that every saving is multiplied by number of donations. Other examples could be the screening for tattoos/piercings or testing for syphilis. |
| Digitalisation allows for more efficient administrative processes in authorities and establishments | To be further quantified | The EU SoHO Platform, financed by the Commission, will facilitate local administration including registration and reporting by professionals as well as authorisations and oversight by authorities. E.g. annual reporting costs are estimated to go down from current EUR 5 000-15 000 to EUR 200-2 000 with an automated reporting tool. |
Digital impacts are also expected, as data in the SoHO sector can become valuable digital assets in the areas of public health and innovation. A single IT system will bring important benefits as it can host flexible solutions, allowing Member States and establishments to maintain and connect with their own system or re-use existing components. It could become an important node in the EU digital ecosystem, and in particular in the future European Health Data Space (EHDS), which aims at opening opportunities and removing barriers to the use and reuse of health data, for the provision of healthcare, personalised medicine, research and innovation, policy making and regulatory activities. To benefit from the EHDS in the future, competent authorities in the field of SoHOs could consider the collaboration with competent bodies under the EHDS at national and EU level, including for aspects related to technical and semantic interoperability.
• Fundamental rights
The proposal would bring positive impacts on some fundamental rights of citizens (health protection, non-discrimination, privacy, informed consent), in particular by strengthening the provisions relating to donor protection and vigilance and the reporting of genetic conditions in children born from medically assisted reproduction with third party donation, and by ensuring that requirements for safety and quality are based on scientific evidence. However, for most of the ethical aspects, in particular the rights of children born from medically assisted reproduction, decisions are taken by Member States at national level.
The proposal maintains the current principle of voluntary and unpaid donation (VUD) in line with Article 3 of the EU Charter of Fundamental Rights that prohibits the commercialisation of the human body. However, the proposal harmonises the differing versions in the blood and the tissue and cell Directives and adapts them to the principle of ‘financial neutrality’ recently recommended by the Council of Europe Committee on Bioethics 12 .
4. BUDGETARY IMPLICATIONS
The legislative financial statement attached to this proposal sets out the budgetary, human and administrative resource implications. Appropriations will be reallocated within the financial envelope of the EU4Health Programme 13 in the Multiannual Financial Framework (MFF) 2021-2027. That Programme was established to respond to the need for further action at Union level to support cooperation and coordination among the Member States. That Programme should be a means to promote strengthened exchange of best practices between Member States, to support networks for the sharing of knowledge or for mutual learning, to address cross-border threats to health so as to reduce the risks of such threats and to mitigate their consequences, and to improve efficiency by avoiding the duplication of activities and optimising the use of financial resources. In that context, some activities organised jointly among Member States, such as inspections or SoHO preparations assessments, could be eligible for Union financial support.
5. OTHER ELEMENTS
• Implementation plans and monitoring, evaluation and reporting arrangements
The Commission will review the monitoring indicators periodically and evaluate the impacts of the legislative act after five years. The monitoring will be possible thanks to the data from reporting obligations on Member States and SoHO entities. The EU SoHO Platform will enable the collection of all elements of the continuous monitoring plan as it automates the extraction of relevant indicators without additional input from stakeholders. For the evaluation, additional data will be collected, in particular on the costs, the usability and the integration across systems. The data platform will be used to transparently publish aggregated indicators of general interest, such as serious adverse occurrences related to SoHOs, insufficiencies of supply or authorised SoHO preparations.
• Detailed explanation of the specific provisions of the proposal
The new Regulation, repealing the Blood Directive 2002/98/EC and the Tissues and Cells Directive 2004/23/EC, and their implementing legislation, is structured around obligations for the different stakeholders: the national competent authorities, the entities handling SoHOs, and the Commission. It includes specific requirements for all organisations that carry out activities that can affect the safety, quality or efficacy of SoHOs used for human application and describes obligations for designated authorities that will verify the proper implementation of the provisions. It will consist of the following main chapters:
Contents
- Chapter I: General provisions
- Chapter II: Competent Authorities
- Chapter III: SoHO Supervisory Activities
- Chapter IV: General Obligations on SoHO Entities
- Chapter V: General Obligations on SoHO Establishments
- Chapter VI: SoHO Donor Protection
- Chapter VII: Recipient and Offspring Protection
- Chapter VIII: Supply Continuity
- Chapter IX: SoHO Coordination Board
- Chapter X: Union Activities
- Chapter XI: EU SoHO Platform
- Chapter XII: Procedural Provisions
- Chapter XIII: Transitional Provisions
- Chapter XIV: Final Provisions
Chapter I contains general provisions of this Regulation. It defines the subject matter and the scope of application of the Regulation. In recognition of the importance of ensuring safety and quality of SoHOs that are not defined by the terms ‘blood’, ‘tissue’ or ‘cell’, such as breast milk and intestinal microbiota, and to future-proof the legislation in this regard, the scope is defined by the broader term SoHOs. Solid organs continue to be regulated by Directive 2010/53/EU and are excluded from the definition of this term. This chapter contains the definitions of the different elements of the Regulation and of the terminology used throughout the text. Furthermore, it introduces the description of SoHO activities and describes the possible more stringent measures set by Member States, in line with Article 168(4)(a) of the TFEU. Certain exclusions are described, along with the partial applicability of this Regulation when SoHOs are used to manufacture products regulated by other Union legislation, or as the starting and raw material thereof.
Chapter II contains the provisions regarding competent authorities for SoHOs, which are responsible for the SoHO supervisory activities. It covers the designation of competent authorities, the possibility to delegate certain SoHO supervisory activities, and general principles for their functioning (independence and impartiality, transparency). It also defines their general responsibilities and obligations. It covers the communication between competent authorities (within the SoHO sector) and consultation and cooperation with authorities of other regulated sectors. It lays down general obligations for authority personnel and provides for competent authorities obligations regarding Commission controls.
Chapter III covers all activities competent authorities undertake vis-à-vis SoHO entities or processes, with the obligation to maintain a register of SoHO entities and a procedure for their registration; the obligation to have a system for the authorisation of SoHO preparations, and a procedure for these authorisations, with provisions for conducting the assessment of SoHO preparations, possibly in a joint process with one or more competent authorities, and further specific obligations for SoHO preparation assessors. This Chapter also covers the obligation to have an authorisation system for SoHO establishments (specific in case of importing SoHO entities) and a procedure for their authorisation (SoHO establishments/importing SoHO entities). It stipulates the obligations for inspections of SoHO establishments and other SoHO entities, possibly via joint inspections, and the specific obligations for inspectors. It provides for the obligations for competent authorities regarding data publication, traceability, vigilance and, SoHO Rapid Alerts.
Chapter IV outlines all the general obligations on SoHO entities, namely their registration, the nomination of a Responsible Person if they release SoHOs for clinical use, the obligations regarding export of SoHOs. It also lays down the obligation for authorisation of SoHO preparations, and the procedure for application for such authorisation. It also covers the obligations for importing SoHO entities regarding their authorisation, and the application for such authorisation. It provides for the obligations for SoHO entities regarding activity data collection and reporting, traceability and coding, the obligation to apply the Single European Code to SoHOs distributed for human application (except for some specific SoHOs), and vigilance notifications.
Chapter V lays down the general obligations on SoHO establishments, a sub-set of SoHO entities that process and store SoHOs. It provides for their authorisation and the application procedure for such authorisation, the obligation to have a Quality Management system in place, and the obligation to designate a physician responsible for specific tasks.
Chapter VI contains the provisions related to the protection of SoHO donors, with standards, and how to implement these standards concerning donor protection.
Chapter VII contains the provisions related to the protection of patients treated with SoHO (recipients) and offspring from medically assisted reproduction, with standards, and how to implement these standards concerning recipient and offspring protection. It also stipulates conditions for the release of SoHOs for human application, and conditions for exceptional release.
Chapter VIII lays down provisions to ensure the continuity of supply of SoHOs. It covers the obligation for Member States to have national SoHO emergency plans (for SoHOs that are critically important for patients) and the responsibilities of competent authorities and entities regarding supply alerts for critical SoHOs. It also stipulates the conditions for derogation from the obligations to authorise SoHO preparations in emergencies, provides for additional emergency measures taken by Member States and finally lays down the obligation for SoHO entities carrying out activities with critical SoHOs to have an emergency plan in place.
Chapter IX establishes the SoHO Coordination Board (SCB) to support Member States in the coordination of implementation of this Regulation and the delegated and implementing acts adopted pursuant to it. This chapter also provides for the composition of the Board and how its functioning is organised.
Chapter X outlines the activities organised at Union level, regarding training and exchange of competent authorities’ personnel, the Commission controls in Member States, and the support provided by the Commission to support the implementation of the Regulation. It also refers to the cooperation with the EDQM, which should address procedures for the development and revision of technical guidelines, including evidence gathering, guideline drafting and public consultation.
Chapter XI describes the EU SoHO Platform that will support information sharing between authorities and with SoHO entities, and outlines its general functionalities.
Chapter XII contains the procedural provisions of the Regulation with regard to confidentiality and data protection obligations. It furthermore contains provisions regarding the exercise of delegation, the urgency procedure and the committee procedure. Finally, it lays down the penalties applicable to infringements of the provisions of this Regulation to be set by Member States.
As regards delegated acts, following the adoption of the proposal, the Commission intends to create an expert group in line with decision C (2016) 3301 in order to advise and assist it in the preparation of delegated acts, as well as on issues related to implementation of the Regulation as regards:
(a)preparing opinions at the request of the Commission on the regulatory status of a substance, product or activity (and consulting equivalent advisory bodies established in other relevant Union legislation);
(b)providing expertise relevant for the development of technical guidelines, other guidelines and technical methods to the Commission;
(c)reviewing reports on activity data and on vigilance data prior to publication by the Commission;
(d)contributing to the continuous monitoring of technical progress and assessment of whether the safety and quality requirements set out in this Regulation are adequate to ensure safety and quality of SoHOs and SoHO preparations and the safety of SoHO donors;
(e)supporting the Commission to exchange views with Union or international level professional associations working in the field of SoHOs on issues of general interest in relation to the applicability of the provisions of this Regulation;
(f)providing expertise to the Commission for the development of guidelines, standards or similar on an international level for SoHOs and their quality and safety, when appropriate;
(g)advising the Commission on the appropriate content and format of Union training programmes for competent authority personnel and supporting the performance of training activities;
(h)providing advice and expertise on the preparation of delegated acts.
The expert group should also provide technical advice to the Commission when it considers that the EDQM guidelines are not sufficient to meet a standard for donor protection or a standard for recipient and offspring protection as provided for in this Regulation.
This Chapter sets the transitional provisions applicable to establishments and SoHO preparations authorised under the former BTC legislation. It stipulates the status of SoHO stored before the application of this Regulation. Finally, it contains the transitional measures related to the date of adoption of certain delegated and implementing acts.
The final chapter stipulates the repeal of Directives 2002/98/EC and 2004/23/EC. It also sets the provision for the evaluation of the Regulation, and its dates of entry into force and application.