On 17 November 2020, the European Commission published an inception impact assessment for a revision of the Directive 2004/23/EC on standards of quality and safety for the donation, procurement, testing, processing, preservation, storage and distribution of human tissues and cells (“Tissues and Cells Directive”), and the Directive 2002/98/EC on standards of quality and safety for the collection, testing, processing, storage and distribution of human blood and blood components (“Blood Directive”). These Directives are collectively referred to in the document as the Blood, Tissues, and Cells Legislation (“BTC”).
The European Commission’s proposal pursues three main objectives. Firstly, to ensure safety and quality standards for patients, donors, and children born from in vitro fertilization; and to ensure enforcement of safety and quality requirements. Secondly, to optimise access to BTC therapies, and avoid shortages of this type of therapies. Thirdly, to ensure that the future framework facilitates the development of innovative BTC therapies.
Identified shortcomings in the current framework
Following an evaluation of the existing BTC legislation, the European Commission identified the following gaps and shortcomings:
- The current safety and quality requirements fail to adequately address frequently changing scientific and epidemiological developments.
- Divergent transposition in EU Member States causes unequal levels of safety and quality of BTC treatments and barriers to the exchange of BTC across the EU.
- The current legislation does not prevent avoidable risks for BTC donors and for children born from donated eggs, sperm, or embryos.
- The current 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. 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.
- There is uncertainty concerning the status of emerging substances susceptible to being considered either BTC or other therapeutics, in particular medicinal products.
- Under the current legislation, the EU is vulnerable to interruptions in the supply of some BTC.
According to the European Commission, these challenges have been emphasized due to the COVID-19 pandemic.
Proposed policy options
In order to tackle the regulatory deficiencies outlined above, the European Commission proposes three different scenarios. The key differences among the three scenarios concern the body in charge of establishing the technical requirements, and the degree of enforcement at EU and national level.
The first policy option relies on strengthened quality and safety requirements defined by BTC establishments, strengthened national inspection, EU audits, and classification advice (“self-regulation system”). The main features of this proposal are the following:
- General safety and quality principles would be provided at EU level. They would be complemented by technical rules and specifications provided by BTC establishments on the basis of documented risk assessments and scientific evidence.
- The proposed legislation would provide for substantially strengthened oversight principles. The European Commission would perform controls in EU Member States, including audits of national systems of inspection, authorisation, and vigilance.
- In order to improve access to and sufficiency of BTC establishments, mandatory monitoring and notification of sufficiency data and measures for emergency supply responses would be introduced at EU level.
- The scope of legislation would include novel substances of human origin currently used but not regulated at the EU level, in order to accommodate innovative therapies.
- Prior authorisation by competent authorities would be required for major changes in the steps of collection, processing, and use of BTC. This authorisation would be based on data demonstrating safety and benefit for patients that justifies any risks associated with the treatment.
The second policy option would be supported by EU-level safety and quality requirements defined by European Expert Bodies, strengthened national inspection, EU audits, and classification advice (“co-regulation system”). This scenario would include the same features than the first policy option, with one relevant difference: BTC establishments would be required to take into account technical rules and specifications provided by authoritative bodies such as the European Centre for Disease Prevention and Control (“ECDC”), and the Council of Europe’s European Directorate for the Quality of Medicines & HealthCare (“EDQM”)
The third policy option provides EU-level safety and quality requirements laid down in EU legislation, improved national inspections systems, and classification advice. Binding technical rules and specifications for their implementation, along with a mechanism for regular updates, would be provided at EU level. This approach would be complemented by similar oversight measures as in the previous policy options. However, the European Commission would not carry out control system audits. The measures relating to ensuring sufficiency and supporting innovation proposed in the other two options would also apply in this scenario.
Following steps
The inception impact assessment is open for feedback until 14 December 2020. After that date, the European Commission will publish an open public consultation for twelve weeks. The potential legislative proposal is expected by the fourth quarter of 2021.
This new development is particularly relevant to manufacturers of medicinal products or medical devices that use BTC as starting materials and those companies supplying equipment such as test kits for the BTC sector.
Authored by Fabien Roy and Silvia López Arnao
