Marketing Authorisation Holder (MAH): The people or company benefiting from a Marketing Authorisation submitted through the "Centralised procedure" to distribute and sell a medicine in the EU. Under EU law, the MAH has a wide role in the pharmacovigilance process: to build and maintain the risk management plan, to collect and analyse safety data, and to ensure adverse events are reported and analysed properly in particular with regular audits of his/her pharmacovigilance system (Directive 2001/83/EC, Art 104.2)
European Medicines Agency (EMA): The EMA is an agency of the EU whose goal is to protect and promote human and animal health. The agency is responsible for the scientific assessment of medicine to be marketed within the EU and the European Economic Area (EEA) and authorised under the centralised procedure. The EMA coordinates pharmacovigilance in the EU and operates services and processes in line with EU legislation through two main committees listed hereafter.
Pharmacovigilance Risk Assessment Committee (PRAC) at the EMA: The PRAC is the EMA's committee responsible for assessing and monitoring the safety of human medicines. The PRAC is responsible for all aspects of the risk management of medicines and for the assessment of the risk management plan provided by the applicant. The PRAC is notably involved in the assessment of ATMPs’ marketing authorisation applications though providing advice to the Committee for Medicinal Products for Human Use (CHMP). The PRAC is composed of a chair, one member and one alternate member appointed by each of the EU Member States, one member and one alternate member appointed by each of the EEA/European Free Trade Association States, six members appointed by the European Commission to ensure the availability of the relevant expertise, one member and one alternate representing healthcare professionals and appointed by the European Commission, and one member and one alternate representing patients’ organisations and appointed by the European Commission.
Committee for Medicinal Products for Human Use (CHMP) at the EMA: The CHMP is the EMA committee responsible for preparing the Agency's opinions on all matters concerning medicines for human use, including the granting of marketing authorisations and post-authorisation measures. In pharmacovigilance, the CHMP acts on PRAC recommendations and may impose or vary risk minimisation measures, post-authorisation safety studies, or other conditions attached to a marketing authorisation. For ATMPs, it receives and integrates Committee for Advanced Therapies and PRAC input before issuing its final opinion. More information on the CHMP here.
Healthcare professionals: Physicians, nurses, pharmacists and other clinical staff serve as the frontline for pharmacovigilance in practice. They monitor patients for both immediate and delayed adverse reactions, document these events, and report suspected safety signals through national and European pharmacovigilance channels. Their role is particularly important for ATMPs where late-emerging toxicities and unusual immune or cellular responses may occur long after treatment administration.
Patients and care providers: They play a vital role in assessing safety in clinical practice. They can report symptoms that arise after discharge from hospital, during long-term follow-up periods, or outside formally monitored clinical settings. Their reports help to identify events that might otherwise go unnoticed during brief clinical consultations or in tightly controlled clinical trials.
Patients' Organisations: Beyond their role as individual notifiers, patients' organisations participate institutionally in pharmacovigilance governance, notably through official representation on the PRAC. They provide a collective perspective on the acceptability of risk-benefit profiles, the adequacy of risk minimisation measures, and the practical impact of safety signals on the concerned communities. For rare diseases, which represent a significant proportion of ATMP indications, patients' organisations often hold unique long-term knowledge of disease natural history and treatment experience that complements regulatory and clinical datasets.
Hospital ATMP units and patient registries: Hospital ATMP units play a practical role in ATMP pharmacovigilance: they handle preparation, administration, local traceability and post-treatment follow-up. Registries and long-term follow-up systems are also important because they help collect outcomes over months or years, which is often required for ATMPs. These structures are particularly valuable when medicines are used under hospital exemption or in highly specialised care pathways.
Research Ethics Committees are independent bodies established by Member States in accordance with national law, whose role is to verify that research protocols comply with applicable ethical principles all along their conduct. Their review covers the scientific relevance and design of the trial, the assessment of risks and benefits, participant information and consent procedures, the suitability of investigators and facilities, and arrangements for vulnerable populations. A positive opinion is required prior to obtain a clinical trial authorisation. In case of a negative opinion from an ethics committee, the Member State shall provide for an appeal procedure.
While ethics committees are not pharmacovigilance bodies as such, their role intersects with pharmacovigilance at several stages. In the pre-authorisation phase, they assess the adequacy of safety monitoring provisions included in clinical trial protocols. This includes adverse event reporting procedure and data safety monitoring board structures, a significant function for ATMPs, given the novelty and complexity of their safety profiles. In the post marketing authorisation phase, ethics committees review post-authorisation safety studies involving new data collection from patients. They also provide ongoing ethical oversight of long-term follow-up protocols. These protocols are frequently imposed as a condition of marketing authorisation for ATMPs, and may span years or decades. Thus, the role of ethics committees complements the regulatory oversight exercised by the EMA including PRAC, and NCAs.
National Competent Authorities (NCAs): Each EU Member State has a designated NCA responsible for the regulation of medicines at national level. In the context of pharmacovigilance, NCAs play a critical role: they receive and process adverse event reports submitted at national level, contribute to signal detection activities within the EudraVigilance network, participate in PRAC assessments, and are responsible for enforcement of pharmacovigilance obligations within their territory. For ATMPs, NCAs are also involved where products are used under hospital exemption, a pathway outside centralised EMA oversight and for which national-level pharmacovigilance governance is the primary safeguard.
European Commission: The European Commission holds the formal decision-making authority in the centralised procedure: it grants, amends, suspends or revokes marketing authorisations on the basis of EMA opinions. In the pharmacovigilance context, the Commission may act upon the PRAC or the CHMP recommendations to impose urgent safety restrictions, require label updates, or initiate referral procedures. It also holds legislative and regulatory missions for the broader EU legislation framework for pharmacovigilance.
EU Network Pharmacovigilance Oversight Group: The EU Network Pharmacovigilance Oversight Group (formerly the ERMS Forum Group) is a key body of the Heads of Medicines Agencies. It is responsible for overseeing the implementation and consistency of pharmacovigilance activities across the EU. Its main tasks are to ensure harmonised pharmacovigilance practices among EU Member States and the EMA, support the coordination of risk management and signal detection activities, and facilitate the exchange of best practices and the resolution of cross-border pharmacovigilance issues. More information here.