Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Involvement of Patients’ and Consumers’ Organisations (PCOs) in activities of the EMA: Development, implementation and outlook ***

Henny Anna Zietze (Abschlußjahr: 2010)

Language: English

Patients representatives are currently involved in many of the Agency's activities and their participation is continually growing. Some procedures are structured and based on legal provisions or guidance documents. However, the further development clearly aims at a structuring of all kinds of interaction. The first time patients were formally involved as member of a scientific committee was in 2000, when the orphan drug legislation came into force and the COMP was established. The EMA MB and other scientific committees (PDCO, CAT) followed this approach. In addition to this involvement of PCOs on a legal basis, a variety of other interactions takes place without specific legal provisions. In addition to their role as full members or observers, patients may be invited either as experts or as representatives of their organisation to join meetings of the scientific committees, SAGs and WPs or other ad-hoc product related discussions. They contribute to guideline preparation and are consulted on the wording of product information and safety communications. PCOs that would like to interact with the EMA have to officially apply for this and prove that they fulfil all criteria that have been developed by the Agency. These criteria ensure that all eligible PCOs have a specific interest in medicinal products and conform to minimum standards with regard to transparency and financial independence. Their participation is published on the EMA website. Dedicated procedural guidance for the involvement of PCOs is available in the following areas:

  • involvement of members of PCOs as experts or representatives of their organisation in committee related activities,
  • review of package leaflets during new marketing authorisation applications,
  • review of package leaflets during the renewal procedure,
  • review of EPAR summaries at the time of initial authorisation of a medicinal product,
  • review of safety communications before publication (e. g. press releases, announcements on withdrawals, product safety etc.),
  • participation in meetings of the PhVWP as observer.


Explicit guidance is currently lacking in the following areas (examples):

  • involvement of PCOs as members in the EMA MB and the scientific committees,
  • involvement of PCOs as observers in any of the Agency's bodies,
  • involvement of PCOs in guideline preparation.


As a platform for exchange between the EMA and PCOs the PCWP has been founded in 2006 following several workshops with representatives of the Agency and patients' organisations. Its focus lays in the monitoring of the progress of this collaboration. Among others, this can be assessed through the implementation status of the proposals for improvement made by the PCWP in the areas of transparency and dissemination of information, patient information, pharmacovigilance, and interaction between EMA/CHMP and PCOs. Up to now, the majority of the proposals that have been evaluated in the frame of this master thesis have been implemented or partly implemented. Some of them, such as the process for PL review during the marketing authorisation procedure can be controversially discussed, as they overlap with existing legal provisions (readability user testing). Others that have not yet been realised, such as the balanced description of benefit and risk in the PL are reflected in the coming work programs of the PCWP and the EMA and will be pursued. In general, the expansion of the involvement of patients in EMA activities is of major interest for the Agency and forms part of its road map to 2015.

Prerequisite for a successful collaboration is that each party is aware of their mutual expectations and knowledge as well as the confidential nature of every product-related discussion. Processes and objectives have to be described in detail for each procedure concerned by the interaction. Although the latter has not been completely achieved so far, the on-going involvement of PCOs in EMA activities is considered valuable. It increases awareness and enhances the understanding of regulatory procedures in the public. In return, the needs of the patients and real-life implications of regulatory decisions can be respected.

Pages: 45

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