The European Medicines Agency has published a report of the workshop held on 26 September 2013 to discuss further development of patient involvement in the evaluation of medicines, in particular in the assessment of benefit and risk that lies at the heart of the regulatory process. The Agency believes that incorporating the knowledge, views and preferences of patients throughout the lifecycle of medicines, from early stages of development through to authorisation and monitoring of safety and efficacy, adds value to the scientific assessment.
Because the views of patients on the benefit and risk of a medicine can differ from those of other stakeholders, and may also vary among patients and at different disease stages, this process often requires innovative approaches and methodologies.
The brought together representatives of patients, consumers and healthcare professionals as well as the pharmaceutical industry and members of the Agency’s scientific committees and staff.
Action points identified included the need to:
- identify where quantitative versus qualitative input is needed, and develop and validate new tools for eliciting values and preferences and representing benefit and risk; these tools need to take account of the way values vary between patients and can change during the patient journey;
- develop means for identifying and managing differences of view among patients, and between patients and other stakeholders;
- consider what training and support is needed to maximise patient involvement at all stages of the process for the full range of disease states, and who should be supplying these;
- further develop the role and timing of patient involvement in later, post-marketing stages of the product lifecycle (e.g. in signal assessment);
- identify and try to minimise legal, regulatory, financial and procedural barriers to patient involvement.
These action points will be considered as part of the on-going review of the existing formal framework of interaction between the Agency and patient and consumer organisations. This framework has been in place since 2005 and defines how the Agency interacts with patients and consumers.
Currently, patient representatives are involved in a number of Agency activities in the capacity of:
- full members of the Pharmacovigilance Risk Assessment Committee (PRAC), Paediatric Committee (PDCO), Committee for Advanced Therapies (CAT) and Committee for Orphan Medicinal Products (COMP): representatives of patient organisations participate in the same way as other committee members;
- experts in the Scientific Advice Working Party (SAWP): companies can request scientific advice on the appropriate tests and studies to be carried out at any stage in the development of a medicine. The EMA has been increasingly involving patient representatives in SAWP meetings as they can bring a unique perspective on areas such as the feasibility of study proposals, patient population, duration and design of studies and relevant outcomes and safety concerns from a patient point of view;
- experts in the various scientific advisory groups (SAGs), which provide specialised advice to the Agency’s scientific committees on the benefit-risk evaluation of specific types of medicines or treatments. Patient representatives are commonly involved as SAG members (in over 80% of cases in 2013).
In addition, a permanent forum for dialogue between the Agency and patient representatives, the Patients’ and Consumers’ Working Party (PCWP), provides recommendations to the Agency and its human scientific committees on all matters of interest to patients in relation to medicines.