Patients to contribute to Committee for Medicinal Products for Human Use discussions on benefit-risk in marketing authorisation evaluations
Patients are active and vital participants in many of the different committees and meetings of the European Medicines Agency (EMA), adding value to decision processes. Now, patients cross a new frontier by participating in discussions with marketing authorisation applicants when invited by the Committee for Medicinal Products for Human Use (CHMP) as part of an initial one-year pilot project.
In a press release, EMA Executive Director Guido Rasi elaborates: «As patients live with their condition on a day-to-day basis, their views on the therapeutic effect of a medicine and its impact on their quality of life – particularly when these are balanced against the risks – may differ from those of other stakeholders. … Involving patients in CHMP discussions brings the patients’ voice into the decision-making process and ultimately contributes to the safe and rational use of medicines.»
The CHMP is responsible for evaluating whether or not the benefits of a medicinal product outweigh the risks and the quality of the medicine in question, before the European Commission can grant a marketing authorisation valid throughout the European Union. The first oral explanation in which patients were invited by the CHMP took place on 23 September 2014. All discussions are fully confidential, with patients signing a confidentiality agreement.
How will it work?
Patient representatives already contribute to benefit-risk evaluations through participation in ad-hoc expert group meetings and scientific advice/protocol assistance procedures. The CHMP pilot builds on this by inviting patients with particular knowledge of the disease or condition under evaluation to participate in specific benefit-risk discussions at CHMP meetings, to be determined on a case-by-case basis, and typically involving scenarios in which the CHMP is likely to deliver a negative opinion, recommend a withdrawal of an application for a product that addresses an unmet need, or would significantly impact patients.
Who will participate?
For each benefit-risk evaluation requiring patient participation, two patients (or carers) with personal experience and knowledge of the particular disease/condition under evaluation will be invited to participate, selected from the EMA network of eligible patient organisations, as well as other networks, and individuals who have indicated a desire to participate. A mentor, most likely from the Working Party with Patients’ and Consumers’ Organisations (PCWP) will accompany patients through the process during the pilot phase. François Houÿez and Richard West, who represent EURORDIS at the EMA, are two of the four mentors. Materials will be distributed ahead of time to help patients participate effectively and appropriately. Patients will not be involved in the voting procedures but would rather be contributing valuable experience and information that will inform and enrich the voting process.
Louise Taylor, Communications and Development Writer, EURORDIS