EMA Launches Pilot to Engage Patients in Risk-Benefit Assessments

From DCAT Value Chain Insights (VCI)

By Regulatory News posted 09-30-2014 12:22


The European Medicines Agency (EMA) has launched a pilot project to involve patients in the assessment of the benefits and risks of medicines in its Committee for Medicinal Products for Human Use (CHMP).

“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,” said Guido Rasi, EMA Executive Director, in an EMA release. “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 conducting the initial assessment of medicines for which a Europe-wide marketing authorization is sought. Assessing whether the benefits of a medicine outweigh its risks lies at the heart of this authorisation process.

As part of the pilot project, patients will be invited to present their views on medicines for which there is an unmet medical need and where the Committee still has concerns or doubts. Patients may also be invited to give their views in cases where the CHMP is considering whether to recommend the withdrawal, suspension or revocation of a marketing authorization, or a restriction of indication of an authorized medicine.

The pilot project will explore how patients can be involved systematically and effectively in oral explanations at the CHMP. A document outlining the main principles of the project is available here. This pilot project stems from a wider EMA strategy to better involve patients in the agency’s activities.

The first medicine to be included in the pilot project contains the active substance afamelanotide. It is intended for the treatment of erythropoietic protoporphyria (EPP), a rare genetic blood disorder which causes an absolute intolerance to light. Between five to ten thousand patients worldwide have EPP. There is currently no authorised medicine for this condition.

At the September meeting of the CHMP, two patients with EPP shared their experiences of living with the condition and answered specific questions from the committee. Their inputs will be considered by the CHMP as part of its assessment of afamelanotide.

The pilot project will run for at least one year to allow a full assessment of the feasibility of involving patients in CHMP oral explanations. A report on the experience gained will be presented to the CHMP at the end of the pilot phase and will address issues including organisational aspects, feedback from the CHMP and patients involved, lessons learned and areas for improvement, as well as a proposal for full implementation of the project.

The pilot project is in line with the work programme of the CHMP which recommends further integrating patients’ views in the assessment of benefits and risks of medicines. It also reflects the EMA's continued emphasis on stakeholder involvement. Although this is the first time that patients have been invited to participate directly in discussions on the benefits and risk of medicines at the CHMP, patient representatives are already involved in many other activities of the EMA. 

Source: EMA  


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