New EMA Pilot Puts Patients At The Centre Of Benefit-Risk Analysis

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EMA pilot puts patients at the heart of benefit-risk discussion for marketing authorisation evaluation

A new EMA pilot program will see two patients (or cares) with in depth experience and knowledge a specific disease directly involved in benefit-risk discussions at Committee for Medicinal Products for Human Use (CHMP) meetings. Previously patients had only been consulted during informal expert group meetings.

Whilst patients will not be involved in the votes held by the CHMP, but will otherwise be immersed within the decision making process. Patient representatives will be accompanied to begin with by a mentor who will guide them through the process and help them to engage with the materials handed out prior to the meeting, so that they may contribute effectively to the meeting.

This moves sees the EMA take another step towards patient integration in the authorisation process. With increasing calls for access to not-yet-approved rare disease therapies by the patient community, the relative weight of small increases in quality of life verses greater risks is something the rare disease community are keen to address. And this is a perspective that only patients and carers are really in a position to answer. Representatives will also be invited to contribute to CHMP discussions regarding the withdrawal, suspension and revocation.

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.”Guido Rasi, EMA Executive Director

The first instance of this pilot took place in September, with two patients with erythropoietic protoporphyria (EPP) joining the CHMP to discuss a potential new treatment. The patient discussed their personal experience living with EPP, a genetic blood disorder that creates an absolute intolerance to light, and answered questions from the committee.

For more information on the pilot, you can find the EMA’s pilot outline here.

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