EMA publishes a report regarding a CHMP pilot project: ‘Involving patients in discussions on benefits and risks of medicines’

The European Medicines Agency (EMA) has recently published a report which describes a pilot project to explore the idea of including patients in benefit/risk discussions at the Committee for Medicinal Products for Human Use (CHMP) meetings.

The pilot study provided patient groups with an opportunity to express their views during the benefit/risk discussions held by the CHMP as part of an oral explanation for the assessment of a marketing authorisation application for a new medicine.

For the purposes of this pilot, at least two patients or carers with personal knowledge and understanding of the therapeutic indication under discussion, were invited on a case-by-case basis to attend CHMP benefit/risk discussions.   Invitations were only issued when the Rapporteurs and EMA product leads could identify a specific benefit to including patients/carers in the oral explanation. For example, if the discussion was for a medicinal product for an unmet medical need where the CHMP wanted to gain insight into the impact on the patient population or where the CHMP wanted to identify the potential impact on a patient population of a recommendation to revoke, suspend or restrict an existing marketing authorisation. The patients included in the study had the opportunity to engage fully in the benefit/risk discussions and actively question the applicants; they were unable to be part of the final voting process.

During the course of the pilot study (September 2014 – December 2016), representatives of the patient population were invited to become involved in the following oral explanations:

  • Scenesse (afamelanotide) – intended for the treatment of erythropoietic protoporphyria
  • Intuniv (guanfacine) –intended for the treatment of ADHD in children & adolescents
  • Tecfidera (dimethyl fumarate) – intended for the treatment of multiple sclerosis
  • Kyndrisa (drisapersen) -intended for the treatment of Duchenne muscular dystrophy
  • Translarna (ataluren) – intended for the treatment of Duchenne muscular dystrophy
  • Translarna (ataluren) – intended for the treatment of Duchenne muscular dystrophy

Following each oral explanation both the included patients and the CHMP members provided feedback to enable the value of the contributions by the participants to be analysed. The results from this feedback proved to be very positive overall with the patient participants reporting that they had greatly improved their understanding of the process involved in assessing a new marketing authorisation. CHMP members largely felt that inclusion of the patients was beneficial.

As a result of the encouraging reactions from all those that were involved in the pilot study, the CHMP committee agreed to increase the opportunities for more regular involvement of patients in relevant CHMP discussions, continuing with their involvement in oral explanations, but also including the possibility of using teleconferences and written consultations at any time during the assessment evaluation as potential alternatives.

The full Pilot Study Final Report: ‘Outcome Report on Pilot to involve patients in benefit/risk discussions at CHMP meetings’, can be found here.