The European Medicines Agency (EMA) has published a final report on the experience gained during its pilot project to involve patients directly in the assessment of the benefits and risks of medicines in its Committee for Medicinal Products for Human Use (CHMP).
During the pilot, which ran from September 2014 to December 2016, patients participated in discussions at the CHMP and gave their views on the benefits and risks of the following medicines, five of which were orphan medicines:
- Scenesse(afamelanotide) for the treatment of erythropoietic protoporphyria (EPP);
- Intuniv(guanfacine) for the treatment of attention deficit hyperactivity disorder (ADHD) in children and adolescents;
- Tecfidera(dimethyl fumarate) for the treatment of multiple sclerosis;
- Kyndrisa(drisapersen) for the treatment of Duchenne muscular dystrophy;
- Translarna(ataluren) for the treatment of Duchenne muscular dystrophy;
- Translarna(ataluren) for the treatment of Duchenne muscular dystrophy.
The responses of participating patients, the EMA and the CHMP are compiled in the report which came to the conclusion that patients should continue to be invited to oral explanations when their input could be valuable to the assessment of a medicinal product. This could be the case, for example, when the Committee is considering whether to recommend the authorisation of a new medicine or the maintenance, suspension or revocation of an existing authorisation, or a restriction of indication of an authorised medicine.
Orphanews June 6, 2017