Involving patients in discussions on benefits and risks of medicines
EMA publishes report on CHMP pilot project
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).
The report concludes that patients should continue to be invited to oral explanations when their input could be valuable to the assessment of a medicine. 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.
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:
- Scenesse (afamelanotide) for the treatment of erythropoietic protoporphyria (EPP) (September 2014);
- Intuniv (guanfacine) for the treatment of attention deficit hyperactivity disorder (ADHD) in children and adolescents (June 2015);
- Tecfidera (dimethyl fumarate) for the treatment of multiple sclerosis (October 2015);
- Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy (May 2016);
- Translarna (ataluren) for the treatment of Duchenne muscular dystrophy (June 2016);
- Translarna (ataluren) for the treatment of Duchenne muscular dystrophy (November 2016).
After each case, feedback was obtained from the patients and the CHMP members who were involved in order to analyse the participation and contribution of the patients. The feedback from CHMP members and patients was positive and confirmed the benefit of including patients in discussions at the CHMP when the patient perspective could compliment the assessment.
Following the positive outcome of the pilot, the Committee also discussed and agreed on the use of additional methods to provide opportunities for more regular involvement, such as inviting patients to participate in CHMP discussions by teleconference, or organising written consultations, which would allow for input outside CHMP meetings and could provide feedback from a larger number of patients.
More details, as well as quotations from those who took part in the pilot, are available in the report published today.
Bringing the patient perspective to the work of the Agency
The project to involve patients in discussions on the benefits and risks of medicines is in line with the work programme of the CHMP which recommends a further integration of patients' views in the assessment of benefits and risks of medicines. It also reflects the Agency's continued emphasis on stakeholder involvement.
In addition to involving patients directly in CHMP discussions, patient representatives are already involved in many other activities, e.g., in the capacity of:
- full members of the Pharmacovigilance Risk Assessment Committee (PRAC), the Paediatric Committee (PDCO), the Committee for Advanced Therapies (CAT) and the Committee for Orphan Medicinal Products (COMP);
- experts within scientific advice procedures;
- experts in the various scientific advisory groups (SAGs), which provide specialised advice to the Agency's scientific committees on the benefit-risk evaluation of specific types of medicines or treatments;
- experts reviewing documents for the public prior to publication, such as package leaflets, EPAR summaries, herbal summaries and safety communications;
- members of the Patients' and Consumers' Working Party (PCWP), through which they provide recommendations to the Agency and its human scientific committees on all matters of interest to patients in relation to medicines.