EMA Seeks Patient Input In Drug Submission Review Process

The European Medicines Agency (EMA) is seeking patient input in the drug review process. The patient-centric pilot project, announced in a press release, will incorporate assessments from patients on the benefits and risks of medicines during Committee for Medicinal Products for Human Use (CHMP) evaluations. Integrating the patient into the drug approval process is slowly becoming a trend in the industry. Indeed, this move follows in the footsteps of Canada’s recent announcement of a patient input pilot program for rare diseases, in cooperation with Hoffmann-La Roche Limited and Hyperion Therapeutics.

Guido Rasi, EMA Executive Director, said, “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.”

EMA Patient-Centric Pilot Project

The EMA patient-centric pilot project will run for at least one year. At the end of the pilot phase, a report to the CHMP will discuss the patient’s experience giving feedback, the feasibility of patient input, organizational aspects, areas for improvement, and proposals for full implementation of patient input in the drug review process. The EMA has made the pilot project’s parameters available to the public, including information on the types of patients being sought.

The EMA states that afamelanotide will be the first medication evaluated with patient input. The agent for the treatment of erythropoietic protoporphyria (EPP) is currently under evaluation by the CHMP. EPP is a rare genetic blood disorder, which causes sufferers to experience a complete intolerance to light. At a September 2014 CHMP meeting, two EPP patients discussed the condition and afamelanotide with committee members, and their input is to be incorporated into CHMP’s assessment.

The EMA states that while the project represents the first time patients have been invited to directly give input, patients are active in other capacities, such as the Pharmacovigilance Risk Assessment Committee (PRAC), the Pediatric Committee (PDCO), the Committee for Advanced Therapies (CAT), the Patients' and Consumers' Working Party (PCWP), and the Committee for Orphan Medicinal Products (COMP).

Patient-Centricity

The trial environment, which centers around the patient, is moving towards patient involvement in all phases of development. Mr. Rasi explained the rationale for patient-centricity, saying, “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.” Listening to the patient enriches the body of knowledge and provides a more accurate assessment of the medication through its lifecycle.