- The European Medicines Agency (EMA) has launched a pilot project that allows patients to weigh in on a drug for which the Committee for Medicinal Products for Human Use (CHMP) harbors any doubts. Patients may also present their viewpoints on drugs that are being considered for market withdrawal.
- This one-year pilot is designed to hear the patient perspective on therapies that are being used to treat an unmet medical need. The EMA also hopes that patients will learn more about the inner workings of the CHMP review process.
- This is the first time that patients have ever been invited to participate in risk/benefit conversations.
The first drug to be reviewed as part of this pilot program is Scenesse (afamelanotide) from Austria-based Clinuvel. If approved, Scenesse will be the first approved therapy for erythropoietic protoporphyria (EPP), a genetic blood disorder that renders affected individuals completely intolerant of light and affects as many as 10,000 people worldwide.
Two patients with EPP have already testified to the CHMP, describing in detail how difficult it is to live with this disease. The endgame is to continue to weigh the benefits and risks of a particular medication using hard evidence and clinical knowledge; however, the role of the patient is now also being considered, which will undoubtedly have some impact on the final outcomes of difficult drug approval decisions.