Is an orphan medicine still an orphan once it gets on the market?

The European Medicines Agency (EMA) will be publishing, as of today, a so-called orphan maintenance assessment report for every orphan-designated medicine which has been recommended for marketing authorization by the Agency.

The new report summarizes the reasoning of the Agency’s Committee for Orphan Medicinal Products (COMP) on whether or not a medicine designated as an orphan medicine during its development still fulfils the designation criteria at the time of its authorization. This is the precondition for it to benefit from 10-year market exclusivity, one of the incentives of the European Union orphan program. The report will be published as part of a medicine’s European Public Assessment Report (EPAR) once the European Commission has adopted its marketing authorization decision.

“The interaction with our stakeholders is a two-way street: they asked for more transparency, we listened to their needs and now we are delivering on our commitment,” explains Bruno Sepodes, the chairman of COMP, adding: “Patients, as well as companies, will better understand the decision-making process once a medicine for a rare disease gets a marketing authorization. And health technology assessment bodies (HTAs) might use this additional information when establishing the cost effectiveness of the medicine.”