EMA begins publishing reports on whether approved medicines still qualify for orphan designation

The European Medicines Agency (EMA) has begun publishing reports on whether newly approved drugs to treat rare diseases still qualify for orphan designation once they receive marketing authorisation.

The reports, known as orphan maintenance assessment reports, will be published for every orphan-designated medicine which has been recommended for marketing authorisation by the EMA. The reports will discuss the Committee for Orphan Medicinal Products (COMP) reasons on whether or not a medicine designated as an orphan medicine during its development still fulfils the designation criteria at the time of its authorisation.

The COMP assesses whether a medicine meets the criteria for orphan designation twice during development; once early on in a medicines development, and then again at the time of marketing authorisation of the new treatment to reconfirm its eligibility for ten year market exclusivity. Until now, these assessments have not been included in the public assessment reports. The report will be published as part of a medicine’s European Public Assessment Report (EPAR) once the European Commission has adopted its marketing authorisation decision.

The EMA states that the reports are being released in response to concerns that some drugs benefiting from incentives for orphan medicines may not still qualify for the designation in the time period between when the designation is first granted and the time of authorisation.

The orphan maintenance assessment reports will be published for all positive and negative COMP opinions, including withdrawals. The reports will include details on the orphan condition and its seriousness, the spread of the condition at the time of maintenance of the designation, and, if applicable, the significant benefit over already authorised medicines.

To read more about the EMA reports on whether approved medicines still qualify as orphans, please click here.