The European Medicines Agency (EMA) will now be publishing “orphan maintenance assessment report” for every orphan-designated medicine which has been recommended for marketing authorisation.
To qualify for orphan designation, a medicine must target a disease that is life-threatening or chronically debilitating that affects less than 5 in 10,000 patients in the EU. If there is already another treatment available for the targeted rare disease, the developer of the new medicine must show that there is a significant benefit to patients compared to the existing options, meaning the medicine provides a clinically relevant advantage or a major contribution to patients.
Orphan designation gives access to a number of incentives to foster research and innovation, including fee reductions for scientific advice and ten year market exclusivity when the medicine is authorised in the EU.
The Committee for Orphan Medicinal Products (COMP) assesses whether a medicine fulfils the criteria for orphan designation at two points in time: early on in a medicine’s development to provide access to incentives supporting the development activities, and again at the time of marketing authorisation of the new treatment to reconfirm its eligibility for ten year market exclusivity.
In the context of increasing demand for greater transparency, these new “orphan maintenance assessment reports” will now summarise the reasoning of the 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 authorisation. These reports 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 orphan maintenance assessment reports will be published for all positive and negative COMP opinions, as well as withdrawals. They will describe 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.
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