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EMA publishes additional reports on decision-making for orphan medicines; initiative addresses requests from stakeholders.

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

The new report summarises 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 authorisation. This is the precondition for it to benefit from ten year market exclusivity, one of the incentives of the European Union (EU) orphan programme. 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 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 Chair of COMP. “Patients, as well as companies, will better understand the decision-making process once a medicine for a rare disease gets a marketing authorisation. And health technology assessment bodies (HTAs) might use this additional information when establishing the cost effectiveness of the medicine”.

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.

The 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 authorisationof the new treatment to reconfirm its eligibility for ten year market exclusivity.

Orphan designation gives access to a number of incentives to foster research and innovation, including fee reductions for scientific advice and ten year market exclusivitywhen the medicine is authorised in the EU.

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.

The first report covers Prevymys, an antiviral medicine that prevents cytomegalovirus reactivation and disease in patients who receive immunosuppressant medicines following an allogeneic hematopoietic stem cell transplant.