EMA Begins Publishing Reports on Whether Approved Medicines Still Qualify as Orphans

The European Medicines Agency (EMA) on Wednesday announced it has begun publishing reports detailing its decision-making on whether newly approved drugs to treat rare diseases still qualify for orphan designation when they receive marketing authorization.

In the EU, drugs intended to treat diseases affecting fewer than five in 10,000 people in the EU qualify for orphan designation, which provides authorized medicines with ten years marketing exclusivity and reduced fees.

Committee for Orphan Medicinal Products' (COMP) currently assesses whether a medicine meets the criteria for orphan designation twice during a product's development, once during development and again when it is authorized. But those assessments were not included in the public assessment reports prior to now.

The new reports, dubbed orphan maintenance assessments, will be published for each newly authorized medicine that was granted orphan designation during its development and will discuss EMA's COMP's rationale for why it decided to maintain or withdraw a product's orphan designation.

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

"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," said COMP Chair Bruno Sepodes.

Alongside the announcement, EMA released its first orphan maintenance assessment for Merck's antiviral drug Prevymys (letermovir), which is used to prevent cytomegalovirus (CMV) reactivation in patients that receive allogenic hematopoietic stem cell transplants.

According to the assessment, Prevymys meets all criteria for maintaining orphan designation, as it is indicated to treat a life-threatening condition that affects less than four in 10,000 patients in the EU and provides a potential clinically significant benefit over previously authorized therapies.

EMA