“Significant benefit” is a key criterion to be taken into account when deciding whether a drug for a rare disease should be awarded orphan designation in the EU.
EU Ruling Raises New Questions For Orphan Designation
An EU court has upheld a decision to revoke the orphan designation awarded to GMP-Orphan's trientine product for Wilson's disease, saying that the product no longer met the “significant benefit” criterion on the grounds of lack of availability of another drug. The case has raised questions about the level of evidence needed to justify a significant benefit.
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