Europe – Regulators Detail Challenges in Defining Orphan Conditions in EU

A new commentary by European regulators and members of the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) in Nature Reviews Drug Discoveryprovides insights into drugmakers seeking orphan designation for their products for difficult to define conditions.

Like the US, the EU offers certain incentives to promote the development of medicinal products to treat rare diseases, including 10 years market exclusivity for approved orphan indications.

However, regulators in the EU and US both have differing interpretations of what constitutes an orphan condition and recent advances in precision medicine are raising challenging questions for regulators in both regions.

“The definition of an acceptable orphan condition is the essential starting point for the assessment of any orphan designation application in the EU,” the authors write, noting that COMP looks at diseases in “broad terms, avoiding designations relating to artificial subsets of a particular condition.”

This is in part due to direction from the European Commission’s Orphan Notice, which states that “sub-setting a condition with the use of biomarkers will not be acceptable unless the sponsor provides solid evidence that the activity of the product should not be shown on the larger population.”…