Eurordis - Rare Disease Europe

This report from the European Medicines Agency highlights the promise of – but also persistent hurdles to – drug repurposing in rare diseases

Drug repurposing – finding new uses for existing medicines – is a key strategy to expand treatment options where no approved therapies exist, especially in rare diseases.

As a member of the Repurposing Observatory Group (RepOG) and its Champions Interest Group, EURORDIS contributed to the EU pilot on drug repurposing, led by the European Medicines Agency and national regulators. The findings of this report include that 68% of submitted projects focused on rare diseases, and five of the six selected targeted rare conditions – confirming the field’s unmet needs and untapped potential.

As a member of the the RepOG’s ‘Champions Interest Group’, EURORDIS supported academic-led efforts and their regulatory engagement.

But the pilot also revealed challenges:

• Academic teams often needed 6+ months to prepare for Scientific Advice.
• Most Marketing Authorisation Holders (MAHs) showed limited interest in new rare indications.
• Only a few projects applied for orphan designation, despite being eligible.