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EMA And FDA Eliminating ‘Needless Differences’ – Particularly For Rare Diseases

 
• By Eliza Slawther

US and EU regulators have invited two companies to work with them on a pilot scheme for assessing drugs for rare genetic diseases, a move that the European Medicines Agency’s Emer Cooke said would help improve “global alignment.”

Francesca Buttigieg, Anthonius de Boer, Steffen Thirstrup and Sabine Haubenreisser at the TOPRA Symposium 2024
Francesca Buttigieg, Anthonius de Boer, Steffen Thirstrup and Sabine Haubenreisser at the TOPRA Symposium 2024 • Source: Pink Sheet

The European Medicines Agency and the US Food and Drug Administration are actively “working towards convergence,” and usually “agree on common endpoints,” for drugs in most therapeutic areas, according to Steffen Thirstrup, chief medical officer at the EMA.

The two regulators are in close communication daily, something that was reflected in the EMA’s recent decision to become an official observer of the FDA’s Project Orbis, Thirstrup said during a panel discussion at the 2024 TOPRA Symposium on 30 September

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