Presentation (CTI)

OBJECTIVES: Orphan drugs, developed to treat rare diseases, address conditions with small patient numbers and significant unmet needs. The Food and Drug Administration (FDA) and European Medicines Agency (EMA) have differing criteria and incentives for orphan drug designation and marketing authorisation (MA). We reviewed orphan drug MAs to understand if there are disparities in access between the US and Europe.
METHODS: The FDA website was searched for all orphan medicines approved between 1^st January 2022 and 31^st December 2023. The EMA website was searched for the corresponding approval in Europe with approval date, orphan designation status, indication, and drug class noted.
RESULTS: During 2022-2023, the FDA approved 140 medicines for orphan conditions. 100 of these have been approved by the EMA of which 51 were approved with orphan designation. The FDA approved 50 medicines for orphan oncology indications of which 38 were also approved by the EMA, 14 (28%) with orphan drug status. The FDA also approved 14 gene or cell therapies of which 8 (57%) have also been approved by the EMA. Of the 6 without European approval, 2 were withdrawn at sponsor company request for commercial reasons. Of the 40 medicines without current European regulatory approval, the majority (29, 71%) were small molecules or proteins. The EMA refused MA for two medicines, two MA applications were withdrawn by the sponsor companies, and an additional one MA was withdrawn at sponsor company request, citing commercial reasons.
CONCLUSIONS: There is a disparity in the number of orphan medicine approvals in the US and Europe. These disparities partly relate to differences in orphan designation criteria between the FDA and EMA and the timeframe for MA. Commercialisation concerns, such as the size of the treatable population and medicine price point, will likely impact the decision on whether to launch a medicine in Europe.