A significant regulatory divergence is emerging between the United States and European Union in the approval of treatments for rare diseases. A retrospective analysis published in PLOS Medicine examined 814 orphan drug approvals by the FDA between 2011 and 2023, revealing that only 29% subsequently received matching European Medicines Agency authorisation with orphan designation. The findings are concerning: one-third of FDA-approved orphan drugs were denied EMA approval entirely, while another 38% received EMA approval without orphan status recognition. This growing disconnect between regulatory pathways suggests divergent scientific or policy standards and threatens equitable patient access to life-saving rare disease treatments across the Atlantic. The trend has intensified in recent years, with approvals from 2017–2023 showing significantly lower odds of EMA authorisation compared to earlier periods.
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