This ERDERA policy brief frames rare diseases as a strategic test case for the proposed European Biotech Act, arguing that fragmented clinical, regulatory, and data ecosystems hinder translation from discovery to patient access. It identifies gaps in the draft Act (including limited recognition of existing infrastructures, narrow biotechnology scope, underdeveloped data frameworks, and skills shortages) and positions the combined ERDERA–European Reference Networks ecosystem as an operational backbone.
It provides seven policy recommendations spanning governance embedding, broader biotech definitions (including RNA, nanomedicine, and AI-enabled technologies), stronger interoperable data infrastructures, support for equitable multinational trials, skills initiatives, and improved investment conditions.