Tracing the path of non-industry-driven medicine development for rare diseases through regulatory interactions at the European Medicines Agency

Abstract

Non-industry organizations, including academia, charities, and public institutes, increasingly contribute to rare disease medicine development. In this study, we reviewed orphan designations (ODs) granted between 2000 and 2022, comparing non-industry and industry outcomes. Of 2734 ODs, 183 (6.7%) originated from non-industry organizations. Protocol assistance was obtained for 33% of non-industry and 35% of industry ODs, with distinct peaks for non-industry ODs in 2015 and 2017 linked to European grant calls promoting regulatory engagement. Only six non-industry ODs led to marketing authorization, all post transfer to industry. Our findings indicate lower transition rates yet partial long-term convergence, highlighting the value of targeted grants and regulatory support.