Tracing the path of non-industry-driven medicine development for rare diseases through regulatory interactions at the European Medicines Agency.
WHY IT MATTERS
This research shows that academic and charity-led rare disease drug projects face significant barriers to reaching patients, suggesting that funding and regulatory support for non-industry developers could unlock more treatment options for rare diseases that pharmaceutical companies might overlook.
Researchers studied how medicines for rare diseases are developed by non-industry groups like universities and charities, compared to pharmaceutical companies. Between 2000 and 2022, only about 7% of rare disease medicine projects came from these non-industry organizations. While these groups got help from regulators at similar rates as companies, very few of their medicines actually made it to patients—only six succeeded, and all had to partner with pharmaceutical companies to finish the job.
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. Authors: Rosenberg et al. Journal: Drug discovery today MeSH: Rare Diseases, Humans, Orphan Drug Production, Europe, Drug Industry, Drug Development, Drug Approval