To help bring new therapeutic uses of authorised medicines addressing an unmet medical need to patients, Article 48 of the new EU Pharmaceutical Regulation creates a specific pathway for non-profit organisations to submit evidence for regulatory assessment. Following a favourable EMA opinion, the MAHs concerned should submit a variation to add the indication to the product information.
Jolanda Lamers-Lemmers represented Starodub in the stakeholder discussion on this topic during the European pharmaceutical legislation working session on 28 September 2026, organized by the Dutch Ministry of Health, Welfare and Sport (VWS) and College ter Beoordeling van Geneesmiddelen (CBG), ahead of the CBG Collegedag.
For non-profit-led projects, the opportunity is significant. However, the real challenge isn’t identifying a promising new use. It’s generating the right evidence and creating a clear pathway to regulatory implementation.
The key question is whether the implementation of Article 48 will bridge the gap between non-profit evidence generation and an authorised, maintained and accessible indication.
What do you see as the biggest implementation challenge: evidence quality, MAH cooperation, funding, data ownership, or lifecycle responsibility?
