A new editorial titled “Repurposing for Small Population Needs” has been published in Frontiers in Medicine. Among the authors is our Head of the Research and Innovation Department Viviana Giannuzzi.

The article provides an overview of the 10 manuscripts, including original research, perspective, mini review and opinion articles, collected in the Research Topic “Repurposing for Small Population Needs”. This collection has highlighted the opportunities from repurposing medicines for small populations reached, expected or failed through industrial and academic approaches and initiatives launched in Europe, while still facing regulatory and economic challenges and barriers.

In this context, the transition from off-label use to authorised indications is often limited by low commercial interest and reimbursement challenges. Dedicated pathways, such as the Paediatric-Use Marketing Authorisation (PUMA), remain underutilised.

The editorial also draws on evidence from EU-funded research programmes, showing how financial and operational constraints can affect the implementation of research&development plans, despite strong scientific rationale.

At the same time, it emphasises the growing role of innovative approaches — including real-world data, pharmacometric modelling, and artificial intelligence— as well as European initiatives supporting repurposing.

Overall, the editorial underlines how drug repurposing represents a concrete opportunity to address unmet medical needs in small populations, namely paediatric and rare diseases, provided that incentives, investment, and collaboration between academia, industry, and institutions are strengthened.

The publication is available here.