A new scientific article entitled “Regulatory innovation for drug repurposing: Ten proposals to accelerate access and safety”, authored by researcher Sara Pintado and professors Sofia Oliveira-Martins and Adelaide Fernandes, was published in the May 2026 issue of the journal Drug Discovery Today.
The article addresses the potential of drug repurposing to accelerate access to therapies in contexts of unmet medical needs, analyses the regulatory challenges that continue to hinder their authorisation, and presents ten strategic measures to facilitate access to treatments and increase the safety of patients with rare diseases in Europe.
Based on the SIMPATHIC project, the authors propose key measures to the European Medicines Agency (EMA) and the European Commission, such as:
- Legal Definition: Establish a legal definition of ‘repositioning’ within the EU to clarify standards and requirements;
- Support Office at the EMA: Provide guidance to academic institutions and non-profit organisations on regulatory processes;
- Simplified Regulatory Pathway: Establish fast-track procedures for situations of unmet medical need;
- Incentives and Transparency: Introduce priority review ‘vouchers’ to attract investment and ensure access to data from original studies;
- Real-World Data: Promote the integration of real-world clinical practice evidence into regulatory decisions.
The document concludes that there is a need for a collaborative model with aligned incentives for all stakeholders. The implementation of these proposals could be crucial to bridging the gap in treatments for rare diseases, removing bureaucratic and financial barriers to ensure that safe and effective medicines reach those who need them more quickly.
