Breaking new ground in bringing repurposed drugs to patients

25 juli 2025

BRUSSELS – The European Medicines Agency (EMA) has released findings from its Pilot Program on drug repurposing, marking a significant step toward establishing new regulatory pathways that could transform how existing medicines are developed for new therapeutic uses across Europe. Anticancer Fund has been a driving force behind this initiative, from its conceptual origins to supporting real-world implementation.

Understanding drug repurposing

Drug repurposing - finding new treatments for diseases from existing medicines used to treat other illnesses - is a development strategy that holds much promise, but is never as simple as first appears. While this approach offers the potential to bring treatments to patients more quickly and cost-effectively than developing entirely new drugs, it faces unique challenges. Repurposing old drugs is especially challenging because many of these drugs are cheap but proving they work in new diseases is expensive, so getting a return on investments is hard.

From concept to reality

The European Medicines Agency (EMA) has released a report on a pilot which provided tailored scientific advice to not-for-profit organisations and academics on repurposing authorised medicines for new indications. The foundation for this initiative was laid in 2018 when the European Commission’s expert group on Safe and Timely Access to Medicines for Patients (STAMP), who also appealed to Anticancer Fund, proposed a framework to support not-for-profit organisations and academics. This led to the Pilot Program conducted by the EMA and national competent authorities (NCA) between 2021 and 2025, which is now reporting its findings.

The aim of the pilot program was to steer a number of real repurposing cases, led by academics and clinicians designated as ‘champions’ in this programme. Some of them worked with not-for-profit organisations, through key steps in the drug approval process. In all, there were 35 applications to join the pilot at the EMA, and of these six were selected – representing a range of diseases, champions and countries.

Real-world impact

Anticancer Fund played its hands-on role by supporting two cases _one involving a drug that prevents transplant organ rejection being repurposed to treat a rare soft tissue sarcoma with no specifically approved treatment, and another to use an old cancer drug to treat a rare and potentially fatal hyperinflammatory disorder. In both cases these are diseases with very high unmet medical needs where commercial development of new drugs has not happened.

These examples showcase Anticancer Fund’s commitment to addressing treatment gaps for patients with challenging conditions and provide them with more treatment options rapidly.

Key findings and regulatory innovation

The key regulatory tool available to the pilot projects was Scientific Advice, a standard part of the commercial development process but not often used by academics and clinicians. A major finding was that the selected projects needed tailored support from the EMA, meaning greater levels of engagement and support to help the champions get the most from the advice process. This more collaborative way of working with clinicians and not-for-profits heralds a potentially ground-breaking future regulatory pathway for drug repurposing – with a significance beyond the pilot programme.

“Repurposing authorised medicines is a proven, cost-effective strategy to meet unmet medical needs. The EU pharmaceutical legislation can play a transformative role by recognising the vital contribution of non-commercial research actors and supporting them with an appropriate regulatory framework”, said Lydie Meheus, managing director of Anticancer Fund, just last week in the European Parliament during a Key Hearing on the reform of the pharmaceutical legislation.

Legislative reform

The proposed reform of the EU pharmaceutical legislation (currently at the Trilogues stage) introduces new legal measures aimed at supporting drug repurposing. This entails a regulatory pathway to evaluate evidence for repurposed drugs submitted by not-for-profit champions – as has been done in the pilot program.

Under the current regime, only the marketing authorisation holder of a drug (i.e. a company with a license to market the drug) is able to apply for a new use to be added to their product.

With the new legislation not-for-profit champions will be able to initiate the process with the EMA. This change would put the EU at the forefront globally, ahead of the USA and UK, in potentially unlocking many new and much-needed treatments for European patients. The positive implications for the competitiveness agenda are clear.

Anticancer Fund's involvement spans the entire journey of this regulatory innovation. Our ongoing commitment is driven by our own clinical trial portfolio, containing several drug repurposing trials. Therefore we have been involved from the beginning, when the ideas were being formulated at STAMP, then working with two of the selected projects. The organisations continues to work with those projects as they develop further.

Looking forward

Anticancer Fund welcomes the findings of the EMA report and remains a strong supporter of a regulatory framework complementary to the commercial development pathway.

We believe this pilot program represents more than just a successful regulatory experiment – it demonstrates the potential for a shift in how Europe tackles drug development for patients with unmet needs. By creating pathways for not-for-profit organisations and academic institutions to champion drug repurposing efforts, the EU is opening doors to numerous treatments that might otherwise remain unexplored.