Science highlights

Unlocking innovation: the power of drug repurposing

Finding new therapeutic uses for existing medicines to treat other diseases or conditions than their initial purpose.

What if?

What if a medicine traditionally used to treat high blood pressure, could be used to treat infantile haemangioma? Or a drug, notorious for causing severe developmental defects when given to pregnant women with morning sickness is successfully used to treat multiple myeloma? What if this is reality?

The reality of drug repurposing

This reality is called drug repurposing, which seeks to find new therapeutic uses for existing medicines to treat other diseases or conditions than their initial purpose. Successful repurposing can provide new treatment options for patients and is of huge importance, especially to those with rare or ultra rare cancers where few therapy options are available.

Bridging the gap

With over 7.000 rare and neglected diseases with unknown molecular basis, but only about 500 treatments available, the gap is significant. Addressing this gap from scratch would take forever, so why not explore the medicines already accessible?

Overcoming barriers

While it seems logical, numerous barriers are holding back this innovation. Economic, regulatory and policy barriers, along with inefficiencies such as a fragmented ecosystem, lack of patient centricity, and inadequate funding throughout the research process, are hurdles along the drug repurposing pathway.

Academic researchers and clinical institutions with drug repurposing ideas often run trials in this field, but the journey frequently ends with a publication in a high-impact journal or a talk at an international conference. Lacking drive, funding, and/or knowledge how to move the needle further, the project ends there.

And that is exactly what the REMEDi4ALL project wants to tackle.

Working together

REMEDi4ALL is a European consortium consisting of 24 partners, ranging from research organisations, patient advocates, funding bodies, regulators, health technology assessment experts and pharmaceutical industry representatives. With EU Commission funding, the initiative is establishing an EU-platform that can facilitate and accelerate the use of existing drugs for new indications.

Launched in September 2022, the consortium has 5 years’ time to build a harmonised, coordinated, and accessible infrastructure where researchers with a good drug repurposing idea that brings value to patients can meet the necessary experts, find guidance tools and resources, to help them along their path to patients. Having the indication on label and the drug reimbursed is the ultimate goal, as this is what really matters to patients with little to no treatment options left.

Creating a one-stop shop

While building the platform into a one-stop shop, learnings are drawn from 4 pre-selected demonstrator projects that allow the consortium partners to identify bottlenecks, find solutions to unforeseen issues, and check the robustness of the tools created. Additionally, the concierge service allows researchers and other stakeholders to already reach out and have a dialogue with the required consortium experts, aiming to support the requestor’s ask while learning where the needs are and which training materials could be developed to support the researchers’ needs.

Facilitating matchmaking

Besides training modules and webinars, the Anticancer Fund and the REMEDi4ALL-partners also set-up a global network of funders. The objective is to facilitate collaboration among all types of funding entities and promote collaboration with research institutions and other stakeholders. This matchmaking is crucial because, without a business case, the money -needed to conduct expensive clinical trials and generate the required strong evidence must come from other sources.

To bring researchers looking for funding closer to those with funds, we have built, together with REMEDi4ALL, the funding calls database. It contains a list of current funding calls related to drug repurposing. While the database is searchable, funders of drug repurposing research are encouraged to enter their call, allowing researchers and funders to find each other easily.

Building momentum in Europe

Successful repurposing means that the patient has access to the medicine, thus that the indication is on label and the drug is reimbursed. The current regulatory framework is one of the barriers, but the pharmaceutical legislation is currently under review, and the proposed text, as drafted by the EU Commission, covers several articles related to drug repurposing, indicating that the untapped potential of drug repurposing is recognised.

Within REMEDi4ALL, we strive to reach a more favourable policy environment, bringing together all stakeholders involved, discussing identified barriers and their practical solutions. As multistakeholder collaboration holds the key to successful repurposing, we gather the global drug repurposing community yearly at the International Drug Repurposing Conference, iDR25, on the 7nd and 8th May 2025, in Amsterdam. Hope to see you there!

Moving forward together

The untapped potential of drug repurposing is immense, and collaboration is essential to unlock this innovation. By working together with our partners of REMEDi4ALL, we overcome barriers, accelerate research, and bring new treatment options to patients in need, faster. So, join us.

Patricia Vandamme
author

Patricia Vandamme is the Policy Officer of the Anticancer Fund. Patricia holds a Master in Pharmaceutical Sciences.