Behind the REPO4EU Platform: In conversation with Harald Schmidt (Maastricht University)

Technically, REPO4EU is the response to a funding call from the European Commission to foster drug repurposing across Europe. 

Drug repurposing means that instead of developing entirely new drugs, we use medicines that are already on the market and find new uses for them. This has been happening for decades, but usually quite serendipitously; our goal is to make this process much more precise and systematic.

The real innovation of REPO4EU is in how we approach this. We want to fundamentally redefine what we currently call a disease. Most diseases today — except for infectious diseases or some rare diseases caused by a single gene — are defined simply by describing a symptom in an organ. Hypertension, asthma, psoriasis, inflammatory bowel syndrome… these sound like precise disease definitions, but they are really just descriptions of symptoms.

As a result, many therapies are imprecise. Not every patient taking the same medication will experience the same benefit, and this also contributes to the high failure rate in drug discovery.

What we introduce instead are predominantly genetically defined disease definitions, more similar to how rare diseases are approached. This allows us to identify much more precise treatment strategies and opens enormous potential for repurposing existing drugs — not only for their original target, but for completely different disease mechanisms.

Because these drugs are already approved, we already know most of their side effects, and we can skip long discovery and preclinical development stages. That means the process is faster, cheaper, and often much more efficient.

Operationally, my role as a coordinator is making sure this unusually large project — with 27 partners and a €25 million budget — moves in the right direction and that all the different pieces come together successfully.

The real advantage of having such a large consortium is that it allows us to bring together the best people across Europe. What we are doing goes far beyond the initial scientific discovery.

In medicine, we currently face what is often called the “valley of death” — a huge gap between basic research and actual patient benefit. We produce enormous amounts of academic research, but very little of it reaches patients in the form of better diagnostics, therapies or prevention strategies. One major reason is that academic success is still measured mostly by publications and grant income, not by whether your work improves patient outcomes.

REPO4EU tries to bridge that gap. We make sure that from the very beginning, researchers are thinking not only about scientific discovery, but also about patents, regulation, clinical validation, market access and reimbursement. We want researchers to think early about whether their discovery can realistically become something that benefits patients.

That requires bringing together experts from many different areas — science, ethics, regulation, business, intellectual property and healthcare systems. No single person can solve this alone.

The REPO4EU platform exists because we believe what we are doing will shape the future of medicine.

Rather than waiting for our clinical results to slowly spread through the academic world, we wanted to provide all the tools, guidelines and workflows directly to researchers so they can apply these methods themselves.

One of our biggest achievements is in bioinformatics. Traditionally, to work with bioinformatics, you needed to be a bioinformatician — someone who could code and use highly specialised software. We wanted to create something that the average biomedical scientist could use without needing that expertise. That required a lot of work because bioinformaticians and biomedical scientists often speak almost different languages.

We now have a very strong tool called drugst.one that makes this possible. Instead of generating a long list of 100 possible drug candidates, we aim for extreme precision — ideally narrowing it down to just two or three highly relevant options.

Beyond that, the platform integrates everything needed for the full translational process: preclinical validation, access to human cell models, clinical trial design, patent strategy, regulatory guidance, reimbursement planning and health technology assessment. It’s not just a collection of links or tools — it’s a fully integrated online workspace that guides researchers through the entire journey from idea to patient impact. And importantly, it can be used globally, not just in Europe.

Nobody in this project works in isolation. You cannot simply sit in your office, do some bioinformatics, and hand the results over to someone else. The process is highly iterative and requires constant dialogue between teams.

For example, after bioinformatics predictions are made, we immediately need discussions around preclinical validation. Can we validate using human cell models? Do we need animal models? Is it safe enough to move directly into clinical trials?

At the same time, we apply an ethics by design approach. We do not want ethics to be something that is considered at the very end, just before a clinical trial starts. Ethics should be part of the process from day one. The very first question should be: is there really an unmet patient need? Does this approach genuinely benefit patients? Are the risks acceptable? If the answer is no, then there is no reason to continue with further experiments.

Likewise, patent strategy and commercial viability must be considered early. If something cannot realistically be developed into a therapy, then even excellent scientific results may end up as a translational dead end.

Everything we do is built around one central goal: not papers, but patients.

Two things. First, that the platform truly works — that it can reliably guide discoveries toward real patient impact; and second, that it is widely used by scientists, clinicians, biotech companies and industry partners.

The goal is scale. We want to accelerate innovation in diagnosis, treatment and prevention as quickly as possible.

Drug repurposing is only one part of this. Precision prevention is another major area. If we can better define disease risk, we can also provide much more personalised prevention strategies, reducing the need for treatment later on. In many cases, lifestyle interventions may be enough if they are tailored precisely enough. Only in a smaller percentage of cases would drugs be needed. So this is really a broader shift toward precision medicine as a whole.

Right now, we are preparing both alpha and beta testing phases of the platform, and feedback is incredibly important. We invite researchers, clinicians and other stakeholders to register via this link, so they can test the platform and tell us what works and what needs improvement.

A very broad range of stakeholders!

Bioinformaticians may want to better understand what happens after the computational predictions. Biomedical scientists can use the tools directly. Biotech companies, pharmaceutical companies, regulators, health technology assessors — they all have a role to play.

Even patient organisations are very important for us. While we do not make specific recommendations for individual patients, we absolutely want patient groups to engage with us and help shape the process.

I think everyone involved in healthcare innovation can benefit from looking a little outside their own silo and understanding how the whole system works. That is exactly what REPO4EU is trying to build.