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DEBRA UK Research Team member attends the International Drug Repurposing conference in May 2026

11th June 2026 (Last updated 10th June 2026)

Dr Abi Witherden, DEBRA’s Research Grants Manager, joined research scientists, clinicians, people with lived experience of rare conditions, representatives of European and American regulatory bodies (EMA and FDA), charities and pharmaceutical companies in Brussels this month for the International Drug Repurposing Conference (iDR26). Titled ‘Navigating the Future’, this year’s theme focused on the complex path from finding the right drug to repurpose for a rare condition, through to patients actually receiving it.

A woman holds a sign about rare disease advocacy; beside her, a presentation slide reads "Success is getting treatments to patients," listing funding, research, and marketing as important steps.

The conference, supported by the EU Repurposing of Medicines for All platform, underscored how drug repurposing continues to gain momentum as a cost-effective and time-efficient approach to addressing unmet medical needs, particularly in rare conditions like EB.

iDR26 brought together delegates from across Europe, North America, and Asia to share cutting-edge research, regulatory insights, lived experience and collaborative opportunities. The programme centred on how repurposing known drugs can shorten development timelines and reduce risks compared with traditional drug discovery pathways. While cutting-edge and innovative new treatments can seem preferable to re-using something that already exists, repurposing drugs whose side effects and doses are already known and are already being commercially manufactured can enable faster progression into clinical trials. Ultimately, drug repurposing has the potential to bring effective new treatments to people living with untreated symptoms more quickly than starting from scratch.

Pre-clinical stages of drug repurposing

The role of computational methods and artificial intelligence (AI) in identifying which drugs to repurpose for a rare condition was highlighted. Machine learning models can be used to analyse huge amounts of information, from molecular structures to electronic health records, and identify areas to focus on far faster than would be humanly possible. The development of methods to screen large numbers of approved drugs in cell models of rare disease is vital and can be used to validate initial drug selections made by AI. The collection of evidence in model systems then allows progression to clinical trials.

A conference audience listens to a presentation titled "Navigating the Future," with a slide summarising key learnings and discussing innovation, legislation, and global impact in patient care.

Presentations from lived experience experts

Several speakers, themselves living with rare conditions, highlighted the frustration of not being able to get prescriptions for medications that they believed would help them before clinical trials could go ahead. It was acknowledged that, while the Yellow Card system exists for reporting unwanted side effects of treatments, there is no national system or expectation that patients or doctors report positive outcomes from drugs used off license. The CureID database was highlighted as a repository for sharing anecdotal reports of success with drugs used off license to treat rare genetic conditions. This could be useful for others with the same condition before evidence can be generated by clinical trials.

 

Presentations from company representatives and scientists

In addition to talks and panel discussions, iDR26 provided a platform for early-career researchers to present their work as posters showcasing various innovative approaches to the different stages of drug repurposing.

One speaker described the initiation of a global registry project for the worldwide EB community that will complement our funding of a global EB atlas and our partnership with NHS England.

A poster by Tamas Vincze described 3D printing of chewable gummies or dissolvable tabs containing a well-known blood pressure drug (Losartan) at doses suitable for children with RDEB. This work could facilitate repurposing this medication to help with EB symptoms.

A poster by Sara Ruiz Arciniegas on repurposing LSD for pain symptoms could have relevance for people living with the chronic pain of EB. We have recently funded a study on cannabinoid oil to reduce pain in EB and a project to identify new ways to reduce EB pain that will begin this year.

A poster by Charissa Witters described the development of a process to rapidly screen drugs that could be repurposed to help wound healing of corneal cells. Damage to the cornea and sight loss are issues in EB and we are currently funding PhD students to work on corneal damage in dystrophic and junctional EB in Australia and JEB at Liverpool University in the UK. 

 

Networking

Networking sessions allowed participants to forge new collaborations, reflecting the conference’s emphasis on community-building.

Two people stand in front of a scientific research poster, smiling at the camera. The poster discusses drug repurposing and knowledge graph research.

The event also addressed challenges that continue to hinder progress. Intellectual property concerns, funding gaps, and limited commercial incentives were cited as persistent barriers.

As the conference concluded, participants expressed optimism that drug repurposing is poised to play an increasingly important role in modern medicine.