For many years now, clinicians, researchers and, above all, EB patients from all over the world have been exchanging ideas at the DEBRA International Congress.

"You are not alone" - was this year's motto of the DEBRA International Patient Conference organised by DEBRA Russia. EB does not only affect a few people, although it is one of the rare diseases. The first virtual DEBRA congress demonstrated this in an impressive way. More than 15,000 participants from over 130 countries show that the topic is a big one and that the virtual format enabled the wider worldwide family of people with EB to participate.

Virtual and free of charge

Actually, the congress should have taken place in Moscow. All participants then travelled to Red Square - but purely virtually and free of charge. The Russian landmark served as the backdrop for the well-functioning online congress platform. For the interaction of the participants, a 3D space was developed with virtual stages, an area for panel discussions, coffee breaks and communication rooms. Participants had the possibility to attend the sessions in their own time zone. There was also a wide range of translation services. In addition to the Medical Track Stage, which focused on clinical and research topics, many people also visited the Patient Track Stage, which provided a platform for people with EB, DEBRA groups worldwide and some entertainment.

 

Current status of EB research, first clinical trial successes and their challenges & clinical care

The distinguished panel of speakers consisted of 40 leading EB experts from around the world, including Austria, Israel, USA, France and Russia. After each session, there was time for individual questions and answers. These were broadcast live and thus enabled a real congress feeling and set the talks presented in advance well into the overall context.

Prof. Johan Bauer, as head of the congress committee, was responsible for the programme from a scientific point of view. All experts agree that the improvement of clinical care and new therapies to address issues like itch, cancer and pain are still urgently needed. And these have never been closer than today, as years of basic research are beginning to bear fruit. Several clinical trials in phase 3 and one already in the registration phase, bring us a big step closer to fulfilling the vision that unites us worldwide: a cure for the 'butterfly children'.

 

Workshops & Networking

Hands-on workshops on very practical topics such as the 'Family Forum with the Health Professionals' and on topics such as oral care, physiotherapy and nutrition rounded off the programme.  In the network areas one could seek exchange in smaller groups.

 

Networking: accelerating therapy development

All the talks in the area of clinical trials showed that not only the clinical endpoints are responsible for the success of a trial. Issues such as quality of life for EB patients and good networking with regulatory authorities and patient organisations come much more into play.

Pharmaceutical companies are increasingly recognising that the involvement of patients and clinicians is central to achieving the common goal. This new direction is good and important. International networking is needed more than ever. A rare disease like EB needs multi-centre trials to ensure that the safety and efficacy of new therapies can be adequately tested.

The framework conditions for the pharmaceutical industry allow this better today than a few years ago. Initial successes, for example in Fabry disease or spinal muscular atrophy, show that investments in the rare disease field are paying off. There are incentive systems on the part of the regulatory authorities, e.g. with the Orphane Drug Designation in the EU. The industry has started to deal with the disease, to understand it and to involve more patients.

 

Our main conclusions from the conference are:

• No EB sufferer worldwide is alone - there are many!
• We urgently need new therapies for 'butterfly children' - this process must be accelerated.
• Therapy development is challenging in principle: small patient groups and the heterogeneity of EB make it even more difficult for 'butterfly children'.
• Collaboration between industry, regulatory authorities, EB clinicians, patient organisations and patients is central to success. The most important task for all stakeholders will be to establish good communication and intensify collaboration.

All presentations on demand

There is a YouTube channel that will make many lectures available on demand by beginning of October. Many thanks to DEBRA Russia for organising this extraordinary congress.

Watch on demand for registered participatns: DEBRA Congress 2021