In today’s blog our CEO Rick tells us about his recent trip to the World Orphan Drug Congress last week.

A big part of our work at Findacure is raising awareness about the work of the rare disease community. This can involve speaking to a wide diversity of people, attending conferences, running races or writing this blog. Last week, we attended one of the larger rare disease conferences in the calendar, the World Orphan Drug Congress Europe 2018, with the aim of telling the European rare disease community about some of the rare disease work in the UK, as well as learning more about what is happening in the rare world today. The event was held in Barcelona, and featured some fantastic talks and discussion about all things rare.

The conference opened with a series of half day workshops that allowed delegates an in-depth look at some major topics in the rare disease field. Luckily for Findacure, we were front and centre, as I was chairing and delivering a large part of the workshop on drug repurposing.

The central question of the workshop was “Is drug repurposing a sufficient business model to drive orphan drug development?”. Between Pan Patziarka, Programme Director at the Anticancer Fund, Dan O’Connor, Medical Assessor at the MHRA, myself we spent a four hour session delving into the world of drug repurposing. Pan and I highlighted the need for repurposing, and the strategic considerations when thinking of running a repurposing project. This inspired a lot of discussion in the room in particular around the need for new incentives for the pharmaceutical industry to work in the field.

After the break Pan discussed some excellent progress in repurposing for rare cancers, including the importance of generating excitement around this approach to drug development in the scientific community. I then highlighted some new models for funding and delivering repurposing projects including the work of the AKU society, the repurposing of Everolimus where the Tuberous Sclerosis Association played a central role, the academic driven MCDS-Therapy, and the social impact bond. Finally Dan wrapped up by discussing new pathways to licence repurposed drugs.

It was great to be so heavily involved in such a detailed session at an international conference. The audience certainly engaged with the topic, and there were some fantastic points of discussion, with patient groups, industry representatives and academics all helping one another to see drug repurposing from a different angle.

After the workshops we launched into the main programme of the conference, which featured sessions on gene therapies, clinical development, access to medicines, and science and strategy. There was some detailed discussion of the European Reference Networks, which are beginning to investigate ways to collaborate more widely with external partners, and some fascinating and often passionate discussion around drug pricing. One of the stand out discussions for me focussed on the progress of project Hercules, a collaborative study to develop models, metrics, and information that can aid the approval and reimbursement of new treatments for Duchenne muscular dystrophy. The team have brought together a mixture of pharmaceutical companies and academics, all of whom are collaborating to better understand the financial impact of Duchenne on the world, and to fund new ways to assess the efficacy and financial impact of new drugs. It is a vital project that is really highlighting the need for a concerted effort to develop evidence to help secure access to new rare disease drugs, and hopefully a model the whole community can learn and benefit from.

It was great to attend the conference, to highlight some of the amazing work being delivered in the patient community, and to connect with rare diseases at the European level. The conference is open and welcoming to patient organisations, and we hope to see more of you there in the coming years.